Cell & Gene: The Podcast: Recent Episodes
Erin Harris
Cell & Gene, the most valuable online resource for delivering in-depth content from authoritative authors and sources to professionals in the CGT sector, introduces Cell & Gene: The Podcast. In each episode, Cell & Gene Chief Editor, Erin Harris, will talk to industry and academic leaders about their current initiatives and how they are moving the sector forward.
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Episode 134 of Cell & Gene: The Podcast features Host Erin Harris' discussion with Shannon Boye, PhD, Founder and CSO of Atsena Therapeutics. Dr. Boye shares how the company is approaching Stargardt disease with a differentiated gene therapy strategy built around delivery, dual-vector design, and a disciplined go/no-go framework. Their conversation also explores what Atsena’s broader clinical experience is teaching the team as it advances new programs and works toward the clinic.
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Episode 133 of Cell & Gene: The Podcast features Host Erin Harris' discussion with Aprea Therapeutics' CEO, Dr. Oren Gilad. They cover the evolution of precision oncology, the science of synthetic lethality, and how Aprea is developing next-generation therapies that target cancer's unique genetic vulnerabilities while improving safety and efficacy. Dr. Gilad also shares insights into biomarker-driven drug development, where precision medicine is headed as researchers pursue more effective treatments for patients with difficult-to-treat solid tumors, and more.
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Episode 132 of Cell & Gene: The Podcast features Host, Erin Harris' conversation with Diakonos Oncology's President and COO, Jay Hartenbach. Together, they explore how the company is advancing a patient-derived dendritic cell therapy designed to generate a stronger immune response against difficult-to-treat solid tumors, including glioblastoma, pancreatic cancer, and refractory melanoma. Their discussion also covers early clinical signals, outpatient administration, and the manufacturing and automation steps needed to make personalized cell therapies more scalable for broader CGT audiences.
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For episode 131 of Cell & Gene: The Podcast, we’re sharing a recent "Better Biopharma" roundtable that Erin Harris had the opportunity to join alongside her Life Science Connect editorial colleagues. Hosted by Tyler Menichiello, this conversation brings together chief editors from across the Life Science Connect network to take a midyear look at what’s shaping the pharmaceutical and biotech landscape in 2026. Featuring perspectives from Katie Anderson, Jeff Buguliskis, Ben Comer, Ray Dogum, Rachel Grabenhofer, Dan Schell, Anna Rose Welch, and Harris, this is a valuable listen for anyone looking to better understand where the industry is headed, and how CGT fits into that broader trajectory. https://www.bioprocessonline.com/solution/better-biopharma
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In Episode 3 of our in vivo-focused special series of Cell & Gene: The Podcast, Ascidian Therapeutics' Founder, President, and CEO Mike Ehlers, M.D., Ph.D., explains how the company is advancing a new class of genetic medicines built on RNA exon editing to durably correct disease at the transcript level without permanently altering DNA. He also shares why in vivo therapies still need to prove precision, durability, and predictability before they become mainstream, and why the field must move beyond proof of concept to reproducible clinical performance across patients and diseases. He discusses the importance of patient selection, tissue specificity, and matching editing efficiency to biological thresholds, and much more.
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In episode 2 of this four-part in vivo-focused special series of Cell & Gene: The Podcast, Host Erin Harris sits down with Gopi Shanker, Ph.D., Chief Scientific Officer at Beam Therapeutics, for an in-depth conversation on the rapidly evolving landscape of in vivo gene editing and precision genetic medicines. Dr. Shanker discusses how next-generation base editing technologies are advancing beyond traditional CRISPR approaches by enabling precise single-base changes without creating double-stranded DNA breaks. They discuss the growing momentum behind in vivo delivery strategies, including targeted LNP technologies designed to reach tissues beyond the liver, as well as the operational and patient-centric advantages these approaches may offer compared to ex vivo therapies.
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In episode 130 of Cell & Gene: The Podcast, Dr. Naji Gehchan, Chief Medical and Development Officer at Kyverna Therapeutics, joins Host Erin Harris to outline the company’s mission to apply CAR T-cell therapy beyond oncology to treat severe autoimmune diseases, highlighting promising Phase 2 data from its investigational therapy, Miv-cel, in stiff person syndrome (SPS). Dr. Gehchan explains that Miv-cel works by deeply depleting pathogenic B cells and enabling a broad immune reset, differentiating it from conventional therapies that require ongoing management. As Kyverna advances a rolling BLA submission, Dr. Gehchan positions this milestone as potentially historic, both for SPS patients and for the broader cell therapy field, signaling an inflection point where CAR T could transform autoimmune disease care in the same way it has oncology.
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This is Episode 1 of a four-episode in vivo-focused special series of Cell & Gene: The Podcast. Host Erin Harris speaks with Cassie Gorsuch, Ph.D., CSO at Precision Biosciences, about the rapid evolution of in vivo gene editing and the scientific, translational, and regulatory hurdles shaping the field. Dr. Gorsuch discusses how Precision Biosciences approaches in vivo therapeutic development through its Arcus platform, with programs targeting chronic hepatitis B and Duchenne muscular dystrophy. They cover the broader challenges facing in vivo gene editing, including delivery limitations outside the liver, balancing specificity and efficiency, mitigating off-target risks, and translating promising preclinical in vivo data into clinical success.
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In episode 129 of Cell & Gene: The Podcast, Host Erin Harris reconnects with Zachary Roberts, M.D., Ph.D., EVP of Research & Development and Chief Medical Officer at Allogene Therapeutics, to explore how allogeneic CAR-T is evolving from a scalability promise into an earlier-line, potentially curative intervention. Their conversation centers on the ALPHA3 trial, where MRD-guided treatment is redefining when and how CAR-T can be deployed, alongside broader implications for diagnostics, patient access, and the shift toward off-the-shelf therapies in community settings.
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In episode 128, Host Erin Harris talks to Tatyana Matveeva, Ph.D., Director of cGMP Operations at George A. "Doc" Lopez, MD Laboratory for Regenerative Cell Therapy, Harvard Medical School and Massachusetts General Hospital, about leading cGMP operations within an integrated ecosystem, where manufacturing, research, and neurosurgery coexist. Dr. Matveeva highlights key operational challenges in scaling autologous therapies, particularly around technology transfer, process reproducibility, and regulatory readiness, emphasizing the need for early collaboration between research and GMP teams. Their conversation also explores rigorous approaches to chain of identity and custody, the importance of extensive simulation runs to ensure robustness, the unique sensitivities of manufacturing cells for neurological applications, and more.
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On Episode 127 of Cell & Gene: The Podcast, Carlos Buesa, Ph.D., Founder and Chairman of the Board at Oryzon talks to Host Erin Harris about how targeting LSD1 is unlocking new treatment approaches across oncology, CNS disorders, and sickle cell disease, with promising early clinical data and a strategy grounded in precision epigenetics.
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On episode 126 of Cell & Gene: The Podcast, Host Erin Harris welcomes Catriona Jamieson, M.D., Ph.D, Director of the UC San Diego Sanford Stem Cell Institute and Professor of Medicine at the School of Medicine. They explore how shifting the medical mindset from “common horses” to rare “zebras” is unlocking earlier detection and more effective treatment of diseases like myelofibrosis. Dr. Jamieson explains how subtle, often overlooked symptoms can mask serious underlying conditions, and how advances in genomic testing are enabling clinicians to identify disease-driving mutations sooner. They cover groundbreaking progress in targeted therapies, which aim to stop cancer progression and overcome treatment resistance.
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On episode 125 of Cell & Gene: The Podcast, Host Erin Harris talks to Halloran Consulting Group's Monika Swietlicka to discuss how the FDA is increasing flexibility in cell and gene therapy development without lowering evidentiary standards, emphasizing a risk-based, holistic approach. They also explore what this means for developers, from platform strategies and global regulatory divergence to the growing role of patient advocacy and the need for early, integrated planning.
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On episode 124 of Cell & Gene: The Podcast, Aurora Therapeutics' CEO Dr. Edward Kaye discusses the company’s strategy for translating CRISPR gene editing into scalable, commercially viable medicines for rare diseases. Aurora is initially targeting phenylketonuria (PKU) using a platform approach that leverages shared components, such as lipid nanoparticles and base editors, while customizing guide RNAs for specific mutations. Dr. Kaye explains how trials, regulatory flexibility, and optimized manufacturing could make it possible to treat many genetic variants efficiently and cost-effectively. Ultimately, Aurora aims to build a repeatable model that expands gene editing access to larger rare-disease populations while keeping patients at the center of development.
