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DTx Pharma's Fatty Acid Ligand Conjugated Oligonucleotide (FALCON) platform facilitates extra-hepatic delivery of siRNA therapeutics.
July 17, 2023
By: Kristin Brooks
Novartis has acquired DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform, for $500 million upfront and additional payments of up to $500 million based on pre-specified milestones. The FALCON platform enables the delivery and activity of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake. DTx Pharma’s lead program is currently in preclinical development, with FDA Orphan Drug Designation, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A), a progressive, neuromuscular, autosomal-dominant disease that can lead to life-long loss of muscle function and disability. DTx Pharma’s lead asset, DTx-1252, is a novel, potential first-in-class, FALCON siRNA candidate targeting PMP22. The asset’s preclinical package demonstrates the reversal of disease in preclinical rodent models and translation to higher species with IND-enabling studies progressing. Novartis has acquired full rights to the FALCON platform and two other early-stage programs in neuromuscular and central nervous system (CNS) indications. “I am thrilled that Novartis will be moving forward with our CMT1A therapeutic program and the FALCON platform. With its resources and capabilities in neuromuscular diseases, Novartis is well positioned to accelerate the development of DTx-1252 and provide hope to patients, who are desperately in need of therapy,” said Artie Suckow, Ph.D., co-founder and CEO of DTx Pharma. “This acquisition underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system,” said Robert Baloh, Global Head of Neuroscience for the Novartis Institutes for BioMedical Research. “We are excited to drive forward these promising preclinical programs and explore the potential of the FALCON platform.”
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