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|Abeona Therapeutics Doses First Patient in Phase 1/2 Trial With ABO-102 Gene Therapy for Patients With Sanfilippo Syndrome Type A (MPS IIIA)|
"MPS IIIA is a profound and deadly lysosomal storage disease with no approved treatments available. Caused by a single gene defect, most of the children with this disease do not reach adulthood. This investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, represents a new treatment paradigm for addressing this relentlessly progressing disease," noted
"This treatment trial is the culmination of more than eighteen years of research and development, and none of it would have been possible without support from the Sanfilippo foundations," say Drs.
The company previously announced that ABO-102 has been granted Orphan Product Designation in the
The clinical study is supported by neurocognitive testing data generated in a 25-subject MPS III Natural History Study, where all patients are through 1 year of follow up assessments.
"Abeona has demonstrated global leadership in supporting clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and we applaud their efforts to bring these potential treatments to clinical trials," said
"This first-in-man gene therapy, which uses an AAV delivered by a single intravenous injection to treat the central nervous system and the peripheral disease manifestations in Sanfilippo patients, helps advance the field of gene therapy with new treatment options for these devastating diseases," noted
About ABO-102 (AAV-SGSH): ABO-102 is an adeno-associated viral (AAV)-based gene therapy for MPS IIIA (Sanfilippo syndrome), which involves a one-time delivery of a normal copy of the defective gene to cells of the central nervous system (CNS) with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in Sanfilippo preclinical models, ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzyme repaired the underlying cell pathology that is the cause of the disease. Preclinical in vivo efficacy studies in Sanfilippo syndrome have demonstrated functional benefits that are sustained for months to years after treatment. A single dose of ABO-102 significantly restored normal cell and organ function, corrected cognitive deficits, increased neuromuscular function and normalized the lifespan of animals with MPS IIIA over 100% for more than one year after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment to replace the defective gene could potentially benefit patients with Sanfilippo syndrome. In addition, safety studies conducted in animal models of Sanfilippo syndrome have demonstrated that delivery of ABO-102 is well tolerated with minimal side effects.
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, management plans for the Company, and general business outlook. 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
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