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In episode 123 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Panteli Theocharous, FIBMS, M.S., Ph.D., FRCPath, about the patient journey in cell therapy trials. They pinpoint key friction points, such as delayed referrals, unpredictable vein-to-vein timelines, and burdensome long-term follow-up, while sharing actionable strategies for simplification. These strategies range from upstream trial design and streamlined consent processes to standardized logistics, hybrid monitoring models, honest risk communication, and engaging patients as true partners in real-world evidence generation.
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In episode 122 of Cell & Gene: The Podcast, Host Erin Harris talks to Paul Romness, CEO of OS Therapies, to learn the company’s mission to address the severe unmet need in pulmonary metastatic osteosarcoma (PMO), a rare pediatric cancer with no established standard of care once it metastasizes. Romness explains how OS Therapies’ off-the-shelf HER2-targeted immunotherapy aims to significantly improve outcomes by stimulating a robust immune response with minimal side effects. He highlights results from a multicenter Phase 2B trial showing markedly improved overall survival rates compared to historical outcomes, details the company’s constructive regulatory interactions with the FDA, and underscores the value of comparative canine biomarkers in development.
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In episode 121 of Cell & Gene: The Podcast, Host Erin Harris talks to Scribe Therapeutics' CEO and Co-Founder Benjamin Oakes about building next‑generation CRISPR and epigenetic editing tools to move genetic medicine beyond rare disease into common cardiometabolic indications. Oakes shares Scribe’s engineered CasX platform and epigenetic silencers, preclinical data from its various programs, and why exquisite specificity and low-dose LNP delivery are essential to treating patients safely. They also explore Scribe’s partnerships with Sanofi and Lilly, the company’s cardiometabolic-first strategy co-developed with Dr. Jennifer Doudna, and Oakes’ conviction that genetic medicines can fundamentally reshape healthspan and the future of preventive cardiovascular care.
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Welcome to episode 120 of Cell & Gene: The Podcast. Host Erin Harris is joined by Rachel Haurwitz, CEO of Caribou Biosciences, to discuss the company’s progress in developing CRISPR-edited, off-the-shelf CAR-T therapies for hematologic malignancies. Their conversation centers on Vispacell, Caribou’s allogeneic CD19 CAR-T for second-line large B-cell lymphoma. Haurwitz explains how Caribou has systematically optimized its allogeneic platform using clinical and translational data. They also cover pivotal Phase 3 trial planning, regulatory considerations, and what to expect next from Caribou’s broader pipeline, including its BCMA-targeted program in multiple myeloma.
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We’re sharing this Better Biopharma episode on Cell & Gene: The Podcast because Better Biopharma is a sister show in the Life Science Connect family, and this conversation touches so many of the same challenges, pressures, and big-picture shifts our audience is thinking about right now. CGT doesn’t live in a bubble, and neither do the people building it. So we wanted to bring this wider, cross-industry conversation straight to you.
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In episode 119 of Cell & Gene The Podcast, Host Erin Harris talks to Dr. Norman Putzki, Global Head Clinical Development, Novartis, about the FDA approval of Itvisma, now the only gene replacement therapy approved for children, adolescents, and adults with spinal muscular atrophy (SMA). Dr. Putzki walks us through the six-year development journey behind the STEER and STRENGTH Phase 3 programs. And we explore what the expanded age-range label means for patients who were previously left behind, why intrathecal, fixed-dose AAV delivery represents a pivotal advance for safety, efficacy, and scalability. He details how the Itvisma program is informing Novartis’ broader gene therapy strategy across neuromuscular and CNS diseases, and more.
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In Episode 118, Host Erin Harris talks to Alexander Cryer, Ph.D., Instructor in Medicine at Mass General Brigham, about a proof of concept strategy that reprograms tumor cells with mRNA lipid nanoparticles to overactivate the cGAS-STING pathway, forcing cancer cells to produce and export large amounts of the innate immune agonist cGAMP to stimulate surrounding immune cells and drive anti-tumor immunity. Dr. Cryer explains the basic biology of cGAS-STING and how his team restored this pathway in tumor cells and leveraged intratumoral LNP delivery to overcome nucleic acid delivery and targeting challenges. He also discusses future directions, the need to move beyond intratumoral administration with more targeted systemic delivery, and the broader concept of turning the tumor’s own abundant cell population and evolutionarily conserved innate immune pathways into therapeutic allies rather than obstacles.
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On episode 117, Host Erin Harris welcomes Paul Perreault, Strategic Advisor, Board Member and former CEO of CSL, who shares insights from his decade leading the company through global expansion and the COVID-19 pandemic. Perreault highlights the importance of patient-centered culture, strategic focus, and adaptive leadership for cell and gene therapy innovators. He offers practical advice on building a values-driven culture that connects employees to patients, the importance of focusing on core competencies to attract capital, and the necessity of continuous leadership development, even advocating for mentorship and emotional intelligence as growth drivers in fast-evolving organizations.
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In episode 116 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Arun Upadhyay, Chief Scientific officer, Head of Research and Development at Ocugen to discuss how the company’s modifier gene therapy platform is redefining treatment possibilities for inherited retinal diseases such as retinitis pigmentosa, Stargardt disease, and geographic atrophy. Dr. Upadhyay explains how Ocugen’s gene-agnostic approach differs from traditional single-gene therapies by targeting shared disease pathways to preserve photoreceptors and slow vision loss across diverse genetic mutations. He also shares key lessons learned from advancing OCU400, the first modifier gene therapy to receive a broad FDA indication for retinitis pigmentosa, including challenges in clinical trial design, endpoint selection, and manufacturing scalability.
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Episode 115 of Cell & Gene: The Podcast features Host Erin Harris' talk with Aliya Omer, Vice President and Global Head of Hematology and Cell Therapy at AstraZeneca. Omer shares valuable insights from her rich experience leading cell therapy development across multiple top companies. She highlights the critical importance of collaboration by breaking down silos across research, manufacturing, regulatory, and commercial teams to deliver innovative therapies efficiently. She also discusses AZ’s diverse and ambitious cell therapy portfolio, encompassing autologous CAR-T, TCR-T, in vivo gene therapies, and regulatory T-cell therapies. She candidly addresses current challenges in manufacturing scalability, patient access, and healthcare system readiness and describes how AZ is prioritizing fast manufacturing platforms and ecosystem-wide partnerships to surmount these hurdles.
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On episode 114 of Cell & Gene: The Podcast, Host Erin Harris talks to Brian Culley, CEO of Lineage Cell Therapeutics, about advancing cell therapy beyond oncology and into transformative treatments for conditions such as spinal cord injury and blindness. Culley shares how Lineage’s allogeneic, off-the-shelf approach, anchored by its OPC1 program, aims to replace lost or damaged cells to restore function, starting with patients who have plateaued after chronic spinal cord injury. He details the DOSED study’s innovative delivery device and thaw-and-inject formulation, both designed to simplify administration, enhance safety, and improve patient access. Harris and Culley also explore Lineage's broader vision for commercial viability in cell transplantation and its collaborative work with the Christopher & Dana Reeve Foundation to drive awareness, research, and investment in spinal cord injury therapies.
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Welcome to Episode 113 of Cell & Gene: The Podcast. Host Erin Harris is joined by Thorsten Gorba, Ph.D., VP Process Development at Aspen Neuroscience to explore how the company is advancing the field of cell therapy manufacturing. Aspen Neuroscience stands at the forefront of integrating machine vision and AI/ML to assess induced pluripotent stem cell (iPSC) colony quality by offering a transformative approach to move beyond the subjective nature of manual evaluation. Dr. Gorba discusses how these technologies bolster reproducibility and scalability and help reduce variability in starting materials. He also covers the regulatory angle, including how the FDA is evaluating digital quality control tools.
