Investing in Cures Summit: Driving Development of the Next Treatments for Retinal Degenerative Diseases
Foundation News
The Foundation Fighting Blindness and the Retinal Degeneration (RD) Fund hosted their annual Investing in Cures Summit (ICS) on March 8 and 9, 2024, to highlight the progress in development of treatments and cures for the entire spectrum of retinal degenerative diseases.
The Foundation Fighting Blindness and the Retinal Degeneration (RD) Fund hosted their annual Investing in Cures Summit (ICS) on March 8 and 9, 2024, to highlight the progress in development of treatments and cures for the entire spectrum of retinal degenerative diseases. Held in Scottsdale, Arizona, ICS convened 170 stakeholders — including researchers, companies, investors, donors, and families — who heard from more than three dozen presenters about the accelerating advancement of therapy-focused research to save and restore vision for the millions affected by inherited retinal diseases (IRDs) and age-related macular degeneration.
“Fifty-two years ago, when the Foundation began, it was impossible to imagine what’s happening today,” said Jason Menzo, the Foundation’s chief executive officer, in his opening remarks. “People diagnosed with an inherited retinal disease are no longer told there’s nothing that can be done, to go home and learn Braille. Instead, they’re getting connected to the Foundation, they’re getting genetic testing, entering the My Retina Tracker Registry, and learning about research efforts and clinical trials are relevant to them. They aren’t feeling isolated and alone — they are welcomed into our community. That’s why the Foundation Fighting Blindness exists.”
He added that today there are about 50 clinical trials underway as well as 90 research projects funded by the Foundation, which is developing its next strategic plan to fund $139 million in research over the next five years.
“If the Foundation didn’t exist, it would be just like it was in 1971, and for decades after, when newly diagnosed patients were told there was no hope,” said Jason. “But we are beyond hope. There is tangible evidence happening every single day to help patients with their disease.”
Next, Rusty Kelley, PhD, managing director of the RD Fund, the Foundation’s venture philanthropy arm, provided an overview and update of the Fund and its investments. He said that the RD Fund was established in 2018 with a dual mandate of providing both clinical and financial returns. “Recognizing that venture capital was essential to advancing therapies into and through clinical trials, the Foundation boldly decided to seed new company formation,” he said. “We subsequently built a diverse, interconnected, transformational, and sustainable eco-system.”
Since its inception, the RD Fund has syndicated with more than 45 top-tier investment firms making 14 investments and leveraging nearly 10 times the RD Fund’s invested capital. That’s more than $700 million invested into the RD Fund’s portfolio companies. The portfolio of assets spans a variety of gene-agnostic and gene specific technologies, including gene therapies, optogenetics, and gene-editing technologies. The venture philanthropy fund is also considering prosthetics and cell-based therapies to support end-stage disease.
Dr. Kelley recognized that recent biotechnology market conditions have been challenging to say the least. “Our intent alone is not protection from failures and setbacks. We are not immune to market calamity, pandemics, or when science tells the truth and the truth hurts, but on the bright side, it appears that a very cold biotech market is starting to wake up, and we have a mature portfolio that should benefit.” Dr. Kelley highlighted the scientific founders, company leadership, and several lead investors within the RD Fund who contributed significantly to building a global retina community.
Serving as the event emcee and tour guide, Chad Jackson, PhD, the Foundation’s senior director of preclinical translational research, kicked off the formal sessions, telling the inspiring story of how he decided to dedicate his education and career to science after watching his courageous and life-loving grandmother endure the adverse effects from ineffective cancer treatments. He encouraged ICS attendees to think about their “why” and what brought them to Scottsdale to learn about the latest advances in sight-saving therapies.
Taking on the Challenge of Whole Eye Transplant (opening keynote on evening of March 8)
Until recently, whole eye transplant for restoring vision was deemed by the ophthalmology research community to be overly ambitious and technically out of reach. However, Cal Roberts, MD, a program manager at the federally funded Advanced Research Projects Agency – Health (ARPA-H), is leading an initiative to take on that challenge, and in five years, perform the first successful (functional) human eye transplant. He spoke about the effort at the dinner preceding the ICS.
Dr. Roberts, a former cornea transplant surgeon and chief executive officer at the Lighthouse Guild, explained that the mission of ARPA-H is to fund high-reward breakthrough technologies that are risky, but if they work, would lead to a major change in healthcare. ARPA-H is modeled after the Defense Advanced Research Projects Agency (DARPA), which led the development of groundbreaking technologies like the Internet and global positioning systems (GPSs).
Dr. Roberts cited three big challenges for whole eye transplant: 1) harvesting donor eyes in a way that will keep the eyes viable in time for transplantation, 2) re-attaching nerves, including the optic nerve, which is comprised of 1.5 million fibers, and 3) re-attaching muscles, veins, and arteries while preventing rejection from the recipient’s immune system. Collaborative teams, including a team led by the Foundation, are now being formed and submitting proposals to address these challenges.
A Brighter Future: Transforming the Treatment Paradigm for Dry Age-Related Macular Degeneration and Geographic Atrophy
Session moderator James Handa, MD, chief of the Retina Division at Wilmer Eye Institute, Johns Hopkins University, underscored the need for more and better therapies for age-related macular degeneration (AMD), especially the dry form. He noted the global prevalence for AMD was about 196 million, double that for Alzheimer’s disease, and is expected to increase to 240 million by 2040. That said, Alzheimer’s disease research funding is triple the funding for AMD research.
The only approaches for reducing AMD risk are the Age-Related Eye Disease Study 2 (AREDS2) supplement, smoking cessation, and a healthy, low-glycemic diet (e.g., Mediterranean).
While more than 50 genetic variants have been identified that increase AMD risk, researchers need to better understand how they cause disease. Also, a better understanding of disease pathways at various stages of vision loss are needed.
Napoleon Ferrara, MD, the Distinguished Professor of Pathology and the Hildyard Endowed Chair in Eye Disease, University of San Diego, is renowned for his discovery of vascular endothelial growth factor (VEGF) and applying knowledge of the protein to develop multiple FDA-approved treatments for wet AMD and cancer. With the advent of several wet AMD therapies, the advanced form of dry AMD, geographic atrophy (GA), is now the leading cause of severe vision loss due to AMD. Dr. Ferrara co-founded the company NVasc, which is developing a therapy for dry AMD and geographic atrophy (GA) based on leukemia inhibitory factor (LIF) to maintain the healthy (non-leaking) vasculature of the retina.
Laura Carter, PhD, chief scientific officer, Character Biosciences, reviewed her company’s 5,000 patient study to track genetic drivers of AMD progression. The work is helping to identify patients at high risk for GA as well as targets for potential therapies. Character is developing a peptide, delivered by intravitreal injection, to reduce lipids, and thereby disperse drusen, to prevent GA. They hope to launch a clinical trial for the approach in late 2024.
Andrew Dick, MD, director of the University College London (UCL) Institute of Ophthalmology, discussed a treatment approach from Cirrus Therapeutics to maintain metabolism of mitochondria, the power suppliers of cells, to treat patients at high risk of developing GA. He noted that improving mitochondrial metabolism can reduce the increase in oxidative stress and inflammation that occurs as people age.
Hemant Khanna, PhD, vice president of preclinical ocular research at Iveric Bio (an Astellas company), discussed IZERVAY™, an FDA-approved C5 complement inhibitor that reduces the growth rate of lesions (areas of cell loss) associated with GA. He acknowledged that while this treatment is a significant advancement, better GA therapies are needed to address the condition more completely. Also, better AMD animal models, including those with an aging macula, will help researchers develop more effective therapies. In addition to his work to better understand AMD pathology, he continues developing “minigene” therapies for delivery of therapeutic IRD genes that are too big for existing viral vectors.
