Genome Editing Applications | Brussels, Belgium | 2-3 December 2015

Get updates on Therapeutic and Biomedical Applications of CRISPRs, ZFNs, TALENs and other Genome Engineering Technologies

Genome Editing Applications 
2-3 December 2015
Sheraton Airport Hotel
Brussels, Belgium

Biomedical researchers have long strived for a reliable and efficient method of making targeted changes to the genome of living cells. With the advent of CRISPR/Cas9 researchers are now able to explore gene function in a much more thorough and cost-effective way.

This year, Lorenz Mayer from Astrazenca will be giving a presentation on their genome wide target discovery and target validation studies. Ines Royaux from Janssen Phamaceutica will also be giving a presentation touching on the team’s first experience of using genome editing for ex vivo gene therapy. Read on to find out more about what you can expect to learn from the speakers at this year's conference.

Get a sneak peek into the new developments and data the speakers will be sharing during their presentations here.

Informa's Genome Editing Applications 2015 conference, taking place on 2-3 December 2015 in Brussels, will showcase the most up-to-date therapeutic and biomedical applications emerging using CRISPRs, ZFNs, TALENs, AAVs and other genome engineering technologies. From improving lead discovery, validation of targeted cell lines and development of transgenic animal models to therapeutic uses in cell therapy & gene therapy.

This inaugural event in Europe follows the enormous success of Genome Editing Applications in March 2015 in the US from our sister company, IBC Life Sciences.

*Genome Editing Applications is co-located with Cell Therapy Manufacturing and Gene Therapy conferences with a shared exhibition area

So far, across the three conferences, over 120 have confirmed to attend with representation from biopharm, pharma and biotech companies - including Novartis Pharma AG, Glaxosmithkline, CRISPR Therapeutics, Astrazeneca, F Hoffmann-La Roche AG, GSK Pharma, Hitachi LTD, Bluebird BIO, Sanofi Pasteur, Janssen Pharmaceutica NV, Merck & CO., INC., Precision Biosciences, INC.

Hear from influential industry experts such as:

REGISTER FOR AS LITTLE AS £799* TODAY

*Based on standard industry rate Biopharm/Pharma/Biotech 2 day conference pass for one attendee. Not including suppliers/vendors.

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New Tools for Genome-Wide Functional Screens Needed to Develop New Drugs

Guest Post: Paul Diehl, PhD, Director of Business Development at Cellecta - Paul will be speaking at IBC's Genome Editing Applications conference March 18-19 in Boston.


Despite the need for new innovative therapies, the rate of novel drug development has remained level with about 35-40 new approvals each year (see figure below). One crucial factor restricting the rate of new drug discovery is the limited set of therapeutic gene targets. There are approximately 1,500 approved therapeutic compounds, but they target only about 300 gene products. Most new compounds in testing are directed toward these same known targets.

However, the potential number of drug targets is at least an order of magnitude greater. DrugBank associates over 4,000 targets with the 7,500 compounds in its database, and GeneCards catalogues over 10,000 genes that have data indicating they “cause, predispose, or protect from diseases.” Why does the current pharmacopeia only exploit less than 10 percent of the potentially targetable gene products then?

The problem is that there is still too much unknown about how most gene products actually work. A tremendous amount of genetic information has been amassed and deposited into online databases. Data about how, under certain conditions in some systems, transcription of certain genes increase or decrease, proteins that interact, and genes that are mutated more frequently in certain systems. Almost all of this data, however, is simply correlative. These massive databases simply do not contain the key information required to really understand how genes produce biology. No amount of deep and thorough analysis of the massive amount of information currently stocked away can illuminate how most genes function because there is a critical dearth of information indicating which genes actually drive the biology.

This lack of information about which genes are responsible for biological responses and disease progression stems from the difficulty in obtaining this data experimentally. Data is needed that causally links gene products to phenotypic changes. This requires assays that assess how the perturbation of certain genes changes the responses or characteristics of cells in a model system, and there are few experimental approaches to generate this sort of data efficiently.

