One of a handful of gene therapy companies with pipelines that include Pompe disease. Today AVROBIO announced a halt to their programs.
Cheryl Wong Po Foo, PhD, Director of Clinical Development, Astellas Gene Therapies, responsible for the advancement of the clinical development program for Pompe disease.
The Spark Therapeutics phase 1/2 gene therapy trial testing SPK-3006 to treat late onset Pompe disease has officially started with the first person being given their first (and theoretically only) dose.
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
This development should provide Audentes with additional resources for their gene therapy programs, including a Phase 1/2 study for Pompe disease.
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.
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
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.
Audentes Therapeutics, a gene therapy biotech company, is on track to file an Investigational New Drug (IND) application in 2019 for Pompe disease.