DTx Pharma

DTx Pharma was a San Diego-based preclinical biotechnology company focused on RNA-based therapeutics for genetic disease, with an emphasis on extra-hepatic delivery of small interfering RNA. Its core technology was the proprietary FALCON platform, which used fatty acid ligand conjugation to improve biodistribution and cellular uptake of oligonucleotide therapeutics beyond the liver. The company’s lead program, DTx-1252, was being developed for Charcot-Marie-Tooth disease type 1A and had received FDA Orphan Drug Designation. In July 2023, Novartis acquired DTx Pharma to expand its xRNA and neuroscience capabilities, including DTx’s platform and early pipeline in neuromuscular and CNS disease.