Latest news with #NTCP
Business Upturn
5 days ago
- Business
- Business Upturn
ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025 Marks the first submitted Clinical Trial Application (CTA) advancing ProQR's Axiomer™ ADAR-mediated RNA editing platform into clinical development LEIDEN, Netherlands & CAMBRIDGE, Mass., June 26, 2025 (GLOBE NEWSWIRE) — ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells. AX-0810 is an investigational ADAR-mediated RNA editing oligonucleotide (EON) designed to selectively modulate NTCP function by reducing toxic bile acid accumulation in the liver, potentially mitigating inflammation, fibrosis, and progression toward liver failure, which are common in cholestatic diseases. 'The submission of this CTA marks a significant milestone for ProQR, representing the first to advance our Axiomer RNA editing platform and pipeline into clinical development,' said Cristina Lopez Lopez, MD, PhD Chief Medical Officer of ProQR. 'This step brings us into the clinic with AX-0810 as we initiate a Phase 1 study to evaluate safety and target engagement in healthy volunteers. Cholestatic liver diseases represent an area of high unmet medical need and by targeting NTCP, AX-0810 introduces a novel therapeutic approach underpinned by human genetics and compelling preclinical data.' The proposed Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810 in healthy adult volunteers. The study will also explore biomarkers to assess early signals of target engagement. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025. About AX-0810 AX-0810 is an investigational GalNac-delivered RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP. These diseases are driven by toxic bile acid accumulation due to impaired bile flow, leading to progressive liver damage. AX-0810 offers a novel therapeutic approach by selectively modulating NTCP function, which decreases bile acid reuptake into the liver. This mechanism is supported by human genetics data showing that naturally occurring NTCP variants safety reduce bile acid reuptake, reinforcing the potential of NTCP modulation by ADAR-mediated RNA editing to improve liver health and alter the course of cholestatic disease. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer 'Editing Oligonucleotides', or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell's own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at Forward Looking Statements for ProQR This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'continue,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look forward to', 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'will,' 'would' and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, ; our Axiomer™ RNA editing technology platform, including the continued development and advancement of our Axiomer platform, the advancement of AX-0810 to clinical trials, including the timing of, and our ability to obtain regulatory clearance; our expectations regarding the initiation of the planned Phase 1 clinical study of AX-0810 in the Netherlands, including our ability to recruit for and complete the study, and the anticipated timing of initial data in Q4 2025; the plan, timing, progress and results of our preclinical studies, planned clinical trials and other development activities, including the release of data related thereto, and the potential of our technologies and product candidates. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228 [email protected] or Investor contact: Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 [email protected]
The Hindu
6 days ago
- Health
- The Hindu
U.T. bags national award for tobacco control measures
Puducherry has bagged the 'Best Performing UT Award' in the Tobacco-Free Youth campaign 2.0, a national recognition conferred by the Union Ministry of Health and Family Welfare. The award acknowledges Puducherry's efforts in implementing tobacco control initiatives under the National Tobacco Control Programme (NTCP) during the year 2024 25, an official release said. The Union Territory was lauded for its robust enforcement of anti-tobacco laws, particularly the yellow line campaigns aimed at preventing the sale of tobacco products near educational institutions. The administration's measure to declare numerous institutions as tobacco-free zones and conduct intensive enforcement drives in collaboration with the police department also came in for commendation, the press note added. According to the release, the highlights of the campaign in Puducherry also included the establishment of 20 new tobacco cessation centres across medical and dental colleges and primary and community health centres. Awareness programmes were also carried out across schools, colleges, and communities. The Department of Health and Family Welfare Services appreciated the sustained efforts of the District Collectorate, Police department, Education department, Social Welfare department, medical and dental colleges, field-level health workers, community health volunteers, and partner organisations for helping achieve the award.
