Duchenne UK and PPMD Joint Call for Therapeutic Projects 2022

Duchenne UK (DUK), a UK-based patient organization, and Parent Project Muscular Dystrophy (PPMD), a USA-based patient organization, are pleased to announce the launch of our 2022 Joint Call for Therapeutic Projects inviting research proposals for submission and review.

Up to $1 million will be awarded to up to two projects seeking to develop innovative therapies to treat Duchenne. Through this 2022 Joint Call, we 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.

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.” 

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.

What sort of projects are we looking to fund?

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.

Why have we chosen to focus on this area?

While the past few years have seen the continued development of gene therapies that may help slow the progression of the disease, we recognize that 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. We seek 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 this patient community.

Who can apply?

Proposals may be submitted from universities, research institutes, and small for-profit companies worldwide. Please read the Terms & Conditions for detailed information on scope, eligibility, project criteria, and funding.

How to submit your proposal?

Please download our Expression of Interest (EOI) form and submit the completed form to research@duchenneuk.org.

In the forms, applicants must acknowledge that they have read and understood the Terms and Conditions.

Submissions, sent as above, must be received by 23:59pm (UK time) on the 6th of December 2022 at the latest.

How will your submissions be reviewed?

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. 

Informational Webinar: 7th of November @ at 10 AM ET / 3 PM UK

Duchenne UK & PPMD will host an informational webinar detailing the opportunity for interested applicants on the 7th of November 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.

Questions?

For more information please contact research@duchenneuk.org.