Abeona, EBRP and EB MRF to collaborate on epidermolysis bullosa treatments

Abeona Therapeutics president and CEO Timothy Miller said: "The addition of the EB gene therapy programs to our clinical pipeline advances our mission of serving those impacted by rare disease.

"The strong Phase 1 clinical data demonstrate safety and initial efficacy one year post treatment, and support a follow-on Phase 2 trial for children suffering from EB."

"This collaboration builds on our strengths in developing gene therapies for devastating rare diseases in partnership with patient groups and academic research centers," said Steven H. Rouhandeh, Executive Chairman of Abeona Therapeutics. "We are proud to work with the EB Research Partnership, EB Medical Research Foundation and Stanford University to accelerate these promising product candidates towards commercialization."

Phase 1 clinical trial results for the lead EB program, EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), were recently presented at the opening Plenary Session of the Society for Investigative Dermatology in May 2016.

Investigators at Stanford are recruiting patients for a Phase 2 trial to begin soon. These novel gene therapy products were developed at the Stanford University School of Medicine and are exclusively licensed to Abeona.

"This collaboration exemplifies the mission of EBRP to advance commercially sustainable research aimed at treating and ultimately curing epidermolysis bullosa," stated Alexander Silver, co-founder and Chairman, EBRP.

"We believe that Abeona can fully realize our mission of progressing research insights from academia into life-changing treatment solutions for EB patients and their families. This partnership also validates EBRP's venture philanthropy model, which is important in getting treatments to patients as soon as possible. We are thankful to the team at Stanford for all their hard work and assistance in forming this partnership."

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of a protein known as type VII collagen. Patients with RDEB develop large, severely painful blisters and chronic wounds from minor trauma to their skin and there are currently no FDA approved treatments.

The Phase 1 clinical trial with gene-corrected skin grafts has shown promising wound healing and safety in adult patients with RDEB. Investigators at Stanford are now recruiting patients for a Phase 2 trial with EB-101 in adolescents age 13 and older to determine the effect of type VII collagen gene-corrected grafts on wound healing efficacy.

About Epidermolysis Bullosa (EB): EB is a group of devastating, life-threatening genetic skin disorders impacting children that is characterized by skin blisters and erosions all over the body.

One of the most severe forms is recessive dystrophic epidermolysis bullosa (RDEB) characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions, and a shortened life span.

Patients with RDEB lack functional type VII collagen owing to mutations in the gene COL7A1 that encodes for C7. C7 is the main component of anchoring fibrils that attach the dermis to the epidermis. EB patients suffer through intense pain throughout their lives, with few or no effective treatments available to reduce the severity of their symptoms.

Along with the life- threatening infectious complications associated with this disorder, many individuals will develop an aggressive form of squamous cell carcinoma (SCC). Abeona's lead EB product, EB-101 (gene-corrected skin grafts), is a gene therapy currently in clinical trials for the treatment of RDEB patients.