Research Area
MyoGene Bio is developing MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy, a devastating muscle wasting disease that leads to premature death. MyoDys45-55 uses AAV delivery of CRISPR to delete DMD exons 45-55 as a way to restore the reading frame and permanently remove mutations for 50% of Duchenne patients. This results in an internally deleted dystrophin protein that has been associated with a very mild disease course in Becker muscular dystrophy and retains 3x more of the protein coding sequence than other types of gene therapy. MyoGene is currently in preclinical development working to translate this therapy to patients as quickly and safely as possible.
Team Description
MyoGene Bio is a small biotech startup co-founded by Drs Courtney Young, Melissa Spencer and April Pyle out of UCLA. Dr Young is CEO and manages a team of 4 people including a program manager and regulatory expert, a research scientist, and two lab technicians. Drs Spencer and Pyle are advisors and collaborators.
Team Members
Team Show
Publications
1. Young CS, Mokhonova E, Quinonez M, Pyle AD, Spencer MJ. Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy. J Neuromuscul Dis. 2017;4(2):139-145. doi: 10.3233/JND-170218. PMID: 28505980; PMCID: PMC5565771.
2. Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell. 2016 Apr 7;18(4):533-40. doi: 10.1016/j.stem.2016.01.021. Epub 2016 Feb 11. PMID: 26877224; PMCID: PMC4826286.