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…]
What is an RMAT and Who Has One? (30 Announced / 40 Granted)
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. [Read more…]
Have We Reached the Age of Advanced Therapies?
In the past few years, there has been a spurt in clinical development of advanced therapies, including cell therapies, gene therapies and tissue engineering products. Several cell-based medicinal products have been approved in the U.S., Europe, China, Japan, South Korea, India, Canada, Australia and New Zealand. A number of start-up companies, hospitals, universities, and large biotech and pharma companies have started testing hundreds of cell-based product candidates in clinical studies.
With a visible acceleration in development, the advanced therapy field has evolved from a curiosity to a vibrant arena for competition. [Read more…]
First iPSC-Derived CAR-T Therapy by CiRA and Takeda Nears Clinical Testing
− iCART Program Advances the Potential for a Highly Scalable, Lower Cost, Off-the-Shelf CAR T-Cell Therapy to Treat Cancer
KYOTO and OSAKA, Japan I July 16,2019 – The Center for iPS Cell Research and Application (CiRA) at Kyoto University and Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that a novel induced pluripotent stem (iPS) cell-derived chimeric antigen receptor (CAR) T-cell therapy (iCART) has been transferred from their T-CiRA research collaboration to Takeda as the program begins process development toward clinical testing. Under the terms of the T-CiRA agreement, Takeda has the global rights to develop and commercialize the iCART product and CiRA will receive development and approval milestones. The teams will continue to collaborate as they prepare the iCART program for a clinical trial in 2021. [Read more…]
U.S. FDA Approved RMATs on the Rise
With recent news from Sangamo Therapeutics and Krystal Biotech, the count of announced RMAT (“Regenerative Medicine Advanced Therapy”) designations has risen to 30.
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…]
