Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society

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.

AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease

AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat 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.

Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

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

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).

Valerion Reports Preclinical Potential for Pompe Treatment

Valerion Reports Preclinical Potential for Pompe Treatment

CONCORD, Mass. – (April, 2017) – Valerion Therapeutics, part of the Alopexx Enterprises portfolio of companies, has announced the development of VAL-1221, a fusion protein that combines its technology with recombinant human acid alpha-glucosidase (rhGAA) to improve delivery of rhGAA to affected tissues in patients with Pompe disease.

Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease

Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease

Concord, MA – (March 3, 2017) – Valerion Therapeutics announced today that it has developed a fusion protein, VAL-1221, which combines its proprietary antibody delivery technology with recombinant human acid alpha-glucosidase (rhGAA) to improve the delivery of rhGAA into affected tissues of patients with Pompe disease (Glycogen Storage Disease, Type II; GSDII).