Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BIVV003 · 1 trial · 1 indication
The percentage of participants who are alive at post-transplantation Day 100 will be calculated using the Kaplan-Meier estimate.
The percentage of participants who are alive at post-transplantation Week 52 will be calculated using the Kaplan-Meier estimate.
The percentage of participants who are alive at post-transplantation Week 104 will be calculated using the Kaplan-Meier estimate.
Successful engraftment is defined by absolute neutrophil count (ANC) greater than or equal to \>=500 cells/microliter (mL) for 3 consecutive days.
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
An SAE is any untoward medical occurrence that at any dose: Results in death, in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
| Arm | Type | Description |
|---|---|---|
| BIVV003 | EXPERIMENTAL | Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. BIVV003 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Hematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus. |
| Name | Type | Description |
|---|---|---|
| Plerixafor | BIOLOGICAL | Plerixafor subcutaneous injection will be administered prior to apheresis. |
| Busulfan | DRUG | Busulfan IV infusion will be administered as myeloablative conditioning therapy. |
| BIVV003 | GENETIC | BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan. |
Inclusion Criteria * Ages 18 to 40 * Confirmation of sickle cell disease (SCD) diagnosis (HbSS or HbS\[beta\]0 genotype) * Severe SCD, defined as having 1 or more of the following manifestations: Clinically significant neurologic event (example \[e.g.\], stroke) or any neurological deficit lasting ...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, Rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
BIVV003 is an investigational gene therapy being developed by Sangamo Therapeutics, Inc. for the treatment of severe sickle cell disease. It is designed as an autologous hematopoietic stem cell transplant, meaning a patient's own stem cells are modified outside the body and then returned to the patient. It is in Phase 1 clinical development.
BIVV003 is being studied for use in patients with severe sickle cell disease. The Phase 1 trial enrolled patients 18 years and older with severe sickle cell disease in the United States. It is an autologous hematopoietic stem cell transplantation approach, so the modified cells come from the patient rather than a donor.
BIVV003 is being developed by Sangamo Therapeutics, Inc., which trades on the Nasdaq under the ticker SGMO. Sangamo is the sponsor of the clinical program evaluating the gene therapy in severe sickle cell disease.
BIVV003 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The Phase 1 study of BIVV003 has been completed, and no trials of the therapy are currently listed as active.
BIVV003 has been evaluated in one clinical trial, NCT03653247, titled a study to assess the safety, tolerability, and efficacy of BIVV003 for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease. That Phase 1 trial is completed, enrolled 7 patients, was conducted in the United States, and was uncontrolled.