rAAV1-CB-hAAT / BCM Families Foundation, Beacon Therapeutics - LARVOL DELTA

Regional Limb Perfusion of AAV1 Gene Therapy Vectors Results in Robust Muscle Transgene Expression in Cynomolgus Macaque (ASGCT 2024) - "We used two vectors packaged in adeno-associated virus serotype 1 (rAAV1), one carrying human AAT (rAAV1-CB-hAAT) that was manufactured using triple transfection and the other rhesus AAT (rAAV1-CB-rhAATmyc) that was used in our last study and manufacturing using HSV...Both vectors successfully transduced skeletal muscle in a safe manner at both lower and higher dosages. The vectors present potential for skeletal muscle gene augmentation therapy at high doses, particularly beneficial for cases with pre-existing liver conditions, offering a ""liver-sparing"" approach."

Gene therapy • Alpha-1 Antitrypsin Deficiency • Bronchiectasis • Chronic Obstructive Pulmonary Disease • Fibrosis • Gene Therapies • Hepatitis C • Hepatology • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases • ELANE • SERPINA1

Gene Therapy’s Renaissance (Genengnews) - "AAV-based gene therapy has made great strides in recent years. Pioneering work from the lab of Terence R. Flotte, MD, dean, provost, and executive deputy chancellor of the University of Massachusetts Medical School, has propelled clinical trials for...alpha-1 antitrypsin (AAT) deficiency....With excellent safety and durability established in a five-year follow-up of its Phase IIa trial, the AAT gene therapy is now in its final investigational new drug–enabling toxicology study stage."

Review • Alpha-1 Antitrypsin Deficiency