Jun
24
2021
On demand

Addressing the challenges of purification & quality control in gene therapy

Recombinant Adeno Associated Virus (rAAV) vectors are one of the most promising mechanisms for therapeutic gene delivery. However, most industrial cell lines exhibit inefficient gene packaging which results in a heterogenous population of vectors composed of empty, partially-loaded, and fully-loaded species. Purifying, identifying, and quantifying these different species is vital from a production and quality control standpoint, since improperly loaded viral capsids do not produce the desired therapeutic effect but may still elicit an unintended immune response.

In this Beckman Coulter Life Sciences webinar, our panelists will focus on several pertinent topics in rAAV process development including current challenges, technologies for overcoming quality control hurdles, best practices for downstream processing, as well as discuss the current trends and future outlook of this rapidly growing therapeutic frontier.

Attendees will learn:

  • Understand the challenges of differing serotypes in rAAV production and quality control
  • Understand the commonly used characterization techniques for AAV capsids
  • Understand the basics of Analytical Ultracentrifugation (AUC) and how it is used to characterize empty, full, and intermediate AAV capsids
  • Learn about best practices in Density Gradient Ultracentrifugation (DGUC) to achieve highest purity in gene therapy products

Further panellists to be announced soon.

Dr. Akash Bhattacharya

Senior Application Engineer, Beckman Coulter Life Sciences

Akash Bhattacharya graduated from Presidency College, India with a major in Physics and went on to a Masters’ in Physics at the Indian Institute of Science where he worked on Quantum Computing. He then moved for doctoral studies in Biophysics to the University of Michigan, Ann Arbor where he joined the lab of Prof. Erik Zuiderweg and worked on NMR spectroscopy methods development in the context of the Structural Biology of Chaperone Proteins.

After Michigan, he worked briefly at Rutgers and then eventually moved to the Dept. of Biochemistry and Structural Biology at the University of Texas Health at San Antonio. Here, he worked with Prof. Dmitri Ivanov and Prof. Borries Demeler on the enzymology of HIV infection and restriction by mammalian proteins. He also worked on projects related to oncology (DNA damage repair) and neuroscience (voltage gated ion channels), using a wide variety of techniques ranging from X-ray crystallography, NMR spectroscopy, fluorescence spectroscopy, molecular dynamics and analytical ultracentrifugation (AUC).

He collaborated with Prof. Demeler to extend AUC methods to novel enzymatic systems resulting in publications in PNAS, Cell Reports, Nature Scientific Reports, etc. Akash joined Beckman Coulter Life Sciences in Oct 2018. He is based in the Colorado R&D center and works on developing new AUC applications. His research interests include extending AUC methodology to new therapeutic areas such as AAV capsids (gene therapy), liposomal drug carriers and others.

Leisha Kopp 

Applications Scientist, Mirus Bio

Leisha Kopp is an Applications Scientist at Mirus Bio LLC, a biotech company providing innovative transfection products to cell culture researchers worldwide. Leisha has over 15 years of molecular biology and mammalian cell culture experience in industrial labs, and her combined bench and business knowledge enables support of scientists in all stages of the drug discovery process - from R&D to commercial manufacturing. Leisha is a graduate of the University of Wisconsin-Madison, with key interests in biotherapeutic antibody discovery and gene therapy.

Dr. Klaus Richter

Group Leader AUC, Coriolis Pharma

Dr. Klaus Richter is the leader of the AUC group at Coriolis Pharma, which is dedicated to the biophysical characterization of drug products and drug substances ranging from small molecules and peptides to viral particles and liposomes. He is a biochemist by training and received a Ph.D. from Technical University of Munich in the field of protein characterization and worked for several years in the area of structure-function and structure-activity relationships of pharmaceutically relevant proteins before joining Coriolis Pharma. Klaus obtained his Habilitation in 2012 and is the (co-) author of more than 70 publications in the field of protein characterization with diverse biophysical methods, in many cases applying analytical ultracentrifugation (AUC) as a tool to investigate protein structure, protein interaction and stability.

