Amyotrophic Lateral Sclerosis (ALS)

Traditional drug development, focused on pathological endpoints, has failed to deliver meaningful interventions to halt the progression of neurodegenerative disease. By targeting fundamental cellular processes, such as RNA-protein interactions, we are leading the discovery of a novel class of targets with pre-clinical efficacy across a number of neurodegenerative diseases.

ALS, like other neurodegenerative diseases, is associated with the emergence of multiple inflammatory stressors that set neurons on a path to premature cellular death (necroptosis). By targeting a complex protein-protein interface, we are able to halt necroptosis without interfering with normal homeostatic-apoptotic pathways. Leveraging CIBS infrastructure and expertise, we are advancing the pre-clinical testing required to move this compound forward.

We are pioneering the development of patient-specific nucleic-acid aptamers capable of neutralizing pathological antibodies in neurogenerative diseases where cellular damage is caused by an autoimmune response.

Further, by virtually screening compounds with demonstrated efficacy in one disease, we are discovering common pathways with other age-associated neurodegenerative diseases, including Alzheimer’s, Parkinson’s and Multiple Sclerosis (MS).