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Untangling Dementia – Scientists used a designer compound to prevent and reverse brain damage caused by tau in mice. Miller lab, Washington University, St. Louis

Reporter: Aviva Lev-Ari, PhD, RN

Designer compound may untangle damage leading to some dementias

NIH-funded preclinical study suggests a possible treatment for Alzheimer’s disease and other neurodegenerative disorders.

In a study of mice and monkeys, National Institutes of Health funded researchers showed that they could prevent and reverse some of the brain injury caused by the toxic form of a protein called tau. The results, published in Science Translational Medicine, suggest that the study of compounds, called tau antisense oligonucleotides, that are genetically engineered to block a cell’s assembly line production of tau, might be pursued as an effective treatment for a variety of disorders.

Cells throughout the body normally manufacture tau proteins. In several disorders, toxic forms of tau clump together inside dying brain cells and form neurofibrillary tangles, including Alzheimer’s disease, tau-associated frontotemporal dementia, chronic traumatic encephalopathy and progressive supranuclear palsy. Currently there are no effective treatments for combating toxic tau.

“This compound may literally help untangle the brain damage caused by tau,” said Timothy Miller, M.D., Ph.D., the David Clayson Professor of Neurology at Washington University, St. Louis, and the study’s senior author.

Antisense oligonucleotides are short sequences of DNA or RNA programmed to turn genes on or off. Led by Sarah L. DeVos, a graduate student in Dr. Miller’s lab, the researchers tested sequences designed to turn tau genes off in mice that are genetically engineered to produce abnormally high levels of a mutant form of the human protein. Tau clusters begin to appear in the brains of 6-month-old mice and accumulate with age. The mice develop neurologic problems and die earlier than control mice.

Injections of the compound into the fluid filled spaces of the mice brains prevented tau clustering in 6-9 month old mice and appeared to reverse clustering in older mice. The compound also caused older mice to live longer and have healthier brains than mice that received a placebo. In addition, the compound prevented the older mice from losing their ability to build nests.

SOURCE

https://www.nih.gov/news-events/news-releases/designer-compound-may-untangle-damage-leading-some-dementias

 

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy

Science Translational Medicine  25 Jan 2017:
Vol. 9, Issue 374,
DOI: 10.1126/scitranslmed.aag0481

Teva – Dr. Sol J. Barer Named Chairman of the Board, Appoints Dr. Yitzhak Peterburg as Interim Chief Executive Officer, Succeeding Erez Vigodman

Reporter: Aviva Lev-Ari, PhD, RN

 

February 06, 2017 4:53 p.m.
Teva Appoints Dr. Yitzhak Peterburg as Interim Chief Executive Officer, Succeeding Erez Vigodman

Dr. Sol J. Barer Named Chairman of the Board

JERUSALEM–(BUSINESS WIRE)–Feb. 6, 2017– Teva Pharmaceutical Industries Ltd. (NYSE and TASE:TEVA) today announced that Dr. Yitzhak Peterburg, who has served as Chairman of the Teva Board of Directors since January 2015, has been appointed Interim President and Chief Executive Officer, effective immediately. This follows the mutual agreement between the Board of Teva and Erez Vigodman that Mr. Vigodman is stepping down. Mr. Vigodman’s service on the Teva Board of Directors has also ended.

In accordance with the Israeli Companies Law, Dr. Yitzhak Peterburg has stepped down from his role as Chairman in order to serve as Interim Chief Executive Officer. Prior to rejoining Teva’s Board of Directors in 2012, Dr. Peterburg led the Company’s innovative R&D efforts as Teva’s Group Vice President, Global Branded Products, from October 2010 until October 2011, after serving on Teva’s Board of Directors from 2009 until July 2010. Previously, he served as President and CEO of Cellcom Israel Ltd. from 2003 to 2005, Director General of Clalit Health Services, the leading healthcare provider in Israel, from 1997 to 2002 and CEO of Soroka University Medical Center, Beer-Sheva, from 1995 to 1997.

The Board has elected Dr. Sol J. Barer, who has been a member of the Teva Board since January 2015, as Chairman. Dr. Barer brings deep knowledge of the global pharmaceutical industry. He was a founder of the biotechnology group at Celanese Corporation, later spun off as Celgene Corporation, where he served in top leadership roles from 1987 to 2011, including as Chairman and CEO from 2007 to 2010.

The Company’s Board of Directors is undertaking a search to identify a permanent Chief Executive Officer with the assistance of a search firm.

“I believe that now is the right time for me to step down,” said Mr. Vigodman. “It has been a privilege to lead Teva, and I am proud of all we have accomplished. I am confident that the Company’s future is bright.”

Dr. Yitzhak Peterburg said, “The Company is focusing on executing its strategic priorities to transform Teva, with immediate focus on realizing the cost synergies and strategic benefits of the Actavis Generics acquisition. I look forward to working with the entire Teva team to conduct a thorough review of the business to find additional opportunities to enhance value for shareholders. Teva has a deep bench of talented leaders and today’s announcement has no impact on our ability to execute going forward. With the strength of our generics pipeline, unique R&D capabilities and unparalleled footprint, coupled with our existing assets and growing pipeline in specialty medicines, I believe in Teva and the Company’s long-term growth prospects.”

Dr. Barer said, “We are grateful to Yitzhak for taking on the role of interim CEO. Teva’s Board of Directors, with its decades of collective pharmaceutical industry experience, will continue to play an active role in driving the Company’s strategy, and I look forward to working with the management team to execute on the value creation opportunities ahead. We intend to conduct a comprehensive search to identify the best person to lead the Company for years to come. On behalf of the Board, I want to thank Erez for his many contributions to Teva over the years and wish him well in the future.”


SOURCE

http://ir.tevapharm.com/phoenix.zhtml?c=73925&p=irol-newsArticle&ID=2243437

http://www.biopharmadive.com/news/teva-ceo-out-erez-vigodman-exit-successor/435574/

Independent Public Relations Professionals with extensive experience in the biotechnology/drug development sector – Please Contact Me

Reporter: Aviva Lev-Ari, PhD, RN

We are seeking an independent public relations professional with extensive experience in the biotechnology/drug development sector to assist on a freelance basis to

  • promote an on line resource in drug development and personalized medicine in the global oncology sector. 

Responsibilities include working with agencies such as PR Newswire to distribute press releases prepared by us in house, and managing our proprietary mailing list and our social media accounts. 

Please, provide a detailed resume with contact information to knowledgebase@newmedinc.com.

 

Institutional Capital Raised by Female Founders in 2016 – A Global Perspective vs the US Economy: Globally 1,272 in the United States 600

Reporter: Aviva Lev-Ari, PhD, RN

2016 REVIEW OF FEMALE FOUNDERS RAISING INSTITUTIONAL CAPITAL

– See more at:

http://femalefoundersfund.com/2016-review-of-female-founders-raising-institutional-capital/#sthash.Pcuj7rVB.dpuf

 

The Data Reflects Several Key Trends
Key Takeaways

Raising a Series A led by an institutional VC remains difficult, but female founders in NYC continued to be the most successful (compared to those in other cities) in 2016.
At 17%, the percentage of total A rounds led by female CEOs in 2016 represents the highest total percentage since Female Founders Fund started tracking the data in 2013.
In addition, New York saw a record number of Series B and C rounds led by female founders in 2016.
Female Founders Fund remains the most active institutional VC firm investing in early-stage female-led companies.
Funds that have traditionally been uninterested in e-commerce have renewed interest in the e-commerce sector following the Dollar Shave Club and Jet.com acquisitions by large strategic investors.

