Regulus Therapeutics Inc (NASDAQ: RGLS) has stated that it will begin studies investigating several oligonucleotides that inhibit miR-155 to determine if they could treat Lou Gehrig’s disease or ALS. The company is conducting this project in partnership with Dr. Howard L. Weiner, and Oled Butovsky, Ph.D. This agreement began in September 2021.
Researchers who will led the study
Dr. Butovsky works at Harvard Medical School as an associate neurology professor
Dr. Weiner, on the other hand, is a neurology professor in the same institution. The researchers will use findings from the study to structure more experiments in animals models and find their properties.
According to Regulus Therapeutics’ CSO, Denis Drygin, the company is interested in treating genetic kidney disorders and advancing its technology to treat CNS diseases with unmet needs. Drygin believes that Dr. Butovsky and Dr. Weiner are critical to the study as they have vast experience in the field.
Drygin points out the high unmet needs in ALS. He notes that the neurodegenerative disease is debilitating and lowers to quality of life in patients. For this reason, be company hopes to provide therapies for ALS. Regulus Therapeutics expects preliminary data later in the year.
The therapy inhibits miR-155
Previous research has realized that miR-155 takes part in ALS pathogenesis. For this reason, Regulus Therapeutics is targeting the molecule as it develops treatments for the disease. Moreover, the ,researchers have found a microglia signature associated with ALS.
When they used ALS animal models, the team discovered that miR-155 in microglia was upregulated. They also linked it to disease progression. Moreover, miR-155 levels were high in the spine of ALS patients.
The team also found that disease was minimized when they blocked miR-155 in the animal models. In addition, the therapy suppressed cytotoxic microglia, causing a remarkable improvement and potentially improving lifespan in patients with ALS.
The Neurology chairman at Brigham and Women’s Hospital, Tracy Batchelor, states that the study would advance the lab’s ALC research. Batchelor expresses her excitement at working with Regulus, with whom their goals align. Both parties hope to uncover ALS pathophysiology and treat people with the disease.
Regulus has its main offices in San Diego, California.
