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.
Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society
CRANBURY, N.J., Oct. 04, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells.
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.
CONCORD, Mass., July 11, 2017 /PRNewswire/ — Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in patients with late-onset Pompe disease.
Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study
CRANBURY, N.J., (May 15, 2017) (GLOBE NEWSWIRE) – Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC).