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Swiss-American biotechnology company
CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. The company does business as CRISPR Therapeutics, Inc
CRISPR_Therapeutics
American biotechnology company
autogetemcel, a CRISPR-based gene therapy for treatment of sickle cell disease. CRISPR Therapeutics Editas Medicine Intellia Therapeutics Prime Medicine
Beam_Therapeutics
Gene editing method
CRISPR gene editing (/ˈkrɪspər/; pronounced like "crisper"; an abbreviation for "clustered regularly interspaced short palindromic repeats") is a genetic
CRISPR_gene_editing
American biotechnology company
Kyverna, and ONK Therapeutics. Intellia has two in vivo programs in ongoing clinical trials. NTLA-2001 is an investigational CRISPR therapy candidate
Intellia_Therapeutics
American biochemist and Nobel laureate (born 1964)
February 19, 1964) is an American biochemist who has pioneered work in CRISPR gene editing, and made other fundamental contributions in biochemistry and
Jennifer_Doudna
American biotechnology company
diagnostic tests using CRISPR-Cas12a and CRISPR-based therapies using its proprietary ultra-small CRISPR systems. Several CRISPR-Cas systems identified
Mammoth_Biosciences
American pharmaceutical company
Vertex announced it would acquire Exonics Therapeutics for up to $1 billion and collaborate with CRISPR Therapeutics, boosting its development of treatments
Vertex_Pharmaceuticals
Gene therapy
beta thalassemia. It was developed by Vertex Pharmaceuticals and Crispr Therapeutics. The most common side effects include low levels of platelets and
Exagamglogene_autotemcel
2018 scientific and bioethical controversy
trials of CRISPR-Cas9 for the treatment of genetic blood disorders was started in August 2018. The study was jointly conducted by CRISPR Therapeutics, a Swiss-based
He_Jiankui_affair
French microbiologist, biochemist and Nobel laureate (born 1968)
first generation of gene therapies. In 2013, Charpentier co-founded CRISPR Therapeutics and ERS Genomics along with Shaun Foy and Rodger Novak. In 2015,
Emmanuelle_Charpentier
Rare autosomal-recessive human disease
fibroblasts. PPL-001 (Papillon Therapeutics) is an ex vivo gene-edited cell therapy in preclinical development. CRISPR Therapeutics received a grant from FARA
Friedreich's_ataxia
PCSK9 inhibitor
VERVE-101 and VERVE-102 are an experimental gene therapy developed by Verve Therapeutics that targets the PCSK9 gene and is intended to reduce blood cholesterol
Verve PCSK9-inhibitor gene therapy
Verve_PCSK9-inhibitor_gene_therapy
Medical technology
Medicine, Intellia Therapeutics, CRISPR Therapeutics, Casebia, Cellectis, Precision Biosciences, bluebird bio, Excision BioTherapeutics, and Sangamo have
Gene_therapy
Biotechnology company
and developing antisense therapy, as well as RNA interference and CRISPR therapeutics. The company was founded in 1989 is based in Carlsbad, California
Ionis_Pharmaceuticals
Archived from the original on 30 June 2020. Retrieved 1 July 2020. "CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing
Timeline_of_biotechnology
Canadian biotechnology company
Acuitas Therapeutics Inc". European Biotechnology Magazine. Retrieved 2026-01-07. Park, Alice (2025-05-15). "A Baby Receives the First Customized CRISPR Treatment"
Acuitas_Therapeutics
Family of inherited blood disorders
from the original on 26 August 2022. Retrieved 26 August 2022. "CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™
Thalassemia
Swiss-American bioengineer and neuroscientist
Swiss-American bioengineer and neuroscientist whose research focuses on CRISPR, genome engineering, transcription and epigenetics, and Alzheimer's disease
Silvana_Konermann
American biologist
MudSKIPPER. Yeo’s work also extends to RNA-targeted therapeutics. His laboratory has explored the use of CRISPR-based tools to target RNA molecules, aiming to
Gene_Yeo
Hereditary blood disorder causing anemia
from the original on 26 August 2022. Retrieved 26 August 2022. "CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™
Beta_thalassemia
American professor of medicine
Matthew Porteus". CRISPR Therapeutics. Retrieved 23 June 2021. "There Are Now Two Pushes to Treat Sickle Cell Disease With CRISPR". CRISPR Medicine News.
Matthew_Porteus
American molecular biologist
Sontheimer co-founded Intellia Therapeutics, a biotechnology company focused on the clinical development of CRISPR-based gene-editing technologies.
