Harvard Develops Universal Gene Therapy for Diamond-Blackfan Anemia; Gilead's Livdelzi Shows 81% Biochemical Response in Phase 3 ASSURE Study

Nov 16, 2024, 03:27 PM

Researchers at Harvard Medical School have developed a universal gene therapy aimed at treating Diamond-Blackfan Anemia, a rare genetic disorder that affects red blood cell production due to mutations in ribosomal proteins. This novel approach targets downstream pathways rather than directly modifying or replacing damaged genes, potentially offering a new avenue for managing hematopoietic disorders. In related developments, Gilead Sciences reported positive results from its Phase 3 ASSURE study of Livdelzi (Seladelpar) for Primary Biliary Cholangitis (PBC), with 81% of participants achieving a biochemical response by month 30. The study also noted that 41% of patients reached normalization of alkaline phosphatase (ALP) levels, and 27% experienced near resolution of itch symptoms. Additionally, Intellia Therapeutics announced that its CRISPR treatment for transthyretin amyloidosis (ATTR) outperformed conventional therapies, with a Phase 1 study demonstrating a consistent reduction in serum TTR levels over 12 months, although a high rate of adverse events was reported, likely due to the advanced underlying disease and the therapy itself.