Posts Tagged ‘research funding’

Real Time Coverage @BIOConvention #BIO2019:  Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3 Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

Derisking Academic Science: The Unmet Need  

Translating academic research into products and new therapies is a very risky venture as only 1% of academic research has been successfully translated into successful products.

Collaboration from Chicago area universities like U of Chicago, Northwestern, etc.  First phase was enhance collaboration between universities by funding faculty recruitment and basic research.  Access to core facilities across universities.  Have expanded to give alternatives to company formation.
Half of the partnerships from Harvard and companies have been able to spin out viable startups.
Most academic PI are not as savvy to start a biotech so they bring in biotechs and build project teams as well as developing a team of ex pharma and biotech experts.  Derisk as running as one asset project.  Partner as early as possible.  A third of their pipeline have been successfully partnered.  Work with investors and patent attorneys.
Focused on getting PIs to get to startup.  Focused on oncology and vaccines and I/O.  The result can be liscensing or partnership. Running around 50 to 60 projects. Creating a new company from these US PI partnerships.
Most projects from Harvard have been therapeutics-based.  At Harvard they have a network of investors ($50 million).   They screen PI proposals based on translateability and what investors are interested in.
In Chicago they solicit multiple projects but are agnostic on area but as they are limited they are focused on projects that will assist in developing a stronger proposal to investor/funding mechanism.
NYU goes around university doing due diligence reaching out to investigators. They shop around their projects to wet their investors, pharma appetite future funding.  At Takeda they have five centers around US.  They want to have more input so go into the university with their scientists and discuss ideas.

Takeda: Data Validation very important. Second there may be disconnect with the amount of equity the PI wants in the new company as well as management.  Third PIs not aware of all steps in drug development.

Harvard:  Pharma and biotech have robust research and academic does not have the size or scope of pharma.  PIs must be more diligent on e.g. the compounds they get from a screen… they only focus narrowly

NYU:  bring in consultants as PIs don’t understand all the management issues.  Need to understand development so they bring in the experts to help them.  Pharma he feels have to much risk aversion and none of their PIs want 100% equity.

Chicago:  they like to publish at early stage so publication freedom is a challenge

Dr. Freedman: Most scientists responding to Nature survey said yes a reproduceability crisis.  The reasons: experimental bias, lack of validation techniques, reagents, and protocols etc.
And as he says there is a great ECONOMIC IMPACT of preclinical reproducability issues: to the tune of $56 billion of irreproducable results (paper published in PLOS Biology).  If can find the core drivers of this issue they can solve the problem.  STANDARDS are constantly used in various industries however academic research are lagging in developing such standards.  Just the problem of cell line authentication is costing $4 billion.
Dr. Cousins:  There are multiple high throughput screening (HTS) academic centers around the world (150 in US).  So where does the industry go for best practices in assays?  Eli Lilly had developed a manual for HTS best practices and in 1984 made publicly available (Assay Guidance Manual).  To date there have been constant updates to this manual to incorporate new assays.  Workshops have been developed to train scientists in these best practices.
NIH has been developing new programs to address these reproducability issues.  Developed a method called
Ring Testing Initiative” where multiple centers involved in sharing reagents as well as assays and allowing scientists to test at multiple facilities.
Dr.Tong: Reproduceability of Microarrays:  As microarrays were the only methodology to do high through put genomics in the early 2000s, and although much research had been performed to standardize and achieve best reproduceability of the microarray technology (determining best practices in spotting RNA on glass slides, hybridization protocols, image analysis) little had been done on evaluating the reproducibility of results obtained from microarray experiments involving biological samples.  The advent of Artificial Intelligence and Machine Learning though can be used to help validate microarray results.  This was done in a Nature Biotechnology paper (Nature Biotechnology volume28pages827–838 (2010)) by an international consortium, the International MAQC (Microarray Quality Control) Society and can be found here
However Dr. Tong feels there is much confusion in how we define reproduceability.  Dr. Tong identified a few key points of data reproduceability:
  1. Traceability: what are the practices and procedures from going from point A to point B (steps in a protocol or experimental design)
  2. Repeatability:  ability to repeat results within the same laboratory
  3. Replicatablilty:  ability to repeat results cross laboratory
  4. Transferability:  are the results validated across multiple platforms?

