Abeona Therapeutics (ABEO) Updates on ABO-102 Phase 1/2 MPS IIIA; Significant Avg. GAG Reduction Noted
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Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at Orphan Drugs & Rare Disease Conference, London UK
October 20, 2016 7:43 AM EDTNEW YORK, NY and CLEVELAND, OH -- (Marketwired) -- 10/20/16 -- Abeona Therapeutics Inc. (NASDAQ: ABEO):
ABO-102 gene therapy well-tolerated at low-dose Cohort of 3 subjects Significant average GAG (heparan sulfate) reduction in urine (57.6% +/- 8.2%) and cerebral spinal fluid (25.6% +/- 0.8%) and reduction in liver (17.1% +/- 1.9%) and spleen volume (17.6% +/- 7.1%) observed at 30 Days post-intravenous injectionCompany to host... MoreAbeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-102 Gene Therapy in Sanfilippo Syndrome Type A
October 18, 2016 7:45 AM EDTNEW YORK, NY, and CLEVELAND, OH -- (Marketwired) -- 10/18/16 --
Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical- stage biopharmaceutical company focused on developing gene therapies for life-threatening rare diseases, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona's lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.
"Receiving... More