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A Complete List of All Publicly Announced RMAT Designations (158)

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To date, what number of U.S. FDA-approved RMATs have been issued and to whom have they been awarded?

BioLife Solutions

The answer is that 158 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced by biotech and pharma companies. However, the FDA states it has received 388 requests and issued 193, which means that a handful are not yet public knowledge. Therefore, a few companies are operating in stealth mode with regard to their RMAT designations and approximately half of RMAT applications get approved (193 approvals / 388 applications = 49.7%).

So far, Rocket Pharmaceuticals stands out as the only company to have secured five RMAT designations from the U.S. FDA. CRISPR Therapeutics follows closely with four RMATs. Four other companies—Allogene, AlloVir, Humacyte, and Mesoblast—have each received three RMATs to date.

Additionally, 16 companies have earned two RMAT designations each. These include: 4DMT, Abeona Therapeutics, Adaptimmune, Athersys, Atsena Therapeutics, Caribou Biosciences, CARsgen Therapeutics, Cook MyoSite, Fate Therapeutics, Intellia Therapeutics, Krystal Biotech, Orca Bio, Ocugen, Orchard Therapeutics, Poseida Therapeutics, and Sangamo Therapeutics.

RMAT Designations by the U.S. FDA

In this article:

List of RMAT Designations

Below is a comprehensive list of publicly announced RMAT designations:

