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CRISPR Patent Battle Determined on 2/15/2017 – USPTO issues a verdict in legal tussle over rights to genome-editing technology

Curator: Aviva Lev-Ari, PhD, RN

Broad Institute prevails in heated dispute over CRISPR patents

Sharon Begley sharon.begley@statnews.com
@sxbegle

In a one-sentence judgment by the Patent Trial and Appeal Board, the three judges decided that there is “no interference in fact.” In other words, key CRISPR patents awarded to the Broad beginning in 2014 are sufficiently different from patents applied for by UC that they can stand. The judges’ full 51-page decision explaining their reasoning stated that the Broad had persuaded them “that the parties claim patentably distinct subject matter.”

https://www.statnews.com/2017/02/15/crispr-patent-ruling/#decision

MIT

The Broad said in a statement that the decision “confirms that the patents and applications of Broad Institute and UC Berkeley are about different subjects and do not interfere with each other.”

UC, Berkeley

In a statement, the University of California said it was pleased that its patent application, which it described as covering “the invention and use of CRISPR gene editing in all cells,” can move forward. “We continue to maintain that the evidence overwhelmingly supports our position that the Doudna/Charpentier team was the first group to invent this technology for use in all settings and all cell types,” it said, “and that the Broad Institute’s patents directed toward use of the CRISPR-Cas9 system in particular cell types are not patentably distinct from the Doudna/Charpentier invention.”

UC said it is considering its legal options, including the possibility of an appeal, but it contended that anyone who wants to develop CRISPR-based treatments for human diseases would have to license not only the Broad’s patents but also those that UC expects to be awarded. “Ours,” Doudna told reporters, “is for the use [of CRISPR] in all cells,” including human ones.

PTAB appeals are heard by the US Court of Appeals for the Federal Circuit, which sits in Washington. In recent years, more than half of PTAB’s decisions have been upheld.

“The Federal Circuit heard three appeals of interferences in 2016,” said Sherkow. “All three were at least affirmed in part. It’s completely unclear whether that’s meaningful — it’s an N of 3–but there you go.” Overall, on 155 appeals since PTAB was created in 2012, the Federal Circuit affirmed 120 on every issue, dismissed or reversed 21 on every issue, and issued partial decisions (that is, upholding parts of a PTAB decision and reversing others) in the other 14.

https://www.statnews.com/2017/02/15/crispr-patent-ruling/#decision

Said UC attorney Lynn Pasahow:

For “all tennis balls,” read “all cells.” For “green tennis balls,” read “eukaryotic cells.”

https://www.statnews.com/2017/02/16/crispr-patent-decision-six-takeaways/

 

What will that mean for licensees of CRISPR patents?

Stanford University Voice

UC believes that any company that wants to use CRISPR to develop human therapies — we’re looking at you, Editas Medicine — will need to license not only the Broad’s patents on eukaryotic cells but also those UC expects to receive on all kinds of cells. “It looks to me as if someone wanting to use the Broad patent would also have to license the UC patent,” agreed law professor Hank Greely of Stanford University. “The UC patent (if granted) would be on any use; the Broad would be on use in eukaryotes. I think someone who wanted to do this in eukaryotes would need to have licenses to both.”

CRISPR-Cas9 is unlikely to be the last genome-editing technology ever discovered. In 2015, Zhang and his colleagues discovered a version called Cpf1, which they’ve now patented and licensed to Editas. “I continue to think the possibility of inventing around the [CRISPR] patents seems very likely,” said Stanford’s Greely. Bacteria “have certainly come up with other ways to reach the same end [of genome editing], ways that aren’t covered by UC’s or the Broad’s claims. That could make either of these patents ultimately of little importance … especially if the licensing conditions give people a strong incentive to come up with invent-arounds.” Science will march on.

https://www.statnews.com/2017/02/16/crispr-patent-decision-six-takeaways/

What does the CRISPR ruling mean for biotech?

By DAMIAN GARDE @damiangarde

FEBRUARY 15, 2017

Editas Medicine, which has aligned with the winning Broad, saw its share price rise more than 25 percent on Wednesday. Intellia Therapeutics, affiliated with UC, fell about 11 percent, while compatriot CRISPR Therapeutics dipped 24 percent.

https://www.statnews.com/2017/02/15/what-does-the-crispr-ruling-mean-for-biotech/

 

Broad Institute wins bitter battle over CRISPR patents

The US Patent and Trademark Office issues a verdict in legal tussle over rights to genome-editing technology.

15 February 2017 Updated:In December 2016, lawyers representing the University of California and the Broad Institute participated in oral arguments before a trio of patent-court judges. University of California attorney Lynn Pasahow said that the team had not yet decided whether it would appeal the verdict on 2/15/2017.

Lawyers representing the University of California filed for an ‘interference’ proceeding, in an effort to have the Broad’s patents thrown out. But on 15 February, patent judges determined that there was no interference, meaning that the Broad’s invention is distinct from that of the University of California, and the Broad patents will stand. The University of California’s patent application will now be referred back to an examiner, but legal challenges could continue.

molecular biologist Jennifer Doudna of the University of California in Berkeley, likened the situation to licensing permission to someone who wants to use green tennis balls. “They will have a patent on the green tennis balls,” she said, referring to the Broad patents. “We will have a patent on all tennis balls.” ”Doudna argued at the press conference that the patent battle had not hampered research, given the speed with which researchers had taken up the technique and companies had rushed to commercialize it.”

The University of California’s invention would cover the design of the RNA molecule that guides the key step in CRISPR–Cas9 gene editing, directing the Cas9 enzyme to a specific site in the genome. But getting that system to work in eukaryotes was an additional inventive step, Coombes says, a patent lawyer at intellectual-property specialists HGF in York, UK.

SOURCE

Nature doi:10.1038/nature.2017.21502

http://www.nature.com/news/broad-institute-wins-bitter-battle-over-crispr-patents-1.21502?WT.ec_id=NEWSDAILY-20170216%20

https://www.statnews.com/2017/02/15/crispr-patent-ruling/#decision

Related articles from nature.com

Other related articles published in this Open Acceaa Online Scientific Journal include the following:

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

 

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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

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Forward Pharma settled with Biogen on their patent dispute on active ingredient for MS Tecfidera (dimethyl fumarate): Biogen will pay $1.25 billion upfront, level of royalties TBD

Reporter: Aviva Lev-Ari, PhD, RN

 

Biogen will pay $1.25 billion upfront, but the level of royalties will be dependent on the interference proceeding hearing decision. The royalty rate could range from 1% to 10%, and could increase to as high as 20%, depending on the outcome – Forward Pharma settled with Biogen on their patent dispute regarding the active ingredient for the blockbuster multiple sclerosis drug Tecfidera (dimethyl fumarate).

 

SOURCE

http://www.biopharmadive.com/news/biogen-settles-in-tecfidera-patent-case/434138/

Biogen’s Tecfidera faces outside pressure

http://www.biopharmadive.com/news/biogen-tecfidera-patent-forward-court/431486/

 

Biogen and Forward Pharma Agree to Enter into Settlement and License Agreement

Biogen to Pay $1.25B in Exchange for License Agreement to Forward Pharma Intellectual Property

Future Payment of Royalties Subject to Resolution of Ongoing Patent Procedures in US and EU

Category:

Tuesday, January 17, 2017 7:30 am EST
“We are very pleased to have reached this settlement with Forward Pharma. We believe this agreement will clarify and strengthen our intellectual property for TECFIDERA, the leading oral therapy for multiple sclerosis”

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen Inc. (NASDAQ: BIIB) today announced that it has agreed to enter into a settlement and license agreement with Forward Pharma, subject to the approval of Forward Pharma’s shareholders and other customary conditions. The license agreement will provide Biogen an irrevocable license to all intellectual property owned by Forward Pharma.

