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Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.
Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s genetherapy Zynteglo, which is already approved in Europe.
Editas Medicine is pausing its ocular genetherapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.
Meanwhile, BioMarin’s genetherapy Roctavian is approved by the FDA for the treatment of hemophilia A. It is a dipeptidyl peptidase-4 inhibitor that keeps insulin levels stable and reduces the amount of glucose produced by the body. But all three drugs have new competitors. Both are anti-CD20 agents. billion in 2022.
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