Our Programs – CRISPR
Posted: July 15, 2018 at 10:46 am
Gene Editing to Treat Disease
The majority of medical therapies available today are directed at managing disease processes, the pathogenic or mis-regulated proteins or molecules associated with disease. However, these pathogenic molecules themselves are typically encoded in or affected by changes in genes or other sequences in the human genome, which encompasses the DNA in all our cells. Gene editing technologies, including CRISPR/Cas9, now offer us the ability to directly modify or correct the underlying disease-associated changes in our genome. Successfully editing or correcting a gene that encodes the dysfunctional or missing protein can in principle result in the expression of a fully normal protein and full correction of the disease.
Gene therapy and other technologies to modify the genome have been in development for many years, and a small number of gene therapies have been approved to treat patients. However, these older approaches have been burdened by challenges to their safety and efficacy and have not yet provided the ability to precisely control a range of different genetic changes.
We believe that CRISPR/Cas9 offers just such an opportunity, particularly to correct DNA changes in somatic (non germ line) cells in patients with serious disease.
CRISPR/Cas9 is a rapid and easy to use gene editing technology that can selectively delete, modify or correct a disease causing abnormality in a specific DNA segment. CRISPR refers to Clustered Regularly Interspaced Short Palindromic Repeats occurring in the genome of certain bacteria, from which the system was discovered; Cas9 is a CRISPR-associated endonuclease (an enzyme), the molecular scissors that are easily programmed to cut and edit, or correct, disease-associated DNA in a patients cell. The location at which the Cas9 molecular scissors cut the DNA to be edited is specified by guide RNA, which is comprised of a crRNA component and a tracrRNA component, either individually or combined together as a single guide RNA (sgRNA). For example, a guide RNA can direct the molecular scissors to cut the DNA at the exact site of the mutation present in the genome of patients with a particular genetic disease. Once the molecular scissors make a cut in the DNA, additional cellular mechanisms and exogenously added DNA will use the cells own machinery and other elements to specifically repair the cut DNA.
There are more than 10,000 known single-gene (or monogenic) diseases, occurring in about 1 out of every 100 births1. Scientists and clinicians are now conducting pioneering research using CRISPR/Cas9 to address both recessive and dominant genetic defects, opening up the potential of gene editing to provide novel transformative gene-based medicines for patients with a large number of both rare and common diseases.
Dr. Emmanuelle Charpentier, one of CRISPR Therapeutics scientific founders, co-invented the CRISPR/Cas9 technology.
The clustered repeats of CRISPR were discovered in 1987 in bacteria2, but their function was unknown. In 2000, these clustered repeat elements were found to be relatively common in bacteria3 hinting to an important role of these elements. The clustered repeats were given the name CRISPR in 2002 and multiple CRISPR-associated (Cas) genes were discovered adjacent to the repeat elements in that same year4.
CRISPR/Cas9 is a rapid and easy to use gene editing technology that can selectively delete, modify or correct a disease causing abnormality in a specific DNA segment. CRISPR refers to Clustered Regularly Interspaced Short Palindromic Repeats occurring in the genome of certain bacteria, from which the system was discovered; Cas9 is a CRISPR-associated endonuclease (an enzyme), the molecular scissors that are easily programmed to cut and edit, or correct, disease-associated DNA in a patients cell. The location at which the Cas9 molecular scissors cut the DNA to be edited is specified by guide RNA, which is comprised of a crRNA component and a tracrRNA component, either individually or combined together as a single guide RNA (sgRNA). For example, a guide RNA can direct the molecular scissors to cut the DNA at the exact site of the mutation present in the genome of patients with a particular genetic disease. Once the molecular scissors make a cut in the DNA, additional cellular mechanisms and exogenously added DNA will use the cells own machinery and other elements to specifically repair the cut DNA.
There are more than 10,000 known single-gene (or monogenic) diseases, occurring in about 1 out of every 100 births1. Scientists and clinicians are now conducting pioneering research using CRISPR/Cas9 to address both recessive and dominant genetic defects, opening up the potential of gene editing to provide novel transformative gene-based medicines for patients with a large number of both rare and common diseases.
