Cell therapy weekly: UK MHRA launches gene therapy consultation
This week: The UK’s Medicines and Healthcare products Regulatory Agency (MHRA; London, UK) has launched a nationwide consultation to modernize gene therapy regulation, OrganaBio LLC (FL, USA) has acquired Excellos Inc.’s (CA, USA) operating assets and Fractyl Health, Inc. (MA, USA) has received Clinical Trial Application authorization in the Netherlands to initiate a Phase I/II study of a gene therapy for type 2 diabetes.
The news highlights:
- UK MHRA launches gene therapy consultation
- Formation of a comprehensive cell therapy service provider
- First gene therapy to enter clinical development for type 2 diabetes
UK MHRA launches gene therapy consultation
In partnership with Northern Ireland’s Department of Health (Belfast, Northern Ireland), the UK MHRA has launched a consultation to update the legal definition of gene therapy medicinal products, which has remained unchanged for over a decade.
The proposed changes aim to modernize the definition to reflect recent advances in gene therapy, synthetic biology and gene editing by classifying products based on mechanism of action rather than outdated criteria. The update will not alter approval processes, affect licensed products or change safety standards, but it will provide greater regulatory clarity, ensure consistent regulation across biologically and synthetically created therapies, and support innovation.
“This proposal reflects how fast-paced scientific progress can be and ensures our regulatory framework remains robust, clear and fit for the future,” commented MHRA Deputy Director Innovative Medicines Jon Beaman. “Our teams have carefully designed these updates to ensure that patient safety remains at the center of any change, while also giving developers, researchers and clinicians greater regulatory certainty, while supporting innovation across the industry.”
The consultation, running from 11 May–22 June 2026, primarily targets developers, manufacturers, researchers, regulatory professionals and clinical experts, though anyone may respond. It seeks feedback on five key proposals:
- Removing the requirement for gene therapies to be biological in origin
- Clarifying when synthetic or recombinant nucleic acids fall within scope
- Ensuring sequence-specific genome-editing products are regulated as GTMPs
- Maintaining the exclusion of infectious disease vaccines from GTMP definitions
- Proposing updates to the Human Medicines Regulations 2012 to support these changes
Formation of a comprehensive cell therapy service provider
OrganaBio has acquired substantially all the operating assets of Excellos, creating a bicoastal cell therapy contract development and manufacturing organization that combines complementary capabilities and expands geographic coverage to deliver more comprehensive cell therapy services. The newly formed Excellos Labs, LLC will oversee San Diego operations going forward.
“This acquisition is the best of both worlds, ensuring continuity for Excellos’ customers as we move into our next phase while adding critical redundancy, expanded geographical reach, and access to additional services and material including cord blood,” explained Tom VanCott, CEO of Excellos.
First gene therapy to enter clinical development for type 2 diabetes
Fractyl Health, Inc. has received Clinical Trial Application authorization in the Netherlands to begin the Phase I/II first-in-human study of RJVA-001, marking what the company believes is the first adeno-associated virus-based gene therapy to enter clinical development for type 2 diabetes.
RJVA-001 is a one-time, beta-cell–targeted gene therapy designed to enable nutrient-responsive GLP-1 expression within the pancreas through a minimally invasive endoscopic infusion, potentially avoiding the high drug levels and side effects associated with systemic GLP-1 therapy.
“For decades, we have managed [type 2 diabetes] as a chronic, progressive disease that inevitably worsens over time. With this authorization, we are preparing to test, for the first time in humans, whether a one-time, pancreas-targeted gene therapy delivered via a routine endoscopic procedure could provide durable metabolic control by enabling physiologic, nutrient-responsive GLP-1 expression at the source of disease,” elaborated Jacques Bergman, Principal Investigator of the RJVA-001 first-in-human study. “Patients who remain inadequately controlled despite maximally tolerated GLP-1 receptor agonists (GLP-1RAs) and multiple oral agents represent a population with significant unmet need. If successful, RJVA-001 could transform how we think about [type 2 diabetes], from a chronic disease you manage every day to one that could potentially be treated once.”
The open-label, multicenter study will evaluate safety, tolerability and efficacy in adults across three escalating dose cohorts (three participants in each cohort), with an optional expansion cohort of up to 20 additional participants. Participants will undergo 12-month monitoring for safety, glucose control, immune response and GLP-1 expression, followed by long-term follow-up for up to 5 years.
