Drug Discovery & DDS– category –
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Ultragenyx Announces Promising Phase 3 ASPIRE Results for Apazunersen (GTX-102), an ASO Therapeutic for Angelman Syndrome
Ultragenyx Pharmaceutical Inc. USA Overview Ultragenyx has reported promising results from its Phase 3 ASPIRE trial of Apazunersen (GTX-102), an ASO therapeutic for Angelman Syndrome. This drug, one of the first investigational agents ta... -
Argo Biopharma Presents Positive Phase II Data for siRNA Therapeutic BW-20805 in Hereditary Angioedema, Demonstrates Significant Reduction in Attack Rate
PR Newswire USA Overview Argo Biopharma announced positive updated Phase II results for its siRNA therapeutic, BW-20805, for Hereditary Angioedema (HAE) at the Bradykinin Symposium 2026. BW-20805 significantly reduced monthly HAE attack ... -
Soufflé Therapeutics Initiates Phase I/II Clinical Trial for Muscle-Targeted siRNA Conjugate SFL-0821 in FSHD
allsci.com USA Overview Soufflé Therapeutics has commenced a Phase I/II clinical trial for SFL-0821, a novel antibody-siRNA conjugate designed for patients with Facioscapulohumeral Muscular Dystrophy (FSHD). SFL-0821 specifically targets... -
Inhaled siRNA Therapy ARO-RAGE Confirms Safety, Tolerability, and Lung Target Engagement in Pulmonary Inflammation Trial
Springer Nature Communities International Overview In a Phase 1/2a clinical trial, the inhaled siRNA therapeutic ARO-RAGE, targeting the Receptor for Advanced Glycation End Products (RAGE), demonstrated a favorable safety profile, good t... -
Bepirovirsen Achieves 20% Functional Cure Rate in Chronic Hepatitis B, Approved in Japan, Filed in China, FDA PDUFA October 2026
OligoTherapeutics.org Japan Overview The antisense oligonucleotide (ASO) bepirovirsen has achieved a 20% functional cure rate in Phase III trials for chronic hepatitis B (CHB) patients, significantly surpassing the 1-3% rate seen with st... -
Arrowhead’s siRNA Therapeutic Plozasiran Achieves Approximately 80% Triglyceride Reduction in Severe Hypertriglyceridemia Patients
TCTMD USA Overview Arrowhead Pharmaceuticals' siRNA therapeutic, plozasiran (Redemplo), demonstrated potent efficacy in SHASTA-3 and SHASTA-4 trials, reducing triglyceride levels by approximately 80% in patients with severe hypertriglyce... -
Ionis ASO Therapy Zanvastro Receives First-Ever FDA Approval for Alexander Disease, Demonstrates Significant Stabilization of Walking Speed
Ionis Pharmaceuticals USA Overview Ionis Pharmaceuticals' antisense oligonucleotide (ASO) therapeutic, Zanvastro (zilganersen), has secured FDA approval as the first treatment for Alexander disease (AxD), a rare and progressive neurodege... -
Gamgee Leverages ChatGPT and AlphaFold to Design Personalized mRNA Cancer Vaccine for Dogs, Accelerating Clinical Trials with AI
Gies Business (Facebook) USA Overview AI is proving to be a catalyst for accelerating all facets of clinical trials, exemplified by Gamgee's use of ChatGPT and AlphaFold to design a personalized mRNA cancer vaccine for dogs, now in clini... -
Superluminal Medicines Secures $60M for AI-Driven Rare Obesity Drug, Clinical Trials Expected This Year
BioPharma Dive USA Overview AI drug discovery startup Superluminal Medicines has raised $60 million to advance its rare obesity therapeutic towards clinical trials. The company's lead candidate targets the G protein-coupled receptor (GPC... -
Isomorphic Labs and Recursion Pharmaceuticals Forge Major AI Drug Discovery Alliances with Pharma Giants
flcube.com International Overview Isomorphic Labs, an Alphabet subsidiary, has secured significant AI-driven drug discovery partnerships with Eli Lilly and Novartis to identify novel small molecule therapeutics for undisclosed targets. C...
