Recent Articles2022-03-14T07:39:44+00:00
27June, 2023

Exa-cel: A Potential Breakthrough with Astounding Results

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

13June, 2023

Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

25May, 2023

Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

15May, 2023

Amazon Prime for DNA – Has A New Era of Oligonucleotide Synthesis Begun?

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

3May, 2023

An ASO Is Granted Accelerated Approval By the FDA

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

24April, 2023

Innovation, Versatile Gene Editing Technologies, and Patent Battles

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

5April, 2023

Can There Be International Agreement on How to Navigate the Future of Genome Editing?

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

Our world is changing at an astonishingly rapid pace and, as described a few weeks ago, CRISPR is poised to become a major source of change in many fields, from medicine to animals to crops. With any new technology, but especially one that has the power to transform the ...

15March, 2023

10 Years of CRISPR – Developing a Previously Unimaginable Idea to Benefit the World

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

CRISPR is now a common term, one that you see even in mainstream media. Yet it is incredible to think that its potential for editing genes was published just ten years ago. In a mere decade, the groundbreaking discovery has been successfully developed to the point that many clinical ...

2March, 2023

The Journey of C. Frank Bennett: Caring and Curiosity Drive the Development of Antisense Therapies

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

The chief scientific officer of Ionis Pharmaceuticals, Dr. C. Frank Bennett, is not someone who comes to work just to do science, but to help his patients. Although he started with little knowledge of antisense drugs when he joined the company more than thirty years ago, his patients have ...

13February, 2023

RSV: Developing Vaccines to Protect the Vulnerable

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

It’s the middle of winter and, at some point during this season, many people will experience a constellation of symptoms that may include coughing, a sore throat, runny nose, headache, body aches, fevers, and fatigue. Most adults would assume they have a cold or the flu, but now, depending ...

Delivering Oligonucleotide Drugs to the Lungs in a World of Influenza, Coronaviruses, Asthma, and Chronic Lung Diseases

June 13th, 2023|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

Martin Egli and Muthiah Manoharan’s Review of Approved Oligonucleotide Therapeutics

May 25th, 2023|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

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 ...

The Journey of C. Frank Bennett: Caring and Curiosity Drive the Development of Antisense Therapies

March 2nd, 2023|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

The chief scientific officer of Ionis Pharmaceuticals, Dr. C. Frank Bennett, is not someone who comes to work just to do science, but to help his patients. Although he ...

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