Monday, August 22, 2016

5 Women to Watch in Boston Biotech

Less than five percent of the CEOs in Fortune 500 companies are women (just one is an African-American woman) but in Boston, several of the most influential biotech executives are women. Perhaps it is the fact that startups with at least one female founder simply perform better. To honor the accomplishments of these pioneering executives, here’s a snapshot at the contributions of five notable women to biotech in Boston.

Susan Windham-Bannister, President and CEO of Biomedical Growth Strategies and the Managing Partner of Biomedical Innovation Advisors

Susan Windham-Bannister was the first President and CEO of the Massachusetts Life Sciences Center where she oversaw a $1 billion investment to accelerate the growth of biotech in Massachusetts. A trail-blazer, she was the first African-American woman to lead a life sciences growth initiative of this scale. Named one of the 10 Most Influential Women in Biotech by the Boston Globe, Windham-Bannister took the reins at the Massachusetts Life Sciences Center in 2008 at a particularly difficult time in the US economy. Windham-Bannister turned this potential disadvantage into a creative force for opportunity, partnering with the private sector to, as a 2013 Northeastern University report found: “attract $3 in outside investment for every public dollar spent, turning just over $300 million in state funds into more than $1 billion worth of backing for the local life sciences sector.”

Susan Windham-Bannister will be the keynote for the Women’s Leadership Symposium and Dinner at part of our Biotech Week Boston event this October.

Mary Lynne Hedley, Ph.D. Co-Founder, President and Board Member, Tesaro

Named “Life Science Entrepreneur of the Year” by the New England Venture Capital Association this past spring, Mary Lynne Hedley Ph.D. has been rightly called a pharmacology pioneer as she has been developing cancer drugs since 1996. Hedley began her career as the co-founder of Zycos, Inc. (which later became MGI Pharma, then Eisai Co Ltd.) moving on in 2009 to become EVP of Operations and Chief Scientific Officer of Abraxis Bioscience. In 2010 Hedley co-founded (with partner Lonnie Moulder) Boston biotech Tesaro, an oncology-focused biopharmaceutical company. The first drug Tesaro brought to the market, called Varubi, manages the side effects of chemotherapy to alleviate suffering and therefore bring some normalcy to the lives of oncology patients. Tesaro is also developing cancer drug Niraparib which we wrote about here. Late last year, Don Seiffert of Boston Business Journal asked Hedley what it was like being one of a tiny minority of women in biotech. Hedley offered: “It’s probably like being a guy in biotech.” Relatively, that rings true – biotech is a tough industry for anyone, with 9 out of 10 companies that begin clinical trials unable to succeed in bringing those drugs to the market.

Katrine Bosley, Chief Executive Officer, Editas Medicine

In 2016, even the least scientifically minded among us cannot have missed the buzz about “CRISPR” technology. Katrine Bosley is at the front row and center of this pioneering technology as the CEO of Editas Medicine. This June Bloomberg called CRISPR “the genetic tool that will modify humanity” and Editas’ mission is to successfully use CRISPR to repair genes that cause mutations that cause a broad range of diseases. In 2014, when Bosley joined Editas, Alex Lash called her one of the “highest profile CEOs of the biotech scene” and her profile has only gotten higher since then. Bosley began her career at Alkermes, moved on to Highland Capital Partners, then Biogen, and then Adnexus (which was bought by Bristol-Myers Squibb).  In her first CEO role, Bosley led Avila Therapeutics into a buyout from Celgene. Prior to Editas, from 2013-2014 she was an Entrepreneur in Residence at the Broad Institute. This May, Fast Company wrote: “Five years ago this was a medical pipedream…With an aggressive timeline and a giant war chest, the Editas CEO may be the first to treat genetic mutations using CRISPR technology…as soon as next year.”

Hannah Mamuszka, Founder Alva10

You may not have heard of Alva10 – yet – but I'll take a bet that you will soon. From stints at some of the most well known Boston based and Global biotechs - Organogenisis, Takeda and ArQule as a researcher and scientist - to almost twelve years as a Director of Pharmaceutical Alliances at Exiqon (previously Oncotech) and most recently VP of Business Development at Exosome, Hannah Mamuszka’s career “has evolved based on the intersections of biotechnology and business development”. The name of her new company Alva10 was inspired by Thomas Edison - whose middle name was, of course, Alva. Mamuszka explains: "Edison was an amazing inventor and thinker, who thought about challenges completely differently than everyone else at the time, and produced radically different results as a product of that thinking. With Alva10, we are emphasizing the value that diagnostics play in personalized, precision medicine, and approaching that value from a completely different perspective than anyone else (that I've seen) in the industry.” The diagnostics she is talking about can analyze “both DNA and RNA in a molecular liquid biopsy”. This ability to translate Big Data into “well validated, broadly distributed diagnostics that are valued in the healthcare system” Mamuszka says is key to precision medicine being realized.

Barbara Fox, Ph.D. Entrepreneur in Residence at Partners Innovation Fund

With a career spanning almost thirty years that started as a Professor of Medicine at the University of Maryland and led to her current role as Founder and CEO of Avaxia Biologics, Barbara Fox has honed her networking skills into a fine art, enabling her companies to compete and win in the highly competitive world of biotech. Prior to Avaxia, Fox was an Affiliated Entrepreneur at Oxford Bioscience Partners, before that President and Chief Scientific Officer of Recovery Pharmaceuticals (now Shire) - a company she founded that develops medicines for the treatment of addiction. Her first position after teaching was Senior Scientist at Immulogic Pharmaceuticals where she quickly moved from that role into Vice President of Discovery Research. At Immulogic Fox directed programs into vaccine development, allergy, autoimmune disease and substance abuse research.

We are pleased to have Barbara Fox speak at Biotech Week Boston’s Bioprocessing International Conference and Exhibition. She will be presenting “Funding a Therapeutic-Focused Company through Angels: The Good, The Bad and the Ugly”. Fox will also be a panelist for: ”How to Overcome the Funding Gap for Biotech Start-ups and Emerging Companies”. Fox will be joined by Joshua Speidel, Latham Biopharm Group and Ohad Karnieli of Karnieli, Ltd.

