Nanoscope Therapeutics Secures FDA RMAT Designation for Stargardt Disease Gene Therapy

Nanoscope Therapeutics, Inc. has achieved Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for its investigational gene therapy, MCO-010 (sonpiretigene isteparvovec), aimed at treating Stargardt disease. This designation is part of a broader regulatory strategy, as MCO-010 has also received Orphan Drug and Fast Track designations for both Stargardt disease and retinitis pigmentosa (RP). The European Medicines Agency (EMA) has granted MCO-010 five orphan designations across various retinal dystrophies, including macular dystrophies and both syndromic and non-syndromic rod- and cone-dominant dystrophies.

Nanoscope strengthens global regulatory pathways for MCO-010 with FDA RMAT and five EMA Orphan DesignationsNanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010FDA grants RMAT designation to Nanscope for Stargardt gene therapy

MCO-010 employs optogenetics to restore vision by transforming retinal cells into light-sensitive cells, utilizing a proprietary adeno-associated virus serotype 2 (AAV2) vector. This gene-agnostic approach allows for treatment without the need for genetic testing or repeat dosing, making it applicable to a wide patient population. The therapy is administered via a single intravitreal injection in an office setting.

FDA grants RMAT designation to Nanscope for Stargardt gene therapy

Clinical trials have shown promising results. The phase 2 STARLIGHT trial (NCT05417126) evaluated MCO-010 in six Stargardt patients over 48 weeks, demonstrating a favorable safety profile. Additionally, a long-term follow-up study, SUSTAIN (NCT06048185), is planned to assess ongoing safety, with completion expected in 2027. Nanoscope is also preparing to initiate a phase 3 registrational trial for Stargardt disease by late 2025.

FDA grants RMAT designation to Nanscope for Stargardt gene therapyNanoscope strengthens global regulatory pathways for MCO-010 with FDA RMAT and five EMA Orphan DesignationsNanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

The RMAT designation signifies the FDA's recognition of MCO-010's potential to address serious conditions with unmet medical needs. Sulagna Bhattacharya, CEO of Nanoscope, emphasized that this designation, alongside previous approvals, validates the company's efforts to expedite the development of therapies for patients with limited treatment options. The company has initiated a rolling submission of its Biologics License Application (BLA) for MCO-010 for RP, with full submission anticipated in early 2026.

Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

While Nanoscope's advancements are notable, the field of gene therapy for retinal diseases is competitive. Other companies, such as VeonGen Therapeutics, are also developing therapies like VG801 for Stargardt disease, which has received similar RMAT designation. This competition may influence the pace and focus of clinical trials and regulatory approvals in the coming years.

Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010FDA grants RMAT designation to Nanscope for Stargardt gene therapyNanoscope strengthens global regulatory pathways for MCO-010 with FDA RMAT and five EMA Orphan Designations

“Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for Stargardt disease and RP [retinitis pigmentosa] is a major validation for our therapies that warrant expedited development and review,” — Sulagna Bhattacharya, CEO of Nanoscope Therapeutics.

Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

“We’ve seen this investigational therapy surpass our expectations in the lab and in patients in clinical trials, and we believe we’re now one step closer to potentially bringing this pioneering therapy to all RP patients.” — Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope.

Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

Nanoscope Therapeutics is at the forefront of developing innovative gene therapies for retinal diseases, with MCO-010 poised to become a significant treatment option for Stargardt disease and RP. The combination of RMAT designation and ongoing clinical trials underscores the potential impact of this therapy on patients suffering from these debilitating conditions.

FDA grants RMAT designation to Nanscope for Stargardt gene therapyNanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010Nanoscope strengthens global regulatory pathways for MCO-010 with FDA RMAT and five EMA Orphan Designations