Rare diseases affect roughly 30 million Americans, yet significant challenges remain in studying and curing these conditions. Since there is often a genetic component to these diseases, the ability to model that genetic mutation in vivo in laboratory animals such as mice or rats is vital to learning more about the specific disease mechanisms and identifying potential therapies. As a leader in generating custom genetically engineered models, Taconic Biosciences is committed to applying our unique capabilities to advance rare disease research. We recently partnered with Cure Rare Disease, a 501(c)(3) nonprofit biotech aiming to advance research and development of therapeutics for ultra-rare genetic disorders such as ADSSL1 gene-related myopathy. This condition is categorized by progressive weakness throughout the body, starting with the lower extremities and continuing through adolescent development into upper body and facial muscle weakness. By knocking-in a mutated ADSSL1 gene in a mouse, researchers will be able to study potential therapeutic interventions for this disease in a custom in vivo model and bring us closer to a cure.
"The work we do at Cure Rare Disease to develop potential treatments for ultra-rare diseases would not be possible without collaborations with dedicated partners, like Taconic Biosciences," explained Rich Horgan, Founder and CEO of Cure Rare Disease. "The knock-in mouse model the Taconic team created will be critical to testing the safety and efficacy of a potential therapeutic to treat ADSSL1, an ultra-rare disease that currently has no treatment or cure."
Learn more about Cure Rare Disease.
Learn more about Cure Rare Disease.