Got any more women in Boston biotech you think we need to write about? We’d love to share them with our audience so Tweet to us at @BiotechWkBoston. And 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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Wednesday, August 17, 2016

Boston’s ‘Neuro’ Network will Ignite Innovation

We asked Martin Tolar, the Founder, President & CEO of Alzheon, Inc. what he thought Boston biotech would look like in 2050 earlier this week, and his response prompted us to invite him to contribute a blog post. What follows is Dr. Tolar's look into the future of biotech, specifically around neurodegenerative disorders.

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

Developing new medicines for neurodegenerative disorders is one of the most challenging areas of drug development that is pushing the new frontiers, and at last with the prospect of real breakthroughs on the horizon. 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.

Let me predict some of the progress that will be made in Alzheimer’s disease, which is the key therapeutic focus for my company, Alzheon.

Biotech companies have already emerged in the land of the ‘giants’ of the big pharmaceutical companies who have traditionally carried out Alzheimer’s drug development, and companies in this region are poised to maintain their position through 2050 for being on the leading-edge of advancing some of the first medicines to Alzheimer’s patients.

In 2050 and beyond, Boston-area biotechs are poised to maintain their position on the leading-edge of bringing some of the first approved medicines to Alzheimer’s patients. Already Boston-area biotechs are among the companies with the most advanced Alzheimer’s drug candidates in Phase 3 clinical trials, and in particular Biogen has led local drug development in this therapeutic area. Biotech companies, including those from Boston, have emerged and earned their place in the ‘land of the giants’ of the big pharmaceutical companies who have traditionally carried out Alzheimer’s drug development.

Alzheimer’s research from the Boston area will span the full spectrum of different treatment approaches for Alzheimer’s disease. New drug mechanisms for targeting Alzheimer’s will continue to emerge, and Boston researchers and biotech companies will continue to spur new frontiers for Alzheimer’s mechanisms. Amyloid-targeting is the most advanced drug development approach for Alzheimer’s, with more recent drug candidates following new science related to the role of tau protein in Alzheimer’s. Boston biotech companies will further advance the most well-established biology and mature learnings in the field of Alzheimer’s which offer the most near-term treatment possibilities. Drug candidates that target beta-amyloid, a pathway that is well-known to play a role in Alzheimer’s, are in Phase 3 trials with data on the near-term horizon, and two Boston-area companies, Biogen and Alzheon, have advanced drug candidates that target beta-amyloid.

Advances in Alzheimer’s drug development will be supported by new understanding of the genetics and sub-populations of Alzheimer’s disease, areas in which Boston’s world-class science and medical ecosystems will continue to make contributions. New insights about genetic markers for Alzheimer’s disease and deeper understanding about the underlying biology and disease progression are guiding better drug development and smarter targeting of patient sub-populations. Again, the Boston ecosystem is ideally suited to bring together scientific researchers, medical thought leaders, and biotech companies to rapidly translate new insights into drug development programs. For example, when clinical research showed that Alzheimer’s patients with the APOE4 gene have a higher risk and burden of disease, drug developers began to explore if patients in APOE4 sub-populations might be higher responders to certain drug candidates. Alzheon is proud to be a pioneer in applying a precision medicine approach to Alzheimer’s, by focusing our drug development on the APOE4 genetically-defined patients with Alzheimer’s.

At this moment in 2016, there is tremendous momentum in the field of Alzheimer’s disease. The ‘neuro network’ of industry, medicine and academia – in Boston and throughout the world – is emerging with greater knowledge about Alzheimer’s and other neurodegenerative disorders, which is driving better informed drug development. Our collective goal is to bring truly effective treatments to market that will change the lives of Alzheimer’s patients, as well as curbing the skyrocketing cost to society estimated at $1 trillion annually in the U.S. by the year 2050. As we look to 2050 and beyond, the future looks brighter than ever for achieving this goal and preventing devastation brought to patients and the impact on society.

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 2 of the event entitled "Innovation in Clinical R&D: Finding a Cure for Alzheimer’s". In this opening keynote Alzheon’s CEO will share the company’s drug development journey in neuroscience, as they innovate towards novel therapeutic solutions for Alzheimer’s Disease. Join us for insights on how innovation at Alzheon has been key in developing a promising pipeline of therapeutics in the clinic.

Martin Tolar

Please follow us on Twitter @BiotechWkBoston for more Boston biotech news and information, and don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is happening this October 4-7; you can learn more by clicking the link below.

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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 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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Monday, August 8, 2016

A Look at Three of Boston’s Biotech Disruptors: Christopher Viehbacher, Michael Pellini and Noubar Afeyan

Disruptors are a special type of innovator who go outside the box and attempt to change preconceived notions and practices. If you take a look at the meaning of the word, “to disrupt” means to “rupture” or break apart, and essentially disruptors do this - discarding the old ways to bring in new ones. We might even take it a step further and look at as the commonly used root of "dis" which means to "do the opposite of" - and see the idea that disruptors are "fixers" essentially putting the pieces of something broken back together. Here’s a look at three disruptors in Boston Biotech who are revolutionizing traditional pharma to radically change the ways cures are being brought about for disease.

Boston Biotech Disruptors

From all the stories that have been in the press about Christopher Viehbacher lately, you see a man who’s an extremely successful change agent that helped Big Pharma save billions. But dig a little deeper and you see a different Christopher Viehbacher, a person who is passionate about “bringing medicines to patients” without “distractions”. Joseph Haas from The Pink Sheet interviewed Viehbacher last year at BioPharm America™ and in the video Viehbacher comes across as fairly triumphant about having left his career in Big Pharma behind and invigorated by his current work as Managing Partner of Gurnet Point Capital and Executive Chairman of Boston Pharmaceuticals.

Interviewer Haas discussed how it’s a current trend that key executives in the industry are moving from Big Pharma to “small pharma” or biotech, and this theme was echoed throughout the entire conversation with Viehbacher. Viehbacher explained his thoughts on how he feels biotechs are simply a better model for efficiency: “There are dys-synergies of scale in this industry. As you get bigger you don’t necessarily get better. ... (Pharma) is an industry that has to reinvent itself all the time… Genzyme was an important tech investment for Sanofi, we weren’t really in biologics, we didn’t have a credible R&D base in the US. Building from scratch would have taken so long. Some of these acquisitions are not just product based but actually help you to shape the strategy of the company.”

