Summary of the 11th Annual Retinal Therapeutics Innovation Summit

The Retinal Therapy Innovation Summit (RTIS), hosted by the Foundation Fighting Blindness and the Casey Eye Institute at Oregon Health & Science University, provides researchers and companies with the latest news on emerging therapies for inherited retinal diseases and dry age-related macular degeneration.

The 11^th annual RTIS was held on May 1, 2026, preceding the annual meeting of the Association of Research in Vision and Ophthalmology (ARVO), in Denver. RTIS featured 25 presentations from retina experts from around the world, and more than 400 professionals from the retinal ophthalmology field attended. While ARVO is the world's largest eye research conference, the Innovation Summit focuses on clinical development and advances for retinal disease therapies.

The meeting was co-organized by Paul Yang, MD, PhD, and Renee Ryals, PhD from the Casey Eye Institute and Amy Laster, PhD, Chad Jackson, PhD, Michelle DiVincenzo, and Chris Adams from the Foundation Fighting Blindness.

Summit Sponsors

• Premiere Sponsor: Casey Eye Institute, Oregon Health and Science University
• Innovator: AAvantgarde Bio, Alkeus Pharmaceuticals, Ascidian Therapeutics, Atsena Therapeutics, Beacon Therapeutics, BlueRock Therapeutics, RestoreVision, Théa / Sepul Bio
• Luminary: Belite Bio
• Discovery: Astellas, Genentech, InFocus Clinical Research, Lexitas, MeiraGTx, Merck, Opus Genetics, Regeneron, Sumitomo Pharma America

KEYNOTE: Gene Therapy in Children with AIPL1-Associated Severe Retinal Dystrophy

Robin Ali, PhD, King’s College London, presented results from a gene therapy program to treat young patients with AIPL1-associated Leber Congenital Amaurosis – a rare early-onset rod/cone dystrophy that causes severe sight impairment and no light perception by age six.

Because the condition is rare and there is a short window for intervention, Dr. Ali pursued compassionate treatment. He obtained a UK Specials License for University of College London Wolfson Gene Therapy Facility, then raised funding for manufacturing and treatment through philanthropy.

Because all retinal structure degenerates past four years old, Dr. Ali’s team identified four patients below age four who still had some retinal structure and treated the better seeing eye with an AAV2/8-CMV-AIPL1 gene supplementation. Patients improved in visual acuity, which was measured by age-appropriate tests including the Sheridan-Gardiner vision test, using flashcards with single letters held at a distance, and a rotating Garber grating test to detect striped patterns. Treatment in very young children appears safe, with no clinically significant intraocular inflammation. Macular edema in one child partially resolved and did not prevent improvement in visual function.

The study shows that patients treated early enough can preserve outer retinal structure and restore function. Dr. Ali notes that while it is difficult to measure outcomes in young children, age-appropriate methods make it possible.

Dr. Ali also underscored the importance of clinical academic centers with manufacturing capabilities in developing treatments for ultra rare diseases. He said this work would not have been possible without a GMP manufacturing facility that could produce the treatment at much lower costs for investigational and compassionate use.

SESSION 1: ULTRA RARE: PROVIDER PERSPECTIVES

Moderator: Chad Jackson, PhD, Foundation Fighting Blindness

Leveraging the “N of 1” Trial Pathway to Treat Ultra-Rare Variants in Inherited Retinal Disease

Shyamanga Borooah, PhD, Shiley Eye Institute, University of California San Diego, presented on peripherin-2 (PRPH2)-associated retinal degeneration (PARD), one of the most common causes of inherited retinal dystrophy.

The U.S. Food and Drug Administration’s “N of 1” framework is a recent pathway for investigators to develop individualized antisense oligonucleotides (ASOs) for ultra-rare indications, allowing treatment of patients with a research investigational new drug. ASOs are single strands of RNA that can treat inherited retinal diseases by restoring essential protein production and cell function.

