Posts

FDA deepens Vertex's Casgevy label to include patients as young as 2

The U.S. Food and Drug Administration (FDA) has expanded the approval of Vertex's Casgevy (exagamglogene autotemcel), a gene‑editing therapy, to include patients as young as 2 years of age with either sickle cell disease (SCD) with recurrent vaso‑occlusive crises (VOCs) or transfusion‑dependent β‑thalassemia (TDT). Casgevy was previously approved only for patients aged 12 years and older, so this marks a significant lowering of the minimum treatment age. The FDA based the expansion on clinical‑study data in children aged 5–under‑12 and extrapolated safety and efficacy to support use down to 2 years. This makes Casgevy the first gene therapy approved for children as young as 2 with SCD and also the first approved genetic therapy for very young children with both SCD and transfusion‑dependent β‑thalassemia. Vertex estimates that about 5,500 additional pediatric patients in the United States now become eligible for this one‑time treatment. The company is also pursuing regulatory revie...

Scribe Therapeutics preps IPO to fund lipid‑lowering genetic medicines through clinical trials

Genentech lays off 103 as part of gRED rethink, advances with $490M breast cancer collaboration

Novartis to Acquire UK Biotech Myricx Bio for Up to $1.5 Billion to Expand ADC Oncology Pipeline

FDA Approvals Outrun Staff Cuts: 23 Novel Drugs Mark Best First Half in Three Years

Ipsen signals more deals to continue buying spree

Sandoz Tests Direct‑to‑Consumer Sales With Omnitrope Growth Hormone Biosimilar

Median Technologies' AI tool wins EU nod for earlier lung cancer detection

AstraZeneca seals near $1.8B renal disease pact with CSPC

PureTech Spins Off Celea with $180M Funding to Advance IPF Drug Deupirfenidone into Phase 3

The Francis Crick Institute and Medicines Discovery Catapult Open KQ Labs Applications, Accelerating the Next Wave of Data-Driven Health Innovation

New Nature Biotechnology Article Highlights the Need for Cell-Cell Interaction Data in AI Biology

Friday Five: Caris Life Sciences hits MCED market