The introduction of anti-VEGF agents has revolutionized the field of medical retina care by reducing rates of vision loss and ...

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

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

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

PDA has released Points to Consider No. 11 Development, Classification, Manufacture, Control, and Testing of Plasmids and Vectors Used in ATMP Production on its bookstore and membership technical ...

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

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

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

Accurate monitoring of particle size and growth is essential to ensure scalability in the development of transfection vectors ...

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

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

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.