Leaders from the companies explain how they navigated to success with the world’s first CRISPR-based medicine – and avoided the pitfalls that claimed other programs.

Plus deals involving Biocytogen, Neurocrine and Ona, Aeterna Zentaris/Ceapro, Teva/Biolojic Design, Pathos AI/Rain Oncology, Inhibitor Therapeutics/Johns Hopkins and more.

The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options. 

Companies aiming to be fast followers in sickle cell disease gene therapy behind Vertex and bluebird have thinned out in 2023, and Beam may struggle to convince investors its base editing approach can compete.