Gene therapy is a hot topic in clinical research today — and for good reason! These technologies have the potential to treat — and in some cases even cure — a wide range of conditions, including rare genetic disorders that previously had no effective therapies. This article will show you the webinar collection of what gene therapy has achieved in treating diseases.
1.siRNA-Based Huntington's Disease Therapies
The webinar titled Optimal Therapeutic Intervention Timepoints for siRNA-Based Huntington's Disease Therapies was held on November 23, 2021, and organized by Creative Biolabs. The speaker is Dr. Markita Landry from the University of California, who talked about how her team developed a near Infrared Catecholamine nanosensor (nIRCat) and used real-time imaging to find out what drives late-disease decreases in evoked dopamine release, and how to use these findings as optimal therapeutic intervention timepoints for siRNA-based HD therapies.
The webinar recording is available to watch for free on the official website of Creative Biolabs.
2.Gene Therapy for Respiratory Diseases
The webinar was held by Advanced Therapy Treatment Centres (ATTC), and the speaker Dr. Eric Alton is Professor of Gene Therapy and Respiratory Medicine, National Heart & Lung Institute, Imperial College London. The webinar summarizes the translational research program’s progress of the UK Respiratory Gene Therapy Consortium, covering clinically relevant gene therapy for cystic fibrosis and other respiratory diseases.
3.Gene Therapy for Eye Diseases
The webinar was also held by ATTC, and the speaker is Dr. Jack Hickmott from the University of British Columbia, working on gene therapy for the rare ocular disorder Aniridia. This webinar explored new technologies in gene therapies and how they are being used in the clinic today. Key points cover the first FDA-approved gene therapy (Luxturna), gene editing trials for inherited vision loss, and a specific kind of protein that can save vision.
4.Gene Therapy for Rare Diseases: Considerations for Both Clinical and Post-Marketing Studies
This webinar held by Quanticate examined the challenges of rare diseases studies focusing on gene therapy treatments, such as appropriate study design, statistical analyses, the use of biomarkers, primary endpoint definition, drug efficacy proving, data sources, pharmacovigilance considerations for the characterization and management of risks, and the use of appropriate data capture and data quality oversight methods.
5.Gene Therapy Webinar
On Wednesday 16th September 2020, Metabolic Support UK held Gene Therapy Webinar to discuss the new gene therapies being developed for non-ketotic hyperglycinemia (NKH) and argininosuccinic aciduria (ASA), and future developments which may also be suitable for those living with other conditions including maple syrup urine disease (MSUD), progressive familial intrahepatic cholestasis type 2 (PFIC2), propionic acidaemia (PA) and ornithine transcarbamylase deficiency (OTC). The speakers are from the University College London.
Continuing advances in cell and gene therapies are transforming how we treat and potentially cure certain diseases and dramatically changing healthcare outcomes. Over the next 10 years, gene therapies are expected to come into their own as a treatment option for a variety of diseases.