Healthcare analytics, AI solutions for biological big data, providing an AI platform for the biotech, life sciences, medical and pharmaceutical industries, as well as for related technological approaches, i.e., curation and text analysis with machine learning and other activities related to AI applications to these industries.
The list includes a few of the area’s largest firms — including Alnylam Pharmaceuticals (Nasdaq: ALNY) and Amag Pharmaceuticals (Nasdaq: AMAG) — and mostly consists of those with little or no revenue. In fact, three of the companies had no revenue in 2015 at all.
At the top of the list is Radius Health (Nasdaq: RDUS), a company which is planning to submit its application for its first-ever drug in coming days, which grew from just 25 employees as of Dec. 31, 2014 to 73 employees a year later (the Waltham-based company has more than 100 employees today). Most of them (48) are in research and development, while 27 are engaged in administration, business development and finance.
“Since our initial IPO in 2014, we have grown exponentially as we evolve from a development stage biopharmaceutical company to a commercial organization,” CEO Robert Ward said in an email. ”We have established a robust and influential presence in Massachusetts, and are looking forward to continuing the momentum with the planned submission of our New Drug Application to the U.S. Food and Drug Administration at the end of this month for our investigational drug abaloparatide for the reduction of fracture risk in postmenopausal osteoporosis.”
The second slot on the list is Tesaro (Nasdaq: TSRO), also in Waltham, which employed 286 people as of the end of last year.
All data is according to the latest federal filings from the companies as compiled by Bloomberg. Click through the attached slideshow to see what other companies made the list.
Philip Hemme, Interviewing Anker Lundemose in Zurich @ 9th Annual Sachs conference for European Biotech CEOs
Reporter: Aviva Lev-Ari, PhD, RN
Philip Hemme, Co-Founder & CEO of Labiotech.eu
Anker Lundemose, CEO of Mission Therapeutics, from just outside of Cambridge (UK). Founded in 2011, Mission Therapeutics, a private drug discovery company which aims to commercialize highly expert research towards the treatment of cancer and other diseases via the ubiquitin cell signaling pathway.
9th Annual ELSCEO Forum & Exhibition, is being held on 15th-16th March 2016 at the Hilton Zurich Airport Hotel, Switzerland. This event will be highly transactional, bringing together an exciting cross-section of venture-funded and small-cap companies with leading investors, pharmas, and scientific thought leaders. We expect around350 delegates and 80 presenting companies. Event’s networking will be powered by online One-2-One meeting system and dedicated meeting facilities to make the event more transactional and productive.
Other related articles published on this Open Access Online Scientific Journal include the following:
14th ANNUAL BIOTECH IN EUROPE FORUM For Global Partnering & Investment 30th September – 1st October 2014 • Congress Center Basel
SACHS Associates, London http://www.sachsforum.com/zurich14/index.html
The late Cambridge Mayor Alfred Vellucci welcomed Life Sciences Labs to Cambridge, MA – June 1976
Reporter: Aviva Lev-Ari, PhD, RN
How Cambridge became the Life Sciences Capital
Worth watching is the video below, which captures the initial Cambridge City Council hearing on recombinant DNA research from June 1976. The first speaker is the late Cambridge mayor Alfred Vellucci.
Vellucci hoped to pass a two-year moratorium on gene splicing in Cambridge. Instead, the council passed a three-month moratorium, and created a board of nine Cambridge citizens — including a nun and a nurse — to explore whether the work should be allowed, and if so, what safeguards would be necessary. A few days after the board was created, the pro and con tables showed up at the Kendall Square marketplace.
At the time, says Phillip Sharp, an MIT professor, Cambridge felt like a manufacturing town that had seen better days. He recalls being surrounded by candy, textile, and leather factories. Sharp hosted the citizens review committee at MIT, explaining what the research scientists there planned to do. “I think we built a relationship,” he says.
By early 1977, the citizens committee had proposed a framework to ensure that any DNA-related experiments were done under fairly stringent safety controls, and Cambridge became the first city in the world to regulate research using genetic material.
1. Peter Fenton
Benchmark
GOT A PAYDAY FROM: Twitter, New Relic and Zendesk.
2. Fred Wilson
Union Square Ventures
GOT A PAYDAY FROM: Twitter, Tumblr and Etsy.
3. Chris Sacca
Lowercase Capital
GOT A PAYDAY FROM: Twitter and Instagram.
4. Josh Kopelman
First Round Capital
GOT A PAYDAY FROM: LinkedIn and OnDeck Capital.
5. Jim Goetz
Sequoia Capital
GOT A PAYDAY FROM: WhatsApp, Palo Alto Networks and Barracuda Networks.
6. Danny Rimer
Index Ventures
GOT A PAYDAY FROM: King Digital Entertainment, Etsy and Net-a-Porter.
7. Steve Anderson
Baseline Ventures
GOT A PAYDAY FROM: Instagram and ExactTarget.
8. Bill Gurley
Benchmark
GOT A PAYDAY FROM: GrubHub, OpenTable and Zillow.
9. Neil Shen
Sequoia Capital (China)
GOT A PAYDAY FROM: JD.com, Alibaba and Qihoo 360.
10. Scott Sandell
New Enterprise Associates
GOT A PAYDAY FROM: Workday, Tableau Software and Spreadtrum Communications.
11. Jim Breyer
Breyer Associates
GOT A PAYDAY FROM: Facebook, Etsy and Legendary Entertainment.
12. Peter Thiel
Founders Fund
GOT A PAYDAY FROM: Facebook, Yammer and Powerset.
13. Sir Michael Moritz
Sequoia Capital
GOT A PAYDAY FROM: LinkedIn and Zappos.
14. Mike Maples, Jr.
Floodgate
GOT A PAYDAY FROM: Twitter, Twitch.tv and Demandforce.
15. Marc Andreessen
Andreessen Horowitz
GOT A PAYDAY FROM: Skype (a deal that was led by his co-founder, Ben Horowitz) and Kno.
16. Aydin Senkut
Felicis Ventures
GOT A PAYDAY FROM: Meraki, Fitbit and Shopify.
17. Jenny Lee
GGV Capital
GOT A PAYDAY FROM: YY.com, Pactera Technology International and 21Vianet.
18. Roelof Botha
Sequoia Capital
GOT A PAYDAY FROM: Instagram and Square.
19. Brad Feld
Foundry Group
GOT A PAYDAY FROM: Zynga, Fitbit, MakerBot and Rally Software.
20. Rebecca Lynn
Canvas Ventures
GOT A PAYDAY FROM: Lending Club, FutureAdvisor and Check.
Maxim Group analysts recommended 6 Israeli biotech firms, with price targets up to 400% higher than market value.
“We recently visited Israel and met with more than 40 companies from stem cell treatments to innovative specialty startups and established diagnostics firms; we returned from our visit impressed and inspired,” wrote analysts at the American investment banking firm Maxim Group about the Israeli biomed industry.
