BSc in Advanced Therapeutic Technologies Webinar

Advanced therapeutic technologies—principally gene therapies, cell therapies, and other advanced therapy medicinal products (ATMPs)—have moved decisively from experimental promise to clinical and commercial reality over the past 18 months.

In 2025, regulators approved a notable wave of new treatments. The US Food and Drug Administration (FDA) authorised six ATMPs, while the European Medicines Agency (EMA) approved four. Among the standouts was Fondazione Telethon’s etuvetidigene autotemcel (Waskyra), the first gene therapy approved for Wiskott-Aldrich syndrome—a rare, life-threatening immune disorder—and the first such approval by a non-profit organisation. Separately, Mesoblast’s Ryoncil became commercially available in the United States in March 2025 after late-2024 FDA approval; it is the first mesenchymal stromal cell therapy cleared for any indication, treating steroid-refractory acute graft-versus-host disease in children and adolescents, a complication carrying high mortality risk.

By 2025, twenty of the thirty largest biopharma companies by market capitalisation had active investments in developing and commercialising ATMPs. Financing for advanced molecular therapy start-ups accelerated sharply in the final quarter of 2025, with fourteen deals totalling $557 million—a 141% increase in value over the previous quarter.

Early 2026 has sustained the momentum. Industry discussions now centre on in vivo gene therapies as the next major wave, with growing big-pharma confidence shifting the question from feasibility to scalable manufacturing, point-of-care delivery, and hospital infrastructure for complex treatments. Regulatory developments are facilitating progress: the FDA moved to publish Complete Response Letters in real time for greater transparency in cell, gene, and RNA therapies, while the International Council for Harmonisation’s Cell and Gene Therapy Discussion Group released recommendations in December 2025 for future ATMP guidelines to address harmonisation needs.

Persistent challenges—manufacturing scale-up, cost, and consistency—persist, but innovations in artificial intelligence, dynamic culture systems, organoids, and biobanking are improving production precision and reliability. In Europe, proposed Biotech Act amendments seek to ease environmental risk assessments for low-risk genetically modified ATMPs and enable more adaptive definitions.

The stakes are high. These therapies offer potential one-time cures for rare genetic diseases, selected cancers, autoimmune conditions, and emerging applications in neurodegeneration, shifting patients from chronic management to disease modification. Expanding access hinges on resolving supply-chain and delivery barriers. Ireland’s established role as a global pharmaceutical hub amplifies the need for specialised talent to support this rapidly expanding sector.