Intellia Says Sickle Cell Therapy Still ‘Wide Open’ As Novartis And Others Exit
Graphite Bio And Sangamo Also Dropping Out
A long-anticipated pruning back of the ev vivo gene therapy pipeline for sickle cell disease has arrived with Novartis, Graphite Bio and Sangamo each dropping projects, but some challengers are looking ahead to a future of in vivo approaches to the condition.
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Chief operating officer Stuart Arbuckle and chief scientific officer David Altshuler talked to Scrip about potential launches in sickle cell disease and pain, and the firm’s expanding pipeline.
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.
Novartis is once again looking to be a pioneer in cell and gene therapy, though this time is selecting a potentially less complex administration method.