Sangamo And Pfizer To Collaborate In Developing ALS Gene Therapy

Pfizer and Sangamo Therapeutics announced on Wednesday that they would partner in the development of a gene therapy to be used in treating ALS. This is a disease which affects the nerve cells contained in the spinal cord and the brain. Additionally the gene therapy to be developed by the two pharmaceutical firms will be used in the treatment of patients suffering from a brain disorder resulting from a gene mutation. About a third of patients suffering from hereditary ALS have this mutation in their genes.

The partnership between Pfizer and Sangamo has coincided with a gain in momentum for gene therapy after a couple of high profile failures decades ago. Recently the Food and Drug Administration updated guidance with a view to assisting in speeding up the development of gene therapies.

Rare type of blindness

Last month a treatment for a rare blindness developed by Spark Therapeutics was approved by Food and Drug Administration. This was the time a gene therapy aimed at treating an inherited disease was getting approved. The deal between Pfizer and Sangamo will see the latter get upfront payments totaling $12 million from the former. There will also be milestone payments of about $150 million.

Pfizer and Sangamo’s partnership comes in the wake of the former being accused by United Kingdom’s Advertising Standards Authority of flouting advertising regulations in its marketing campaigns. On January 3, the Advertising Standards Authority issued a ruling to the effect that an ad developed for Imedeen, a collagen beauty shot developed by Pfizer exaggerated its claims.

“We considered that because the claim in the ad stated a particular site of action (the deep dermal layer of the skin), … it went beyond the authorized claim,” said the UK advertising watchdog in its ruling.

Leukemia treatment

Also earlier this week in the United Kingdom Pfizer welcomed a decision by the National Institute for Health and Care Excellence to reassess the benefits of its treatment Besponsa, as a therapy for leukemia. Last year in August Pfizer had lodged an appeal after the National Institute for Health and Care Excellence rejected the drug as a therapy for adult patients suffering from refractory or relapsed CD22-positive B-cell precursor acute lymphoblastic leukemia.

The reasons National Institute for Health and Care Excellence gave for the rejection included the cost-effectiveness of the treatment. During its INO-VATE clinical trial the Besponsa treatment was found to double the complete remission rates vis-à-vis the current standard treatment.

Leave a Reply

Your email address will not be published. Required fields are marked *