Vertex Pharmaceuticals
NEWS
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.
The U.K.’s Medicines and Healthcare products Regulatory Agency on Thursday provided conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel.
As Vertex and CRISPR Therapeutics await the monumental potential approval of exa-cel, bluebird bio, Iovance and Rocket Pharmaceuticals wait patiently in the wings.
In the third quarter, Vertex Pharmaceuticals reported revenue of $2.48 billion, a 6% increase from the same period in 2022. However, these figures fell short of Wall Street’s consensus estimates.
The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.
In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.
New Phase I/II trial results show that one more type 1 diabetes patient achieved insulin independence after treatment with Vertex Pharmaceuticals’ investigational stem cell therapy VX-880.
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