Rare skin disease gene therapy EB-101 enters Phase III clinical

Currently, most patients who plan to enroll in the VIITAL study have completed the pre-screening process at Stanford University, and the company expects the first patients in the VIITAL study to receive treatment in the first quarter of 2020RDEB is a rare connective tissue disease characterized by severe skin trauma that can cause pain and can lead to systemic complications that affect life expectancy and quality of lifeThe COL7A1 gene in RDEB patients is defective, preventing them from producing functional type VII collagen, which is necessary to fix the skin and epidermisAt present, there is no approved treatment for the diseaseEB-101 is an autonomous, gene-corrected cell therapy that is currently under Phase III clinical development for the treatment of RDEBEB-101 treatment involves gene transfer, introducing the COL7A1 gene into the patient's own skin cells (keratogenesformation cells) and transplanting it back to the patient's body table to make normal type VII collagen expression and promote wound healingData from Stanford University's Phase I/IIa clinical trial of EB-101 show that gene-corrected cell therapy provides patients with RDEB with lasting wound healing lasting from 2 to 5 years, including the largest and most challenging wounds affecting most RDEB patientsIn the United States, EB-101 has been awarded Advanced Therapy in Regenerative Medicine (RMAT), Breakthrough Drug Qualification (BTD), Rare Paediatric Disease Qualification (RPD), and in the United States and the European Union, EB-101 has been granted orphan drug eligibility (ODD)"With the launch of the critical Phase III study to evaluate EB-101 treatment of RDEB, we expect 2020 to be a transformational year for Abeona," said DrJo?o Siffert, CEO,AbeonaWe look forward to the first case of this quarter to receive EB-101 treatment to launch the final phase of development of this important projectEB-101 has the potential to be the first approved treatment for RDEB, the only durable treatment for large chronic wounds that are very painful and debilitatingin addition to EB-101,, Abeona Therapeutics has a number of therapeutic gene and cell therapies for sanfilippo syndrome type A (MPS IIIA), Sanfilippo syndrome type B (MPS IIIB), infant Barton disease (CLN1 disease), infant Barton disease (CLN3), cystic fibrosis (CF), retinopathy, etcend of December 2019, the company's gene therapy ABO-102 was granted priority drug qualification (PRIME) by the European Medicines Agency (EMA)ABO-102 is an adeno-related virus 9 (AAV9) gene therapy, administered through single-dose intravenous infusions, for the treatment of Type A of Sanfilippo syndrome (MPS IIIA) Sanfilippo syndrome Type A (MPS IIIA) is a rare lysosome accumulation disorder (LSD) that mainly affects the central nervous system (CNS) and there is currently no approved treatment The disease is characterized by neurodevelopment and rapid decline of the body Children with reduced sexual language and cognitive ability and abnormal behavior Other symptoms include sleep problems and frequent ear infections In addition, in early childhood, there are also obvious facial features, eyebrows thick or single eyebrows, plump lips, excessive body hair, liver and spleen swelling The disease is caused by a genetic mutation that causes a lack of SGSH enzymes, which break down glycomine polysaccharides, which accumulate in systemic cells, leading to a rapid decline in health associated with the disease ABO-102 delivered a functional copy of the SGSH gene to cells of the central nervous system (CNS) and peripheral organs via the AAV9 vector, which is intended to address the underlying cause of the disease, sGSH enzyme deficiency The lack of this enzyme leads to abnormal build-up of glycomine polysaccharides in the brain and the body, leading to sexual cell damage, neurodevelopment and physical decline in the United States, ABO-102 has been awarded advanced medical products (RMAT), fast-track eligibility (FTD), rare pediatric disease eligibility (RPD), orphan drug eligibility (ODD) In the European Union, ABO-102 is awarded TO PRIME and ODD by THE EMA original origin: Abeona Therapeutics Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Ive Dystrophic Epidermolysis Bullosa