Sangamo Therapeutics and Pfizer entered a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFPTFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C90RF72 gene.
ALS and FTLD are part of a spectrum of neurodegenerative disorders caused by mutations in the C90RF72 gene that involve hundreds of additional repetitions of a six base pair sequence of DNA.
Sangamo will receive a $12 million upfront payment from Pfizer. Sangamo will be responsible for the development of ZFPTF candidates. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization for the C90RF72 ZFPTF 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.
"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."