Disease or Syndrome – Page 7

Eli Lilly Expands RNA Pipeline with $1.3 Billion Rznomics Hearing Loss Pact

1 month ago talkbio0Tagged Deafness, Eli, Eli Lilly, gene therapy, pharmaceutical partnerships, RNA Editing, RNA therapeutics, Rznomics, Sensorineural Hearing Loss (disorder), trans-splicing ribozyme

Eli Lilly; Rznomics; RNA editing; hearing loss; gene therapy; pharmaceutical partnerships; biotech; trans-splicing ribozyme; sensorineural hearing loss; RNA therapeutics

Neurogene Details New Safety Measures After Young Patient’s Death in Rett Syndrome AAV Gene Therapy Trial

1 month ago talkbio0Tagged AAVS1 gene, ASGCT, Clinical Trials, Dosage, gene therapy, Individual Adjustment, Lymphohistiocytosis, Hemophagocytic, Measures, MECP2 gene, Neurogene, NGN-401, Rett Syndrome, Safety, United States Food and Drug Administration

Neurogene; AAV gene therapy; Rett syndrome; NGN-401; ASGCT; safety measures; HLH; clinical trial; MECP2 gene; FDA; dose adjustment

FDA Delays Decision on Biohaven’s Troriluzole for Rare Disease, Plans Advisory Committee Review

1 month ago talkbio0Tagged Advisory Committees, Ataxia, Spinocerebellar, Biohaven, Deferred, disease drug, Extension, Infrequent, Neurodegenerative Disorders, PDUFA, Troriluzole, United States Food and Drug Administration

Biohaven; FDA delay; troriluzole; spinocerebellar ataxia (SCA); rare disease drug; PDUFA extension; advisory committee; neurodegenerative disease

Kyverna Therapeutics Cuts 16% of Staff in Q1 2025 Amid Pipeline Focus

1 month ago talkbio0Tagged Autoimmune Diseases, CAR-T, KYV-101, Kyverna, layoffs, pipeline development, Staff

Kyverna Therapeutics; layoffs; biotech; staff reduction; Q1 2025; CAR-T; autoimmune diseases; pipeline development; KYV-101

Boehringer Ingelheim Expands Oncology Pipeline via Strategic Collaboration with Tempus AI

1 month ago talkbio0Tagged Aortic Valve Insufficiency, Biological Markers, Boehringer Ingelheim, Cancer Research, Discover, Drug Development, Neoplasms, real-world data, strategic collaboration, Tempus AI

Boehringer Ingelheim; Tempus AI; oncology; cancer research; real-world data; AI; biomarker discovery; drug development; strategic collaboration

Novo Nordisk and Septerna Forge $2.2B+ Collaboration on Oral Obesity and Diabetes Drugs

1 month ago talkbio0Tagged cardiometabolic diseases, Collaboration, Diabetes Mellitus, Non-Insulin-Dependent, G-Protein-Coupled Receptors, Gastric Inhibitory Polypeptide, Glucagon Receptor, Glucagon-Like Peptide 1, Nordisk, Obesity, oral drugs, Septerna, small molecule therapies

Novo Nordisk; Septerna; GPCR; oral drugs; obesity; type 2 diabetes; cardiometabolic diseases; GLP-1; GIP; glucagon receptors; small molecule therapies; collaboration; pharmaceuticals

GSK Acquires Phase III-Ready Liver Drug Efimosfermin for Up to $2B to Enter MASH Arena

1 month ago talkbio0Tagged Analog, Boston Pharmaceuticals, drug acquisition, efimosfermin alfa, fibroblast growth factor 21, Liver diseases, MASH, Phase III, SLC17A5 gene, SmithKline Beecham

GSK; Boston Pharmaceuticals; efimosfermin alfa; Phase III; liver disease; MASH; SLD; drug acquisition; FGF21 analog

Arcturus Prunes Early-Stage Pipeline to Focus on mRNA Therapies

1 month ago talkbio0Tagged Arcturus, biotech strategy, cash runway, Cystic Fibrosis, Kostaive, mRNA therapies, Ornithine carbamoyltransferase deficiency, pipeline reduction, vaccine programs

Arcturus Therapeutics; pipeline reduction; mRNA therapies; cystic fibrosis; ornithine transcarbamylase deficiency; Kostaive; vaccine programs; biotech strategy; cash runway

RFK Jr. Defends HHS Staff Cuts and Reorganization at House and Senate Hearings

1 month ago talkbio0Tagged Budgets, CHARGE Syndrome, Congress (U.S. Legislature), health coverage, HELP Committee, House Appropriations Committee, Medicaid, reorganization, RFK Jr., Senate, Staff

RFK Jr.; HHS; staff cuts; reorganization; Congress; House Appropriations Committee; Senate HELP Committee; budget cuts; Medicaid; health coverage

OpenAI Debuts HealthBench Dataset to Evaluate AI Models in Real-World Medical Scenarios

1 month ago talkbio0Tagged AI safety, Aortic Valve Insufficiency, Evaluation, HealthBench, healthcare benchmark, language models, medical AI, open-source dataset, OpenAI, Physicians, realistic medical scenarios, rubrics

OpenAI; HealthBench; AI healthcare benchmark; large language models; medical AI evaluation; physician rubrics; realistic medical scenarios; AI safety; open-source dataset