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Cleveland Clinic Trial Shows Gene-Editing Therapy Dramatically Lowers Dangerous Blood Fat Levels

Cleveland Clinic Trial Shows Gene-Editing Therapy Dramatically Lowers Dangerous Blood Fat Levels

A Phase 1b clinical trial conducted by Cleveland Clinic has demonstrated that a single-dose CRISPR-Cas9 gene-editing therapy, CTX310, significantly reduced triglyceride levels in patients suffering from severe hypertriglyceridemia. The findings were presented at the American Heart Association’s annual meeting in Chicago. The trial involved 10 adults with severe hypertriglyceridemia, a condition that elevates the risk of heart disease. Thirty days after receiving a single intravenous infusion of CTX310, participants experienced an average reduction of 63% in triglyceride levels, with some reductions reaching up to 85%. The therapy targets the ANGPTL3 gene, which is a key regulator of lipid metabolism. Beyond triglycerides, the treatment also lowered non-HDL cholesterol, apolipoprotein B, and triglyceride-rich lipoprotein cholesterol levels. The treatment was generally well tolerated, with no serious side effects reported.

Merck Announces Positive Phase 2b Results for Efinopegdutide in MASH with Fibrosis

Merck Announces Positive Phase 2b Results for Efinopegdutide in MASH with Fibrosis

Merck Announces Positive Phase 2b Results for Efinopegdutide in MASH with Fibrosis

FDA Expands Approval for Inhaled Insulin Afrezza for Pediatric Type 1 Diabetes Patients

FDA Expands Approval for Inhaled Insulin Afrezza for Pediatric Type 1 Diabetes Patients

The FDA has expanded its approval for Afrezza, an inhaled insulin, for use in children aged six years and older with diabetes, including type 1 diabetes. This medication offers an alternative to injected mealtime insulin, delivering a dry powder through the lungs into the bloodstream for rapid absorption. The INHALE-1 clinical trial, which evaluated Technosphere insulin (Afrezza) against injected rapid-acting insulin analogs in 230 children and adolescents aged 4 to 17 years, showed mixed results regarding glucose control. While the primary analysis did not establish noninferiority for HbA1c outcomes, a sensitivity analysis did meet this standard. Participants using inhaled insulin reported significantly greater treatment satisfaction and experienced less weight gain. However, Afrezza carries an FDA boxed warning regarding acute bronchospasm in patients with chronic lung disease, making it unsuitable for children with asthma or other chronic lung conditions. Lung function must be evaluated before treatment...

UCLA-Led Study Develops Clinical-Scale CRISPR Gene Correction for Sickle Cell Disease

UCLA-Led Study Develops Clinical-Scale CRISPR Gene Correction for Sickle Cell Disease

A new study has demonstrated the successful development of a clinical-scale manufacturing process capable of directly correcting the mutation responsible for sickle cell disease (SCD) in patients’ blood-forming stem cells. The experimental therapy, named CRISPR_SCD001, utilizes CRISPR-Cas9 technology along with a short, single-stranded DNA template to repair the sickle mutation within the beta-globin gene (HBB). The process involves collecting CD34-positive cells from mobilized peripheral blood, culturing them, and then using electroporation to introduce the CRISPR-Cas9 complex and corrective DNA template. These edited autologous cells are then intended for reinfusion into the patient after conditioning. This approach differs from currently approved gene therapies for SCD, which either add an anti-sickling gene via a lentiviral vector or edit a regulatory region to restore fetal hemoglobin production. CRISPR_SCD001 aims to directly repair the disease-causing mutation without a viral donor template. The man...

