Sangamo BioSciences and CHDI Foundation announce collaboration to develop ZFP Therapeutic® for Huntington’s disease
Richmond, California and New York – Sangamo BioSciences, Inc. (Nasdaq: SGMO) and CHDI Foundation, Inc. have announced a collaboration to develop a novel therapeutic for Huntington’s disease (HD) based on Sangamo’s proprietary zinc finger DNA-binding protein (ZFP) technology. The ZFP therapeutic approach will target the huntingtin gene that causes HD, an inherited neurodegenerative disease for which there are currently no therapies available to slow disease progression.
“We’re very excited to bring Sangamo’s ZFP technology platform to Huntington’s disease,” said Ignacio Munoz-Sanjuan, Ph.D., Vice President, Biology at CHDI. “Our collaboration aims to develop ZFP transcription factors that will alter huntingtin expression, which has the potential to directly modify disease progression. Moreover, the versatility of ZFP technology makes it a powerful platform that can be deployed to impact various aspects of HD pathology and we look forward to working with Sangamo to develop multiple innovative therapeutic approaches for HD.”
“We are very pleased to be collaborating with CHDI, who bring tremendous experience and resources in HD to the development of a novel ZFP Therapeutic for this devastating disease,” said Philip Gregory, D. Phil, Sangamo’s vice president, research and chief scientific officer. “The causative genetic defect in HD – mutations in the huntingtin gene – provides a validated target for intervention using a ZFP-based approach. Our collaboration will initially focus on developing ZFP Therapeutics to lower the levels of the mutant huntingtin protein in mouse and human cells. This collaboration highlights the unique ability of our ZFP technology to drive potentially therapeutic outcomes at the genetic level.”
“The addition of a program in HD expands our portfolio of programs in monogenic and rare diseases,” said Edward Lanphier, Sangamo’s president and CEO. “Sangamo is committed to the development of novel ZFP Therapeutics in areas of unmet medical need, such as HD, for which there are no disease-modifying therapies.”
Sangamo has ongoing research stage and preclinical programs in rare disease indications including blood coagulation disorders such as hemophilia, hemoglobinopathies including beta-thalassemia and sickle cell disease and immune function disorders such as X-linked severe combined immunodeficiency disorder.
About Huntington’s Disease
Huntington’s disease is an autosomal dominant neurodegenerative genetic disorder that causes motor, cognitive, and behavioral dysfunction. It is estimated that 120,000 people in the U.S. have the genetic mutation that causes Huntington’s disease and will experience symptoms during a normal lifetime. The average lifespan for patients after onset of motor dysfunction is approximately 10 to 20 years. There are currently no disease-modifying therapies available to slow the progression of Huntington’s disease.
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately-funded, not-for-profit, scientific research organization exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease. As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. Our scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing project management to ensure that our common goals remain in focus. More information about CHDI can be found at www.chdifoundation.org.
About Sangamo BioSciences, Inc.
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy. Sangamo also has a Phase 1/2 clinical trial and two ongoing Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS as well as a Phase 1 trial of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on Parkinson’s disease, monogenic diseases, neuropathic pain and nerve regeneration. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins called zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company’s website at www.sangamo.com.
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.
This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs and their applications in the treatment of Huntington’s disease, strategic partnerships with collaborators and clinical trials of ZFP Therapeutics. Actual results may differ materially from these forward-looking statements due to a number of factors, including technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. See the company’s SEC filings, and in particular, the risk factors described in the company’s Annual Report on Form 10-K and its most recent quarterly report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
|Sangamo BioSciences, Inc.||CHDI Foundation, Inc.|
|Elizabeth Wolffe, Ph.D.||Simon Noble, Ph.D.|