Viehbacher explained what he sees as a big pitfall with many Big Pharmas – the inability to take risks: “What people forget about this industry – and that was particularly difficult to communicate in a big company - our business is inherently risky. If you don’t take risks, you will not advance. The question is ‘How do you manage the risk?’ That’s partly through a portfolio strategy… you need to be in multiple therapeutic areas, in multiple products. That means diversifying your sources of innovation, if you’re just betting on your own teams all the time you’re not necessarily going to come up with innovation. You have to mix sourcing of innovation from outside and from inside.”

He goes on to discuss the freedom given to biotechs versus the demands placed on Big Pharma: “You have to accept that some of the things you are going to do are not going to work out. I used to look at Regeneron, they would have Fidelity as a big investor, Sanofi had fidelity as a big investor – but it’s not the same Fidelity. And the expectations when you’re Big Pharma are buy backs on dividends. You’re a big cap company, they want you to have this predictability and sustainability of results. It became more difficult to take on risk and do innovation. In biotech there’s an assumption that this is risky and there’s an assumption that it’s going to take a while to see a payoff. Biotechs aren’t so concerned about quarterly earnings. That is one of the other reasons why I was quite happy to shift, if you really want to be involved in science and innovation you have to have patient capital and you can’t be on that treadmill of quarterly earnings. There will always be a role for biotech and big pharma but they really have to think about themselves in a complimentary way instead of a competitive way.”

How does Boston Pharmaceuticals disrupt? Well for one thing they have “no specific therapeutic area in mind” and focus on drugs targets that have already proven to have a benefit – and develop them with outside providers. Their site describes what they do well and simply: they acquire clinical stage molecules which they develop through clinical phases and then either partner and out license or keep and commercialize. The copy for the Gurnet Point Capital website strongly echoes Viehbacher’s disruptive vision: “We partner with life science leaders who have the vision and drive to transform their businesses. Those leaders are born risk-takers. Original thinkers with big ideas and bigger ambitions. Explorers who question the status quo. They are driven to succeed and are uncompromising in their quest to make things happen, even if the solutions aren’t easy or obvious.”

Michael Pellini is also a Big Pharma veteran who has transitioned to biotech. The President and CEO of Foundation Medicine, an organization that uses genetics to help select the right drugs to treat cancer patients, he and his organization are disrupting the siloed approach to cancer research to make cures happen faster and more effectively. He discusses his reasons for this disruption in a Pershing Square Foundation presentation last year.

Pellini begins: “Our aim is to democratize the precision medicine initiative and the work going on at academic centers around the US - not to be competitive with the academic centers – but let’s face it 85% of the patients in the United States never end up at Sloan Kettering, Weill Cornell or MD Anderson - they are treated in the communities around the United States.”

Pellini explains the need for what Foundation Medicine fundamentally does: “We extract information…we’re all building these databases that have to come together and they should absolutely not just come together inside my company or inside a medical center…they have to truly come together over time. (You need to combine) learning from the West Coast, Southeast, New York City, Boston, Hong Kong – because you’re going to see patients that are these snowflakes, tumors represented as snowflakes all around the world even the experts might not have seen enough of any one patient diagnosis to know exactly what to do with that patient. That issue is magnified many times over in the communities around the United States.” He emphasizes that “all data needs to be connected” and he urges the scientific community to “take the learnings from Silicon Valley.”

Moderna Therapeutics, a company co-founded by Noubar Afeyan, was given the distinction of being the #1 disruptor by CNBC last year, one of just a handful of non-tech companies in that list. Afeyan is the founder, Senior Managing Partner and CEO of Flagship Ventures, the firm that launched Moderna as well as several others. In fact, in his 30 plus year career, Afeyan has founded over 38 life science and tech startups, making him the perfect person to - know when and how NOT to start a start-up. An immigrant to the US at age 13, Afeyan credits being an immigrant as a big part of his success. He said to the Armenian Mirror-Spectator this spring: “What keeps you from innovating is being comfortable…If you’re an immigrant, then you’re used to being out of your comfort zone.”.

Two years ago Afeyan gave an inspiring talk to a group of scientists at Imperial College in London, and interestingly enough his talk was less about scientific innovation than it was about the business aspect of how to make scientific discoveries successful. Afeyan opened his talk with the affirmation that the most important innovation in the last fifty years wasn’t the internet, biotherapeutics or even genome sequencing but “startup ventures” as an entity. Afeyan makes this point: “Startup ventures are a fairly new phenomenon. When I was at MIT in the mid-1980s there was maybe one company every year of any note that was being created.”

He goes on to describe his experience of founding multiple startups, and offers the idea that startups as the way they are now will radically change. Afeyan is at the epicenter of that change with his organization Flagship Ventures: “Flagship ventures asks the question, can you think systematically about innovation, instead of doing it when it comes to you or when an opportunity is presented. Venture Labs is an institutional attempt to create first of their kind companies around technologies developed to solve a problem – not around advancement of science.”

Please follow us on LinkedIn at our company page or on Twitter @BiotechWkBoston for more Boston biotech news and information, and don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is happening this October 4-7; you can learn more by clicking the link below.

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Monday, August 1, 2016

How Venture Philanthropy is Helping 5 Boston Biotechs Develop Cures

Researching and developing lifesaving medicines can take as long as 15 years and the costs are high – recently estimated at $2.6 billion by the Tufts Center for the Study of Drug Development. Venture capital can be hard to get, especially in the early stages of the drug pipeline. Broadview Ventures summed up the situation in their Venture Philanthropy in the Healthcare Marketplace report: “Venture capitalists are risk averse. NIH is retracting. Big Pharma is refocused on marketing and commercialization, as the productivity of R&D declines. This widening translational gap threatens to strand promising products and cures.”

Luckily venture philanthropy is increasingly fulfilling those financial needs with important bridge funding. And where once philanthropies focused on funding academic and institutional research, more and more are beginning to take a look at biotech companies, and essentially helping to build those bridges from bench to bedside in order to expedite the delivery and approval of life saving therapeutics. Chris Colecchi of Broadview Ventures was quoted in their report: “Venture philanthropy can disrupt the status quo with fewer dollars than people think. Modest amounts of money can fund early stage research in that translational space.” Indeed their research shows that 51% of the funding that venture philanthropies are giving focuses on the preclinical stage of development, a “key unmet need in the funding landscape”. But just exactly who is funding medical research, and what research are they funding? Here are examples of five Boston biotechs who are working with patient advocacy foundations to develop cures.

