adeno-associated virus (AAV)

Medicine May Open ATA845 Trials to People With AAV Antibodies

Astellas Gene Therapies has obtained the rights to develop medication that could help reduce preexisting immune responses against the viral carrier used in ATA845, its investigational gene therapy for adults with late-onset Pompe disease (LOPD). Called IdeXork, or Xork, the investigational medication was developed by Selecta Biosciences,…

Amicus CEO John Crowley Outlines Progress in Pompe, Fabry, Batten Therapies

Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, and in the nearly 10 years since the release of “Extraordinary Measures” — a tearjerker starring Brendan Fraser as Crowley and Harrison Ford as short-tempered scientist Robert Stonehill — biotech has seen a huge transformation, both…

Gene Therapy Candidate Holds Promise for Correcting Pompe Disease in Muscles and Brain, Mouse Study Suggests

Amicus Therapeutics announced positive preclinical data showing that its investigational gene therapy for Pompe disease is effectively delivered and leads to significant glycogen reduction at key tissues in mice, including the brain and spinal cord.

Gene Therapy Using Improved Virus Holds Promise for Pompe Disease Treatment, Mouse Study Shows

Gene therapy, using an improved version of a virus as a delivery vehicle, greatly improved muscle and respiratory function in a mouse model of Pompe disease. The study, “Systemic delivery of AAVB1-GAA clears glycogen and prolongs survival in a mouse model of Pompe disease,” was published in…