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Overcoming data-driven challenges to minimize toxicity in gene therapy development

04 May 2026
Gene Therapy Development
  • Does the physical titer or genome titer correlate with (or match) the functional titer?
  • How is rAAV genome integrity characterized, specifically with respect to partial versus full-length genomes?
  • What residual plasmid impurities or reverse packaging species are present, and how do they impact drug potency?
  • How are host genomic DNA impurities detected and characterized?
Industry Expert
Mehmet Karaca, Director | Molecular Biology Sequencing - Sarepta Therapeutics