preclinical data

Muscle-directed gene therapy found effective in new rat model of IOPD

A gene therapy designed to travel into muscles resulted in widespread production of the enzyme that’s missing or faulty in Pompe disease, according to a study that also describes a new rat model of the infantile-onset form of the disease. Researchers also observed that rats given a single…

Spark Therapeutics’ SPK-3006 Shows Promising Preclinical Data for Treating Pompe Disease

Spark Therapeutics has announced promising preclinical data from animal studies regarding its experimental gene therapy, SPK-3006, to treat Pompe disease. With these positive results, the company plans to conduct a Phase 1/2 clinical trial in 2019 evaluating the safety of SPK-3006 in adult Pompe patients. The preclinical data were…