The Maxim analysts have made “buy” recommendations for six Israeli biomed companies: Brainstorm, BioLine, Galmed, Intec Pharma, and Pluristem. The target price set by Maxim for their shares is 100-400% higher than their market value.
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Meanwhile, Teva also received a “buy” from Maxim, at a price 32% higher than market value. The report considers the prospect of 40 public and private companies.
“We saw a medical renaissance in Israel,” wrote Maxim analysts, Jason Kolbert and Jason McCarthy. They warned of a value gap between companies traded in Israel and similar companies in the US. “We are expecting the gap to close through dual-listing of the companies as they near commercialization. At those stages we will see global joint ventures, capital funding, and a greater presence in the US market.”
Maxim drew emphasis on several Israeli companies involved in “paradigm-changing technologies”, including private firms BioSight, PolyPid, Anima Biotech, Quark, and Izun and public companies Bonus, CollPlant, Kadimastem, VBL, Brainsway, Alcobra, and Compugen.
“The biotech industry has developed uniquely, with innovation centers around the globe. Israel is one of them,” wrote the analysts, noting the country has a high ratio of PhDs per capita. They said “the small companies are becoming the source for innovation while their large counterparts provide their expertise in production, regulation, and commercialization.”
They believe Israel has increased its importance to the global biotech field, estimating the number of Israeli companies in the sector to have passed 600, with revenue of $2 billion.
They further wrote, “The Israeli government is involved in a variety of efforts to encourage global companies to expand their direct involvement in research & development or production facilities in Israel.”
Published by Globes [online], Israel business news – www.globes-online.com – on March 10, 2016
SHARK TANK FOR WOMEN Entrepreneurs in LIFE SCIENCES
Reporter: Aviva Lev-Ari, PhD, RN
Next week Women in Bio (WIB), JLABs, and Johnson & Johnson Innovation are co-hosting The LobsterPot. This will be a “Shark Tank” style event eight female executives from startups raising funds will pitch to five senior-level women at investment firms.
Here’s some additional info on the event:
Date: March 16th, 2016
Time: 6:00 pm – 9:00 pm (ET)
6:00 pm – 6:30 pm Registration & Networking
6:30 pm – 8:00 pm Company Pitches & Investor Feedback
In premises of finance, a unicorn is a startup company having valuation at or more than $1 billion. The Wall Street Journal and Dow Jones VentureSource has compiled a list of 146 venture-backed private unicorn companies. Interestingly, it also includes ten companies in healthcare industry. It is exciting to observe that many biotech companies have made it to the list, which is another assurance of the global renaissances in biotech industry and hence securing the faith of the investors.
In this post, I would like to introduce you to these companies as it is worth knowing how they did it. Hope you will find it interesting!
1. Stemcentrx Inc. : Cancer Stem Cells Killer Biotech ‘unicorn’
Valuation: $5bn
Total equity funding: $250M
Specialties: Solid Tumor Cancer Stem Cell Discovery & Targeted Therapeutics
Location: South San Francisco, USA
Founded: 2008
Investors: Artis Ventures, Founders Fund (A Peter Thiel Company)
CEO: Brian Slingerland (co-founder)
Mission
Stemcentrx mission is to develop therapies that cure and significantly improve survival for cancer patients by pioneering new approaches to eliminate cancer stem cells, which initiate and perpetuate tumors.
Why a unicorn company?
Advancing from a stealth startup to this valuation, the biotech company showed new approach towards cancer research and drug development with already five investigational drugs in human clinical trialsto their credit. The three out of the five molecules have demonstrated clinical responses in patientswith small cell lung cancer, triple-negative breast cancer, ovarian cancer, and peritoneal cancer. In the pipeline they have additional novel targets for other major cancer types.
2. Moderna- modern future of messenger RNA therapeutics?
Investors: Alexion Pharmaceuticals, AstraZeneca, Flagship Ventures, Invus Group, RA Capital Management, Viking Global Investors, Wellington Management
CEO: Stéphane Bancel
Mission
This Company is enabling messenger RNA science to develop a new generation of transformative medicines for patients with their Core “Expression” Platform: messenger RNA Therapeutics (a trademark platform).
Why a unicorn company?
Moderna has already started the first-in-human clinical trials of its inaugural candidate early this year and proposed to move 5 more into the clinic trials by the end of 2016. Comprehensively, this company has developed more than 200 patent applications with more than 10,000 claims which diversely ranges from novel nucleotide chemistries to specific drug compositions. With load of cash and significant partnerships with other giants such as–AstraZeneca, Merck and Alexion, this company have many clinical ambitions for coming years.
3. Intarcia Therapeutics- changing the picture of Type 2 Diabetes
Valuation: $1.8bn
Total equity funding: $598M
Specialties: proprietary subcutaneous delivery system
Location: Boston, MA, United States
Founded: 1995
Investors: Adams Street Partners, Alta Partners, Bank Jullus Baer, Baupost Group, Bay City Capital, BIM, Bio21 Venture Capital, Brentwood Venture Capital, BSI Industries, CBG Compagnie Bancaire, Charter Venture Capital, Delphi Ventures, Farallon Capital Management, Fidelity Investments, Foresite Capital Management, Franklin Resources, Garfin, GC&H Investments, GGV Capital, Glynn Capital Management, Greenspring Associates, InterWest Partners, LGT Bank, Vienna, Lombard Odier Darier Hentsch & Cie, New Enterprise Associates, New Leaf Venture Partners, Omega Fund Management UK, Ontario Teachers’ Pension Plan Board, Pacific Growth Equities, PAC-LINK Management, Picet, Quilvest Ventures, RA Capital Management, Raiffeisen Centrobank, Trefoil, Triazis Trust, Venrock Associates
CEO: Kurt Graves
Mission
Intarcia is committed to develop disruptively innovative and life-changing therapies that merge medicine with technology and transform therapeutic categories.
Why a unicorn company?
Intarcia have a proprietary subcutaneous delivery system platform technology and a lead product named ITC 650, which is poised to transform the global diabetes market, as it can be a potential once-a-year type 2 diabetes treatment. ITC 650 is currently in phase 3 development and the company expects to file for regulatory approval in the U.S. in 2016.
4. Oxford Nanopore: the Next-Gen Sequencing master
Valuation: $1.5bn
Total equity funding: $344M
Specialties: Immuno-oncology
Location: Oxford, U.K
Founded: 2005
Investors: Illumina, Invesco Perpetual, Invesco Private Capital, IP Group, Lansdowne Capital, Odey Asset Management LLP, Redmile Group, Top Technology Ventures
CEO: Gordon Sanghera (co-founder)
Mission
Oxford Nanopore is developing and selling a new generation of nanopore-based electronic systemsfor analysis of single molecules, which can includes DNA, RNA and proteins.
Why a unicorn company?
This company wants to get your DNA online, which is almost reality with their next-gen gene sequencer known as MinION which can plugs into USB ports. Other than that they have developed PromethION and GridION systems for the high-throughput/high sample number real-time biological analyses.According to company, their products can be ubiquitously used in scientific research, personalized medicine, crop science, security and defense and environmental applications.
