Opportunity in
Autoimmune Disease

Multiple sclerosis (MS) is an autoimmune disease of the central nervous system (CNS) in which myelin is damaged by the body’s own immune cells. This myelin damage disrupts neural circuits in the brain, hampers communication between the brain and the rest of the body, and makes nerves vulnerable to cell death and neurodegeneration. Two distinct disease processes play a role in MS: acute processes that cause MS relapses and chronic smoldering processes in the CNS that cause gradual disease progression independent of relapse activity (PIRA). In patients with progressive MS, the chronic smoldering pathology is typically dominant, and current MS therapies have little or no effect on this pathology. This presents an enormous unmet need for MS patients.

We are developing ABA-101 for the treatment of progressive MS. ABA-101 is an autologous Treg cell therapy that is uniquely positioned to directly ameliorate chronic smoldering MS pathology as well as promote repair of damaged myelin in CNS tissue of MS patients. ABA-101 is created by engineering a patient’s own Tregs to express a T cell receptor (TCR) that specifically recognizes degraded myelin within the CNS. Because of their specificity for the site of myelin degradation, ABA-101 Tregs are expected to become active in the affected tissues in the CNS, where they can suppress damaging immune activity and help restore homeostasis.

ABA-101 was specifically designed for MS patients with progressive disease who have imaging evidence of ongoing smoldering inflammatory CNS tissue injury. In preclinical studies, ABA-101 was shown to be safe and demonstrated tissue-specific trafficking, persistence, and robust suppression of inflammation, supporting its potential therapeutic effect. Abata is currently conducting a first-in-human clinical trial of ABA-101.

Learn more at ClinicalTrials.gov.

The figure below illustrates chronic smoldering pathology in progressive MS and how ABA-101 will work.

Type 1 diabetes (T1D) is an autoimmune disease in which the immune system destroys the insulin-producing beta cells in the pancreas, leading to dependence on exogenous insulin treatment for survival. Even with the best glucose monitoring and insulin administration technologies, the underlying pathology of T1D remains untreated and the disease presents a significant burden with various short- and long-term complications that impact both life expectancy and quality of life. No therapies to prevent or cure T1D currently exist, leaving a substantial unmet need for T1D patients.

T1D patients still have functional beta cells at the time of diagnosis, and preserving these cells could have transformative impact on patients’ lives. We are therefore developing ABA-201, an autologous cell therapy designed to halt the destruction of insulin-producing beta cells. Specifically, ABA-201 uses TCR-engineered regulatory T cells to suppress the immune cells attacking beta cells, thereby preserving insulin production. Additionally, this therapeutic approach offers the potential to promote repair of damaged tissue, which may further increase or extend insulin production in the pancreas.

We plan to initially enroll patients with newly diagnosed T1D in our clinical studies of ABA-201. Ultimately, we aim to treat even earlier to prevent the onset of symptomatic disease. We are currently completing late pre-clinical studies for ABA-201 and expecting to begin clinical studies in T1D patients in 2025.