Biogen secures 'gene switch' technology from Sangamo

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Biogen has entered a new agreement with US-based company Sangamo Therapeutics to develop and commercialise two gene therapies for neurodegenerative and neurological disorders, including Alzheimer and Parkinson disease.

Sangamo was established in 1995 and has become a global leader in the development of gene therapy. It has a range of existing collaborations with major global biopharmaceutical companies, including Pfizer, Takeda and Sanofi.

Sangamo’s genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), is currently delivered via an adeno-associated virus to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. 

“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,” said Alfred Sandrock, Executive Vice President of R&D at 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, 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.”

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

Biogen also has exclusive rights to nominate up to nine additional undisclosed targets over a target selection period of five years.

The companies said Sangamo will perform early research activities, costs for which will be shared, aimed at the development of the combination of proprietary CNS delivery vectors and ZFP-TFs targeting therapeutically relevant genes. Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.

Sangamo will be responsible for GMP manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity. Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.

Sangamo will receive US$350 million comprised of US$125 million in a license fee payment and US$225 million from the sale of new Sangamo stock, or approximately 24 million shares at US$9.21 per share. In addition, Sangamo may receive up to US$2.37 billion in other development, regulatory, and commercial milestone payments, including up to US$925 million in pre-approval milestone payments and up to US$1.445 billion in first commercial sale and other sales-based milestone payments. Sangamo will also be eligible to receive from Biogen tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration.