Yearly Archives: 2019


Opinion: Neighborhood Health Clinic contributes to the overall wellbeing of Naples – Naples Daily News

Leslie Lascheid, CEO Published 12:44 p.m. ET Oct. 16, 2019

On Monday, October 7, 2019, Gregg Semenza, MD, Ph.D., Director of Vascular Research at the Institute for Cell Engineering, Johns Hopkins University School of Medicine and American Cancer Society Research Professor, received a call he will never forget.

Shortly before 4 a.m., the Nobel Committee called Semenza to inform him that hed won the Nobel Prize for Physiology or Medicine for his discovery of hypoxia-inducible factor 1 (HIF-1), the protein complex that turns off and on our genes in response to low oxygen levels.

Leslie Lascheid(Photo: rolandscarpa[photography])

Specifically, the role HIF-1 plays in cancer metastasis (spreading), metabolism, stem cell maintenance, immune evasion and chemotherapy resistance.

Awarded jointly to Semenza; William G. Kaelin Jr., Harvard University; and Sir Peter J. Ratcliffe, University of Oxford in England, at the Karolinska Institute in Stockholm; his finding has far-reaching implications for cancer treatment, coronary artery disease, blood disorders, diabetes, eye diseases and other conditions.

As the C. Michael Armstrong Professor of Pediatrics at Johns Hopkins, Dr. Semenzas relationship opened a window of opportunity for the clinic since C. Michael Armstrong, who funds Semenzas position serves on our executive board as treasurer. As a result of the connection, we were able to bring Semenza to tour the clinic and update our medical committee and volunteer physicians on his research inFebruary 2018.

Dr. Semenza has visited Naples as part of the Neighborhood Health Clinics ongoing efforts to provide the 250 physicians who volunteer at our campus access to the latest research, technological advances and practices available.

With Armstrong on the Board, our ability to bring such leaders to Naples has expanded. In fact, he arranged for Dr. Ben Carson, neurosurgeon and United States Secretary of Housing and Urban Development (HUD), to tour and host a private physician seminar in 2014.

The importance of these visits is multifaceted. First, the information these leaders share not only benefits the uninsured workers our 385 medical/dental volunteers provide during 11,000 patient visits annually, but enhances the care they offer in their private practices and hospital service across the area.

Its also a meaningful way for the clinic to say thank you to the professionals who so generously donate their time and expertise providing more than 27,095 medical and dental procedures annually.

Educational opportunities like these also provide another avenue for the clinic to fully engage our board members, as well as show our supporters that our charity is a good steward of the resources entrusted to us. Finally, its another way the clinic contributes to the overall wellbeing of Naples.

When my parents Dr. Bill and Nancy Lascheid started the Neighborhood Health Clinic in 1999, they couldnt have imagined that we would provide more than 107,000 patient visits in the years that followed.

They did see that we would continue to expand to meet the growing needs of the areas uninsured workers, which now exceeds 50,000 women and men.

So, as we congratulate clinic-friend Dr. Semenza on his accomplishments, we also honor all the medical researchers working diligently to ensure nonprofits like the Neighborhood Health Clinic truly do provide our communities both hope and healing.

For more information, including volunteer opportunities, please visit http://www.NeighborhoodHealthClinic.org, or call 239.261.6600.

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Opinion: Neighborhood Health Clinic contributes to the overall wellbeing of Naples - Naples Daily News

New research center to explore muscle health and disease – Newswise

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Newswise With collaborating labs across the University of Washington campus and at other Seattle-area institutions and beyond, the Center for Translational Muscle Research will encompass a myriad of muscle science and disease investigations. Studies will range from the basics of muscle-related proteins, genes and cell biology to the design of potential treatments for devastating muscle diseases. At present, only symptom management and supportive care is available for many of these conditions.

The latest advances in such areas as gene therapy and stem cell biology are putting medical science closer to finding options for people with as-yet incurable muscle conditions that cause disability and shorten lives. A few of these disorders eventually result in neuromuscular breathing weakness or failure requiring mechanical ventilation.

