2024 Breakthrough: 9 New Cell and Gene Therapies Approvals

2024 was a remarkable year for cell and gene therapies (CGT) with the FDA approving nine innovative products. Read more below about these approved CGT therapies and new indications.

FIG 1. CGT Approved Products in 2024

RYONCIL - Mesoblast Limited (MESO)

➯ 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 

➯ Ph3 trial: 70% achieved overall response by Day 28 of treatment

SYMVESS - Humacyte (HUMA)

➯ 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 (Private)

➯ 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 (PTCT)

➯ 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 - Autolus Therapeutics (AUTL)

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

➯ the 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 (ADAP)

⇨ 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 (PFE)

⇨ 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 - Orchard Therapeutics (a  Kyowa Kirin company)

⇨ 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 - Iovance Biotherapeutics (IOVA)

⇨ 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?

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Article History:

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

This article is not investment, tax, or legal advice. Please do your own diligence and seek advice from professional advisors representing your interests.