Aldevron and Integrated DNA Technologies Manufacture
FARGO, N.D. & CORALVILLE, Iowa--(BUSINESS WIRE)-- Aldevron, a global leader in the production of DNA, RNA and protein, together with Integrated DNA Technologies (IDT), a global leader in genomics solutions, announced the successful manufacture of the world's first personalized CRISPR gene editing drug product to treat an infant with urea cycle disorder (UCD). With no current cure for UCDs, the Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania (Penn) engaged Aldevron and IDT, both part of Danaher Corporation (NYSE: DHR) to manufacture a novel mRNA-based personalized CRISPR therapy in six months—three times faster than the standard timeline for gene editing drug products.
The technically complex, N of 1 therapy required a new guide RNA (gRNA) sequence, new mRNA-encoded base editor, custom off-target safety services and a clinically validated lipid nanoparticle (LNP) formulation, marking an industry milestone.
Share
The technically complex, N of 1 therapy required a new guide RNA (gRNA) sequence, new mRNA-encoded base editor, custom off-target safety services and a clinically validated lipid nanoparticle (LNP) formulation, marking an industry milestone that demonstrates how the U.S. continues to lead the way in mRNA gene editing therapies to improve human health for all. The outcome is featured in a study published today in The New England Journal of Medicine and provides proof of concept for the potential of safe, and effective, personalized CRISPR therapy in the future.
'We are unique in our ability to deliver this innovative treatment in such a short timeline,' said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. 'This CRISPR therapy was made under exceptional circumstances—not something our industry is built to do consistently—given the steadfast focus and dedication of the Aldevron and IDT teams to leverage years of expertise and strong partnerships to do what was needed to improve this patient's outcome. Collaboration between Aldevron, IDT, and Acuitas allowed for this innovation to happen, and the future of rare disease treatment is now brighter as a result.'
This accomplishment is complementary to the goal of the Danaher-IGI Beacon for CRISPR Cures to develop platform approaches that can be easily modified to develop gene-editing medicines for hundreds of devastating diseases. Launched in January 2024, the Beacon unites the finest scientific minds in gene editing at the IGI, with the R&D and manufacturing talent, technology and expertise from across Danaher's various operating companies, to create transformative solutions.
'What we've accomplished together sets a new gold standard for operationalizing the future of medicine,' said Sandy Ottensmann, VP/GM, Gene Writing & Editing at IDT. 'The implications of this work are profound and illuminate how collaborations between academic medicine and industry can enable major science wins. The opportunity ahead lies in continuously leveraging our CRISPR toolbox to innovate and operationalize more potential treatments and help patients in desperate search of cures.'
Aldevron provided the mRNA and worked with Acuitas Therapeutics, a private biotechnology company specializing in the development of LNP delivery systems for nucleic acid therapeutics. Together with the gRNA and safety services provided by IDT, the companies delivered a customized in vivo base-editing therapy, in a significantly compressed timeline, as a transformational therapy for the infant patient. Collaboration between all partners' quality and regulatory teams also led to successful EIND approvals.
'This study is an important milestone,' said corresponding author of the NEJM study Kiran Musunuru, MD, PhD, MPH, ML, MRA, the Barry J. Gertz Professor for Translational Research and Director of the Genetic and Epigenetic Origins of Disease Program in the Perelman School of Medicine at the University of Pennsylvania. 'The impact of this work extends beyond this particular patient and category of clinical indications—it suggests a potential roadmap for transforming CRISPR therapies for other inborn errors of metabolism and life-threatening genetic diseases. It's an exciting future for personalized medicine.'
The patient was treated at CHOP by co-corresponding author and physician-scientist, Rebecca C. Ahrens-Nicklas, MD, PhD. The infant, who was diagnosed with UCD, suffered from neonatal-onset CPS1 deficiency and was unable to remove ammonia from the body.
To learn more about Aldevron and IDT's capabilities, visit www.dhrls.co/aldevronidtworldsfirst.
About Aldevron
Aldevron is a premier manufacturing partner, producing high-quality plasmid DNA, mRNA, proteins, and other key components for the development of vaccines, gene and cell therapies, immunotherapies and other treatments. As a part of the Danaher Corporation family of global science and technology companies, Aldevron supports thousands of scientists who are developing revolutionary, lifesaving treatments for millions of people. To learn more about how Aldevron is advancing biological science, visit www.aldevron.com/about-us and follow the company on LinkedIn, Facebook and YouTube.
About IDT
Building from a strong foundation of innovation, expertise, and reliability, Integrated DNA Technologies (IDT) has evolved from an oligo manufacturer to a leading genomics provider. We work shoulder-to-shoulder with scientific and global health partners to enable genomics breakthroughs at scale. Our vision of enabling researchers to rapidly move from the lab to life-changing advances reflects our ongoing commitment to a healthier, brighter future for all.
IDT is proud to be part of Danaher, a global science and technology leader. Together we combine our capabilities to accelerate the real-life impact of tomorrow's science and technology to improve human health.
For more information about IDT, visit www.idtdna.com and follow the company on LinkedIn, X, Facebook, YouTube, and Instagram.