Upon the effectiveness of the settlement and license agreement, Biogen will provide Forward Pharma a cash payment of $1.25 billion. Under certain circumstances outlined in the agreement, Biogen will pay Forward Pharma royalties on net sales of Biogen products for the treatment of multiple sclerosis that are covered by a Forward Pharma patent and have dimethyl fumarate (“DMF”) as an active pharmaceutical ingredient.

“We are very pleased to have reached this settlement with Forward Pharma. We believe this agreement will clarify and strengthen our intellectual property for TECFIDERA, the leading oral therapy for multiple sclerosis,” said Michel Vounatsos, Chief Executive Officer of Biogen.

The settlement and license agreement does not resolve the issues pending in the ongoing Interference Proceeding in the U.S. or the Opposition Proceeding in the EU. Biogen and Forward Pharma intend to permit the Patent Trial and Appeal Board (PTAB), the U.S. Court of Appeals for the Federal Circuit, the European Patent Office, and the Technical Board of Appeal and the Enlarged Board of Appeal, make a final determination in the proceedings before them.

Summary of Details and Conditions of the Agreement

The agreement to enter into a settlement and license agreement (the “License Agreement”) announced today was reached between Biogen’s wholly owned subsidiaries, Biogen Swiss Manufacturing GmbH and Biogen International Holding Ltd., and Forward Pharma A/S, a Danish limited liability company (“Forward Pharma”) and additional related parties and is subject to the approval of Forward Pharma’s shareholders and other customary conditions. The approval of two-thirds of Forward Pharma’s voting share capital is required to approve the License Agreement. Shareholders representing approximately 77% of Forward Pharma’s voting share capital have irrevocably agreed to vote in favor of the License Agreement. Forward Pharma has agreed to convene an extraordinary general meeting on February 1, 2017 to obtain the approval of its shareholders.

The License Agreement will have a perpetual term and provide for the grant to Biogen of an irrevocable, co-exclusive license to all intellectual property owned by Forward Pharma in the U.S. (the “U.S. Licensed Intellectual Property”). The co-exclusive U.S. license may be converted into an irrevocable exclusive license subject to the conditions in the License Agreement, which include the absence of legal restraints and the receipt of all necessary regulatory approvals. The License Agreement will also provide for the grant to Biogen of an irrevocable, exclusive license to all intellectual owned by Forward Pharma anywhere else in the world (collectively, the “Designated Countries Licensed Intellectual Property”).

Upon the execution and delivery of the License Agreement, Biogen will pay Forward Pharma a non-refundable cash payment of $1.25 billion which will not affect Biogen’s 2016 Non-GAAP financial results. Under certain circumstances, Biogen will also be obligated to pay Forward Pharma future royalties on net sales of Biogen products for the treatment of multiple sclerosis that are covered by a Forward Pharma patent and have dimethyl fumarate (“DMF”) as an active pharmaceutical ingredient.

Biogen will only be obligated to pay Forward Pharma royalties in the U.S. if Forward Pharma obtains patent rights covering treatment of a human for multiple sclerosis by orally administering 480 mg per day of DMF arising from the interference proceeding between the Company and Forward Pharma that is currently pending at the Patent Trial and Appeal Board (“PTAB”) of the United States Patent and Trademark Office (the “Interference Proceeding”). If royalties are payable in the U.S. and Biogen holds a co-exclusive license, a royalty of 1% will be payable from January 1, 2023 until the earlier of the expiration, unenforceability or invalidation of the patents included in the U.S. Licensed Intellectual Property. If Biogen holds an exclusive license, a royalty of 10% will be payable from January 1, 2021 to December 31, 2028 and a royalty of 20% will be payable from January 1, 2029 until the earlier of the expiration, unenforceability or invalidation of the patents included in the U.S. Licensed Intellectual Property.

