Abeona Therapeutics Expands ABO-102 Gene Therapy Clinical Trials

Abeona Therapeutics (NASDAQ:ABEO) announced an expansion to its gene therapy clinical trials for patients with MPS IIIA in the US, Europe, and Australia.
As quoted in the press release:

“We have completed the necessary regulatory and ethical committee approvals and site initiations in Europe and Australia in order to accelerate enrollment,” stated Juan Ruiz, M.D., Ph.D., Abeona’s Chief Medical Officer. “We remain very encouraged by the improvements observed in clinically relevant biomarkers post-dosing of ABO-102, including durable reductions in heparan sulfate measured in the CNS, reduction of organ disease pathology, and signals of CNS improvement or stabilization at one-year follow-up in Cohort 1 subjects, and look forward to providing a more fulsome clinical update at important clinical conferences, including ESGCT, this fall,” he continued.

The planned expansion will enroll an additional eight to ten MPS IIIA subjects, with total enrollment of fourteen to sixteen subjects to be completed by 1Q2018. Per the design of the clinical trial, subjects will receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system.

Click here to read the full press release.

Source: globenewswire.com