Target ALS and Regeneron Partner to Expand Access to a Novel C9orf72 ALS/FTD Mouse Model
January 27, 2026
Target ALS is pleased to announce a new partnership with Regeneron to enable access to a novel mouse model of ALS and frontotemporal dementia (FTD) driven by C9orf72 repeat expansions, the most common genetic cause of both diseases.
This collaboration reflects a shared commitment to accelerating therapeutic discovery by providing high-quality, disease-relevant research tools and making them broadly available to the ALS community.
A critical genetic driver of ALS and FTD
A GGGGCC hexanucleotide repeat expansion in the C9orf72 gene accounts for a significant proportion of familial ALS and FTD cases, and a meaningful subset of sporadic disease. Despite its prevalence, modeling C9orf72-driven pathology in vivo has been technically challenging, limiting the field’s ability to rigorously evaluate therapeutic strategies.Leveraging its deep expertise in neurodegenerative disease research, Regeneron has developed a unique knock-in mouse model expressing 545 C9 G4C2 repeats. This model has been extensively characterized and recapitulates key, age-dependent features of disease pathology, including TDP-43 aggregation, a hallmark of ALS and FTD. Details on the generation of the model were published in Kojak et al., 2024, Nucleic Acids Research.
Accelerating discovery through shared access
In an effort to speed progress toward effective therapies, Regeneron is making this model available to Target ALS–funded investigators through a contract research organization (CRO). Researchers will be able to use the model to evaluate potential therapeutic approaches relevant to people living with C9orf72 repeat expansion–associated disease.Importantly, access to the model is being provided in a way that preserves scientific independence. Investigators retain control of their data and intellectual property, and are not required to share confidential information. This approach ensures that the model can be used widely, rigorously, and responsibly across academia and industry.
A trusted conduit for the research community
This partnership underscores Target ALS’s role as a trusted, neutral convener within the ALS ecosystem, capable of working effectively across industry, academia, government and the patient community to enable progress at scale.
“This partnership reflects the trust that the community places in Target ALS,” said Manish Raisinghani, MBBS, PhD, Chief Executive Officer of Target ALS. “It’s the result of a decade-long relationship built on shared values and a commitment to doing what’s right for people living with ALS. Regeneron chose to work with us as a conduit to the broader research community because they know this model will be used thoughtfully, rigorously, and for maximum impact. This arrangement enables the community to focus on what matters most: advancing therapies.”
Commitment to open science
Target ALS is deeply grateful to Regeneron for its leadership and commitment to open science, and in particular to Dr. Aarti Sharma, Director of Motor Neuron Diseases, whose vision and collaboration made this partnership possible.
Additional information on how Target ALS–funded investigators can access the C9orf72 repeat expansion mouse model will be shared in the coming months.
By expanding access to rigorously characterized, disease-relevant models, partnerships like this one help ensure that promising ideas can be tested faster, more rigorously, and with greater confidence, bringing the field one step closer to effective treatments for ALS and FTD.
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