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Cambridge Healthtech Institute’s 13th annual two-part conference on Advances in Gene Editing and Gene Silencing will cover the latest in the use of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9-based gene editing and RNA interference (RNAi) for use in drug discovery and for developing novel drug therapies.

Part 1 will cover the use of CRISPR/Cas9 and RNAi for identifying new drug targets and therapies. It will bring together experts from all aspects of basic science and clinical research to talk about how and where gene editing and RNAi can be best applied. What are the different tools that can be used and what are their strengths and limitations? How does the CRISPR/Cas system compare to RNAi and other gene editing tools, such as Transcription Activator-like Effector Nucleases (TALENs) and zinc finger nucleases (ZFNs), and do they have any complementary uses? 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 target discovery, disease modeling, and for creating cell and viral therapies.

Final Agenda


RECOMMENDED ALL ACCESS PACKAGE:

• September 19 Short Course: Introduction to Gene Editing

• September 19 Symposium: Understanding CRISPR: Mechanisms and Applications

• September 20-21 Conference: Advances in Gene Editing and Gene Silencing - Part 1

• September 21-22 Conference: Advances in Gene Editing and Gene Silencing - Part 2

• September 21 Short Course: Functional Screening Strategies Using CRISPR and RNAi


Day 1 | Day 2 | Download Brochure


Tuesday, September 20

7:00 am Registration Open and Morning Coffee


KEYNOTE SESSION:
GENOME EDITING FOR IN VIVO APPLICATIONS

8:05 Chairperson’s Opening Remarks

Bryan R. Cullen, Ph.D., James B. Duke Professor of Molecular Genetics and Microbiology and Director, Center for Virology, Duke University

8:20 AAV for Gene Therapy and Genome Editing

James Wilson, M.D., Ph.D., Professor, Department of Pathology and Laboratory Medicine, Perelman School of Medicine; Director, Orphan Disease Center and Director, Gene Therapy Program, University of Pennsylvania

In vivo delivery of nucleic acid therapeutics remains the primary barrier to success. My lab has focused on the use of vectors based on adeno-associated virus (AAV) for achieving success in pre-clinical and clinical applications of gene replacement therapy. Most of the current academic and commercial applications of in vivo gene replacement therapy are based on endogenous AAVs we discovered as latent viral genomes in primates. These vectors are reasonably safe and efficient for application of gene replacement therapy. The emergence of genome editing methods has suggested more precise and effective methods to treat inherited diseases in which genes are silenced or mutations are corrected. AAV vectors have been the most efficient platform for achieving genome editing in vivo. We will review our attempts to achieve therapeutic genome editing in animal models of liver disease using AAV.

9:20 Using CRISPR/Cas to Target and Destroy Viral DNA Genomes

Bryan R. Cullen, Ph.D., James B. Duke Professor of Molecular Genetics and Microbiology and Director, Center for Virology, Duke University

A number of pathogenic human DNA viruses, including HBV, HIV-1 and HSV1, cause chronic diseases in humans that remain refractory to cure, though these diseases can be controlled by antivirals. In addition the DNA virus HPV causes tumors that depend on the continued expression of viral genes. Here, I will present data demonstrating that several of these viruses can be efficiently cleaved and destroyed using viral vectors that express Cas9 and virus-specific guide RNAs, thus providing a potential novel approach to treatment.

9:50 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing

10:35 Targeted Endonucleases as Antiviral Agents: Promises and Pitfalls

Keith R. Jerome, M.D., Ph.D., Member, Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center; Professor and Head, Virology Division, Department of Laboratory Medicine, University of Washington

Genome editing offers the prospect of cure for infections such as HIV, hepatitis B virus, herpes simplex, and human papillomavirus, by disruption of essential viral nucleic acids or the human genes encoding receptors needed for viral entry. This talk will highlight the most recent laboratory data and the challenges still ahead in bringing this technology to the clinic.

11:05 Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

Daniel 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 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 deliver RNA, both in vitro and in vivo. A number of delivery formulations have been developed with in vivo efficacy, and show potential applications for the treatment of genetic diseases, viral infections and cancers.

11:35 PANEL DISCUSSION: CRISPR/Cas: A Realistic and Practical Look at What the Future Could Hold

Moderator: Bryan R. Cullen, Ph.D., James B. Duke Professor of Molecular Genetics and Microbiology and Director, Center for Virology, Duke University

Participants: Session Speakers

Each speaker will spend a few minutes sharing their viewpoints and experiences on where things stand with using the CRISPR/Cas system for in vivo applications. Attendees will have an opportunity to ask questions and share their opinions.

