Blog

Co-chair panel Bioassays and statistics FAQs to FACTS

In this session, Nancy Sajjadi and Steve Walfish introduce 4 speakers and posit several questions to address hot topics in bioassay design, development and validation:  Generating and evaluating the standard curve, establishing equivalence…

Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

This paper, cited over 200 times, summarizes a phase 1 dose escalation study, 13 subjects with hemophilia A received by peripheral intravenous infusion a retroviral vector carrying a B-domain–deleted human factor VIII (hFVIII) gene. This was…

Defining a Detailed Approach to Using the Adenovirus Reference Material (ARM)

This white paper encouraged advocates of the adenovirus standard to carefully examine its use in the field. It was originally presented at conference in 2003 but the article has recently been made open access (https://www.bioprocessingjournal.com/afp/J25-Sajjadi.pdf)…

Analysis of testes and semen from rabbits treated by intravenous injection with a retroviral vector encoding the human factor VIII gene: no evidence of germ line transduction

In this paper, we demonstrated that a PCR assay to detect vector was fit for its intended purpose.  In collaboration with Kim Boekelheide at Brown University, my group addressed a key regulatory concern for integrating vectors- potential for…

Working toward an adenoviral vector testing standard

This paper summarizes the proceedings of a conference held to discuss the development of an adenoviral vector standard.  In response to the tragic death of Jesse Gelsinger, dose related toxicity became a focus in the field of gene therapy.…