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A Platform Approach to Functional Potency Assay Development for Gene Therapy Products

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Originally Presented:
Wednesday, November 20, 2019
Analytical Development Summit
Boston, MA

Presenter: Karen Doucette, MBA
Director of Operations, ACF Bioservices

What you will learn:

  • Streamlining development of functional potency assays across diverse product types with custom and complex MOA
  • Bridging the gap between R&D and cGMP for commercially viable potency assays
  • Case studies for in vivo and ex vivo and non-vector based therapies

 

A Platform Approach to Functional Potency Assay Development for Gene Therapy Products

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Presentation Request:

Potency Assay Development for Cell and Gene Therapy Products

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Abstract:

THE ISSUE
Approved biological products are required to be accompanied by analytical tests to demonstrate safety, purity and potency that are validated and performed under current Good Manufacturing Processes (cGMP) conditions. CGT products present specific challenges for developing in vitro potency assays due to their complex mechanisms of action, complicated manufacturing processes, variable critical quality attributes (CQAs) and reduced preclinical and early stage clinical data with which to inform development.

THE PROCESS
Developing in vitro potency assays for CGT products must include at least two steps: demonstration of the vector’s ability to transfer genetic material into a cell and demonstration of function. The development of an in vitro potency assay to accompany an approved CGT product involves a series of activities that can be divided into four key stages.

THE SOLUTION
ACF has a long history of creating well controlled, biologically relevant test systems and validating them to support FDA approvals. Our experience includes creating and characterizing custom engineered cell lines to silence or overexpress target genes.

Using Empty AAV Capsids to Reduce Inhibitory Effect of Neutralizing Antibodies on AAV Transduction

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Using Empty AAV Capsids to Reduce Inhibitory Effect of Neutralizing Antibodies on AAV Transduction

Jiu Jiang, PhD1, Steven Wright, PhD2, and Ismael Hidalgo, PhD1
1Absorption Systems LLC, 436 Creamery Way, Suite 600, Exton, PA 19341
2Deaprtment of Biology, Carson-Newman University, 1646 Russel Ave, Jefferson City, TN 37760

This poster was presented at AAPS PharmSci 360, 2019

Currently, gene therapy is becoming more promising for treating numerous genetic disorders. The non-pathogenic adeno-associated viruses (AAVs) are among the most frequently used viral vectors for gene therapy. However, patients with pre-existing immunity against AAVs have neutralizing antibodies (NAb) that may hinder viral transduction; thus, they
are less likely to benefit from this type of therapy. Since the prevalence of NAb in the human population can reach up to 60%-70% for some serotypes such as AAV2, systematic exclusion of seropositive patients from treatment would greatly limit the potential impact of gene therapy in humans. For this reason, there is a need to understand how NAbs can interact with AAVs and explore strategies to allow expansion in the application of gene therapy in individuals with NAb. The purpose of this study was to investigate, in an in vitro cell-based assay, the potential utility of empty capsids of AAV2 in overcoming the negative effect of NAb on AAV2-mediated transduction.

In Vitro and In Vivo Assessment of the Potential of Supersaturation to Enhance the Absorption of Poorly Soluble Basic Drugs

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In Vitro and In Vivo Assessment of the Potential of Supersaturation to Enhance the Absorption of Poorly Soluble Basic Drugs

Yuri Bukhtiyarov, Jibin Li, Nicole Spivey, Svitlana Silchenko, Christopher Force, Carlos Hidalgo, Yuehua Huang, Albert J. Owen, Ismael J. Hidalgo
Absorption Systems, LLC

This poster was presented at AAPS PharmSci 360, 2019

Inducing supersaturation is an attractive approach for increasing the systemic absorption of poorly soluble drugs. Delayed precipitation of weakly basic drugs in supersaturated state following their transition from the acidic gastric environment to the near-neutral proximal small intestinal fluid is emerging as a promising tactic for achieving higher transitional solubility and improved bioavailability of such drugs. The purpose of this study was to assess the effect of pH-shift induced supersaturation on drug dissolution and permeation in vitro, and to evaluate the approach of extending supersaturation to enhance drug oral absorption in vivo.