Alex McKeown, PhD, team leader of drug discovery and translational development at Apellis, reviewed his company’s FDA-approved C3 complement inhibitor, SYFOVRE®, for reducing the growth rate of lesions in people with GA. He said that the research community needs to better understand why GA keeps progressing after treatment, why there is tremendous disease variability among GA patients, and what factors beyond complement activation contribute to disease progression. He also would like to better understand which patients will best respond to specific treatments, suggesting that perhaps multiple treatments will be needed for some cases.
Fireside Chat: A View from the Top – How Large Firms Perceive Therapeutics in the Retina Space
Paul Biondi, an executive partner at Flagship Pioneering was interviewed by Kelly Lisbakken, a managing director in the Healthcare Investment Banking Group at Wedbush PacGrow and a board director for the Foundation’s RD Fund. Mr. Biondi, who works with growth company CEOs and their teams to achieve the best value their companies, said that therapy developers can benefit by pursuing innovation for rare diseases, because: 1) therapies can be more quickly delivered to rare disease patients, 2) the biology for rare diseases is in some cases better known, 3) the unmet medical need is clear, and 4) payers have less fear to reimburse for rare disease therapies. He noted that the pendulum is swinging back toward a more favorable investment environment for rare diseases after a challenging period. Mr. Biondi said that interesting science is coming out of the ophthalmology sector.
Unlocking the Power of Delivery: Advancing Gene Therapy with Innovative Platforms
Moderated by Ash Jayagopal, PhD, chief scientific and development officer at Ocuphire Pharma, the session reviewed various emerging IRD gene therapies and the innovative systems in development for gene delivery.
Gaurav Sahay, PhD, is professor of Pharmaceutical Sciences and co-director of the Center for Innovative Drug Delivery and Imaging, at the College of Pharmacy at Oregon State University. With co-founder Mark Pennesi, MD, PhD, he launched the company EnterX Biosciences to develop lipid nanoparticle delivery systems for IRD gene-editing therapies and mRNA treatments. His team has shown that their innovative system can deliver large therapeutic genes into photoreceptors of large animals. He said the lipid nanoparticle delivery approach is scalable and modular, and that genetic cargo can be injected multiple times without significant immune reactions that might otherwise occur with viral delivery. Also, the system can deliver bigger payloads than conventional adeno-associated virus (AAV) systems.
Caroline Man Xu, PhD, co-founder and chief executive officer at ViGeneron, reviewed her company’s pipeline of emerging gene therapies. ViGeneron’s VG901 is a gene therapy delivered by intravitreal injection for people with retinitis pigmentosa (RP) caused by mutations in CNGA1. It is currently in a Phase 1/2 clinical trial at University of Tubingen in Germany. VG801, a dual-vector ABCA4 gene therapy for people with Stargardt disease, is in IND-enabling studies to prepare for submission of an Investigational New Drug (IND) application to obtain authorization to advance the gene therapy into a clinical trial. The company is also developing a gene therapy for wet AMD. ViGeneron plans to use a virtual reality (VR) system as a clinical trial endpoint and is willing to make the VR technology available to other companies and therapy developers.
Shannon Boye, PhD, is a professor and chief of the Division of Cellular and Molecular Therapy in the Department of Pediatrics at the University of Florida and co-founder, director, and chief scientific officer at Atsena Therapeutics, a gene therapy company funded by the RD Fund. Atsena reported exciting results for its fully enrolled Phase 1/2 gene therapy clinical trial for people with Leber congenital amaurosis 1 (LCA1) which is caused by mutations in the GUCY2D gene. The therapy has performed safely and showed robust efficacy for patients receiving the high dose. Some patients are now reading food labels, seeing snowflakes, and walking outside at night. The company is working toward a Phase 3 clinical trial. Three patients have been dosed in the company’s Phase 1/2 gene therapy clinical trial for X-linked retinoschisis (XLRS). In the XLRS trial, an innovative spreading vector (AAV-SPR) is used to deliver more treatment across the retina, including the fovea (central retina responsible for perceiving the most detail), without injecting underneath the fragile foveal region. The company is also in IND-enabling studies for an Usher syndrome type 1B gene therapy which uses a dual, spreading vector. Dr. Boye noted that it is incumbent on IRD therapy developers to educate the FDA about new clinical trial endpoints and criteria that are most relevant to IRD patients.
