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 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.
SAN FRANCISCO, CA – February 26, 2015 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced its support for Rare Disease Day®, to be held on February 28th, 2015. The theme of this year’s Rare Disease Day is Living with a Rare Disease. In honor of the day, Audentes is pleased to announce the launch of a website.
SAN FRANCISCO, CA – December 2, 2014 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced the closing of a $42.5 million Series B financing.
SAN FRANCISCO, CA. – July 18, 2013 – Audentes Therapeutics Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing.