The drugs will be developed using Sangamo’s zinc finger protein (ZFP) technology, which is delivered through adeno-associated virus (AAV)

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Biogen, Sangamo sign licensing agreement to develop gene therapies. (Credit: Arek Socha/Pixabay.)

US-based biotechnology firm Biogen has signed a global licensing agreement with genomic medicine company Sangamo Therapeutics to develop and commercialise therapies addressing several neurological diseases.

Under the terms of 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.

In addition, the company will hold rights to nominate up to nine additional undisclosed targets over a target selection period of five years.

The drugs will be developed using Sangamo’s zinc finger protein (ZFP) technology, which is delivered through adeno-associated virus (AAV) to target the expression of genes related to neurological diseases.

Biogen research and development executive vice president Alfred Sandrock 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.”

Biogen will pay up to $2.72bn as license, milestone payments and stock sale

Sangamo will conduct early research activities, and finance the development for a combination of advanced CNS delivery vectors and ZFP-TFs, targeting therapeutically relevant genes.

Biogen will later assume responsibility and share costs for the investigational new drug-enabling studies, clinical development, related regulatory approvals, and global marketing.

Upon closing of the transaction, Biogen will pay $350m, comprising $125m license fee payment and $225m from the sale of new Sangamo stock, or approximately 24 million shares at $9.21 per share.

Sangamo would receive up to $2.37bn in other development, regulatory, and commercial milestone payments, including $925m in pre-approval milestone payments and $1.4bn in the first commercial sale and other sales-based milestone payments, in addition to the royalties.

The closing of the transaction is subject to the completion of the review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the US.

Sangamo CEO Sandy Macrae said: “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.

“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.”