RARE Daily

Ring Publishes Study of Novel Gene Delivery Platform that Could Enable Redosable Gene Therapies

April 2, 2024

Rare Daily Staff

Ring Therapeutics said it has made available a pre-print publication in bioRxiv featuring the first gene delivery vector system based on a human commensal virus, the anellovirus, enabling potentially redosable, programmable viral vectors.

The company said the data support the promise of its Anellogy platform, harnessing the unique biology of commensal anelloviruses to engineer the next generation of durable viral vectors.

The publication suggests the potential of Ring’s platform to generate a new class of viral vectors that could successfully evade the immune system and exhibit tissue-specific tropism, potentially overcoming some of the major challenges facing gene therapy today.

The Anellovector demonstrated transduction in multiple tissue types including in the mouse eye during interim analysis at nine months and achieves comparable gene expression to dose-matched AAV9.

“We set out to develop an entirely new vector system based on human commensal viruses to address the many hurdles facing current genetic medicine delivery – and we’ve achieved that goal at remarkable speed,” said Tuyen Ong, CEO of Ring. “Building on our breadth of publications showcasing anellovirus diversity, structure, tropism, and immune privilege, this new publication shows we can successfully harness the unique characteristics of anelloviruses to generate the Anellovector – the first novel viral vector in decades.”

He said the work serves as the foundation for multiple vectors coming out of Ring’s Anellogy platform and will inform upcoming IND-enabling studies.

In the publication, researchers outlined their development of a novel vector packaging system called SATURN, which packages vector payloads into the capsids of anelloviruses. The technology could enable the Anellogy platform to take advantage of the diversity of anelloviruses.

In vitro function of Anellovectors in retinal pigment epithelial cells was validated, and viral genomes were detected in the nuclei of cells by in situ hybridization. In vivo studies in mice demonstrated durable function for nine months after subretinal administration and achieved comparable expression to dose-matched AAV9 when transduced by the intracerebroventricular route of administration.

Photo: Tuyen Ong, CEO of Ring

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