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Kln-1010

Hematologic Disease and Disorders

Kelonia Therapeutics, Inc.gene_therapyPhase 1Active
CC
43/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
28/ 100
Competitive Position
95/ 100

Competitive context

31 Active assets in the Hematologic Disease and Disorders cohort.

Asset / SponsorPhaseRating
Cyclophosphamide
Baptist Health South Florida
Phase 2/3BBB
Fludarabine
Baptist Health South Florida
Phase 2BB
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
Vertex Pharmaceuticals Incorporated
Phase 3BB
Busulphan
Baptist Health South Florida
Phase 2BB
Kogenate
Bayer
Phase 4BB

View all 50 →

Same molecule, other indications

Kln-1010 is also rated in:

neoplasmDormant
CC
Indicative ·
43 / 100
View asset →
vascular hemostatic diseaseDormant
CC
Indicative ·
43 / 100
View asset →
plasma cell myelomaDormant
CC
Indicative ·
43 / 100
View asset →

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