CRISPR-based gene editing revolutionized medicine—what's next for the firm that helped develop it?
CRISPR-Cas 9 is a gene-editing tool that made it possible to rewrite any organism's genetic code and tackle genetic diseases more effectively.
Known as genetic scissors, CRISPR identifies a DNA sequence that is cut by an enzyme called Cas 9. It then changes or replaces that sequence with a different section of DNA.
For this discovery, co-inventors Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in Chemistry in 2020.
"By our interest in the lab to find new molecules that could have a role in in the bacterium streptococcus pardonus , we came across a very neat mechanism that allows to really recognize the virus that infects the bacterium in a very, very specific minor at the level of the genome of the virus. And we exploited this natural mechanism to develop the CRISPR-Cas9 technology," Emmanuelle Charpentier said in an interview with CNBC's The Edge.
In 2013, Charpentier co-founded CRISPR Therapeutics to fulfil her lifelong goal of finding cures for diseases.
A decade later, the company and its partner Vertex Pharmaceuticals developed CASGEVY, a therapy to treat blood disorders beta thalassemia and sickle cell disease.
"With CASGEVY, we're taking the bone marrow cells from the patient, making the edit for that particular patient and we're putting it back into the patient, and it reconstitutes the hematopoietic system of the patient. We're making a drug just for you," CRISPR Therapeutics' CEO Samarth Kulkarni told The Edge.
CASGEVY is a one-time therapy that costs $2.2 million per patient and can be administrated on patients 12 years of age and older. In 2023, it became the first CRISPR-based gene editing therapy to be approved by the Federal Drug Administration.
CRISPR Therapeutics currently has seven clinical and ten pre-clinical programs across oncology, autoimmune cardiovascular disease and diabetes, and is investigating next generation editing modalities.
Watch the video above for the full interview with Professor Charpentier from Berlin, Germany, and a tour of CRISPR Therapeutics' facilities in Boston, Massachusetts.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
20 hours ago
- Yahoo
DIA 2025 Global Annual Meeting Spotlights FDA Leadership with Opening Plenary, FDA Town Hall, and Global Regulatory Delegations
FDA Commissioner Dr. Martin A. Makary to join EMA, MHRA, and AAAS leadership for Opening Plenary; FDA Town Hall to feature moderated discussion with agency leadership on current regulatory priorities WASHINGTON, May 28, 2025--(BUSINESS WIRE)--DIA, a global non-profit organization of life sciences professionals, will convene the world's leading regulators, researchers, and innovators from nearly 50 countries at the DIA 2025 Global Annual Meeting, held June 15–19 in Washington. As the life sciences industry confronts rapid scientific advances and shifting global health policies, the Global Annual Meeting offers an opportunity for stakeholders to align in real time on regulation, innovation, and global access. This year's program will feature more than 200 sessions across 12 content tracks, including insights from 35 U.S. Food and Drug Administration (FDA) speakers and 103 confirmed participants from global regulatory agencies. The event's emphasis on transparency and cross-border collaboration reflects growing demand for unity amid fragmented global health systems. At the forefront of this year's agenda is FDA Commissioner Dr. Martin A. Makary, who will share insights on Monday, June 16, during the conference's Opening Plenary. Dr. Makary will be joined by Emer Cooke, Executive Director of the European Medicines Agency (EMA) and Chair of the International Coalition of Medicines Regulatory Authorities (ICMRA), who will be honored among the Fellows of DIA and recipients of the Global Inspire Awards; Lawrence Tallon, Chief Executive Officer of the UK's Medicines and Healthcare products Regulatory Agency (MHRA); and Dr. Sudip Parikh, Chief Executive Officer of the American Association for the Advancement of Science (AAAS). Keynote speakers Jimi Olaghere, a sickle cell disease advocate whose experience receiving CRISPR-based gene therapy marks a milestone in patient-led innovation, and Dr. Doug Melton, a distinguished research fellow at Vertex Pharmaceuticals known for his pioneering work in stem cell biology, will also participate in the Opening Plenary, addressing regulatory cooperation, public trust, and the path forward for global innovation. "Global progress depends on shared priorities, open access, and evidence-based policy," said Marwan Fathallah, DIA's President and Global Chief Executive. "DIA's Global Annual Meeting offers a rare opportunity to hear directly from regulators on the front lines of policy and innovation — and to confront the challenges affecting patients worldwide." Also on the agenda is the FDA Town Hall, a moderated forum that invites attendees to engage directly with FDA leadership on the agency's current regulatory