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On episode 112 of Cell & Gene: The Podcast, Michael Quigley, Ph.D., Chief Scientific Officer and Global Head of Research at Sanofi talks to Host Erin Harris about the establishment of Sanofi’s dedicated Genomic Medicine Unit (GMU). Dr. Quigley emphasizes in vivo delivery and process optimization to improve patient experience, scalability, and global access. They discuss the importance of partnerships with academia, industry, and regulators, and Dr. Quigley discusses how advances in AI are accelerating research efficiency, molecule optimization, and experimental design across Sanofi’s portfolio. He also points to the breakthroughs likely to revolutionize immunology and gene therapy, such as solutions to pre-existing immunity barriers, improved tissue-specific delivery, regulated and reversible gene therapies, and more precise gene editing.
Cell & Gene: The Podcast and Cell & Gene are part of the Life Science Connect family of resources.
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On episode 111 of Cell & Gene: The Podcast, Host Erin Harris talks to Ernexa Therapeutics' President and CEO, Sanjeev Luther, about how the company is advancing cell therapy innovation for cancer and autoimmune disease through engineered induced pluripotent stem cell-derived mesenchymal stem cells (iMSCs). Luther shares how Ernexa’s synthetic, standardized, off-the-shelf iMSCs are designed to overcome historic hurdles in MSC therapy, including variability, scalability, and manufacturing complexity, by leveraging master cell banks, precise gene editing, and novel product engineering. He also shares insights into the process of developing pro- and anti-inflammatory iMSCs for oncology and autoimmunity.
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This is Episode 4 of "FDA Fridays," a special series from Cell & Gene: The Podcast, Chief Editor Erin Harris speaks with regulatory affairs expert Daniela Drago, Partner, NDA Partners, about how shifting FDA and CBER priorities under the current administration could influence the trajectory of cell and gene therapy development. From the impact of leadership changes and evolving policy directives to the role of funding allocations and public health priorities, Drago sheds light on what CGT developers and sponsors should be watching most closely. She explores regulatory modernization trends, including digital health integration, real-world evidence, and decentralized trial models, and offers practical guidance on how companies can best prepare for potential regulatory shifts while maintaining alignment with FDA expectations for safety, efficacy, and manufacturing rigor.
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In this episode of Cell & Gene Podcast episode, Host Erin Harris talks to Stanford School of Medicine Ph.D. student, Yuanhao Qu, about his work developing CRISPR-GPT, an AI-driven multi-agent system designed to automate genetic experimental design and data analysis, making CRISPR experiments more efficient and accessible, even for non-experts. Qu explains how CRISPR-GPT addresses key challenges such as guide design, delivery methods, off-target prediction, and protocol generation, and shares how collaborations with Princeton helped shape the tool’s architecture and evaluation. Qu also discusses Biomni, a general-purpose biomedical AI agent aimed at supporting a broad range of life science applications, and how the two systems complement each other as building blocks toward an "AI scientist" capable of accelerating discovery across biomedicine. Qu emphasizes the importance of rigorous evaluation, productivity gains, and ethical guardrails to ensure these tools are powerful yet safe for the future of biomedical research.
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This is Episode 3 of "FDA Fridays," a 4-week special series from Cell & Gene: The Podcast. Host Erin Harris talks to Bambi Grilley, Ph.D., Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine and Chief Regulatory Officer for ISCT, a leading expert at the forefront of pediatric cell and gene therapy. Dr. Grilley shares her wealth of experience and unique perspective on the most pressing barriers to accessing transformative treatments for children, from logistical and financial challenges faced by families, to systemic and regulatory hurdles in getting therapies from the lab to the clinic.
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Episode 2 of FDA Fridays, the special series from Cell & Gene: The Podcast, features Host Erin Harris' discussion with regulatory expert Monika Swietlicka, Principal, Regulatory Strategy, Halloran Consulting Group about the key strategies and challenges in navigating FDA regulation for cell and gene therapies. Their conversation covers the importance of defining a therapy’s intended role (curative, chronic, or adjunctive) early in development, the different regulatory expectations based on disease context and patient population, and the crucial impact of unmet medical need on regulatory flexibility. Swietlicka emphasizes the necessity of a clear, scientifically sound regulatory narrative, especially when precedent is lacking, and advocates for early, robust investment in CMC to minimize risks. They also explore the complexities of using surrogate endpoints, planning for long-term patient follow-up under uncertainty, and the value of integrating commercial considerations from the outset. Swietlicka calls for cell and gene therapy developers to adopt a commercial mindset and proactive regulatory engagement to achieve clinical and commercial success.
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In Episode 109 of Cell & Gene: The Podcast, Host Erin Harris talks to Will Chou, M.D., CEO of Passage Bio about the promise and challenges of developing gene therapies for frontotemporal dementia (FTD), a neurodegenerative disease with strong genetic underpinnings. Dr. Chou discusses the biological basis of FTD and the complex scientific, manufacturing, and regulatory hurdles that come with targeting the brain. They discuss the latest interim results from the upliFT-D study of Passage Bio’s PBFT02, including safety and tolerability findings, dose cohort comparisons, and the evolving strategies around immunosuppression. Dr. Chou also highlights upcoming milestones for PBFT02 and what they could mean for advancing gene therapies in neurodegenerative diseases.
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This is Episode 1 of a special series of Cell & Gene: The Podcast, "FDA Fridays." This first episode features Host, Erin Harris' conversation with Kate Spratt, Strategic and Non-Clinical Regulatory Consultant at Spratt Advanced Cell & Gene Therapy Regulatory Consulting. Spratt emphasizes the importance of early and continuous engagement with regulatory authorities, especially through structured meetings like INTERACT and pre-IND sessions. Harris and Spratt cover CMC readiness, filing for RMAT/CIT, BLA approvals and regulatory expectations, and more.
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Cell & Gene: The Podcast Host, Erin Harris, talks to Orca Bio CEO and Co-Founder Nathaniel Fernhoff as he recounts the company’s origins and evolution, tracing its roots to pioneering stem cell research at Stanford University and Dr. Irv Weissman's lab. Seeking to create safer, more effective cures for blood cancers such as AML, ALL, and MDS, Orca Bio developed high-precision cell therapies that utilize purified regulatory T cells and stem cells to minimize complications, such as graft-versus-host disease. Orca Bio spun out of academia to establish its own scalable production platform and clinical pipeline, and Fernhoff explains Orca's flagship product, Orca T, which recently achieved a pivotal milestone by outperforming standard stem cell transplants in a Phase 3 trial.
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On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Ralf Schmid, Ph.D., Associate Director of Preclinical Research at Novartis Biomedical Research, about the evolving use of large animal models in gene therapy development. Dr. Schmid discusses the current reliance on non-human primates (NHPs), their growing logistical and ethical challenges, and the emerging interest in alternatives like genetically engineered pigs and sheep. He outlines key considerations around safety, biodistribution, and immunogenicity that still necessitate large-animal testin —particularly for CNS-targeted AAV therapies — and emphasizes the need for thoughtful study design, responsible sourcing, and diversification in model systems. Dr. Schmid also previews his participation in the upcoming Next Generation Gene Therapy Vectors Summit and reflects on the future of preclinical safety testing in a landscape aiming to balance innovation, rigor, and compassion.
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In this episode of Cell & Gene: The Podcast, George Magrath, CEO of Opus Genetics, shares with Host, Erin Harris, how the company is advancing gene therapies for inherited retinal diseases (IRDs), with a focus on LCA5, a rare and severe form of childhood blindness. Built on the foundational science of Dr. Jean Bennett and leveraging the legacy of Luxturna, Magrath shares how Opus is developing subretinal AAV-based therapies targeting seven distinct mutations. He also explains why gene therapy is particularly well-suited to IRDs, shares compelling early clinical data, and discusses the promise of early intervention in preserving vision and enabling proper neural development.
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In this episode of Cell & Gene: The Podcast, Host Erin Harris interviews Dr. Gloria Matthews, Chief Medical Officer of Aegle Therapeutics, a clinical-stage regenerative medicine company focused on developing therapies for rare and severe dermatologic and immunologic disorders. Dr. Matthews explains how Aegle is pioneering the use of extracellular vesicles (EVs) derived from mesenchymal stem cells, which offer advantages over traditional cell-based therapies. Their discussion highlights Aegle's lead product, AGLE-102 as well as the regulatory and manufacturing challenges of scaling EV-based therapies. They cover the broader therapeutic potential of EVs in areas such as graft-versus-host disease, ocular, and orthopedic disorders, and more.
