Foundation Fighting Blindness: Recent Episodes
Foundation Fighting Blindness
The Foundation Fighting Blindness is committed to finding treatments and cures for blinding retinal diseases that affect more than 10 million people in the United States. To learn more, go to FightingBlindness.org.
Dr. Alicia Kemble explains how the Foundation is using venture philanthropy to drive treatments for inherited retinal diseases even closer to the finish line.
Sepul Bio recently announced that it completed enrollment of its Phase 2b safety and efficacy clinical trial for ultevursen – an investigational RNA therapy to treat patients who have retinitis pigmentosa (RP) associated with mutations in the USH2A gene.
FDA approves Lumvoa, a new treatment for thyroid eye disease by Foundation Fighting Blindness
Belite Bio submits new drug application to FDA for its Stargardt treatment by Foundation Fighting Blindness
New host Maddie Mossman interviews retinal surgeon Dr. Staropoli, the first medical doctor to win a NASCAR race.
2020 Llura Liggett Gund Award recipient honored for pioneering gene therapy that transformed vision research
Data monitoring committee recommends proceeding with registration-enabling study for ATSN-201
MeiraGTx Announces the Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP) by Foundation Fighting Blindness
Strategic funding extends cash runway through 2029 and advances multiple inherited retinal disease programs
VG801 shows sustained vision improvements through 12 months with favorable safety profile
Potential cell therapy, DSP-3077, gains special regulatory status to support development for inherited retinal disease.
First-of-its-kind small molecule therapy targets protein misfolding at the root of RHO-adRP
episode is Ben's final for Eye on the Cure, but the podcast will continue on with a new host. Stay tuned!
Novel neuroprotective approach could benefit RP patients regardless of genetic mutation
Beyond radio show featuring Foundation Fighting Blindness CEO Jason Menzo. Jason discusses the Foundation's mission and the remarkable progress achieved over the past 55 years in advancing diagnostics, treatments, and cures for blinding diseases. He also shares insights into ongoing advocacy efforts on Capitol Hill to preserve critical vision research funding through the FDA and National Eye Institute. The conversation concludes with an invitation for listeners to register for VISIONS 2026, the Foundation's global conference taking place June 12–13 in Fort Worth, Texas.
Dr. Prem Subramanian, of the University of Colorado Sue Anschutz-Rodgers Eye Center and School of Medicine, talks with host Ben Shaberman about thyroid eye disease (TED), a condition that is often misdiagnosed and can cause a range of symptoms including discomfort and vision loss. Dr. Subramanian discusses potential causes, symptoms, and therapeutic approaches. This episode is sponsored by Amgen.
Josh Steinberg and his father, Jon, talk with host Ben Shaberman about their close-knit family's journey with Josh's vision loss and eventual diagnosis of retinitis pigmentosa, as well as their volunteer leadership roles in driving the Foundation's mission. Jon is Chair of the Foundation's Science Committee and Vice Chair for Science on the Board. Josh is the former Chair of the Strategic Council and also a Board director.
Lindsey Rambo, a special education teaching assistant, mom, and wife, talks to host Ben Shaberman about her journey with Leber congenital amaurosis 5 (LCA5), participation in Opus Genetics' LCA5 gene therapy clinical trial, and her appearance on Good Morning America to tell her story about meaningful vision improvements.
The company has also fully enrolled the Phase 2/3 VISTA clinical trial for its XLRP gene therapy.
Dr. Deniz Dalkara, a research director at INSERM in France, talks to hose Ben Shaberman about her emerging gene-agnostic, gene therapy designed to restore cone-mediated vision in people with advanced retinitis pigmentosa. Known as SPVN20 and clinically developed by SparingVision, the treatment recently moved into a clinical trial in Europe. Dr. Dakara also discusses her earlier work in retinal gene and optogenetic therapy development. This episode is sponsored by Boehringer Ingelheim.
Tinlarebant slowed disease progression by 36 percent in the Dragon Phase 3 clinical trial.
Greta Streimikyte, a blind para-athletic running champion from Ireland, talks to host Ben Shaberman about growing up with substantial vision loss from retinopathy of prematurity and her competitive running career. Greta has won gold in the women's 1500 meters at the 2018 and 2021 European Para Athletic Championships and is a three-time Paralympian. This episode is sponsored by Boehringer Ingelheim.
The approach may provide better visual acuity than current optogenetic therapies in clinical trials.
The emerging therapy improved vision for some participants in an earlier clinical trial.
Durable vision improvements were also observed out to 18 months for previously dosed adult patients in the trial.
Lance Baldo, MD, CEO at Beacon Therapeutics, talks to host Ben Shaberman about his company's fully enrolled Phase 3 VISTA clinical trial for laru-zova, a gene therapy for X-linked retinitis pigmentosa (RPGR mutations) that's showed promising results in earlier trials. They also discuss Beacon's forthcoming clinical trial for its dry age-related macular degeneration gene therapy.
NACA is a gene-agnostic, oral medication designed to preserve vision in people with retinitis pigmentosa and Usher syndrome.
September 5, 2025. Dr. George Magrath, CEO of Opus Genetics, talks with host Ben Shaberman about the company's evolution and progress in clinically developing gene therapies for several inherited retinal diseases including those caused by mutations in the genes: LCA5, BEST1, RHO, RDH12, MERTK, NMNAT1, and CNGB1.
The Foundation funded earlier, proof-of-concept studies for the Best1 gene therapy and later launched Opus to develop several IRD gene therapies.
The trial is evaluating a cell dose that is 50 percent higher than the highest dose used in a previous Phase 2b trial.
August 8, 2025. Dr. Christina Ohnsman talks to host Ben Shaberman about Tern Therapeutics, a start-up company she co-founded to develop gene therapies for Batten disease and potentially other conditions. Dr. Ohnsman reviews early results for a Batten disease (CLN2) retinal gene therapy clinical trial. She also reflects on her previous work as a pediatric ophthalmologist and roles with Spark Therapeutics and Regenxbio.
