Fractyl Health’s trial to evaluate a glucagon-like peptide-1 (GLP-1) gene therapy has been approved by European authorities.
The open-label, first-in-human, Phase I/II study will investigate RJVA-001 in patients with obesity and type 2 diabetes.
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The study has been granted Clinical Trial Application authorisation in the Netherlands. Fractyl also plans to conduct the study at sites in Australia, where a clinical trial application has been submitted and regulatory feedback is expected in Q3 2026. Fractyl is hoping to dose the first patient with RJVA-001 and report preliminary data in H2 2026.
Fractyl believes RJVA-001 is the first adeno-associated virus (AAV)-based gene therapy candidate to enter clinical development for type 2 diabetes.
Patients will receive a GLP-1 washout before receiving RJVA-001 delivered via endoscopic ultrasound-guided intrapancreatic infusion. Three escalating dose cohorts will be followed by an optional expansion cohort of up to 20 additional participants treated at the selected optimal dose.
Patients will be monitored for 12 months for safety, glucose control, immune response, and GLP-1 expression, and enrolled in a long-term follow-up study for up to five years.
Primary endpoints include safety and tolerability, while secondary endpoints assess preliminary efficacy through continuous glucose monitoring, including time-in-range and other glycaemic control measures.
RJVA-001 is a one-time, beta-cell–targeted gene therapy designed to enable nutrient-responsive, physiologic GLP-1 expression within the pancreas. RJVA-001 remains in preclinical development in the US, with no Investigational New Drug (IND) application having yet been filed with the US Food and Drug Administration (FDA).
Dr Harith Rajagopalan, CEO of Fractyl Health, said: “GLP-1 medicines have changed what is possible in obesity and type 2 diabetes, but they require chronic, high-dose systemic exposure that many patients cannot or do not sustain. RJVA-001 takes a different path: a potential one-time, pancreas-targeted gene therapy designed to enable the body to produce GLP-1 in response to meals: physiology, not pharmacology. With this authorisation, RJVA-001 becomes the first AAV gene therapy candidate to enter clinical development for type 2 diabetes.”
The company also has its post-GLP-1 weight miniatous therapy, Revita, in a pivotal study (NCT06484114), with a primary completion date in February 2027, according to ClinicalTrials.gov. Revita is an outpatient endoscopic procedure.
Fractyl also has a second gene therapy, RJVA-002, in preclinical development.
GlobalData’s latest report, “GLP-1 Innovation Landscape 2026: Industry Insights,” reveals that the use of glucagon-like peptide-1 receptor agonist (GLP-1RA) drugs is rapidly expanding beyond treatments for diabetes and into mainstream weight management because they are effective at increasing satiety and reducing food intake, resulting in rapid weight loss.
Use among patients without diabetes but with overweight or obesity in the US rose 700% from 2019 to 2023, according to researchers from the University of Pennsylvania and Cedars-Sinai Medical Center.
While injectables remain the growth engine through 2031, the convenience and affordability of the GLP‑1RA pill format is expected to expand the user base, with pill market sales expected to rise from $3.2bn in 2025 to $34.3bn in 2031, according to GlobalData’s Pharmaceutical Intelligence Centre.
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