  1. AbelZeta – C-CAR168 for the Treatment of Refractory SLE, Including LN (05/27/25)
  2. 4DMT – 4D-150, a gene therapy for the treatment of Diabetic Macular Edema (05/01/25)
  3. 4DMT – 4D-150, a gene therapy for the treatment of Intravitreal Treatment of Wet AMD (12/21/23)
  4. Abeona Therapeutics – EB-101 for treatment of Recessive RDEB (01/29/18)
  5. Abeona Therapeutics – ABO-102 for treatment of Sanfilippo Syndrome Type A (MPS IIIA) (04/23/18)
  6. AccurEdit Therapeutics – ART001, an in vivo gene editing therapy developed to treat transthyretin amyloidosis (ATTR) by reducing the production of the TTR protein in the liver, using LNP technology (05/29/25)
  7. Adaptimmune Therapeutics – ADP-A2M4 for the treatment of Synovial Sarcoma (12/03/19)
  8. Adaptimmune Therapeutics – ADP-A2M4CD8 for the treatment of patients with platinum resistant ovarian cancer (11/08/22)
  9. Adverum Biotechnologies, Inc. – Ixo-vec, its clinical-stage gene therapy product candidate, for the treatment of wet AMD (8/1/24)
  10. Affimed N.V. – A combination therapy of acimtamig and Artiva Biotherapeutics’ AlloNK® for treating relapsed/refractory Hodgkin Lymphoma (12/05/24)
  11. AffyImmune – AIC100, a CAR T-cell therapy, for the treatment anaplastic thyroid cancer (7/23/24)
  12. Allogene Therapeutics – ALLO-715 for the treatment of relapsed/refractory multiple myeloma (04/21/21)
  13. Allogene Therapeutics – ALLO-501A for the treatment of relapsed/refractory LBCL (06/08/22)
  14. Allogene Therapeutics – ALLO-316, an AlloCAR T™ Investigational Product for Adult Patients with Advanced or Metastatic Renal Cell Carcinoma (10/29/24)
  15. Allovir – Posoleucel (Viralym-M, ALVR105) for the treatment of BK Virus-associated Hemorrhagic Cystitis after HSC Transplant (06/11/19)
  16. Allovir – Posoleucel (Viralym-M, ALVR105) for the treatment of adenovirus (AdV) infections post-allogeneic stem cell transplantation (01/05/22)
  17. Allovir – Posoleucel (Viralym-M, ALVR105) for prevention of clinically significant infections and disease from six devastating viruses that commonly impactpatients following allogeneic hematopoietic cell transplant (04/20/22)
  18. Angiocrine Bioscience – AB205 for the treatment of organ vascular niche injuries for the prevention of severe toxicities in lymphoma patients (11/11/20)
  19. Ask Bio – AB-1005, its investigational gene therapy for the treatment of Parkinson’s disease (02/19/25)
  20. Asterias Biotherapeutics (Acquired by Lineage Cell Therapeutics) – AST-OPC1 for treatment of Spinal Cord Injury (SCI) (10/02/17)
  21. Athersys – MultiStem for the treatment of Ischemic Stroke (10/05/17)
  22. Athersys – MultiStem for the treatment of Acute Respiratory Distress Syndrome (ARDS) (09/23/20)
  23. Atsena Therapeutics – ATSN-101, an investigational gene therapy, for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (11/14/23)
  24. Atsena Therapeutics – ATSN-201, a gene therapy to treat X-linked Retinoschisis (04/17/25)
  25. Audentes Therapeutics – AT132 for the treatment of IX-linked Myotubular Myopathy (XLMTM) (08/21/18)
  26. Autolus Therapeutics – Obecabatagene autoleucel (obe-cel), a CD19-directed autologous CAR-T therapy, for the treatment of adult relapsed / refractory B-Acute Lymphocytic Leukemia (04/25/22)
  27. Aurion Biotech – AURN001, an allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease (06/19/24)
  28. Avobis Bio – AVB-114, clinical-stage implantable cell therapy, for the treatment of Crohn’s perianal fistulas (10/03/2025)
  29. AxoGen, Inc. – Avance® Nerve Graft for Peripheral Nerve Repair (10/29/18)
  30. Beam Therapeutics – BEAM-302 is a liver-targeting LNP therapy delivering a base editor and guide RNA to correct the mutation causing alpha-1 antitrypsin deficiency (AATD) (05/29/25).
  31. Beacon Therapeutics – laru-zova, a gene therapy for treating X-linked retinitis pigmentosa (XLRP), a severe, inherited retinal disease (01/28/25)
  32. BioMarin Pharmaceutical – Valoctocogene roxaparvovec, a gene therapy for adults with severe hemophilia A (03/08/21)
  33. Bionic Sight – BS01, a gene therapy for the treatment of retinitis pigmentosa (RP) patients with advanced stage vision loss (02/18/25)
  34. Biosolution, Co., Ltd. – CartiLife for autologous cartilage implantation (08/01/23)
  35. bluebird bio – Lyfgenia (lovo-cel) for the treatment of Severe Sickle Cell Disease (10/01/17)
  36. BlueRock Therapeutics, a subsidiary of Bayer – Bemdaneprocel, previously known as BRT-DA01, for the treatment of Parkinson’s disease (06/03/24)
  37. BridgeBio Pharma, Inc. – BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease (09/10/24)
  38. Bristol-Myers Squibb – Liso-cel, an autologous anti-CD19 CAR-T cell therapy for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) (12/18/19)
  39. Caladrius Biosciences – CLBS14 (CD34+ cell therapy program) for the treatment of Refractory Angina (10/01/17)
