Gottlieb at BIO 2018: 40 Gene Therapy Approvals by 2022
Published: Jun 07, 2018 By Mark Terry
As the 2018 BIO International Convention wraps up in Boston today, it kicked off with a fireside chat with Dr. Scott Gottlieb, Commissioner of the U.S. Food and Drug Administration (FDA). Gottlieb was interviewed by Jim Greenwood, president and chief executive officer of BIO.
In a wide-ranging discussion, Gottlieb said he expects the FDA to approve 40 gene therapies by 2022 and might see cures for diseases like sickle cell anemia within 10 years. He also noted that there are complexities in gene therapy that are related to product issues as compared to clinical issues, which is requiring the agency to approach approvals differently. Gottlieb indicated that agency will publish six guidance documents regarding gene therapies soon. . .
Gottlieb also said the agency may be willing to accept more uncertainty for certain gene and cell therapies, particularly with those earning the new Regenerative Medicine Advanced Therapy (RMAT) designation. He noted that as of the end of April, there had been 62 RMAT designation submissions since the inception of the program in December 2016, and 19 designations have been granted. Of these 19 products, 14 also have orphan designation.
Here is a list of companies/products that have received FDA regenerative medicine advanced therapy designation or RMAT. Many of these are Cryoport clients . . .
Abeona Therapeutics (EB-101 Gene Therapy in Epidermolysis Bullosa)
Abeona Therapeutics (ABO-102 Gene Therapy in MPS IIIA)
Asterias (AST-OPC1, spinal cord injury)
Athersys (MultiStem)
BlueBird Bio (Lentiglobin in SCID)
Capricor (CAP1002, Duchenne Muscular Dystrophy)
Enzyvant (DiGeorge syndrome)
Fortress Biotech (Cellvation’s CEVA101, traumatic brain injury)
Humacyte (Vascular Access for Hemodialysis)
jCyte (Retinitis Pigmentosa)
Juno (JCAR017; CAR-T)
Kiadis Pharma (ATIR101, blood disorders)
Mallinckrodt/Stratatech’s Stratagraft
Mesoblast (Heart Failure, mesenchymal precursor cell therapy)
MiMedx Group (AmnioFix for osteoarthritis)
Vericel Ixmyelocel-T
