Share this story

MassBiologics announces partnership with PTC Therapeutics to manufacture gene therapy for rare pediatric condition

MBL-exterior-20-660.pngMassBiologics of UMass Medical School has entered into an agreement with PTC Therapeutics to manufacture biological investigational new drugs and commercial pharmaceutical products for use in humans.

“We are delighted to collaborate with PTC Therapeutics to create new medicines for rare diseases,” said Mark S. Klempner, MD, executive vice chancellor for MassBiologics. “For more than 125 years, MassBiologics has researched, developed, manufactured and distributed medicines for better lives.”

MassBiologics will provide dedicated manufacturing and quality control teams at its South Coast facility, which specializes in the manufacturing of viral vectors, a delivery system by which genetic material can be delivered to cells to treat disease.

Initially, MassBiologics will manufacture PTC Therapeutics’ novel investigational therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic condition caused by a mutation in the dopa decarboxylase (DDC) gene, resulting in a lack of functioning AADC enzyme. AADC deficiency results in delays or failure to reach developmental milestones such as head control, sitting, standing, walking or talking, low muscle tone (also known as muscular hypotonia), severe, seizure-like episodes involving involuntary eye movement (also known as oculogyric crises), autonomic abnormalities, and the need for life-long care. 

“UMass Medical School is proud to partner with PTC Therapeutics, a company with a demonstrated commitment to finding therapies for patients with rare conditions, and an equally promising track record,” said Chancellor Michael F. Collins.

“This innovative partnership demonstrates our commitment to achieving impact,” said Jim Glasheen, PhD, executive vice chancellor for innovation and business development. “Whether a partner is an academic innovator, a non-profit foundation or a for-profit company—we stand ready to help our collaborators change the world.”