Slide 1: Key challenges and lab solutions in your global cell and gene therapy trials
Presentation Topic Key challenges and lab solutions in your global cell and gene therapy trials Dr. Maryland Franklin Vice President and Enterprise Head Cell and Gene Therapy Labcorp ©2024 Labcorp. All rights reserved. 1
Slide 2: Key Challenges and Lab Solutions in your Global Cell and Gene Therapy Trials
Key Challenges and Lab Solutions in your Global Cell and Gene Therapy Trials Maryland Franklin, PhD Vice President and Enterprise Head, Cell and Gene Therapy March 28, 2024 + ©2024 Labcorp. All rights reserved. 879410
Slide 3: Presentation agenda
Presentation agenda 1. Cell & gene therapy landscape in Asia Pacific 2. Labcorp’s global cell & gene therapy approach 3. Key challenges and laboratory solutions for globalization of cell & gene therapies ©2024 Labcorp. All rights reserved. 3
Slide 4: Cell and Gene Therapy Landscape – Asia Pacific
Cell and Gene Therapy Landscape – Asia Pacific ©2024 Labcorp. All rights reserved. 4
Slide 5: Cell and gene therapy landscape – 2023
LABCORP CELL & GENE THERAPY Cell and gene therapy landscape – 2023 North Asia Europe Total (includes all America Pacific regions) Developers 1,184 925 568 2,762 Clinical Trials 978 805 360 1,920 Investment $8.1B $2.1B $1.2B $11.7B Q4 2023 Report, Feb 2024 and 2023 State of the Industry, Jan 2024 All
Slide 6: Cell and gene therapy landscape – Asia Pacific
LABCORP CELL & GENE THERAPY Cell and gene therapy landscape – Asia Pacific Therapeutic class of all active assets (n=1307) 69% of all assets are in preclinical development Citeline Data as of February 9, 2024 Filtered by Development Status - Compounds (is Active), Drug Country (is China, S. Korea, S
Slide 7: Cell therapy landscape – Asia Pacific
LABCORP CELL & GENE THERAPY Cell therapy landscape – Asia Pacific Active assets by drug country region (n=2,109) Therapeutic class of assets in Asia (n=889) Drug Country Number Asia Country Number Region of Assets of Assets Asia 889 China 525 Europe EU 206 Japan 96 Europe Non-EU 109 South Korea 168
Slide 8: AAV gene therapy landscape – Asia Pacific
LABCORP CELL & GENE THERAPY AAV gene therapy landscape – Asia Pacific Active assets by drug country region (n=460) Drug Country Number Asian Country Number Region of Assets of Assets Asia 92 China 54 Europe EU 53 Japan 14 Europe Non-EU 33 South Korea 23 North America 267 Singapore 1 Oceania 9 South
Slide 9: Global Cell and Gene Therapy Approach - Labcorp
Global Cell and Gene Therapy Approach - Labcorp ©2024 Labcorp. All rights reserved. 9
Slide 10: CGT dedicated leadership
LABCORP CELL & GENE THERAPY CGT dedicated leadership Brian Caveney, MD Maryland Franklin, PhD Jon DiVincenzo VP and Enterprise Head of Cell EVP and President, Central Laboratories EVP and President, Early Development and Gene Therapy and International Research Laboratories and Chief Medical and Scie
Slide 11: Expertise and insights across development stages
LABCORP CELL & GENE THERAPY Expertise and insights across development stages 750+ PRECLINICAL STUDIES Conducted using cell & gene products • Supporting novel & in the last 5 years (mostly in vivo) complex testing programs CMC Analytical Testing HELPED ADVANCE • Bringing new therapies to EIGHT FDA ap
Slide 12: Commitment to cell & gene therapy
LABCORP CELL & GENE THERAPY Commitment to cell & gene therapy Labcorp has deep experience & expertise with a commitment for growth 90% Growing Dedicated Oncology* capacity/ Including 6 of 6 Specialized C> Team capabilities in global strategy, client CAR-T therapies C> facilities Molecular BioA,
Slide 13: Cell and gene therapy – APAC
LABCORP CELL & GENE THERAPY Cell and gene therapy – APAC Proactively prepare for market trend and client need Building a Cross-Functional China CGT Team Multi-Disciplinary CGT Solution Enabling Client‘s Success • Core team in China including CLS, BioA and Safety, Efficacy, Target Identification Immu
Slide 14: A hybrid model supporting commercialization within APAC or going global
A hybrid model that supports your commercialization vision within the APAC region or going global with your therapeutic Global network and capabilities to support APAC biotechs going global Excellent delivery in APAC through • Global standard execution • Unmatched regulatory leadership with proven t
Slide 15: Key Challenges in Globalization of CGTs
Key Challenges in Globalization of CGTs ©2024 Labcorp. All rights reserved. 15
Slide 16: Challenges in cell and gene therapy development
Challenges in cell and gene therapy development Regulatory Study Global Regulatory Expertise and Guidelines & Complexity & Environment Precedents Clinical Design Experience There is Continuity much to Outsourcing Advances in Study Throughout Capabilities and the Technology consider. Decisions & Mode
Slide 17: Five key challenges in global development of CGTs
Five key challenges in global development of CGTs Biomarkers and companion diagnostics 1 Manufacturing and CMC analytical testing • Scaling up and scaling out 4 • Patient identification • Patient enrollment • Consistency of product • Patient monitoring • Potency testing • Market access • CQAs 2 Nonc
Slide 18: Chemistry Manufacturing and Controls (CMC) Analytical Testing
1 Chemistry Manufacturing and Controls (CMC) Analytical Testing ©2024 Labcorp. All rights reserved. 18
Slide 19: Manufacturing and CMC analytical testing is a key challenge
Manufacturing and CMC analytical testing is a key challenge Problem Statement • Cell and gene therapies are scientifically and technically complex, and due to the novelty and complexity of the molecular characterization and manufacturing processes, the validation of analytical methods can be especia
Slide 20: Nonclinical Testing
2 Nonclinical Testing ©2024 Labcorp. All rights reserved. 20
Slide 21: Early Development nonclinical lab capabilities and bioanalytical solutions
Early Development nonclinical lab capabilities and bioanalytical solutions for cell & gene therapy Ann Arbor, MI – Murine - Discovery • Preclinical Oncology (PCO) Models Huntingdon, UK • In vivo Non-Viral Gene Therapy * • Lead Asset Adoptive Cell Identification Safety/Tox Indianapolis, IN • In vivo/
Slide 22: Expanded offerings for new modalities, same scientific excellence
MOLECULAR BIOANALYTICAL Expanded offerings for new modalities, same scientific excellence Capabilities, technologies and automation 3 global sites • 3,000+ Nonclinical, clinical • Breadth of technology • committed to delivering results 80+ 70+ PCR assays and global staff cell and gene 50+ Comprehens
Slide 23: Multiple global teams support a fast-track gene therapy program
GENE THERAPY FOR A RARE NEURODEGENERATIVE DISORDER Multiple global teams support a fast-track gene therapy program THE CLIENT NEEDED: Early development support to Strong partnership for IND Reliable support & rapid advance from preclinical to program, GLP studies, CMC & turnaround to clinical stage
Slide 24: Clinical Development and Testing Strategies
3 Clinical Development and Testing Strategies ©2024 Labcorp. All rights reserved. 24
Slide 25: Global central labs and related specialty testing facilities
CENTRAL LABORATORIES Global central labs and related specialty testing facilities Indianapolis, IN Greenfield, IN Harrogate, U.K. Esoterix York, U.K. Madison, WI Westborough, MA Monogram Biosciences Dianon, Esoterix South San Francisco, CA OmniSeq Shelton, CT Mechelen, Belgium Colorado Coagulation L
Slide 26: Global cell & gene therapy experience
LABCORP CELL & GENE THERAPY Global cell & gene therapy experience Key therapeutic area experience with expertise across development phases Oncology Hematology Global Central Labs, 750+ in vivo experience BioA, CMC Ophthalmology studies across early- and Analytical Combined across US + EU + APAC late
Slide 27: APAC cell & gene therapy experience
LABCORP CELL & GENE THERAPY APAC cell & gene therapy experience APAC key therapeutic area experience with expertise across development phases Oncology Local Combined experience in expertise across 50+ in vivo early-stage and Central Labs, Ophthalmology studies growing in late- BioA and I&I* Specific
Slide 28: Comprehensive laboratory support for gene therapy development
LABCORP CELL & GENE THERAPY Comprehensive laboratory support for gene therapy development Preclinical GMP CMC Release Clinical Pharmacodynamic (PD) response/efficacy/research and Critical quality attributes (CQAs) Patient selection/predictive biomarkers development of novel delivery modalities • Cli
Slide 29: Comprehensive laboratory support for adoptive cell therapy development
LABCORP CELL & GENE THERAPY Comprehensive laboratory support for adoptive cell therapy development Preclinical GMP CMC Release Clinical In vitro analysis Critical quality attributes Pharmacodynamic response/efficacy • Transduction efficiency (flow cytometry) • Vector copy number, titre and viability
Slide 30: Biomarkers and Companion Diagnostics
4 Biomarkers and Companion Diagnostics ©2024 Labcorp. All rights reserved. 30
Slide 31: Cell & gene therapy considerations for laboratory assessment
LABCORP CELL & GENE THERAPY Cell & gene therapy considerations for laboratory assessment Target Safety Patient ID/ PK/PD Engagement Assessment Stratification Cellular Therapy Related Biomarkers Gene Therapy Related Biomarkers Tumor and immune cell T cell persistence and Disease Indication Biodistrib
Slide 32: Assay/testing requirements for gene therapy programs are very complex
EXAMPLE OF TESTING COMPLEXITY IN AAV GENE THERAPY PROGRAMS The assay/testing requirements for gene therapy programs are very complex Biodistribution Gene expression (qPCR, IHC) (ELISA, IHC, RNA-scope) Transgene Analysis ddPCR tAb/nAb Potency For Titer Assays Assay Many Progressive Bespoke Testing AA
Slide 33: Patient Identification and Enrollment
5 Patient Identification and Enrollment ©2024 Labcorp. All rights reserved. 33
Slide 34: Harness the power of Labcorp diagnostic data
UNIQUE HIGH-VALUE DATA SET Harness the power of Labcorp diagnostic data to develop tomorrow’s breakthroughs >35 billion >6,500 >150 million test results diagnostic assays U.S.-based patients Real-time data Longitudinal data Test results uploaded daily View results over time • Support trial design •
Slide 35: We're dedicated to helping you advance the future of medicine
We’re dedicated to helping you advance the future of medicine We commit to providing you with the global scale and support you need—and the personalized experience you deserve. Learn more today at biopharma.labcorp.com/CGT ©2024 Labcorp. All rights reserved. 35