12:05 pm Presentation to be Announced

12:35 Session Break

Millipore Sigma12:45 Luncheon Presentation: Building a Better Research Story: Screening with shRNA and CRISPR

Ryan Raver, Ph.D., Global Product Manager, Functional Genomics, MilliporeSigma

Parallel RNAi and CRISPR-Cas9 screens have opened up new opportunities for assay development, screening and validation. In partnership with the Wellcome Trust Sanger Institute, MilliporeSigma has developed the first whole genome arrayed CRISPR library. Topics include evaluation of complementary shRNA and CRISPR screens, and the efficacy of each. As each technology has distinct biological processes, the advantages and disadvantages of each for uncovering novel targets will be discussed in detail.


1:25 Refreshment Break in the Exhibit Hall with Poster Viewing


COMPLEMENTING THE USE CRISPR & RNAi FOR DISEASE MODELING

2:05 Chairperson’s Remarks

Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

2:15 Genetically Modified Mouse Models of Cancer Using Gene Depletion and Deletion

Ricky Johnstone, Ph.D., Assistant Director of Research Division; Co-Head, Cancer Therapeutics Program and Head, Gene Regulation Laboratory, Peter MacCallum Cancer Centre, Australia

Genetically engineered mouse models (GEMMs) have been extensively used to functionally identify genes involved in cancer. Gene depletion and deletion techniques using shRNA and CRISPR respectively are effective methodologies to determine the putative tumor suppressor role of various genes and their importance in mediating therapeutic responses to anti-cancer agents. I will present data showing the use of shRNA and CRISPR to demonstrate tumor suppressor functions of individual genes in GEMMs and the importance of distinct genes in tumor maintenance.

2:45 Use of CRISPR/Cas9-Based Gene Editing to Model and Treat Retinal Degenerative Disease

Donald Zack, M.D., Ph.D., Guerrieri Professor of Genetic Engineering and Molecular Ophthalmology, Johns Hopkins University

The combination of human stem cell retinal modeling, CRISPR/Cas9 genome editing, and high content screening technology provides unparalleled opportunities for the study of retinal biology, disease modeling, drug screening, and the development of novel gene therapy-based treatment approaches. The use of these technologies to model and identify new targets for diseases involving retinal ganglion cells, photoreceptors, and retinal pigment epithelial cells will be discussed.

3:15 Sponsored Presentation (Opportunity Available)

3:45 Refreshment Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced

4:25 Introduction to Technology Panel: Trends in CRISPR & RNAi Technologies

Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

4:55 Technology Panel: Trends in CRISPR & RNAi Technologies

Moderator: Ralph Garippa, Ph.D., Director, RNAi Core Facility, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center

Panelists: Opportunity Available for Sponsoring Companies

This panel will bring together 4-5 technical experts from leading technology and service companies to discuss trends and improvements in library design, assay reagents and platforms, and data analysis tools that users can expect to see in the near future.

5:25 Welcome Reception in the Exhibit Hall

6:25 End of Day

Day 1 | Day 2 | Download Brochure


Wednesday, September 21

7:30 am Registration Open and Morning Coffee


EXPLORING THE VERSATILITY OF CRISPR/Cas9

8:00 Chairperson’s Opening Remarks

Geoffrey Bartholomeusz, Ph.D., Associate Professor and Director, Target Identification and Validation Program, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center

8:10 Functional Genomics Using CRISPR-Cas9: Technology and Applications

Neville Sanjana, Ph.D., Core Faculty Member, New York Genome Center and Assistant Professor, Department of Biology & Center for Genomics and Systems Biology, New York University

The easy programmability of CRISPR/Cas9 suggests a new way to interrogate gene function at the DNA level instead of the transcript level. By combining genome engineering with functional genomic screens, we have developed genome-wide libraries for negative and positive selection screening in human and mouse cells. In addition, we have developed techniques for adapting CRISPR screens into noncoding regions of the genome, where it can be challenging to identify functional elements.

8:40 Harnessing the Versatile CRISPR-Cas9 Systems for Cancer Modeling Platforms

Geoffrey Bartholomeusz, Ph.D., Associate Professor and Director, Target Identification and Validation Program, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center

The specificity of the CRISPR/Cas9-based gene editing technology derived from the microbial defense system is driving innovative applications from basic biology to biotechnology and medicine. To successfully utilize this gene editing system to further understand the operations of complex genomes, it is imperative to minimize interfering off target biological mechanisms. We highlight such outcome identified during the optimization of our CRISPR systems.

9:10 Towards Combinatorial Drug Discovery: Mining Heterogenous Phenotypes from Large Scale RNAi/Drug Perturbations

Arvind Rao, Ph.D., Assistant Professor, Department of Bioinformatics and Computational Biology, The University of Texas MD Anderson Cancer Center

In this talk, we will outline methodologies and share our experiences for the interpretation of cellular phenotypes obtained via high throughput microscopy on drug screens in patient-derived glioma cell lines as well as of Triple negative breast tumors, in 3D culture. The large volume and heterogeneity in cellular phenotypes across the population make this a challenging task. We will also describe frameworks to quantitatively interpret such heterogeneity and prioritize hits (genes from RNAi, drugs from screens).

