HUDSON, NY - March 3, 2015
- Taconic Biosciences today announced its partnership with the Rare Genomics Institute (RGI) to support the 2015 Rare Disease Science Challenge: Be HEARD (Helping Empower and Accelerate Research Discoveries). The Challenge, part of a wide-ranging portfolio of activity timed to coincide with Rare Disease Day, provides personalized rare disease support by connecting individuals who have rare diseases with researchers who can customize projects using cutting-edge technologies to advance disease understanding.
Taconic's support of this initiative underscores our continuing commitment to raise awareness of rare diseases, which affect a small percentage of the population yet have overwhelming effects. Half of those affected by rare disease are children, more than half of whom won't survive to their 5th birthday. Almost none of the rare diseases has a drug treatment approved by the US Food and Drug Administration (FDA) and none has a cure. And many have no organization providing support or research. Taconic Biosciences is proud to support the advancement of rare disease research as a Corporate Sponsor and Ultimate Technology Sponsor
of the 2015 Be HEARD Challenge. This is the organization's second year participating in the challenge. In 2014 Taconic donated custom generation of a genetically engineered mouse model that is now being used to study Sanfilippo disease through a grant from the National Institutes of Health (NIH). This genetic metabolism disorder causes severe brain damage and typically results in hearing loss, vision loss, organ damage and bone deformities.
"We are pleased to sponsor the Be HEARD Challenge for a second consecutive year with the award of a novel mouse model bred to study cohorts," said Taconic CEO Bob Rosenthal. "Since most rare diseases have a genetic basis, the development of a genetically modified mouse model that recapitulates the disease can provide insights into its mechanistic basis. Further the model can be used for target identification and ultimately in preclinical work to test therapies for both efficacy and safety. Recent advancements at Taconic with CRISPR gene editing technology
allow for generating the model more quickly than traditional methods, getting this important tool into the hands of investigators faster than ever."
For the 2015 challenge, Taconic is donating an end-to-end project that includes custom model generation, model characterization, cryopreservation, and rapid expansion breeding of a cohort of mice ready for use in studies. The genetically engineered mouse model will be derived using the advanced CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing technique.
"The CRISPR/Cas9 methodology is already showing great promise as widely applicable for generating mouse models significantly faster than traditional genetic engineering methods," explained Jim Vitale, product manager, Taconic Biosciences. The company holds the first CRISPR license, obtained under certain patents from The Broad Institute, and was one of the first to market with the service in May 2014. "Taconic has invested considerable scientific development resources to improve and apply CRISPR in order to speed access to complex models. By donating a project using this technology, we continue our commitment to facilitating biomedical research and advancing drug discovery." Taconic is honored to join RGI and 18 other sponsoring organizations supporting rare disease research, which together have contributed over $600,000 in cutting-edge technology, services and resources. "We are delighted to partner with Taconic Biosciences," said Jimmy Lin, President of the Rare Genomics Institute. "Taconic's donation of a complete project using advanced gene editing technology will help us provide researchers with valuable tools for developing rare disease treatments."
The Rare Disease Challenge is open globally to researchers, foundations, or anyone who has a rare disease research idea that is constrained by limited resources. Research proposals can be submitted between February 28, 2015 and May 17, 2015. Proposals will be evaluated by an expert panel of scientists from both RGI and the sponsored companies, with the final winner(s) selected based on the scores achieved. Prizes include more than $600,000 of sponsored technology and/or services from participating organizations.
About Rare Genomics Institute
RGI is a nonprofit that makes cutting edge research and technologies, as well as scientific and medical experts, accessible to all families with a need, as they would do for their own loved ones if they were diagnosed with a rare disease. RGI does so through the provision of three innovative services: First, a researcher network: access to top genomics and biotechnology researchers who are willing to partner with patients in research. Second, a micro-funding website: an online fundraising platform and social network that helps patient communities raise funds. Third, a clinical network: connecting patients with clinicians and genetic counselors that will help interpret and translate the individualized research findings. RGI's latest development is expanding its research network from genomics to experts in model organisms, drug development and repurposing, and other high throughput technologies to help rare disease patients accelerate research and treatment options within their lifetimes. Learn more about the Rare Genomics Institute