CK0803

CK0803 is a novel allogeneic cell therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Based on the search results, I can provide comprehensive information about this investigational therapy. ## Description CK0803 is a novel, allogeneic, neurotrophic T regulatory (Treg) cell therapy derived from clinical-grade umbilical cord blood units. It is manufactured using Cellenkos' proprietary CRANE technology, which generates disease-specific cell products. CK0803 Tregs have high cell surface expression of CD11a and CXCR3, which allows them to leverage the CXCR3/CXCL10 axis to engage with inflamed microglia in patients with ALS. This mechanism targets neuroinflammation, a key pathological feature in ALS and other neurodegenerative disorders. The therapy does not require HLA or ABO matching to recipients, and multiple doses can be manufactured from a single umbilical cord blood unit. The final product is cryopreserved until ready for use at the point of care. In clinical trials, the treatment protocol includes four weekly infusions followed by five monthly infusions. ## Development Status CK0803 received FDA clearance for its Investigational New Drug (IND) application in September 2022. The first patient was dosed in the REGALS trial (NCT05695521) in May 2023. As of March 2024, the trial had progressed to its second cohort following a positive recommendation from the Data Safety Monitoring Board (DSMB), which found no serious adverse events associated with the therapy. The REGALS trial consists of a Phase 1 safety run-in study of 6 individuals with ALS, followed by a Phase 1b randomized, double-blind, placebo-controlled trial with an additional 60 patients. The primary outcome measures include treatment-limiting toxicities and a combined assessment of function and survival (CFAS) based on survival time and change in ALS Functional Rating Scale-Revised (ALSFRS-R) score. In addition to ALS, CK0803 is also being investigated in preclinical studies for Parkinson's disease and Chronic Inflammatory Demyelinating Polyradiculoneuropathy.