TGM-148 is designed to combine hemostatic efficacy with a favorable safety profile and low treatment burden
Tangram’s second asset to enter the clinic in 2026
Phase 1 trial will evaluate TGM-148 in healthy volunteers, with data anticipated in 2027
LONDON, Oct. 29, 2026 (GLOBE NEWSWIRE) -- Tangram Therapeutics ("Tangram"), a company committed to unlocking transformative RNAi medicines for meaningful patient impact, today announced the dosing of the first participant in its Phase 1 trial evaluating TGM-148, a novel GalOmic medicine for the treatment of bleeding disorders, in healthy volunteers. TGM-148 is Tangram’s second GalOmic medicine to enter the clinic this year.
There are up to five million people living with bleeding disorders worldwide1. Von Willebrand disease (VWD), the planned entry indication for TGM-148, is the most common inherited bleeding disorder, with approximately 140,000 people diagnosed with the condition in the United States alone². People living with severe VWD experience frequent bleeding and significantly reduced quality of life, yet prophylactic treatment options remain limited and burdensome, involving multiple intravenous infusions per week.
TGM-148 is a GalOmic siRNA targeting Protein Z-dependent Protease Inhibitor (ZPI), a natural regulator of coagulation. Target identification was enabled by Tangram’s proprietary network biology approach, with human genetic data providing further evidence for the safety of ZPI downregulation. By silencing ZPI, TGM-148 combines robust hemostatic efficacy with low thrombotic risk through its novel context-specific mechanism of action, enhancing clot formation at the site of active bleeding when clotting is impaired. With an anticipated regimen of four or fewer subcutaneous doses a year, TGM-148 has the potential to substantially reduce the burden of prophylaxis in VWD.
The ongoing Phase 1 trial will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending doses of TGM-148 in healthy volunteers. Initial data are anticipated in 2027. VWD is planned as the first clinical indication, with the opportunity to expand into a broader range of rare bleeding disorders, supported by preclinical data generated in four distinct genetic models.
“At Tangram, we set out to create impactful medicines that can add differentiated options to the treatment paradigm, and TGM-148 is a clear example of our approach. For VWD patients, our ambition is to develop a prophylactic option that safely protects against bleeds and fits around their lives. Initiating our Phase 1 trial brings us closer to realizing our ambition, in VWD and beyond,” said Dr. Laura Roca-Alonso, Chief Executive Officer.
“This Phase 1 study is designed to rigorously characterize the safety and pharmacology of TGM-148 in healthy volunteers, a stringent setting in which to assess its profile. These insights will enable the program to accelerate into patient studies in VWD and other rare bleeding disorders. Establishing a strong safety foundation is essential as we advance TGM-148 to patients in need,” added Dr. Sonya Montgomery, Chief Development Officer.
About TGM-148
TGM-148 is a first-on-target GalOmic GalNAc-conjugated small interfering RNA (GalNAc-siRNA) designed to selectively silence Protein Z-dependent Protease Inhibitor (ZPI) in the liver. Its context-specific mechanism of action aims to enhance clotting in response to bleeding in those with bleeding disorders, while maintaining a favorable safety profile.
In preclinical studies, TGM-148 demonstrated efficacy in four genetic models of bleeding disorders and was well tolerated at supratherapeutic doses in wildtype animals. Together, these findings support TGM-148’s wide therapeutic window and provide a strong rationale for its development across a broad range of bleeding disorders, with VWD selected as the first indication. By offering the potential for quarterly or less frequent subcutaneous administration, TGM-148 is also designed to lower the barriers to prophylaxis, supporting broader uptake, better outcomes, and improved quality of life for patients.
About Tangram Therapeutics
Tangram Therapeutics is a clinical-stage biotech committed to unlocking transformative RNAi medicines for meaningful patient impact. Real progress in the treatment of diseases with high unmet need calls for drug discovery and development that goes beyond the obvious. Tangram pursues differentiated target genes, developing innovative medicines enabled by GalOmic, the Company’s proprietary RNAi chemistry platform designed to selectively silence disease-associated genes in hepatocytes. Tangram is advancing a pipeline of investigational RNAi medicines for real-world patient benefit, including TGM-312 for the treatment of MASH and TGM-148 for the treatment of bleeding disorders.
Learn more at www.tangramtx.com.
Media Contact
press@tangramtx.com
Investor Relations Contact
Ami Bavishi or Nick Colangelo
Gilmartin Group LLC
TangramTx@Gilmartinir.com
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References
- Al-Samkari H et al. Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report. Am J Hematology 2025.; Castaman G et al. Von Willebrand disease: classification and epidemiology. Haematologica 2026. World Federation of Hemophilia, Report on the Annual Global Survey 2024. Komodo claims data.
- Weyand A et al. ASH 2024
