By targeting RNA rather than DNA, Amber Bio aims to treat multiple genetic diseases with fewer safety risks.
Gene editing technology using Crispr holds a lot of promise for treating genetic disorders like type 1 diabetes and sickle cell anemia. Studies have demonstrated effectiveness by changing patients’ DNA to correct mutations. The FDA is weighing approval of at least one treatment using this technology by the end of the year.
But the technique has drawbacks: DNA editing treats only one mutation at a time, causes permanent genetic modifications and has potentially irreversible safety risks.
That’s where San Francisco-based Amber Bio comes in. It uses Crispr gene editing tools to target RNA, rather than DNA, which has the potential to correct a broader variety of genetic disorders while reducing safety risks. On Thursday, the company announced that it had emerged from stealth with a $26-million seed funding round that was led by Playground Global. Eli Lilly, Andreessen Horowitz, Hummingbird Ventures and other firms participated in the round as well. The new capital is geared toward advancing the company’s RNA editing platform and hiring high caliber scientists to join its development team.
Amber cofounder Basem Al-Shayeb says that the current approach many biotech and pharmaceutical companies are taking to gene editing is like having a word processor “Individually searching for correcting different typos across hundreds of different files.”
That approach involves editing the RNA in the cell, which is what cells use to make needed proteins by copying instructions on what to make from DNA. By targeting RNA rather than DNA with its Crispr tools, Al-Shayeb believes they could correct the outcomes of genetic defects without running into many of the challenges involved in making changes to the DNA itself.
In 2021, Al-Shayeb, a 29-year-old alumnus of the Forbes 30 Under 30 list for Science, says he met Jacob Borrajo through mutual friends and that the two soon became an “unconventional team.” At the time, Al-Shayeb was studying Crispr protein tools in Nobel Prize winner Jennifer Doudna’s lab at the University of California, Berkeley. Borrajo, 34, meanwhile, was working on new RNA technologies at the Broad Institute, a biotechnology institution at MIT and Harvard. The two were both intrigued by not only treating common mutations but those that are deemed untreatable.
After realizing the potential of the research they were working on, the pair founded Amber Bio in 2022. The company is currently interested in applying its technology to genetic disorders of the eye, though it declined to disclose any specific disease targets.
Amber isn’t the only biotech company looking into the potential of using gene editing tools in combination with RNA. Other competitors include Korro Bio, Wave Life Sciences and Shape Therapeutics. But Jory Bell, a general partner at Playground Global, thinks the pair are up to the challenge. “We think it’s a lot easier to find world class scientists and support them into becoming successful entrepreneurs than the other way around.”
Al-Shayeb and Borrajo are clear eyed about the challenges of both developing a new way to tackle gene therapies while also trying to attract talent in a crowded industry, but are hopeful the seed investment will help them attract more scientists. In particular, they are hopeful that their company will be able to move forward in development with therapies that might be useful for more than one disease.
“We want to innovate new genetic medicines that can extend broadly across diverse patient populations without the need to custom tailor to each specific mutation,” says Borrajo. “That’s what gets us at Amber really excited and that’s what we’re building today.”
This article was first published on forbes.com and all figures are in USD.
