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|Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in Spain|
“The authorization of our ABO-101 trial in
Subjects in the Phase 1/2 trial receive a single, intravenous infusion of ABO-101, which uses an AAV vector to introduce the functional NAGLU gene to treat patients with MPS IIIB disease. Subjects will be evaluated at multiple time points post-injection for safety assessments and efficacy parameters. The clinical program is supported by a Natural History Study which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments.
ABO-101 has been granted Rare Pediatric Disease Designation in the U.S., and Orphan Product Designation in both the U.S. and the
About ABO-101 (AAV-NAGLU): ABO-101 is Abeona’s first-in-human, adeno-associated viral (AAV)-based gene therapy for MPS III (Sanfilippo syndrome). Treatment involves a one-time intravenous delivery of a functioning copy of the N-acetyl-α-D-glucosaminidase (NAGLU) gene to cells of the central nervous system (CNS) and peripheral organs, with the aim of correcting the effects that result from the genetic aberrations that are the root cause of the disease. Following administration of a single dose in Sanfilippo preclinical animal models, ABO-101 induced cells in the Central Nervous System and peripheral organs to produce the missing NAGLU enzyme, which then restored underlying sugar (glycosaminoglycan or GAG) storage pathology to normal levels in cells. In preclinical in vivo efficacy studies in Sanfilippo syndrome animal model, ABO-101 demonstrated functional benefits that continue for months after treatment. A single dose of ABO-101 significantly restored normal cell and organ function, corrected cognitive defects, increased neuromuscular function and normalized the lifespan of animals with MPS IIIB after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with Sanfilippo syndrome. Safety and efficacy studies of AAV gene therapy treatments for Sanfilippo syndrome have recently been published in several peer-reviewed scientific journals.
About MPS IIIB: (also known as Sanfilippo syndrome type B) is a genetic, progressive, and devastating rare lysosomal storage disease. In patients with MPS IIIB, genetic mutations result in a marked decrease in NAGLU enzyme activity, which leads to accumulation of heparan sulfate (HS) in the brain and other organs as well as progressive brain atrophy with cortical gray matter volume loss. The accumulation of abnormal HS results in neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. MPS IIIB typically presents in children during the first few years of life, and 70% of patients do not reach 18 years of age. There are no approved treatments for MPS IIIB.
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 Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. Such statements include that we are encouraged by the results of the US trial, and that we plan to add clinical sites for the trial in three European countries. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, 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 secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions; risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the