priorities. The session, scheduled for Thursday, June 19, will offer attendees the opportunity to submit questions for open discussion. Starting the week with star power and inspiration, Grammy-nominated artist and songwriter Aloe Blacc is set to perform at both the DIA Inspire Awards and the Planet Word Networking Reception. Renowned physician and global health leader, best known for his pivotal role in the eradication of smallpox, Dr. Larry Brilliant will also speak at the Inspire Awards on June 15. Tickets for the Inspire Awards ceremony are available to the public for the first time and can be purchased here. You can also purchase tickets for the Planet Word Networking Reception here. To register for the DIA 2025 Global Annual Meeting or view the full event program, visit Members of the media may request credentials by emailing diaglobal@ About DIA DIA is a leading global non-profit life science membership association that drives collaboration in drug, device, and diagnostics development in pursuit of a healthier world. Founded in 1964 with headquarters in Washington, D.C., and offices in Europe and Asia, DIA provides unparalleled networking opportunities, educational resources, scientific research publications, and professional development programs to members in more than 80 countries. Learn more at and connect with DIA on LinkedIn, Facebook, X (Twitter), and Instagram. View source version on Contacts Media Contacts Katie KennedyGregory FCAdiaglobal@ 610-731-1045 Kali GillGregory FCAdiaglobal@ 610-731-4179 Sign in to access your portfolio
Scientific American
21 hours ago
- Scientific American
White House Budget Plan Would Devastate U.S. Space Science
Late last week the Trump Administration released its detailed budget request for fiscal year 2026 —a request that, if enacted, would be the equivalent of carpet-bombing the national scientific enterprise. 'This is a profound, generational threat to scientific leadership in the United States,' says Casey Dreier, chief of space policy at the Planetary Society, a science advocacy group. 'If implemented, it would fundamentally undermine and potentially devastate the most unique capabilities that the U.S. has built up over a half-century.' The Trump administration's proposal, which still needs to be approved by Congress, is sure to ignite fierce resistance from scientists and senators alike. Among other agencies, the budget deals staggering blows to NASA and the National Science Foundation (NSF), which together fund the majority of U.S. research in astronomy, astrophysics, planetary science, heliophysics and Earth science —all space-related sciences that have typically mustered hearty bipartisan support. On supporting science journalism If you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today. The NSF supports ground-based astronomy, including such facilities as the Nobel Prize–winning gravitational-wave detectors of the Laser Interferometer Gravitational-Wave Observatory (LIGO), globe-spanning arrays of radio telescopes, and cutting-edge observatories that stretch from Hawaii to the South Pole. The agency faces a lethal 57 percent reduction to its $9-billion budget, with deep cuts to every program except those in President Trump's priority areas, which include artificial intelligence and quantum information science. NASA, which funds space-based observatories, faces a 25 percent reduction, dropping the agency's $24.9-billion budget to $18.8 billion. The proposal beefs up efforts to send humans to the moon and to Mars, but the agency's Science Mission Directorate —home to Mars rovers, the Voyager interstellar probes, the James Webb Space Telescope (JWST), the Hubble Space Telescope, and much more —is looking at a nearly 50 percent reduction, with dozens of missions canceled, turned off or operating on a starvation diet. 'It's an end-game scenario for science at NASA,' says Joel Parriott, director of external affairs and public policy at the American Astronomical Society. 'It's not just the facilities. You're punching a generation-size hole, maybe a multigenerational hole, in the scientific and technical workforce. You don't just Cryovac these people and pull them out when the money comes back. People are going to move on.' Adding to the chaos, on Saturday President Trump announced that billionaire entrepreneur and private astronaut Jared Isaacman was no longer his pick for NASA administrator —just days before the Senate was set to confirm Isaacman's nomination. Initial reports —which have now been disputed —explained the president's decision as stemming from his discovery that Isaacman recently donated money to Democratic candidates. Regardless of the true reason, the decision leaves both NASA and the NSF, whose director abruptly resigned in April, with respective placeholder 'acting' leaders at the top. That leadership vacuum significantly weakens the agencies' ability to fight the proposed budget cuts and advocate for themselves. 