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|Abeona Therapeutics Announces Publication of Preclinical Data Supporting Clinical Translation of Juvenile Batten Disease Gene Therapy|
"This is the first demonstration of a systemic delivery route to restore CLN3 function, and highlights the importance of selecting the right vector, promoter and delivery route to treat the symptoms of this devastating disease," said
Researchers concluded that a single intravenous injection "led to widespread virus biodistribution in the brain, spinal cord, and eye" that was capable of "improving motor function, attenuating microglial and astrocyte activation, and reducing lysosomal pathology, all hallmarks of JNCL" at an age when significant lysosomal pathology had already manifested.
"The data support the clinical translation of ABO-201 for patients with juvenile Batten disease, and demonstrates AAV delivery to target tissues in the central nervous system as well as peripheral organs led to resolution of the underlying disease pathology," stated
The publication article can be accessed by clicking on the following link: (http://www.jneurosci.org/content/36/37/9669.short).
About ABO-201: ABO-201 (AAV-CLN3) is an AAV-based gene therapy which has shown promising preclinical efficacy in delivery of a normal copy of the defective CLN3 gene to cells of the central nervous system with the aim of reversing the effects of the genetic errors that cause for Juvenile neuronal ceroid lipofuscinosis (JNCL) (also known as Juvenile Batten disease). JNCL is a rare, fatal, autosomal recessive (inherited) disorder of the nervous system that typically begins in children between 4 and 8 years of age. Often the first noticeable sign of JNCL is vision impairment, which tends to progress rapidly and eventually result in blindness. As the disease progresses, children experience the loss of previously acquired skills (developmental regression). This progression usually begins with the loss of the ability to speak in complete sentences. Children then lose motor skills, such as the ability to walk or sit. They also develop movement abnormalities that include rigidity or stiffness, slow or diminished movements (hypokinesia), and stooped posture. Beginning in mid- to late-childhood, affected children may have recurrent seizures (epilepsy), heart problems, behavioral problems, and difficulty sleeping. Life expectancy is greatly reduced, and there are no approved treatments for JNCL.
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 are looking forward to advancing multiple important new therapeutic candidates for the treatment of epidermolysis bullosa, that we plan to accelerate up to three new promising EB product candidates toward commercialization, that encouraging signs of early biopotency had been observed in urinary and CSF GAG (heparin sulfate) measurements as well as potential disease-modifying effects in the liver and spleen in our ABO-102 program, 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