What the new muscle research center offers patients with these diseases, many of whom are racing against time, is hope, said center director Michael Regnier, professor of bioengineering, a jointly operated department of the University of Washington College of Engineering and the UW School of Medicine.

A few of the several diseases for which the new center initially will be seeking answers are

Time is of the essence for many patients eagerly awaiting treatment progress in muscle diseases that are characterized by a decline and weakening.

Clinical partners include pediatric experts in rare genetic disorders and physical medicine physicians who care for patients with neuromuscular disorders at the UW Medical Center Rehabilitation Medicine Clinic and other settings.

One of several areas in which the center is expected to take a major leadership role is in growing human stem cells, some of which will be derived from patient cells, to produce disease-in-a-lab-dish models. These models will improve understanding of how the disease pathology begins and develops, and will also serve for testing possible treatments.

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New research center to explore muscle health and disease - Newswise

Magenta Therapeutics Appoints Jan Pinkas as Senior Vice President, Head of Translational Sciences and Announces Transition of Chief Scientific Officer…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced the appointment of Jan Pinkas, Ph.D., as Senior Vice President, Translational Sciences. The Company also announced that Mike Cooke, Ph.D., Chief Scientific Officer, will leave Magenta to pursue other opportunities.

We have made tremendous progress at Magenta, with two clinical programs in multiple trials and with our targeted antibody-drug conjugates (ADCs) for patient preparation advancing toward the clinic, said Jason Gardner, D. Phil., Chief Executive Officer and President, Magenta. Jan is an expert drug developer who will provide critical translational input and help us accelerate the advancement of our programs as we work to make cures possible for more patients.

Magenta is uniquely positioned as the only company taking a comprehensive approach to unlocking the power of stem cell transplant medicine, said Dr. Pinkas. I am very excited to be part of the team that is building and expanding upon this foundational and innovative work to bring potentially transformative therapies to patients.

Dr. Pinkas is a seasoned scientist with deep expertise in leading drug development programs, specifically ADCs. Prior to joining Magenta, he was Head of Translational Research & Development at Immunogen, where he led nonclinical and translational research and development-related activities for all programs in discovery through late-stage clinical development. Dr. Pinkas earned his undergraduate degree in biology from Johns Hopkins University and his doctorate in Molecular and Cellular Biology from the University of Massachusetts at Amherst.

As Magenta has evolved into a clinical-stage company with a robust pipeline of preclinical assets, Mike has made tremendous contributions. He has built a world-class research organization and advanced our pipeline. Mike and I agreed that now, with a well-established Magenta research platform that is generating strong conditioning ADCs, validated targets, and discovery biology, it is the right time for Mike to explore other opportunities. We wish him well in his new adventure and will always be grateful for his scientific contributions, added Dr. Gardner.

I am very proud of Magentas rapid progress since our launch three years ago, and I am particularly proud of the cutting-edge scientific work that has come from our platform, said Dr. Cooke. I am confident that the scientific groundwork we have laid will help ensure that Magenta achieves its vision to transform the lives of many patients.

About Magenta TherapeuticsHeadquartered in Cambridge, Mass., Magenta Therapeutics is a clinical-stage biotechnology company developing novel medicines for patients with autoimmune diseases, blood cancers and genetic diseases. By creating a platform focused on critical areas of unmet need, Magenta Therapeutics is pioneering an integrated approach to allow more patients to receive one-time, curative therapies by making the process more effective, safer and easier.

Forward-Looking StatementThis press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Appoints Jan Pinkas as Senior Vice President, Head of Translational Sciences and Announces Transition of Chief Scientific Officer...

An Oncologist Asks When Its Time to Say Enough – The New York Times

Raza documents the failure of chemotherapy to help the great majority of patients with metastatic disease, and the immense cost and suffering involved. She castigates pharmaceutical companies (as have many others) for concentrating on drugs that often fail and at best achieve, on average, a few extra months of life. She quotes research that in the United States, over 14 years, 42.4 percent of the 9.5 million cancer cases had lost all of their life savings within two-plus years.

