May 31, 2020

The Niche

Knoepfler lab stem cell blog

RMAT List (42)

Here is a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm names are to relevant press releases. A special thanks to some folks at ARM for helping me keep the list up to date.

It was updated as of May 6, 2020 with 42 total:

  1. Abeona Therapeutics (EB-101 Gene Therapy in Epidermolysis Bullosa)
  2. Abeona Therapeutics (ABO-102 Gene Therapy in MPS IIIA)
  3. AdaptImmune (ADP‑A2M4 for the Treatment of Synovial Sarcoma)
  4. AlloVir (Viralym-M for hemorrhagic cystitis)
  5. Asterias/BioTime (AST-OPC1, spinal cord injury)
  6. Athersys (MultiStem)
  7. Audentes (AT132 for the Treatment of X-Linked Myotubular Myopathy)
  8. AxoGen (Avance for nerve injuries)
  9. BlueBird Bio (Lentiglobin in SCID)
  10. Bristol-Myers Squibb (Liso-cel, CAR-T cell therapy for large B-cell lymphoma)
  11. Caladrius (CD34+ cell therapy for angina)
  12. Capricor (CAP1002, Duchenne Muscular Dystrophy)
  13. CARsgen (CT053 CAR-T therapy for Multiple Myeloma)
  14. Cellerant Therapeutics, Inc/Fibrocell. (Romyelocel-L to Prevent Infections During Neutropenia)
  15. Enzyvant (DiGeorge syndrome)
  16. ExCellThera (ECT-001 for blood cancers
  17. Fibrocell (FCX-007 Gene Therapy for the Treatment of RDEB)
  18. Fortress Biotech/Cellvation (CEVA101, traumatic brain injury)
  19. Humacyte (Vascular Access for Hemodialysis)
  20. Iovance (Lifileucel for metastatic melanoma)
  21. jCyte (Retinitis Pigmentosa)
  22. Immunicum AB (Ilixadencel in Kidney Cancer)
  23. Juno/Celgene (JCAR017; CAR-T)
  24. Kiadis Pharma (ATIR101, blood disorders)
  25. Krystal Biotech (Gene therapy KB103 for skin disease)
  26. Magenta (MGTA-456 for Inherited Metabolic Disorders)
  27. Mallinckrodt/Stratatech’s Stratagraft
  28. Mesoblast (Heart Failure, mesenchymal precursor cell therapy)
  29. MiMedx Group (AmnioFix for osteoarthritis)
  30. Mustang Bio/St. Judes (MB-107 lentiviral gene therapy for X-linked Severe Combined Immunodeficiency (XSCID))
  31. NightStar Therapeutics (NSR-REP1 for choroideremia)
  32. Orchard Therapeutics (OTL-103 for the Treatment of Wiskott-Aldrich Syndrome)
  33. Poseida Therapeutics (P-BCMA-101 for multiple myeloma)
  34. Rocket Pharma (RP-L102 for Fanconi Anemia)
  35. SanBio (SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury)
  36. Sangamo Therapeutics (SB-525 for Hemophilia A.)
  37. Talaris (FCR-001 for prevention of renal transplantation rejection)
  38. Tessa (CAR-T for Hodgkin Lymphoma)
  39. TissueTech (cryopreserved human umbilical cord investigational biologic product TTAX02 for spina bifida in-utero.)
  40. Vericel (Ixmyelocel-T for cardiovascular disease)
  41. ViroMed (VM202 is a DNA based therapy for ALS)
  42. Voyager Therapeutics (VY-AADC for the Treatment of Parkinson’s Disease)