Disclaimer: CGMP refers to products manufactured under ICHQ7; IDT engineering runs and CGMP gRNA are for development and investigational use only. The performance characteristics of this product have not been established. This product is not intended to be used as final drug product. The purchaser is solely responsible for all decisions regarding the intended use of the product and any associated legal or regulatory obligations.
About Danaher
Danaher is a leading global life sciences and diagnostics innovator, committed to accelerating the power of science and technology to improve human health. Our businesses partner closely with customers to solve many of the most important health challenges impacting patients around the world. Danaher's advanced science and technology - and proven ability to innovate - help enable faster, more accurate diagnoses and help reduce the time and cost needed to sustainably discover, develop and deliver life-changing therapies. Focused on scientific excellence, innovation and continuous improvement, our approximately 63,000 associates worldwide help ensure that Danaher is improving quality of life for billions of people today, while setting the foundation for a healthier, more sustainable tomorrow. Explore more at www.danaher.com.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Associated Press
an hour ago
- Associated Press
Lead Plaintiff Deadline is June 16, 2025 for Investors of Ibotta, Inc.
NEW YORK, NY - June 8, 2025 ( NEWMEDIAWIRE ) - Kaplan Fox & Kilsheimer LLP announces that a class action lawsuit has been filed against Ibotta, Inc. ('Ibotta' or the 'Company') (NYSE: IBTA) on behalf of Ibotta investors. CLICK HERE TO JOIN THE CASE If you are an investor in Ibotta and have suffered losses, you may CLICK HERE to contact us. You may also contact Kaplan Fox by emailing [email protected] or by calling (646) 315-9003. DEADLINE REMINDER: If you are a member of the proposed Class, you may move the court no later than June 16, 2025 to serve as a lead plaintiff for the purported class. If you have losses we encourage you to contact us to learn more about the lead plaintiff process. You need not seek to become a lead plaintiff in order to share in any possible recovery. Ibotta purports to be a technology company that allows consumer packaged goods brands to deliver digital promotions to consumers through the Ibotta Performance Network. On April 18, 2024, Ibotta conducted its Initial Public Offering ('IPO'), offering 6,560,700 million shares of Class A common stock at a price of $88 per share. The complaint alleges that throughout the Class Period, Defendants made false and/or misleading statements and/or failed to disclose that; (i) Ibotta's data measurement system did not provide accurate, precise, and real time client campaign and consumer data measurement; (ii) the Company's business mix had shifted and was generating less revenue; and (iii) Ibotta had 'exhausted' its clients' budgets, negatively impacting fourth quarter 2024 revenue and expected first quarter 2025 revenue. According to the action, on February 26, 2025, after market hours, in connection with reporting fourth quarter 2024 and full year 2024 financial results, Ibotta's CEO Bryan W. Leach ('CEO Leach') explained just how deficient Ibotta's data measurement technology was by stating that 'it has become clear that we need to bring to market a more rigorous form of measurement that goes beyond the industry standard return on ad spend, or ROAS, framework.' Further CEO Leach allegedly announced that Ibotta would transform into a programmatic advertising company, which according to the complaint demonstrates that, at the time of the IPO, Ibotta's data measurement infrastructure was not suited for heavy reliance on third party platforms. On this news, the price of Ibotta's stock fell $29.08, or nearly 46%, to close at $34.09 on February 27, 2025, more than 60% lower than the IPO price of $88 per share. WHY CONTACT KAPLAN FOX - Kaplan Fox is a leading national law firm focusing on complex litigation with offices in New York, Oakland, Los Angeles, Chicago and New Jersey. With over 50 years of experience in securities litigation, Kaplan Fox offers the professional experience and track record that clients demand. Through prosecuting cases on the federal and state levels, Kaplan Fox has successfully shaped the law through winning many important decisions on behalf of our clients. For more information about Kaplan Fox & Kilsheimer LLP, you may visit our website at This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and ethical rules. If you have any questions about this Notice, your rights, or your interests, please contact: CONTACT: Pamela A. Mayer KAPLAN FOX & KILSHEIMER LLP 800 Third Avenue, 38th Floor New York, New York 10022 (646) 315-9003 [email protected] Laurence D. King KAPLAN FOX & KILSHEIMER LLP 1999 Harrison Street, Suite 1560 Oakland, California 94612 (415) 772-4704 [email protected] Contacting or submitting information to Kaplan Fox & Kilsheimer LLP does not create an attorney-client relationship, nor an obligation on the part of Kaplan Fox to retain you as a client. View the original release on
Yahoo
4 hours ago
- Yahoo
This Monster Growth Stock Is Up 167% in the Past Year and Disrupting the Healthcare Space