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On this episode of Cell & Gene: The Podcast, Host Erin Harris welcomes Monika Swietlicka, Principal, Regulatory Strategy at Halloran Consulting Group to discuss the key regulatory challenges facing cell and gene therapy companies. Swietlicka highlights manufacturing complexity and clinical trial design limitations. On gene editing, she notes that the FDA is focused on off-target effects and demands robust data, including functional validation. She emphasizes the importance of early definition of critical quality attributes and a strong comparability strategy to ensure product consistency. She also underscores the growing role of real-world evidence, especially in rare disease settings with limited clinical trial data.
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Host Erin Harris talks to Generate:Biomedicines' EVP of R&D, Dr. Alex Snyder about the convergence of AI, machine learning (ML), and synthetic biology in the development of next-generation therapies. They cover how AI is transforming drug discovery by enabling the rapid design and optimization of therapeutic candidates, particularly in complex fields like immuno-oncology and cell therapy. Dr. Snyder shares how that in the context of CAR-T therapies, AI-driven approaches are used to design and refine each component of the CAR construct. And, Dr. Snyder sets the stage for a broader conversation about how the integration of AI and synthetic biology is not only accelerating drug development timelines but also expanding the realm of what’s possible in cell-based therapeutics.
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In this episode of Cell & Gene: The Podcast, Host Erin Harris sits down with Peter Altman, Ph.D., CEO of BioCardia, to discuss the company’s evolution from a cardiac biotherapeutic delivery firm to a developer of autologous and allogeneic cell therapies for cardiovascular and pulmonary diseases. Altman highlights BioCardia’s CardiAMP program, a precision medicine approach that pre-screens patients based on their cell profiles to improve trial outcomes and reduce costs. He explains why no cardiac cell therapy has yet received FDA approval, citing challenges in delivery, immune rejection, and arrhythmia risks. Altman outlines BioCardia’s near-term roadmap, including regulatory submissions in the U.S. and Japan and the launch of a second trial focused on patients most likely to benefit.
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In this episode of the Cell & Gene podcast, Host Erin Harris speaks with Alicia Zhou, Ph.D., CEO of the Cancer Research Institute (CRI), about CRI’s mission to advance cancer immunotherapy and the critical role of federal funding, particularly from the NIH, in supporting basic and translational cancer research. Dr. Zhou highlights CRI’s focus on funding early-stage science and clinical trials to move immunotherapy toward curing all cancers. She covers the serious implications of recent U.S. Government funding cuts, including NIH budget freezes and halted grant review processes, which are already impacting biomedical research institutions, graduate programs, and clinical trials. In light of the NIH budget cuts, Dr. Zhou explains how CRI is stepping up by committing an additional $2.5 million from its reserves to fund 10 postdoctoral fellowships.
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On the 100th episode of Cell & Gene: The Podcast, Host, Erin Harris, sits down with Audrey Greenberg, Founder and CEO of AG Capital Advisors. Audrey is the award-winning executive, board director, and strategic advisor with a proven track record of launching, scaling, and successfully exiting multi-billion-dollar ventures. They explore the biggest challenges and opportunities facing CGT biotech leaders today; the funding crisis in biotech, the evolving investment landscape, and whether the traditional VC-driven model is still sustainable. They also examine what it really takes to scale a biotech company from startup to market leader, the role of AI-driven drug discovery, and how big tech’s growing influence could reshape the industry. You'll hear their discussion on leadership challenges in biotech, the need for more diverse voices at the top, and what must change to empower the next generation of women leaders in CGT, and more.
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In this episode of Cell & Gene: The Podcast, Host Erin Harris dives into the world of retinal gene therapy with Kenji Fujita, M.D., Chief Medical Officer of Atsena Therapeutics. Atsena develops treatments for inherited retinal diseases, including X-linked retinoschisis (XLRS), a rare genetic condition that currently has no FDA-approved therapy. They talk through the impact of XLRS on patients, the challenges of gene delivery to the retina, and how Atsena’s AAV vector technology is designed to overcome these hurdles. They also discuss the significance of the Fast Track designation recently granted to Atsena’s XLRS program, the role of patient advocacy in rare disease research, and the key milestones to watch for in the months ahead.
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In this episode, Host Erin Harris sits down with Dr. Simrit Parmar, Founder of Cellenkos, to explore how the company is pioneering umbilical cord blood-derived Regulatory T cell (Treg) cell therapies for autoimmune and inflammatory diseases. Dr. Parmar shares the vision behind Cellenkos, the advantages of cord blood-derived Tregs over other sources, and how their CRANE technology platform enhances precision in targeting diseases. They also discuss key findings from their Phase 1b trial for CK0804, the challenges of scaling up off-the-shelf Treg therapies, and what’s next for Cellenkos in 2025.
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Omkar Kawalekar, Ph.D., Senior Manager, NextGen Therapies Manufacturing & Supply Chain Lead, Deloitte Consulting joins Host Erin Harris to share his expert take on how distributed manufacturing models address capacity constraints and supply chain risks, the role of automation in reducing batch-to-batch variability, as well as the power of digital technologies in optimizing production processes. He also covers the key considerations for CGT companies when choosing between in-house manufacturing and CDMO partnerships, and much more.
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In this episode, Erin Harris sits down with Faraz Ali, CEO of Tenaya Therapeutics, to explore the cutting-edge world of gene therapies for heart diseases. As Tenaya makes significant strides in developing innovative treatments, Ali shares insights on Tenaya's gene therapy platform, their in-house manufacturing capabilities, and the potential impact of their therapies on prevalent heart conditions. They also discuss the complexities of commercializing gene therapies, industry-wide developments, and Tenaya's roadmap for 2025. This conversation offers a unique glimpse into the future of cardiac care and the transformative potential of gene therapies in treating heart diseases.
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Helen Sabzevari, Ph.D., President and CEO of Precigen, joins Host, Erin Harris to discuss how the company utilizes gorilla adenovirus vectors. These vectors offer significant advantages in delivering large genetic payloads, a crucial factor in developing effective gene therapies. Sabzevari also discusses Precigen's recent BLA submission for PRGN-2012, which includes Phase 1/2 pivotal study results where over 50% of patients achieved Complete Response and more than 85% experienced decreased surgical interventions.
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Christopher Horan, Chief Technical Operations Officer, Artiva Biotherapeutics joins Cell & Gene: The Podcast Host, Erin Harris, to discuss the advantages natural killer (NK) cells have offer over T cells in terms of safety and efficacy for autoimmune disease. They take a deep dive into the key factors that make NK cells promising for ‘off-the-shelf’ cell therapy products. They cover Artiva's "manufacturing first" approach to enabling scalable NK cell production, and much more.
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Cell & Gene: The Podcast Host Erin Harris talks to Christopher Choi, Ph.D., MBA, SVP Industry Partnership, Director of GMP and Cell Manufacturing Facility, and Associate Professor of Oncology at Roswell Park Comprehensive Cancer Center about the Center's Buffalo, NY-based cell and gene therapy hub slated to open in early 2025. They cover the increasingly important role of automation and digital technologies in CGT manufacturing. Choi also offers his advice to new and emerging CGT biotechs on how to correctly tackle manufacturing as they begin their product development journey.
Email Erin at erin.harris@cellandgene.com with topics you'd like us to cover in future episodes.
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Cell & Gene: The Podcast Host Erin Harris talks to Lothar Roessig, M.D., SVP, Integrated Product Team Lead, Congestive Heart Failure at Asklepios BioPharmaceutical, Inc. (AskBio) about the biggest challenges in developing gene therapies for cardiovascular diseases, specifically congestive heart failure (CHF). They discuss AB-1002, the gene therapy the company is developing for the treatment of CHF and why a gene therapy approach is significantly different from traditional therapies.
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On this episode of Cell & Gene: The Podcast, Host Erin Harris, invites BlueRock Therapeutics' Dr. Amit Rhakit to take a deep dive into therapy options for Parkinson's disease (PD). They discuss how BlueRock's bemdaneprocel's mechanism of action differs from traditional PD treatments. They cover the limitations of current PD medications, how cell therapies might offer a different approach to long-term symptom management, and how BlueRock is doing traditional dopamine replacement therapy differently.
