Non-Profits Create Next-Generation Medicines that Treat Ultra-Rare Diseases
Hundreds of millions of people worldwide are living with a rare disease. Most rare diseases do not currently have a cure. So, once diagnosed with a rare disease, most patients and their families are merely given strategies to manage the disease symptoms and try to minimize the impact on their lives. Sadly, those who receive a diagnosis are the ...
Oligonucleotides Safely Cross the Blood-Brain Barrier to Knock Down Gene Expression
Neurodegenerative diseases, brain tumors, and infectious diseases and inflammatory conditions that impact the brain wreak havoc on a person’s health and are notoriously difficult to treat. Oligonucleotide drugs could provide solutions to many pathological brain conditions if certain challenges could be overcome. Researchers at Tokyo Medical and Dental University, Takeda Pharmaceutical, and Ionis Pharmaceuticals may have found a solution. ...
Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics
Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these problems and there are multiple solutions that have proven to work in some capacity. In a previous article, we delved ...
OTS President’s Paper Pick – August 2021
By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian D Gillmore, Ed Gane, Jorg Taube, Justin Kao, Marianna Fontana, Michael L Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, Adam P Boyd, Jeffrey E Cehelsky, Mark D McKee, Andrew Schiermeier, Olivier Harari, Andrew Murphy, Christos ...
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines and the recent news of phenomenal interim results for Intellia’s investigational genome editing treatment for ATTR amyloidosis. Neither of these would have been possible without a lesser-known component – lipid nanoparticles. This delivery system has been decades in the making and, fortunately for humanity, ...
A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared interim data from the first ever human study using systemically administered CRISPR as a gene editing therapy. Early results that surpassed expectations show that not only is the CRISPR-based therapy to treat ATTR amyloidosis safe, it also precisely edited the desired gene in the ...
Non-Profits Create Next-Generation Medicines that Treat Ultra-Rare Diseases
Hundreds of millions of people worldwide are living with a rare disease. Most rare diseases ...
Oligonucleotides Safely Cross the Blood-Brain Barrier to Knock Down Gene Expression
Neurodegenerative diseases, brain tumors, and infectious diseases and inflammatory conditions that impact the brain wreak ...
Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics
Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many ...
OTS President’s Paper Pick – August 2021
By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian ...
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines ...
A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared ...
