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Presented by:
THE AGE OF
GENE EDITING
25th February 2016
Steffi Friedrichs
OECD Working Party on
Biotechnology, Nanotechno...
Outline – Gene Editing 101
2
• What is it? - Technological Advances
• Who is doing it? – the main Players
• What is it use...
What is Gene Editing?
3
• Gene/Genome Editing:
– High-precision manipulation of individual strands of DNA (i.e.
at the ‘by...
What is Gene Editing?
4
• Gene/Genome Editing:
– High-precision manipulation of individual strands of DNA (i.e.
at the ‘by...
How does Gene Editing work?
Example: CRISPR-Cas9
5
1. Sequencing of a genome
(i.e. the DNA-code is read) …
2. The gene edi...
The main Players in Gene Editing
6
… and their work:
Source: The Economist ‘Genome editing - The age of the red pen’, 22.0...
Applications of Gene Editing (1)
7
Current Applications:
• Health / Medicine:
– Fast, targeted breeding of mouse models (e...
Applications of Gene Editing (2)
8
(potential) Future Applications:
• Health / Medicine:
– Treatment of diseases (i.e. by ...
IP Issues arising from Gene Editing:
Patentability and Priority
9
Patent applications relating to CRISPR-Cas9:
– Northwest...
IP Issues arising from Gene Editing:
Patent Values and Economics
10
In August 2015, several high-profile investors,
includ...
Potential Safety Issues
11
• Off-target edits/mutations
• Unknown effects, such as:
– In agriculture: creation of poisons
...
Benefits arising from Gene Editing
12
• Fast eradication of malaria-carrying mosquitos (~ 3.2 billion people – nearly
half...
Regulatory Issues arising
from Gene Editing
13
• Since April 2015: EU is contemplating, if gene-edited plants should be
cl...
Ethical Issues arising from Gene Editing
14
• ‘Playing God’: gene-editing is fast and easy (i.e. readily available and
app...
THANK YOU!
Steffi Friedrichs
Biotechnology, Nanotechnology and Converging
Technologies
t: + (33-1) 85 55 60 27
e: steffi.F...
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The age of gene editing - Workshop on innovations in food and agriculture system: Policies to foster productive and sustainable solutions

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The workshop took place in Paris on 25-26 February 2016. Its central aim was to discuss with experts how scientific, technological, and farm practice innovation can improve productivity and sustainability in the food and agricultural sector, with a focus on international collaboration on gene editing techniques. It was introduced in the form of a presentation entitled ‘The Age of Gene editing’, produced by Steffi Friedrichs (STI), which played a pivotal role during the expert discussions.

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The age of gene editing - Workshop on innovations in food and agriculture system: Policies to foster productive and sustainable solutions

  1. 1. Presented by: THE AGE OF GENE EDITING 25th February 2016 Steffi Friedrichs OECD Working Party on Biotechnology, Nanotechnology and Converging Technologies (BNCT)
  2. 2. Outline – Gene Editing 101 2 • What is it? - Technological Advances • Who is doing it? – the main Players • What is it used for? – current (and future) Applications and Products • Why are we talking about it? – the Policy Issues surrounding Gene Editing: – IP issues (i.e. Patents, Open Science, etc.) – Risks and Benefits – Regulatory Issues (e.g. applicability of GM Regulation) – Public Acceptance
  3. 3. What is Gene Editing? 3 • Gene/Genome Editing: – High-precision manipulation of individual strands of DNA (i.e. at the ‘byte’-level of information storage in organic life) – Currently, three main techniques are used: Taken from: http://www.technologyreview.com/review/524451/genome-surgery/ “Collectively these technologies have created a scientific paradigm that envisions the genome as an infinitely editable piece of software.” (Gersbach, 2014).
