RARE Daily

Novartis and Biogen Terminate Collaborations with Sangamo for Neurodevelopmental Disorders

March 20, 2023

Rare Daily Staff

Sangamo Therapeutics said that Novartis and Biogen both have terminated collaboration and licensing agreements with the biotech for gene regulation therapies for neurodevelopmental disorders that were potentially worth billions.

Both Novartis and Biogen attributed the terminations to recent strategic pipeline reviews and prioritizations.

The agreement with Novartis, which will terminate on June 11, signed at the end of July 2020 and due to expire at the end of July 2023, covered programs to research gene regulation therapies to treat three neurodevelopment disorders.

Under their original agreement, Sangamo received $75 million upfront and was eligible to earn up to $720 million in milestones. Sangamo granted Novartis an exclusive, royalty bearing and worldwide license to develop, manufacture and commercialize certain of its zinc finger protein transcription factors (ZFP-TFs) targeted to three undisclosed genes that are associated with neurodevelopmental disorders, including autism spectrum disorder and intellectual disability. Over the three-year collaboration period, which was scheduled to expire on July 27, 2023 in conjunction with Sangamo’s planned delivery to Novartis of ZFP-TFs for each of the three programs, Sangamo performed early research activities for each gene target and manufactured the ZPF-TFs required for such research, the costs of which have been fully reimbursed by Novartis.

Sangamo said it will investigate alternative options to advance the neurodevelopmental disorder programs from the collaboration dependent on the outcome of a broader strategic review of its preclinical CNS pipeline.

The agreement with Biogen, which will terminate on June 15, was originally signed in February 2020 and focused on programs to research and develop gene regulation therapies to treat neurological diseases.

Under their original agreement, Sangamo granted Biogen an exclusive global license to develop, manufacture and commercialize certain zinc finger protein (“ZFP”) and/or AAV-based products directed to up to 12 neurological disease gene targets selected by Biogen. Biogen selected four of these: Sangamo’s ST-501 product candidate to treat tauopathies, Sangamo’s ST-502 product

candidate to treat synucleinopathies including Parkinson’s disease, a third product candidate targeting Type 1 Myotonic Dystrophy (DM1), a neuromuscular disease, and a fourth undisclosed neurological disease gene target.

For each gene target selected by Biogen, Sangamo performed early research activities, costs for which were shared by the companies, aimed at the development of the combination of proprietary CNS delivery vectors and ZFP-TFs (or potential other ZFP products) targeting therapeutically relevant genes. For three of the four product candidates, Sangamo had achieved predetermined proof of mechanism objectives and had advanced research activities to late-stage preclinical testing with lead candidates. Sangamo’s research activities for all targets were scheduled to conclude no later than April 2027.

As part of their deal, Biogen paid Sangamo a $125 million upfront license fee and purchased $225 million of Sangamo common stock. Sangamo was eligible to earn up to $2.37 billion in milestone payments, if Biogen selected all 12 collaboration targets.

Sangamo says that it believes that its available cash, cash equivalents, and marketable securities will be adequate to fund its operations at least through the next 12 months and does not expect the termination of the Novartis and Biogen agreements to impact this estimate.


Stay Connected

Sign up for updates straight to your inbox.