Emmanuelle Charpentier: revision 1 to 2

Revision 1

Revision 2

categories: ["people", "genetics"]

tags: ["crispr", "gene editing", "rna", "nobel prize", "microbiology", "bacteria"]

summary: "French microbiologist who identified the second RNA that CRISPR-Cas9 requires, co-authored the 2012 paper that made the system programmable, and shared the 2020 Nobel Prize in Chemistry."

summary: "French microbiologist who identified the second RNA that CRISPR-Cas9 requires, co-authored the 2012 paper that made the system programmable, and shared the 2020 Nobel Prize in Chemistry."

updated: "2026-07-28"

updated: "2026-10-29"

---

```infobox

{ "label": "Born", "value": "11 December 1968, Juvisy-sur-Orge, France" },

{ "label": "Nationality", "value": "French" },

{ "label": "Education", "value": "PhD, Université Pierre et Marie Curie, 1995" },

{ "label": "Known for", "value": "tracrRNA; programmable CRISPR–Cas9", "link": "/wiki/crispr-cas9" },

{ "label": "Known for", "value": "tracrRNA; programmable CRISPR-Cas9", "link": "/wiki/crispr-cas9" },

{ "label": "Field", "value": "Bacterial pathogens; RNA biology" },

{ "label": "Affiliation", "value": "Max Planck Unit for the Science of Pathogens, Berlin" },

{ "label": "Co-founded", "value": "CRISPR Therapeutics, 2013" },

Charpentier was born in Juvisy-sur-Orge, south of Paris, studied biochemistry and microbiology at the

Université Pierre et Marie Curie, and completed a doctorate in 1995 on molecular mechanisms of

antibiotic resistance, with the research carried out at the Institut Pasteur. She then spent six years

in the United States — at Rockefeller University working on *Streptococcus pneumoniae*, then at NYU

in the United States: at Rockefeller University working on *Streptococcus pneumoniae*, then at NYU

Medical Center, St. Jude Children's Research Hospital and the Skirball Institute.

She returned to Europe in 2002 and moved repeatedly: the University of Vienna and the Max F. Perutz

School from 2013. In 2015 she became a director at the Max Planck Institute for Infection Biology in

Berlin, and in 2018 the Max Planck Society established the Max Planck Unit for the Science of Pathogens

with her as founding director. Five countries and two decades of moves separate the doctorate from the

permanent position, and both of the papers the Nobel rests on were published while she was at Umeå.

permanent position. Both of the papers the Nobel rests on were published while she was at Umeå.

## Small RNAs in a streptococcus

The subject that runs through Charpentier's career is how *Streptococcus pyogenes* — the group A

The subject that runs through Charpentier's career is how *Streptococcus pyogenes* regulates itself

streptococcus, cause of scarlet fever, necrotizing fasciitis and a large burden of ordinary throat

with small non-coding RNA. The organism is the group A streptococcus, cause of scarlet fever,

infection — regulates itself with small non-coding RNA. Her group's early work described an RNA that

necrotizing fasciitis and a large burden of ordinary throat infection. Her group's early work described an RNA that

controls the synthesis of virulence factors in that organism, and the CRISPR result came out of the same

programme: a survey of the bacterium's small-RNA transcriptome, run with Jörg Vogel's group.

That survey turned up an abundant small RNA transcribed from beside the CRISPR locus. The 2011 paper

reporting it showed that this trans-encoded RNA — tracrRNA — base-pairs with the repeat portion of the

reporting it showed that this trans-encoded RNA, tracrRNA, base-pairs with the repeat portion of the

CRISPR precursor transcript, and that the resulting duplex is processed into mature CRISPR RNAs by the

host enzyme RNase III in the presence of the protein then called Csn1, now known as Cas9.[^deltcheva2011]

> [!key] Why the second RNA mattered

> The type I and type III systems studied at the time process their guides without a partner RNA. That

> The type I and type III systems studied at the time process their guides without a partner RNA, so

> the type II system needed one was not predictable from them, and nothing about Cas9 could be

> nothing about them predicted that the type II system would need one. Cas9 could not be

> reconstituted correctly without it. The single guide of 2012 is a fusion of the two RNAs this paper

> identified.

[[crispr-cas9]].

Virginijus Šikšnys's group in Vilnius reached an overlapping conclusion independently, showing that a

Cas9–crRNA complex from *Streptococcus thermophilus* cuts DNA at a sequence matching its guide. That

Cas9-crRNA complex from *Streptococcus thermophilus* cuts DNA at a sequence matching its guide. That

paper was received by its journal in May 2012 and published in September, after the *Science*

paper.[^gasiunas2012] The 2018 Kavli Prize in Nanoscience was awarded to Charpentier, Doudna and Šikšnys

together; the Nobel statutes permit three laureates and the 2020 chemistry prize named two.

> [!debate] Which experiment is the invention

> The 2012 work was biochemistry in a tube. Editing inside human cells was reported months later by

> other groups, among them one led by [[george-church]]. Whether the tube experiment already contains

> other groups, among them one led by [[george-church]]. Both the credit dispute and the patent

> the invention, or whether making it work in a eukaryotic nucleus was the inventive step, is the

> litigation turn on whether the tube experiment already contains the invention or whether making it