2016 Series A Rounds — NYC — Female CEO
Rockets of Awesome — $12.5 million — December — Rachel Blumenthal
Ellevest — $9.0 million — September — Sallie Krawcheck
CoheroHealth — $9.0 million — November — Melissa Manice
Away — $8.5 million — September — Steph Korey
Primary– $8.0 million — June — Galyn Bernard
goTenna — $7.5 million — March — Daniela Perdomo
LOLA — $7.0 million — December — Alex Friedman and Jordana Kier
Uncharted Play — $7.0 million — September — Jessica Matthews
Thrive Global — $7.0 million — August — Arianna Huffington
Everplans — $6.4 million — June — Abby Schneiderman
pymetrics — $6.1 million — February — Frida Polli
Sakara Life — $4.8 million — January — Whitney Tingle, Danielle DuBoise
Shoppable — $3.5 million — August — Heather Marie
MMLaFleur — Sarah LaFleur

2016 Series A Rounds — Bay Area — Female CEO

Cortexyme — $15.0 million — January — Casey Lynch
FOVE — $11.0 million — March — Yuka Kojima
Front — $10.0 million — May — Mathilde Collin
REBBL — $10.0 million — December — Sheryl O’Loughlin
Nima — $9.2 million — May — Shireen Yates
Rocksbox — $8.7 million — March — Meaghan Rose
LaunchDarkly — $8.7 million — December — Edith Harbaugh
Modsy — $8.0 million — February — Shanna Tellerman
ThirdLove — $8.0 million — February — Heidi Zak
Node — $7.5 million — June — Falon Fatemi
Shippo — $7.0 million — September — Laura Behrens Wu
Mobilize — $6.5 million — September — Sharon Savariego
Neurotrack — $6.5 million — January — Elli Kaplan
Sourcery — $5.0 million — September — Na’ama Moran
Luka — $4.4 million — April — Eugenia Kuyda
SupportPay — $4.1 million — December — Sheri Atwood
Zybooks — $4.0 million — February — Smita Bakshi
Schoola — $3.6 million — May — Stacey Boyd

– See more at:

http://femalefoundersfund.com/2016-review-of-female-founders-raising-institutional-capital/#sthash.Pcuj7rVB.dpuf

 

 

Series A Rounds in 2016

Our 2016 analysis began with an overall review of Series A rounds globally, nationally and regionally.

2017 Research Graph 1

Series A Rounds Raised Globally, Nationally and Regionally in 2016

Series A Rounds in 2016:

 

 

 

2016 Series A Rounds in 2016:

Globally: 1,272
United States: 600
Bay Area: 187
NYC: 84
Boston: 31
Los Angeles: 38
Seattle: 26
Austin: 7
Washington D.C.: 17

2015 Series A Rounds in 2015:

Globally: 1,164

United States: 664

Bay Area: 205

NYC: 96

Boston: 50

Los Angeles: 40

Seattle: 25

Austin: 22

Washington D.C.: 17

– See more at:

http://femalefoundersfund.com/2016-review-of-female-founders-raising-institutional-capital/#sthash.Pcuj7rVB.dpuf

The total number of Series A rounds in the U.S. decreased by 10% in 2016. Of the seven regions that we track in the U.S., Seattle is the only region that experienced an increase in the number of Series A raises in 2016, at 4%.

While overall Series A activity declined slightly in Los Angeles, there were two large Series A raises for female-led businesses — (i) HopSkipDrive, led by CEO Joanna McFarland, which raised $10.2 million in January 2016 from Upfront Ventures and FirstMark Capital; and (ii) Hutch, led by CEO Beatrice Fischel-Bock, which raised $5 million in July 2016 from Founders Fund. Los Angeles remains among the most female entrepreneur-friendly cities in the U.S.

 

VC’s investing in female-led companies in 2016.

Female Founders Fund remained the most active investor, participating in 3 of the 14 — or 21% — of all female-led A rounds in NYC. – See more at: http://femalefoundersfund.com/2016-review-of-female-founders-raising-institutional-capital/#sthash.Pcuj7rVB.dpuf

– See more at:

http://femalefoundersfund.com/2016-review-of-female-founders-raising-institutional-capital/#sthash.Pcuj7rVB.dpuf

On Investment Platforms for Private Funds and Investment Platforms for Private Placements – SEC Update

Reporter: Aviva Lev-Ari, PhD, RN

 

SEC Update

In the last few years, we have seen a number of important developments in the securities laws related to finders and broker-dealer registration requirements. Below we provide an overview of the broker-dealer registration requirement as it relates to finders who assist in matching issuers with investors or buyers and the latest developments in this area.

Overview

The distinction between being classified as a finder and a broker-dealer can have significant consequences. An unregistered broker-dealer may face sanctions from the Securities and Exchange Commission (SEC), and it may be unable to enforce payment for its services. In addition, transactions involving an unregistered broker-dealer may create a right of rescission in favor of the investors, allowing the investors the right to require the issuer to return the money invested. One example of the consequences of an unregistered broker-dealer occurred in the Ranieri Partners SEC enforcement action. In that action the SEC brought charges against a private-equity firm, its managing director, and a consultant because of the consultant’s failure to register as a broker-dealer. The SEC’s order found that the private equity firm paid transaction-based fees to a consultant, who was not registered as a broker-dealer, for soliciting investors for private fund investments.1

The federal securities laws do not specifically define the term “finder” or outline what finders can do. Instead, finders must avoid being deemed a broker or dealer under the federal securities laws unless they register as such with the SEC and the Financial Industry Regulatory Authority (FINRA). A broker is defined as “any person engaged in the business of effecting transactions in securities for the accounts of others.”2 A dealer is defined as a person that is “engaged in the business of buying and selling securities … for such person’s own account,” but excludes a person that buys and sells securities for its own account, but not as part of a regular business.3Because the broker definition is the one that finders have the most trouble with, this discussion is focused on what activities may cause a finder to fall within the definition of a broker required to register with the SEC and FINRA.

  • M&A Brokers
  • FINRA Guidance
  • Investment Platforms for Private Placements
  • Investment Platforms for Private Funds
  • Crowdfunding
  • Potential Regulatory Action
Conclusion

A determination of whether an intermediary is acting as a finder or an unregistered broker-dealer is a very fact-specific analysis and can often be very complex. Unfortunately for unwary entrepreneurs, company executives, and equity fund sponsors, frequently a third party assisting with capital-raising will be acting as a broker-dealer, not a finder, and therefore should not be engaged unless properly registered. It is likely that we will see further clarification or new rules from regulators in the future; regardless, it is important to always carefully consider the involvement of finders or broker-dealers in any capital-raising endeavor.

If you have any questions regarding the use of finders, or capital raising in general, please contact the Venable lawyer with whom you work, one of the authors of this article, or a member of our Corporate Finance and Securities Group.

SOURCE
https://www.venable.com/finders-and-unregistered-broker-dealers-12-04-2015/?utm_source=Mondaq&utm_medium=syndication&utm_campaign=View-Original

Top 50 Women in CRISPR : Women in CRISPR, Legal Status of Inventions and Declaration of the Heroes in CRISPR

Curator: Aviva Lev-Ari, PhD, RN

2.1.5.6

2.1.5.6   Top 50 Women in CRISPR : Women in CRISPR, Legal Status of Inventions and Declaration of the Heroes in CRISPR, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 2: CRISPR for Gene Editing and DNA Repair

Part 1: Top 50 Women in CRISPR : Women in CRISPR 

See List, below

SOURCE

Part 2: UPDATED – Status “Interference — Initial memorandum” – CRISPR/Cas9 – The Biotech Patent Fight of the Century: UC, Berkeley and Broad Institute @MIT

Reporter: Aviva Lev-Ari, PhD, RN

SOURCE

https://pharmaceuticalintelligence.com/2016/01/06/status-interference-initial-memorandum-crisprcas9-the-biotech-patent-fight-of-the-century/

Part 3: The Heroes of CRISPR

in CELL, December, 2015

Eric S. Lander1,2,3,*

1, Broad Institute of MIT and Harvard, 415 Main Street, Cambridge, MA 02142, USA

2Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA

3Department of Systems Biology, Harvard Medical School, Boston, MA 02115, USA

*Correspondence: lander@broadinstitute.org

Three years ago, scientists reported that CRISPR technology can enable precise and efficient genome editing in living eukaryotic cells. Since then, the method has taken the scientific community by storm, with thousands of labs using it for applications from biomedicine to agriculture. Yet, the preceding 20-year journey—the discovery of a strange microbial repeat sequence; its recognition as an adaptive immune system; its biological characterization; and its repurposing for genome engineering—remains little known. This Perspective aims to fill in this backstory—the history of ideas and the stories of pioneers—and draw lessons about the remarkable ecosystem underlying scientific discovery.

SOURCE

http://dx.doi.org/10.1016/j.cell.2015.12.041

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Sharma, C.M., Hoffmann, S., Darfeuille, F., Reignier, J., Findeiss, S., Sittka, A., Chabas, S., Reiche, K., Hackermu¨ ller, J., Reinhardt, R., et al. (2010). The primary transcriptome of the major human pathogen Helicobacter pylori. Nature 464, 250–255. Siksnys, V., Gasiunas, G., and Karvelis, T. (2012). RNA-directed DNA cleavage by the Cas9-crRNA complex from CRISPR3/Cas immune system of Streptococcus thermophilus. U.S. Provisional Patent Application 61/613,373, filed March 20, 2012; later published as US2015/0045546 (pending). Sontheimer, E., and Marraffini, L. (2008). Target DNA interference with crRNA. U.S. Provisional Patent Application 61/009,317, filed September 23, 2008; later published as US2010/0076057 (abandoned). Sorek, R., Kunin, V., and Hugenholtz, P. (2008). CRISPR–a widespread system that provides acquired resistance against phages in bacteria and archaea. Nat. Rev. Microbiol. 6, 181–186. Sternberg, S.H., and Doudna, J.A. (2015). Expanding the Biologist’s Toolkit with CRISPR-Cas9. Mol. Cell 58, 568–574. Travis, J. (2015). GENETIC ENGINEERING. Germline editing dominates DNA summit. Science 350, 1299–1300. Urnov, F.D., Miller, J.C., Lee, Y.-L., Beausejour, C.M., Rock, J.M., Augustus, S., Jamieson, A.C., Porteusm, M.H., Gregory, P.D., and Holmes, M.C. (2005). Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435, 646–651. van der Oost, J., Westra, E.R., Jackson, R.N., and Wiedenheft, B. (2014). Unravelling the structural and mechanistic basis of CRISPR-Cas systems. Nat. Rev. Microbiol. 12, 479–492. Wright, A.V., James, K., Nun˜ ez, J.K., and Doudna, J.A. (2016). Biology and applications of CRISPR systems: Harnessing nature’s toolbox for genome engineering. Cell 164, this issue, 29–44. Zetsche, B., Gootenberg, J.S., Abudayyeh, O.O., Slaymaker, I.M., Makarova, K.S., Essletzbichler, P., Volz, S.E., Joung, J., van der Oost, J., Regev, A., et al. (2015). Cpf1 Is aSingleRNA-Guided Endonuclease of a Class 2 CRISPR-Cas System. Cell. Zhang, F., Cong, Le, Lodato, S., Kosuri, S., Church, G.M., and Arlotta, P. (2011). Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat. Biotechnol. 29, 149–153. Zhang, F. (2012). Systems Methods and Compositions for Sequence Manipulation. U.S. Provisional Patent Application 61/736,527, filed December 12, 2012; later published as US008697359B1 (awarded)

Top 50 Women in CRISPR : Women in CRISPR

SOURCE
A B C D E F G H I J
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Women in CRISPR/Cas9 genome editing research – List Version 3
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First Name
Last Name Organisation Location Country Position Website
Twitter Handle
Field of Research
Research Interest
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Divaki Bhaya Stanford Univeristy Stanford, CA USA Professor https://dpb.carnegiescience.edu/labs/bhaya-lab
Evolution and Ecology – microbial diversity – Plant Biology
Research in my lab is driven by an interest in understanding how photosynthetic microorganisms perceive and evolve in response to environmental stressors, such as light, nutrients and viral attack.We work both with model organisms and with cyanobacteria in naturally occurring communities. Recently,we have started to develop synthetic biology-inspired approaches to use in cyanobacteria.
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Jill Banfield University of California Berkeley Berkeley, CA USA Professor http://nanogeoscience.berkeley.edu Evolution and Ecology – microbial diversity
The study system for this project is an aquifer adjacent to the Colorado River in Rifle, Colorado, USA.Research addresses knowledge gaps related to the roles of subsurface microbial communities in biogeochemical cycling. Given the link between the carbon cycle and global climate change, a particular interest in this work is the impact of microorganisms on carbon compounds buried in the terrestrial subsurface, both through respiration and carbon fixation.
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Denis Bauer
Commonwealth Scientific and Industrial Research Organisation (CSIRO)
Sydney Australia Head of laboratory http://people.csiro.au/B/D/Denis-Bauer.aspx @allPowerde Computational biology – Technology development
Dr. Denis Bauer is the team leader of the transformational bioinformatics team in CSIRO’s ehealth program. Her expertise is in high throughput genomic data analysis, computational genome engineering, as well as Spark/Hadoop and high-performance compute system.
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Pilar Blancafort Harry Perkins Institute for Medical Research Perth Australia Associate Professor Cancer biology – Technology Development
The Blancafort laboratory focuses on the development of novel approaches to target cancers that are currently refractory to treatment and associated to poor outcome, such as triple negative breast cancers and ovarian cancers. At present, there are no targeted approaches to combat these tumors with chemotherapy and radiation the only treatment options. The laboratory generates novel functionalised molecules able to specifically target these tumors with minimal toxicity to normal cells. Our emphasis is in advanced stage metastatic tumors, which quasi invariably develop resistance. Ultimately we wish to revert the behavior of metastatic cells by sensitizing these treatment resistant tumors to chemotherapy regimes.
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Alexa Burger University of Zurich Zurich Switzerland
Senior postdoctoral fellow
http://www.imls.uzh.ch/en/research/mosimann/labmembers.html @aburger2009 Zebrafish – Technology development
CRISPR application in Zebrafish (ribonucleic complex and increase mutation efficiency)
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Emmanuelle Charpentier Max Plank Institute Berlin Germany Professor http://www.mpiib-berlin.mpg.de/research/regulation_in_infection_biology Host-pathogens interaction
Our research relates to the field of Molecular Infection Biology. We are overall interested in understanding the molecular mechanisms governing physiology-, virulence- and infection-associated processes in Gram-positive bacterial pathogens. We use a combination of genetic, genomic, molecular, biochemical, physiological and cell infection approaches to study mechanisms of gene expression at the transcriptional and post-transcriptional level in horizontal gene transfer, adaptation to stress, physiology or virulence. In particular, we do research on CRISPR, the adaptive immune system that protects bacteria against invading genetic elements; the small regulatory RNAs that interfere with bacterial pathogenicity; protein quality-control that regulates bacterial adaptation, physiology and virulence; and the mechanisms of bacterial recognition by immune cells.
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Sylvia Comporesi Kings College London London UK Lecturer https://silviacamporesiresearch.org/about/ @silviacomporesi Bioethics
I am a bioethicist with an interdisciplinary background in medical biotechnologies, ethics and philosophy. I am a tenured Lecturer (the UK equivalent to Assistant Professor) in Bioethics & Society in the Department of Global Health & Social Medicine (formerly, Social Science, Health & Medicine) at King’s College London, where I direct the Master’s in Bioethics & Society.
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Elena Conti Max Plank Institute Martinsried Germany
Group leader and Director
http://www.biochem.mpg.de/4877968/Research Structural Biology – RNA biology
Our group has a long-standing interest in RNA metabolism, with a particular focus on the molecular mechanisms of eukaryotic RNA transport and degradation.
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Jennifer Doudna University of California Berkeley Berkeley, CA USA Professor http://rna.berkeley.edu/index.html @doudna_lab RNA biology – Adaptive immunity
Exploring molecular mechanisms of RNA-mediated gene regulation
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Caixia Gao Chinese Academy of Science Beijing China Professor http://enpcce.genetics.cas.cn/PN/CXG/ACXG/ Plant biology (Wheat) – Technology development
The main research goal of our laboratory is to develop high-throughput transgene technologies for common wheat (Triticumaestivum L.) and maize (Zea mays) and other major crops to satisfy the needs of crop improvement and gene discovery.
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Carine Giovanangeli Museum National d’Histoire Naturelle Paris France Director of Research http://biophysique.mnhn.fr/site/Modifications+génomiques+et+réponses+cellulaires DNA repair mechanisms – Technology development
Nowadays, we are mainly focusing on novel artificial DNA binding domains, the TALE repeats (transcription-activator like effector) and CRISPR/Cas9 system. We use the CRISPR/Cas or TALE as nucleases (TALEN) to study DNA repair in mammalian cells as well as DNA probes to study genome dynamics (see Repeated DNA sequences and chromatin).
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Natalia Gomez-Ospina Stanford Univeristy Stanford, CA USA Clinical Instructor https://med.stanford.edu/profiles/natalia-gomez-ospina?tab=bio Stem cell biology – Clinical therapy
Dr. Gomez-Ospina was born and raised in Medellin, Colombia. She began her undergraduate studies in petroleum engineering at the Universidad Nacional de Colombia before moving to Colorado. She double majored at the University of Colorado Boulder, completing her bachelor’s degree in Molecular Cellular and Developmental Biology as well as Biochemistry. She graduated summa cum laude and wrote an honors thesis entitled “Role of the quiescent center in the regeneration of the root cap in Zea Mays.” She then completed her combined MD, PhD at Stanford Medical School, where her PhD work focused on understanding the novel functions of voltage-gated calcium channels. Her PhD thesis, “The calcium channel CACNA1C gene: multiple proteins, diverse functions,” was published in Cell. After completion of her dual degrees, she did her preliminary year in internal medicine at Santa Barbara Cottage hospital before starting residency in Dermatology at Johns Hopkins Hospital. She completed residency in Medical Genetics at Stanford Hospital and clinics. She is currently doing her post-doctoral research with Dr. Matthew Porteus in Pediatric Stem Cell transplantation, where she is developing a genome editing strategy in stem cells as a curative therapy for metabolic diseases. In addition to her research, Dr. Gomez-Ospina is a clinical instructor in Medical Genetics. For her clinical practice she sees patients with suspected genetic disorders, and is also in charge of the enzyme replacement service for lysosomal storage disorders at Lucile Packard Children’s hospital. She has been the lead author in research studies in The New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics.
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Asma Hatoum-Aslan The University of Alabama Tuscaloosa, AL USA Assistant Professor http://bsc.ua.edu/asma-hatoum-aslan/ @crisprcas10 Host-pathogens interaction
Bacterial infectious diseases are a major cause of mortality worldwide. The rise in antibiotic resistant infections, coupled with the sharp decline in the discovery of new and clinically useful classes of antibiotics, underscores an urgent need for alternative strategies to combat bacterial infections. Small noncoding RNA pathways have recently been recognized as important regulators of bacterial pathogenesis, and the challenge lies in gaining a detailed understanding of these processes. My research uses the tools of biochemistry and molecular genetics to unravel the mechanisms of small RNA-mediated pathways and enable the development of novel anti-microbial therapeutics.
