Tag Archive for: Vertex Pharmaceuticals

Vertex Pharmaceuticals on Wednesday scooped up clinical-stage immunotherapy company Alpine Immune Sciences for $4.9 billion in cash in the largest acquisition of 2024, targeting autoimmune and inflammatory diseases.

Sionna Therapeutics announced it raised another round of funding as the company looks to challenge Vertex Pharmaceuticals’ dominant cystic fibrosis business.

The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.

In late 2023, Vertex Pharmaceuticals and CRISPR Therapeutics made history by gaining the first FDA approval for a CRISPR-based drug, exagamglogene autotemcel (Casgevy) for the treatment of sickle cell disease with vaso-occlusive crisis. This historic achievement coincided with a record number of 14 review designations awarded by the FDA to CRISPR-based therapies in 2023, according to GlobalData.

After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.

Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.

In its announcement, the Boston, MA–based company said that the deaths were “unrelated” to the study treatment and that the study pause was “protocol-specified.”

The company’s non-opioid painkiller candidate VX-548 decreased Numerical Pain Rating Scale scores by at least two points after 12 weeks in a mid-stage diabetic peripheral neuropathy study.

The agency approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.