New investors participating in this financing include Bpifrance Large Venture and funds managed by Tekla Capital Management LLC. Dynacure’s existing investors Andera Partners, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Kurma Partners and Pontifax also meaningfully participated. In connection with the financing, Henry Skinner, Ph.D., Senior Vice President of Tekla Capital Management, will join the Dynacure board of directors.

• “We are excited to partner with Dynacure as they pioneer the use of antisense therapy for the treatment of Myotubular and Centronuclear Myopathies,” said Ellen Hukkelhoven, Managing Director of Perceptive Advisors. “Perceptive has long been committed to supporting novel approaches in rare diseases and we look forward to working with the dedicated team at Dynacure to bring DYN101 and other medicines to patients faster.”

DYN101 is an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM). Proceeds from the financing will be used to advance Dynacure’s lead program, DYN101, through an ongoing Phase 1/2 clinical trial in adults, expand the DYN101 clinical program to include treatment of pediatric patients and to continue to grow the company and its pipeline.

• “Since the company’s inception in 2016, Dynacure has made significant progress in a very short time, resulting in the first CNM patient dosed with their antisense medicine, DYN101, in March 2020,” said Laurent Higueret, Investment Director at Bpifrance Large Venture. “We view this financing as our commitment to supporting Dynacure as they advance DYN101 and become one of the industry’s future rare disease leaders.”
   
• “With the support of our outstanding group of investors, this financing positions us well to advance DYN101 through Phase 1/2 clinical evaluation in both adult and pediatric patients and expand our pipeline into other rare muscle disorders to strengthen Dynacure’s growth,” said Stephane van Rooijen (M.D. MBA), Chief Executive Officer of Dynacure.

CNMs are serious, rare, life-threatening disorders that affect skeletal muscles from birth. Centronuclear Myopathies affect between 4,000 and 5,000 patients in the EU, US, Japan and Australia1. DYN101 is being developed in collaboration with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery. Preclinical efficacy observed in multiple mouse models of the disease gives confidence in the ability to demonstrate potential disease-modifying results in humans.