Rethymic
allogeneic processed thymus tissue
Species
no relevantRat
Longest study
39 wk
Genotox
Not conducted Not conducted
Carcinogenicity
Not conducted Not conducted
Repro
Not conducted Not conducted
First dose
Not conducted Not stated publicly
Speciesopen answer page →
Species
No relevant species; nude rat pharmacology studies only, no tox
“Based on the acceptable clinical safety profile of RVT-802 and the lack of relevant animal species/models, no nonclinical toxicology studies with RVT-802 were performed.”
Why that species
Nude rats used to model engraftment and thymopoiesis of allogeneic thymus; no relevant tox species
“Studies were conducted in nude rats to to identify potentially optimal culture conditions of allogeneic neonatal thymus that result in engraftment and development of an endogenous T cell population”
Surrogate molecule
No surrogate; human product tested in athymic nude rats (pharmacology only)
“Allogeneic Thymus Transplantation in Nude Rats RVT-802-001”
Study lengthopen answer page →
Longest pivotal study
39 weeks
“thymopoiesis was observed at the pre-specified time points (1 and 9 months”
Pivotal GLP studies
No toxicology studies; pharmacology only (in vitro thymus organ culture, nude rat transplants to 9 months)
“no nonclinical toxicology studies with RVT-802 were conducted.”
Route in tox
Implantation (nude rat; clinical use is quadriceps muscle implant)
“RVT-802 is surgically implanted into the quadriceps muscle.”
Skipped studiesopen answer page →
Genotoxicity
Not conducted
“No genotoxicity studies were conducted.”
Carcinogenicity
Not conducted; published mouse thymus-transplant literature reviewed
“No carcinogenicity/tumorigenicity studies were conducted.”
Reproductive & developmental
Not conducted; published thymectomy literature suggests minimal risk
“No DART studies were conducted.”
Safety pharmacology
Not conducted
“No safety pharmacology studies were conducted.”
First doseopen answer page →
First-in-human starting dose
Not stated in public reviews
NOAEL / key findings
No NOAEL; no toxicology studies; dose range from clinical use
“The recommended dose range is to 22,000 mm2”
Risksopen answer page →
Immune & cytokine safety
Donor-derived T cells declined as endogenous recipient T cells developed in rats; no dedicated immunotoxicity study
“there was a time-dependent decrease in donor-derived T cells in the peripheral blood as the endogenous T cell population developed.”
Biodistribution & persistence
No cell distribution studies conducted
“No cell distribution studies were conducted.”
Modality-specific risk
Alloreactivity and tumorigenicity risk assessed only through literature; considered minimal
“these data combined with the existing clinical safety data for RVT-802, indicate that the risk of carcinogenicity after RVT-802 transplantation in humans appears to be minimal.”