Shanghai-based ReJuKon Biopharm Inc. has announced that its Category 1 drug RJK002 has received tacit clinical trial approval from China’s Center for Drug Evaluation (CDE) for the treatment of amyotrophic lateral sclerosis (ALS). RJK002 is recognized as the first clinically approved adeno-associated virus (AAV) gene therapy for ALS in China and marks a significant advancement in the global landscape of ALS treatments.
The drug is also noted as the world’s first AAV gene therapy targeting abnormal protein aggregation associated with ALS. In October 2023, RJK002 was granted orphan drug designation (ODD) in the US, following compelling evidence of its neuroprotective and survival-extending therapeutic effects demonstrated in preclinical animal models of ALS. RJK002 is now set to undergo a multi-center investigator-initiated trial in China.- Flcube.com