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Rita Johnson-Greene, COO at Alliance for Regenerative Medicine (ARM) joined Cell & Gene: The Podcast Host, Erin Harris, on site during 2024 Meeting on the Mesa to record their discussion. Johnson-Greene shared information about the Joint Clinical Assessment (JCA) in Europe and its potential impact on cell and gene therapies, how globalization impacts patient access, an update on ARM's Grow Internship Program, as well as a few key takeaways from the event that sector professionals can anticipate impacting the CGTs in the next 12 months and beyond.
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On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Dennis Williams, Senior Vice President of Late Stage Development at Adaptimmune. Williams shares valuable insights into the groundbreaking world of TCR T-cell therapies, with a particular focus on the TECELRA trial. The trial led to the FDA approval of Tecelra, marking a significant milestone as the first engineered T-cell therapy for solid tumors. Williams delves into the complexities of developing this innovative treatment for synovial sarcoma, a rare and aggressive form of soft-tissue cancer. He also highlights the unique challenges and promising outcomes associated with TCR T-cell therapies, offering a comprehensive understanding of this cutting-edge approach in cancer immunotherapy.
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This episode of Cell & Gene: The Podcast was recorded on site during ARM's 2024 Meeting on the Mesa in Phoenix. Tune in to hear Host Erin Harris talk to Miguel Forte, M.D., Ph.D., CEO of Kiji Therapeutics, President of ISCT, and Executive Committee Member of ARM about Kiji's clinical plan and why iPSC-MSC therapy for inflammatory diseases shows great promise. They also cover this year's conference and what to expect during and after the event.
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Carsten Linnemann, Ph.D., CEO of Neogene Therapeutics and Head of Oncology Cell Therapy Clinical Development, AstraZeneca joins Cell & Gene: The Podcast’s Erin Harris to discuss T-cell receptor (TCR) therapies for solid cancers. They talk through key challenges and opportunities in developing TCR therapies targeting neoantigens as well as the potential benefits of TCR-T therapies for cancer treatment. Linnemann shares his thoughts on the future landscape of cell therapy in oncology and beyond. Previous episodes of Cell & Gene: The Podcast have focused on TCR therapies, including Episode 50, featuring Affini-T Therapeutics’ Co-Founder, CEO, and President Dr. Jak Knowles. Be sure to check out that episode, too.
https://www.cellandgene.com/doc/targeting-oncogenic-drivers-for-solid-tumor-cancers-with-affini-t-therapeutics-dr-jak-knowles-0001
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Ray Therapeutics' Co-Founder and CEO, Paul Bresge, joins Host Erin Harris to talk about optogenetics and how the company’s optogenetics approach differs from other gene therapies on the market developed to restore vision in retinal diseases like retinitis pigmentosa.
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Tessera Therapeutics' CEO, Dr. Michael Severino, joins Host Erin Harris to discuss Gene Writing, the biotech's genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Severino discusses why delivery has always been a challenge for gene editing technologies and why Gene Writing is different when it comes to efficient delivery.
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Allogene Therapeutics' EVP of R&D and CMO, Dr. Zachary Roberts, penned an original article for Cell & Gene titled, The Path Forward for CLL is Allogeneic. In his article, Dr. Roberts explains that chronic lymphocytic leukemia (CLL) is the most common leukemia in the U.S. And that while CLL remains a disease that is managed, it is not one that is often cured. In this episode, Host Erin Harris follows up Roberts' article with in-depth questions around existing issues with current CAR T approaches, "auto versus allo," and why allogeneic is the path forward. They also discuss how and why allogeneic therapies are truly scalable.
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Applications (BLAs) of recent approvals and complete responses for novel products or rare diseases as well as BLAs currently under review. Benton shares information about FDA Draft Gudiances, including Platform Technology Designation Program for Drug Development: Guidance for Industry. She also provides a regulatory outlook for CGT developers.
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Debra Miller, CEO and Founder and Dr. Michael Kelly, Chief Scientific Officer at CureDuchenne talk to Host Erin Harris about the FDA approval for the expansion to the label indication for ELEVIDYS from Sarepta Therapeutics as well as how CureDuchenne works with various biotechs developing gene therapies for Duchenne muscular dystrophy (DMD). Miller and Kelly also share their advice and recommendations for companies developing drugs for DMD - while much progress has been made, more innovation is needed.
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Lucas Harrington, Co-Founder and Chief Scientific Officer of Mammoth Biosciences, joins Erin Harris to shed light on the biotech's proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. They discuss why in vivo delivery continues to be a bottleneck against the clinical advancement of gene-editing-based medicines and what ultracompact CRISPR systems are designed to do.
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KSQ Therapeutics' CSO, Micah Benson, Ph.D., joins Erin Harris to discuss how Tumor-Infiltrating Lymphocytes (TILs) as a treatment modality have the potential to treat a variety of solid tumor types. Benson explains KSQ's Phase 1/2 clinical study, KSQ-001EX, which consists of TILs in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing. In addition to solid tumors, Benson also addresses the therapeutic potential for autoimmune disease.
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Strand Therapeutics' CEO and Co-Founder Jake Becraft joins Erin Harris to discuss genetic regulation and how the company's programmable mRNA constructs combine genes for self-replication derived from RNA viruses with genetically programmed logic circuits that control the location, timing, and intensity of expression of therapeutic proteins within the patient’s body, enabling precise and controlled delivery. They also cover the role synthetic biology plays to genetically program mRNA to deliver therapies.
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Elixirgen Therapeutics' Aki Ko joins Erin Harris to detail the company's Bobcat mRNA therapeutic and how it differs from traditional exon-skipping drugs and AAV-microdystrophin in treating DMD. Regarding delivery, Ko explains the advantage of the mRNA approach over the AAV approach.
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Dr. Bambi Grilley is Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine AND she is Chief Regulatory Officer at ISCT. Dr. Grilley talks to us about her role as CRO at ISCT and the global regulatory landscape for cell and gene therapies. She takes a deep dive into the pediatric patient journey, and she shares her expectations for ISCT 2024 Vancouver.
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In anticipation of ISCT 2024 in Vancouver, Canada, Dr. Bruce Levine is back on Cell & Gene: The Podcast to share information about the event as well as his expertise on the future of CAR-T therapy for solid tumors, autoimmune disease, and more. He talks about his time and learnings as President Elect, President, and Immediate Past President of ISCT. He covers Cellicon Valley and Philadelphia's success as on-going CGT hub, and much more.
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Dr. Marcus Conant is CMO at Addimmune, a biotech startup developing a cell therapy for HIV that spun out of cell and gene therapy biotech American Gene Technologies (AGT). He spent his career on the front lines of HIV treatment and research and remains an advocate for the HIV patient. He formed the Kaposi’s Sarcoma Research & Education Foundation in 1982, which later became the San Francisco AIDS Foundation. On this episode, Dr. Conant shares they why behind cell and gene therapy to treat HIV, and he explains Addimmune’s lead asset, AGT103-T, which is designed to provide broad protection using silencing RNA to inhibit binding/entry of the virus, halt replication, and prevent escape.
BlueRock Therapeutics' SVP Head of Development, Dr. Ahmed Enayetallah, joins Cell & Gene: The Podcast Host, Erin Harris, to discuss the company's phase I clinical trial for Parkinson’s disease, which continues to show positive trends at 18 months. They cover the important role induced pluripotent stem cells' (iPSCs) play in the trial, and they also discuss the company's investigational cell therapy, bemdaneprocel.
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In 2021 on Episode 13, Vor Bio’s President and CEO, Dr. Robert Ang shared data about the company’s lead engineered hematopoietic cell (eHSC) product candidate. Dr. Ang is back to talk to Cell & Gene: The Podcast Host, Erin Harris, about how Vor has treated 8 patients demonstrating clinical proof of concept and is also in the clinic with a CAR-T that could be used in combination with shielded stem cell transplants. They also talk through Vor’s decision to build an internal cell therapy manufacturing facility co-located with their Cambridge, MA-based headquarters.
Orca Bio's CMO, Dr. Scott McClellan, joins Erin Harris on this episode of Cell & Gene: The Podcast to discuss how and why toxicities continue to stymie allogeneic cell therapies. They cover Orca Bio's plan to potentially lowering the risk of GvHD as well as Orca-T, the investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies.
