Targeting– tag –
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CRISPR Gene Therapies Expand Beyond Initial FDA Approvals, Advancing to Late-Stage Trials for Diverse Indications
Ubie Doctor's Note USA Overview As of mid-2026, two CRISPR gene therapies, Casgevy (for sickle cell disease and beta-thalassemia) and EDIT-101 (for Leber congenital amaurosis type 10), have received FDA approval, establishing foundationa... -
China’s NMPA Grants World’s First Approval for Solid Tumor CAR T-Cell Therapy Satri-cel Targeting CLDN18.2 Positive Gastric Cancer
OncLive USA Overview OncLive's June 2026 GI oncology regulatory roundup highlights China's NMPA approval of CARsgen Therapeutics' satricabtagene autoleucel (satri-cel), the first solid tumor CAR T-cell therapy for Claudin18.2-positive ad... -
KATU News: Experimental Gene Therapy Targeting Aging Cells Based on Yamanaka Factors Enters Early Glaucoma Trials
KATU News (YouTube) USA Overview According to KATU News, an experimental gene therapy inspired by Professor Shinya Yamanaka's Nobel Prize-winning iPSC research is targeting aging cells and holds the potential to open a new chapter in agi... -
Penn Medicine’s AI Framework Uncovers Novel GPNMB Target for CAR T-cell Therapy, Advancing Solid Tumor Treatment
Penn Medicine USA Overview Researchers at Penn Medicine have developed an innovative artificial intelligence (AI)-powered framework to efficiently identify novel target antigens for CAR T-cell therapy. This AI framework successfully demo... -
CRISPR’s First Clinical Evidence for HBV cccDNA Elimination, Prime Editing Advances to Inaugural Human Trial
CRISPR Medicine News USA Overview Precision Biosciences' PBGENE-HBV has demonstrated the first clinical evidence of eliminating viral cccDNA in chronic Hepatitis B patients, marking a significant milestone for CRISPR-based therapies in i... -
Synthetic Biology Firm Syntax Bio Expands Series A to $14.4M, Boosting Preclinical Research for CRISPR-Based Type 1 Diabetes Beta-Cell Therapy
Fierce Biotech USA Overview Synthetic biology company Syntax Bio announced an expansion of its Series A funding round to $14.4 million, pushing total capital raised over $25 million. This capital injection will primarily drive the develo... -
Engineered Exosomes Breach Blood-Brain Barrier, Unlocking New Vistas in Neuro-Oncology
MDPI (論文) Global Overview The blood-brain barrier (BBB) critically impedes drug delivery to the brain, posing a major challenge for treating central nervous system diseases, particularly brain tumors. Engineered exosomes are emerging a... -
Exosomes: Unlocking Nature’s Nanoparticles for Precision Medicine, Navigating Translational Hurdles
Frontiers (論文) Global Overview Exosome research is rapidly advancing, revealing substantial potential in drug delivery and regenerative medicine due to their inherent biocompatibility and targeting capabilities. However, clinical trans... -
SonoThera Raises $125M Series B to Advance Safer Ultrasound-Mediated Gene Therapies for Duchenne Muscular Dystrophy and ADPKD into Clinical Development
BioSpace USA Overview SonoThera has secured $125 million in Series B financing to advance its gene therapy programs for Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) into clinical development.... -
Beam Therapeutics Receives FDA IND Clearance for BEAM-304, Advancing Base Editing Therapy for Phenylketonuria (PKU) into Clinical Development
GlobeNewswire USA Overview Beam Therapeutics Inc. announced that the FDA has cleared its Investigational New Drug (IND) application for BEAM-304, a base editing therapy for Phenylketonuria (PKU). PKU is a rare genetic metabolic disorder ...
