RAPA-501

RAPA-501 is an autologous, epigenetically reprogrammed hybrid T-cell therapy developed to treat amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The product is manufactured ex vivo from a patient’s own T-cells, which are collected via apheresis and then reprogrammed to yield a population enriched for both regulatory T-cell (TREG) and helper Th2 phenotypes. These cells express the transcription factors FOXP3 and GATA3, as well as surface markers CD39, CD73, and CD103. The dual anti-inflammatory phenotype of these cells enables them to suppress effector T-cell inflammatory molecules and central nervous system microglial inflammation. The goal of this approach is to restore immune balance in ALS patients by increasing functional regulatory immune activity, potentially slowing disease progression. Administration is via intravenous infusion; up to four infusions are given over 18 weeks in ongoing clinical trials[1][4][5][6][8].

Other names

Autologous hybrid TREG/Th2 cell therapyRAPA-501 Autologous T cellsRAPA501 Autologous T cellsRAPA 501 Autologous T cellsRAPA-501 T cell therapyRAPA501 T cell therapyRAPA 501 T cell therapy