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Rachel Haurwitz Caribou Biosciences Berkeley, CA USA
President and Chief Executive officer
http://cariboubio.com/about-us/management-team Biotech – Technology development
Rachel is a co-founder of Caribou Biosciences and has been President and CEO since its inception. She has a research background in CRISPR-Cas biology, and is also a co-founder of Intellia Therapeutics. In 2014, she was named by Forbes Magazine to the “30 Under 30” list in Science and Healthcare, and in 2016, Fortune Magazine named her to the “40 Under 40” list of the most influential young people in business. Rachel is an inventor on several patents and patent applications covering multiple CRISPR-derived technologies, and she has co-authored scientific papers in high impact journals characterizing CRISPR-Cas systems. Rachel earned an A.B. in Biological Sciences from Harvard College, and received a Ph.D. in Molecular and Cell Biology from the University of California, Berkeley.
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Sara Howden Murdoch Children Research Institute Melbourne Australia Senior Research Fellow Stem cell biology – Technology development
Around 10-20% of kidney disease is inherited. In children with kidney disease, this is closer to 50% although in many instances, the disease-causing mutation is unknown, therefore limiting treatment options. In our research group, we investigate the genes required for normal kidney development and what happens as a result of genetic or environmental damage during development. This knowledge is used to try to recreate kidney stem cells. We have developed methods for generating mini-kidneys from human stem cells that represent models of the human organ. We hope to use these mini-kidneys to screen drugs for kidney toxicity, as models with which to understand kidney disease, to generate cells for the treatment of kidney disease and eventually to bioengineer replacement organs.
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Nina Hoyland-kroghsbo Princeton University Princeton, NJ USA Postdoctoral fellow http://molbiolabs.princeton.edu/bassler/members Host-pathogens interaction
Research Interest: The global threat of multi-drug resistant bacteria urgently demands alternatives to conventional antibiotics. Two promising alternatives to traditional antibiotics are bacteriophage (phage) therapy and inhibitors of bacterial cell-cell communication, known as quorum sensing (QS). Bacteria in high cell density maximally engage in QS. These cells are particularly vulnerable to phage infections, which could rapidly spread and kill the population. QS-control of antiphage activities would enable bacteria to specifically activate defenses when they are at the highest risk of infection. I am investigating to what extent bacteria use QS to regulate their antiphage defenses. Whereas QS-inhibitory compounds are generally studied for their capacity to inhibit bacterial virulence, I will study whether they additionally have the ability to increase the vulnerability of pathogenic bacteria to phages.
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Danwei Huangfu Memorial Sloan Kettering New York, NY USA Head of laboratory https://www.mskcc.org/research-areas/labs/danwei-huangfu Stem cell biology – Technology development
The ability to program naïve cells or to reprogram differentiated cells into particular fates will open the door to the discovery of novel therapeutics for diseases such as diabetes. The goal of my lab is to understand the fundamental principles that govern the identity of a cell, and to use these principles to manipulate cell fates for regenerative medicine. In pursuit of this goal, we employ a variety of approaches including cellular programming and reprogramming through gene transduction, directed differentiation of embryonic stem (ES) cells, chemical screening, mouse genetics, adult tissue injury and regeneration, and tissue/cell transplantation.
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Maria Jasin Memorial Sloan Kettering New York, NY USA Head of laboratory https://www.mskcc.org/research-areas/labs/maria-jasin DNA repair mechanisms – DSB
Human chromosomes are constantly assaulted by challenges to their integrity as a result of either environmental agents that damage DNA or from normal DNA metabolism. The failure to repair damaged DNA faithfully is ultimately responsible for many human diseases, especially cancer. This laboratory focuses on the repair of 1 particular lesion in DNA, the double-strand break (DSB). DSBs arise from agents, such as ionizing radiation, and can also occur spontaneously during DNA replication. Our emphasis is on repair of DSBs by homologous recombination, with a particular interest in the role of homologous recombination in maintaining genetic stability. Understanding the repair of DSBs is not only important for basic science and health concerns, but also impacts on molecular genetic manipulations of mammalian genomes
21
Josephin Johnston The Hasting Centre Garrison, NY USA Director of Research http://www.thehastingscenter.org/team/johnston/ @bioethicsjosie Bioethics
Josephine Johnston is an expert on the ethical, legal, and policy implications of biomedical technologies, particularly as used in human reproduction, psychiatry, genetics, and neuroscience.
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Helene Jousset-Sabroux The Walter and Eliza Hall Institute for Medical Research Melbourne Australia Head of laboratory http://www.wehi.edu.au/people/hélène-jousset-sabroux
High Throughput Screening – Technology Development
The screening laboratory offers a wide range of expertise gained from both industrial and academic backgrounds, resulting in a professional ability to develop high capacity cellular or biochemical assays. We offer liquid handling robotics, plate readers and computing programs to increase the scale and speed of assays, and leverage automation to quickly assess the activity of a large number of compounds.
23
tamsin Lannagan University of Adelaide Adelaide Australia
Senior postdoctoral fellow
Cancer biology – Technology Development
My role within the group is to develop and assess novel mouse models of colorectal cancer, using colonoscopy techniques that are very similar to patient surveillance in humans. In addition, I am developing an in vitro method of growing mouse and human stem cells from the colon with their associated connective tissue. This will allow us to further investigate these support cells in normal growth and cancer. Both systems will be directly therapeutically relevant, allowing us to assess preclinical targeting of molecular pathways relevant to colorectal cancer.
24
Hong Li Florida State University Tallahassee, FL USA Professor http://biophysics.fsu.edu/hongli/ Structural Biology – RNA biology
A diverse range of RNA:protein, RNA:RNA and protein:protein interactions occur at the level of transcription and translation as well as post-transcriptional modifications. RNA:protein interactions are particularly interesting not only because they play important functional roles in assembly and biological processes, but also because the rules of their interactions are still poorly understood owing to the scarce structural data. Unlike DNA molecules, RNA can fold into a range of structures for interacting with proteins and small molecules. We hope, by providing exceptionally detailed images of the molecular events along the assembly and functional pathways, to unveil the underlying basis for assembly and functions involving RNA and partner proteins.
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Jennifer Listgarten Microsoft Research Cambridge, MA USA Senior Researcher http://www.jennifer.listgarten.com Computational biology – Technology development
My area of expertise is in machine learning and applied statistics for computational biology. I’m interested in both methods development as well as application of methods to enable new insight into basic biology and medicine.
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Shirley Liu Dana Farber Cancer Institute – Harvard Cambridge, MA USA Head of laboratory http://liulab.dfci.harvard.edu Computational biology – Technology development
We are developing the computational methods for the design (SSC), analysis (MAGeCK), hit prioritization (NEST), and visualization (VISPR) of genome-wide CRISPR screens. We are also using this technology to identify key genes in breast and prostate tumor progression and drug resistance. We also develop CRISPR screen platforms to understand the functions of enhancers and long-noncoding RNAs, and identify synthetic lethal gene pairs in cancer that leads to optimized cancer precision medicine.
27
Anita Marchfelder Ulm University Ulm Germany Head of laboratory https://www.uni-ulm.de/en/nawi/nawi-molbot/research/anita-marchfelder/ Host-pathogens interaction
All prokaryotic cells have to fend off foreign genetic elements like for instance viruses. To do that they have developed several different defence strategies. The recently discovered new defence strategy is the so called prokaryotic immune system also called CRISPR/Cas (CRISPR: clustered regularly interspaced short palindromic repeats, Cas: CRISPR-associated). It is adaptive, since cells can become immune against new invaders and it is heritable, since the information about the invader is stored in the genome. The CRISPR/Cas system consists of clusters of repetitive chromosomal DNA in which short palindromic DNA repeats are separated by spacers, the latter being sequences derived from the invader. In addition, a set of proteins, the Cas proteins, is involved in this defence reaction. We are investigating the CRISPR/Cas system in the halophilic archaeon Haloferax volcanii. Haloferax encodes a type I-B CRISPR/Cas system with eight Cas proteins and three CRISPR RNAs.