On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Life Biosciences' CSO, Sharon Rosenzweig-Lipson, Ph.D., about the Boston-based biotech's cellular rejuvenation therapies for the treatment of age-related diseases. They cover Life Biosciences' lead program, a gene therapy called OSK that is being advanced in two optic neuropathies – a rare eye disease of aging called non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. They also cover they why behind gene therapy as a modality, as well as Life Biosciences' partnership with Forge Biologics to manufacture AAV for the cellular rejuvenation technology.
Rare Disease Day is February 29, 2024, and in honor of this important day, Cell & Gene: The Podcast Host Erin Harris sits down with Jackson Laboratory’s (JAX) Rare Disease Translational Center (RDTC)'s VP, Cat Lutz. They discuss RDTC's mission, the specific qualifications that make up a rare disease, how the study of rare diseases can potentially advance progress in common diseases, and much more.
On this episode of Cell & Gene: The Podcast, Christopher McDonald, Global Head of Technical Operations at Kite, a Gilead company, talks to Host Erin Harris about Kite's recent FDA approval of manufacturing process change resulting in reduced median turnaround time for Yescarta. They talk through the most significant steps Kite took to reach this approval, lessons learned from having worked with the FDA, scaling up technologies for cell therapy manufacturing, and more.
The Dedham Group's Jennifer Klarer, M.Sc.Eng., Partner and Head of Cell & Gene Therapy, joins Erin Harris on this episode of Cell & Gene: The Podcast for a deep dive into pricing and value for CGTs. Klarer shares a detailed breakdown of the most misunderstood aspects of cell and gene therapy pricing, value, and affordability. She details how manufacturers can get ahead of addressing time to treatment issues typically experienced at launch. They cover obtaining adequate investment dollars, and much more.
Paul Lammers, M.D., M.Sc., CEO of Triumvira Immunologics joins Cell & Gene: The Podcast to talk to Host Erin Harris about TAC, the company's proprietary T cell Antigen Coupler, which has both autologous and allogeneic approaches. They also discuss targeting relapsed or refractory HER2-positive solid tumors and CLDN18.2-positive solid tumors. And they cover a realistic outlook on the evolution of cancer treatment.
Kristin Yarema, Ph.D., CEO, Poseida Therapeutics sits down with Cell & Gene: The Podcast Host Erin Harris to discuss four of the biggest challenges the CGT sector faces when it comes to the development of off-the-shelf therapies. Kristin shares how 2024 might see some real progress in allogeneic therapies.
For this third and final episode recorded at ARM's 2023 Meeting on the Mesa, Cell & Gene: The Podcast Host, Erin Harris, sat down with Greg Kunst, president, CEO, and Board member at Aurion Biotech. Listen in as they discuss the cell therapy used to treat corneal endothelial disease and how Greg navigated the event as a first-timer attending Meeting on the Mesa.
Vittoria Biotherapeutics’ CEO, Nick Siciliano, joins Erin Harris on this episode of Cell & Gene: The Podcast to discuss the company's mission to expand the applications for CAR-based therapeutics by employing novel cell engineering and gene editing technologies.
Richard Wilson, Senior Vice President, Primary Focus Lead, Genetic Regulation at Astellas sat down with Cell & Gene: The Podcast's Erin Harris at ARM's 2023 Meeting on the Mesa to discuss the gene therapy deal making landscape as well as Astellas' recent deals and collaborations. They also discussed Astellas' strategic approach since having acquired Audentes back in 2020.
Ken Mills, CEO, President, and Director at REGENXBIO joins Erin Harris for this episode of Cell & Gene: The Podcast to discuss the company's progress with its Phase I/II Affinity Duchenne trial of RGX-202 for the treatment of Duchenne Muscular Dystrophy. They discuss the future of AAV gene therapy as well as achieving scalability with consistent yield and product purity in gene therapy manufacturing.
Alliance for Regenerative Medicine's COO, Rita Johnson-Greene, joined Cell & Gene: The Podcast host, Erin Harris, in person at the 2023 Meeting on the Mesa to discuss this year's event. Johnson-Greene also shared what's ahead for 2024 as the event heads to a new location. They discussed major topics and trends at the event as well as ARM's GROW internship program.
A pioneer and an icon in diabetes research, Camillo Ricordi, M.D., is the Professor of Surgery, Director of Cell Transplant Center and Director Emeritus of the Diabetes Research Institute at the University of Miami Miller School of Medicine, Florida (UM-DRI). He joins Cell & Gene: The Podcast's Erin Harris to discuss the biggest and most troubling challenges facing the treatment of Type 1 Diabetes (T1D). They also discuss the future of cell and organ transplantation in treating T1D as well as the latest innovations in biopreservation.
Umoja Biopharma's CEO, Andy Scharenberg, M.D., joins Cell & Gene: The Podcast's Erin Harris to discuss the evolution of allogeneic, in vivo CAR-T cell therapeutics. They discuss The Climb, or The Colorado Laboratory and Innovation Manufacturing Building, which is Umoja’s lentiviral vector development and manufacturing facility. They also cover recent progress in lentiviral vector manufacturing.
The FDA's Center for Biologics Evaluation and Research (CBER)'s Dr. Peter Marks makes his second appearance on Cell & Gene: The Podcast. This time Dr. Marks talks to Host, Erin Harris, about base editing and prime editing and their potential to meet unmet medical need. They discuss what clinical holds say about the FDA’s views on gene editing. They also discuss heritable genome editing.
As SVP Cell Therapy Franchise Lead at Bristol Myers Squibb, Lynelle Hoch leads her team to bring more curative cell-based therapies to patients. She joins Cell & Gene: The Podcast's Host, Erin Harris, to discuss the near-term future of CAR-T. They also take a deep dive into the various innovations and patient access progress being made in both solid tumors and hematologic malignancies in the CGT sector at large as well as at Bristol Myers Squibb.
On this episode of Cell & Gene: The Podcast, Rachel Haurwitz, Ph.D., CEO at Caribou Biosciences joins Host Erin Harris to discuss the evolution of genome editing, its present state, as well as its undeniable potential. They also discuss Caribou's chRDNA genome editing technology and how it differs from CRISPR-Cas9.
In June 2023, PDA held its Advanced Therapy Medicinal Products Conference in Baltimore, MD. At the conference, Stephan Krause, Ph.D., Executive Director, AS&T, CT Global Quality at Bristol Myers Squibb, sat down with Cell & Gene: The Podcast Host Erin Harris to discuss the event's major themes and topics as well his focus areas for cell therapy in 2023 and beyond.
Dr. Francois Vigneault, Co-Founder, President, and CEO at Shape Therapeutics joins Cell & Gene: The Podcast's Erin Harris to discuss how RNA technologies are shaping the future of gene therapy. They also discuss AI's role in gene therapy as well as the how the CGT sector is benefiting from RNA, AI, and synthetic biology.
Dr. Andrew Anzalone, Head of the Prime Editing Platform and Scientific Co-Founder of Prime Medicine joins Cell & Gene: The Podcast to talk with Host Erin Harris about prime editing - what it is and why it is a major technological advancement in gene editing. Anzalone explains why prime editing has a broad applicability for different tissue and cell types to be used in different therapeutic applications.
Glenn Wright, President and CEO of Parenteral Drug Association (PDA), sat down with Cell & Gene: The Podcast's Erin Harris at the 2023 PDA ATMP conference to discuss not only the conference's theme, “Navigating Through CMC Challenges,” but also Wright's take on CGT manufacturing strategy based on his years in industry. Wright shares his insight on the past, present, and future of the cell and gene therapy space.
Dr. Usman "Oz" Azam is CEO of Inspirna now but has spent much of his career in the cell and gene therapy space dedicating years as Novartis’ Global Head of Cell & Gene Therapy, President and CEO of Tmunity Therapeutics, and more. Given Oz’s tenure in cell and gene therapy space, I invited him to Cell & Gene: The Podcast to get his valuable take on the state of the space. During our discussion, we cover best practices in engaging with the FDA, the potential in solid tumor cancer treatment, the biggest yet solvable hurdles facing the sector today, and much more.
Affini-T Therapeutics combines a validated TCR discovery platform with synthetic biology switches to attack tumor biology at its root cause. Co-Founder, CEO, and President Dr. Jak Knowles joins Cell & Gene: The Podcast Host Erin Harris to discuss how the company engineers immune cells to target oncogenic driver mutations, such as KRAS, the most prevalent oncogenic driver mutation in solid tumors, to minimize potential tumor escape mechanisms.
