Glycogen Storage Disease IIIA

As several well-funded companies race to make generative AI models that can design new protein therapies from scratch , one small startup has found a new use for existing AI systems: compressing large proteins to better package them in gene therapies or deliver them to hard-to-reach places like the back of the eye. The startup, called ZipBio, has raised $4 million in seed funding for the idea, it told Endpoints News exclusively. “The inspiration came from the compression of technology in the digital world,” CEO Roee Farber said in an interview. Of course, shrinking the structure of a protein and retaining its function isn’t as simple as compressing digital documents into a zip file. The startup is trying to isolate small parts of proteins that are most important for making a drug and shave off the rest. The problem is that the rest of the protein is there for a reason. Removing it can cause the entire thing to collapse. ZipBio’s solution is to use generative AI to create a new backbone that holds that key part of the protein in place. “In the past, nobody succeeded with that. Now it’s something we can do relatively trivially,” Farber said. The company hasn’t published details about how its approach works, but Farber said ZipBio is using and refining publicly available AI models for protein design, rather than creating its own. The startup was founded in 2023, began work on its lead drug just eight months ago, and is already testing it in animals, Farber said. That drug candidate builds on Regeneron’s antibody Eylea as a starting point — an eye therapy that made the company $5.89 billion last year. Eylea inhibits two forms of VEGF, a protein that can spur unwanted blood vessel growth in the eyes. But just blocking the A and B forms of the protein doesn’t fully treat everyone. ZipBio has created a compact inhibitor of VEGF-C and VEGF-D and meshed it with Eylea, using AI to create a hybrid molecule that Farber says is compact enough to travel to the back of the eye, something that a simple fusion of two full antibodies likely wouldn’t do. Clinical trials could begin as soon as 2026. The startup is also working with the French nonprofit Généthon to create a compact version of a protein that’s too big to fit into AAVs — the most common viral vectors used in gene therapy — to treat a rare disease called glycogen storage disorder III, a form of muscular dystrophy. ZipBio’s seed funding was led by NFX, a venture capital firm that invests in tech startups at a seed stage or earlier. MoreVC, which focuses on early-stage Israeli tech startups, also contributed. ZipBio’s team of 12 is currently split between Seattle and Tel Aviv, Israel.

HYOGO, Japan--( BUSINESS WIRE )-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the Phase I study of JR-441 in individuals with mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome type A). JR-441 is a recombinant form of heparan N-sulfatase that is developed using JCR’s proprietary J-Brain Cargo ® blood-brain barrier (BBB)-penetrating technology. JR-441 addresses a significant unmet medical need by crossing the BBB and targeting the neurological symptoms of MPS IIIA within the central nervous system. "MPS IIIA is a rare and life-threatening lysosomal storage disorder for which there are currently no approved treatment options for individuals living with this devastating disease, and there is a dire need to address the neurological signs and symptoms of MPS IIIA,” said Shin Ashida, Chairman and President of JCR Pharmaceuticals. “We are pleased that the PMDA’s review of the Phase I study plan is complete, and that clinical development program is progressing. We are committed to continuing to advance the JR-441 clinical development program with the goal of meeting the unmet medical needs of this patient community. We look forward to sharing updates on the program when they are available.” The European Commission (EC) and the U.S. Food and Drug Administration (FDA) granted JR-441 orphan drug designation (ODD) for the treatment of MPS IIIA. JR-441 is currently being studied in an ongoing Phase I/II clinical trial in Germany (JR-441-101, NCT06095388 ), apart from this Japanese Phase I study. The impact of this announcement on JCR's consolidated financial results for this fiscal year (ending March 2025) is expected to be minor. About Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, is an autosomal recessive disease caused by pathogenic mutations in the SGSH gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. The accumulation of heparan sulfate in several types of cells of the body, especially in the central nervous system in the brain, results in severe neurological deterioration, cognitive impairment, mild somatic involvement, and shortened lifespan. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. 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