University of Pennsylvania

Gene therapy trials for hereditary blindness show significant improvement in night vision for patients with Leber congenital amaurosis 1 (LCA1) and RLBP-1 associated retinal dystrophy, with plans for future Phase III trials.

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.

The Inflation Reduction Act (IRA), effective 2026, aims to reduce Medicare spending by $6 billion in 2026, sparking debate over its impact on drug innovation and availability. It targets high-spending Medicare drugs, potentially reducing small molecule development and affecting therapeutic areas like oncology and endocrinology. Industry reactions are mixed, with some companies adjusting strategies in response. The IRA's long-term effects on innovation and patient access remain uncertain.