5. CureVac: oldest player of messenger RNA (mRNA) therapeutics
Valuation: $1.2bn
Total equity funding: $265M
Specialties: messenger RNA (mRNA) therapeutics
Location: Tubingen, Germany
Founded: 2000
Investors: Dievini Hopp BioTech Holding, Bill & Melinda Gates Foundation
CEO: Ingmar Hoerr (co-founder)
Mission
CureVac stands for creating diverse therapeutic possibilities based on revolutionary vaccination with the utilization of natural, chemically unmodified mRNA.
Why a unicorn company?
CureVac is the first company in messenger RNA (mRNA) therapeutics area with two candidates in phase III and diverse portfolio in the different stages of clinical trials. This company has partnerships with various multinational corporations and organizations which also includes Bill & Melinda Gates Foundation. Most recently, CureVac announces that its mRNA-based, first-in-man prophylactic vaccine against rabies “MERAN” has been approved by the WHO as suffix for mRNA drug substances.
6. Adaptive Biotechnologies: the immuno-oncology master
Valuation: $1bn
Total equity funding: $425M
Specialties: immuno-oncology
Location: Seattle, USA
Founded: 2009
Investors: Alexandria Real Estate Equities, BD Biosciences, Casdin Capital, Celgene, Illumina, Lab Corp. of America Holdings, Matrix Capital Management, Rock Springs Capital, Senator Investment Group, Tiger Management, Viking Global Investors
CEO: Chad Robins (co-founder)
Mission
Developing revolutionary immune-based discoveries which combines high-throughput sequencing and state-of-art bioinformatics to profile T-cell and B-cell receptors which can be utilized to transform the diagnosis and treatment of immune-mediated diseases including cancer, autoimmune disorders, and infectious disease.
Why a unicorn company?
This company has ground-breaking immunosequencing technology which can help in translating into clinical diagnostics and therapeutic development. Also it is validating a novel clinical diagnostic as a reliable measure of “immunocompetence” to predict and monitor response to immune-modulating cancer therapies. The immunoSEQ®Assayis another major product to characterize immune reconstitution post-cord blood transplantation. Adaptive also has a sensitive and definitive method to diagnose cutaneous T-cell lymphoma. A new proprietary pairSEQ™ Assay enables high-throughput pairing of T cell receptor (TCR) α and β sequences and thus provides ultimate chances of the identification of highly promising TCRs for adoptive T-cell therapy.
The MassBio Annual Meeting focuses on the most critical challenges facing the Massachusetts life sciences industry. The meeting program is designed by a steering committee of industry leaders and the agenda encompasses keynote presentations, panel discussions, interactive working sessions and extensive networking opportunities.
The MassBio Annual Meeting also includes the Innovative Leadership Award Luncheon, which honors an industry leader for his or her contribution to moving the life sciences industry forward.
AGENDA
Day 1
UPDATED on 3/21/2016
John Maraganore to be Honored with Innovative Leadership Award
MassBio is pleased to announce that John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals will be awarded the 2015 Henri A. Termeer Innovative Leadership Award at MassBio’s 2016 Annual Meeting.
John will accept his award and make keynote remarks at the Awards Luncheon on March 31.
Read a few recent news stories on John Maraganore and Alnylam:
Just Announced: Kate Marshall to Deliver Opening Keynote!
MassBio is thrilled to announce that Kate Marshall, high school honors student, athlete and CF advocate, will share her experiences as a patient and how the biotech industry has changed her life.
John Orloff, Executive Vice President, Head of Research & Development and Chief Scientific Officer, Baxalta
Helen Sabzevari, CSO & Co-Founder, Compass Therapeutics
Biosimilars
Sige Gutman, Chair, Life Sciences Patent Practice, Proskauer Rose (Moderator)
Jim Roach, CMO, Momenta
Alex Waldron, Vice President of Global Commercial Operations, Epirus Biopharmaceuticals
Science in Space
Dr. George Church, Professor of Genetics, Harvard Medical School & Director, PersonalGenomes.org
Kris Kimel, Co-Founder & Chairman of the Board, Space Tango
Mike Roberts, Deputy Chief Scientist, Center for the Advancement of Science in Space
Dr. Ting Wu, Professor of Genetics, Harvard Medical School, Director, Consortium for Space Genetics and Director, Personal Genetics Education (pgEd.org) Project(Moderator)
Tony Coles to give closing keynote at Annual Meeting!
Dr. Coles is a founding investor and the chairman and chief executive officer of Yumanity Therapeutics, a Cambridge, MA-based biotechnology company focused on transforming drug discovery for neurodegenerative diseases caused by protein misfolding such as Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS).
Dr. Coles also serves as chairman and chief executive officer of TRATE Enterprises, LLC, a privately held company.
Previously, Dr. Coles was chairman and chief executive officer of Onyx Pharmaceuticals, Inc., which was acquired by Amgen in late 2013. Under his leadership, Onyx introduced two new innovative cancer medicines to patients and established the company’s international presence outside of the U.S. Prior to joining Onyx in 2008, he was president, chief executive officer and a member of the board of directors of NPS Pharmaceuticals, Inc. Before joining NPS Pharmaceuticals in 2005, Dr. Coles was senior vice president of commercial operations at Vertex Pharmaceuticals Inc., and earlier, held a number of executive positions at Bristol-Myers Squibb Company. Additionally, from 1992 until 1996, Dr. Coles held a number of positions of increasing responsibility at Merck & Co., Inc.
Educated at Johns Hopkins University, he earned an M.D. from Duke University and a master’s degree in public health from Harvard University. He completed his cardiology and internal medicine training at Massachusetts General Hospital and was a research fellow at Harvard Medical School.
Dr. Coles currently serves on the board of CRISPR Therapeutics, a biopharmaceutical company focused on developing transformative gene-based medicines for patients with serious diseases He also serves as a member of the board of directors of McKesson Corporation (NYSE: MCK), is vice chair of the board of trustees for Johns Hopkins University and is a member of the board of trustees for Johns Hopkins Medicine. In March 2015, Dr. Coles was named to the National Institutes of Health (NIH) working group tasked with charting the course for President Obama’s Precision Medicine Initiative, now part of the PMI Cohort Program Advisory Panel. Dr. Coles also serves as a member of the council for the Smithsonian’s National Museum of African American History and Culture in Washington, D.C.; a member of the board of trustees for The Metropolitan Museum of Art in New York City; and a member of the Council on Foreign Relations, an independent, nonpartisan membership organization, think tank, and publisher.
Governor Charlie Baker to make opening remarks at the Annual Meeting!
Since taking office in January, 2015, Governor Charlie Baker has been making Massachusetts a great place to live, work, start a business and raise a family while delivering a customer service oriented state government that is as thrifty, creative and hard working as the people of Massachusetts.Governor Baker called on an expert, bipartisan team of Republicans, Democrats and Independents to lead his cabinet.
