Pact to develop 3D models could help speed new rare disease treatments
2 US companies partnering to advance human-based models
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Two U.S.-based companies are teaming up to advance personalized human-based disease models that could help speed the discovery of novel therapies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency.
Ixcells Biotechnologies and Rosebud Biosciences, both headquartered in California, have entered into a partnership to develop “scalable, patient-relevant human models” that use “complex 3D biology,” according to a press release from Ixcells.
The collaboration combines Ixcells’ induced pluripotent stem cells (iPSCs) — a type of stem cell that can be used to generate nearly any cell type in the body — with Rosebud’s expertise in growing three-dimensional (3D) human organoids. These organoids are miniature lab-grown tissues that mimic the structure and function of human organs.
“Our partnership with iXCells allows us to make advanced organoid systems more accessible to researchers who need models that better represent human biology,” said Kitch Wilson, Rosebud’s CEO and cofounder. “By combining our complementary strengths, we can support deeper exploration of disease biology and help drive the next generation of therapeutic discovery.”
Together, the companies aim to create more accurate systems for evaluating the safety, toxicity, and tissue-specific effects of potential therapies — longstanding challenges in rare disease research.
“This collaboration enhances our end-to-end platform and expands our ability to deliver next-generation models that reflect human biology with greater depth and accuracy,” said Steve Smith, CEO of Ixcells. “In areas such as rare diseases, where predicting human-specific safety and response earlier can significantly impact development timelines and patient outcomes, these integrated systems provide critical insight.”
Rare disease research hampered by lack of reliable disease models
Research into rare neurological disorders such as AADC deficiency, a genetic condition that affects the nervous system, has long been hampered by the lack of reliable human disease models. A dearth of such models has made it difficult to predict how experimental therapies will behave once tested in patients.
Therapeutic development has often relied on animal models or simplified cell systems that do not fully reflect human brain biology.
By integrating Rosebud’s organoid generation and characterization expertise into Ixcells’ existing iPSC platform, iPSCore, this partnership will give researchers access to complex, human-relevant systems that better replicate tissue-level biology and therapeutic responses, the companies say.
The combined workflow brings together Ixcells’ scalable, modular iPSC platform with Rosebud’s AI-driven organoid technology, which is designed to generate reproducible human tissue data for drug discovery and safety assessment. According to the companies, this approach could provide more biologically representative models for studying disease mechanisms and testing new treatments.
The expansion reflects “growing demand” for human-based systems that can better support translational decision-making, according to the companies. This particularly the case in rare diseases, where patient populations are small and clinical trial opportunities are limited.
“Our focus has always been on anticipating what our partners will need next, and Rosebud brings impressive depth in organoid science that aligns perfectly with that vision,” Smith said. “This partnership reflects the strategic direction we are taking as iXCells scales its platform to support more complex, translationally focused programs for our partners.”
