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Korea Herald
17-07-2025
- Business
- Korea Herald
SK bioscience Submits IND for Phase 1/2 Clinical Trial of Adjuvanted Influenza Vaccine Candidate
SEONGNAM, South Korea, July 17, 2025 /PRNewswire/ -- SK bioscience, a global innovative vaccine and biotech company committed to promoting human health from prevention to cure, today announced that the company has submitted an Investigational New Drug (IND) application to the Ministry of Food and Drug Safety (MFDS) for a Phase 1/2 clinical trials of a new influenza vaccine candidate, 'NBP607B'. The candidate incorporates an adjuvant into its existing cell-based influenza vaccine, 'SKYCellflu' to enhance protective efficacy. SK bioscience previously utilized adjuvants in its COVID-19 vaccine, 'SKYCovione', and now seeks to expand this technology to influenza vaccines as part of its broader platform strategy. NBP607B contains an adjuvant developed by the Vaccine Formulation Institute (VFI), a Swiss-based non-profit vaccine research organization. Comprising multiple immune-boosting components, the adjuvant is expected to induce strong immune responses and antibody production in elderly individuals. SK bioscience has proactively conducted non-clinical studies since 2023 and reported promising results. The Phase 1/2 clinical trial is scheduled to begin during the upcoming Northern Hemisphere flu season, enrolling approximately 320 older adults in Korea and abroad. The study will evaluate the vaccine's immunogenicity and safety compared to an approved high-immunogenicity flu vaccine, with interim results expected by 2027. This marks the first attempt by a Korean company to submit an IND to develop a high-immunogenicity influenza vaccine using an adjuvant. If successful, the company plans to leverage the platform for other vaccines and establish a competitive edge in the global high-value vaccine market. The development aligns with global health authorities' increasing recommendations for enhanced flu vaccines in high-risk groups. The U.S. Centers for Disease Control and Prevention (CDC)'s Advisory Committee on Immunization Practices (ACIP) recommends high-dose or adjuvanted influenza vaccines for adults aged 65 and older. The World Health Organization (WHO) also supports the use of adjuvanted vaccines for vulnerable populations. In Korea, the Korea Disease Control and Prevention Agency (KDCA) has indicated that domestically developed high-immunogenicity vaccines may be considered for inclusion in the National Immunization Program (NIP) if they meet appropriate criteria. According to market research firm Mordor Intelligence, the global vaccine market is projected to grow from USD 83.9 billion in 2025 to USD 114.8 billion in 2030, with an average annual growth rate of 6.5%. Demand for high-immunogenicity vaccines is expected to rise continuously due to global aging and the increasing number of immunocompromised individuals and those with chronic illnesses. SKYCellflu, SK bioscience's existing cell-based influenza vaccine, has already been recognized for its innovation. It became the world's first cell-based flu vaccine to receive prequalification (PQ) from the WHO and is currently approved in 11 countries, with supply through international procurement programs by United Nations Children's Fund (UNICEF) and Pan American Health Organization (PAHO). Jaeyong Ahn, CEO of SK bioscience, said, "We believe the combination of our proven SKYCellflu platform and our experience in adjuvanted vaccine development positions us well for success. We aim to establish a differentiated presence in the high-immunogenicity vaccine market while building a flexible platform for future infectious disease preparedness."
