Pfizer and Sangamo Partner on Gene Therapy for Lou Gehrig's Disease

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The companies aim to develop a potential zinc finger protein transcription factor-based gene therapy for treating Lou Gehrig’s disease.

On Jan. 3, 2018, Pfizer announced a collaboration with Sangamo Therapeutics, a genomics company, to develop a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

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

In Sangamo’s ZFP-TF technology, an engineered ZFP, which is designed to identify and bind to a precise sequence of DNA, is introduced. Once bound to the target DNA sequence, a transcriptional repressor domain attached to the ZFP suppresses the expression of that gene. Under their collaboration, Sangamo and Pfizer intend to investigate allele-specific ZFP-TFs. Their aim is to differentiate the mutant C9ORF72 allele from the wild type allele and to specifically down-regulate expression of the mutant form of the gene.

This technology enables the targeting of a broad range of diseases that require regulation of endogenous gene expression, yet it differs from other gene therapy approaches or zinc finger nuclease-mediated genome editing. The latter are designed to replace or correct a missing or mutated gene or DNA sequence, whereas the ZFP-TF technology is designed to suppress expression of specifically targeted genes. Sangamo is developing ZFP-TFs as a new therapeutic approach for diseases of the central nervous system.


The new collaboration with Pfizer follows an earlier collaboration and license agreement that the companies entered in May 2017. Under the earlier deal, Sangamo and Pfizer aim to develop and commercialize potential gene therapy products for hemophilia A. In addition to its collaborations with Pfizer, Sangamo has also established a collaboration with Shire for Huntington’s disease.

Source: Pfizer