LRP2

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Judo Bio , a biotechnology company pioneering oligonucleotide medicines delivered to the kidney, today announced that the company will present mechanistic and preclinical data that demonstrate the use of megalin receptors for intracellular delivery of ligand conjugated-siRNA therapeutics to silence production of target mRNA in the kidney. These conjugated siRNA are developed by Judo Bio’s proprietary STRIKE (Selectively Targeting RNA Into KidnEy) platform, and are internalized by the recycling receptor megalin. The data will be presented at the American Society of Nephrology (ASN) Kidney Week taking place October 23-27 in San Diego. Details of the poster presentation are as follows: Title: Targeted Delivery of siRNA to Proximal Tubule Cells in Kidney Session Name: Genetic Kidney Diseases: Models, Mechanisms, and Therapies [PO1202-3] Session Date and Time: October 26, 2024 from 10:00 a.m. to 12:00 p.m. PT Location: San Diego Convention Center, Exhibit Hall Poster Number: SA-PO637 About Judo Bio Judo Bio is pioneering oligonucleotide medicines delivered to the kidney, opening the way for new genetic medicines for systemic and renal diseases. With its STRIKE (Selectively Targeting RNA Into KidnEy) platform, the company is using a proprietary approach to create ligand-RNA conjugate drugs designed for receptor-mediated update by specific kidney cell types, resulting in gene silencing of disease-modifying target genes. Judo Bio’s initial pipeline programs are megalin-STRIKERs that use the megalin receptor family to selectively deliver siRNA therapeutics to the proximal tubule of the kidney to silence mRNA expression of specific solute carrier proteins (SLCs), thereby inhibiting the uptake of circulating solutes linked to systemic diseases. Located in Cambridge, MA, Judo Bio’s team and advisors include experts in oligonucleotide therapies and innovative drug development. For more information, visit and follow us on LinkedIn . Contacts Media contact: Kathryn Morris The Yates Network LLC 914-204-6412 kathryn@theyatesnetwork.com

The kidney hasn’t always gotten much love from genetic medicine. The organ is vital to our health, but is often viewed as a liability for drug developers. It can flush out otherwise helpful therapies, and many efforts to deliver medicines to it have been foiled by its intricate and effective plumbing. A new startup says that it’s finally figured out how to shuttle RNA-based medicines directly into specific cells of the kidney. After three years in stealth, the Cambridge, MA-based startup, Judo Bio, launched on Monday with $100 million in a combined seed and Series A financing, co-led by Atlas Venture, The Column Group and Droia Ventures. CSO Alfica Sehgal said the idea for the company came during a brainstorming session about the lack of precise treatments in kidney care. “The kidney is not straightforward. It’s a very complex organ with dozens of specialized cell types,” Sehgal told Endpoints News in an interview. To try and solve the problem, the company borrowed a page from one of genetic medicine’s best-known delivery targets — the liver. Alnylam, which pioneered the first gene-silencing treatments, figured out that attaching a sugar molecule known as GalNAc to a siRNA drug would help the treatment get swept up by certain receptors on liver cells. So Judo’s team began to wonder if a similar pairing could be discovered to shuttle siRNA medicines into kidney cells. The startup focused on a kidney receptor known as megalin, which acts like a hungry scavenger, sucking up all manner of proteins and other biomolecules, Sehgal said. Judo has been working on finding molecules that they can use to get siRNA drugs swept up by megalin receptors in the proximal tubular epithelial cells of the kidney, she added. Judo isn’t disclosing the chemical makeup of its megalin-targeting molecules, but in a poster presentation at an RNA conference in Montreal this week, it revealed that in a mouse study, its approach increased the exposure of siRNA in the kidney up to 30-fold compared with naked RNA molecules. One injection lowered gene expression by 50% to 70%, and the effect lasted four weeks. Now more genetic medicine developers are beginning to look at the organ. In August, Versant Ventures and Novartis launched Borealis Biosciences with $150 million to develop RNA medicines for kidney diseases — it disclosed even fewer details about how it plans to deal with delivery. Alnylam leaders have also pointed to the kidney as a potential area of interest as part of its effort to expand the reach of its siRNA medicines beyond the liver. Judo CEO Rajiv Patni, who was recruited to lead the startup last month, told Endpoints it will initially use its siRNA drugs to shut down the production of solute carrier proteins, which line the tubes of the kidney and control levels of molecules like amino acids and glucose in the blood. A class of diabetes drugs known as SGLT2 inhibitors works by blocking certain solute carrier proteins. The startup Jnana Therapeutics, which Otsuka Pharmaceutical bought in a deal worth at least $800 million in August, was focused on making small molecule inhibitors to some of the hundreds of other proteins in the solute carrier family. Judo aims to make genetic medicines that tamp down on these proteins with longer-lasting effects. Patni, who was most recently the head of R&D at Reata Pharmaceuticals before Biogen acquired the company for $7.3 billion last year, won’t say what diseases Judo will focus on first, but he is interested in both rare metabolic diseases like phenylketonuria and urea cycle disorders, as well as more common conditions including diabetes, gout, heart failure and hypertension. “We’re using kidneys as the access point,” Patni said. The company also plans to target kidney cells known as podocytes to directly treat renal diseases, Patni added. But he declined to say when he expects Judo to have a drug candidate ready for the clinic or how long the company’s new funding will last.

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 Alnylams 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 whats 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 startups chief scientific officer, while John Maraganore, Alnylams former CEO, sits on its advisory board.The companys platform is built around so-called ligand-siRNA conjugates, which pair the gene silencing nucleic acid with a targeting molecule. Judos 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 fats.Bound to megalin, Judos 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, Judos 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, theyre 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 Judos primary moves, a strike, is also the acronym for the companys platform, which is being developed to knock down certain genes via the kidney.The company is presenting preclinical data at the Oligonucleotide Therapeutics Societys annual meeting this week that it says show ligand-siRNA conjugates can durably knock down targeted gene expression. '