Raza also accuses research scientists and her fellow oncologists of unshakable hubris, convinced as we are that we possess the power to untangle the intricacies of as complex a disease as cancer. She dismisses much current research with the comment that it is pure arrogance to think the problem can be solved by a few molecular biologists; research, she says, should be based on studying humans, not mice. She goes on to say: Our lives are at stake. Our future is at stake.

Cancer is overwhelmingly a disease of old age, even though ads for cancer charities invariably show pictures of children and young women. It is worth noting that most of the patients whose stories Raza recounts are relatively young as well. She writes, An effective treatment for cancer can only be developed essentially after we understand how life works, how we age, since aging and cancer are two sides of the same coin. Fine words, but the reader can be forgiven for feeling that they smack of the same hubris afflicting those molecular biologists, toiling away in the lab with their mouse models.

So what is the answer? Raza suggests the first cancer cell that gives rise to a tumor is like a grain of sand that precipitates the collapse of a sand pile. Research, she says, should concentrate on finding these early changes, before an actual tumor develops. There is research going on along these lines, but Raza argues that its funding is insufficient compared with the resources being poured into new drug development.

A quantum leap is required, and this will involve genomics, transcriptomics, proteomics, metabolomics; indeed, panomics. It will also involve smart bras and special toilets real-life technologies in various stages of development, she assures us. I am in no position to know whether these technologies represent a paradigm shift in the treatment of cancer, or whether they are akin to the magical thinking that geoengineering will save us from the unfolding apocalypse of climate change, or to the gullibility that gave rise to the Theranos scandal.

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An Oncologist Asks When Its Time to Say Enough - The New York Times

CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies’ Respective Cell Therapy Programs in Oncology – SynBioBeta

CRISPR Therapeutics to receive non-exclusive access to certain KSQ IP for its allogeneic CAR-T programs

KSQ Therapeutics to receive non-exclusive access to certain CRISPR IP for its autologous cell therapies, including its existing eTILTM cell franchise

ZUG, Switzerland & CAMBRIDGE, Mass.(BUSINESS WIRE)CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development, today announced a license agreement whereby CRISPR Therapeutics will gain access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ will gain access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The financial terms of the agreement are not being disclosed.

We are thrilled to gain access to CRISPR Therapeutics foundational IP estate through this agreement, said David Meeker, M.D., Chief Executive Officer at KSQ Therapeutics. Our eTILTM programs involve editing gene targets in human TILs that were discovered at KSQ by applying our proprietary CRISPRomics approach to immune cells in multiple in vivo models. This agreement clears an important path for us to be able to bring these programs through development and commercialization, leveraging CRISPR Therapeutics proprietary editing technology.

The gene targets within the scope of the license agreement were identified using KSQs proprietary CRISPRomics drug discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery. These specific targets were uncovered in screens to identify genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology.

KSQ has built an industry-leading platform to screen for novel gene targets using its technology, and has identified a group of targets that could help unlock the full potential of adoptive cell therapy in oncology, said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. As a result of this license agreement, CRISPR Therapeutics will have the opportunity to bring these novel targets into our leading allogeneic CAR-T development platform to further strengthen our future programs in this important therapeutic area.

About KSQ Therapeutics

KSQ Therapeutics is using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics drug discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SECs website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies' Respective Cell Therapy Programs in Oncology - SynBioBeta

Accomplishments of Dr. Steven Rosenberg in Cancer Immunotherapy Inspire Young Researchers in Japan & XIV Fujio Cup Quiz on Stem Cells Is Won by…

TOKYO Utilizing the capability of ones own immune system to tackle cancer, an out-of-the box idea was the brain child of Dr. Steven Rosenberg almost three decades ago. His initiative which paved way for a new chapter in oncology, inspired many young scientists and clinicians in the NCRM NICHE 2019 held in Tokyo, Japan, as his acceptance speech in the Edogawa NICHE Prize ceremony was videocast.

