Disease or Syndrome – Page 19

Novartis; Argo Biopharma; RNA interference (RNAi); siRNA; cardiovascular pipeline; ANGPTL3; dyslipidemia; biotech collaboration; drug development; milestone payments

FDA Announces New Rare Disease Approval Programme

2 months ago talkbio0Tagged Accelerated, Approved, Biological Factors, conditional approval pathway, drug review, Endpoints, Infrequent, real-world data, streamlined process, Surrogate, United States Food and Drug Administration

FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process

Wave Life Sciences Shares Dip After Mixed Data Update on GSK-Partnered AATD Therapy

2 months ago talkbio0Tagged alpha 1-Antitrypsin Deficiency, Clinical Trials, protein levels, RNA Editing, SmithKline Beecham, Wave Life Sciences, WVE-006

Wave Life Sciences; GSK; AATD; RNA editing; WVE-006; clinical trial; protein levels; stock dip; biotech

Paul Offit, RFK Jr. Critic, Removed from Key FDA Vaccine Panel

2 months ago talkbio0Tagged Advisory Committees, CHARGE Syndrome, Paul Offit, Policy, removal technique, RFK Jr., United States Food and Drug Administration, vaccine panel, Vaccines, VRBPAC

Paul Offit; FDA; vaccine panel; RFK Jr.; removal; vaccine policy; VRBPAC; advisory committee; HHS

UK Pharma Trade Group Calls for Overhaul of Government’s Life Sciences Investment Evaluation and Medicines Pricing Schemes

2 months ago talkbio0Tagged ABPI, Biological Science Disciplines, Evaluation, government review, innovation, Investments, Nance-Horan syndrome, pharma trade group, Pricing, statutory scheme, UK, valproic acid glucuronide

UK life sciences; ABPI; VPAG; pharma trade group; investment evaluation; medicines pricing; government review; NHS; innovation; statutory scheme

Editas Medicine Reboots With Cholesterol Gene Therapy (EDIT-401) as Lead Asset

2 months ago talkbio0Tagged Clinical Development, EDIT-401, Editas Medicine, Gene Modification, gene therapy, Hyperlipidemia, in vivo, LDL Cholesterol Lipoproteins, LDLR gene, strategic pivot

Editas Medicine; EDIT-401; gene therapy; hyperlipidaemia; LDL cholesterol; LDLR gene; in vivo gene editing; strategic pivot; clinical development

Ionis Builds Case for Wider Tryngolza Use in Severe Lipid Disorders Following Positive Phase 3 Results

2 months ago talkbio0Tagged drug indication, expansion, Familial chylomicronemia syndrome, Hypertriglyceridemia, Ionis Pharmaceuticals, Lipid Metabolism Disorders, Olezarsen, Pancreatitis, Acute, Severe (severity modifier), TRYNGOLZA, United States Food and Drug Administration

Ionis Pharmaceuticals; Tryngolza; olezarsen; severe hypertriglyceridemia; lipid disorder; Phase 3 trial; familial chylomicronemia syndrome; acute pancreatitis; FDA; drug indication expansion