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Intellia and Ultragenyx’s Clinical Updates, Novo Holdings’ Catalent Acquisition, and NanoString Bankruptcy; Simon Barnett
Feb 9, 2024
Gene therapy updates for rare diseases by Intellia and Ultragenyx, Novo Holdings' acquisition of Catalent for $16.5B to keep up with drug demand, NanoString bankruptcy after patent infringement lawsuit, and an interview with Simon Barnett on advancing biotech's digitization and the intersection of machine learning and biophysics in structural biology.
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Quick takeaways
- Intellia Therapeutics' gene therapy, NTLA 2002, shows promising results in treating hereditary angioedema with a significant decrease in monthly attacks.
- UltraGenics' AV gene therapy UX111 demonstrates significant improvements in cognitive function and reduction in harmful substances for San Filippo Syndrome type A.
Deep dives
Intellia's Gene Therapy for Hereditary Angioedema Shows Promising Results
Intellia Therapeutics has published findings in the New England Journal of Medicine on their gene therapy, NTLA 2002, which treats hereditary angioedema. The phase one trial showed that the therapy was safe at different doses and led to a 95% decrease in monthly angioedema attacks. NTLA 2002 has already been designated a priority medicine by the EMA, and a phase three study is planned for the third quarter of 2024.
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