Disease or Syndrome

Zhongmou Therapeutics Reports First-in-Human Success of Optogenetic Gene Therapy ZM-02 for Retinitis Pigmentosa

21 hours ago talkbio0Tagged AAV Vector, First-in-Human Clinical Trial, gene therapy, Ophthalmology specialty, Optogenetic Gene Therapy, PsCatCh2.0, Retinitis Pigmentosa, Vision Restoration, Zhongmou, ZM-02

Zhongmou Therapeutics; Optogenetic Gene Therapy; ZM-02; Retinitis Pigmentosa; First-in-Human Clinical Trial; Vision Restoration; AAV Vector; PsCatCh2.0; Gene Therapy; Ophthalmology

Gan & Lee Pharmaceuticals Initiates Phase 3 Clinical Study (GRADUAL-3) of the First Chinese Once-Monthly GLP-1 RA for Weight Management

2 days ago talkbio0Tagged Chinese Language, Clinical Research, Gan & Lee Pharmaceuticals, Glucagon-Like Peptide 1, GRADUAL-3, innovation, once-monthly, Phase 3, Rheumatoid Arthritis, Weight maintenance regimen

Gan & Lee Pharmaceuticals; Phase 3 clinical study; GRADUAL-3; once-monthly GLP-1 RA; weight management; Chinese innovation

Vera Therapeutics Appoints James R. Meyers to Its Board of Directors

2 days ago talkbio0Tagged Appointments, Atacicept, Autoimmune Diseases, Biological Factors, biotech executive, board of directors, Gilead Sciences, IGA Glomerulonephritis, IntraBio Ltd., James, Vera Therapeutics

Vera Therapeutics; James R. Meyers; Board of Directors; appointment; biotech executive; IgA nephropathy; atacicept; autoimmune diseases; Gilead Sciences; IntraBio Ltd; biopharmaceutical

FDA Delays Decision on Ascendis’ Dwarfism Drug by Three Months

3 days ago talkbio0Tagged Achondroplasia, Approved, Ascendis Pharma, CNP gene, Deferred, Dwarfism, navepegritide, PDUFA, post-marketing requirement, TransCon, United States Food and Drug Administration

FDA; Ascendis Pharma; TransCon CNP; navepegritide; achondroplasia; dwarfism; PDUFA; post-marketing requirement; delay; approval

Otsuka’s Anti-APRIL Antibody (Voyxact/Sibeprenlimab) Receives FDA Approval for IgA Nephropathy (IgAN)

3 days ago talkbio0Tagged anti-APRIL antibody, Approved, IGA Glomerulonephritis, Monoclonal Antibodies, Otsuka, Proteinuria, Sibeprenlimab, United States Food and Drug Administration, Voyxact

Otsuka; anti-APRIL antibody; Voyxact; sibeprenlimab; FDA approval; IgA nephropathy; IgAN; proteinuria; monoclonal antibody

CMS Unveils Negotiated Prices for Ozempic, Wegovy, and Other Drugs in Second Round of IRA Talks

3 days ago talkbio0Tagged drug price, Effectiveness, Inflation Reduction Act, Medicare Part D, Myasthenic Syndromes, Congenital, negotiation, Ozempic, Prices, semaglutide, Wegovy

CMS; Ozempic; Wegovy; Inflation Reduction Act; drug price negotiation; Medicare Part D; semaglutide; negotiated prices; 2027 effective date

FDA OKs Sarepta Study of New Immune Suppression Approach with Elevidys; Gilead Acquires Preclinical TREX1 Program

3 days ago talkbio0Tagged acute liver injury, ELEVIDYS, gene therapy, Gilead, Muscular Dystrophy, Duchenne, sirolimus, Therapeutic immunosuppression, TREX1 gene, United States Food and Drug Administration

FDA; Sarepta; Elevidys; immunosuppression; Duchenne muscular dystrophy; Gilead; TREX1; gene therapy; sirolimus; acute liver injury

Dupixent Gains EU Approval as First Targeted Therapy for Chronic Spontaneous Urticaria in Over a Decade

3 days ago talkbio0Tagged Approved, Chronic Spontaneous Urticaria, Dupixent, EU, Interleukin-13, interleukin-4, Monoclonal Antibodies, Regeneron, sanofi, type 2 inflammation

Dupixent; chronic spontaneous urticaria (CSU); EU approval; Sanofi; Regeneron; monoclonal antibody; IL-4; IL-13; CUPID phase 3 trials; type 2 inflammation

BostonGene to Showcase Transformative Impact of AI-Driven Profiling with Six Abstracts at the 67th American Society of Hematology Annual Meeting & Exposition

3 days ago talkbio0Tagged 67th, AI-driven, annual, Asymmetric Septal Hypertrophy, Biological Markers, BostonGene, Clinical Trials, Hematology (discipline), immune profiling, meeting, Molecular Profiling, Multiple Myeloma, Neoplasms, Precision - temporal, Stratification

BostonGene; AI-driven molecular profiling; hematology; 67th ASH Annual Meeting; immune profiling; multiple myeloma; patient stratification; clinical trials; biomarkers; precision oncology

Novo Nordisk’s Amycretin Significantly Reduces Weight and Blood Sugar in Phase 2 Type 2 Diabetes Trial

3 days ago talkbio0Tagged Administration, Oral, Agonist, amycretin, Amylin Receptor Agonists, Diabetes Mellitus, Non-Insulin-Dependent, Glucagon-Like Peptide 1, Glycosylated hemoglobin A, Novo Nordisk, Phase 2 clinical trial, subcutaneous administration, Weight Loss

Novo Nordisk; Amycretin; Type 2 diabetes; Weight loss; HbA1c reduction; GLP-1 agonist; Amylin receptor agonist; Phase 2 clinical trial; Subcutaneous administration; Oral administration