Updated FDA granted RMAT List (129)

Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT.

Sometimes people call these “r mat” with a space.

I also include FDA data on RMAT submissions via an FDA RMAT resource page. At this point as of April 18, 2025, I have found 129 grant RMATs. 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 or company announcements.

FDA RMAT list, RMAT list
FDA table on data related to their review of regenerative medicine advanced therapy designation or RMAT applications. Screenshot from FDA site with data from 02/12/2025.

Note that the FDA has recently updated its RMAT totals to indicate that it has granted a total of 152 RMATs. You can see the interesting table above from the FDA about the number of total applications, those granted, and those denied or withdrawn. In every year more RMATs were denied or withdrawn than granted. I wonder how many applications were from stem cell clinics? The agency continues to deny a sizable number of applications.

RMAT list

  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. AdaptImmune (ADP-A2M4CD8 for the treatment of patients with platinum resistant ovarian cancer)
  5. Adverum Biotechnologies (Ixo-vec for the Treatment of Wet AMD.)
  6. Affimed RV (Acimtamig and Artiva’s AlloNK for Relapsed/Refractory Hodgkin Lymphoma Treatment)
  7. AffyImmune (AIC100 in Recurrent Anaplastic Thyroid Cancer)
  8. Allogene Therapeutics (ALLO-715, an AlloCAR T Cell Therapy for Relapsed/Refractory Multiple Myeloma
  9. Allogene Therapeutics (ALLO-501A for Large B Cell Lymphoma)
  10. Allogene Therapeutics (ALLO-316, an AlloCAR T for adults with advanced or metastatic kidney cancer)
  11. AlloVir (Viralym-M for hemorrhagic cystitis)
  12. AlloVir (Viralym-M for adenovirus infections after stem cell transplants.
  13. AlloVir (Viralym-M for viral infections more generally after transplants. This seems to overlap with the previously listed one).
  14. Angiocrine (AB205 Cell therapy for organ vascular niche injuries for the prevention of severe toxicities in lymphoma patients)
  15. Arcellx (CART-ddBCMA for refractory multiple myeloma)
  16. Ask Bio (AB-1005 for Parkinson’s)
  17. Athersys (MultiStem)
  18. Athersys (MuliStem for ARDS)
  19. Atsena Therapeutics  (ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)
  20. Audentes (AT132 for the Treatment of X-Linked Myotubular Myopathy)
  21. Aurion Biotech (AURN001 for corneal edema secondary to corneal endothelial disease)
  22. Autolus (obe-cel, for the treatment of adult B-ALL)
  23. AxoGen (Avance for nerve injuries)
  24. Beacon Therapeutics (laru-zova for  X-linked retinitis pigmentosa)
  25. BioMarin (Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A)
  26. Bionic Sight (BS01 for  retinitis pigmentosa)
  27. BlueBird Bio (Lentiglobin in SCID)
  28. BlueBird Bio (lovotibeglogene autotemcel (lovo-cel) for certain cases of sickle cell)
  29. BridgeBio Pharma, Inc. (BBP-812 for Canavan disease)
  30. Bristol-Myers Squibb (Liso-cel, CAR-T cell therapy for large B-cell lymphoma)
  31. Caladrius (CD34+ cell therapy for angina)
  32. Capricor (CAP1002, Duchenne Muscular Dystrophy)
  33. Caribou Bio (CB-010, an Allogeneic Anti-CD19 CAR-T Cell Therapy for LBCL)
  34. CARsgen (Zevor-cel or CT053 CAR-T therapy for Multiple Myeloma)
  35. CARsgen (CT041 CAR-T therapy for specific GI cancers)
  36. Cartesian Therapeutics (Descartes-08 for myasthenia gravis)
  37. Cellerant Therapeutics, Inc/Fibrocell. (Romyelocel-L to Prevent Infections During Neutropenia)
  38. Cellular Biomedicine Group or CBMG. C-CAR039 for relapsed or refractory diffuse large B cell lymphoma)
  39. Cook MyoSite (AMDC-USR for Urinary Sphincter Repair)
  40. Cook MyoSite (AMDC-USR for Urinary Sphincter Repair)
  41. CRISPR Therapeutics (CTX001 for hemoglobinopathies. This is with Vertex)
  42. CRISPR Therapeutics (CTX110 for specific B cell tumors)
  43. CRISPR Therapeutics (CTX130 for cutaneous T cell lymphomas)
  44. CRISPR Therapeutics  (CTX112 for treating relapsed or refractory CD19-positive B-cell malignancies)
  45. Deverra Therapeutics (DVX101 (Dilanubicel) for Acute Myeloid Leukemia)
  46. Direct Biologics (ExoFlo for COVID-19). This one is highly controversial in my view. There was political pressure on then Commissioner Steve Hahn to move this forward for Direct Biologics. My review of the key ExoFlo Phase I trial paper found many red flags too.
  47. DiscGenics (IDCT or rebonuputemcel for disc repair)
  48. Doron Therapeutics (MOTYS for knee osteoarthritis)
  49. Editas (EDIT-301 for severe sickle cell disease)
  50. Elixirgen Therapeutics (EXG-34217 for Telomere Biology Disorders)
  51. Enzyvant (DiGeorge syndrome)
  52. ExCellThera (ECT-001 for blood cancers)
  53. Fate Therapeutics (FT516 for Relapsed / Refractory DLBCL)

