Therapy Areas: Cardiovascular
Expansion Therapeutics Raises USD 80m series B Financing to Treat Neurodegenerative Diseases
29 September 2021 - - US-based biotechnology company Expansion Therapeutics, Inc has closed an USD 80m series B financing, the company said.

Cormorant Asset Management led the financing with participation from new investors Westlake Village BioPartners, Surveyor Capital (a Citadel company) and Logos Capital as well as series A investors RA Capital Management, 5AM Ventures, Kleiner Perkins, Sanofi Ventures and Novartis Venture Fund.

Proceeds from the financing will be used to advance the company's small molecule RNA platform (SMiRNA) to identify clinical candidates in myotonic dystrophy type 1, amyotrophic lateral sclerosis, frontotemporal dementia and various tauopathies.

Expansion focuses on structured RNA targets that are evolutionarily conserved or genetically demonstrated to cause disease.

Disease-driving RNA structures amenable to ligand binding are identified by combining patient genetics with deep informatic analysis and then screened against Expansion's proprietary RNA-focused libraries for small molecules that modulate the RNA target in a functionally relevant manner.

Expansion's novel structural biology SMiRNA platform is then utilized for the design and advancement of lead-like chemical matter that ultimately delivers an oral medicine to treat disease.

Expansion is focused on neurological diseases where few treatment options are available for patients such as DM1 which is a system wide disorder affecting muscle, cardiac and brain function. A small molecule medicine that treats all aspects of the disease offers patients a significant benefit.

As part of its initiative to broaden its portfolio of small molecule medicines, Expansion also recently in-licensed two new research programs from Scripps Research, including a program targeting tau, an important driver of dementia disorders.

The tau program builds on the observation that defective pre-mRNA encoding the production of tau protein is involved in the formation of neurofibrillary tangles and has been linked to tauopathies such as Alzheimer's disease, frontotemporal dementias, progressive supranuclear palsy and other neurodegenerative diseases.

Most recently, Expansion's scientific founder, Matthew D. Disney, Ph.D. and colleague, Alicia Angelbello, Ph.D., were recipients of the American Chemical Society's Nobel Laureate Signature award for their pioneering work developing RNA-targeting medications for genetic diseases, including DM1.

Expansion Therapeutics is a biotechnology company focused on developing transformative oral medicines for severe RNA-mediated diseases including neurodegenerative disorders.

Based on exclusive worldwide rights to groundbreaking research from the laboratory of Matthew D. Disney, Ph.D., at Scripps Research, Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule RNA modulators.

Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of severe RNA-mediated disease indications. Headquartered in Boston, Massachusetts, Expansion's research facility is located in Jupiter, Florida.
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