Codon-Optimized Myelin Autoantigen Sequences Including MOG, PLP, and MBP
This first-in-class antigen-specific immunotherapy is designed to restore immune homeostasis in multiple sclerosis (MS) patients through the in vivo induction of myelin-specific regulatory T cells (Tregs). By selectively reprogramming pathogenic immune responses while preserving systemic immunity, this approach represents a precision immunology strategy aimed at re-establishing durable immune tolerance to neuroantigens.
MS is a chronic autoimmune and neuroinflammatory disorder characterized by immune-mediated destruction of myelin within the central nervous system, leading to progressive neurological dysfunction, disability, and substantial long-term healthcare burden. Current disease-modifying therapies—including Ocrelizumab, Natalizumab, interferon beta therapies, and sphingosine-1-phosphate receptor modulators—primarily rely on broad immune suppression or generalized immune modulation. While these agents can reduce relapse frequency and delay disease progression, they require chronic administration and are associated with significant safety risks, including opportunistic infections and progressive multifocal leukoencephalopathy (PML). Importantly, these therapies do not restore antigen-specific immune tolerance or address the underlying immune dysregulation driving disease progression, particularly in progressive MS, where effective therapeutic options remain limited.
The rapidly expanding MS therapeutics market is valued at approximately $30.3 billion in 2026 and is projected to exceed $56 billion by 2036, reflecting a compound annual growth rate (CAGR) of 6.4%1. Despite substantial commercial growth, currently approved therapies remain limited in their ability to induce durable remission or reverse established autoimmune pathology. Researchers at the University of Florida have developed a liver-directed antigen-specific immunotherapy platform engineered to induce regulatory T cells (Tregs) against disease-driving neuroantigens in vivo. By leveraging the liver’s naturally tolerogenic immune environment and expressing codon-optimized MOG, this platform re-educates the immune system to recognize myelin as “self.” The result is a targeted restoration of immune tolerance with the potential to induce durable remission, reverse pathogenic neuroinflammation, and establish a potentially transformative therapeutic paradigm for MS and other autoimmune diseases.
Application
An antigen-specific regulatory T cell inducing gene immunotherapy platform for the induction of durable immune tolerance in autoimmune disease, with initial applications in multiple sclerosis and future platform expansion opportunities across Type 1 diabetes, rheumatoid arthritis, systemic lupus erythematosus, and additional immune-mediated disorders
Advantages
- Disease-modifying, antigen-specific Treg induction capable of suppressing or reversing pre-existing neurological pathology rather than solely reducing relapse frequency in multiple sclerosis
- Single-administration, liver-directed immunotherapy designed to establish durable immune tolerance and restore long-term immune homeostasis without chronic immunosuppressive treatment
- Modular antigen-encoding platform architecture enabling rapid expansion into additional autoimmune indications, including rheumatoid arthritis, systemic lupus erythematosus, and Type 1 diabetes
- Antigen-specific immune modulation minimizes risks associated with broad immunosuppression, including opportunistic infections, systemic immune compromise, and progressive multifocal leukoencephalopathy (PML)
Technology
The platform consists of a liver-directed antigen-specific immunotherapy system engineered to restore immune tolerance through the in vivo generation of regulatory T cells (Tregs). The technology utilizes an adeno-associated viral vector to deliver codon-optimized sequences encoding disease-relevant myelin autoantigens, including myelin oligodendrocyte glycoprotein (MOG), proteolipid protein (PLP), and/or myelin basic protein (MBP), enabling targeted and sustained antigen expression associated with multiple sclerosis pathology. Hepatic antigen presentation within the liver’s naturally tolerogenic microenvironment promotes the induction and expansion of antigen-specific Tregs capable of selectively suppressing pathogenic immune responses while preserving systemic immune function. These Tregs circulate systemically and migrate to sites of neuroinflammation within the central nervous system, where they suppress autoreactive effector T cells, reduce inflammatory damage, and inhibit ongoing demyelination.
The platform may be further enhanced through combination with transient immunomodulatory agents, including anti-CD3 antibodies or rapamycin, to optimize the Treg-to-effector T cell ratio and strengthen early tolerance induction. Through substitution of disease-relevant antigens, the technology is adaptable across a broad range of autoimmune indications, establishing a scalable precision immunology platform designed to restore immune balance and address the root cause of autoimmune pathology.
Brochure
