LAMA1

Modalis Therapeutics Reports Data Supporting Development of a Transformative Epigenome Editing Therapeutic, MDL-101: a First-in-Class Epigenome Editing approach for the Treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2-CMD)

Retrieved on: 
tisdag, maj 7, 2024
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Dicarboxylic acid, Metadata, DSM-IV codes, CRISPR, DOI, Safety, LAMA1, LAMA2, AAV, Tokyo Stock Exchange, DNA, Therapy, Patient, Congenital muscular dystrophy, Gene expression, Manuscript, BioRxiv, Vaccine

The company reported data demonstrating exceptional durability, robust efficacy and safety in dyW mouse disease model of LAMA2-CMD and in adult and juvenile NHPs.

Key Points: 
  • The company reported data demonstrating exceptional durability, robust efficacy and safety in dyW mouse disease model of LAMA2-CMD and in adult and juvenile NHPs.
  • LAMA2-CMD is a severe, early onset congenital muscular dystrophy caused by the absence of the LAMA2 protein.
  • No approved therapies to address the root cause of this condition exist or are in clinical trials.
  • "We are thrilled to post our comprehensive preclinical data supporting the development of MDL-101 on bioRxiv.