The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy


 Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.

There is considerable enthusiasm surrounding the FDA’s approval. CRD has been developed to treat patients who are too often reminded that there is no cure and therefore no hope. CRISPR technology is presumed to stabilize or even reverse Duchenne’s progression.


The newly created CRD-TMH-001 drug treats exon 1 mutations on the dystrophin gene using CRISPR technology.

What is CRISPR

CRISPR is a technology that can edit genes. It is a way of finding DNA inside a cell.

CRD-TMH-001 is administered one time only. The patient stays in the hospital for a few days to avoid adverse reactions.

Once released, the patient’s progress is followed for fifteen years in accordance with FDA guidelines. Cure Rare Disease is not able to comment once the clinical trial begins, thus protecting the patient’s confidentiality.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy, a rare disease, leads to muscle wasting that worsens over time.  The disease is caused by an alteration (mutation) in the gene coding for the production of the dystrophin protein. Duchenne muscular dystrophy affects all types of muscles and eventually causes cardiac and respiratory failure.

Approximately one in thirty-five hundred male births are affected, yet it is rarely found in female births.

The Partnership

For the past three years, a team of leading scientists, clinicians, and researchers focused their attention on the development of therapies to treat rare neuromuscular disorders. Several laboratories that participated were the Labs at Charles River and Yale University’s Lek Lab.

Richard Horgan is the CEO and founder of Cure Rare Disease. He commented that the approval is just the starting point. He said that Cure Rare Disease plans to follow this approval with additional therapeutics that treat rare diseases and added that they will break barriers to help patients needing effective treatments.

CRD’s CEO hopes that the Medicare & Medicaid Centers appreciate the heavy financial burdens facing people with rare diseases. He suggests using a reimbursement program (fee-for-service) to help rare disease patients.

Now that the FDA has given an all-clear for the onset of the dosing of CRD-TMH-001, it is expected to begin imminently at the UMass Medical School. This is the first-in-human new drug that uses CRISPR-transactivator technology in the treatment of a rare mutation that causes Duchenne muscular dystrophy.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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