Erik_J._Sontheimer
Drug candidate designation in the United States
(Press release). Retrieved 2021-07-01. Capricor Therapeutics Inc. (2019-09-24). "Capricor Therapeutics to Meet with FDA to Discuss CAP-1002 to Treat Duchenne
Regenerative medicine advanced therapy
Regenerative_medicine_advanced_therapy
Discovery-phase pharmaceutical company
biotechnology company which is developing therapies for rare diseases based on CRISPR gene editing technology. Editas headquarters is located in Cambridge, Massachusetts
Editas_Medicine
US non-profit biotechnology company
Woodbridge, Connecticut that is working to create novel therapeutics using gene therapy, gene editing (CRISPR technology) and antisense oligonucleotides to treat
Cure_Rare_Disease
Pharmaceutical Executive
Alyftrek. Under her tenure, and in collaboration with CRISPR Therapeutics, Vertex developed Casgevy, a CRISPR-based gene-editing therapy for sickle cell disease
Reshma_Kewalramani
American nonprofit scientific research institute
started forming to commercialize CRISPR in various ways, including Caribou Biosciences, Editas Medicine, and CRISPR Therapeutics. While Doudna was involved
Innovative_Genomics_Institute
2019 TV documentary series
overview of genetic engineering, particularly the DNA-editing technology of CRISPR, from the perspective of scientists, corporations and biohackers working
Unnatural Selection (TV series)
Unnatural_Selection_(TV_series)
American pathologist and molecular biologist
TAL effector, TALENs, and the RNA-guided CRISPR/Cas9 system. In addition to demonstrating the use of the CRISPR/Cas9 system in vivo through the zebrafish
J._Keith_Joung
Medical condition
2023. Retrieved 8 December 2023. "Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, Casgevy (exagamglogene
Hemoglobin_D
Group Philippe Drouet (Executive MBA), Chief Commercial Officer of CRISPR Therapeutics Que (Thanh) Dallara (MBA), Member of Executive Board, Executive Vice
List_of_INSEAD_alumni
Technologies to genetically improve human bodies
PMID 29091160. Chavez M, Chen X, Finn PB, Qi LS (January 2023). "Advances in CRISPR therapeutics". Nature Reviews. Nephrology. 19 (1): 9–22. doi:10.1038/s41581-022-00636-2
Human_genetic_enhancement
American immunologist
and in combination with BH3 mimetics. "Building up its I/O ops, CRISPR Therapeutics allies with Marcela Maus at Mass General". Endpoints News. Retrieved
Marcela_Maus
French American probiotics researcher
Board of Intellia Therapeutics. His research focuses on CRISPR-Cas9 in bacteria. In 2017, Barrangou was named Editor-in-Chief of The CRISPR Journal, a peer-reviewed
Rodolphe_Barrangou
CEO of biotechnology company
2019. In 2015 Leiden established a collaboration between Vertex and CRISPR Therapeutics that led to the discovery and development of Casgevy, (exagamglogene
Jeffrey_Leiden
American bioengineer and molecular biologist
is an American bioengineer, entrepreneur, and investor specializing in CRISPR, machine learning, synthetic biology, and gene therapy. He is an assistant
Patrick_Hsu
American biotechnology company
BioTherapeutics Has Secured $60M to Proceed with HIV Clinical Trial". Philadelphia. Retrieved 16 November 2023. Krieger, Lisa (28 October 2023). "CRISPR
Excision_BioTherapeutics
Type of genetic engineering
end joining (NHEJ), resulting in targeted mutations. The development of CRISPR gene editing in 2015 improved the efficiency, specificity, and practicality
Genome_editing
American gene therapy recipient (born 2024)
2024) is the first person to have received personalized gene therapy using CRISPR gene editing. Born with an extremely rare (one in a million) genetic condition
KJ_Muldoon
Treatment of an inherited blood disorder
2023. Retrieved 4 December 2023. "Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, Casgevy (exagamglogene
Management_of_thalassemia
Chinese–American biochemist (born 1981)
well known for his central role in the development of optogenetics and CRISPR technologies. In 2025, he was awarded National Medal of Technology and Innovation
Feng_Zhang
U.S. nonprofit organization
Ingelheim Bristol-Myers Squibb Camurus Catalyst Pharmaceuticals CRISPR Therapeutics CSL Behring Cytokinetics Daiichi Sankyo Editas Medicine Eli Lilly
National Organization for Rare Disorders
National_Organization_for_Rare_Disorders
American biotechnology company
billion in milestone payments. Beam Therapeutics Ledford, Heidi (19 May 2025). "World first: ultra-powerful CRISPR treatment trialled in a person". Nature
Prime_Medicine
Genetic modification technique
or CRISPRs. CRISPR-associated (cas) genes encode cellular machinery that cuts exogenous DNA into small fragments and inserts them into a CRISPR repeat
Gene_knockdown
Russian-American biologist
customized CRISPR treatment six months after birth for an infant born with a deadly genetic disease, CPS1 deficiency. He is a cofounder of Tune Therapeutics, an
Fyodor_Urnov
American biomedical research nonprofit
RNA" with potential advantages in simplicity and precision compared to CRISPR. The research was described in two papers published back-to-back in Nature
Arc_Institute
Israeli biotechnology company
Pluri Inc., formerly Pluristem Therapeutics, is an Israeli company engaged in the development of human placental adherent stromal cells for commercial
Pluri_Inc.
