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PYRIDOXAL ISONICOTINOYL HYDRAZONE

Beta-Thalassemia

Case Western Reserve UniversitySmall moleculePhase 2Dormant
CCC
55/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
40/ 100
Competitive Position
81/ 100

Competitive context

17 Active assets in the Beta-Thalassemia cohort.

Asset / SponsorPhaseRating
DESFERRIOXAMINE B
Ain Shams University
Phase 4BBB
Deferasirox
Kevin H.M. Kuo, MD, MSc, FRCPC
Phase 4BBB
Hydroxyurea
National Heart, Lung, and Blood Institute (NHLBI)
Phase 2/3BB
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Daniel Bauer
Phase 3BB
Deferiprone
Carelon Research
Phase 4BB

View all 39 →

Same molecule, other indications

PYRIDOXAL ISONICOTINOYL HYDRAZONE is also rated in:

Anemia (Iron-Loading)Dormant
CCC
Indicative ·
55 / 100
View asset →
Hematologic DiseasesDormant
CCC
Indicative ·
55 / 100
View asset →
HemoglobinopathiesDormant
CCC
Indicative ·
55 / 100
View asset →

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