Clinical Trials

Date: 2016-09-15

Type of information: Publication of results in a medical journal

phase: preclinical

Announcement: publication of results in Molecular Therapy Methods & Clinical Development

Company: Abeona Therapeutics (USA - NY)

Product: ABO-102 (SC AAV-SGSH)

Action mechanism:

gene therapy. ABO-102 (AAV SGSH) is a next generation adeno-associated viral (AAV)-based gene therapy for MPS IIIB. This gene therapy involve a one-time delivery of a genetically modified virus to deliver a normal copy of the defective gene to cells of the central nervous system and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in preclinical animal models of Sanfilippo syndrome ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in animals with Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose significantly restored normal cell and organ function, corrected cognitive defects that remained months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals.

Disease: mucopolysaccharidosis type IIIA (Sanfilippo B syndrome)

Therapeutic area: Rare diseases - Genetic diseases

Country: USA

Trial details:

Latest news:

• On September 15, 2016, Abeona Therapeutics announced that preclinical data supporting clinical trials for ABO-102 (AAV-SGSH), the AAV-based single intravenous gene therapy program for MPS IIIA, (Sanfilippo Type A), were published in the June issue of Molecular Therapy Methods & Clinical Development. Researchers concluded that "… an intravenous injection of scAAV9-U1a-hSGSH vector, leading to restoration of SGSH activity and reduction of glycosaminoglycans (GAG) throughout the central nervous system and somatic tissues at a dose…". Other benefits included improved learning ability, increased survival, and improved GAG storage pathology in the CNS, leading researchers to note that: "The study suggests that there is potential for gene therapy intervention in MPS IIIA at intermediate stages of the disease, and extends the clinical relevance of our systemic scAAV9-hSGSH gene delivery approach." Abeona Therapeutics previously announced that ABO-102 preliminary measures of clinically relevant biomarkers in the ABO-102 Phase 1/2 clinical trial provided promising signals of potential systemic and CNS clinical benefits for patients suffering with MPS IIIA. The program has been granted Orphan Product Designation in the USA and received the Rare Pediatric Disease Designation. Abeona is currently working toward opening two additional clinical sites to test ABO-102, one in Spain and one in Australia.