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).
â€śAndelyn will manufacture the gene therapy under GMP and sterile conditions in specially qualified cleanrooms,â€ť a spokesperson for Andelyn told BioProcess Insider.
â€śThe 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 -60Â°C to await administration. The one-time administration is then expected to be a long-termÂ durableÂ therapeuticÂ replacing or adding the gene activity that was missing.â€ť
According to the CDMO, while the partnership is an â€śaccelerated process to help get the therapy ready for manufacturing, there is no definitive end dateâ€ť and the collaboration â€śhas been established for long-term engagement.â€ť
The production of clinical grade material will take place in one of Andelynâ€™s three facilities:
â€śThe 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 Andelynâ€™s clinical and commercial GMP manufacturing facility which will produce the GMP clinical material for INADcureÂ therapy that will be used in clinical trials,â€ť 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.