Cambridge Healthtech Institute’s Inaugural

New Frontiers in Gene Editing

Transitioning From the Lab to the Clinic

February 19-20, 2015 | The InterContinental San Francisco | San Francisco, CA
Part of the 22nd International Molecular Medicine Tri-Conference


Gene editing is rapidly progressing from being a research/screening tool to one that promises important applications downstream in drug development and cell therapy. Cambridge Healthtech Institute’s inaugural symposium on New Frontiers in Gene Editing will bring together experts from all aspects of basic science and clinical research to talk about how and where gene editing can be best applied. What are the different tools that can be used for gene editing, and what are their strengths and limitations? How does the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas system, compare to Transcription Activator-like Effector Nucleases (TALENs), zinc finger nucleases (ZFNs) and other systems and where are they being used? Scientists and clinicians from pharma/biotech as well as from academic and government labs will share their experiences leveraging the utility of gene editing for functional screening, creating cell lines and knock-outs for disease modeling, and for cell therapy.

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Thursday, February 19

7:30 am Registration and Morning Coffee


9:00 Chairperson’s Opening Remarks

Joseph C. Wu, M.D., Ph.D., Director, Stanford Cardiovascular Institute and Professor, Department of Medicine/Cardiology & Radiology, Stanford University School of Medicine


Genome Edited Induced Pluripotent Stem Cells (iPSCs) for Drug Screening

Joseph C. Wu, M.D., Ph.D., Director, Stanford Cardiovascular Institute and Professor, Department of Medicine/Cardiology & Radiology, Stanford University School of Medicine

Dr. Wu’s lab is focusing on human iPSCs for cardiac disease modeling, drug discovery, and regenerative medicine. We have been using ZFN, TALEN, and CRISPR to create isogenic iPSC lines that carry various cardiovascular diseases (e.g., LQT, HCM, DCM) as well as reporter genes for in vitro and in vivo tracking. We are also using this approach for improving the efficiency of high throughput drug screening.

9:40 Exploration of Cellular Stress and Trafficking Pathways Using shRNA and CRISPR/Cas9-Based Systems

Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University

We have developed high-complexity shRNA libraries (25 shRNAs/gene) that greatly reduce false negatives/false positives for RNAi screens, and have adapted these libraries to knock down gene pairs to perform systematic genetic interaction maps in mammalian cells. We have used these maps to study ER-trafficking toxins, identify novel protein complexes, and gain insights into retrograde trafficking. We are using this strategy together with the CRISPR/Cas9 system for functional genomics efforts and identification of novel drug targets.

10:10 Gene Editing in Patient-Derived Stem Cells for in vitro Modeling of Parkinson’s Disease

Birgitt Schuele, M.D., Associate Professor and Director of Gene Discovery and Stem Cell Modeling, The Parkinson’s Institute

Recent development of “genome editing” technologies to introduce site-specific genome modifications in disease relevant genes lay the foundation for new approaches to understand direct genotype-phenotype correlations at the molecular level in human disease. With the introduction of next-generation sequencing, many new genetic variants have been identified in Parkinson’s related genes; however, it is currently challenging to interrogate their functional relevance. Human-derived genome edited cell lines will be a way to analyze variants in a high-throughput format.

10:40 Coffee Break with Exhibit and Poster Viewing


11:15 Massively Parallel Combinatorial Genetics to Overcome Drug Resistance in Bacterial Infections and Cancer

Timothy K. Lu, M.D., Ph.D., Associate Professor, Synthetic Biology Group, Department of Electrical Engineering and Computer Science and Department of Biological Engineering, Synthetic Biology Center, Massachusetts Institute of Technology

Complex biological phenotypes can result from the interplay of multiple genetic factors but deciphering the multifactorial genotypes that underlie these phenotypes is challenging. We have developed technologies for the scalable and barcoded assembly of high-order combinatorial genetic libraries. These strategies enable multiplexed tracking of individual genetic combinations with next-generation sequencing in pooled screens. We have used these technologies to perform massively parallel combinatorial genetics in bacteria and human cells and to modulate relevant phenotype.

11:45 Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

Daniel G. Anderson, Ph.D., Professor, Department of Chemical Engineering, Institute for Medical Engineering & Science, Harvard-MIT Division of Health Sciences & Technology and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing and characterizing RNA delivery and gene editing systems. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery RNA, both in vitro and in vivo. A number of delivery formulations have been developed with in vivo efficacy, and show potential therapeutic application for the treatment of genetic disease, viral infection, and cancer.

