The Tokyo-based biotech firm expects RV-001 to be able to alter light sensitivity for patients with limited or no vision regardless of genotype.

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

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

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Robin Fields ProPublica is a nonprofit newsroom that investigates abuses of power. Sign up to receive our biggest stories as ...

PARIS, France I February 12, 2025 I GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and ...

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

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

CSL Behring's gene therapy Hemgenix four years post-infusion data continue to show sustained efficacy and safety in adults with haemophilia B: King of Prussia, Pennsylvania Monday ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

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

Qure is planning soon to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162 for SOD1 ...