Study on Hold – Talk Bio

FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose

2 days ago talkbio0Tagged C3 complement inhibitor, Capillary Leak Syndrome, Dosage, gene therapy, Glycogen Storage Disease Type IIb, Individual Adjustment, Phase 2 Clinical Trials, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial

FDA Delays Draw Contrasting Responses: Neurizon Cites Agency Strain, Omeros Takes Softer Tone

5 days ago talkbio0Tagged agency staffing shortages, Amyotrophic Lateral Sclerosis, delays, narsoplimab, NUZ-001, Omeros Corporation, regulatory review, Study on Hold, Transplantation, United States Food and Drug Administration

FDA delays; Neurizon Therapeutics; Omeros Corporation; regulatory review; NUZ-001; narsoplimab; ALS; transplant drugs; agency staffing shortages; clinical hold

Sarepta Therapeutics Faces FDA Crackdown after Third Patient Death; George Tidmarsh Named Top FDA Drug Regulator; Multiple Drugs Rejected; Pharma Invests Billions in US Manufacturing

1 month ago talkbio0Tagged drug rejection, ELEVIDYS, gene therapy, George Tidmarsh, Liver Failure, Acute, pharmaceutical manufacturing, reshoring, Study on Hold, United States Food and Drug Administration

Sarepta Therapeutics; FDA; gene therapy; Elevidys; acute liver failure; clinical hold; George Tidmarsh; drug rejection; reshoring; pharmaceutical manufacturing

Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy

1 month ago talkbio0Tagged AAVS1 gene, ELEVIDYS, gene therapy, Liver Failure, Acute, Muscular Dystrophies, Limb-Girdle, Patient Death, Safety, Study on Hold, United States Food and Drug Administration, vector

Sarepta Therapeutics; limb-girdle muscular dystrophy; SRP-9004; gene therapy; patient death; acute liver failure; FDA clinical hold; AAV vector; Elevidys; safety concerns

FDA Places Clinical Hold on Five Gilead HIV Studies Due to Safety Concerns

2 months ago talkbio0Tagged Biktarvy, CD4 + T-cell, Clinical Trials, Gilead Sciences, GS-1720, GS-4182, HIV, Lenacapavir, Lymphocyte Count measurement, safety signal, Study on Hold, United States Food and Drug Administration

FDA; Gilead Sciences; HIV; clinical hold; GS-1720; GS-4182; clinical trials; safety signal; CD4+ T-cell; lymphocyte count; Biktarvy; lenacapavir

Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold

3 months ago talkbio0Tagged acute systemic infection, Adverse event, Capillary Leak Syndrome, gene therapy, Glycogen Storage Disease Type IIb, Patient Death, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; FDA; patient death; capillary leak syndrome; acute systemic infection; RP-A501; adverse event