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

BioViva’s journey in gene therapy began with its use of an adeno-associated virus (AAV) vector. The company’s lead gene therapy, BV-702, is AAV-based and is in preclinical development for ...

In the assessment of 12-month price targets, analysts unveil insights for 4D Molecular Therapeutics, presenting an average ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

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.

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

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

The adeno-associated virus (AAV) vector-based therapy is given as a one-shot intravenous infusion, delivering a gene for Factor IX, a clotting factor that is deficient in haemophilia B.

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 ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for ... carried by an adeno-associated virus (AAV) vector, which is administered into ...

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The following highlights showcase the most pivotal, innovative research published across multiple therapeutic areas in ...