Keyword: Vertex Pharmaceuticals
Merck licensed the drugs for $230 million upfront two years ago but has now granted Vertex the right to use them in certain gene-editing applications.
GSK chairman bows out; Editas CEO signs off; Roche dispatches exec to take over Genentech; and Vertex fires COO for code of conduct violation.
After a year's delay, Editas Medicine will start enrolling patients in a phase 1/2 trial of its CRISPR-based treatment for LCA10, a rare form of blindness.
The lack of efficacy seen in the phase 2b is another, potentially terminal, blow to the prospects of the Rho inhibitor.
The FDA lifted a clinical hold it placed on Vertex and CRISPR Therapeutics’ gene-edited stem cell therapy for sickle cell disease.
Inscripta aims to broaden access to CRISPR by offering new enzymes for free, and to boost R&D by outfitting scientists with a full suite of tools.
CRISPR Therapeutics and Vertex Pharmaceuticals will test their gene therapy in patients with beta thalassemia.
The partnership will leverage a platform that collates and analyzes genotype-phenotype studies to uncover insights into the underpinnings of disease.