Manufacturing Stumble: FDA Rejects ITM Radiopharma’s Neuroendocrine Tumor Therapy
In a stark reminder of the immense logistical complexities surrounding radiopharmaceuticals, the U.S. Food and Drug Administration (FDA) has issued…
Category: Genetics
FDA Expands Casgevy Gene Therapy Approval to Children as Young as Two with Sickle Cell Disease
In a monumental milestone for pediatric hematology, the U.S. Food and Drug Administration (FDA) has expanded the approval of Casgevy,…
Vertex Is Buying More Than a Rare-Disease Asset
The most revealing life-sciences development of the last two days is not a trial readout or a regulatory letter. It…
Gene Editing Is Moving Into Childhood, and the System Is Not Ready
The most important feature of a curative therapy is not always its mechanism. Sometimes it is the age at which…
Gene Therapy Is Entering Its Durability Audit Era
The latest update from Taysha Gene Therapies is easy to misread as a conventional rare-disease data release. The company announced…
Beyond the Short Read: How Long-Read Sequencing is Solving the Unsolvable
A landmark study demonstrates that long-read genome sequencing can replace 15 separate diagnostics for rare genetic diseases, while also illuminating…
A Milestone in Gene Therapy: Restoring Sound and the Base Editing Debate
The FDA’s landmark approval of an in vivo gene therapy for hearing loss signals a new era for biotechnology, even…