Enabling IND Approval: Preclinical in vivo Considerations for Novel Cell and Gene Therapies
Recorded: July 2022
Preclinical evaluation of cell and gene therapy (CGT) products in vitro and in vivo helps characterize product profile throughout development. The cell and gene therapy space is expanding significantly, with many candidate therapies advancing into clinical testing and many more progressing through preclinical pipelines. It is a critical time to ensure your preclinical road map is well thought through to position your product for success. As the number of early-stage candidate products continues to increase, so does the need to define effective preclinical development strategies that support clinical translation. Given the diverse and often multifunctional nature of these therapies, it is not possible to define a one-size-fits-all preclinical program for cell and gene therapy. Instead, preclinical studies need to be customized to support the feasibility of the proposed administration route and appropriate application procedure, address the candidate product’s specific therapeutic properties, and fully characterize any potential safety concerns. This webinar will highlight through case studies the benefit of experience and how a data driven science based approach using clinically relevant modalities and end points can support a successful IND submission.
Key Messages
- The diversity and biological properties of GT products necessitate a flexible testing strategy
- Preclinical study designs for CGT should be product specific (no “one-size-fits-all” approach and supported by scientific rationale/data)
- The correct animal model has to be selected for assessment of bioactivity and safety which demonstrates a biological response similar to that expected in humans
- Study protocols should be reviewed and approved by an Institutional Animal Care and Use Committee (IACUC) before conducting studies and all study tasks should be performed in an AAALAC accredited facility in accordance with facility Standard Operating Procedures (SOPs) and consideration of the 3Rs
- The right end points have to be selected which can demonstrate efficacy and functional rescue with appropriate imaging modalities which enable visualization of anatomical and physiological changes
- Correctly designed POC and safety studies with clinically relevant endpoints/outcomes will help establish benefit: risk profile in the IND submission
- A qualified and experienced preclinical team with experience is key
Moderated by:
Vatsala Naageshwaran, M.A.,M.S - Vice President Business Operations at Pharmaron
Speakers:
Key Opinion Leader: Sean Ainsworth - Chief Executive Officer at Immusoft
Sean has more than 20 years’ experience in pharmaceuticals and biotechnology. Previously, he founded and led RetroSense Therapeutics, a gene therapy company he advanced into human clinical trials and sold to Allergan in 2016 in a deal valued at up to $555 million.
Earlier, he was deeply involved in the launch of Compendia Bioscience, Inc., and GeneVivo, LLC. At both he assisted in developing business models, licensing technologies, building management teams, and securing capital and first customers. Life Technologies acquired Compendia BioScience in 2012.
Sean founded Ainsworth BioConsulting in 2004 to provide licensing, as well as strategic and business planning services to the life science and entrepreneurial community. His clients included large pharma, small biotechs, universities, CROs, and venture investment funds. He has worked with clients at all stages of development.
Sean earned an M.B.A. in strategy and finance from Washington University in St. Louis. He holds a B.S. in Microbiology from University of California, San Diego.
Pharmaron Speaker: Linda L. Manza, Ph.D. - Sr. Director of Scientific Operations at Pharmaron
Linda received a Ph.D. in Pharmacology and Toxicology from the University of Arizona, Tucson, a M.S. in Toxicology from San Diego State University, Graduate School of Public Health, and a B.A. in Biological Sciences and Chemistry from the University of California Santa Barbara, College of Creative Studies. Linda started out in the nursing field and once she finished her masters worked as a toxicologist in small pharma and a contract research organization. She then returned to school for her Ph.D. During that time, she was in the Army reserve and was in charge of a 440-member hospital unit during peace time and was the Company Commander of a 40-person Medical Support unit deployed in support of Operation Iraqi Freedom. Following the completion of her PhD she worked in Quality Assurance for a mid-sized pharmaceutical company for six years, the last four as the Associate Director for GLP Compliance. In September of 2013 Linda started her own consulting company and assisted small companies with lab management, study design and quality assurance issues. In November of 2015 she began working at Absorptions Systems as the Director of Quality and Compliance until 2019 when she transitioned to the Director of Scientific Operations. Linda is currently Senior Director of Scientific Operations for Pharmaron with oversight for all preclinical study execution and data delivery.
Pharmaron Speaker: Sandeep Kumar, Ph.D. - Director, Ophthalmology at Pharmaron
Dr. Sandeep Kumar is Director of Ophthalmology at Pharmaron. He serves as Principal Investigator or Study Director for preclinical ophthalmic studies and interface with different teams including in-vivo scientists, QC/QA, Account Managers/Sales. Dr. Kumar has 10+ years of experience working in ocular research field. He worked previously at Karolinska Institute, Sweden; Moran Eye Center, University of Utah; Cullen Eye Institute at Baylor College of Medicine, USA. Originally, trained as a biochemist, Dr. Kumar has made significant contributions in creating animal models for eye disease research. He has transgenically generated and characterized a mouse model for Polypoidal Choroidal Vasculopathy (PCV), a variant of Age Related Macular Degeneration (AMD), which mimics human PCV condition. Dr. Kumar’s scientific papers has been published in prestigious journals such as Proceedings of the National Academy of Science, Circulation Research, Investigative Ophthalmology Vision Science, Clinical genetics, BBA and Molecular and Cellular Biochemistry etc. His research interests are focussed on developing animal models for ocular diseases to use them for new treatment strategies.