The European Medicines Agency has awarded Priority Medicines (PRIME) designation to Abeona Therapeutics’ rare disease therapy ABO-102.

ABO-102 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS).

The therapy is dosed in a one-time intravenous infusion using an AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs.

It has been developed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that causes progressive cell damage and neurodevelopmental and physical decline.

The PRIME designation for the ABO-102 programme “recognises the urgent need for a treatment option for children suffering from MPS IIIA, and underscores the potential of ABO-102 to modify the course of this devastating lysosomal storage disease,” said João Siffert, Abeona’s chief executive.

The PRIME initiative provides companies with access to enhanced support for the development of medicines targeting an unmet medical need.

It allows sponsors enhanced interaction and early dialogue with the regulator regarding promising medicines, as well as the possibility of accelerated assessment of medicines applications, to help patients benefit from them as early as possible.