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, FDA, EMA, and biosimilar approvals. He can be contacted at connect@pharmashots.com
