News | August 29, 2016

Abeona Therapeutics Completes Enrollment Of Low-Dose Cohort For ABO-102 In Phase 1/2 Clinical Trial For MPS IIIA Patients

  • Low-dose cohort (n=3) enrollment completed for ongoing study to evaluate ABO-102 (AAV-SGSH), first-in-man AAV gene therapy by single intravenous injection in patients with MPS IIIA, a rare lysosomal storage disease
  • Encouraging safety profile and early biopotency observed in initial subjects

Abeona Therapeutics Inc. , a clinical-stage biopharmaceutical company focused on developing gene and plasma-based therapies for life-threatening rare genetic diseases, announced recently 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.

About ABO-102 (AAV-SGSH)
ABO-102 is an adeno-associated viral (AAV)-based gene therapy for MPS IIIA (Sanfilippo syndrome), which involves a one-time delivery of a normal copy of the defective gene to cells of the central nervous system (CNS) with the aim of reversing the effects of the genetic errors that cause the disease. ABO-102, has been well tolerated in initial subjects of the low-dose cohort with no safety or tolerability concerns identified through 30-day post-injection in patients suffering from MPS IIIA, or Sanfilippo syndrome Type A, a rare autosomal recessive disease, is caused by genetic mutations that result in a deficiency of SGSH enzyme activity, leading to abnormal accumulation of GAG (specifically, heparan sulfate) in the CNS and systemic tissues and organs. This accumulation of heparan sulfate results in neurocognitive decline, speech loss, loss of mobility, and premature death. Encouraging signs of early biopotency have been observed in urinary and CSF GAG (glycosaminoglycan, specifically, heparan sulfate) measurements, as well as potential disease-modifying effects in the liver and spleen of the initial subjects enrolled and treated in the trial. The clinical study is supported by neurocognitive evaluations, biochemical assessments and MRI data generated in a 25-subject MPS III Natural History Study, also conducted at Nationwide Children's Hospital, where patients continued through one-year of follow up assessments.

About Abeona
Abeona Therapeutics Inc. is a clinical stage company developing gene and plasma-based therapies for life-threatening rare genetic diseases. Abeona's lead programs are ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB), respectively. Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha (alpha-1 protease inhibitor) for inherited COPD, utilizing its proprietary SDF (Salt Diafiltration) ethanol-free process. For more information, visit

Source: Abeona Therapeutics Inc.