FDA Approves New Cancer Therapies for Bladder and Rectal Cancers, Expands Keytruda Indication

FDA Approves New Cancer Therapies for Bladder and Rectal Cancers, Expands Keytruda Indication

The U.S. Food and Drug Administration (FDA) has recently approved several new cancer treatments and expanded indications for existing therapies. On October 14, 2026, the FDA approved nogapendekin alfa inbakicept-pmln (Anktiva) in combination with bacillus Calmette-Guérin (BCG) for adults with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC). This approval expands the prior indication to include patients with high-grade papillary-only tumors, based on the efficacy demonstrated in the QUILT-3.032 trial. Additionally, on October 16, 2026, the FDA granted traditional approval to pembrolizumab (Keytruda) or pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex) for adult and pediatric patients with unresectable or metastatic tumor mutational burden–high (TMB-H) solid tumors. This converts a previous accelerated approval and refines the TMB-H definition to ≥ 13 mutations/megabase. Furthermore, on October 19, 2026, the PD-1 inhibitor dostarlimab (Jemperli) received accelerated approval for adult...

Capricor Therapeutics' Deramiocel Cardiac Results for Duchenne Muscular Dystrophy Win American Heart Association Award

Capricor Therapeutics' Deramiocel Cardiac Results for Duchenne Muscular Dystrophy Win American Heart Association Award

Capricor Therapeutics announced that the cardiac results from its HOPE-3 Phase 3 trial of Deramiocel for Duchenne muscular dystrophy (DMD) received the Outstanding Research Award in Pediatric Cardiology at the American Heart Association (AHA) Scientific Sessions 2026. The presentation, led by Jonathan Soslow, M.D., MSCI, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center, included new cardiac MRI data on myocardial fibrosis and a comparison with untreated patients from a natural history dataset. The HOPE-3 trial randomized 106 DMD patients to Deramiocel or placebo over 12 months, meeting its primary endpoint of upper limb function. Key cardiac findings showed a +1.8 percentage point improvement in left ventricular ejection fraction (LVEF) for all patients treated with Deramiocel compared to placebo, and a more pronounced +2.8 percentage point improvement in LVEF for patients with cardiomyopathy. Additionally, treated patients showed slower progression of heart scarring, with 3.0 f...

Mirum Pharmaceuticals Achieves Primary Endpoint in AZURE-4 Phase 3 Study for Chronic Hepatitis Delta Virus Treatment

Mirum Pharmaceuticals Achieves Primary Endpoint in AZURE-4 Phase 3 Study for Chronic Hepatitis Delta Virus Treatment

Mirum Pharmaceuticals Achieves Primary Endpoint in AZURE-4 Phase 3 Study for Chronic Hepatitis Delta Virus Treatment

Study Reveals Most Eligible U.S. Teens Miss Out on Advanced Obesity Treatments

Study Reveals Most Eligible U.S. Teens Miss Out on Advanced Obesity Treatments

A new study from Ann & Robert H. Lurie Children's Hospital of Chicago, published in JAMA Pediatrics, indicates that the majority of eligible U.S. adolescents are not receiving advanced obesity treatments, despite the availability of new medications and evidence supporting weight-loss surgery. Researchers analyzed electronic health records of over 1.4 million U.S. adolescents who met the criteria for metabolic and bariatric surgery between 2017 and 2025. The study found a significant increase in the use of GLP-1 medications, such as semaglutide and liraglutide, during this period. Prescribing rates for GLP-1s surged from 301 prescriptions per 100,000 eligible adolescents in early 2017 to 10,676 per 100,000 by the end of 2025, a 35-fold increase. Conversely, bariatric surgery rates, which had steadily increased until 2022, saw a 29% decline by the end of 2025, coinciding with the FDA approval of semaglutide for adolescent obesity. Despite these developments, less than 1% of eligible adolescents underwent sur...

CRISPR Therapeutics Reports Positive Phase 1b Results for CTX310 in Severe Hypertriglyceridemia

CRISPR Therapeutics Reports Positive Phase 1b Results for CTX310 in Severe Hypertriglyceridemia

CRISPR Therapeutics has announced positive Phase 1b clinical trial results for its investigational gene-editing therapy, CTX310, designed to treat severe hypertriglyceridemia (sHTG). The data, presented at the American Heart Association (AHA) Scientific Sessions 2026, showed a mean 63% reduction in triglycerides (TG) and a 65% reduction in circulating angiopoietin-like protein 3 (ANGPTL3) at Day 30 following a single-course treatment. Participants in the trial had a median baseline triglyceride concentration exceeding 1,000 mg/dL, indicating significant lipid abnormalities. All ten patients in the Phase 1b cohort had diabetes, 30% had a history of pancreatitis, and 20% had existing atherosclerotic cardiovascular disease, and all had failed prior triglyceride-lowering therapies. The therapy works by permanently disabling the ANGPTL3 gene in liver cells using CRISPR-Cas9 technology, which prevents the production of functional ANGPTL3 protein. This allows lipoprotein lipase (LPL), an enzyme that breaks down t...

