A moonshot in autoimmune disease. Elevating cell therapy.

Innovating Treg engineering and manufacturing to turn the tide in serious autoimmune and inflammatory disease

Opportunity in Autoimmune Disease

Despite the development of innovative therapies for some severe autoimmune diseases, the majority of patients – including those with progressive multiple sclerosis (MS) – have inadequate or no therapeutic options.

People living with these debilitating diseases need groundbreaking new therapies that are tissue-specific and long-lasting and promote repair.

Our Approach

We are bringing an entirely new approach to the treatment of autoimmune disease by engineering regulatory T cells (Tregs) as targeted therapies that stop immune-mediated destruction, restore homeostasis – a state of harmony – and promote repair in affected tissues.

Our Treg Cell Therapies

Tissue-specific

By engineering Tregs to express a TCR that recognizes a tissue-restricted antigen, we selectively target the site of disease. Our Treg cell therapies will not impose systemic immune suppression.

Robust

Our therapies employ the intrinsic polypharmacy arsenal of Tregs to suppress multiple mechanisms of inflammation, restore homeostasis and promote repair.

Durable

The durability of our autologous TCR-engineered Tregs may allow the effects of a single dose to last for years, or even a lifetime.

Abata’s TCR-engineered Tregs interact with antigen presenting cells that display tissue-specific antigens in tissues affected by autoimmune disease. As a result, the Tregs potently suppress the ongoing aberrant immune response and restore homeostasis in the diseased tissue.

Use of the native TCR pathway uniquely maximizes the full effect of Tregs, helps to maintain a stable Treg phenotype for optimal safety and stimulates tissue residency for durability. TCRs are also exquisitely sensitive to antigens for robust activation.

Our Tregs are terminally differentiated, stable and – unlike induced Tregs originating in the periphery – resistant to pro-inflammatory triggers.

Tregs used in our cell therapies are autologous, meaning they are derived directly from a patient’s own cells, minimizing risk of rejection once engineered cells are delivered back to the patient.

Abata’s first program in progressive MS builds on well-understood technology in autologous cells to create a rapid path to the clinic.

Product Engine

Based on the innovative work of our founders, we are developing a differentiated, cutting-edge product engine to create engineered Treg cell therapies for autoimmune and inflammatory disease.

TCR Discovery

Expertise to identify the right TCR / antigen combination in each disease for targeted Treg activation in affected tissues

Treg Product Development

Knowledge and experience to develop proprietary processes for engineering, manufacturing and supplying high-quality, stable Treg cell therapies

Translational Science

Molecularly and genetically defined patient stratification and biomarker assays to rapidly assess Treg function and efficacy

Partnership

We have partnered with ElevateBio, leveraging ElevateBio BaseCamp, to accelerate development of our Treg therapies. Together we deploy a proprietary process to isolate, engineer and expand mature, thymically derived Tregs.

Pipeline

Progressive Multiple Sclerosis

Abata is rapidly advancing its early programs with plans to initiate three clinical studies by 2025.

Our initial pipeline includes our lead program for progressive multiple sclerosis (MS), as well as programs in Type 1 diabetes and inclusion body myositis – all tissue-specific autoimmune diseases with substantial unmet need and a strong rationale for our Treg approach. We will also develop therapies for other tissue-specific inflammatory and autoimmune diseases where we believe Tregs can make a difference.

MS is an autoimmune disease of the central nervous system (CNS).

Myelin, a critical component of a functioning CNS, wraps and insulates nerve fibers to protect them and enable proper transmission of electrical signals. In the CNS of MS patients, myelin is damaged by the body’s own immune cells, thereby exposing nerve fibers. 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, with long-lasting, often irreversible impact. Progressive MS is characterized by increasing clinical disability independent of acute relapsing pathology.

Our lead program targets MS patients with non-relapsing progressive disease, imaging evidence of ongoing inflammatory tissue injury and an HLA genotype of DR2a/b – estimated to be about 45,000 patients in the U.S. today. There are no approved therapies for non-relapsing progressive MS.

Success in this program supports expansion in two ways: additional HLA haplotypes and ultimately all progressive MS patients, including those who still experience relapses.

Our Treg cell therapy will be armed with a TCR that selectively recognizes degraded myelin. The figure below illustrates progressive MS pathology and how our TCR-Tregs will work.

HEALTHY BRAIN TISSUE

T cells transiently scan the surfaces of APCs but don’t recognize their specific antigen and move on. As a result, no immune response is initiated.

PROGRESSIVE MS: CHRONIC LESION

Inflammatory T cells in a stable, long-lasting aggregate relsease inflammatory mediators (dotted arrows) that activate macrophages. Macrophages at the lesion edge continuously degrade myelin. Existing MS therapies do not directly address this pathology.

TREG CELL THERAPY FOR PROGRESSIVE MS

A TCR enables Treg activation through antigen-dependent APC interaction. Activated Tregs release factors (green arrows) that suppress lymphoid aggregates, block new lymphocytes and inhibit macrophages. Tregs also produce factors that may promote myelin repair.

Additional Programs

Type 1 Diabetes

Modulating T cells has been clinically demonstrated to affect disease progression in patients with Type 1 diabetes (T1D). Our T1D program will treat patients with the genetic HLA haplotype DR3-DQ2 (over half of patients) who are early in autoimmune pathogenesis, ultimately aiming to prevent the onset of symptomatic disease and insulin dependence.

Inclusion Body Myositis

Inclusion body myositis (IBM) is a debilitating disease characterized by inflammation that leads to muscle damage and weakness. Today, there are no therapies. Because Tregs play a role in muscle regeneration, we believe Treg cell therapies have the potential to address the unique pathogenic process in IBM. Our Treg cell therapy will treat patients with the genetic HLA haplotype DR3, an estimated 75% of patients.