Why Tessera Believes Its Gene Writing Platform Can Go Way Beyond CRISPR
Interview: Bio-Entrepreneur On The Hunt For The Definitive Genetic Medicine Platform
As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.
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The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.
The company presented interim data for NTLA-2002 in hereditary angioedema, along with interim data for Regeneron-partnered NTLA-2001 in transthyretin amyloidosis with cardiomyopathy. The company plans a rapid expansion of its programs.
The US firm has started a rolling US BLA for its infectious disease candidate, SER-109, following positive data from a second late-stage study, a milestone which could draw attention to firms in the microbiome space.