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Hope for sickle cell disease patients as gene therapy gets more approvals SHEM OIRERE
January 19, 2024
After years of exploration on gene therapy for sickle cell disease (SCD) and transfusion-dependent β-thalassemia.
FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years FDA
December 20, 2021
FDA has granted accelerated approval for Oxbryta (voxelotor) tablets to treat sickle cell disease in pediatric patients aged four up to 11 years. FDA had previously granted accelerated approval for Oxbryta for patients aged 12 years and older with sickle
Orphan Drug Designation Granted for Plasma-Derived Hemopexin Therapy for Sickle Cell Disease americanpharmaceuticalreview
November 11, 2020
CSL Behring announced its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the ...
Cyclerion Announces Sickle Cell Disease Study Results americanpharmaceuticalreview
October 22, 2020
Cyclerion Therapeutics announced top-line results from its STRONG-SCD study of olinciguat, an investigational, orally-administered, once daily, vascular sGC stimulator for the potential treatment of sickle cell disease (SCD).
Fulcrum to Initiate FTX-6058 Trial for Sickle Cell Disease americanpharmaceuticalreview
October 12, 2020
Fulcrum Therapeutics announced the Investigational New Drug application (IND) is now in effect for its Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease.
FDA grants GlycoMimetics rare paediatric disease designation for rivipansel for treatment of sickle cell disease pharmaceutical-business-review
October 10, 2020
GlycoMimetics announced that the U.S. Food and Drug Administration (FDA) has granted the Company a Rare Pediatric Disease designation for rivipansel for the treatment of sickle cell disease in patients 18 years old and younger.
Vertex, CRISPR Therapeutics’ sickle cell gene therapy wins PRIME designation pharmatimes
September 27, 2020
Vertex Pharmaceuticals and CRISPR Therapeutics have been awarded a Priority Medicines (PRIME) designation from the European Medicines Agency for their CRISPR/Cas9 gene therapy, designed to treat severe sickle cell disease (SCD).
FORMA Therapeutics receives FDA orphan drug designation for FT-4202 to treat sickle cell disease pharmaceutical-business-review
April 20, 2020
FORMA Therapeutics, a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers.
Fulcrum Completes IND-enabling Safety Studies for Sickle Cell Disease Treatment americanpharmaceuticalreview
April 14, 2020
Fulcrum Therapeutics announced it is on track for submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for FTX-6058 ...
FDA approves novel treatment to target abnormality in sickle cell disease fda
November 26, 2019
The U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.

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