  40. Cabaletta Bio – Rese-cel in myositis, which is a disabling, multi-system autoimmune disease (05/15/25)
  41. Candel Therapeutics – CAN-2409 (aglatimagene besadenovec), targeting newly diagnosed, localized prostate cancer in intermediate-to-high-risk patients (05/29/2025)
  42. Capricor Therapeutics – CAP-1002 for the treatment of Duchenne Muscular Dystrophy (02/05/18)
  43. Caribou Biosciences – CB-010, an allogeneic anti-CD19 CAR-T cell therapy for relapsed or refractory large B cell lymphoma (11/29/22)
  44. Caribou Biosciences – CB-011, an allogeneic anti-BCMA CAR-T cell therapy for relapsed or refractory multiple myeloma (r/r MM) (03/31/26)
  45. CARsgen Therapeutics – CT053 (CAR-T therapy) for the treatment of Relapsed or Refractory Multiple Myeloma (10/28/19)
  46. CARsgen Therapeutics– CT041 for patients with advanced gastric or gastroesophageal junction adenocarcinoma (GC/GEJ) with Claudin18.2-positive tumor (01/10/22)
  47. Cartesian Therapeutics – Descartes-08, an autologous mRNA CAR-T directed against the B cell maturation antigen (BCMA), for the treatment of myasthenia gravis (05/22/24)
  48. Cellerant Therapeutics – Romyelocel-L for prevention of Infections During Neutropenia (07/02/18)
  49. Cellular Biomedicine Group Inc (CBMG) – C-CAR039 for use in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) (01/12/22).
  50. Cook Myosite – Iltamiocel (autologous muscle cells) for treatment of stress urinary incontinence (SUI) following surgical treatment (12/17/20)
  51. Cook Myosite – Iltamiocel for the treatment of adult females with chronic fecal incontinence (06/01/22)
  52. CRISPR Therapeutics – CTX001™ for the treatment of Severe Hemoglobinopathies (05/11/20)
  53. CRISPR Therapeutics – CTX110™, an allogeneic CAR-T cell therapy targeting CD19+ B-cell malignancies (11/22/21)
  54. CRISPR Therapeutics – CTX130™, an allogeneic CAR T cell therapy targeting CD70, for the treatment of Mycosis Fungoides and Sézary Syndrome (09/28/2022)
  55. CRISPR Therapeutics – CTX112 for treating relapsed or refractory CD19-positive B-cell malignancies (12/09/24)
  56. Deverra Therapeutics – DVX101 (Dilanubicel) for the treatment of Acute Myeloid Leukemia (03/19/24)
  57. Direct Biologics – Exoflo, an extracellular vesicle (EV) drug product, to treat Covid-19-linked acute respiratory distress syndrome (04/12/22)
  58. DiscGenics – Injectable Disc Cell Therapy (IDCT or rebonuputemcel), an injectable, allogeneic discogenic progenitor cell therapy for the treatment of symptomatic lumbar degenerative disc disease (DDD) (01/26/23)
  59. Doron Therapeutics – MOTYS™, its lead placental tissue-derived biologic for the treatment of symptoms associated with knee osteoarthritis (02/19/2025).
  60. Editas Medicine, Inc. – EDIT-301, an investigational, gene editing medicine for the treatment of severe sickle cell disease (10/16/23)
  61. Elixirgen Therapeutics – EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (02/20/25)
  62. Encoded Therapeutics – ETX101, an AAV9-based gene regulation therapy for the treatment of SCN1A+ Dravet syndrome (02/09/25)
  63. enGene – Detalimogene voraplasmid (also known as “detalimogene” and previously EG-70), an investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (06/25/25)
  64. Enzyvant – Rethymic (originally RVT-802) for the treatment of DiGeorge Syndrome (04/17/17)
  65. Eureka Therapeutics – ECT204, an ARTEMIS® CAR (AbTCR) T-cell therapy for advanced hepatocellular carcinoma (liver cancer) (01/12/2026)
  66. ExCellThera – ECT-001 for the treatment of Hematologic Malignancies (04/23/19)
  67. Fate Therapeutics – FT516, a gene-edited iPSC-derived NK cell therapy, for relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (12/13/21)
  68. Fate Therapeutics – FT819, an off-the-shelf, iPSC-derived CAR T cell therapy in Phase 1 development for the treatment of active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (04/14/25)
  69. Fibrocell – FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (05/29/19)
  70. Fortress Biotech (Subsidiary Cellvation Inc.) – EVA101 for the treatment of Traumatic Brain Injury (11/08/17)
  71. Galapagos NV – GLPG5101 for the treatment of Relapsed/Refractory Mantle Cell Lymphoma (08/06/25)
  72. Genascence – GNSC-001, a gene therapy that blocks interleukin 1 (IL-1) for the treatment of knee osteoarthritis (07/16/25)
  73. Grace Sciences – GS-100, an investigational AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency (04/15/26)
  74. Gradalis – Vigil® (Gemogenovatucel-T), a personalized immunotherapy for Advanced Ovarian Cancer (02/05/25)
  75. Helixmith Co., Ltd. (Previously ViroMed) – VM-202 for the treatment of Diabetic Peripheral Neuropathy (05/30/18)
  76. Hope Biosciences – Adipose-derived mesenchymal stem cells (HB-adMSCs) for treatment of patients living with relapsing-remitting multiple sclerosis (08/27/25)