Joseph Senn, PhD, head of research and development at Saliogen, discussed his company’s Saliogase enzyme, which seamlessly inserts new DNA of any size into precise genomic locations. The enzyme and therapeutic DNA are delivered using non-viral approaches including lipid nanoparticles. SalioGen is developing therapies for IRDs. Furthest along in development is a treatment for IRDs caused by ABCA4 mutations (e.g., Stargardt disease). The ABCA4 therapy has worked successfully in mouse models and is now being evaluated in a large animal model. The RD Fund has an investment in the company.
Leah Byrne, PhD, chief scientific officer and cofounder at Avista Therapeutics, and assistant professor in the department of ophthalmology at the University of Pittsburgh, reviewed her company’s innovative approach for identifying the optimal adeno-associated virus (AAV) based vectors to deliver therapeutic genes for specific retinal conditions. Avista’s scAAVengr platform provides head-to-head comparison of millions of viruses to see what works best at the level of single cells. The company has shown that its AAV vectors can perform 30 times more efficiently than existing, gold-standard AAV vectors. The Foundation has provided funding for development of this platform.
Genetic Medicines: Expanding the Addressable Patient Populations through Advances in Gene Editing
Moderated by Rachel Huckfeldt MD, PhD, assistant professor of ophthalmology at Harvard and clinician scientist at Mass Eye and Ear, the session covered alternative techniques and approaches to traditional gene therapy. Dr. Huckfeldt noted the need for alternatives because only 50 percent of the most prevalent genes are addressable with existing gene therapy technologies.
Ben Yerxa, PhD, chief executive officer at Opus Genetics, discussed how his company is taking on development of IRD gene therapies that other companies aren’t noticing. A company launched by the Foundation and seed invested by the RD Fund, Opus has moved a gene therapy for LCA5 into a Phase 1/2 clinical trial at the University of Pennsylvania. LCA5 causes severe vision loss, affecting about 500 people in the US and EU. The first three patients in the trial treated at the low dose had improvements in full-field sensitivity (FST) which measures a patient’s overall retinal sensitivity. The three patients also showed vision improvements based on a virtual reality test. Opus’ program for Best disease is ready to go into a clinical trial, pending additional investment. Its RDH12 program is ready for toxicology studies pending additional investment. The company needs to conduct one more animal study to move its RHO gene therapy into a human study. (Mutations in RHO are a leading cause of autosomal dominant RP.) Also, it had a pre-IND meeting with the FDA regarding clinical development of its NMNAT1 (LCA9) gene therapy.
Bence Gyõrgy, MD, PhD, is a team leader for the Ophthalmic Translational Research Group at the Institute of Molecular and Clinical Ophthalmology Basel in Switzerland. His team is developing a base-editing treatment targeting the G1961E mutation in ABCA4, which is a common cause of Stargardt disease. His approach uses an AAV to deliver the base editor. His team has tested it on retinal explants and in organoids, mice, and large animals. Thus far, the treatment has been highly efficient in editing both photoreceptors and retinal pigment epithelial (RPE) cells. RPE cells provide critical support for photoreceptors.
Jeremy Duffield, MD, PhD, chief scientific officer, Prime Medicine, discussed his company’s emerging prime editing therapies which can correct mutations in as many as 90 percent of genetic conditions. The approach can edit genetic regions of varying sizes by nicking DNA rather than causing more invasive, double-strand breaks. Prime’s emerging RHO treatment is delivered by an AAV and can correct mutations for about 60 percent of RHO patients (using multiple editors). The company is also targeting specific regions of the USH2A gene, which, when mutated, causes Usher syndrome type 2A or non-syndromic RP.
