Exa-cel: A Potential Breakthrough with Astounding Results
A new therapy that uses the Nobel Prize-winning CRISPR technology may be the latest breakthrough in gene editing therapy. Exagamglogene autotemcel (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, addresses two debilitating blood disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). If approved, it will mark the world’s first therapy based on CRISPR technology and could transform the lives ...
Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases
There are many different respiratory viruses, from influenza and respiratory syncytial virus (RSV) to coronaviruses, parainfluenza viruses, rhinoviruses, and adenoviruses. Some are highly pathogenic and can trigger respiratory failure and death. These viruses also facilitate secondary bacterial infections, often resulting in pneumonia, meningitis, and other acute inflammatory pathologies (1). These acute viral respiratory tract infections (AVRIs) are among the top ...
Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics
Martin Egli and Muthiah Manoharan recently co-wrote an outstanding review that is a great resource for anyone in our community. The review, titled “Chemistry, structure and function of approved oligonucleotide therapeutics,” is a collection of information about the eighteen nucleic acid therapeutics that have been approved in the last 25 years. Readers will gain knowledge from their insights and ...
Amazon Prime for DNA – Has A New Era of Oligonucleotide Synthesis Begun?
Phosphoramidite chemistry has been used for decades to write synthetic DNA, but challenges arise when longer, more complex oligos are needed. After years of innovation fueled by the goal of meeting this need, Ansa Biotechnologies announced de novo synthesis of a 1005 base long oligonucleotide with industry-leading accuracy. Not only that, but they are also beginning an early access program ...
An ASO Is Granted Accelerated Approval By the FDA
Tofersen, an antisense oligonucleotide treatment for SOD1-ALS, a rare form of amyotrophic lateral sclerosis (ALS), was recently granted accelerated approval by the U.S. FDA. This is quite significant, as approval was granted based on a biomarker rather than clear clinical improvement of the disease. This flexible approach to granting approval may have far-reaching implications for the treatment of many ...
Innovation, Versatile Gene Editing Technologies, and Patent Battles
Last June, at a genome editing summit in Lisbon, following a much-anticipated presentation by Tessera Therapeutics about their new gene editing technology, an audience member asked a question that is now the basis of a patent dispute: how was it different from the prime editing technology created by Harvard and Broad Institute biochemist David Liu? Gene editing has become ...
Exa-cel: A Potential Breakthrough with Astounding Results
A new therapy that uses the Nobel Prize-winning CRISPR technology may be the latest breakthrough in ...
Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases
There are many different respiratory viruses, from influenza and respiratory syncytial virus (RSV) to coronaviruses, parainfluenza ...
Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics
Martin Egli and Muthiah Manoharan recently co-wrote an outstanding review that is a great resource ...
Amazon Prime for DNA – Has A New Era of Oligonucleotide Synthesis Begun?
Phosphoramidite chemistry has been used for decades to write synthetic DNA, but challenges arise when longer, ...
An ASO Is Granted Accelerated Approval By the FDA
Tofersen, an antisense oligonucleotide treatment for SOD1-ALS, a rare form of amyotrophic lateral sclerosis (ALS), ...
Innovation, Versatile Gene Editing Technologies, and Patent Battles
Last June, at a genome editing summit in Lisbon, following a much-anticipated presentation by Tessera ...
