Companies Announce Collaboration to Develop Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The focus of this partnership will be the development of FCX-007. This is Fibrocell’s leading gene therapy candidate which is being developed to treat recessive dystrophic epidermolysis bullosa (RDEB). The partnership will combine the specialties and expertise of both companies. Fibrocell focuses on gene therapy and Castle Creek focuses on epidermolysis bullosa.

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. Recessive dystrophic epidermolysis bullosa is caused by mutations of the COL7A1 gene and is characterized by painful oral lesions. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer. 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.

Optimizing Drug Development

Under the agreement, Castle Creek will provide funding for further development of the therapy, which will allow Fibrocell to focus on some of its other candidates. In exchange, Castle Creek will obtain exclusive rights for commercialization and licensing in the USA. The partnership will help benefit the long term goals of both companies. In addition, it improves the chances of FCX-007 actually gaining official approval.

A Phase 3 clinical trial of 15-20 patients with recessive dystrophic epidermolysis bullosa is expected to begin in the second quarter of 2019.

 


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