The FDA has approved the Vertex Pharmaceuticals CRISPR/Cas9 gene-edited cell therapy called Casgevy for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of Casgevy for sickle cell disease [SCD], it is exciting to now secure approval for TDT well ahead of the […]

Vertex Pharmaceuticals’ Casgevy is a one-time therapy used to treat people with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The administration of Casgevy requires experience in stem cell transplantation thus Vertex is engaging with experienced hospitals to establish a network of authorized treatment centers throughout the U.S. to offer Casgevy to patients. Casgevy is made specifically for […]

As the first two programs signed under its collaboration with Flagship Pioneering, Novo Nordisk has entered into separate research collaborations with Omega Therapeutics and Cellarity. The companies, both founded by Flagship Pioneering in 2017, are based in Cambridge and Somerville, respectively. The Omega collaboration will leverage its proprietary platform technology to develop a potential new […]

Bristol Myers Squibb has agreed to acquire Boston-based biopharma Karuna Therapeutics for $330 per share in a transaction valued at $14 billion. Karuna is focused on therapies for psychiatric and neurological conditions with a lead asset called KarXT, an antipsychotic with a novel mechanism of action and differentiated efficacy and safety. Karuna’s NDA for KarXT […]

Watertown-based Tome Biosciences launched in mid-December 2023 with $213 million in Series A and B funding from investors and moved to acquire California biotech Replace Therapeutics just two weeks later. Tome promises “to usher in a new era of genomic medicines based on programmable genomic integration (PGI).” PGI enables the insertion of any DNA sequence, […]