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Karen Maxwell University of Toronto Toronto Canada Assistant Professor http://individual.utoronto.ca/maxwell_lab/ @theMaxwellLab Host-pathogens interaction
The Maxwell lab studies the phages that infect and kill the human bacterial pathogens Escherichia coli, Pseudomonas aeruginosa and Staphylococcus aureus. Infections caused by these bacteria create a significant disease burden, and the increasing incidence of antibiotic resistant infections caused by these pathogens is one of our most serious health threats.
29
Barbara J Meyer University of California Berkeley Berkeley, CA USA Head of laboratory http://mcb.berkeley.edu/labs/meyer/ Nematode – Technology development
Targeted Genome-editing Across Highly Diverged Nematode Species. Thwarted by the lack of reverse genetic approaches to enable cross-species comparisons of gene function, we established robust strategies for targeted genome editing across nematode species diverged by 300 MYR. In our initial work, a collaboration with Sangamo BioSciences, we used engineered nucleases containing fusions between the DNA cleavage domain of the enzyme FokI and a custom-designed DNA binding domain: either zinc-finger motifs for zinc-finger nucleases or transcription activator-like effector domains for TALE nucleases (TALENs). In those experiments, we allowed the DNA double-strand breaks to be repaired imprecisely by non-homologous end joining (NHEJ) to create mutations in precise locations.
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Shondra Miller Washington University St Louis, MO USA Director of Research http://geic.wustl.edu/technology/ Stem cell biology – Technology development
The Genome Engineering and IPSC Center (GEiC) was formed by the consolidation of two pre-existing cores, the Genome Engineering Center and the Induced Pluripotent Stem cell (iPSC) core, both established by the Department of Genetics in the past few years. These two Centers were established to facilitate functional genomic studies through the use of patient-derived iPSCs and the generation of modified cells and organisms using genome editing technologies.
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Hiromi Miura Tokai University School of Medicine Kanagawa Japan Assistant Professor https://www.researchgate.net/profile/Hiromi_Miura Mouse – Technology development
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Kathy Niakan The Francis Crick Institute London UK Head of laboratory https://www.crick.ac.uk/research/a-z-researchers/researchers-k-o/kathy-niakan/ Stem cell biology – Technology development
The allocation of cells to a specific lineage is regulated by the activities of key signalling pathways and developmentally regulated transcription factors. The focus of our research is to understand the influence of signalling and transcription factors on differentiation during early human development.
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Kate O’Connor-Giles University Wisconson Madison Madison, WI USA Head of laboratory http://oconnorgiles.molbio.wisc.edu Drosophila -Technology development
We are also developing genetic technologies for identifying and gaining genetic control of neuronal subtypes to determine their characterize their roles in neural circuits. Working with the laboratories of Jill Wildonger and Melissa Harrison, we recently adapted the CRISPR/Cas9 system for use in Drosophila. CRISPR is a novel technique that is revolutionizing genome engineering. Developed from bacteria where the CRISPR/Cas9 system functions in acquired immunity, CRISPR technology enables highly efficient and specific editing of targeted genomic sequences – opening the door to routine genome engineering. The many applications of CRISPR technology include modifying the genomes of model organisms to probe gene function, conferring disease resistance to agricultural organisms, and correcting disease-causing mutations in humans. We are capitalizing on this advance to develop novel genome engineering approaches that overcome current technological limitations to understanding neural circuits. Visit our flyCRISPR and flyCRISPR Optimal Target Finder sites for more details on our genome engineering work.
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April Pawluk University of California Berkeley Berkeley, CA USA Postdoctoral fellow http://rna.berkeley.edu/people.html @AprilPawluk Host-pathogens interaction
Bacteria and their cognate viruses, known as bacteriophages, are in a constant battle for survival. Among many mechanisms that bacteria possess to defend against bacteriophage infection, one of the most widespread and sophisticated is the CRISPR-Cas system. Setting CRISPR-Cas apart from other defence systems is the fact that it is an adaptive immunity system: one that can acquire the ability to target newly encountered invaders in a sequence-specific manner. Although much has been uncovered about the targeting mechanisms of CRISPR-Cas systems, very little is known about how they select and capture genetic snapshots of bacteriophages for later use as guides for the “seek and destroy” machinery. I leverage biochemical and structural biology approaches to investigate the CRISPR-Cas adaptation process in detail.
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Jennifer Phillips University of Oregon Eugene, OR USA Research Fellow http://zfin.org/ZDB-PERS-040915-1 @ClutchScience Zebrafish – Technology development
Our laboratory studies the molecular genetic basis of human diseases, particularly Usher syndrome, the leading cause of combined deafness and blindess, and other diseases of the eye and ear.
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Wenning Qin Biogen inc Cambridge, MA USA Director of Research https://www.biogen.com @wenningqin Mouse – Technology development
Wenning has been focusing on and exploring into genetic engineering technologies in her entire professional career. Her association includes Monsanto Biosciences, Pharmacia Corporation, Pfizer Incorporated and the Jackson Laboratory. She currently directs the Genetically Engineered Models group of Biogen, leveraging into genetic engineering to advance drug discovery pipeline for Biogen. Over the years, she acquired extensive knowledge and experience in design and creation of genetically engineered models, using random transgenesis, conventional gene targeting as well as CRISPR/Cas9 technology.
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Rakhi Rajan The University of Oklahoma Norman, OK USA Assistant Professor http://www.ou.edu/cas/chemistry/directory/faculty/rakhi-rajan.html RNA biology – Adaptive immunity
Protein-nucleic acid interactions are key to fundamental life processes such as DNA replication, transcription, recombination, and protein synthesis. Deciphering the mechanism of protein-nucleic acid interactions is invaluable for understanding human disease pathways and infections. The primary focus of my lab is to characterize protein-DNA/RNA interactions structurally, biochemically, and biophysically. The immediate emphasis is the study of the recently discovered bacterial and archaeal immune system, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). CRISPR is an RNA-based adaptive immune system that inactivates foreign DNA/RNA entering the cell, based on the sequence similarity of small RNAs, called CRISPR RNA (crRNA) to the invading genetic element. The process requires several proteins called CRISPR associated (Cas) proteins. The CRISPR/Cas9 system has revolutionized the genome editing field due to the ease with which targeted double-stranded DNA breaks can be achieved in cells, using a guide RNA and Cas9 protein. The long-term goals of my laboratory are to understand the role of CRISPR/Cas system in pathogenicity and virulence of bacteria, characterize the mechanism of adaptation of bacteria to phage infection, and to determine the signaling mechanisms of the CRISPR/Cas system. We incorporate molecular biology, biochemistry, X-ray crystallography, and additional biophysical tools to characterize these protein-nucleic acid interactions.
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Dipali Sashital Iowa State University Ames, IA USA Assistant Professor http://www.sashitallab.org @dsashital RNA biology – Adaptive immunity
RNA-protein (RNP) complexes are central to many fundamental processes of gene regulation and genome maintenance in all kingdoms of life. The RNA components of these molecular machines often carry out diverse functions, acting as guide, template, scaffold, or catalyst. Despite this versatility, RNAs require protein partners to function, and the interactions that form between these components often dictate the overall activity of the RNP complex. Our lab is interested in understanding the molecular mechanisms underlying the function of RNPs from diverse cellular pathways. To that end, we combine a broad range of biochemical, structural and cellular tools to study RNA and protein structure, interactions and function.
