In a deal that could bring more than $2.1 billion in payments to Arbor Biotechnologies Inc., 90-year-old Chiesi Group gained exclusive and global rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1, an ultra-rare disease caused by a mutation in the AGXT gene, as well as an option to go after a limited number of additional targets. Read More
A top-line readout of the 26-week phase IIa Cbeyond trial showed nimacimab, Skye Bioscience Inc.’s peripherally restricted CB1 inhibitor antibody for weight loss, fell short of statistical significance as a monotherapy vs. placebo on the primary endpoint of weight loss, sending the company’s shares down 60%. Skye executives, however, offered a more optimistic outlook for the findings, which they said provide clear direction for moving forward. Read More
The Nobel Committee announced today that it has awarded the 2025 Nobel Prize in Physiology or Medicine to three scientists for their discovery of regulatory T cells, which are a critical part of the way the body prevents autoimmune attacks. Read More
Even though the U.S. CDC is operating on a skeleton crew due to the partial government shutdown, it is updating its immunization schedules to adopt the COVID-19 and chickenpox vaccine recommendations the Advisory Committee on Immunization Practices (ACIP) made at its September meeting. Read More
Newco Trogenix Ltd. has emerged from incubation and raised £70 million (US$94.1 million) in a series A, as it prepares the ground for a U.S/U.K. clinical trial of a novel gene therapy construct in glioblastoma multiforme that is due to start at the beginning of 2026. Read More
BioWorld Staff Writer Randy Osborne reflects on the transformative progress in gene therapy – from tools like CRISPR and AAV vectors to major breakthroughs in diseases like hemophilia, sickle cell and beta-thalassemia. Read More
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Alto, Roche, Santhera. Read More