Rare Disease Moonshot’s Post

🔔 Have you heard the latest news this July? 😎 You might know that the European Joint Programme on Rare Diseases (RD) is shaping the RD research ecosystem, but do you know how? We're thrilled to share that the upcoming European Rare Diseases Research Alliance (ERDERA) is set to take public-private partnerships to the next level. With over 170 partners across 36 countries, ERDERA will enhance RD research by offering crucial services like data processing, regulatory compliance, innovative methodologies, and training support. This ensures strategic coherence and resource efficiency both globally and nationally. Ultimately, ERDERA aims to establish a European Federated Rare Disease Research organization, sustaining long-term public and private support and transforming the landscape of RD research. That's the #RDMoonshotspirit! EURORDIS-Rare Diseases Europe EuropaBio - the European Association for Bioindustries EUCOPE - European Confederation of Pharmaceutical Entrepreneurs EFPIA - European Federation of Pharmaceutical Industries and Associations BBMRI-ERIC EATRIS ECRIN (European Clinical Research Infrastructure Network) Critical Path Institute (C-Path) Daria Julkowska Yanis Mimouni Halftermeyer Juliane Yanna Petton #clementmoreau Roseline Favresse Birute Tumiene https://lnkd.in/eHXJA_6g

🌟 Exciting News from the Rare Diseases International Congress! 🌟 🌍💼 Don't miss this pivotal session at the upcoming International Congress on Rare Diseases and Orphan Drugs – a dynamic panel discussion on "Navigating Access Pathways for Orphan Drugs in Greece ". At the heart of the conversation is the pressing need to improve access to life-changing orphan drugs for rare disease patients in Greece. Joined by esteemed experts and stakeholders, we will delve into the complexities and challenges surrounding access pathways, aiming to pave the way for tangible solutions. Key Discussion Points: 🔍 Explore the current landscape of orphan drug access in Greece. 💡 Identify barriers and gaps in the system. 🤝 Showcase collaborative strategies to enhance patient access and advocacy efforts. 🌐 Leverage international best practices for local implementation 🎤 We are thrilled to welcome Yvoni Papastelatou, Sanofi Country Lead, Greece & Cyprus and Specialty Care General Manager, Greece & Cyprus and PhARMA Innovation Forum Greece (PIF) BoD Member tomorrow at Day 1 of proceedings at Technopolis City of Athens Gasholder 1 – Auditorium “Miltiadis Evert” joining this Panel Discussion on "Navigating Access Pathways for Orphan Drugs in Greece". Having worked in the pharmaceutical sector for more than 9 years she brings a wealth of knowledge and experience into the discussions. Save the Date: [Thursday February 29th 12:45 PM - 14:00 PM] 🗓️ Hybrid 🔥 Join proceedings! As we continue to champion the cause for rare disease patients, let's collaborate, advocate, work together to ensure equitable access to essential treatments and drive positive change for the Rare Disease community worldwide! Every voice counts, we can make a difference! 💪🧬 🌐 Register Today: https://bit.ly/3HkkWxA 🌍Spread the Word: Tag your colleagues, friends, and anyone passionate about rare diseases. Let's make ICORD 2024 a gathering of minds committed to making a difference! #ICORD2024 #rarediseasesconference24

🌍 𝐄𝐱𝐜𝐢𝐭𝐢𝐧𝐠 𝐧𝐞𝐰𝐬. We are heading to BIO-Europe 2024! 🌍 We can't wait to attend #bioeurope 2024, one of the biggest life science partnering events! It’s the perfect chance to connect, collaborate, and change the future of precision healthcare together. 𝐖𝐡𝐚𝐭 𝐭𝐨 𝐞𝐱𝐩𝐞𝐜𝐭: ✦ 5,000+ delegates ✦ 2,500+ unique companies ✦ 27,000 one-to-one meetings ✦ 5,000+ published licensing opportunities ✦ 65+ countries represented ✦ 110+ presenting companies (Sabiad included 😉) This is the place to be for life sciences professionals! 🧪 𝐀𝐛𝐨𝐮𝐭 𝐒𝐚𝐛𝐢𝐚𝐝 Founded in 2022 as a spin-off from the University Medical Center Groningen UMCG, Sabiad is building on 20+ years of research led by Prof. Jan Maarten van Dijl We’re leading the way in developing theranostic solutions for bacterial infections, particularly 𝐒𝐭𝐚𝐩𝐡𝐲𝐥𝐨𝐜𝐨𝐜𝐜𝐮𝐬 𝐚𝐮𝐫𝐞𝐮𝐬 (𝐒𝐀). You will learn how you can accurately target, detect, and treat Staphylococcus aureus (SA) infections with our innovative diagnostic and therapeutic solution. At this event, we’ll introduce our 𝐒𝐭𝐚𝐩𝐡𝐌𝐚𝐫𝐤 theranostic platform, 𝐩𝐨𝐰𝐞𝐫𝐞𝐝 𝐛𝐲 𝐭𝐡𝐞 1𝐃9 𝐚𝐧𝐭𝐢𝐛𝐨𝐝𝐲. Discover how our in vivo and ex vivo applications transform infection management with pinpoint accuracy. Are you attending BIO-Europe 2024? 🔗 Save 300€ by purchasing your ticket before November 1st! 𝐑𝐞𝐠𝐢𝐬𝐭𝐞𝐫 𝐡𝐞𝐫𝐞 → https://lnkd.in/dnuNZ2D Drop a comment below or send us a direct message if you’ll be there. We’d love to arrange a time to meet and explore how we can collaborate at the event! #bioeurope2024 #precisionmedicine #infectioncontrol #theranostics #StaphMark

