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Breakthrough in Genetic Medicine: Beam Therapeutics Shows Potential with BEAM-302 for Alpha-1 Antitrypsin Deficiency
Beam Therapeutics announces groundbreaking results from its Phase 1/2 trial of BEAM-302, demonstrating the first-ever clinical genetic correction for Alpha-1 Antitrypsin Deficiency (AATD), offering hope for a potential one-time cure for this genetic disorder...
New RNA-Guided System Uncovered, Offering Promise for Precision Gene Editing Therapies
TIGR (Tandem Interspaced Guide RNA) systems use RNA guides to target specific DNA sites, offering a compact and modular alternative to CRISPR. Scientists at MIT and the Broad Institute discovered over 20,000 TIGR-associated proteins, some capable of precise DNA cutting, potentially expanding genome...
Avance Biosciences™ Registers with CLIA, Expanding Clinical Trial Testing Capabilities
Avance Biosciences™ announces CLIA registration, expanding its high-complexity laboratory testing capabilities for clinical trials, regulatory submissions, and companion diagnostics. This milestone strengthens its commitment to high-quality, regulatory-compliant testing solutions that accelerate drug...
Gene Therapy Restores Vision in Children with AIPL1-Related Retinal Dystrophy
A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...
World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England
Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel. Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia...
FDA Approves First T Cell Receptor (TCR) Gene Therapy
Tecelra is the first FDA-approved T cell receptor (TCR) gene therapy. The product is an autologous T cell immunotherapy composed of a patient’s own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen expressed by cancer cells in synovial sarcoma...