The FDA has accepted Nanoscope Therapeutics’s Biologics License Application (BLA) for MOGENRY (sonpiretigene isteparvovec; MCO-010), an optogenetic therapeutic for the treatment of retinitis pigmentosa (RP).
This update comes nearly 14 months after the company first initiated rolling submission for the regulatory submission package.
Let’s start with the basics.
- See here for a rundown on optogenetics.
- Click here for some background on Nanoscope’s multi-characteristic opsin (MCO)-based science.
Lastly: Check out this recap on the company’ MCO technology platform, which serves as the basis for its optogenetic therapeutic approach to targeting retinal disease-caused vision loss.
- A note on this: The platform secured a U.S. patent earlier this year, giving its vision-restoring methods U.S. intellectual property protections through 2039 (and potentially beyond).
Alrighty, now to the therapeutic in question.
MOGENTRY is a mutation-agnostic optogenetic gene therapy that uses adeno-associated viral vector serotype 2 (AAV2) to deliver an MCO transgene.
- How: via a single, in-office intravitreal (IVT) injection.
Take a closer look at this asset, including what sets it apart from other retinal disease treatments.
Anything else?
The FDA previously granted MOGENTRY Orphan Drug and Fast Track designations for its RP indication.
To note: Fast Track designation qualified it not only for an expedited priority review application process but also enabled the package’s submission on a rolling basis.
Also worth noting: This submission was reportedly the first for any gene-agnostic gene therapy for retinal disease.
Speaking of this submission … what was it based on?
That would be results from the phase 2b/3 RESTORE trial (NCT04945772) and a long-term follow-up trial.
Regarding that first study: We previously reported its primary endpoint was met, demonstrating a statistically significant improvement in best-corrected visual acuity (BCVA) from Week 52 for both then-MCO-010 dose groups—as well as the therapeutic’s durable effect beyond Week 52.
What about that long-term follow-up study?
The company released 3-year vision improvements in October 2025 from a long-term extension (dubbed REMAIN) of the RESTORE trial.
See here for our coverage on the 152-week data, in which MOGENRY’s favorable safety and tolerability profile was maintained following just one IVT injection.
So … has the FDA assigned a PDUFA date yet?
Not yet! The first step was acceptance—now we wait to see if the federal agency will set a Prescription Drug User Fee Act (PDUFA) target action date to make MOGENRY the first gene-agnostic therapy to restore vision in legally-blind RP patients.
The timeframe: within the next 6 months (which would bring us to March 2027).
Got it. And in the meantime, any other clinical developments to keep an eye on?
Did we mention MOGENRY is also under clinical evaluation for a few other retinal disease indications?
The most prominent (after RP): Stargardt disease, for which we previously covered the therapy’s promising phase 2 safety and efficacy performance in the STARLIGHT trial.
- See here for a look at those other retinal disease targets.