2015 Archived Content

Cambridge Healthtech Institute’s Inaugural 
Gene Editing for Immunotherapy

Enabling Technologies to Advance Immune-Based Therapies

August 27-28

Enabling technologies for immune-based therapies are evolving just as rapidly as immunotherapies themselves to keep up with the growing interest and demand.  This has brought new life to an old concept: using a patient’s own immune cells to attack disease cells. Cambridge Healthtech Institute’s Inaugural Gene Editing for Immunotherapy will focus on platforms that enable targeting of a broader range of molecules associated with disease biology. Special focus will be given to emerging gene editing techniques using CRISPR and CAS9 and next-generation sequencing in support of immunotherapies. Finally, studies that showcase more personalized approaches to immunotherapies will be presented. Overall, this event will provide attendees with a comprehensive view into the tools moving immunotherapies forward from bench to bedside.

Day 1 | Day 2 | Download Brochure | Speaker Biographies
UD: Unpublished Data | CS: Case Study

Recommended Dinner Short Course*
Adoptive Therapy with CAR T Cells
*Separate registration required


7:30 am Registration & Morning Coffee


8:10 Chairperson’s Opening Remarks

Gary K. Lee, Ph.D., Associate Director, Genome Editing, Sangamo BioSciences

Rodger Novak

Rodger Novak, M.D., Ph.D., CEO & Founder, CRISPR Therapeutics 

Within less than two years after its inception the CRISPR-Cas system has truly democratized genome editing with many areas of research being transformed due to ease of use and broad applicability of the technology. With such an enormous impact on many areas of life science the translation of the CRISRP-Cas technology into human therapeutics seems to be a logical consequence. However, besides many exciting opportunities a number of challenges will have to be addressed; some of them more obvious than others.

9:00 Characterization of Cas9-Mediated Genome Editing in Human T Cells

Grant-WelsteadG. Grant Welstead, Ph.D., Scientist II, Editas Medicine

Genome editing via CRISPR/Cas9 promises to provide a novel class of therapies for a variety of human diseases. To better understand the utility of Cas9-mediated gene editing for engineering human T cells, we surveyed a variety of delivery modalities and assessed the functionality of different Cas9 variants in human T cells. Data from this work will be presented.

9:30 Late-Breaking Presentation: Targeted Activation of Endogenous Human Regulatory Elements by a CRISPR-Cas9-Based Acetyltransferase

Isaac HiltonIsaac B. Hilton, Ph.D., Postdoctoral Fellow, Biomedical Engineering and Center for Genomics and Computational Biology, Duke University

Technologies that enable targeted manipulation of epigenetic marks could be used to precisely control cell phenotype or interrogate the relationship between the epigenome and transcriptional control. Here, we describe a programmable, CRISPR-Cas9-based acetyltransferase consisting of the nuclease-null dCas9 protein fused to the catalytic core of the human acetyltransferase p300. The fusion protein catalyzes acetylation of histone H3 lysine 27 at its target sites, leading to robust transcriptional transactivation from targeted promoters and enhancers. In contrast to previous dCas9-based activators, the acetyltransferase activates genes from enhancer regions and with an individual guide RNA. These results support the concentrated acetylation of chromatin as a causal mechanism of transactivation and provide a robust tool for manipulating gene regulation.


10:00 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 In vivo Genome Editing using Staphylococcus aureus Cas9

Ann-RanFei Ann Ran, Ph.D., Post-Doctoral Fellow, Laboratory of Dr. Feng Zhang, Broad Institute; Junior Fellow, Harvard Society of Fellows

The Cas9 nuclease from the bacterial CRISPR (clustered regularly interspaced short palindromic repeats) adaptive immune system is a powerful tool for facilitating targeted genome editing in a number of eukaryotic species. Cas9 can be programmed by short guide RNAs to induce multiplexed gene knockout or homology-directed repair with high efficiency. Recently, we have identified an additional small Cas9 nuclease from Staphylococcus aureus that can be packaged, along with its guide RNA, into a single adeno-associated virus (AAV). We demonstrate the use of this system for effective gene modification in adult animals and further expand the Cas9 toolbox for in vivo genome editing.

