REGENECYTE® is StemCyte's FDA-licensed allogeneic hematopoietic progenitor cell therapy derived from cord blood, approved under US License 2280 on November 20, 2024. It is the first commercial HPC, Cord Blood product indicated for use in unrelated donor hematopoietic progenitor cell transplantation. REGENECYTE® also holds FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Long COVID, and FDA cleared a compassionate-use Expanded Access program in January 2026.
REGENECYTE®®
(HPC, Cord Blood)
An allogeneic cord blood hematopoietic progenitor cell therapy indicated for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.
Full Prescribing Information (FDA)Indications & Usage
REGENECYTE® is indicated for use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.
The risk-benefit assessment for an individual patient depends on patient characteristics including disease, stage, and risk factors; characteristics of the graft; and other available treatments or types of hematopoietic progenitor cells.
Mechanism of Action
Hematopoietic stem/progenitor cells from REGENECYTE® migrate to the bone marrow where they divide and mature. The mature cells are released into the bloodstream, partially or fully restoring blood counts and immune function.
In patients with enzymatic abnormalities due to certain severe storage disorders, mature leukocytes from transplantation may synthesize enzymes that circulate and improve cellular functions of native tissues.
Dosage & Administration
For intravenous use only. Do not irradiate. The recommended minimum dose is 2.5 × 10⁷ nucleated cells/kg at cryopreservation. Matching for at least 4 of 6 HLA-A, HLA-B, and HLA-DRB1 alleles is recommended. Infuse over 15 to 60 minutes. Monitor the patient during and for at least six hours after administration.
Clinical Studies
The effectiveness of REGENECYTE® was demonstrated across three studies: the prospective COBLT study (Study 1, n=324), retrospective docket and public data (Study 2, n=1,299), and a retrospective review of the REGENECYTE® database (Study 3, n=54).
| Endpoint | COBLT (n=324) | Docket Data (n=1,299) | REGENECYTE® (n=54) |
|---|---|---|---|
| Neutrophil Recovery (Day 42) | 76% (71–81%) | 77% (75–79%) | 91% (81–97%) |
| Platelet Recovery d100 (20K) | 57% (51–63%) | — | 72% (58–83%) |
| Platelet Recovery d100 (50K) | 46% (39–51%) | 45% (42–48%) | 73% (54–88%) |
| Median Neutrophil Recovery | 27 days | 25 days | 22 days |
| Median Platelet Recovery (20K) | 90 days | — | 50 days |
| Median Platelet Recovery (50K) | 113 days | 122 days | 64 days |
95% CI shown in parentheses. Source: FDA Prescribing Information, US License Number 2280.
Warnings and Precautions
REGENECYTE® carries a Boxed Warning for fatal infusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure. Administration should occur in a setting equipped to manage allogeneic transplantation complications. The following risks apply.
Adverse Reactions
The most common infusion-related adverse reactions (≥5%) from the pooled safety population are hypertension, vomiting, nausea, bradycardia, and fever. Mortality from all causes at 100 days post-transplant was 25%. Among the 54 patients in Study 3 (REGENECYTE®), 7.4% experienced an infusion reaction.
| Reaction | Any Grade | Grade 3–4 |
|---|---|---|
| Any reaction | 65.4% | 27.6% |
| Hypertension | 48.0% | 21.3% |
| Vomiting | 14.5% | 0.2% |
| Nausea | 12.7% | 5.7% |
| Sinus bradycardia | 10.4% | 0 |
| Fever | 5.2% | 0.2% |
| Sinus tachycardia | 4.5% | 0.2% |
| Allergy | 3.4% | 0.2% |
| Hypotension | 2.5% | 0 |
| Hemoglobinuria | 2.1% | 0 |
| Hypoxia | 2.0% | 2.0% |
Infusion-Related Adverse Reactions. Reactions occurring in ≥1% of infusions in the COBLT Study (n=442 infusions). Source: FDA Prescribing Information, Table 1.
Study 3 (REGENECYTE®), voluntary reports
Among 54 patients who received REGENECYTE® at the recommended TNC dose, voluntary reports for 388 infusions yielded information on infusion reactions. 7.4% of patients (n=4) experienced an infusion reaction. Most common reactions occurring in >1% of patients: hypertension (3.7%), nausea (3.7%), vomiting (1.9%), hypotension (1%), and chest pain (1%).
Report Adverse Reactions
To report SUSPECTED ADVERSE REACTIONS, contact StemCyte at 1-626-646-2485 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
All clinical data from FDA Full Prescribing Information: REGENECYTE® (HPC, Cord Blood)
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