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Pfizer, Sangamo Tie Up for Development of ALS Gene Therapy

Pfizer has now teamed up with the California-based Sangamo Therapeutics to develop a therapy using the latter’s zinc finger protein transcription factor (ZFP-TF) gene regulation technology to treat amyotrophic lateral sclerosis and frontotemporal lobar degeneration linked to mutations of chromosome 9 open reading frame 72 (C9orf72).

ALS and FTLD 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, the company said in its announcement.

Sangamo receives a $12 million upfront payment from Pfizer, and is also eligible to receive

potential development and commercial milestone payments of up to $150 million, as well as tiered royalties on net sales.

We are excited to continue our collaborative relationship with Pfizer with this new program using Sangamo’s zinc finger protein technology to develop a potential gene therapy for patients with certain forms of ALS and FTLD, devastating diseases with very limited treatment options,” said Dr. Sandy Macrae, Chief Executive Officer of Sangamo. “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.

We look forward to working with Sangamo on potential treatments for devastating diseases related to genetic mutations of the C9ORF72 gene,” said Greg LaRosa, Senior Vice President and Chief Scientific Officer, Pfizer Rare Disease. “Pfizer is proud of the progress we have made in the area of gene therapy, which offers tremendous promise to patients and their families.

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