'What's more inefficient than a rudderless agency without an empowered leadership?' Dreier asks. Actions versus Words During his second administration, President Trump has repeatedly celebrated U.S. leadership in space. When he nominated Isaacman last December, Trump noted 'NASA's mission of discovery and inspiration' and looked to a future of 'groundbreaking achievements in space science, technology and exploration.' More recently, while celebrating Hubble's 35th anniversary in April, Trump called the telescope 'a symbol of America's unmatched exploratory might' and declared that NASA would 'continue to lead the way in fueling the pursuit of space discovery and exploration.' The administration's budgetary actions speak louder than Trump's words, however. Instead of ushering in a new golden age of space exploration—or even setting up the U.S. to stay atop the podium—the president's budget 'narrows down what the cosmos is to moon and Mars and pretty much nothing else,' Dreier says. 'And the cosmos is a lot bigger, and there's a lot more to learn out there.' Dreier notes that when corrected for inflation, the overall NASA budget would be the lowest it's been since 1961. But in April of that year, the Soviet Union launched the first human into orbit, igniting a space race that swelled NASA's budget and led to the Apollo program putting American astronauts on the moon. Today China's rapid progress and enormous ambitions in space would make the moment ripe for a 21st-century version of this competition, with the U.S. generously funding its own efforts to maintain pole position. Instead the White House's budget would do the exact opposite. 'The seesaw is sort of unbalanced,' says Tony Beasley, director of the NSF-funded National Radio Astronomy Observatory (NRAO). 'On the one side, we're saying, 'Well, China's kicking our ass, and we need to do something about that.' But then we're not going to give any money to anything that might actually do that.' How NASA will achieve a crewed return to the moon and send astronauts to Mars—goals that the agency now considers part of 'winning the second space race'—while also maintaining its leadership in science is unclear. 'This is Russ Vought's budget,' Dreier says, referring to the director of the White House's Office of Management and Budget (OMB), an unelected bureaucrat who has been notorious for his efforts to reshape the U.S. government by weaponizing federal funding. 'This isn't even Trump's budget. Trump's budget would be good for space. This one undermines the president's own claims and ambitions when it comes to space.' 'Low Expectations' at the High Frontier Rumors began swirling about the demise of NASA science in April, when a leaked OMB document described some of the proposed cuts and cancellations. Those included both the beleaguered, bloated Mars Sample Return (MSR) program and the on-time, on-budget Nancy Grace Roman Space Telescope, the next astrophysics flagship mission. The top-line numbers in the more fleshed-out proposal are consistent with that document, and MSR would still be canceled. But Roman would be granted a stay of execution: rather than being zeroed out, it would be put on life support. 'It's a reprieve from outright termination, but it's still a cut for functionally no reason,' Dreier says. 'In some ways, [the budget] is slightly better than I was expecting. But I had very low expectations.' In the proposal, many of the deepest cuts would be made to NASA science, which would sink from $7.3 billion to $3.9 billion. Earth science missions focused on carbon monitoring and climate change, as well as programs aimed at education and workforce diversity, would be effectively erased by the cuts. But a slew of high-profile planetary science projects would suffer, too, with cancellations proposed for two future Venus missions, the Juno mission that is currently surveilling Jupiter, the New Horizons mission that flew by Pluto and two Mars orbiters. (The Dragonfly mission to Saturn's moon Titan would survive, as would the flagship Europa Clipper spacecraft, which launched last October.) NASA's international partnerships in planetary science fare poorly, too, as the budget rescinds the agency's involvement with multiple European-led projects, including a Venus mission and Mars rover. The proposal is even worse for NASA astrophysics—the study of our cosmic home—which 'really takes it to the chin,' Dreier says, with a roughly $1-billion drop to just $523 million. In the president's proposal, only three big astrophysics missions would survive: the soon-to-launch Roman and the already-operational Hubble and JWST. The rest of NASA's active astrophysics missions, which include the Chandra X-ray Observatory, the Fermi Gamma-Ray Space Telescope and the Transiting Exoplanet Survey Satellite (TESS), would be severely pared back or zeroed out. Additionally, the budget would nix NASA's contributions to large European missions, such as a future space-based gravitational-wave observatory. 