🧬 This Week in Rare Diseases: FDA Approvals, Gene Therapies, Global Screening Expansions and More!
This week’s Rare Diseases roundup delivers pivotal news from across the globe — including FDA approvals, cutting-edge gene therapy developments, major funding announcements, and international efforts to expand early diagnosis. Whether you’re tracking novel RNA-based platforms or following regulatory progress, these updates will keep you informed and ahead in the rare disease landscape.
🔬 Key highlights:
🇬🇪 Georgia Expands Access to Cystic Fibrosis Care
The Georgian government adds inhalation therapies, antibiotics, vitamins, and hospital care to its rare disease program, with funding reaching 55 million GEL.
đź“š NORD Launches Free Clinical Data Literacy Course
In partnership with the FDA and C-Path, NORD debuts “From Records to Research,” empowering patients and caregivers to interpret clinical data and engage in research.
🧪 World’s First Gene Therapy for p47-CGD Delivered at GOSH
UCL and GOSH pioneer the first-ever gene therapy for p47 Chronic Granulomatous Disease — developed, manufactured, and administered under one roof. The first patient, 19-year-old Remi, reports a dramatic improvement in quality of life.
🧬 BeginNGS Launches in Qatar for Newborn Genomic Screening
BeginNGS partners with Sidra Medicine to screen newborns for over 1,000 treatable rare diseases, accelerating early intervention across the region.
đź’° Arnatar Therapeutics Debuts with $52M for RNA Drug Development
Emerging from stealth, Arnatar’s platform (DARGER) integrates siRNA and ASO to treat previously untreatable diseases. Its lead asset, ART4, targets Alagille syndrome.
âś… FDA Approves Dawnzera for Hereditary Angioedema Prevention
Ionis’ donidalorsen, now approved as Dawnzera, is the first RNA-targeted prophylactic for HAE — reducing attack rates by 94 percent at one year. Orsini is named exclusive pharmacy partner.
đź“„ INOVIO Begins BLA Submission for INO-3107 in RRP
INOVIO initiates rolling BLA submission for INO-3107, targeting recurrent respiratory papillomatosis. With Breakthrough Therapy designation, full FDA acceptance is expected by end of 2025.
📢 Stay Ahead in Rare Disease Research!
âś… Like, share, and subscribe for weekly updates on orphan drugs, rare conditions, and clinical trial breakthroughs
#RareDiseases #GeneTherapy #OrphanDrugs #FDAApproval #NewbornScreening #RNAtherapeutics #CysticFibrosis #p47CGD #HAE #AlagilleSyndrome #INOVIO #HealthcareInnovation #LucidQuest #ClinicalResearch #BeginNGS #RareDiseaseAwareness