The lab will run the inaugural study for Project Mosaic, a groundbreaking initiative to develop patient-specific models of sporadic ALS and fix a pipeline where 99% of treatments fail.

Mosaic Neuroscience and iXCells Biotechnologies USA, Inc. (“iXCells”) today announced a groundbreaking pilot project aimed at making it easier to develop precision drugs for ALS, a notoriously complex and deadly disease.

Launched as part of “Project Mosaic” – an ambitious effort to transform how drugs are developed for ALS by adapting methods from cancer research – the pilot is designed to advance industrial methods of creating patient-specific models of sporadic ALS (sALS), which accounts for 90% of ALS cases. If successful, Mosaic Neuroscience, the public benefit company organizing Project Mosaic, hopes to use the patient-specific models, multiomics data, and advanced AI to help drug makers develop precision medicines for sALS.

“Disease models are one of the most overlooked bottlenecks in ALS drug development,” said Bernie Zipprich, Mosaic’s founder and CEO. “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. These models will give us a path to develop precision therapies for the majority of patients. We’re thrilled to be partnering with iXCells to take the first steps in this critical journey.”

IXCells Biotechnologies was chosen through a competitive process by the Mosaic Neuroscience team, with input from the project's scientific advisors, based on the company’s experience in ALS modeling, its R&D capacity, biomanufacturing abilities, scientific leadership, and commitment to innovation for ALS. Its new iPSCore platform, designed for precision drug development in rare diseases, made iXCells a natural 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.”

Turning Adversity into Innovation

Diagnosed with ALS in 2020 at the age of 32, Zipprich – a Harvard and Wharton-trained healthcare entrepreneur – launched Mosaic in 2024 with the goal of addressing the high rate of sALS clinical trial failures by modernizing the way ALS drugs are tested before human trials.

ALS often progresses differently in different patients and can vary biologically at the cellular level as well. But this diversity is not reflected in the cell and animal models used by most researchers to develop drugs. In fact, most ALS research is based on rare familial forms which affect fewer than 10% of patients. Mosaic contends that this is a key reason why 99% of treatments trialed for sALS fail.

“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,” said Eugene Brandon, PhD, Mosaic’s CSO and a neuronal stem cell industry veteran. “It’s like trying to develop drugs for liver cancer using only HER2-positive breast cancer cells.”

A New Model for Precision Neurology

Project Mosaic builds on a growing body of academic work suggesting that, like cancer, ALS is not a single disease – but rather a “mosaic” of overlapping subtypes sharing common characteristics. More than 50 genes have been identified as important ALS triggers in a subset of cases, indicating diverse and distinct causes. Multiple labs have found strong evidence of sALS subtypes in data derived from hundreds of autopsied patients. And other researchers, including USC’s Justin Ichida, PhD, a project advisor, have found evidence that genetic differences across sALS patients may contribute to differences in how patients respond to drugs.

The pilot with iXCells is a landmark first attempt to industrialize disease models that reflect this diversity. To do this, the pilot will use patient-derived stem cell lines from Answer ALS – previously profiled by Johns Hopkins researchers Jeff Rothstein, PhD, and Alyssa Coyne, PhD – to generate transcriptomic signatures indicative of sALS. The goal is to show that these signatures are unique and consistent for each patient. Later work will investigate how these patterns can help match patients to drugs.

Working with iXCells serves another key Project Mosaic goal: shortcutting the time it usually takes for academic advances to achieve industry adoption and patient impact. Following the pilot, iXCells plans to make sALS models with standardized phenotypes available to researchers and drug makers. At the same time, Project Mosaic will advance to stage two: inviting additional academic and industrial labs to cross-validate and build on the pilot results.

“Project Mosaic is not just about cutting-edge research,” said Zipprich. “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.”

About Mosaic Neuroscience

Mosaic Neuroscience is a public benefit biotechnology company founded to develop precision medicine tools for sporadic ALS and other neurodegenerative diseases. Its flagship initiative, Project Mosaic, brings together leading academic and industrial labs to create and validate patient-specific ALS models.

About iXCells Biotechnologies

iXCells Biotechnologies (“iXCells”) is a leading San Diego, California-based cell technology company focused on generating a wide variety of predictive human disease models. In addition, iXCells is pursuing custom and patient-specific services leveraging its expertise in the preparation, handling, engineering and differentiation of induced pluripotent stem cells (iPSCs) and in the isolation of primary cells along with associated services for descriptive and functional cell characterization, compound screening and toxicology testing.

The company’s mission is to supply a diverse range of human disease models, derived from ethically sourced patient and healthy donor tissues, to support academic, pharmaceutical, and clinical researchers globally. By providing predictive human cell models, we aim to deepen the understanding of the molecular mechanisms underlying disease onset and progression as much as slow-down or reversion of disease, and accelerate the discovery and development of new medicines and innovative therapies to further advance precision medicine.

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“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.”