While the drop in Ultragenyx stock certainly provides a lower entry point for new investors, the more prudent choice right ...
Industry data shows small molecules achieve 5–10% approval rates over 15–20 years; antisense oligonucleotides (“ASOs”) are shifting the odds through rational design. Six new antisense drugs gained FDA ...
A Korean research team has successfully demonstrated that antisense oligonucleotide (ASO) therapy can restore hearing in a mouse model of hereditary deafness caused by KCNQ4 mutations, marking a ...
A cross-section from a healthy brain and one with Huntington’s disease. The huntingtin protein causes nerve cells in the brain to break down and die. Jenna El-Attar/ Guest Artist Huntington’s disease ...
HT-KIT triggered significant tumor cell death in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis as early as 24 hours post-treatment, with statistically ...
Hoth Therapeutics Inc. (NASDAQ:HOTH) on Tuesday announced combined findings from multiple preclinical programs evaluating its precision antisense candidate HT-KIT. HT-KIT is a precision antisense ...
Typically, the goal of ALS treatments is to slow the disease or halt progression. But new data from the antisense oligonucleotide (ASO) jacifusen for FUS-ALS show that two patients went beyond the ...
CAMBRIDGE, Mass., April 02, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an ...
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