Hims & Hers is gaining market share in the telehealth sector and has a long runway to disrupt the healthcare industry. It is acquiring a company in order to enter Europe. The stock is expensive, but it may still be a great investment over the long term. 10 stocks we like better than Hims & Hers Health › Telehealth has gone through a major boom-and-bust cycle. One promising stock emerging from the bust is Hims & Hers (NYSE: HIMS). Through nifty marketing and an insurance-circumventing subscription model that delivers medicine directly to your front door, the company is taking a lot of share in the telehealth market. The stock has traded up 449% since going public, and it is up a staggering 158% in the past year. Disruptive innovation helped bring shareholders of Hims & Hers stock major gains. But does that make the stock a buy today? People in the United States get frustrated dealing with health insurance -- as you may know from personal experience. Hims & Hers aims to slowly disrupt the market with an innovative approach that bypasses insurers. It helps customers easily get generic medications that help deal with sexual health, hair loss, mental health, and other common concerns, by having prescriptions and shipments sent straight to their doors through monthly subscriptions. This model has helped Hims & Hers dominate the telehealth prescription market and reach $1.78 billion in trailing-12-month revenue. It's now trying to further expand its offerings by adding the branded weight loss drug Wegovy to its marketplace through a partnership with Novo Nordisk (NYSE: NVO). Previously, Hims & Hers sold weight loss drugs under an exemption because of supply shortages for the products, but with those shortages now resolved, it has to work with patent holders such as Novo Nordisk. Along with weight loss, it's also aiming to get into testosterone and menopause-related prescriptions. Today, Hims & Hers has 2.4 million active customers. Management believes there are over 100 million people who could utilize one of its products, giving the company a huge runway to grow. A key factor will be the new partnership for marketing Wegovy, which is an expensive subscription at an introductory discount offer of $549 a month. Usage of such drugs is growing like a weed, and could be a new growth avenue for Hims & Hers to pursue. Another huge step for Hims & Hers is international expansion. While countries vary in their approaches to healthcare and insurance, most people want easy-to-use products, affordable prices, and convenient at-home shipping regardless of where they live. Management hopes to supercharge international growth with its proposed buyout of competitor Zava in Europe. Zava serves the western European market with 1.3 million active customers in the United Kingdom, France, Germany, and Ireland. The combined company can utilize Hims & Hers' marketing expertise, increasing scale, and partnerships to bring this sought-after model to Europe. Global disruption of the healthcare space will give Hims & Hers an even larger runway for growth, while also allowing it to invest in new innovations -- including at-home patient testing and its own compounding manufacturing facility. Hims & Hers has grand ambitions to disrupt healthcare with its direct-to-consumer model, and Zava will give it even more scale to keep accelerating growth. It will be exciting to see what the combined company can do over the next decade. You can feel the excitement with Hims & Hers and its explosive revenue growth. Sales grew 111% year over year last quarter, and are expected to hit at least $2.3 billion in 2025. (They were just $100 million in 2020.) The company has a goal of reaching $6.5 billion in sales by 2030, which would make it one of the fastest-growing companies in the world this decade. This fast growth has created some high expectations for Hims & Hers stock. It now has a price-to-earnings ratio (P/E) of 79, which is a high trailing earnings multiple even for a fast-growing company. However, revenue is growing so quickly and with such high margins that the company may grow into this high valuation by the end of the decade. As noted, management has a goal of $6.5 billion in revenue in 2030. With 20% bottom-line profit margins -- easily doable with 77% gross profit margin over the last 12 months -- that would equate to roughly $1.3 billion in annual earnings in 2030. Today, the market cap is $12.3 billion, which would mean a P/E of just around 9.5 by 2030 if the market cap did not change (which is an unlikely scenario, but demonstrates that there is potential for the valuation to drop). Even with some shareholder dilution that raises the number of shares outstanding, the stock would be trading at a P/E of around 10 to 12 at the current share price (which is also likely to change). If you believe this rapid growth will continue over the long term, Hims & Hers stock will grow into its valuation. If you have any doubts about this pace of growth, shares should be considered overvalued at the moment. Before you buy stock in Hims & Hers Health, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Hims & Hers Health wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $674,395!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $858,011!* Now, it's worth noting Stock Advisor's total average return is 997% — a market-crushing outperformance compared to 172% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of June 2, 2025 Brett Schafer has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Hims & Hers Health. The Motley Fool recommends Novo Nordisk. The Motley Fool has a disclosure policy. This Monster Growth Stock Is Up 167% in the Past Year and Disrupting the Healthcare Space was originally published by The Motley Fool
Business Wire
17 hours ago
- Business Wire
ABL Investors Have Opportunity to Join Abacus Global Management, Inc. Fraud Investigation with the Schall Law Firm
LOS ANGELES--(BUSINESS WIRE)-- The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of Abacus Global Management, Inc. ('Abacus' or 'the Company') (NASDAQ: ABL) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. Abacus is the subject of a report issued by Morpheus Research on June 4, 2025, titled: 'Abacus Global Management: This $794 Million SPAC Is Yet Another Life Settlements Accounting Scheme Manufacturing Fake Revenue by Systematically Underestimating When People Will Die.' According to the report, the Company changed its portfolio valuation methodology to make it appear more profitable than it actually is. The report also alleges that the Company uses unusual methodologies to calculate life expectancy estimates and its co-founders have 'red flags' in their past. If you are a shareholder who suffered a loss, click here to participate. We also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach us through the firm's website at or by email at bschall@ The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation. This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.
