The nonprofit group CureDuchenne won $50,000 at a recent Shark Tank-like competition in Irvine, California, to pitch scientific projects.
CureDuchenne won the prize after co-founder Debra Miller presented Eric Olson’s CRISPR/Cas9 gene editing research, which CureDuchenne is sponsoring and which could be transformational for patients with Duchenne muscular dystrophy (DMD).
Debra and Paul Miller founded CureDuchenne after their son was diagnosed with DMD. Today, the organization leads DMD-specific physical therapy plans, funds human clinical trials and fights DMD in many other ways.
“It is very humbling to think that we might actually play a role in finding a new treatment that would help our son and other children like him,” Debra Miller, CEO of CureDuchenne, said in a press release. “There’s a huge unmet need for new therapies like gene editing and we need to work together and take advantage of every resource to accelerate the discovery process.”
The pitch competition closed the inaugural RARE Partnering and Investor Forum, part of Global Genes’ 6th Annual RARE Patient Advocacy Summit. Besides CureDuchenne, other competitors included Boston’s Children Hospital and its subsidiary, Myos; Chameleon Biosciences; Cincinnati Children’s Hospital; Circumvent Pharmaceuticals and Orphagen Pharmaceuticals.
“We are thrilled to sponsor competitions like this that demonstrate that the Scientist.com marketplace can help anyone — a concerned parent, a patient, an academic researcher or an industry scientist — plan and run research experiments and develop new cures,” added Scientist.com CEO Kevin Lustig. “Scientist.com was designed to democratize science.”
The event was hosted by Morrie Ruffin, manager of the Alliance for Regenerative Medicine. The judges who awarded CureDuchenne were Graeme Martin, Jeremy Springhorn, Kush Parmar and Toby Freyman.
“The people in this community are all extraordinary. They’re all champions,” said Global Genes CEO Nicole Boice. “When we all work together, patients win, companies win and researchers win because it takes a village to cure a rare disease.”