Lighting the Way to a Cure

  • Financials
  • Patient Library
  • Blog
  • Calendar
FSHD Header Logo
mobile-logo

  • Who We Are
        • About Us
          • History
          • Mission
          • Our Team
          • Financials
        • Our Impact
          • Therapeutic Accelerator
          • Education
          • Advocacy
          • Our Partners
        • Journey To A Cure
          • 2022 Donor Impact Report
  • Understanding FSHD
        • What is FSHD
          • Symptoms
          • Diagnosis
          • Early-onset FSHD
          • Genetic Testing
        • Living with FSHD
          • Newly Diagnosed?
          • Finding Care
          • Symptom Management
          • Patient Library
        • FSHD University
          • Physical Health
          • Wellness
          • Research
  • Get Involved
        • Support Us
          • Ways to Give
          • Become a Torchbearer
          • Raise Awareness
          • Volunteer
          • Fundraise
        • Advance Research
          • Patient Involvement
          • True Cost of FSHD Study
          • Clinical Trials
        • Connect
          • Find Your Local Chapter
          • Join the Community
          • World FSHD Alliance
          • Online Communities
        • Events
          • Walk & Roll to Cure FSHD
          • Events Calendar
          • FSHD 360 Regional Conferences
          • FSHD Connect Conference
  • Research
        • For Patients & Families
          • Advance Research
          • True Cost of FSHD Study
          • TestFSHD genetic testing
          • Clinical Trials
        • For Researchers
          • Apply for a Grant
          • Research We Have Funded
          • Resources
          • Int’l Research Congress
        • For Clinicians
          • FSHD Masterclass
          • ICD-10
        • For Industry
          • Therapeutic Accelerator
          • Clinical Trial Research Network
          • Drug Development Pipeline
          • Voice of the Patient Report
          • Project Mercury
  • News
    • Media Library
    • Blog
      • Living with FSHD
        • Health and Medicine
        • PT and Exercise
        • Tips and Tricks
        • Mental Health
      • FSHD Society News
      • FSHD Research
      • Fundraising & Events
      • Media & PR
    • FSHD Society Radio
    • The FSHD Advocate
  • Search
  • Financials
  • Patient Library
  • Blog
  • Calendar
  • JOIN US
  • Donate
  • JOIN US
  • Donate

Sanofi and miRecule to partner on FSHD drug program

Home / FSHD Research / Sanofi and miRecule to partner on FSHD drug program

Posted on: Oct 04, 2022

Bringing two technologies together for a best-in-class therapy

miRecule, Inc., a biotech based in Gaithersburg, Maryland, today announced a strategic collaboration and exclusive license agreement with pharmaceutical giant Sanofi to develop and commercialize a novel treatment for FSHD. Sanofi is a multinational corporation valued at $36 billion, making it the ninth largest pharmaceutical company in the world. The two companies will collaborate to combine miRecule’s anti-DUX4 RNA therapy with Sanofi’s NANOBODY technology to target the therapy to muscles. The companies aim to produce a “best-in-class” treatment to slow or stop the progression of FSHD by suppressing the expression of the DUX4 gene, the underlying cause of the disease.

“Under the terms of the collaboration agreement, miRecule will grant Sanofi an exclusive worldwide license to intellectual property rights to the FSHD therapy,” miRecule said. “miRecule and Sanofi will collaborate on research activities through lead candidate selection.” Once a lead candidate has been selected, Sanofi will assume sole responsibility for  investigational new drug (IND) enabling studies—laboratory studies to produce data to demonstrate to the Food and Drug Administration (FDA) that the drug is likely to be safe, effective, and suitable to test in human clinical trials. If the FDA provides IND clearance, Sanofi would shepherd the drug through clinical trials, regulatory approval, and commercializing the therapy globally.

What does this partnership mean for patients?

The FSHD Society took an early interest in miRecule when its co-founder and CEO, Anthony Saleh, PhD, reached out after his own family was diagnosed with FSHD. The Society, Friends of FSH Research, and National Institute of Neurological Disorders and Stroke (NINDS, Grant 5R44NS119147-02) funded early-stage research. Last week, miRecule announced that SOLVE FSHD has agreed to invest $1 million toward the next phase of development.

The Society hoped that as miRecule hit its milestones, a large pharmaceutical company would become interested and provide the substantial resources and expertise needed to develop miRecule’s technology and take it to market. The partnership with Sanofi is great news because miRecule now has those needed resources.

"We are thrilled to enter our first major licensing transaction with a partner of Sanofi’s caliber and capabilities as a global leader in the development and commercialization of rare disease therapies,” said Saleh. “Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life.”

"We look forward to working with miRecule to bring together our two groundbreaking technologies synergizing in a best-in-class therapy designed to suppress the underlying cause of FSHD,” said Pablo Sardi Global Head of Rare and Neurologic Diseases Research, Sanofi. “We are excited to embark on this collaboration with miRecule as we work together to bring hope to the FSHD community.”

The FSHD Society closely follows develops in FSHD therapy development. Make sure you don't miss important announcements. Sign up below.

Read miRecule’s press release here.

More about miRecule’s technology

Dr. Saleh described miRecule’s research at the 2020 FSHD Connect conference. Watch:

 

Sign Up Today!

Never miss an update

Sign up to receive important news about developments in FSHD, delivered directly to your inbox.

Sign Up for email alerts
Share

Filed Under: FSHD Research

Leave a Reply Cancel reply

Your email address will not be published. Required fields are marked *

SEARCH

Categories

  • FSHD Research
  • FSHD Society News
    • Extraordinary Measures
    • Journey to a Cure
  • Fundraising & Events
  • General
  • Living with FSHD
    • Health and Medicine
    • Mental Health
    • PT and Exercise
    • Tips and Tricks
  • Media & PR

Sign up for Email Alerts

Stay up to date with the latest FSHD news and updates from FSHD Society.

CONNECT WITH US

Proud Member World FSHD Alliance (1)
Footer-Logo-min

General Mailing Address:

75 North Main Street, Suite 1073
Randolph, MA 02368 U.S.A.

Mail Donations to:

Department 960
PO Box 4106
Woburn, MA 01888-4106

(781) 301-6060

  • DONATE
  • Donate
  • Join Us
  • Inclusivity & Equity
  • Privacy Policy
  • Contact Us
  • Glossary Of Scientific Terms
Digital Marketing by