As we look ahead to 2018, the most remarkable trend is the accelerating pace at which human applications of gene and cell therapy are advancing through the translational pipeline. The year 2017 witnessed the first two Food and Drug Administration (FDA)-approved gene therapy drugs in the United States, Kymriah and Yescarta, both anti-CD19 chimeric antigen receptor (CAR) T-cell therapies for B-cell cancers. In each case, the gene therapy component is a lentiviral vector, which is used to introduce the CAR into the T cells. These two new additions join other worldwide approved gene therapy products: Gendicine (Ad-P53 for cancer) and Oncorine (oncolytic Ad for cancer) in China, and Glybera (rAAV1-LPL for genetic hyperlipidemia) and Strimvelis (retro-ADA for immune deficiency). In addition, the 16-0 vote of the FDA's Cellular Tissue and Gene Therapies Advisory Committee in October 2017 has paved the way for FDA approval of voretigene neparvovec in late 2017. Other nucleic acid–based therapies are also now achieving similar milestones, including Spinraza, an exon-skipping therapy for spinal muscular atrophy (SMA) type 1, which was approved in 2017, and Patisirin, an siRNA therapy for TTR-induced amyloidosis, which has received Breakthrough Designation and for which a rolling biologics licensing application has been initiated. The progress of various cell-based therapies is even more complex, ranging from those in more established use, such as hematopoietic stem cells, through various cell therapies in clinical trials, such as mesenchymal stem cells, to those that remain preclinical.
Within the Human Gene Therapy family of journals, we are similarly optimistic and accelerating our pace to keep up with the rapidly evolving world of gene and cell therapy. Our plans for the coming year will include a special issue on CAR-T cells from guest editors Drs. Jennifer Adair and Cameron Turtle, which will include an interview with Dr. Michel Sadelain, as he reflects on the maturation of this technology and its entry into the world of clinical medicine, as well as a number of other original research papers and scholarly reviews on the topic. In addition, we anticipate another special issue on DNA vaccines as a technologic platform, with Dr. Shan Lu as guest editor.
In keeping with the global scope of gene and cell therapy research, we are most enthusiastic as we anticipate the special issue on gene therapy in China, timed for February 2018 to coincide with the celebration of the New Year in that nation. We hope that this issue will serve to highlight major achievements of gene and cell therapy researchers in China and will help to orient scientists outside of China to the major centers of excellence in gene therapy field in the world's most populous country. We are very grateful to Drs. Yuquan Wei and Depei Lu for agreeing to serve as guest editors for this ground-breaking special issue. We hope that it will serve as a first step in helping the gene therapy communities of East and West to come together in furthering the aims of our field and harness the power of the genomic revolution for the benefit of humanity.
