Showing posts with label Gene Editing. Show all posts
Showing posts with label Gene Editing. Show all posts

Monday, August 15, 2016

Experts Predict what Boston Biotech Might Look Like in 2050

This past July Karl Thiel wrote in Biospace: “Never has there been so much transformative technology, seemingly right around the corner… Hugely exciting new technologies like CAR-T, emerging technologies like CRISPR-Cas, and perhaps-ready-for-prime-time technologies like RNAi, gene therapy, and antisense all seem to be on the cusp of revolutionizing healthcare.” It’s hard to deny the fact that so many amazing groundbreaking biotechnologies have been developed, so quickly, in the past few years. If this growth rate continues there is really no telling what is on the horizon for biotech. I asked several experts in the Boston area – from biotech CEOs to bioengineers – what they thought Boston biotech would look like in 30 or so years.

What does Boston biotech look like in 2050?


Jeffrey Karp, Brigham and Women's Hospital and Karp Labs

In 2050, Boston’s population will have significantly swelled resulting from the booming biotech, medtech, and pharma industries, and new innovative colleges that have formed. People will be much more in control of their health than they are today - most people will have had their genome sequenced and will wear devices whose data will be used to minimize implications of the genome findings. Data from wearable devices will help promote lifestyle modifications to maximize health and be used by clinicians to tailor patient specific treatments. Life expectancies will continue to rise and quality of life past 65 will improve with regenerative therapies for hearing loss, cancer, and cardiovascular disease. This will in part be achieved through controlling stem cell populations inside the body with small molecules. People will also frequently visit stem cell infusion clinics for routine therapy for multiple diseases and tissue defects. The future is quite bright for Boston!

Catch Jeffrey Karp at Biotech Week Boston's Cell and Gene Therapy Bioprocessing and Commercialization event this October. Jeffrey's talk is entitled "MSCs on steroids".


Robert Langer, David H. Koch Institute Professor, MIT

I think in 2050 the Boston area will be the center of the biotech universe even more than it is today, and I expect we will see a host of new technologies including RNA therapies, nanotechnology, tissue engineering, gene editing and technologies that are not even on our radar screen today affecting the lives of billions all over the world.


Martin Tolar, MD, PhD, Founder, President and CEO Alzheon, Inc.

Boston is one of the most active areas for research and drug development in neuroscience and neurodegenerative diseases. The Boston ecosystem that fuels biotechnology innovation includes a brain trust of experts in science, medicine and biotechnology collaborating for new ways to treat neurodegenerative disorders.

By the year 2050, the outlook is promising for new life-changing medicines to emerge from the Boston hub for these devastating diseases of the brain that represents some of the greatest challenges in human health, currently with very limited treatment options for patients: Alzheimer’s, ALS, Huntington’s, Parkinson’s diseases. We are on the cusp of bringing new medicines to millions of patients in need.

You can see Martin Tolar at Biotech Week Boston's Partnerships in Clinical Trials event this October. Martin will present the opening keynote for day two of the event entitled "Innovation in Clinical R&D: Finding a Cure for Alzheimer’s".


C. Michael Gibson, Founder and Chairman of Wikidoc.org and Professor of Medicine at Harvard Medical School

Randomized clinical trials will no longer need to pay to build a new database for each trial and will not be using large number of nurses and doctors to identify and follow patients. Instead, national health databases will be used to identify patients with disease or those at risk of disease, and with the patient's consent they will be randomized to a therapy and followed using this database and a more limited number of nurses at centralized centers. Digital devices will collect and transmit data. Obviously, therapy will be much more highly targeted based upon genomics, proteinomics and other "ics."


Phillip Sharp, Ph. D.  Koch Institute at MIT. Dr. Sharp won the Nobel Prize in Physiology or Medicine 1993 for the discovery of RNA splicing (in 1977) and founded Biogen in 1978.

Biotech in Cambridge and Boston will be thriving in 2050 having generated numerous treatments for Alzheimer's, Parkinson’s, better control of cancers, schizophrenia and depression. Delivery of medical care will continue to move from hospitals to more diverse settings and intense use of IT and engineering will individualize healthcare and reduce its cost.


Got any predictions for 2050? We’d love to share them with our audience so Tweet to us at @BiotechWkBoston. Don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is a hub for life sciences, technology, and business and fosters cross-disciplinary interaction and collaboration to break down silos and spark change. Biotech Week Boston will showcase the most comprehensive science and innovative technologies while fostering partnerships to unlock the full potential of what science and business can achieve. Learn more by clicking the link below.






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Friday, June 17, 2016

Gene Editing in CHO Cells

The team behind Cell Line Development & Engineering event have recently produced an exclusive whitepaper titled "Gene Editing in CHO Cells". Below you will find a brief summary of the whitepaper and download the complete whitepaper now.


Whitepaper Summary:

Recently, several exciting advances in CHO cell line engineering have received significant media and research attention due to efforts in genome sequencing, systems biology, and bioinformatics combined with the relatively new field of targeted gene editing platforms. Three key gene editing technologies have been at the forefront of the recent developments in CHO cell line engineering. Early efforts to introduce targeted site specific edits to the CHO genome focused on implementing the zinc finger nucleases (ZFNs) and the transcription activator-like effector nucleases (TALENs). The ZFN platform has been successfully deployed in a variety of post translational modification applications aimed at increasing specificity of recombinant protein production, but the efficiency of this platform can be limited in mammalian cell lines.

The clustered regularly interspaced short palindromic repeats associated 9 (CRISPR/Cas9) targeted gene editing system has recently exploded onto the research scene in almost every organism. This targeted gene editing platform allows for the creation of multiplexed edits in a single cost-effective step with a specificity previously unachievable in the genome editing arena. This complex is composed of short guide RNAs (sgRNAs) and a CRISPR-RNA that form a site specific construct that is complimentary to the target DNA, which introduces a double stranded DNA break upon binding. Repair of the break site by endogenous enzymes then creates a highly specific change to the DNA which can be customized for a variety of applications.

These advances in genome editing have helped enable high-throughput development of CHO cell lines that can be utilized as economically viable commercial expression vectors. The CRISPR/cas9 gene editing system has shown to be an extremely useful tool for customizing the metabolic pathways of CHO cell lines for use in biopharmaceutical production. One of the most useful applications of this exciting technology has been the creation of multiplexed targeted knock out screening systems. Previously, knock out experiments had to rely on mutagenesis, drug knock out, or media screening to identify the effect of a mutation on a desired cell type. These methods are inefficient and sometimes lead to less desirable off-target effects. It is now possible to develop very large gene knockout libraries to be targeted by CRISPR/cas9 using bioinformatics software specific to this platform.

Customizing metabolic pathways in CHO cell lines is of paramount importance for developing “cell factories” capable of biopharmaceutical production. Reducing the energy expenditure associated with mitochondria production and oxidative metabolism is one approach that has been shown to increase the efficiency of the cell by directing metabolism towards production of the target product. Modulating these pathways has traditionally been accomplished using interfering RNAs, but the specificity of the CRISPR/cas9 platform offers another tool with which researcher can customize the energy profile of CHO cell lines destined for biopharmaceutical production.

Future efforts in the field will be focused on increasing the efficiency of the CRISPR/cas9 system, as this platform is poised to become the model for the majority of biopharmaceutical development. Currently, 60 to 70% of all biopharmaceutical production is accomplished with recombinant mammalian cell lines, and this market share is expected to grow. Multiplexed editing efforts will also continue to increase the rate at which specific customizable CHO cell lines can be produced, as this process is highly critical to increasing the rate at which CHO cell line engineering moves forward.




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