In neuromuscular diseases, the most widely used vector for transporting genetic material is the natural adeno-associated virus (AAV). However, a large proportion of injected vectors do not reach ...

Roche will apply Dyno Therapeutics’ engineered adeno-associated virus (AAV) capsid platform to develop next-generation AAV vectors for gene therapies targeting unspecified neurological diseases ...

Biotech stocks are surging as groundbreaking neuroscience and gene therapy innovations take center stage. Amid this backdrop, ...

Recently, several techniques have been developed to enhance vector tropism, such as genetically inserting peptides on the AAV capsid to modify their interactions with known cell receptors.

Explore the role and challenges of AAV characterization, the significance of capsid filling, and the best methods to enhance ...

Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The firm’s clinical program is a LVV-based gene therapy for ...

Similarly, GoQURE incorporates gene silencing with gene replacement within one AAV vector. This approach removes the mutated gene's toxic products while repairing function by replacing the faulty ...

Existing gene therapies have primarily used a small number of naturally occurring AAV vectors limited by low delivery efficiency as well as problems with pre-existing immunity and manufacturability.

Capillary electrophoresis (CE) is transforming viral vector manufacturing for next-generation biopharmaceuticals. Traditionally used for quality control, CE can now be used to profile viral proteins ...

In the GAN trial, participants who were baseline AAV9 seropositive showed higher anti-AAV9 neutralizing antibody titers in the serum and experienced faster vector clearance after CSF-AAV ...