Ring Therapeutics Presents New Data on Anellogy™ Platform at the 27th Annual American Society of Gene & Cell Therapy Conference

Expanded payload capacity of Anellovectors widens the therapeutic potential of this novel class of viral vectors

Data showcasing transduction and redosability of Anellovectors in non-human primates (NHP), enhancing the likelihood of clinical success

Cambridge, Mass. May 07, 2024 – Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced new preclinical data demonstrating successful transduction and redosability of its Anellovector within non-human primates, as well as expanded payload capacity for the vector beyond the natural genome size of anelloviruses (ANV). The data, which will be presented as one oral presentation and two posters at the 27^th Annual America Society of Gene & Cell Therapy (ASGCT) conference, showcases the viability of Anellovectors as the next generation of viral vectors.

“I am thrilled that our Ring team has achieved this significant milestone in non-human primates showcasing transduction, redosability, and expanded payload capacity of Anellovectors,” said Tuyen Ong, MD, MBA, Chief Executive Officer of Ring Therapeutics and CEO-Partner at Flagship Pioneering. “Only a few short years ago, we set out to create a safe and effective viral vector from a human commensal virus, and the NHP data provides critical insights into how Anellovectors may work in humans. Additionally, the expanded payload capacity of Anellovectors widens the therapeutic potential of this novel vector class allowing even more patients to be potentially treated. These data demonstrate our significant progress in advancing Anellovectors towards the clinic, positioning Ring another step closer to delivering life-changing therapies for patients.”

Presentation details and key highlights below:

Expanded Payload Capacity

Title: Anellovectors: Expanding the Payload Capacity of Anellovirus-Based Vectors Beyond the Wild-Type Genome Capacity

Abstract number: 450

Presenter: Cato Prince

Presentation location, date, and time: Exhibit Hall, Wed, May 8, 12:00 PM ET

• Using vector genomes of increasing size, we have demonstrated that Anellovectors are capable of packaging vector genomes up to 5.0kb, representing up to 65% beyond the wild-type genome capacity.
• The overall potential for Anellovector cargo capacity appears to exceed the ~4.7 kb cargo capacity of Adeno-associated viruses (AAV).
• Unlike AAV, which appears to exhibit a strict packaging limit, Anellovectors allow for the packaging of genomes larger than the wild-type genome.
• A combination of qPCR and next-generation sequencing was used to verify that the encapsidated expanded cargo DNA is intact, circular, and single-stranded.
• Expanding the Anellovector’s payload capacity builds upon its intrinsic characteristics of immune favorability and tropism in efforts to reach the broadest possible patient population in need of gene therapies.

Transduction and Redosability in NHPs

Title: Anellovector Derived from Human Retinal Pigment Epithelium Delivers and Expresses a Therapeutically Relevant DNA Payload in the Retina of Nonhuman Primates
Abstract number: 444

Presenter: Parmi Thakker

Presentation location, date, and time: Exhibit Hall, Wed, May 8, 12:00 PM ET

• Anellovectors, when administered intravitreally in NHPs, demonstrated transgene infectivity and expression in ocular tissues.
• Additionally, we observed significant increase in transgene infectivity and expression in the neuroretina of the eye that received a repeat dose compared to the eye treated only once, showcasing the Anellovector’s potential for redosing as well with no signs of ocular toxicity.​
• These data are the first demonstration of therapeutic payload delivery with an ANV vector and represent an initial investigation into the potential use of this novel platform to treat eye diseases such as wet AMD.

Anellovector Platform Overview

Title: Anellovectors, a Gene Delivery Platform Based on Commensal Human Anelloviruses, Have the Potential to Evade the Immune System and Deliver DNA Payloads to a Broad Range of Tissues in a Redosable Manner
Speaker:  Chris Wright, MD, PhD – Chief Medical Officer at Ring Therapeutics

Session Title and Location: Emerging Viral Vectors – Ballroom 4, Baltimore Convention Center

Presentation time: Thurs, May 9, 5:00 – 5:15 PM ET