Yahoo
16-07-2025
- Business
- Yahoo
Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD Further Supporting Advancement of Novel Gene Editing Approach for the Treatment of Duchenne Muscular Dystrophy Towards Clinic
- New preclinical data for the PBGENE-DMD final clinical candidate demonstrates an increase in dystrophin positive muscle cells across key muscle types, potentially driven by editing of muscle satellite cells - - PBGENE-DMD is a first-in-class in vivo gene editing approach for up to 60% of Duchenne Muscular Dystrophy patients, specifically those impacted by dystrophin mutations in the 'hot spot' region between exons 45-55 - - Precision targeting to submit an Investigational New Drug (IND) and/or Clinical Trial Application (CTA) for PBGENE-DMD in 2025 with clinical data expected in 2026 - DURHAM, N.C., July 16, 2025--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the Company's first-in-class in vivo gene editing approach for Duchenne muscular dystrophy (DMD), towards first-in-human clinical investigation. "In a long-term durability study in a DMD diseased mouse model, we have observed up to a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius, up to 85% of cells were dystrophin-positive," said Cassie Gorsuch, PhD., Chief Scientific Officer at Precision BioSciences. "These new data build upon the preclinical data shared at ASGCT in May 2025, demonstrating that PBGENE-DMD treatment resulted in significant and sustained improvement of maximum force output at the same three- and nine-month timepoints. This broad increase in dystrophin-positive cells, along with the increased dystrophin protein detected in tissues, further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells, which were also observed in this study. We believe these results demonstrate the unique potential of the PBGENE-DMD gene editing approach to produce a sustained functional benefit without the need for AAV persistence, which is required of microdystrophin approaches. We look forward to presenting the complete dataset at a future scientific conference. Given the totality of preclinical evidence, we remain excited and steadfast in advancing this program towards clinic." Currently, there are no approved treatments or treatments in development that significantly improve muscle function over time to beneficially alter the long-term prognosis of DMD. PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those afflicted carry mutations in the 'hot spot' region between exons 45-55. Precision's approach is designed to permanently edit a patient's own DNA sequence, resulting in naturally produced, near full-length dystrophin protein known to be functional in humans. The final IND-enabling toxicology studies are currently underway with IND and/or CTA filing targeted in 2025 and initial clinical data expected in 2026. The Company believes that its current cash runway will be sufficient to progress both PBGENE-HBV, its current Phase 1 asset, and PBGENE-DMD through Phase 1 clinical readouts. About PBGENE-DMD PBGENE-DMD is Precision's development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near full-length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address up to 60% of the DMD patient population. In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body's ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement. About Precision BioSciences, Inc. Precision's two lead programs, PBGENE-HBV, for chronic Hepatitis B, and PBGENE-DMD, for Duchenne Muscular Dystrophy, are focused on areas with large patient populations with high unmet need. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline prioritizes in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners' and licensees' product candidates and gene editing approaches including PBGENE-DMD; the potential of PBGENE-DMD to drive meaningful improvement in functional and durable benefit over time for up to 60% of patients with DMD; the design on PBGENE-DMD to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein proven known to be functional in humans; the approach of using a single AAV to deliver PBGENE-DMD to excise exons 45-55 of the dystrophin gene with the aim of restoring a near-full length dystrophin protein within the body to improve functional outcomes; the increased dystrophin protein detected in the tissues and improved muscle function that was observed over time in the DMD mouse model may be attributable to edited satellite cells could be driving sustained replenishment of edited myocytes, resulting in increased dystrophin protein expression, dystrophin -positive cells, and improved muscle function over time; the expected timing of regulatory processes and clinical operations (including IND and/or CTA filings, studies, enrollment and clinical data for PBGENE-DMD; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "appear," "approach," "believe," , "confidence", "contemplate," "could," "design" "designed," "estimate," "expect," "goal," "intend," "look," "may," "mission," "plan," "possible," "potential," "predict," "project," "pursue," "should," "strive," "suggest," "target," "will," "would," or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. View source version on Contacts Investor and Media Contact: Naresh TannaVice President of Investor Sign in to access your portfolio