Biogen will only be obligated to pay Forward Pharma royalties in countries other than the U.S. if Forward Pharma obtains patent rights covering treatment of a human for multiple sclerosis by orally administering 480 mg per day of DMF in the opposition proceeding against Forward Pharma’s European patent EP 2801355 (Application No. 14172398.1) (the “Opposition Proceeding”). If royalties are payable in countries other than the U.S., a royalty of 10% of Net Sales of applicable infringing products will be payable on a country-by-country basis, from January 1, 2021 to December 31, 2028, and a royalty of 20% will be payable on a country-by-country basis from January 1, 2029 until the earlier of the expiration, unenforceability or invalidation of the patents included in the Designated Countries Licensed Intellectual Property in each country.

The License Agreement does not resolve the issues pending in the Interference Proceeding or the Opposition Proceeding. Biogen and Forward Pharma intend to permit the PTAB and the U.S. Court of Appeals for the Federal Circuit, as applicable, and the European Patent Office and the Technical Board of Appeal and the Enlarged Board of Appeal, as applicable, to make a final determination in the proceedings before them.

About Biogen

Through cutting-edge science and medicine, Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological, autoimmune and rare diseases. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For more information, please visit www.biogen.com. Follow us on Twitter.

Safe Harbor

This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements relating to: Biogen’s commercial business, the obligation to make, the anticipated amount of, and the timing of, royalty payments under the License Agreement, the timing, outcome and impact of administrative, regulatory, legal and other proceedings related to patents and other proprietary and intellectual property rights, the strength and value of intellectual property rights, and the approval of the License Agreement and the transactions contemplated by the License Agreement by Forward Pharma’s shareholders and regulatory authorities and tribunals. These forward-looking statements may be accompanied by such words as “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: risks relating to management and key personnel changes; failure to compete effectively; difficulties in obtaining and maintaining adequate coverage, pricing and reimbursement for our products; potential future healthcare reforms; the occurrence of adverse safety events; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; uncertainty of success in developing, licensing or acquiring other product candidates or additional indications for existing products; and other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the SEC.

These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.

Contact:

Biogen Media Contact:
Biogen Inc.
Jason Glashow, 781-464-3260
or
Biogen Investor Contact:
Biogen Inc.
Matt Calistri, 781-464-2442

SOURCE

http://media.biogen.com/press-release/investor-relations/biogen-and-forward-pharma-agree-enter-settlement-and-license-agreem

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Four patents and one patent application on Nanopore Sequencing and methods of trapping a molecule in a nanopore assigned to Genia, is been claimed in a Law Suit by The Regents of the University of California, should be assigned to UCSC

Reporter: Aviva Lev-Ari, PhD, RN

 

The university claims that while at UCSC Roger Chen’s research focused on nanopore sequencing, and that he along with others developed technology that became the basis of patent applications filed by the university. However, when Chen left the university in 2008 and cofounded Genia, he was awarded patents for technology developed while he was at UCSC, but those patents were assigned to Genia and not the university, according to the suit.

In the suit, the university notes four patents and one patent application assigned to Genia that it claims should be assigned to UCSC: US Patent Nos., 8,324,914; 8,461,854; 9,041,420; and 9,377,437; and US Patent Application 15/079,322. The patents and patent applications all relate to nanopore sequencing and specifically to methods of trapping a molecule in a nanopore and characterizing it based on the electrical stimulus required to move the molecule through the pore.

Genia was founded in 2009, and in 2014, Roche acquired the startup for $125 million in cash and up to $225 million in milestone payments. Earlier this year, the company published a proof-of-principle study of its technology in the Proceedings of the National Academy of Sciences.

Roche’s head of sequencing solutions, Neil Gunn, said that Roche would announce a commercialization timeline in 2017.