Some techniques, such as genomic recombination technologies like transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), can provide the needed data by disrupting or otherwise altering genetic targets in a precise manner. However, functional analysis tools must be able to assay the roles of large numbers of genes simultaneously. Collecting data on this scale requires parallel measurements in genome-wide functional assays, which is difficult with these gene manipulation techniques.

There are really only two technologies that currently form the basis of techniques for broad-based genetic screens that assess the functions of large numbers of genes in a single assay: RNA interference (RNAi) and CRISPR knockout screens.

Although RNAi and CRISPR loss-of-function screening approaches are effective, their potential to elucidate gene function remains somewhat limited. One limitation is that either approach only looks at the effects produced by the disruption of a single gene within the system. In reality, though, it is rare that one gene produces one trait. Most phenotypes are caused by multiple interacting genes. While loss-of-function screens can be designed to look for interacting genes, such as Bassik, et al. has shown, paired gene screens can only be run on a limited set of a few hundred genes, so efficient combinatorial assessments of interacting genes across the genome is really out of reach at the moment.

Also, disruption of gene function by knockdown or knockout provides only a limited range of functional assays. Genes may also be activated or otherwise functionally modulated to produce biological responses. Although RNAi can only be used for loss-of-function screens, CRISPR has shown some potential in screening for the activation or gain of genetic function. There is the potential, then, for the development of more sophisticated CRISPR-based screens that assay for genetic changes activating new pathways or bringing about novel biological transformations that may well elucidate disease development.

Thus, while RNAi and CRISPR loss-of-function screens provide one of the few effective approaches currently available for genome-wide functional screening, they are not sufficient to meet the daunting challenge of understanding how genes produce biology. Progress in identifying novel therapeutic targets will continue at a glacial pace until more advanced techniques are developed to address the challenge of teasing out the complex genetic networks that drive and control cellular responses and processes.

  
New Drug Approvals and R&D Expenditures on Annual Basis. The number of new drugs with novel mechanisms approved each year by the FDA (left axis) as compared with the annual expenditures reported by pharmaceutical companies in the 2014 Biopharmaceutical Research Industry Profile published by PhRMA (http://www.phrma.org/profiles-reports).

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Genetic Engineering: It's Not Frankenstein

Today's guest post comes from Genome Editing Applications sponsor Horizon Discovery.

Genetic Engineering: It's Not Frankenstein

Author: Hannah Murfet (PCQI, BSc). Quality and Regulatory Manager

Genetic engineering often faces unjustified bad press with regard to genetically modified crops, however genetic engineering continues to provide great promise for drug discovery, assessment and production. We must apply care and regulation to allow continued advances to focus of human treatment and not enhancement, science should not allow us to create a monster.

Genetic engineering put simply is the adjustment of the instructions of our cells, the smallest functional unit of life. The genome is the sequence of information required to code for an organism. this consists of a long code represented by 4 letters (G, A, T and C). The sequence of these letters codes for the proteins and how often they are produced. Changes in the coding of these 4 letters take a range of forms from single letter changes, insertions and duplications.

Genetic engineering is routinely applied in medical treatment, particularly with the mass production of human insulin in bacteria since 1982 (1). In this example the gene for insulin was inserted into bacteria, the bacteria then produce insulin protein which can then be purified for medicinal use. The future of this technology has continued with antibody therapy, where immune proteins are engineered and produced for targeted treatment of cancer and autoimmune disease.

Scientists are able to make use of changes in coding to further our understanding of disease models such as cancer, where changes in the 4 letters can lead to changes in the way our cells behave (2). Cell and animal models can be used in early stage drug trials to determine effectiveness and resistance.

The future of genetic engineering takes this a stage further to target the cells in our bodies. This technology makes use of a virus to insert a gene to treat disease where the coding defective, Glybera is the first drug of this kind to be recommended for approval (3). The cost of these therapies is currently high, but the field of genetic engineering is continuing to evolve and new technologies such as CRISPR are being adopted (4).