12:15 pm Advanced Breeding in Plants Using Precision Genome Editing

Greg Gocal, Senior Vice President, Research and Development, Cibus

Precision editing of crop genomes is a central tool in advancing agricultural biotechnology. Cibus, founded in 2001, leads this space with its technology platform called RTDS™. Combining nucleases with GRONs yields rapid and precise spelling changes in native genes.

12:30 Session Break

12:40 Luncheon Presentation: Loss-of-Function Genetic Screening with shRNA and CRISPR Libraries

Paul Diehl, Ph.D., Director, Business Development, Cellecta, Inc.

Genome-wide loss-of-function screens provide a direct approach to identify the genes regulating biological responses and find new therapeutic targets. While RNAi screens have proven an effective tool, CRISPR/Cas9 provides an alternative approach. To complement our established shRNA screening platform, we have developed pooled format genome-wide modular sgRNA libraries for cost-effective CRISPR knockout screens. Pooled sgRNA and shRNA libraries were used to identify lethal interactions in isogenic PDX-derived cell line pairs.

1:15 Session Break


1:50 Chairperson’s Remarks

Eric N. Olson, Ph.D., Professor and Chairman, Department of Molecular Biology, The University of Texas Southwestern Medical Center.


Preventing Muscle Disease by Genomic Editing

Eric N. Olson, Ph.D., Professor and Chairman, Department of Molecular Biology, The University of Texas Southwestern Medical Center

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. We used CRISPR/Cas9-mediated genome editing to correct the dystrophin gene (Dmd) mutation in the germline of mdx mice, a model for DMD. The degree of muscle phenotypic rescue in mosaic mice exceeded the efficiency of gene correction, likely reflecting the progressive contribution of corrected cells to regenerating muscle. Progress toward the correction of DMD in adult myofibers will be discussed.

2:30 CRISPR-Cas: Tools and Applications for Genome Editing

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

Recently, the Cas9 nuclease from the bacterial CRISPR (clustered regularly interspaced short palindromic repeats) adaptive immune system has been adapted for targeted genome editing in a number of plant and animal species. Cas9 can be programmed by short guide RNAs to induce multiplexed gene knockout or homology-directed repair with robust efficiency. We have further identified additional small Cas9 orthologs that can be delivered by adeno-associated virus for effective gene modification of somatic tissues in vivo.

3:00 Refreshment Break with Exhibit and Poster Viewing

3:30 Anti-HIV Therapies: Genome Engineering the Virus and the Host

Paula M. Cannon Ph.D., Associate Professor, Molecular Microbiology & Immunology, Biochemistry, and Pediatrics, Keck School of Medicine, University of Southern California

By taking advantage of cellular repair pathways, targeted nucleases such as, zinc finger nucleases (ZFNs) can be used to achieve precise gene knockout, gene editing, or gene addition. For anti-HIV applications, nucleases can disrupt the CCR5 co-receptor gene, be used to insert anti-HIV genes at a designated site, or inactivate the viral genome that persists in infected cells. We use humanized mouse models to help us evaluate the translational potential of these different applications of targeted nuclease technologies.

4:00 Nuclease-Based Gene Correction for Treating Single Gene Disorders

Gang Bao, Ph.D., Robert A. Milton Chair Professor in Biomedical Engineering, Department of Biomedical Engineering, Georgia Institute of Technology and Emory University

We have developed a clinically applicable gene correction technology to treat sickle cell disease (SCD), which is caused by a single (A-T) mutation in the beta-globin gene. To treat SCD, we constructed TALENs and CRISPR/Cas9 systems that specifically target beta-globin gene and systematically evaluated their on- and off-target cleavage in different cells. We also quantified the nuclease-induced gene modification rates due to homologous recombination and non-homologous end joining. These studies significantly facilitated our pre-clinical investigation using mouse models.

4:30 Genome Editing for Genetic Diseases of the Blood

Matthew Porteus, M.D., Ph.D., Associate Professor of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine

A potentially ideal approach to the curative treatment of genetic blood diseases is to directly modify the hematopoietic stem cell in a precise fashion using genome editing. With the development of multiple different nuclease platforms, including zinc finger nucleases, TAL effector nucleases, and RNA-guided endonucleaes of the CRISPR/Cas9 family this can now be approached in a variety of different ways. We have focused on using this strategy for a number of different diseases and in this presentation will focus on our progress for severe combined immunodeficiency.

