Nanoscope Therapeutics


Nanoscope Therapeutics is advancing gene therapies to restore sight in patients suffering from retinal degenerative diseases using ambient light-sensitive molecules. The company develops optogenetic gene therapies for conditions such as Retinitis Pigmentosa, Stargardt disease, and dry age-related macular degeneration, aiming to provide vision restoration where no cure currently exists. Their pipeline includes optogenetics-based retinal regeneration therapy and a proprietary non-viral laser gene delivery platform. Recognized as a top retina company, Nanoscope's MCO gene therapy has a unique mechanism of action—fast, broadband, and ambient-light sensitive—allowing vision restoration in everyday settings. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt disease.

Industries

biotechnology
health-care
therapeutics

Nr. of Employees

small (1-50)

Nanoscope Therapeutics


Patents

Optogenetic modulation by multi-characteristic opsins for vision restoration and other applications thereof

US-12459977-B2

View Details

Products

Ambient‑light‑sensitive opsin gene therapy platform (intravitreal AAV delivery)

A gene therapy platform delivering engineered ambient‑light‑sensitive opsin genes to retinal cells via intravitreal administration to restore functional vision across retinal degenerative diseases in a mutation‑agnostic manner.


Services

Design and conduct of early and later‑stage clinical trials, including randomized, double‑masked, sham‑controlled multicenter studies; endpoint selection and clinical data dissemination.

Laboratory development of retinal organoids and in‑vivo animal models, OCT‑integrated label‑free functional assays, and targeted delivery methods for assessing gene therapy potency and efficacy.

Advisory and operational support for regulatory filings and agency interactions, including IND preparation, End‑of‑Phase meeting preparation, and orphan designation strategy.

Expertise Areas

  • Optogenetic gene therapy for retinal degenerative diseases
  • Preclinical retinal model development and organoid‑based assays
  • Retinal gene delivery methods (AAV vectors and non‑viral optical approaches)
  • Design and execution of ophthalmic clinical trials including randomized sham‑controlled studies
  • Show More (4)

Key Technologies

  • Ambient‑light‑sensitive optogenetic proteins
  • AAV‑based intravitreal gene delivery
  • Non‑viral laser/optical gene delivery
  • Intravitreal injection administration
  • Show More (5)

News & Updates

Nanoscope plans to submit a Biologics License Application (BLA) to the FDA for MCO-010 to treat Retinitis Pigmentosa.

Nanoscope received IND clearance from the FDA to begin a Phase 2 trial of MCO-010 for Stargardt disease.

Nanoscope received a $1.5 million Phase 2b SBIR grant from the NIH to develop its ambient light activatable optogenetic gene therapy for AMD.


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