Dr. Borooah’s work was funded by the N-Lorem Foundation within an academic non-profit model. UCSD completed the natural history study. The study participant – a 64-year-old-female with macular degeneration – was treated with an ASO targeting a specific PRPH2 variant and has seen no serious adverse events, though it is too early to make safety and efficacy conclusions.

Dr. Borooah suggested that careful selection of the patient and non-profit partner is important and that navigating academic institutional review board and contractual agreements with those unaware of “N of 1” style studies can add complexity to the approval process.

Antisense Oligonucleotides for the Treatment of Rare Eye Diseases Such as LCA10 and Usher Syndrome Type 2A

Bart Leroy, MD, PhD, Ghent University, also discussed the use of antisense oligonucleotides (ASOs) to treat inherited retinal diseases, focusing on the ASO RNA therapies sepofarsen and ultevursen.

Sepofarsen targets CEP290-associated LCA10 caused by the common c.2991+1655A>G variant. In the Phase 2/3 ILLUMINATE study, 8/15 sepofarsen-treated eyes improved in visual function. HYPERION, the Phase 3 clinical trial, is a placebo-controlled paired-eye design that is actively recruiting globally with 25% enrolled to date. Sepofarsen is available through compassionate use to previous ProQR trial participants free of charge in seven countries.

Ultevursen targets USH2A-associated retinitis pigmentosa due to mutations in exon 13. During Phase 1/2 trials, 29 participants dosed with ultevursen had no serious adverse events or adverse events leading to discontinuing treatment. ​The Phase 2b sham-controlled study LUNA is screening last potential participants and plans to close enrollment in the next few months.

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies (Baseline Results)

José-Alain Sahel, MD, University of Pittsburgh, offered updates on the Universal Rare Gene Study (Uni-Rare). Uni-Rare includes a registry open to 1,500 people with mutations in over 380 rare IRD genes, as well as a natural history component for up to 100 patients per gene for select genes. Dr. Sahel likened the study’s infrastructure to a stadium—if it is built once, it can be reused for many subsequent studies.

As of March 2026, 33 global sites and 623 participants representing 103 genes are included in the study. Enrollment is anticipated to complete December 2026.

Since many genes may only have one or a few patients enrolled, study researchers proposed grouping genes together based on factors including biological mechanisms, age, and protein function. 64% of genes have been grouped into one of twenty categories. Geneticists will continue refining the best way to group genes before running analyses.

Uni-Rare is funded in part by the Foundation.

SESSION 2: ULTRA RARE: Systemic Hurdles for “N of 1” Trials

Moderator: Paul Yang, MD, PhD, Casey Eye Institute, Oregon Health and Science University

Discussion of FDA Evolving Regulatory Paths for Retinal Diseases

William M. Boyd, MD, U.S. Food & Drug Administration, discussed what FDA looks for when approving new drugs and devices. New drugs must provide benefits that outweigh risks when used by the intended population, shown in adequate and well-controlled trials. FDA does not accept random experience, isolated cases, or reports lacking details for new drug or new device applications.  

Dr. Boyd highlighted the FDA Rare Disease Innovation Hub, which serves as a collaboration between Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research to improve outcomes for patients. It focuses on products intended for smaller populations or for diseases where the disease progression is variable and not fully understood.

He also said that FDA is accepting requests to participate in the Commissioner’s National Priority Voucher Pilot Program, a pathway to reduce review times for drug and biological product applications and manufacturing or efficacy supplements.

Personalized Intervention for Inherited Retinal Diseases: The Example of Stargardt Disease

Rob Collin, PhD, Radboud University Medical Centre, discussed using antisense oligonucleotides to treat Stargardt disease through a non-profit collaboration with the Dutch Center for RNA Therapeutics. Dr. Collin and team designed an “N of 1” treatment in the Netherlands ​for a 42-year-old with bi-allelic mutations in ABCA4. Pre-clinical safety trials in rabbits showed mild inflammation in the vitreous and slightly reduced ERG, comparable to ultevursen, which is currently approved for use in a clinical trial. To understand the patient’s disease progression, the team followed the patient for more than four years to find that visual acuity varied, declining in a non-linear manner, while there was a linear increase in atrophy.

