Vertex has announced positive results from its Phase 3 program in the treatment of moderate-to-severe acute pain using its selective NaV1.8 inhibitor called VX-548. NaV1.8 plays a critical role in pain signaling in the peripheral nervous system. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class […]

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, […]

AstraZeneca has announced plans to acquire Seattle-based Icosavax, a clinical-stage biopharm focused on developing vaccines using a protein virus-like particle (VLP) platform. The proposed acquisition, valued at up to $1.1 billion, will build on AstraZeneca’s expertise in respiratory syncytial virus (RSV), strengthening AstraZeneca’s late-stage pipeline with Icosavax’s lead investigational vaccine candidate, IVX-A12, a potential first-in-class, […]

Moderna and Merck have announced follow-up data from the Phase 2b clinical trial evaluating Moderna’s investigational INT (individualized neoantigen therapy) mRNA-4157 (V940) in combination with Merck’s Keytruda in patients with high-risk melanoma (Stage III/IV) following surgery. Treatment with the combination therapy reduced the risk of recurrence or death by 49% and the risk of developing […]