The 5th @Philly Cell and Gene Therapy Annual Conference — organized by SAPA-GP — marks a five-year milestone, bringing together scientists, clinicians, entrepreneurs, regulators, investors, and industry leaders across one of the world’s most dynamic CGT hubs.
Two days of expert-led scientific sessions, panel discussions, company showcases, and networking across Gene Therapy, CMC, Cell Therapy, RNA Therapy, Clinical & Regulatory, Business, and Career Development. Conference Chair: Xinjun Zhang, Ph.D. (President, SAPA-GP). Held at the Sheraton Valley Forge Hotel, King of Prussia, PA.
Advancing Genetic Medicine — Peter Marks, Ph.D., M.D., SVP, Molecule Discovery & Head of Infectious Disease, Eli Lilly and Company. (Session Chair: Haichen Yang, M.D., M.B.A.)
Hemophilia Gene Therapy and Lessons from Translational Investigation — Lindsey George, M.D., Director of Clinical In Vivo Gene Therapy, University of Pennsylvania & Children’s Hospital of Philadelphia.
Ocular Gene Therapy — From Rare to Common Diseases — Abhi Gupta, M.B.A. (Ocugen).
Beyond Anti-VEGF: Emerging Frontiers in AMD — Yin Shen, M.D., Ph.D. (Augelux Therapeutics).
Developing Safe, Effective and Affordable AAV Gene Therapy Drugs — Daozhan Yu, Ph.D. (AAVnerGene).
What AUC can teach us about AAVs and LNPs — Borries Demeler, Ph.D. (Canada 150 Research Chair).
Cell-Derived Nanoparticles for T-Cell Activation — Pedro A. Moura, Ph.D. (BlueWhale Bio).
CMC Isn’t the Problem — When You Start Is — Patrick M. Dentinger (CellPort).
Building Success: the Business Case for Advanced Therapies in Greater Philadelphia (Cent II) — Moderator: Bryan Tsao, Ph.D. (Chamber of Commerce): Rebecca Grant, DVM (Philadelphia Dept. of Commerce); Jeff Alexander, Ph.D. (Lilly Gateway Labs); Kurt Imhof (Life Sciences PA).
The Current Trends of Investment and Deal-Making in Biotech (Cent III) — Moderator: Leon Tang, Ph.D. (InScienceWeTrust): Al Hawkins (Syndeo); Pavel Khrimian (Deka Biosciences); Madee Gooriah (Legend Biotech); Rui Che (KPMG); Morgan Conn (Pharmacallis Advisory).
Mianus Accelerator: Clinical Development with “China Speed” — Steve Yang, M.B.A., C.F.A. (Merit/Mianus Capital, Cent I).
AAV Viral Vectors in Non-Human Primates — Guodong Zhang, Ph.D. (TriApex Laboratories, Cent II).
BioRMB — Next-Generation Purification for AAV — Oleg Shikazh (ChromaTan, Wayne).
Session Chair: CJ (Chunjuan) Song, Ph.D., Conference Co-director, VP R&D, Exegenesis Bio.
Receptor-targeting AAV Variants for Efficient CNS Transduction — Zhenhua Wu, Ph.D. (Exegenesis Bio).
Symptomatic delivery of a BBB-crossing AAV to restore TDP-43 function — Philip C. Wong, Ph.D. (Johns Hopkins).
First-in-Human Astrocyte-to-Neuron Conversion in Parkinson’s — Jingmin Zhou, Ph.D. (Genemagic Bio).
Advancing siRNA Manufacture — Chris (Xiaocen) Li, Ph.D. (Hongene Biotech).
Quality Attributes of Empty, Partial, Full and High-Density rAAV Capsids — Bashkim Kokona, Ph.D. (Genentech).
From Ex Vivo to In Vivo CAR-T: Lentiviral Vector Manufacturing — Eric Lin (ProBio).
Crystal Jin, Ph.D. (Bound Therapeutics); Sarah Johnson, Ph.D., M.B.A. (Jecho Laboratories); Dora Mitchell, Ph.D. (Interius Biotherapeutics); Stella Vnook, Ph.D. (Aviva Ventures); Jing Yang, Ph.D. (BaseCure Therapeutics).
Company Showcase (Cent II): Building Effective Preclinical Strategies for CGT — Yan Zhou, M.D., Ph.D. (Medicilon USA).
From Ex Vivo to In Vivo Therapies (Grand): OTC-HOPE: First In-Vivo, Liver-Directed, AAV-Mediated Gene Insertion in Infants — George A. Diaz, M.D., Ph.D. (iECURE); plus Vibha Jawa, Ph.D. (EpiVax).
CARs Inside (Cent II): In Vivo CAR-T Revolution — Zhenghong Gao, Ph.D. (Uni-Pioneers); Transient Immunomodulation with mRNA-LNPs — Edward F. Kreider, M.D., Ph.D. (UPenn); circRNA-LNP iPSC Reprogramming & in vivo CAR-T — Jeffrey Hung, Ph.D. (uBrigene).
Lake N. Paul, Ph.D. (BioAnalysis), followed by a panel discussion with all CMC speakers.
Bojing Fu (Philadelphia Dept. of Commerce); John Hu, Ph.D. (HIO Biosciences); Yi Xie (InvestHK); Laurent Metz (Cortexx Medical Intelligence).
Immune Responses to AAV Gene Delivery for Muscle and CNS — Terence Flotte, M.D., Provost & Dean, UMass Chan Medical School.
