An FDA advisory committee offered up lower vector doses, stricter exclusion criteria, and long-term animal studies to grapple with liver toxicities associated with adeno-associated virus (AAV) ...

As gene therapy evolves to target additional indications—including more common indications—the choice of delivery platform is bound to become more interesting. At present, the most common delivery ...

AAVs are attractive viral vectors for gene therapy due to their lack of pathogenicity, low immunogenicity, broad tropism and persistent transgene expression in both proliferating and quiescent cells.

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...

WATERTOWN, Mass. - Gene therapy has traditionally been conceptualized as a one-time, curative treatment option; however, research shows that there may be a need for subsequent doses years after ...

Adeno-associated viral vectors comprise the majority of recent gene therapy development programs due to their broad tissue-tropism and relatively low immunogenicity. Recent gene therapy approvals, ...

What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...

Viral vices have been center stage throughout the ongoing pandemic, but to the everyday scientist, viruses are essential tools in the research toolbox. Viruses are vectors not of disease, but of ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a collaboration and license agreement with Roche (SIX: RO ...

Dyno Therapeutics CEO and co-founder Eric Kelsic, PhD, likens his company’s development of transformative AI-designed adeno-associated virus (AAV) vectors for gene therapy to climbing a mountain—a ...