Modalis Therapeutics’ MDL-101 Gains the US FDA’s Orphan Drug Designation to Treat Congenital Muscular Dystrophy Type 1A (LAMA2-CMD)
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- The US FDA has granted orphan drug designation to the company’s MDL-101 for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD)
- MDL-101 is an experimental therapy aimed at treating LAMA2-CMD through epigenetic editing. This therapy has the potential to deliver a one-time, lasting treatment for individuals affected by LAMA2-CMD.
- Modalis' CRISPR-GNDM technology provides an innovative method for modulating gene expression without altering the patient's DNA. MDL-101 is designed to meet an unmet medical need by stimulating the expression of the related gene LAMA1 in muscle tissues, thus compensating for the impaired function of LAMA2
Ref: Modalis | Image: Modalis
Related News:- Modalis Therapeutics Unveils Epigenome Editing Therapy MDL-101 for Treatment of LAMA2-Deficient CMD
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A passionate content writer with expertise in delivering high-quality and engaging content, Dipanshu is a keen reader and a versatile writer. Dipanshu dedicatedly covers news ranging from biopharma, life sciences, biotech, and MedTech to diagnostics and animal health companies. He can be contacted at connect@pharmashots.com