Five years ago the Juvenile Diabetes Research Foundation (JDRF) stepped in to help Boston biotech Selecta Biosciences develop a vaccine for type 1 diabetes, specifically a therapeutic which would prevent the autoimmune response that causes type 1 diabetes. Since JDRF’s inception, JDRF has contributed more than $2 billion to T1D research and is currently funding 50 human clinical trials of potential T1D therapies. Selecta’s ability to begin this research with the help of JDRF’s funding led them to the success they have now; just this June they raised $70 million in an IPO. Selecta describes their technology as being: “designed to communicate precise instructions to the immune system and to expand the use of vaccines to immunotherapies that enable a new generation of biologic therapies to treat autoimmune diseases, allergies, and cancer.” You can learn more about JDRF grants and the work they do here.

Akashi Therapeutics is unusual in that it was founded not by VCs but by nonprofit disease advocacy groups for the main purpose of developing a cure for Duchenne muscular dystrophy. Duchenne is a disease which affects about 300,000 people a year, primarily boys, and leads to progressive decline in muscle function and early death. Akashi Therapeutics was founded in 2010 – known then as Dart Therapeutics - and by 2014 it had raised $2.5 million dollars from 25 different patient advocacy groups. One of those groups was CureDuchenne. Akashi has had to suspend an experimental treatment called HT-100 for Duchenne following the tragic death of one child who was being given a high dose of the drug. (You can get an update from their CEO Marc Blaustein here where he talks about next steps for the HT-100 study.) Early this year CureDuchenne’s president and CEO Debra Miller spoke with Boston Business Journal about the situation: “We are confident that the team at Akashi will be able to isolate the problem soon. Anti-fibrotic drugs are needed, and CureDuchenne will continue to fund a research pipeline so that we can treat all aspects of this devastating disease.” CureDuchenne is currently funding 10 clinical trials and 7 preclinical studies as well as the Center for Duchenne Muscular Dystrophy at UCLA. You can apply for a research grant directly on their website here.

Early this year The Boston Globe described The Cystic Fibrosis Foundation and their then President Robert J Beall as having “pioneered” the model of “venture philanthropy”. In fact, The Cystic Fibrosis Foundation have invested a great deal of their resources in the Boston area: with early investment in Vertex Pharmaceuticals, a current alliance with Shire PLC and their own research lab in Bedford, Massachusetts. Late this spring Boston’s Editas Medicine made an exciting announcement – that The Cystic Fibrosis Foundation would be giving them up to $5 million for their research on CRISPR/Cas9 based medicines. CFF’s press release says that in 2015 they: ”gave approximately $15 million in grants to nearly 50 scientific laboratories to fund CF research on emerging technologies such as gene editing, gene delivery and stem cell research.” Learn more about CFF research awards here.

Last year Emulate was awarded a grant from the Michael J. Fox Foundation to apply the company’s “organs-on-chips” for the investigation of drug candidates for Parkinson’s disease. Organs on chips basically simulate the real thing for testing, to give more predictive data as well as a deeper understanding of both drug and disease. This funding led to Emulate eventually raising $28 million in a Series B Round this past March, as well as partnerships with both Merck and Johnson & Johnson. The Michael J. Fox Foundation website shows recent grants to Boston’s own MGH and MIT, and their press says they have funded more than $600 million in research to date fundamentally altering the “trajectory of progress toward a cure” for Parkinson’s disease. You can learn more about MJFF grants here.

Back in 2012, Constellation Pharmaceuticals began a partnership with The Leukemia & Lymphoma Society (LLS) to “advance the development of a novel BET inhibitor.” LLS provided $7.5 million in funding for their Phase I trial. Since then Constellation has established an alliance with Genentech and gone on to get $55 million in financing from venture capital. Their website describes why they develop drugs that target epigenetic function: “Our research has shown that when epigenetic regulatory events occur aberrantly, the proteins that regulate these events can become drivers of disease. Inhibiting these targets with novel agents promises to be a powerful avenue to develop important treatments serving unmet medical needs.” LLS invested a total of $67.2 million in cancer research last year. You can learn more about partnering with LLS here.

Please follow us on LinkedIn at our company page or on Twitter @BiotechWkBoston for more Boston biotech news and information, and don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is happening this October 4-7; you can learn more by clicking the link below.

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Monday, July 25, 2016

5 Boston Biotech Breakthroughs We Could See in 2017

Early this year, Tufts Center for the Study of Drug Development reported that “developing new drugs has become more complex and more expensive than ever.” What is increasing the cost? For one, Tufts CSDD reports, “a typical Phase III protocol now entails an average of 167 procedures” which is 60% more than it was just 16 years ago in 2000. With statistics like these it’s not surprising that 9 out of 10 companies that begin clinical trials never bring a drug to market. However, even with these odds biotech companies are persevering and in some cases thriving, especially in Boston’s Biotech Hub. Boston is a hotbed of discovery and innovation, with startups attracting VC investment and Big Pharma organizations like Merck building new labs here even as they cut teams in other parts of the country. Right now Boston has several promising drugs in the pipeline, with Phase III studies completed and FDA approvals on the horizon. Read about five success stories we could see coming out of Boston biotechs in 2017.

Biotech Week Boston

According to their data, Sarepta Therapeuticsdrug for Duchenne muscular dystrophy (DMD) “Eteplirsen” has been effective in a Phase III clinical study. Because DMD is both rare and serious, Sarepta has been a candidate for a sped up review process from the FDA, however as Robert Weissman from the Boston Globe recently reported an “influential advisory committee” has recommended “to reject its experimental treatment”. Sarepta’s March press release explains: “The FDA has granted Eteplirsen Priority Review status, which is designated for drugs which provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to Eteplirsen, as well Orphan Drug Designation and Fast Track Status.” What does Eteplirsen do? Sarepta explains: “The underlying cause of DMD is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Our investigational therapies for DMD are designed to skip an exon in the dystrophin pre-m RNA to enable the synthesis of a functional shorter form of the dystrophin protein.”