Susvimo is an FDA-approved treatment that continuously delivers drug through a tiny, implanted capsule
July 25, 2025. Dr. Christine Curcio, one of the world's foremost experts in age-related macular degeneration (AMD) pathogenesis and the associated risk of advanced vision loss, talks to host Ben Shaberman about her epical career and recent Proctor Medal lecture with Dr. Cynthia Owsley at the 2025 meeting of the Association for Eye and Vision Research (ARVO). Her work has led to a highly effective test for predicting who is at greatest risk of losing vision from AMD.
July 11, 2025. Richard Small, CEO of Neurotech, talks to host Ben Shaberman about ENCELTO™, the first and only FDA-approved treatment for adults with idiopathic Macular Telangiectasia Type 2 (MacTel). The therapy is a tiny, implantable capsule, the size of a rice grain, that delivers neurotrophic factors to preserve the patient's retinal cells and vision. The approach was previously developed to treat retinitis pigmentosa and age-related macular degeneration.
June 13, 2025. Dr. Philip Beales, a co-founder of Axovia Therapeutics, talks to host Ben Shaberman about Bardet Biedl syndrome, ciliopathies, and his company's emerging gene therapy for Bardet Biedl syndrome type 1 (BBS1) which is moving toward a clinical trial.
May 23, 2025. Lenore von Krusenstiern, MD, PhD, at BlueRock Therapeutics talks to host Ben Shaberman about the company's clinical trial for a photoreceptor replacement therapy for people with inherited retinal conditions such as retinitis pigmentosa and cone-rod dystrophy. The approach, which uses induced pluripotent stem cells to develop photoreceptor precursors, comes out of the lab of stem cell pioneer David Gamm, MD, PhD, University of Wisconsin-Madison.
Part B of the trial will enroll nine adult patients as well as three pediatric patients.
Cindy Elden, who co-founded the Usher III Initiative with her late father, talks to host Ben Shaberman about her journey with Usher syndrome type 3 (USH3), the decades-long work and persistence of her non-profit, and the clinical trial it launched for an USH3 small molecule therapy.
April 3, 2025. In this episode of Eye on the Cure, Jen Bernstein at Horizon Government Affairs, and Sharon King of Taylor’s Tale, talk to host Ben Shaberman about how to schedule and conduct a visit with your House Member in your local district to advocate for important issues. With federal eye research funding at great risk and a Congressional district work period coming in mid-April, now is an ideal time to schedule a visit with your House Member.
March 21, 2025. Professor Michel Michaelides, a world-renowned clinical researcher for inherited retinal diseases at Moorfields Eye Hospital and University College London, talks to host Ben Shaberman about the extraordinary vision improvements for young blind children receiving gene therapy for Leber congenital amaurosis 4 (LCA4). Professor also talks about emerging therapies in clinical trials for X-linked retinitis pigmentosa and Stargardt disease.
The technology was previously funded by the Foundation for the treatment of retinitis pigmentosa and dry age-related macular degeneration.
March 7, 2025. Aaron Nagiel, MD, PhD, a retina surgeon at Children’s Hospital Los Angeles, talks to host Ben Shaberman about how gene therapy is administered to the retina, what the surgery and recovery is like for patients, and the vision improvements for young patients receiving LUXTURNA, the first FDA-approved gene therapy for the eye or an inherited condition. Dr. Nagiel also discusses other retinal gene therapies in clinical development.
In earlier clinical trials, the emerging gene therapy greatly reduced the treatment burden associated with injections of approved anti-VEGF therapies.
February 21, 2025. Ricky Enger, the Director of Practical Help for Hadley, talks with host Ben Shaberman about her personal and professional journeys as someone born completely blind and the many assistive and accommodative technologies that are helpful to people with low vision. At Hadley, Ricky develops technology workshops and other community initiatives for older adults new to vision loss.
Though unaffected by vision loss, she brought great passion to driving the Foundation’s mission.
January 24, 2025. Aniz Girach, MD, the chief medical officer at SpliceBio, talks to host Ben Shaberman all about the company’s emerging protein-splicing therapy for Stargardt disease which has received clearance from the FDA to move into a clinical trial.
January 10, 2025. Damon Lembi talks to host Ben Shaberman about his journey as a college baseball star, father, and CEO of Learnit (a live learning platform), and how the diagnosis of an inherited retinal disease informs his leadership style, personal growth, and relationships. Damon is also a best-selling author and host of the Learn-It-All podcast.
December 20, 2024. Dr. Sheldon Rowan, a professor and researcher at Tufts University, talks to host Ben Shaberman about the impact of the glycemic index, microbiome, and diet on the development of age-related macular degeneration and optimization of overall retinal health. Dr. Rowan will also discuss his grant from the Foundation and the Free Family for studying diet and AMD risk. The grant is also enabling Dr. Rowan to evaluate an FDA-approved drug and an emerging gene therapy as potential AMD preventions.
The company has also launched a natural history study of patients to inform the planned clinical trial.
December 6, 2024. Bob Bell, PhD, chief scientific officer at Ascidian Therapeutics, talks with host Ben Shaberman about STELLAR, the company's Phase 1/2 clinical trial for its emerging RNA-rewriting therapy for Stargardt disease (ABCA4 mutations). Dr. Bell also provides overviews of Stargardt disease, the role of RNA in cells, and the advantages of RNA re-writing over other genetic treatment approaches. Learn more about the clinical trial at STELLAR (https://stellarstargardttrial.com/). Learn more about the prescreening study for STELLAR at PRESCREEN (https://stellarstargardttrial.com/#participation-criteria).
The Foundation funded Dr. David Gamm for development of the induced pluripotent stem cell approach to be used in the trial.
Sharon King, co-founder and president of Taylor’s Tale, talks to host Ben Shaberman about her family's journey when her daughter Taylor was diagnosed with Batten disease, a life-limiting, neurodegenerative disease that includes vision loss as a symptom. Taylor’s Tale is raising awareness of, and research funding for, Batten disease. Sharon is also a tireless advocate for the needs of the rare disease community.
WhiteCaneDay is here! Let's celebrate the strength and independence the white cane symbolizes! Do you use a white cane or other tools? Share your story with #ShareYourVision!www.FightingBlindness.org/ShareYourVision
Researchers hope the molecule will slow the vision and hearing loss associated with USH3.
