Sangamo, Biogen Team Up to Develop Gene Therapies for Neurological Diseases

Sangamo, Biogen Team Up to Develop Gene Therapies for Neurological Diseases
Sangamo and Biogen are partnering up to develop gene therapies for Alzheimer’s and Parkinson’s disease, as well as another neuromuscular disease target and up to nine undisclosed neurological disorders. Initially, the collaboration will focus on two molecules in Sangamo's pipeline: ST-501 for tauopathies such as Alzheimer’s, ST-502 for synucleinopathies including Parkinson’s disease, and a third yet-to-be-disclosed neuromuscular target. “As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance. We aim to develop and advance these programs forward to investigational new drug applications,” Alfred Sandrock Jr., MD, PhD, executive vice president of research and development at Biogen, said in a press release. The companies' approach will leverage Sangamo's proprietary zinc finger protein (ZFP) technology, designed to target nearly any sequence in the human genome (the complete set of DNA). ZFPs are a class of transcription factors, or proteins that regulate gene expression by recognizing certain parts of the DNA. Once bound to the target gene, transcription factors can switch it on or off as required by cells. Gene expression is the process by which information in a gene is synthesized to create a working product, such as a protein. Delivered via a modified, harmless form of a virus, called an adeno-associated virus, the therapy modulates — either repressing or activating — the expression of key genes involved in neurological diseases. This therapy is being designed as a one-time treatment and routes of administration include intracranial, intrathecal (via the spinal canal), a
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