Leading researchers from the NIH discuss the discovery of Pompe disease, Pompe disease research studies at the NIH, and the recognition of a new phenotype for Pompe disease following the introduction of enzyme replacement therapy.
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.
Audentes Therapeutics, a gene therapy biotech company, is on track to file an Investigational New Drug (IND) application in 2019 for Pompe disease.
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.
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. – (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.
CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and poster1 at the 13thAnnual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation.