Last week in Part 1 of our series, we presented observations from industry leaders into how they plan for success by installing comprehensive quality management systems and laying down detailed groundwork for an optimized transition to GMP-compliant starting materials and technologies. This week in Part 2 of our series, cell therapy manufacturers present their own perceptions of what constitutes an ideal cell therapy starting material supplier, as well as what can be done on their end to ensure their cell therapy retains consistent quality and potency.
The emergence of cell and gene therapies onto the global stage is generating growing excitement, as unprecedented clinical success fuels the expectation of a promising new chapter in medicine. Alongside an extraordinary increase in the number of cell and gene therapies entering clinical trial, there are a lower yet still significant number of companies launching the commercialized manufacture of their cell therapy products.
The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies. [1] The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years:
HemaCare Corporation’s new global headquarters is officially open for business! The company started 2019 in a brand new 40,000 square foot space in a newly modernized 44-acre corporate campus located in the heart of the San Fernando Valley at Northridge. The design and construction of the cutting-edge facility provide a spacious workspace that promotes the well-being of its occupants, while significantly expanding the company’s on-site capabilities.
Manufacturing of clinical grade Tregs for cell based therapies
Graft-versus-host disease (GVHD) is a grave matter of concern for allogeneic hematopoietic stem cell transplantation (HSCT) where newly transplanted donor cells regard the recipient's body as foreign and attack them. This life-threatening complication is due to the fact that donor T-cells, present within the transplant recognize recipient major and/or minor histocompatibility antigens, become activated and attack target organs of GVHD. Although a variety of pharmacologic agents are available, such as calcineurin inhibitors and steroids to boost the tolerance against the clinical transplantation still more than 50% of severe GVHD patients are refractory to these current therapies[1]. Moreover, the long-term use of such agents impairs immune reconstitution, and diminishes T-cell mediated graft-versus-leukemia (GVL) responses. Therefore, there is a pressing need to develop specific cell-based therapies which may offer alternative approaches to prevent and/or to control severe GVHD.