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First CRISPR drug could be approved soon – DW – 11/02/2023

A CRISPR drug to treat sickle cell disease may be approved by the US drug regulator next month.

New yorkUnited statesHoward university centerDistrict of columbiaEdward ivyJames taylorGil wolfeGene technologySickle cell disease association of americaUniversity of buffaloVertex pharmaceuticalsDrug administrationVice chief medical officerSickle cell disease associationSickle cell disease

Potential cure for sickle cell disease raises few concerns for FDA panel

Vertex Pharmaceuticals has hailed its treatment as "transformative" for patients with sickle cell disease. The FDA will decide on approval soon.

United statesNicole verdunTaby ahsanDaniel bauerGil wolfeFrancis collinsKomudi singhJon lapookDavid altshulerVertex pharmaceuticalsGene therapies advisory committeeNational institutes of healthDrug administrationOffice of therapeutics productsNational institutesSaharan africa

argenx SE: argenx Highlights Data Evaluating VYVGART in Neuromuscular Autoimmune Disease at AANEM and MGFA Scientific Sessions

argenx SE: argenx Highlights Data Evaluating VYVGART in Neuromuscular Autoimmune Disease at AANEM and MGFA Scientific Sessions
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United statesUnited kingdomNoord hollandMjamesf howardGil wolfeDemyelinating polyneuropathyYuebing liA gordon smithPushpa narayanaswamiGlenn phillipsCynthia qiLuc truyenTom hughesBen petokJamesf howard jrAntoine azar

Sickle cell cure: FDA advisory committee considers 1st CRISPR gene editing treatment exa-cel

Sickle cell cure: FDA advisory committee considers 1st CRISPR gene editing treatment exa-cel
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United statesDaniel bauerFyodor urnovLakiea baileyStephanie krogmeierGil wolfeVertex pharmaceuticals incorporatedBiomedical sciencesDrug administrationDepartment of molecularUniversity of californiaUniversity at buffalo jacobs school of medicineVertex pharmaceuticalsSickle cell consortiumBlood disorders centerBoston based vertex pharmaceuticals

FDA considers first CRISPR gene editing treatment that may cure sickle cell - Boston News, Weather, Sports

(CNN) At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive<a class="excerpt-read-more" href="https://whdh.com/news/fda-considers-first-crispr-gene-editing-treatment-that-may-cure-sickle-cell/">Read More</a>

United statesDaniel bauerStephanie krogmeierLakiea baileyFyodor urnovGil wolfeDepartment of molecularUniversity of californiaVertex pharmaceuticalsSickle cell consortiumBiomedical sciencesBlood disorders centerVertex pharmaceuticals incorporatedDrug administrationUniversity at buffalo jacobs school of medicineCell biology