(MENAFN- EIN Presswire) Leading Pharmaceutical companies are advancing the AAV vector gene therapy pipeline and its future growth potential. LAS VEGAS , NV, UNITED STATES, February 7, 2025 ...

Gene therapy is usually reserved for severe and ... These rats were randomly selected to receive AAVlac (a recombinant AAV vector expressing ß-galactosidase 18) or PBS vehicle.

The candidate in question is VY9323, an AAV gene therapy combining a highly potent pri-miRNA against superoxide dismutase 1 ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

Viralgen and Axovia Therapeutics are set to form a partnership to progress the development and manufacturing of an adeno-associated virus vector serotype 9 (AAV9)-based gene therapy for retinal ...

The nano-complexes released the loaded-AAV intracellularly ... approach to viral vector treatments, which are currently limited by neutralizing antibodies. Gene therapy is a treatment method ...

Viralgen and the CTNNB1 Foundation have come together to manufacture an investigational gene therapy designed to treat CTNNB1 ...

Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

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

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Patients with CTNNB1 Syndrome will benefit from this joint effort to develop and manufacture a new AAV investigational gene therapy ... a clinically validated vector that is expected to enter ...