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Oligonucleotide Therapeutics Society •

Targeting the Kidney: The Promise of RNA-Based Therapeutics - Oligonucleotide Therapeutics Society

When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, she received her first kidney transplant. Gutierrez was born with polycystic kidney disease (PKD), a genetic condition that makes fluid-filled cysts grow on the kidney and can lead to kidney failure.
Growing up, Gutierrez had to stop attending school due to her frequent doctor visits and three-times-a-week dialysis treatments, which each lasted three hours. While the transplant was initially successful, within...
Oligonucleotide Therapeutics Society •

2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases - Oligonucleotide Therapeutics Society

In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). The approvals — the second largest in 30 years — include an oligonucleotide therapy for blood cancer, an antisense oligonucleotide shown to significantly drop triglyceride levels, and an mRNA vaccine targeting the respiratory syncytial virus.
 
IMETELSTAT: a first-in-class telomerase inhibitor
On June 6, 2024, Imetelstat (RYTELO) was approved...
Oligonucleotide Therapeutics Society •

Transporting Therapeutics: A Novel Approach to Deliver ASOs to the Brain - Oligonucleotide Therapeutics Society

Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not without its risks or drawbacks (1). However, a recent study by Denali Therapeutics, as well as other scientific research in the field, offers promising techniques to ov...
Oligonucleotide Therapeutics Society •

Oligonucleotide Treatment Advances Offer Relief for Patients with Familial Chylomicronemia Syndrome - Oligonucleotide Therapeutics Society

At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn’t stop crying and wasn’t eating. Concerningly, her triglyceride levels were in the 20,000’s, far over the normal 150 level. Darlene was referred to a lipid specialist at John Hopkins University Hospital and, after being genetically tested, was diagnosed with familial chylomicronemia syndrome (FCS), a rare, highly debilitating, and life-threatening disease.
FCS is a gen...
Oligonucleotide Therapeutics Society •

The Man Behind the Molecules: John Maraganore's RNAi Legacy - Oligonucleotide Therapeutics Society

The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role in making RNA interference (RNAi) a successful therapeutic modality while CEO at Alnylam Pharmaceuticals. However, before joining and after leaving Alnylam, he was and continues to be influential in the biotechnology sphere, with his leadership skills, optimism, and willingness to take risks still changing the RNAi landscape.
Biogen and Millennium Pharmaceuticals: drug developments and important acqu...
Oligonucleotide Therapeutics Society •

Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials - Oligonucleotide Therapeutics Society

A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as Angelman syndrome (AS) has achieved a favorable safety profile and lessening of overall symptoms in trial participants.
In a recent press release, Ionis announced the detailed HALOS study results for the multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582. Consistent and encouraging results were demonstrated on measures assessing all functional domains, including communicatio...
Oligonucleotide Therapeutics Society •

Encouraging Progress in the Hunt for a Huntington's Treatment - Oligonucleotide Therapeutics Society

Described as a combination of Alzheimer’s disease, Amyotrophic lateral sclerosis, and Parkinson’s disease all at once, Huntington’s disease is a rare and debilitating neurological disorder passed down within affected families. With a 50% chance of a child inheriting it from a parent with the HD mutation, the fatal disease often leaves a devastating family legacy.
In the United States alone, there are approximately 40,000 people with symptomatic Huntington’s disease, and more than 200,000 are at...
Oligonucleotide Therapeutics Society •

ALS: From Genetic Complexity to Treatment Challenges and Advances - Oligonucleotide Therapeutics Society

Amyotrophic Lateral Sclerosis (ALS), commonly called Lou Gehrig’s disease, is a neurodegenerative disorder of the motor neurons that causes progressive muscle weakness and respiratory failure (1). ALS is the most common motor disease among adults (2), affecting around 60,000 people in the U.S. and Europe. Life expectancy for those with the fatal disease is typically 2-5 years after diagnosis (1), and onset is usually in late middle life (3). While 90-95% of ALS cases are sporadic, meaning there’...
Oligonucleotide Therapeutics Society •

Inherited Retinal Disorder: In Vivo CRISPR Therapy Provides Vision Improvements - Oligonucleotide Therapeutics Society

Inherited retinal disorders (IRDs) are the leading cause of visual impairment, affecting approximately 1 in 2,000 people of all ages worldwide (1). There are many types of IRDs, each caused by a gene variant that affects how the retina functions, with one of the most severe being Leber Congenital Amaurosis (LCA). The early-onset condition is the leading cause of inherited blindness in children and occurs in about three out of 100,000 newborns.
LCA is often caused by a mutation in the CEP290 gene...
Oligonucleotide Therapeutics Society •

Angelman Syndrome Treatment Efforts and a Novel Prenatal Strategy - Oligonucleotide Therapeutics Society

Imagine finding out your child may never walk, talk, feed, or dress themselves. Never have a job, get married, or have children. Never live independently. This long list of milestones never to be met is the experience for many parents with children diagnosed with Angelman syndrome (AS), a rare, early-onset neurodevelopmental disorder affecting approximately one in 12,000 to 20,000 people globally.
The Angelman syndrome story of Colin, who was born long, lean, and with a headful of blond hair, st...
Oligonucleotide Therapeutics Society •

Treating Genetic Causes of Dravet Syndrome - Oligonucleotide Therapeutics Society

In September 2014, identical twins Hudson Blake and Grady Lee were born and appeared seemingly perfect. However, when their mom went to return to work when the boys were 12 weeks old, she started to notice they both had an odd shiver. When Hudson began shivering again one day, his mom took him to the ER, where he started having another episode, and it was confirmed as a seizure. One week later, Grady had a similar seizure and was admitted to the ER. The family says the following year was a rolle...
Oligonucleotide Therapeutics Society •

Hope of New Treatments for Chronic Liver Disease MASH (NASH) - Oligonucleotide Therapeutics Society

In the early stages of Metabolic Dysfunction-Associated Steatohepatitis (MASH), many patients are asymptomatic, though some may feel weak or fatigued or have an ache in their upper right abdomen. While lifestyle changes can help patients diagnosed early enough, more advanced stages of the disease have fewer options. However, recent successful clinical trials are providing hope that this may change.
The Growing Prevalence of Metabolic Dysfunction-Associated Steatohepatitis (MASH)
Hepatic Steatosi...
Oligonucleotide Therapeutics Society •

RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials - Oligonucleotide Therapeutics Society

When Peggy’s mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be diagnosed with Alpha-1 Antitrypsin Deficiency (AATD). Knowing it was a genetic condition, the doctors tested Peggy and her brother for it. Peggy, 21 years old and an avid runner with complete lung function, had the full deficiency of the disease. Int...
Oligonucleotide Therapeutics Society •

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 •

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 •

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 •

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 •

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 •

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
Oligonucleotide Therapeutics Society •

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
Oligonucleotide Therapeutics Society •

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 •

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 •

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
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