Vertex Pharmaceuticals
NEWS
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
Infusions of Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have begun; Moderna targets 10 approvals through 2027; more oral obesity drug data; the latest from ESMO and more.
Last month, Vertex said sickle cell patients had not yet received infusions of its gene therapy Casgevy. That’s now changed, as the company races with bluebird bio’s Lyfgenia.
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
Patients in the U.K. with transfusion-dependent beta-thalassemia will have access to Vertex Pharmaceuticals’ gene editing therapy Casgevy, thanks to an agreement with the National Health Service on the price.
The size of the global immunology market is projected to nearly double by 2032 to a quarter of a trillion dollars. Here are this year’s standout deals in the space.
Poor efficacy data for two early-stage candidates for the rare disease alpha-1 antitrypsin deficiency have convinced Vertex Pharmaceuticals to terminate their development.
The FDA will have six months to review Vertex Pharmaceuticals’ suzetrigine, potentially the first new class of drugs for acute pain in more than 20 years, according to the company.
As the yearslong litigation over ownership of CRISPR gene editing continues, investors have forged ahead with funding the technology’s development by biopharma.
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