To help spread awareness during October’s Blindness Awareness Month, the Foundation Fighting Blindness invites you to share your unique experiences with blinding diseases through our social media campaign, #ShareYourVision. Just as no two fingerprints are alike, our perception of the world through our eyes is equally distinct. Let’s come together as a beacon of hope, emphasizing the resilience found in the uniqueness of our sight. Join us this October and share your story through written word, audio, or video and inspire others! Learn more at: www.FightingBlindness.org/ShareYourVision
Dr. Kia Washington, a professor in the Division of Plastic and Reconstructive Surgery at the University of Colorado, Anschutz Medical School, talks to host Ben Shaberman about her work in whole eye transplantation. Dr. Washington is also the first Black female plastic surgeon in the country to hold the title of full professor and she discusses the challenges of ensuring diversity and inclusion in her field. This episode is sponsored by Johnson & Johnson.
To help spread awareness during October’s Blindness Awareness Month, the Foundation Fighting Blindness invites you to share your unique experiences with blinding diseases through our social media campaign, #ShareYourVision. Just as no two fingerprints are alike, our perception of the world through our eyes is equally distinct. Let’s come together as a beacon of hope, emphasizing the resilience found in the uniqueness of our sight. Join us this October and share your story through written word, audio, or video and inspire others! Learn more at: www.FightingBlindness.org/ShareYourVision
“Tatel’s humility and tenacity shine. The result is a stirring reflection on an extraordinary life,” Publishers Weekly
Dr. Zuhal Butuner, chief medical officer at Sepul Bio, talks with host Ben Shaberman about her company's two emerging RNA therapies – sepofarsen (for LCA10) and ultevursen (for USH2A) – formerly in clinical trials sponsored by ProQR. Dr. Butuner discusses how the RNA treatments work, what was learned from earlier clinical trials, and Sepul Bio's plans to move the therapies back into human studies.
Hannah Scanga, the supervisor of genetic counseling and the ocular genetics service at the University of Pittsburgh Medical Center, talks to host Ben Shaberman about the basics of genetic testing and counseling, as well as deeper topics such as inheritance patterns, reasons for variations in disease severity, and variants of unknown significance.This episode of Eye on the Cure Podcast is sponsored by Johnson & Johnson.
Leonide Saad, PhD, founder and chief scientific officer of the biotech Alkeus, talks to host Ben Shaberman about his company’s emerging therapy, gildeuretinol, a modified form of vitamin A which has performed well in clinical trials for people with Stargardt disease.
Check out this exclusive Beacon Story podcast with WROC’s Adam Chodak and professional Para triathlete Deb Chucoski as they discuss living with Stargardt disease and the amazing achievements of those who refuse to let it define them. Deb’s journey is a testament to the power of resilience and determination. Hear her story and how the Paralympic Games provide a platform for athletes with vision loss and any disability to showcase their extraordinary talents! This special Foundation Fighting Blindness Beacon Story podcast episode is sponsored by Alkeus Pharmaceuticals.
Previously, the formula was thought to only reduce risk of early age-related macular degeneration advancing to late-stage disease
Karen Petrou, the new board chair at the Foundation, talks to host Ben Shaberman about her vision for the Foundation moving forward. Karen has been an active director on the Foundation’s board for nearly two decades. She is co-founder and managing partner of Federal Financial Analytics, a firm providing analytical and advisory services on legislative, regulatory, and public-policy issues affecting financial services companies. Karen has RP and lives in Washington, DC, with her guide dog, Ike.
Honoring John Corneille, JD, on his retirement from the Foundation Fighting Blindness and the Lulie's Light Awards.
Speakers: Todd Durham, PhD; Kristin Macdonald and Thomas Mendel, MD, PhDThis session will provide clinician and participant perspectives on inherited retinal disease clinical trials and discuss the importance of natural history studies for interventional clinical trials.
Speakers: Dan Chung, DO, MA; Michael Voevodsky, MBA abd Jami Kern, MBA, PhDThis session will discuss gene agnostic therapeutic approaches, which are applicable to multiple retinal diseases and patients at early to mid-stage of disease progression.
Speakers: Paul Bernstein, MD, PhD and Abigail Fahim, MD, PhDThis session will discuss how dietary supplements and lifestyle choices can influence eye health (e.g. smoking, exercise).
Speakers: Bikash Pattnaik, PhD and Ramiro Maldonado, MDThis session will feature clinicans and researchers discussing recent updates for Leber Congenital Amaurosis (LCA).
Speakers: Kenji Fujita, MD and Renee Ryals, PhDThis session will discuss therapies that are gene and/or mutation specific, including gene therapy and genome editing.
Emerging Technologies: Retinal Organoids, RNA Editing, Retinal Prosthetics, and Artificial Intelligence.Speakers: Robert Bell, PhD; Karl Wahlin, PhD; Hossein Ameri, MD, PhD and Kerry GoetzThis session will discuss hot topics om retinal biology, including retinal organoids, RNA editing, retinal prosthetics, and the use of AI in ophthalmology.
Speaker: Abigail Fahim, MD, PhDThis session will feature clinicans and researchers discussing recent updates for other retinal diseases, including choroideremia and X-linked retinoschisis.
Speakers: Nieraj Jain, MD and Andrew Goldberg, PhDThis session will feature clinicans and researchers discussing recent updates for specific retinal diseases.
Stargardt Disease and Age Related Macular Degeneration.Speakers: Thomas Wubben, MD, PhD; Jason Miller, MD, PhD and Esther Biswas-Fiss, PhDThis session will feature clinicans and researchers discussing recent updates for Stargardt disease and age-related macular degeneration.
Speakers: Robert Hyde, MD, PhD and Miranda Scalabrino, PhDThis session will feature clinicans and researchers discussing recent updates for Retinitis Pigmentosa.
Speakers: David Corey, PhD and Benjamin Bakall, MD, PhDThis session will feature clinicans and researchers discussing recent updates for Usher Syndrome.
Speaker: Francisco Richardson, ODLearn about devices, technologies, and low vision adaptations for work, home and education.
Speakers: Leslie Hoskins and Alyssa OtisLearn what to expect when applying for a guide dog, including review of the application materials and potential outcomes. Additionally, an in-depth discussion on the skills required to be approved for a guide dog from the International Guide Dog Federation (IGDF) standards.