  4. 4. What is Gene Editing? 4 • Gene/Genome Editing: – High-precision manipulation of individual strands of DNA (i.e. at the ‘byte’-level of information storage in organic life) – Currently, three main techniques are used: Taken from: http://www.technologyreview.com/review/524451/genome-surgery/ “Collectively these technologies have created a scientific paradigm that envisions the genome as an infinitely editable piece of software.” (Gersbach, 2014). See: Report of the Workshop on Environmental Risk Assessment of products derived from New Plant Breeding Techniques (February 2014), OECD Series on Harmonisation of Regulatory Oversight in Biotechnology, No. 61, ENV/JM/ MONO(2016)5. CRISPR: Clustered Regularly-Interspaced Short Palindromic Repeats
  5. 5. How does Gene Editing work? Example: CRISPR-Cas9 5 1. Sequencing of a genome (i.e. the DNA-code is read) … 2. The gene editing toolset is created (by adapting an existing defence mechanism used by bacteria): Target-DNA (i.e. DNA to be edited) Nuclease (i.e. enzyme that cuts DNA) Guide-RNA (attached to nuclease) 3. The Guide-RNA unzips the Target-DNA, and the Nuclease cuts the Target-DNA. Option 2: insertion of a new DNA strand Option 1: switching off the entire gene (‘KO’) Taken from: https://www.youtube.com/watch?v=2pp17E4E-O8 Youtube clip by Mc Govern Institute for Brain Research at MIT. non-GMO!? non- detectable
  6. 6. The main Players in Gene Editing 6 … and their work: Source: The Economist ‘Genome editing - The age of the red pen’, 22.08.2015.
  7. 7. Applications of Gene Editing (1) 7 Current Applications: • Health / Medicine: – Fast, targeted breeding of mouse models (e.g. 2 years shortened to 4 months) for advanced understanding of the roles of specific genomes and cures for genetic disorders, or for fertility studies with human embryo cells (cf. Francis Crick Institute, UK, February 2016) – Study of the gene-editing tool itself (i.e. safety issues, off-target effects, efficacy, etc.) (cf. China, April 2015) – Trials to eradicate malaria-transmitting mosquitos (by making them infertile / preventing mating / infecting them with bacteria / etc.) • Agriculture: – Fast, targeted breeding of plants with special resistances (e.g. drought resistance), or with special abilities (e.g. self-fertilization or self-pollination), or for special purposes (e.g. therapeutic applications) • Environment: – Studies to inhibit the spreading of invasive species (e.g. farmed salmon)
  8. 8. Applications of Gene Editing (2) 8 (potential) Future Applications: • Health / Medicine: – Treatment of diseases (i.e. by elimination of genetic mutations that cause a disease, or by modification of human somatic (i.e. non- reproductive) cells): HIV/Aids, haemophilia, sickle-cell-anaemia, some cancers, etc. – Elimination of heritable genetic diseases from a family line (i.e. by KO- or repair of the human germ line in embryonic cells) • Agriculture: – Fast, targeted and ‘cheap’ breeding of plants and animals with specific desirable attributes (cf. super-muscly pigs created by deleting a gene, which inhibits muscle growth, versus the Belgian Blue beef bred through traditional breeding techniques) Taken from: http://www.nature.com/news/super-muscly-pigs-created-by-small-genetic-tweak-1.17874 “Super-muscly pigs created by small genetic tweak.” (Nature 523 (2015) 13-14).
