US-based Abeona Therapeutics has begun its enrolment of high-dose subjects for the ongoing Phase I/II clinical trial of ABO-102 (AAV-SGSH) to treat Sanfilippo syndrome type A (MPS IIIA).
ABO-102 is an adeno-associated viral (AAV)-based gene therapy designed to deliver a normal copy of the defective gene to cells of the central nervous system (CNS) in order to reverse the effects of the genetic errors that cause the disease.
The ongoing Phase I/II trial will evaluate safety and preliminary indications of the efficacy of a single ABO-102 intravenous injection.
MPS IIIA is a lysosomal storage disease that affects every cell and organ and leads to neurocognitive decline, speech loss, loss of mobility, and premature death.
It is caused due to genetic mutations, resulting in a deficiency of SGSH enzyme activity and abnormal accumulation of GAG in the CNS, systemic tissues and organs.
Abeona Therapeutics presedient and chief executive officer Timothy Miller said: “The combination of CSF and urinary heparan sulfate GAG reduction, liver and spleen volume reduction, and neurological effects support our world leading gene therapy treatment paradigms for patients with MPS III."
The single injection given to the subjects in the low-dose and high-dose cohorts will deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene.
The subjects will be assessed at multiple time points during the initial six months post-injection for safety assessments and initial signals of biopotency.
The ABO-102 MPS IIIA programme has secured orphan product designation in the US and Europe, as well as the rare pediatric disease designation in the US.