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

Anellovivers有效载荷的扩大扩大了这类新型病毒载体的治疗潜力

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

数据显示了非人类灵长类动物（NHP）中无细胞载体的转导和可氧化性，提高了临床成功的可能性

CAMBRIDGE, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- 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).

马萨诸塞州剑桥市，2024年5月7日（环球通讯社）--Ring Therapeutics是一家生命科学公司，由Flagship先锋创立，旨在通过其共生病毒组平台彻底改变遗传药物，今天宣布了新的临床前数据，证明其在非人类灵长类动物中成功转导和可氧化载体，以及扩大载体的有效载荷能力，使其超过无病毒（ANV）的自然基因组大小。

The data, which will be presented as one oral presentation and two posters at the 27th Annual America Society of Gene & Cell Therapy (ASGCT) conference, showcases the viability of Anellovectors as the next generation of viral vectors..

这些数据将在第27届美国基因与细胞治疗学会（ASGCT）年会上以一份口头报告和两张海报的形式呈现，展示了无细胞载体作为下一代病毒载体的可行性。。

'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.

Ring Therapeutics首席执行官兼Flagship先锋首席执行官Tuyen Ong博士（MBA）说：“我很高兴我们的Ring团队在非人灵长类动物中取得了这一重要里程碑，展示了无载体的转导，可氧化性和扩展的有效载荷能力。”就在几年前，我们开始从人类共生病毒中创建一种安全有效的病毒载体，NHP数据为无载体如何在人类中发挥作用提供了重要见解。

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.”.

此外，anellovivers扩大的有效载荷能力扩大了这种新型载体类别的治疗潜力，允许更多的患者接受潜在治疗。这些数据表明，我们在将无载体推向临床方面取得了重大进展，为患者提供了改变生命的疗法。”。

Presentation details and key highlights below:

演示细节和关键亮点如下：

Expanded Payload Capacity

扩大有效载荷容量

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

标题：无细胞载体：将基于无细胞病毒的载体的有效载荷能力扩展到野生型基因组能力之外

Abstract number: 450

摘要编号：450

Presenter: Cato Prince

主持人：Cato Prince

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

展示地点、日期和时间：展览厅，星期三，5月8日，美国东部时间下午12:00

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.

使用越来越大的载体基因组，我们已经证明，无载体能够包装高达5.0kb的载体基因组，比野生型基因组容量高达65%。

The overall potential for Anellovector cargo capacity appears to exceed the ~4.7 kb cargo capacity of Adeno-associated viruses (AAV).

无载体载药量的总体潜力似乎超过了腺相关病毒（AAV）约4.7 kb的载药量。

Unlike AAV, which appears to exhibit a strict packaging limit, Anellovectors allow for the packaging of genomes larger than the wild-type genome.

与似乎表现出严格包装限制的AAV不同，AnelloVector允许包装比野生型基因组大的基因组。

A combination of qPCR and next-generation sequencing was used to verify that the encapsidated expanded cargo DNA is intact, circular, and single-stranded.

qPCR和下一代测序的组合用于验证包裹的扩增货物DNA是完整的，环状的和单链的。

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.

扩大无载体的有效载荷能力建立在其免疫有利性和向性的内在特征的基础上，以努力达到需要基因治疗的尽可能广泛的患者群体。

Transduction and Redosability in NHPs

NHPs的转导和可氧化性

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

标题：源自人类视网膜色素上皮的无细胞载体在非人灵长类动物的视网膜中传递并表达治疗相关的DNA有效载荷

Abstract number: 444

摘要编号：444

Presenter: Parmi Thakker

礼物：Parmi Thakker

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

展示地点、日期和时间：展览厅，星期三，5月8日，美国东部时间下午12:00

Anellovectors, when administered intravitreally in NHPs, demonstrated transgene infectivity and expression in ocular tissues.

当在NHP中玻璃体内给药时，Anellovivers表现出转基因感染性和在眼组织中的表达。

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.​

此外，我们观察到，与仅接受一次治疗的眼睛相比，接受重复剂量治疗的眼睛的神经视网膜中转基因感染性和表达显着增加，这表明无细胞载体具有氧化的潜力，并且没有眼部毒性的迹象。​

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.

这些数据是ANV载体治疗有效载荷传递的首次证明，代表了对这种新型平台治疗湿性AMD等眼病的潜在用途的初步研究。

Anellovector Platform Overview

Anellovector平台概述

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

标题：无细胞载体是一种基于共生人类无细胞病毒的基因传递平台，有可能逃避免疫系统，并以可氧化的方式将DNA有效载荷传递给多种组织

Speaker: Chris Wright, MD, PhD – Chief Medical Officer at Ring Therapeutics

演讲者：Chris Wright，医学博士，博士，Ring Therapeutics首席医疗官

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

会议标题和地点：新兴病毒载体——巴尔的摩会议中心第4舞厅

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

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

About Ring Therapeutics

关于Ring Therapeutics

Ring Therapeutics is revolutionizing the genetic medicines and nucleic acid space by harnessing the most abundant and diverse member of the human commensal virome, anelloviruses. The company developed the Anellogy™ platform which focuses on anelloviruses to potentially treat a broad range of diseases.

Ring Therapeutics通过利用人类共生病毒体中最丰富和多样的成员，即无细胞病毒，正在彻底改变遗传药物和核酸空间。该公司开发了Anellogy™平台，该平台专注于anelloviruses，可以治疗多种疾病。

Through harnessing the unique properties of these commensal viruses, the Anellogy™ platform generates diverse vectors that exhibit both tissue-specific tropism and the potential to be re-dosed. Ring Therapeutics, founded by Flagship Pioneering in 2017, aims to develop and further expand its portfolio by leveraging its platform to unlock the full potential of gene therapy and nucleic acid medicines, enabling a variety of mechanisms that successfully deliver therapeutic cargo to unreachable organs and tissues.

通过利用这些共生病毒的独特特性，Anellogy™平台产生了多种载体，这些载体既具有组织特异性，又具有重新给药的潜力。Ring Therapeutics于2017年由Flagship Expandative创立，旨在通过利用其平台来开发和进一步扩大其投资组合，以充分发挥基因治疗和核酸药物的潜力，从而实现各种机制，成功地将治疗货物输送到无法到达的器官和组织。

To learn more, visit https://www.ringtx.com/ or follow us on X (Twitter) at @Ring_tx..

要了解更多信息，请访问https://www.ringtx.com/或者在X（推特）@Ring_tx上关注我们。。

Press Contacts

新闻联系人

Ring Therapeutics Media

Ring Therapeutics媒体

Brittany Leigh, PhD

Brittany Leigh博士

LifeSci Communications

LifeSci通信

bleigh@lifescicomms.com

bleigh@lifescicomms.com

813-767-7801

813-767-7801