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Prime Medicine Reverses Course, Seeks FDA Approval for Gene Therapy in CGD After Shelving

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Prime Medicine announced on March 3, 2026, it will seek FDA approval for its prime editing gene therapy, previously administered to only two patients in a Phase 1/2 trial for chronic granulomatous disease (CGD) 1 3 . The therapy uses prime editing, a CRISPR-based technology developed by David Liu, to insert two missing DNA letters into blood cells, addressing CGD which causes life-threatening infections and inflammation 1 . This biologics license application (BLA) follows 'breakthrough' Phase 1/2 data for PM359 and aims for accelerated approval after final FDA alignment 3 . The move tests the FDA's commitment to speeding gene-editing treatments amid recent scrutiny over neurological gene therapies 1 . Sources: 1. https://www.statnews.com/2026/03/03/prime-medicine-seeks-fda-approval-cgd-disease-gene-editing-treatment/ 3. https://www.stocktitan.net/sec-filings/PRME/8-k-prime-medicine-inc-reports-material-event-d1d4c545da83.html

FDA Accelerates Rare Disease Drug Approvals with New Frameworks and Record 2025 Approvals

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3T Biosciences Appoints Rebeca Villarreal-Barragan, MBA, as Head of Clinical Operations

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Orca Bio Presents Clinical Data on High-Precision Cell Therapies at 52nd EBMT Annual Meeting

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Neko Health Appoints Dr. Sunita Mishra as Chief Medical Officer

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Celldex Reports Strong Retreatment Results With Barzolvolimab In Cold Urticaria and Symptomatic Dermographism

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North Carolina Exits Job Development Investment Grants for Two Gene Therapy Companies in Durham

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UniQure's Huntington's Gene Therapy AMT-130 Faces FDA Roadblock, Requiring New Trial

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Aardvark Therapeutics Stock Plunges 54% Following Phase 3 Trial Pause

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Ascendis Pharma Enters Achondroplasia Market with FDA Accelerated Approval of Weekly Drug YUVIWEL

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RFK Jr. Reshuffles HHS Leadership Amid Midterm Focus

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CHMP Recommends Moderna's mCombriax Flu-COVID Vaccine for Adults 50+

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Biocytogen Announces Clinical Milestone with First Patient Dosed in Phase 1 Trial of IDEAYA's First-in-Class B7H3/PTK7 Bispecific TOP1 ADC IDE034

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