The action date for FDA review was late May, but the FDA requested more data from Sarepta in June. Sarepta explained the dire need for a Duchenne cure, and how they could potentially meet that need, in their March press release: “It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13% of people with the disease having mutations addressable by Eteplirsen/exon 51 skipping.” This potential for a cure gives new hope for those affected by this disease, a hope which has been increasingly channeled into activism. Fierce Biotech reported last month that: “Hundreds of patient advocates, patients and families turned out to give their raucous support for an approval during the (FDA) panel review and vote.” In addition, the Washington Post reported that families whose sons have taken Eteplirsen have documented the “apparent halt in their sons' decline” on Facebook and YouTube. Note: since this article was written, biotech Santhera was set back by potentially three years by the FDA decision for them to run another Phase III study for their Duchenne’s drug. The jury is out on how this decision will affect Sarepta’s chances. Matthew Herper of Forbes had this to say on Twitter right after that decision: “I don’t think there is any read-through from Santhera to Sarepta.”

Back in 2013, Robert Weissman reported on this devastating Boston biotech setback: “It took Aveo Pharmaceuticals Inc. seven years to develop a much anticipated kidney cancer drug (Tivozanib). Federal regulators needed only about four hours to crush the company’s hopes.” Three years later, after a corporate restructuring (the SEC has threatened to ban three of its former executives), loss of Biogen and Astellas as research partners, a rebrand as Aveo Oncology and then gain of $17 million in funding, Aveo has given the go ahead from the FDA to begin a Phase III trial of Tivozanib for renal cell carcinoma. What does Tivozanib do? Aveo’s website describes it as: “a potent, selective, long half-life inhibitor of all three vascular endothelial growth factor (VEGF) receptors that is designed to optimize VEGF blockade while minimizing off-target toxicities.” Why blockade VEGF? Aveo explains: “vascular endothelial growth factor (VEGF) pathway plays a significant role in angiogenesis, which is critical in cancer”. With perhaps lessons learned from how they work with the FDA, success may be on the horizon for this drug, eagerly awaited by patients and patient advocates.

In partnership with NSGO (Nordic Society of Gynaecological Oncology), Tesaro just had the first successful Phase III trial of a PARP inhibitor Niraparib (NOVA) for ovarian cancer. Tesaro’s NDA and MDA submissions “are planned for Q4 2016”. Niraparib (BRAVO) is also in a Phase III trial for the treatment of breast cancer. One of the youngest companies of the five on our list, founded in 2010, it is also one of the most successful, with a commercial drug for chemotherapy side effects called Varubi, a $500 million deal with Janssen for commercial use of Niraparib for prostate cancer, and plans to add 100 employees by the end of the year.

Paratek Pharmaceuticals’ website describes their efforts as “working to change how bacterial infections are treated.” Having just received positive results in Phase III trials for Omadacycline, they are poised to fulfill that promise. Paratek believes that Omadacycline will solve a great deal of the issues with the bacterial resistance doctors are increasingly dealing with. Paratek says that Omadacycline is “Active against drug resistant pathogens, including methicillin-resistant Staphylococcus aureus, penicillin-resistant and multi-drug resistant Streptococcus pneumonia da, and vancomycin-resistant Enterococcus species.”

ArQule is currently in two Phase III trials for Tivantinib to treat hepatocellular carcinoma, the most common type of liver cancer, in partnership with Daicchi Sankyo and Kyowa Hakko Kirin. The National Cancer Institute gives a great explanation of what exactly Tivantinib does: ”Tivantinib binds to the c-Met protein and disrupts c-Met signal transduction pathways, which may induce cell death in tumor cells overexpressing c-Met protein or expressing constitutively activated c-Met protein.” In more layman’s terms, it’s a “kinase inhibitor” a type of research in which ArQule specializes, having “eight kinase inhibitors into human clinical trials with a ninth about to enter the clinic.” In late June ArQule was featured by Zack’s Investment Research Firm with the call to action “Forget Valeant, Invest in These Attractive Stocks Instead.”

Two Boston biotechs that may not have an impending breakthrough, but are looking fairly certain (if anything is certain in this biotech world) to have success by 2020, are Alnylam and Boston Biomedical. Andrew McConaghie raved about Alnylam this June in Pharma Phorum, saying it was: “one biotech company which is tipped to deliver on huge expectations, thanks to a rigorous approach to validating its science.” Alynlam proposes to have three drugs approved and another ten in the pipeline by 2020. Alynylam’s core focus is RNAi therapeutics, and Pharma Phorum describes their pipeline as: ” based on targets in the liver, but covering a broad range of diseases; rare genetic diseases, cardiometabolic conditions and hepatic infectious diseases, such as hepatitis B.”

Boston Biomedical’s Napabucasin was just granted Orphan Drug Designation from the FDA in the treatment of gastric cancer this June. Orphan Drug status is given to: “Drugs that are not developed by the pharmaceutical industry for economic reasons but which respond to public health need.” Boston Biomedical is currently in Phase III trials for this drug, which inhibits cancer cell pathways by targeting the STAT3 pathway.

Please follow us on LinkedIn at our company page or on Twitter @BiotechWkBoston for more Boston biotech news and information, and don’t forget to check in every week for our Biotech Week Boston blog series. Biotech Week Boston is happening this October 4-7; you can 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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Friday, June 10, 2016

Innovative Development Strategies and Applications for Bispecific Antibodies

The team behind the Next Generation Protein Therapeutics Summit have recently produced an exclusive whitepaper titled "Innovative Development Strategies and Applications for Bispecific Antibodies". Below you will find a brief summary of the whitepaper and download the complete whitepaper now.

