Novartis acquires RNA medicine delivery startup DTx Pharma for $500 M

DTx Pharma's Fatty Acid Ligand Conjugated Oligonucleotide (FALCON) platform facilitates extra-hepatic delivery of siRNA therapeutics

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DTx Pharma, a US-based preclinical stage biotechnology startup addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform, has been acquired by Swiss multinational pharmaceutical corporation Novartis.

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 programme is currently in preclinical development, with FDA Orphan Drug Designation, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a progressive, neuromuscular, autosomal-dominant disease that can lead to life-long loss of muscle function and disability.

Currently, there are no approved therapeutics addressing PMP22, the underlying genetic cause of CMT1A, for the estimated 150,000 patients living with the debilitating disease in the United States and Europe.

In addition to the CMT1A programme, Novartis has acquired full rights to the FALCON platform and two other early-stage programmes in neuromuscular and central nervous system (CNS) indications.

Under the terms of the agreement, Novartis will make an upfront payment of $500 million and additional payments of up to $500 million upon completion of pre-specified milestones.

Published on : 18th July, 2023