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Featured:
uniQure Reports Encouraging Early Clinical Case Data for AMT-260 AAV Gene Therapy in Refractory MTLE read more »
Beam Therapeutics’ BEAM-302 Granted FDA Orphan Drug Designation for Alpha-1 Antitrypsin Deficiency read more »
Ocugen’s AAV Therapy OCU410ST Receives U.S. FDA Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies read more »
News:
Intellia Therapeutics Provides Update on Phase 3 MAGNITUDE Study; Reports Grade 4 Liver Enzyme Elevation in One Patient read more »
Taysha AAV Therapies Achieves FDA Alignment on Pivotal Trial Design and Reports Positive Phase 1/2 Data for TSHA-102 in Rett Syndrome read more »
TQ Therapeutics Acquires Juno Therapeutics GmbH, Bolstering Cell Therapy Development read more »
FDA Places Rocket Pharmaceuticals’ RP-A501 Danon Disease Clinical Trial on Hold read more »
XellSmart Biopharmaceutical Receives NMPA and FDA Approval for World’s First Clinical Trial of Allogeneic iPSC-Derived Neural Regenerative Cell Therapy for Spinal Cord Injury read more »
UniXell Biotech Announces Successful First-in-Human Dosing of Autologous iPSC-Derived Cell Therapy (UX-DA001) for Parkinson’s Disease read more »
AbelZeta Pharma Presents Promising Early Clinical Data for C-CAR168 in Refractory Autoimmune Diseases at LUPUS 2025 read more »
King Faisal Specialist Hospital & Research Centre and Cellenkos Inc. Forge Landmark U.S.-Saudi Clinical Trial Alliance for Advanced Cell Therapies read more »
High-Cost Trend in New U.S. Drugs Continues, Driven by Rare Disease Therapies read more »
CureDuchenne Ventures Invests $1 Million in Entos Pharmaceuticals for Duchenne Gene Therapy read more »
Articles:
In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking read more »
Stromal Gene Therapy Mediates Prolonged Protection Against Corneal Neovascularization Induced by an Aggressive Angiogenic Insult read more »
Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components read more »
Airway applied mRNA vaccine needs tailored sequence design and high standard purification that removes devastating dsRNA contaminant read more »
Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy read more »
Perspective: Advancing safe and effective DNA therapeutics using bioactive lipid nanoparticles read more »
Disease Modifying, Multidimensional Efficacy of Putaminal CaV1.3-shRNA Gene Therapy in Aged Parkinsonism Male and Female Macaques read more »
Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy read more »
CD97-directed CAR-T cells with enhanced persistence eradicate acute myeloid leukemia in diverse xenograft models read more »
Allogeneic CAR T cells: A new player in the field and the peculiar opportunities of the hospital exemption path read more »
Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes read more »
Cross-species tropism of AAV.CPP.16 in the respiratory tract and its gene therapies against pulmonary fibrosis and viral infection read more »
Novel Photoreceptor-Specific Promoters for Gene Therapy in Mid-to-Late Stage Retinal Degeneration read more »
A suite of enhancer AAVs and transgenic mouse lines for genetic access to cortical cell types read more »
AAV vector development, back to the future read more »
Evolution-guided protein design of IscB for persistent epigenome editing in vivo read more »
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
