Vertex now looks likely to gain US approval for the first ever CRISPR gene edited therapy by the end of the year, but could face immediate competition from Bluebird Bio.
Vertex looks on course to gain approval for its sickle cell disease gene therapy exagamglogene autotemcel (exa-cel) by the end of this year, with the US Food and Drug Administration having confirmed it will make its decision by 8 December. Its chief rival, bluebird bio, is not far behind with its candidates, however.
Vertex is also seeking the therapy’s approval as a treatment for transfusion-dependent beta thalassemia (TDT), but the US regulator has...
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US FDA approval of Niktimvo for third-line chronic GVHD, a competitor to Sanofi’s Rezurock, came less than two months ahead of anticipated approval of Syndax’s leukemia drug revumenib.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.
2seventy bio sold full rights to a hemophilia A program and in vivo gene editing technology to partner Novo Nordisk.
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Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.
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