AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

The AAV Innovation Summit 2025, hosted by Form Bio, will bring together top experts in gene therapy to discuss the latest ...

This article examines viral and non-viral vectors in gene therapy, highlighting their mechanisms, advantages, and limitations ...

Explore a comprehensive guide to standardizing large scale production and clarification of lentiviral vectors.

Amid a broader push to beef up cell and gene therapy manufacturing capacity in Europe, Novartis has opened the doors to a €40 ...

Using “cure” to refer to “obtaining a normal life” also fits — if you ignore previously lost education, career opportunities ...

Signs an Amended & Restated License Agreement for Multiple Technologies for Gene Therapy for Type 1 and Type 2 Diabetes Forms a Wholl ...

The Mater Hospital has successfully treated the first patient in Ireland with a ground-breaking ocular gene therapy. The gene ...

The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.