Parviz Shamlou, Vice President and Executive Director at Thomas Jefferson University joins Cell & Gene: The Podcast to talk to Host Erin Harris about Jefferson Institute for Bioprocessing (JIB), the full-service development and training organization specializing in biopharmaceuticals and biologics. Shamlou explains how JIB will move forward cell and gene therapies as well as JIB’s partnership with NIBRT (National Institute for Bioprocessing Research & Training), and much more.
Shankar Ramaswamy, M.D., CEO & Co-Founder, Kriya Therapeutics joins Cell & Gene: The Podcast's Erin Harris for a discussion on the evolving role computational biology plays in gene therapy development. They also discuss the company's lessons learned from building its own North Carolina-based in-house manufacturing facility as well as Ramaswamy's insight on the future of gene therapy.
Dr. Linda Marbán, Ph.D., CEO of Capricor Therapeutics, a clinical-stage biotech focused on the development of cell and exosome-based therapeutics for the treatment and prevention of serious diseases, joins Cell & Gene: The Podcast's Erin Harris to discuss the current state and the near-term future of exosomes. Marbán discusses Capricor’s exosome program and the company’s proprietary allogeneic cardiosphere-derived cells (CDCs) and engineered exosomes. She also covers the company's HOPE clinical trial series' progress to treat Duchenne muscular dystrophy (DMD).
In December 2022, Atara Biotherapeutics’ Ebvallo received European Commission approval as the first-ever therapy for adults and children with EBV+ PTLD. The ground-breaking approval represents the first approval of an allogeneic T-cell immunotherapy ever, globally. Atara Bio's CEO, Pascal Touchon, talks to Cell & Gene: The Podcast's Erin Harris about the biopharma's path to regulatory approval, commercialization plans for Ebvallo, an in-depth look at EBV+ PTLD, the connection between EBV and serious diseases like multiple sclerosis (MS), and much more.
Cell & Gene: The Podcast Host, Erin Harris, welcomes Romesh Subramanian, Ph.D., CEO of Ascidian Therapeutics, a Boston-based biotech focused on rewriting RNA to the pod. During the episode, Subramanian defines RNA exon editing, and they discuss the differences between RNA exon editing and gene editing. With current programs in ophthalmology, neurological and neuromuscular disorders, as well as rare diseases, Subramanian explains the overwhelming potential of RNA exon editing.
When it comes to manufacturing, many cell and gene therapy companies struggle to automate the diverse processes needed to scale manufacturing. London-based PA Consulting's Associate Partner, Paolo Siciliano, talks to Cell & Gene: The Podcast's Erin Harris about how lean, small biotechs with limited resources and limited funds can strive for automation success. They also discuss what cell and gene therapy companies should do today to address tomorrow’s scalability challenges and what the long-term effects on the business may be from such upfront work.
On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Andy Ramelmeir, EVP Head of Technical Operations at Sangamo Therapeutics, a biotech based in Brisbane, California that applies cell and gene therapy to combat Hemophilia A and other genetic diseases. Ramelmeir explains the why behind the company's decision to build its own Phase 1/2 cGMP AAV in-house facility. He also talks broadly about manufacturing cell and gene therapies and the potential and realistic solutions to needed to drive down manufacturing costs.
Miguel Forte is Entrepreneur in Residence at AdBio, a VC firm based in France, and President Elect of ISCT. Forte and Cell & Gene: The Podcast host, Erin Harris, break down market outlook perspective for the cell and gene therapy space. He provides valuable tips to employ when seeking VC partnership, and he covers ISCT's primary focus areas and what to expect from ISCT 2023 Paris.
MustangBio's Robert Sexton, VP Program and Alliance Leadership, explains the why behind the need for this role in any CGT company. Having worked for Novartis, Sanofi, Legend Biotech, and more, with responsibilities having included quality, operations, and order management, Sexton is currently responsible for aspects of CMC. As such, he addresses the industry's top CMC challenges as well as sound advice for future scale up.
Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) within the Food and Drug Administration (FDA) sits down with Cell & Gene: The Podcast host Erin Harris to discuss the most anticipated areas of innovation in clinical development, and he offers recommendations to companies in their engagement with the FDA in order to make manufacturing more streamlined and cost effective. Dr. Marks also provides advice to listeners about how and when to engage with CBER, and much more.
Shoreline Biosciences' Chief Scientific Officer Dr. Robert Hollingsworth shares his insight on intelligently designed allogeneic off-the-shelf, standardized, and targeted iPSC-derived natural killers (NK) and macrophage cellular immunotherapies. He explains the company's preclinical testing on a potential NK cell therapy for acute myeloid leukemia. We discuss how the field recognizes the importance of off-the-shelf NK cell therapy not only for cost reduction but also for improving safety measures. As such, Dr. Hollingsworth talks through why NK cells necessary to move immunotherapies forward.
BlueRock Therapeutics' President and CEO Seth Ettenberg, Ph.D. talks to Cell & Gene: The Podcast listeners about the company's Phase 1 Clinical Trial for Advanced Parkinson’s Disease. Ettenberg also covers the most promising therapeutic applications for iPSCs currently in development as well as the major regulatory challenges that the field faces for their clinical use. We also cover how far iPSCs have come and what future progress may entail.
Orgenesis' CEO, Vered Caplan explains the business model behind providing cell and gene therapies that may be manufactured at the point of care. She details how working with hospitals and healthcare providers to enable them to participate in CGT development on their sites and providing these therapies in-house by adapting them to closed system manufacturing rather than relying on the typical biotech model is an important step forward in CGT manufacturing.
Capstan Therapeutics' CEO, Laura Shawver, Ph.D., talks us through the company's in vivo CAR therapies and what led to the in vivo cell engineering breakthrough that has been decades in the making. Shawver explains the promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo. She also details best practices to garner funding and investment partnership.
PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases has focused on delivery. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.
On the heels of Congress' reauthorization of the Prescription Drug User Fee Authorization (PDUFA) Act, BridgeBio's Chief Regulatory Affairs Officer, Adora Ndu, explains the Commitment Letter, the programs that may be rolled out under PDUFA VII, why right-sizing CBER will help the cell and gene sector going forward as well as how sponsor companies can prepare for 2023 from a regulatory perspective.
Arrowhead Pharmaceuticals is a biopharmaceutical company based in Pasadena, CA that develops medicines that treat intractable diseases by silencing the genes that cause them. Javier San Martin, Arrowhead's CMO, talks to Cell & Gene: The Podcast listeners about the similarities and differences between RNAi and CRISPR. He also explains the roadmap for RNAi as well as the company's pipeline development strategy, which includes pre-clinical to Phase 3 trials.
Will Junker, Head of Manufacturing Quality at Kite Pharma, talks to Cell & Gene: The Podcast listeners about the challenges associated with capacity planning, whether to partner with a CDMO, and why it's important to prioritize inventory over time-to-market. We also discuss why developing a better understanding of the vector manufacturing process is critical to bringing continuous and secure supply to market.
With 20+ gene therapy targets in Novartis Gene Therapies’ pipeline, President Chris Fox and her team are hyper-focused on newborn screenings as the pathway to helping more and more patients. The company's initial gene therapy, Zolgensma, for spinal muscular atrophy (SMA) has been approved in more than 40 regions and countries and has been used to treat more than 2,300 patients worldwide. Fox details what’s next on the commercialization front for the therapy, and she shares advice with Cell & Gene: The Podcast listeners about what it takes to commercialize a therapy.
Sernova Corp. is a clinical-stage regenerative therapies company currently focused on insulin-dependent diabetes. CEO and President, Philip Toleikis, Ph.D., details the company's on-going clinical trial to treat Type 1 diabetes (T1D) as well as the very real potential to progress from simply masking symptoms to having a functional cure for T1D. Toleikis also provides his take on the near-term future of regenerative medicine.
Emily Moran, VP of Vector Manufacturing and Avi Nandi, VP of Process Development at the Center for Breakthrough Medicines share key considerations for biotechs looking to partner with a CDMO. During this epsiode, biotechs will learn how to reduce risk, lower costs, and how to better manage expectations when it comes the biotech / CDMO relationship.
