Speakers: Patrick Trapa, Biogen; Nancy Chen, Moderna; Frank Gibbons, Takeda; Chris Scull, Scull Bio Consulting ; Diane Ramsden, Korro Bio; Adam Amaral, Intellia Therapeutics; Patrick Au, Ensoma; Vaishali Dixit, Mersana
Organizers: Hank Lin, Toxicology Consultant; Hanlan Liu, Be Biopharma; Chuck Locuson, Agios Pharmaceuticals; Ekta Kadakia, Biogen
Date: 2024-11-06
Time: 8:15-17:10 Eastern Time
Registration fee: Regular attendees: $295; Academic/Students/Postdocs/Out-of-Pocket: $45
Location: Webcast from PBSS Boston.
Major Sponsor:
Vendor show vendors registered to date:
Registration: http://www.PBSS.org
Registration deadline:2024-11-06  (it will close sooner if the seating cap is reached)

About the Topic

Both commercial and clinical pipeline of new modalities have vastly increased in recent years; notably the antibody drug conjugate, SiRNA/ASO, and gene and cell therapies-based therapeutics in development has been growing at an incredible pace. The investigational new drug application (IND) application is a crucial step to achieve therapeutics, and understanding the requirements for a successful IND filing is utmost important to ensure fast and efficient development. While the principles and basics are similar across different modalities for an IND filing such as safety/PK studies, there are important aspects that need to be considered for antibody drug conjugate, SiRNA/ASO, and gene and cell therapies-based therapeutics. In this workshop, we will cover the best practices for a successful IND package of antibody drug conjugate, SiRNA/ASO and gene and cell therapies. Our experienced speakers will discuss the preclinical studies for antibody drug conjugate, SiRNA/ASO, and gene and cell therapies IND packages. This workshop will be suitable for novice drug developers and small molecule drug development experts who are interested in learning these new modalities’ therapeutic development.

• Adam is currently a director of bioanalytic and DMPK at Intellia. Since joining Intellia in 2017, Adam has supported the advancement of novel CRISPR/Cas9 gene editing therapies from non-clinical to clinical stages of development. His contributions in the non-clinical PK, PD and safety data analysis, as well as modeling, have been instrumental to the successful first-in-human dosing regimens employed in the first two in vivo CRISPR gene editing programs. Additionally, Adam has served as the non-clinical lead for several development candidates and manages a productive bioanalysis lab to support early-stage platform and research studies.
  
• Christopher Scull is a drug development professional with expertise in preclinical development of complex biologics. Dr. Scull’s has managed IND-enabling programs at academic centers and at biotech startups. Since 2017 Chris has worked as a drug development and regulatory consultant, helping biotech companies navigate meetings with the FDA and supporting more than 40 IND applications. In 2024 Chris founded his own consulting firm to continue providing preclinical support to companies transitioning from R&D to a clinical stage of development. Dr Scull also serves as a reviewer at CIRM, where he provides funding recommendations for translational, preclinical, and early phase programs.
• Diane Ramsden is a Senior Director and ADME BA group leader within Korro Bio. She has worked in the pharmaceutical industry for over 20 years and has a range of experience in project representation from early discovery stage to post marketing in multiple therapeutic and disease areas and across modalities. She has authored over 75 peer reviewed articles, book chapters and abstracts, holds three patents, one trade secret and has given over 30 invited talks. She is an editorial board member for Drug Metabolism and Disposition and serves as a member of the scientific leadership team with PBSS Boston.
• Frank Gibbons is currently Scientific Director of DMPK & Modeling at Takeda Pharmaceuticals, Cambridge, MA, where he leads a team of talented modelers providing model-based support for preclinical drug development in diseases associated with the gastro-intestinal system or inflammation. Prior to that, he led preclinical modeling support for Takeda’s recombinant AAV gene therapy programs in rare lysosomal storage disorders. With a PhD in physics from Northeastern University, Boston, he has over 20 years’ experience applying quantitative techniques to biological problems, and has experience in DMPK, PK/PD and QSP modeling, pharmacometrics and clinical pharmacology.
• Nancy Chen is currently working as Senior Director heading the DMPK department at Moderna. She received her PhD degree from University of Western Ontario in Canada focusing on Pharmacokinetics and Drug Metabolism. Nancy started her industry career at PPDM Merck working on biologics and siRNA platform for 5 years. She joined Shire in 2015 and eventually became the group leader within DMPK for Lysosomal Storage Disease and Rare Genetic Disorders, focusing on different therapeutic modalities such as small molecules, therapeutic proteins and gene therapy. After the acquisition of Takeda, Nancy continued to lead the DMPK project rep group, supporting various modalities in rare disease, immune-oncology and autoimmune disease.
• Patrick Au is a founding team member and VP of Translational Research and Early Development at Ensoma. He was previously Senior Director of Regulatory, Pharmacology and Toxicology at Casebia Therapeutics. Prior to that, he worked in Pharmacology/Toxicology and Regulatory Affairs at Spark Therapeutics and Adverum Biotechnologies (formerly Avalanche Biotech). Patrick began his career at the FDA as a Pharmacology/Toxicology Reviewer in CBER’s Office of Cellular, Tissue and Gene Therapies. He received his Ph.D in Medical Engineering from Harvard-MIT Division of Health Sciences and Technology and his B.S. in Chemical Engineering at Johns Hopkins University.
• Patrick Trapa currently leads the DMPK department at Biogen. He received an SB and Ph.D. from the Department of Materials Science and Engineering at MIT, and an M.B.A. from the University of Rhode Island. After receiving his doctorate, he co-founded a lithium polymer-battery company and later served as lecturer at MIT. During that time, he consulted with NASA on high- temperature metallurgy projects. He then ran the specialty-battery division at a publicly traded battery company before entering Pfizer in 2006. There, he held roles in preformulations as well as ADME, PK/PD and systems modeling and led quantitative translation in multiple therapeutic areas.
• Dr. Vaishali Dixit is currently Head of DMPK at Mersana Therapeutics. She holds a PhD in Medicinal Chemistry from the University of Florida and has conducted postdoctoral work at the University of Washington. She has over 15 years of experience in drug discovery and development and has contributed to several regulatory submissions. She has worked in multiple modalities including small molecules, ADCs, and protein degraders, spanning programs from early discovery into early development. She has participated in industry consortia on PBPK models for DDI and Special Populations resulting in peer reviewed publications. Prior to joining Mersana, Vaishali has held roles of increasing responsibilities at Vertex, Eisai and Kymera Therapeutics.