Precision Signs Multiple Agreements with Novartis to Develop an In Vivo Gene Editing Therapies for Sickle Cell Disease and Beta Thalassemia


Precision to receive a $75M up front, ~$1.4B in additional milestones & is also eligible to receive research funding along with royalties on sales of licensed products & will be responsible for R&D, manufacturing & commercialization activities
Precision to develop a single ARCUS nuclease designed for safe & efficient in vivo gene insertion as a one-time transformative treatment option for hemoglobinopathies. Novartis to get an exclusive license to ARCUS nuclease to create in vivo treatment options for the same indication
The collaboration combines Precision’s ARCUS genome editing platform & gene insertion capabilities with Novartis’ drug discovery & gene therapy expertise for curative treatment in an inherited blood disorder

Ref: Bussinesswire | Image: Novartis

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