Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.
February 28, 2019 will be the twelfth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organisations from countries and regions all over the world will hold awareness-raising activities.
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.
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study of AT-GAA in Patients with Pompe Disease
Amicus Therapeutics has announced that the first patient has been dosed in their Phase 3 study of AT-GAA for patients with Pompe disease.
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.
Amicus Therapeutics announces positive data from their Phase 1/2 clinical study (ATB200-02) to investigate AT-GAA in both ERT-Naive and ERT-Switch patients with 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.