Duchenne UK and Parent Project Muscular Dystrophy Announce 2022 Joint Call for Therapeutic Projects to Find Transformative Treatments for Duchenne Muscular Dystrophy

News provided by

Parent Project Muscular Dystrophy (PPMD)

Oct 26, 2022, 10:00 ET

Up to $1 Million Will Be Committed to Fund Research Proposal/s Which Address Challenges in 'First Generation' Gene Therapies and Opportunities in Cell-Based and Regenerative Therapies for the Treatment of Duchenne Muscular Dystrophy

WASHINGTON, Oct. 26, 2022 /PRNewswire/ -- 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 2022 Joint Call for Therapeutic Projects inviting research proposals for submission and review.

Up to $1 million will be awarded to up to two research projects seeking to develop innovative therapies to treat Duchenne. Through this 2022 Joint Call, the organizations hope to find projects to address the challenges in 'first generation' gene therapies and take advantage of opportunities in the cell-based and regenerative therapies field to ensure new, safer, and more effective treatments reach the clinic.

While the past few years have seen the continued development of gene therapies that may help slow the progression of the disease, there remain many barriers to these novel genetic therapies being available to all individuals living with Duchenne. Challenges with the safe delivery of viral-based gene therapy and the efficacy of those potential strategies remain paramount.

As the science in the field of cell and gene therapies continues to advance, Duchenne UK and PPMD endeavor to help usher novel therapies forward to combat this disease. The 2022 Joint Call seeks to identify those novel cell and gene therapies that can address and surmount current challenges and have a clear path to the clinic and importantly, the Duchenne community.

PPMD and Duchenne UK invite proposals for ambitious, translational projects driving the next generation of transformative Duchenne therapies which have the potential to disrupt the current therapeutic landscape, leading to marked improvements in the safety, efficacy, and delivery profile over current standard of care therapies and therapies currently in clinical development. These will involve novel genetic therapeutic approaches, including: gene therapy and gene editing, that use delivery models and strategies that address the current challenges of using viral delivery in the clinic; novel cell-based therapeutic approaches; other regenerative approaches such as those targeting the host muscle stem cells.

PPMD's Vice President, Research and Clinical Innovation, Eric Camino, PhD, and Duchenne UK's Director of Research, Alessandra Gaeta, PhD 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 once again partner with each other and offer this joint call of up to $1 million. The scientific developments in the space of cell and gene therapy continue to grow at a rapid pace. As novel approaches are developed, we want to ensure we are helping shepherd them along the pathway to translation."

To submit an application for consideration, please click here to learn more. All submissions will be carefully reviewed, with an initial Expression of Interest to be reviewed by a joint Science Review team composed of each organization's scientific leads, CEOs, and scientific advisors as needed. Subsequently, there will be a thorough review of proposals by a specially convened peer review panel. 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.

The organizations will host an informational webinar detailing the opportunity for interested applicants on November 7th at 10 AM ET / 3 PM UK time. Click here to register to attend. Participation in this meeting is encouraged but not required for applicants.

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 five 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

As the leading Duchenne muscular dystrophy (DMD) charity in the UK, Duchenne UK aims to end Duchenne's devastating impact. We connect scientists, the pharmaceutical industry, the NHS and families to advance and accelerate medical research, while bringing everyone affected by DMD the care and support they need.

Our president is Her Majesty The Queen Consort. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

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

SOURCE Parent Project Muscular Dystrophy (PPMD)