A Boston biotech founded by researchers at the Broad Institute of MIT and Harvard has landed $157 million for its big plans to develop pills for cancer, immune conditions, neurological diseases, and a rare metabolic disorder.
Jnana Therapeutics announced Tuesday that it raised $107 million in series C financing, more than doubling the $100 million it raised in its previous two rounds. Jnana also struck its second drug discovery collaboration with the pharma giant Roche that will earn the startup $50 million up front plus potential milestone payments that could exceed $2 billion.
In addition, the five-year-old startup began its first clinical trial. Jnana is testing an experimental pill that it developed to help lower blood levels of a protein component that is toxic to people with a rare genetic disease called phenylketonuria, or PKU.
Harvard University chemist Stuart Schreiber and Massachusetts General Hospital gastroenterologist Dr. Ramnik Xavier cofounded Jnana in 2017. Both researchers are members of the Broad Institute.
The startup’s name (pronounced ja-na-na) comes from a Sanskrit word “meaning knowledge or wisdom gained from experience,” said cofounder and chief executive Joanne Kotz, a former director at the Broad Institute.
Jnana got its start in an incubator space inside the Boston drug company Vertex Pharmaceuticals. The startup has since grown to about 75 employees and now has a lab of its own in Boston’s Seaport District. “We’ve really enjoyed being in the Seaport and are excited to be part of the growing biotech ecosystem here,” Kotz said.
The firm recently began testing the safety of its PKU drug in healthy volunteers and will later test it in people with the disease. PKU is an inherited condition caused by a broken or missing enzyme that’s crucial for metabolizing phenylalanine, one of the 20 building blocks in proteins. Without the enzyme, phenylalanine can rise to dangerously high levels that damage the brain.
People with PKU must maintain strict diets to avoid phenylalanine. But the molecule is so ubiquitous that it’s even found in some foods and drinks that don’t list protein on their nutrition labels. Although there are some medications for the disease, they don’t work well for everyone.
Jnana’s drug is designed to increase the excretion of phenylalanine in urine and lower its levels in the blood by blocking a transporter protein in the kidneys that normally reabsorbs the molecule into the bloodstream. Kotz expects early studies of the drug to wrap up in 2024.
The startup is far from alone in its ambitions to bring new treatments to PKU patients. Because the cause of the disease is well understood, a large number of biotech firms are developing drugs for it, including gene therapies that could yield a cure. But that competition isn’t deterring Jnana.
“We believe that an oral once-a-day or twice-a-day compound that can treat any patient, at any age, with any genetic background, will be highly valuable,” said Joel Barrish, Jnana’s president, chief scientific officer, and cofounder.
Going forward, most of the company’s internal focus will be on immune-mediated diseases and cancer, Barrish added, rather than rare metabolic diseases. The new partnership with Roche will focus on cancer, immune and neurological diseases.
Jnana was built around a class of techniques known as chemoproteomics, which enable scientists to screen large numbers of chemical compounds to find ones that interact with disease-causing proteins. Those compounds then become the starting points for designing new drugs.
The company initially used the technique to hone in on a class of transport proteins that move molecules from one place to another in the body, and have been implicated in many diseases, including PKU. Jnana’s first partnership with Roche, formed in 2020, was focused on these transport proteins.
Since then, Jnana has “pushed the platform broadly” to find drugs for other tough-to-target proteins, Kotz said, including transcription factors, which control how large swathes of genes are turned on or off and play a role in many cancers. Jnana’s second partnership with Roche is focused on this new line of research.
Kotz said that the series C financing should sustain the company for more than two years. The round was led by Bain Capital Life Sciences, and several of Jnana’s previous investors, including Pfizer Ventures, chipped in as well.