Drug Developer Receives $900,000 for the Development of a Gene Therapy for Epidermolysis Bullosa

According to a story from pm360online.com, the gene therapy company Fibrocell Science, Inc., recently received an investment of $900,000 from the Epidermolysis Bullosa Medical Research Foundation (EBMRF) and the EB Research Partnership, Inc. This funding will go towards the continued development of the company’s investigational product FCX-007, a gene therapy that is intended to treat recessive dystrophic epidermolysis bullosa (RDEB), a rare and ultimately lethal skin blistering disease. Fibrocell is dedicated to the development of gene therapies to treat disorders of the skin and connective tissue.

About Epidermolysis Bullosa

Epidermolysis bullosa is a group of genetic conditions which are most characterized by unusual blistering of the mucous membranes and skin. These painful blisters can occur with only minor friction or abrasion. The disease can be fatal in severe cases. These disorders are all caused by genetic mutations; the various subtypes of epidermolysis bullosa are classified by which genes are affected. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer.

Recessive dystrophic epidlermolysis bullosa is a severe form which is characterized by chronic inflammation, iron deficiency anemia, and damage to the esophagus. Most patient die before age 30 of cancer or other complications. There is no cure for the condition; treatment primarily focuses on symptom management, such as controlling pain, cleaning wounds, prevention or treatment of complications, nutritional support, and controlling infections. Males and females are affected at equal rates. The disease can seriously shorten lifespan, and in severe cases, patients do not survive beyond infancy. There is a dire need for more effective treatment approaches for epidermolysis bullosa. To learn more about these disorders, click here.

About FCX-007

FCX-007 has the potential to offer a therapy for patients who are in desperate need of a treatment option that addresses the underlying cause of the disease. Epidermolysis bullosa patients have few options that can help provide sustained, long term relief from their symptoms. FCX-007 is currently being tested as a therapy for RDEB in a Phase 2 clinical trial, the Fibrocell also plans to initiate a Phase 3 trial with the therapy in early 2019. The drug has earned Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the US Food and Drug Administration.


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