Despite advancements, most rare diseases lack effective therapies, underscoring the necessity for a strategic overhaul in the approach to research and development. To address the multifaceted challenges in rare disease research, a unified framework that encompasses the recommendations across clinical trials, diagnostics, and translational research is proposed. Central to this framework is the role of public-private partnerships in bridging gaps in research and development. By aligning efforts, leveraging resources, and fostering innovation, significant strides can be made towards erasing the "white spots" in rare disease research. Read our research needs recommendations: 👉 our one pager/factsheet https://lnkd.in/dmthy73t and in greater details: 👉 about clinical trials https://lnkd.in/d2yiVGZv 👉 about diagnosis https://lnkd.in/dvRih7cr 👉 about translational research https://lnkd.in/d9M6G2Gr EFPIA - European Federation of Pharmaceutical Industries and Associations EURORDIS-Rare Diseases Europe Critical Path Institute (C-Path) EATRIS BBMRI-ERIC ECRIN (European Clinical Research Infrastructure Network) EUCOPE - European Confederation of Pharmaceutical Entrepreneurs EuropaBio - the European Association for Bioindustries European Patients' Forum european joint programme on rare diseases ejp rd International Rare Diseases Research Consortium (IRDiRC) Together for Rare Diseases Innovative Health Initiative (IHI)

The 2nd ERA4Health workshop took place on February 8-9 in Brussels, fostering a unique blend of expert insights, collaborative discussions, and practical knowledge sharing. 💡 This workshop, hosted by ECRIN, gathered funders, clinical study experts, EMA (ACT EU), European Commission, and HaDEA representatives. The primary objective was to delve into funding of Multinational Investigator-Initiated Clinical Studies (IICS), particularly addressing public health-driven, non-commercial studies. The first day included presentations from different initiatives currently funding or planning to fund multinational studies in different topics (infectious diseases, cancer, vaccines, rare diseases, comparative-effectiveness) and set ups (European, international). The models and funding strategies introduced were then used as a foundation for the second day, reserved for ERA4Health partners. It focused on discussions of possible adaptations of the current Joint Transnational Call funding mechanism to the objectives, of the planned ERA4Health Pilot Call on IICS. We thank all the participants for their invaluable contributions. ECRIN will continue to work with the funders in ERA4Health and other stakeholders to support the creation of a sustainable funding mechanism for IICS relying on the work carried out in this workshop. ✍️ #FundingMechanisms #InvestigatorInitiatedClinicalStudies #Workshop

Earlier this week, our colleagues Julia Lischke and Lianne Hulshof attended the 5th AMR Accelerator Cross-Project meeting in Basel. The AMR accelerator is comprised of nine projects that are focused on progressing the development of new medicines to treat or prevent resistant bacterial infections. Lygature is involved in two of these projects: UNITE4TB and GNA NOW, providing partnership management and communication & dissemination support. The UNITE4TB Consortium is working to accelerate the development of new tuberculosis drug regimens, while GNA NOW is working to progress potential Gram-negative antibiotics from lead optimization to the completion of Phase I trials. Sharing insights from the meeting, Julia, who was there on behalf of both Lygature and UNITE4TB said: "The AMR accelerator pipeline is filled with interesting candidates and there is active collaboration between the projects on the exchange of ideas and perspectives. Perhaps more importantly though, there is a well-managed handover of assets from preclinical to clinical studies between the projects. This facilitates the most efficient use of resources. "For Lygature the cross-project meeting is a very good networking opportunity, nicely facilitating idea generation for new collaborations. For UNITE4TB, it is an essential means of understanding what assets are being developed and how they are progressing, helping to optimally facilitate the cross-project collaboration." Lianne added from the GNA NOW perspective: "It is encouraging to see the amount of energy being put into this multi-project collaboration. New opportunities are constantly being found and explored. Together, we are working towards the common goal of finding solutions to the ever-growing problem of antimicrobial resistance." Keen to learn more about our involvement in these and other projects in the Global Health space and beyond? Get in touch and discover how we're helping to pioneer the development of new treatments for patients in need. #AMR #EndTB #GlobalHealth #PublicPrivatePartnerships  Innovative Health Initiative (IHI)