'This is the most powerful fleet of missions in the history of the study of astrophysics from space,' says John O'Meara, chief scientist at the W. M. Keck Observatory in Hawaii and co-chair of a recent senior review panel that evaluated NASA's astrophysics missions. The report found that each reviewed mission 'continues to be capable of producing important, impactful science.' This fleet, O'Meara adds, is more than the sum of its parts, with much of its power emerging from synergies among multiple telescopes that study the cosmos in many different types, or wavelengths, of light. By hollowing out NASA's science to ruthlessly focus on crewed missions, the White House budget might be charitably viewed as seeking to rekindle a heroic age of spaceflight—with China's burgeoning space program as the new archrival. But even for these supposedly high-priority initiatives, the proposed funding levels appear too anemic and meager to give the U.S. any competitive edge. For example, the budget directs about $1 billion to new technology investments to support crewed Mars missions while conservative estimates have projected that such voyages would cost hundreds of billions of dollars more. 'It cedes U.S. leadership in space science at a time when other nations, particularly China, are increasing their ambitions,' Dreier says. 'It completely flies in the face of the president's own stated goals for American leadership in space.' Undermining the Foundation The NSF's situation , which one senior space scientist predicted would be 'diabolical' when the NASA numbers leaked back in April, is also unsurprisingly dire. Unlike NASA, which is focused on space science and exploration, the NSF's programs span the sweep of scientific disciplines, meaning that even small, isolated cuts—let alone the enormous ones that the budget has proposed—can have shockingly large effects on certain research domains. 'Across the different parts of the NSF, the programs that are upvoted are the president's strategic initiatives, but then everything else gets hit,' Beasley says. Several large-scale NSF-funded projects would escape more or less intact. Among these are the panoramic Vera C. Rubin Observatory, scheduled to unveil its first science images later this month, and the Atacama Large Millimeter/submillimeter Array (ALMA) radio telescope. The budget also moves the Giant Magellan Telescope, which would boast starlight-gathering mirrors totaling more than 25 meters across, into a final design phase. All three of those facilities take advantage of Chile's pristine dark skies. Other large NSF-funded projects that would survive include the proposed Next Generation Very Large Array of radio telescopes in New Mexico and several facilities at the South Pole, such as the IceCube Neutrino Observatory. If this budget is enacted, however, NSF officials anticipate only funding a measly 7 percent of research proposals overall rather than 25 percent; the number of graduate research fellowships awarded would be cleaved in half, and postdoctoral fellowships in the physical sciences would drop to zero. NRAO's Green Bank Observatory — home to the largest steerable single-dish radio telescope on the planet — would likely shut down. So would other, smaller observatories in Arizona and Chile. The Thirty Meter Telescope, a humongous, perennially embattled project with no clear site selection, would be canceled. And the budget proposes closing one of the two gravitational-wave detectors used by the LIGO collaboration—whose observations of colliding black holes earned the 2017 Nobel Prize in Physics—even though both detectors need to be online for LIGO's experiment to work. Even factoring in other operational detectors, such as Virgo in Europe and the Kamioka Gravitational Wave Detector (KAGRA) in Japan, shutting down half of LIGO would leave a gaping blind spot in humanity's gravitational-wave view of the heavens. 'The consequences of this budget are that key scientific priorities, on the ground and in space, will take at least a decade longer—or not be realized at all,' O'Meara says. 'The universe is telling its story at all wavelengths. It doesn't care what you build, but if you want to hear that story, you must build many things.' Dreier, Parriott and others are anticipating fierce battles on Capitol Hill. And already both Democratic and Republican legislators have issued statement signaling that they won't support the budget request as is. 'This sick joke of a budget is a nonstarter,' said Representative Zoe Lofgren of California, ranking member of the House Committee on Science, Space, and Technology, in a recent statement. And in an earlier statement, Senator Susan Collins of Maine, chair of the powerful Senate Committee on Appropriations, cautioned that 'the President's Budget Request is simply one step in the annual budget process.' The Trump administration has 'thrown a huge punch here, and there will be a certain back-reaction, and we'll end up in the middle somewhere,' Beasley says. 'The mistake you can make right now is to assume that this represents finalized decisions and the future—because it doesn't.'
