RMAT– tag –
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FDA Approves Orca-T, First Regulatory T-Cell Therapy to Improve Chronic GVHD-Free Survival in Blood Cancer Patients Undergoing Allogeneic HSCT
American Society of Clinical Oncology (ASCO) Post USA Overview The US FDA has approved Orca-T (Tregzi, Orca Bio), the first regulatory T-cell (Treg)-based immunotherapy to improve chronic graft-versus-host disease (GVHD)-free survival in... -
FDA RMAT Designations Hit Record 48 in 2025, Signaling CGT Pipeline Maturity and Market Acceleration
Pharmaceutical Technology UK Overview FDA Regenerative Medicine Advanced Therapy (RMAT) designations surged from 2024, reaching a record 48 in 2025, according to Pharmaceutical Technology analysis. This unprecedented increase reflects th... -
FDA RMAT Designations Reach 162 Total: Rocket Pharmaceuticals and CRISPR Therapeutics Lead
BioInformant USA Overview The FDA's publicly announced Regenerative Medicine Advanced Therapy (RMAT) designations have reached a total of 162, with approximately half of all RMAT applications being approved, according to BioInformant dat... -
CRISPR’s First Clinical Evidence for HBV cccDNA Elimination, Prime Editing Advances to Inaugural Human Trial
CRISPR Medicine News USA Overview Precision Biosciences' PBGENE-HBV has demonstrated the first clinical evidence of eliminating viral cccDNA in chronic Hepatitis B patients, marking a significant milestone for CRISPR-based therapies in i... -
Top 10 Emerging Healthcare Technologies in 2026: CRISPR Gene Editing Established as Therapeutic Category, Regenerative Medicine Nearing Clinical Application
Slate USA Overview An article on 10 emerging healthcare technologies to watch in 2026 highlights that CRISPR gene editing is moving beyond the lab to become a bona fide therapeutic category. Additionally, regenerative medicine and stem c... -
Prime Medicine’s Autologous Prime Editing Hematopoietic Stem Cell Therapy PM359 for p47phox-Deficient Chronic Granulomatous Disease Receives FDA RMAT Designation
BiopharmaWatch (Prime Medicine Press Release) USA Overview Prime Medicine announced its investigational autologous prime editing hematopoietic stem cell therapy, PM359, for the treatment of p47phox-deficient Chronic Granulomatous Disease... -
Qihan Biotech’s Universal Dual-Target Hypoimmune CAR-T Therapy QT-019B Receives FDA RMAT and Breakthrough Therapy Designations
PackGene Biotech China Overview Qihan Biotech's universal dual-target CAR-T therapy, QT-019B, has received both FDA Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designations (BTD). This off-the-shelf allogeneic ... -
uniQure Announces Plan for BLA Submission of Huntington’s Disease Gene Therapy AMT-130, FDA Accepts Phase 1/2 3-Year Data for Accelerated Approval
uniQure Press Release USA Overview uniQure has announced that the FDA agreed in a Type B meeting that 3-year analysis data from its Phase 1/2 trial of gene therapy AMT-130 for Huntington's Disease (HD) would be acceptable as primary evid... -
FDA Grants RMAT Designation to Cellectis’ Allogeneic CD22 CAR-T Lasme-cel for Relapsed/Refractory B-ALL
Targeted Oncology USA Overview The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Cellectis' lasmecabtagene timgedleucel (lasme-cel; UCART22), an allogeneic CD22-targeting CAR-T cell therapy, for relapsed/re... -
FDA Expedited Pathway Guide: Strategic Selection of Fast Track, Breakthrough Therapy, and RMAT
Boyd Consultants USA Overview The FDA offers several expedited programs to accelerate innovative drug development, including Fast Track, Breakthrough Therapy, and Regenerative Medicine Advanced Therapy (RMAT). This article outlines the d...
