Fractyl Health(US:GUTS) Globenewswire·2025-05-19 11:00Core Viewpoint - Fractyl Health is advancing a first-in-human study for RJVA-001, a gene therapy targeting pancreatic beta cells to treat inadequately controlled type 2 diabetes (T2D) and obesity, with preliminary data expected in 2026 pending regulatory approval [1][9]. Company Overview - Fractyl Health, based in Burlington, MA, focuses on innovative treatments for obesity and T2D, aiming to shift from chronic management to disease-modifying therapies [8]. - The company is pioneering the Rejuva platform, which develops next-generation adeno-associated virus (AAV)-based gene therapies targeting metabolic diseases [9]. Product Details - RJVA-001 is designed to express glucagon-like peptide-1 (GLP-1) locally in pancreatic beta cells, potentially reducing side effects associated with systemic GLP-1 therapies [1][2]. - The therapy utilizes a minimally invasive endoscopic ultrasound-guided infusion directly into the pancreas, allowing for nutrient-triggered secretion of GLP-1 [2][4]. Clinical Study Design - The upcoming Phase 1/2 study will be an open-label, multicenter trial assessing the safety, tolerability, and preliminary efficacy of RJVA-001 in adults aged 35-70 with T2D [4][6]. - Participants will be monitored for 12 months post-treatment, with a long-term follow-up study extending up to 5 years [5]. Study Objectives and Endpoints - Primary endpoints include safety and tolerability, while secondary endpoints focus on changes in HbA1c, fasting plasma glucose, and time-in-range metrics [7]. - Exploratory endpoints will assess beta-cell function, metabolic biomarkers, cardiovascular risk markers, and transgene expression [7]. Future Implications - If successful, RJVA-001 could redefine diabetes treatment from daily management to a potential one-time therapy, significantly impacting patient care [5]. - The company aims to develop a platform of therapies that offer superior durability, potency, and tolerability compared to conventional GLP-1 drugs [3].