Friday’s advisory committee meeting to discuss Sarepta’s DMD gene therapy could have broad implications for the field

ProQR’s sepofarsen, an antisense oligonucleotides, stumbles in late-stage trial for LCA10

ProQR is readying to advance a second RNA-based antisense oligonucleotide therapy for inherited retinal diseases into pivotal testing on the back of positive early data for QR-421a late last

How gene therapy manufacturing bottlenecks are impeding academic translation