Acid alpha-glucosidase (GAA) enzyme

Astellas Gene Therapy for Pompe Disease Clears Important Safety Hurdle

Acid alpha-glucosidase (GAA) enzyme, Adeno-Associated Virus (AAV) Vectors, Astellas Pharma, BioSpace, Clinical Trials, Gene Therapy, Interim Data, Metabolic Diseases, Neutralizing Antibodies, Pompe disease, R&D, Safety Data

At WORLDSymposium, Astellas Pharma presented interim safety data from a Phase I/II study assessing the company’s AAV gene therapy for the rare, autosomal metabolic disease Pompe disease.

Data presented at WORLDSymposium reinforces Sanofi’s robust rare disease pipeline

Acid alpha-glucosidase (GAA) enzyme, Business, Clinical Data, Enzyme Replacement Therapy (ERT), Muscle Cells, Pompe disease, Product Pipeline, Rare Diseases

Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.

Fast Track, Breakthrough and Priority Reviews: Sanofi and ViiV

Acid alpha-glucosidase (GAA) enzyme, Biologics License Application (BLA), Breakthrough Therapy Designation, Bruton's Tyrosine Kinase (BTK) Inhibitors, Chronic Immune Thrombocytopenia (ITP), Clinical Trials, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, HIV pre-exposure prophylaxis (PrEP), HIV Prevention, Muscle Disease, Orphan Drug Designation, PDUFA, Pompe disease, Priority Review Status, R&D, Sanofi

The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.