SIOUX CENTER, Iowa—CHDI Foundation, Inc. and ExemplarGenetics recently announced a collaborative research agreement for the creationof multiple miniature swine models of Huntington's disease. Under theagreement, the companies will create two gene-targeted miniature swine modelsof Huntington's disease, one that represents the human disease phenotype andone that can be used for the development of genetic-based therapies for thedisease. Financial details were not disclosed.
"CHDI is pleased to support this important project for thedevelopment of new large animal models, wherein the genetic mutation is beinggenerated in a context that is very similar to that found in HD. We hope thatsuch models will advance our understanding of pre-manifest stages of HD and beuseful for preclinical therapeutic studies," David S. Howland, Ph.D., directorof In-Vivo Biology at CHDI, said in a press release.
CHDI brings its expertise in the disease to the table forthe collaboration, as the company focuses exclusively on therapeutics forHuntington's disease. Exemplar Genetics brings its specialization in creatinggene-targeted swine models of human disease, which offer better accuracy andmore biological similarities than mice or rats. The company currently has modelsof diseases such as cystic fibrosis, muscular dystrophy, cancer, cardiovasculardisease and neurological disease in its pipeline, among others.
"We are excited to be partnering with CHDI in thedevelopment of these new models of Huntington's disease," John Swart, Ph.D.,president of Exemplar Genetics, said in a press release. "Our experience andthat of our research partners is demonstrating that gene targeted miniatureswine models have some distinct advantages over the more commonly used rodentmodels. Our models mimic human disease giving researchers a much clearer windowinto disease mechanisms and should result in accelerated therapeuticdiscovery."
Huntington's disease is passed down genetically, and causes nerve cells in certain parts of the brain to degenerate.According to the Huntington's Disease Society of America, one out of every10,000 Americans have the disorder, with approximately 200,000 at risk ofdeveloping it. The disease most commonly develops in a patient's 30s or 40s,though a rare early-onset form of the disorder does occasionally strikechildren and adolescents. Huntington's leads to unusual movements, dementia,speech impediments and behavior changes, leading to disability that worsensover time. There is no cure and no methods to stop its progression, and peoplewith Huntington's usually die within 15 to 20 years, according to PubMedHealth.
The collaboration is CHDI's second in as many months withHuntington's as its focus. In late May, CHDI also announced a researchcollaboration with Lundbeck for the investigation of a targeted therapy for thedisease. CHDI will be conducting pre-clinical studies on an investigativecompound of Lundbeck's, with a focus on the compound's effect on P2X receptorsthat may be involved in the disease.
"We look forward to working with this distinguished group ofscientists who share our dedication to the HD community. CHDI is a beacon ofhope for the HD community and devotes itself entirely to finding therapies thathave the potential to improve the lives of HD families," Staffan Schüberg,president of Lundbeck, said in a press release of his company's collaborationwith CHDI. "Given our shared commitment, we are thrilled to partner with themand hope that our research will lead to advancements in HD therapeutic options.We are also pleased that this announcement coincides with HD Awareness Month tohelp draw attention to the need to find therapies for this degenerative neurologicaldisease."