Foundation Board Chair David Brint sits down with Judge David Tatel to discuss vision loss, his career and latest book.
Speakers: Christy Vanek and Janni Lehrer-SteinJoin us for a fireside chat conversation on advocacy across the personal, workplace, and legislative levels. We will explore how advocacy relates to personal identity, what it means to act as an advocate, and personal strategies for making sustainable advocacy choices.
Speaker: James AshworthThis session will discuss practical tips and strategies for traveling with vision loss on Southwest Airlines, information such as accessible transportation options, navigating airports, finding accessible accommodations, and using assistive technology. The session aims to empower participants with the knowledge and confidence to explore air travel regardless of their level of vision loss.
Speakers: Todd Durham, PhD; Kristin Macdonald and Thomas Mendel, MD, PhDThis session will focus on empowering patients to decide if participation in a clinical trial is right for them and discuss how natural history studies pave the way for interventional clinical trials.
Science Plenary SessionSpeakers: Todd Durham, PhD; Kristi Lee, MS, CGC and Ramiro Maldonado, MDThis session will discuss the importance of genetic testing and give an overview of the Foundation's My Retina Tracker® Registry.
Speakers: Paul Bresge; Eric Daniels, MD and Ethan Rossi, PhDThis session discusses optogenetics, a method of endowing non-photoreceptor cells with light-sensing capabilities. This is the most applicable to late-stage patients.
Speakers: Paul Bresge; Eric Daniels, MD and Ethan Rossi, PhDThis session discusses optogenetics, a method of endowing non-photoreceptor cells with light-sensing capabilities. This is the most applicable to late-stage patients.
Speakers: Katherine Uyhazi, MD, PhD and Srinivasa Sripathi, PhDStem cell therapies are based on the premise that we could restore vision if we could replace missing or nonfunctional functional cells, either through transplantation or by inducing existing eye cells to create more photoreceptors. These therapies are likely to be most beneficial for mid- to late-stage patients.
Speakers: Katherine Uyhazi, MD, PhD and Srinivasa Sripathi, PhDStem cell therapies are based on the premise that we could restore vision if we could replace missing or nonfunctional functional cells, either through transplantation or by inducing existing eye cells to create more photoreceptors. These therapies are likely to be most beneficial for mid- to late-stage patients.
Francois Paquet-Durand, PhD, a professor from the University of Tübingen and co-founder of the company Mireca, talks to host Ben Shaberman about the long journey of an emerging, Foundation-funded drug for the treatment of certain forms of retinitis pigmentosa. The episode highlights the challenges that scientists often encounter when developing a drug for a retinal disease, and the persistence and tenacity that are required to keep it moving forward.
FDA-approved in 2021, the tiny implantable capsule provides sustained delivery of treatment, but was recalled in 2022.
For this episode of Eye on the Cure, Michael Telias, PhD, an assistant professor at the University of Rochester in New York, talks with host Ben Shaberman about forthcoming clinical trials to evaluate the FDA-approved drug disulfiram (aka, Antabuse) for improving vision in people with retinitis pigmentosa and other retinal diseases. Disulfiram was originally developed to deter people from abusing alcohol.
Dr. Peter Campochiaro, a clinician-scientist and the Eccles Professor of Ophthalmology and Neuroscience at the Johns Hopkins University School of Medicine, talks to host Ben Shaberman about N-acetylcysteine (NAC), a drug that shows promise for preserving cone vision (central vision and acuity) in people with retinitis pigmentosa. The powerful antioxidant has moved into a 30+ site, Phase 3 clinical trial called NAC Attack.
Researchers believe the FDA-approved drug can reduce the hyperactivity of ganglion cells to improve vision.
Peter Wallsten, a veteran newspaper journalist and head of investigative reporting at The Washington Post, talks to host Ben Shaberman about his evolving career as a reporter and editor and the impact that Stargardt disease has had on his journey and ambitions. Peter also reflects on recently receiving a Pulitzer Prize for his team's 15-part series on the role of the AR-15 assault rifle in mass shootings and how the weapon has become one of the best-selling firearms in the US.
The emerging treatment is designed to work for people with RP and other conditions regardless of the gene mutation causing disease
No serious adverse events were reported. One patient had encouraging improvement in retinal sensitivity.
Matthew Whitaker talks to host Ben Shaberman about his remarkable achievements and success as a young jazz pianist and composer. Matthew was born three months prematurely and weighed less than two pounds. The oxygen he needed to stay alive also caused total blindness. But now 23 years old, the renowned music prodigy has recorded four albums, received several honors and awards, and appeared on TV shows such as Showtime at the Apollo, the Today Show, Ellen, and 60 Minutes.
The XLRS gene therapy was well tolerated and resolved damaging schisis cavities in patients receiving the lowest dose.
The company’s innovative Gene Coding™ platform enables seamless insertion of DNA into selective genomic locations.
Kapil Bharti, PhD, a senior investigator and director of the Intramural Research Program at the National Eye Institute (NEI), talks with host Ben Shaberman about his clinical trial underway for an innovative cell therapy for geographic atrophy (advanced dry age-related macular degeneration) as well as a clinical trial of the drug metformin for Stargardt disease.
The company reported positive two-year results for its Phase 2 clinical trial of MCO-010.
Opus Reports Vision Improvements for Patients in LCA5 Gene Therapy Clinical Trial by Foundation Fighting Blindness
Hema Ramkumar, MD, a retinal specialist, surgeon, and founder of the company Oculogenex, talks with host Ben Shaberman about her emerging therapy that targets mitchondria to slow the progression of intermediate dry age-related macular degeneration and potentially other retinal diseases. She also discusses her experiment that will put mice in space so researchers can gain a better understanding of retinal degeneration.
Peter Quinn, PhD, a principal investigator and associate research scientist at Columbia University, talks to host Ben Shaberman about the promise of emerging CRISPR/Cas9 gene editing therapies, including base and prime editing approaches, for inherited retinal diseases. Dr. Quinn also reviews gene editing projects ongoing in his lab for patients with mutations in CRB1 and PRPH2.
Known as retinal progenitors, the company’s jCells® are designed to preserve photoreceptors in people with retinitis pigmentosa (RP) and other retinal diseases.