NCRM NICHE, an active knowledge gaining academic event since 2006 in which young scholars from all over the world compete for the Fujio Cup Quiz (FCQ) in regenerative medicine is evolving to be an open innovation platform according to Dr. Shojiro Katoh, Chairperson of Edogawa Evolutionary Lab of Science (www.eels.tokyo), a co-host. He added that the FCQ motivated his team research on 16 different themes in Regenerative Medicine in various clinical specialties, among which two have completed clinical pilot studies with successful outcome viz., corneal endothelial regeneration and urethral stricture repair.

The XIV edition of FCQ contest that witnessed teams from Malaysia, Indonesia and India in the finals, was won by Reshma Romanas and Aayurshi Agrahari of Kasturba Medical College, India. Alumni of the FCQ are now eligible to nominate the awardees for Edogawa NICHE Prize which was established in 2018 to honour scientists or clinicians who develop novel solutions in healthcare, based on inter-disciplinary interactions. Dr. Steven A. Rosenberg, Chief of surgery, National Cancer Institute, NIH, USA is the recipient of the award in 2019. The award portrays such accomplished role models to the FCQ Elites according to the organizers who have instituted Joyce & James Till Travel Grant with a generous grant by Prof James Till, that supports travel of yesteryears FCQ Elites, who are now accomplished researchers in their own rights to meet and inspire the FCQ Elites of today, thus bringing together science and generations across nations.

NCRM NICHE is supported by a consortium with EELS as knowledge partners and JBM Inc., as industry partners based in Tokyo which has set up a hybrid cell culture cum biomaterials lab for taking forward the cell therapy and tissue engineering innovations to bed side, with future plans to propagate them globally through networking with like-minded academic and industry partners.

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Accomplishments of Dr. Steven Rosenberg in Cancer Immunotherapy Inspire Young Researchers in Japan & XIV Fujio Cup Quiz on Stem Cells Is Won by...

Tucson Tech: Ventana Medical Systems founder tells the rest of the story in new book – Arizona Daily Star

If youve been in Tucson long, you may have heard the story of how Ventana Medical Systems was founded here more than 30 years ago by a University of Arizona pathologist who invented an automated instrument to deliver fast diagnostics for cancer.

And you may have also heard what seemed to be the end of the story: How Ventana grew into a multi-billion-dollar public company and was acquired by Swiss drug giant Roche for $3.4 billion in 2008.

Now, company founder and UA professor emeritus Dr. Thomas Grogan has penned the rest of the story.

Grogans new book, Chasing the Invisible: A Doctors Quest to Abolish the Last Unseen Cancer Cell is a remarkable story, a loving memoir with elements of a spy thriller, a medical whodunit and a compelling business story.

It also has an intriguing cast of characters, including Grogans own mother, former Libyan strongman Moammar Gadhafi, an Inuit woman, intrepid scientists and Wall Street money men.

Grogan, 74, who retired from the UA about nine years ago and stepped down in 2017 from active duty at what is now Roche Tissue Diagnostics, said he may have learned as much about himself as he did about book publishing during the writing process.

Its been a great experience. The amazing part of writing a memoir is you relive what happened, Grogan said. And my story is one part personal, one part diagnostic medicine, one part technology, one part entrepreneurship and one part big pharma and Roche.

But getting the book published wasnt easy, said Grogan, who noted that his work was rejected by a dozen traditional publishers.

He finally reached a deal with Virginia-based Koehler Books to print the book under a co-publishing deal.

The initial critique from Koehlers editor was decidedly mixed.

The editor said, Okay, its interesting, its well-written but its a lousy memoir because you dont talk about yourself, Grogan said.

At one point in his draft, the editor demanded Grogan explain a passage noting that he had grown up living in dangerous places in dangerous times.

