DM1 is an inherited genetic disorder linked with the DMPK gene. People diagnosed with DM1 experience muscle loss and weakness, difficulty breathing, cataracts, heart conditions, intellectual disability, and early death. According to NORD, DM1 affects approximately one in 8,000-20,000 people in the general population, with significant geographic and ethnic variation.
“DM1 is a devastating disease for which there is no treatment that addresses its underlying cause, so we are eager to carry out our collaboration with Pfizer to accelerate the development of new treatments into this space,” said Ameet Nathwani, M.D., Chief Executive Officer of Dewpoint. “We are excited about applying our platform to a wide array of diseases where biomolecular condensates may play a role in key disease pathways, as Dewpoint believes they do in DM1.”
“The development of treatments for rare diseases, such as DM1, is a complex and rapidly evolving area of science, underscoring the importance of emerging research like this,” said Uwe Schoenbeck, Senior Vice President and Chief Scientific Officer, External Science & Innovation at Pfizer. “Through our combined expertise, we believe this collaboration has the potential to advance novel therapeutic solutions for patients in need.”
About Biomolecular Condensates
Biomolecular condensates, formed through a process called phase separation, are membraneless droplets inside cells that facilitate molecular interactions and help cells perform vital functions. Condensates have been shown to play a critical role in key biological processes and in serious, intractable diseases across areas including neurodegeneration, cancer, inflammation, infectious disease, metabolic disease, and rare genetic disorders. The first condensates were observed more than 100 years ago. It is only in the last dozen years, though, that scientists—including Dewpoint founders Tony Hyman of the Max Planck Institute in Dresden and Rick Young of the Whitehead Institute—have begun to understand the dynamic nature and function of condensates. Dewpoint develops drugs that exploit this biology. Prior to the discovery of biomolecular condensate function, it was unknown how the right molecules could find each other at the right time to catalyze important processes in the crowded molecular environment of the cell.
About Dewpoint Therapeutics
Dewpoint Therapeutics is the first company to apply the emerging understanding of biomolecular condensates to drug discovery. Dewpoint believes that a vast range of conditions have pathways that are regulated by condensates or arise from the dysfunction of condensates — including cancer, neurodegeneration, infectious disease, and metabolic disease. Dewpoint scientists work in Boston, Dresden, and Berlin to translate condensate biology into treatments for the toughest diseases.
Scientists or investors interested in biomolecular condensates can also visit condensates.com for news and updates in the field.