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Skin disease solution
NEW YORK & CLEVELAND—Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on delivering gene- and plasma-based therapies for life-threatening rare diseases, is collaborating with EB Research Partnership (EBRP) and EB Research Medical Foundation (EBMRF) on gene therapy treatments for epidermolysis bullosa (EB). This group of devastating rare genetic skin disorders impacting children is characterized by skin blisters and erosions all over the body.
According to Dr. Timothy Miller, president and CEO of Abeona Therapeutics, “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.”
As he explained, recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering skin disease caused by the 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.
“Our lead EB-producing candidate is EB-101 (gene corrected skin grafts), an ex-vivo gene therapy developed at Stanford University for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) that has demonstrated promising Phase 1 results in adult RDEB patients,” Miller said. The Phase 1 clinical trial with gene-corrected skin grafts has shown “promising wound healing and safety in adult patients with RDEB.”
“Phase 2 clinical trials in adolescents are expected to begin in the second half of 2016,” he added. Investigators at Stanford University 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.
As Miller explained, “The EB-201 (AAV DJ Col7A1) preclinical candidate uses a novel gene therapy approach known as homologous recombination, which works with a targeted AAV-mediated gene editing and delivery approach to correct gene mutations in skin cells (keratinocytes). The AAV DJ delivers a native COL7A1 gene fragment into affected cells and induces homologous recombination to fix a genomic copy of the mutated gene. This novel approach possesses relatively high recombination frequency and low off-target changes and doesn’t require the introduction of bacterial proteins (such as CRISPR/Cas9) to stimulate recombination, so potentially it can be used for gene editing in vivo.”
Abeona was originally founded with 12 different patient foundations from around the world to advance novel therapies for rare disease patients, according to Miller. Abeona was already working with investigators at Stanford on a different gene therapy technology and noted an “outstanding” EB program, he said. The research and development program, sponsored by the EB Research Partnership and EB Medical Research Foundation, had already advanced EB-101 into early clinical studies, with additional programs in the lab, but needed an industry partner to accelerate development and commercialization. The EBRP and EBMRF are “dedicated, committed and progressive patient foundations that recognize the importance of translational development of research from the bench to bedside,” Miller commented.
There are approximately 2,500 patients with RDEB in the United States.
“We believe EB-101 is a leading gene therapy candidate which may significantly advance RDEB treatment for patients and their families, as well as present a promising commercial opportunity which builds on Abeona’s leadership in rare disease,” noted Miller.
“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 of 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.”
Steven H. Rouhandeh, executive chairman of Abeona Therapeutics, summarized, “This collaboration builds on our strengths in developing gene therapies for devastating rare diseases in partnership with patient groups and academic research centers. 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.”