Beam-101

Hemoglobinopathy

Beam Therapeutics Inc.Active

30/ 100

Provisional rating · Methodology v0.2

Rating breakdown

25 Active assets in the Hemoglobinopathy cohort.

Asset / Sponsor	Phase	Rating
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 3	BB
Hydroxyurea National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Phase 3	BB
Deferasirox Novartis	Phase 4	BB
Fludarabine Masonic Cancer Center, University of Minnesota	Phase 2	B
Ribavirin Azienda Ospedaliera V. Cervello	Phase 4	B

View all 41 →

Beam-101 is also rated in:

sickle cell diseaseDormant

Indicative ·

41 / 100

Order a full diligence report on this asset.

Submit your data room for a Verified Rating. The public-data rating remains visible alongside.