Cell & Gene Therapy Tracker
BIOPHARMAWIRE | RESOURCE LIBRARY
Cell & Gene Therapy Tracker
Global progress in CGT development and approvals — approved products and key late-stage pipeline programs
Updated Q4 2025 | BioPharmaWire Editors | Sources: FDA, EMA, company disclosures
Cell and gene therapy has moved from experimental science to established medicine faster than almost any other drug class in history. As of late 2025, more than 20 CGT products have received regulatory approval in the US or EU, with dozens more in pivotal trials. This tracker covers approved products and selected late-stage pipeline programs across four modalities: CAR-T and engineered cell therapies, in vivo gene therapies, gene editing, and TCR-T therapies. It is updated quarterly.
Key Themes Heading Into 2026
- CRISPR moves to standard of care. Casgevy’s approval for sickle cell disease and beta-thalassaemia marks the first CRISPR therapy to reach patients. Confirmatory data and real-world evidence will define whether gene editing becomes the preferred curative approach over gene addition.
- Manufacturing remains the binding constraint. Autologous CAR-T timelines of 4-6 weeks and seven-figure price tags continue to limit patient access. Allogeneic (off-the-shelf) cell therapy programs in Phase 2/3 are the most closely watched development in the field.
- Solid tumors are the next frontier. Nearly all approved CGTs target blood cancers. TCR-T and next-generation CAR-T programs targeting solid tumor antigens (MAGE-A4, PRAME, GD2) are the most active area of early-phase development.
- Pricing and reimbursement pressure intensifies. With single-treatment costs ranging from $375,000 to over $3.5 million, payer negotiations and outcomes-based contracts are becoming standard. The EU’s HTJA joint assessment process will add new complexity for multi-market launches from 2025 onward.
- In vivo gene editing enters pivotal trials. Intellia’s NTLA-2001 for transthyretin amyloidosis and several base editing programs are moving into Phase 3, representing a potential step-change from ex vivo approaches.
CGT Tracker: Approved Products & Late-Stage Pipeline
Status key: Approved BLA/MAA Filed Phase 3 Phase 1/2
Product | Company | Modality | Indication | Status | Market |
CAR-T & CELL THERAPIES | |||||
Kymriah (tisagenlecleucel) | Novartis | CAR-T (CD19) | B-cell ALL, DLBCL | Approved | US / EU |
Yescarta (axicabtagene) | Kite/Gilead | CAR-T (CD19) | DLBCL, FL | Approved | US / EU |
Breyanzi (lisocabtagene) | Bristol Myers Squibb | CAR-T (CD19) | DLBCL, CLL | Approved | US / EU |
Abecma (idecabtagene) | BMS / 2seventy | CAR-T (BCMA) | Multiple myeloma | Approved | US / EU |
Carvykti (ciltacabtagene) | J&J / Legend Biotech | CAR-T (BCMA) | Multiple myeloma | Approved | US / EU |
Aucatzyl (obecabtagene) | Autolus | CAR-T (CD19) | B-cell ALL | Approved | US |
Turoctocog alfa pegol | Allogene / Pfizer | AlloCAR-T (CD19) | B-cell NHL | Phase 3 | US / EU |
BMS-986353 | Bristol Myers Squibb | CAR-T (GPRC5D) | Multiple myeloma | Phase 2 | US |
CT103A | IASO Bio | CAR-T (BCMA) | Multiple myeloma | Phase 3 | China / US |
GENE THERAPIES (IN VIVO) | |||||
Zolgensma (onasemnogene) | Novartis | AAV9 gene therapy | Spinal muscular atrophy | Approved | US / EU |
Luxturna (voretigene) | Spark/Roche | AAV2 gene therapy | RPE65 retinal dystrophy | Approved | US / EU |
Hemgenix (etranacogene) | CSL Behring | AAV5 gene therapy | Haemophilia B | Approved | US / EU |
Roctavian (valoctocogene) | BioMarin | AAV5 gene therapy | Haemophilia A | Approved | US / EU |
Elevidys (delandistrogene) | Sarepta | AAV gene therapy | Duchenne MD | Approved | US |
Beqvez (fidanacogene) | Pfizer | AAV gene therapy | Haemophilia B | Approved | US |
SPK-8011 | Spark Therapeutics | AAV gene therapy | Haemophilia A | Phase 3 | US / EU |
AMT-061 next-gen | uniQure | AAV5 gene therapy | Huntington’s disease | Phase 2 | US / EU |
ST-920 | Sangamo / Sanofi | AAV gene therapy | Fabry disease | Phase 3 | US / EU |
GENE EDITING THERAPIES | |||||
Casgevy (exa-cel) | Vertex / CRISPR Tx | CRISPR-Cas9 | Sickle cell / Beta-thal | Approved | US / EU / UK |
Lyfgenia (lovotibeglogene) | bluebird bio | Lentiviral gene addition | Sickle cell disease | Approved | US |
NTLA-2001 | Intellia Therapeutics | CRISPR in vivo | Transthyretin amyloidosis | Phase 3 | US / EU |
EDIT-301 | Editas Medicine | CRISPR-AsCas12a | Sickle cell disease | Phase 1/2 | US |
ARO-HSD | Arrowhead / Intellia | Base editing | Liver disease | Phase 2 | US |
BEAM-101 | Beam Therapeutics | Base editing | Sickle cell disease | Phase 1/2 | US |
TCR-T & ENGINEERED CELL THERAPIES | |||||
Afami-cel (afamitresgene) | Adaptimmune | TCR-T (MAGE-A4) | Synovial sarcoma | Approved | US |
Lifileucel (lifileucel) | Iovance | TIL therapy | Melanoma | Approved | US |
IMA203 | Immatics | TCR-T (PRAME) | Solid tumors | Phase 2 | US / EU |
MC-0518 | Medigene | TCR-T (AFP) | Hepatocellular carcinoma | Phase 1/2 | EU |
This tracker is an editorial product of BioPharmaWire based on publicly available regulatory and company disclosures as of Q4 2025. It is not exhaustive. Pipeline status is subject to change. It does not constitute investment advice. For live coverage of CGT approvals and trial readouts, follow our News and Innovation sections.