Rob Collin, PhD, is a professor at Radboud University in the Netherlands and chief scientific officer at Astherna. His lab and company are developing therapies called antisense oligonucleotides (ASOs) which work like band aids to cover up faulty regions in RNA. Dr. Collin explained that DNA (genes) are like recipes and RNA are like copies of recipes. Our cells read RNA to make critical proteins much like a chef reads the copy of a recipe from a book to make spaghetti. Changes to RNA aren’t permanent with ASO treatment (i.e., they need to be re-administered) but are delivered by an intravitreal injection which can be administered in a doctor’s office. The company’s treatments for specific ABCA4 mutations worked well in a retinal organoid model. Astherna is also targeting some USH2A mutations. Dr. Collin’s ASO research for Stargardt disease is currently funded by the Foundation. Earlier Foundation-funded research from his lab led to the development of an USH2A ASO therapy from ProQR, a previous RD Fund portfolio company that licensed its ophthalmology assets to Théa.
Robert Bell, PhD, senior vice president and head of research at Ascidian Therapeutics, presented its most advanced RNA exon-editing therapy which can address mutations in approximately 70 percent of people with IRDs, including Stargardt disease, caused by ABCA4 mutations. Exons are the genetic regions that cells use to encode proteins. Most mutations occur in exons. The Ascidian treatment is delivered by an AAV. The company has authorization from the FDA to launch a Phase 1/2 clinical trial. Enrollment for the trial is expected to begin later in 2024. Ascidian has a second ABCA4 program for addressing the remaining 30 percent of mutations not addressed by the first.
Basem Al-Shayeb, PhD, co-founder and chief technology officer at Amber Bio, received his doctorate at UC Berkeley under the mentorship of Jennifer Doudna, PhD, a CRISPR co-inventor and Nobel Laureate. Dr. Al-Shayeb discussed his company’s innovative RNA-editing platform. The approach can edit large regions of RNA thereby by addressing many/most of the mutations for a given disease-causing gene. The emerging treatment is delivered using an AAV.
Cell Therapies: The Future of Vision Restoration
Kapil Bharti, PhD, scientific director and senior investigator at the National Eye Institute (NEI), moderated the session and also reviewed his emerging RPE cell therapy for people with GA. The RPE cells are developed from induced pluripotent stem cells (iPSCs), which are derived from the patients’ own blood, and placed on a biodegradable scaffold to optimize their orientation and survival when transplanted. The treatment, which covers about half the macula, is in a Phase 1/2 clinical trial at the NEI. Dr. Bharti noted that the NEI also has a clinical trial underway for metformin for people with Stargardt disease and is developing a metformin eye formulation for intermediate AMD. Metformin is a drug approved by the FDA for diabetes. In a mouse study, metformin reduced the accumulation of lipofuscin, the damaging deposits that accumulate in the retinas of people with Stargardt disease.
Mandeep Singh, MD, PhD, holds the Andreas C. Dracopolous Professorship of Ophthalmology at Wilmer Eye Institute, Johns Hopkins University. He discussed an emerging cell therapy being developed by Hopkins-spinout Agnos, in which donor cells merge with the recipients’ cells to replenish missing or defective proteins. The gene agnostic approach is for people with recessive (and potentially dominant) retinal diseases. In a lab study of the approach, mice regained 20 to 30 percent of their vision. A device for subretinal delivery is being designed for the pediatric population. The Foundation has previously funded Dr. Singh’s stem cell research.
Valerie Chichagova, PhD, is the associate director of iPSC technology and head of retina at NewCells Biotech, a company developing cell-based models and tools, including retinal organoids, to support the drug discovery efforts of companies and researchers in the biotech community. NewCells enables their clients to de-risk and improve drug discovery. NewCells projects have included development of retinal organoid disease models for gene therapy lead optimization.
Jogin Desai, PhD, is founder and chief executive officer at EyeStem, which is planning to launch a clinical trial in India for an RPE cell therapy for people with GA. The company also has a program in lab studies for photoreceptor replacement. EyeStem is focused on frugal innovation to make therapies more accessible to all people, regardless of their economic status. EyeStem plans to employ scalable manufacturing to reduce production costs.