Yahoo
a day ago
- Yahoo
CRISPR Market Research and Competitive Landscape Report 2025 - Global Forecast to 2030: Rising Adoption in Research and Therapy Drives Growth with Thermo Fisher Scientific and Merck Leading
Growth is fueled by increased adoption in research and industry, advancements in CRISPR tools, and rising investments in biotechnology. Key applications include drug discovery and gene therapy, with pharmaceutical and biotech companies dominating the market. North America leads the sector, driven by innovative research and strong investment. Amidst this expansion, companies like Thermo Fisher Scientific and Merck KGaA emerge as market leaders, shaping the competitive landscape with new developments and collaborations. CRISPR Market Dublin, June 04, 2025 (GLOBE NEWSWIRE) -- The "CRISPR Market by Offering [Product (Kits, Enzymes, Libraries), Service (gRNA Synthesis, Cell Line Development, Screening, Validation)], Application (Drug Discovery, Development, Agriculture) Competitive Landscape, Company Profiles -Global Forecast to 2030" has been added to offering. The global CRISPR market is set to expand from USD 3.21 billion in 2025 to USD 5.47 billion by 2030, registering a robust CAGR of 11.2% The report presents an analysis of market drivers, focusing on the rising demand for CRISPR-based therapies and ongoing technological advancements. It identifies key opportunities for CRISPR in areas such as vaccines and genetic disease treatments. A comprehensive evaluation of market shares and growth strategies of leading companies is included, along with an overview of challenges, particularly regulatory hurdles, that affect market progression. Growth is fueled by the technology's expanding use in research, clinical, and industrial applications, supported by increased investments in biotechnology R&D and the growing prevalence of genetic disorders. As CRISPR tools and delivery systems improve, and regulatory frameworks become more supportive, the integration of CRISPR into research and therapeutic pipelines accelerates. Leading companies include Thermo Fisher Scientific Inc., Merck KGaA, Agilent Technologies, GenScript, Takara Bio, Creative Biogene, Fujian SunyBiotech, Caribou Biosciences, Lonza Group, Danaher Corporation, among others. Products Dominated the Market in 2024 The CRISPR market is divided into products and services. The products segment, which includes CRISPR kits & enzymes and CRISPR libraries, leads the market. These products are essential in academic, clinical, and industrial research for genome editing and therapeutic development. This segment's growth is driven by the demand for standardized and innovative CRISPR products. Drug Discovery & Development: A Fast-Growing Application Segment Within applications, drug discovery & development is the largest and fastest-growing segment, leveraging CRISPR to find gene-disease links and streamline drug functions. This reduces timelines and increases success probabilities. Pharmaceutical and biotechnology firms are pivotal in incorporating CRISPR for personalized medicine and functional genomics, further driving the technology's adoption. Pharmaceutical & Biotechnology Companies' Market Dominance in 2024 These companies led the CRISPR products market in 2024, thanks to increased investment in cell and gene therapies, and the need for precise genetic tools. Their financial strength allows them to implement advanced CRISPR technologies effectively, bolstering their market position. North America's Leadership in the CRISPR Market North America, with its prominent research institutions and substantial biotech investments, captured the largest CRISPR market share in 2024. The region's emphasis on medical research and personalized medicine offers it a competitive edge. European markets, especially in Germany, the UK, and France, are also seeing robust growth due to heightened R&D activity. Key Attributes: Report Attribute Details No. of Pages 267 Forecast Period 2025 - 2030 Estimated Market Value (USD) in 2025 $3.21 Billion Forecasted Market Value (USD) by 2030 $5.47 Billion Compound Annual Growth Rate 11.2% Regions Covered Global Market Dynamics Drivers Growing Demand for Crispr-Based Gene Therapies Advancements in Crispr Technology Growing Government and Private Investments and Funding for Crispr Technology Restraints Technical Challenges Associated With Crispr Technology Opportunities Development of New Vaccines and Treatments for Genetic Diseases Expanding Industrial and Agricultural Applications of Crispr Challenges Regulatory Hurdles for Crispr Advancements Technology Analysis Key Technologies Crispr-Cas Systems Grna Design & Optimization High-Throughput Crispr Screening Complementary Technologies Synthetic Biology Single-Cell Genomics Next-Generation Sequencing Adjacent Technologies Stem Cell Engineering Synthetic Dna & Gene Synthesis Microfluidics Company Profiles Thermo Fisher Scientific Inc. Merck Kgaa Agilent Technologies, Inc. Genscript Revvity Lonza Danaher Corporation Takara Bio Inc. Creative Biogene Fujian Sunybiotech Co., Ltd. Editco Caribou Biosciences, Inc. Hera Biolabs Origene Technologies, Inc. Cellecta, Inc. Mammoth Biosciences, Inc. Applied Stemcell New England Biolabs Toolgen, Inc. Genecopoeia, Inc. Twist Bioscience Synthego Egenesis Inscripta, Inc. Precision Biosciences For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Attachment CRISPR Market CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
