$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to $1 million in funding for gene therapy research in Duchenne muscular dystrophy (DMD).

The organizations are specifically looking for proposals that address the immune system’s response to gene therapy, a major challenge to the overall success of the technique.

“Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD,” Emily Crossley, Duchenne UK’s CEO, and Pat Furlong, CEO of PPMD, said in a joint statement. “We are pleased to partner with each other and offer this grant of $1 million (US).”

DMD is characterized by the breakdown of muscle fibers over time. The disorder is caused by a mutation in the DMD gene, leading to no production or small amounts of functional dystrophin, a protein with a key role in muscle fiber growth and protection.

Gene therapy is meant to provide a source of the normal DMD gene to patients’ muscle cells, ultimately aiming to restore production of functional dystrophin.

A number of gene therapies are currently being investigated to treat DMD. Some of these approaches are designed to produce so-called micro-dystrophin, which is a shorter but functional version of dystrophin. Examples include SRP-9001 by Sarepta Therapeutics, SGT-001 by Solid Biosciences, and PF-06939926 by Pfizer.

Another potential gene therapy, called GALGT2 and also developed by Sarepta, aims to enhance the expression of other proteins essential for muscle function to compensate for reduced dystrophin. This different approach makes GALGT2 a surrogate gene therapy.

All current gene therapy candidates for DMD require the use of a virus to deliver the healthy DNA into the patients’ cells. Although the viruses have been modified to be harmless, patients may have natural immunity against the specific viral vectors used in the therapies, rendering the treatments ineffective.

Such patients are ineligible for treatment, but others may develop immunity after the first gene therapy dose, which represents a significant obstacle to continued treatment.

Duchenne UK and PPMD intend to fund research that addresses these concerns, including approaches that suppress or circumvent the immune system, or those that focus on a new method of delivery.

“Gene therapy is offering great promise, but there are challenges associated with the immune response which are limiting the rate of progress and a barrier to ensuring all patients can have access to these potentially transformative therapies,” said Crossley and Furlong. “We hope that this call will encourage groups working in this area to submit applications and engage with us in bringing new treatments to the clinic.”

Scientists seeking to apply can find more information here. All applications will be reviewed initially by a team with members from both organizations, followed by a scientific advisory board for a more thorough review.

By collaborating in this funding call, the two organizations hope to expedite the development of gene therapies for people with DMD.