​Dr. Collin’s team propose treating the patient with a 160 ug intravitreal dose, followed by 80 ug every six months. They plan to treat one eye, using the other as a control, and will measure progression of atrophy as the main endpoint. ChemGenes / N=1 Collaborative support the GMP manufacturing of the treatment.

SESSION 3: Small Molecules to the Rescue

Moderator: Alicia Kemble, PhD, Foundation Fighting Blindness

Successful Protection of Cones from Degeneration & Dysfunction with Oral Dosing of BRX011

Maureen A. McCall, PhD, University of Louisville, used drug BRX011 as a neuroprotective agent that works independent of mutation to treat autosomal dominant retinitis pigmentosa. Dr. McCall’s work is a partnership with BioJiva and funded by the Foundation’s Brint Family Translational Research Award.

Dr. McCall and her team used transgenic pigs expressing the human P23H rhodopsin variant to evaluate if replacing docosahexanoic acid (DHA) with a modified form has therapeutic benefit. DHA is found in high concentrations in the photoreceptor outer segment.

Pigs were treated orally with deuterated DHA (BRX011) mid-disease or pre-disease. More stable, deuterated bonds may resist the negative effects of IRD-induced oxidative stress. There was no change in wild type cone and rod morphology or function after treatment, establishing safety and leading to an Investigational New Drug acceptance. D-DHA maintains cone structure and function in disease, and surprisingly, was also able to rescue rod function.

Nacuity’s Positive Phase 2 Results for Its Gene Agnostic Therapy for Retinitis Pigmentosa Using NPI-001

Halden Conner, Nacuity Pharmaceuticals, discussed safety and efficacy data from a Phase 1/2 randomized clinical trial for NACA (NPI-001) – an oral treatment for retinitis pigmentosa associated with Usher syndrome. NACA is a small molecule antioxidant designed to address oxidative stress. The study included 49 patients with Usher syndrome with some rod loss but mostly viable cones. Patients were randomized to control or treatment of 500 mg/day.

NACA showed excellent safety and tolerability, with mostly mild adverse events. It significantly slowed photoreceptor loss by 50% over two years, with significant effect between six months and two years.

Nacuity plans a confirmatory two-year study with 80 patients in sites in Australia and the U.S. in 2026. The Foundation’s RD Fund is an original investor in Nacuity.

Topline Results from a 2-Year Phase 3 Study of Oral Tinlarebant in Adolescent Patients with Stargardt Disease

Nathan L. Mata, PhD, Belite Bio, Inc, presented results from the two-year Phase 3 DRAGON clinical trial of tinlarebant in adolescents with Stargardt disease. Tinlarebant is a once-a-day oral tablet that binds to RBP4, reducing retinol delivery to the eye to slow the accumulation of toxic vitamin A byproducts called bisretinoids.

Patients included were 12-20 years old with a clinical diagnosis of Stargardt disease and at least one mutation in the ABCA4 gene.

The trial found 5 mg/day tinlarebant was well-tolerated, significantly slowed atrophic lesion growth by 36% compared to placebo, and reduced RBP4 levels by 80% on average.

In 2026, tinlarebant became the first Stargardt treatment submitted to FDA for new drug approval. Belite Bio used data from the Foundation’s largest ever natural history study of patients with Stargardt disease (ProgSTAR) to inform their clinical trial design.

SESSION 4: Retinal Stem Cell Therapies: Clinical

Moderator: Katie Cording, PhD, Foundation Fighting Blindness

Retinal Pigment Epithelium Cell Therapy in Geographic Atrophy Secondary to Age-related Macular Degeneration: Three-Year Results from the OpRegen Phase 1/2a Study

Eyal Banin, MD, PhD, Hadassah-Hebrew University Medical Center, covered three-year results of OpRegen cell therapy in patients with geographic atrophy (GA). The treatment involves transplanting retinal pigment epithelium cells derived from human embryonic stem cells into the subretinal space.  