Whitepaper Summary:

The phenomenal growth of the bispecific antibody arena has culminated in 60 unique constructs, more than 30 in clinical development, and two on the market as therapeutics for a wide variety of cancer types and numerous diseases/disorders. Bispecific antibodies are specially engineered antibodies which simultaneously bind to two different epitopes on the same antigen or different antigens, increasing selectivity and effectiveness. [1]

The focus in incorporating bispecific antibodies within oncology applications has been in either blocking multiple and redundant signaling pathways involved in oncogenesis or redirecting immune effector cells to be in close proximity to tumor cells. In non-oncology applications, a major developmental effort has gone into blocking pro-inflammatory cytokines.[2, 3]

Despite successes in development there are some critical hurdles to overcome and there is a need for innovation and improvement. Manufacturability issues such as low expression yields and product instability/short half-life have hindered development. Challenges lie in the need for rapid discovery of lead bispecific antibodies with optimal selectivity for their targets, and a need for rapid purification techniques. Adverse effects from immunogenicity, mainly caused by a “cytokine storm,” can stifle clinical trials.[3]

Development efforts have provided some solutions to these hurdles. Researchers at Eli Lily are using mathematical modeling parameters to make predictions about how engineered antibody properties will affect binding to cell surface antigens, ultimately optimizing developability. [4]Another novel strategy involves monitoring target/ligand binding of bispecific antibodies through surface plasmon resonance (SPR), which allows users to view the dynamics of bispecific antibody binding and dissociation events with two targets. [5]

The short half-life of scFv-based bispecific antibodies is a major drawback compared to that of IgG-like bispecific antibodies. Successful half-life extension, and in some cases, recycling, has been achieved by attaching a variety of components: PEG chains [6], human serum albumin, and Fc fragments [1]. In another novel approach, human mesenchymal stromal cells (MSCs) can be genetically modified to produce and secrete bispecific antibodies that accumulate near tumors continuously throughout the lifetime of the patient.[7]

A plethora of unique applications are being investigated for bispecific antibodies. One is in delivery of therapeutic antibodies across the blood-brain barrier for neurological conditions. [8]Another innovative application involves engaging bispecific antibodies to deliver drug, nanoparticle or radiolabel payloads to tumor sites. [1]Bispecific antibody-based immunoassays are being developed for diagnosis of patients with various infectious diseases: SARS, hepatitis B, tuberculosis, as well as E. coli infections. [9]Another application involves tackling the rising threat of antibiotic resistance through specially designed constructs effective against antibiotic resistant bacteria such as Pseudomonas aeruginosa. [10]

This exciting and fast moving arena includes many creative design formats, and innovative solutions for numerous development and manufacturing issues. There are still many unmet needs, but the field is bound to yield many more successes.

  1. Fan, G., et al., Bispecific antibodies and their applications. Journal of Hematology & Oncology, 2015. 8(1): p. 1-14.
  2. Spiess, C., Q. Zhai, and P.J. Carter, Alternative molecular formats and therapeutic applications for bispecific antibodies. Mol Immunol, 2015. 67(2 Pt A): p. 95-106.
  3. Spasevska I, D.M., Klein C, Dumontet C, Advances in Bispecific Antibodies Engineering: Novel Concepts for Immunotherapies. J Blood Disord Transfus 2015. 6(243).
  4. Rhoden, J.J., G.L. Dyas, and V.J. Wroblewski, A Modeling and Experimental Investigation of the Effects of Antigen Density, Binding Affinity, and Antigen Expression Ratio on Bispecific Antibody Binding to Cell Surface Targets. J Biol Chem, 2016.
  5. Karllson, R., Applications of Surface Plasmon Resonance for Detection of Bispecific Antibody Activity. Biopharm International, 2015. 28(10): p. 38-45.
  6. Kontermann, R.E., Strategies for extended serum half-life of protein therapeutics. Curr Opin Biotechnol, 2011. 22(6): p. 868-76.
  7. Aliperta, R., et al., Bispecific antibody releasing-mesenchymal stromal cell machinery for retargeting T cells towards acute myeloid leukemia blasts. Blood Cancer Journal, 2015. 5: p. e348.
  8. Couch, J.A., et al., Addressing Safety Liabilities of TfR Bispecific Antibodies That Cross the Blood-Brain Barrier. Science Translational Medicine, 2013. 5(183): p. 183ra57-183ra57.
  9. Byrne, H., et al., A tale of two specificities: bispecific antibodies for therapeutic and diagnostic applications. Trends Biotechnol, 2013. 31(11): p. 621-32.
  10. DiGiandomenico, A., et al., A multifunctional bispecific antibody protects against Pseudomonas aeruginosa. Science Translational Medicine, 2014. 6(262): p. 262ra155-262ra155.

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Friday, June 3, 2016

Creating New Pathways for the Development, Translation, and Delivery of Immunotherapies

The team behind the Next Generation Protein Therapeutics Summit have recently produced an exclusive whitepaper titled "Creating New Pathways for the Development, Translation, and Delivery of Immunotherapies". Below you will find a brief summary of the whitepaper and download the complete whitepaper now

 Whitepaper Summary:

The last decade has seen substantial growth of immunotherapy treatments for cancer. The new immunotherapies have produced exciting results in terms of response rates to treatment for certain cancers, such as melanoma, which have been refractory to treatment, once substantial metastasis has occurred. The US Food and Drug Administration (FDA) has approved several antibodies against immune checkpoint inhibitors based on the encouraging results in clinical trials. Other types of immune therapies are also being developed which may also be useful in the treatment of cancer.

New antibody/cytokine fusion proteins, anti-CD 137 monoclonal antibodies (mabs) for stimulating cells of the immune system, more refined uses of cytokines in immunotherapy treatments, cancer vaccines, use of genetically engineered T cells to specifically target tumors, new techniques in stimulating the innate immune system and combinations of immunotherapies have great potential for being effective treatment options for cancer treatment in the future.

Challenges, however, remain to perfect these therapies for use in the clinic for cancer treatment. The nature of these challenges are scientific, clinical and regulatory. Scientific challenges include defining the appropriate tumor types for an appropriate immunotherapy, the conditions under which various cells of the immune system would be activated and determining how best to manipulate them to allow for maximum tumor cell destruction, finding better ways to circumvent cancer cell suppression of immune cell responses and using new techniques to prevent or ameliorate the nonspecific injury to normal tissue that can occur as a side effect of immunotherapeutic treatment.

Clinical challenges include ensuring there is adequate quality control in manufacturing process to supply the agent in pure enough form for clinical trials, obtaining enough patients that meet inclusion criteria, designing clinical trials that will yield adequate information to assess the safety and efficacy of the treatment and defining appropriate endpoints for a cancer clinical trial to effectively interpret clinical data gained from the clinical trial.

