HI-Bio: A Biogen Company’s Post

💡 Breaking Barriers in Rare Disease Research - From development planning to regulatory submissions, ERGOMED partners with you to design and execute innovative, patient-centric clinical research programs for patients and families living with the challenge of a rare disease. Ready to learn more? Meet our team at Booth #437 at World Orphan Drug Congress USA 2024 next week! Together, let's make a difference. #RareDisease #PatientCentricity #Innovation #ClinicalResearch

Last week I was in Las Vegas, this week, San Francisco! I'm honored to be speaking at Clinical Trials in Rare Diseases on Engaging patients beyond just participating in a trial: the role of biopharma companies in supporting patients. #PatientCentricity means truly putting the patients at the forefront of your research and development. Supporting patients throughout the entire lifecycle of rare disease research and clinical trials and beyond as well as giving them a voice in the process is the only way to maximize your ROI! #clinicaltrials #patientadvocacy #patientengagement #patientsupport #research #treatment #patientvoice #biotech #pharma #healthtech #realworldevidence #realworlddata #researchanddevelopment #patientexperience #patientsolutions #orphandrugs #raredisease

Did you miss our latest webinar? No problem! We've compiled a comprehensive library of over 20 market access webinar recordings, available on demand for your convenience. 💡 Our goal is to provide valuable resources for your market access journey and keep your team at the forefront of developments in the market access landscape. Here are some of the exciting topics we've recently explored: - Navigating EU HTA: The Future of Supranational HTA Initiatives; Lessons Learned So Far - The Value of Delphi Panels in Accelerating Patient Access - Breaking Down Barriers to Access in Europe: Getting It Right the First Time for Rare Diseases Discover the full range of market access webinars, exclusively available for biopharma companies. Request your recordings here: https://lnkd.in/exewTNnb #webinar #marketaccess #webinarrequest

We are proud to share two recognitions in this year’s Fierce Pharma Fierce 50 report. Lotte Bjerre Knudsen, Chief Scientific Advisor, Research & Early Development, and Uli Stilz, Head of the Bio Innovation Hub, are breakthrough and innovation honorees. Lotte’s advocacy work around a single molecule to treat diabetes and obesity has revolutionized healthcare. Our Bio Innovation Hub in Cambridge, MA, under Uli’s leadership, harnesses cutting-edge technology to co-create next-gen therapeutics with partners across North America. Lotte and Uli’s recognitions serve as a testament to their leadership and our commitment to driving innovation. Together, we're forging new paths in addressing cardiometabolic diseases, fueled by our world-class scientists and exceptional partners who are redefining the boundaries of innovation and drug discovery. https://lnkd.in/eA9dkkQq #NovoNordiskUS #NovoNordiskWay #Fierce50

We’re excited to share a major development at AGYANY Pharma, founded by Professor Ari ZIMRAN in November 2022. With decades of expertise in Gaucher Disease, Prof. Zimran is leading the charge to advance the treatment of GBA1-associated Parkinson’s Disease (GBA1-PD) through pioneering clinical trials with pharmacological chaperones like Ambroxol. Our mission extends beyond generating crucial data for future therapies. We are committed to developing innovative treatments that could transform the care landscape for both Gaucher Disease and GBA1-PD. Recently, we secured key patents for the use of Ambroxol in treating these conditions—a pivotal moment in our journey. Our vision is clear: to deliver the first safe and effective therapies for these devastating diseases, meeting the most pressing needs of patients and setting a new standard in care. #Biotech #HealthcareInnovation #GaucherDisease #ParkinsonsDisease #MedicalResearch