Time of India
6 days ago
- Health
- Time of India
India's Health ministry gets Bloomberg Philanthropies Award for Global Tobacco Control
New Delhi: India's Ministry of Health and Family Welfare was honoured with the prestigious Bloomberg Philanthropies Award for Global Tobacco Control in the 'O' category, which recognises efforts in offering help to quit tobacco use under a policy package of the World Health Organisation (WHO). Given under the MPOWER strategy, the award was presented by Michael R Bloomberg during a ceremony announced late night on June 23 (local time) at the World Conference on Tobacco Control in Dublin, Ireland. The award celebrates India's exemplary efforts in providing cessation support to millions of tobacco users. Alongside India, governments and NGOs from Mauritius, Mexico, Montenegro, the Philippines, and Ukraine were also recognised for their contributions to curbing tobacco use. "The achievements recognised today reflect what's possible when governments and civil society act decisively to protect health. These awardees have not only implemented proven tobacco control measures; they've also done so under complex circumstances including conflict and industry interference," said Dr Tedros Adhanom Ghebreyesus, WHO Director General. Bloomberg, founder of Bloomberg Philanthropies, who presented the award said, "We commend their leadership, their commitment to evidence-based policies, and their contribution to saving millions of lives. "The global tobacco control movement has helped save tens of millions of lives, making it one of the most successful public health efforts in history." He is also the WHO Global Ambassador for Noncommunicable Diseases and Injuries, and the mayor of New York City. "Bloomberg Philanthropies has long worked closely with the World Health Organisation and others who are leading this vitally important work, and the winners of this year's Awards for Global Tobacco Control prove how much progress is possible," he said. India's recognition highlights the Modi government's approach through the National Tobacco Control Programme (NTCP). The NTCP has established more than 675 tobacco cessation centres across district hospitals, delivering 1.35 million counselling sessions in financial year 2023-24. The National Tobacco Quitline Services (NTQLS), launched in 2016, supports 16 Indian languages with around 100 counsellors, handling 42,000 calls monthly. The mandatory display of "QUIT TODAY, CALL 1800-11-2356" on tobacco packs, as per the Cigarettes and Other Tobacco Products Act (COTPA) 2003, has boosted call volumes by more than 30 per cent. "India has been making consistent efforts to help people quit tobacco through the NTCP," said Dr Pratima Murthy, director and senior professor of psychiatry, National Institute of Mental Health and Neuro Sciences in India. "This includes the establishment of tobacco cessation clinics, screening and intervention in primary care at the Ayushman Arogya Mandirs, and establishing Cessation and National Tobacco Quitlines. "These comprehensive efforts indicate India's commitment to reduce tobacco use and promote public health. It is an absolute honour to receive on behalf of the Indian government the Bloomberg Philanthropies Award for Global Tobacco Control in the 'O' category," Dr Murthy added. India's leadership includes banning e-cigarettes under the Prohibition of Electronic Cigarettes Act (PECA) 2019, setting a global standard, and mandating anti-tobacco warnings on OTT streaming platforms to protect the youth. Other award recipients include Mauritius for plain packaging, Mexico for its advertising ban, Montenegro for tax increases, the Philippines for surveillance partnerships, and Ukraine for smoke-free policies. During the ceremony, Michael R Bloomberg announced a 20 million dollars initiative to advance tobacco control in low- and middle-income countries, supporting governments and NGOs and bolstering India's NTCP efforts. This builds on the Bloomberg Initiative's success, saving 35.2 million lives and reducing cigarette sales by 820 billion since 2012. Tobacco use claims 1.3 million lives annually in India, or 3,500 daily. Yet, the Global Adult Tobacco Survey (GATS) reports a six per cent decline in tobacco use from 34.6 per cent (2009-10) to 28.6 per cent (2016-17), a 17.3 per cent relative reduction. Integration with the National TB Elimination Programme and more than 300 cessation centres in medical and dental colleges reflect India's holistic approach.
Indian Express
23-06-2025
- Health
- Indian Express
India among 6 countries conferred with Bloomberg Philanthropies Awards for Global Tobacco Control
India is among the six countries that received 2025 Bloomberg Philanthropies Awards for Global Tobacco Control for prioritizing tobacco cessation approaches to reach a significant number of tobacco users in the country. The National Tobacco Control Cell of Ministry of Health and Family Welfare, Government of India, was presented with the award at the World Conference on Tobacco Control held in Dublin, Ireland, on Monday. India was presented the 'O' category award for promoting tobacco cessation. The 'O' is the MPOWER policy package of the World Health Organisation (WHO) that stands for 'Offer help to quit tobacco use.' The awards celebrated several governments and NGOs making progress to reduce tobacco use. Among the other countries that received the award were Mauritius, Mexico, Montenegro, Philippines and Ukraine. The WHO Global Tobacco Epidemic 2025 report, developed with support from Bloomberg Philanthropies, was also launched on the occasion. In India, a whole range of services including tobacco