Cart
×
Funding the science. Advancing the cure.
Since 2014, CureDuchenne Ventures has applied rigorous scientific expertise to identify, fund, and advance the most promising Duchenne muscular dystrophy research, from early-stage discovery through FDA approval. That investment is paying off: approved therapies exist where none did before, a historic pipeline of new treatments is approaching regulatory review, and individuals living with Duchenne are outliving every previous generation. This is what happens when donor dollars are guided by science.
|
$28M+
invested in 53 Duchenne research programs since 2003
|
$170
in biotech/pharma investment for every $1 CureDuchenne commits*
|
4
drugs pursuing FDA approval in 2026/2027 were directly supported by CureDuchenne
|
*For every $1 CureDuchenne invests, an additional $170 flows into those companies from other investors, not to CureDuchenne directly.
Over a decade of impact.
Our scientific team has evaluated over a thousand programs and invested in 53 research projects across the Duchenne landscape, from exon skipping to gene therapy to next-generation steroids to cardiac treatment. Some of those investments have yielded FDA-approved therapies. Others have attracted acquisition by major pharmaceutical companies, validating the science and bringing it to larger development pipelines. Others have advanced the fundamental understanding of Duchenne biology in ways that make the next generation of treatments possible.
CureDuchenne supported companies responsible for 6 of the 8 FDA-approved Duchenne therapies. Four more CureDuchenne-backed therapies are approaching FDA review in 2026 and 2027.
The pipeline is advancing.
More CureDuchenne-backed programs are nearing FDA review. Four CureDuchenne-backed therapies are approaching FDA review in 2026 and 2027, the most concentrated period of Duchenne drug approvals in history. Each represents a different scientific approach, together expanding the population of individuals who may benefit from treatment, including, for the first time, individuals with Becker muscular dystrophy.
|
PDUFA: Aug 22, 2026
Capricor Therapeutics: DeramiocelMutation-agnostic (broad population) CureDuchenne Ventures invested $1.5 million in Capricor in 2015. If approved, deramiocel would be the first Duchenne therapy approved by the FDA for its demonstrated benefits to both heart and skeletal muscles. Because it is mutation-agnostic, it has the potential to benefit nearly all individuals with Duchenne. |
Filing 2026
Avidity Biosciences/Novartis: Del-zotaExon 44 (~7% of Duchenne population) CureDuchenne Ventures invested in Avidity Biosciences in 2018, supporting development of an antibody-oligonucleotide platform that dramatically improves exon-skipping therapy delivery to muscles. Del-zota has demonstrated approximately 32% dystrophin restoration in clinical trials. Avidity was acquired by Novartis for $12 billion in 2025. |
|
Filing 2026
Dyne Therapeutics: Z-rostudirsenExon 51 (~11% of Duchenne population) CureDuchenne Ventures invested $2 million in Dyne Therapeutics in 2020, supporting the FORCE platform, which uses an antibody fragment to target exon-skipping therapies directly to skeletal, cardiac, and smooth muscle, with substantially improved dystrophin restoration compared to first-generation therapies. |
FDA filing anticipated 2027
Edgewise Therapeutics/Servier: SevasemtenDuchenne and Becker muscular dystrophy, mutation-agnostic (broad population) CureDuchenne Ventures invested in Edgewise Therapeutics in 2019, supporting development of sevasemten, a first-in-class muscle-stabilizing therapy that protects muscle fibers from damage regardless of genetic mutation. If approved, sevasemten would be the first therapy for Becker muscular dystrophy, addressing a critical unmet need. Servier acquired Edgewise’s sevasemten programs in 2026. |
Additional Duchenne therapies are also approaching FDA review that were not funded by CureDuchenne Ventures. CureDuchenne Ventures provides advice and guidance to all companies throughout their therapeutic development process, and CureDuchenne also helps the community stay informed about all programs in development through webinars, clinical trial education, and 1:1 family guidance.
Progress is real. But no treatment yet stops Duchenne in its tracks.
More individuals with Duchenne are living longer than ever before, and CureDuchenne’s scientific investment has helped make that possible. But current therapies slow this disease; they do not stop it. Too many are still waiting for treatments that address their specific biology, and a cure remains out of reach. The next wave, redosable gene therapy, next-generation combination approaches, has the potential to reach everyone. We will not stop until it does. Let’s keep going. Because every child deserves the chance to grow up.