ToplineThe Food and Drug Administration approved the use of Casgevy, a therapy from Vertex Pharmaceuticals and CRISPR Therapeutics that uses CRISPR gene-editing to treat the serious blood disorder transfusion-dependent beta thalassemia—marking the second major U.S. regulatory approval for the emerging gene-editing technology.FILE - This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and ... [+] Prevention shows crescent-shaped red blood cells from a sickle cell disease patien ...

- Approximately 1,000 patients in the U.S. 12 years of age and older are now eligible for this one-time treatment- ZUG, Switzerland and BOSTON, Jan. 16, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfu ...

Shares of CRISPR Therapeutics (CRSP -0.91%) climbed 54% in 2023, according to data provided by S&P Global Market Intelligence. The biotech company is developing a number of promising gene-therapy treatments for serious illnesses, but it doesn't currently have any products for sale on the market.CRISPR Therapeutics passed multiple important milestones last year, and the stock moved higher when that news was released. The company is navigating the tricky and expensive regulatory-approval process for its first ...

CRISPR Therapeutics (CRSP -0.91%) has put itself on the map of the healthcare industry as a serious player in gene editing. Now that it has an approved treatment in Casgevy, there's undoubtedly going to be more of a spotlight on the company.Gene-therapy treatments are growing in popularity, and for a larger healthcare company that is perhaps looking into getting into the business, it could be more attractive to simply buy CRISPR Therapeutics than to develop its own therapies.Here's why CRISPR could be an at ...

Cathie Wood has become a top investor, thanks to her investments in innovation. Wood identifies companies developing cutting-edge technologies with game-changing potential and gets in on them early. This way, her funds can benefit as these products or services take the world by storm. This helped the superstar investor's flagship fund, Ark Innovation ETF, soar 67% last year.Wood invests across various industries -- but you can find many of her favorite companies in the area of healthcare. From gene editing ...

Summary of CRISPR Therapeutics AG Conference Call Company Overview - **Company**: CRISPR Therapeutics AG (NASDAQ: CRSP) - **Event**: 42nd Annual J.P. Morgan Healthcare Conference - **Date**: January 9, 2024 - **CEO**: Dr. Sam Kulkarni Key Points Industry and Company Background - CRISPR Therapeutics is at the forefront of gene editing technology, specifically CRISPR Cas9, which has been revolutionary in biomedicine [2][6] - The company has diversified its portfolio into oncology, cardiovascular medicine, and diabetes, with several data catalysts expected in 2024 [3][4] Product Development and Pipeline - **CASGEVY**: The approval of CASGEVY for sickle cell disease and thalassemia is highlighted as a historic achievement, with a focus on expanding its addressable population through a targeted conditioning agent [6][7] - **In Vivo Approaches**: The company is advancing in vivo editing of hematopoietic stem cells (HSCs) and has received a $15 million Gates grant to support this work [8] - **2024 Priorities**: The company aims to develop three additional franchises: immuno-oncology, cardiovascular, and type 1 diabetes, with multiple clinical trials ongoing [9][10] Manufacturing and Operational Efficiency - CRISPR Therapeutics has established its own state-of-the-art manufacturing facility, which won the Facility of the Year Award, allowing for scalable production [5][19] - The company reports a significant reduction in cost of goods sold (COGS), down 4x from CTX110 to CTX112, enhancing its ability to bring therapies to market efficiently [19][20] Competitive Landscape and Future Outlook - The company is optimistic about the potential of allogeneic CAR-T therapies, particularly in autoimmune diseases, and is moving forward with urgency in this area [12][14] - The potential for solid tumors is emphasized, with CTX131 showing promising results in renal cell carcinoma [16][18] - The company is exploring partnerships to expand its capabilities and manage its extensive pipeline, with increasing interest from big pharma in cell and gene therapies [48] Financial Health and Growth Strategy - CRISPR Therapeutics has a strong balance sheet to support its growth initiatives, with five assets in the clinic and seven different clinical readouts expected in 2024 [29][28] - The company is focused on maintaining flexibility in its manufacturing strategy, balancing in-house capabilities with outsourcing for efficiency [50][51] Conclusion - CRISPR Therapeutics is positioned for significant growth in 2024, with a robust pipeline and innovative gene editing technologies that could transform treatment paradigms across multiple diseases [30][29]

CRISPR Therapeutics AG (NASDAQ:CRSP) 42nd Annual J.P. Morgan Healthcare Conference January 9, 2024 5:15 AM ET Company Participants Dr. Sam Kulkarni - Chief Executive Officer Conference Call Participants Wong Hooi - JPMorgan Wong Hooi I think we'll get started now. Thanks everyone for joining of CRISPR Therapeutics Presentation at Day 2 of JPMorgan's Healthcare Conference. I'm Wong Hooi from JPMorgan's healthcare investment banking team, and it's my pleasure to be introducing you to Dr. Sam Kulkarni, CEO of ...

Evgenii Kovalev/E+ via Getty ImagesInvestment Thesis The field of gene therapies and gene editing technology is the field of the future, but it is still in its infancy. CRISPR Therapeutics (NASDAQ:CRSP) has been a pioneer in this field, where scientists, for the first time ever, could be able to cure diseases and illnesses such as diabetes, hemophilia, cancer, and sickle cell disease. Diseases with genetic and systemic issues could possibly be cured with these advanced gene therapies. But, there have be ...

Betting on healthcare’s future via healthcare stocks is one of the safest long-term bets you can make. Though changes and advances happen rapidly, healthcare is one industry that is here to stay. It offers a unique investment opportunity at the intersection of innovation and demand.The best healthcare stocks to buy today aren’t necessarily the old, legacy pharma giants or even bulk medical device manufacturers. Though each sector has its strengths and weaknesses, many of the more mature healthcare stocks ha ...

Key PointsGene-editing is making headlines with the FDA approval of Casgevy, the first gene-editing treatment utilizing ex-vivo CRISPR/Cas9 for sickle cell disease (SCD), granted to Vertex Pharmaceuticals and CRISPR Therapeutics.Editas is focused on in-vivo treatment for SCD utilizing their EDIT-301 technology, which uses the proprietary enzyme CRISPR/Cas12 but also collects licensing fees for Cas9 from Vertex.Intellia’s co-founder, Jennifer Doudna, won the 2020 Nobel Prize for pioneering work in CRISPR tec ...