Agenda

8:00–8:50 AM Registration and Breakfast

8:55–9:00 AM Welcome Remarks by Hua Tu, Ph.D., CEO of LakePharma

9:00–9:35 AM Session #1 A Platform for Identifying Strategies

for Reversing Cancer and Restoring the Immune Response

Dean Felsher, MD, Ph.D. Director of Oncology Research,

Director of Translational Research and Applied Medicine at Stanford University

9:40–10:15 AM Session #2 Directed Evolution of Novel

Adeno-Associated Viral Vectors for Clinical Gene Therapy

David Schaffer, Ph.D. Director of the Berkeley Stem

Cell Center at UC Berkeley

10:20–10:40 AM Break

10:45–11:20 AM Session #3 Industrializing the Manufacture of Cell

and Gene Therapies Madhusudan Peshwa, Ph.D. Chief Technology Officer

of Global Cell Therapy at GE Healthcare

11:25–12:00 PM Session #4 Comparison of Baculovirus (BV)-Sf9

and HEK293 Expression Systems for the Production of Adeno-Associated Viral (AAV) Vectors

Michael Boychyn, Ph.D. Vice President of Process

Development at REGENXBIO Inc.

12:00–1:15 PM Lunch in Baden Room A and B

12:40–1:10 PM Parallel Lunch Workshops (see page 2)

1:15–1:20 PM Brief Remarks by Norm Garceau, Ph.D., VP, Technical Operations, LakePharma

1:20–1:55 PM Session #5 Synthetic Lethal Approaches to

Cancer Therapy Alan Ashworth. Ph.D., FRS President of Helen Diller Family

Comprehensive Cancer Center at UCSF

2:00–2:35 PM Session #6 Clinical and Pre-Clinical Approaches

Using T Regulatory Cells to Prevent or Treat Graft-versus-Host Disease

Everett Meyer, MD, Ph.D. Medical and Scientific Director,

Cellular Therapy Facility at Stanford University

2:35–3:00 PM Poster Break

3:05–3:40 PM Session #7 The Combination of Immune Cells and

Oncolytic Virus Creates A Unique Multi-mechanistic Biotherapy for Treatment of Refractory Solid Tumors

Pamela Contag, Ph.D. CEO, Bioeclipse

3:45–4:20 PM Session #8 From CRO to CRDMO, LakePharma’s

Technology Development in Meeting Next-Generation Therapeutic Needs

Hua Tu, Ph.D. CEO of LakePharma

4:30–5:30 PM Happy Hour and Networking

2019 LakePharma Symposium on Next-Generation TherapeuticsOctober 10, 2019 | South San Francisco Conference Center

Agenda

Workshop 1Panel Discussion on Cell Therapy GMP Manufacturing

LakePharma | Salon A Room

Join us as we discuss some of the common issues and latest innovations around GMP manufacturing for cell therapy applications.

Panelists

• Pam Contag, Ph.D., CEO, Bioeclipse

• Everett Meyers, MD, Ph.D., Medical and Scientific Director, Cellular Therapy Facility at Stanford University

• Andy Kogelnik, MD, Ph.D., Director, Open Medicine Institute

• Hua Tu, Ph.D., CEO, LakePharma

Workshop 2Alt-R CRISPR Genome Editing: Innovation Driving Solutions for HDR, Specificity, and Off-Target Analysis

Integrated DNA Technologies, Inc. | Salon B Room

Genome editing by CRISPR systems has proven to be groundbreaking in medical research and has great implications for the treatment of human disease. CRISPR-Cas9 and Cas12a (Cpf1) proteins introduce double-stranded breaks (DSBs) at targeted genomic loci, which are repaired by endogenous cellular pathways such as non-homologous end joining (NHEJ) and homology-directed repair (HDR). CRISPR systems demonstrate unparalleled editing efficiency in a broad range of host species and cell types but suffer from concerns related to target site specificity and editing precision.

Here, we describe methods for detecting and measuring the frequency of off-target effects (OTEs) including multiplexed enrichment for next-generation sequencing and data analysis to fully characterize on- and off-target editing events. In addition, we present novel mutant CRISPR enzymes with improved on- and off-target editing profiles: Alt-R HiFi S.p. Cas9 nuclease and Alt-R A.s. Cas12a Ultra nuclease. Finally, we describe methods to improve the rate of HDR-mediated repair by careful selection of repair template characteristics and the use of Alt-R HDR Enhancer. We implement these findings in the Alt-R CRISPR HDR Design Tool, a novel bioinformatics tool for ssDNA HDR template design.

Workshop 3Expanding SPR Throughput Orders of Magnitude to Accelerate Therapeutic Antibody Discovery

Carterra | Salon C Room

Surface Plasmon Resonance (SPR) is the de facto technique for measuring the binding kinetics and affinities of antibody interactions but has been relegated to a secondary role in discovery due to its limited throughput. Here, we introduce the Carterra LSA, a high throughput SPR platform that is disrupting antibody analytics by enabling screening and characterization to be performed in the same step. The LSA can measure the binding kinetics and affinities of hundreds of interactions in parallel and perform comprehensive epitope binning experiments on up to 384 antibodies per chip. With the additional advantages of minimal sample consumption and industry-leading analysis software, the LSA is streamlining the library-to-leads triage, saving time, cost and resources in generating clinically-ready molecules.

Workshop 4Integrated Workflow Solutions for Development of Cellular Therapies

Thermo Fisher Scientific | Salon D Room

Advancements in cellular therapies has mandated the rapid development of closed automated systems, integrated workflows and analytical solutions to increase consistency while decreasing costs. Challenges in manufacturing cell therapy products include laborious and contamination prone cell isolation techniques, scale-up of cell engineering technologies, and standardization of assays to ensure identity, purity, and potency. To address these key challenges, we have developed tools that allow for efficient and reliable isolation, engineering, and characterization of cell products.

The Rotea Counterflow Centrifuge System provides cell processing of numerous therapeutic cell types with a high level of reproducibility at every level of R&D and clinical manufacturing. The Rotea is able to separate, isolate, wash & concentrate cells while maintaining a closed system that ensures sterility of the product. Further, users can design protocols to fit their intended cell type and application allowing for a modular system that delivers high levels of cell recovery and viability. Using an integrated workflow with qualified media and reagents, isolated cells can be engineering using the LV-MAX Lentiviral production system, which facilitates production of high titer virus in a scalable manner. Finally, identity and purity of the resulting cell therapy product can be measured using the traditional flow-based Essential Human Phenotyping kits and a novel qPCR-based PureQuant Methylation assay. Importantly, the PureQuant assays remove variability and input requirements associated with flow cytometry, allowing for decreased cell input and standardized data interpretation. These assays, together with downstream functional assays, provide comprehensive methods to qualify cell therapy products. Together, these tools enable a streamlined workflow to develop cell products from R&D through clinical manufacturing.

All workshops are from 12:40–1:10 PM.

Thank You To Our Sponsors

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More information, earlier.

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Explore the full kinetic and epitope diversity of large antibody libraries at the start of the drug discovery process. Carterra’s LSA

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Find out more at: thermofisher.com

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