The panel then discussed the role of journals and funders to drive reproduceability in research.  They felt that editors have been doing as much as they can do as they receive an end product (the paper) but all agreed funders need to do more to promote data validity, especially in requiring that systematic evaluation and validation of each step in protocols are performed..  There could be more training of PIs with respect to protocol and data validation.

Other Articles on Industry/Academic Research Partnerships and Translational Research on this Open Access Online Journal Include

Envisage-Wistar Partnership and Immunacel LLC Presents at PCCI

BIO Partnering: Intersection of Academic and Industry: BIO INTERNATIONAL CONVENTION June 23-26, 2014 | San Diego, CA

R&D Alliances between Big Pharma and Academic Research Centers: Pharma’s Realization that Internal R&D Groups alone aren’t enough

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The Bioscience Crowdfunding Environment: Will Crowdfunding be the Bigger, New VC

Reporter: Stephen J. Williams, Ph.D.


Pharmaceutical Consulting Consortium International Inc. (PCCI) recently presented their 7th annual Roundtable “CROWDFUNDING FOR LIFE SCIENCES: A BRIDGE OVER TROUBLED WATERS?”, a panel discussion on how this new funding mechanism applies to early stage life science companies and changes the funding landscape.

A major provision in the recently passed JOBS Act resulted in Securities & Exchange Commission (SEC) rule changes revolutionizing the way companies can raise capital, with some figures in the range of $11 trillion dollars. Companies, startups, and entrepreneurs can, in a manner, now go directly to the individual investor and raise capital. This method is generally referred to as CROWDFUNDING.

As explained by Mark Roderick, moderator for the meeting, there are two main types of approved crowdfunding:

  • Donation-based Crowdfunding – Popularized by the crowdfunding platform Kickstarter, this method of raising capital can accept small donations from anyone for an idea/project to be completed. The donor may either get a free token of appreciation or access to enjoy the fruits of the project, for example, a watching a movie funded by the donor. Some scientific researchers have used Kickstarter as a method to fund their research.
  • Investor-based Crowdfunding– This type of crowdfunding involves the actual transfer of securities, and investors must qualify according to rules set by the SEC and go thru brokers, or portals, like the bioscience and healthcare internet portal Poliwogg.

Investor-based crowdfundingwas discussed at the meeting.  There are five different mechanisms with this type of funding: Title II (Rule 506c), Title II, Title IV, Existing Regulation A, and Rule 504. The main focus of the meeting was on Title II as, according to Mr. Roderick, involves the mechanism most suited for biotech startups, while rules for Title III still need to be finalized.

Title II crowdfunding requires that “accredited” or “qualified” investors (those who make at least $200,000/year or net worth $1 million US) go through licensed dealer internet nodes (or Portals) like Poliwog. The Portal will have lists of startups they deem legitimate which investors can choose from. For instance the Epilepsy Foundation uses Poliwog to fund certain projects.

The panelists discussed matters including:

  • How crowdfunding is different than other mechanisms like venture capital
  • What are the regulations and financial responsibilities for both biotech and crowdfunder
  • Liabilities
  • Due-diligence issues

The panelists included:

  1. Mark Roderick, moderator. Mark is an attorney at Flaster/Greenberg PC (@CrowdfundAttny on Twitter) and has developed great experience and expertise in the details of crowdfunding. He maintains a Crowdfunding blog www.crowdfundattny.com, which contains information and links about the JOBS Act and crowdfunding.
  2. Barbara Schiberg, Managing Director at BioAdvance, a Mid-Atlantic bioangel investment community.
  3. Samuel Wertheimer, PhD, CIO Poliwogg, a crowdfunding internet portal.
  4. Darrick Mix, Partner, Duane Morris LLP, corporate lawyer with experience in the JOBS act
  5. Donlon Skerret, PCCI President and CEO of NanoScan Imaging and serial entrepreneur

The Opportunity















Recent estimates place Title II Crowdfunding capacity to $1 Trillion.

Venture Capital (VC) had estimated only $5 Billion bio-investment in 2013.

Where does the rest go?


