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Dec 19 — This Week in Gene and Cell Therapy

Dec 19, 2025

 
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Featured:

Dec 17 4DMT Reports Positive 1-Year Efficacy for AAV Cystic Fibrosis Gene Therapy read more »
Dec 15 Link Cell Therapies Launches with $60M Series A to Advance Logic-Gated CAR-T for Solid Tumors read more »

News:

Dec 17 Addition Therapeutics Emerges from Stealth with $100 Million to Advance All-RNA Gene Insertion Technology read more »
Dec 16 AAV Gene Therapy Using AAV-Ant1 Successfully Restores Mitochondrial Function to Halt Dilated Cardiomyopathy in Mouse Models read more »
Dec 16 Genflow Biosciences Reports Strong Safety Profile After Completing Dosing of AAV SIRT6 Gene Therapy in Canine Clinical Trial read more »
Dec 15 Kyverna’s CAR-T Therapy Succeeds in Stiff Person Syndrome Trial and Targets First Autoimmune Approval read more »
Dec 15 CIRM Awarded $7.4 Million Grant to Advance Novel AAV Gene Therapy for Rare Muscular Dystrophy read more »
Dec 15 FDA Grants Orphan Drug Designation to Immusoft’s Engineered B Cell Therapy for the Treatment of MPS II read more »
Dec 15 AAV Gene Therapy Successfully Restores Hearing in Hereditary Deafness Models Using Targeted GRE Expression read more »
Dec 15 XOMA Royalty Expands Genetic Medicine Portfolio with Acquisition of Generation Bio and its Advanced ctLNP Delivery Platform read more »
Dec 12 Moderna Partners with Nanexa to Develop Long-Acting Injectable mRNA Medicines read more »
Dec 11 Tenaya’s AAV Gene Therapy TN-401 Shows Early Safety and Arrhythmia Reduction in ARVC Patients read more »

Articles:

Dec 17 AAV-delivered engineered suppressor tRNA rescues visual function in mice with an inherited retinal disease read more »
Dec 16 Optimization of AAV tools to target Muller glial cells for retinal gene therapy read more »
Dec 16 A Combinatorial mRNA Therapy for Treating Rheumatoid Arthritis and Osteoarthritis by Inhibiting Inflammation and Promoting Cartilage Regeneration read more »
Dec 16 Partial restoration of mitochondrial dysfunction by AAV-Ant1 protects from dilated cardiomyopathy in Ant1-/- plus mtDNA mutant mice read more »
Dec 16 Cell-specific delivery of GJB2 restores auditory function in mouse models of DFNB1 deafness and mediates appropriate expression in NHP cochlea read more »
Dec 15 Organ-specific delivery of an mRNA-encoded bispecific T cell engager targeting glypican-3 in hepatocellular carcinoma read more »
Dec 14 AAV-delivered CRISPR-Cas9 elicits persistent retinal immune responses compared with transient responses to RNP read more »
Dec 13 Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system read more »
Dec 11 mRNA vaccine developed for sequential selective organ-to-cell targeting of glioma read more »
Dec 11 Cyclase-Associated Protein 2 gene delivery as a potential multi-target approach for preventing synaptic failure in Alzheimer’s disease read more »
Dec 11 In vivo base editing rescues ADPKD in a humanized mouse model read more »
Dec 10 Redefining Multiple Sclerosis with CAR-T Cell Therapy read more »
Dec 10 DR5 CAR-T cells target melanoma and suppress MDSCs with minimal toxicity read more »
Dec 09 Introducing arginine residues into scFv in CAR-T cells shows promising antitumor effects read more »
Dec 09 Administration of barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport read more »
Dec 09 Lentiviral Hematopoietic Stem Cell Gene Therapy ameliorates GM1-gangliosidosis in mice read more »
Dec 09 Gene-corrected regulatory T cell therapy for IL2RA deficiency read more »
Dec 09 AAV gene therapy for GBA1 related diseases read more »
Dec 09 Personalized CRISPR knock-in cytokine gene therapy to remodel the tumor microenvironment and enhance CAR T cell therapy in solid tumors read more »

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PackGene Biotech, Inc.
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