Gene therapy expands beyond oncology — and brings new challenges

1. May 30, 2025

Gene therapies now account for most ATMP clinical trials, with a balanced distribution between in vivo and ex vivo approaches. Oncology remains dominant (38%), but there is increasing activity in metabolic (13%) and hematological (10%) areas.

Notably, in December 2023, the FDA approved Casgevy — the first CRISPR-based therapy — for sickle cell disease and beta-thalassemia. By Q3 2024, over half of newly initiated gene therapy trials targeted non-oncology indications.

Axcellant has extensive experience in this space, supporting clinical programs involving CAR-T cells, cancer vaccines, and stem cells. We help biotech teams manage the regulatory, logistical, and operational complexity behind these groundbreaking therapies.

Discover our capabilities in ATMP trial delivery.