🎉 Celebrating 40 years of EU Research and Innovation! This year marks the 40th anniversary of the first EU funding programme dedicated to science, research and innovation. 🎯 For the last 21 years, the European Commission has been supporting #GlobalHealth research first under the European & Developing Countries Clinical Trials Partnership and now under Global Health EDCTP3. Today we are excited to spotlight 4 success stories that are the result of years of investments in research and capacity-building in sub-Saharan #Africa under the EDCTP1 and EDCTP2 programmes: ⭐ A simpler and safer treatment for #CryptococcalMeningitis   ⭐ A new #Malaria vaccine for children, R21 Matrix M ⭐ A successful treatment for #Schistosomiasis in children ⭐ The first oral treatment for #SleepingSickness As we celebrate #EUfunded research and innovation, these groundbreaking outcomes demonstrate the global impact of collaborative efforts in improving and advancing health and scientific progress! #ResearchImpactEU #EU40YearsRI Find out more about our work 👇 🔗https://lnkd.in/eiCBCUb7 -------- Iliana Ivanova, Irene Norstedt, EU Science, Research and Innovation, European Medicines Agency World Health Organization, Lygature, Merck European Vaccine Initiative (EVI) Global Health Innovative Technology (GHIT) Fund Pediatric Praziquantel Consortium Drugs for Neglected Diseases initiative - DNDi

🌍 Sustainable Funding: Key to Advancing Antimicrobial Resistance Research & Development 🦠 The threat of antimicrobial resistance (AMR) remains one of the most pressing challenges in global health. The GNA NOW Consortium, coordinated by Lygature and part of the AMR Accelerator, is leading the charge to develop novel Gram-negative antibacterials to tackle severe enteric infections caused by pathogens like Shigella, Campylobacter, E. coli, and Salmonella. But initiatives like GNA NOW and the AMR Accelerator can’t do it alone. They need sustained investment from governments, the private sector, and global stakeholders to ensure that R&D capacity remains strong and capable of delivering life-saving antibiotics. Public-private partnerships like GNA NOW and those under the Accelerator demonstrate that through collaboration, risk-sharing, and long-term commitment, it’s possible to bring new treatments to patients and strengthen the fight against AMR. 🔗 Find out more about the GNA NOW Consortium and the joint AMR Accelerator Call to Action ahead of the UN High-Level Meeting on AMR later this week 👉 https://lnkd.in/eSejxSDR GNA NOW is a #PublicPrivatePartnership funded by the Innovative Medicines Initiative, now Innovative Health Initiative (IHI). The Consortium consists of GSK (Scientific Lead), BIOASTER, Helmholtz Centre for Infection Research, North Bristol NHS Trust, University of Liverpool, INSERM, University of Poitiers, Erasmus MC, Medical University of Vienna, Fraunhofer-Gesellschaft, and Lygature (Coordinator). EFPIA - European Federation of Pharmaceutical Industries and Associations #AntimicrobialResistance #AMR #GlobalHealth #SustainableFunding #UNGAHLM #WeWontRest

We are happy to attend the prestigious #PrixGalien Forum Poland, where industry leaders will discuss the future of healthcare innovation, including topics such as AI in healthcare, mental health therapies, and more. Justin Gandy, Managing Director of MSD Polska, will participate in the “Pharma CEO” panel.  Our company #MSDPolska has been selected as a finalist in three categories in the prestigious Prix Galien Poland awards: 🏆 Best Pharmaceutical Product – Recognizing our immunotherapy in oncology, in new indications. 🏆 Best Orphan/Rare Disease Drug – For our new therapy in pulmonary arterial hypertension (PAH), a rare and debilitating condition that profoundly impacts patients’ quality of life. 🏆 Best Health Awareness Campaign – For our “Let’s Win Against HPV” campaign, which unites doctors, patients, and the public in the fight against HPV-related diseases. The Prix Galien is known worldwide as a pinnacle of medical and pharmaceutical recognition, with editions in countries like the U.S., France, and Canada. It celebrates advancements that make a transformative difference in healthcare, honouring pioneering work in medical science and health awareness.   🌟 We are proud of our innovations in healthcare! We are cheering for all the innovators who, like us, are finalists of the #PrixGalienPolska and contribute to improving health outcomes for Polish patients and the overall health of the Polish community. Together, we can change the future! 💪🇵🇱 #Innovations #Health #Poland To learn more about this prestigious event, visit: https://lnkd.in/dgzVm_-E Read also: https://lnkd.in/eF2G2wh5 #prixgalien2024 #StayTuned #PrixGalienPoland #HealthcareInnovation #Immunotherapy #HPVPrevention #PulmonaryHypertension #HealthAwareness

📢 Fondazione Telethon is among the promoters of a call to action issued today on the implementation of the EU Joint Clinical Assessment for ATMPs.   🔬 New rules for the clinical assessments of medicines at Union level were established to streamline the process of bringing novel therapies to patients. However, stakeholders are concerned that unless the approach is modernised to adequately evaluate advanced therapies, which have unique features compared to traditional drugs and often target rare diseases, this initiative will in fact create another barrier to patient access.   🔬 The appeal urges all those involved in the JCA to recognise and use all types of available clinical evidence, including single-arm trials and real-world evidence.   🔬 In particular, Fondazione Telethon warned about the ethical dilemma associated with adopting a randomised controlled trial over a single arm trial when developing therapies for rare and ultra-rare diseases; this would, in most cases, imply giving a placebo to patients with no therapeutic alternative.   💙 More than 30 organizations, including patient groups, scientific societies, research foundations and medical institutes, signed the appeal.