Nanoscope Therapeutics, Inc. has initiated its submission of a Biologics License Application (BLA) to the FDA for multi-characteristic opsin (MCO)-010, a gene-agnostic gene therapy for the treatment of retinitis pigmentosa (RP).
First up, this company and its science.
Nanoscope is targeting optogenetics (a therapeutic approach utilizing light to turn specific cells on and off) as a potentially groundbreaking new treatment method for retinal diseases.
- Check out all the conditions being targeted
How: Via the development of therapies (MCOs such as the aforementioned MCO-010) that “functionalize” (in the company’s words) retinal cells to become light (photo)sensitive.
- The basis for how this is done can be attributed to Nanoscope’s optogenetic technology.
And what happens when these retinal cells become light sensitive?
These “highly dense bipolar” retinal cells then preserve the visual processing network of neurons in the retina by either circumventing or supplementing dead photoreceptors.
In the context of RP: Over 100 genes (that are known) and nearly 3,000 corresponding mutations have been attributed to the retinal disease.
Alrighty, now tell me how MC0-010 works into this.
MCO-010 is an ambient-light activatable MCO optogenetic agent formulated to reprogram healthy retina cells—turning them photosensitive—via a single, in-office, intravitreal (IVT) injection (with no need for genetic testing).
How this is done: A proprietary adeno-associated virus serotype 2 (AAV2) and promoter technology are used to deliver MCO genes into retinal bipolar cells to enable vision in different color environments.
- The intended result for RP patients: Vision restoration featuring enhanced contrast and definition in real-world environments.
Nice! So how is MCO-010 unique in its RP treatment?
MCO-010 is gene-agnostic, which translates to mean that it addresses the broad genetic diversity of RP … regardless of the underlying mutation (and unlike standard gene therapies, which target specific genes).
- More on this: Gene-agnostic therapeutic approaches target common pathogenic pathways that lead to retinal degeneration—providing efficacy regardless of the specific gene defect for a wider range of inherited retinal disease (IRD) patients.
And importantly: This BLA submission of MCO-10 reportedly marks the first for any gene-agnostic gene therapy for retinal disease.
Before we get more into this BLA, talk about MCO-010’s clinical journey so far.
To start: The therapeutic has received both FDA Orphan Drug and Fast Track designations for RP (as well as for Stargardt disease, which it is also under clinical development for).
- Take note: Fast Track designation also qualified MCO-10 for priority review (expediting the application review process to within 6 months)
And in regards to its RP clinical experience?
MCO-010 was evaluated in two doses (versus sham) during the phase 2b RESTORE trial (NCT04945772).
- The primary endpoint was evaluating the efficacy of a single MCO-010 injection via best-corrected visual acuity (BCVA).
As for the data: As we reported in March 2024, the study met its primary endpoint, demonstrating a statistically significant improvement in BCVA from Week 52 for both MCO-010 dose groups—and the therapeutic’s durable effect beyond Week 52.
Now about this rolling BLA submission …
The rolling submission of a BLA is typically permitted by companies who have received Fast Track designation (which also grants them that aforementioned priority review eligibility and more frequent meeting with the federal agency).
And what does this process entail?
A company may submit sections of its application to the FDA as they become available—in lieu of one full submission with all required sections (such as preclinical and clinical data as well as manufacturing and labeling information).
Importantly: A company must receive FDA agreement prior to beginning its rolling submission.
- In this case: Nanotech reported in October 2024 that the planned “rolling submission” of its BLA was acknowledged and accepted following a meeting with the FDA.
So when does the company anticipate a full BLA submission?
With the first modules of its application already reported to be submitted, Nanotech shared plans for a complete BLA submission by early 2026.
The big-picture potential of this: If eventually FDA approved, MCO-010 could become the first gene-agnostic therapy to restore vision in legally-blind RP patients.