Expansion said much of the funds will go into its SMiRNA platform to find clinical candidates in myotonic DM1, FTD, ALS, and several other tauopathies.
Expansion Therapeutics today raised $80 million from a Series B financing round to support its growing RNA-led therapy research.
The Boston-based biotechnology firm is known for its focus on developing oral therapies for severe RNA-mediated diseases. In a statement, the company said much of the funds it generated will go into its small molecule RNA platform SMiRNA to find clinical candidates in myotonic dystrophy type 1 (DM1), frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), and several other tauopathies.
Expansion’s initiatives are anchored on finding structured RNA targets that are genetically, or evolutionary demonstrated to cause disease. The illness-causing RNA structures are screened against the company’s proprietary RNA-focused libraries for small molecules that modulate the RNA target. SMiRNA will be utilized to design lead-like chemical matter to create an oral medication to treat neurological diseases.
The research is vital as most patients suffering from neurological illnesses only learn to live with the disease with no hope for a cure. Expansion’s efforts could lead to a significant change in the lives of those suffering from severe and debilitating illnesses that offer minimal options.
“Drugging RNA with small molecules has the potential to dramatically transform the lives of patients... This financing milestone demonstrates significant investor confidence in the leadership team, the science and our mission to accelerate the preclinical and clinical development of our novel medicines utilizing our proprietary approach,” said Renato Skerlj, Ph.D., the president and chief executive officer of Expansion Therapeutics.
As part of its growth plans, Expansion recently in-licensed two research initiatives from Scripps Research, which includes a program that targets tau — a key driver of dementia disorders. The tau program builds on the observation that tau protein production contributes to the formation of the neurofibrillary tangles linked to tauopathies like frontotemporal dementia, Alzheimer’s disease, and other neurodegenerative illnesses.
“Recent advances in understanding RNA structure and the science of targeting RNA with small molecules offer tremendous opportunities to transform the way RNA-mediated diseases are treated. We’re extremely enthusiastic about the promise that the SMiRNA™ platform holds to deliver disease-modifying therapies for a wide range of devastating neurological disorders, particularly neurodegenerative diseases,” commented Andy Phillips, Ph.D., the managing director of Cormorant Asset Management, one of the top contributors to the Series B funding round.
The other companies participating in the fundraising event are Surveyor Capital, Westlake Village BioPartners, Logos Capital, RA Capital Management, 5AM Ventures, Sanofi Ventures, Novartis Venture Fund, and Kleiner Perkins.
