Pfizer is paying $12m upfront to Sangamo Therapeutics in a deal focusing on the development of gene therapies for motor neuron disease, also known as amyotrophic lateral sclerosis (ALS).
The deal – which also includes $150m in potential milestones – focuses on the use of Samgamo’s zinc finger protein (ZFP) technology to address mutations in the C9ORF72 gene, linked to ALS and another neurodegenerative disease called frontotemporal lobar degeneration (FTLD).
This is the second collaboration between the two companies, which are already working on a gene therapy candidate for haemophilia A that started phase I/II clinical testing last summer.
Around a third of all cases of familial ALS is caused by mutations in the C9ORF72 gene, which leads to hundreds of additional repetitions of a six base pair sequence of DNA. This genetic disruption ultimately leads to the irreversible deterioration of neurons.
Sangamo’s technology involves introducing a ZFP carrying a repressor sequence into the C9ORF72 gene, to down-regulate its expression and – hopefully – prevent the neurons from degenerating.
Sangamo has responsibility for the initial development of the C9ORF72 gene-targeting ZFP canddiates, with Pfizer “operationally and financially responsible for subsequent research, development, manufacturing and commercialization … and any resulting products”.
The deal comes as excitement about gene therapy is gathering pace again after a long period in doldrums punctuated by clinical failures and concerns about the safety of some of the viral vectors used to deliver genetic sequences.
Towards the end of last year, the FDA approved its first gene therapy – Spark’s Luxturna – and this week GlaxoSmithKline won NICE backing in the UK for its Strimvelis therapy for bubble baby syndrome, adding to confidence that a commercial model is viable for advanced therapies.
Sangamo is hoping to ride the wave of resurgence in gene therapy, and CEO Sandy Macrae says the “precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation”.