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2024 Breakthrough: 9 New Cell and Gene Therapies Approvals

Jan 233 min read

We've come a long way since the first monoclonal antibody, Muromonab-CD3 (OKT3) was approved in 1986. It took more than 30 years after that to have our 1st approval of a cell-based gene therapy, Kymriah (2017). Since then, a growing number of new cell and gene therapies have been approved. In 2024, new Cell and gene therapies (CGT) continued to make there way to the market, with the FDA approving nine innovative products in 2024 (FIG. 1). These therapies include stem cell therapies, gene therapies, and CAR-T therapies. Five of the 9 approvals occurred in the last 2 months of the year. Read more below about these approved CGT therapies and their indications.


FIG 1. CGT Approved Products in 2024.



  • RYONCIL (remestemcel-L) is the first FDA-approved mesenchymal stromal cell (MSC) therapy.

  • Indicated for children aged 2 months & older including adolescents & teenagers with steroid-refractory acute graft versus host disease (SR-aGvHD).

  • Allogeneic bone marrow-derived. 

  • Phase 3 trial: 70% achieved overall response by Day 28 of treatment.



SYMVESS - Humacyte

  • SYMVESS (acellular tissue engineered vessel-tyod) a bioengineered human tissue.

  • Approved for use in adults as a vascular conduit for extremity arterial injury to prevent limb loss when autologous vein grafts are not feasible.

  • First-in-class bioengineered human tissue designed to be a universally implantable vascular conduit.

  • Demonstrated high patency rates (blood flow) and low rates of amputation and infection in clinical trials.



REGENECYTE - StemCyte

  • REGENECYTE, a cord blood stem cell therapy

  • First commercial biotech company to secure a biologics license for cord blood stem cell therapy.

  • Approved for unrelated donor hematopoietic progenitor cell transplantation in patients with disorders affecting the hematopoietic system.

  • Company advancing Phase II clinical trials for REGENECYTE™ in long COVID syndrome and acute stroke.



KEBILIDI - PTC Therapeutics

  • KEBILIDI (eladocagene exuparvovec-tneq) for AADC deficiency.

  • First ever approval for gene therapy directly administered to the brain. 

  • rAAV2-based gene therapy. 

  • Broad label including children and adults. 



  • AUCATZYL® (obecabtagene autoleucel) indicated for adult B-cell acute lymphoblastic leukemia (r/r B-ALL)

  • First CAR-T therapy approved with no required REMS program 

  • Approval based on FELIX trial in adult patients with r/r B-ALL:

    • 63% overall complete remission (OCR)

    • 42% complete remission within 3 months

    • Median duration of remission: 14.1 months



TECELRA - Adaptimmune

  • TECELRA (afamitresgene autoleucel) is indicated for adults with advanced synovial sarcoma who have undergone prior chemo & meet specific genetic criteria (HLA and MAGE-A4)

  • First approved engineered cell therapy for solid tumor  

  • Accelerated approval: based on initial response rates and further studies required to confirm long-term benefits



BEQVEZ - Pfizer

  • BEQVEZ (fidanacogene elaparvovec-dzkt), one-time gene therapy for hemophilia B.

  • Indicated for adults with moderate to severe hemophilia B on FIX prophylaxis or with severe bleeding history, lacking AAVRh74var antibodies.

  • Enables patients to produce their own FIX, eliminating the need for frequent infusions.

  • Reduces bleeding episodes and significantly decreases the treatment burden vs. standard care.



  • Lenmeldy (atidarsagene autotemcel), a hematopoietic stem cell gene therapy. 

  • The only therapy for children with early-onset metachromatic leukodystrophy (MLD) in the U.S. 

  • Lenmeldy known as Libmeldy in EU.



  • AMTAGVI™ (lifileucel) is the first FDA-approved T cell therapy for a solid tumor cancer. 

  • First treatment option for advanced melanoma after anti-PD-1 and targeted therapy. 

  •  Approved under accelerated approval. 



What do you think was the most exciting approval in the cell and gene therapies space in 2024?


And check out our free PDUFA calendar on biopharmIQ, to see upcoming FDA approval decision (PDUFA) deadlines. Want to schedule a demo of how to use our online platform to search for upcoming PDUFAs, clinical trial dates, combined with targeted sponsor info?


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Article History:

MV/AV/RF/DG (1/23/25)


Not legal, investing, or tax advice.


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