Abeona Therapeutics (ABEO) Concludes ABO-102 Phase 1/2 Enrollment for MPS IIIA Patients

August 29, 2016 7:06 AM EDT
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Abeona Therapeutics Inc. (Nasdaq: ABEO) announced that enrollment has been completed for the low-dose cohort (n=3) in its ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes a single injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death.

"Completing enrollment of the low-dose cohort is an important milestone in the advancement of ABO-102 for patients with Sanfilippo syndrome type A," said Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. "We look forward to providing an update of topline data from the completed cohort and the anticipated commencement of the high-dose cohort in the coming months."

The company recently announced that ABO-102 is well tolerated in initial subjects and preliminary biopotency appears promising. Abeona's MPS IIIA program, ABO-102, has been granted Orphan Product Designation in the USA and received the Rare Pediatric Disease Designation.

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