CureDuchenne Ventures was formed in 2014 after the successful exit from an equity investment in Prosensa (acquired by BioMarin Pharmaceuticals for $680M).
CureDuchenne’s portfolio includes wide-ranging projects aimed at finding treatments for Duchenne. This includes investments in companies pursuing dystrophin-restoring approaches as well as those developing anti-inflammatory and other mechanisms contributing to the disease. We also look for novel technologies and platform approaches aimed at overcoming the limitations of existing therapies in development. Investments from CureDuchenne Ventures have successfully leveraged more than $3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies, and have resulted in several successful exits.
CureDuchenne Ventures’ impact fund deploys donor dollars to fund Duchenne muscular dystrophy treatments with the singular vision of funding a cure for the entire Duchenne community.
Our robust pipeline of therapies comes from a diverse array of cutting edge technologies used to treat all aspects of Duchenne. CureDuchenne Ventures’ early funding helps Duchenne science attract future investments made by VC firms, biotech companies and pharmaceutical companies.
A successful investment made by CureDuchenne Ventures is a success for the entire Duchenne community. 100% of investment returns into research, resources, and support for families living with Duchenne.
CureDuchenne Ventures, LLC is the investment arm of CureDuchenne, a national nonprofit dedicated to curing Duchenne muscular dystrophy, the most common and lethal form of muscular dystrophy.
FOUNDED AND FUNDED
Collaborative Trajectory Analysis Project:
to enhance Duchenne clinical trial design
to develop gene-editing therapies for Duchenne; assisted acquisition by Vertex
developing therapies to improve muscle function and enhance steroid tolerance
to generate novel viral capsids for use in Duchenne gene therapy
oligonucleotide therapeutics agents for exon skipping to muscle, diaphragm and heart
acquired UNC vector core, worked to facilitate Pfizer acquisition
to support development of cardiac-targeting cell therapy
EVADER technology shields gene therapy virus from the immune system
3DNA gene therapy platform aims to deliver full-length dystrophin and allow for redosing
Dyne’s FORCE™ platform enhances the delivery of exon skipping therapeutics to skeletal, cardiac and smooth muscle with the potential to improve efficacy and reduce dosing frequency
to target metabolic processes in Duchenne muscle to make them more resistant to activity-induced damage
treating devastating diseases with intracellular biologics
their GMAB platform technology efficiently delivers a rance of nucleic acid payloads, including mRNA, DNA, siRNA and ASOs
to develop a t-RNA-based therapeutic to target premature stop codon mutation
next-generation gene therapy to enable lower dose and fuller-length dystrophin
Kevin Flanigan at Nationwide Children’s:
to develop U7 gene therapy to treat rare duplication mutations
LocanaBio: develop AAV-delivered exon-skipping therapies
to support development of new therapy to combat bone loss in Duchenne
to develop a CRISPR/Cas9 gene editing approach to address up to 50% of Duchenne mutations
to develop a virus-free gene therapy platform for delivering full-length dystrophin
to develop peptide-conjugated oligonucleotides for enhanced exon skipping in skeletal and cardiac muscle
a spin-out from Leiden University and assisted subsequent acquisition by BioMarin
to encourage development of stop-codon read through drugs
a spin-out from USC seeking to develop novel anti-fibrosis therapy
to develop an anti-inflammatory with less side effects than current glucocorticoids
when on clinical hold, enabling first Duchenne drug approval by FDA
to support development of their RNA-fix modified CRIPR system to edit mutations in Duchenne RNAne
a neuromuscular focused CRO to accelerate drug development for Duchenne
specialty prescribing application for patients suffering from Duchenne muscular dystrophy
CureDuchenne Ventures supports Duchenne research by using philanthropic donations to encourage development of new Duchenne drugs. Through an impact financing model we can provide both grant funding to academic groups, as well as equity financing to biotech and pharmaceutical companies.
90% of proceeds from equity positions in companies are reinvested to support research into next-generation technologies.
Every dollar donated has the ability to fund multiple projects and have a lasting impact on the treatment of Duchenne. Help us create a self-sustaining fund which will redeploy proceeds from exited positions in companies to fund the next-generation of Duchenne research.
CureDuchenne Ventures’ Impact Model of Funding Research
We find, fund and foster early-stage science using philanthropic dollars. We amplify our funding power by exiting our position in companies and reinvesting those returns into emerging scientific research opportunities.
FIND the next stage Duchenne research that needs additional financial support to make it out of the lab to commercialization.
FUND the science with DONOR dollars through grants, loans, or equity financing.
FOSTER development of the technology with:
- In-house company creation and drug development expertise
- Introduction to Duchenne research and clinical resources
- Aligning of strategic partnerships
- Additional capital investments as needed
- Achieve proof-of-concept and find pharma partners at the right time