Together, they are fulfilling his commitment to building stronger and safer communities for our children and families; keeping our roads and bridges safe and reliable; protecting our natural resources; and ensuring our schools and students are successful and safe.
From resolve in the face of unprecedented snow and freezing temperatures, to working to fix the MBTA, the Department of Children and Families, the Health Connector and Registry of Motor Vehicles, to balancing budgets despite billions in deficits all without raising taxes – the Baker Political Administration is making state government truly work for the people of Massachusetts.
Avvinity will have exclusive rights in oncology to use Alphamer therapeutic platform, invented by a Nobel Laureate and developed by Centauri: A Case of a Joint Venture Model, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)
Avvinity will have exclusive rights in oncology to use Alphamer therapeutic platform, invented by a Nobel Laureate and developed by Centauri: A Case of a Joint Venture Model
Reporter: Aviva Lev-Ari, PhD, RN
Horizon Discovery Group plc Enters Immuno-Oncology Therapeutic Development and Forms Joint Venture with Centauri Therapeutics Limited
·Newly formed company Avvinity Therapeutics will be jointly managed by Horizon, via its Research Biotech business, and Centauri
·Joint venture creates a differentiated new player in the rapidly growing immuno-oncology market, currently valued at £25 billion
·Avvinity will have exclusive rights in oncology to use Alphamer therapeutic platform,invented by a Nobel Laureate and developed by Centauri
·Horizon to invest up to £5.3 million, with an initial outlay of £2.5 million, in a joint venture to identify novel immuno-oncology therapeutics
·Horizon will contribute its gene editing technology platform and oncology expertise and will also benefit from service revenues from the joint venture
·Formation of the joint venture represents a strategic move by Horizon to capture the upside potential of its IP, platform technologies and capabilities in immuno-oncology, synthetic lethality and cell therapy based therapeutics
Cambridge, UK, 2 March 2016: Horizon Discovery Group plc (LSE: HZD) (“Horizon” or “the Company”), the leading international gene editing company, announces today that it has formed an immuno-oncology joint venture, Avvinity Therapeutics (“Avvinity”), with Centauri Therapeutics Limited (“Centauri”), a UK-based biotechnology company focused on the discovery and development of novel molecules targeting life-threatening infectious diseases. This transaction represents part of Horizon’s previously outlined strategy to invest up to £10 million, further leveraged by its IP, technology platforms and know-how, to identify the next generation of molecular and cellular cancer therapeutics.
Avvinity will combine Horizon’s gene editing, immunology, oncology and drug discovery capabilities with Centauri’s Alphamer technology to provide a powerful and proprietary platform to discover and develop novel immuno-oncology therapeutics, for both solid tumours and leukaemias. Avvinity will be targeting an immuno-oncology market currently worth £25 billion per year and expected to grow to approximately £50 billion per year by 2020.(1)
Under the terms of this agreement, Horizon will out-license certain background intellectual property relating to its translational genomics and drug discovery platforms, and will invest up to £5.3 million over two tranches with the first tranche of £2.5m committed, and the second to be committed at Horizon’s discretion pending the progress of three development programs. Centauri will license background IP and expertise on its Alphamer technology to Avvinity, which will have exclusivity for the field of oncology for an initial three year period and can be extended via the issue of further equity concurrently with the raise of new investment.
Avvinity will be managed jointly by Horizon and Centauri, and based on the investment of IP, technology and the first tranche of funding; Horizon will own 33% of Avvinity’s equity, representing 50% of the most-preferred class of voting shares. Upon completion of the 2nd tranche of funding Horizon will own 49.99% of Avvinity’s equity, representing 50% of the most-preferred class of voting shares. The joint venture will be managed within Horizon as part of the Company’s Research Biotech business (formerly Horizon’s Leveraged business unit).
Neither Horizon nor Centauri will be obliged to provide further funding to Avvinity, though both retain pre-emption rights and may elect to participate in future funding rounds. Subject to achieving key development milestones, Avvinity plans to raise significant new external investment to take its innovative drugs into clinical trials, at which time the value of Horizon’s stake in the business would be highly-material.
Dr. Darrin M. Disley, Chief Executive Officer, President Research Biotech of Horizon Discovery Group plc, said: “By combining Horizon’s deep understanding of the genetic basis of cancer alongside its gene editing, drug discovery and emerging immuno-oncology toolbox, with Centauri’s unique Alphamer technology and knowledge of its use, we have created an exciting new company to spearhead Horizon’s move into targeted therapeutic development. We are confident this joint venture will break new ground in the development of immunotherapies, and bring significant value creation to Horizon shareholders.
“The establishment of Avvinity is in line with our hybrid Research Biotech strategy to not only work with partners but also take advantage of the therapeutic upside potential of the most exciting new areas of personalised and genomic medicine in a risk-managed way.”
Dr. Mike Westby, Chief Executive Officer of Centauri Therapeutics Limited, commented: “Alphamers are an entirely novel way to target disease and represent an exciting new approach for recruitment of host immunity. At Centauri we have invested to build the Alphamer platform and assembled the drug discovery expertise necessary to exploit the platform in infectious diseases. Through this joint venture with Horizon, we look forward to applying our combined know-how and capabilities to develop Alphamers as important new immuno-oncology medicines, particularly for cancer indications that have proven intractable to date.”
Alphamer technology is based on chemically synthesized molecules that redirect naturally occurring antibodies in the human immune system to selected pathogens or cancer cells. One end of a molecule binds a cell-surface target on a pathogen or cancer cell using an aptamer, while the other end presents specific epitopes that attach to the circulating antibodies. The result of this redirection is cell death and subsequent recruitment of the T-Cell mediated pathways to clear the body of the pathogen or cancer cell.
Alphamers promise key advantages over conventional antibody and antibody-drug conjugate molecules in immuno-oncology applications, including the ability to target cancers driven by both wild type (“normal”) gene overexpression as well as mutant (”abnormal”) gene overexpression, and by exhibiting a short half-life in the body yielding reduced toxicity and systemic side-effects. Considerable investment has been made in the Alphamer technology over the past four years and its ability to engage the immune system to destroy bacteria has been confirmed.(2)
Platform technologies based around alternative immuno-oncology approaches have secured high valuations once early positive results in clinical trials have been achieved: Amgen’s 2012 acquisition of Micromet valued Micromet at $1.16b, and in 2013, Spirogen was acquired by AstraZeneca for up to $440m.
A conference call for analysts, investors and media will take place at 09:30 GMT today hosted by Darrin Disley, Chief Executive Officer and President, Research Biotech, and Richard Vellacott, Chief Financial Officer, who will run through a presentation followed by a Q&A session. The presentation will be made available shortly before the call at: https://www.horizondiscovery.com/about-us/investor-relations/corporate-videos-and-presentations.
The dial-in numbers for the conference call are:
UK: 08006940257
Standard International: +44 (0) 1452 555566
Conference call ID: 62637628
A replay of the call is available approximately four hours after the call concludes. For those unable to attend it live, or who would like to listen to it again, call +44 (0)1452550000 and quote the conference call ID: 62637628.
