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Exagamglogene autotemcel, sold
under the
brand name Casgevy, is a gene
therapy used for the
treatment of
sickle cell
disease and transfusion-dependent...
-
Lovotibeglogene autotemcel, sold
under the
brand name Lyfgenia, is a
lentiviral gene
therapy used for the
treatment of
sickle cell disease. The most common...
-
Atidarsagene autotemcel, sold
under the
brand name
Libmeldy among others, is a gene
therapy treatment for
metachromatic leukodystrophy developed by Orchard...
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Elivaldogene autotemcel, sold
under the
brand name Skysona, is a gene
therapy used to
treat cerebral adrenoleukodystrophy (CALD). It was
developed by Bluebird...
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Betibeglogene autotemcel, sold
under the
brand name Zynteglo, is a gene
therapy for the
treatment for beta thal****emia. It was
developed by
Bluebird Bio...
-
positive results tend to
occur with very
young people.
Betibeglogene autotemcel, sold
under the
brand name Zynteglo, is a gene
therapy for the treatment...
- the
United States for its one-time
treatment Zynteglo (betibeglogene
autotemcel). Gene
editing therapies aimed at
increasing fetal hemoglobin production...
-
neurological symptoms,
plasma VLCFA levels remained elevated.
Elivaldogene autotemcel is
pending authorization by the
European Commission as of May 2021[update]...
-
Crizanlizumab B06AX02
Betibeglogene autotemcel B06AX03
Voxelotor B06AX04
Mitapivat B06AX05
Exagamglogene autotemcel "ATC (Anatomical
Therapeutic Chemical...
-
Award (FOYA)
award in 2022. The company’s lead program,
exagamglogene autotemcel, or exa-cel (formerly CTX001), was
granted regulatory approval by the...
