Modalis Therapeutics presented supportive preclinical data of MDL-101 in the preprinted paper “Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo” on bioRxiv, to treat LAMA2-CMD
In the preclinical study, a single dose of MDL-101 using MYOAAV (an AAV vector) elevated LAMA1 in skeletal muscles for 12 mos., improved muscle pathophysiology, prolonged lifespan, and increased body weight of dyW mouse
In adult & juvenile NHPs, MYOAAV version MDL-101 has shown enhanced muscle tropism and induced LAMA1 to function restoration level across skeletal muscle tissues without AEs, depicting its clinical efficacy
Ref: Modalis Therapeutics | Image: Modalis Therapeutics
Related News:- X4 Pharmaceuticals’ Xolremdi Receives the US FDA’s Approval for the Treatment of WHIM Syndrome
PharmaShots! Your go-to media platform for customized news ranging for multiple indications. For more information connect with us at connect@pharmashots.com