Mr. Skerret noted that bioangels can only take you so far but thinks that crowdfunding may fill this “valley of death”.



Crowdfunding is SELLING SECURITIESso there is liability, disclosure and nondisclosure issues.

Title II contains 580 pages of regulations and SEC needs a licensed intermediary.




Barbara Schiberg also noted that with VCs or bioangels groups you also get s support network, basically their rolodex of contacts and KOL’s and experts. With Crowdfunding like Poliwog they just handle linking investors with entrepreneur. Any contact is done through social media and the crowd.


BioAdvance hires experts – may take months to years to get expert opinion


Poliwog only has responsibility to investor to make sure company is legitimate. They don’t do extensive due diligence like bioangels. Most crowdfunding do not have extensive networks of professionals.



To obtain a video recording of this meeting and get more information please go to PCCI’s web site at http://www.rxpcci.com/meetings.htm.


Other posts on this site related to FUNDING and Bio Investing include:


PCCI’s 7th Annual Roundtable “Crowdfunding for Life Sciences: A Bridge Over Troubled Waters?” May 12 2014 Embassy Suites Hotel, Chesterbrook PA 6:00-9:30 PM

10 heart-focused apps & devices are crowdfunding for American Heart Association’s open innovation challenge

Importance of Funding Replication Studies: NIH on Credibility of Basic Biomedical Studies

Partnerships & Funding

Updated: Investing and Inventing: Is the Tango of Mars and Venus Still on

Transforming Biotech & Pharma: LinkedIn is the Quiet Force by Timmerman

Technion-Cornell Innovation Institute in NYC: Postdocs keep exclusive license to their IP and take a fixed dollar amount of Equity if the researchers create a Spinoff company




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Tycho Brahe, where art thou? Today’s Renaissance of the Self-Funded Scientist!

Curator: Stephen J. Williams, Ph.D.

Every scientist usually can describe an event or an admired historical figure as their pivotal point of inspiration which led them to embark on a scientific career.

I will admit there were two points of inspiration: the first was Jacques Cousteau while watching his program Undersea World of Jacques Cousteau.

The other (and please don’t laugh) was reading about the intellectual duel and collaboration between two of the greats in astronomy and mathematics: Tycho Brahe and Johannes Kepler, two historical figures responsible for our modern-day understanding of the universe and planetary motion.  For some reason I had romanticized the study of science, envisioning days in the laboratory wearing renaissance garb while striking medieval vogue poses (just kidding). But back then, accurately determining planetary motions and mapping the stars was a real big deal, as trade ships would rely on the positioning of stars as their heavenly GPS system.  Otherwise you might be trying to establish a new trade route to India and wind up somewhere… say America.


Tycho Brahe (1546-1601; born Tyge Ottesen Brahe) was a Danish nobleman and scientist who made the most accurate measurement of planetary motion and positioning of the stars, which enabled another great astronomer, Johannes Kepler, to deduce the laws of planetary orbits.  His measurements allowed Kepler to prove Copernicus’s sun centered theory (Earth revolves around the sun). An interesting history of Brahe, The crazy life and crazier death of Tycho Brahe, history’s strangest astronomer, gives some in-depth look at this intriguing historical figure.

Now back then science, as is the case now, costs money; and the two ways to get that money was either find a wealthy backer (like a king) or have a rich uncle who leaves a great inheritance.  Well Tycho did have a rich uncle who left him a lot of money, but instead of just sitting around spending it on jewelry, he used a great portion of his inheritance to build his 1st observatory to make his important measurements and also discover a supernova (published in De Nova Stella), breaking the dogma at the time that stars never changed their appearance or position.

(Photo Credit: Wikipedia)

There have been other examples of self-funded scientists including:

i.            Luther Burbank (b:1845) who led the way for plant genetics.  After developing the Burbank potato he used the money from his nursery business to buy a farm to conduct plant breeding experiments

ii.            Dr. Edward Jenner who used his own funds to develop the first smallpox vaccine and later awarded money from Parliament for his development

iii.            Ritu Levi Montalcini, M.D.:  Dr. Montalcini discovered nerve growth factor together with Stanley Cohen (both awarded Nobel Prize).  After earning an MD in Turin, Italy in 1938 she was unable to work as Mussolini banned Jews from holding professional positions.  So she moved to Belgium but when the Nazi’s took over she fled back to Turin and made a secret lab to study the development of neurons in chick embryos.

