[Source]
PTC Therapeutics, Inc. has announced a collaboration with Aldevron, LLC to secure GMP manufacturing capacity for PTC's gene therapy portfolio, including the Friedreich ataxia and Angelman syndrome programs in PTC's growing gene therapy pipeline.
"Our strategic collaboration with Aldevron represents our continued commitment to produce and provide the highest quality product to patients," said Neil Almstead, Ph.D., Chief Technical Operations Officer of PTC Therapeutics. "Our gene therapy pipeline is addressing the unmet needs of multiple patient populations, and we feel an urgent need to develop safe products with the utmost speed. The development of relationships with top-tier companies like Aldevron aligns with our goal of partnering with the best collaborators as we drive meaningful improvements in the lives of patients."
"It is truly an honor to work with PTC's motivated team of experts. They are making enormous contributions to the future of genetic medicine," said Michael Chambers, Founder and CEO of Aldevron. "This is Aldevron's mission – to serve scientists and researchers who are relentlessly pursuing cures for people who need them."
The central nervous system (CNS) gene therapy platform at PTC uses targeted micro-dosing applied directly to areas involved in the disorder. This allows for greater efficacy, durability, lower risk of immunogenicity or other off-target effect, and efficient and scalable manufacturing. PTC has an advancing gene therapy pipeline for CNS disorders, including programs for AADC deficiency, Friedreich Ataxia, and Angelman Syndrome. PTC is preparing a BLA for the treatment of AADC deficiency for submission to the FDA in the fourth quarter of 2019.