Three biotech companies, Actio Biosciences, AIRNA Therapeutics, and Alesta Therapeutics, have recently raised significant funding to advance treatments for genetic diseases. Actio Biosciences is developing treatments for rare neurological conditions, while AIRNA Therapeutics is working on a “RNA cure” for alpha-1 antitrypsin deficiency. Alesta Therapeutics is focusing on orally available small-molecule therapies for genetic disorders. These companies are making progress in addressing the challenges of treating genetic diseases.
Forecast for 6 months: We expect to see the launch of phase 1b trials for Actio Biosciences’ ABS-0871, a TRPV4 inhibitor for Charcot–Marie–Tooth disease type 2C, and the start of clinical testing for Alesta Therapeutics’ ALE1, an orally active inhibitor for hypophosphatasia.
Forecast for 1 year: By the end of 2026, we anticipate the completion of phase 1/2 trials for AIRNA Therapeutics’ AIR-001, a treatment for alpha-1 antitrypsin deficiency, and the initiation of phase 2 trials for Actio Biosciences’ ABS-1230, a treatment for KCNT1-related epilepsy.
Forecast for 5 years: In the next five years, we expect to see the approval of multiple treatments for genetic diseases, including those developed by Actio Biosciences, AIRNA Therapeutics, and Alesta Therapeutics. This could lead to improved treatment options and better outcomes for patients with these conditions.
Forecast for 10 years: By 2035, we anticipate significant advancements in the field of genetic disease treatment, with the development of more effective and targeted therapies. This could lead to a reduction in the number of patients with genetic diseases and improved quality of life for those affected.