Dr. Emmanuelle Charpentier, one of CRISPR Therapeutics scientific founders, co-invented the CRISPR/Cas9 technology.
The clustered repeats of CRISPR were discovered in 1987 in bacteria2, but their function was unknown. In 2000, these clustered repeat elements were found to be relatively common in bacteria3 hinting to an important role of these elements. The clustered repeats were given the name CRISPR in 2002 and multiple CRISPR-associated (Cas) genes were discovered adjacent to the repeat elements in that same year4.
The function of the CRISPR-Cas system in bacteria as an immune defense mechanism was hypothesized by Mojica in 20055 and experimentally validated at the food ingredient company, Danisco, in 20076.
In 2011, Dr. Charpentiers lab discovered an essential component of the CRISPR-Cas system, tracrRNA, in bacteria7. The following year she and colleagues described how the Cas9 endonuclease works together with crRNA and tracrRNA to form functional molecular scissors to cut at a specific DNA sequence in the genome8. In this same publication the authors also described how to modify, or re-program, the system to direct the molecular scissors to cut at essentially any DNA sequence; how to modify the RNA components into a single guide RNA, simplifying the system into only 2 components; and how to modify the Cas9 molecular scissors to make nicks in the DNA by only cutting one of the two DNA strands. These foundational discoveries enabled transformative gene editing in a wide range of cells, tissues and species, including for the potential benefit of patients suffering from serious genetic diseases.
CRISPR/Cas9 is an easy, effective technology for gene editing that has enabled a wide range of new studies and transformed many areas of research. Thousands of academic laboratories across the world are carrying out research using the technology. Rapid adoption of CRISPR/Cas9 by the broader academic community and the collective efforts of their research are in turn driving tremendous progress in the field.
We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier. This IP is directed broadly to CRISPR/Cas9 genome editing and includes many different applications of the technology. We have filed additional IP and will continue to do so in support of our mission to bring transformative gene-based medicines to patients with serious diseases.
In 2011, Dr. Charpentiers lab discovered an essential component of the CRISPR-Cas system, tracrRNA, in bacteria7. The following year she and colleagues described how the Cas9 endonuclease works together with crRNA and tracrRNA to form functional molecular scissors to cut at a specific DNA sequence in the genome8. In this same publication the authors also described how to modify, or re-program, the system to direct the molecular scissors to cut at essentially any DNA sequence; how to modify the RNA components into a single guide RNA, simplifying the system into only 2 components; and how to modify the Cas9 molecular scissors to make nicks in the DNA by only cutting one of the two DNA strands. These foundational discoveries enabled transformative gene editing in a wide range of cells, tissues and species, including for the potential benefit of patients suffering from serious genetic diseases.
CRISPR/Cas9 is an easy, effective technology for gene editing that has enabled a wide range of new studies and transformed many areas of research. Thousands of academic laboratories across the world are carrying out research using the technology. Rapid adoption of CRISPR/Cas9 by the broader academic community and the collective efforts of their research are in turn driving tremendous progress in the field.
We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier. This IP is directed broadly to CRISPR/Cas9 genome editing and includes many different applications of the technology. We have filed additional IP and will continue to do so in support of our mission to bring transformative gene-based medicines to patients with serious diseases.
