Biogen, Sangamo to Develop Novel Gene Therapies

Biogen and Sangamo Therapeutics have begun a collaboration to develop and commercialize gene therapies for Parkinson’s and Alzheimer’s diseases, as well as an undisclosed neuromuscular disease.

The five-year collaboration will join Sangamo’s science expertise and proprietary technology with Biogen’s clinical development capabilities. The goal is to submit investigational new drug applications to the U.S. Food and Drug Administration (FDA) and move forward to clinical trials.

“We have extensive experience in the discovery and preclinical development of genomic medicines for neurological disease. Biogen is dedicated to neuroscience and has the resources and expertise to translate these investigational therapies to the clinic,” Bryan Zeitler, PhD, director of gene regulation at Sangamo Therapeutics, said in a press release.

Sangamo has a proprietary zinc finger protein (ZFP) technology, designed to target almost any sequence in the human genome, which is the complete set of DNA present within each of our cells. Delivered via a modified harmless form of a virus, called an adeno-associated virus (AAV), the technology 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.

“Our genomes encode hundreds of zinc finger proteins that are expressed naturally in the brain. Rather than creating a double-stranded break like traditional gene editing, an engineered ZFP-TF has been shown to precisely tune down the expression of the genes that encode the aberrantly folded, neurotoxic proteins implicated in many neurodegenerative diseases,” Zeitler said.

ZFPs are part of Sangamo’s genome regulation technology. In the case of Parkinson’s, One of these therapeutic candidates is ST-502, which delivers ZFPs that target and repress the activity of the alpha-synuclein (SNCA) gene, which provides instructions for making the alpha-synuclein protein. Abnormal aggregates, or clumps, of this protein are thought to underlie the development of Parkinson’s disease.

Another candidate, 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.

“We know we can build these cutting-edge gene-regulation tools. But in order to achieve our goal of delivering them to patients with devastating neurological disorders, we wanted to collaborate with a partner who has the experience of taking that next step — the biological expertise, and clinical and commercial infrastructure,” said Jeff Miller, PhD, senior director of innovation design and technology at Sangamo.

“Now we have the potential to develop innovative treatments to patients and bring hope to so many suffering from challenging neurological diseases,” Miller added.

The agreement allows Biogen and Sangamo to nominate up to nine more therapeutic targets over a five-year period.