One Million US Dollars Committed to Fund Research Proposal/s Which Support Enhancing Gene Therapy Delivery for the Treatment of Duchenne Muscular Dystrophy

 June 2, 2020

Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), and Duchenne UK, a UK-based patient organization, are pleased to announce the launch of their joint 2020 Research Grant Call, inviting research proposals for submission and review. Up to $1 million (US) will be awarded to a project or projects seeking to minimize the immunological issues of gene therapy to treat Duchenne.

Over the last few years, there has been huge progress in the treatment of the root cause of Duchenne through the delivery of replacement (micro) dystrophin using gene therapy. Several gene therapy programs are now being tested in clinical trials on patients with Duchenne. Although it is still early stages, there is evidence that the presence of microdystrophin protein is detected in treated patients. 

A common feature of all these gene therapy programs is their use of a virus to deliver the therapeutic material. Challenges can arise from an individual’s immune system preventing safe and successful delivery.

A significant number of patients have a natural immunity to the virus (and so are ineligible for treatment), but even those without immunity will develop it after the first administration, making second dosing (if required) very difficult.

We hope to find projects through our Grant Call to advance the understanding of the best ways to safely, effectively, and repeatedly deliver gene therapies to the muscles where they are needed.

PPMD and Duchenne UK invite proposals which are investigating ways to mitigate the immune response both in terms of pre-existing immunity and immunity induced by receiving a gene therapy dose. These may focus on ‘damping down’ or ‘circumventing’ the immune system itself or may be investigating alternative delivery methods that promote a minimal immune response.

PPMD’s Founding President & CEO, Pat Furlong, and Duchenne UK’s CEO, Emily Crossley explained in a joint statement:

Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD. We are pleased to partner with each other and offer this grant of $1 million (US). 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. 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.

To submit an application for consideration, please click here to learn more.

All submissions will be carefully reviewed, initially by a joint Science Review team and by the CEOs from DUK and PPMD. Subsequently there will be a thorough review of proposals by a specially convened Scientific Advisory Board.

By partnering together to identify and support these cutting-edge projects Duchenne UK and PPMD seek to accelerate the timeline of translation to patients in need of these exciting therapies.

About Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne—our mission is to end Duchenne. 

We demand optimal care standards and strive to ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won three FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org and follow PPMD on Facebook, Twitter, Instagram, and YouTube.

About Duchenne UK

Duchenne UK has one clear aim – to end Duchenne. We are the largest funder of Duchenne research in the UK. We connect leading researchers with industry, governing bodies and families to challenge every stage of drug development. Together, we will find treatments and a cure for this generation of patients with Duchenne.

For more information about Duchenne UK: visit www.duchenneuk.org.

CONTACT:      Will Nolan, SVP of Communications (PPMD): [email protected]

                        Molly Hunt, Communications Manager (Duchenne UK) at [email protected]

ENDS