Now as many government science budgets are tightening some scientist are returning to self-funding and alternative models in order to continue their research.

The Ronin Institute

One such example is the Ronin Institute, founded by Dr. Jon Wilkins, Ph.D., where scientists who may not have institutional support, band together in a sort of virtual Institute which supports publication and grantsmanship.  The mission and values of the Ronin Institute (which can be found here) includes creating new models for the conducting, funding, collaboration, and dissemination of scholarly research to get researchers back to what they do best: RESEARCH.

A wonderful and inspiring article, written by Samuel Arbesman and co-authored by Jon Wilkens, can be found in Wired magazine.

The following is an excerpt from the article about independent researchers:

One of us (Jon Wilkins), has set out to promote and support independent scholarly research through the founding of the Ronin Institute. The Ronin Institute acts as an aggregator for the fractional scholars of the world, providing an institutional affiliation, connection with other fractional scholars, and support for conference travel and grant applications.

When people are doing something that they are passionate about, they work harder and produce a better product. Thus, underemployed scholars represent in some sense a good that is currently trading well below its actual value. By providing a mechanism for those who wish to conduct research, we can allow these people to engage in their passions while growing the base of scholarly knowledge, which in turn has the potential to create further economic growth.

Through the Ronin Institute, we will be harnessing the skills and talents of thousands of underemployed researchers.

Some more articles on the Ronin Institute can be found on their site at http://ronininstitute.org/press/

Two other great articles on “gentlemen scientists” or self-funding scientists can be found at the Singular Scientist blog post entitled “Self-Funding in Science” and a 1998 Science article by Jon Cohen entitled Scientists Who Fund Themselves.  In each case, scientists felt freed up from the financial overhead accompanied with big institutions and realized more time for their research.

Alternate Funding Source: CROWDSOURCING

The passage of the JOBS act has relieved some of the pressures off obtaining funding for companies through crowdfunding mechanisms.  Scientists are also turning to crowdsourcing mechanisms to fund their research.  An article in the Washington Times (Scientists discover ‘crowdfunding’ as a way of replacing research grants) highlights some of the successes and science-related crowdfunding sites that exist.

Science-related Crowdfunding sites include:

i.            Rockethub

ii.            SciFund Challenge

iii.            Microryza

iv.            Kickstarter

v.            Petridish.org

Digital Tools and Lab Space for the Self-Funding Scientist

Dr. Elizabeth Iorns, breast cancer researcher and founder and CEO of Science Exchange, an online marketplace for ordering science experiments from various nationwide and worldwide labs, explains in a three-part Nature blog post “Research 2.0.1: The future of research funding” how the traditional government-based grant-funding model may transition into a more crowdfunding model.  For example Science Exchange allows you to order common laboratory procedures (for example immunohistochemistry or bioinformatics analysis or gene sequencing) from a list of participating labs in the marketplace.  Prices are usually reasonably priced.

Finding Lab Space: Biohacker Labs

The last piece of the puzzle is finding rented space and equipment to do research.  A new type of laboratory space, small, nimble, and priced and equipped to fit the independent researcher is cropping up.  Termed biohacker labs or hackubators, these small rented communal spaces are different from the traditional bio-incubators or science centers which sprang up decades ago to foster the biotech revolution.  This phenomenon is explained quite nicely in a Science article by Virginia Gewen “Biotechnology: Independent Streak”.  These spaces can go for $100-400 a month, much less than $900 a month for incubator space. Most of the investigators highlighted in the article get funds through crowdsourcing.

One such hackubator lab is Bio, Tech and Beyond, a DIY lab in San Diego which supports numerous projects using 3D printing, cell culture, and sequencing.  These type of DIY biolabs are springing up all over, based on the idea from tech hacker DIY labs, although before the expense seemed to be the limiting factor.  Now it appears the internet is once again revolutionizing another industry, namely that of the independent bio researcher

…. Sans the 16th century fashion (what a shame!)

