Biotech Start-Up Ring Therapeutics Hopes to Fix a Key Shortcoming of Gene Therapies – Barron’s

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Gene therapies were once touted as a lifetime cure for crippling, costly inherited disorders. But clinical trials of the treatments are showing signs that some may lose effectiveness after five or six years. If that happens, patients could find themselves unable to receive the treatment again because they have developed antibodies to the engineered viruses that deliver most gene therapies.

Ring Therapeutics hopes it can provide gene therapies with a second act.

The privately held start-up in Cambridge, Mass., published a study Tuesday in the science journal Cell Host & Microbe about a family of viruses that Ring has engineered to deliver genetic medical treatments repeatedly because they dont provoke immune defenses. Called anelloviruses, this diverse family of viruses appears to have lived inside us, without causing disease, for as long as humans have existedsimilar to the many innocuous bacteria in our guts microbiome. Rings customized versions of these viruses could safely solve gene therapys inability to repeat dosing.

This could be a completely transformative approach to gene therapy, Ring chief executive Tuyen Ong told Barrons. Ring has protected its anellovirus technology with a moat of patent applications.

With just one peer-reviewed publication on Rings work, its still early days. The start-up has tested what it calls its Anellogy engineered-virus technology in test tubes and lab animals. It has yet to plan clinical trials in humans. If other gene therapy trials are any guides, those trials will last several years.

To bring its anellovirus technology into clinical trials, Ring said Wednesday that it has raised $117 million in a second round of funding led by its founding venture backers at Flagship Pioneering. More than an investment firm, Flagship has created and incubated dozens of biotech firms, including Covid-19 vaccine innovator Moderna (ticker: MRNA).

Ring chairman and Flagship partner Avak Kahvejian said that the growing scientific interest in the health implications of our bacterial microbiome inspired Flagship in 2017 to start searching for viruses that might also lurk harmlessly in our cells. A few anelloviruses has been discovered in the 1990s. The Flagship researchers found an unexpected variety of these viruses in different tissues of our bodies. No ones shown that these viruses cause disease. Because anelloviruses have a circle of DNA, the new venture was called Ring Therapeutics.

Most gene therapies on the marketor in clinical trialsget their therapeutic genes into a patients cells by packing a DNA cargo into hollowed-out versions of another kind of virus known as adeno-associated viruses, or AAV. Companies likeRegenxbio(RGNX) have specialized in engineering AAVs as vectors for carrying gene therapies.

But AAV vectors trigger an immune response, so they can be administered just once in a patients lifetime. If AAV gene therapy results change with time, as some clinical trials hint, they couldnt be re-administered. Because they have thrived in humans for millennia, anellovirus vectors dont seem to have that problemand Ring says that immune responses havent been seen in lab animals.

Ring CEO Ong said the company has been able to engineer anelloviruses that can carry gene therapy cargoes of similar size to the carrying capacities of AAV vectors. Ring can make vectors that target different parts of the body. And most important, its anellovirus vectors can be potentially dosed repeatedly.

Mother Nature handed us this amazing set of aces, said Ong, which have a natural advantage over other forms of viral delivery.

Corrections & amplifications: Ring Therapeutics raised $117 million in its recent private funding round. An earlier version of this article incorrectly reported the amount as $112 million.

Write to Bill Alpert at william.alpert@barrons.com

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Biotech Start-Up Ring Therapeutics Hopes to Fix a Key Shortcoming of Gene Therapies - Barron's