Beacon Therapeutics has reported vision improvements for five of eight patients receiving the high dose of its X-linked retinitis pigmentosa (XLRP) gene therapy in the Phase 2 SKYLINE clinical trial. Known as AGTC-501, the emerging gene therapy is for patients with mutations in RPGR, the gene most frequently associated with XLRP. Interim, 12-month results for SKYLINE were reported on February 7, 2024, by Mark Pennesi, MD, PhD, director, Ophthalmic Genetics at the Retina Foundation of the Southwest, at the 47th Macula Society Meeting.
Dr. Jose Sahel, one of the most honored and accomplished leaders in the retinal disease research space, talks with host Ben Shaberman about his establishment and leadership of renowned ophthalmology clinical research centers at the University of Pittsburgh Medical Center and Institut de la Vision in Paris. They also discuss emerging inhertied retinal disease therapies from SparingVision, a biotech co-founded by Dr. Sahel.
ACDN-01 is the only genetic medicine entering clinical development for Stargardt disease.
Justin Kauflin, a renowned jazz pianist who lost his vision as a boy, talks to host Ben Shaberman about his extensive touring, numerous collaborations, and close friendship with legendary trumpeter Clark Terry. Their story was captured in the hit documentary film Keep On Keepin' On. Justin became a jazz pro at the age of 14, toured globally with Quincy Jones, and was voted jazz artist of the year by VEER magazine.
Dr. Robert MacLaren, a world-renowned retinal surgeon, clinical trial investigator, and research lab leader at the University of Oxford, talks to host Ben Shaberman about the successes and challenges in retinal gene therapy development and clinical trials, as well as advances in robotics for improving retinal surgery outcomes.
Jason Comander, MD, PhD, clinical researcher, surgeon, and director of the Inherited Retinal Disease Disorders Service at Mass Eye and Ear, talks to host Ben Shaberman about his administration of LUXTURNA gene therapy to RPE65 patients, his team's re-analysis of vitamin A therapy for retinitis pigmentosa, and passion for retinal research and patient care.
Known as antisense oligonucleotides, the treatments performed encouragingly in ProQR’s clinical trials
James Rath, a renowned filmmaker and accessibility consultant, talks to host Ben Shaberman about his vision loss journey and travel documentary, Blindspots. The new streaming program, on CuriosityStream.com, follows James as he explores the globe in search of accessible adventures, remarkable blind personalities, and great food.
Michael Voevodsky, MBA, CEO at MitoChem Therapeutics, talks to host Ben Shaberman about his company's emerging, gene-agnostic treatment designed to boost mitochondrial function for preserving vision in people with retinitis pigmentosa, age-related macular degeneration, and other retinal conditions.
The company’s molecule is designed to restore some vision to people with ultra-low or no vision caused by advanced retinitis pigmentosa and other retinal diseases.
The report also concludes that vitamin E supplementation accelerates vision loss for RP patients.
Omar Mahroo, MB, PhD, talks with host Ben Shaberman about electrophysiological testing for the retina, namely the electroretinogram (ERG), and its important role in evaluating and diagnosing inherited retinal diseases. Dr. Mahroo is a consultant ophthalmologist and retinal specialist at Moorfields Eye Hospital and St. Thomas’ Hospital in London, and Professor of Retinal Neuroscience at University College London.
Rachelle Lin, OD, MS, FAAO, an optometrist and assistant professor at the Southern California College of Optometry at Marshall B. Ketchum University, talks with host Ben Shaberman about the critical role of optometrists in providing care and low-vision resources for patients with inherited retinal diseases and other eye conditions.
Ocugen Reports Updated Results for Clinical Trial of Modifier Gene Therapy by Foundation Fighting Blindness
David Birch, PhD, at the Retina Foundation of the Southwest in Dallas, talks to host Ben Shaberman about his productive and impactful 40-plus-year career as a retinal disease researcher and clinical trial investigator. Dr. Birch also discusses the challenges and opportunities for advancing more therapies through trials and out to the people who need them.
September 1, 2023. Brendan Creemer talks to host Ben Shaberman about how he is taking on the diagnosis of Usher syndrome 1F by dedicating his education and career to becoming a researcher for retinal disease treatments and cures.
First cohort of patients has been dosed in Phase 1/2 clinical trial taking place in Pittsburgh and Paris
The AAV.SPR gene delivery system used in the trial is designed to more safely reach targeted retinal cells
Known as antisense oligonucleotides, the treatments performed encouragingly in ProQR’s clinical trials
Artur Cideciyan, PhD, one of the world’s foremost authorities in measuring vision and structural changes in people with retinal diseases, talks with host Ben Shaberman about current imaging tools, functional tests, and his experience as an investigator in several prominent clinical trials at the University of Pennsylvania, Scheie Eye Institute.
Rachel Huckfeldt, MD, PhD, a clinical researcher and assistant professor of ophthalmology at Mass Eye and Ear, talks with host Ben Shaberman about caring for patients with inherited retinal diseases (IRD), the challenges and opportunities with clinical trials, and the educational and professional journey that led her to the IRD research field.
Alex Cohen, PhD, a marketing professor with retinitis pigmentosa, talks with host Ben Shaberman about his career in the hospitality industry and launch of Accessible Pharmacy, a comprehensive pharmacy service for people with vision loss.
Jami Kern, PhD, chief clinical officer at Nacuity Pharmaceuticals, talks with host Ben Shaberman about NACA, the company's emerging gene-agnostic therapy for Usher syndrome and retinitis pigmentosa in a Phase 2 clinical trial in Australia. Their conversation includes a discussion about oxidative stress, which NACA is designed to mitigate, and timing for the efficacy readout from the Phase 2 study.
Kari Branham, a genetic counselor with Kellog Eye Center at the University of Michigan, talks to host Ben Shaberman about a variety of topics related to genetic testing and counseling for people with inherited retinal diseases. Their conversation includes discussion of: common misconceptions about genetic testing, variants of unknown significance, when re-testing makes sense, and when and if family members should be tested.
The company is expanding the trial to enroll younger patients with less advanced disease
Aaron Osborne, MD, chief medical officer and chief development officer at Nanoscope Therapeutics, talks about vision restoration in clinical trials of the company's optogenetic therapy for people with advanced vision loss from retinitis pigmentosa and Stargardt disease.