Patients with less advanced GA had structural benefit and greater gains in visual acuity that lasted at least three years after a single injection. ​Structural benefits included restoration of outer retinal structure, re-appearance of an RPE layer, and features associated with recovery of photoreceptors. ​

Treatment effects were more prominent in patients where the area of atrophy was covered more extensively with the OpRegen bleb compared to those with limited coverage. ​

The Phase 2a GAlette study evaluating the success of subretinal delivery of OpRegen cell therapy for GA is currently enrolling.

Results of Phase 1/2a Trial of Eyecyte-RPE for the Treatment of Geographic Atrophy Secondary to Dry Age-related Macular Degeneration

Jogin Desai, MBBS, Eyestem Research, presented clinical trial results using retinal pigment epithelium derived from induced pluripotent stem cells to treat patients with geographic atrophy. All nine subjects in the first two cohorts gained on average 13 letters in six months. Nearly all – eight out of nine – subjects in first three cohorts saw improvement in vision without any serious adverse effects.

Lower doses appear to have better efficacy than higher doses (100k-200k vs 300k cells). Patients saw predictable vision improvement at four to eight weeks.

Eyestem plans to explore these initial signs of efficacy and safety in Phase 2 trials in India and a pivotal trial in the U.S. They are currently investigating the durability of the treatment effect, if repeat dosing would be possible, and how the immune system reacts to treatment of the other eye, among other questions.

Adult RPE Stem Cell Therapy for Geographic Atrophy

Jeffrey Stern, MD, PhD, Neural Stem Cell Institute, used stem cells derived from the retinal pigment epithelium to treat geographic atrophy. Cells originate from donor eyes and differentiate into RPE. Dr. Stern and team found that four-week-old RPE cells fully rescued vision in the retina in a rat model of retinal degeneration.

They formed the company Luxa to evaluate the potential treatment in a Phase 1/2a clinical trial for safety and efficacy. One-time treatment of poorer-seeing patients improved visual acuity by about 20 letters on average over the first year. The improvement remained even when immune suppressants were removed. Those with better vision treated with a lower dose saw vision stabilization.

The Phase 1/2 study is ongoing and Luxa is actively enrolling and planning for Phase 2b for larger numbers of patients.

The Llura Liggett Gund Award – Dr. Eric Pierce, Gene Editing for Inherited Retinal Degenerations

Eric Pierce, M.D., Ph.D., Harvard Medical School and Ocular Genomics Institute, was named the 2026 recipient of the Llura Liggett Gund Award, the highest award from the Foundation Fighting Blindness. The award recognizes Dr. Pierce’s contributions to inherited retinal disease research, including work that led to the first FDA-approved gene therapy for an inherited disease and leadership of the world’s first CRISPR genome-editing study in humans.

After accepting the award, Dr. Pierce provided an overview of gene editing for inherited retinal degenerations.

EDIT-101 was the first instance of in vivo human gene editing, targeting a mutation in the CEP290 gene, and demonstrating that gene editing can be done in the eye safely and effectively.

Dr. Pierce said that genetic therapies show great promise for the treatment of inherited diseases​. He also addressed future directions, suggesting the priorities below to increase success rate:

• Improving our understanding of disease genetics/biology
• Taking full advantage of novel technologies, including novel delivery technologies
• Identifying and developing additional approvable endpoints, including better patient-reported outcome measures
• Building natural history data
• Identifying surrogate biomarkers
• Updating trial design
• Having realistic expectations

SESSION 5: Retinal Gene & RNA Editing: Preclinical

Moderator: Renee Ryals, PhD, Casey Eye Institute, Oregon Health and Science University

Gene-Agnostic Read Through of Translation Termination Using Engineered tRNA

Bikash Pattnaik, PhD, University of Wisconsin-Madison, discussed a new gene-agnostic therapeutic concept.​ Instead of targeting a specific gene, Pattnaik’s team developed anticodon-engineered transfer RNA (ACE-tRNA) to target a class of mutations—nonsense mutations—that cause many diseases. This could lead to a treatment beyond one gene.

