Freeline’s FLT180a could still prove to be the best-in-class gene therapy for hemophilia B but its cash and time are running out as rivals grow closer to market.
Freeline Therapeutics is weighing up what to do next with its lead gene therapy candidate, the hemophilia B treatment FLT180a, after a Phase I/II study failed to provide the compelling efficacy data it needed.
While the safety and initial efficacy data were encouraging from the first three patients in the trial, Freeline fell short...
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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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