Critical Path Institute (C-Path)

Great news to share! 🎉 Congratulations to C-Path President and COO Kristen Swingle on her appointment to the Board of the Institute for Advanced Clinical Trials for Children. This esteemed nonprofit is dedicated to driving innovation and expediting pediatric drug development — a mission that resonates deeply with C-Path's own commitment to advancing pediatric research and data sharing. We're thrilled to share in Kristen's enthusiasm as she embarks on this journey and look forward to the collaboration and shared learning that will follow. "𝘐 𝘢𝘮 𝘩𝘰𝘯𝘰𝘳𝘦𝘥 𝘵𝘰 𝘫𝘰𝘪𝘯 𝘵𝘩𝘦 𝘣𝘰𝘢𝘳𝘥 𝘰𝘧 𝘐𝘈𝘊𝘛4𝘊, 𝘢𝘯 𝘰𝘳𝘨𝘢𝘯𝘪𝘻𝘢𝘵𝘪𝘰𝘯 𝘵𝘩𝘢𝘵 𝘪𝘴 𝘢𝘵 𝘵𝘩𝘦 𝘧𝘰𝘳𝘦𝘧𝘳𝘰𝘯𝘵 𝘰𝘧 𝘥𝘳𝘪𝘷𝘪𝘯𝘨 𝘪𝘯𝘯𝘰𝘷𝘢𝘵𝘪𝘰𝘯 𝘪𝘯 𝘱𝘦𝘥𝘪𝘢𝘵𝘳𝘪𝘤 𝘥𝘳𝘶𝘨 𝘥𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵,” 𝘒𝘳𝘪𝘴𝘵𝘦𝘯 𝘴𝘢𝘪𝘥. “𝘌𝘯𝘴𝘶𝘳𝘪𝘯𝘨 𝘵𝘩𝘢𝘵 𝘤𝘩𝘪𝘭𝘥𝘳𝘦𝘯 𝘩𝘢𝘷𝘦 𝘢𝘤𝘤𝘦𝘴𝘴 𝘵𝘰 𝘴𝘢𝘧𝘦 𝘢𝘯𝘥 𝘦𝘧𝘧𝘦𝘤𝘵𝘪𝘷𝘦 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵𝘴 𝘪𝘴 𝘢 𝘮𝘪𝘴𝘴𝘪𝘰𝘯 𝘵𝘩𝘢𝘵 𝘳𝘦𝘴𝘰𝘯𝘢𝘵𝘦𝘴 𝘥𝘦𝘦𝘱𝘭𝘺 𝘸𝘪𝘵𝘩 𝘮𝘦. 𝘐 𝘭𝘰𝘰𝘬 𝘧𝘰𝘳𝘸𝘢𝘳𝘥 𝘵𝘰 𝘤𝘰𝘯𝘵𝘳𝘪𝘣𝘶𝘵𝘪𝘯𝘨 𝘵𝘰 𝘐𝘈𝘊𝘛4𝘊’𝘴 𝘦𝘧𝘧𝘰𝘳𝘵𝘴 𝘵𝘰 𝘢𝘥𝘷𝘢𝘯𝘤𝘦 𝘳𝘦𝘴𝘦𝘢𝘳𝘤𝘩 𝘢𝘯𝘥 𝘪𝘮𝘱𝘳𝘰𝘷𝘦 𝘵𝘩𝘦 𝘭𝘪𝘷𝘦𝘴 𝘰𝘧 𝘤𝘩𝘪𝘭𝘥𝘳𝘦𝘯 𝘵𝘩𝘳𝘰𝘶𝘨𝘩 𝘮𝘰𝘳𝘦 𝘦𝘧𝘧𝘪𝘤𝘪𝘦𝘯𝘵 𝘢𝘯𝘥 𝘪𝘮𝘱𝘢𝘤𝘵𝘧𝘶𝘭 𝘤𝘭𝘪𝘯𝘪𝘤𝘢𝘭 𝘵𝘳𝘪𝘢𝘭𝘴." #CPath #IACT4C #clinicaltrials #pediatrics #collaboration #drugdevelopment #datasharing #globalhealth

A reminder that Public Review for the Cardiac Imaging Supplement closes 30 September! CDISC is pleased to announce the draft version 1.0 of the Cardiac Imaging Supplement to the Duchenne Muscular Dystrophy Therapeutic Area User Guide v1 has entered the Public Review period to solicit your feedback. This supplement is being developed in partnership with the Critical Path Institute (C-Path) Duchenne Regulatory Science Consortium (D-RSC). The guide includes new controlled terminology, SDTM and ADaM examples for several cardiovascular tests. Public review is a key quality step in our Standards Development Process. CDISC relies on your input to ensure neutral, consensus-based data standards are developed and adopted by a diverse global community interested in improving research processes and quality for the benefit of all. Thank you for contributing your time and expertise. Instructions for providing comments: https://lnkd.in/e3BeFfj5 Public Review closes 30 September 2024. #cleardataclearimpact

C-Path is excited to announce its membership with the Kidney Health Initiative! C-Path, in conjunction with the Biomarker Data Repository initiative, is proud to join KHI as the newest member. KHI was created by American Society of Nephrology and the FDA as public-private partnership that would enhance patient safety and foster innovation in kidney disease. More than 100 members have joined the KHI community since its inception in 2012. Their mission is to catalyze innovation and the development of safe and effective patient-centered therapies for people with kidney diseases. To review the current list of KHI members, please visit the KHI website: https://lnkd.in/gji87ahk. We are excited for this new partnership, KHI! Learn more about the BmDR project here: https://lnkd.in/gsYT23fE Nicholas King Katrina Peron, MS, RAC #CPath #BmDR #Biomarkers #Data #ASN #KHI #collaboration #drugdevelopment #datasharing #clinicaltrials #KidneyHealthIntiative #KidneyDisease #PatientCenteredTherapies

🚨Our publication about ‘Regulatory Readiness Level framework’ is out! We propose the implementation of a Regulatory Readiness Level framework. Designed to streamline regulatory pathways for innovation in Rare Diseases, the framework aims at making rare disease drug development more attractive and sustainable for approval and access. Allowing public–private collaborative work to further stimulate the integration of high-quality regulatory science in research can demystify regulatory requirements and potentially reduce investment risks. By leveraging existing infrastructures, resources and developing innovative tools such as Drug Development Tools, the Rare Disease Moonshot initiative strives to create a more efficient and sustainable ecosystem for rare disease research. The initiative's strength lies in its emphasis on multistakeholder collaboration, bringing together expertise from academia, industry, patient organisations, and regulatory bodies. This collaborative, regulatory-science-focused framework promises to accelerate the development of much-needed treatments for patients with rare and ultra-rare conditions. https://lnkd.in/dT2isCJR Thank you to all the authors and the advisors 👇 Cécile Ollivier Dr Solange Corriol-Rohou, MD Marta del Álamo Roseline Favresse Johanna Kostenzer, PhD Mathieu Boudes, PhD Anton Ussi Klaus Viel R. Michael Linden Magda Chlebus Ralf Herold Victor Maertens Stefania Alessi Claire Skentelbery Adrien Samson Manuela Pausan Daria Julkowska Gareth Baynam EFPIA - European Federation of Pharmaceutical Industries and Associations EURORDIS-Rare Diseases Europe EUCOPE - European Confederation of Pharmaceutical Entrepreneurs EuropaBio - the European Association for Bioindustries EATRIS ECRIN (European Clinical Research Infrastructure Network) BBMRI-ERIC Critical Path Institute (C-Path) european joint programme on rare diseases ejp rd International Rare Diseases Research Consortium (IRDiRC) #RDMoonshot #LaunchR #RareCareCentre #Rare Diseases Moonshot-Australia (RDMA)

Interested in C-Path updates? Be sure to subscribe at https://lnkd.in/gEXxG5Nd For 20 years, C-Path has been providing vital infrastructure to generate a neutral environment for everyone working in drug development to collaborate, not compete. Let's improve lives, together. #CPath #drugdevelopment #neutral #datasharing #globalhealth #collaboration #regulatoryscience

C-Path is excited to share that our Critical Path for Lysosomal Diseases Consortium has been awarded a grant from the Ara Parseghian Medical Research Fund at the University of Notre Dame. This generous support will strengthen our collaborative efforts in advancing research and developing treatments for Niemann-Pick Disease Type C. NPC is a rare and devastating genetic disorder that affects children, leading to severe brain and liver damage and often resulting in early death.    The APMRF was established by Cindy and Mike Parseghian, whose three youngest children (grandchildren to Legendary Notre Dame Coach, Ara Parseghian) were diagnosed with NPC. The foundation supports NPC research and is dedicated to finding a cure. “We are deeply honored to receive this grant from the Ara Parseghian Medical Research Fund,” said C-Path CEO Klaus Romero, M.D., M.S., FCP. “This support will significantly enhance our efforts to bring together the brightest minds from relevant fields to collaborate and accelerate the development of life-changing therapies for the NPC community.” Sean Kassen Cindy Parseghian Krista Casazza Read all the details, here: https://lnkd.in/duvtWWH3 #CPath #NiemannPick #NPC #raredisease #AraPaseghian #NotreDame #drugdevelopment #datasharing #collaboration

