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|Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa|
Abeona’s Fourth Gene Therapy Program to Receive EMA Orphan Designation
EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) has Demonstrated Promising Efficacy and Safety in Ongoing Phase 1/2 Clinical Trial
“EB-101 is Abeona’s fourth gene therapy program to be granted EMA Orphan Designation and it further builds on our commercial portfolio of clinical-stage gene therapies that have received
The ongoing phase 1/2 clinical trial with gene-corrected skin grafts has shown promising wound healing and safety in patients with RDEB. Investigators at
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 initial four patients of the clinical study demonstrated that treatment with EB-101 restored type VII collagen (C7) 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, and 50% at 12 months compared with baseline wound sites.
“The encouraging EB-101 clinical results advances our support to address the significant unmet medical needs that RDEB patients experience and underscores our commitment to collaborating with outstanding research groups such as
About EB-101: EB-101 is an autologous, ex-vivo gene therapy in which COL7A1 is transduced into autologous keratinocytes for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). EB-101 has been well tolerated to date and demonstrated promising efficacy in the ongoing phase 1/2 clinical trial in RDEB patients (NCT01263379).
About Epidermolysis Bullosa (EB): EB is a group of devastating, life-threatening genetic skin disorders 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 belief that the designation by the EMA is an important validation of the scientific and clinical translation of our products for severely underserved patient populations. 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 ability to successfully continue our 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