being developed by Intellia Therapeutics and was initially known by the investigational drug name NTLA-2002. Markman, Jon. "CRISPR Breakthrough Brings First
Lonvoguran_ziclumeran
American molecular biologist, biochemist, and organic chemist (born 1973)
with CRISPR nucleases for human therapeutics), Pairwise Plants (genome editing for agriculture), Beam Therapeutics (base editing for human therapeutics),
David_R._Liu
Genetically modified human embryo
involve directly editing the genome before birth, using technologies such as CRISPR. A controversial example of this can be seen in the 2018 case involving
Designer_baby
Overview of the events of 2020 in science
Sickle Cell Disease Is Thriving". NPR.org. Retrieved 1 July 2020. "CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing
April–June_2020_in_science
American cardiologist
diseases, and is a leading expert in genome-editing techniques, particularly CRISPR-Cas9. His lab was the first to develop an efficient technique to genetically
Kiran_Musunuru
Topics referred to by the same term
Cofactors Required for SP1 activation, e.g., CRSP3 The stock ticker for CRISPR Therapeutics This disambiguation page lists articles associated with the title
CRSP
Clinical phase American pharmaceutical company
Park), North Carolina, founded in 2015 as an NCSU spin‑out. It develops CRISPR-Cas3–enhanced bacteriophage therapies (“crPhage”) targeting drug-resistant
Locus_Biosciences
Type of mutation in a DNA sequence
initiating chain termination, upon encountering premature stop codons. CRISPR-Cas9 based single nucleotide substitutions have been used to generate amino
Nonsense_mutation
Existential threat from biological sources
covered risks including viral agents. Since then, new technologies like CRISPR and gene drives have been introduced. While the ability to deliberately
Biotechnology_risk
American scientist and life sciences industry leader
biopharmaceutical companies, including Beam Therapeutics, Hemab Therapeutics, Kymera Therapeutics, Rapport Therapeutics, and Takeda Pharmaceuticals. He is also
John_Maraganore
American developmental biologist
mice with multiple sclerosis. Tesar also identified CRISPR and antisense oligonucleotide therapeutics that restored myelination and extended the lifespan
Paul_J._Tesar
American physician-scientist known for work in gene therapy
Kriya Therapeutics". Kriya Therapeutics. April 8, 2024. Retrieved April 17, 2025. "Mark A. Kay, M.D., Ph.D. – Tenaya Therapeutics". Tenaya Therapeutics. Retrieved
Mark_A._Kay
Introduction of foreign genetic material into a host
FA, Cox D, et al. (February 2013). "Multiplex genome engineering using CRISPR/Cas systems". Science. 339 (6121): 819–23. doi:10.1126/science.1231143.
Transgenesis
American physician scientist and rapper
donations". Houston Chronicle. Retrieved September 4, 2024. "Vertex and CRISPR Therapeutics Announce US FDA Approval of Casgevy (exagamglogene autotemcel) for
Rayne_Rouce
American scientist who co-discovered mTOR
in human cells, most notably through the use of RNA interference and the CRISPR-Cas9 system. 2009 Paul Marks Prize for Cancer Research 2014 NAS Award in
David_M._Sabatini
American molecular biologist
cardiovascular therapeutics. His most recent work has provided a new strategy for correction of Duchenne muscular dystrophy using CRISPR gene editing.