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Nikki Shariat Gettysburg College Gettysburg, PA USA Assistant Professor https://sites.google.com/site/nikkishariat/home-1 RNA biology – Adaptive immunity
The Shariat Lab research interests are in prokaryote small RNA regulation and function, specifically in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPRs). These elements are present in nearly half of all sequenced bacterial genomes and comprise several unique short sequences, called spacers, which are interspaced by conserved direct repeats. Spacers are derived from exogenous nucleic acids, such as bacteriophage genomes and plasmids. The spacers are transcribed into CRISPR RNAs (crRNAs), which are subsequently targeted to complementary nucleic acids, resulting in degradation of the target. Due to acquisition of new spacers, CRISPRs provide a remarkably dynamic adaptive immune system in both bacteria and archaea.
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Bettina Schmid Deutsches Zentrum fur Neurodegenerative Erkankungen Helmotz Germany Head of laboratory https://www.dzne.de/en/sites/munich/research-groups/schmid.html Zebrafish – Technology development
Our group uses the advantages of the zebrafish, Danio rerio, as an in vivo model system to address some of the unresolved questions in Alzheimer’s disease, Parkinson’s disease, Frontotemporal Lobar Degeneration (FTLD), and Amyotrophic lateral Sclerosis (ALS).
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Kimberley Seed University of California Berkeley Berkeley, CA USA Assistant Professor http://www.kimseedlab.com/#introduction Host-pathogens interaction
The ability of V. cholerae to prevent phage predation is critical for its evolutionary fitness and epidemic potential. In turn, as obligate bacterial parasites, phages must co-evolve to overcome this resistance or they will face extinction. Our research is aimed at understanding the bacterial immunity and opposing phage immune evasion strategies at play in this dynamic co-evolutionary arms race. We use comparative genomics and complementary molecular approaches to identify and experimentally validate such strategies in disease associated phage and V. cholerae isolates.
42
Kaylene Simpson Peter McCallum Cancer Centre Melbourne Australia Associate Professor
High Throughput Screening – Technology Development
The Victorian Centre for Functional Genomics (VCFG) at Peter Mac offers biomedical researchers Australia-wide the ability to perform novel discovery-based functional interrogation all genes in the genome, or selected boutique collections using multiple platforms including CRISPR/cas9, small interfering RNA (siRNA), micro RNA (miRNA) and long non-coding RNA (lncRNA) and short hairpin RNA (shRNA).
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Joyce Van Eyck Cornell Univeristy Ithaca, NY USA Assistant Professor http://bti.cornell.edu/explore-bti/directory/joyce-van-eck/#research-overview Plant Biology (Tomato) – Technology Development
The focus of research in the Van Eck laboratory is biotechnological approaches to the study of gene function and crop improvement. For our studies, we apply several genetic engineering strategies to two major food crops: potato and tomato. The development of biotechnological techniques has made it possible to design and introduce gene constructs into plant cells and recover plants that express the introduced genes. Genes of interest to us have the potential to strengthen a plant’s resistance to disease, improve fruit characteristics, and enhance nutritional quality.
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Stineke Van Houte University of Exeter Exeter UK Research Fellow http://www.exeter.ac.uk/esi/people/researchandtechnical/van_houte/ Host-pathogens interaction
I am a biologist with a broad interest in host-parasite interactions, from an evolutionary, ecological and molecular perspective. Currently I work as a Marie-Curie fellow in the lab of Professor Angus Buckling on the evolution of immunity against virus infections in Pseudomonas bacteria. My PhD research at the Laboratory of Virology, Wageningen University (the Netherlands) focused on manipulation of host insect behaviour by baculoviruses, insect-specific viruses that cause lethal disease in caterpillars.
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Leslie Vosshall The Rockfeller Univeristy New York, NY USA Head of laboratory http://vosshall.rockefeller.edu @pollyp1 Insect – Technology development
The overall goal of work in our laboratory is to understand how complex behaviors are modulated by external chemosensory cues and internal physiological states. The lab takes a multi-disciplinary approach spanning cell biology, genetics, neurobiology and behavior. Our early focus has been to study how the brain interprets olfactory signals in the environment that signal food, danger, or potential mating partners. We have been studying these problems in three model organisms: the fly, the mosquito and the human. The majority of the early work in the laboratory was carried out in the genetically tractable vinegar fly, Drosophila melanogaster, which displays a rich repertoire of chemosensory behaviors despite having a nervous system with only 100,000 neurons. In this animal, we have studied the functional neuroanatomy of the olfactory system, how this system perceives sex pheromones, and the structure and function of the insect odorant receptors.
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Kan Wang Iowa State University Agron,IA USA Professor http://www.agron.iastate.edu/personnel/userspage.aspx?id=266 Plant biology (Maize) – Technology development
As the rapid development in plant genomics research identifies more genes, their functional analysis relies on strategies such as complementation, overexpression, or gene silencing. Plant genetic transformation is a critical technology required in the application of these strategies.
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Rachel Whitaker University of Illinois at Urbana Champaign Urbana, IL USA Associate Professor http://www.life.illinois.edu/whitaker/ Evolution and Ecology – Adaptive immunity
My lab combines population genomics with laboratory-based genetic and genomic experimental techniques to study the evolutionary ecology of microbial populations. We take a comparative approach, examining interactions within and between species using wild strains from natural populations isolated across spatial and temporal scales. Currently we are working on two critical forces that define the evolutionary process in all organisms: host-virus co-evolution and recombinational gene flow. We have a particular interest in how the unique biology of organisms in the Archaeal domain is reflected in genome architecture and how the CRISPR-Cas immune system functions in microbial populations.
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Susan Woods University of Adelaide Adelaide Australia Senior Research Fellow https://researchers.adelaide.edu.au/profile/susan.woods#career Cancer biology – Technology Development
Susan’s current project focuses on colorectal cancer. This is the second most common cancer type in Australia, costing us over $1 billion dollars annually. There are minimal effective treatments for advanced disease. The lab has recently identified a new stem cell that gives rise to a layer of cells that support the intestinal lining. We are investigating whether similar support cells can promote the formation of colorectal cancer from cells lining the intestine, and if we can prevent it using a new therapeutic approach.
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Luhan Yang eGenesis Cambridge, MA USA Co-founder and CSO http://www.egenesisbio.com/founding-team.html Biotech – Technology development
Luhan is leading the effort to eradicate PERVs from the porcine genome and engineer human compatibility in porcine cells. She previously developed the highly programmable genome-engineering tool, CRISPR/Cas9, for use in mammalian cells, and pioneered the first isogenic human stem cell lines to model human diseases at the tissue level. She was named among the “30 Under 30” in Science and Healthcare by Forbes Magazine (2014) and was a laureate of the “Young Entrepreneur Initiative” competition (2014). Luhan holds B.S. degrees in Biology and Psychology from Peking University and a Ph.D. in Human Biology and Translational Medicine from Harvard Medical School.
50
Yan Zhang University of Michigan Ann Arbor, MI USA Assistant Professor https://medicine.umich.edu/dept/biochem/yan-zhang-p RNA biology – Technology development
CRISPR-Cas is a RNA-guided, genetic interference pathway in prokaryotes that enables acquired immunity against invasive nucleic acids. Nowadays, CRISPRs also provide formidable tools for facile, programmable genome engineering in eukaryotes. Cas9 proteins are the “effector” endonucleases for CRISPR interference; and have recently begun to be also recognized as important players in other aspects of bacterial physiology (e.g. acquisition of new spacers into CRISPRs, endogenous gene regulation, and microbial pathogenesis, etc.).My laboratory is broadly interested in CRISPR biology and mechanism. We will use Neisseria species as our model system, and E. coli and human cells as additional platforms. We employ complementary biochemical, microbiological, genetic and genomic approaches. We are also interested in working with the broader scientific community to develop and apply novel CRISPR-based tools to tackle diverse biological questions.

