Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease

The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease. The post Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a…

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ISPOR 2026

Excited to be attending ISPOR 2026 next week. I’m looking for some great plenary sessions, presenting my research on “The Extent of Treatment Response and Preference Heterogeneity in Major Depressive Disorder: Implications for Population-Level Resource Allocation” with co-authors Nadine Zawadzki and Cheryl Neslusan, and meeting up with old colleagues. See you in Philadelphia!

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Sarepta CEO: ‘We Have, I Believe, a Very Laudable History of Being Extraordinarily Transparent’

Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing public for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program. The…

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Senate Committee on Appropriations Approves FY 2026 Labor, Health and Human Services, Education, and Related Agencies (Labor HHS) Appropriations Bill & Accompanying Report

The Senate Committee on Appropriations approved its FY 2026 Labor, Health and Human Services, Education, and Related Agencies (Labor HHS) appropriations bill, accompanying report, and amendments on July 31, 2025. While most U.S. global health funding is provided to the State Department through a separate appropriations bill, the Labor HHS appropriations bill includes funding for…More

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