  9. 9. IP Issues arising from Gene Editing: Patentability and Priority 9 Patent applications relating to CRISPR-Cas9: – Northwestern University in September 2008 (Erik Sontheimer and Luciano Marraffini, 61/099,317); – Vilnius University in March 2012 (Virginijus Siksnys and others, 61/613,373); – UC Berkeley in May 2012 (Jennifer Doudna and others, 61/652,086); and – ToolGen in October 2012 (Jin Soo Kim and others, 61/717,324) – Broad Institute, MIT, in December 2012 (Feng Zhang and others, 8,697,359) Rejected by USPTO USPTO pending Rejected by USPTO Rejected by USPTO Granted by USPTO (April 2014) • 11th January 2016: declaration of interference between Doudna’s patent application and Zhang’s issued patents • 16th February 2016: Caribou Biosciences Inc., co-founded by Doudna, wins a patent on ‘Compositions and methods of nucleic acid-targeting nucleic acids’ (i.e. it does not mention Cas9)
  10. 10. IP Issues arising from Gene Editing: Patent Values and Economics 10 In August 2015, several high-profile investors, including the Bill & Melinda Gates Foundation and Google Ventures, pumped US$120 million into the genome-editing firm Editas Medicine of Cambridge, Massachusetts. Big Agriculture is following suit: DuPont forged an alliance with the genome-editing firm Caribou Biosciences in October, and announced its intention to use CRISPR–Cas9 technology to engineer crops. • 11th January 2016: declaration of interference between Doudna’s patent application and Zhang’s issued patents • 16th February 2016: Caribou Biosciences Inc., co-founded by Doudna, wins a patent on ‘Compositions and methods of nucleic acid-targeting nucleic acids’ (i.e. it does not mention Cas9) • Worries about ‘Evergreening’ of gene-editing patents In 2011, Caribou Biosciences Inc. raised USD$11 million for cell-engineering, drug screening and agricultural and industrial biotech.
  11. 11. Potential Safety Issues 11 • Off-target edits/mutations • Unknown effects, such as: – In agriculture: creation of poisons – In mammals: diseases/defects (e.g. gene tp53 expresses a tumor-suppressing protein (p53), which also causes premature ageing) Taken from: http://www.origene.com/CRISPR-CAS9/Product.aspx?SKU=KN200003
  12. 12. Benefits arising from Gene Editing 12 • Fast eradication of malaria-carrying mosquitos (~ 3.2 billion people – nearly half of the world's population – are at risk of malaria. ~ 438 000 malaria deaths (in 2015) … these numbers already represent a 60% decrease in mortality rates due to increased prevention). • Gene-editing studies provide insights into fertility: – 2/3 of human embryos fail to develop successfully, – every year, 7.9m children, 6% of total births worldwide, are born with a serious defects of genetic origin • Reduction of time (and cost) of traditional breeding techniques, while providing the same results (i.e. products are indistinguishable from those resulting from traditional breeding techniques). • Pharma companies hope to explore the function of every gene in the human genome. [1] Source: Financial Times (January 2016): http://www.ft.com/cms/s/0/9fd0529e-bb6a-11e5-b151-8e15c9a029fb.html#ixzz40tvGgvHr
  13. 13. Regulatory Issues arising from Gene Editing 13 • Since April 2015: EU is contemplating, if gene-edited plants should be classified as ‘genetically modified’ (i.e. GMOs)  The final verdict is expected towards the end of March 2016  Some gene-editing products have already been confirmed as non-GMO by several countries (i.e. US, Canada, Germany, Sweden, Argentina, …)  The PROBLEM: the gene-editing technique can be used in different ways, resulting in some products that are GMO and others that are not  The EU Food Safety Authority (EFSA) noted that all ‘non-natural’ plants would be classed as GM, but: “You can tell if a crop has been genetically modified, but it is impossible to tell if a plant has been subject to gene editing. It is closer to old-fashioned breeding techniques than it is to genetic modification technology.” (Professor Wendy Harwood, John-Innes Centre, UK)
  14. 14. Ethical Issues arising from Gene Editing 14 • ‘Playing God’: gene-editing is fast and easy (i.e. readily available and applicable toolkit) • The boundaries to ‘creating life’ are blurred: why stop at the insertion of naturally occurring genetic variants? Why not create/write new variants/DNA-code with synthetic biology? (e.g. programme cells to die, if they become cancerous) • The boundaries of curable diseases/defects and fixable disabilities are blurred; a new form of ‘eugenics’ might devalue the humanity of the disabled • The danger of over-hype: similar to stem-cell therapies, expectations might be too high and must be managed
  15. 15. THANK YOU! Steffi Friedrichs Biotechnology, Nanotechnology and Converging Technologies t: + (33-1) 85 55 60 27 e: steffi.FRIEDRICHS@oecd.org Skype: steffifriedrichs

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