Yahoo
15-07-2025
- Business
- Yahoo
Rocket Pharmaceuticals Receives FDA IND Clearance for BAG3-DCM Gene Therapy RP-A701
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is one of the best low priced pharma stocks to buy now. On June 30, Rocket Pharmaceuticals announced that the US FDA cleared its Investigational New Drug/IND application for RP-A701. RP-A701 is a first-in-class AAVrh.74-based gene therapy candidate designed to treat BAG3-associated Dilated Cardiomyopathy (BAG3-DCM). BAG3-DCM is a rare and inherited heart condition caused by mutations in the BAG3 gene (Bcl2-associated athanogene 3). This leads to early-onset, rapidly progressing heart failure characterized by progressive ventricular enlargement and impaired systolic function, resulting in high morbidity and premature mortality. Scientific equipment in a lab setting, revealing the cutting edge of biotechnology research. A loss of BAG3 leads to the accumulation of misfolded and damaged proteins, impairing the heart's ability to contract. Rocket Pharmaceuticals estimates that ~30,000 individuals in the US are affected by BAG3-DCM. Current therapies don't prevent disease progression and are associated with complications. Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is a late-stage biotechnology company that develops gene therapies for rare diseases in the US. While we acknowledge the potential of RCKT as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Forbes
11-07-2025
- Business
- Forbes
The Hidden Power Of Questions In The Age Of AI
Bob Pearson , Chair, The Next Practices Group. getty When a child learns to speak, they pepper us with questions—an instinct rooted in survival. In 2013, a British study of 1,000 mothers found that children asked their parents more than 300 questions per day at an hourly rate that rivals the pace of the Prime Minister's Questions time. Questions help us navigate life and our roles within organizations—clarifying expectations, accelerating learning, building relationships and managing risk. To understand how questions help cut through complexity, consider Shannon's Theorem, which was created by Claude Shannon, the father of information theory. The theorem offers an equation for how much data can be sent across a communications channel in the presence of noise. Shannon was working at Bell Labs, so he was mainly focused on channels like a telephone line or a radio band. At organizations today, however, we still need to understand how to eliminate the noise that distracts us as we toil away on our projects. This is the role of questions: to help us focus. Understanding which questions to ask at a given time point helps reduce uncertainty, which is fundamental to how we utilize machine learning, decision trees and the field of data science. This ability to ask the right question—especially as AI becomes central—isn't just a technical skill but a foundational one. The Value Of Questions Within Organizations If we know which questions to ask at each key point for a given task, we can increase knowledge and reduce uncertainty. Relevant questions help us shape workflow, meet customer needs, teach teams and build trust. Of course, being human can also be our biggest obstacle. Too often, we stifle questions, prioritizing output over whether work is done optimally or scalably. So, what is the importance of questions within organizations, and why do we need to improve how we use them? To start, questions can lead to new information, frame a problem or check our own bias. Questions can just as easily unlock innovation as they work their magic to keep us on track, so we scale workflow efficiently. Imagine two scenarios. In the first, you are leading an SAP transformation project for a Forbes 2000 company that will exceed $300 million in cost and occur over three years. Your job is to make it happen on time and on budget. If you break down your project, you have 12 workstreams and 100 individual tasks per workstream. That is 1,200 different time points where you want to ensure your team understands what to do, how to handle unforeseen issues and accomplish each task. Email, teams and spreadsheets are not enough. Now, imagine your friend leads the development of a new drug in BioPharma. She said she has 60 key decision points in discovery/preclinical, 20 for an Investigational New Drug submission and 40 for Phase 1 trials. Getting a new drug into the clinic has 120 key decision points. In each case, we can proactively identify the top five questions that align with each decision. As your team gets ready for each decision point, they look at the five questions to ask themselves and their team. Did they address a certain problem? Did they categorize the expense associated with this action? Do they have a reason to believe this action could be improved? This process of structured questioning is incredibly powerful, but also time-consuming. That's where AI enters the picture. How AI Can Shape Question Asking The subject matter experts of the world are the heroes here. They have been there, done that and know what questions