CRISPR gene editing

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Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress
Globenewswire· 2025-10-09 15:01
Preclinical data demonstrates proof-of-concept for EDIT-401 with ≥90% mean LDL-C reduction in non-human primates and mouse modelsCAMBRIDGE, Mass., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today reported in vivo preclinical proof-of-concept data for EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (LDL-C), at the 32nd Annual European Society of Gene and Cell Therapy (ESGCT) Congress i ...
X @Cathie Wood
Cathie Wood· 2025-09-12 03:09
Thank you, @Shea_ARK, for surfacing the potential value of one of the most important biology breakthroughs of our time: CRISPR gene editing. Many analysts seem to think that one-time cures will not be a good business, @ARK Invest begs to differ! $CRSP, $NTLA, $BEAM, $PRMEARK Invest (@ARKInvest):Cardiovascular disease is the world’s leading killer. With CRISPR-based therapies targeting lipid metabolism genes, a one-time treatment could reshape prevention and open a ~$2.8T market. Read @Shea_ARK's analysis in ...
BBH Vs. IBB: How To Prepare For The Next Biotech Growth Cycle With BBH
Seeking Alpha· 2025-09-01 12:03
Industry Overview - The biotechnology industry is recognized as a rapidly growing sector within healthcare, driven by innovations such as AI that support drug research and CRISPR gene editing [1] Investment Insights - For investors looking to build positions in healthcare, investing in biotech companies presents significant opportunities [1]
Maravai LifeSciences(MRVI) - 2023 FY - Earnings Call Transcript
2023-11-16 15:00
Financial Data and Key Metrics Changes - The company reported a significant reliance on a single customer for the majority of its revenue last year, particularly related to the COVID vaccine, which has influenced its market narrative [11] - The nucleic acid production segment accounts for more than three-quarters of revenue, indicating a strong focus on this area moving forward [15] - The company maintains a net cash position of $580 million, which provides a solid financial foundation for future growth [19] Business Line Data and Key Metrics Changes - The nucleic acid production segment includes TriLink, Glenn Research, and AlphaZyme, which are critical for mRNA and other nucleic acid-based therapies [15] - The biologic safety testing segment, primarily through Cygnus Technologies, contributes about a quarter of revenue and is characterized by high margins [17] Market Data and Key Metrics Changes - The company is positioned in the mRNA and gene therapy markets, which are expected to see significant growth in the coming years, driven by advancements in programmable medicine [10][20] - The market for biologics is broad, with the company covering various cellular expression systems, which enhances its competitive positioning [18] Company Strategy and Development Direction - The company aims to focus on the discovery phase of drug development, seeking to be the first choice for customers in early-stage programs [7][8] - There is an emphasis on expanding capabilities through infrastructure investments made during the pandemic, which are expected to support future growth [13][20] - The company plans to continue its acquisition strategy to enhance its portfolio and drive both organic and inorganic growth [19] Management's Comments on Operating Environment and Future Outlook - Management acknowledges the current challenges in visibility for future orders, particularly in the COVID-related business, and is working closely with customers to understand their needs [26][28] - There is optimism regarding the potential announcement of a CRISPR medicine that could revitalize interest in the sector [34] - The long-term model anticipates a return to pre-pandemic growth rates, supported by a robust infrastructure capable of handling increased business without significant new investments [38] Other Important Information - The company has made cost adjustments to align its structure with the base business, reflecting a strategic response to current market conditions [28] - The CleanCap technology is expected to evolve from a COVID-specific application to broader respiratory vaccine applications [29] Q&A Session Summary Question: Update on non-COVID opportunities for CleanCap - The company plans to report on clinical trials and visibility in Q4, noting expected delays in programs due to funding concerns [22][24] Question: Revenue guidance for CleanCap in 2024 - The company is deferring guidance for 2024 due to a lack of visibility and is working with customers to understand their needs [26][28] Question: Incremental revenue from new facilities - The company is strategically opening new facilities to align with customer needs, with Flanders I expected to launch in Q2 [30][31] Question: Materiality of new partnerships - The partnerships are part of a broader strategy to engage customers early in the discovery process, which is expected to drive future development [32][33] Question: Long-term revenue targets and market indicators - The company is optimistic about returning to prior growth rates and expects to leverage its existing infrastructure to support this growth [34][36] Question: Capital allocation and stock buyback considerations - The company remains focused on M&A opportunities and maintaining a strong cash position for future investments [39]