testing labs and mobile cessation measures among others have been set up to reduce tobacco usage in the country. This includes a well established national toll-free quit line, handling around 50,000 calls per month and free cessation support services at primary care facilities and hospitals. Tobacco cessation services are provided at 675 district hospitals, 302 dental institutions, 361 medical institutions and across 607 non-communicable diseases (NCD) clinics, oral health clinics and community health centres. There are four quit lines at NIMHANS, Vallabhbai Patel Chest Centre, New Delhi, Tata Memorial Centre and Borooah Cancer Institute in Guwahati. Between 2007 and 2023, the global average smoking prevalence has reduced from 22.3 per cent to 16.4 per cent, a relative reduction of 26 per cent over 16 years. According to the report, the adult daily smoking prevalence in 2023 in India was at 7 per cent. While experts said that smokeless tobacco was a big concern in India, several steps have been taken to address multiple challenges. 'India has been making consistent efforts to help people quit tobacco through the National Tobacco Control Programme (NTCP). This includes the establishment of tobacco cessation clinics in various locations (including dental and medical facilities), screening and intervention in primary care at the Ayushman Arogya Mandirs, establishing Cessation and National Tobacco Quitlines, integrating the NTCP with existing health services and national programmes, enacting legislation, providing training and awareness to healthcare providers, as well as public awareness campaigns,' Dr Pratima Murthy, Director and senior professor of Psychiatry at the National Institute of Mental Health and Neuro Sciences, who received the award on behalf of the Ministry of Health and Family Welfare, told The Indian Express. 'Tobacco free youth campaigns and the TeleManas initiative also represent efforts to address the underlying reasons for initiation of tobacco use. These comprehensive efforts indicate India's commitment to reduce tobacco use and promote public health. It is an absolute honour to receive the award on behalf of the Indian Government,' added Dr Murthy. Michael Bloomberg, founder of Bloomberg LP and Bloomberg Philanthropies and WHO Global Ambassador for Noncommunicable Diseases and injuries, presented the award. 'Since Bloomberg Philanthropies started supporting global tobacco control efforts in2007, there has been a sea change in the way countries prevent tobacco use, but there is still a long way to go,' said Bloomberg. Meanwhile, the WHO Global Tobacco Epidemic 2025 report reveals that the most striking gains have been in graphic health warnings, one of the key measures under the WHO Framework Convention on Tobacco Control (FCTC), that make the harms of tobacco impossible to ignore. At the award ceremony, Michael R. Bloomberg also announced a new $20 million Accelerator Fund to help advance progress in countries where tobacco control is stalled. Bloomberg Philanthropies will work with partners to proactively engage governments and NGOs in several low- and middle-income countries that either lack best-practice tobacco control measures or are close to implementing all best-practice measures. Anuradha Mascarenhas is a journalist with The Indian Express and is based in Pune. A senior editor, Anuradha writes on health, research developments in the field of science and environment and takes keen interest in covering women's issues. With a career spanning over 25 years, Anuradha has also led teams and often coordinated the edition. ... Read More
Time of India
20-06-2025
- Health
- Time of India
Arunachal bags award for tobacco-free youth campaign
1 2 Itanagar: Arunachal Pradesh has been recognised as the best performing state under the smaller states category for its outstanding implementation of the Tobacco-Free Youth Campaign (TFYC) 2.0, an initiative of the Union health and family welfare ministry. The award was conferred by ministry's additional secretary V Hekali Zhimomi and director of drug and general disease (electronic medical record) L Swasti Charan and was received by National Tobacco Control Programme (NTCP) in-charge state programme officer Hatobin Mai and NTCP state consultant Karpa Marde from Arunachal Pradesh. Conducted from Sept 23 to Nov 24 last year, the campaign aimed at creating mass awareness among the youth about the dangers of tobacco use, encouraging cessation and shielding them from the manipulative strategies of the tobacco industry. The campaign also promoted the establishment of tobacco-free educational institutions and empowered rural communities through public engagement and stronger law enforcement. The campaign was successfully implemented across all 26 NTCP districts, including Aalo, Bomdila, Daporijo, Itanagar Capital Region, Pasighat, Roing, Seppa, Tezu, Yupia, Yingkiong, and Ziro among others. Under TFYC 2.0, the state recorded remarkable achievements: 2,250 IEC activities were conducted, along with 851 village-level awareness campaigns. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like One of the Most Successful Investors of All Time, Warren Buffett, Recommends: 5 Books for Turning... Blinkist: Warren Buffett's Reading List Click Here Undo A total of 279 gram panchayats passed resolutions declaring their jurisdictions tobacco-free, while 404 villages were officially declared tobacco-free. Additionally, 2,120 educational institutions achieved tobacco-free status under the the Tobacco Free Educational Institutions (ToFEI) guidelines, and 3,356 institutions were covered through awareness drives by the district tobacco control cells.
