Global Regulatory Report

Developed by ISCT in partnership with Clarivate, the Global Regulatory Report provides an overview of the cell and gene therapy regulatory landscape, including pipeline, late-stage (Phase III and pre-registration), approved products, covering cell, genetically modified cell and gene therapies.

FIFTH GLOBAL REGULATORY REPORT: 2025

Highlights:

  • 322 active clinical trials are represented in the 2025 global pipeline, with activity concentrated in early‑stage development (Phase I–II), reflecting sustained R&D momentum.
  • Five‑year regulatory approval trends show consistent activity across regions, spanning both genetically modified and non‑genetically modified cell therapy products.
  • Eight products received first‑ever global approval in 2025:
    • BBM‑H901 (dalnacogene ponparvovec)
    • Encelto™ (revakinagene taroretcel)
    • Papzimeos™ (zopapogene imadenovec)
    • Pulidekai® (puzolcabtagene autoleucel)
    • Qartemi® (varnimcabtagene autoleucel)
    • Ruibosheng (amimestrocel)
    • Waskyra™ (etuvetidigene autotemcel)
    • Zevaskyn® (prademagene zamikeracel)
  • Major global regulatory and standards updates were issued by key authorities, including the British Pharmacopoeia, EMA, European Pharmacopoeia, FACT‑JACIE, Health Canada, HSA, ICH, MHRA, TGA, and the US FDA.

Report Coming Soon.

FOURTH GLOBAL REGULATORY REPORT: 2024 H2

Highlights:

  • 3063 therapies in the global pipeline (+7.5% increase from H1 2024)
  • 91 products are under regulatory review globally (+7.1% increase from H1 2024) of which 13 products are in the pre-registration phase and 78 products are in Phase III clinical trials (+1.3% increase from H1 2024)
  • 106 products approved globally of which 4 products received their first approvals:
    • RegeneCyte (HPC, Cord Blood)
    • Akuugo (vandefitemcel)
    • Tecelra (afamitresgene autoleucel)
    • Aucatzyl (obecabtagene autoleucel)
    • Ryoncil (remestemcel-L-rknd)
  • Key Global Legislative/Framework Changes by British Pharmacopoeia, EMA, FACT-JACIE, Health Canada, ICH, Medsafe, OGTR, US FDA

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THIRD GLOBAL REGULATORY REPORT: 2024 H1

Highlights:

  • 2848 therapies in the global pipeline (–3.8% decrease from H2 2023)
  • 85 products are under regulatory review globally of which 8 products are in pre-registration phase and 77 products are in Phase III clinical trials
  • 99 products approved globally (–50% decrease from H2 2023) of which 3 producs received their first approvals:
    • Amtagvi (Lifileucel)
    • CT-053 (Zevorcabtagene Autoleucel)
    • Beqvez (fidanacogene elaparvovec)
  • Key Global Legislative/Framework Changes by British Pharmacopoeia, EMA, ICH, NIH,
    and US FDA

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Highlights:

  • 2960 therapies in the global pipeline (2% increase from H1 2023)
  • 80 products are under regulatory review globally of which 71 products are in Phase III clinical trials
  • 96 products approved globally (9% increase from H1 2023)
  • 6 products received their first approvals globally including.
    • Elevydis (delandistrogene moxeparvovec-rokl)
    • Lantidra (donislecel)
    • Fucaso® (equecabtagene autoleucel)
    • CNCT19 (inaticabtagene autoleucel)
    • Casgevy™ (exagamglogene autotemcel)
    • Lyfgenia™ (lovotibeglogene autotemcel)
  • Key Global Legislative/Framework Changes by US FDA,  EMA, ICH, and NZ Ministry of Environment

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Highlights:

  • 87 cell and gene therapies approved globally, and 71% of them are non-genetically modified cell products.
  • 81 cell and gene therapies under the late development stages (Phase III or pre-registration)
  • Key Global Legislative/Framework changes by EU Commission, US FDA, Health Canada, TGA, NZ Ministry of Health, WHO
  • Carvykti®, received TGA Approval for the treatment of people with multiple Myeloma
  • Vyjuvek™, received FDA BLA Approval for the treatment of wounds in patients with Dystrophic Epidermolysis Bullosa

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The International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists, and industry partners with a shared vision to translate cell and gene therapy into safe and effective therapies to improve patients' lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory, and Commercialization. Through strong relationships with global regulatory agencies, academic institutions, and industry partners, ISCT drives the advancement of research into a standard of care.

Comprised of over 4,500 cell and gene therapy experts across five geographic regions and representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders, and organizations invested in cell and gene therapy translation. For more information about the organization, visit ISCT.

Clarivate™ is a leading global provider of transformative intelligence offering enriched data, insights & analytics, workflow solutions and expert services in the areas of Academia & Government, Intellectual Property and Life Sciences & Healthcare. Visit Clarivate.