Society News

The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role in making RNA interference (RNAi) a successful therapeutic modality while CEO at Alnylam Pharmaceuticals. However, before joining and after leaving Alnylam, he was and continues to be influential in the biotechnology sphere, with his leadership skills, optimism, and willingness to take risks still changing ...

Link Source: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024). Gene editing has swiftly advanced since CRISPR was discovered, with CRISPR being developed not only to provide therapeutic gene editing treatments for humans but also for uses such as basic research, as a diagnostic tool, and for large-scale genetic screens, as well ...

Within the world of rare diseases exist conditions so sparse and infrequent they’re called nano-rare — or N of 1  — diseases and typically affect only one to thirty people worldwide (1). While less than 10% of rare diseases have approved treatments, nano-rare or ultra-rare diseases are often entirely excluded from drug development programs (1). However, the sphere of ...

A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as Angelman syndrome (AS) has achieved a favorable safety profile and lessening of overall symptoms in trial participants. In a recent press release, Ionis announced the detailed HALOS study results for the multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582. ...

Described as a combination of Alzheimer's disease, Amyotrophic lateral sclerosis, and Parkinson's disease all at once, Huntington's disease is a rare and debilitating neurological disorder passed down within affected families. With a 50% chance of a child inheriting it from a parent with the HD mutation, the fatal disease often leaves a devastating family legacy. In the United States ...