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Rivogenlecleucel

Hemoglobinopathy

Explore 26 assets in Hemoglobinopathy →
Floatz Rating
C38/100
Confidence
Indicative
v0.2
Sponsor
Bellicum Pharmaceuticals
Modality
Cell therapy
Development Phase
Phase 1/2
Status
Dormant
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
24High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT03733249CT.gov
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
—Terminated———
NCT02231710CT.gov
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
—Terminated———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Hemoglobinopathy

AssetSponsorPhaseRating
Rivogenlecleucel (this asset)Bellicum PharmaceuticalsP1/2C · 38
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 TranscriptionVertex Pharmaceuticals IncorporatedP3BB
HydroxyureaNational Institute of Blood and Marrow Transplant (NIBMT), PakistanP3BB
DeferasiroxNovartisP4BB
FludarabineMasonic Cancer Center, University of MinnesotaP2B
Pegyinterferon-Alfa-2AAzienda Ospedaliera V. CervelloP4B
BusulfanumBeam Therapeutics Inc.P2B
RibavirinAzienda Ospedaliera V. CervelloP4B
ThiotepaUniversity of California, San FranciscoP2B

+32 more in the Hemoglobinopathy cohort

Other indications for Rivogenlecleucel

IndicationSponsorPhaseRating
Acute Myeloid Leukemia—P2/3CCC · 48
Myelodysplastic Syndrome—P2/3CC · 44
Inborn Error Of Immunity—P1/2C · 39

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Rivogenlecleucel in Hemoglobinopathy. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/rivogenlecleucel-hemoglobinopathy

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