In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some ...

A liver biopsy was performed in one participant 10 years after the receipt of gene therapy in order to conduct a molecular analysis under a separate protocol (EudraCT number, 2018-001333-40).

The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College ...

IRF8, interferon regulatory factor 8; BMAL1, brain and muscle ARNT-like 1; PPARγ, peroxisome proliferator-activated receptor γ; HFD, high-fat diet; CD, chow diet; AAV, adeno-associated virus ...

For each organoid, 1 x 1010 vg of each AAV serotype was placed into the organoid medium. GFP expression was evaluated by flow cytometry after 7 days.

The results underscore the potential of KRIYA-497, an investigational, intramuscularly-administered AAV gene therapy designed to express native fibroblast growth factor 21 (FGF21) protein.

“I am very pleased to announce the signing of this license agreement for our proprietary AAV capsid platform, JUST-AAV,” said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals.

Furthermore, this purification process is considered universal. The scientists successfully adapted it to a variety of AAV serotypes and genes of interest, including AAV8-GFP and AAV5-GFP.

They chose adeno-associated virus (AAV) as a gene delivery vector, as it exhibits low toxicity and immunogenicity, and stable gene expression in the nervous system. They developed an AAV construct ...