Pfizer and Sangamo to develop amyotrophic lateral sclerosis therapies

Pfizer has announced a new drug research collaboration with Sangamo that will see the companies working to develop innovative treatments for amyotrophic lateral sclerosis (ALS).

The partners will work together to create a potential gene therapy using zinc finger protein transcription factors to treat ALS and frontotemporal lobar degeneration linked to mutations of the C9ORF72 gene.

Both conditions are part of a spectrum of neurodegenerative disorders caused by mutations in this gene, leading to the deterioration of motor neurons, in the case of ALS, or neurons in the frontal and temporal lobes among FTLD patients. There are currently no cures for either disease.

Sangamo's ZFP-TF technology will be used to suppress expression of the gene in question, with the research to investigate allele-specific ZFP-TFs with the potential to differentiate mutant C9ORF72 alleles from wild-type alleles.

Greg LaRosa, senior vice-president and chief scientific officer for Pfizer's rare disease business, said: "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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