Horizon is a leading international gene-editing company that supplies products, services and research programmes that enable genomics research and the development of personalised and genomic medicines. Horizon has a diverse and global customer base of over 1,400 unique organisations across more than 50 countries, including major pharmaceutical, biotechnology and diagnostic companies as well as leading academic research centres. The Group supplies its products and services into multiple markets, estimated to total in excess of £29 billion in 2015.
Horizon’s core capabilities are built around its proprietary translational genomics platform, a highly precise and flexible suite of gene editing tools (rAAV, ZFN and CRISPR) able to alter almost any gene sequence in human or mammalian cell-lines.
Horizon offers over 23,000 catalogue products, almost all of which are based on the application of gene editing to generate in vitro and in-vivo models that accurately model the disease-causing genetic anomalies found in diseases like cancer. These ‘patients-in-a-test-tube’ are being used by customers to: understand the genetic drivers of disease; identify targets of therapeutic intervention that can moderate or correct these genetic drivers; develop novel medicines and companion diagnostic tests that result in the right patient getting the right medicine.
Horizon also provides custom in vitro and in vivo disease model generation services, biopharmaceutical manufacturing cell lines and generation services, quantitative molecular reference standards and contract research and custom screening services.
In addition, Horizon through its Research Biotech business deploys the Company’s intellectual property, gene-editing platform, products, services and know-how in cancer research, drug discovery and immunology to develop its immuno-oncology, synthetic lethality and cell therapy platforms which aim to deliver novel drug treatments into the pharmaceutical pipeline.
Horizon is headquartered in Cambridge, UK, and is listed on the London Stock Exchange’s AIM market under the ticker “HZD”. For further information please visit: www.horizondiscovery.com.
Centauri Therapeutics is a UK-based biotechnology company focused on the discovery and development of novel molecules targeting life threatening diseases.
Centauri Therapeutics has established a core R&D facility at Discovery Park in Sandwich, Kent, with an experienced team of industry scientists focused on discovery, optimisation and development of novel Alphamers targeting acute hospital acquired infections. The company is currently focussed on the development of Alphamers against anti-microbial resistant (AMR) pathogens, which pose an increasing threat to human health.
Centauri Therapeutics’ Executive team is led by Mike Westby, Chief Executive Officer (previously Pfizer, Roche) and Stuart Lawson, Chief Financial Officer (CEO of the private investment group Animatrix Capital LLP, and previously KPMG). Clive Dix is Non-Executive Director and Chairman of the Board (previously Convergence Pharmaceuticals, PowderMed, PowderJect, Glaxo Wellcome).
The ubiquitin system produces a protein that greatly restricts the development of cancerous tumors.
A new study by researchers at the Technion-Israel Institute of Technology could hold one key to control cancer cell growth and development. In a paper published in the April 9, 2015 edition of CELL, the team reports on the discovery of two cancer-suppressing proteins.
Distinguished Professor Aaron Ciechanover. Photographer: Dan Porges
The research was conducted in the laboratory of Distinguished Professor Aaron Ciechanover, of the Technion Rappaport Faculty of Medicine. The team was led by research associate Dr. Yelena Kravtsova-Ivantsiv and , included additional research students and colleagues, as well as physicians from the Rambam, Carmel and Hadassah Medical Centers, who are studying tumors and their treatment.
The heretofore-undiscovered proteins were found during ongoing research on the ubiquitin system, an important and vital pathway in the life of the cell, which is responsible for the degradation of defective proteins that could damage the cell if not removed. The ubiquitin system tags these proteins and sends them for destruction in the cellular complex known as the proteasome. The system also removes functional and healthy proteins that are not needed anymore, thereby regulating the processes that these proteins control.
Usually, the proteins that reach the proteasome are completely broken down, but there are some exceptions, and the current line of research examined p105, a long precursor of a key regulator in the cell called NF-κB. It turns out that p105 can be broken down completely in certain cases following its tagging by ubiquitin, but in other cases it is only cut and shortened and becomes a protein called p50.
NF-κB has been identified as a link between inflammation and cancer. The hypothesis of the connection between inflammatory processes and cancer was first suggested in 1863 by German pathologist Rudolph Virchow, and has been confirmed over the years in a long series of studies. Ever since the discovery (nearly 30 years ago) of NF-κB, numerous articles have been published linking it to malignant transformation. It is involved in tumors of various organs (prostate, breast, lung, head and neck, large intestine, brain, etc.) in several parallel ways, including: inhibition of apoptosis (programmed cell death) normally eliminates transformed cells; acceleration of uncontrolled division of cancer cells; formation of new blood vessels (angiogenesis), which are vital to tumor growth; and increased resistance of cancerous cells to irradiation and chemotherapy.
The dramatic effect of these proteins on cancer growth: above the two tumors in the foreground (the control group) are tumors that express high levels of the proteins
As noted, the precursor p105 is “handled” by the ubiquitin system in one of two parallel and equally prevalent ways. It is either destroyed completely, or shortened and transformed to p50. The current research deciphers the decision-making mechanism that determines which process will be applied to the protein: when a ubiquitin system component called KPC1 is involved in the process and attaches ubiquitin to p105, the protein is shortened to become p50. When ubiquitination is mediated by another component of the system (and without KPC1), p105 is degraded.
The ubiquitin molecule within all living cells
The decision between these two options has significant implications on the cell, as the presence of high levels of KPC1 (which generates p50) and p50 (the product of the process) – with the accompanying disruption of the normal ratios between the processes – suppresses the malignant growth and apparently protects the healthy tissue. The current research was conducted on models of human tumors grown in mice, as well as on samples of human tumors, and a strong connection was discovered between the suppression of malignancy and the level of the two proteins, clearly indicating that the increased presence of KPC1 and/or p50 in the tissue can protect it from cancerous tumors.
Professor Ciechanover, who is also the president of the Israel Cancer Society, notes that many more years are required “to establish the research and gain a solid understanding of the mechanisms behind the suppression of the tumors. The development of a drug based on this discovery is a possibility, although not a certainty, and the road to such a drug is long and far from simple.”
Professor Ciechanover won the Nobel Prize in Chemistry in 2004 (jointly with Professors Avram Hershko – also from the Technion – and Irwin Rose, of the Fox Chase Cancer Center) for the discovery of the ubiquitin system. The current line of research is a continuation of that discovery.
President Reuven Rivlin and Indian Prime Minister Narendra Modi, March 29, 2015 (photo credit: Courtesty Tomer Reichmann)
President Reuven Rivlin and Indian Prime Minister Narendra Modi, March 29, 2015 (photo credit: Courtesty Tomer Reichmann)
Days after the Technion announced that a team led by Nobel Prize laureate Professor Aaron Ciechanover had discovered how proteins could be used to suppress cancer and control tumor growth and development, the institute revealed that it had entered into an exclusive agreement with India’s Sun Pharmaceuticals — the world’s fifth-largest specialty generic pharmaceutical company and India’s top pharmaceutical company.
