FDA Approved Regenerative Medicine Advanced Therapies Increasing

Krystal Biotech received a Regenerative Medicine Advanced Therapy designation for its gene therapy KB103 this past June 24, Sangamo Therapeutics received it’s RMAT designation on July 5, 2019 for its gene therapy SB-525.

Gene therapy KB103 is being investigated for wound closure capacity in those with severe generalized recessive dystrophic epidermolysis bullosa; and with the approval gene therapy SB-525 has become the first hemophilia A gene therapy to receive an RMAT designation. 

30 RMATs have been announced, according to the US FDA 108 requests have been received and 40 have been granted, which means 10 companies are operating in stealth mode regarding acquiring their RMAT designations, and approximately 37% of applications become approved. 

The Regenerative Medicine Advanced Therapy designation allows for faster, more streamlined approval of regenerative products in America, and these companies represent some of the foremost innovative competitors in the marketplace. 

Regenerative medicine companies are emerging with Century Therapeutics emerging from their stealth mode on this past July 1st with their mission to produce large quantities of allogeneic, homogeneous iPSC derived cellular therapeutics.