Undisclosed mRNA target (ETX-394)

The target of ETX-394 is a novel, undisclosed mRNA transcript identified by Tangram Therapeutics (formerly e-therapeutics) using its proprietary HepNet™ computational platform [13, 33]. This target is specifically expressed in hepatocytes and is involved in the molecular pathways driving metabolic dysfunction-associated steatohepatitis (MASH) [15, 25]. ETX-394 is an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to silence this target gene through the RNA interference (RNAi) pathway [18, 23]. By promoting the sequence-specific degradation of the target mRNA, ETX-394 aims to modulate disease biology that is distinct and differentiated from the company's lead MASH program, ETX-312 [13, 32]. Preclinical proof-of-concept studies are currently underway to evaluate the candidate's ability to reduce hepatic fat accumulation, inflammation, and fibrosis [15, 26]. As a precision genetic medicine, ETX-394 represents a targeted approach to treating chronic liver diseases with high unmet medical need [13, 25]. The use of the GalOmic™ platform ensures potent and durable silencing with the potential for infrequent subcutaneous dosing [13, 18].

Other names

ETX-394 targetTGM-394 targetUndisclosed MASH target 2

RNA interference (RNAi) mediated by a GalNAc-conjugated small interfering RNA (siRNA) that leads to the sequence-specific degradation of the target mRNA in hepatocytes [13, 18].