Ionis Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development ofRNA-targeted therapies
By leveraging its proprietary antisense technology, Ionis focuses on treating a range of serious diseases, including neurological disorders, cardiovascular conditions, and rare diseases. The company's innovative approach allows for precise targeting of specific genes to modulate protein production, providing potential treatment options for patients with unmet medical needs. With a robust pipeline of therapies in various stages of clinical development, Ionis is dedicated to advancing the field of genetic medicine and improving patient outcomes.
Denali's DNL343 missed efficacy goals in the HEALEY ALS trial but was well-tolerated. Analysts await detailed biomarker data and subgroup analyses in 2025.
William Blair starts coverage on Denali Therapeutics with an Outperform rating, citing its transport vehicle platform and promising therapies for neurodegenerative diseases.
Ionis Pharmaceuticals releases positive results from Phase 1/2 study of ION582 for Angelman syndrome, showing consistent clinical improvements in communication, cognition, and motor function. Safety profile is favorable. Phase 3 study planned for 2025.