5:00 Close of Day

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Friday, February 20

8:00 am Morning Coffee


8:25 Chairperson’s Remarks

Charles A. Gersbach, Ph.D., Assistant Professor, Department of Biomedical Engineering, Center for Genomic and Computational Biology, Duke University

8:30 Genome Engineering Tools for Gene Therapy and Regenerative Medicine

Charles A. Gersbach, Ph.D., Assistant Professor, Department of Biomedical Engineering, Center for Genomic and Computational Biology, Duke University

Genome engineering tools, including zinc finger proteins, TALEs, and the CRISPR/Cas9 system, can be used to both edit gene sequences and control the expression of endogenous genes for applications in medicine and basic science. For example, we have used each of these tools for gene editing to restore the expression of the dystrophin protein that is mutated in cells from Duchenne muscular dystrophy patients. In other studies, we have developed gene regulation tools that can be applied to cell reprogramming for disease modeling and regenerative medicine.

9:00 Preventing Transmission of Mitochondrial Diseases by Germline Heteroplasmic Shift Using Transcription Activator-like Effector Nucleases (TALENs)

Keiichiro Suzuki, Ph.D., Research Associate, Gene Expression Laboratory, The Salk Institute for Biological Studies

Mitochondrial diseases include a group of maternally inherited genetic disorders caused by mutations in the mitochondrial DNA (mtDNA). In most of these patients, mutant mtDNA coexists with wild type mtDNA, a situation known as mtDNA heteroplasmy. Pre-implantation genetic diagnosis can only help to reduce, but not fully prevent, the transmission of mitochondrial diseases. We will report a novel strategy towards preventing germline transmission of mitochondrial diseases by induction of mtDNAheteroplasmy shift.


Precise Single-base Genome Engineering for Human Diagnostics and Therapy

Bruce R. Conklin M.D., Investigator, Roddenberry Center for Stem Cell Biology and Medicine, Gladstone Institutes and Professor, Division of Genomic Medicine University of California, San Francisco

Dr. Conklin’s research focuses using genome engineering to find therapies for life threatening human genetic diseases. Current projects us human induced pluripotent stem cells (iPSCs) to model cardiac, hepatic and neurological diseases. The Conklin lab has recently developed a method that significantly increases the ability to perform scarless single-base genome editing to induce or revert disease mutations. These studies allow the construction of robust in vitro human disease models, and also provide a path to cell therapy with gene corrected cells.

10:00 CRISPR Goes Viral

Shawn Shafer, Ph.D., Market Segment Manager, Functional Genomics, Sigma-Aldrich

While other genome editing reagents exist, the CRISPR system has significant advantages in terms of cost, design, and construction.  Most recently, the CRISPR system has been ported into lentiviral particles, allowing researchers to realize the previously unattainable goal of high throughput whole genome knockout screens.  Completed screens and their significance will be discussed in this talk, as will the future of this promising new technology and its potential applications. 

10:30 Coffee Break with Exhibit and Poster Viewing


11:00 Gene Editing on the Cusp of Exciting Opportunities for Human Therapeutics

Rodger Novak, M.D., CEO, 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.

11:30 Advancing the CRISPR/Cas9 Technology Platform for Therapeutic Applications

Alexandra Glucksmann., Ph.D., COO, Editas Medicine

Genome editing technologies, including the CRISPR/Cas9 system, allow for precise and corrective molecular modifications to treat the underlying cause of genetic diseases.Key to the successful translation of CRISPR/Cas9 systems to the clinic is the optimization of the technology within the context of specific therapeutic applications. This presentation will focus on Editas Medicine’s approach to improving both activity and specificity of CRISPR/Cas9-mediated gene editing in parallel with the development of delivery solutions for therapeutic applications.

12:00 pm Small Molecules Modulating CRISPR Editing

Sheng Ding, Ph.D., William K. Bowes, Jr. Distinguished Investigator, Gladstone Institute of Cardiovascular Disease, and Professor, Department of Pharmaceutical Chemistry, University of California San Francisco

CRISPR-Cas9 system has emerged as an effective tool for genome editing, but challenges remain. To enhance CRISPR-mediated gene editing, we screened chemical libraries and had identified distinct small molecules that can enhance either HDR-based gene knock-in or NHEJ-based knock-out. The use of small molecules provides a simple and effective strategy that enhances precise genome engineering applications and facilitates the study of DNA repair mechanisms in mammalian cells.

12:30 Close of Symposium

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