LBSL is caused by biallelic variants in the DARS2 gene, which encodes mitochondrial aspartyl-tRNA synthetase. The complete etiology of LBSL is yet unclear; however, loss of function variants may impair mitochondrial protein synthesis resulting in select neuronal loss and progressive white matter damage.
using AAV vectors to deliver a healthy copy of DARS2 directly into the Central Nervous System
to target dosing to the CNS and for minimally invasive delivery
to assess response and guide dosing in human trials
Restoration of DARS2 expression in key CNS regions, reduced cortical loss, and improved motor phenotypes in mouse models
Normalization of lactate levels and increased neurite outgrowth in patient derived neurons
IND submission for DEOS-101 planned for early 2027
🧪 In vivo data show sustained transgene expression in mice
🧬 Reduction of pathological markers (e.g., Lactate) in disease models
🧠Survival, ataxia and gait rescue observed in LBSL mouse models