Finally I admitted to him, if I tell that part of the story I have to say something I was raised in the family to never talk about that my father was a CIA officer in the Middle East and Africa, Grogan said.

With his mothers blessing, Grogan detailed his familys life on the Mediterranean island of Cyprus in the early 1950s, when Cypriots rebelled against the last vestiges of British rule as Greece and Turkey began fighting for control.

Grogan recounted how his mother fearlessly drove the family car through an angry mob of Cypriots to deliver him and his brother to a Christmas pageant rehearsal at church.

Theres this whole episode where were attacked by a mob, and my mom says, Get down! And hits the accelerator, he said.

Grogans book starts with a scene about five years ago, when his 90-year-old mother asks him for advice after doctors discovered a cancerous tumor in her skull they believed may have been a recurrence of earlier breast cancer and suggested she consider hospice care.

She decided to get further tests and instead of metastatic breast cancer, the tumor was identified as a large B-cell lymphoma.

Well, I dont see giving up yet, she says. After all, they havent done a biopsy and we dont know the nature of the beast yet.

After testing with Ventana instruments and treatment with a Roche drug, Grogans mother recovered and remains disease-free today, Grogan said.

Grogan relates his inspiring meeting with a Libyan doctor who treated Gadhafis sister and later won funding for Ventana instruments from late dictator.

He also tells the story of how during a fishing trip to Alaska he met a native Inuit woman with cancer.

Grogan helped her understand the importance of testing to get the right treatment, and after her successful treatment, the need to keep the invisible disease from recurring using the analogy of how mosquitoes return every summer.

The book also describes Ventanas diagnostic technology in detail, but in terms most laymen can understand.

Grogans story of how he started Ventana seems to parallel his own life, overcoming obstacles, fighting the unseen, never giving up.

He relates how, after filing for a business license to start Ventana in 1985, a UA lawyer told him he could already be a felon because state law prohibited state employees from forming private businesses while on the state payroll.

That law was changed to accommodate Ventana and has led to the formation of countless faculty startups.

Grogan details how he was rejected by 35 investor groups before finding investors to bankroll Ventanas development, how the company went public and at one point losses had piled up to nearly $50 million, and how convincing the famed Cleveland Clinic to adopt the companys instruments helped the company break into the market.

He details how Roches advances were initially viewed with skepticism but what began as a hostile takeover put the company on a path to global acceptance as a standard of cancer diagnostics.

Despite his editors best efforts to get Grogan to talk about himself, he spends much of the book praising the efforts of colleagues, including his UA pathology department head, the late Dr. Jack Layton, for encouraging his work; key investor John Patience for his unwavering support; and his UA lab team and fellow pathologists for their hands-on work to develop and perfect the game-changing diagnostics.

Grogan does acknowledge he has one talent.

I had my own talents, but my success had to do with the ability to create alliances, Grogan, said, describing how he lured top talent with the promise of transforming medicine.

The reason to read this is, theres something to learn about global medicine, something to learn about how it is that this hidden world is really something thats actionable, he said.

Because its like the story I tell about the Inuit woman theres a strength you gain when you know what youre dealing with.

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Contact senior reporter David Wichner at dwichner@tucson.com or 573-4181. On Twitter: @dwichner.

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Tucson Tech: Ventana Medical Systems founder tells the rest of the story in new book - Arizona Daily Star

The Medicine Cabinet Of The Future: If You Can Imagine A Drug, This Company Can Make It – Forbes

Codexis CEO John Nicols has made his company a protein engineering powerhouse, and in doing so has become an important resource for the biopharma industry. If you can imagine a drug, chances are his company can help make it.

I have previously written how big pharma has been slow to innovate and adopt the latest synthetic biology tools, which could vastly speed the creation of new treatments and vaccines.

Im changing my mind.

Last week, on the heels of SynBioBeta 2019, Codexis held its annual Protein Engineering Forum in Palo Alto, California. The forum brought together leading scientists and engineers to share the latest in protein engineering, which is being revolutionized by the tools of synthetic biology. The forum aimed to see where science is taking proteins, and what proteins can offer the world.

