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Dyne Reports on Response Predictive Biomarkers for Neurodegenerative Disorder Genetic Medicine
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The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.
Hoth Therapeutics Eyeing Human Trials for HT-KIT Antisense Therapy in Early 2026
After seeing promising results in preclinical studies, the firm hopes to begin Phase I trials shortly after getting IND clearance.
Isarna to Discuss ASO for Wet AMD, Diabetic Macular Edema With Regulators
Phase II trial results indicated that treatment with the firm's antisense oligonucleotide led to stable or improved best-corrected visual acuity.
FD Foundation Spinout Tikun Therapeutics Charges Ahead Studying Therapies for Familial Dysautonomia
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The New York-based public benefit corporation is advancing three drug candidates for the extremely rare condition.
Amylyx Doses First Patient in Phase I Trial of ASO to Treat AML
The company expects early cohort data this year from the trial assessing the calpain-2-targeted antisense oligonucleotide therapy.
Sep 10, 2024
Ionis Pharmaceuticals Prices $500.3M Stock Offering
Apr 8, 2024
Apr 4, 2024
Oct 30, 2023