Ractigen receives orphan drug designation for ALS therapy
RAG-17 has been designed to act on and knockdown SOD1 expression in patients with pathogenic mutations that are known to cause ALS. The chemistry of the therapeutic is
The adeno-associated viral serotype rh10 (AAVrh10) gene therapy FBX-101 is delivered intravenously after hematopoetic stem cell transplant (HSCT) infusion. The designation for this lead adeno-associated virus (AAV) drug