CureDuchenne Ventures

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.


Collaborative Trajectory Analysis Project:
to enhance Duchenne clinical trial design

Exonics Therapeutics:
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

Bamboo Therapeutics:
To support development of a microdystrophin gene therapy for Duchenne, and expand manufacturing capabilities.  Bamboo and its Duchenne gene therapy program was acquired by Pfizer in 2016. 

Capricor Therapeutics:
to support development of cardiac-targeting cell therapy

Chameleon Biosciences:
EVADER technology shields gene therapy virus from the immune system

Code Biotherapeutics:
3DNA gene therapy platform aims to deliver full-length dystrophin and allow for redosing

Dyne Therapeutics:
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

Edgewise Therapeutics:
to target metabolic processes in Duchenne muscle to make them more resistant to activity-induced damage

Entrada Therapeutics:
treating devastating diseases with intracellular biologics

Gennao Bio:
their GMAB platform technology efficiently delivers a rance of nucleic acid payloads, including mRNA, DNA, siRNA and ASOs

hC Bioscience:
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

Myosana Therapeutics

Myosana Therapeutics:
to develop a virus-free gene therapy platform for delivering full-length dystrophin

PepGen Logo

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

PTC Therapeutics:
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

Shape Therapeutics:
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.

Impact Model

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

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Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.