Neurogene

Genetic Therapies

NGN-401 Clin1/Clin2 Rett syndrome; NGN-201 Clin0 Krabbe disease; NGN-402 RD neurodevelopmental condition

Neurogene is a clinical-stage biotech company developing life-changing gene therapies for patients battling devastating neurological diseases. Their mission is to transform these complex and often incurable conditions into treatable ones. Neurogene utilizes adeno-associated virus (AAV) vectors, in addition to other viral and non-viral platforms to deliver functional copies of genes or gene editing tools directly into a patients' cells. Their proprietary platform, EXACT allows for precise control over transgene expression, minimizing potential side effects associated with conventional gene therapy approaches. Neurogene has constructed a state-of-the-art manufacturing facility to support their clinical development activities, ensuring quality and potentially expediting clinical trials.