DURHAM – Precision BioSciences, a genome-editing company based in Durham, announced today the opening of its highly prized production facility for in-house manufacturing of its unique technologies.
Calling it the Manufacturing Center for Advanced Therapeutics (MCAT), Precision touts the facility as “the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor (CAR) T cell therapy products.”
CAR T cell therapies are opening the door to a whole new way to fight cancer. But they also require a highly specialized manufacturing process, in which scientists engineer T cells – a type of immune system cell that recognizes invading germs or cancer cells – to enable them to carry a tumor-targeting “warhead” called a chimeric antigen receptor (CAR).
Traditional CAR T cell therapy technologies isolate cells from cancer patients’ blood and re-engineer them to specifically target receptors on tumor cells. The reprogrammed cells are multiplied in a laboratory and then returned to the same patient, to target that patient’s tumor. These are called autologous CAR T cells, and their creation involves a complex and lengthy process.
Precision’s proprietary ARCUS genome editing technology enables the production of CAR T cells derived from healthy donors rather than relying on the patient’s own blood. These “off-the-shelf” products are called allogeneic CAR T cells. This approach aims to overcome the manufacturing-related limitations of traditional autologous CAR T therapies and enable a broader range of malignancies to be targeted.
Precision’s product candidates can be made in advance, manufactured in large batches and then cryopreserved for shipment, storage and off-the-shelf use.
Precision’s ARCUS genome-editing platform also streamlines the therapeutic process because it removes a T cell receptor in order to prevent graft versus host disease, avoiding the need for donor-patient matching required in traditional tissue donation procedures. ARCUS editing also enables targeted insertion of the CAR gene into a single, specific location in the T cell genome for more controlled, consistent expression.
“As part of our mission to overcome cancer and provide valuable new treatment options for patients, we are rapidly advancing a pipeline of next-generation, off-the-shelf CAR T product candidates and we anticipate, once optimized, this platform will be able to support two new clinical programs per year,” said Matt Kane, chief executive officer and co-founder of Precision.
“Given the potential output of our platform, we’ve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.”
Currently, the new facility is undergoing CQV (commissioning, qualification, and validation) in preparation for cGMP manufacturing to begin in the fourth quarter of 2019. It is a multi-product facility designed to produce three different drug substances: allogeneic CAR T cells, messenger RNA (including formulations development) and adeno-associated viral vectors. Precision intends to initially use this new manufacturing center to create clinical trial material for its Phase I/II clinical trials in 2020. Precision BioSciences’ MCAT facility is designed to meet regulatory requirements in the United States, Europe and Japan.
Precision is developing product candidates in three innovative areas: its allogeneic CAR T immunotherapy, gene correction therapies, and, through its recent agricultural spinout, Elo Life Systems, food.
With the MCAT opening, Precision still has plenty of room to grow. It’s now leasing 17,300 square feet of its headquarters building, which contains 143,000 square feet of leasable space. The facility can allow the current staff of 10 to expand to 24 employees.
(C) N.C. Biotech Center