CureDuchenne Ventures was formed in 2014 after the successful exit from an equity investment in Prosensa (acquired by BioMarin Pharmaceuticals for $680M).

As of 2017, CureDuchenne’s portfolio includes 12 wide-ranging projects with 3 successful exits. Investments from CureDuchenne Ventures have successfully leveraged more than $1.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne.

Mission: 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 earnings are re-invested back into Duchenne research to further the goal of a cure.

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

  • Exonics Therapeutics: developing gene-editing therapies for Duchenne
  • Myotherix: developing therapies to improve muscle function and enhance steroid tolerance

FUNDED

  • TRiNDS: a neuromuscular focused CRO to accelerate drug development for Duchenne
  • Bamboo Therapeutics: acquired UNC vector core, worked to facilitate Pfizer acquisition
  • RASRx: a spin-out from USC seeking to develop novel anti-fibrosis therapy
  • Collaborative Trajectory Analysis Project: to enhance Duchenne clinical trial design
  • Kevin Flannigan at Nationwide Children’s: to develop U7 gene therapy to treat rare duplication mutations
  • Prosensa: a spin-out from Leiden University and assisted subsequent acquisition by BioMarin
  • Sarepta: when on clinical hold, enabling first Duchenne drug approval by FDA
  • PTC Therapeutics: to encourage development of stop-codon read through drugs

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.

100% 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 funding opportunities range from $50K-$5M (depending on project needs). In addition to financing, we have extensive knowledge on partnerships and collaborations with the FDA, pharmaceutical, and biotechnology industry.

TYPES OF FUNDING:

  • Grants
  • Loans
  • Equity Financing

Objective 1

Fund the totality of Duchenne’s expression to identify novel biological targets, pharmaceutical assets, and research programs that exhibit the most potential to advance rapidly to clinical trial proof of concept studies.

Objective 2

Treat the whole disease by sponsoring a variety of approaches that are needed to treat Duchenne’s many genetic variants, and because of the complexity of the human genome, a variety of approaches are needed to develop the most effective treatments. Without the protein dystrophin, inflammation will lead to the infiltration of fat, scar, or fibrous tissue in muscle. The most life threatening damage can be to the heart and diaphragm muscles, resulting in cardiac and respiratory failure as the leading causes of death for those with Duchenne. In addition, Duchenne boys suffer from a loss of independence resulting from fibrosis leading to loss of ambulation and reduced mobility. Consequently, in addition to supporting research aimed at replacing the missing dystrophin protein; CureDuchenne Ventures has backed projects for heart failure, respiratory failure, anti-inflammatory, and anti-fibrotic treatments.

Objective 3

Develop new biomarkers, statistical models, and clinical endpoints to facilitate drug development through financing and strategic partnerships with leading experts, scientists and companies in the industry.

As of 2017, CureDuchenne’s portfolio includes 12 wide-ranging projects with 3 successful exits. Investments from CureDuchenne Ventures have successfully leveraged more than $1.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne.

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

FIND the next stage Duchenne research that needs additional financial support to make it out of the lab to commercialization.

FUND

FUND the science with DONOR dollars through grants, loans, or equity financing.

FOSTER

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

Portfolio

Research Focus
Show All
Anti-fibrosis
Anti-inflammatory
Gene Therapy
Cardiac Function
Clinical Trial Design
Gene Editing/CRISPR
Clinical Trial Support
Statistical Analysis
Stop Codon Read-Through
Utrophin Upregulation