Abeona Therapeutics (ABEO) Announces Presentation of ABO-102 Phase 1/2 data in MPS IIA

October 14, 2016 7:03 AM EDT
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Abeona Therapeutics Inc. (Nasdaq: ABEO) announced that top-line results of the low-dose cohort for ABO-102 in Phase 1/2 clinical trial for Sanfilippo type A (MPS IIIA) patients will be presented at the Orphan Drugs and Rare Disease Congress taking place in London, UK, from October 19th through October 20th. The company's ABO-102 program, enrolling at Nationwide Children's Hospital (Columbus, Ohio), is the first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for MPS IIIA, a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children. Abeona management will provide an update on the ABO-102 and other programs on a conference call during the conference.

Abeona Recent Highlights:

  • October 5, 2016: announced Data Safety Monitoring Board Approved ABO-102 Dose Escalation for Second Cohort in a Phase 1/2 Clinical Trial for Sanfilippo Syndrome Type A
  • September 26, 2016: enrolled First Patient in Phase 2 for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa
  • September 21, 2016: announced the exclusive worldwide license of the AIMâ„¢ AAV capsid portfolio for next generation gene therapies from University of North Carolina at Chapel Hill
  • September 8, 2016: enrolled 5th Patient in Phase 1/2 Gene Therapy Clinical Trial for Epidermolysis Bullosa
  • August 29, 2016: completed enrollment of the Low-Dose Cohort for ABO-102 in Phase 1/2 Clinical Trial for MPS IIIA Patients (n = 3)
  • August 9, 2016: announced a collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University for the development of treatments for recessive dystrophic epidermolysis bullosa (RDEB)
  • August 4, 2016: announced it had received European regulatory approval by the Agencia Espanola de Medicamentos y Productos Sanitarios for a Phase 1/2 trial for ABO-102 (AAV-SGSH) to be conducted at Cruces University Hospital (Bilbao, Spain)
  • August 2, 2016: provided an update on the initial subjects enrolled in the ABO-102 clinical trial, stating that ABO-102 had been well tolerated with no safety or tolerability concerns identified through 30-days post-injection, and that encouraging signs of early biopotency had been observed in urinary and CSF GAG (heparan sulfate) measurements as well as potential disease-modifying effects in the liver and spleen


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