Read the rest here:
Our Programs - CRISPR
- What's the Latest in CRISPR Gene-Editing Technology? - Technology Networks - March 12th, 2024
- In vivo genome-wide CRISPR screening identifies CITED2 as a driver of prostate cancer bone metastasis | Oncogene - Nature.com - March 12th, 2024
- Investigating the mechanisms underlying resistance to chemoterapy and to CRISPR-Cas9 in cancer cell lines ... - Nature.com - March 12th, 2024
- Here's Why CRISPR Therapeutics Stock Climbed 34% in February - The Motley Fool - March 12th, 2024
- SXSW Panel Recap: The First CRISPR Foods Have Arrived - Austin Chronicle - March 12th, 2024
- CRISPR-Cas systems: Overview, innovations and applications in human ... - March 4th, 2024
- CRISPR Therapeutics Stock Has 32% Upside, According to 1 Wall Street Analyst - The Motley Fool - March 4th, 2024
- Missed Out on CRISPR Therapeutics? My Best Gene-Editing Stock to Buy and Hold - The Motley Fool - March 4th, 2024
- MEGA-CRISPR tool gives a power boost to cancer-fighting cells - Nature.com - February 23rd, 2024
- 3 Biotech Stocks to Buy That Have CRISPR-Like Breakthrough Potential - InvestorPlace - February 23rd, 2024
- CRISPR 'will provide cures for genetic diseases that were incurable before,' says renowned biochemist Virginijus iknys - Livescience.com - February 23rd, 2024
- Opinion: The Promise and Challenges of CRISPR-Based Treatments - BioSpace - February 23rd, 2024
- There's Reason For Concern Over CRISPR Therapeutics AG's (NASDAQ:CRSP) Massive 26% Price Jump - Simply Wall St - February 23rd, 2024
- Move over, CRISPR: RNA-editing therapies pick up steam - Nature.com - February 23rd, 2024
- If You Invested $10000 in CRISPR Therapeutics in 2019, This Is How Much You Would Have Today - The Motley Fool - February 23rd, 2024
- CRISPR Therapeutics Joins Rank Of Stocks With 95-Plus Composite Rating - Investor's Business Daily - February 23rd, 2024
- CRISPR Therapeutics (NASDAQ:CRSP) Hits New 12-Month High on Better-Than-Expected Earnings - AmericanBankingNEWS - February 23rd, 2024
- CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results - GlobeNewswire - February 23rd, 2024
- CRISPR Therapeutics AG (CRSP) Moves 6.9% Higher: Will This Strength Last? - Yahoo Finance - February 23rd, 2024
- Advancements in RNA for HIV Treatment: CRISPR Cas9, mRNA Therapeutics, and Next-Generation Sequencing ... - Medriva - February 23rd, 2024
- Intellia Therapeutics Charges Ahead: A Glimpse into the Future of CRISPR-Based Therapies - BNN Breaking - February 23rd, 2024
- The FDA Approved The First CRISPR-Based Therapy. What's Next? - Science Friday - February 5th, 2024
- Using CRISPR technology, researchers succeed in growing tomatoes that consume less water without compromising yield - Phys.org - February 5th, 2024
- CRISPR-Cas9 gene-editing tool repairs defective T cells to treat rare hereditary disease - News-Medical.Net - February 5th, 2024
- New CRISPR Technology Increases Recognition of Cancer Cells by the Immune System - Inside Precision Medicine - February 5th, 2024
- Is CRISPR Therapeutics a Buy in the New Bull Market? - The Motley Fool - February 5th, 2024
- Stocks Flashing Renewed Technical Strength: CRISPR Therapeutics - Investor's Business Daily - February 5th, 2024
- AI at Davos, new CRISPR therapies and health tech's bad marketing - Marketplace - January 20th, 2024
- FDA expands use of newly approved CRISPR therapy - Axios - January 20th, 2024
- CRISPR-based therapy receives expanded approval for beta thalassemia - STAT - January 20th, 2024
- CRISPR Therapeutics And Vertex's CRISPR Breakthrough: How And Why They Got There First - Scrip - January 20th, 2024
- Pharmalittle: We're reading about a CRISPR approval, selling meds directly to patients, and more - STAT - January 20th, 2024
- Here's Why CRISPR Therapeutics Stock Rose 54% Last Year - The Motley Fool - January 20th, 2024
- Vertex's CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease - MedCity News - January 20th, 2024
- Groundbreaking CRISPR/Cas9-based Genome Editing Therapy Secured the Second FDA Approval - geneonline - January 20th, 2024
- What Does It Mean for Investors if CRISPR Therapeutics Gets Bought Out in 2024? - The Motley Fool - January 20th, 2024
- First FDA-approved CRISPR-based gene therapy cleared for 2nd indication - LabPulse - January 20th, 2024
- CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY ... - GlobeNewswire - January 20th, 2024
- CRISPR Gene Editing And Its Role In Hematology | TheHealthSite.com - TheHealthSite - January 20th, 2024
- Doudna institute hatches plan to 'cure hundreds of diseases' left behind by CRISPR revolution - STAT - January 11th, 2024
- Discover the recent progress of nonviral delivery carriers for CRISPR/Cas9 systems - News-Medical.Net - January 11th, 2024
- How CRISPR could yield the next blockbuster crop - Nature.com - January 11th, 2024
- Weight-loss drugs, malaria vaccines and more: CRISPR innovations headline the science breakthroughs of 2023 - Genetic Literacy Project - January 11th, 2024
- CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook - GlobeNewswire - January 11th, 2024
- CRISPR Therapeutics AG (CRSP) 42nd Annual J.P. Morgan Healthcare Conference (Transcript) - Seeking Alpha - January 11th, 2024
- CRISPR to be used to genetically modify crops - FoodNavigator.com - January 11th, 2024
- Casgevy approval unlikely to be followed up by another CRISPR drug in near future - BioPharma-Reporter.com - January 11th, 2024
- Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY, for the Treatment of Sickle Cell ... - Business Wire - January 11th, 2024
- The Science Behind CRISPR: Germline Genome Editing and Its Applications - Medriva - January 11th, 2024
- Here's Why 2024 Could Be a Big Year for CRISPR Therapeutics - The Motley Fool - January 11th, 2024
- Revolutionizing acne treatment with CRISPR technology - Labiotech.eu - January 11th, 2024
- What Is CRISPR Gene Editing and How Does It Work? - December 25th, 2023
- This first CRISPR treatment is just the beginning. Heres what's next - Fast Company - December 25th, 2023
- The Age of Crispr Medicine Is Here - WIRED - December 25th, 2023
- 6 Words That Explain Why CRISPR Stock Isn't Soaring Despite the Recent FDA Approval for Its Gene-Editing Therapy - Yahoo Finance - December 25th, 2023
- Crispr Therapeutics Medical Chief Morrow to Resign - The Wall Street Journal - December 25th, 2023
- Crispr Therapeutics chief medical officer is resigning - MarketWatch - December 25th, 2023
- 3 Reasons to Buy CRISPR Therapeutics Stock Like There's No Tomorrow - Yahoo Finance - December 25th, 2023
- CAR T Therapy May Cause Rare Cancer & How CRISPR Could Be The Solution - Forbes - December 25th, 2023
- CRSP Stock Alert: CRISPR Therapeutics Is Losing Its Medical Chief - InvestorPlace - December 25th, 2023
- With the promise of saving millions of lives, CRISPR medicine is born - EL PAS USA - December 25th, 2023
- Casgevy: the world's first CRISPR therapy - Epigram - December 25th, 2023
- The Basics of CRISPR Gene Editing - Cleveland Clinic Health Essentials - November 27th, 2023
- Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing - November 27th, 2023
- What is CRISPR gene editing, and how does it work? - The Conversation - October 16th, 2023
- What is CRISPR/Cas9? - PMC - National Center for Biotechnology Information - October 16th, 2023
- CRISPR, 10 Years On: Learning to Rewrite the Code of Life - April 26th, 2023
- What Is CRISPR, and Why Is It So Important? - Scientific American - March 23rd, 2023
- Global CRISPR Technology Market Is Projected To Grow At A 22% Rate Through The Forecast Period - EIN News - March 14th, 2023
- What is CRISPR and why is it controversial? | CNN - February 2nd, 2023
- CRISPR | Description, Technology, Uses, & Ethical Concerns - February 2nd, 2023
- In vivo CRISPR screening reveals nutrient signaling processes ... - PubMed - December 12th, 2022
- What is CRISPR? | New Scientist - October 16th, 2022
- CRISPR-Cas9 Structures and Mechanisms - PubMed - October 16th, 2022
- A CRISPR cure for HIV? Gene-editing technology may be able stop viral replication in its tracks and wipe out infections - Genetic Literacy Project - October 16th, 2022
- Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene... - October 16th, 2022
- More Foods Will Be Gene-Edited Than You Think - The Epoch Times - October 16th, 2022
- What is CRISPR? - MD Anderson Cancer Center - September 21st, 2022
- CRISPR infusion eliminates swelling in those with rare genetic disease - Science - September 21st, 2022
- Crispr Therapeutics becomes the latest biotech to open in the Seaport - The Boston Globe - September 21st, 2022