Innovation in Gene Therapy Starts at UPMC Children’s

UPMC Children’s is on its way to becoming the lead site for development and human clinical trials of a new gene therapy treatment for VLCADD (Very Long Chain Acyl CoA Dehydrogenase Deficiency), a rare genetic disorder that is identified through newborn screening.

UPMC Children’s Chief of Genetic and Genomic Medicine Dr. Jerry Vockley recently received a grant from Kriya Pharmaceuticals that will empower him to develop the ground-breaking treatment. “Kriya reached out to me because we at UPMC Children’s are renowned for being able to develop and move new therapies into clinical trials,” Dr. Vockley shares.

VLCADD can lead to extremely weak heart and skeletal muscle, cardiorespiratory failure, and repeated bouts of low blood sugar, and it can be life-threatening in children if not treated. Dr. Vockley has already developed the only FDA-approved medication for VLCADD, and this grant will enable him to advance care to the next level.

Dr. Vockley’s research around VLCADD is one of numerous gene therapy research studies happening at UPMC Children’s. Gene therapy is an experimental treatment that aims to modify the genetic information of the patient’s disease-causing cells, thereby curing or lessening the severity the disease. It is a field considered by many to be the future of medicine.

So, why exactly is UPMC Children’s excelling in gene therapy? “We are such a strong clinical institution, and we have that translational mindset,” Dr. Vockley says. “We can do the research and treat the patients simultaneously, which makes us a great partner for pharmaceutical companies who want to pursue new treatments and therapies.”

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