DTx Pharma is a preclinical stage biotechnology company focused on developing small interfering RNA (siRNA) therapeutics for rare diseases. The company's proprietary Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform enables the delivery and activity of siRNA therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake.
DTx Pharma's lead candidate, DTx-1252, is a potential first-in-class FALCON siRNA therapeutic targeting PMP22, the underlying genetic cause of Charcot-Marie-Tooth Disease Type 1A (CMT1A). By repressing PMP22, DTx-1252 has demonstrated reversal of CMT1A in preclinical rodent models, inducing remyelination of axons, improving electrophysiological measurements, and increasing muscle mass, grip strength, coordination, and agility. DTx-1252 received Orphan Drug Designation from the FDA in June 2023 for the treatment of CMT1A.
In addition to DTx-1252, DTx Pharma's pipeline includes two other preclinical programs focused on neuromuscular and central nervous system (CNS) indications, leveraging the FALCON platform's versatility in delivering siRNA therapeutics to extrahepatic tissues.
In July 2023, Novartis acquired DTx Pharma for an upfront payment of USD 500 million and potential additional milestone payments of up to USD 500 million. The acquisition provides Novartis with access to the FALCON platform and DTx Pharma's pipeline, including DTx-1252 for CMT1A, aligning with Novartis' commitment to developing life-changing medicines for neuromuscular diseases and disorders of the nervous system.
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