Overview

Broadening the Possibilities of Gene Therapy

The genetics of many rare hematological diseases and immune disorders are relatively well understood, yet translating this knowledge into effective in vivo therapies has been constrained by delivery challenges.

Limitations in targeting, payload capacity, and the inability to support repeat dosing have forced trade-offs in therapeutic design, leaving many serious conditions inadequately addressed.

STRM MVs naturally home to the bone marrow, enabling targeted delivery of genetic payloads with the potential for repeat dosing. This unlocks a broader landscape of in vivo therapies, freed from traditional delivery constraints.

A Significant Unmet Need and Opportunity in Fanconi Anemia

Fanconi anemia (FA) is a rare inherited DNA-repair disorder caused by mutations in genes that help cells fix damage during normal replication. Patients develop progressive bone marrow failure and have significantly increased lifetime risk of blood cancers and solid tumors. These underlying genetic defects make FA a compelling target for precision genetic therapies.

Advances in genome engineering and gene replacement now allow targeted modification of a patient’s own hematopoietic stem cells to correct or compensate for the faulty genes that drive FA.

• CRISPR-based technologies aim to precisely repair disease-causing mutations or introduce functional gene sequences while preserving long term stem cell function.
• Engineering approaches such as base editing, prime editing, and refined gene insertion strategies are being explored.
• Gene replacement methods seek to restore DNA repair capacity by delivering functional copies of defective genes, such as FANCA mRNA, potentially providing long-lasting therapeutic benefit through repeat dosing.

Next Generation CAR-T: In Vivo Delivery

In vivo CAR T therapy reprograms immune cells directly in the body to recognize and eliminate disease, offering the potential to significantly reduce costs, accelerate treatment, and expand access to more patients.

Current gene editing and delivery technologies are limited in terms of targeting specificity, payload capacity, repeat dosing, and immune activation. In contrast, STRM MVs enable targeted delivery to immune cells within the bone marrow, carrying gene editing technology or CAR-encoding RNA, for in vivo programming of immune cells while supporting repeat dosing and reduced immunogenicity.

This combination positions STRM.BIO to unlock the full potential of in vivo CAR-T, transforming a promising concept into a scalable, clinically viable therapeutic modality.