The rapid advancement of cell and gene therapies offers unprecedented potential to treat complex diseases, but navigating both scientific and financial challenges remains a significant hurdle. From developing robust clinical trials to securing funding for research, biotech companies face a dual challenge in bringing these transformative treatments to market. This article explores the key obstacles and innovative solutions that are driving the next wave of breakthroughs in cell and gene therapy.
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November 28, 2023
Facing the Dual Scientific and Financial Challenges to Advance Cell and Gene Therapies
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Repeatability and Intermediate Precision of a Genome Integrity Assay for rAAV by Long-Read Sequencing
Truncated recombinant AAV genomes can compromise transgene expression and the therapeutic efficacy1 of gene therapy vectors, making genome integrity a critical quality attribute, highlighting the need for robust analytical methods. Long‑read...
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June 01, 2026
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ddPCR-Based Quantification and Fragment Analysis of Residual E1A in rAAV Products: Method Validation and Correlation with Total Host Cell DNA
Abstract Gene therapy using adeno-associated virus (AAV) vectors has emerged as a promising strategy, offering new hope for patients with genetic disorders. However, during rAAV manufacturing, residual host-cell DNA (hcDNA) and plasmid DNA from the...
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June 01, 2026
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Multivariate Approach to Small-Scale Model Qualification in Upstream Adeno-Associated Virus Production
Abstract AAV gene therapy manufacturing requires scalable processes and reliable small-scale models (SSMs) to support process characterization and regulatory expectations. Ensuring that SSMs accurately represent large‑scale operations is essential...
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June 01, 2026