Eric_N._Olson
with a focus on gene therapy, cell therapy, and RNA vaccine platforms. CRISPR-based technologies transitioned further from laboratory research into clinical
2025_in_biotechnology
American entrepreneur and philanthropist (born 1979)
T-cells engineered using the CRISPR gene-editing technology. The trial was the first in the United States to test CRISPR-modified cells in humans. The
Sean_Parker
Public medical school in Worcester, Massachusetts, US
is chaired by molecular biologist Phillip D. Zamore and vice-chaired by CRISPR biologist Erik J. Sontheimer. The RTI brings together basic scientists and
UMass_Chan_Medical_School
First cloned mammal (1996–2003)
biotechnology company PPL Therapeutics, based near Edinburgh. The funding for Dolly's cloning was provided by PPL Therapeutics and the Ministry of Agriculture
Dolly_(sheep)
President and Senior Investigator, Gladstone Institutes
stem cell expert Shinya Yamanaka and Jennifer Doudna, co-inventor of the CRISPR gene editing technology. He also added a number of early career scientists
Deepak_Srivastava
development of new therapeutics. He was named by Eric Lander in “The Heroes of CRISPR”3 as inspiring Broad Institute interest in developing CRISPR as a tool for
Michael_S._Gilmore
American immunologist
A; Gönen, M; Sadelain, M (2017). "Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection". Nature. 543 (7643): 113–117. Bibcode:2017Natur
Michel_Sadelain
Species of fungus
boulardii has also been used as a model organism in molecular biology, and the CRISPR–Cas9 genome-editing system has been demonstrated to function effectively
Saccharomyces_boulardii
American venture capital firm
Impulse Space Saildrone Terra Industries Kela Aera Therapeutics Benchling eGenesis Eikon Therapeutics No longer operating: Tempo Automation Entrepreneurship
Lux_Capital
American businessman
San Francisco. Retrieved May 27, 2026. Han, Ashley (July 17, 2025). "A CRISPR approach to neurodegenerative diseases". University of California. Retrieved
Brook_Byers
Process that prevents the expression of a gene
knockdown mechanism since the methods used to silence genes, such as RNAi, CRISPR, or siRNA, generally reduce the expression of a gene by at least 70% but
Gene_silencing
(CRISPR-Cas) system has been extensively studied to target a specific DNA sequence using a single guide RNA (sgRNA). For ATF applications the CRISPR-Cas
Artificial transcription factor
Artificial_transcription_factor
Mammalian protein found in humans
2023). "CRISPR gene editing shown to permanently lower high cholesterol". Ars Technica. Ars Technica. Retrieved 15 November 2023. "VERVE-101: CRISPR-Based
PCSK9
Medical condition
This procedure is high-risk. More recently, it has become possible to use CRISPR gene editing technology to modify the patient's own HSCs in a way that reduces
Sickle_cell_disease
Interdisciplinary branch of biology and engineering
2012: Charpentier and Doudna labs publish in Science the programming of CRISPR-Cas9 bacterial immunity for targeting DNA cleavage. This technology greatly
Synthetic_biology
Chinese neuroscientist
which autistic conditions in the brain could be reversed in mice using CRISPR gene editing. After mice were induced with MECP2 mutations, the MECP2 protein
Qiu_Zilong
Professor of Biostatistics and Computational Biology at Dana-Farber and Harvard
Institute and Harvard T.H. Chan School of Public Health. She co-founded GV20 Therapeutics and, in early 2022, left DFCI to become its full-time CEO. Xiaole Shirley
Xiaole_Shirley_Liu
professor in the school's department of radiation oncology. Sheltzer uses CRISPR/Cas9 technology to study aneuploidy and cancer genomics. Through the use
Jason_Sheltzer
American ophthalmologist and geneticist
Members" (PDF). Precision Medicine, CRISPR, and Genome Engineering – Moving from Association to Biology and Therapeutics | Stephen Tsang | Springer. "Recent
Stephen_Tsang
antagonist KNX-101 – 15-lipoxygenase (15-LOX; ALOX15) inhibitor LATER – CRISPR-dCas9 gene-editing PAX-01 – undefined mechanism of action Opiranserin -
List of investigational analgesics
List_of_investigational_analgesics
French biopharmaceutical company
company employed nearly 300 people in early 2014. With the advent of the CRISPR (clustered regularly interspaced short palindromic repeat) genome editing
Cellectis
in vitro detection of off-target genome editing events in DNA caused by CRISPR/Cas9 as well as other RNA-guided nucleases in living cells. Similar to LAM-PCR
GUIDE-Seq
American gene editing company
potential off-targeting. An ARCUS nuclease is also much smaller in size than CRISPR spCas9. It can use either adeno-associated virus (AAV) vectors or lipid
Precision_BioSciences
Drug delivery method
classes of lipid nanoparticles (LNPs) to enable targeted delivery of therapeutics to specific organs in the body. The SORT molecule alters tissue tropism
Selective_organ_targeting
by the Israel Innovation Authority to develop genome editing tools using CRISPR and artificial intelligence. Evogene states that its computational platform
Evogene
Israeli-American researcher
Lab". Ahituv Lab. Retrieved 2024-03-05. Matharu, Navneet (2018-12-13). "CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by
Nadav_Ahituv
American professor of medicine and bioengineering
mapping techniques, including synthetic-lethal interaction mapping with CRISPR/Cas9 (with Prashant Mali) and characterization of differential interactions
Trey_Ideker
Antimicrobial substance active against bacteria
(CRISPR). These viral DNA sequences enable the nuclease to target foreign (viral) rather than self (bacterial) DNA.Although the function of CRISPR-Cas9
Antibiotic
Artificial enzymes
can be used to precisely alter the genomes of higher organisms. Alongside CRISPR/Cas9 and TALEN, ZFN is a prominent tool in the field of genome editing.
