Audentes Therapeutics provides updates on their AAV-based genetic medicine program for serious rare neuromuscular diseases.
In what could eventually be very good news for Pompe patients, Amicus Therapeutics presents positive data from their preclinical gene therapy program for Pompe disease.
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
Aldevron will supply plasmid DNA to fulfill Sarepta Therapeutics’s needs for its gene therapy clinical trials and commercial supply.
Audentes Therapeutics, a gene therapy biotech company, is on track to file an Investigational New Drug (IND) application in 2019 for Pompe disease.
Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies
Focussing on Pompe, Fabry, CDKL5, Amicus Therapeutics begins a collaboration with the Gene Therapy Program at the Perelman School of Medicine at University of Pennsylvania.
Spark Therapeutics announced new preclinical data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease.
Audentes Therapeutics presented pre-clinical work in support of an IND submission to study AT982 in both infantile and late onset Pompe disease patients.
Sarepta Therapeutics signs agreement with Lacerta Therapeutics, gaining rights to CNS-targeted Gene Therapy programs
Sarepta has expanded its presence in gene therapy by licensing programs from Lacerta, including exclusive rights to Lacerta’s candidate for Pompe disease.
Cambridge, MA, September 20, 2017 – AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following a Phase 1 Fabry program and a pre-Phase 1/2 Gaucher program.