Successfully reported this slideshow.
We use your LinkedIn profile and activity data to personalize ads and to show you more relevant ads. You can change your ad preferences anytime.
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...
Upcoming SlideShare
Loading in …5
×

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

9,753 views

Published on

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.

Published in: Science
  • Be the first to comment

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

×