Floatz logo

Busulfanum

Immunodeficiency Disease

Explore 9 assets in Immunodeficiency Disease →
Floatz Rating
BB66/100
Confidence
Indicative
v0.2
Sponsor
University of California, San Francisco
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
51High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT03609840CT.gov
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
—Completed———
NCT02660281CT.gov
URMC Related Haplo-identical Donor BMT
—Completed———
NCT03016806CT.gov
Umbilical Cord Blood Transplantation From Unrelated Donors
—Recruiting———
NCT01666080CT.gov
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
—Recruiting———
NCT01049854CT.gov
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
—Completed———
NCT00744692CT.gov
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
—Completed———
NCT00801931CT.gov
Double Cord Blood Transplant for Patients With Malignant and Non-malignant Disorders
—Terminated———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Immunodeficiency Disease

AssetSponsorPhaseRating
Busulfanum (this asset)University of California, San FranciscoP2BB · 66
MelphalanSidney Kimmel Comprehensive Cancer Center at Johns HopkinsP2BB
ThiotepaUniversity of California, San FranciscoP2BB
FludarabineSidney Kimmel Comprehensive Cancer Center at Johns HopkinsP2BB
Human ImmunoglobulinsGrifols Therapeutics LLCP3B
AlemtuzumabSidney Kimmel Comprehensive Cancer Center at Johns HopkinsP2B
CyclophosphamideSidney Kimmel Comprehensive Cancer Center at Johns HopkinsP2B
Anti-Human T-Lymphocyte Immunoglobulin, RabbitNew York Medical CollegeP2B
TacrolimusSidney Kimmel Comprehensive Cancer Center at Johns HopkinsP2B

+12 more in the Immunodeficiency Disease cohort

Other indications for Busulfanum

IndicationSponsorPhaseRating
Myelodysplastic/Myeloproliferative Neoplasm—P2/3BBB · 76
Graft Versus Host Disease—P4BBB · 74
Disease Related To Hematopoietic Stem Cell Transplant—P3BBB · 74

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. Busulfanum in Immunodeficiency Disease. 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/busulfanum-immunodeficiency-disease

Need depth?

Order a full diligence report on this asset.

Order report →

Are you the sponsor?

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

Contact →