Medical writing

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

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

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

FDA Draft Guidance - An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients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as an emerging and promising treatment for rare diseases, the US Food and Drug Administration (FDA) has released a draft guidance for their development.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

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

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

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)

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

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

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

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

Reducing Noise in Hospitals Can Improve the Wellbeing of Patients and Staff

It's well established that a quiet environment promotes healing. Yet, the noise levels of the places built for healing and recovery in the United States have been steadily increasing since the 1960s and are more than double the acceptable level (1). From the beeps and whirs of machines to conversations among the staff, noise is one of the main disruptions to patients in health care settings (2). Unwanted sounds are more than a little annoyance; studies show elevated noise can affect sleep, injur

Can Coronary Artery Disease and Carotid Stenosis Be Reversed or Prevented?

The arteries play an essential role in the vascular system; they transport oxygen and nutrients to the body's tissues. However, like any part of the body, they're susceptible to damage and disease. Coronary artery disease and carotid stenosis are two arterial diseases that, if left untreated, can lead to serious complications, including heart attack and stroke. Lifestyle habits and genetics both influence the health of our arteries, and at TCIM, our functional approach aims to find the root caus