CureDuchenne’s mission is to improve the lives of everyone affected by Duchenne through accelerating research to find the cure, improving care and empowering the Duchenne community. Duchenne is a devastating muscle disease and is the most common form of muscular dystrophy, occurring in approximately one in every 5,000 male births. Those affected with Duchenne lose their ability to walk, feed and breathe independently, and eventually succumb to heart and respiratory failure.
However, gene therapies provide hope for the Duchenne community. CureDuchenne is working to support researchers in the space via their Biobank program as well as families living with Duchenne with their Cares program. CureDuchenne is bringing the community together to provide interactive education and outreach to help manage the challenges of Duchenne.
CureDuchenne Senior Director of Advocacy, Erin Frey, explains how the organization is working to fuel hope for Duchenne patients and families:
What does the future of gene therapy look like for Duchenne patients?
We’re seeing exciting advancements when it comes to gene therapies for Duchenne patients. Science is moving at a rapid speed. However, we must work to ensure our policies keep pace - there are a number of challenges we need to address. This is why we are working with IGT to ensure deeper consideration by federal agencies of the Duchenne community’s insights throughout the drug development process and beyond, into the assessment of value to help ensure coverage. Eventually, when there is an approval of a gene therapy to treat Duchenne, the Duchenne community will need access to them and CureDuchenne will be there to help them navigate access.
What investments has CureDuchenne made in the realm of gene therapy?
CureDuchenne Ventures focuses on vetting and funding early-stage Duchenne science, to then be the catalyst for the projects we've funded to attract the additional investment needed to bring that science to clinical trials and approval. We hope to accelerate the drug development pipeline, with a vision of bringing transformative treatments and cures for everyone with Duchenne. Standout projects include:
· In 2016, we funded Bamboo Therapeutics to support IND-enabling studies and scale-up of vector manufacturing resulting in an acquisition by Pfizer that has enabled it to progress to a phase 3 clinical trial for a gene therapy to treat Duchenne .
· In 2017, CureDuchenne co-founded a company, Exonics Therapeutics, with Dr. Eric Olson at University of Texas Southwestern. CureDuchenne provided the seed funding to enable Exonics to achieve strong proof of concept for its CRISPR Cas9 gene editing research for Duchenne. In 2019, Vertex Pharmaceuticals acquired Exonics and is working toward clinical trials.
· In 2018/2019, CureDuchenne funded preclinical research which seeks to overcome the limitations of current gene therapy technologies: 4D Molecular Therapeutics, Chameleon Biosciences and Myosana Therapeutics. These companies are investigating methods to avoid or overcome immune reactions to current AAV-based therapies and deliver a more robust gene.
Can you share more about the importance of newborn screening for Duchenne?
Screening has proven to improve health outcomes for every disease that is screened for. Now that dystrophin-restoring exon skipping is a possible treatment path in some cases of Duchenne, steroid use at an early age has become standard of care, and gene therapy may be arriving in the coming years, it’s even more important to identify children who can be helped before the disease begins to show symptoms at all. Having been involved with newborn screening in a myriad of ways over the last several years, we see it as the best way to find everyone who has Duchenne as early as possible.
We are actively engaged in the effort to determine the best ways to modernize the newborn screening public health program to keep pace with innovation, and supportive of federal funding efforts for newborn screening and the eventual reauthorization of the Newborn Screening Saves Lives Act.
As a Patient Advocate Advisory Council member, what do you hope IGT will accomplish?
We see the Institute for Gene Therapies as a highly motivated and nimble organization that convenes innovative leaders from industry, research, and patient advocacy. There is great value in IGT providing the space for those leaders to engage in robust discussion to arrive at focused strategic policies that will deliver tangible outcomes that benefit families.
CureDuchenne is working to drive real change for those with Duchenne muscular dystrophy and their loved ones, pioneering education, and support programs. We know IGT is here to help.