Nanoscope Therapeutics, Inc.
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
Nr. of Employees
small (1-50)
Nanoscope Therapeutics, Inc.
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.
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
Clinical development services for ophthalmic gene therapies
Design and conduct of early and later‑stage clinical trials, including randomized, double‑masked, sham‑controlled multicenter studies; endpoint selection and clinical data dissemination.
Preclinical research and assay development
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.
Regulatory strategy and submission support for ocular gene therapies
Advisory and operational support for regulatory filings and agency interactions, including IND preparation, End‑of‑Phase meeting preparation, and orphan designation strategy.
Clinical development services for ophthalmic gene therapies
Design and conduct of early and later‑stage clinical trials, including randomized, double‑masked, sham‑controlled multicenter studies; endpoint selection and clinical data dissemination.
Preclinical research and assay development
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.
Regulatory strategy and submission support for ocular gene therapies
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
Key Technologies
- Ambient‑light‑sensitive optogenetic proteins
- AAV‑based intravitreal gene delivery
- Non‑viral laser/optical gene delivery
- Intravitreal injection administration