Dr. Carl June, an HIV gene therapy pioneer and the father of CAR-T cell therapy, joins Cell & Gene: The Podcast to discuss the current state of CAR-T therapy, what's on the short-term horizon for immunotherapy, on-going manufacturing capacity issues, an update on Emily Whitehead, the first child to receive CAR-T cell therapy on an experimental basis, and more.
TC BioPharm's CEO Bryan Kobel explains gamma delta t cells (GDT) in depth as well as the advantages of using GDT cells as a cell therapy vehicle. He discusses why the publicly-traded, clinical-stage cell therapy company conducts and manages all clinical trials in house as well as the unique challenges and benefits that come along with doing so.
Poseida Therapeutics is a broad platform technology company led by CEO, Mark Gergen. During this episode, Gergen explains how and why the company is moving away from AAV delivery in favor of nanoparticle delivery. He shares some of the biggest hurdles the CGT sector faces when it comes to the development of off-the-shelf therapies as well as the manufacturing challenges Poseida has had to overcome and how they did it.
Autologous cell therapy clinical trials and products are very complex, and the stakeholder investment is diverse. Immatics' Director, Clinical Operations, Michael Mehler, explains why new hybrid roles operate in clinical operations, clinical supply chain, strategic operations, and medical affairs to help bridge the gap between the sponsor company and the clinical site for clinical trials in an autologous setting. Mehler talks through the main aspects of a site operations lead and why the role is imperative to commercial success as well as how he sees the role evolving in the near term. He also explains what size CGT companies will benefit most from these new hybrid roles.
Cell & Gene: The Podcast is produced by Cell & Gene in partnership with Applied Biosystems by Thermo Fisher Scientific.
Dr. Metin Kurtoglu, COO at Cartesian Therapeutics, explains the clinical-stage biotech's autologous RNA cell therapies, Descartes-08 for Myasthenia Gravis and Descartes-11 for multiple myeloma. He also explains Descartes-25, their allogeneic RNA cell therapy and the reasons for the move to "off-the-shelf," and why the future of RNA cell therapy is not confined to rare and fatal diseases. We also discuss post-care for patients having received infusions.
Encoded Therapeutics' Chief Manufacturing Officer Andy Stober explains the unique commercial manufacturing issues in gene therapy and how to address them. Stober also shares some of the lessons he's learned, which may educate the next generation of gene therapy scientists to accelerate future manufacturing success. We also talk about what developers need to consider regarding the ethical responsibilities in gene therapy.
Amit Kumar, Ph.D., President and CEO at Anixa Biosciences details the company’s progress on its Phase 1 trial for ovarian cancer. We also discuss why the company’s ovarian cancer program is integral for initial proof of concept for other solid tumor indications. He covers why CAR-T therapy uses an existing hormone/hormone receptor relationship to create a targeted approach to treat ovarian cancer. Dr. Kumar shares how the field of cell therapy may evolve in the short term, and much more.
Celyad Oncology’s CMO, Dr. Charlie Morris, shares the benefits and challenges of the allogeneic approach versus the autologous approach and why a non-gene edited shRNA approach may result in better efficacy and safety for CAR-T therapies.
Rocket Pharmaceuticals CEO Dr. Gaurav Shah offers his take on why gene therapy developers have been plagued by clinical holds, whether the FDA’s AAV-focused CTGTAC meeting's takeaways will hold up throughout 2022, and more.
BlueSphere Bio's CEO Dr. David Apelian explains the difficulties associated with treating solid tumor cancers as well as what the CGT sector needs to get right in the short term and why.
Cue Biopharma's Anish Suri, Ph.D., explains why the clinical-stage biopharma is engineering a novel class of injectable biologics to selectively engage and modulate targeted T cells directly within the body to transform the treatment of cancer.
REGENXBIO's VP of Regulatory and Science Policy, Nina Hunter, Ph.D., shares potential solutions to gene therapies' regulatory roadblocks and why the accelerated approval pathway is so pertinent.
Selecta Biosciences' President and CEO Dr. Carsten Brunn details the company's ImmTOR platform, which can mitigate unwanted immune responses that cause immune-related toxicities that impose significant treatment challenges with current therapies.
Cellevolve Bio's founder and CEO. Dr. Derrell Porter shares his experience with starting up a cell therapy company and why we, as an industry, need to advocate for more Black leaders and leaders of color in the biotech industry.
Dr. Michael Singer, CSO at Cartesian Therapeutics, details why RNA has advantages over other cell therapies, the company's three RNA trials, and the therapeutic benefit of multiple modifications.
Vor Biopharma's CEO Dr. Robert Ang explains the company's lead engineered hematopoietic cell (eHSC) product candidate, genome engineering of HSCs, and the cell manufacturing process needed to leverage genome engineering technologies for the cells they're creating.
Fernanda Copeland, Global Head, Patient Advocacy & Engagement at AVROBIO, explains how to incorporate community education, patient feedback, and more to improve clinical trial development and recruitment.
Carisma Therapeutics’ Dr. Barton breaks down the latest in CAR-M technology as well as the company's Phase 1 First-in-Human Study of Adenovirally Transduced Anti-HER2 CAR Macrophages in Subjects with HER2 Overexpressing Solid Tumors.
XyloCor Therapeutics' EXACT clinical trial is a Phase 1/2 multicenter, open-label, single arm, dose escalation trial for coronary artery disease — the most common type of heart disease — with gene therapy at its center.
Dr. Amy Nicole Nayar, VP of US Patient Advocacy and Government Affairs at Novartis Gene Therapies explains what patient engagement should encompass in the cell and gene field as well as the unique challenges cell and gene therapy companies face where a patient engagement officer provides essential help.
BridgeBio's CEO of Gene Therapy, Dr. Eric David, explains the company's gene therapy programs for Canavan disease and congenital adrenal hyperplasia, their manufacturing plans, and the platform underpinning their novel approach to gene therapy.
Spark Therapeutics' Clinical Development Lead, Hematology, Dr. Tiffany Chang explains the company's data from its ongoing Phase 1/2 clinical trial of investigational SPK-8011 for hemophilia A, the largest gene therapy trial in this disease to date as well as the misconceptions and unknowns about the disease.
Karen Kozarsky, Ph.D., Co-Founder and CSO of SwanBio, explains how the company is pioneering a deep and varied pipeline of gene therapies for genetically defined neurological diseases with a focus on the spinal cord.
Bob Levis was diagnosed with Chronic Lymphocytic Leukemia in 2002 and was one of the early experimental trial participants at Penn Medicine for Dr. Carl June’s CAR-T therapy. Bob shares his remarkable journey with CLL as well as his important work with the CLL Society.
Dr. Murat Kalayoglu, CEO at Cartesian Therapeutics, details the cell and gene therapy sector’s advancements in RNA cell therapy and the why behind the company's decision to build its own wholly-owned GMP manufacturing facility.
University of Pennsylvania’s Dr. Bruce Levine explains his road to adoptive immunotherapy trials, overcoming the gap between academia and industry, and how Philadelphia earned the "Cellicon Valley" name for its continued innovation in cell and gene therapy — and more.
In this episode of Cell & Gene: The Podcast, University of Pennsylvania Health System’s CEO Kevin Mahoney talks to Host Erin Harris about how hospital systems must evolve to meet the needs of cell and gene therapy patients as these complex therapies are commercialized. They discuss what comprises UPHS and the ongoing innovation it brings to advanced therapies as well as how the Health System stays patient-focused at all costs.
During this first episode of Cell & Gene: The Podcast, Host Erin Harris talks to Enzyvant’s CEO Rachelle Jacques about the biotech’s lead asset, investigational regenerative therapy RVT-802 for congenital athymia. Jacques provides an update on FDA approval and details what’s next for RVT-802. She explains tissue-based therapies — how far they’ve come and the work that’s left to be done. And, they discuss what being patient-focused means in the world of rare diseases.
Cell & Gene, the online resource that delivers in-depth content from authoritative authors and sources to professionals in the CGT sector, is thrilled to introduce “Cell & Gene: The Podcast.” Cell and gene therapies offer huge potential to treat a wide range of diseases and their possibilities represent a new era in medicine. “Cell & Gene: The Podcast” will explore approved and pipeline therapies, new trends in manufacturing, the long, regulated road to commercialization, and everything in between. In each episode, Cell & Gene Chief Editor, Erin Harris, will talk to an industry or academic leader about their current initiatives and how they are moving the sector forward. (Music by Royalty Free Music From Bensound)