REGISTER NOW 📆 C-Path's Predictive Safety Testing Consortium is delighted to announce its second public workshop on evidentiary considerations for regulatory assessment and qualification of complex in vitro models - CIVMs.  Health authorities, academia professionals, model developers, and representatives from the pharmaceutical industry are encouraged to participate in this workshop. It offers a valuable platform for attendees to engage in discussions and deliberations regarding the evidentiary considerations essential for regulatory assessment and qualification of CIVMs. Through collaborative efforts, attendees will strive to reach a consensus on the standards and features necessary to enhance the performance of CIVMs as a reliable tool for drug development and regulatory assessment. The meeting agenda features sessions led by key opinion leaders, who will address general considerations for qualification. Additionally, interactive breakout sessions will concentrate on various organ systems and disease models. During these breakouts, participants will actively engage in discussions and deliberations to explore the evidentiary considerations crucial for regulatory assessment and qualification of complex in vitro models. Join us September 26 - 27, 2024 in Bethesda, Maryland. Register now: https://lnkd.in/gU5rSSi4 #CPath #PSTC #CIVM #workshop #drugdevelopment #datasharing #regulatoryscience #collaboration #globalhealth

C-Path's Translational Therapeutics Accelerator proudly announced today a $250,000 grant award to Peter Wipf, Ph.D., a distinguished researcher and professor at the University of Pittsburgh and his colleagues Dr. Zhou Wang UPMC Chair in Urological Research and Dr. Joel B. Nelson, the Frederic N. Schwentker Professor and Chairman of the Department of Urology. This funding will support their pioneering research aimed at developing a novel small-molecule drug to combat castration-resistant prostate cancer. Prostate cancer is the second leading cause of cancer-related deaths in people assigned male at birth in the United States, claiming over 30,000 lives annually. While first line therapy is effective initially, many patients experience relapse due to the reactivation of the androgen receptor (AR). Drs. Nelson, Wang and Wipf’s innovative approach targets a particular constitutively active variant of AR that plays an important role in this reactivated state drug-resistant relapse. “C-Path’s TRxA is committed to funding research that addresses significant medical challenges, and Drs. Nelson, Wang and Wipf’s project epitomizes this mission,” said TRxA Executive Director Maaike Everts, Ph.D. “We are enthusiastic about the potential impact of this research on the treatment of CRPC and the improvement of patient outcomes. Importantly, this highly collaborative program combines medicinal chemistry, cancer biology, and the translational and clinical expertise to drive this project to clinical solutions for the patients.” Full details, here: https://lnkd.in/dxZaQs8D #CPath #TRxA #UniversityofPittsburgh #translational #therapeutic #prostatecancer #datasharing #druddiscovery #drugdevelopment #urology #collaboration

We are thrilled to share that C-Path has entered into a strategic data-sharing partnership with Vivli, an independent nonprofit dedicated to advancing global clinical trials data sharing. This collaboration marks a significant step forward in breaking silos and making rare disease data more accessible to researchers and stakeholders worldwide. The partnership will see data sets from Biogen, including the phase II PASSPORT trial in progressive supranuclear palsy and two choroideremia datasets, available on both C-Path's Rare Disease Cures Accelerator Data and Analytics Platform and Vivli's Clinical Trials Data Platform. This alignment reinforces our shared commitment to the FAIR principles of data sharing, ensuring that valuable research data is findable, accessible, interoperable, and reusable. “We are thrilled to announce that Vivli, RDCA-DAP®, and Biogen collaborated to integrate a Progressive Supranuclear Palsy clinical trial dataset into both partnering platforms, as well as two datasets in choroideremia, our first data in rare eye disorders,” shared Alexandre Bétourné, PhD, PharmD, PMP, Executive Director for RDCA-DAP at C-Path. Together, we're accelerating the development of treatments for rare diseases by fostering collaboration and data sharing among academic researchers, industry leaders, healthcare organizations, and patient advocacy groups. Julie Wood Janet Krause #CPath #Vivli #DataSharing #RareDiseases #Collaboration #ClinicalTrials #RDCADAP #DrugDevelopment

Exciting News! C-Path has received its first data transfer of natural history data on single large-scale mitochondrial DNA deletion syndromes from The Champ Foundation. This data transfer is a significant milestone, bringing in the most comprehensive dataset of individuals with SLSMDS from The Champ Foundation Registry. It includes patient-reported information on symptoms, medical history, and standardized assessments of quality of life, care partner burden, and functional outcomes. The Champ Foundation, a dedicated patient advocacy group, is on a mission to find treatments and a cure for SLSMDS, including Pearson syndrome. Their commitment to sharing this invaluable data with C-Path is a huge step forward in our shared goal of advancing research. Dr. Elizabeth Reynolds, Co-Founder and Executive Director of The Champ Foundation, shared her enthusiasm: “We are excited to collaborate with C-Path to further research on Pearson syndrome and other single large-scale mitochondrial deletion syndromes. This data transfer represents a significant step towards better understanding these rare diseases and ultimately finding effective treatments.” This collaboration is poised to unlock new insights and accelerate the path to better treatments for those affected by these ultra-rare disorders. We look forward to an impactful partnership. Full details, here: https://lnkd.in/gNhBjKzm Amanda Klein Jeff Reynolds #CPath #RareDisease #DataSharing #TheChampFoundation #MitochondrialDisease #PearsonSyndrome #drugdevelopment #globalhealth #collaboration