Dr. Pattnaik’s potential treatment targets mutations that disrupt channel function due to mutations in KCNJ13. Dr. Pattnaik delivered ACE-tRNA via an adenovirus to human-induced pluripotent stem cells to restore the protein and rescue channel function. Subretinal delivery of the ACE-tRNA in a mouse model rescued visual function.

The engineered tRNA creates a full-length protein​ that restores ion conductance​ and tissue function. The tRNA provides codon-specific but gene-agnostic targeting​ and does not interfere with transcription or translation​.

Prime Editing Rescues Aberrant Splicing Defect Caused by Pathogenic Variants in PRPH2

Peter M.J. Quinn, PhD, University of Pennsylvania, discussed prime editing to correct DNA mutations in the peripherin-2 (PRPH2) gene. Mutations in this gene, whose protein product is located in the outer segment of rod and cone photoreceptor cells, can cause multiple inherited retinal diseases, for which there are currently no treatments.

Prime editing uses CRISPR-Cas9 to introduce a single strand break in DNA to correct a single erroneous base that leads to an inherited retinal disease. Dr. Quinn used prime editing to correct the c.828+1G>A variant in PRPH2 in human induced pluripotent stem cells (hiPSCs). The resulting hiPSC cells did not have any detectable off-target mutations or karyotype abnormalities. Correcting this mutation restored the original PRPH2 transcript and reduced levels of the mutant transcript.

The work highlights prime editing as a potential precise, safe method to correct mutations in PRPH2-related inherited retinal diseases.

This work was supported by the Foundation.

SESSION 6: Retinal Gene Augmentation: Preclinical

Moderator: Preeti Subramanian, PhD, Foundation Fighting Blindness

Anc80 Gene Therapy Platform to Treat Vision Loss Caused by RPGRIP1 Mutations

Ashley Winslow, PhD, Odylia Therapeutics, introduced OT-004, a gene replacement therapy for patients with RPGRIP1-associated retinal dystrophy, including Leber congenital amaurosis 6, cone-rod dystrophy 13, early-onset retinitis pigmentosa, and achromatopsia. Odylia uses the Anc80 viral vector to deliver the human RPGRIP1 gene into photoreceptors to preserve or restore vision in patients, through a single subretinal injection.

OT-004 improves vision by preserving or restoring photoreceptor function in the subretinal area. The therapy is expected to be less than a year to clinical trials, though in need of funding or a partner at this time. OT-004 has received orphan drug designation and rare pediatric disease designation.

The work has received funding from the Foundation.

A Tripartite AAV System with Engineered Lox Sites Enables Efficient Delivery of the EYS Gene for Retinal Gene Therapy

Seongjin Seo, PhD, University of Iowa College of Medicine, discussed Uni-STAR – a new approach to deliver large genes using up to four AAV vectors. While AAVs are currently the most broadly used vehicle to deliver genes for gene therapy, they are limited in the size of the gene they can carry, up to about 4.2 kb​. The eyes shut homolog (EYS) gene is a major cause of autosomal recessive retinitis pigmentosa, and, at about 9.5 kb, is challenging to deliver.

Dr. Seo uses a tripartite AAV-EYS system to deliver and express the full-length EYS gene in mouse retinas. His work demonstrates that Uni-STAR is a scalable platform for delivering large genes beyond AAV capacity.  

Because mice lack a functional copy of EYS, future research is needed to explore larger animal models that express EYS. Future directions also include applying the Uni-STAR platform to other large genes ​such as CEP290​, CDH23 (USH1D), PCDH15 (USH1F), MYO7A (USH1B), and USH2A.

Fibrin Hydrogel Platforms for Retinal Gene and Cell Therapy

Brittni A. Scruggs, MD, PhD, Mayo Clinic, uses fibrin hydrogels as a new way to deliver gene therapy. Luxturna – the first gene therapy for patients with Leber Congenital Amaurosis 5 – is well-tolerated with good outcomes but comes with risks: in 429 eyes across 18 studies, 24.7% of eyes had serious adverse events, including chorioretinal atrophy.  

