Regulatory
A second nod for the first CRISPR medicine
The FDA expanded its approval of Casgevy, a genome-editing medicine from Vertex Pharmaceuticals and CRISPR Therapeutics, to include patients with beta thalassemia, an inherited blood disorder.
As STAT's Adam Feuerstein reports, the FDA's decision comes a month after the agency approved Casgevy as a one-time treatment for sickle cell disease, making it the first CRISPR-based medicine available in the U.S.
Casgevy uses the gene-editing technology CRISPR to genetically modify a patient's own blood stem cells to produce high levels of fetal hemoglobin — the healthy, oxygen-carrying form of hemoglobin that is produced during fetal development but normally shuts down soon after birth.
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In the lab
Chemo causes hair loss. A startup wants to change that
Among the most dreaded side effects of chemotherapy is the near guarantee that patients will lose their hair. "It's more than vanity," said Sarah Schellhorn, a medical oncologist at Yale Cancer Center. "It's about looking sick. It's about how they are perceived by others."
Perseus Therapeutics, a biotech company founded last year, has an idea to change that. As STAT's Angus Chen reports, Perseus is in the early stages of developing an antibody treatment that would protect hair follicle cells from the effects of chemo.
It all stems from the work of a Duke University grad student, who was studying whether boosting a protein called TSLP would accelerate wound healing in mice. It didn't, but the treated rodents grew markedly hairier than their counterparts, setting in motion experiments that led to Perseus' lead program.
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Catalysts
Biotech's big any-day-now news
While the J.P. Morgan Healthcare Conference brought a fair bit of news, a few of biotech's most hotly anticipated happenings went without a mention, meaning they've become any-day-now disclosures that are sure to move markets.
Vertex Pharmaceuticals will soon have data from three Phase 3 studies testing whether its novel, non-addictive pain treatment can improve the lives of patients who have recently undergone surgery. The company said it expects results in "early 2024," whenever that might be, and the multibillion-dollar future of its pain research relies on success.
Eli Lilly is awaiting FDA approval for donanemab, its treatment for Alzheimer's disease. The company hasn't disclosed an expected decision date, but Wall Street figures it'll be this quarter. The mystery here isn't whether donanemab will be approved — the supporting evidence appears to meet the FDA's established standards — but how Lilly will price, position, and launch its product.
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