  77. Humacyte – Humacyl for Vascular Access for Hemodialysis (03/20/17)
  78. Humacyte – Acellular Tissue Engineered Vessel (previously Human Acellular Vessel) for urgent arterial repair following extremity vascular trauma (05/04/23)
  79. Humacyte – Acellular Tissue Engineered Vessel (ATEV) for advanced peripheral artery disease (PAD) (07/01/24)
  80. IASO Biotherapeutics – BCMA CAR-T CT103A (Equecabtagene Autoleucel) for relapsed/refractory multiple myeloma (02/12/23)
  81. iECURE – ECUR-506, an in vivo targeted gene insertion therapy for the treatment of neonatal onset ornithine transcarbamylase (OTC) deficiency (01/07/26)
  82. Immatics N.V. – IMA203, a TCR-T cell therapy, for multiple relapsed and/or refractory HLA-A*02:01-positive and PRAME-expressing cancers, including cutaneous melanoma, uveal melanoma, endometrial carcinoma, synovial sarcoma, and ovarian cancer (10/24/23).
  83. Immix Biopharma (and its subsidiary Nexcella) – NXC-201, an investigational autologous BCMA-directed CAR-T therapyfor relapsed/refractory (r/r) light chain (AL) amyloidosis (02/10/25).
  84. Immunicum AB – Ilixadencel, activated allogeneic dendritic cells that act as an immune primer for the treatment of metastatic Renal Cell Carcinoma (05/06/20)
  85. Immunity Bio – ANKTIVA® and CAR-NK (PD-L1 t-haNK) for the reversal of Lymphopenia in patients receiving standard-of-care chemotherapy/radiotherapy and in relapsed locally advanced or metastatic pancreatic cancer (02/27/25).
  86. Intellia Therapeutics – NTLA-2002, an in vivo CRISPR-based investigational therapy, for the treatment of Hereditary Angioedema (03/21/23)
  87. Intellia Therapeutics – Nexiguran ziclumeran (nex-z or NTLA-2001), a CRISPR-based therapy for treating hereditary transthyretin amyloidosis with polyneuropathy (11/25/24)
  88. Iovance – Lifileucel for the treatment of Advanced Melanoma (10/11/18)
  89. iRegene – NouvNeu001, an iPSC-derived, allogeneic dopaminergic progenitor cell therapy to replace lost dopaminergic neurons, restore disrupted neural circuits, and enable dopamine production in Parkinson’s disease patients (01/19/26)
  90. jCyte – jCell for the treatment of Retinitis Pigmentosa (05/02/17)
  91. Juno Therapeutics (with Celgene Corp) – JCAR017 for the treatment of Lymphoma (large B cell NHL) (11/01/17)
  92. Kiadis Pharma – ATIR101 for the treatment of Leukemia (9/20/17)
  93. Krystal Biotech – KB103 for wound closure for RDEB Patients (06/24/19)
  94. Krystal Biotech – KB707, an immunotherapy candidate targeting non-small cell lung cancer (02/09/2026)
  95. Kyverna Therapeutics – KYV-101, an autologous, human CD19 CAR T-cell product candidate, for the treatment of patients suffering from refractory stiff-person syndrome (07/15/24)
  96. Lexeo Therapeutics – LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy (11/13/24)
  97. Longeveron – Lomecel-B™ for the treatment of Mild Alzheimer’s Disease (07/10/24)
  98. Luxa Biotechnology – RPESC-RPE-4W, an adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (02/18/25)
  99. Lyell Immunopharma – LYL314, a CAR T-cell therapy, for the treatment of relapsed or refractory (R/R) large B-cell lymphoma (04/15/25)
  100. Magenta Therapeutics – MGTA-456 for Multiple Inherited Metabolic Disorders, including Cerebral Adrenoleukodystrophy (09/04/19)
  101. Mallinckrodt Pharmaceuticals – Stratagraft for Thermal Burns (07/18/17)
  102. March Biosciences – MB-105, a first-in-class autologous CD5-targeted CAR-T cell therapy, in development for relapsed/refractory CD5-positive T-cell lymphoma (11/12/25).
  103. Medeor Therapeutics – MDR-101 to prevent kidney transplant rejection without chronic use of immunosuppressive drugs (09/22/20)
  104. MeiraGTx – AAV2-hAQP1 for the treatment of Grade 2/3 Radiation-Induced Xerostomia (12/09/24)
  105. Mesoblast – MPC-150-IM for Heart Failure (12/21/17)
  106. Mesoblast – Rexlemestrocel-L in the treatment of chronic low back pain (CLBP) associated with disc degeneration (02/08/23)
  107. Mesoblast – Rexlemestrocel-L (Revascor®) for the treatment of children with hypoplastic left heart syndrome (HLHS), a congenital heart condition (12/04/24)
  108. MiMedx Group – AmnioFix® Injectable for Knee Osteoarthritis (03/09/18)
  109. Mustang Bio (St. Judes)– MB-107 for X-linked Severe Combined Immunodeficiency (“Bubble Boy” Disease) (08/22/19)
  110. Myrtelle Inc. – rAAV-Olig001-ASPA for the treatment of Canavan disease (CD) (04/02/24)
  111. Nanoscope Therapeutics – MCO-010, a one-time, in-office therapy for the treatment of Stargardt disease (09/02/25)
  112. Nature Cell Co., Ltd. – JointStem for the treatment of severe degenerative arthritis (10/25/24)
  113. Neurogene – NGN-401, an investigational gene therapy for Rett Syndrome (08/07/24)