Zinc-finger_nuclease
Biological process of gene regulation
miRNAs because the Dicer enzyme is not involved. It has been suggested that CRISPR interference systems in prokaryotes are analogous to eukaryotic RNAi systems
RNA_interference
Type of muscular dystrophy
Duchenne muscular dystrophy (DMD). Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in the
Duchenne_muscular_dystrophy
Organism altered by genetic engineering
host genome. Recent advancements using genome editing techniques, notably CRISPR, have made the production of GMOs much simpler. Herbert Boyer and Stanley
Genetically_modified_organism
American nanotechnologist (born 1968)
rapid results and has developed diagnostic tools that use DNA barcodes and CRISPR technology, making cancer diagnostics more affordable and accessible for
Sangeeta_Bhatia
Gene editing technique
methodologies similar to precursor genome editing technologies, including CRISPR/Cas9 and Base editing. Prime editing has been used on some animal models
Prime_editing
approaches for retinopathy. He and his collaborators applied a new generation of CRISPR technology called base editing and prime editing as a treatment for inherited
Krzysztof_Palczewski
Pathogen resistance to medications
across the cell surface. CRISPR-mediated resistance evolution Clustered regularly interspaced short palindromic repeats (CRISPR) and associated Cas proteins
Drug_resistance
British scientist
cholerae with VPI-1 site-specific recombination characteristics contains CRISPR-Cas and type VI secretion modules. (2016) Genomic Microbial Epidemiology
Ian_Charles
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
Boy/Male
Latin
Curly-haired.
Girl/Female
Scottish American Irish
Abbreviation of Christine. Follower of Christ.
Male
Portuguese
Portuguese form of Latin Christophorus, CRISTÓVÃO means "Christ-bearer."Â
Boy/Male
English Latin
Curly-haired. The 3rd century martyr St. Crispin is known as patron of shoemakers.
Male
Spanish
Spanish form of Latin Christophorus, CRISTÓBAL means "Christ-bearer."Â
Male
Romanian
Pet form of Romanian Cristian, CRISTI means "Christian."
Girl/Female
Scottish Irish
Abbreviation of Christine. Follower of Christ.
Girl/Female
Hindu, Indian, Mexican, Russian
Crisp; Calm; Reserved
Male
English
English name derived from Latin Crispinus, CRISPIN means "curly(-headed)."
Surname or Lastname
English
English : variant spelling of Crisp.
Boy/Male
Latin
Curly-haired.
Girl/Female
Scottish Irish
Abbreviation of Christine. Follower of Christ.
Surname or Lastname
English
English : nickname for a man with curly hair, from Middle English crisp, Old English crisp, cryps (Latin crispus), reinforced in Middle English by an Old French word also from Latin crispus.Americanized spelling of the German cognate Krisp, from Middle High German krisp, krispel ‘curly-haired man’.Americanized form of German Krisp, from a short form the medieval personal name Krispin (see Crispin).
Boy/Male
American, Australian, British, Christian, Dutch, English, French, German, Greek, Hebrew, Latin, Portuguese, Swedish
Curly-haired
Boy/Male
Indian, Latin
Curly Haired
Surname or Lastname
English
English : variant spelling of Christie.
Surname or Lastname
English
English : occupational name for a maker of pouches, from the plural of Middle English crippes ‘pouch’.English : metathesized form of Crisp.German : variant spelling of Krips, a variant of Krebs.
Boy/Male
Greek
Anointed.
Girl/Female
Spanish
Appointed one. A Christian.
Surname or Lastname
English and French
English and French : from the Middle English, Old French personal name Crispin, Latin Crispinus, a family name derived from crispus ‘curly-haired’ (see Crisp). This name was especially popular in France in the early Middle Ages, having been borne by a saint who was martyred at Soissons in ad c. 285 along with a companion, Crispinianus (whose name is a further derivative of the same word).English and French : diminutive of Crisp.
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS
CRISPR THERAPEUTICS