A new state-of-the-art facility dedicated to plasmid manufacturing will be in operation by August 2019 in Gerenzano, Italy. The new site will couple with the existing GMP facility active in the Cell and Gene (C&G) therapy space. With 800m2 (8,000 square feet) of cleanrooms, the two facilities of Anemocyte, the first Biotech Manufacturing Organisation (BMO), will address manufacturing needs of Somatic Cells, Non-Viral Modified Cells, Vesicles and Plasmids for Viral Vectors. [Read more…]
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…]
As of today, the list of publicly announced RMAT’s has risen to 30. However, the U.S. FDA states it has received 108 total requests for RMAT designations and granted 40.
RMAT stands for “Regenerative Medicine Advanced Therapy” designation. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.
In this article:
- RMAT List
- RMAT Reviews by The FDA
- Regenerative Medicine Advanced Therapy (RMAT)
- RMAT Definition
- Future RMAT Designations
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…]