Search for potential participants in ongoing gene transfer clinical trials in Mucopolysaccharidosis type III (Sanfilippo A/B) and Neuronal Ceroid Lipofuscinosis (CLN) type 1
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for life- threatening paediatric rare genetic diseases. We are currently conducting clinical trials using adeno-associated virus serotype 9 (AAV9)-based vectors for correction of enzymatic defect in lysosomal storage disorders. These studies may be of interest to your patients. Other AAV-based gene therapies have [...]