Right Drug, Right Time

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

Right Drug, Right Time

Author: Hannah Murfet, Vice-Chair CQI Next Generation Network

“Right first time” is a concept familiar to the quality profession, but it is taking on a whole new meaning since the emergence of personalised medicine. Personalised medicine aims to eradicate an often random and reactive approach to patient care by employing techniques such as genetic testing to ensure treatment is more likely to be effective and safe in a specific patient population.

Traditional approaches to drug treatment generally take a population-wide trial and error approach. That is to say that the drugs used for treatment are known to be generally effective, but their specific effect on an individual patient is often unknown. Drugs are typically tested on large patient populations in order to test for safety and efficacy. As the patient population is seldom uniform, the efficacy is reduced as the drug works well for some and not others. Personalised medicine has the opportunity to change this by allowing us to use the right drug at the right time – a prime example of root cause analysis.

The successes of personalised medicine are staking up, and include genetic testing for breast cancer, chronic myeloid leukaemia and colorectal cancer. One very well-known example is screening for BRCA1 and BRCA2 gene mutations to predict whether a patient needs preventative breast cancer treatment. Other examples include targeted therapy based on chromosome alterations in patients with chronic myeloid leukaemia and KRAS gene mutations used as biomarkers to predict the success of specific drug treatments for colorectal cancer. Seemingly, the possibilities for personalised medicine are endless.

While the upfront costs of personalised medicine are initially higher, they will be reduced in the long term as the cost of quality is reduced by removing the expense of ineffective and repeated treatments. It’s a tremendously exciting time for the pharmaceutical and medical device fields, but I also feel those in the quality profession can take something from this. The application of the right drug, at the right time, has parallels with the work of the quality profession. We aim to use the right tool, the right approach and the right improvement at the right time. So let’s take a ‘personalised medicine’ approach to business improvement. I’m sure the results will be just as powerful for improving quality.

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 post is co-posted with the CQI Blog.

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Novartis invests in gene editing technology

Announced earlier this week, Novartis will be joining in forces with Atlas Venture, a partnership created to back start up Intellia Therapeutics which will focus on gene editing technology in the oncology field. One of the key players in the start- up team is the former chief scientific officer of AbbVie, John Leonard and they will be based in Camebridge, Massachusetts.  The key technology CRISPR Cas9, which according to Fierce Biotech, works by developing  host bacterial cells incorporate short bursts of DNA sequences from invaders that can trigger disease which has the potential to create huge shifts in oncology treatment.  They are in-licensing this technology from Caribu Biosciences.

Plans for the initial work include:

Initially Intellia plans to focus on ex vivo work, where cells are collected from patients and then modified for therapeutic purposes before being injected back into patients--with applications relevant to CAR-T and checkpoint inhibitors. An in vivo approach can later be used to correct genes found inside cells, with applications in ophthalmic, central nervous system, muscle, liver, anti-infective and other disease.

This March at the Genome Editing Applications event, Caribou Biosciences will be joining us at the event to share more about this technology in the presentation Crisper Designs for Cas9-mediated Genome Engineering.  For more information on this session and the rest of the program, download the agenda.  If you'd like to join Caribou Biosciences in Boston March 18-19, as a reader of this blog, when you register to join us and mention code D15223BLG to save 20% off the standard rate. Have any questions or want to get involved? Reach out to Jennifer Pereira.

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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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Heart Attack Vaccination Could Be on the Way

This post was contributed by @MikeMadarasz

Heart attacks are the leading natural killer worldwide and claim approximately one million lives every year.  A staggering figure, researchers with the Harvard Stem Cell Institute and University of Pennsylvania claim to have discovered a treatment that could potentially reduce the risk of heart attack by up to 90%. 

Scientists at the Harvard Stem Cell Institute and University of Pennsylvania have collaborated on a “genome editing” approach that has dramatically reduced cholesterol levels in mice with a single injection.  Kiran Musunuru MD, PhD, leader of the HSCI team working on this project, called the success of the first iteration of the experiment “pretty remarkable”.

The research was focused on altering a gene known as PCSK9 which is found in the liver and recognized as being a regulator of cholesterol.  In 2003, a group of researchers in France discovered that a rare mutation in this gene was positively correlated with high cholesterol and early heart attacks. 

More recently, a research group in Texas has discovered that a separate mutation in this gene also has the opposite effect.  The “good” mutation is present in only 3% of the population, but those carrying this specific gene have bad cholesterol levels ranging from 15-28% lower.  Consequently, their risk of heart attack is 47-88% lower. 

This lead Musunuru and his team to deduce that if they could replicate the effect, they’d be able to protect people from heart attack.   

The research reached a turning point in 2013 with an improvement to a technology called Cas9.  Said Musunuru, “Cas9 is a protein that will create a break in DNA, and the CRISPR is an RNA component that will bind to a matching sequence and directs the Cas9 to that sequence in the DNA in which you are interested. This creates a break where you want it. The cell can then repair itself, though often with errors, which is useful if you want to disrupt a gene to create a ‘knockout’ of the gene.”

“What we were thinking was that with this genome editing technology we can do something we couldn’t do before—make permanent changes in the genome at the level of the DNA; we can actually go to the source. So the question was whether we can use genome editing to make normal people like people born with the ‘good’ mutations.” Based on the results with mice, the answer is a resounding “yes”.

The group saw a 35-40% reduction in cholesterol levels in these mice which they say could translate into about reduction in heart attacks as high as 90% in humans.  Says Musunuru, “It could be a one-time treatment, a permanent alteration. If you used this in a population, you could reduce the occurrence of heart attack by 30 percent, 50 percent, 90 percent.”

Get the full article from HSCI here.  

Get more on the latest in the field of cell therapy at this year’s Cell Therapy Bioprocessing conference.  Join us September 15-16 in Arlington, VA.  Save $100 as a reader of this blog when you register to join us and mention code XB14189BLOG.

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