{"id":135438,"date":"2025-09-05T20:14:06","date_gmt":"2025-09-05T20:14:06","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/135438\/"},"modified":"2025-09-05T20:14:06","modified_gmt":"2025-09-05T20:14:06","slug":"fda-grants-rmat-designation-to-nanscope-for-stargardt-gene-therapy","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/135438\/","title":{"rendered":"FDA grants RMAT designation to Nanscope for Stargardt gene therapy"},"content":{"rendered":"

Nanoscope Therapeutics, Inc. has received Regenerative Medicine Advanced Therapy (RMAT) designation<\/a> from the FDA for multi-characteristic opsin (MCO)-019, its gene-agnostic gene therapy for Stargardt disease.<\/p>\n

The news coincides with the company receiving a major regulatory boost in Europe\u2014and follows just months after Nanoscope moved one step closer toward a potential regulatory approval<\/a> for the therapeutic\u2019s retinitis pigmentosa (RP) indication in the United States.<\/p>\n

Lots of developments! Where to start?<\/p>\n

We\u2019ll begin with a brief rundown on Nanoscope\u2019s science.<\/p>\n

As we reported over the summer<\/a>, the company\u2019s therapeutic approach is based on utilizing optogenetics (learn more about this<\/a>) to develop new therapeutics for inherited retinal diseases (IRDs)\u2014including Stargardt and RP.<\/p>\n

Referred to as MCOs, these therapeutics transform retinal cells to become light-sensitive (photosensitive) and \u201chighly dense bipolar retinal cells\u201d that preserve neurons\u2019 visual processing network within the retina.<\/p>\n

The foundation for this approach: Nanoscope\u2019s optogenetic technology.And how does MCO-010 come into play?<\/p>\n

This is an ambient-light-activated optogenetic therapeutic that reprograms healthy retinal bipolar cells into photosensitive cells.<\/p>\n

How:<\/p>\n

Via a single, in-office intravitreal (IVT) injection\u2014with no need for genetic testingSpecifically, by using a proprietary adeno-associated virus serotype 2 (AAV2) vector and promoter system to deliver MCO genesThese genes, in turn, allow for vision restoration.What makes this therapeutic different from other IRD treatments?<\/p>\n

Its gene-agnostic approach.<\/p>\n

Now to this FDA designation \u2026 which isn\u2019t the first, right?<\/p>\n

That\u2019s correct. As we previously reported, MCO-010 has already received Orphan Drug<\/a> and Fast Track<\/a> designation for both Stargardt and RP.<\/p>\n

As for RMAT designation: This is reserved for regenerative medicine therapies (RMT) that use the body\u2019s own cells to promote healing and repair tissues or organs.<\/p>\n

Specifically: A candidate must be intended to treat, modify, reverse, or cure a serious condition (with preclinical evidence to back it up).<\/p>\n

See here<\/a> for more on this, including the requirements a company must meet.So how has MCO-010 performed in clinical trials so far?<\/p>\n

Promising, to say the least. Looking at its Stargardt indication, the therapeutic was studied in the phase 2 STARLIGHT trial (NCT05417126<\/a>).<\/p>\n

What to know about this study: Investigators evaluated a single IVT-administered dose of MCO-010 among six Stargardt patients (\u226516 years of age) over a 48-week period.<\/p>\n

See here<\/a> for details on primary and secondary outcomes that were measured.<\/p>\n

The data: As we reported in 2023<\/a>, MCO-010 demonstrated a favorable safety profile consistent with that observed in prior<\/a> phase 2 research<\/a> for its RP indication. Check out the full findings<\/a>.<\/p>\n

To note: A long-term safety follow-up study (dubbed SUSTAIN; NCT06048185<\/a>) to the STARLIGHT trial is also in the works, with a 2027 estimated completion date.Noted. Any phase 3 plans yet?<\/p>\n

Indeed there are \u2026 Nanoscope actually shared an update last September<\/a> following an end-of-phase-2 meeting<\/a> with the FDA for MCO-010\u2019s Stargardt indication.<\/p>\n

Among the outcomes from that meeting: A proposed phase 3 registrational trial design for Stargardt, including its primary and secondary endpoints<\/a>.<\/p>\n

So when is this study expected to kick off?<\/p>\n

According to the company: by the end of 2025 (late Q4).<\/p>\n

As a side note: Nanoscope also expects to initiate a phase 2 trial on MCO-010 for a new IRD indication\u2014geographic atrophy (GA)\u2014in Q4 2025.<\/p>\n

Nice! And in the meantime, what else should we know about MCO-010?<\/p>\n

As we mentioned earlier, its RP indication has already made further progress towards regulatory approval in the United States.<\/p>\n

The potential: For MCO-010 to become the first approved therapy for both RP and Stargardt patients.<\/p>\n","protected":false},"excerpt":{"rendered":"Nanoscope Therapeutics, Inc. has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for multi-characteristic opsin (MCO)-019,…\n","protected":false},"author":2,"featured_media":135439,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[50],"tags":[200,79],"class_list":["post-135438","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","tag-genetics","tag-science"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/135438","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=135438"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/135438\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/135439"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=135438"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=135438"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=135438"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}