HealthOne of the world's best gene-editing researchers wants to "break the barrier" of science with his mysterious new company

One of the world’s best gene-editing researchers wants to “break the barrier” of science with his mysterious new company

One of the world’s best gene editing researchers has spoken with Business Insider about your company’s ambition to solve one of the thorniest problems in the field.

CRISPR pioneer Feng Zhang spoke with Business Insider about Aera Therapeutics, his Boston-based biotech. On Thursday, the company announced that it had raised $193 million in a combined Series A and Series B funding round led by Arch Venture Partners, GV and Lux ​​Capital.

Aera researchers hope to address the persistent lack of effective methods for delivering genetic drugs—or drugs that manipulate life’s genetic codes in DNA or RNA—into the body. If it works, Aera could help extend methods as powerful as Nobel Prize-winning CRISPR gene-editing tools to more parts of the body, potentially treating more diseases.

“The goal is to break the barrier that genetic medicine is facing,” Zhang told Business Insider in his first interview about the company. “Right now, one of the biggest barriers is administration. That’s one of the main challenges that Aera is trying to make an impact on.”

Founded in September 2021, Aera has not made its plans public so far. In October of last year, Business Insider unveiled Aera’s plans to bring gene editing to more parts of the body, and in February posted that the startup had hired former Alnylam executives to run the company. Its CEO told Business Insider that the company has grown to around 50 employees, with plans to hit 75 or 80 by the end of 2023.

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The startup joins a growing list of startups founded by Zhang, one of the world’s leading gene-editing researchers, including base-editing biotech Beam Therapeutics, CRISPR company Editas Medicine and gene-editing startup Arbor Biotechnologies. When starting another company, Zhang believes it is important to have a dedicated team that deals with results. As with his other companies, Zhang advises Aera while remaining a researcher at the Broad Institute.

Despite its enormous potential, CRISPR has been faced with a management dilemma

Akin Akinc

Part of Aera’s story begins in the Utah lab of Jason Shepherd, a neurobiologist who wanted to better understand how the brain makes memories. He studied a memory-forming protein called ARC and found that it evolved from, and even acted like, a virus to enter cells.

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After Shepherd published his findings in 2018, Zhang found that the ARC protein was not an abnormality. She discovered dozens of proteins in the human body that basically looked and acted similar to viruses. In an article by Science In 2021, Zhang’s lab showed that they could also create potential drugs from these proteins by injecting them with a bit of messenger RNA that can take CRISPR or other gene-editing tools inside cells to treat a disease. Aera later acquired Shepherd’s startup, which was pursuing a similar management idea.

According to Akin Akinc, CEO of Aera, the company now has a collection of about 80 such proteins, about 50 of which come from Zhang’s initial paper. The company found out more through further investigation. The company is still in the early stages of research, trying to better understand these proteins and determine which ones are best suited for use as medicines. According to Akinc, the company has not yet determined which diseases it will focus on first.

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“We are laying the foundations for the future,” he adds.

But Akinc is already clear on one important detail: Aera can synthetically create these delivery vehicles, which the company calls protein nanoparticles. In other words, Aera won’t have to rely on cell culture to create its treatments. This has turned out to be a difficult and delicate process for other management ideas.

Aera hopes to help delivery technologies catch up with the emergence of genetic platforms such as CRISPR gene editing, mRNA and RNA interference. Until now, these technologies have been largely limited to the treatment of liver or eye diseases. If Zhang’s proteins work, these genetic modification methods could reach new organs, meaning they could treat more diseases.

“In the last decade there has been an incredible evolution in the number of genetic medicine modalities available, but I would argue that progress in delivery systems has lagged behind,” says Akinc. “That, as a result, has limited the wide and widespread application of genetic medicines.”

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