Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.
Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.
2018
(há 8 anos)
Fundação
2023
(há 3 anos)
IPO
NASDAQ
Listagem
