Epigenetic Editing with CRISPR-GNDM: MDL-101 is a Novel Muscle-Tropic AAV Vector for the Treatment of LAMA2-CMD

Time: 9:00 am
day: Conference Day Two


  • MDL-101 demonstrates promising efficacy in dyW mouse disease model of LAMA2-CMD, with optimal vector biodistribution, robust LAMA1 expression and significant improvement in the disease phenotype
  • MDL-101 demonstrates a range of safety profiles in NHPs, exhibiting strong biodistribution to skeletal muscles, robust expression of GNDM, and therapeutically significant induction of LAMA1 mRNA and protein
  • With the successful completion of Process Development for MDL-101 vector production and IND-enabling discussions with the FDA, MDL-101 stands poised to advance toward clinical development