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FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients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 ...
Transitioning from Trainee to Faculty – A conversation with Prof. Bethany Powell Gray
Time: 11am - 12pm EST Date: July 14, 2022 Description: Join us for an open discussion with Dr. Bethany Powell Gray, who is an Assisstant Professor at the John Hopkins University School of Medicine, where we will talk about the postdoc to faculty ...
Positive Results from Tofersen VALOR Trial and OLE Integrated Data
People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed ...
Trainee Spotlight Series: Yan Li and Roberto El Khoury
Title: Trainee Spotlight Series: Yan Li and Roberto El Khoury Time: 11am EST Date: July 7, 2022 Description: This Trainee Spotlight features Yan Li, a Ph.D. student at the University of California, Los Angeles, and Roberto El Khoury a Ph.D. candidate at McGill ...