Philanthropy and Beyond: Funding Innovation in Rare Disease Therapeutics
Brandon Nixon, co-founder and chief executive officer at the Nixon Vision Foundation, talked with Jason Menzo about the challenging vision loss journey for his wife, Janine, who was ultimately diagnosed with a retinal disease caused by mutations in the PRPH2 gene.
Initially told by a doctor to not worry about spots on her retinas, Janine eventually lost vision and sought answers for her progressive condition, which had also affected her grandmother, though was never accurately diagnosed. The Nixons subsequently made a commitment to advance PRPH2 science, bring affected people together, and provide emotional support to families and patients.
In seeking answers for Janine’s condition, the family saw that the Foundation Fighting Blindness had supported much of the research into IRDs and was a global hub of resources and knowledge for people with retinal conditions. They launched a $10 million PRPH2 research initiative with the Foundation and the University of California, San Diego, and hosted a two-day workshop in March 2023 to bring researchers, clinicians, and families together to kick-off a new, global PRPH2 initiative.
The Foundation will soon be announcing two new grants, supported by this new initiative in partnership with the Nixon Vision Foundation, to address gaps in PRPH2 knowledge. PRPH2 is one of the most common genes associated with inherited retinal diseases. Mutations in PRPH2 can cause RP and macular degeneration.
Gene Agnostics: Broad Spectrum Therapeutic Strategies to Address Retinal Disease
Moderated by Dan Chung, DO, chief medical officer at SparingVision, the session featured emerging therapies designed to work independent of patients’ mutated genes. Gene-agnostic approaches can potentially be of benefit to people whose mutated, disease-causing gene hasn’t been identified or those with genes that aren’t being targeted by emerging genetic therapies.
Dr. Chung said SparingVision’s lead gene-agnostic treatment candidate, SPVN06, expresses protein called rod-derived cone viability factor to stabilize the function of cones and mitigate oxidative stress. The company is currently dosing RP patients in its third dose-escalation cohort for its Phase 1/2 clinical trial, PRODYGY. The company is also planning to launch a Phase 1/2 clinical trial for its therapy designed to bestow light sensitivity to dormant cone cells, an approach for people with more advanced RP and related conditions. The RD Fund is a founding investor in SparingVision.
Shankar Musunuri, PhD, chief executive officer and co-founder at Ocugen, discussed his company’s development of gene-modifier therapies to address retinal degeneration. Gene modifiers work like master regulators to modify expression of multiple retinal genes, bringing homeostasis and a healthy environment to enhance cell survival. Ocugen is currently planning a Phase 3 clinical trial for OCU-400 for people with RP. It plans to enroll 150 people total in an RHO group and a gene-agnostic group. The company is also dosing patients for its OCU-410 Phase 1/2 clinical trials for people with Stargardt disease and dry AMD.
Halden Conner, chief executive officer and president at Nacuity, said that many researchers believe that N-acetylcysteine amide (NACA), his company’s emerging anti-oxidative treatment for RP and Usher syndrome, “is the most effective antioxidant on the planet.” He noted that oxidative stress increases dramatically after rods are lost, resulting in the destruction of cones. (Rods are usually lost first in conditions like RP and Usher syndrome). NACA entered a Phase 2 clinical trial in Australia about three and a half years ago for 49 people with Usher syndrome. The interim results for the trial, which will conclude in about one year, look promising. The company is strategizing for patient recruitment for a US trial.
Paul Bresge is co-founder and chief executive officer at Ray Therapeutics, a company developing an optogenetic therapy for restoring vision in people with advanced forms of RP, AMD, and other retinal conditions. Mr. Bresge said that Ray has engineered the most light-sensitive protein in nature. Therefore, the treatment should work in ambient light; patients will not need to wear glasses or goggles to boost the light signal coming into the eye. The protein was engineered to express in bipolar cells or ganglion cells. A clinical trial is planned.
