🧬 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.
📚 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.
💰 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.
✅ 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.
📄 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!
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