Sickle Cell Gene Therapies Reach Patients, Moderna Cuts, Obesity Pill Race and ESMO 2024

Infusions of Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have begun; Moderna targets 10 approvals through 2027; more oral obesity drug data; the latest from ESMO and more.

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After their groundbreaking approval last year, infusions of Vertex and CRISPR Therapeutics’ and bluebird bio’s sickle cell gene therapies have begun, bringing hope to patients and the companies closer to realizing revenue. Meanwhile, bispecifics and anti-TIGIT therapies were all the rage at ESMO 2024 as BioNTech, GSK and iTeos, BMS and more reported positive results across multiple cancers.

Last week, Moderna announced it would slash its R&D budget by $4 billion as it targets 10 new approvals through 2027. Possibly boding well for this target, the biotech features prominently on our list of 5 late-stage mRNA vaccines to watch. Separately, the oral obesity drug race continues to heat up, with Terns Pharmaceuticals, Roche and Novo Nordisk all reporting new data from their respective trials. And in the equally hot radiopharmaceuticals space, Sanofi inked a $110 million licensing deal with RadioMedix to develop a neuroendocrine tumor candidate.

Finally, BioSpace takes a deep dive into the HEALEY ALS Platform Trial, which has so far seen quick failures and small victories as leaders and early participants remain hopeful.

Heather McKenzie is senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.
Jef Akst is managing editor of BioSpace. You can reach her at jef.akst@biospace.com. Follow her on LinkedIn and Twitter @JefAkst.
Greg Slabodkin is news editor at BioSpace. You can reach him at  greg.slabodkin@biospace.com. Follow him on LinkedIn.
Annalee Armstrong is senior editor at BioSpace.
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