Regulatory challenges can occur at multiple levels for companies or entities trying to gain FDA approval for their immunotherapy technique or product. These challenges can be at the preclinical level, clinical or manufacturing levels. Adherence to regulations governing Good Laboratory Practices, Good Clinical Practices, and Good Manufacturing Practices can be problematic when trying to take immunological reagents or cellular treatments from the research laboratory to the cancer treatment clinic.

Despite the challenges when conducting research and treatment with immunotherapies, the intense level of research and data being generated with immunotherapies for cancer will ensure that a wide variety of new therapies will be possible in the coming years.

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Tuesday, May 3, 2016

Preparing for future bioprocessing challenges with the Cellca CHO Expression Platform

Therapeutic protein development and production is a very technically challenging, time consuming and expensive endeavor.

What are some of the challenges? There are numerous critical decisions that must be made with regard to mammalian cell line choice (CHO, NS0, etc.), appropriate gene and marker expression systems, cell culture medium and feed optimization, clone selection and scale-up strategies. Biopharmaceutical developers should be careful to choose options which deliver the following:

• Maximum protein titer
• Scalability (5L – 1000L)
• Long-term Stability
• Cost effectiveness
• Shortened development timelines
• No burdensome IP concerns

Why is it important and critical to understand these challenges? There are considerable economic considerations to be made; one should maximize financial budgets by minimizing expenditures on equipment and technologies required for in-house development and characterization of the therapeutic protein. It’s important to make efficient and productive investments in R&D; technology and process development requires highly trained and expert staff. There are also commercial or market considerations which will affect return on investments; to maximize IP and patent life the therapeutic protein must be developed and commercialized as quickly as possible to gain maximum market share in a highly competitive environment. First to market is paramount for biosimilar developers.

How has the CRO industry responded? “Turn-key” cell line development and bioanalytical testing CROs are currently open for business and accessible for outsourcing nearly all upstream, downstream and biomolecular analytical activities. Sartorius Stedim Biotech offer the most comprehensive, scalable and unique bioprocess portfolio to support Biosimilar development and manufacturing. With Sartorius Stedim Biotechs’ recent acquisition of Cellca (cell line development) and BioOutsource (bioanalytical and biosafety testing), there is now established a fully functional, and integrated CRO uniquely positioned to support clients development programs across the entire product lifecycle.

The result of outsourcing biopharmaceutical development and production to experienced CROs will have major positive implications for shortening the time to commercialization at significantly reduced costs.

To learn more about the industry leading Cellca CLD platform which delivers stable, high titre clones, (95% of projects have delivered titres >3.0g/litre) in a proven scalable fed batch manufacturing process in a cost and time efficient manner please join our talk by Dr. Brian Wendelburg at IBC Cell Line Development & Engineering Conference, San Francisco, 14th June at 1.25pm.

Author Bio:

Dr. Brian Wendelburg received his Ph.D. in Molecular Biophysics from the Institute of Molecular Biophysics at Florida State University. He then completed his postdoctoral training at the University of North Carolina’s Lineberger Comprehensive Cancer Center. His professional career has involved diverse roles in sales and business development activities at leading Biotech companies such as Cepheid, Affymetrix and Miltenyi Biotec where he gained a strong technical background in genomics, proteomics and cell and gene therapy applications. He joined the Sartorius-Stedim Biotech team in the spring of 2016 and is currently Sr. Field Marketing Manager for the BioOutsource and Cellca divisions.

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Tuesday, April 26, 2016

Cell Culture Media: Balancing Resources with Results

Why does medium matter?

It all starts with media. Without an appropriate growth medium, no biopharmaceutical process would exist. The proper medium allows for cells to grow and generate product. Going beyond the fact that cell culture processes would simply not exist without cell culture media, the medium (and associated feeds for fed-batch processes) represents a large portion of the cost of a biopharmaceutical process. Cell culture media are complex entities that encompass large quantities of raw materials as well as a high level of technical expertise, driving up the cost per litre.

What are the advantages of an optimized medium formulation compared to an off the shelf product? 

Many people will choose to start with standard off the shelf products for their cell culture processes. While there are a wide variety of products available on the market, there are several important reasons to consider using an optimized medium formulation instead. The first is to tailor a medium formulation specifically for your own cell line. Every cell line will respond differently to standard products, and optimizing a medium formulation is the best way to ensure that you use a medium that works best for your application and requirements. Additionally, as you generate different cell lines with varying genetic constructs for new products, your cells may begin to respond differently. Having an optimized medium formulation allows you to adapt to these changes while maintaining growth and productivity. Using a standard product also means you are bound to a single vendor with no access to proprietary formulations. With your own optimized medium, you have full access to the formulation, with the ability to manufacture it with a vendor of your choice.

What factors should I consider when developing an optimized medium?

As discussed previously, optimizing a medium formulation to get you the best growth and productivity is the main driving factor, and allows you to minimize the amount of medium used and therefore your overall COGS. It is important to also consider what the criteria are for your optimized medium. For many industries, having a fully chemically defined, non-animal origin medium is critical. This minimizes lot to lot variability and is highly desirable from a regulatory standpoint. Ease of use is also important to consider. This encompasses storage aspects, such as shelf life, and shipping and handling conditions. Ease of use also involves simplifying the process for the operator. As media and feeds become increasingly complex, how difficult does it become to formulate? Will operators have to perform risky pH adjustments with large volumes of corrosive reagents? Will high temperatures be needed to dissolve certain components, and can these high temperatures be achieved as the industry moves towards more single-use technologies? These and other factors must be considered when developing an optimized medium formulation.

What resources are required to develop an optimized medium?

The actual raw materials that form the bulk of a cell culture medium are generally inexpensive. The resources required for an optimized medium formulation that drive up the ultimate cost per liter are time, personnel, equipment, and expertise. With the increasing use of multivariate approaches (Design of Experiments, Principal Component Analysis), the use of high throughput systems become highly advantageous, reducing personnel requirements. Finally, expertise in media development is something that comes only with years of experience in the industry and cannot be bought with any amount of money.

So how do I develop an optimized medium formulation?

