Dive Brief:

• A new biotechnology company called Judo Bio is taking a strike at kidney diseases, armed with $100 million and a RNA drug platform it claims can get to targets that historically have been challenging for researchers to hit.
• Formed three years ago, Judo is backed by a handful of investors that include Atlas Venture, which incubated the startup and co-led its Series A, as well as The Column Group and Droia Ventures.
• With its technology, Judo is following in the footsteps of Alnylam Pharmaceuticals, which pioneered use of a biological process called RNA interference to make a new class of drugs. Unlike Alnylam’s first medicines, which are directed at the liver, Judo claims it can reliably sneak its RNA medicines into the kidney.

Dive Insight:

First discovered in 1998, RNA interference gave drugmakers a tool to silence genes that encode disease-causing proteins. In the nearly three decades since, Alnylam has used that tool to develop five approved medicines for a range of rare and common diseases. (A sixth, from Novo Nordisk-owned Dicerna Pharmaceuticals, is also approved.)

Those medicines work via what’s known as small interfering RNA, or siRNA, which can mute messenger RNA and thereby prevent problematic proteins from being made by a cell.

Judo sees itself as a successor of sorts. Its leadership includes Alnylam veteran Alfica Sehgal, who now serves as the startup’s chief scientific officer, while John Maraganore, Alnylam’s former CEO, sits on its advisory board.

The company’s platform is built around so-called ligand-siRNA conjugates, which pair the gene silencing nucleic acid with a targeting molecule. Judo’s ligands shuttle the RNA payload to specific types of kidney cells, where they bind to a receptor known as megalin that helps cells absorb vitamins and solutes.

Bound to megalin, Judo’s drug is ingested by the cell and, once inside, works to silence mRNA expression of solute carrier proteins. These proteins typically act as transporters and their dysfunction is linked to a range of diseases.

According to Rajiv Patni, Judo’s CEO, using a “proven” mechanism like siRNA allowed the company to pursue more challenging drug targets like those in the kidney.

“The kidney is a huge risk — in part but not entirely — because of its complexity,” Patni said. “As a drug developer, you're always thinking about a tissue target that is the holy grail.”

The kidney's complexity is also why Judo chose to focus on the organ. It's where blood is filtered and, while other drugs can reach the organ, they’re typically excreted without any helpful effect.

The company has not disclosed disease targets, but Patni says Judo is looking at systemic diseases such as diabetes and hypertension, as well as kidney-specific conditions.

Judo shares its name with the form of martial arts known for taking a weakness and turning it into a strength, said Sehgal, the Alnylam vet. One of Judo’s primary moves, a strike, is also the acronym for the company’s platform, which is being developed to knock down certain genes via the kidney.

The company is presenting preclinical data at the Oligonucleotide Therapeutics Society’s annual meeting this week that it says show ligand-siRNA conjugates can durably knock down targeted gene expression.

Editor’s note: This story has been updated to clarify the biological activity of megalin.