2022 Mary Ann Liebert Publishers, Inc. Young Investigator Award Winner Guizhi (Julian) Zhu, PhD
Guizhi (Julian) Zhu, PhD Virginia Commonwealth University Biography: Guizhi (Julian) Zhu is currently an Assistant Professor of the Department of Pharmaceutics and Center for Pharmaceutical Engineering and Sciences at Virginia Commonwealth University (VCU). He is a co-founder and CSO of a startup company AmpedRNA ...
OTS President’s Pick Paper – September 2022
Today’s President’s pick is a bit unusual. I was watching television and noticed a new commercial. A jolly man was happily proclaiming that he had a new medication for controlling his high cholesterol. He seemed very pleased. We’ve all seen these commercials many times before, ...
The Personal Journey of Stanley Crooke, the Creation and Development of Ionis, and Beyond
More than 30 years ago, Dr. Stanley Crooke founded Ionis Pharmaceuticals — a biotech company that helped pioneer the now successful technology of antisense oligonucleotides (ASO) despite overwhelming challenges and widespread disbelief. After stepping down last year as the company's CEO, Crooke recently published ...
A Landmark Permanent Treatment for Children Born with a Deadly Immune Disorder
In June 2022, the first stem cell gene therapy of Dutch origin was successfully given to a patient by researchers at the Leiden University Medical Centre (LUMC). A baby with severe congenital immune disorder (SCID) received the complex but one-time treatment, marking the first ...
Treating Cardiovascular Disease by Changing a Single Letter of DNA
A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. On July 12, biotechnology company Verve Therapeutics announced that the first patient had received a novel gene editing medicine designed to permanently reduce "bad" LDL cholesterol — the fatty ...
Interview with Pawan Kumar, PhD
Pawan Kumar, PhD Principal Scientist, Takeda Pharmaceuticals How did you become interested in the field of oligonucleotides? I was first exposed to the field of therapeutic oligonucleotides as an exchange PhD student in the Hrdlicka’s lab at the University of Idaho, where I developed ...
Patient Advocacy and Therapeutic Development for Huntington’s Disease
Time: 11am - 12pm EST Date: August 11, 2022 Description: Dan O'Reilly, PhD, a former OTS Trainee BoD member, focuses his research on developing siRNA therapeutics for Huntington’s Disease. Join us as Dr. O'Reilly discusses patient advocacy and Huntington’s Disease research. ...
FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients
In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. As oligonucleotide therapeutics continue to develop as an emerging and promising treatment for ...
OTS President’s Pick Paper – July 2022
By: David Corey Establishing an environment in which rigorous scientific inquiry is practiced: A personal journey. Stanley Crooke Nucleic Acids Research, gkac526, https://doi.org/10.1093/nar/gkac526 Published: 08 July 2022. https://academic.oup.com/nar/advance-article/doi/10.1093/nar/gkac526/6633881?login=true The Story of Ionis Pharmaceuticals For those new to the field, Ionis Pharmaceuticals has always been ...
A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye
Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its potential in treating disease. A key mechanism that induces gene-silencing in the RNA pathway is ...