Advancing Precision Medicine: Oligonucleotide and Genome Editing Therapeutics — Laura Sepp-Lorenzino, Ph.D., Principal, GNMmeds LLC.
How to apply and operate gene therapy IIT in China — Fang Lu, M.D. (West China Hospital).
UGX-201 Optogenetic Gene Therapy for Advanced Retinitis Pigmentosa — Peirong Lu, Ph.D., M.D. (First Affiliated Hospital of Soochow University).
Navigating IIT in China — Heping Xie, M.S. (PanaCRO). Ocular Gene Therapy / Glaucoma — Bo Liang, Ph.D. (IVIEW). Panel discussion follows, joined by Cheng Wang, Ph.D. (Innovec Biotherapeutics).
Bioanalysis of Antibody-Oligonucleotide Conjugates — Long Yuan, Ph.D. (Biogen).
Bioanalytical & Immunogenicity Strategies for RNA Therapeutics — Rashmi Jalah, Ph.D. (Johnson & Johnson).
Challenges and Solutions in Bioanalysis for RNA Therapies — Yunlin Fu, Ph.D. (Novartis).
Reprogramming Immune Interactions with Immune Engineering & Genome Editing — Xiaoyu (Ariel) Zhou, Ph.D. (Wistar Institute / UPenn).
Universal Cell Therapy for Solid Tumors — Adam Snook, Ph.D. (Thomas Jefferson University).
In Vivo CAR-T and Gene Editing: the Manufacturing Reality — Peng Wang, Ph.D. (Lonza).
Panelists: Adetoun Adeniji-Adele, Ph.D. (Integral Molecular); Phil Brooks, B.S. (Graduate Philadelphia); Ralf D. Wiedemann, J.D. (Immigration Attorney); Linda Zhao, M.B.A., M.S. (MSQ Ventures).
Business (Wayne): Science, Interrupted — A Biotechnology Origin Story — David Cole, Ph.D., M.B.A. (Science History Institute).
MCE’s GMP Small Molecules Empowering Cell and Gene Therapy — Wei Li, Ph.D. (MedChemExpress, Cent I).
Unlocking the Frontier of mRNA-Based Therapeutics Analysis with CE — Mukesh Malik, Ph.D. & Novel Analytical Workflows for CGT — David Colquhoun, Ph.D. (Sciex, Cent II).
Paving the Way for Gene Therapy Approval in an Evolving Regulatory Environment — Jim Wang, Ph.D., M.B.A. (Regeneron).
A Regulatory Playbook: Avoiding Disasters and Finding Wins — Daniela Drago, Ph.D. (NDA Partners).
What to Expect from FDA in 2026 and Beyond for CGT — Jae Kim, J.D. (DLA Piper).
DMPK Considerations for Oligonucleotide-Protein Conjugates — Babak Basiri, Ph.D. (Dyne Therapeutics).
Predicting Human PK/Dose of GalNAc-Conjugated siRNAs — Xianwei Zhang, Ph.D. (Alnylam).
Drug Metabolism & PK of Oligonucleotides — Ganesh Moorthy, Ph.D. (Frontage Laboratories).
Targeted Nanomedicine Across Biological Barriers — Jia Nong, Ph.D. (UPenn).
Bespoke Gene Editing Therapies for Urea Cycle Disorders — Sarah A. Grandinette, Ph.D. (UPenn).
Precision A3G Base Editors for Disease Modeling and Correction — Tyler Daniel, Ph.D. (UPenn).
Panelists: Wei Gao, Ph.D. (Merck); Joanna Ghayad, Pharma.D. (Executive Coach); Evelyn Guo, M.D., M.B.A. (Genmab); Michael Kalos, Ph.D. (Next Pillar Consulting); Donna Serdula, B.A. (Vision Board Media).
Oncolytic Virus Therapy for Triple-Negative Metastatic Breast Cancer — Ashok Srivastava, M.D. & Elias Tharakan, B.S. (AyurDatta Solutions).
Development of Novel CAR Products: Historical Perspective and Future Considerations — John Rossi, M.S. (New Co).
RNA Oligonucleotide Therapeutics: Toxicity, PD, PK & First-in-Human Dose — Xiaochun Chen, Ph.D. (Joinn Laboratory).
Chemistry-Guided RNA Therapeutics via Anticlastic RNAs — Bhaskar Halami, Ph.D. (Alloy Therapeutics).
ModDetect Antibodies for Detection of Modified Oligonucleotides — Seth Farrier, B.S. (Rockland Immunochemicals).
Panelists: Xiao Huang, Ph.D. (Drexel); Xiaoyu (Ariel) Zhou, Ph.D. (Wistar / UPenn); Peng Wang, Ph.D. (Lonza).
The 5th @Philly Cell & Gene Therapy Annual Conference is made possible by the generous support of our sponsors.
Partners: DIA · The Chamber of Commerce for Greater Philadelphia · iSWT Community · bioSeedin · MBoC (ASCB) · Science History Institute · BioLabs for Advanced Therapeutics · Hope for Rare Foundation · Frontage · Life Sciences Pennsylvania · Keystone LifeSci Collaborative · Pennsylvania Biotechnology Center.
Conference Chair: Xinjun Zhang, Ph.D. · Co-Chairs: Lu Wang, Ph.D., M.B.A. and Evelyn Guo, M.D., M.B.A. Organized by SAPA-GP.
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