11:15 Zinc Finger Nuclease Driven Gene Modification of T Cells

Gary-LeeGary K. Lee, Ph.D., Associate Director, Genome Editing, Sangamo BioSciences

The ability to engineer the precise genetic modification of human cells to extend their potential therapeutic application is now being realized via the use of zinc finger nucleases (ZFNs). ZFNs are customizable, sequence-specific endonucleases that can be designed to introduce a discrete cleavage event at any user-chosen location within the genome. This talk will describe recent applications of this technology to genetically modify T cells.

11:45 PANEL: Advantages and Disadvantages of Different Gene Editing Technologies

  • Current and future therapeutic applications
  • Generating knock-outs
  • Checking for off-target effects
  • Future and emerging gene editing techniques and technologies

Moderator: Gary K. Lee, Ph.D., Associate Director, Genome Editing, Sangamo BioSciences


Rodger Novak, M.D., Ph.D., CEO & Founder, CRISPR Therapeutics

Fei Ann Ran, Ph.D., Post-Doctoral Fellow, Laboratory of Dr. Feng Zhang, Broad Institute and Junior Fellow, Harvard Society of Fellows

André Choulika, Ph.D., CEO, Cellectis 


12:15 pm Sponsored Presentation (Opportunity Available)

12:30 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:00 Session Break


2:00 Chairperson’s Remarks

Alexander Marson, M.D., Ph.D., UCSF Sandler Faculty Fellow, Medicine and Diabetes Center, University of California San Francisco

2:05 Gene Editing in T Cells for Enhanced Adoptive Immunotherapies

Andrew-ScharenbergAndrew M. Scharenberg, M.D., Professor, Pediatrics; Adjunct Professor, Immunology, University of Washington School of Medicine; Co-Director, Program for Cell and Gene Therapy, Seattle Children’s Hospital

Adoptive transfer of engineered autologous primary human T cells has emerged as a potentially transformational approach to therapy of cancer and immunologic diseases. Rare cleaving nuclease technologies are an important enabling technology for achieving the promise of adoptive T cell therapies, as they allow for precise engineering of targeting, function, and control mechanisms. This talk will cover translational application of rare cleaving nuclease technologies using newly developed methods to facilitate high efficiency recombination and multiplex genomic modifications.

2:35 Engineering Human T Cell Circuitry

Alexander Marson, M.D., Ph.D., UCSF Sandler Faculty Fellow, Medicine and Diabetes Center, University of California San Francisco

T cell genome engineering holds great promise for cancer immunotherapies. We have overcome the poor efficiency of CRISPR/Cas9 genome engineering in primary human T cells using Cas9: single-guide RNA ribonucleoproteins (Cas9 RNPs). Cas9 RNPs can promote targeted genome sequence replacement in primary T cells by homology-directed repair (HDR), which was previously unattainable. This provides technology for diverse experimental and therapeutic applications.

3:05 Allogeneic Universal CAR-T Cells Engineered With TALEN®

André ChoulikaAndré Choulika, Ph.D., CEO, Cellectis

The allogeneic production of CAR T-cells would allow the development of cost-effective, “off-the-shelf” frozen products that are capable of being stored and distributed worldwide. In addition, it would render the product available to patients that do not have enough T-cells to undergo an autologous therapeutic process. This talk will cover the high performance of TALEN® technology in T-cell genome editing from single allele modification to simultaneous multiple allele knock-outs.

3:35 Refreshment Break


4:05 Engineering T Cells through Genome Editing

Matthew-PorteusMatthew Porteus, M.D., Ph.D., Associate Professor, Pediatrics, Cancer Biology, Stanford University School of Medicine

T-cells are one of the most potent therapeutic cell types in the body because they can mediate beneficial effects through the elimination of infections and cancers but also can mediate pathologic effects through inflammation and auto-immunity. Moreover, they are also the primary pathologic target for HIV infection. We have been using nuclease-mediated genome editing to engineer T-cellls that contain multiple genetic blocks to HIV infection. In this talk, I will discuss the advances we have made in using engineered nucleases, particularly the CRISPR-Cas9 system using modified synthetic single-guide RNAs, as a method to efficiently edit the genome of T-cells.