9:40 Coffee Break in the Exhibit Hall with Poster Viewing

10:25 CRISPR in Stem Cell Models of Eye Disease

Alexander Bassuk, M.D., Ph.D., Associate Professor of Pediatrics, Department of Molecular and Cellular Biology, University of Iowa

Induced pluripotent stem cells (iPSCs) generated from patient fibroblasts could potentially be used as a source of autologous cells for transplantation in retinal disease. Patient-derived iPSCs, however, would still harbor disease-causing mutations. To generate healthy patient-derived cells, we used CRISPR/Cas9 to precisely repair a point mutation that causes retinal degeneration. This important proof-of-concept finding supports the development of personalized iPSC-based transplantation therapies for retinal disease.

10:55 CRISPR in Mouse Models of Eye Disease

Vinit Mahajan, M.D., Ph.D., Assistant Professor of Ophthalmology and Visual Sciences, University of Iowa College of Medicine

Massive parallel sequencing enables identification of numerous genetic variants, but determining pathogenicity of any one mutation is daunting. The most commonly studied preclinical model of retinal degeneration is homozygous for two different mutations in the same gene. We used the CRISPR/Cas9 gene editing system to identify the causative variant and rescue neurofunction. This is among the first examples of CRISPR-mediated repair in a sensory system.

11:25 Enjoy Lunch on Your Own



2:40 Refreshment Break in the Exhibit Hall with Poster Viewing

3:20 End of Conference



Day 1 | Day 2 | Download Brochure

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 PREMIER SPONSOR

Cellecta

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SPONSORSHIPS & EXHIBITS

The exhibit hall was sold out in 2015, so please contact us early to reserve your place. To customize your sponsorship or exhibit package for 2016, contact:

Jon Stroup
Sr. Business Development Manager
781-972-5483
jstroup@healthtech.com

 

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IPR-Special-Report-Packages  

SEPTEMBER 19 SYMPOSIA:

Next-Generation Histone Deacetylase Inhibitors

Strategies for Tackling Rare Genetic Diseases

Understanding CRISPR: Mechanisms and Applications

Autoimmunity – Small Molecule Approaches

NK Cell-Based Cancer Immunotherapy

Medical Dermatology Therapeutic R&D and Technical Innovation

CONFERENCES

SEPTEMBER 20-21

Targeting Histone Methyltransferases and Demethylases

Targeting the Ubiquitin Proteasome System

Targeting the Microbiome
– Part 1

GPCR-Based Drug Discovery - Part 1

Advances in Gene Editing and Gene Silencing – Part 1

Gene Therapy Breakthroughs

Antibodies Against Membrane Protein Targets – Part 1

Targeting Cardio-Metabolic Diseases

Targeting Ocular Disorders

SEPTEMBER 21-22

Targeting Epigenetic Readers and Chromatin Remodelers

Kinase Inhibitor Discovery

Targeting the Microbiome
– Part 2

GPCR-Based Drug Discovery - Part 2

Advances in Gene Editing and Gene Silencing – Part 2

Translating Cancer Genomics

Antibodies Against Membrane Protein Targets – Part 2

Metabolomics in Drug Discovery

TRAINING SEMINAR: Data Visualization

SHORT COURSES*

Monday, September 19
8:00 - 11:00 am

(SC1) Immunology Basics for Chemists

(SC2) Designing Peptide Therapeutics for Specific PPIs

(SC3) Phenotypic Screening and Chemical Probe Development

(SC4) Medical Dermatology Therapeutic R&D and Technical Innovation - Part 1

Monday, September 19
2:00 - 3:00 pm

(SC5) GPCR Structure-Based Drug Discovery

(SC6) RNA as a Small Molecule Drug Target

(SC7) Using IP Landscape Studies to Improve Your Confidence

(SC8) Medical Dermatology Therapeutic R&D and Technical Innovation - Part 2

Monday, September 19
3:30 - 6:30 pm

(SC9) Targeting of GPCRs with Monoclonal Antibodies

(SC10) Introduction to Targeted Covalent Inhibitors

(SC11) Contact Lens Drug Delivery Systems

(SC12) Introduction to Gene Editing

Monday, September 19
7:00 - 9:30 pm

(SC13) Convergence of Immunotherapy and Epigenetics for Cancer Treatment

Wednesday, September 21
7:00 - 9:30 pm

(SC14) Cancer Metabolism: Pathways, Targets and Clinical Updates

(SC15) Introduction to Allosteric Modulators and Biased Ligands of GPCRs

(SC16) Functional Screening Strategies Using CRISPR and RNAi

(SC17) Challenges and Opportunities in DNA Methyl Transferase (DNMT) Inhibitors as Therapeutics