Biogen-Sangamo Collaboration Comforts Millions Affected by Neurodegenerative Disorders
By Ruchi Jhonsa, Ph.D.
Neurodegenerative disorders affect millions of people worldwide, yet there is no definitive cure for them. In 2016, an estimated 5.4 million Americans were living with Alzheimer’s disease and these numbers are believed to rise even more by 2020. The risk of being affected by the disease increases with age. So even though new medical innovations increase the average human lifespan they cannot reduce the chance of getting affected by neurodegenerative disorders. This creates a critical need to understand the causes of neurodegenerative diseases and develop new approaches for treatment and prevention.
To strengthen the fight against neurodegenerative diseases, Biogen and Sangamo Therapeutics shook hands to develop and commercialize gene therapy for Alzheimer’s, Parkinson’s and an undisclosed neuromuscular disorder, and up to nine additional undisclosed neurological disease targets. Biogen will use Sangamo’s proprietary zinc finger protein (ZFP) technology to modify the expression of key genes involved in neurological diseases. For the same, Biogen will pay $350 million along with $2.37 billion in milestone payments. Sangamo is also eligible to receive royalties on potential net commercial sales of products arising from the collaboration. This news would be welcomed by patients who are still waiting for Biogen’s first new Alzheimer’s disease drug, Aducanumab.
Alfred Sandrock Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen said, “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.”
Zinc Finger Technology
Very similar to the CRISPR technology, zinc finger nucleases (ZFNs) first recognize and bind to specific sequences of target DNA by virtue of the DNA binding domain. Following recognition, they make a double-stranded break in the DNA at a precise location by using the DNA cleavage domain, which can be used to either activate or repress the expression of specific genes to achieve a desired therapeutic effect. Sangamo engineers these ZFNs for targeting any nucleotide, at any desired location without any off-target effects such that any gene of interest can be targeted with high precision. Biogen’s interest in the technology grew when ZFN’s tailor-made for tau (ST-501) and alpha synuclein (ST-502) showed potential in preclinical studies.
“The combination of Sangamo’s proprietary zinc finger technology, Biogen’s unmatched neuroscience research, drug development, and commercialization experience and capabilities, and our shared commitment to bring innovative medicines to patients with neurological diseases establishes the foundation for a robust and compelling collaboration,” said Stephane Boissel, Head of Corporate Strategy at Sangamo. “This collaboration exemplifies Sangamo’s commitment to our ongoing strategy to partner programs that address substantial and diverse patient populations in disease areas requiring complex clinical trial designs and commercial pathways, therefore bringing treatments to patients faster and more efficiently, while deriving maximum value from our platform.”
According to the agreement, Biogen will hold exclusive global rights to ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, and a third undisclosed neuromuscular disease target. Additionally, Biogen will also have exclusive rights to choose up to nine targets over a target selection period of five years. Sangamo will be responsible for the early research activities, which will be required for the development of CNS delivery vectors and zinc finger proteins targeting therapeutically relevant genes. However, the two companies will share the cost of the research. Sangamo’s manufacturing facility will also be used for GMP manufacturing activities for the initial clinical trials for the first three products. Biogen’s major responsibilities will come later when the drug will be put through new drug enabling studies and clinical studies as well as when expertise in regulatory interactions and global commercialization will be required. All the manufacturing activities beyond the first clinical trial of the first three products of collaboration will be taken care of by Biogen.
“There are currently no approved disease modifying treatments for patients with many devastating neurodegenerative diseases such as Alzheimer’s and Parkinson’s, creating an urgency for the development of medicines that will not just address symptoms like the current standards of care, but slow or stop the progression of disease,” said Sandy Macrae, the CEO of Sangamo. “We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genomic level.
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