Assembly Biosciences, Inc.的动态

We’ve made significant progress since signing our collaboration with Gilead, with four programs now in the clinic and a number of important data readouts expected in the next year. This new funding will support the advancement of our pipeline, driving us closer to delivering meaningful impact for people living with serious viral diseases.

?? FDA’s STAR Pilot Program: Accelerating Rare Disease Therapies ?? Exciting news for the rare disease community! The FDA has launched the *Support for Therapeutic Advancement and Research (STAR) Pilot Program*, an initiative designed to speed up the development of therapies for rare diseases. STAR aims to foster collaboration, support innovative trial designs, and provide early, ongoing feedback to developers of rare disease treatments. By streamlining the path from research to regulatory approval, the program hopes to deliver more effective, timely solutions to patients with critical, unmet needs. Let’s celebrate this step forward in making transformative therapies more accessible to rare disease patients! #FDA #RareDiseases #STARProgram #DrugDevelopment #PatientAdvocacy #HealthcareInnovation

With over 7,000 rare diseases affecting approximately 300 million people globally, the collective burden of these conditions is profound. When it comes to rare disease clinical development, Perspectives covers a wide range of topics. Here are 2024’s top posts: Orphan Drug Designation for Rare Diseases https://hubs.ly/Q030pVy00 Understanding Market Exclusivity for Orphan Drug Products https://hubs.ly/Q030pVJ70 Quantitative Strategies for Rare Disease Clinical Trials https://hubs.ly/Q030pVzL0 Sample Size Re-Estimation for Rare Disease Clinical Trials https://hubs.ly/Q030pVPL0 Thank you to Natalia Mu?hlemann, Boaz Adler, Valeria Mazzanti, and the Strategic Consulting team for sharing your insights!

??? Championing Hope in Rare Disease Clinical Trials?? At PHARMExcel, we are honoured to play a part in advancing treatments for rare diseases, bringing hope to patients and families who often face immense challenges. While rare diseases may affect smaller populations, their impact is deeply felt—and we’re committed to making a difference. Our team is driven by compassion, expertise, and a shared purpose. By working together with our rare disease partners, sponsors, and investigators, we strive to deliver clinical trials that are thoughtfully designed and patient-focused. ?? Here’s how we make an impact: Specialised Expertise: We have widespread experience in rare disease trials, managing their unique complexities with care and precision. Collaboration at Heart: We believe that working closely with all stakeholders—patients, families, sponsors, and investigators—is key to delivering successful trials and meaningful outcomes. ?? Rare disease research is more than science—it’s a mission of hope. At PHARMExcel, we’re proud to be part of that mission, walking alongside our partners and patients every step of the way. Read more about how we support rare disease research https://lnkd.in/e59RUx57 Together, we can transform challenges into opportunities and help bring life-changing therapies to those who need them most. #ClinicalResearch #RareDiseases #PHARMExcel #PatientCentricity #ClinicalTrials #CRO

Introducing the Innovative Trial Designs Track at #iDR25! This track will explore how advanced clinical trial designs are revolutionising drug repurposing. ? Learn how different trial methods address challenges like small population sizes and limited disease knowledge. ? Discover how unique statistical approaches tailored for small populations can enhance trial efficiency. ? Explore the impact of these innovative designs on the drug repurposing landscape to benefit patients. ?? Join us to uncover the future of clinical trials. Early bird tickets are available here for a limited time: https://loom.ly/xGqLsX4 Beacon for Rare Diseases, Anticancer Fund, EURORDIS-Rare Diseases Europe, ECRIN (European Clinical Research Infrastructure Network), EATRIS, Teamit #InnovativeTrials #DrugRepurposing #ClinicalResearch #PatientImpact

As per this FDA article below, an estimated 10,000+ rare diseases affect more than 30 million people - that's a lot of clinical trials where it can become challenging to recruit/enroll eligible subjects, while there's often a delayed diagnosis/misdiagnosis. If you play a role in one of these trials, we should talk about how our Judi for Eligibility solution can support you by streamlining/automating the central review process to help achieve a final determination in a timely manner. https://lnkd.in/eYKX_8aA #judiforeligibility #raredisease #collaboration

How are biotechs raising the bar for fatal liver disease treatments? ?? Acute on-chronic liver failure is a major healthcare issue with high unmet needs for patients, and a massive burden for healthcare systems. ?? French biopharma Genfit aims to take on rare, life-threatening liver diseases with a range of therapeutic targets. ?? ?? Labiotech spoke to GENFIT’s CSO Dean Hum about what treatments are on the way to treat the debilitating condition. ?? ?? Tune into the latest episode of the Beyond Biotech Podcast to find out about novel therapies addressing liver disease! ?? https://lnkd.in/d59ENPAK #BeyondBiotechPodcast #liverdisease #biotechinnovation #healthcaresolutions #rarediseases #medicalresearch

We are excited to announce the esteemed speakers for Goodwin’s Annual Rare Disease Symposium 2025!! Join us on February 5th to gain insights from: - FDA's Rare Disease Innovation Hub - NORD - BIO - Biotech CEOs - Rare Disease Researchers, Patients, and Organizations - Rare Disease Policy Experts And stick around to spend time networking with your peers in the rare disease community. Mark your calendars and RSVP at the link provided in the comments! #RareDiseaseSymposium #LifeSciences

“Given the small [rare disease] patient populations, it’s important not to make inclusion and exclusion criteria so strict that recruitment becomes impossible. Genetic diseases often have variable phenotypes or clinical presentations, so data-informed choices about parameters are essential to maintain scientific rigor while maximizing enrollment potential… By aligning trial criteria with real-world patient experiences, we can enhance the relevance and feasibility of clinical trials.” -Sara Riordan, vice president, Strategy In the latest issue of Clinical Trial Vanguard our Sara Riordan sits down with Moe Alsumidaie to discuss the unique challenges in clinical trial patient recruitment ? https://lnkd.in/drXNSS9e

ICT Spring 2024 is now live! ?? Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry