Funds aim to accelerate development of best-in-class antibody-RNA conjugate to treat facioscapulohumeral muscular dystrophy
GAITHERSBURG, Maryland and VANCOUVER, British Columbia – September 29, 2022 (View original source.) – miRecule, Inc., an innovator of next-generation RNA therapeutics, and SOLVE FSHD, a venture-philanthropy organization catalyzing the pace of innovation to accelerate a cure for facioscapulohumeral muscular dystrophy (FSHD), today announced that SOLVE FSHD has invested US$1 million in miRecule to support the discovery of the company’s anticipated best-in-class antibody-RNA conjugate (ARC) designed to be a disease-modifying treatment for the underserved FSHD patient population.
Categorized as a rare-disease, FSHD is the second most common type of muscular dystrophy – affecting more than one million individuals worldwide with no approved treatments. Patients with causative genetic mutations experience lifelong deterioration of muscle function and progressive disability. miRecule discovered its anti-DUX4 RNA therapy through its proprietary DREAmiRTM platform technology. The RNA therapy is conjugated to a muscle specific antibody utilizing miRecule’s NAVIgGatorTM chemistry. The resulting molecule has the potential to be a best-in-class treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. This groundbreaking approach would enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive disease.
In addition to the investment, Executive Director Dr. Eva Chin, Ph.D. , will join miRecule’s Scientific Advisory Board. miRecule expects to benefit from Dr. Chin’s decades of experience in drug development for neuromuscular disorders to help guide preclinical development of the program.
Dr. Eva Chin, Executive Director, Solve FSHD
“We are excited to support miRecule and to be a part of this unique and cutting edge approach to a disease-modifying therapy for FSHD. The goals of miRecule in identifying a muscle-specific antibody conjugated DUX4 inhibitor for FSHD is aligned with our Founder Chip Wilson’s mission of being a catalyst towards a cure for FSHD. Anthony’s leadership of miRecule with their expansion into rare neuromuscular disease and focus on FSHD has been inspirational. His continued integration with the patient community and focus on a novel approach is accelerating the path to new therapies. At Solve FSHD, we are honored to be part of the miRecule support network.”
Anthony Saleh, Founder and Chief Executive Officer, miRecule
"Chip and Neil have created a unique organization to accelerate a cure for FSHD through SOLVE, and we are thrilled to have their support to help miRecule advance our groundbreaking approach targeted at the cause of this devasting disease. We also look forward to the contributions from someone of Eva’s caliber as her guidance will prove invaluable to ensure our development program’s success. With the added support of the patient community, miRecule and SOLVE will work tirelessly to bring forward an effective treatment for FSHD.”
miRecule’s founder and CEO has a strong commitment to developing a therapy to treat FSHD as the disorder runs in his family. miRecule started its program in 2019 with strong support from the patient and academic community, receiving funding from the FSHD Society, the Friends of FSH Research, and National Institute of Neurological Disorders and Stroke (NINDS, Grant 5R44NS119147-02).
miRecule, Inc. is a biotechnology company focused on advancing next-generation RNA therapeutics utilizing personalized genomic patient data to create highly tailored therapeutics treating a variety of diseases, including cancer and muscular dystrophy. The company’s proprietary DREAmiR™ genomics-based discovery platform identifies critical RNA targets for drug development in specific subsets of patients that would benefit from treatment. miRecule then creates proprietary RNA therapeutics with improved pharmacology and joins them with antibodies for targeted delivery to disease tissue. miRecule’s lead candidate, MC-30 for head and neck cancer, replaces the potent tumor suppressor activity of microRNA-30 which is lost in half of head and neck cancer patients. miRecule’s second program that is now licensed by Sanofi, MC-DX4 for the treatment of Facioscapulohumeral muscular dystrophy (FSHD), eliminates expression of the DUX4 gene, which causes the disease in 95% of FSHD patients. For more information, please visit: www.mirecule.com.
About SOLVE FSHD
SOLVE FSHD is a Vancouver-based venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. FSHD is one of the most prevalent forms of muscular dystrophy, and the organization will work as a catalyst to accelerate drug developments and treatments for FSHD2 while further benefiting FSHD1. Established by renowned Canadian entrepreneur and philanthropist Chip Wilson, the founder of technical apparel company lululemon athletica inc. Chip has committed $100 million to kick-start funding into projects that support the organization’s mission to find a cure for FSHD by 2027.
The goal of SOLVE FSHD is to find a solution that can stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. The organization will fund innovative biotech and biopharma research and development activities that accelerate novel treatments and therapies for FSHD. For more information, please visit: www.solvefshd.com.
miRecule, Inc. Contacts
Communications – Stacy Roughan, media@miRecule.com
Investor Relations – Anthony Saleh, firstname.lastname@example.org