CRISPR Therapeutics Reports Positive Phase 1b Data for CTX310 in Severe Hypertriglyceridemia Patients

CRISPR Therapeutics Reports Positive Phase 1b Data for CTX310 in Severe Hypertriglyceridemia Patients

CRISPR Therapeutics has announced promising clinical data from its Phase 1b trial of CTX310, an investigational gene-editing therapy designed to treat severe hypertriglyceridemia (sHTG). The data, presented at the American Heart Association (AHA) Scientific Sessions 2026, showed that a single course of CTX310 led to significant reductions in key lipid markers. Participants with a median baseline triglyceride (TG) level above 1,000 mg/dL experienced a mean reduction of 65% in circulating angiopoietin-like protein 3 (ANGPTL3) and 63% in triglycerides at Day 30. Additionally, the therapy resulted in mean reductions of 51% in non-HDL cholesterol, 23% in apolipoprotein B, and 16% in LDL cholesterol. The trial, which included 10 participants, reported no treatment-related serious adverse events and no clinically relevant elevations in liver transaminases, indicating a favorable safety profile. The U.S. Food and Drug Administration (FDA) has already granted Regenerative Medicine Advanced Therapy (RMAT) designatio...

Cleveland Clinic Study Reveals Gene-Silencing Therapy's Dual Impact on High Cholesterol and Triglycerides

Cleveland Clinic Study Reveals Gene-Silencing Therapy's Dual Impact on High Cholesterol and Triglycerides

A new study led by Cleveland Clinic has demonstrated that a single dose of ARO-DIMER-PA, a gene-silencing medication, significantly reduced both 'bad' cholesterol (LDL-C) and triglycerides in patients diagnosed with mixed hyperlipidemia. The findings were presented at the American Heart Association’s Scientific Sessions 2026 in Chicago. Dr. Luke Laffin, study author and medical director of the Cleveland Clinic Coordinating Center for Clinical Research, highlighted that this is the first time a single drug has successfully blocked two separate disease pathways in the liver simultaneously. The medication, a small interfering RNA (siRNA), targets and silences both APOC3 and PCSK9 genes, which are involved in lipid metabolism. While other siRNA medications typically target only one gene, ARO-DIMER-PA's dual action led to dose-dependent reductions in PCSK9 and APOC3 proteins by 50-89% and 43-95% respectively. Consequently, LDL-C was reduced by 26-65% and triglycerides by 56-78%. The drug was generally well-tole...

American Heart Association Study Links Energy Drinks to Increased Cardiovascular Events in Young Adults

American Heart Association Study Links Energy Drinks to Increased Cardiovascular Events in Young Adults

New research from the American Heart Association (AHA), set to be presented at the AHA Scientific Sessions in Chicago, indicates a potential link between energy drinks and stimulant-containing supplements and serious heart problems in young adults. A retrospective observational analysis of over 28,000 FDA reports on cardiovascular events revealed that cardiac arrest was reported approximately 11 times more often in cases involving caffeine and stimulant products compared to those without. Heart attacks were reported 4.5 times more frequently, and abnormal heart rhythms or palpitations about four times more often. Elevated heart rate, chest pain, and fainting were also more common. Approximately 45% of these reported cardiovascular events associated with stimulant products occurred in adults aged 18 to 34. The stimulants examined included caffeine, methylxanthines, guarana, yerba mate, synephrine, and yohimbine, found in various products like soft drinks, meal replacement shakes, and vitamins. Dr. Pamela Be...