It’s unclear how the lawsuit will impact that commercialization, but Mick Watson, director of ARK-Genomics at the Roslin Institute in the UK, speculated in a blog post that if the suit is decided in favor of UCSC, it could result in a very large settlement and potentially even the end of Genia.

 

SOURCE

https://www.genomeweb.com/sequencing/university-california-files-suit-against-genia-cofounder

http://www.opiniomics.org/university-of-california-makes-legal-move-against-roger-chen-and-genia/

 

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Interview with Bill Zurn, Scientist and Inventor in http://www.globalinnovationmagazine.com October 2016″

Reporter: Aviva Lev-Ari, PhD, RN

 

BILL ZURN’S drill bit & cylinder patent was issued on Oct 11, 2016. 

US Patent 9,464,487

http://pdfpiw.uspto.gov/.piw?PageNum=0&docid=09464487&IDKey=9542CA372E67&HomeUrl=http%3A%2F%2Fpatft1.uspto.gov%2Fnetacgi%2Fnph-Parser%3FSect1%3DPTO1%2526Sect2%3DHITOFF%2526d%3DPALL%2526p%3D1%2526u%3D%25252Fnetahtml%25252FPTO%25252Fsrchnum.htm%2526r%3D1%2526f%3DG%2526l%3D50%2526s1%3D9%2C464%2C487.PN.%2526OS%3DPN%2F9%2C464%2C487%2526RS%3DPN%2F9%2C464%2C487

zurn_interview_global_innovation_mag_10-04-2016_page_1

 

zurn_interview_global_innovation_mag_10-04-2016_page_2

Permission to Re-Publish Interview with Bill Zurn

“This interview was first featured in www.globalinnovationmagazine.com October 2016″.

From: clifford.thornton@gmail.com

Date: Fri, 14 Oct 2016 02:21:39 -0400

Subject: Fwd: Request permission to re-publish William Zurn Interview – Leaders in Pharmaceutical Business Intelligence (LPBI) Group

To: wilzur@msn.com

CC: avivalev-ari@alum.berkeley.edu; jamesoflynn@hotmail.com; clifford.thornton@gmail.com

Bill,

Per James O’Flynn and his forwarded Email below, he is fine with you re-publishing the interview in LPBI.  He has granted you permission for that initiative. 

He has requested, as a condition of that permission, to note in the related LPBI publication/ re-publishing, “This interview was first featured in www.globalinnovationmagazine.com October 2016″.

Regards,

Cliff

———- Forwarded message ———-

From: james oflynn <jamesoflynn@hotmail.com>

Date: Fri, Oct 14, 2016 at 2:01 AM

Subject: Re: Request permission to re-publish William Zurn Interview – Leaders in Pharmaceutical Business Intelligence (LPBI) Group

To: Clifford Thornton <clifford.thornton@gmail.com>

That’s fine, I would like it noted in their publication though i.e. ‘This interview first featured in www.globalinnovationmagazine.com October 2016′

Best

James 

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LIVE – The CRISPR/Cas9 Revolution and Gene Editing: 2016 WARREN ALPERT FOUNDATION PRIZE SYMPOSIUM

Reporter: Aviva Lev-Ari, PhD, RN

Each year the recipient(s) of the Warren Alpert Foundation Prize are recognized at a scientific symposium hosted by Harvard Medical School.

OCTOBER 6, 2016 –

1:30PM TO 5:30PM

2016 WARREN ALPERT FOUNDATION PRIZE SYMPOSIUM

The CRISPR/Cas9 Revolution and Gene Editing

In honor of Rodolphe Barrangou, Emmanuelle Charpentier,  Jennifer Doudna, Philippe Horvath, Viginijus Siksnys for remarkable contributions to the understanding of the CRISPR bacterial defense system and the revolutionary discovery that it can be adapted for genome editing.