4:35 Discovery of Novel Targets for Genetic Engineering of Tumor-Specific T Cells

Kai-WucherpfennigKai W. Wucherpfennig, M.D., Ph.D., Professor, Dana-Farber Cancer Institute and Harvard Medical School

A key challenge in the cancer immunology field is the discovery of the most suitable targets for therapeutic intervention. We recently reported a novel RNA-interference (RNAi)-based approach for systematic discovery of such targets in the tumor microenvironment in vivo utilizing pooled shRNA libraries as a screening tool. I will discuss how this unbiased method can be used to develop innovative cancer therapeutics.

5:05 End of Day

5:00 Dinner Short Course Registration

Day 1 | Day 2 | Download Brochure | Speaker Biographies
UD: Unpublished Data | CS: Case Study


8:00 am Morning Coffee


8:25 Chairperson’s Opening Remarks

Adrian Bot, M.D., Ph.D., Vice President, Translational Medicine, Kite Pharma, Inc.

8:30 PANEL: Characterizing T Cell Therapies in the Clinic

  • Mechanistics aspects of adoptive cell therapy
  • On-treatment and predictive immunotherapy biomarkers
  • Relationships between clinical and immunological response

Moderator: Adrian Bot, M.D., Ph.D., Vice President, Translational Medicine, Kite Pharma, Inc.


Chantale Bernatchez, Ph.D., Assistant Professor, Melanoma Medical Oncology, MD Anderson

Luise U. Weigand, Ph.D., Team Leader, Cell Biology, Immunocore Ltd.

9:00 FEATURED PRESENTATION: Personalized Cellular Immunotherapy against Novel Cancer Targets

Oliver Schoor, Ph.D., Director, Discovery, immatics biotechnologies GmbH


9:30 Engaging Macrophages as Effectors of Cancer Immunotherapy

Kipp A. Weiskopf, Ph.D., Research Scientist, Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine

Macrophages are innate immune cells that possess immense potential as effectors of cancer immunotherapy. Their ability to attack cancer can be unleashed by disrupting the CD47-SIRPa interaction, which acts as a myeloid-specific immune checkpoint. CD47-blocking therapies synergize with monoclonal antibody therapies, and their combination could yield personalized immunotherapeutic regimens that could be tailored to individual patients or tumor subtypes.

10:00 Coffee Break

Harjeet Singh
10:30 Engineering T Cells for One and All

Harjeet Singh, Ph.D., Research Investigator, Pediatrics, MD Anderson Cancer Center

The administration of genetically modified T cells is championed by academia and industry alike. T cells are precision tools and one challenge now is to render them suitable for broad appeal as immunology is translated into immunotherapy. To help democratize T-cell therapy, I will reveal strategies how immune cells can be engineered ex vivo using a transposon/transposase system for in vivo applications. I will discuss how this non-viral approach to gene therapy can be combined immunotherapy to redirect specificity and improve the effector functions of T cells manufactured for clinical trials. For example, T cells can be genetically modified to express chimeric antigen receptors (CARs) to redirect specificity for cell-surface tumor-associated protein antigens and carbohydrates on fungi. I will reveal how the Sleeping Beauty (SB) system can be adapted and used to stably express CARs and TCRs to improve the therapeutic potential of clinical grade T cells. These clinical data serve as a foundation for additional genetic engineering to co-express transgenes to improve persistence as well as provide the opportunity for genome editing to eliminate undesired endogenous genes to improve T-cell potency and broaden their distribution and application.


11:00 Novel Tumor Target Discovery Approaches for Cancer Immunotherapy

Pranay Khare, Ph.D., Consultant, Gene and Cell Therapy

Limited data and success has been observed in solid tumors using currently available tumor antigen. We recently published our Phase I clinical findings on “Delivery of Carcinoembryonic antigen targeted chimeric antigen receptor T cells (CAR-T) cells in solid tumor”. Tumor antigen’s specificity, targeting capability, exclusivity and accessibility determine the therapeutic benefit and toxicity. This talk will discuss the use of novel patented technology that can identify novel glycosylated polypeptides, proteins and/or receptors on tumor of your choice.

11:30 Close of Gene Editing for Immunotherapy

Day 1 | Day 2 | Download Brochure | Speaker Biographies

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