Deos Therapeutics is using leading edge science to develop gene therapies and antisense oligonucleotide therapies for patients with rare genetic conditions. Our team brings together proven methods in vector design, innovation in therapeutic delivery, and a partner ecosystem with unequalled experience in the scientific, regulatory, and commercial spaces to bring these therapies to patients.
LBSL is a rare, inherited leukodystrophy caused by variants in the DARS2 gene, leading to progressive white matter degeneration, motor dysfunction, and in some cases, cognitive decline—often beginning in childhood. There are currently no disease-modifying treatments available.