A few years ago, investors were calling on Vertex to buy in late-stage innovation, but its focus on audacious drug development goals looks set to pay off with a groundbreaking CRISPR-based sickle cell disease cell therapy approaching the market. Its R&D head David Altshuler talked to Scrip about its approach.
Vertex and its partners CRISPR Therapeutics could be just months away from achieving a two-in-one historic breakthrough: the approval of exagamglogene autotemcel (exa-cel), the first ever cell therapy for sickle cell disease and beta-thalassemia and also the first ever CRISPR gene-edited medicine.
A US Food and Drug Administration approval (a decision is expected by 8 December) would represent a major step forward...
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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.
![Spotlight On RDCs, AOCs As Leading Novel Bioconjugates[Updated]](https://web.insights.norstella-labs.com/resizer/v2/3M7ZAWZOBNK7NG4A4WDOIFG6DU.jpg?smart=true&auth=392a1fdb3cfacb29445720c412410e598dbcc5f90f14857652cd498bd0faa0fb&width=400&height=225)
