Oligonucleotide Therapeutics Society • 3rd April 2024

Jennifer Doudna - Seeking to Improve the World with CRISPR

At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had been in an all-day meeting; feeling exhausted and not thinking too much about the imminent Nobel Prize announcements, she turned off her phone ringer and went to bed. Still waking from her deep sleep, she assumed Ledford was calling to ask her to comment on so

Oligonucleotide Therapeutics Society • 21st March 2024

FDA Approved the First CRISPR Treatment - Potential Cure for People with Sickle Cell Disease

Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like changing weather, would put her in the hospital. Every four to six weeks, she would go to the hospital to have a catheter pull out four to five units of her blood and receive replacement blood to keep her healthy. Then, in 2019, w

Oligonucleotide Therapeutics Society • 21st February 2024

Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol

Cardiovascular disease is the leading cause of death in the United States, exceeding all types of cancer, unintentional injury, and stroke combined. However, updated data from Novartis’s open-label trial shows promising long-term results for inclisiran (Leqvio), the first and only small interfering RNA (siRNA) therapy to reduce low-density lipoprotein cholesterol (LDL-C).

ORION-8 is part of an extensive clinical trial program known as VictORION, which was co-created with international healthcar

Oligonucleotide Therapeutics Society • 9th February 2024

The First FDA Approval for a GalNAc-conjugated ASO

It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has become easily irritated, or you’ve started to lose weight or develop heart problems. Hereditary ATTR amyloidosis can present differently among patients and with symptoms found in far more common diseases, so it is often misdiagnosed. Fortunately, treatment for the disease is expanding, with the most recent therapy offering an at-home solution.

On Dec. 21, 2023, AstraZeneca and Ionis Pharmaceuticals

Oligonucleotide Therapeutics Society • 23rd January 2024

Base Editing in Clinical Trials to Treat Acute Lymphoblastic Leukemia

Base editing has experienced a rapid rise in use since it first came on the scene in 2016, with multiple trials underway, testing its ability to treat conditions with precise, single-letter changes to DNA. The technique presents a potentially more accurate and safer method compared to nuclease-based approaches; however, it’s not without its risks. Currently, researchers are investigating how base editing can be used to treat an aggressive form of leukemia, which leaves its victims with a long-te

Oligonucleotide Therapeutics Society • 5th September 2023

Fanzor: A Programmable RNA-Guided System In Eukaryotes Similar to CRISPR

Researchers have uncovered the first programmable RNA-guided system in eukaryotes that could be even more precise than CRISPR gene-editing. The discovery — led by Feng Zhang at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard — demonstrates that DNA-modifying proteins exist across all kingdoms of life (1).

In a recently published study in the scientific journal Nature, the team explains how the system is based on a protein called Fanzor, which uses RNA

28th August 2023

RNAi: The Persistence of a Few Leads to Success

Oligonucleotide Therapeutics Society • 27th June 2023

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 of people suffering from these blood diseases.

Both

Oligonucleotide Therapeutics Society • 24th April 2023

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 one of the most game-changing scientific technologies of the 21st century. The ability to precisely modify genes in living o

Oligonucleotide Therapeutics Society • 2nd March 2023

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

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 remained his greatest source of inspiration in creating life-changing therapies. With more than a dozen antisense drugs now on the market, his caring and curious mindset has led to great success.

Frank Bennett

Oligonucleotide Therapeutics Society • 31st January 2023

Inclisiran’s Interesting Path to Treating LDL Cholesterol

When The Medicines Company licensed inclisiran, they envisioned a drug for the masses that would save the healthcare system money and be reasonably priced for patients. Despite the company having little experience in the oligonucleotide therapeutics field, it went all in on the drug, selling off its other products and downsizing its workforce. It was a gamble, but if successful, it could revolutionize the treatment of heart disease. So, they trusted the science and the story of bringing the drug

Oligonucleotide Therapeutics Society • 19th January 2023

Alnylam’s 20-Year Journey of Discovery

Twenty years ago, a group of scientists intent on developing a new class of medicine using RNAi technology came together and formed Alnylam Pharmaceuticals, a name symbolizing their passion for discovery. The bright center star of Orion’s belt, Alnilam, has long been used as a celestial navigator, guiding explorers in their mission to discover the world. Like many explorers, the path for Alnylam was not always clear, and they appeared lost at times. Despite a constant wave of external disbelief

14th November 2022

Centyrins Effectively and Safely Deliver siRNAs to Multiple Muscle Tissues

Oligonucleotide Therapeutics Society • 8th September 2022

The Personal Journey of Stanley Crooke, the Creation and Development of Ionis, and Beyond

More than 30 years ago, Dr. Stanley Crooke founded Ionis Pharmaceuticals — a biotech company that helped pioneer the now successful technology of antisense oligonucleotides (ASO) despite overwhelming challenges and widespread disbelief.

