Foundation Fighting Blindness

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.

Despite missing the primary endpoint, the treatment improved vision for some patients.

The company is planning a Phase 2/3 clinical trial for therapy in late 2025.

Part B of the trial will enroll nine adult patients as well as three pediatric patients.

The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.

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.

Patients with worse vision at trial enrollment had greatest vision improvements.

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.

MeiraGTx hopes to apply for marketing approval in the UK for the gene therapy.

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.

Perceive Pharma is a spin out of Perceive Biotherapeutics.

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.

The Foundation is a founding investor in Atsena.

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.

The company is also planning to conduct the clinical trial at sites in the EU.

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.

Known as ultevursen, the USH2A RNA therapy was licensed from ProQR.

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. Learn more about the prescreening study for STELLAR at PRESCREEN.

When the Lemay-Pelletier family from Montreal learned that three of their four children were losing vision from retinitis pigmentosa, they made the bold decision to spend 15 months traveling the world to give their kids as many visual memories as possible. Podcast host Ben Shaberman talks to the family about their many incredible adventures and the National Geographic documentary (to stream on Disney+ in late 2024) that beautifully captured the journey.

The companies are planning a Phase 3 clinical trial for the promising gene therapy.

The Foundation funded Dr. David Gamm for development of the induced pluripotent stem cell approach to be used in the trial.

The 36-participant trial will take place at multiple sites in Australia.

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.

Merger will provide additional capital for gene therapy development.

#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

The drug is the first USH3 treatment to move into a human study.

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

Known as 4D-150, the wet AMD gene therapy performed well in a Phase 2b clinical trial.

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.

The USH1B trial is the first ever for a dual AAV gene therapy delivered to the retina.

The Phase 3 trial is expected to begin in Q1 2025.

Hannah Scanga, the supervisor of genetic counseling and the ocular genetics service at the University of Pennsylvania 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.

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.

Speakers: Todd Durham, PhD; Kristin Macdonald and Thomas Mendel, MD, PhD This 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, PhD This 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, PhD This session will discuss how dietary supplements and lifestyle choices can influence eye health (e.g. smoking, exercise).

Honoring John Corneille, JD, on his retirement from the Foundation Fighting Blindness and the Lulie's Light Awards.

VISIONS 2024 | Karen Petrou "Vision for the Future" by Foundation Fighting Blindness

Speaker: Abigail Fahim, MD, PhD This session will feature clinicans and researchers discussing recent updates for other retinal diseases, including choroideremia and X-linked retinoschisis.