In the mid-1990s, Hiroyuki Nakai, now an adeno-associated virus (AAV) researcher at the Oregon Health and Science University, moved to the United States. Nakai quickly became fascinated with the ...
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, ...
Adeno-associated viruses (AAVs) are used in nearly a dozen FDA-approved therapies and more than 900 R&D projects to deliver therapeutic payloads to cells. Yet, large-scale AAV production is still a ...
CNS-directed AAV gene therapy shows milder immune responses compared to systemic administration, reducing peripheral immune activation and systemic toxicities. Direct CNS delivery can bypass ...
Adeno-associated viruses (AAV) have emerged as pivotal delivery vehicles for gene therapy due to their overall safety (less immunogenic), efficiency (broad tropism), and long-term gene expression.
Several quality attributes (QAs) must be monitored to ensure an effective and safe final product when developing adeno-associated virus (AAV) vectors as drug products. Thermal stability, aggregate ...
PHILADELPHIA--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, ...