Cellenkos gets orphan drug status for off-the-shelf Treg in ALS

The U.S. FDA has granted orphan drug designation to Cellenkos’ allogeneic, off-the-shelf Treg cell therapy, CK0803, for amyotrophic lateral sclerosis.

CK0803 is an umbilical cord blood-derived cell therapy that consists of regulatory T-cells. The cells have a high cell surface expression of CD11a that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia in patients with ALS.

The results of a proof-of-concept compassionate use clinical study with 6 ALS patients was published earlier this year in NEJM Evidence, demonstrating that Cellenkos' Tregs can lead to durable slowing of the ALS disease progression. Preliminary data from the phase 1 trial of CK0803, shows disease slowing for both spinal and bulbar onset ALS, especially in patients that started treatment early on in their disease.