Accurate Quantification and Characterization of Adeno-Associated Viral Vectors

Mitigation strategy for Unwanted Immunogenicity of Therapeutic Proteins

Taking immunogenicity assessment of therapeutic proteins to the next level

Immunogenicity of biotherapeutics for biosimilars and biobetters.

Immunogenicity assessment of biotherapeutic products: An overview of assays and their utility.

Characterization of immunogenicity response to multiple domain biotherapeutics

Recommendation for risk-based bioanalytical strategy for the immunogenicity assessment for biologics

Antisense Oligonucleotide Volanesoren lowers triglyceride levels in Familial Chylomicronemia Syndrome

Correlation of anti-AAV9 Preexisting antibody with In Vivo Transduction and NAGLU activity

Nonclinical Non-human Primate Studies for Hemophilia investigate impact of preexisting antibodies against AAV5 on therapeutic efficacy

Industry trends on use of surrogate biomaker as endpoint for clinical benefit

GIVLAARI (givosiran, siRNA) for treatment of Acute Hepatic Porphyria

MALS (Multi Angle Stable Light Scattering)

Analytical Platform Review: Mass Spectrometers (LC-MS) for Protein Characterization and Quantification

Digital Droplet PCR (ddPCR) For Viral Vectors Biodistribution Studies For Gene Therapy

FDA raise concern over data manipulation for Zogensma (Gene therapy for SMA)

Engineering Immunodominant Residues of Cas9 reduces Cell Mediated Cytotoxicity

AAV-CRISPR mediated gene editing corrects Duchene Muscular Dystrophy phenotype

AVV5 mediated microRNA delivery for treatment of ALS

Pre-existing Immune Responses against Cas9 protein (CRISPR) add complexity to CRISPR based Gene Therapy