Andelyn Biosciences has partnered with the INADcure Foundation to bring the first-ever gene therapy for Infantile Neuroaxonal Dystrophy (INAD) to clinical trials.
Contract development manufacturing organization (CDMO) Andelyn will deliver adeno associated-associated virus vectors (AAV) in a ready-to-use format to the INADcure Foundation. The foundation was formed in 2017 to accelerate development for the treatment of INAD and other types of PLA2G6-related neurodegeneration (PLAN).
“Andelyn will manufacture the gene therapy under GMP and sterile conditions in specially qualified cleanrooms,” a spokesperson for Andelyn told BioProcess Insider.
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“The adherent gene therapy production replicates this virus with the gene inserted and grows by over 1-1000 billion-fold. This virus will then be purified, and a large battery of 25-50 tests are conducted to make sure the manufacturer has achieved its goal of safe and effective therapy. Finally, the gene therapy will be aseptically transferred to a vial, and then cooled to a long-term storage temperature of approximately -60°C to await administration. The one-time administration is then expected to be a long-term durable therapeutic replacing or adding the gene activity that was missing.”
According to the CDMO, while the partnership is an “accelerated process to help get the therapy ready for manufacturing, there is no definitive end date” and the collaboration “has been established for long-term engagement.”
The production of clinical grade material will take place in one of Andelyn’s three facilities:
“The Andelyn Plasmid Center (APC) will produce any plasmids contracted; the Andelyn Development Center (ADC) will be the partner group to advance preclinical work including process development, analytical development, and preclinical manufacturing. The Andelyn Corporate Center (ACC) is Andelyn’s clinical and commercial GMP manufacturing facility which will produce the GMP clinical material for INADcure therapy that will be used in clinical trials,” the spokesperson said.
Andelyn opened the ACC located in Columbus, Ohio in October and said that the 200,000 square-foot gene therapy production plant has the capability to support any scale of gene therapy viral vector manufacturing.
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