Adeno-Associated Virus 9 Gene Therapy Granted Fast Track Status for CLN1 Disease

The Food and Drug Administration has granted Fast Track designation to ABO-202 (Abeona Therapeutics Inc), a one-time adeno-associated virus 9 (AAV9) gene therapy for the treatment of CLN1 disease, also known as infantile neuronal ceroid lipofuscinosis or infantile Batten disease.

CLN1 disease is a rapidly-progressing rare lysosomal storage disease caused by a defect in the CLN1 gene which leads to deficiencies in the PPT1 enzyme. The absence of this enzyme primarily affects the CNS and results in visual impairment, motor and cognitive decline, seizures and ultimately early death. Currently, this disease affects 2 to 4 out of every 100,000 children in the US and there are no approved treatments.

ABO-202 is designed to deliver a functional copy of the PPT1 gene to the CNS and peripheral organs using a combination of intravenous (IV) and intrathecal administrations. Preclinical data showed that early treatment with ABO-202 normalized survival and improved neurological function in CLN1 mice. In addition, the combination of IV and intrathecal infusions improved efficacy over either route alone.

“Receiving Fast Track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” said João Siffert, MD, Chief Executive Officer.

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The Company will initiate a phase 1/2 clinical trial evaluating ABO-202 in patients with CLN1 disease and will provide guidance on the timing of the trial later this year.

ABO-202 has also received Orphan Drug designation and Rare Pediatric Disease designation from the FDA.

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