New Gene-Editing Biotech Era Therapeutics From Pioneer Feng Zhang

  • CRISPR pioneer Feng Zhang is building a new gene-editing startup, Insider has learned.
  • Aera Therapeutics is working to improve the delivery of CRISPR — a key challenge in gene-editing.
  • Arch Venture Partners and GV have invested in Aera, which has raised about $200 million.

CRISPR pioneer Feng Zhang is in the process of launching yet another ambitious gene-editing startup, backed by approximately $200 million from some of biotech’s biggest investors, Insider has exclusively learned.

The Cambridge, Massachusetts-based biotech, named Aera Therapeutics, hopes to crack a persistent challenge in the gene-editing field: delivering genetic goods, including CRISPR, into different organs inside the body. Based on some of Zhang’s research published last yearAera is developing better gene carriers to reach more cells and organs than currently possible, ultimately hoping to expand the number of diseases that CRISPR can treat.

The weather is still in stealth mode, meaning it hasn’t been publicly announced. An insider confirmed its existence by reviewing corporate filings along with interviewing people familiar with the company.

Aera Therapeutics could be the FedEx of gene-editing companies

Genetic technologies like CRISPR and mRNA have progressed rapidly in the past few years, but how to deliver these medicines into the body remains a “multibillion-dollar question,” said Josh Wolfe, a cofounder and managing partner of the VC firm Lux Capital. Wolfe compared the opportunity to the rise of delivery giants FedEx and UPS, which grew alongside the internet and online commerce.

“I am convinced the most important piece of the next chapter is who is going to be the FedEx and UPS to do targeted delivery, not to a town or a city, but to a specific apartment or home, or in this case, a specific cell or organ,” Wolfe told Insider.

Aera was incorporated in September 2021, shortly after Zhang’s research was published in Science. The company currently is hiring up and has already acquired VNV, a Lux-backed biotech startup that was pursuing a similar delivery idea, according to Jason Shepherd, a University of Utah neurobiologist who cofounded VNV and now consults for Aera.

Aera has recently hired a CEO and raised about $200 million, Shepherd added, although he declined to name the CEO.

The startup’s only backers listed in its Massachusetts corporate filings are Arch Venture Partners and GV, with Arch’s managing partner Robert Nelsen, GV venture partner Issi Rozen, and GV general partner David Schenkein listed as the company’s current officers and directors. Nelsen and GV both declined to comment.

Aera is the latest company founded by CRISPR pioneer Feng Zhang

Zhang is not listed on the corporate filings, but is a cofounder of the company, says Shepherd.

The 40-year-old Zhang has become a prolific gene-editing researcher and company creator at the Broad Institute. Aera is at least the seventh startup Zhang has cofounded, joining a list that includes base-editing biotech Beam Therapeutics, CRISPR company Editas Medicine, and gene-editing startup Arbor Biotechnologies. Zhang did not respond to an interview request from Insider.

Genetic medicines have two key parts: the cargo and the container. For a gene-editing medicine, for instance, the cargo is the DNA-modifying CRISPR. The container is the packaging that carries that cargo into the body and inside of cells.

Most of Zhang’s previous startups — and the vast majority of the gene-editing field — have focused on developing new cargo.

The space has gravitated to using two types of containers: lipid nanoparticles, which are microscopic balls of fat, or hollowed-out viruses. Both have limitations. Lipid nanoparticles tend to land in the liver, which is ideal for editing liver cells but less helpful in reaching other organs. Hollowed-out viruses can only happen a tiny amount of cargo inside, and many biotechs are grappling with toxicity issuesas the body’s immune system reacts to a foreign virus.

Aera hopes its approach can reach more organs and cause less of an immune reaction, Shepherd said.

A surprise discovery about a memory gene leads to a new delivery idea

A headshot of Jason Shepherd, University of Utah neurobiologist

Jason Shepherd, University of Utah neurobiologist

Jason Shepherd

While Area is based on a Cambridge biotech incubator called Alexandria LaunchLabs, some of its research came from Shepherd’s neurobiology lab in Utah. He studied a memory-forming protein in our brains called ARC, and found that it evolved from, and even acts like, a virus. This protein forms shells that viruses use to protect themselves and enter cells.

Shepherd published these findings in a 2018 Cell paperand venture-capitalists came calling, interested in seeing if this ARC protein could be a new delivery tool to deliver RNA into cells.

Lux and Shepherd started a stealth biotech to see if it could use the ARC protein to deliver a desired piece of RNA. Just as that startup — called VNV, or Virus Not Virus — was gearing up to start animal testing and raise a Series A, Zhang published a Science paper in August 2021. That study found there were many virus-like particles in the human body like ARC.

While the ARC protein seems tailored for brain delivery, Zhang’s suite of proteins could reach other organs, Shepherd said.

Zhang called his system SEND — a loose-fitting acronym for Selective Endogenous Encapsidation for Cellular Delivery.

“The biomedical community has been developing powerful molecular therapeutics, but delivering them to cells in a precise and efficient way is challenging,” Zhang said in an August 2021 statement about his research. “SEND has the potential to overcome these challenges.”

Zhang initially named the startup TranSEND, but renamed it this past July to Aera after running into copyright concerns, Shepherd said. Shepherd added the company isn’t exclusively focused on delivery, as it also has licensed some of Zhang’s research on gene-editing technologies.

“The promise of being able to impact something as important as gene therapy is super exciting,” Shepherd said.

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