Retinitis Pigmentosa Research Advances

MeiraGTx acquires rights to bota-vec, seeks rapid regulatory filing

In April 2026, MeiraGTx acquired botaretigene sparoparvovec (bota-vec), a gene therapy for X-linked retinitis pigmentosa (XLRP) caused by mutations in RPGR, from Johnson & Johnson. Data from J&J’s Phase 3 LUMEOS study, which enrolled 95 patients treated in both eyes, showed meaningful improvements across multiple measures of vision. While the trial did not meet its primary endpoint, there was consistent signals from the secondary measures.

The company plans to file for regulatory approval in the U.S., Europe, and Japan as quickly as possible, with the potential for a commercial launch in 2027. The FDA has granted bota-vec Fast Track and Orphan Drug designations, and regulators in the European Union have granted Priority Medicines (PRIME) and Orphan Drug designations—all of which are designed to expedite the regulatory review process.

 

Sumitomo Pharma America recieves Orphan Drug Designation for cell therapy

In March 2026, Sumitomo was granted FDA Orphan Drug Designation for DSP-3077, an investigational cell therapy for the treatment of retinitis pigmentosa (RP). DSP-3077 is a investigational product made up of retinal sheets, which are transplanted into the eyes of patients with RP. It is currently being evaluated in a Phase 1/2 clinical study.

Benefits of orphan drug designation include tax credits for clinical trial costs, exemption from FDA user fees, and the potential for seven years of market exclusivity if the drug is eventually approved. These incentives help offset the significant costs and risks associated with developing treatments for rare diseases.

 

Octant Bio begins clinical testing of small molecule therapy for RHO-adRP

Octant Bio dosed the first healthy volunteer in its Phase 1/2 clinical trial of OCT-980, an investigational oral medication for rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). This is the first clinical program for Octant Bio.

OCT-980 aims to correct the underlying protein misfolding that characterizes many forms of RHO-adRP. When rhodopsin proteins are misfolded, they cannot properly function within photoreceptor cells and cause cellular stress, leading to progressive vision loss and eventual blindness. By targeting the root cause—helping proteins fold correctly and move to their proper locations within cells—OCT-980 has the potential to improve low-light vision and halt disease progression. As an oral medication, it could offer patients a convenient, non-invasive treatment option for this sight-threatening condition.

 

SparingVision launches Phase 1/2 trial for advanced RP

SparingVision, a Paris-based company developing therapies for inherited retinal diseases (IRDs), has dosed the first patient in its NYRVANA Phase 1/2 clinical trial for SPVN20, a gene-agnostic gene therapy designed to restore cone vision for people with advanced retinitis pigmentosa (RP) and potentially other IRDs. The trial was initiated in Belgium and will expand to France and Ireland. The company plans to seek clearance in 2026 to conduct the trial in the US. The RD Fund, the Foundation’s venture philanthropy arm, is an original investor in SparingVision. The Foundation also provided significant funding to Deniz Dalkara, PhD, Institut de la Vision, for preclinical development of an approach similar to the one used in SPVN20.

 

Beacon completes enrollment for its Phase 2/3 VISTA clinical trial for XLRP; launches Phase 2 LANDSCAPE trial

Beacon Therapeutics has reported vision improvements for patients receiving laru-zova, the company’s RPGR X-linked retinitis pigmentosa (XLRP) gene therapy, in the Phase 2 DAWN clinical trial. In DAWN, 15 male patients, who received the treatment in one eye in a previous trial, had their second eyes dosed. The average improvement in low luminance visual acuity (LLVA) for the second eyes of patients was 16 letters or about 3 lines on an eye chart. (LLVA is the ability to read letters on an eye chart in dim light.) Improvements in microperimetry, which measures light sensitivity at multiple points in the retina, were also observed. With enrollment completed in the pivotal Phase 2/3, the company expects to report results in 2026. The newly-launched LANDSCAPE trial will evaluate the safety and efficacy of bilateral dosing of the gene therapy, called laru-zova. In January 2026, the RD Fund announced an investment in Beacon.

 

Nacuity reports reduced photoreceptor loss in clinical trial for oral antioxidant therapy

Dallas-based Nacuity reported that NACA, its oral antioxidant, reduced photoreceptor loss by 50 percent in a Phase 2 clinical trial in Australia. The trial is for people with Usher syndrome (retinitis pigmentosa with hearing loss). The Foundation Fighting Blindness is investing up to $7.5 million to advance the promising, emerging drug for retinitis pigmentosa, Usher syndrome, and related conditions. Known as N-acetylcysteine-amide (NACA), the molecule is designed to slow vision loss by protecting retinal cells from oxidative stress. In previous Foundation-funded lab studies at Johns Hopkins University, NACA slowed retinal degeneration in rodent models of RP. Johns Hopkins and the National Eye Institute are also conducting an international Phase 3 clinical trial for NAC — the original, FDA-approved formulation of N-acetylcystieine. Nacuity plans to launch a Phase 3 trial in 2026.

 

Sepul Bio advances LCA10 and USH2A RNA (antisense oligonucleotide) therapies into clinical trials

Théa has launched a new business unit, Sepul Bio, to continue clinical development of two RNA therapies: sepofarsen for people with LCA10 caused by the IVS26 mutation in the CEP290 gene and ultevursen for people with exon 13 mutations in the USH2A gene. Both therapies, originally developed by ProQR, had shown efficacy in earlier clinical trials. Sepul Bio has dosed the first patients in its LUNA Phase 2b clinical trial for ultevursen and its Phase 3 HYPERION for sepofarsen.

