Verve Therapeutics, a biotech based in Cambridge, MA, has progressed its second base edited gene therapy, aimed at significantly lowering low-density lipoprotein (LDL) cholesterol, to the clinic in a Phase I trial. The company is using base editing, a more precise gene editing technique than CRISPR-Cas9, to create novel therapies for genetic cardiovascular conditions. Verve 102, which is being tested in the Phase I study, is designed to inactivate the PCSK9 gene in the liver to significantly reduce levels of LDL cholesterol in people with heterozygous familial hypercholesterolemia or premature ..read more

Broken String Biosciences and the Francis Crick Institute are partnering to develop novel applications for the start-up’s proprietary DNA break-mapping platform, INDUCE-seq, to investigate the impact of genomic instability in the development of amyotrophic lateral sclerosis (ALS). The partnership will examine DNA break-mapping technology and advance understanding of genomic instability in the development of ALS. Felix Dobbs, PhD, CEO, Broken String Biosciences, said, “This collaboration with the Crick Institute is validation of our differentiated approach to DNA break-mapping; enabling our tea ..read more

A study by researchers at Mass General Brigham has revealed that patient adverse events are prevalent and persistent in outpatient settings including visits to primary care physicians, specialty care, day surgeries, and other settings. The research analyzed patient care in 2018 at four Boston-area healthcare systems and provides insights on the most common forms of adverse healthcare events and the populations most likely to be affected. The report, published in The Annals of Internal Medicine, also suggests interventions that can improve patient safety in these care settings. “Our study is an ..read more

Female mammals are more susceptible to autoimmune diseases because of how their X-chromosome is inactivated, new research from France suggests. Scientists “turned back on” the inactive X gene in a mouse model, which led to signs of autoimmune disease. It’s likely the findings will relate to people as well. The study was published in Science Advances and the lead author is Céline Morey, PhD, Université Paris Cité, CNRS. “There are several indications that the same kind of regulation also occurs in humans. The X-chromosome inactivation process is conserved amongst all female eutherian mammals ..read more

Screening for preeclampsia using a combination of maternal history, ultrasound screening, and a panel of blood biomarkers could help improve early detection rates of the condition, which could improve both maternal and infant health in those affected. Writing in the journal Hypertension, lead study author Emmanuel Bujold, MD, professor in the department of obstetrics and gynecology at the Université Laval in Canada, and colleagues report that their screening test was more accurate at detecting both preterm and early onset preeclampsia than currently recommended methods, which focus on maternal ..read more

New research, led by University of Cambridge investigators, shows they have developed a new vaccine technology that provides a broad range of protection against coronaviruses, even those that don’t yet exist. This approach to vaccine development is called “proactive vaccinology,” which seeks to build disease-fighting immunity before some pathogens emerge. Their approach, reported in Nature Nanotechnology, trains the immune system to recognize the specific regions of eight coronaviruses including SARS-CoV-1, SARS-CoV-2, and several others that are currently circulating in bats and have the pote ..read more

Colonoscopy screening intervals could be extended for people without a family history of colorectal cancer (CRC), research suggests, allowing them to avoid unnecessary invasive examinations. The decade-long interval between screenings could potentially be extended to 15 years for those whose first colonoscopy is negative for the cancer, without resulting in major adverse consequences. The study, published in JAMA Oncology, adds to an evolving body of evidence that supports extending the historical 10-year screening interval for individuals at average CRC risk. ‘This study provides evidence for ..read more

Researchers at the Medical College of Georgia (MCG) have found that targeting a specific receptor in the eye may benefit many patients with neovascular age-related macular degeneration, who don’t respond well to current treatments.  Age-related macular degeneration is a leading cause of blindness worldwide and occurs mostly in the elderly and people with metabolic diseases, such as diabetes and obesity. It is characterized by increased blood vessel growth in the eye, damaging the macula at the back of the eye, which is essential for translating light into image signals.  Current trea ..read more

Researchers at the University of California, Los Angeles (UCLA) have discovered that a known network of inflammatory molecules could be used as biomarkers to calculate a person’s risk of developing cerebral small vessel disease, a common cause of stroke and cognitive decline.  In current clinical practice, the only way to understand a person’s susceptibility to developing cerebral small vessel disease is a combination of medical imaging techniques, family history, and assessing other risk factors. Moreover, physicians may only discover a person is at risk of having a stroke after they hav ..read more

Artificial intelligence can filter through electronic health records (EHRs) to identify patients with rare, undiagnosed diseases, research suggests. A machine-learning algorithm was able to spot more than half of individuals who went on to be diagnosed with the inherited disease of common variable immunodeficiencies (CVID). The group of immune disorders leaves patients susceptible to infections and autoimmunity and is characterized by antibody deficiency and impaired B cell responses. The findings are published in the journal Science Translational Medicine. “Immune deficiency patients elude di ..read more