Spark Therapeutics announced new preclinical data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease. This was announced during the late-breakling session of the International Annual Congress of the World Muscle Society. This is a one-time investigational gene therapy that is engineered to use the liver to express the needed enzyme. Positive results have been seen at the 10-month point in mouse studies.
- Audentes Therapeutics’ “Be Bold for Courageous Patients” Program to Support Patients Living with Rare Diseases
- Amicus Therapeutics establishes a 75,000 sq. ft. Gene Therapy Center of Excellence
- Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
- Rare Disease Day 2019
- Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
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