Charley's Fund

Summit is a UK-based company focused on the discovery and development of novel drug candidates to treat areas of high unmet medical need.  One of two focus areas is Duchenne Muscular Dystrophy.  Summit is developing a drug candidate that is designed to upregulate utrophin, a protein that can compensate for lack of dystrophin.  Charley’s Fund, [...]

Halo Therapeutics was founded to acquire and develop a powerful anti-fibrotic drug that shows promise as a treatment for Duchenne and other orphan diseases. In animal studies, halofuginone has delayed and in some cases even reversed fibrosis of the muscles, a major factor in the downstream pathology of Duchenne. The sole owners of Halo Therapeutics [...]

Charley’s Fund has collaborated with Galapagos, a Belgian biotech company, to develop the company’s SARM drug (selective androgen receptor modulator) as a Duchenne treatment. Galapagos was already developing the drug for cachexia (cancer-induced muscle wasting). Charley’s Fund supported an efficacy study and a dose ranging study in the mdx mouse to determine if this SARM [...]

This NJ-based biotech company is developing oral medications that may delay muscle degeneration in Duchenne. Several classes of compounds have been identified for four protein targets and are being optimized for safety and efficacy.