During his Proctor Award Lecture, Dr. Pierce reviewed encouraging clinical trial results for Editas’ CRISPR/Cas9 treatment for people with LCA10
News | Budd Tucker Honored with Award for Advancements in Retinal Therapy Development by Foundation Fighting Blindness
April 28, 2023. Henry Klassen, MD, PhD, co-founder of jCyte and clinical researcher at UC Irvine, talks to host Ben Shaberman about jCyte's clinical trial results and future plans for developing its cell-based therapy for preserving vision caused by retinitis pigmentosa, Usher syndrome, and related retinal conditions.
Clinical trial participants with advanced vision loss from RP were able to identify fruits and vegetables
April 14, 2023. Electronica musician and composer Jason Taylor, aka Aether, talks with podcast host Ben Shaberman about his craft, inspirations, and how vision loss from retinitis pigmentosa has influenced his work. Jason's ambient, ethereal music has been featured in a variety of popular video games including: viewfinder, mythwrecked: ambrosia island, Rocket League, and Kingdoms and Castles. He has a new EP out called Moonstone. Learn more at aetheraudio.co.uk and @atheraudio.
March 31, 2023. Marinna Madrid, PhD, co-founder and chief product officer at Cellino, talks about her groundbreaking work in developing lasers as a doctoral student and her company’s innovative approach for manufacturing personalized cell-based therapies including those for retinal diseases.
Most participants with advanced retinitis pigmentosa (RP) in the trial had improved navigation and/or object discrimination in reduced lighting conditions
March 10, 2023. Paul Bresge, chief executive officer at Ray Therapeutics, talks to host Ben Shaberman about his company’s emerging optogenetic therapy for restoring vision to people with advanced forms of retinitis pigmentosa (RP), Stargardt disease, and geographic atrophy (advanced dry age-related macular degeneration). Paul also reflects on his prior role as chief executive officer at jCyte and how the diagnosis of RP for his daughter changed the trajectory of his career.
February 24, 2023. Dean Bok, PhD, a distinguished professor and renowned researcher at UCLA, talks with host Ben Shaberman about his pivotal discovery in the late 1960s of the daily renewal process for photoreceptors in the retina and other early, groundbreaking discoveries about retinal diseases that laid a foundation for therapy development for years to come.
February 10, 2023. Rebecca Alexander — a psychotherapist, keynote speaker, author, fitness instructor, and disability rights advocate — talks candidly with host Ben Shaberman about her journey with Usher syndrome. Reflecting on her own experiences, she discusses how people with vision loss and their loved ones can practice good emotional care and advocate for their needs.
January 20, 2023. Paul Bernstein, MD, PhD, from the Moran Eye Center, University of Utah, and a member of the Foundation’s Scientific Advisory Board talks to host Ben Shaberman about his clinical practice for retinal disease patients, his early research into vitamin A metabolism in the retina, and nutrition for people with retinal conditions such as retinitis pigmentosa and Stargardt disease.
January 6, 2023. For the first Eye on the Cure episode for 2023, host Ben Shaberman highlights some of the promising clinical advancement made in therapy development over the last year. His recap includes discussions of emerging treatments from: SparingVision, Atsena Therapeutics, Alkeus, Belite, Apellis, Iveric Bio, MeiraGTx, AGTC, 4DMT, and Opus Genetics, which, after the recording of the episode, reported its acquisition of rights to gene therapies for Best disease and RP (RHO).
December 16, 2022. Rando Allikmets, PhD, from Columbia University, talks to host Ben Shaberman about his remarkable and serendipitous discovery more than two decades ago of ABCA4, the gene associated with Stargardt disease. The breakthrough changed the trajectory of his career, changed the landscape of Stargardt disease research, and led to the development of gene therapies and visual cycle modulators.
The emerging therapy is designed to work independent of the mutated gene causing retinitis pigmentosa
The LCA5 gene therapy will be the first emerging Opus treatment to move into a human study
December 2, 2022. Host Ben Shaberman interviews Sue Bramhall who launched Mind's Eye Travel to host journeys around the world for people with vision loss. Affected with RP, Sue channels her passion for travel and planning to organize group adventures that include great eating, tactile experiences, and other non-visual experiences for her guests. Sighted guides also join the excursions to help along the way. Upcoming trips include Europe, Africa, and New Zealand. Learn more at www.MindsEyeTravel.com.
November 18, 2022. Kenji Fujita, MD, chief medical officer at Atsena Therapeutics, talks to host Ben Shaberman about retinal disease gene therapies including positive results from his company's gene therapy clinical trial for people with Leber congenital amaurosis 1 (GUCY2D mutations) as well as Atsena's emerging gene therapies for X-linked retinoschisis and Usher syndrome type 1B.
November 4, 2022. In this episode of Eye on the Cure, host Ben Shaberman talks with LambdaVision's Nicole Wagner, PhD, and Jordan Greco, PhD, about the company's emerging protein-based artificial retina, the promise it holds for restoring vision, and the low-gravity, manufacturing experiments that have been conducted at the International Space Station.
A blinding disease diagnosis can be overwhelming. Some good advice can go a long way. Do you have any advice for someone newly diagnosed? #ShareYourVision
Ask. Questions. #ShareYourVision to tell others how to best support you.
Learn more about how you can #ShareYourVision at: www.FightingBlindness.org/ShareYourVision
Monica Pruitt shares her #ShareYourVision story about being diagnosed with Usher syndrome.
Dan Day shares his #ShareYourVision story about being diagnosed with retinitis pigmentosa.
Blindness is not a complete blackout for everyone; it's a spectrum. What does blindness mean to you? #ShareYourVision this October. Learn more at: www.FightingBlindness.org/ShareYourVision
Individuals sit in front of a black background and share about how others can best help them as part of our #ShareYourVision campaign. Learn more about how you can #ShareYourVision at www.FightingBlindness.org/ShareYourVision
Chad shares his #ShareYourVision story about being diagnosed with retinitis pigmentosa.
Raquel shares her #ShareYourVision story about being diagnosed with retinitis pigmentosa.
JJ Martinez shares her #ShareYourVision story about being diagnosed with retinitis pigmentosa.
