Vertex Pharmaceuticals– tag –
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FDA Approves Vertex’s Zanvastro: First Disease-Modifying ASO Therapy for Alexander Disease
Healio USA Overview The U.S. FDA has approved Zanvastro (zilgannersen), an antisense oligonucleotide (ASO) therapy from Vertex Pharmaceuticals, for pediatric and adult patients with Alexander disease. This marks the first disease-modifyi... -
Vertex Pharmaceuticals Initiates Phase I/II for Type 1 Diabetes Cell Therapy, Expands Renal Pipeline at Morgan Stanley Conference
MarketBeat USA Overview Vertex Pharmaceuticals unveiled its ambitious growth strategy beyond cystic fibrosis at the Morgan Stanley 24th Global Healthcare Conference, emphasizing its renal pipeline and a groundbreaking cell therapy progra... -
Biotech M&A Heats Up: Merck KGaA Acquires Bio-Techne for $11.3B to Bolster Life Science Tools for Cell and Gene Therapy
Fintent United States Overview Fintent's September 8, 2026, report forecasts intensified M&A activity in healthcare and life sciences, highlighting Merck KGaA's $11.3 billion acquisition of Bio-Techne. This strategic move aims to str... -
Merck KGaA to Acquire Bio-Techne for $11.3 Billion, Bolstering Life Science Tools for Cell and Gene Therapy Workflows
BioBucks United States Overview Merck KGaA has agreed to acquire life science tools provider Bio-Techne for $11.3 billion, a strategic move to significantly enhance its life science division. This acquisition deepens Merck KGaA's exposur... -
Single-Dose CRISPR Therapy Slashes LDL Cholesterol by 52.5%, Triglycerides by 47.8% in Landmark Phase 1 Trial
Medical Xpress USA Overview Vertex Pharmaceuticals' VERVE-101, a one-time CRISPR-Cas9 gene editing therapy, has achieved significant results in a Phase 1 trial, safely reducing LDL cholesterol by 52.5% and triglycerides by 47.8% in patie... -
August Sees Mixed Fortunes in Gene Therapy: Ultragenyx’s GSDIa Therapy Gets Accelerated Approval, Epicrispr Raises $90M, While REGENXBIO’s RGX-121 Faces Clinical Hold
Vertex AI Search USA Overview August 2026 brought both significant advancements and setbacks in the next-generation therapeutics space. Ultragenyx secured FDA accelerated approval for GENGLYCOS, a gene therapy for GSDIa, offering a new o... -
CRISPR Therapeutics Diversifies Pipeline into In Vivo Gene Editing and Off-the-Shelf CAR-T Cell Therapies, Leveraging Casgevy Commercialization Experience
Venture Atlas USA Overview CRISPR Therapeutics is strategically diversifying its pipeline, leveraging its commercialization experience with Casgevy, into in vivo gene editing programs (CTX310, CTX320, CTX340, CTX460) and allogeneic off-t... -
Vertex Pharmaceuticals’ CASGEVY Receives FDA Label Expansion for Over 5,500 US Children with Sickle Cell and Beta-Thalassemia, Driving Soaring Sales
ScanX USA Overview Vertex Pharmaceuticals' gene therapy, CASGEVY (exagamglogene autotemcel), received FDA label expansion for patients aged 2 and older with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This... -
Vertex Pharmaceuticals Initiates Phase 3 Trial for iPSC Islet Cell Therapy VX-880 for Type 1 Diabetes, Showing High Rates of Insulin Independence
Facebook (Breakthrough T1D) USA Overview Vertex Pharmaceuticals has launched a Phase 3 clinical trial for zimislecel (VX-880), its stem cell-derived islet cell therapy for severe type 1 diabetes. Prior trials demonstrated remarkable effi... -
Vertex Pharmaceuticals Secures FDA IND Approval for iPSC-Derived Type 1 Diabetes Therapies, Boosts 2026 Revenue Forecast
Vertex Pharmaceuticals USA Overview Vertex Pharmaceuticals announced robust Q2 2026 financial results and significantly raised its full-year revenue guidance, propelled by key pipeline advancements. The company secured FDA Investigationa...
