▎WuXiEdited by Kant Content Team

Dewpoint Therapeutics today announced a strategic research collaboration agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) to jointly advance one of Dewpoint'sUnder ResearchTAR DNATDP-43 TargetingSmall molecule condensate modulators (c-mod) for the treatment of patients with amyotrophic lateral sclerosis (ALS).

Leveraging its unique expertise in the field of condensate biology, Dewpoint has discovered a small molecule drug capable of addressing the TDP-43 mislocalization issue.TDP-43 is a key splicing factor, closely associated with various neurodegenerative diseases and represents one of the core pathological features in over 97% of ALS patients.The abnormal accumulation of TDP-43 in membraneless cytoplasmic structures leads to impaired neuronal function in ALS patients, and these abnormal structures are referred to as biomolecular condensates.

Through high-throughput screening technology, Dewpoint Therapeutics discovered a c-mod capable of alleviating pathological TDP-43 condensates. This small molecule drug corrects the dysfunction of the disease-causing condensates (i.e., "condensate diseases") by selectively separating TDP-43, restoring its proper localization in the nucleus, and maintaining normal cellular stress responses.This therapy not only restored the normal splicing activity of TDP-43 but also significantly improved neuronal health and enhanced multiple key biomarkers associated with ALS in animal models.

▲Dewpoint Therapeutics Pipeline (Source:Dewpoint Therapeutics Official Website）

According to the terms of the agreement, Dewpoint will receive an upfront payment and is eligible to receive milestone payments based on specific near-term research and development goals.After achieving these milestones, MTPC will obtain an exclusive option and conduct global clinical development and commercialization of the project.TheThe total transaction amount can reach up to 480 million US dollars.

References:

[1] Dewpoint Therapeutics and Mitsubishi Tanabe Pharma Corporation Enter Research Collaboration to Advance Small Molecule Condensate Modulator for ALS. Retrieved December 4, 2024 from https://dewpointx.com/dewpoint-therapeutics-and-mitsubishi-tanabe-pharma-corporation-enter-research-collaboration-to-advance-small-molecule-condensate-modulator-for-als/

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