- Pfizer Inc. on Wednesday said it would partner with Sangamo Therapeutics Inc. to develop a gene therapy for amyotrophic lateral sclerosis (ALS) and a related neurodegenerative disorder, deepening the pharma giant's investment into exploring DNA-based treatments for rare diseases.
- Under the collaboration deal, Pfizer will pay Sangamo $12 million upfront — and as much as $150 million in milestone payments — to tap the California biotech's zinc finger protein technology. The companies previously teamed up in May to work on a gene therapy for hemophilia A.
- Sangamo will be responsible for developing potential candidates using zing finger protein transcription factors, after which Pfizer will take over for any subsequent research, development and commercialization.
Amid the enthusiasm over the potential of CRISPR-based gene therapy, Sangamo's zinc finger technology tends to get overlooked. The biotech has a full pipeline of therapeutic candidates, however, and is currently recruiting for four small open-label studies in hemophilia and two lysosomal storage disorders.
In May, Pfizer paid $70 million upfront to gain access to Sangamo's SB-525 candidate for hemophilia A, which recently entered the clinic at the end of last summer.
The most recent collaboration deal will expand that work into neurological disease, exploring the application of zinc finger proteins for treatment of ALS or frontotemporal lobar degeneration (FTLD) tied to mutations of a gene known as C9ORF72.
In contrast to gene editing or gene replacement, Pfizer and Sangamo will explore use of so-called transcription factor-mediated gene regulation. With this approach, Sangamo's zinc finger proteins latch on to a relevant sequence of DNA. A transcriptional repressor domain attached to that protein then acts to suppress the expression of the gene.
The companies plan to target the C9ORF72 gene — mutations of which can lead to ALS or FTLD. A third of cases of familial ALS result from these types of mutations, Pfizer said in a statement.
Sangamo isn't Pfizer's only research pathway into the gene therapy field. In 2016, the pharma giant bought Bamboo Therapeutics for $150 million, picking up a manufacturing facility for adeno-associated viruses. AAVs, as they are more commonly referred to, are crucial components for delivery of gene therapies into the body.
Pfizer also has an ongoing collaboration with Spark Therapeutics Inc. to develop a gene therapy for hemophilia B.