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, 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.”

For more information and to submit an application for consideration, visit Parent Project Muscular Dystrophy. 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. Register here to attend. Participation in this meeting is encouraged but not required for applicants.

[Source(s): Parent Project Muscular Dystrophy, PR Newswire]

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