CureDuchenne Ventures

Exonics Therapeutics: to develop gene-editing therapies for Duchenne; assisted acquisition by Vertex Myotherix: developing therapies to improve muscle function and enhance steroid tolerance 4DMT: to generate novel viral capsids for use in Duchenne gene therapy Avidity: oligonucleotide therapeutics agents for exon skipping to muscle, diaphragm and heart Baebies: accelerate newborn screening for Duchenne muscular dystrophy Bamboo Therapeutics: acquired UNC vector core, worked to facilitate Pfizer acquisition 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 Kevin Flannigan at Nationwide Children’s: to develop U7 gene therapy to treat rare duplication mutations Mesentech: to support development of new therapy to combat bone loss in Duchenne Myosana Therapeutics: to develop a virus-free gene therapy platform for delivering full-length dystrophin PepGen: to develop peptide-conjugated oligonucleotides for enhanced exon skipping in skeletal and cardiac muscle Prosensa: a spin-out from Leiden University and assisted subsequent acquisition by BioMarin PTC Therapeutics: to encourage development of stop-codon read through drugs RASRx: a spin-out from USC seeking to develop novel anti-fibrosis therapy ReveraGen: to develop an anti-inflammatory with less side effects than current glucocorticoids Sarepta: 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 TRiNDS: a neuromuscular focused CRO to accelerate drug development for Duchenne ZappRx: 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