Codexis CEO John Nicols opened the two-day forum by remarking on the extraordinary success protein engineering has had in bringing real-world applications to market faster and more often, and the hope that the meeting would represent another milestone in the progress of proteins.

It certainly seemed to be. Here are a few examples of how biopharma is making the jump to lightspeed:

Accelerating evolution to make drugs with biology: I recently wrote about Nobel Laureate Frances Arnolds pioneering principles for nudging nature to do what she does best: evolve. Merck has taken this to heart by using her directed evolution methods to go from chemistry-based to biology-based production of its diabetes drug, Januvia. The result: an efficient, economical, and environmentally friendly process for manufacturing a range of drugs.

Unleashing computers: To respond to a wider range of more complex diseases, companies like Amgen are making their R&D pipeline more flexible and higher volume. To do this, they are integrating the latest in computation and experimental methods. This promises to speed the development of protein therapeutics, which can replace abnormal or deficient proteins in diseases like arthritis, cardiovascular disease, and blood disorders.

Cascade reactions: The ability to engineer extremely effective enzymes the proteins that catalyze chemical transformations allows biologists to put many steps in a single pot, going from starting materials to finished product in one go. This makes the process more sustainable and more efficient; to paraphrase one participant, If its sustainable, its also cheaper. Researchers at biopharma giant GSK reported that most of their projects are going in this direction. GSKs commitment to the environment is reflected in giving projects an environmental score and cascade reactions are a green dream.

The DNA to build it all: Using the power of silicon to write the DNA needed for just about any biopharma application, CEO Emily Leproust described how her company helped researchers develop a rapid response tool to make antibodies to fight global viral threats like Zika and Ebola. The company also spun out Twist Biopharma with an emphasis on creating new antibody drugs for difficult-to-target diseases.

The protein revolution isnt limited to biopharma, either. Here are a few other areas highlighted at the forum where protein design is having big impacts:

Cannabinoids: Ive previously written about the benefits of brewing cannabinoids as you would beer, such as producing rare cannabinoids not easily purified from plants. Companies like Invizyne are going one step further by taking biology pathways out of cells and into cell-free systems. Invizyne is just one player in a field of heavy-hitters pursuing cannabinoids, including Amyris, Ginkgo Bioworks, and Intrexon.

Bioplastics: Theres a buzz about bioplastics. Protein engineers are evolving enzymes to break down plastic in the environment. Conversely, they can and have created bioplastics from biological sources, ones that are more biodegradable than their petrochemical cousins.

Biofuels: In the earliest days of synthetic biology, pioneers like Jay Keasling went after biofuels. As a commodity chemical, this was a very challenging first target: its a commodity chemical that must be produced on a tremendous scale, and compete economically with the petrochemical industry. (Thats why much of the industry retreated to higher-value chemicals from the top of the barrel.) Fast-forward to 2019: James Liao, Keasling, and other researchers have tools to radically change the way we ferment ethanol and other fossil fuel replacements, hinting at the new golden age of biofuels to come.

What does this mean for manufacturing?

The science and technology we use to make proteins are going to transform manufacturing, but how? Will manufacturing continue to require central manufacturing facilities with their high capital equipment costs? Or are markets going to benefit from faster, cheaper, and better ways of making chemicals and materials?

We think about this all the time, said one participant. We see a day when, instead of large, expensive manufacturing facilities, you will have your entire biomanufacturing platform on a skid. Add glucose and your enzyme cocktail, and it will produce your final product, maybe even in pill form. You could pick it up, put it on a plane, send it anywhere in the world that its needed.

Where do big data and AI fit in?

Industry has invested billions in R&D to bring products to the market. In doing so, it has accumulated enormous amounts of data. Much of that data is about failed attempts, such as drug candidates that fail in the late stages of clinical testing. Companies tend to share info about successes, but not about failures. So all that data remains locked in company databases, waiting for the day we can learn from it with computer algorithms and other sources of big data.