 

 

PYC moving PRPF31 RNA therapy into Phase 3 clinical trial

PYC Therapeutics reported promising results from two Phase 1/2 clinical trials in the US and Australia for VP-001, its emerging RNA therapy for people with retinitis pigmentosa 11 (RP11) caused by mutations in the gene PRPF31. The trial investigators reported mean improvements in low luminance visual acuity (LLVA), the ability to read lines on an eye chart in dim light, for patients who received multiple therapeutic doses of VP-001. Additionally, the trial investigators reported mean improvements in microperimetry, which measures light sensitivity at different locations (foci) in the retina, for patients receiving multiple therapeutic doses in the two trials.

 

BlueRock’s photoreceptor cell therapy moves into clinical trial

BlueRock Therapeutics, a cell therapy company and wholly owned subsidiary of Bayer AG, has launched CLARICO, a Phase 1/2 clinical trial for OpCT-001, a photoreceptor cell therapy for people with inherited retinal diseases such as retinitis pigmentosa and cone-rod dystrophy. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the US.

 

Phase 3 clinical trial of NAC launched for RP patients

Johns Hopkins University (JHU) has fully enrolled a Phase 3 clinical trial of N-acetylcysteine (NAC) for the treatment of retinitis pigmentosa (RP). Known as NAC Attack, the 45-month study has enrolled approximately 438 patients at 30 sites throughout the US, Canada, Mexico, and Europe. Study participants have been assigned randomly 2:1 to either the treatment or placebo group, respectively. If the NAC group shows benefit (i.e., slowing of vision loss) at 21 months, participants in the placebo group will begin to receive the treatment. JHU’s Peter Campochiaro, MD, and Xiangrong Kong, PhD, designed the trial and are its lead investigators. The National Eye Institute is providing more than $20 million in funding for NAC Attack. NAC is an oral antioxidant approved in 1963 by the US Food & Drug Administration for acetaminophen (Tylenol^®) overdose. It is also used for treating cystic fibrosis and pulmonary diseases.

 

Ocugen conducting a Phase 3 clinical trial for its modifier gene therapy

The biotech company Ocugen has fully enrolled 140 participants in a Phase 3 clinical trial for its gene-agnostic, modifier gene therapy under development for people with retinitis pigmentosa (RP). One arm of the trial will enroll approximiately 70 participants with mutations in the RHO gene. The other arm will approximately 70 participants with mutations in other genes causing RP. In each arm, participants will be randomized 2:1 to treatment and untreated control groups, respectively. Results are expected in the beginning of 2027.

OCU400 is a one-time therapy injected underneath the retina to deliver copies of the NR2E3 gene to improve regulation of multiple functions in the retina, including photoreceptor maintenance and development, metabolism, phototransduction, inflammation, and cell survival. OCU400 uses a human-engineered, adeno-associated virus (AAV), which works like a container system, to deliver NR2E3 copies into the recipients’ photoreceptors.

 

Four companies conducting clinical trials for their optogenetic therapies for advanced RP

Ray Therapueitcs, GenSight, Bionic Sight, and Nanoscope have each launched clinical trials for  their optogenetic therapies for RP and potentially other retinal diseases. The treatments are designed to provide vision to people who are completely blind from conditions such as retinitis pigmentosa and Usher syndrome. Ray, GenSight, and Bionic Sight are designed to work by bestowing light sensitivity to ganglion cells in patients who have lost all of their photoreceptors. Nanoscope is targeting bipolar cells. All companies have reported some modest restored vision in their early-stage trials. Nanoscope completed its Phase 2 clinical trial and is seeking approval from the FDA for its approach.

 

Kiora reports vision restoration in Phase 1/2 clinical trial for photoswitch therapy

Kiora Pharmaceuticals has reported some vision restoration for participants in ABACUS-1, its Phase 1/2 clinical trial in Australia for KIO-301, a molecule designed to bestow light sensitivity to retinal ganglion cells in people with advanced retinitis pigmentosa (RP) and other retinal diseases. The company is also enrolling a 36-patient Phase 2 clinical trial in Australia for KIO-301. The goal of the study is to evaluate the safety and efficacy of 3 injections (every 6 weeks) of KIO-301. Known as a photoswitch, the molecule enables retinal ganglion cells to respond to light, thereby working like a back-up system for lost photoreceptors. Retinal ganglion cells, which are downstream from photoreceptors, often survive in advanced retinal disease but don’t naturally respond to light.

 

SparingVision conducting clinical trial to evaluate sight-saving protein for RP

SparingVision has completed dosing for all 33 patients in its Phase 1/2 clinical trial for its emerging therapy to preserve cone-mediated vision. A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. Scientists demonstrated that RdCVF prevented or slowed the degeneration of cones, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision.

 

FDA approves Spark’s vision-restoring gene therapy

In 2017, Spark Therapeutics’ vision-restoring RPE65 gene therapy received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition. Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. Study participants with severe vision loss reported putting away their navigational canes, seeing stars, being able to read, and recognizing faces of loved ones. Vision restoration has persisted for at least three years. The treatment is also designed to work for people with retinitis pigmentosa (RP) caused by RPE65 mutations. The Foundation invested about $10 million in more than a decade of lab research that made possible the RPE65 gene therapy clinical trial at the Children’s Hospital of Philadelphia (CHOP).

 

 

APRIL 2026