NEWPORT BEACH — CureDuchenne has made a second investment into Tevard Biosciences Inc. to push forward a therapy with potential to restore muscle-function proteins.
Boston-based Tevard is pursuing a treatment targeting a subgroup—representing about 12% of the Duchenne population—with a mutation resulting in shortened, unfinished proteins. No approved treatments exist for this mutation type.
CureDuchenne made its first investment in Tevard in 2023 after a prior drug, developed by PTC Therapeutics, failed to meet the FDA’s and European regulators’ criteria for regulatory approval.
“The FDA just pulled the plug on it completely, so we started talking with Tevard a few years back and they were addressing the same population with a potentially much more effective treatment,” Debra Miller, co-founder and chief executive of CureDuchenne, told the Business Journal.
Officials declined to disclose the amount of the investment.
Therapy Restores 70% of Key Muscle Protein
Duchenne muscular dystrophy is a progressive muscle-wasting disease, affecting 1 in every 5,000 male births.
The root cause of the disease is genetic mutations that affect the production of dystrophin, a protein essential for muscle function. By the age of 10 to 12, many children with Duchenne need a wheelchair for mobility and don’t live past their 20s.
Tevard’s suppressor transfer RNA (tRNA) platform aims to bypass the mutation and restore production of full-length proteins.
Last September, the company presented preclinical data showing an average of 70% restoration of dystrophin protein in mice.
“We’ve never seen as much as 70% dystrophin restoration in a Duchenne individual before,” said CureDuchenne’s Chief Scientific Officer Dr. Michael Kelly. “If this works as well in humans as it is doing in mice, then this will be absolutely transformative for those patients.”
CureDuchenne’s investment will help the company submit an Investigational New Drug (IND) application before it progresses to human clinical trials, according to Kelly.
“They’ve got all of their ducks lined up scientifically, and it’s a matter of now just moving it through,” Kelly said.
Portfolio Companies Near Finish Line
Tevard is just one of several companies CureDuchenne has invested in nearing potential drug approvals.
Miller said three to four of the companies they’ve invested in are close to having an approved drug within the next 12 months.
The closest to approval is Capricor Therapeutics’ investigational cell therapy that’s intended to improve heart and muscle function in Duchenne patients.
“It’s enough to hopefully slow down this disease,” Miller said, whose son Hawken has Duchenne.
Capricor has an FDA target action date of Aug. 22 after the agency previously declined to approve its Biologics License Application last year.
Another company that could secure approval before year-end is San Diego-based Avidity Biosciences Inc..
Miller said the company has reported “tremendous results” with its exon-skipping technology. The therapeutic approach directs cells to bypass faulty sections of a gene, allowing them to produce a shorter but functional version of a protein.
“It’s probably the most effective drug in-human we’ve seen so far,” Miller said.
CureDuchenne has also invested in Boulder, Colorado-based Edgewise Therapeutics. While the company is not up for approval, it’s expected to complete its pivotal trial this year for Becker muscular dystrophy (BMD)—a milder form of Duchenne. It would be the first approved treatment for BMD.
In total, the nonprofit has invested $27 million in 51 different research projects to date, according to its 2025 annual report.
Beyond clinical efforts, CureDuchenne opened a second clinic at Rady Children’s Health Orange County, formerly CHOC, last September and is committing nearly $1.5 million over three years to fund the clinic, as well as expand clinical trials and research for Duchenne and Becker muscular dystrophy—a milder form of Duchenne.
“One would think that in this affluent area of Orange County that there might not be an unmet need, but Rady addresses the Inland Empire all the way down through San Diego, so it’s a real blessing to have this clinic in a drivable distance for them,” Miller said.
