FDA Accepts Nanoscope's BLA for MOGENRY, an Optogenetic Gene Therapy for Severe Vision Loss in Retinitis Pigmentosa
核心洞察
The FDA has accepted and filed Nanoscope Therapeutics (搜索)' Biologics License Application for MOGENRY (搜索) (sonpiretigene isteparvovec, MCO-010) in retinitis pigmentosa (搜索) with severe vision loss.
The filing is supported by the Phase 1/2a trial and the RESTORE Phase 2b/3 sham-controlled trial, which met its primary and key secondary endpoints at weeks 52 and 76.
MOGENRY (搜索) is a one-time, in-office intravitreal optogenetic gene therapy that requires no genetic testing or surgical suite and showed no treatment-related serious adverse events.
The U.S. Food and Drug Administration has accepted and filed the Biologics License Application (BLA) from Nanoscope Therapeutics (搜索), Inc. for MOGENRY (搜索) (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration in patients with retinitis pigmentosa (搜索) (RP) who have severe vision loss. The Dallas-based biotechnology company announced the acceptance on Sept. 9, 2026, noting that the submission was made on a rolling basis under Fast Track designation and came nearly 14 months after the rolling submission was first initiated.
"FDA acceptance and filing of our BLA for MOGENRY (搜索) is a crucial milestone that brings us a critical step closer to offering a one-time, in-office treatment option to the retinitis pigmentosa (搜索) community with severe vision loss, who have no approved therapy today," said Sulagna Bhattacharya, chief executive officer of Nanoscope Therapeutics (搜索). "The strength of our RESTORE data, together with the durability we have observed through long-term follow-up in the REMAIN study, give us confidence in MOGENRY's potential to become a new standard of care for patients living with RP having severe vision loss."
RESTORE and REMAIN Data Underpin the Filing
The BLA is supported by positive efficacy and safety data from the Phase 1/2a trial (NCT04919473) and the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial (NCT04945772). According to the company, RESTORE met its primary and key secondary endpoints, demonstrating improvements in visual acuity at weeks 52 and 76. In the trial, the primary endpoint was met with a statistically significant improvement in best-corrected visual acuity (BCVA) from Week 52 for both MCO-010 dose groups, along with a durable treatment effect beyond Week 52. MOGENRY (搜索) was well tolerated, with no treatment-related serious adverse events reported.
Most patients dosed in RESTORE have continued into the REMAIN long-term extension study, which provides the long-term follow-up data included in the BLA. The company previously reported 3-year vision improvements from REMAIN in October 2025, with 152-week data showing that MOGENRY (搜索)'s favorable safety and tolerability profile was maintained following just one intravitreal injection.
"Restoring vision is the most challenging metric to achieve in ophthalmic gene therapy — and MOGENRY (搜索) seems to be clearing that bar, with years of improved vision alongside it. That combination makes this a milestone moment, not just for our patients with an unmet need and Nanoscope, but for our entire field," said Allen C. Ho, M.D., professor of ophthalmology at Thomas Jefferson University, director of Retina Research at Wills Eye Hospital, and chief medical advisor for Nanoscope Therapeutics (搜索). "MOGENRY does not require genetic testing or administration in a surgical suite, which could enable broad adoption by community retina practices. If approved, MOGENRY could usher in a new era of RP treatment within reach of patients who do not have access to a tertiary academic center."
SriniVas Sadda, M.D., A. Ray Irvine, Jr., MD, Endowed Chair in Clinical Ophthalmology and professor at the University of California, Los Angeles David Geffen School of Medicine and the Doheny Eye Institute, and chair of Nanoscope's Visionary Advisory Committee, emphasized the durability of the observed effect. "As retina specialists, we've watched plenty of promising drug candidates fail to show, let alone sustain, benefits for patients. What sets MOGENRY (搜索) apart is a meaningful benefit, with evidence of sustained effect for years. Long-term durability is exactly what physicians need to see in a one-time treatment and know their patients can truly rely on it," he said.
A Gene-Agnostic Approach to Photoreceptor Loss
MOGENRY (搜索) is an investigational, one-time, in-office, intravitreal optogenetic gene therapy built on Nanoscope's multi-characteristic opsin (搜索) (MCO) platform. The therapy uses an adeno-associated viral vector serotype 2 (AAV2) to deliver an MCO transgene. Nanoscope's MCO technology employs a proprietary genetically engineered synthetic opsin designed for optimized performance in terms of high light sensitivity across a broad spectrum and fast kinetics. By delivering a multi-characteristic opsin gene to the highly dense bipolar retinal cells, MOGENRY makes these surviving cells directly light-sensitive, enabling them to utilize the remaining visual circuitry following photoreceptor loss.
The company states that MOGENRY (搜索) does not require genetic testing, invasive surgery, or repeat dosing, and is designed for administration within existing retina office workflows. The MCO platform secured a U.S. patent earlier this year, providing U.S. intellectual property protections for its vision-restoring methods through 2039 and potentially beyond. According to one report, the submission was the first for any gene-agnostic gene therapy for retinal disease.
Disease Burden in Retinitis Pigmentosa
Retinitis pigmentosa (搜索) is a group of rare inherited disorders in which photoreceptor cells degenerate progressively, leading to impaired vision and eventual blindness. RP is linked to more than 1,000 mutations in over 100 genes. It is one of the leading causes of blindness among the working-age population in the U.S., affecting more than 100,000 people, of whom over 25,000 are legally blind. The rate of vision loss varies with the underlying mutation, but patients lose approximately 0.03 LogMAR — about 1.5 letters — per year on average, equivalent to roughly one line of vision on an eye chart every three years. A majority of patients are legally blind (worse than 20/200) by the age of 60.
Regulatory Designations and Broader Development Program
The FDA previously granted MOGENRY (搜索) Orphan Drug and Fast Track designations for the RP indication. The Fast Track designation qualified the therapy not only for an expedited priority review application process but also enabled the package's submission on a rolling basis. No Prescription Drug User Fee Act (PDUFA) target action date has been assigned yet; the timeframe for a decision is expected within the next six months, which would bring the agency to March 2027.
Beyond RP, MCO-010 is under clinical evaluation in additional retinal disease indications. The company has reported promising results from the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (搜索) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and Stargardt disease, along with RMAT designation for Stargardt disease, and EMA Orphan designations covering non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. MCO-010 has also received Sakigake and Orphan designations for inherited retinal dystrophies (IRDs) in Japan and an Orphan designation for IRDs in Saudi Arabia. A Phase 2 program for MCO in geographic atrophy (搜索) is expected to start in 2026, and other IND-ready programs include Leber congenital amaurosis (搜索) (LCA).
If approved, MOGENRY (搜索) would become the first gene-agnostic therapy to improve vision in patients with RP having severe vision loss, and the company suggests it has the potential to become the standard of care for this population, administered as a one-time, in-office injection without the need for genetic testing.