Dr. Audrey Chang

Vice President of Quality Control and Analytical Service, Vigene Biosciences

Dr. Chang is a world leader in biologics QC and analytical testing; and is currently leading the QC and Analytical Development group at Vigene BioSciences. Her 25 year career has focused on leading the QC development efforts for biologics with a recent focus on cell gene therapy and novel modalities biological products. Audrey is the author of more than 15 papers and has served as a keynote speaker for many conferences and meetings. Audrey earned her Ph.D in Biology from John Hopkins University and B.S. from University of Maryland (College Park).

Shawn Sternisha, Ph.D

Senior Field Applications Scientist, Beckman Coulter Life Sciences

Shawn Sternisha is currently a Senior Field Applications Scientist in the Biotechnology Business Unit at Beckman Coulter Life Sciences where he develops applications and supports centrifuge product lines. Shawn attended Illinois State University for his undergraduate studies and went on to earn his Ph.D. in Biochemistry at Florida State University. His doctoral research was focused on understanding how glucokinase regulates glucose homeostasis at the molecular level. Shawn has also previously worked at GlaxoSmithKline where he was involved in investigating novel imaging modalities for monitoring mammalian cell cultures. Shawn is broadly interested in enzyme structure-function relationships and mechanisms of action, cell and gene therapy, and expanding applications of biochemical and biophysical techniques (namely Analytical Ultracentrifugation). Shawn currently resides in Miami, FL with his fiancée Patty and their dog Ruby. In his free time, he enjoys cooking, running, traveling, and learning Spanish.

SPEAKERS

Dr. Akash Bhattacharya
Senior Application Engineer, Beckman Coulter
Dr. Akash Bhattacharya
Senior Application Engineer, Beckman Coulter
Leisha Kopp
Applications Scientist, Mirus Bio
Leisha Kopp
Applications Scientist, Mirus Bio
Klaus Richter, PhD
Group Leader AUC, Coriolis Pharma
Klaus Richter, PhD
Group Leader AUC, Coriolis Pharma
Dr. Klaus Richter is the leader of the AUC group at Coriolis Pharma, which is dedicated to the biophysical characterization of drug products and drug substances ranging from small molecules and peptides to viral particles and liposomes. He is a biochemist by training and received a Ph.D. from Technical University of Munich in the field of protein characterization and worked for several years in the area of structure-function and structure-activity relationships of pharmaceutically relevant proteins before joining Coriolis Pharma. Klaus obtained his Habilitation in 2012 and is the (co-) author of more than 70 publications in the field of protein characterization with diverse biophysical methods, in many cases applying analytical ultracentrifugation (AUC) as a tool to investigate protein structure, protein interaction and stability.
Dr. Audrey Chang
Vice President of Quality Control and Analytical Service, Vigene Biosciences
Dr. Audrey Chang
Vice President of Quality Control and Analytical Service, Vigene Biosciences
Shawn Sternisha
Senior Field Applications Scientist, Beckman Coulter Life Sciences
Shawn Sternisha
Senior Field Applications Scientist, Beckman Coulter Life Sciences
Shawn Sternisha is currently a Senior Field Applications Scientist in the Biotechnology Business Unit at Beckman Coulter Life Sciences where he develops applications and supports centrifuge product lines. Shawn attended Illinois State University for his undergraduate studies and went on to earn his Ph.D. in Biochemistry at Florida State University. His doctoral research was focused on understanding how glucokinase regulates glucose homeostasis at the molecular level. Shawn has also previously worked at GlaxoSmithKline where he was involved in investigating novel imaging modalities for monitoring mammalian cell cultures. Shawn is broadly interested in enzyme structure-function relationships and mechanisms of action, cell and gene therapy, and expanding applications of biochemical and biophysical techniques (namely Analytical Ultracentrifugation). Shawn currently resides in Miami, FL with his fiancée Patty and their dog Ruby. In his free time, he enjoys cooking, running, traveling, and learning Spanish.