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

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Upwardly to
$1 Million
Will Exist Committed to Fund Enquiry Proposal/s Which Accost Challenges in ‘Get-go Generation’ Gene Therapies and Opportunities in Jail cell-Based and Regenerative Therapies for the Handling of Duchenne Muscular Dystrophy

, /PRNewswire/ —Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to cease
Duchenne muscular dystrophy (Duchenne),  and
Duchenne Great britain, a UK-based patient organisation, are pleased to denote the launch of their 2022 Joint Telephone call for Therapeutic Projects inviting research proposals for submission and review.

Up to
$i one thousand thousand
will exist awarded to up to 2 research projects seeking to develop innovative therapies to treat Duchenne. Through this 2022 Joint Call, the organizations hope to find projects to accost the challenges in ‘first generation’ gene therapies and accept advantage of opportunities in the prison cell-based and regenerative therapies field to ensure new, safer, and more effective treatments achieve the clinic.

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

As the science in the field of cell and cistron therapies continues to advance, Duchenne Great britain and PPMD endeavor to help usher novel therapies forward to gainsay this disease. The 2022 Joint Call seeks to identify those novel jail cell and gene therapies that can address and surmount current challenges and have a clear path to the dispensary and chiefly, the Duchenne community.

PPMD and Duchenne UK invite proposals for ambitious, translational projects driving the next generation of transformative Duchenne therapies which accept the potential to disrupt the electric current therapeutic landscape, leading to marked improvements in the safety, efficacy, and delivery profile over electric current standard of care therapies and therapies currently in clinical evolution. 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 commitment in the dispensary; novel cell-based therapeutic approaches; other regenerative approaches such as those targeting the host muscle stem cells.

PPMD’due south Vice President, Research and Clinical Innovation,
Eric Camino, PhD, and Duchenne UK’due south Director of Inquiry,
Alessandra Gaeta, PhD explained in a articulation 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 more partner with each other and offering this joint phone call of up to
$ane one thousand thousand. The scientific developments in the space of jail cell and gene therapy go on to grow at a rapid footstep. As novel approaches are developed, we want to ensure we are helping shepherd them forth the pathway to translation.”

To submit an awarding for consideration,
please click here to learn more than. All submissions volition be carefully reviewed, with an initial Expression of Interest to exist reviewed by a joint Scientific discipline Review team composed of each organization’due south scientific leads, CEOs, and scientific advisors every bit needed. Subsequently, there volition be a thorough review of proposals past a specially convened peer review panel. By partnering together to place and support these cut-border projects Duchenne United kingdom 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
10 AM ET
iii PM
Uk fourth dimension.
Click here to register to attend.
Participation in this meeting is encouraged but not required for applicants.

About Parent Project Muscular Dystrophy

Parent Projection Muscular Dystrophy (PPMD) is the largest nigh comprehensive nonprofit arrangement in
the United States
focused on finding a cure for Duchenne—our mission is to cease Duchenne.

Nosotros demand optimal care standards and strive to ensure every family has access to expert healthcare providers, cutting-border treatments, and a customs of support. Nosotros invest deeply in treatments for this generation of Duchenne patients and in inquiry that will benefit futurity generations. Our advocacy efforts accept secured hundreds of millions of dollars in funding and won 5 FDA approvals.

Everything we practice—and everything we have washed since our founding in 1994—helps those with Duchenne live longer, stronger lives. We volition not rest until we end Duchenne for every unmarried person affected past the disease. Bring together our fight against Duchenne at
and follow PPMD on Facebook, Twitter,
Instagram, and YouTube.

Nearly Duchenne UK

As the leading Duchenne muscular dystrophy (DMD) charity in the United kingdom, Duchenne UK aims to terminate Duchenne’s devastating bear on. We connect scientists, the pharmaceutical industry, the NHS and families to accelerate and advance 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
Andy Farrell.

For more data about Duchenne UK: visitduchenneuk.org.

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SOURCE Parent Project Muscular Dystrophy (PPMD)

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