Verve Hits The Clinic With Gene-Edited Cholesterol-Lowering Drug
The company dosed the first patient in its study of base editor VERVE-101, a possible single-dose treatment for HeFH that joins a growing list of PCSK9-targeted therapy candidates.
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‘Epigenetic Engineering’ Gene Therapy From Epic Bio Shows Early Promise
The San Francisco-based company has taken a step forward with proof of concept for its ‘switch on or switch off’ approach to gene regulation via non-cutting CRISPR molecules.
US FDA Guidance Aims To Tackle Challenges In Multi-Endpoint Trials
Final guidance targets the ‘multiplicity problem.’ At about half the length of the 2017 draft, the final removes some sections, truncates other and drops several examples.
Trials In Focus: FDA Guidance Aims To Tackle Challenges In Multi-Endpoint Trials
Beam Therapeutics also announced that it dosed the first patient in its Phase I/II sickle cell disease trial; Vyne stepped into the increasingly competitive vitiligo space, while Tracon moved further into sarcomas in its checkpoint inhibitor program, and Telix started a small trial of its drug in brain cancer.