Settings
Save and close
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.
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.
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.