Other posts on this site about Science Funding, Crowdsourcing, and Open Innovation include:

10 heart-focused apps & devices are crowdfunding for American Heart Association’s open innovation challenge

Importance of Funding Replication Studies: NIH on Credibility of Basic Biomedical Studies

Digital Health: SXSW Interactive 2014, March 9, 2014, Startup Village, Hilton Austin Downtown, 4th Floor


Fourth Annual QPrize Competition to Fund the World’s Next Groundbreaking Startups by Qualcomm Ventures

Gamification of Genomics and Proteomics Research

Collaborations and Open Access Innovations – CHI, BioIT World, 4/29 – 5/1/2014, Seaport World Trade Center, Boston

The Fatal Self Distraction of the Academic Publishing Industry: The Solution of the Open Access Online Scientific Journals

e-Recognition via Friction-free Collaboration over the Internet: “Open Access to Curation of Scientific Research”

Investing and inventing: Is the Tango of Mars and Venus Still on

2013 Genomics: The Era Beyond the Sequencing of the Human Genome: Francis Collins, Craig Venter, Eric Lander, et al.

conceived: NEW Definition for Co-Curation in Medical Research

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AACR announces AACR Progress Report 2013

Stephen J. Williams: Curator

The American Association for Cancer Research (AACR) presented a webinar of the highlights of their yearly progress report (released yesterday and available on the AACR website) on the recent advances and current status of cancer research and cancer research’s impact on health outcomes in the United States.  This report, compiled by staff of AACR, with special thanks to the efforts of Dr. Karen Honey, Ph.D, reports on the current achievements in cancer research including developments in immunotherapies, new drug approvals, health outcomes, newly approved imaging modalities, and the current state of affairs of funding for cancer research and clinical trials.  The report also describes the impact and timeline of discoveries leading to the use of genomics and personalized medicine in cancer treatment.  The last portion of the report is an “AACR Call to Action”, imploring cancer patient activists, scientists, and citizens to write their representatives in Washington for increased funding for cancer research and clinical trials.  The report and presentation will be given to lawmakers on Capital Hill on Spetmeber 19, 2013 as part of Hill Day’s Rally for Medical Research.

The presentation, given on September 18, 2013 at the National Press Club in Washington DC) was headed by AACR CEO Dr. Marge Foti, M.D., Ph.D. with presentations given by

  • Dr. Charles Sawyers, M.D. (Memorial Sloan Kettering)
  • Dr. Drew M. Pardoll, M.D., Ph.D. (Sidney Kimmel Cancer Center, Johns Hopkins)
  • 3 cancer survivors

Below is a brief summary of each of their talks.  The downloadable AACR Progress Report 2013 can be found here and a link to the video can also be found at the AACR website.

Marge Foti, M.D., Ph.D. (Chief Executive Officer, American Association Cancer Research)

Although Dr. Foti mentioned the grim statistic in the US 580,000 this year will die of cancer, she gave multiple statistics on the great progress the US has achieved since staring the “War on Cancer” in 1971 and the future progress which lies ahead.  Notably (from the report)

  • From 1990 to 2012 over 1 million cancer patients lives have been saved
  • There are over 13 million cancer survivors today
  • For the year 2012-2013 FDA has approved
  1. 11 new cancer drugs
  2. 3 new uses of previously approved drugs
  3. 3 new imaging modalities and protocols for cancer detection

However Dr. Foti also stressed the speed of progress is being pressured by diminishing federal funds for cancer research and clinical trials.  Dr. Foti noted:

  • In mid 90’s there was a doubling of federal funds to the NCI
  • Since 2003 however funding has not kept up with “biomedical inflation” (not risen adjusted for current inflation)
  • Sequester has been a big pressure on biomedical and cancer research capacity
  • Funding cuts also decrease the number of patients that can enroll in clinical trials

Charles Sawyers, M.D. (Howard Hughes Medical Institute investigator and Director at Memorial Sloan-Lettering Cancer Center)

Dr. Sawyers’s research work involves the signaling pathways involved in conferring growth advantage to cancerous cells.  His work led to the development of numerous targeted therapies such as imatinib (Gleevec) for CML (chronic myeloid leukemia).  He referred to these therapies as “precision medicine” and noted there were only 5 such therapies 10 years ago but now 17 such precision medicines five years ago for cancer, “ a complex host of diseases”.

