February 24, 2025

FDA Lifts Clinical Hold on Entrada’s Duchenne Muscular Dystrophy Drug ENTR-601-44 After Two Years

2 months ago talkbio0Tagged ELEVATE-44, ENTR-601, Entrada, exon, Muscular Dystrophy, Duchenne, Phase 1b Trial, skipping, Study on Hold, United States Food and Drug Administration

Duchenne muscular dystrophy, ENTR-601-44, Entrada Therapeutics, FDA, clinical hold, exon 44 skipping, ELEVATE-44-102, Phase 1b trial

Roche rolls out new DNA ‘sequencing-by-expansion’ approach

2 months ago talkbio0Tagged DNA, Roche (company)

Roche (company), DNA

FDA Approves Mirum’s Ctexli as First Treatment for Rare Lipid Disorder CTX

2 months ago talkbio0Tagged Approved, bile acid therapy, cerebrotendinous xanthomatosis, chenodeoxycholic acid, Ctexli, CTX, Lipoidosis, Metabolic Diseases, Mirum, United States Food and Drug Administration, Xanthomatosis, Cerebrotendinous

Ctexli, chenodiol, cerebrotendinous xanthomatosis (CTX), FDA approval, Mirum Pharmaceuticals, rare metabolic disorder, lipid storage disease, bile acid therapy

FDA Reverses Course, Rehires Hundreds of Recently Fired Probationary Workers

2 months ago talkbio0Tagged Department of Health and, federal agencies, HHS, mass layoffs, probationary workers, Transplant Registry Unified Management Program, United States Food and Drug Administration

FDA, probationary workers, rehiring, mass layoffs, federal agencies, Trump administration, Department of Health and Human Services (HHS)

Bluebird Bio’s Fall from Grace: Gene Therapy Pioneer Sells for $29M After $10B Valuation

2 months ago talkbio0Tagged Acquisition, Capital, Carlyle, DKK1 gene, Family Turdidae (organism), Financial Stress, gene therapy, Industry, Private Equity

Bluebird Bio, gene therapy, private equity, Carlyle, SK Capital, financial challenges, acquisition, biotech industry