U.S. biotech company Fractyl Health is launching the world's first clinical trial of a glucagon-like peptide-1 (GLP-1) gene therapy.
As GLP-1 class treatments that mimic the action of the hormone GLP-1, which is naturally secreted in the gut when eating, have emerged, the paradigm for treating diabetes and obesity has changed dramatically.
Current GLP-1 treatments on the market are administered as weekly or monthly injections or taken daily, but the industry is focused on the attempt to develop a therapy that induces the body to generate the GLP-1 hormone on its own with a single administration.
Fractyl Health said on the 11th (local time) that RJVA-001, its GLP-1 gene therapy under development, received Phase 1/2 clinical trial approval from Dutch regulators.
This is the first case of a GLP-1 gene therapy entering human clinical trials.
The trial targets adult patients with type 2 diabetes whose blood sugar control remains insufficient despite using existing antihyperglycemic agents and GLP-1 receptor agonists. The company will begin recruiting patients from June 1, with plans to dose the first patient and secure initial data in the second half of this year.
RJVA-001 takes a fundamentally different approach from existing obesity and diabetes drugs such as Wegovy by Novo Nordisk and zepbound and Mounjaro by Eli Lilly and Company, which currently dominate the market.
RJVA-001 is designed to use an adeno-associated virus (AAV) vector to enable cells in the patient's body to produce GLP-1 directly. The company said the therapy is designed to respond only at mealtimes to secrete GLP-1. To reduce the risk of hypoglycemia or gastrointestinal side effects that can occur when GLP-1 is excessively secreted, it is structured to mimic the body's natural metabolic response.
The Korea Biotechnology Industry Organization said, "If this gene therapy succeeds in development, it is expected to fundamentally change the paradigm of diabetes and obesity treatment that currently requires lifelong, regular injections or pills."
However, there are many hurdles to clear before commercialization.
Because gene therapies act in the body for a long time after a single administration, safety verification standards are very strict. Key questions include how stably GLP-1 expression can be controlled and whether there are long-term side effects. The current stage is still only a Phase 1/2 trial assessing initial safety and tolerability.
Fractyl Health is also pursuing trials in Australia following the Netherlands. The company has submitted an investigational new drug application (IND) to Australian regulators and said it expects approval in the third quarter of this year.
Fractyl Health, founded in 2010, is a biotech company specializing in metabolic diseases and listed on Nasdaq in 2024 under the ticker GUTS. Co-founder and CEO Harris Rajasopalan is known as an MD-PhD researcher trained at Stanford University and Johns Hopkins University School of Medicine.
The company recorded a net loss of about $141 million (about 209 billion won) in 2025 due to continued research and development (R&D) investment. As of the end of last year, its cash-equivalent assets were about $81.5 million (121.3 billion won). Given this, the company may seek financing to secure operating funds through early 2027.