Sangamo Therapeutics as well as also Pfizer said on Wednesday they would likely work together to develop a gene therapy to treat ALS, a disease in which affects nerve cells inside the brain as well as also the spinal cord.
The gene therapy will also be used to treat patients which has a brain disorder caused by mutations of a gene in which is actually linked to about a third of hereditary ALS cases.
The partnership comes at a time when gene therapy is actually gaining momentum, after several high profile failures inside the late 1990s as well as also early 2000s, with the U.S. Food as well as also Drug Administration recently updating guidance to help speed up development of such treatments.
The agency last month approved Spark Therapeutics’s treatment for a rare form of blindness, marking the first approval of a gene therapy for an inherited disease. The company announced Wednesday the treatment would likely cost $850,000.
Under the terms of the deal, Sangamo will receive a $12 million upfront payment coming from Pfizer as well as also one more $150 million in milestone payments, the companies said.
ALS (amyotrophic lateral sclerosis), whose sufferers include renowned British physicist Stephen Hawking, attracted international attention in 2014 with the “Ice Bucket Challenge”, in which people posted videos of pouring ice-cold water on themselves to encourage donations to research.
Shares of Sangamo were up 4.3 percent at $18.30, while those of Pfizer were unchanged.