Dr. Sawyers reflected this is the “most serious funding crisis in decades” and we are “already losing momentum” due to the current funding crisis.

Drew M. Pardoll, M.D. Ph.D. (Professor, Co-Director Division Immunology, Johns Hopkins)

Dr. Pardoll is a leader in the fielod of immunotherapy for cancer and his work is pioneering a new clas of immunotherapies, such as PD1 inhibitors, which supports the cancer patient’s own immune system to fight and kill the patient’s own cancer cells.  Dr. Pardoll had mentioned early work on immunotherapy had revealed its potential but researchers are now realize this is the “5th pillar of cancer therapy”.  Because of research done in the early 2000’s, cancer researchers such as Dr. Pardoll figured out mechanisms how to make these immunotherapies more reproducible in clinical trials.  This led to the discovery of CTLA4 and PD1 as major regulators of the immune tolerance to cancer cells (see post Combined anti-CTLA4 and anti-PD1 immunotherapy shows promising results against advanced melanoma).

Dr. Pardoll also mentioned how he, and others, noticed that the pharmaceutical industry is now looking to academia to keep driving the science and that patient advocates are very important partner in the discovery process.

Moving presentation were also given by three cancer survivors (breast cancer, ovarian cancer, and  childhood leukemia) which all attested that without ground-breaking clinical research they might not have survived their deadly cancer.

Please see the following website below about the Rally for Medical Research to see how you can get involved in supporting cancer research in the US, and contacting your representative.

Rally for Medical Research Hill Day

September 18, 2013

Federal funding for medical research is in jeopardy, threatening the health of Americans. On September 18, 2013, a broad coalition of groups from the medical research advocacy community will meet with House and Senate offices in Washington, D.C., to urge Congress to invest in the National Institutes of Health for the health and economic security of our nation.

Sponsoring organizations will join the Rally for Medical Research Hill Day to raise awareness during a critical time about the urgent need for a sustained investment in the NIH to improve health, spur more progress, inspire more hope and save more lives.

More articles on Progress on the War on Cancer from this site include:

2013 Perspective on “War on Cancer” on December 23, 1971

2013 American Cancer Research Association Award for Outstanding Achievement in Chemistry in Cancer Research: Professor Alexander Levitzki

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Curator: Meg Baker, PhD, Reg Patent Agent


Novartis provides funding for research of modified T cell treatments. Successful application of the technique was demonstrated in a clinical study led by Dr. Carl June, a pathologist at the University of Pennsylvania, for CLL (chronic lympocytic leukemia), one of the most common types of leukemia. The initial study report was in August 2011 http://www.uphs.upenn.edu/news/News_Releases/2011/08/t-cells/

The concept of doctoring T-cells genetically was first developed in the 1980s by Dr. Zelig Eshhar at the Weizmann Institute of Science in Rehovot, Israel. It involves adding gene sequences from different sources to enable the T-cells to produce what researchers call chimeric antigen receptors, or CARs — protein complexes that transform the cells into, in Dr. June’s words, “serial killers.” See http://www.nytimes.com/2011/09/13/health/13gene.html?pagewanted=all

Dr. June describes the new therapy as “ultrapersonalized” because  the treatments involve extracting a  patient’s immune cells and using deactivated HIV-1 to deliver genes into the cells, and later infusing in the re-educated cells back into patient’s system.  The treatment is characterized as “training the immune system” to attack cancer.  Thus, it is hoped that the technology can be applied more broadly to cancer therapy.

Read more: Novartis backs UPenn’s pioneering cancer immunotherapies – FierceBiotech http://www.fiercebiotech.com/story/novartis-backs-upenns-pioneering-cancer-immunotherapies/2012-08-06#ixzz25tGYGQ9N
Subscribe: http://www.fiercebiotech.com/signup?sourceform=Viral-Tynt-FierceBiotech-FierceBiotech

NYT article: http://www.nytimes.com/2012/08/06/business/novartis-and-penn-unite-on-anticancer-approach.html

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