> question underneath both the credit dispute and the patent litigation, and it has no answer that both

> work in a eukaryotic nucleus was the inventive step. No answer is accepted by both sides.

> sides accept.

## Companies and translation

Charpentier co-founded CRISPR Therapeutics in 2013 with Rodger Novak and Shaun Foy, and ERS Genomics

the same year to license her patent rights outside human therapeutics. CRISPR Therapeutics and Vertex

Pharmaceuticals went on to develop the sickle cell and beta-thalassemia treatment approved in 2023 as

[[casgevy]], the first approved medicine using the system — an ex vivo cell therapy that requires

[[casgevy]], the first approved medicine using the system. It is an ex vivo cell therapy that

chemotherapy conditioning and a hospital stay, and whose price places it out of reach of most of the

requires chemotherapy conditioning and a hospital stay, and its price places it out of reach of most

patients the disease affects, as discussed under [[access-and-inequality]].

of the patients the disease affects, as discussed under [[access-and-inequality]].

The clinical record beyond that remains thin. Delivery, not editing chemistry, is the binding

The clinical record beyond that remains thin. The binding constraint is delivery rather than editing

constraint: in vivo work has concentrated on the liver, where [[lipid-nanoparticles]] reliably deposit

chemistry: in vivo work has concentrated on the liver, where [[lipid-nanoparticles]] reliably deposit

their cargo, and the [[crispr-off-target-effects|off-target and large-deletion problems]] created by

double-strand breaks have pushed much of the field towards [[base-editing]], [[prime-editing]] and

[[epigenome-editing]], which avoid cutting both strands.

the Broad's applications, covering use in eukaryotic cells, were granted first under an accelerated

route. The Patent Trial and Appeal Board ruled for the Broad on priority in 2022. In May 2025 the

Federal Circuit vacated that determination and sent it back, holding that the board had conflated the

legal standards for conceiving an invention and for reducing it to practice.[^fedcir2025] Nothing about

legal standards for conceiving an invention and for reducing it to practice.[^fedcir2025] The ruling

who invented what was decided by that ruling, which returned the question to the board rather than

decided nothing about who invented what; it returned the question to the board. The dispute has now

answering it. The dispute has now run more than a decade across two continents, and the American

run more than a decade across two continents, and the American position has been reversed on appeal

position has been reversed on appeal before; this article does not track its current posture, and a

before. This article does not track its current posture, and readers who need the present state of

reader who needs the present state of the American patents should check the docket rather than rely

the American patents should check the docket rather than rely on an encyclopedia entry.

on an encyclopedia entry.

The credit question is separate from the legal one and has its own history. A 2016 perspective essay in

*Cell* by Eric Lander, founding director of the Broad Institute, narrated CRISPR's development in a way

The 2020 prize was awarded "for the development of a method for genome editing" and was the first

science Nobel given to two women and no one else.[^nobel2020] Charpentier is the less publicly visible

of the two laureates and has stayed with the subject she started in: her Berlin unit is devoted to how

of the two laureates and has stayed with the subject she started in. Her Berlin unit studies how

bacterial pathogens regulate themselves, with CRISPR one system among several, rather than to genome

bacterial pathogens regulate themselves, with CRISPR one system among several, rather than genome

engineering or its applications.

That distance is itself part of the record the field argues over. CRISPR arrived from curiosity-driven

That distance is part of the record the field argues over. CRISPR came out of curiosity-driven

microbiology, not from a programme aimed at editing genomes, which is the case regularly made for

microbiology rather than a programme aimed at editing genomes, and it is regularly cited in the case

funding basic work whose applications cannot be named in advance. The counter-observation is that the

for funding basic work whose applications cannot be named in advance. Against that, the same tool

same tool has since made a set of governance problems urgent — [[germline-editing|heritable editing]]

has since made a set of governance problems urgent: [[germline-editing|heritable editing]] and its

and its one confirmed clinical use in the [[he-jiankui-affair]], [[gene-drive|self-propagating drives]]

one confirmed clinical use in the [[he-jiankui-affair]], [[gene-drive|self-propagating drives]] in

in wild populations, and the [[dual-use-research|dual-use]] questions that attach to cheap, precise

wild populations, and the [[dual-use-research|dual-use]] questions that attach to cheap, precise

sequence modification — that a laboratory studying streptococcal virulence had no reason to anticipate

sequence modification. A laboratory studying streptococcal virulence had no reason to anticipate

and no standing to settle. The institutions that must settle them are the subject of

these problems and no standing to settle them. The institutions that must settle them are the subject of

[[governance-of-genome-editing]].

## See also