CRISPR Medicine News launched its Global Clinical Trials Database 2.0, a manually curated resource covering over 300 gene-editing clinical trials. The database includes ex vivo and in vivo therapies, LTFU studies, and clinical data, with open and paid access levels.
Data presented at EHA show that sickle cell disease patients have high interest in gene therapy, with 71% eager to learn more, despite limited awareness and concerns about safety and cost. Early clinical data from approved CRISPR-based therapies demonstrate significant reductions in vaso-occlusive crises and hospital admissions.
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
Nature Communications findings showed NR2F6 deletion revived exhausted CAR-T cells and boosted activity against solid tumors. In murine models, modified cells drove tumor regression, prolonged survival and resistance to rechallenge.
The FDA has issued draft guidance creating a "Plausible Mechanism Framework" to accelerate approval of individualized therapies for ultra-rare diseases where traditional clinical trials are not feasible. The guidance focuses on gene editing and RNA-based treatments targeting specific genetic abnormalities.
Researchers discovered that adding three common amino acids to lipid nanoparticles dramatically improves mRNA and CRISPR gene editing delivery, boosting efficiency up to 20-fold and raising gene editing success rates from 25% to nearly 90%.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Ocugen will host a conference call on March 4, 2026 at 8:30 a.m. ET to discuss fourth quarter and full year 2025 financial results and provide a business update, with a pre-market earnings announcement the same day.
The pharmaceutical industry is experiencing recovery driven by innovation in drug development, aggressive M&A activity, and expansion of drug development services. The sector faces both opportunities and headwinds including pipeline setbacks and regulatory pressures.
