Company’s in vivo HSC engineering platform drives durable multi-lineage CAR immune cell generation in mice, addressing key limitations of CAR therapies for solid tumors

Ensoma, an in vivo hematopoietic stem cell (HSC) engineering company with a mission to advance the future of medicine through one-time therapies, today announced new preclinical data demonstrating proof-of-concept for its in vivo, HSC-derived CAR-M, NK, and T cell platform, including its potential to durably generate lineage-restricted CAR cells in solid tumors. The data will be presented in two poster sessions this week at the Society for Immunotherapy of Cancer (SITC) 40^th Annual Meeting, taking place November 5-9 in National Harbor, Md.

“While ex vivo CAR-T therapies have transformed treatment for blood cancers, use in solid tumors has been limited by multiple factors, including poor T cell infiltration and persistence in the immunosuppressive tumor microenvironment, as well as manufacturing cost and complexity,” said Jim Burns, CEO of Ensoma. “By engineering HSCs in vivo, we can develop off-the-shelf therapies that turn the body into its own cell factory—capable of continuously producing multiple CAR immune cell types that work together against solid tumors. These data move us closer to realizing this vision as we advance toward our first in vivo, HSC-derived CAR-M, NK, and T development candidate early next year.”

Ensoma SITC poster presentations:

In vivo HSC engineering with Ensoma’s virus like particles (VLPs) generates lineage-restricted, multiplexed CAR-M, NK, and T cells to cooperatively mediate solid tumor control in pre-clinical models

Abstract Number: 302

Poster Presentation Time/Date: Saturday, November 8, 5:10-6:35 pm EST

Location: Gaylord National Resort and Convention Center - Lower Level Atrium - Prince George's ABC

Presenter: Yiwen Zhao, Ph.D., Ensoma

This study in HER2-positive orthotopic tumor-bearing mouse models, validates proof-of-concept for anti-tumor activity driven by in vivo CAR therapy via HSC engineering. Administration of VLPs encoding lineage-specific HER2 CARs successfully generated durable CAR-expressing myeloid, NK, and T cells from HSCs that:

• Exhibited tumor suppression in vivo and ex vivo
• Remodeled the cold solid tumor microenvironment, marked by macrophage M1 polarization, increased lymphocyte recruitment, and production of inflammatory cytokines and chemokines.

Discovery of lineage specific regulatory elements for development of in vivo CAR immune cell therapy via hematopoietic stem cell engineering

Abstract Number: 1019

Poster Presentation Time/Date: Friday, November 7, 5:10-6:35 pm EST

Location: Gaylord National Resort and Convention Center - Lower Level Atrium - Prince George's ABC

Presenter: Alvin Pratama, Ph.D., Ensoma

This research supports the ability of the Ensoma platform to precisely identify and validate genetic regulatory elements that have the potential to drive robust lineage-restricted CAR expression in effector immune cells, potentially improving safety and functional control. Using Ensoma’s HSC-targeted VLPs to deliver lineage-restricted CAR payloads, the team achieved stable integration and selective CAR expression across myeloid, NK and T cells in human CD46 transgenic mouse models. The lineage-restricted CAR cells displayed potent, antigen-dependent cytotoxicity and cytokine production comparable to ubiquitous CAG-driven CARs, supporting the platform’s potential for precise, scalable and lineage-controlled in vivo CAR delivery.

About Ensoma

Ensoma is developing potentially curative medicines for genetic diseases, immune disorders and cancer through in vivo hematopoietic stem cells (HSCs) engineering. Our platform combines proprietary high-efficiency gene integration and base editing systems with high-capacity virus-like particles (VLPs) toward our goal of providing one-time administration of durable genetic medicines. The delivery system is based on VLPs that preferentially bind to HSCs, delivering DNA to the nucleus. With a 35-kilobase cargo capacity, these VLPs can carry a diverse range of sophisticated genomic engineering tools capable of introducing changes from single base edits to large multi-gene insertions, along with control elements for HSC-lineage cell specific expression. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genetic medicines. Ensoma is based in Boston. For more information, visit ensoma.com.

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