How can we encourage companies to share with the rest of the world? Can the government play a role in providing sharing incentives? If companies could find good ways to share that information, with the right incentives for everyone involved, it would open up a huge range of new possibilities for the industry. Maybe advanced encryption techniques could allow companies to share some data and conceal others.

What does it all mean for you and me?

Some of us are already benefiting from advances in protein science, such as the diabetes medicine Januvia I mentioned above, where the same product is now made with reduced pollution and waste. But for others, the revolution in protein engineering is shortening the time it takes to take a drug from bench to bedside.

At the Codexis forum, there was a confidence on the part of the biopharma participants that synthetic biology tools and technologies will soon be able to make just about any drug or vaccine you can think of. As one participant put it, When were asked, Do you have an enzyme that can make that?, the answer isnt yes or no. The answer is yes or not yet.

In the future, we can expect entirely new products. For example, an engineered probiotic is now available to prevent hangovers.

Perhaps the most important area is the one we can all relate to the most: human disease. Gjalt Huisman, Senior VP of Strategic Development at Codexis, has spent most of his career in biotherapeutics, and he shared an experience he had at a conference on phenylketonuria a rare but potentially devastating disease.

I was speaking at a conference with patients and their family members in the audience, as well as scientists, Huisman said. After our session, this older man came up to me and said, simply, My granddaughter has PKU, and we count on you. It was a revelation to me, and a reminder of the big job we have ahead of us in the real world.

With a little courage and the right tools, biopharma is poised to make me a believer.

Acknowledgment: Thank you to Kevin Costa for additional research and reporting in this post.

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The Medicine Cabinet Of The Future: If You Can Imagine A Drug, This Company Can Make It - Forbes

Past, Present and Future: How Non-Clear Cell Kidney Cancer Has Evolved – Curetoday.com

Those with non-clear cell kidney cancer have much to look forward to as oncologists understanding of the disease is evolving.

BY Kristie L. Kahl

At A Vision of Hope: A Kidney Cancer Educational Symposium, which was hosted by the Judy Nicholson Kidney Cancer Symposium and Penn Medicine Abramson Cancer Center, Narayan discussed the past, present and future of the treatment of non-clear cell kidney cancer.

Non-clear cell kidney cancer accounts for 15% to 20% of kidney cancers and consists of papillary type 1 and 2, chromophobe and unclassified disease, as well as several additional less common subtypes. However, with all of these types, oncologists understanding of treatment for non-clear cell kidney cancer has evolved over the last several years, Narayan explained.

Where, instead of viewing this as a single uniform entity, we began to appreciate that this actually represents a spectrum of different diseases, all with distinctive molecular and genetic clinical courses and variable responses to treatment, he said.

In the past, most initial trials evaluated agents only for clear cell kidney cancer which comprises 80% of patients with little consensus on how to treat non-clear cell disease.

Two trials evaluated systemic treatments for non-clear cell kidney cancer the ESPN and ASPEN trials, both comparing Sutent (sunitinib) with Afinitor (everolimus). Although both drugs appeared effective in patients with clear cell kidney cancer, outcomes were merely modest for those with non-clear cell disease.

Moving forward to present day, oncologists began to think about the disease differently. We've begun to change our thinking in terms of how we think about non-clear cell kidney cancer. Instead of acting as lumpers, we begin to think (of the disease) more as splitters and recognize that this is truly a heterogeneous disease, Narayan said.

With this, oncologists began to think about their improved understanding of the molecular underpinnings of each subtype of the disease, and defined characteristics based on chromosomal alteration, tumor metabolism and more, he added.

As part of the Cancer Genome Atlas, a collaborative project to create comprehensive maps of the key genomic changes in different cancer types, oncologists were able to define pathways among each subtype.