Harvard Medical School
The Joseph B.Martin Conference Center
77 Avenue Louis Pasteur
Boston, MA 02115

RSVP: HMS_events@hms.harvard.edu
Seating available on a first-come first served basis

Opening Remarks

Barbara J McNeil, MD, PhD, Acting Dean, HMS

Warren Alpert a very prestiguos Prize for advancements in Medicine, Treatment of disease and alleviation of suffering

Clifford Tabin, MD, Prof. of Genetics at HMS

  • CRISPR as regulatory system

Featured Speakers include:

Rodolphe Barrangou, PhD
Todd R. Klaenhammer Distinguished Scholar in Probiotics Research
North Carolina State University
CRISPR-mediated immunity in bacteria: discovery and applications

and

Philippe Horvath, PhD
Senior Scientist
Dupont
CRISPR-mediated immunity in bacteria: discovery and applications

  • CRISPR-Cas: basics, history, applications, future
  • cas1 (larger number of spacers) and Cas 2 almost universal
  • DNA repeate in preKariotes, 2002 Milk coagulation, dairy industry – lactic acid, bacteriaphaging – failure of fermentation

History

  • 2005 outstanding spacer polymorphism
  • CRISPR genotype – phase sensitivity & resistance correlation
  • CRISPR – Mechanism of Action
  • 8/2005 Patent of bacteriaphage – Eureka:comercialization in 1990 200 sequence resistance to phage – Anti Phaging Hypothesis
  • certain spacers in genome cross immunity against the phage –
  • Spacers: engineeringCRISPR-encoded immunity resistence against phaging: ad spacer gain resistence
  • cas9 disruption >> loss of phage resistence in dairy bacteria
  • csn2 disruption -.. no subsequence acquisition of spacers
  • no phenotypic resistance loss
  • RNSi – A putative RNA-interference-based immune system
  • Science 2007
  • Discovery of the CRISPR motif (PAM): resistence in Streptococcus Thermophilus
  • Immunity is mediated by small CRISPR RNAs (crRNAs)
  • CRISPR Immunity – DNA encoded in bacteria
  • CRISPR/Cas bacterial immune system cleaves bacteriaphage and plasmid DNA, Nature 11/2010
  1. Immunization
  2. Interference with expression of immunity – with invading nucleus by viral DNA infection

Applications for CRISPR

  1. Bacterial strain typing
  2. natural vaccination against phages: CRISPerization (cultivation, plating)
  3. Natural genetc tagging
  • signature in the genome – genetic tag
  • strain identification
  • Patent for phage genome editing in 2009
  • Lethal self-targegting in bacteria programmable antimicrobial is death
  • genotype of interest selected
  • Agriculture applications: contamination in food, starters probiotics

Perspective on last Decade  

  • phage resistance phenotype
  • In silico & predictions in vitro
  • success in Crops, Food, Animals
  • Matters: IP (file for Patent, convert, publish), PR, Reg

Emmanuelle Charpentier, PhD
Prof. Dr.; Scientific Member of the Max Planck Society, Max Planck Director
Professor, Umeå University
The transformative genome engineering CRISPR-Cas9 technology: lessons learned from bacteria

  • Non-infectious Disease: Cancer, Heart Genetic, Brain
  • Infectious Diseases: Transmiable & Comnunicative
  1. Bacteria
  2. Viruses
  3. funcgi
  4. parasites
  • Enzyme Cas9 S. Pyogenes: Group A Strep
  • spacer acquisition – crRNA expression and maturationng CRISPR-CAS evolved into 6 types
  • Human Bacterial host
  • An mRNA : Type II CRISPR -Cas locus: TracrRNA – pre-crRNA
  • Cas9 requires tracrRNA:crRNA to cleave DNA
  • Genome editing with sequence specific nucleatease
  • RNA -programmable CRISPR -Cas9
  • Applications of CRISPR-Cas9 in human medicine: sequencing of Human genome – gene therapy to an organ, genetic predisposition of diseases
  • trcrRNA is associated to Type II CRISPR-Cas
  • Interchangeability among dual-RNA-Cas9 orthologs
  • Cpf1 – Type V-A
  • Adaptive Immune system
  • Mechnism of maturation of CRISPR-RNA