Right now all the work in this field is creating some positive enhancements for medicine. With careful control and regulation the prospects for drug development and treatment have massive potential, however we must take care of the ethical implications of genetic alteration, particularly anything that may lead to human enhancement - we certainly don't want to create any monsters!

(1) http://www.brighthub.com/science/genetics/articles/21983.aspx

(2) http://www.oapublishinglondon.com/article/607

(3) http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.html

(4) http://www.bio-itworld.com/2014/5/22/advances-genetic-engineering-american-society-microbiology-meeting.html



Genome Editing Applications will take place will take place March 18-19, 2015 in Boston.  Find out more about the program.  As a reader of this blog, you can save 20% off the standard rate when you register to join us and mention code D15223BLG.  

This is co-posted on LinkedIn

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George Church and Feng Zhang to keynote inaugural Genome Editing Applications conference

IBC Life Sciences, the organizer of market leading events in drug discovery and biologics development, today announced that George Church and Feng Zhang, two world-renowned scientists, will provide the keynote presentations at the Genome Editing Applications conference to be held on March 18-19, 2015 at the Hilton Boston Back Bay, Boston, MA. The Genome Editing Applications conference is the first comprehensive event to focus on the applications of genome editing technologies and genome engineering in therapeutic development, drug discovery and screening and other research applications.

"The emerging applications of genome editing tools hold enormous potential for the development of powerful life saving therapies. Genome editing technologies also offer significant advantages in drug discovery, target validation, screening and other pharmaceutical research applications, including development of gene therapies, cell therapies and immunotherapy. In George Church and Feng Zhang, we are excited to have two of the most respected leaders in the field to share their insights on current and future technologies and applications in genome editing and genome engineering, “said Michael Keenan, Conference Director, IBC Life Sciences.

The event features a compelling program that enables delegates to:

• • Learn about therapeutic applications of genome editing in cardiovascular disease, gene therapy, stem cell therapy, CART cell immunotherapy, hemophilia B and other advances in in vivo genome editing
• • Gain an in-depth update on the latest technology advances and applications of CRISPR-Cas9 including a discussion of off-target issues, delivery to cells/tissues, improving specificity and multi-genic alterations
• • Apply genome editing as a research and drug discovery tool in target identification and validation, genome-scale screening, gene targeting, cell line validation and transgenic animal models
• • Compare the advantages and disadvantages of CRISPRs, ZFNs, and TALENs for various applications and discover ways to navigate the IP landscape of genome editing technologies for your own projects

For more information about the Genome Editing Applications conference and how to register, visit the webpage.

About the Genome Editing Applications conference
IBC’s Genome Editing Applications conference will showcase the most up-to-date therapeutic and biomedical applications emerging using CRISPRs, ZFNs, TALENs, AAVs and other genome engineering technologies. From validation of targeted cell lines and development of transgenic animal models to therapeutic uses in cell therapy, gene therapy and immunotherapy, this event will also highlight applications in drug target identification/validation and lead discovery and explore how these technologies are being used for whole-genome and functional screening as well as basic research applications. A special emphasis on CRISPR will highlight the design of novel CRISPR constructs and discuss practical aspects of CRISPR including off-target effects, how to improve specificity and delivery to specific cell types, including potential applications in multi-genic alterations. By attending this applications-focused conference you will learn about advances in all of the latest genome editing technologies, including comparisons, advantages and disadvantages of each. You will also learn how to position your company for success in genome editing in the face of the still-evolving IP landscape of CRISPR and other genome editing approaches.

About IBC Life Sciences
IBC Life Sciences, a division of Informa, is your connection to the life sciences industry. Through conferences, webinars, podcasts and its suite of integrated marketing solutions, IBC Life Sciences delivers expert knowledge, valuable connections and customized offerings so customers can meet their goals. www.IBCLifeSciences.com

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