Expansion Therapeutics issued a press release this week highlighting funding for the development of therapies targeted at expanded repeat disorders, including myotonic dystrophy (DM1 and DM2).

New Biotech Company

Expansion Therapeutics, which launched in late 2017, is testing a small molecule platform technology developed by Dr. Matt Disney at the Scripps Research Institute Florida. Expansion identified DM1 as an early focus, while DM2 and other expanded repeat disorders are also candidate indications for the technology. The small molecule strategy in DM is to selectively bind CUG and CCUG repeats and displace muscleblind, thereby allowing restoration of normal RNA splicing.

Significant First-Round Funding

Expansion Therapeutics reports that it received $55.3M in Series A financing from a strong group of investors, including 5AM Ventures, Kleiner Perkins, Novartis Venture Fund, and Sanofi Ventures. Series A financing is the first round of financing given to a new business. Interest from the venture capital arms of the major pharmaceutical firms, Novartis and Sanofi, is an encouraging sign, as not only are they experienced in evaluating candidate therapeutic opportunities, but they have the resources to help move the program into clinical development.

The initial focus of Expansion Therapeutics efforts is on discovery and development of RNA targeted molecules. This will necessarily involve considerable preclinical and regulatory efforts before a candidate therapeutic for DM1 and DM2 could reach clinical trials. MDF will continue to work with Dr. Disney and Expansion Therapeutics to help foster their drug development efforts on behalf of patients living with myotonic dystrophy.