"If you never had to do anything hard, you would never learn how creative, strong, assertive, resilient you are." What have you learned about yourself?
Brett Knight shares his #ShareYourVision story about being diagnosed with retinoschisis.
Martha Steel shares her #ShareYourVision story about being diagnosed with Usher syndrome.
To help spread awareness during October Blindness Awareness Month, we’re inviting you to share your vision-loss journey through written word, audio or video as a part of our October “Share Your Vision” campaign. You can choose whether to share your story with a small select group of people you know or to share your story more broadly. We are here to help either way! Visit www.FightingBlindness.org/ShareYourVision to learn more.
To help spread awareness during October Blindness Awareness Month, we’re inviting you to share your vision-loss journey through written word, audio or video as a part of our October “Share Your Vision” campaign. Visit www.FightingBlindness.org/ShareYourVision to learn more. To see the video for this audio, please visit our YouTube channel at https://youtu.be/ywe64utKr5Q.
October 7, 2022. Podcast host Ben Shaberman talks with Jacque Duncan, MD, a retinal specialist and clinical researcher at UCSF and chair of the Foundation's Scientific Advisory Board (SAB), about her work with adaptive optics for hi-resolution imaging of the retina, her challenging but rewarding role on the SAB, and how the research pipeline enables her to convey hope to all patients and families affected by inherited retinal diseases.
To help spread awareness during October Blindness Awareness Month, we’re inviting you to share your vision-loss journey through written word, audio or video as a part of our October “Share Your Vision” campaign. Visit www.FightingBlindness.org/ShareYourVision to learn more. To see the video for this audio, please visit our YouTube channel at https://www.youtube.com/watch?v=68Mhyh8o1PI.
Michelle Glaze shares her #ShareYourVision story about being diagnosed with retinitis pigmentosa.
September 23, 2022. Dr. Sandy Greenberg talks to host Ben Shaberman about his incredible life's journey of going blind suddenly at the age of 19 to earning advanced degrees from Columbia and Harvard, receiving a White House Fellowship, serving on the Council of Foreign Relations, and founding the company which developed the computer system for the lunar excursion module that transported the first men to the moon. His compelling memoir, "Hello Darkness, My Old Friend," reflects on these and many other accomplishments as well as his lifelong friendship with Art Garfunkel. Dr. Greenberg and his wife, Sue, launched the Campaign to End Blindness to generously fund vision-restoring research.
September 9, 2022. Jean Bousquet, a certified orientation and mobility specialist with the Office of Rehabilitation Services in Rhode Island, talks to podcast host Ben Shaberman about navigational canes, guide dogs, phone apps, and many other orientation and mobility resources for people with low vision.
The company’s drug is designed to reduce accumulation of toxins that cause vision loss.
Host Ben Shaberman talks with Steven McCoy, who overcame early challenges as a kid with Usher syndrome, to become the first black, deaf-blind journalist. Steven found his way forward through an impressive career with Disney and his own projects including his "Sessions with Steven" podcast and forthcoming memoir and television film about his remarkable personal journey.
August 5, 2022. Stem cell expert David Gamm, MD, PhD, talks with host Ben Shaberman about his innovative work in stem cell therapy development for retinal diseases, including his research at the University of Wisconsin-Madison and the company Opsis, which he co-founded. Their conversation covers a variety of topics including induced pluripotent stem cells, the benefits of scaffolds for cell orientation in the retina, and scale-up manufacturing.
July 22, 2022. Brian Strem, PhD, CEO of Kiora Pharmaceuticals, talks to Ben Shaberman about his company's emerging gene-agnostic small molecule for restoring vision for people with advanced retinitis pigmentosa and potentially other retinal diseases. Kiora recently received authorization in Australia to launch a trial for the treatment.
July 8, 2022. Jeff Stern, MD, PhD, and Sally Temple, PhD, co-founders of the Neural Stem Cell Institute, talk with host Ben Shaberman about their discovery of stem cells in the human retinal pigment epithelium and their newly launched clinical trial for the cells in people with dry age-related macular degeneration.
Achromatopsia, Choroideremia, Blue Cone Monochromacy, Cone-rod Dystrophy and X-linked Retinoschisis. Speakers: Christine Kay, MD and Robert Hufnagel, MD, PhD
June 24, 2022. Rachel Wong, PhD, professor and chair of the Department of Biological Structure at the University of Washington in Seattle, discusses the beauty and complexity of retinal circuitry and why the work in her lab is so important to the advancement of treatments and cures for people with inherited retinal diseases. Dr. Wong delivered the prestigious Friedenwald Award Lecture at the 2022 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO).
June 10, 2022. Frank Bruni, a popular NY Times columnist, book author, and Duke journalism professor, talks with host Ben Shaberman about his eclectic career, journey with vision loss, and latest book, "The Beauty of Dusk."
May 6, 2022. Host Ben Shaberman talks with Ash Jayagopal, chief scientific officer at Opus Genetics, a company launched by the Foundation Fighting Blindness to advance gene therapies for inherited retinal diseases into clinical trials. The company is targeting rare retinal conditions, including forms of Leber congenital amaurosis, that aren't being addressed by other companies.
Alkeus Pharmaceuticals reported that its emerging treatment for Stargardt disease slowed the growth of retinal atrophy – i.e., the area of damage to the retina – by approximately 30 percent per year for patients in its Phase 2 TEASE clinical trial at seven clinical research centers in the US.
April 22, 2022. John Flannery, PhD, a renowned gene therapy researcher from UC Berkeley and co-founder of Vedere Bio, talks with host, Ben Shaberman, about the promise of optogenetics, a gene-agnostic approach for restoring vision for people with advanced retinal diseases.
April 8, 2022. Host Ben Shaberman chats with Judge David Tatel, who, since 1994, has served on the US Court of Appeals — DC Circuit. He was appointed to the court by President Bill Clinton when Ruth Bader Ginsburg was appointed to the Supreme Court. During the podcast interview, David reflects on his impressive career, his adjustments to vision loss from XLRP, and his beloved guide dog, Vixen.
March 25, 2022. Jazz singer Katriona Taylor talks with host Ben Shaberman about her journey with Stargardt disease and her new album, Blind Passion, which includes covers of songs from iconic singers such as Stevie Wonder, Ray Charles, and Diane Schuur. Katriona also shares a performance of her original song "In the Name of Love."
