Nanoscope Therapeutics, Inc. has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for multi-characteristic opsin (MCO)-019, its gene-agnostic gene therapy for Stargardt disease.
The news coincides with the company receiving a major regulatory boost in Europe—and follows just months after Nanoscope moved one step closer toward a potential regulatory approval for the therapeutic’s retinitis pigmentosa (RP) indication in the United States.
Lots of developments! Where to start?
We’ll begin with a brief rundown on Nanoscope’s science.
As we reported over the summer, the company’s therapeutic approach is based on utilizing optogenetics (learn more about this) to develop new therapeutics for inherited retinal diseases (IRDs)—including Stargardt and RP.
- See here for a look at those IRDs.
Referred to as MCOs, these therapeutics transform retinal cells to become light-sensitive (photosensitive) and “highly dense bipolar retinal cells” that preserve neurons’ visual processing network within the retina.
- The foundation for this approach: Nanoscope’s optogenetic technology.
And how does MCO-010 come into play?
This is an ambient-light-activated optogenetic therapeutic that reprograms healthy retinal bipolar cells into photosensitive cells.
How:
- Via a single, in-office intravitreal (IVT) injection—with no need for genetic testing
- Specifically, by using a proprietary adeno-associated virus serotype 2 (AAV2) vector and promoter system to deliver MCO genes
- These genes, in turn, allow for vision restoration.
What makes this therapeutic different from other IRD treatments?
Its gene-agnostic approach.
- See here for what we mean by this.
Now to this FDA designation … which isn’t the first, right?
That’s correct. As we previously reported, MCO-010 has already received Orphan Drug and Fast Track designation for both Stargardt and RP.
As for RMAT designation: This is reserved for regenerative medicine therapies (RMT) that use the body’s own cells to promote healing and repair tissues or organs.
Specifically: A candidate must be intended to treat, modify, reverse, or cure a serious condition (with preclinical evidence to back it up).
- See here for more on this, including the requirements a company must meet.
So how has MCO-010 performed in clinical trials so far?
Promising, to say the least. Looking at its Stargardt indication, the therapeutic was studied in the phase 2 STARLIGHT trial (NCT05417126).
What to know about this study: Investigators evaluated a single IVT-administered dose of MCO-010 among six Stargardt patients (≥16 years of age) over a 48-week period.
- See here for details on primary and secondary outcomes that were measured.
The data: As we reported in 2023, MCO-010 demonstrated a favorable safety profile consistent with that observed in prior phase 2 research for its RP indication. Check out the full findings.
- To note: A long-term safety follow-up study (dubbed SUSTAIN; NCT06048185) to the STARLIGHT trial is also in the works, with a 2027 estimated completion date.
Noted. Any phase 3 plans yet?
Indeed there are … Nanoscope actually shared an update last September following an end-of-phase-2 meeting with the FDA for MCO-010’s Stargardt indication.
Among the outcomes from that meeting: A proposed phase 3 registrational trial design for Stargardt, including its primary and secondary endpoints.
So when is this study expected to kick off?
According to the company: by the end of 2025 (late Q4).
As a side note: Nanoscope also expects to initiate a phase 2 trial on MCO-010 for a new IRD indication—geographic atrophy (GA)—in Q4 2025.
Nice! And in the meantime, what else should we know about MCO-010?
As we mentioned earlier, its RP indication has already made further progress towards regulatory approval in the United States.
- Case in point: In July 2025, Nanoscope began a rolling submission of its Biologics License Application (BLA)—with a full submission planned for early (Q1) 2026.
The potential: For MCO-010 to become the first approved therapy for both RP and Stargardt patients.