Rare Disease Translational Center

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Partner with us to provide patients with rare disease and families an efficient plan from diagnosis to therapy.

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The Rare Disease Translational Center team standing in a group.

Careers with the RDTC

Are you passionate about rare disease research?Apply now and join our growing team.

Available positions

Your Premier Rare Disease Research Partner

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.

Our Mission

To empower rare disease solutions through partnership, innovation, and scaled pre-clinical pipelines, to deliver targeted therapies from lab to clinic swiftly and effectively.

Our Approach


Dr. Cat Lutz looking through a microscope
The strategies implemented within the Rare Disease Translational Center laid the foundation for the approval of Spinraza, the first FDA-approved drug for spinal muscular atrophy.



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Ongoing projects

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Models created

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The Rare Disease Translational Center’s Lead Programs

Current phase
Model Generation
Model Characterization
Therapeutic Assessment
IND Enabling
Clinical Trials
Rett Syndrome
Gene Editing
Alternating Hemipelgia of Childhood (AHC)
Gene Editing, ASO, Gene Therapy
HNRNPH2-related neurodevelopmental disorder
Cornelia de Lange Syndrome
Gene Therapy
PGAP3-congenital disorder of glycosylation
Gene Therapy
Charcot-Marie-Tooth Type 4J
Gene Therapy *
Infantile neuroaxonal dystrophy (INAD)
Gene Therapy
Huntington’s Disease
Gene Editing
Spinal Muscular Atrophy
Gene Editing
Friedreich’s Ataxia
Gene Editing
POLG Mitochondrial Disorders
Gene Editing, Gene Therapy
Multiple Sulfatase Deficiency
Gene Therapy *

* Accepted into the Bespoke Gene Therapy Consortium


Your Platform

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.

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Your Pipeline

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



Your Partner

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.

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