The latest figures on the gene and cell therapy market released by the Alliance for Regenerative Medicine for the fourth quarter of 2025 show a clear lead for the United States over Europe, both in the number of clinical trials (916 vs 337) and in total investments ($2.5 billion vs $0.6 billion), despite a nearly identical number of developers (703 vs 685).
On the regulatory front, the new “plausible mechanism pathway” announced by the FDA at the end of last year is designed to accelerate access to personalized gene editing therapies and other treatments targeting unmet medical needs. Also in the United States, Baby KJ became the first child to receive an in vivo gene editing treatment for the ultra-rare CPS1 deficiency—a condition caused by the lack of the carbamoyl phosphate synthetase 1 enzyme, leading to toxic ammonia accumulation in the body. The CRISPR-based therapy was developed in just six months from diagnosis, with FDA authorization granted in only one week.
In Europe, meanwhile, the publication of the Biotech Act by the European Commission on December 16, 2025, is being seen as a turning point for the biotech medicines sector. The initiative combines financial incentives with regulatory improvements aimed at supporting the development of gene and cell therapies.
European pipelines lag behind
The report also highlights a robust U.S. pipeline, with at least twenty gene and cell therapy products already approved (three in 2026) or nearing regulatory submission.
By contrast, Europe’s clinical pipeline remains significantly smaller, with only five products approaching or having reached approval in 2026. Among them is the gene therapy developed by Fondazione Telethon for Wiskott-Aldrich syndrome, approved by the European Commission on January 16, 2026. The therapy had also received FDA approval in December 2025 and marks a milestone as the first case worldwide of a gene therapy for a rare genetic disease fully developed by a non-profit organization. Two additional approvals are expected in Europe this year, involving a cell therapy for metastatic melanoma and a gene therapy for retinitis pigmentosa.
On the corporate front, the quarter was marked by Bristol Myers Squibb’s $1.5 billion acquisition of Orbital Therapeutics, alongside several new strategic partnerships. Notably, Italy’s Chiesi has entered into a collaboration with Arbor Biotechnologies focused on genome editing for the treatment of primary hyperoxaluria type 1, with an upfront commitment of $115 million from Chiesi.