Dr. Scruggs developed fibrin hydrogels for retinal gene therapy​, seeding retinal pigment epithelium cells on top of the fibrin hydrogel and then implanting it underneath the retina. Dr. Scruggs demonstrated that in pigs, the fibrin dissolves, leaving a layer of retinal pigment epithelium cells in place. She used fibrin hydrogels in a human to treat a macular hole without causing inflammation. Dr. Scruggs also used fibrin hydrogels to deliver an AAV to the retina in pigs, without doing a subretinal injection.

Because hydrogels do not cause inflammation and are easy to handle and store, their use could advance gene therapy for retinal degenerations, as well as for other ocular or systemic disorders​.

SESSION 7: Retinal Gene Augmentation: Clinical

Moderators: Angela Bowman, PhD, and Todd Durham, PhD, Foundation Fighting Blindness

Safety and Efficacy of OPGx-RHO Silence-and-Replace Gene Therapy for RHO-adRP: Evidence Across Two Large Animal Models

Ashwath Jayagopal, PhD, Opus Genetics, reported two company programs, including OPGx-RHO – a mutation-independent potential treatment for rhodopsin-associated autosomal dominant retinitis pigmentosa​ – in pig and dog models. Research collaborators used an AAV to target mutant rhodopsin and deliver a functional copy.

There was no toxicity in dogs during the study. The team found that while the low dose was well-tolerated, higher doses led to increasing retinal inflammation, helping to establish the ideal dose with no adverse effects at 4.74 × 10¹⁰ vg/eye. In the pig model, the treatment led to good cone preservation at the mid/low dose and dose-dependent preservation of rods and outer nuclear layer thickness.

Dr. Jayagopal also highlighted that Opus Genetics plans to conduct Phase 3 dosing for OPGx-LCA5 in 2026​ and expects three-month results from Phase 1/2 trials for OPGx-BEST1 mid-2026.

The work was funded by the Foundation’s Brint Family Translational Research Award. Opus Genetics is a company founded by the Foundation and is an RD Fund portfolio company.

Intein-Based Dual-AAV Subretinal Gene Therapy for Stargardt Disease

Aniz Girach, MD, SpliceBio, discussed SB-007, a potential treatment for Stargardt that uses two AAV vectors to restore expression of the ABCA4 protein. Genetic mutations in the ABCA4 gene typically reduce the ABCA4 protein, which causes build-up of waste by-products that lead to photoreceptor death and vision loss. Because the ABCA4 gene is larger than the packaging size of AAV vectors, two vectors are used to restore the gene by delivering it in two halves.  

The team was able to express a functional human ABCA4 gene in rodents, pigs, and non-human primates. The protein was delivered to the appropriate location in photoreceptor outer segments.

An observational study, POLARIS, is recruiting 180 patients across 21 sites in the U.S. and Europe and will enroll fast progressors into the Phase 1/2 study. A dose-escalation study (ASTRA) finds that SB-007 has a good safety profile in line with other AAV sub-retinal gene therapies.

Design and Early Clinical Insights from LUCE: Dual-Vector MYO7A Gene Therapy in Usher Syndrome Type 1B

Jayashree Sahni, MD, PhD, AAvantgarde Bio, presented a potential gene therapy treatment for Usher Syndrome Type 1B, which causes profound deafness, vestibular dysfunction, and early onset retinitis pigmentosa, leading to progressive, irreversible decline of visual function. AAvantgarde Bio uses a dual AAV system to deliver the MYO7A gene that underlies USH1B.

The Phase 1/2 LUCE trial of patients with biallelic mutations in the MYO7A gene found no serious adverse events or dose-limiting toxicities in low- or mid-dose patients, although best corrected visual acuity in patients has not recovered since receiving the high dose. Several patients saw improvements in visual function six months after receiving either the low or mid dose.

