- December 24, 2021
- Posted by: Stratford Team
- Category: Business
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Validated ability to Drug the Genome™ to address both rare and common diseases without the limitations of early precision genetic medicine technologies; the Company’s delivery shuttle enables pharmacology across programs in multiple tissues, including in the brain and muscle, after subcutaneous administration
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Nominated the development candidate for the myotonic dystrophy type 1 (DM1) program with potential for best-in-class features; initiated IND-enabling studies; and expect to submit an IND filing to the FDA in 4Q CY2022
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Established clinical development and CMC teams at new Cambridge, Mass. site; finalized formulation for systemic routes; and scaled-up manufacturing to support GLP toxicology and Phase 1/2 clinical trials for the DM1 program
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Expect to initiate scale-up and toxicology activities for the development of a systemically administered allele-selective NT-0100 program to treat Huntington’s disease (HD) in CY2022; and targeting an IND filing to…
