Ensoma to Present Preclinical Data and Manufacturing Advancements for In Vivo HSC Engineering Platform to Treat Cancer, Immune Disorders and Genetic Diseases at ASGCT 28th Annual Meeting

Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo therapies designed to precisely and durably engineer the hematopoietic system to treat chronic diseases, today announced it will present preclinical data and manufacturing advancements at the American Society of Gene & Cell Therapy (ASGCT) 28^th Annual Meeting, hosted May 13-17 in New Orleans.

The company’s poster presentations will highlight key programs that leverage its in vivo HSC engineering platform. Ensoma combines a unique delivery system with an advanced gene engineering toolkit, offering the potential for durable, transformative therapies to address chronic diseases, improve patient access and significantly reduce treatment burden. The platform uses virus-like particles (VLPs) that preferentially bind to HSCs, efficiently deliver DNA to the nucleus and de-target the liver. With a 35-kilobase cargo capacity, these VLPs carry a diverse range of genomic engineering constructs, including control elements that enable specific cargo expression in targeted cells, from single base edits to large multi-gene insertions.

“We look forward to presenting preclinical data at ASGCT that underscore the potential of our in vivo HSC engineering platform to address complex genetic disorders and cancer,” said Robert Peters, Ph.D., chief scientific officer of Ensoma. “Exciting new findings from our non-human primate studies reinforce the precision and effectiveness of our VLPs, showcasing their ability to safely deliver genetic therapies with targeted biodistribution and expression. With this strong progress, Ensoma remains on track to initiate our first clinical trial in the second half of 2025—marking a major step toward bringing this groundbreaking therapy to patients.”

“Building on this momentum, our novel adenovirus production process is a significant milestone for Ensoma, ensuring the scalable and high-quality production of VLPs needed for clinical trials,” said Dan Leblanc, chief technology officer of Ensoma. “This robust suspension manufacturing process is delivering consistent product yield and quality to support the clinical use of EN-374 for the treatment of X-CGD. It’s incredibly rewarding to see our platform move closer to delivering potentially transformative therapies to patients.”

Poster Presentations at ASGCT 28^th Annual Meeting:

• Title: In Vivo Engineering of Hematopoietic Stem Cells with Virus-like Particles to Generate Multi-Lineage CAR Immune Cell Therapy for Cancer (1783)
  Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15
  Location: Poster Hall I2

Presenter: Chirayu Chokshi, Ph.D., Ensoma

Data Summary: Ensoma will present updated data from its preclinical HER2 CAR program. An HSC-targeted VLP encoding lineage-specific regulatory elements to direct CAR expression resulted in robust generation of HSC-derived CAR-M, NK and T cells in vivo. This lineage-restricted, multi-cellular CAR therapy mediated tumor control and microenvironment remodeling, supporting Ensoma’s technology as a highly differentiated approach to addressing solid tumors.

• Title: Novel In Vivo Gene Therapy Approach to Hematopoietic Stem Cell (HSC) Engineering Creates Durable HSC-Derived Neutrophils to Treat X-Linked Chronic Granulomatous Disease (1780)

Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15

Location: Poster Hall I2

Presenter: Sravya Kattula, Ph.D., Ensoma

Data Summary: Ensoma will present updated preclinical data from its X-CGD program with EN-374, which highlights the use of its VLP platform to efficiently modify HSCs in vivo and restore neutrophil function. In this preclinical study in a CGD mouse model, EN-374 provided durable gene correction in neutrophils to restore CYBB protein expression and activity. This study sets the foundation for Ensoma’s in vivo HSC gene therapy to reach the clinic and be applied to a range of genetic disorders that thus far have only been addressed with ex vivo gene therapies.

• Title: Acute Safety and Biodistribution Profile of Hematopoietic Stem Cell (HSC) Targeting Virus-like Particles Based on Helper-dependent Adenovirus Serotype 5/35++ in Non-human Primates (1779)
  Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15
  Location: Poster Hall I2

Presenter: Patrick Au, Ph.D., Ensoma

Data Summary: Ensoma will present data from a non-human primate study evaluating the safety, biodistribution and transgene expression profiles of its HSC-targeting VLPs. Results showed favorable tolerability without any clinical signs of toxicity and a well-characterized biodistribution, supporting the continued development of Ensoma’s VLP-based platform as a safe and effective solution for in vivo HSC engineering.

• Title: Development and Scale-up of a Novel Adenovirus Production Process (2016)
  Poster Presentation Time/Date: 6:00-7:30 p.m. CT, Tuesday, May 13
  Location: Poster Hall I2

Presenter: Chapman Wright, Ph.D., Ensoma

Data Summary: Ensoma will present the establishment of a novel, serum-free suspension manufacturing process for large-scale, high-efficiency VLP manufacturing. The presentation will address adaptation of an adherent cell line to a serum-free suspension cell culture, followed by a design-of-experiment strategy for efficient development of a clinical-scale production process.

About Ensoma

Ensoma is developing potentially curative medicines through precision in vivo cellular engineering. Our platform combines class-leading proprietary base editing and high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) to provide one-time, durable genetic medicines in an outpatient procedure. We are focused on in vivo engineering of hematopoietic stem cells (HSCs) to address genetic diseases, immune disorders and cancer. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genomic medicines. Ensoma is based in Boston. For more information, visit ensoma.com.

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