Abeona up on PRIME designation for gene therapy ABO-102 for the treatment of MPS IIIA

US cell and gene therapy company Abeona Therapeutics (Nasdaq: ABEO) saw its shares gain 4.2% to $3.75 in after-hours trading Tuesday, after it announced that the European Medicines Agency has granted the designation of Priority Medicines ( PRIME) for its ABO-102, which studies its punctual gene therapy based on adeno-associated virus 9 (AAV9) for the treatment of Sanfilippo syndrome type A (MPS IIIA).

The PRIME designation is based on clinical and non-clinical data from the study Transpher A global clinical trial Phase I/II evaluating a single dose of ABO-102 for the treatment of children with MPS IIIA.

"The PRIME designation from the EMA for the ABO-102 program recognizes the urgent need for a treatment option for children with MPS IIIA, and highlights the potential of ABO-102 to modify the progression of this disease overload lysosomal devastating,” said Dr João Siffert, president and chief executive.