BOSTON, Massachusetts, January 7, 2024 – Dewpoint Therapeutics Inc., a biotechnology company that translates the novel science of condensates into breakthrough therapeutics, today announced the filing of multiple patent applications covering novel compounds and their use in the potential treatment of amyotrophic lateral sclerosis (ALS).
Composition of matter covered by these applications aims to treat ALS by modulating TDP-43 – a protein implicated in the pathology of ALS and other neurological diseases – in a condensate-selective manner. Specifically, claims cover chemical space and compositions around several compound series, and their use in treating ALS, and other TDP-43 mediated neurological diseases. These patents build upon Dewpoint’s considerable patent estate, including the recently granted foundational platform patents directed to methods of screening condensate-associated specificity and drug partitioning.
ALS is a devastating neurodegenerative disease, caused by a combination of genetic and environmental factors. Despite the genetic diversity between ALS patients, the majority share a common trait: a condensatopathy. A condensatopathy is an aberration in a membraneless compartment (condensate) that functions as a central node of dysfunction in complex diseases and across multiple patient populations. TDP-43, a protein that performs its normal functions in the nucleus, is erroneously localized in cytoplasmic condensates, driving disease through a combination of gain and loss of function. By taking a non-traditional approach to target the condensate in motor neurons, Dewpoint discovered c-mods that correct pathological markers of ALS in disease-relevant models; they reduce TDP-43 cytoplasmic inclusions, improve nuclear TDP-43 function, and reduce neurodegeneration. C-mods targeting the TDP-43 condensatopathy are the first small molecules to potentially address the underlying pathology of >97% of ALS patients.
These patents, the first of a series of Dewpoint-discovered new chemical entities, mark a pivotal milestone in the company’s progress towards transformative drugs for patients with diseases such as ALS. Over the next few years, the Dewpoint platform is set to produce many new drugs utilizing innovative condensate mechanisms to tackle a variety of diseases.
Dr. Ameet Nathwani, CEO of Dewpoint Therapeutics commented:
“We are thrilled to expand our already robust patent portfolio, a significant leap forward in our mission to redefine the treatment of diseases deemed intractable or difficult to treat, such as ALS.”
Condensates are membraneless organelles that form dynamically throughout the cell via a process called phase separation. These subcellular compartments organize and concentrate molecules within cells to enable certain key biochemical processes. The dysregulation of biomolecular condensates has been observed in many diseases, including cancer, diabetes, and neurological disorders. Condensate-modifying drugs (c-mods) potentially provide novel therapeutic options for complex diseases and historically undruggable targets.
ABOUT DEWPOINT THERAPEUTICS
Dewpoint is the first company to apply the emerging understanding of biomolecular condensates to drug discovery for the vast range of conditions that are regulated by condensates or arise from the dysfunction of condensates. Dewpoint drives drug discovery though a proprietary end-to-end experimental and AI-powered platform. Dewpoint currently has programs across an ambitious pipeline spanning oncology, neurodegenerative, cardiopulmonary, and metabolic disease indications, and collaborations with leading global academic and pharmaceutical partners, including Bayer and Novo Nordisk. Dewpoint scientists work in Boston, Dresden, and Frankfurt to translate condensate biology into medicine for diseases previously considered untreatable. Learn more at Dewpointx.com and follow us on X and LinkedIn.