Today on #WorldAlzheimersDay, C-Path honors those affected by Alzheimer’s and reaffirms our commitment to advancing research and drug development. Founded in 2008, the Critical Path for Alzheimer’s Disease is a global, neutral convener, bringing together diverse stakeholders across industry, regulatory agencies, and academia within a pre-competitive forum. By leveraging patient-level data contributions, CPAD enhances regulatory decision-making tools, addresses critical unmet needs, and accelerates therapeutic innovation to improve the lives of those living with Alzheimer’s and related dementias. We are also proud to share that Diane Stephenson, Executive Director of CPAD, will co-chair the “Advancing Regulatory Science to Deliver Precision Medicines” panel at the 2024 NIH Alzheimer’s Summit next week. This important discussion will bring together experts from the FDA to explore how regulatory science can help deliver precision treatments for Alzheimer’s patients. Learn more about CPAD, here: https://lnkd.in/gZjPjKin #CPath #WorldAlzheimersDay #AlzheimersResearch #CPAD #FDA #PrecisionMedicine #NIHSummit

🌟 Breaking Barriers: Embracing the Moonshot Initiative in Rare Diseases! 🌙🚀 Exciting news for the rare disease community! We're thrilled to announce that Magda Chlebus, Executive Director Science Policy and Regulatory Affairs, European Federation of Pharmaceutical Industries and Associations (EFPIA), a visionary leader in healthcare innovation, will be presenting the groundbreaking Moonshot Initiative at the upcoming International Congress on Rare Diseases and Orphan Drugs. 🎤✨ Unveiling the Moonshot Initiative: 🚀 Mission: The Moonshot Initiative is a bold and transformative approach to addressing the challenges faced by the rare disease community. Magda Chlebus will unveil the mission, strategies, and collaborative efforts that make this initiative a beacon of hope for patients and caregivers. 🌈 Visionary Impact: Explore how the Moonshot Initiative aims to accelerate research, enhance patient care, and foster collaboration among stakeholders. Save the Date: [Friday 1st of March 09.20 – 10.00 AM] 🗓️ Digital How to Tune In: Secure your virtual seat now to witness this groundbreaking presentation. Register and be part of a transformative moment that could shape the future of rare disease interventions. 🔗 [Registrer here: https://bit.ly/3HkkWxA] Spread the Excitement: Tag your colleagues, fellow advocates, and anyone passionate about driving change in the rare disease landscape. Let's amplify the impact of the Moonshot Initiative together! #rarediseasesconference24 #MoonshotInitiative #RareDiseases #HealthcareInnovation #ICORD2023 #PatientAdvocacy Don't miss this extraordinary opportunity to be part of a paradigm-shifting conversation! 🌐🌟

Last night, was one of those nights where I did something I rarely do. I watched a "soapy" just to switch off from my work day. Which one? Grey's Anatomy. I don't think I've ever seen a full episode of this before and it was pretty torrid but what I found interesting was that one of patients in this episode had cystic fibrosis. He was very pragmatic and said that he had lived a lot longer than anyone expected him to, so he felt lucky he'd made it to 26 years old. Any my brain went - wait! What??? That's not right..... And then I realised, this episode first aired in 2005 when the median survival for CF patients was less than 30 years. It would be another 7 years before Vertex' first product, Kalydeco, would be approved by the FDA and a few months later, in 2013, I would submit this registration application to the TGA in Australia. The median survival for cystic fibrosis patients has now almost doubled thanks to new treatments for this disease. We are all very focused on finding the next drug target, starting the next clinical trial, submitting the next registration application, and rightly so. But sometimes, it's good to reflect on how far we've come in the orphan drug space in recent years and the impact this has had on patients and their families and friends. Next week I'll be attending the ARCS Conference in Sydney and will be part of the new BioBeacon sessions. Companies will be presenting on their new products in development and panels of experts will provide opinions on some of the pitfalls they may face, questions to ask etc. I very much look forward to being a member of this panel and hearing about what may come to market, from the Australian biotech ecosystem, in another 10-20 years time. #ARCS #biotech #innovation Adjutor Healthcare Group

Sunday Spotlight: At #ASRS2024, Arshad M. Khanani, MD, presented late-breaking RHONE-X data showing more than 90% of patients experienced the absence of DME after four years of faricimab (Vabysmo; Genentech). Check out our interview for expert perspective on how this data can influence decision-making in #ophthalmology: https://lnkd.in/eFtfnm5S

Join a free webinar on January 30th to illustrate some of the challenges and considerations of successfully executing hAME studies.