Dec 14, 2022, 07:00 ET AOC 1001 delivered siRNA to skeletal muscle and produced meaningful DMPK reduction in 100% of participants with a 45% mean reduction in DMPK after a single dose of 1 mg/kg or two doses of 2 mg/kg AOC 1001 produced splicing improvement of…
A new review outlines how lipid-based nanomedicines could deliver pathway-specific siRNA to treat hepatocellular carcinoma, highlighting delivery design, preclinical candidates and clinical constraints.
Oppenheimer cut Dyne Therapeutics to perform, citing small trial size and data risk for DYN-101 in DM1. - Oppenheimer downgraded Dyne Therapeutics (DYN) to perform from outperform, saying that upcoming data for DYN-101 (zeleciment basivarsen, or z-basivarsen), its candidate for myotonic dystrophy…
The full CMI on the next page has more details. If you are worried about being given this medicine, speak to your doctor or nurse. 1. Why am I being treated with DBL Desferrioxamine Mesylate for... NOTICE: This Consumer Medicine Information…
A new review argues that lipid nanoparticles, already clinically validated as mRNA carriers, could unify cancer therapy around restoring the p53 tumour suppressor.
Givinostat (Duvyzat; ITF Therapeutics) in combination with corticosteroids delayed Duchenne muscular dystrophy (DMD) progression and demonstrated long-term safety and tolerability in ambulatory patients aged 6 and older, according to a study published in Annals of Clinical and Translational Neurology.1 The findings, which…
Interested in Alnylam Pharmaceuticals, Inc.? Here are five stocks we like better. Brezosiran reduced cerebrospinal fluid mutant huntingtin in a Phase Ib study, with mean reductions of 60%–64% at three months at the highest dose and individual reductions reaching 83%. The treatment…
- Data from a pooled dose group of participants initially enrolled in the multiple ascending dose portion of the trial to be compared against a matched natural history cohort - - Dyne also intends to provide the mean baseline value for video hand…
- Data from a pooled dose group of participants initially enrolled in the multiple ascending dose (MAD) portion of ACHIEVE showed functional improvement versus baseline, versus placebo and versus a matched natural history cohort across multiple measures at 12 months - - Data continued…
Preclinical data demonstrate subnanomolar silencing potency, achieving an IC50 tens of times lower than the corresponding values for two clinical-stage reference molecules targeting the same target. In non-human primates, a single dose maintained robust target-protein suppression for months, demonstrating a durable…
RNA drugs: siRNA, antisense oligonucleotides and RNA-editing therapies, their trials, and the companies making them such as Alnylam, Ionis, Arrowhead and Wave.
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