News

Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
InvestorsHub on MSN9d
Precision BioSciences Shares Surge Following FDA Rare Pediatric Disease Designation
Shares of Precision BioSciences, Inc. (NASDAQ:DTIL) jumped 17% on Wednesday after the company announced that its gene therapy ...
Muscular Dystrophy News8d
Precision’s gene-editing therapy gets FDA’s rare pediatric disease status
The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...
TMCnet10d
Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome
The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines ...
Minovia Therapeutics Announces FDA Fast Track and Rare Pediatric Disease Designations for MNV-201 in Pearson Syndrome
Regulatory Milestone Underscores Urgency and Promise of First-in-Class Mitochondrial Cell TherapyHaifa, ISRAEL, June 30, 2025 (GLOBE NEWSWIRE) -- Minovia Therapeutics Ltd. (“Minovia” or the “Company”) ...
Contract Pharma9d
Precision BioSciences Gains FDA Rare Pediatric Disease Designation for PBGENE-DMD
Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).
Minovia Gets FDA Designations Supporting Development of Pearson Syndrome Treatment
Minovia Therapeutics said Monday that the Food and Drug Administration granted fast-track and rare-pediatric-disease designations to its lead investigational compound. The drug, MNV-201, is in Phase 2 ...
Health Affairs7dOpinion
Congress Should Remove The Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It
Expansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs ...
Investing.com South Africa9d
Precision BioSciences stock jumps after FDA grants rare pediatric designation
Investing.com -- Precision BioSciences, Inc. (NASDAQ: DTIL) stock surged 17% on Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric ...
Parliamentarian reverses course on rare disease drug price provision in tax bill
The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for ...
Novadip receives RMAT designation for NVD003, its regenerative treatment for congenital pseudarthosis of the tibia
Novadip is developing NVD003, an autologous therapy derived from adipose stem cells, as a potential single treatment to save ...
The Bakersfield Californian10d
Longeveron® Announces Completion of Enrollment of Pivotal Phase 2b Clinical Trial Evaluating ...
Top-line trial results are anticipated in the third quarter of 2026, after the final follow-up at 12-monthsLaromestrocel Biological License Application (BLA) submission for full traditional approval f ...