Speakers: Lakshmi Amaravadi, AstraZeneca; Valerie Clausen, Alnylam; Tripti Gaur, AffyImmune Therapeutics; Thomas Klem, Solid Biosciences; Rama Boyanapalli, Bristol Myers Squibb; Anka Ehrhardt, Merck
Organizers: Karen Cao, Beam Therapeutics; Jaya Goyal, Novo Nordisk; Haojing Rong, Atlas Venture NewCo
Date: 2024-09-12
Time: 8:15-16:30 Eastern Time
Registration fee: Regular attendees: $295; Academic/Students/Postdocs/Out-of-Pocket: $45; Additional vendor show rep: $125; Major Sponsorship: $3000; Vendor Show: $695, Lunch Sponsorship: $1250; On-site Registration: $500
Location: Marriott Cambridge, 50 Broadway, Cambridge, MA 02142
Major Sponsor: (2)Frontage Laboratories, Inc.; PPD, Part of Thermo Fisher Scientific
Vendor show vendors registered to date: (7)Aliri; Altasciences; BioAgilytix; Celerion; NovaBioAssays LLC; Resolian; Worldwide Clinical Trials
Registration: http://www.PBSS.org
Registration deadline:2024-09-11  (it will close sooner if the seating cap is reached)

About the Topic

This in-person workshop focuses on the critical role of biomarkers in drug development for regulatory approval, particularly for rare diseases, with industry experts from biotechs/pharma companies to share case studies, insights and strategies. It aims to enhance understanding of biomarker strategies, validation, and their pivotal role in therapy development.

Topics include the biomarker framework, small molecule biomarkers in clinical studies, minimal residual disease monitoring in CAR-T cell therapies, phenylalanine as a biomarker for PKU gene therapy, neurodegenerative disease biomarkers, and analytical challenges.

Participants have the opportunity to connect with industry experts, fostering a community dedicated to advancing biomarker-enabled clinical development.

---Lakshmi Amaravadi is the Head of Oncology Bioanalytical Strategy Function at Astra Zeneca. She oversees development and implementation of PK, PD, and Immunogenicity strategies to support preclinical & clinical programs across multiple modalities. Lakshmi has >30 years of industry experience in drug development across multiple therapeutic areas including Oncology, Neurology, autoimmune, metabolic, and rare diseases. During her tenure at Biogen, Sanofi, Shire, and Boston Pharma she contributed successfully to >45 INDs and >12 BLA/NDAs & registrations. She is an active leader of AAPS scientific communities and served as a Board Member. She is passionate about developing innovative translational approaches to Drug Development, mentoring next generation of scientists. She earned her Ph.D. in Cell, Molecular and Developmental Biology from Indiana State University & fellowship at Walther Oncology Center, Indianapolis.

---Valerie Clausen is a Director of Bioanalytical Sciences at Alnylam Pharmaceuticals. She leads the bioanalytical outsourcing group managing clinical and nonclinical sample analysis for RNAi therapeutics in development, including approved RNAi therapeutics: lumasiran, givosiran, vutrisiran and patisiran. Valerie’s role includes supporting PK, biomarkers and ADA bioanalysis. Prior to her current role, Valerie supported assay development and sample screening for discovery and development projects at Alnylam and other biopharmaceutical companies. Valerie has a doctorate in Biological Chemistry from the University of Minnesota Twin Cities and 20 plus years of experience in the discovery and development of novel biopharmaceuticals.

---Tripti Gaur is a seasoned leader in clinical biomarkers and exploratory research, specializing in biomarker strategies for cell and gene therapies targeting oncology as well as rare genetic diseases. Tripti is the Executive Director and Head of Translational Medicine at AffyImmune Therapeutics, Inc., where she supports clinical studies developing affinity-tuned CAR T cell therapies for solid cancer. Prior to this, she held similar roles with increasing responsibilities at Mustang Bio, CRISPR Therapeutics, and Takeda/Shire/Dyax. Tripti earned her PhD in molecular biology from Delhi University, India, and received a postdoctoral training in regenerative medicine and cancer biology at UMass Medical School.

---Thomas Klem is Head of Bioanalytical Sciences at Solid Biosciences supporting the company’s nonclinical and clinical programs for cardiac gene therapies.

Prior to Solid, he led the bioanalytical group at Homology Medicines for that company’s AAV-based metabolic gene therapy programs.

Tom started his bioanalytics career at Shire (now Takeda), taking roles of increasing responsibility from the bench, to vendor oversight, to bioanalytical SME on clinical program teams. From Shire, he moved to Sarepta Therapeutics, leading immunogenicity programs for exon-skipping oligonucleotide therapeutics. He received his BS degree in microbiology at UCLA and PhD in biochemistry from Purdue University.

---Ramakrishna (Rama) Boyanapalli is the Senior Director at Bristol Myers Squibb, specializing in translational and biomarker strategy and discovery. With over 18 years of industry experience, Rama has made significant contributions to neuroscience and immunology, focusing on translational biology, biomarker discovery, development, and assay development. His extensive career includes pivotal roles at Wave Life Sciences, Shire Pharmaceutics (now part of Takeda) and Nivalis Therapeutics. Rama's expertise and leadership continue to drive advancements in biomarker research, enhancing the development of innovative therapeutic strategies.

---Anka Ehrhardt is a biophysicist with a Ph.D. in human physiology. She has led successful teams in industry, including at Merck and BMS, driving biomedical research through innovative technologies and strategies. Dr. Ehrhardt designed and implemented clinical biomarker strategies for Huntington's disease at CHDI Foundation. Currently, at Merck, she provides strategic and technical leadership for the development of biologics products.