#USA #San Diego #Duchenne MD #MyoGene Bio #PPMD Venture

PPMD Invests in Gene Editing Research

In an exciting development for the Duchenne muscular dystrophy (DMD) community, Parent Project Muscular Dystrophy (PPMD) has announced a $400,000 funding initiative directed towards MyoGene Bio. This partnership is a part of PPMD's Venture Pathways program, aimed at accelerating therapeutic advancements for DMD and Becker muscular dystrophy.

Duchenne muscular dystrophy is a severe genetic condition affecting the muscle tissue and is currently diagnosed in approximately one in every 5,000 live male births in the United States. The disorder arises from mutations in the DMD gene, which is responsible for producing dystrophin, a crucial protein that helps maintain muscle integrity. With no potential cure established, innovative solutions like gene editing are becoming increasingly important.

MyoGene Bio has developed an advanced gene editing strategy referred to as MyoDys45-55. This approach specifically targets a significant deletion occurring in exons 45-55 of the DMD gene. By excising this region, which is commonly mutated in the DMD gene, MyoGene aims to create an in-frame deletion that would allow the body to produce a functional dystrophin protein again. This innovative strategy closely mimics a naturally occurring deletion associated with a milder form of muscular dystrophy, Becker muscular dystrophy, which may account for the preservation of muscle function in these patients.

Dr. Eric Camino, Vice President of Research and Clinical Innovation at PPMD, highlighted the organization's ongoing commitment to advancing gene therapy. He stated, "Gene editing offers a revolutionary approach to permanently fix the genetic variations present in DMD patients. It is essential for preparing future treatment options that can significantly enhance patient outcomes."

The funding from PPMD will enable MyoGene to conduct critical experimental studies comparing the behavior of the corrected dystrophin protein against other dystrophin variants. This step is crucial as it will pave the way towards clinical studies, assessing the long-term safety and efficacy of their innovative approach.

Overall, the total financial support for MyoGene sums up to $1.42 million, thanks to contributions from other organizations like Duchenne UK and How Women Invest. This funding not only emphasizes the importance of advocacy support in this field but also aims to attract further investment into MyoGene's ongoing research.

Courtney Young, CEO and Co-founder of MyoGene Bio, expressed gratitude towards PPMD for their vital contribution. "Funding from patient advocacy groups such as PPMD is pivotal for advancing our research initiatives and amplifying the need for innovation within therapeutic development for Duchenne. With this investment, we can leverage our efforts to secure larger grants that will significantly contribute to the advancement of our gene therapy."

PPMD’s financial backing of MyoGene aligns with its rich history of investing over $55 million into various Duchenne and Becker muscular dystrophy research initiatives since its establishment 30 years ago. It displays the organization’s dedication not only to current treatment breakthroughs but also its commitment to future generations facing the challenges posed by these disorders. By investing in biopharmaceutical advancements, PPMD is poised for financial returns that could be reallocated towards research and development within this crucial sector.

As the DMD landscape continues to evolve, partnerships such as PPMD and MyoGene Bio reflect the collaborative spirit needed to expedite the journey toward effective treatment solutions.

For more information about PPMD’s initiatives and ongoing efforts, visit their website or follow them on social media channels. Every contribution is a step towards improving the lives of those affected by Duchenne muscular dystrophy and, ultimately, towards finding a cure.