Myonexus Therapeutics has announced that it has secured $2.5 million to advance limb girdle muscular dystrophy (LGMD) gene therapies into the clinic. This seed financing will enable Myonexus to initiate a phase 1/2a clinical trial testing MYO-101, the company’s LGMD2E gene therapy, in early 2018.
Myonexus’ pipeline also includes MYO-102, a LGMD2D gene therapy that is currently in phase 1/2a clinical trial, and MYO-201, a LGMD2B gene therapy currently in phase 1 clinical trial. The company is also developing gene therapies for LGMD2C and LGMD2L, which are currently undergoing preclinical testing.
Dr Bruce Halpryn, Chief Operating Officer at Myonexus, said:
There are currently no approved treatments for the limb-girdle muscular dystrophies. Initial LGMD human clinical studies demonstrated expression in muscles exposed to MYO-102 and MYO-201. MYO-101’s Phase 1/2a clinical trial represents the first intravenous systemic exposure, potentially providing the first evidence of functional improvements in LGMD patients following treatment with these gene therapies.
Myonexus’ gene therapies use an adeno-associated virus (AAV) to deliver the desired gene into the body (you can read more about this in our AAV feature article). They were originally developed by Dr Louise Rodino-Klapac and Dr Jerry Mendell and their research teams at Nationwide Children’s Hospital. Both researchers continue to work closely with Myonexus, with Dr Rodino-Klapac serving as its Chief Scientific Officer and Dr Mendell as a clinical development consultant.
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