Six reasons why you can’t miss ELRIG’s CRISPR in Drug Discovery 2019: From Targets to Therapeutics

ELRIG’s first CRISPR in Drug Discovery: From Targets to Therapeutics meeting will focus on the latest drug discovery applications of the rapidly developing CRISPR/Cas9 technology. Taking place at the King’s Centre in Oxford from 27-28 February 2019, the event will bring together a panel of knowledgeable scientists and researchers from across academia and industry.

Through a range of presentations and vendor technology spotlights, you will have the unique opportunity to explore the applications of CRISPR/Cas9 technology in identifying novel drug targets, developing advanced cellular and in vivo disease models for target validation, and therapeutic genome editing.

If you wish to keep up with the latest developments in this area and gain insights into how you can increase your chances of success with your drug discovery research, you can’t afford to miss CRISPR in Drug Discovery: From Targets to Therapeutics. Read our interview with Dr Matthew Porteus, Professor of Paediatrics at the Division of Stem Cell Transplantation and Regenerative Medicine at Stanford University to find out why!

Reason #1: Delve into the potential of CRISPR/Cas9 technology for drug discovery

It’s no secret that CRISPR/Cas9 has already been widely adopted in laboratories conducting gene editing. Yet as the technology continues to become more sophisticated, researchers are able to push the boundaries in this area even further.

“CRISPR/Cas9 technology offers a much simpler method of gene editing, allowing us to easily engineer nucleases to make a specific DNA double-strand break at our gene of interest,” explains Dr Porteus. “A piece of ‘donor’ DNA is then used to repair the break and introduce precise sequence changes into the gene. This really is the most time- and cost-efficient way of gene editing.”

What is especially remarkable with CRISPR/Cas9 technology is that it’s worked for every target site it’s been applied to so far. Additionally, while the specificity of the technology has been the subject of much debate, recent research results demonstrate that the method is highly specific, making it suitable for developing more effective therapies to treat serious genetic diseases.

“The way I see it, CRISPR/Cas9 technology is essentially a new type of drug in the making, in the sense that it’s giving us the opportunity to change the DNA sequence of a cell,” says Dr Porteus. “There are no other therapeutics like it out there. For many years, we’ve been wishing for such a drug to become available. Now, efforts are focussed on getting it to the patients who need it the most.”

Reason #2: Keep up with the latest CRISPR/Cas9 technology innovations

Describing an advancement achieved in his own laboratory, Dr Porteus explains: “The traditional method of delivering the CRISPR/Cas9 system in the cells using DNA plasmids works very well in cultured cell lines. However, it has proven highly ineffective when applied to clinically-relevant cells that must be manipulated to generate a therapeutic effect. To address this, we discovered a system capable of delivering the Cas9 protein complex to the guide RNA as the molecule gets delivered. This has been a ground-breaking development.”

At the same time, many laboratories have discovered new improved versions of Cas9 that are more specific and able to retain on-target activity that is comparable to the original protein to ensure sufficient levels of potency. Despite these advances, it will still be some time before we see CRISPR/Cas9 treatment systems active in the clinic. “We need to be careful with our expectations of this technology. Whereas there’s been much progress when it comes to developing new and better tools, more time will be needed to implement CRISPR/Cas9 in the clinic. We can safely say that the CRISPR/Car9 promise will be realised in the next five to ten years,” comments Dr Porteus.

Reason #3: Discover what’s in the future for CRISPR/Cas9 technology

Within just a few years, CRISPR/Cas9 technology has been successfully used to develop pre-clinical models with extremely high efficiencies. This is particularly exciting as these models ultimately form the foundation for human clinical trials. When it comes to genome editing in hematopoietic stem cells (used to repopulate the blood and immune system) using the CRISPR/Cas9 method, Dr Porteus’ laboratory has not only achieved knock-out efficiencies of 90%, but also correction frequencies of 30-70%.

Dr Porteus believes CRISPR/Cas9 technology will first be used to treat two classes of diseases. The first one will be oncology, where gene editing could be used to produce CAR T-cells that will be more active, broadly used, and perhaps even safer. The second application of the technology will be to treat genetic diseases. Ultimately, CRISPR/Cas9 technology will be used across an extensive range of diseases, including into aspects of regenerative medicine.

“These are exciting times when we look forward to discover how we can use genome edited cell products that are more off-the-shelf rather than bespoke and personalised, making them much more easily available to more patients,” add Dr Porteus. “In our laboratory, we specifically aim to use genome editing in hematopoietic stem cells to develop cures for patients with serious genetic diseases, such as sickle cell disease.”

Reason #4: Find out why a research career can be so rewarding

“Choosing between a career in academic research or industry depends on where your passion lies and your own personal vision of what you wish to accomplish,” says Dr Porteus. “That’s the most important thing. As a researcher at heart, I believe that a research career can be particularly rewarding, as it gives you the opportunity to work closely with patients and witness the difference your work could really make to change their lives and their future.

“During my medical school training, for example, I worked with patients who suffered from sickle cell disease and it was not long before I realised that the available medicines to treat those patients had not been driven by our genetic understanding of the disease and were therefore ineffective. That’s when I became interested in the field of T-cell therapy and more specifically in the idea of what we now call gene editing and how this can be used to treat diseases caused by a specific mutation, as is the case with sickle cell disease. I kept wondering why we couldn’t develop a method to fix that mutation. So, it’s really exciting that after many years that idea is finally coming to fruition through the application of CRISPR/Cas9 technology.”

Reason #5: Learn from the experts

Whether you’re an experienced academic researcher, a seasoned industry expert, a recent graduate or a student, the CRISPR in Drug Discovery: From Targets to Therapeutics meeting will offer you a dedicated hub for learning and innovation in the field of CRISPR/Cas9 technology. Over the course of two days, the event’s programme will feature a range of educational talks, poster presentations and technology spotlight sessions delivered by experts such as Dr Porteus and covering:

  • The application of large-scale CRISPR functional genomics studies for drug target identification and validation
  • How genome engineering can be used to develop even the most complex biological disease models
  • State-of-the-art CRISPR genome engineering technologies, as well as their current and future therapeutic applications

There will also be the chance to hear our platinum sponsors, Horizon, Merck and Synthego, all industry leaders in gene editing tools, discuss their latest contributions in this field.

Reason #6: Plant the seeds for fruitful collaborations

In alignment with ELRIG’s vision to bridge the gap between academia and industry, the inaugural CRISPR in Drug Discovery: From Targets to Therapeutics event will bring together academic researchers and pharmaceutical/biotechnology industry professionals to exchange ideas and information. The meeting will offer an open-access forum for delegates to meet with their peers and explore potential synergies and provides the ideal opportunity to connect with those who fall outside your scientific expertise. By doing so you can gain new insights and a deeper understanding of CRISPR/Cas9 technology.

“Innovation in the field of CRISPR/Cas9 technology is situated at the crossroads of academia and the pharmaceutical/biotechnology industry,” explains Dr Porteus. “We’ve already reaped the benefits of strategic collaborations that have allowed us to marry the scientific expertise of academic researchers with the tools, resources and drug development knowhow of vendors, to advance the technology. Such partnerships are going to be increasingly important when it comes to developing the therapies of the future.”

Don’t miss out. Register now!

Now that you know why you shouldn’t miss ELRIG’s 2019 CRISPR in Drug Discovery meeting, it’s time to register to attend the event for free. You can find out more about the event here or quickly and easily access all necessary information on your mobile phone or tablet by downloading ELRIG’s free app.