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Under the agreement, researchers from the Technion and Sun will conduct studies on how high concentrations of two proteins can protect tissue from tumors. A study published in the medical journal Cell this week discussed how the proteins can suppress malignancies.
Along with Ciechanover, the research team included Dr. Gila Maor and Professor Ofer Binah. In a statement, Ciechanover said that the research held a great deal of promise of an effective drug for treating cancer, “although this is not a certainty, and the road to such a drug is long and far from simple.”
The deal with Sun is just one of several R&D ventures between Israel and India, on both the business and government levels. So far, the two countries have signed seven bilateral economic and R&D agreements, including one that fosters joint projects on space travel and satellite development.
The joint program aims at attracting additional, world-class support from institutions and individuals who are dedicated to eradicating cancer through focused and efficient research.
Two of the world’s preeminent academic and research institutions — New York University’s Langone Medical Center and Haifa’sTechnion-Israel Institute of Technology — have made a “groundbreaking step forward to advance global collaboration in the fight against cancer formally.
On Wednesday night, they announced a $9 million gift from philanthropists Laura and Isaac Perlmutter that will fund two major, joint research endeavors with potentially far-reaching impact in advancing cancer research. The joint program aims at attracting additional, world-class support from institutions and individuals who are dedicated to eradicating cancer through focused and efficient research, they said in a joint statement.
The first $3 million of the grant will finance six cancer-focused research projects to be conducted by teams spearheaded by co-investigators from both NYU and the Technion. The remaining $6 million will be used to establish a state-of-the-art research facility on Technion’s campus that will support these and other research projects and focus mainly on the emerging field of cancer metabolomics.
“NYU Langone and the Technion have a shared, longstanding commitment to advancing cancer research,” said Dr. Dafna Bar-Sagi, senior vice president and vice dean for science at the New York hospital, chief science officer at NYU School of Medicine and a principal architect of the NYU Langone-Technion partnership. “We are now at a great moment in our institutions’ illustrious histories, a point from which we can jointly leverage the talent and creativity of our researchers toward accelerating breakthroughs. The foresight and the generosity of the Perlmutters, particularly at this time of financial challenge in funding for basic research, will have tremendous impact.”
“Bringing together the unique expertise of researchers from both NYU and the Technion will hopefully enable us to overcome some of the most difficult challenges in treating cancer patients,” said Technion Prof. Aaron Ciechanover, the 2004 Nobel Prize Laureate in Chemistry and Distinguished Research Professor and head of the David and Janet Polak Cancer and Vascular Biology Research Center at the Technion Faculty of Medicine.
Renowned cancer biologist Dr. Benjamin Neel, an expert in the field of cell signal transduction, recently joined the Langone faculty as director of the Perlmutter Cancer Center, and Dr. Eyal Gottlieb, a world leader in cancer metabolism, has been recruited to lead the new research facility at the Technion funded by the Perlmutter gift. Neel will work closely with Ciechanover to lead the collaborative cancer research effort between the two institutions, they said.
In addition, Neel will oversee at NYU the building of world-class translational programs in immunotherapy, cancer genetics/targeted therapies and epigenetics, imaging, as well as expanded programs in clinical care, community outreach and supportive oncology.
The innovative method, developed at the Technion, identifies persons at risk for developing stomach cancer and for detecting tumors at an earlier stage. The prestigious journal Gut, which published the research, notes that the detection method is quick, simple, inexpensive and non-invasive.
Innovative gastric cancer-detection technology
Innovative gastric cancer-detection technology developed by the Technion can be used for the early detection of stomach cancer and for identifying persons at risk for developing the disease. The new detection method, based on breath analysis, has significant advantages over the existing detection technology: Gut reports that the new method is quick, simple, inexpensive and non-invasive.
Gastric cancer is one of the most lethal forms of cancer and in most cases, its diagnosis involves an endoscopy (the insertion of a tube into the esophagus, requiring that the patient fast and receive an intravenous sedative). Treatment is aggressive chemotherapy, radiation and the full or partial removal of the stomach. The disease develops in a series of well-defined steps, but there’s currently no effective, reliable, and non-invasive screening test for picking up these changes early on. Thus, many people succumb to stomach cancer only because it was not diagnosed in time.
The new technology, developed by Prof. Hossam Haick of the Wolfson Faculty of Chemical Engineering, can be used to detect premalignant lesions at the earliest stage, when healthy cells start becoming cancerous.
The research, published in Gut as part of the doctoral thesis of Mr. Haitham Amal, was conducted in conjunction with a Latvian research group headed by Prof. Marcis Leja, based on the largest population sample ever in a trial of this type. 484 people participated in the trial, 99 of whom had already been diagnosed with stomach cancer. All the participants were tested for Helicobacter pylori, a bacterium known to increase the risk for stomach cancer, and two breath samples were taken from each person.
The first sample from each participant was analyzed using the GCMS technique, which measures volatile organic substances in exhaled breath. The researchers noted that GCMS technology cannot be used to detect stomach cancer because the testing is very expensive and requires lengthy processing times and considerable expertise to operate the equipment.
The second breath sample was tested using nanoarray analysis, the unique technology developed by Prof. Haick, combined with a pattern recognition algorithm.
The findings:
Based on the concentrations of 8 specific substances (out of 130) in the oral cavity, the new technology can distinguish between three groups: gastric cancer patients, persons who have precancerous stomach lesions, and healthy individuals.
The new technology accurately distinguishes between the various pre-malignant stages.
The new technology can be used to identify persons at risk for developing gastric cancer.
The diagnosis is accurate, regardless of other factors such as age, sex, smoking habits, alcohol consumption and the use of anti-oxidant drugs.
In short, the nano-array analysis method developed by Prof. Haick is accurate, sensitive technology that provides a simple and inexpensive alternative to existing tests (such as GCMS). This new technology offers early, effective detection of persons at risk for developing stomach cancer, without unnecessary invasive tests (endoscopy). In order to assess the accuracy and effectiveness of the new, a wide-scale clinical trial is currently under way in Europe, with thousands of participants who have cancerous or pre-cancerous tumors.
About Prof. Hossam Haick
Prof. Hossam Haick, who joined the senior staff at the Technion Wolfson Faculty of Chemical Engineering in 2006, has been working since that year on the development of innovative, non-invasive technology for detecting cancer and other diseases. This technology is based on an “electronic nose” – an apparatus capable of detecting illnesses by analyzing a patient’s exhaled breath.
