Mosaic Neuroscience and iXCells Biotechnologies USA, Inc. announced a pioneering pilot project designed to revolutionize ALS drug development through patient-specific disease modeling. The collaboration represents the inaugural study for Project Mosaic, an ambitious initiative aimed at addressing the devastating 99% failure rate of treatments for sporadic ALS (sALS), which affects 90% of ALS patients.
Addressing Critical Gaps in ALS Research
The partnership tackles a fundamental problem in ALS drug development: the mismatch between disease models used in research and the reality of patient diversity. According to Bernie Zipprich, Mosaic's founder and CEO, "Disease models are one of the most overlooked bottlenecks in ALS drug development. In cancer, testing drugs in patient-derived cells is standard before human trials. Since you can't biopsy an ALS patient's brain, Project Mosaic is creating 'neurobiopsies' – lab-grown neurons that replicate an individual's disease."
Current ALS research predominantly relies on models based on rare familial forms of the disease, which affect fewer than 10% of patients. Eugene Brandon, PhD, Mosaic's CSO and neuronal stem cell industry veteran, emphasized the disconnect: "The vast majority of ALS patients have the sporadic form of the disease, but nearly all of our preclinical tools are based on the familial forms. It's like trying to develop drugs for liver cancer using only HER2-positive breast cancer cells."
Revolutionary Approach to Precision Neurology
Project Mosaic builds on emerging scientific evidence suggesting ALS is not a single disease but rather a "mosaic" of overlapping subtypes with distinct characteristics. More than 50 genes have been identified as important ALS triggers in subsets of cases, indicating diverse and distinct causes. Multiple laboratories have found strong evidence of sALS subtypes in data derived from hundreds of autopsied patients.
The pilot project will utilize patient-derived stem cell lines from Answer ALS, previously profiled by Johns Hopkins researchers Jeff Rothstein, PhD, and Alyssa Coyne, PhD. The goal is to generate transcriptomic signatures indicative of sALS that are unique and consistent for each patient. Future work will investigate how these patterns can help match patients to specific drugs.
Strategic Partnership and Technology Platform
iXCells Biotechnologies was selected through a competitive process based on the company's experience in ALS modeling, R&D capacity, biomanufacturing abilities, scientific leadership, and commitment to ALS innovation. The company's new iPSCore platform, designed for precision drug development in rare diseases, made it an ideal partner for Project Mosaic.
"What excites us about Project Mosaic is not just the urgency – it's the rigor," said Nianwei Lin, PhD, President and CSO of iXCells. "This isn't just another iPSC study. It's a systematic effort to make sALS disease models a new preclinical standard."
Personal Mission Driving Innovation
The initiative stems from personal experience with the disease. Zipprich, a Harvard and Wharton-trained healthcare entrepreneur, was diagnosed with ALS in 2020 at age 32. He launched Mosaic in 2024 with the specific goal of modernizing how ALS drugs are tested before human trials.
"Project Mosaic is not just about cutting-edge research," Zipprich explained. "It's about fixing the broken ALS pipeline – at the speed this disease requires. This can only happen if ALS nonprofits, academic researchers, and industry work together in new ways. Someone new is diagnosed every 90 minutes and people are dying every day. We have no time to waste."
Future Implementation and Industry Impact
Following the pilot study, iXCells plans to make sALS models with standardized phenotypes available to researchers and drug makers. Project Mosaic will then advance to stage two, inviting additional academic and industrial laboratories to cross-validate and build on the pilot results.
The collaboration aims to accelerate the translation of academic advances into industry adoption and patient impact. By creating industrialized disease models that reflect the diversity of sporadic ALS, the project seeks to establish a new preclinical standard that could significantly improve drug development success rates for the majority of ALS patients.