to ask at each point. AI can then supplement their knowledge to create a detailed list of questions for every decision point. As the user touches any point on the SAP transformation, the key questions to address will appear. Those questions will be linked to background information, and questions answered by other project members will become available, as they apply to that particular task. Now, questions are a quality check and a way to contribute to the enterprise workflow. It's a team sport. AI is ensuring that knowledge gained anywhere in the world is being shared precisely to the right time points in a project in real time. AI platforms can learn, in real time, what questions are most effective, what answers are most important and what type of backup information helps teach, educate and answer our questions. Questions and content can be translated into any language, enabling ideas to emerge from anywhere. To achieve this vision, we need to adjust a fundamental habit that drives us today. Ever since Google first set sail in 1998, we have been conditioned to write a few keywords or phrases, so our questioning ability is rusty. Now, with generative AI, we must flex our 'question muscles,' as we realize that the value of information we receive is dependent on our ability to ask the optimal question. This emerging skill—known as prompt engineering—is critical for tapping into AI's full potential. Keep in mind that our effective use of generative AI will help us mirror our own intelligence. Conclusion We start life as curious humans. We should never let that trait dissipate. Our job, with AI, is to remain as curious and disciplined as we were as toddlers. We are just now applying this approach to major global projects. A map of questions for each decision point. An AI back end to share answers and related content. The ability of any team member to contribute to the knowledge of the enterprise. Exploring the effective use of AI is more important than lamenting on what jobs will go away. What is required? Pretty simple. We need to change our habits and approach as we embrace the advances of AI. The 'curious companies' will complete that SAP transformation for $200 million and a year earlier, or they will create a more effective clinical trial design of a new treatment, based on a new style of learning and scaling. The question we are left with is, when do we make this a reality? Forbes Communications Council is an invitation-only community for executives in successful public relations, media strategy, creative and advertising agencies. Do I qualify?
Business Wire
10-07-2025
- Business
- Business Wire
Eolas Therapeutics Secures Full Development Rights to AZD4041
PALM BEACH GARDENS, Fla.--(BUSINESS WIRE)--Eolas Therapeutics today announced that it has entered into a new agreement with AstraZeneca to assume full development rights to AZD4041, an investigational therapy targeting the orexin-1 receptor, for the treatment of opioid use disorder (OUD) and other substance use disorders. Terms of the agreement are confidential. Eolas Therapeutics Takes the Lead on Promising Treatment for Opioid Addiction and other Drug Addictions. Share AZD4041 is a highly selective orexin-1 receptor antagonist that was being co-developed by Eolas and AstraZeneca with support from the National Institute on Drug Abuse (NIDA). The compound targets neural circuits that regulate craving, reward, and stress - key drivers of addiction. Preclinical and early clinical data support the potential of AZD4041 as a first-in-class, non-opioid therapeutic for substance use disorders, including opioid, stimulant, and alcohol use disorders. Under this agreement, Eolas will assume full responsibility for the program under the existing Investigational New Drug (IND) application and will advance AZD4041 into Phase II clinical trials with support from a NIDA UG3 cooperative agreement. "We are grateful to AstraZeneca for their collaboration and confidence in our ability to lead this program forward," said Albert Man, Chief Executive Officer of Eolas Therapeutics. "Substance use disorders remain among the most urgent and under-treated public health challenges. AZD4041 demonstrated promising preclinical data on addictive behaviors and has completed Phase 1 studies. Clinically significant drug-drug interactions are not anticipated within the range of doses which Eolas will test for efficacy. This compound represents a unique opportunity to develop a non-opioid therapeutic that we believe could significantly improve outcomes for people living with opioid dependence and other addictions." Eolas is committed to a rigorous, patient-centered development strategy to fully realize the therapeutic potential of AZD4041 across multiple indications. About Eolas Therapeutics, Inc. Eolas Therapeutics, Inc. is a clinical stage therapeutic development company committed to treating diseases of addiction and other neurological disorders. Our lead program, is a first-in-class compound to treat opioid-use disorder and other drug addictions. It is the first therapeutic to directly address the brain circuitry of addiction rather than the drug-signaling pathway. For more information, please visit
