RareMoon | Orphan Drug Regulatory Affairs

We are once again thrilled to announce that RareMoon has made significant progress last week in our mission to help our clients advance their orphan drug programs! Last week’s responses from the FDA included securing FOUR Rare Pediatric Disease Designations, a Fast Track Designation, an Orphan Drug Designation and an EMA Orphan Drug Designation!   This marks another breakthrough in our quest to develop and advance transformative therapies for rare diseases. We extend our deepest gratitude to our exceptional team and partners whose dedication made this accomplishment possible. Stay tuned for weekly updates as we continue to achieve these designations and push therapies to those patients who need it most. #RareDisease #Pharmaceuticals #HealthcareInnovation #OrphanDrugs #Biotechnology #LifeSciences

Conmpliments to another successful client 🚀 PentixaPharm AG has received Orphan Drug Designation from the European Medicines Agency for Pentixafor, a promising new therapy targeting the CXCR4 receptor in lymphoma. This milestone accelerates the path to providing crucial treatment options for patients. Kudos to the PentixaPharm AG team on this achievement! 👏 #HealthcareInnovation #Oncology #Lymphoma #PentixaPharm

Congratulations to our clients and team for securing both an ODD and RMAT designation this week! 🎉 RareMoon is proud to be part of this journey and look forward to continued success.👏 #ODD #RMAT #Innovation #Healthcare #Milestone

Innovative advancements in gene therapy treatments are emerging, driven by machine learning. Researchers at MIT and Harvard's Broad Institute have created a machine-learning model that enhances the precision of gene delivery systems. This new tool, Fit4Function, designs viral capsids with improved targeting accuracy for specific organs, which could transform how we treat various genetic conditions. The ability to predict these outcomes across different species is a game-changer, potentially leading to more effective and widely accessible gene therapies. #genetherapy #raredisease #healthcare #innovation #therapy https://lnkd.in/gTCPYpkn

👏 Another amazing weekly update from RareMoon! This week our clients have received more successful designations including three Rare Pediatric Disease Designations and one RMAT (Regenerative Medicine Advanced Therapy) designation. A huge thank you to our talented team, dedicated partners, and visionary clients for making this possible. Together, we're making a real difference in the lives of those who need it most. 🌟 #RareDisease #PediatricCare #RMAT #HealthcareInnovation #RegulatoryAffairs #LifeSciences #PatientCare

The FDA’s Center for Drug Evaluation and Research (CDER) is making strides in addressing rare diseases through various initiatives. With over 7,000 rare diseases affecting millions, CDER's Accelerating Rare disease Cures (ARC) Program is central to these efforts. Key initiatives include enhancing collaboration across FDA centers, developing the Rare Disease Innovation Hub, and implementing expedited review processes. These efforts aim to overcome the challenges of drug development for rare diseases, ensuring faster access to effective therapies for patients. #raredisease #FDA #CBER #healthcare #innovation Explore the full article: https://lnkd.in/dTacjaRM.

New client designation received! Actio Biosciences' ABS-0871 has received FDA Orphan Drug and Rare Pediatric Disease designations for the treatment of Charcot-Marie-Tooth disease subtype 2C (CMT2C), a rare and debilitating condition. This recognition underscores the potential impact of ABS-0871 in addressing unmet medical needs for those affected by this disease. These designations not only accelerate the development of this promising therapy but also bring hope to patients and families facing CMT2C. Congratulations to Actio as well as our team at RMC for the successful designations!

🔬 Breakthrough at Lawson Health Research Institute!    Researchers have developed a novel AI-driven technology, EpiSign, capable of diagnosing rare diseases and prenatal exposure-related birth disorders through a simple blood test. This pioneering method measures a patient's epigenome, providing accurate diagnoses for over 100 genetic diseases and identifying conditions like fetal valproate syndrome. This advancement holds significant promise for improving patient care and treatment outcomes globally.   Read more: https://lnkd.in/gumeWr77

📒 Noteworthy update from the FDA! The launch of the Rare Disease Innovation Hub marks a major step forward in developing treatments for rare diseases. This initiative aims to harness advances in drug target identification and gene therapy to address high unmet medical needs. The Hub will focus on collaboration, regulatory science, and community engagement to expedite the approval of safe and effective treatments for the 30 million Americans affected by rare diseases. This initiative underscores the FDA's commitment to improving patient outcomes. Read more: https://lnkd.in/gRAguC3F

🔬 Interesting Discovery in Genetics! Researchers from the Wellcome Sanger Institute, the University of Cambridge, and AstraZeneca have discovered that "gene misbehavior" is surprisingly common in healthy individuals. This phenomenon, where inactive genes become active, affects over half of our genes. The findings, published in the American Journal of Human Genetics, could revolutionize precision medicine by enhancing our understanding of genetic expression and its role in health and disease. Read more about this groundbreaking study here: https://lnkd.in/gwe3RCdE #genetics #innovation #genetherapy