  114. Neurona Therapeutics – NRTX-1001 for the treatment of Focal Epilepsy (06/18/24)
  115. Nightstar Therapeutics – NSR-REP1 for Choroideremia (progressive vision loss) (06/14/18)
  116. Novadip – NVD003, a tissue regeneration product for the treatment of congenital pseudarthosis of the tibia (06/21/25)
  117. Novartis – Kymriah® for patients with relapsed or refractory (r/r) follicular lymphoma (04/22/20)
  118. Obsidian Therapeutics – OBX-115 for the treatment of patients with unresectable or metastatic melanoma that is resistant to immune checkpoint inhibitor (ICI) therapy (09/03/2024)
  119. Ocugen – NeoCart® for the repair of full-thickness lesions of the knee cartilage in adults (05/24/22)
  120. Ocugen – OCU400 for teatment of Retinitis Pigmentosa associated with RHO mutations (12/19/23)
  121. Opus Genetics – OPGx-LCA5, a gene therapy for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene (05/07/25)
  122. Orca Bio – Orca-T, an experimental allogeneic cell therapy for patients with blood cancers who are eligible for a hematopoietic stem cell transplant (10/15/20)
  123. Orca Bio – Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies (04/28/26)
  124. Orchard Therapeutics – OTL-103 for Wiskott-Aldrich Syndrome (07/29/19)
  125. Orchard Therapeutics – OTL-200 for Metachromatic Leukodystrophy (MLD) (01/14/21)
  126. Organogenesis – ReNu® for Knee Osteoarthritis (01/11/21)
  127. Pacira BioSciences – PCRX-201 for treatment of Osteoarthritis of the Knee (03/13/24)
  128. PolarityTE – SkinTE® for a chronic cutaneous ulcers and skin conditions (05/13/2022)
  129. Poseida Therapeutics – P-BCMA-101 for Relapsed/Refractory Multiple Myeloma (11/05/18)
  130. Poseida Therapeutics – P-BCMA-ALLO1, an investigational stem cell memory T cell (TSCM)-based allogeneic CAR-T cell therapy for patients with relapsed/refractory multiple myeloma (09/16/24).
  131. ProKidney – REACT® (Renal Autologous Cell Therapy) for slowing the progression of Chronic Kidney Disease (CKD) and improving kidney function (01/18/22)
  132. Ray Therapeutics – RTx-015 for Retinitis Pigmentosa (04/01/26)
  133. Regenxbio – RGX-121, an investigational gene therapy for Hunter syndrome (05/24/2023)
  134. Rocket Pharmaceuticals – RP-L102 for Fanconi Anemia (11/27/18)
  135. Rocket Pharmaceuticals – RP-L201, an investigational gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I) (03/09/21)
  136. Rocket Pharmaceuticals – RP-A501, the company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease (02/07/2023)
  137. Rocket Pharmaceuticals – RP-L301, gene therapy to treat pyruvate kinase deficiency (PKD) (05/23/2023)
  138. Rocket Pharmaceuticals – RP-A601, an investigational AAV-based gene therapy for the treatment of PKP2-arrhythmogenic cardiomyopathy (07/18/25)
  139. Rznomics – RZ-001, an investigational candidate for the treatment of hepatocellular carcinoma (05/08/26)
  140. SanBio – SB623 for Chronic motor deficits secondary to TBI (09/20/19)
  141. Sangamo Therapeutics – SB-525 (giroctocogene fitelparvovec) for Severe Hemophilia A (07/05/19)
  142. Sangamo Therapeutics – ST-920 ( isaralgagene civaparvovec) for Fabry disease (02/12/24)
  143. Senti Bio – SENTI-202 for adults with relapsed or refractory Acute Myeloid Leukemia (12/09/25)
  144. StemCyte – HPC Cord Blood Therapy for the treatment of Long COVID-19 (10/03/24)
  145. Takeda – Alofisel for the treatment of perianal fistulas (02/10/21)
  146. Talaris – FCR-001 for preventing renal transplantation rejection (04/18/19)
  147. Taysha Gene Therapies – TSHA-102 for the treatment of Rett Syndrome (05/02/24)
  148. Tessa Therapeutics – CD30-directed autologous CAR-T cell therapy for relapsed or refractory CD30-positive Hodgkin lymphoma (02/27/20)
  149. TissueTech, Inc. – Cryopreserved human umbilical cord product, TTAX02 (04/16/20)
  150. TScan Therapeutics – Two T-cell receptor (TCR)-engineered T cell (TCR-T) therapy candidates, TSC-100 and TSC-101, for the treatment of hematologic malignancies (05/29/24)
  151. Ultragenyx Pharmaceutical – UX11, an AAV gene therapy proposed for the treatment of Sanfilippo syndrome type A (05/30/24)
  152. uniQure – Investigational Gene Therapy AMT-130 for the treatment of Huntington’s Disease (06/03/24)
  153. United Therapeutics – miroliverELAP®, an investigational external liver assist product for the treatment of acute liver failure (04/08/26)
  154. University of California San Francisco (UCSF) – Lentiviral gene therapy for Artemis-SCID (02/14/2022)
  155. VeonGen Therapeutics – VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies (08/21/25)
  156. Vericel Corporation – Ixmyelocel-T for Dilated Cardiomyopathy (05/10/17)
  157. Voyager Therapeutics – VY-AADC for Advanced Parkinson’s disease (06/21/18)
  158. Wugen – WU-CART-007 for the treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) (05/20/2024)

