Solid Biosciences Completes $50M in Series C Financing to Advance Duchenne Program

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

Solid DMD

Solid Biosciences of Cambridge, Mass., has completed the initial closing of a $50 million Series C round of financing to advance the company’s clinical portfolio of Duchenne muscular dystrophy (DMD) medications and programs.

RA Capital Management and Bain Capital Life Sciences are the principal investors, with input also from RTW Investments, Foresite Capital, Cormorant Asset Management, entities affiliated with Leerink Partners and an unnamed healthcare fund. Existing investors Perceptive Advisors, Janus Capital Management and Biogen also participated.

Solid is a company exclusively working to find meaningful therapies for Duchenne. Founded in late 2015 by former investment banker Ilan Ganot — whose son, Eytani, was born with the disease — the company brings together experts in science, technology and healthcare to drive forward a portfolio of candidates that demonstrate therapeutic potential.

Currently, Solid is advancing programs across four platforms: corrective therapies, disease-modifying therapies, disease understanding and assistive devices. Its lead candidate, SGT-001, is an adeno-associated viral (AAV) vector-mediated gene therapy whose first clinical trials will start in late 2017.

SGT-001 is aimed at rescuing functional dystrophin expression in muscle. Dystrophin is the deficient protein in DMD. In preclinical trials, a unique dose of SGT-001 was capable of stabilizing the dystrophin glycoprotein complex and restoring muscle function, with improvements observed in muscle strength and protection against damage induced by muscle contraction.

“The proceeds will enable us to move SGT-001 through clinical development and maintain momentum in our efforts to identify and develop a new generation of meaningful DMD therapies,” Ganot said in a press release.

Solid will continue to secure manufacturing capacity to support its program’s long-term clinical and commercial requirements. One such effort includes the recent Solid Biosciences merger with Solid GT, the company’s subsidiary focused on gene therapy development. The boards of directors will also be consolidated.

“Solid has put together one of the most compelling scientific portfolios in DMD, starting with its innovative gene therapy program,” said Rajeev Shah, portfolio manager and managing director at RA Capital. “We are excited to support a company that has the right business model and people in place to successfully bring forward life-changing therapies for patients with this devastating disease.”

SGT-001 received orphan drug designation last October from both the U.S. Food and Drug Administration and the European Commission.