Pfizer has partnered with US-based biotechnology firm Sangamo Therapeutics to use zinc finger protein transcription factors (ZFP-TFs) for the development of a gene therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) associated with C9ORF72 gene mutations.
Sangamo’s ZFP-TF technology works through the introduction of an engineered zinc finger protein (ZFP) designed to detect and bind to a precise DNA sequence, where ZFP’s transcriptional repressor domain suppresses gene expression.
The firms plan to assess allele-specific ZFP-TFs that have the potential to distinguish mutant C9ORF72 and wild type alleles, and for specific down-regulation of the expression of the gene’s mutant form.
Sangamo Therapeutics CEO Dr Sandy Macrae said: “The precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation.
“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.”
The firm initially formed an alliance with Pfizer in May last year to develop and commercialise potential gene therapy products for Haemophilia A.
Under the new collaboration, Pfizer will make an upfront payment of $12m to Sangamo, which will develop the ZFP-TF candidates.
Pfizer will be responsible for the operational and financial aspects of the subsequent research, development, manufacturing and commercialisation of the ZFP-TF programme and any related products.
Furthermore, the firm exercises an option to pay around $150m of potential development and commercial milestone payments, and tiered royalties on net sales.