STAT+: Novo strikes $2.1 billion deal with Omeros for rare disease drug

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Good morning. Today we catch up on some fascinating discussions from the STAT Summit, including talk of scaling therapies like the gene-editing treatment given to Baby KJ. Also, an oncologist talks about the importance of funding immunotherapy research, and GSK CEO Emma Walmsley addresses the FDA’s leucovorin push.

The need-to-know this morning

• Praxis Precision Medicines said its experimental treatment for essential tremor, a neurological disease that causes involuntary shaking of the limbs, achieved the primary goals of two Phase 3 studies.

Baby KJ’s gene editing sparks a scaling of ambition

At the STAT Summit in Boston yesterday, Kyle and Nicole Muldoon shared the story of how their son, Baby KJ — once facing certain death from an ultra-rare liver disorder — became the first patient treated with a bespoke CRISPR-based gene-editing therapy that partially corrected his condition, STAT’s Megan Molteni writes.