Total RMATs Received and Awarded by FDA

To date, the U.S. FDA has received at least 388 requests for RMAT designations and granted 193.

The table below presents the cumulative number of RMAT requests that the FDA has received by year.

YEAR # REQUESTS GRANTED DENIED WITHDRAWN
2017 31 11 18 2
2018 47 18 27 2
2019 37 17 18 2
2020 34 13 21 0
2021 24 9 13 2
2022 30 14 15 1
2023 35 17 18 0
2024 59 43 14 2
2025 91 50 22 3
TOTAL 388 193 166 14

*Source: U.S. FDA 

What is an RMAT?

RMAT is an acronym that stands for “Regenerative Medicine Advanced Therapy.” The designation was introduced in December 2016 as part of the 21st Century Cures Act.

Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs. An RMAT submission request must be made in conjunction with an Investigational New Drug application (IND), either concurrently or as an amendment to an existing IND.

Products eligible for an RMAT designation include:

  • Cell therapies
  • Gene therapies
  • Therapeutic tissue engineering products
  • Human cell and tissue products
  • Combination products using such therapies or products

RMAT Definition

By definition, an RMAT is an award from the U.S. FDA that allows for faster, more streamlined approvals of regenerative medicine products within the United States, such as cell and gene therapies, tissue engineering products, and combination products. RMAT designations make innovative products eligible for quicker development and review of a marketing application.

An RMAT designation is similar to the breakthrough therapy designation that is available to drugs.

Benefits of an RMAT include increased opportunities to meet with FDA officials, as well as early meetings to discuss potential surrogate or intermediate endpoints.

21st Century Cures Act

The 21st Century Cures Act was passed by the 114th U.S. Congress in December 2016. The act authorize $6.3 billion in funding, most of which is allocated for the National Institutes of Health (NIH).

The bill, passed by both houses of Congress and signed into law by President Obama, puts the U.S. on the same playing field as other countries that support accelerated of innovative medicines. Other regions that have accelerated pathways include Japan, South Korea, and the EU. The bill encourages late-stage development in regenerative medicine, recognizing the field’s potential to address severe unmet medical needs.

Congress included several provisions related to cell and gene therapies in the 21st Century Cures Act, the most important of which is the RMAT designation.

RMAT Reviews by The FDA

For more detail, the following is from the U.S. FDA:

As described in Section 3033 of the 21st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:

  1. The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
  2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  3. Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.
Who do you expect to appear next on this RMAT list? Share your guesses in the comments below.
2.5/5 - (786 votes)

About Cade Hildreth, Founder & President

Cade Hildreth is the Founder of BioInformant.com, the world's largest publisher of stem cell industry news. Cade is a media expert on stem cells, recently interviewed by the Wall Street Journal, Los Angeles Business Journal, and Vogue Magazine. 

Comments

  3. S Dalvi says

    Cade this is impressive and useful information for the industry and those scientist who want to bring products to market legally.
    Thank you.

    Reply

  8. richard deutsch says

    i just saw OpRegen announced by Roche for its license with lineage cell therapeutics september 30 2024?

    Reply

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