There are areas of overlap with clear cell kidney cancer, but also some very important and unique differences with each of the subtypes of non-clear cell, Narayan explained. So as we start to think about how to use this information to improve the treatments for patients with non-clear cell disease, were really starting to have a change in the treatment paradigm. Instead of simply extrapolating treatments from clear cell kidney cancer, and testing them in non-clear cell patients, it would be ideal to have biology-driven clinical trials that assess specific treatments for specific subtypes.

He added that there are challenges with this ideology, however, because it is difficult to accrue patients in a trial for uncommon kidney cancers, but the benefit of having uniform biology to test the rationale for new treatment strategies could help.

Currently, researchers have evaluated Sutent, Cabometyx (cabozatinib), savolitinib (an experimental drug) and Xalkori (crizotinib) in patients with papillary renal cell carcinoma types 1 and 2. More recently, however, Sutent and Cabometyx have become the only two horses in the race, Narayan said.

In the future, he is hopeful that oncologists understanding of overlap and the differences seen with conventional clear cell kidney cancer will continue to evolve.

In thinking about the treatment of non-clear cell kidney cancer, especially as we move to the future, it's clear that our understanding of this disease is evolving, Narayan concluded. Our clinical trials will hopefully evaluate treatment strategies to target unique biology.

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Past, Present and Future: How Non-Clear Cell Kidney Cancer Has Evolved - Curetoday.com

Study focuses on repair and reversal of damage caused by Huntington’s disease – UCLA Newsroom

UCLA research identifies a potential strategy that may lead to treatment for the disorder

UCLA

The image shows astrocytes (in purple) with mutant huntingtin protein inclusions (green). Some of these were located within astrocytes as shown in the expanded image on the right.

A new study examining the role that star-shaped brain cells called astrocytes play in Huntingtons disease has identified a potential strategy that may halt the disease and repair some of the damage it causes.

Astrocytes interact with and support neurons, or nerve cells, and other brain cells. Although astrocytes outnumber neurons, little is known about how they interact with synapses, the junctions between neurons that enable them to communicate and convey messages to each other.

The study, led by UCLA researchers and published in the journal Science Translational Medicine, found that Huntingtons disease damages astrocytes at the early stages of the disease, which contributes to the neuropsychiatric symptoms that develop as the disease progresses.

Huntingtons is caused by a mutation in the huntingtin gene. People with Huntingtons experience depression, irritability and other neurological and behavioral problems. They may also have difficulty processing information and controlling their bodys movements.

Its likely that we will not understand brain diseases without also understanding what happens to the cells that actually form the brain, including astrocytes, said Baljit Khakh, the studys lead investigator and a professor of physiology and neurobiology at the David Geffen School of Medicine at UCLA.

Khakh led a team that previouslypioneereda method that enables scientists to look inside thebrainsof mice to observe astrocytes influence over nerve-cell communication in real time. The scientists are able to see how interactions between synapses and astrocytes change over time, and as a result of neurological diseases.

For the recent study, researchers observed the progression of Huntingtons disease in samples from the brains of deceased humans, and in living mice that carry the gene mutation. They found that by suppressing the mutation in astrocytes, they were able to stop the disease progression in mice and repair some of the damage that can be seen when examining the cells closely.

We believe that if we are able to stop the progression of the disease in astrocytes and neurons, then we may be able to restore activity in the brain to what it was before the disease developed, Khakh said.

Study author Blanca Diaz-Castro, a former UCLA postdoctoral scholar, said that while its well-known that the mutation causes the cell death in neurons, this is the first study to identify how the mutation affects astrocytes.

We believe the findings will lead to further studies on astrocytes in brain diseases, she said.

The study also established a database that can now be used for future studies of astrocytes in many neurodegenerative diseases. Khakh said the findings add to a growing body of evidence that suggests impaired astrocytes play a role in many neurological diseases, such as Huntingtons, ALS, multiple sclerosis and Alzheimers.

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Study focuses on repair and reversal of damage caused by Huntington's disease - UCLA Newsroom