University of Pennsylvania

Gene therapy Beqvez (fidanacogene elaparvovec) significantly reduces bleeding episodes in hemophilia B patients by 71% after a single infusion, with over half experiencing no bleeds, according to a clinical trial published in the New England Journal of Medicine. The therapy, approved by the FDA in April 2024, uses a virus to deliver a working factor IX gene to the liver, enabling continuous production of the clotting factor. Patients report a 'hemophilia-free state of mind' post-treatment.

Beqvez gene therapy significantly reduces bleeding episodes in hemophilia B patients, with 71% average drop and 50% bleed-free outcomes post-infusion, per NEJM study. FDA approved in April 2024, offering a single-dose alternative to regular factor IX infusions.

Gene therapy Beqvez significantly reduces bleeding episodes in hemophilia B patients by 71% post-infusion, with over half experiencing no bleeds. The therapy, approved in April 2024, delivers a working factor IX gene to the liver via a virus, potentially offering a single-dose, life-changing treatment.

Analysis of WHO ADR data shows suicidality and self-harm with semaglutide, but not in clinical trials or a large cohort study. Both studies excluded patients with mental health history, leaving unanswered if GLP-1 receptor agonists worsen symptoms in such patients. Rapid weight loss due to semaglutide may exacerbate mental distress in vulnerable patients, necessitating continued vigilance and further research.

Nomic and PICI collaborate to profile ~3000 blood samples from RADIOHEAD study using nELISA™ to understand immunotherapy responses, aiming to improve patient care through precision medicine.

Chiesa Fuxench discusses the lack of research on psoriasis in Hispanic populations, emphasizing the need for diverse clinical trial populations and culturally relevant care. She highlights the importance of including diverse skin tones in clinical trials and awareness campaigns, noting the potential impact on treatment selection and patient quality of life. Chiesa Fuxench calls for greater diversity in dermatology to better understand genetic risk factors and improve care for all patients.

Climate change could triple U.S. heat deaths by mid-century, disproportionately affecting poor and minority populations. Deaths from extreme cold may decrease slightly, but heatwaves will more than offset this. Projections show a doubling to tripling of extreme temperature-related deaths by mid-century, depending on carbon emissions scenarios. Efforts to reduce greenhouse gas emissions and mitigate extreme temperature impacts are crucial.

Carisma Therapeutics, founded in 2016, pivoted from CAR-M to CAR-Monocyte therapy, focusing on CT-0525, which received FDA Fast Track designation. The company, now public, expanded its collaboration with Moderna into autoimmune diseases, securing funding into Q3 2025. CT-0525 aims to address solid tumors more effectively than previous CAR-M therapies.

EDG-7500, an oral cardiac sarcomere modulator, showed robust LVOT gradient reductions in obstructive HCM patients without affecting LVEF, according to Phase 1 and CIRRUS-HCM trial data. Edgewise Therapeutics initiated the 28-day CIRRUS-HCM trial for both obstructive and non-obstructive HCM patients, with initial 28-day data expected in Q1 2025.

Kesimpta (ofatumumab) demonstrated efficacy in reducing disability progression and disease activity in relapsing multiple sclerosis (RMS) patients, according to ALITHIOS and OLIKOS trials. First-line treatment with Kesimpta for up to six years produced less disability and disease progression in recently diagnosed (≤3 years) and treatment-naïve RMS patients compared to those switching from Aubagio. Continuous use of Kesimpta led to fewer disability worsening events and progression independent of relapse activity events, particularly in treatment-naïve patients. The OLIKOS trial found no new gadolinium-enhancing T1 lesions at 12 months in clinically stable RMS patients switching from IV anti-CD20 therapy to Kesimpta.