Jennifer Doudna, PhD
Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences/HHMI Investigator
University of California, Berkeley
The Future of Genome Engineering: Biology, Technology and Ethics

  • Biology
  • Technology  – Gene Editing
  • Ethics

BIOLOGY

  • Adaptation – acquire and maintain genetic memory Prokaryotic cells
  • crRNA Biogenesis
  • Interference
  • Supercoiled plasmid target helps for the integration reaction
  • Integration preceeds via a 3′-OH nucleophilic attack, 3′ – PO4
  • What directs Cas1-Cas2 to the leader side of CRISPR Loci
  • Integration Host Factor (IHF): alpha and beta
  • IHF is required for spacer acquisition in vivo
  • mechanism of spacer integration for DNA repair and repeat replication
  • Harness integrase for genomic tagging

TECHNOLOGY

  • CAS9 is a dual-RNA guided DNA Endonuclease
  • Cas9 programmed by single chimeric RNA
  • Most CISPR systems target dsDNA
  • PAM binding drives DNA target recognition: protospacer — PAM– dsDNA
  • C2c2 is an RNA-activated RNase: cis Cleavage vs trans Cleavage – used to detect specific RNA

INTERFERENCE

  • Chromatin search
  • DNA repair
  • RNA targeting

ETHICS

  • CRISPR based white mushrooms programmed to resist browning
  • human gene modification

 

Virginijus Siksnys, PhD
Professor and Chief Scientist/Department Head, Institute of Biotechnology
Vilnius University
From mechanisms of microbial immunity to novel genome editing tools

  • bacteria can absorb interference
  • superinfection survival
  • defense islands in genomes
  • CCGG-family: specificity of restriction enzymes that recognize different nucleotides
  • meganucleases: ZFN, TALEN
  • CRISPR-Cas are transportable: CRISPR3 was transferred to e-Coli and plasmid
  • isolate Cas9 protein – RNA-guided endonuclease – adaptive immunity in bacteria
  • generate Cas9 variants
  • Cas9 – restriction enzyme – targeting 2 sites on a pUC18 plasmid
  • Cas9 specificityis encoded by crRNA: REases
  • Cas9- versatilegenome editing tool: induce DNA breaks, gene editing of Human cells, animals plants
  • Cas target is composite – >1000 Cas9 orthologues are known: 20 nt protospacer PAM sequence PAM Assay: PAM depends on Cas9 concentration
  • RNP assembly Cas9
  • Type II-CCRISPR-Cas for B. laterosporus
  • PAM preference for Blat Cas9
  • Maze genome
  • Off-target cleavage: role of PAM, PAM contribute to cleavage at off-target site: Stringent PAM restriction on Cas9

 

 

Invited Speakers:

Luhan Yang, PhD
Chief Scientific Officer
eGenesis
Rewriting the pig genome to transform Xenotransplantation

  • Organ transplantation unmet needs
  • natural bioreactor for organ transplants manufacturing

Obstacles for Xenotransplantation

viral transmission

  • immunological Incompatibility
  • New tools: CRISPR-Cas9 multiplexible genome engineering
  • Infectivity of virus  – Infectivity is real: gRNA to destroy catalytic PERVs
  • Eridicate of PERVs activates in PK15 cells
  • generate viable PERV free embryo
  • Genotyping of Clone 40
  • viral transmission

immunological Incompatibility

  • a disruptive technology across tissue and organ types
  • therapeutic applications

FUTURE

  • write the Genome
  • Next Generation of Gene Editing Tools

 

Austin Burt, PhD
Professor of Evolutionary Genetics
Imperial College London
Developing CRISPR-based gene drive for malaria control