Prof. Haick, a native of Nazareth, completed his Ph.D. studies at the Technion by the time he was 27 and went to the Weizmann Institute of Science in Rehovot and Caltech Institute of Technology in California. He returned to the Technion in 2006 and his research group was awarded one million euros in grants by the European Union, which was very impressed by his research into artificial olfactory systems. Today he heads a consortium that includes Siemens and several universities, research institutes and companies in Germany, Austria, Finland, Ireland, Latvia and Israel. Since joining the senior faculty in the Chemical Engineering Department in 2006, Prof. Haick has won dozens of awards, grants and international honors. These include the Marie Curie Excellence Grant, European Research Council (ERC) grant and the Bill & Melinda Gates Award. Prof. Haick was nominated to MIT’s list of the 35 leading young scientists worldwide, received the Knight of the Order of Academic Palms, from the French Government and won the Hershel Rich Technion Innovation Award (twice), as well as the Tenne Prize for Excellence in the Science of Nanotechnology. He has also been recognized for his outstanding teaching skills and is the recipient of the Yanai Prize for Academic Excellence. In 2014, at the initiative of the president of the Technion, Prof. Haick headed an MOOC (Massive Open Online Course) in nanotechnology and nano-sensors that had an enrollment of 42,000.
This month is Breast Cancer Awareness Month in Israel and around the world. Innovative technology developed at the Technion Faculty of Biomedical Engineering will enable the prediction of cancer metastasis after the appearance of breast cancer. The technology, whose efficacy has been proven in preliminary laboratory-trials, is entering into advanced testing using cells from patients undergoing surgery.
In contrast to benign cells (right), metastatic cells (left) penetrate into the gel and disappear inside it, thanks to their unique characteristics
Assistant Professor Daphne Weihs recently achieved a research breakthrough: the unique technology that she developed – a biomechanical method for early detection of metastatic cancer – was approved by the Ethics Committee. This means that the technology that was found to be effective in tests on cell lines will advance to trials with tumor cells collected directly after surgery, in cooperation with Rambam Healthcare Campus.
According to Assistant Professor Weihs, the practical concept is that “during or immediately after a biopsy or surgery on a malignant tumor, the system will enable the medical team to quantitatively evaluate the likelihood of the presence or development of tumor metastases in other organs, and to propose which organ or organs are involved. Such knowledge will make it possible to act at a very early stage to identify and curb these metastases and, moreover, to prevent the primary tumor from metastasizing further.
Cancer is a general name for a wide family of diseases – more than 200 – whose common denominator is that the cell division rate becomes uncontrolled and the cells become immortal. In other words, the cancer mechanism disrupts the normal cell division process and converts it into “wild” and rapid division. Since the cells do not age and do not die, the original, primary tumor expands, invades and takes over more and more nearby tissue. In addition, apart from spreading to its immediate vicinity, a tumor that has become very aggressive “knows” how to send metastases to more distant tissues through the lymph and circulatory systems. Metastases (secondary tumors) are usually more dangerous than the primary tumor because it is difficult to identify them at their inception. When they are detected at an advanced stage, treating them medically is more complicated and the medical prognosis is typically not good.
“In fact, most cancer-related deaths are caused by metastases rather than by the primary tumor, and therefore vast resources are invested in developing methods for early detection of metastases,” explains Assistant Professor Daphne Weihs. “Early detection means timely and more effective treatment. The new approach that we are developing will enable early prediction of the likelihood of the formation of metastases and where in the body their development is probable. This prediction is based on identifying the biomechanics of the primary tumor cells, and does not require us to know the specific genetic makeup of the tumor.”
Diagnostic system developed by Technion professor is to pair with tiny smell-sensitive sensor that can go anywhere
By David Shamah February 3, 2015, 2:05 pm
A patient uses the NaNose breathalyzer (Photo credit: Courtesy Technion)
Writers
David Shamah
An innovative early disease detection system that uses the sense of smell is going mobile.
The NaNose breathalyzer technology developed by Professor Hossam Haick of the Technion will soon be installed in a mobile phone – to be called, appropriately, the SniffPhone. A tiny smell-sensitive sensor will be installed onto a phone add-on, and using specially designed software, the phone will be able to “smell” users’ breath to determine if they have cancer, among other serious diseases.
By identifying the special “odor” emitted by cancer cells, the NaNose system can detect the presence of tumors, both benign and malignant, more quickly, efficiently and cheaply than previously possible, said Haick.
“Current cancer diagnosis techniques are ineffective and impractical,” he said. NaNose technology, he said, “could facilitate faster therapeutic intervention, replacing expensive and time-consuming clinical follow-up that would eventually lead to the same intervention.”
According to research done by Haick’s team, the NaNose system has a 90 percent accuracy rate.
The smartphone device is just a vehicle to implement the NaNose technology that can be taken anywhere and used in any circumstances, including in rural areas of the developing world where bringing in sophisticated testing equipment is impossible.
The plan calls for a chip with NaNose technology to be installed in a device that is attached to a smartphone, and for an app to read the sensor data, analyzing it on the device or uploading it to the cloud for processing.
NaNose technology will be especially useful in battling lung cancer, said Haick. According to US government statistics, lung cancer kills more Americans annually than the next three most common cancers — colon, breast, and pancreatic — combined. The reason, doctors say, is because lung cancer is so difficult to detect. Currently, the only way to detect early-stage lung cancer is through an extensive process involving blood tests, biopsies, CT scans, ultrasound tests, and other procedures — and even then, detection is difficult.
“Mostly the patient arrives for diagnosis when the symptoms of the sickness have already begun to appear,” said Haick, describing the drawbacks in current detection protocols. “Months pass before a real analysis in completed. And the process requires complicated and expensive equipment such as CT and mammography imaging devices. Each machine costs millions of dollars, and ends up delivering rough, inaccurate results.”
Dr. Hossam Haick (Photo credit: Courtesy)
The NaNose-based system, on the other hand, doesn’t require anything more than a patient’s breathing into the device in order to come up with an initial diagnosis. Lung cancer tumors produce chemicals called volatile organic compounds (VOCs), which easily evaporate into the air and produce a discernible scent profile. Haick’s NaNose chip detects the unique “signature” of VOCs in exhaled breath. In four out of five cases, the device differentiated between benign and malignant lung lesions and even different cancer subtypes.
The project is being funded by the European Commission, which has given the consortium developing it a six million euro grant. The developers include universities and research institutes from Germany, Austria, Finland, Ireland and Latvia, as well as Irish cell biology research firm Cellix, with the NaNose system the centerpiece of the technology. That Israeli-developed component will be delivered by an Israeli start-up called NanoVation-GS, a spinoff of the Technion. Professor Haick serves as the start-up’s Chief Science Officer.
“The SniffPhone is a winning solution. It will be made tinier and cheaper than disease detection solutions currently, consume little power, and most importantly, it will enable immediate and early diagnosis that is both accurate and non-invasive,” said Haick. “Early diagnosis can save lives, particularly in life-threatening diseases such as cancer.”
Anyone who knows a person in the midst of chemotherapy is aware that anti-cancer drugs often take a very harsh toll on the body. This is one reason scientists have been trying to develop improved means of drug delivery for years. Now, a Technion research team discovered a way to improve drug delivery to tumors using Nanostructured Porous Silicon (PSi) particles (instead of an IV drip), a method that’s emerging as a promising new platform for drug delivery. In the future, PSi could be used in cancer treatments, potentially offering an alternative to traditional chemotherapy, which is notorious for its agonizing side effects.