There are several approaches to media optimization. Some methods include titration, reverse engineering of other media, and metabolomics. While some of these methods are effective, when implemented on their own, they are time consuming, labor intensive, and heavily resource-dependent. A modern, DoE based approach to media development for CHO based processes is the CHOptimizer® Media Builder. CHOptimizer® consists of three distinct phases. The package is designed to be integrated into our ambr™15 system for automation and ease of use. In the first phase, four chemically defined, non-animal origin based media are blended in different ratios, according to a mixtures design DoE approach. Subsequent phases incorporate spent media analysis and fractional factorial DoE approaches to develop an optimized medium formulation, feed formulation, and corresponding feeding strategy. CHOptimizer® base media are developed using the expertise of Lonza Biologics, and combines a modern, high throughput approach with traditional ideas to deliver an optimized medium in a short timeline, with field based support. It also offers full access to the formulation for ultimate flexibility in the future.

To learn more about CHO media optimization join Sartorius Stedim Biotech’s workshop, chaired by Dr. Michael Gillmeister, Lonza at the IBC Cell Line Development & Engineering Conference, San Francisco, 13th June at 11.45am.

About the author:
Dr. Michael Gillmeister received his Ph.D. in Chemical and Biomolecular Engineering from the Johns Hopkins University in collaboration with the University of Maryland School of Medicine specializing in glycosylation, transient protein production, and neurobiology. In 2009, Mike joined the Gibco® research and development group and was responsible for next-generation media and sera projects. He then led media and process development projects to modulate product quality and maximize titer for PD-Direct® Custom Media Services using high-throughput and bioreactor technologies. Currently, Mike leads Protein Expression Media R&D and the CHOptimizer™ media optimization service for Lonza Walkersville.

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Wednesday, April 20, 2016

Australia's Future in Global Peptide Drug Development

Paul Watt, Chief Scientific Officer, Phylogica Ltd, Australia sat down with the TIDES team to discuss the future of global peptide drug development in Australia as well as the current challenges for intracellular drug delivery and the important roles that academic institutions have in the development and commercialization of peptide therapeutics. Below you will find a brief excerpt from this exclusive interview. To access the complete interview, click here.

In terms of investment and innovation, where do you see Australia's future in global peptide drug development?

Australians have had a long history in peptide discovery. For example the pioneering work of Professor Mario Geysen enabled the parallel synthesis of peptides in the early 80's. More recently Australia has accumulated multiple high achieving peptide scientists such as Paul Alewood (eg. synthesis toxin peptides including use of selenolanthionine bridges), Richard Lewis and Glenn King (ion channel active venom peptides) (including ion channel inhibitors). David Craik (cyclotides and structural biology of complex peptides), John Wade (neuropeptides/relaxins). Australian peptide innovation has been successfully commercialised with the establishment of multiple companies, including Mimotopes, Auspep, Protagonist and Phylogica. As many of these companies mature, I see a bright future in the development of the Australian peptide industry. In addition several peptides discovered in Australia have been subject of multiple alliances with big Pharma offering further commercial potential as they progress through clinical development.

To learn more from Paul, join him at TIDES, May 9-12, 2016 in Long Beach, CA. Where he will be discussing how to design oligonucleotides with better drug-like properties to accelerate your products from discovery/preclinical to the clinic and to market.  

Register for TIDES now and save $100, use the code B16180BLOG100.

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Monday, April 18, 2016

The Next Generation Protein Therapeutics Summit: Earn a complimentary pass as a Guest Blogger!

Earn a complimentary all-access pass to The Next Generation Protein Therapeutics Summit by serving as a Guest Blogger at the event. As a Guest Blogger, you’ll have access to the Next Generation Protein Therapeutics, and the co-located Cell Line Development & Engineering, and Bioconjugates: From Targets to Therapeutics events’ comprehensive agenda attracting the best insights from around the world, right in San Francisco, California in June.

The Next Generation Protein Therapeutics Summit
and the co-located 
Bioconjugates: From Targets to Therapeutics and Cell Line Development & Engineering
June 13 - 15, 2016
Parc 55 Hotel, San Francisco, CA

The Next Generation Protein Therapeutics Summit's image depicts Nanobody protein therapeutic molecule

We are looking for an industry expert with interest in the following topics:

• Speed of discovery and development
• Alternative administration routes
• Potency and payload capacity
• New potential targets in cancer
• Half-life
• Robustness of discovery and preclinical development of bispecific antibodies
• Generation of human bispecific antibodies
• Efficiency of CMC development
• Development of large scale, high yield and manufacturing process

• High-Throughput Platforms for Cell Line Development
• Advances in Host Cell Engineering
• Improving Processes and Product Quality
• Improving Biosimilarity
• Assuring Clonality and Stability
• Innovations in Alternative Expression Systems and Novel Host Cell Lines
• Cell Line Development and Modification for Novel Modalities 
• Genome Editing in CHO cells
• Cell Line Development and Modification for Difficult-to-Express Proteins
• Manufacturing Assessment Strategies
• Understanding CHO Metabolisms

• Intellectual Property and Linker Payloads 
• Progress in Combination Therapies 
• Breakthrough Discoveries and Advances in ADCs
• Analytical and Characterization Strategies 
• Eliminating Heterogeneity: Advancements in Site-Specific Conjugation 
• Innovations in ADC Design and Development
• Regulatory Considerations for ADCs 
• From Discovery to Commercialization: Readiness for Manufacturing & Commercialization
• Perfecting the Chemistry Behind Conjugation 
• Conjugate Vaccines and Novel Conjugate Technologies

...and who would like to learn more about protein therapeutics!

The premise is to provide protein therapeutics related articles, whitepapers, and overall original content with a strong focus on the whole protein engineering & design industry.
What You get is:

FREE pass to the conference (valued up to $2,999.00);
• Access to extensive social learning activities;
• Exclusive admission to a networking community in the industry of your interest!

You also have a chance to GAIN exposure through our blog with over 2000 unique visitors monthly and more than 20 related LinkedIn groups with over 65,000 subscribers combined!

Learn more about the The Next Generation Protein Therapeutics Summit event by visiting our website.

Interested & want to learn more about this opportunity? Please contact Ksenia Newton at Feel free to share your short biography, links to your blog or writing samples, along with a few sentences about why we should choose you to become the Guest Blogger for the Next Generation Protein Therapeutics Summit 2016!
We hope to have you join us in San Francisco!

* Guest Bloggers are responsible for their own travel and lodging.

* All content is subject to IBC approval.

Stay tuned: 

Visit the website:
Future of Biopharma Blog

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