Avrobio‘s AVR-RD-03 Gene Therapy Effective in Pompe Mouse Model

AVR-RD-03, Avrobio‘s experimental blood stem cell gene therapy for classic infantile-onset Pompe disease, was found to be safe and effective in a mouse model of the disease. The findings, presented as a poster at the 2022 annual meeting of the American Society of Gene & Cell Therapy (ASGCT)…

Asthma Medicine Salmeterol Improves Effectiveness of Gene Therapy for Pompe Disease, Mouse Study Suggests

Salmeterol, a vasodilator approved for the treatment of asthma and chronic obstructive pulmonary disease (COPD), has shown potential to improve the effectiveness  of gene therapy for Pompe disease. Studies in mice demonstrated that administering salmeterol as an add-on treatment to gene therapy can significantly reduce the amount…

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.