  • Genetically MODIFICATION of the mosquito strains that brings Malaria to Humans
  • Driving Y chromosom – convert all population of mosquitos to MALE: don’t bite, don’t transmit and do not contribute to next generation
  • Homing: natural process endonuclease genes in many microbes
  • Find Gene needed for female fertility: Ovary  expression : sterile non-sterile
  • gene needed for vector competence
  • target gene validation: number of Larvae
  • CRISPR-based homing at target gene – Frequency
  • Issues arising form this approach: Resistance, ecological and biodiversity, Governance and acceptance, step by step development pathway

 

Warren Alpert Foundation Prize Recipients

2016

For remarkable contributions to the understanding of the CRISPR bacterial defense system and the revolutionary discovery that it can be adapted for genome editing.

2015

For their pioneering discoveries in chemistry and parasitology, and personal commitments to translate these into effective chemotherapeutic and vaccine-based approaches to control malaria – their collective work will impact millions of lives globally particularly in the developing countries.

2014

For seminal contributions to our understanding of neurotransmission and neurodegeneration.

2013

For their seminal contributions to concepts and methods of creating a genetic map in the human, and of positional cloning, leading to the identification of thousands of human disease genes and ushering in the era of human genetics.

2012

For the discovery, preclinical and clinical development of bortezomib to FDA approval and front line therapy for the treatment of patients with multiple myeloma.

2011

In recognition of their extraordinary contributions to medicine and innovations in bioengineering.

2010

For the expansion and differentiation of human keratinocyte stem cells for permanent skin restoration in victims of extensive burns.

2008-2009

For the discovery, characterization and implementation of laser panretinal photo-coagulation, which is used to treat proliferative diabetic retinopathy.

2007

For work leading to the development of a vaccine against human papillomavirus.

2006

For their contribution to the development of the breast cancer therapy Herceptin, the first target-directed cancer treatment for solid tumors.

2005

For discovering angiogenesis and its relationship to disease, and for championing the concept of anti-angiogenic therapies.

2004

For her seminal contributions to the understanding of how the antitumor agent Taxol kills cancer cells.

2003

For their pioneering work on the purification, characterization, and cloning of human interferon-alpha.

2002

For his pioneering work in understanding the role of vitamin A supplementation in preventing blindness and life-threatening infections in children in the developing world.

2001

For their pioneering work in cardiovascular research which has dramatically reduced the mortality rate for heart attacks.

2000

For their research that contributed to the development of a drug that effectively treats chronic megelogenous leukemia and other forms of cancer.

1999

For their research in the development of statins which lower the level of cholesterol in the heart.

1998

For elucidating the pathway forming the leukotrienes and their role in bronchial asthma.

1997

For their discovery of human immune deficiency virus (HIV).

1996

For their discoveries of molecules that regulate the growth and differentiation of bone marrow cells in health and disease.

1995

For the development of the lung surfactant used for treating pulmonary hyaline membrane disease.

1994

For identifying Helicobacter pylori as the organism that causes gastric and duodenal ulcers.

1993

For developing a complete description of thalassemia at the molecular level.

1992

For discovering the enzymatic basis of Gaucher’s disease leading to its effective treatment.

1991

For designing a powerful new approach to the treatment of high blood pressure and congestive heart failure.

1989

For pioneering the use of DNA in the diagnosis of congenital anemias.

1988

For defining the genetic basis of muscular dystrophy.

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2016 Award Winners of the Lasker Foundation Awards in Physiology, Virology and Science Education

Reporter: Aviva Lev-Ari, PhD, RN

The Lasker Awards, among the most respected prizes in medicine, will go to six researchers who made major discoveries in physiology and virology, and to a scientist who has tirelessly promoted science education, the Albert and Mary Lasker Foundation announced on Tuesday.

The awards, honoring basic medical research, clinical research and special achievement, each come with a $250,000 prize and a nice omen: 87 Lasker laureates have also won Nobel Prizes.

 

2016 Award Winners

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