Investing in Retinal Therapeutics
Owen Smith, partner, 4BIO Capital, and session moderator, said that 2023, from a patient and scientific perspective, was a good year in biotech. There were 75 new therapies approved, including two new approvals in ophthalmology, Iveric Bio’s IZERVAY and Apellis’ SYFOVRE, both for GA. Also, 23 biotechs were acquired for more than $1B, a record. Most activity was in oncology, cardiology, and neurology. However, 2023 was a difficult year in biotech investing, with $17 billion invested over 350 investment rounds. He added the year was challenging for those seeking investments in cell, gene, and RNA therapies. 4BIO reviews about 1,000 opportunities a year and has been working with both SparingVision and Ray Therapeutics, so they bring helpful insights to their discussions with emerging biotechs seeking investments.
William Link, PhD, is founder and managing partner at Flying L Partners, which only invests in the ophthalmology market. The company has been involved with 30 ophthalmology companies, nine of which have focused on back of the eye. He said that the last few years have been the most challenging times in the last several decades. The normal business cycle was impacted significantly by COVID. Overall, ophthalmology has delivered nice returns. Investments in back-of-the-eye therapies are attractive, though the market is complex and crowded. A lesson learned: Getting into human studies early has helped immensely for many companies. He’s observed therapy developers who spent too much time in the lab, only to find new, tough issues once they enter human studies.
Julia Kaufman, PhD, is director, Boehringer Ingelheim Venture Fund USA, which invests in early regenerative, transformative companies. She said that investing sentiment has improved, though it has been difficult to raise money over the past few years. Her team is looking for strong intellectual property, product and company differentiation, biology, and delivery mechanisms. They avoid stacked risk, a scenario in which a company is developing many, new high-risk technologies for a given therapy or portfolio.
Kazufumi Nakamura, is the investment director at Mitsui Global Investment, Inc. (MGI), the venture capital arm of Mitsui Group, focusing on early-stage biotech therapeutics. They are evaluating unique diverse technologies, including those for dry AMD, glaucoma, Stargardt disease, and other IRDs.
Rusty Kelley, PhD, managing director of the RD Fund, said that he sees opportunity in 2024; the investing climate appears to be improving, though he’s observed more interest from strategic partners than venture capital firms, which haven’t fully moved back into rare disease and retina. Interest in mergers and acquisitions has picked up after a period when the public markets in biotech were shut down.
Charting the Course Ahead: Summit Rundown and What’s Next
To put a wrap on ICS, Alicia Kemble, PhD, a venture analyst at the RD Fund, introduced George Peinado, the founder of GAP Investments, father of three boys, and husband of 27 years. He told his family’s compelling journey that led them to the Foundation Fighting Blindness.
George said that his middle son, Luke, was born hearing impaired, though, ironically, was the best listener of his three boys. With hearing aids, Luke hears well and is a strong student and hockey player. However, he began having vision issues in high school. Glasses and contacts never fully corrected his vision. Eventually, during his senior year, an ophthalmologist looked at the back of his eyes and saw thinning of his retinas. That’s when George and his wife began researching his symptoms and suspected Usher syndrome was the cause.
When Luke was officially diagnosed by a retinal specialist with the progressive condition, he was devastated. “So, we left the clinic, hugged it out, and that was the last time I ever saw Luke cry,” said George. “He’s a resilient guy and the best person I know.”
Shortly thereafter, the family got Luke an appointment with Dr. Rachel Huckfeldt at Mass Eye and Ear. “She was awesome — very smart and passionate. She gave us the first words of encouragement and hope. She said that while there was no cure, the science was moving very fast and the progression of vision loss was very slow,” said George. Subsequent genetic testing confirmed that mutations in USH2A were causing Luke’s hearing and vision loss.
Luke moved on in his life while George and his wife “consumed every word we could about RP and Usher syndrome. We felt we had to do something.” They subsequently found the Foundation, contacted Foundation Board Chairman David Brint, who invited them to the 2022 ICS at Half Moon Bay in California.
“As a career private equity guy, I was immediately drawn to the RD Fund. The more I dug in, the more I was encouraged. My wife and I made a four-year commitment to the RD Fund and are big supporters of the mission, the people, and their processes for investing capital to cure blindness,” said George. “I believe it comes down to shots on goal and eventually you’re going to score.”
