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FDA approves world-first gene editing treatment: Sickle cell therapy gives hope to 100,000 Americans with incurable disease - but it ll cost millions of dollars per DOSE

In a ground-breaking decision, the Food and Drug Administration approved the first gene therapies to treat people with sickle cell disease

United statesSan antonioUnited kingdomRegina hartfieldSamarth kulkarniJulie kanterLos angelesReshma kewalramaniVertex pharmaceuticalsSickle cell disease association of america incDrug administrationBoston based vertex pharmaceuticalsCrispr therapeuticsSwitzerland based crispr therapeuticsNobel prizeBluebird bio

First Time Ever CRISPR Gene-Editing Therapy Approved in the US

First Time Ever CRISPR Gene-Editing Therapy Approved in the US
theepochtimes.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from theepochtimes.com Daily Mail and Mail on Sunday newspapers.

Lyfgenia casgevyRobert chiarlePeter marksAkshay sharmaJulie kanterTechnology suggestive of cancer risksDrug administrationJude children research hospitalUniversity of alabamaLifespan comprehensive sickle cell centerBiologics evaluationBluebird bioNew treatment options currentlyBone marrow transplantationCellular therapyJude children

Lovo-cel gene therapy transformative for sickle cell disease

Lovo-cel gene therapy transformative for sickle cell disease
healio.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from healio.com Daily Mail and Mail on Sunday newspapers.

Julie kanterMindy valcarcelBymark leiserUniversity of alabama at birminghamSickle cell clinicBluebird bio

Long-term Follow-up Data From bluebird s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events -December 09, 2023 at 11:32 am EST

Oral presentation to include data on 47 patients through five years of follow-up Endpoints of sVOE-CR and VOE-CR achieved in 94% and 88% of evaluable patients respectively 100% of adolescents .

United statesJulie kanterRichard colvinDivision of hematologyExchange commissionDrug administrationAmerican society of hematologyAmerican societyAnnual meetingAlabama birmingham adult sickle cell clinicMarketing long term follow up studyPrescribing informationPrivate securities litigation reform actAnnual reportQuarterly reportsCurrent reports

Bluebird gene therapy, now approved for sickle cell, shows durable benefit in study update

BioPharma

United statesJulie kanterAlexis thompsonDrug administration onUniversity of alabamaVertex pharmaceuticalsAmerican society of hematologyDrug administrationAmerican societyRich colvinLike lyfgeniaGene therapy

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