As more cell-based therapies transition towards the market, hurdles surrounding safety, efficacy, and manufacturing are becoming increasingly apparent. The advancement of gene delivery technologies is fundamental to driving more clinical prospects of cell therapies.
Overall, the growth in Asia’s bioprocessing capacity, particularly in China, has been impressive, albeit from a low baseline. Capacity growth over the past five years has been over 20 percent annually. New facility construction and expansions reflect the demand for biologics for domestic consumption, while other facilities are beginning to develop manufacturing strategies for GMP production for major markets, with capacity involving commercial-scale stainless steel and single-use bioreactors.
Robert Hariri, M.D., Ph.D., is an engineer, a jet aviator, a movie producer, a professor and a surgeon. However, it’s his experience as a biomedical scientist that led to him to be a successful serial entrepreneur, including his most recent biopharma venture — Celularity Therapeutics.
GenSight Biologics believes it is on the cutting edge of ophthalmology, gene therapy, and drug development. The company is focused on finding treatments for patients with neurodegenerative diseases and is developing products that are a combination of gene therapy, its mitochondrial targeting sequence technology, and optogenetics.
Delivering cell and gene therapies is an expensive and highly complex process, and there are a number of critical metrics that manufacturers should consider when selecting treatment sites. Subsequent activities — preparing sites to receive and initiate therapy, and managing site training and ongoing certification — can be equally, if not more, challenging.
When I look at the state of the contract manufacturing and development industry, I see an industry ripe for disruption. It isn’t inconceivable to envision an outsider coming in to shake things up.
A recent CDMO press release essentially asserted superiority via proximity: Geography as God of outsourcing. I contacted two biopharma executives familiar with the Boston/Cambridge scene for reactions: Michael Kaufman, Senior Vice President, CMC, Mersana Therapeutics, and 15-year veteran of Merck; and George Avgerinos, Senior Vice President of Biologics Operations, Fortress Biotechnologies, and 22-year veteran of BASF/Abbott/Abbvie.
Many biotechnology/pharmaceutical organizations are in the early stages of developing their risk management process. For the most part, we tend to see this process developing at the clinical trial level. The struggle with implementing these practices solely at the trial level is that it quickly becomes inconsistent and obsolete. Study teams are taking the time to assess risk as it applies to their trial, but the information gained is seldom shared cross-functionally or upward through an organization. It is more of a “going through the motions” type practice — which is then filed away, never to be utilized again.
While pursuing hid PhD in chemical engineering at MIT, Armon Sharei developed an interest in novel cell therapies. That interest led him to a discovery that today is the foundation of SQZ Biotech, a cell-therapy company where he serves as CEO. Sharei discovered a new method of inserting materials into cells more effectively than anything that currently existed.
Mary Elizabeth Williams is a journalist and an author. She is also a patient who has with metastatic melanoma who participated in a Phase 1 immunotherapy clinical trial. There is much that pharma can learn from her in terms of how companies approach clinical trials and drug development.
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