Sangamo Therapeutics’ Focus on Rare Diseases
Nathalie Dubois-Stringfellow, PhD, Senior Vice President of Product Development & Management at Sangamo Therapeutics discusses the company’s focus on rare diseases.
Sangamo Therapeutics, Inc has several gene therapies in clinical development to treat various rare conditions, including hemophilia A and Fabry disease, as discussed by Dr. Dubois-Stringfellow.
Hemophilia A is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Symptoms will vary and are dependent on the level factor VIII present. In the more severe forms of the disease, persons can experience bleeding following an injury and may have frequent spontaneous bleeding episodes, often into their joints and muscles. Treatment for hemophilia is factor VIII replenishment. Most hemophilia A patients take a recombinant factor VIII product to control bleeding and numerous products are available. Sangamo is working with Pfizer to develop a gene therapy for hemophilia A.
Fabry disease is a rare X-linked lysosomal storage disorder that results in the cellular buildup of globotriaosylceramide. Characteristic features of Fabry disease include acroparesthesias, angiokeratomas, hypohidrosis, corneal opacity, gastrointestinal problems, tinnitus, and hearing loss. Enzyme replacement therapy is the current standard treatment for this rare condition that has to be infused every two weeks. Sangamo is developing a gene therapy for Fabry disease and currently sponsoring a Phase1/2 clinical trial.
To learn more about Sangamo’s pipeline, visit https://www.sangamo.com/programs/
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