Date: 2018-01-03
Type of information: Development agreement
Compound: gene therapy using zinc finger protein transcription factors (ZFP-TFs)
Company: Pfizer (USA - NY) Sangamo Therapeutics (USA - CA)
Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases
Type agreement: development
Action mechanism:
- gene therapy. Sangamo’s zinc finger protein transcription factors (ZFP-TF)-mediated gene regulation approach is designed to either selectively repress or activate the expression of a specific gene or DNA sequence with a single administration. This technology enables targeting of a broad range of diseases requiring regulation of endogenous gene expression and differs from other approaches such as gene therapy or zinc finger nuclease-mediated genome editing, which are designed to replace or correct a missing or mutated gene or DNA sequence.
- Sangamo is developing ZFP-TFs as a novel therapeutic approach for diseases of the central nervous system (CNS). In keeping with the company’s strategy to externalize development of ZFP-TFs for CNS diseases, Sangamo has established collaborations with Pfizer for ALS and FTLD and with Shire for Huntington’s disease. Sangamo is also developing ZFP-TFs to down-regulate the expression of tau, a protein associated with Alzheimer’s disease and frontotemporal dementia (FTD).
Disease: gene therapy using zinc finger protein transcription factors (ZFP-TFs)
Details:
- • On January 3, 2018, Sangamo Therapeutics and Pfizer announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. These diseases are part of a spectrum of neurodegenerative disorders caused by mutations in the C9ORF72 gene that involves hundreds of additional repetitions of a six base pair sequence of DNA. This ultimately 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.
- Under this collaboration, Sangamo and Pfizer will investigate allele-specific ZFP-TFs with the potential to differentiate the mutant C9ORF72 allele from the wild type allele and to specifically down-regulate expression of the mutant form of the gene.
Financial terms:
- 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.
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