  54. Fate Therapeutics (FT819 to Treat Moderate to Severe Systemic Lupus Erythematosus (SLE))

  55. Fibrocell (FCX-007 Gene Therapy for the Treatment of RDEB)
  56. Fortress Biotech/Cellvation (CEVA101, traumatic brain injury)
  57. Gradalis (Vigil (Gemogenovatucel-T) for Advanced Ovarian Cancer)
  58. Helixmith (VM202 is a DNA based therapy for Diabetic Peripheral Neuropathy)
  59. Humacyte (Human Acellular Vessel (HAV) for urgent arterial repair following extremity vascular trauma)
  60. Humacyte (Vascular Access for Hemodialysis)
  61. Humacyte  (Acellular Tissue Engineered Vessel (ATEV) for advanced peripheral artery disease)
  62. IASO Biotherapeutics (BCMA CAR-T CT103A for relapsed/refractory multiple myeloma)
  63. Immatics (IMA203 GEN1 for multiple PRAME-expressing cancers, including cutaneous and uveal melanoma)
  64. Immix Biopharma (NXC-201 CAR-T for relapsed/refractory (r/r) light chain (AL) amyloidosis.
  65. Immunicum AB (Ilixadencel in Kidney Cancer)
  66. Immunity Bio (ANKTIVA and CAR-NK (PD-L1 t-haNK) for the reversal of lymphopenia in some cancer patients)
  67. Intellia Therapeutics (NTLA-2002 for angioedema)
  68. Intellia Therapeutics (Nexiguran ziclumeran (nex-z or NTLA-2001 for hereditary transthyretin amyloidosis with polyneuropathy)
  69. Intellia Therapeutics (Nexiguran ziclumeran, also known as nex-z or NTLA-2001 for the treatment of transthyretin or TTR amyloidosis with cardiomyopathy.
  70. Iovance (Lifileucel for metastatic melanoma)
  71. jCyte (Retinitis Pigmentosa)
  72. Juno/Celgene (JCAR017; CAR-T)
  73. Kiadis Pharma (ATIR101, blood disorders)
  74. Krystal Biotech (Gene therapy KB103 for skin disease)
  75. Kyverna Therapeutics (KYV-101 CAR-T for patients suffering from refractory stiff-person syndrome)
  76. Lexeo Therapeutics (LX2006 for Friedreich ataxia cardiomyopathy)
  77. Lineage Cell Therapeutics (OPC1, spinal cord injury)
  78. Lineage Cell Therapeutics (OpRegen, dry AMD)
  79. Longeveron (Lomecel-B for mild Alzheimer’s)
  80. Luxa Biotechnology (RPESC-RPE-4W for dry AMD)

