Krystal Biotech received an RMAT designation for its gene therapy KB103 on June 24, 2019. KB103 is being studied for its wound closure capacity in patients with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”). The FDA also granted a RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation. [Read more…]
Brad Taylor, PhD and Dominic Clarke, PhD
HemaCare Corporation 8500 Balboa Boulevard Suite 130 Northridge CA 91325
Public discourse on cell and gene therapy is focused on novel approaches to long-established procedures such as bone marrow transplants, as well as on truly innovative treatments such as cancer immunotherapy and stem cell therapy for autoimmune disorders and other serious ailments. Positive clinical trial results and the demonstrated success of advanced therapies such as Kymriah® and Yescarta® are fueling a sense of excitement on what could be accomplished with these medicines—and driving a significant increase in the number of cell therapies in the clinical trial pipeline. As of last year, there were nearly 300 different cell and gene therapies in development, and that number is on target to grow. [Read more…]
With a swell of R&D activity flowing into the development of cell and gene therapies over the past decade, a manufacturing shortage gripped the industry. This caused industry participants to express grave fears that the manufacturing shortage would threaten industry growth and lengthen time-to-market for these new types of living therapies.
While there remains a shortage of skilled workers, the manufacturing capacity available to the cell and gene therapy marketplace has turned a proverbial corner, entering an era of prolific growth.
Aggressive construction of cell and gene therapy manufacturing facilities is now being undertaken by biotech companies seeking their own production capabilities, as well as by Contract Development and Manufacturing Organizations (CDMOs) who plan to serve third-party clients. [Read more…]
ElevateBio, a newcomer to the cell and gene therapy sector, is taking a different approach than the competition. Founded only months ago in early 2019, the company plans to aggregate a portfolio of cell and gene therapy companies that will develop, manufacture and commercialize life-transforming medicines.
It has launched with an unusual business model, which is to be a hybrid CDMO for cell and gene therapy companies, as well as an investment holding company. It will accomplish this feat by offering “R&D and cGMP manufacturing facilities to be shared across several portfolio companies, each with a different disease focus.”
Key to ElevateBio’s plan is the construction of an R&D and cGMP manufacturing facility in Cambridge, MA, that it calls BaseCamp. The complex will act as a centralized research and manufacturing group to support a portfolio of promising cell and gene therapy companies, each which will have a different disease focus.
I had the honor of interviewing Dr. Ruby Yanru Chen-Tsai, Chief Scientific Officer and Co-founder of Applied StemCell Inc. (ASC), a fast growing biotechnology company in Milpitas, California. Specializing in gene-editing technologies and stem cell innovation, ASC offers an optimized series of tools for basic research study, drug discovery, bio-processing, bio-production and preclinical applications.
In this fascinating interview, we discuss Dr. Yanru Chen-Tsai’s impressive background, ASC’s unique product portfolio, its TARGATT™ gene editing technology, and the company’s future trajectory. [Read more…]