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.

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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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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