After stepping down last year as the company’s CEO, Crooke recently published a perspective of his journey in creating Ionis. But to understand what drove Crooke to doggedly pursue his mission of creating one of the top antisense drug companies, it’s helpful to

Oligonucleotide Therapeutics Society • 27th August 2022

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 time stem cell gene therapy has treated this specific form of the disease.

SCID is a rare congenital disorder in which a genetic error in the blood stem cells causes newborns to have a non-functioning immune system, leavi

Oligonucleotide Therapeutics Society • 16th August 2022

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 molecule that causes arteries to accumulate plaque and harden over time. The single-course treatment, called VERVE-101, uses base editing to turn off the PCSK9 gene in the liver to decrease LDL cholesterol

Oligonucleotide Therapeutics Society • 26th July 2022

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 rare diseases, the US Food and Drug Administration (FDA) has released a draft guidance for their development.

Although the mission of the FDA is to protect public he

TCIM • 13th June 2022

Why Is It So Difficult to Get Quality Sleep?

We all want to get a good night's sleep and wake up feeling refreshed. But, too often, it seems we're left staring at our ceilings at 3 am, wondering why our minds won't shut off, or we will get a full night's sleep but wake up still feeling exhausted. Several things could be causing these terrible periods of sleep; addressing the underlying cause and adopting positive sleep hygiene habits can help.

What Interferes with Getting a Night of Good Sleep?

We recently discussed the complex relations

Oligonucleotide Therapeutics Society • 6th June 2022

CRISPR Babies and the Scientist Behind Their Editing

At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had used CRISPR-Cas9, a genetic engineering tool, to edit the CCR5 gene of three embryos with the goal of making them resistant to HIV (1). What followed the breaking story was not scientific praise but shock and horror.

He Jiankui’s pariah status in the scientific community is almost certainly not the

Oligonucleotide Therapeutics Society • 31st May 2022

CRISPR Patent Rights and Their Effect on the Industry

A decade ago, Jennifer Doudna of the University of California, Berkley and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology drafted blueprints for a groundbreaking gene editing technique. The two scientists had found a way to cut and make precise changes to DNA — a discovery that won them a Nobel Prize. This was the start of CRISPR, a technology that biotech companies now use to develop treatments for genetic conditions like sickle cell disease and inherited blindness (1)

TCIM • 17th May 2022

How Sleep and Stress Are Intertwined

We've all been there, a night where you cannot fall asleep — the stress of a presentation or life events endlessly looping through your mind, keeping you awake. Not only does poor sleep exacerbate stress, but it also affects our physiological health and quality of life. From academic performance to recovering from (or preventing) COVID-19, sleep is essential for a healthy, happy life.

The Anatomy and Physiology of Sleep

There are two types of sleep humans alternate between during the sleep cyc

Oligonucleotide Therapeutics Society • 22nd April 2022

A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD

Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body’s muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty and fibrotic, typically leading to heart failure and death by age 30. There is no cure, and the approved drugs each help a small portion of patients and cannot target the heart muscle. However, a new treatment could help significantly more people with DMD and its associated heart issues.

Muscular dystr

TCIM • 5th April 2022

Are Your Uncomfortable Gut Symptoms Due to SIBO?

Small intestinal bacteria overgrowth (SIBO) is a common but under-diagnosed condition. Historically viewed as a controversial diagnosis due to its wide-ranging clinical presentations and overlap with other conditions, it has recently gained support as a viable explanation for gastrointestinal problems (1). Although antibiotics remain the most common treatment method for SIBO, functional medicine's holistic approach of addressing the root cause and restoring balance to the gastrointestinal tract

Oligonucleotide Therapeutics Society • 28th February 2022

A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy

Typically, a single copy of a gene is enough to support normal growth and development but in a small subset of genes, the loss of one copy can cause serious diseases that are often difficult to treat. One of these conditions, Dravet Syndrome, has no treatment options that directly target the cause of the disease. However, new research from Stoke Therapeutics Inc. may have found a solution that changes how these disorders are targeted and treated.

The study results of Stoke Therapeutics Inc.’s n