High cholesterol can quietly damage arteries for years, raising the risk of heart disease and other cardiovascular problems. Now, researchers from the University of Barcelona and the University of Oregon have developed an experimental strategy that could offer a new…
BioPharm International FAQ: What FDA’s Individualized Gene Editing Draft Guidance Means for Developers FDA’s draft framework would let sponsors add patient-specific genome editing variants to an approved BLA using in vitro data. Here is what the guidance proposes, what evidence…
A new mathematical model comparing chemotherapy and CAR T-cell therapy for leukemia finds that simulated T-cell infusion produces a stronger and longer-lasting reduction in leukemia cells than chemotherapy alone. The chemotherapy simulations produced a nuanced picture. Subject of Research: Mathematical…
CRISPR Gene Therapy for Sickle Cell and Thalassemia Shows Strong Efficacy, Bioengineer.org
Bioengineer.org Subject of Research: In vivo prime editing as a gene-correction strategy for genetic diseases Article Title: Research progress of gene correction for genetic diseases via in vivo prime editing News Source: Juliet Wilcox. (November 5, 2026). Prime Editing Moves Toward…
"It costs us time, and in this disease, time is measured in transfusions," Vita said, describing setbacks in the company's manufacturing plans.¹ DBA is an inherited bone marrow failure disorder characterized by impaired red blood cell production. Some patients respond…
AllSci Philadelphia-based Verismo Therapeutics, a subsidiary of HLB Innovation, reported that SynKIR-310, a CD19-targeted KIR-CAR T therapy, produced an 80% objective response rate (ORR) in 4 of 5 evaluable patients and a 60% complete response rate (CRR) across the first two dose cohorts in…
Posted by cfelixcpa | Nov 9, 2025 | SF STAT! | 0 | One-time infusion of investigational CRISPR-Cas9 therapy found to safely reduce LDL cholesterol by 50% and triglycerides by about 55% in Phase 1 trial November 8, 2025 – Results from a Cleveland Clinic Phase 1, first-in-human clinical trial showed that a one-time…
So, for many patients, the best option is automated red blood cell exchange (aRBCX), a procedure where patients have their red blood cells removed by apheresis and replaced with donor cells. (Sickle cell disease causes misshapen red blood cells, which…
Marco Davila, Senior Vice President, Translational Research at Roswell Park Comprehensive Cancer Center, shared Journal for ImmunoTherapy of Cancer’s post on LinkedIn, adding: “Thank you, Journal for ImmunoTherapy of Cancer, for highlighting our work! Proud of our team and grateful…
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