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

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Sangamo and Biogen are teaming up to develop gene therapies for Parkinson’s and Alzheimer’s disease, as well as another neuromuscular disease target and up to nine undisclosed neurological disorders.

The collaboration will leverage Sangamo’s proprietary zinc finger protein (ZFP) technology, designed to target almost any sequence in the human genome, which is the complete set of DNA. Delivered via a modified, harmless form of a virus, called an adeno-associated virus (AAV), the therapy modulates — either repressing or activating — the expression of key genes involved in neurological diseases.

Gene expression is the process by which information in a gene is synthesized to create a working product, like a protein.

“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, research and development at Biogen, said in a press release.

“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, 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,” Macrae said.

ZFPs are part of Sangamo’s genome regulation technology. Delivered using AAVs, ZFPs can selectively activate or repress specific genes. In the case of Parkinson’s, ST-502 delivers ZFPs that target and repress the activity of the alpha-synuclein (SNCA) gene, which codes for the alpha-synuclein protein. Abnormal aggregates, or clumps of this protein are thought to underlie the development of Parkinson’s disease.

ST-501 works under the same principle but targets the protein tau, thought to be a key player in the development of Alzheimer’s disease.

In preclinical studies, strategies using these gene therapies have shown promising therapeutic effects.

“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,” Boissel added.

Under the terms of the collaboration, Biogen has the exclusive global rights for ST-502, ST-501, and a third undisclosed neuromuscular disease target.

Moreover, Biogen has the exclusive rights to select up to nine additional undisclosed targets over a period of five years.

Sangamo will be responsible for early research for these gene therapies, with the costs shared by the companies. Biogen will take over research aimed at clinical development, regulatory interactions, and global commercialization, assuming responsibility for such costs.

Within the licensing agreement, Biogen will pay Sangamo $350 million upfront. Sangamo is eligible to receive up to $2.37 billion in potential milestones, as well as royalties once the therapies are marketed.