Edwards Lifesciences closes $690m a buy of Valtech Cardio and most of the heart valve repair technologies it’s developing

Reporter: Aviva Lev-Ari, PhD, RN

 

Valtech’s  Cardioband device is designed to reshape the mitral valve using specially designed anchors, aka 

transcatheter structural heart disease technologies.

Valtech won CE Mark approval in the European Union for Cardioband in September 2015 but the device is not approved for the U.S. market.

Israel-based Valtech was the target of a previous takeover attempt by HeartWare International that was spiked early this year after a proxy war. (HeartWare itself was acquired by Medtronic (NYSE:MDT) for $1.1 billion in August.)

 

Deal Terms:

The deal, announced in November 2016, calls for $340 million in up-front cash and another $350 million in milestones over 10 years. It does not include Valtech Cardio’s trans-septal mitral valve replacement program; that business is slated to be spun out on its own before the buyout’s closing, expected in early 2017, but Edwards said last year that it’s due to keep an option to buy.

SOURCE

http://www.massdevice.com/edwards-lifesciences-closes-690m-valtech-cardio-buy/?utm_source=newsletter-170124&utm_medium=email&utm_campaign=newsletter-170124&spMailingID=10291384&spUserID=MTU0MTAzNDg3OTA5S0&spJobID=1081981757&spReportId=MTA4MTk4MTc1NwS2

Edwards Lifesciences closes $690m Valtech Cardio buy

JANUARY 24, 2017 BY

EDWARDS LIFESCIENCES COMPLETES ACQUISITION OF VALTECH CARDIO
IRVINE, Calif., Jan. 23, 2017 – Edwards Lifesciences Corporation (NYSE: EW), the global leader in patient-focused innovations for structural heart disease and critical care monitoring, today announced that it has closed its acquisition of Valtech Cardio Ltd., a privately held company based in Israel and developer of the Cardioband System for transcatheter repair of the mitral and tricuspid valves. Edwards announced in November that it had signed an agreement to acquire Valtech.
Under the terms of the merger agreement, Edwards paid $340 million in stock and cash for Valtech at closing, subject to typical adjustments. In addition, there is the potential for up to $350 million in pre-specified milestone-driven payments over the next 10 years. Edwards’ financial guidance provided at its Investor Conference in December incorporated the expected financial impact of the transaction in 2017.
“We look forward to the Valtech team joining Edwards. We believe their knowledge, experience and the Cardioband technology are valuable additions to Edwards,” said Michael A. Mussallem, Edwards’ chairman and CEO. “This therapy has the potential to be a breakthrough structural heart therapy to help many patients in desperate need, and we look forward to gaining valuable insights from its commercial use in Europe.”
The Cardioband System is not approved for sale in the United States. The mitral application of the Cardioband System has received CE Mark in Europe.
About Edwards Lifesciences

Edwards Lifesciences, based in Irvine, Calif., is the global leader in patient-focused medical innovations for structural heart disease, as well as critical care and surgical monitoring. Driven by a passion to help patients, the company collaborates with the world’s leading clinicians and researchers to address unmet healthcare needs, working to improve patient outcomes and enhance lives. For more information, visit http://www.edwards.com and follow us on Twitter @EdwardsLifesci.
SOURCE

 

Personalized Medicine Meeting – Discussion on FDA Regulation with Outgoing FDA Commissioner, Pharma and Investors

Reporter: Aviva Lev-Ari, PhD, RN

The agency has long felt that the current oversight provided by the Centers for Medicare & Medicaid Services under CLIA has critical gaps and is putting patients at risk. However, for more than two decades, the lab industry has aggressively fought against FDA regulation by threatening legal action and lobbying Congress. Despite industry objections, the FDA two years ago pushed ahead a draft guidance outlining a risk-based oversight plan for LDTs. However, following the November elections, the agency said it would hold off on finalizing those plans in order to consider input from other groups and await further input from the new administration.

Then, a week before Donald Trump was sworn into office, the agency released a discussion paper outlining a revised regulatory framework for LDTs based on more than 300 comments to its draft guidance, a public workshop, and meetings with stakeholders. The paper allowed the agency, without issuing enforceable regulations, to publicly respond to the lab industry’s concerns about burdensome requirements, demonstrate that it had listened to critics of the draft plan, and lay out the rationale, once again, for why FDA needed to step in to look at aspects of test development that CMS doesn’t.

“I think the community better take this really seriously,” Califf said discussing LDT regulation at the PMWC. While on the one hand regulation shouldn’t stifle innovation, he noted that doctors can’t be left to figure out which test they should order. “We’ve got to come up with some middle ground, so regardless of where you are in the US you can get a reproducible laboratory result,” he said, especially when patients’ treatment decisions depend on those results. “I think that’s upcoming work for this year,” Califf added.

Some industry observers have noted that in a crowded immunotherapy market, the availability of multiple PD-L1 tests, the availabilty of FDA approved kits and unapproved LDTs that seemingly gauge the same analyte, and the companion versus complementary diagnostic categories, are actually confusing physicians. This has led to some to suggest that drugmakers work with regulators to advance one test.

Additional Sources

  • New Complementary Dx Category Provides Regulatory Flexibility, but Poses Real WorldChallenges

GenomeWeb , 2016

  • At CDx Harmonization Meeting, Drugmakers Take First Step Toward Exploring Test Differences

GenomeWeb , 2015

  • In Approving Opdivo With Dakos Complementary Test, FDA Advances Rx Personalization Option

GenomeWeb , 2015

  • In 2016, Personalized Medicine Saw More CDx Deals, Flexible FDA, No LDT Guidance

GenomeWeb , 2017

  • Agilents Dako Lands FDA Approval for Lung Cancer Complementary Dx

GenomeWeb , 2015

  • In 2015, Precision Medicine Options Grew; FDA, Labs Still at Odds; Payment Remained Mostly Elusive

GenomeWeb , 2015

Outgoing FDA Commissioner, Pharma, Investors Discuss Regulation at Personalized Medicine Meeting

Jan 24, 2017 | Turna Ray

SOURCE

https://www.genomeweb.com/molecular-diagnostics/outgoing-fda-commissioner-pharma-investors-discuss-regulation-personalized?utm_source=SilverpopMailing&utm_medium=email&utm_campaign=Daily%20News:%20NSF,%20USDA%20Award%20Microbiome%20Research%20Grants%20-%2001/24/2017%2004:15:00%20PM