March 11, 2022. Dr. Nancy Holekamp, a retinal specialist, clinical trial investigator, and key opinion leader in the retinal disease space, discusses new therapies and research for wet and dry age-related macular degeneration and some emerging treatment approaches for inherited retinal diseases.
This episode is sponsored by Apellis Pharmaceuticals.
February 25, 2022. Host Ben Shaberman talks with Nieraj Jain, MD, a retinal surgeon and expert in inherited retinal diseases from Emory on a wide range of clinical topics including retinal surgeries, vitamin A for IRD patients, cataract surgery for IRD patients, and his role in the Phase 3 XLRP gene therapy clinical trial being conducted by MeiraGTx/Janssen.
February 11, 2022. Ben Shaberman talks with Michael Stone, a 17-time Ironman triathlete, about the adventures and challenges of being a prolific athlete with vision loss and a really fast guide dog.
January 28, 2022. Eye on the Cure host, Ben Shaberman, talks with world-renowned researcher Tom Reh, PhD, who is developing an approach to enable the degenerating retina to regenerate its own new photoreceptors.
January 14, 2022. Podcast host, Ben Shaberman, has a fun chat with Ben Mehl, an actor with Stargardt disease who plays a completely blind librarian on the hit Netflix series "You."
The clinical trial is for RHO and NR2E3 mutations, but the retinal gene therapy has the potential to benefit people with a variety of other mutated genes.
December 10, 2021. Gordon Gund, co-founder and chairman emeritus of the Foundation, joins host Ben Shaberman to share reflections on 50 years of fighting blindness and what has made the Foundation successful in driving its mission to eradicate the entire spectrum of retinal degenerative diseases.
November 19. 2021. Michael Kalberer talks with Ben Shaberman about his perspective on living with cerebral palsy and Leber congenital amaurosis as well as his vision improvements after receiving an emerging CRISPR/Cas9 treatment in Editas' BRILLIANCE clinical trial. Michael can be reached at michaeljkalberer@gmail.com. His website is at michaeljkalbererpresents.com.
November 5, 2021. Ben Shaberman talks with Dave Knop, PhD, vice president of process development at AGTC, about how emerging gene therapies for inherited retinal diseases are manufactured for use in human studies.
The US Food & Drug Administration (FDA) has approved Susvimo, Genentech’s port delivery system (PDS) with ranibizumab, for the treatment of wet age-related macular degeneration (AMD). The PDS, a refillable capsule the size of a rice grain, provides continual release of ranibizumab, a protein that blocks the growth of vision-robbing, leaky blood vessels which are the hallmark of wet AMD. The PDS is implanted at the surface of the eye during a one-time, outpatient, surgical procedure. The PDS may be refilled as infrequently as twice a year. Susvimo will be available to patients in the coming months.
October 22, 2021. Michael Young, PhD, from Harvard Medical School, joins host Ben Shaberman to talk about the latest in stem cell research for retinal diseases.
October 8, 2021. Silvia Sorensen, PhD, an associate professor at the University of Rochester in the Center for Community Health and Prevention, talks with host Ben Shaberman about mental health issues and management for people with low vision.
September 24, 2021. Dr. Naveed Shams, chief scientific officer at ProQR, discusses RNA therapies and ProQR's three clinical trials targeting: Leber congenital amaurosis (a CEP290 mutation), USH2A exon 13, and retinitis pigmentosa (RHO-P23H).
September 10, 2021. Stephen Daiger, PhD, Director of the Laboratory for Molecular Diagnosis of Inherited Eye Diseases at UT Houston, discusses the challenges and advancements in identifying the genetic mutations that cause retinal degenerative diseases.
August 27, 2021. Host Ben Shaberman provides an overview of the Foundation's no-cost genetic testing program for people in the U.S. with inherited retinal diseases.
August 13, 2021. Host Ben Shaberman talks with Bonnie Swenor, MPH, PhD, Director of the Johns Hopkins Disability Health Research Center, about her journey from the sudden, unexpected onset of vision loss to becoming a researcher and advocate for people with all disabilities.
July 30, 2021. Ben Shaberman interviews Drs. Philip Troyk and Frank Lane of the Illinois Institute of Technology about their clinical trial for the Intracortical Vision Prosthesis, a pioneering system for restoring vision to people who are completely blind.
July 16, 2021. Ben Shaberman chats with Morten Bonde, senior art director at Lego Group in Copenhagen and author of the book "Sentenced to Blindness – Now What?"
July 2, 2021. Jill Dolgin, the executive director of patient advocacy at AGTC, talks to Ben about her role in helping patients understand clinical research. They also discuss AGTC's current gene therapy clinical trials for X-linked retinitis pigmentosa and achromatopsia.
News | AGTC Reports Encouraging Results from Phase 1/2 Gene Therapy Clinical Trial for Achromatopsia by Foundation Fighting Blindness
June 18, 2021. Host Ben Shaberman interviews Dr. Daniel Chung, chief medical officer at SparingVision, which is developing a gene-agnostic therapy to preserve cone vision in people with retinitis pigmentosa, Usher syndrome, and related conditions. A clinical trial launch for the emerging treatment, known as SPVN06, is planned for late 2021 or early 2022.
May 28, 2021. Sheila Nirenberg, PhD, a professor at Cornell and founder at Bionic Sight talks with Ben Shaberman about her company's innovative optogenetic therapy which provides hope for vision restoration for people with advanced vision loss from retinal diseases. The approach has shown encouraging results in an early stage clinical trial.
May 14, 2021. In the inaugural Eye on the Cure podcast, host Ben Shaberman explains why the retina is such a great target for therapy development and also provides some updates from the clinical trial frontlines.
Precise and Sensitive Outcome Measures are Essential for Getting Vision Treatments through Clinical Trials
Gene-editing approaches are often better suited for autosomal dominant retinal diseases than gene replacement therapies
Cellular treatment provided significant improvements in visual acuity for subpopulation of patients with better vision
The gene-agnostic approach shows promise for people with late-stage RP and related diseases.
Beacon Story | Carolyn Covington | Life After Blindness by Foundation Fighting Blindness