JAX joins $46M initiative to accelerate genetic medicines for rare neurodevelopmental disorders
By JAX Communications
Article | November 10, 2026
The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) is a collaborator on a new $46 million international research initiative led by the University of Oxford and Boston Children’s Hospital to develop a scalable approach to designing and testing genetic medicines for children with neurodevelopmental disorders, including certain genetic conditions associated with profound autism.
The Genetic Medicines Hub is one of six research hubs within the Aligning Research to Impact Autism (ARIA) ecosystem.
While advances in genetic sequencing have rapidly increased understanding of the biological basis of many neurodevelopmental conditions, translating these discoveries into scalable therapeutic strategies remains a significant challenge.
Rather than pursuing one condition at a time, the Genetic Medicines Hub will establish systematic methods to design, test, and optimize these molecules across multiple genetic targets. This approach is intended to improve efficiency, reduce duplication, and enable progress across a wider range of rare conditions.
JAX’s Rare Disease Translational Center (RDTC) is bringing expertise in developing and characterizing research models, generating genetic and biological data, and building shared resources that can help researchers better understand rare diseases and evaluate potential therapies.
“For rare disease patients, the biggest barrier has never been scientific ambition, it's been data. JAX has built its rare disease mission around open, deeply annotated genomic resources, and this collaboration is a direct extension of that commitment, ensuring rare conditions have the depth of evidence needed to drive genetic medicine development forward,” said Cat Lutz, vice president of the Rare Disease Translational Center at JAX.
At the core of the program is the development of a platform-based approach to genetic medicines, focused initially on antisense oligonucleotides (ASOs), which are short, synthetic molecules that can be designed to interact with RNA and modify gene expression. ASOs have been used to successfully treat disorders of the central nervous system, offering an opportunity for rapid translation.
“Genetic neurodevelopmental disorders have long faced a bottleneck between gene discovery and therapeutic development. By uniting world-class teams and building a shared, transparent blueprint for ASO innovation, we aim to make that bottleneck disappear,” said Tim Yu, associate professor of pediatrics at Boston Children’s Hospital. “This Genetic Medicines Hub will allow us to move from scientific insight to actionable therapies with unprecedented speed — and to do so in a way that benefits the entire field.”
The program will initially focus on a set of genes associated with NDDs, including ARID1B, CHD2, GRIN2B, RNU4-2, SCN2A, and SLC6A1, working hand-in-hand with another research hub, the ARIA IMPACT Network and gene-specific Patient Advocacy Groups.
The Genetic Medicines Hub will combine cutting-edge experimental and computational capabilities, including:
- High-throughput laboratory systems to scale molecular testing
- Novel screening approaches to assess biological impact at the protein level
- AI-informed design tools to optimize candidate molecules
- Integrated data platforms to enable sharing of results, protocols, and insights
Together, these elements are designed to create a reproducible, scalable pipeline for genetic medicine development and to generate a shared resource for the wider research community.
The Hub brings together complementary strengths across Boston Children’s Hospital, the University of Oxford, JAX, and N=1 Collaborative, combining expertise in human genetics, functional genomics, clinical research, and therapeutic development.
“This program represents a step change in how we approach developing genetic medicines. By building a platform that can be applied across multiple genes, we can move beyond fragmented efforts and begin to scale our impact,” said Stephan Sanders, professor of pediatric neurogenetics at the department of pediatrics at the University of Oxford. “Importantly, this work is grounded in collaboration and open science, ensuring that the knowledge generated benefits the broader community.”
“Reaching more patients requires more than advancing individual programs—it requires a field that learns together,” said Nicole Nolen, executive director of N=1 Collaborative. “This collaboration creates an opportunity to turn what is learned into shared approaches that can be adopted and built upon across the community.”
Together, the teams aim to develop safe and effective ASO therapies for several NDDs while establishing a sustainable framework for advancing genetic medicines across a much wider range of conditions.
Read the original announcement for additional details about the Genetic Medicines Hub and its participating institutions.
Media Contact: Patrick Skahill, [email protected], 860-839-3309.
Rare Disease Translational Center
The mission of the RDTC is to empower rare disease solutions through partnership, innovation, and scaled pre-clinical pipelines, to deliver targeted therapies from lab to clinic swiftly and effectively.
View more
Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory launch the Center for Therapeutic Genetics
A new collaboration to develop precision medicines for those with rare diseases, using infrastructure and repeatable practices that will be shared with others.
View more