  81. Lyell Immunopharma (LYL314 for the Treatment of Relapsed and/or Refractory Large B-Cell Lymphoma)

  82. Magenta (MGTA-456 for Inherited Metabolic Disorders)
  83. Mallinckrodt/Stratatech’s Stratagraft
  84. Medeor Therapeutics (MDR-101 for prevention of kidney transplant rejection)
  85. MeiraGTx (AAV2-hAQP1 for Grade 2/3 Radiation-Induced Xerostomia)
  86. Mesoblast (Heart Failure, mesenchymal precursor cell therapy Rexlemestrocel-L)
  87. Mesoblast (Back pain, Rexlemestrocel-L)
  88. Mesoblast (Rexlemestrocel-L (Revascor®) for pediatric hypoplastic left heart syndrome)
  89. MiMedx Group (AmnioFix for osteoarthritis)
  90. Mustang Bio/St. Judes (MB-107 lentiviral gene therapy for X-linked Severe Combined Immunodeficiency (XSCID))
  91. Myrtelle Inc. (rAAV-Olig001-ASPA for the treatment of Canavan disease)
  92. Nature Cell (JointStem for severe degenerative arthritis)
  93. Neurogene (NGN-401 for Rett Syndrome)
  94. Neurona Therapeutics (NRTX-1001 for the treatment of Focal Epilepsy). See more on Neurona
  95. NightStar Therapeutics (NSR-REP1 for choroideremia)
  96. Novartis (Kymriah for follicular lymphoma)
  97. Ocugen (NeoCart for generating new cartilage for OA)
  98. Ocugen (OCU400 for Retinitis Pigmentosa with RHO mutations)
  99. Orca Bio (Orca-T (NCT04013685 for patients with blood cancers eligible for HSCT)
  100. Orchard Therapeutics (OTL-103 for the Treatment of Wiskott-Aldrich Syndrome)
  101. Orchard Therapeutics (OTL-200 for Metachromatic Leukodystrophy)
  102. Organogenesis (ReNu candidate for osteoarthritis of the knee)
  103. Pacira BioSciences (PCRX-201 for knee osteoarthritis)
  104. Pfizer (Fidanacogene elaparvovec for hemophilia B)
  105. PolarityTE (SkinTE for cutaneous ulcers)
  106. Poseida Therapeutics (P-BCMA-101 for multiple myeloma)
  107. Poseida Therapeutics (P-BCMA-ALLO1 CAR-T cell therapy for relapsed/refractory multiple myeloma)
  108. ProKidney (REACT for serious kidney disease)
  109. Regenxbio (RGX-121 for Hunter syndrome). This is a gene therapy.
  110. Rocket Pharmaceuticals (RP-L102 for Fanconi Anemia)
  111. Rocket Pharmaceuticals (RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I)
  112. Rocket Pharmaceuticals (RP-L301 for pyruvate kinase deficiency)
  113. Rocket Pharmaceuticals (RP-A501 for Danon Disease)
  114. SanBio (SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury)
  115. Sangamo Therapeutics (SB-525 for Hemophilia A)
  116. Sangamo Therapeutics  (ST-920 ( isaralgagene civaparvovec) for Fabry disease)
  117. StemCyte (HPC Cord Blood for Long COVID-19) I don’t see a formal product name for this one
  118. Takeda (Darvadstrocel for Perianal fistulas)
  119. Talaris (FCR-001 for prevention of renal transplantation rejection)
  120. Taysha Gene Therapies (TSHA-102 for Rett Syndrome)
  121. Tessa (CAR-T for Hodgkin Lymphoma)
  122. TissueTech (cryopreserved human umbilical cord investigational biologic product TTAX02 for spina bifida in-utero.)
  123. TScan Therapeutics (TSC-100 and TSC-101 for hematologic malignancies) Is this one or two RMATs?
  124. UCSF (Artemis-SCID treatment: I don’t see a product name here)
  125. Ultragenyx Pharmaceutical (UX11 for Sanfilippo syndrome type A)
  126. uniQure (AMT-130 for Huntington’s)
  127. Vericel (Ixmyelocel-T for cardiovascular disease)
  128. Voyager Therapeutics (VY-AADC for the Treatment of Parkinson’s Disease)
  129. Wugen (WU-CART-007 for relapsed/refractory T-cell acute lymphoblastic leukemia and lymphoma)

References

  1. FDA RMAT page, current as of March 18, 2025
  2. FDA definition of RMATs.

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