Safety and Efficacy of ATSN-201 in Patients with X-linked Retinoschisis: An Update on The LIGHTHOUSE Study

Shannon Boye, PhD, Atsena Therapeutics, discussed progress on ATSN-201, a potential subretinal gene therapy to treat X-linked retinoschisis. A mutation in the RS1 gene makes the retinoschisin protein non-functional, leading to extreme tearing of the retina – called a schisis – and profound vision loss in males typically in their 50s and 60s.

Because of the fragile retina tissue in XLRS patients, ATSN-201 uses a novel capsid that spreads laterally after being injected subretinally to restore the retinoschisin protein and close the tearing of the retina.

The LIGHTHOUSE Phase1/2 studies found that ATSN-201 closed the foveal schises in seven out of nine treated eyes among adults treated at different doses and in four out of six eyes in six months among adults treated with different volumes. Schisis closure corresponds with functional improvements. Early data from safety studies in children finds minimal inflammation.

The Foundation’s RD Fund is an original investor in Atsena.

One-Year Safety, Visual Function, and Biomarker Outcomes of IVB107/PUMCH-E101 Gene Therapy for RDH12-Associated Retinopathy

Cheng Wang, PhD, Innovec Biotherapeutics, discussed updates on IVB107, a gene therapy under development for RDH12-Associated Retinopathy. RDH12 mutations lead to severe retinal degeneration and can be diagnosed as retinitis pigmentosa, Leber Congenital Amaurosis, and early-onset severe retinal dystrophy​.

IVB107 is adeno-associated virus vector which delivers RDH12 and is designed for intravitreal injection. It restores enzymatic activities in rats, as well as in patient-derived retinal organoids.

Patients with severe visual field impairments, macular atrophy, pigment proliferation, retinal thinning, and structural disorganization were treated with either low or high doses. IVB107 was generally safe, with a total of 40 treatment-emergent adverse events (TEAEs) and 16 ocular TEAEs reported. There was a mean BCVA improvement of 6.5 in study eyes. IVB107 has received special prescribing authorization under Hainan Boao Pilot Zone policies in China. In the U.S., it has been granted fast track designation and has received IND acceptance from the FDA.

The most advanced progress in Gene Replacement Therapy for Bietti Crystalline Corneoretinal Dystrophy (BCD)

Liping Yang, MD, PhD, Peking University Eye Center, discussed ZVS101e, a potential gene replacement therapy for Bietti Crystalline Corneoretinal Dystrophy (BCD), a rare autosomal recessive IRD caused by mutations in the CYP4V2 gene. Dr. Yang used an AAV2 vector to introduce a normal copy of the gene through subretinal injection.

In Phase 1/2 clinical trials, 50% (3/6) of low dose patients improved more than 15 letters on the eye chart one year after treatment. Average BCVA gains were 14 letters.

During the Phase 3 clinical trial, 11 (35%) participants in the treatment group achieved a BCVA improvement of more than 15 letters after 24 weeks. One patient gained 37 letters, and another gained 16 letters on BCVA. The treatment slowed progression of BCD.

ZVS101e was granted Breakthrough Therapy Designation in China and obtained Advanced Therapy for Regenerative Medicine certification from the FDA.

Phase 1/2 Trial of Subretinal Gene Therapy FT-002 in Participants with RPGR-associated XLRP

Ruifang Sui, MD, PhD, Peking Union Medical College Hospital, discussed nine-month results from pediatric RPGR-associated X-linked retinitis pigmentosa patients treated with the potential gene therapy FT-002. Mutations in the RPGR gene account for more than 70% of XLRP cases. Pediatric patients included males who were 13 years old on average, with confirmed mutation of the RPGR gene. Patients were treated with a subretinal injection of 2×10¹¹ vg/eye.

Six children were enrolled, and they did not experience serious adverse events or treatment-related ocular inflammation. Children had sustained improvement in low level visual acuity for nine months. The Phase 2 clinical trial is currently underway, with more data in children and adults planned for the last quarter of 2026.