Partner with us to provide patients with rare disease and families an efficient plan from diagnosis to therapy.
Learn more about our collaborations
Are you passionate about rare disease research?Apply now and join our growing team.
Our team serves those with rare disease by accelerating the pre-clinical phase. Our vision is to provide patients with an efficient path from diagnosis to therapy, allowing them to live longer, healthier lives.
To empower rare disease solutions through partnership, innovation, and scaled pre-clinical pipelines, to deliver targeted therapies from lab to clinic swiftly and effectively.
* Accepted into the Bespoke Gene Therapy Consortium
A mouse model is a foundational research platform to accelerate your preclinical research pipeline. Our team of scientists work closely with you to determine if a rare disease model exists and if it fits your therapeutic strategy. If no suitable model is available, our genetic engineers employ state-of-the-art technologies and strategies to effectively model patient variants. We aim to future-proof your model by designing it to be a tool to test a broad range of translational therapeutic approaches.
Our team of scientists work with you to craft preclinical pipelines with a focus on outcomes that translate directly to clinical practice. Our commitment to rigorous, robust, and reproducible research, positions your studies to be Investigational New Drug (IND) supporting. We are redefining the testing process to make high-quality preclinical work accessible and efficient by employing a parallel approach which, accelerate lead therapies to the clinic faster. This approach was the foundation for the successful clinical trials of Spinraza, the first FDA-approved drug for spinal muscular atrophy (SMA).
Design your preclinical platform
We collaborate with a diverse range of global entities, from families who have just received a diagnosis to biotech and pharmaceutical groups. As your partner, our research team not only executes your studies but also becomes an integral part of your scientific research team. We tenaciously tackle even the most complex research problems, drawing on decades of expertise in rare disease.
Our Stories
Unravel Biosciences and The Jackson Laboratory announce collaboration to accelerate the discovery of promising…
Read MoreJoin JAX RDTC Program Manager Paige Martin and Study Director Matt Simon as they take us through…
Read MoreThe funding will support construction of a dedicated facility for rare disease research…
Read MoreIn collaboration with Ohio’s Nationwide Children’s Hospital, JAX is testing a gene therapy for PGAP3-Congenital…
Read MoreMembers of the RDTC and Bar Harbor Preclinical Services participated in a walk to raise awareness for ALS.
Read MoreThe Rare Disease Translational center is driving the rare disease research pipeline.
Read MoreRepresentatives from JAX attended the 2023 National Organization for Rare Disorders Rare Diseases and Orphan Products Breakthrough…
Read MoreFor the first time, there's an FDA approved treatment for Friedreich’s ataxia (FA). This is a major step forward for the FA…
Read MoreParents of a child with INAD, a rare nervous-system disorder, partner with the JAX Rare Disease Translational Center to test therapies.
Read MoreSupported by an NIH grant, Cat Lutz will lead a multi-institutional team to develop and validate new gene editing-based therapeutic…
Read MoreA new consortium will positively impact rare disease patients and families and is a powerful and gratifying next step for those gene therapies …
Read MoreA team including The Jackson Laboratory has received a $2 million grant to research the cause of…
Read MoreThe Rare Disease Translational Center connects JAX to young patients and their families.
Read MoreTerry Pirovolakis and his family are going to the ends of the earth to create a treatment for his son Michael’s rare disease…
Read MoreCat Lutz, Ph.D., M.B.A. was recently named vice president of the Rare Disease Translational Center at The Jackson…
Read MoreRare disease families and experts offer advice on how to navigate the complicated and daunting journey from diagnosis to potential treatment.
Read MoreChris Brannigan is preparing to take off his boots and walk nearly 1200 miles down the eastern seaboard of the…
Read MoreAmyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. As of now, there is no…
Read MoreSenior Director of JAX Rare and Orphan Disease Center, Cat Lutz, Ph.D., M.B.A. is honored for her tireless efforts in rare disease…
Read MoreA JAX research team led by Cat Lutz launched a project to develop gene therapy strategies and to test small molecules for the treatment of multiple sulfatase…
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