Abata Therapeutics Announces First Lead in Development, ABA-101, a Novel Treg Cell Therapy for the Treatment of Progressive Multiple Sclerosis

BOSTON–(BUSINESS WIRE)–Abata Therapeutics, a company focused on translating the biology of regulatory T cells (Tregs) into transformational medicines for patients living with severe autoimmune and inflammatory diseases, today announced that it has selected its first development candidate, ABA-101, which has the potential to benefit the 45,000 patients with progressive multiple sclerosis (MS) in the US who currently have no treatment options.

“The progressive pathology of MS has not been effectively treated because existing therapies do not address the central nervous system compartmentalized inflammation that drives that aspect of the disease,” said Richard M. Ransohoff, MD, co-founder and chief medical officer at Abata and a venture partner. at Third Rock Ventures. “Our approach of exploiting the natural role of Tregs in the immune system allows us to address complex inflammatory mechanisms, offering new hope for patients without treatment options. Our work to date in the development of ABA-101 validates our approach to the use of TCR-targeted Treg therapies for the treatment of autoimmune diseases. We look forward to working with our new CSO, Ellen Cahir-McFarland, to translate the science behind ABA-101 into clinical studies in the first half of 2024.”

“Since our inception, we have been focused on advancing transformative therapies for people with autoimmune diseases. We believe our Treg product mechanism has yielded a candidate in ABA-101 that is truly differentiated and may have significant benefit for people living with progressive multiple sclerosis,” said Samantha Singer, MS, MBA, president and chief executive officer of Abata. “ABA-101 uses the TCR to target our product specifically to the site of ongoing pathology and offer the potential for a powerful, highly durable effect. Working with our partners at ElevateBio BaseCamp, we rapidly established a core manufacturing process in less than one year, demonstrating that we can successfully engineer and expand Tregs for use as therapies. Overall, this work gives us great confidence not only in our ABA-101 program, but also in our additional programs for type 1 diabetes and inclusion body myositis.”

“At ElevateBio, we enjoy working with scientific innovators like Abate, helping to empower their new cell and gene therapies,” said Michael Paglia, MS, Chief Operating Officer of ElevateBio BaseCamp. “It has been a pleasure to work with the Abata team to create and refine a manufacturing process that allows them to expand antigen-specific TCR-engineered Tregs in yields that will support the clinical development of ABA-101 and serve as a template for their future programs. We look forward to continuing our partnership.”

The hypothesis for ABA-101 as a Treg treatment for progressive MS is firmly rooted in the scientific literature. Single-cell data link chronic active lesions in patients with an adaptive immune system, raising the possibility that Treg may have a therapeutic effect in progressive MS. Additionally, EAE (experimental autoimmune encephalomyelitis) studies in mice show proof of biology, including TCR-Treg trafficking, persistence, biological activity, and suppression of inflammation—all critical elements for successful treatment. Additional non-clinical studies identify Tregs’ role in myelin regeneration, a further potential benefit of the approach. Abata will use MRI to select patients for inclusion as clinical trial participants, as chronic active lesions (recognizable by MRI) correlate with disease progression in progressive MS studies in natural history.

On ABA-101 and progressive multiple sclerosis

ABA-101 is Abata’s autologous Treg therapy in development for the treatment of progressive MS. It targets MS patients with progressive, nonrelapsing disease who have the DRB1*15:01 genetic haplotype and who have imaging evidence of ongoing inflammatory tissue injury—an estimated 45,000 patients in the US today. ABA-101 was created by minimally engineering the patient’s own Tregs to express a TCR that specifically recognizes immunogenic myelin fragments in the CNS—an approach that offers a potentially strong safety profile and a highly localized anti-inflammatory effect at the site of disease. In vitro, ABA-101 demonstrated antigen-specific, dose-dependent Treg functionality, anti-inflammatory cytokine production, and suppression of inflammatory factor production (T cell-derived cytokines). ABA-101 also maintains its Treg phenotype under inflammatory conditions.

About Abata Therapeutics

Abata Therapeutics is focused on translating the biology of regulatory T cells (Tregs) into transformational medicines for patients with severe autoimmune and inflammatory diseases. Founded by pioneers in Treg biology, TCR and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers, Abata has developed a differentiated manufacturing engine to create engineered Treg cell therapies that are tissue-specific, robust and durable. In addition to its lead program in progressive multiple sclerosis, Abata has programs in type 1 diabetes and inclusion body myositis – two other tissue-specific autoimmune diseases with significant unmet needs and a strong rationale for Abata’s Treg approach. The company was launched in 2021 by Third Rock Ventures, with participation from a diverse syndicate of investors, including ElevateBio, Lightspeed Venture Partners, Invus, Samsara BioCapital and JDRF T1D Fund. The abbey is located in Cambridge, Massachusetts. Visit abatatx.com or follow us Twitter or LinkedIn for more information.

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