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AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE

Type 1 Gaucher Disease

AVROBIOCell therapyDormant
D
30/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
15/ 100
Competitive Position
95/ 100

Competitive context

0 Active assets in the Type 1 Gaucher Disease cohort.

Asset / SponsorPhaseRating
ISOFAGOMINE
Amicus Therapeutics
Phase 2BB
Elelyso
Pfizer
Phase 4BB
at2101
Amicus Therapeutics
Phase 1CC

Same molecule, other indications

AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE is also rated in:

beta thalassemiaDormant
B
Indicative ·
60 / 100
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beta-thalassemia majorDormant
CCC
Indicative ·
55 / 100
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Transfusion-dependent Beta-ThalassemiaDormant
CCC
Indicative ·
55 / 100
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