Rare Daily Staff

Sangamo Therapeutics and Pfizer said they will collaborate to develop a potential gene therapy using zinc finger protein transcription factors to treat a certain form of the neurodegenerative disease amyotrophic lateral sclerosis, or ALS.

The two companies will target ALS that is linked to mutations of the C9ORF72 gene, as well as frontotemporal lobar degeneration linked to mutations of the same gene.

ALS and FTLD are part of a spectrum of neurodegenerative disorders caused by mutations in the C9ORF72 gene that involve hundreds of additional repetitions of a six base pair sequence of DNA. This leads to the deterioration of motor neurons, in the case of ALS, or neurons in the frontal and temporal lobes, in the case of FTLD. Currently, there are no cures to halt or reverse the progression of ALS or FTLD. The C9ORF72 mutation is linked to approximately one-third of cases of familial ALS.

“The precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation,” said Sandy Macrae, CEO of Sangamo. “Collaboration with the right partner for a given therapeutic application is a key component of our corporate strategy and enables us to pursue the vast opportunity set of our platform.”

Under the terms of the collaboration agreement, Sangamo will receive a $12 million upfront payment from Pfizer. Sangamo will be responsible for the development of ZFP-TF candidates. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization for the C9ORF72 ZFP-TF program and any resulting products. Sangamo is eligible to receive potential development and commercial milestone payments of up to $150 million, as well as tiered royalties on net sales.

Sangamo’s ZFP-TF technology involves introducing an engineered zinc finger protein, which is designed to identify and bind to a precise sequence of DNA. Once bound to the target sequence of DNA, a transcriptional repressor domain attached to the ZFP suppresses expression of the gene.

In May 2017, Sangamo and Pfizer entered into an exclusive, global collaboration and license agreement for the development and commercialization of potential gene therapy products for Hemophilia A, including SB-525, which entered the clinic in August 2017.

January 3, 2018

Photo: Sangamo CEO Sandy Macrae