The silicon “carriers” used in this study to deliver chemotherapy drugs behave differently in cancerous tumors than they do in healthy tissues. Therefore, the findings could help scientists to design nano-carriers that deliver drugs to tumors, instead of treating patients with traditional, intravenous chemotherapy. However, it would take years to develop and apply this new type of drug delivery method, which would potentially be taken orally.
So far, these nano-silicon “containers” have been studied in vitro – outside of a living organism – rather than in an environment that behaves more closely to that of a tumor in a cancer patient’s body. The Technion research team looked at what happens to PSi particles when they’re injected into the area around the tumor in mice. The significant differences in the area around a cancerous growth and regular healthy tissue have been widely described and studied; however, the effect on these porous silicon “containers,” or carriers, was unknown until now.
Prof. Ester Segal of the Technion – Israel Institute of Technology, who led this joint study with the Massachusetts Institute of Technology (MIT) and the Harvard Medical School, said the team has “shown for the first time that bio-materials in general, and Nanostructured Porous Silicon in particular, behave differently when they are injected (or implanted) at the tumor micro-environment.”
Revolutionizing cancer treatments
Silicon materials could revolutionize treatments in a way that no existing drug delivery does. Prof. Segal tells NoCamels that the silicon containers “could deliver drugs over a long period of time – weeks or even months”, something no existing chemotherapeutic delivery mechanism can do currently.
Cancer cells
The special properties of these porous nano-silicon carriers lie in their large surface area, which can ferry many or large drug molecules. Additionally, due to their biodegradability they’re able to break down into harmless silicic acid, which is expelled through urination. They are also biocompatible, so they do not stimulate any inflammation or clotting. Another benefit to these nano-silicon containers is their versatility. They can be ingested, injected or implanted, and they can be designed to carry a wide range of dosage sizes. In the process of their study, lab members also developed an approach to determining how biomaterials will react in settings more similar to their eventual clinical purpose – treating cancer, for example.
In a separate study, Tel Aviv University scientists recently founda strategy that would stop brain tumor cell proliferation with similar nano-particles. “It is a basic, elegant mechanism and much less toxic than chemotherapy,” TAU’s Prof. Dan Peer said in a statement.
These works underline the importance of such studies in successfully developing bio-delivery materials that will have therapeutic benefits in the near future.
“Nano-skeletons’ (in red) delivered to human tissue infected by prostate cancer. The infected cells are colored in blue (PIP) and green (cytoplasmic); it is possible to see how the ‘nano-skeletons’ reach them
Florida native Dr. Beth Schoen, is part of a team developing a novel platform for delivering anti-cancerous drugs directly to its mark as part of her postdoctoral research at the Technion
Beth Schoen, born in Hollywood Florida, came to the Technion to conduct her postdoctoral research at age 26. In her very limited spare time she plays soccer for the leading all women’s soccer team – Maccabi Hadera – and studies Hebrew. “The Hebrew thing is no simple matter,” she confesses, “but I’m willing to make the effort, because it’s clear to me that Israel is where I want to live.”
Dr. Beth Schoen completed her undergraduate degree at the University of Florida, and her doctorate at Michigan State University in chemical engineering. “My doctoral studies focused on synthetic organic chemistry, particularly on the development of polymers with unique thermodynamic attributes especially resistant to high temperatures. These types of materials are used in part for the production of jet engine parts, body armor and Nomex (used for making fire-resistant gloves and overalls). One of our tasks was to create soft sheets that were not brittle, to be worn to be both bulletproof and fire resistant. It was a theoretical study, but as part of the process I also produced some of these polymers and tested them.”
Dr. Schoen planned to come to the Technion as part of her doctoral studies, but, she adds, “It didn’t work out, so I started to check where I could best fit in here in my future studies.” She decided to join Prof. Marcelle Machluf’s laboratory, at the Faculty of Biotechnology and Food Engineering, “I was eager to move from chemistry to biology and pursue cancer research in particular. I was very glad for the tremendous opportunity that Marcelle gave me in taking me on – perhaps it was because of my experience in nanomaterials and polymers.”
Prof. Marcelle Machluf’s research team consists of 17 female and 3 males students, researchers and technicians working on two main projects: (1) the development of scaffolds to rehabilitate damaged heart-tissue, and (2) the development of new technology to deliver drug treatment to damaged (sick) tissue (specifically related to cancer therapy). In an interview with her she focused on the second project.
“The current treatment for cancer involves radiotherapy and chemotherapy usually administered through intravenous infusion. The cancer drugs available are extremely effective, yet the way they are put to use in present day treatment, they also cause damage to healthy tissues. These are very potent drugs – they are intended to kill cancer cells – and on their way they also end up killing healthy ones.”
“The greatest damage is caused to rapidly dividing cells, which are similar to cancer cells. Hair follicle cells, for example, are a type of rapidly dividing cells and they damage easily from these types of treatment, which explains the hair loss in patients undergoing chemotherapy. Other side-effects include nausea and hearing loss, sometimes even leading to deafness. The drug Cisplatin for example, is a type of chemo drug used to treat various types of lung and breast cancers; some of its side-effects include damage to renal and immune system functioning, putting patients at risk to infections and diseases.”
These impediments are what fuel Prof. Machluf’s drive to develop a new drug delivery platforms capable of delivering anti-cancer drugs directly to the tumor without damaging healthy tissues on its way. “This is the top priority of cancer treatment: to develop a ‘magic bullet’ that target cancer cells,” explains Prof. Machluf. “And our new platform may be the solution to this great challenge.”
The new platform is based on ‘depleting’ specific cells – mesenchymal stem cells – so that there is nothing left of them save for the membrane. This membrane, called a ghost cells can be down sized to nano-vesicles, termed nano-ghosts, which can be loaded with any drug and delivered by injection directly into the blood stream. The immune system falls for the trap and does not recognize the ‘intruder,’ instead it treats these cells as if they were naturally part of the system and sends them to the afflicted area. On the way to their target they do not release the drug they are carrying and therefore do not do any damage to healthy tissues. Only upon reaching the malignant tissue, which they know how to identify, do they break down and secrete their contents at the site of the tumor cells.
This original idea was tested in a long series of experiments, and the results are very impressive: these nano-ghosts are in fact tumor selective, no matter the type of tumor. They ‘dash’ straight to the malignant tissue without emitting their drug on the way and without damaging healthy cells. Moreover, this unique ‘parcel’ increases the effectiveness of the treatment by ten-fold. Animal studies have shown that the employment of nano-ghosts for anti-cancer drug delivery have led to an 80% delay of prostate cancer – an unprecedented rate.
Still, there is a lot of work ahead, as Prof. Machluf’s research team works on improving the mechanism of this novel new platform: some of them are focusing on compatibility with specific drugs while others, like Dr. Beth Schoen, are concentrating on improving the nano-ghosts “This platform must be very precise,” explains Schoen. “It must be able to endure travelling through the entire human body, and release its contents only inside the tumor.”
2012pharmaceutical
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