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|Abeona Therapeutics Announces JAMA Publication of Positive Phase 1 Study Results for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa|
Typically, wounds in patients with RDEB, also known as "butterfly skin" syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body. Results from the clinical study demonstrated that treatment with EB-101 restored Type VII collagen expression at the dermal-epidermal junction at the graft sites in 90% of the biopsy samples at 3 months post-treatment, in 66% at 6 months post-treatment, and in 42% samples at 12 months post-treatment. Importantly, correct type VII collagen localization was observed at anchoring fibrils. Wounds that demonstrated type VII collagen at graft sites displayed 87% healing at 3 months, 67% at 6 months, 50% at 12 months compared with baseline wound sites.
"Phase 1 data indicate that EB-101 COL7A1 ex-vivo gene transfer has a favorable safety profile and capable of cutaneous type C7 delivery, highlighting the potential of durable cell-based RDEB therapy in humans in devastating non-healing chronic wounds associated with high levels of morbidity and mortality," noted
The Phase 1 clinical trial with gene-corrected skin grafts has shown promising wound healing and safety in patients with RDEB. Investigators at
"The clinical data demonstrate that EB-101 gene therapy corrected the underlying genetic deficit in RDEB patient wounds for months to over a year, and the wounds closed -- which is remarkable for a disease where the patient's skin can blister and erode every day," said
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. The most severe form, recessive dystrophic epidermolysis bullosa (RDEB), is 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 (C7) owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils that attach the dermis to the epidermis. EB patients suffer through intense pain throughout their lives, with no effective treatments available to reduce the severity of their symptoms. Along with the life-threatening infectious complications associated with this disorder, many individuals often develop an aggressive form of squamous cell carcinoma (SCC).
This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the