CRISPR pioneer Feng Zhang is again making a mark in biotechnology, this time with a new startup focused on expanding the kinds of diseases genetic medicines can treat.
Aera Therapeutics officially debuted Thursday, announcing a combined Series A and B funding of $193 million. The company, which is headquartered in Boston, is formed around technology that’s meant to help solve one of genetic medicine’s most vexing challenges.
Gene therapy, which encompasses RNA-based medicines as well as gene replacement and gene editing applications, has been a hot area of research for biotech startups and pharmaceutical giants alike over the past decade, spurred by its potential to create powerful, long-lasting treatments for many diseases.
However, delivering gene therapies to the right area of the body for them to work remains a major obstacle. Infused genetic medicines are often limited to diseases of the liver, where they will usually end up, or to “ex vivo” applications, involving the modification of cells outside of the body.
Genetic medicines are also expensive to develop and manufacture, due in part to the high costs of the components that make them work, such as engineered viruses.
In 2021, Zhang's lab at the Broad Institute of MIT and Harvard identified a collection of proteins in the human genome that could form “capsid-like” structures capable of transporting genetic cargo within the body. One of the proteins, known as PEG10, could be engineered to "package, secrete and deliver specific RNAs," according to a Science journal article published that year.
He took that research to Bob Nelsen of Arch Venture Partners and Issi Rozen of GV, two investors with experience building gene editing companies. In September 2022, Zhang tapped former Alnylam Pharmaceuticals executive Akin Akinc to become Aera’s CEO and, more recently, longtime Alnylam leader John Maraganore to be its chairman.
The company isn’t yet working on building a pipeline of medicines, Akinc said. Instead, it’s first spending time developing its technology and identifying potential applications.
“The greatest unmet need today in terms of the genetic medicine space is delivery,” Akinc said in an interview with BioPharma Dive. “Ultimately, what we're after is a genetic medicine, and that means combining those two.”
Akinc claims Aera can address the safety and immunogenicity issues sometimes seen with current gene-based treatments. Aera is starting with human proteins, which its researchers hypothesize could lower the risk of provoking unwanted immune responses.
Cracking the code for delivery may also lead to new targets, perhaps in the central nervous system, heart or lungs, Akinc said. And it could also help with some of the problems associated with manufacturing the complex medicines.
“We’re less interested in trying to do a different way of solving a problem that's already been solved with lipid nanoparticles or viral vectors,” Akinc said, referring to the two most common ways to deliver genetic medicines.
Aera’s platform is built on work done in Zhang’s lab, where he also developed the research that led to the formation of Editas Medicine and Beam Therapeutics. Some of that technology also comes from a biotech it acquired called VNV, which Akinc described as doing “similar work” to Aera.
Arch and GV, which both bet big on gene editing company Prime Medicine, are joined by Lux Capital in leading Aera’s private financing rounds. The cash raised so far will be put to building out its team, currently numbering roughly 50 employees, Akinc said.
The company’s board of directors also includes Maraganore, former Vertex Pharmaceuticals president Vicki Sato and ex-Agios Pharmaceuticals CEO David Schenkein.
Maraganore, who advises several other biotechs, among them Beam and Orbital Therapeutics, said Aera’s technology could be “disruptive for the future of genetic medicine.”
“I'm trying to make new Alnylams, and companies like Orbital, Beam and Aera are great examples of companies I think have the potential to be the Alnylam of the future," Maraganore said.