Global innovation briefing · Updated 5 November 2026
Cell and gene therapy is advancing on several fronts at once: new genetic indications, regional approvals and stronger expectations for manufacturing control. For developers and health systems, the useful question is how each development changes the evidence and infrastructure needed to deliver treatment.
United States: new genetic indications
On 26 March 2026, the FDA granted accelerated approval to Kresladi for a defined pediatric population with severe leukocyte adhesion deficiency type I. The indication covers patients with biallelic ITGB2 variants who lack an available HLA-matched sibling donor. This is an indication-specific decision, with confirmatory evidence obligations attached to accelerated approval. [1]
On 23 April, FDA approval of Otarmeni introduced a dual-AAV gene therapy for genetically defined OTOF-associated hearing loss. Its eligibility conditions include preserved outer hair-cell function and no previous cochlear implant in the same ear. The development illustrates how genomic diagnosis and organ-specific assessment increasingly shape patient selection. [2]
Europe: another rare-disease milestone
Waskyra received an EU-wide marketing authorisation on 9 January 2026 for eligible patients with Wiskott–Aldrich syndrome. It uses the patient's genetically modified blood stem cells and requires specialist delivery. EMA describes long-term safety monitoring, including a 15-year study, as part of the evidence programme. [3]
Japan: a different regulatory model
Japan granted conditional, time-limited marketing approvals to the iPSC-derived products RiHEART and AMCHEPRY on 6 March 2026. These cover cardiac and Parkinson's-related applications respectively. Their status must be described accurately: further post-marketing confirmation is part of the Japanese pathway. [4]
The newest manufacturing signal
On 2 November 2026, the FDA issued final potency-assurance guidance for cell and gene therapies. It describes an integrated approach combining process and material controls, manufacturing-stage testing and release assays. The practical implication is that potency should be designed into development rather than left to a final laboratory result. [5]
What this means for GCC development
HERMS interpretation: build a product-by-product readiness map linking genetic eligibility, reference-country approval, local regulatory classification, treatment-centre capability, chain of identity and long-term follow-up. An overseas approval can inform planning; it does not itself authorise use in another jurisdiction. Compare manufacturing changes and clinical delivery requirements before selecting a localisation or technology-transfer strategy.
Sources and further reading
- FDA: Kresladi approval, 26 March 2026
- FDA: Otarmeni approval, 23 April 2026
- EMA: Waskyra assessment and authorisation
- AMED: Japan's iPSC therapy approvals
- FDA: final potency-assurance guidance, November 2026
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