deoxycytidine + deoxythymidine

deoxycytidine + deoxythymidine is an investigational combination therapy consisting of two small molecule nucleosides being developed by Suneet Agarwal at Boston Children's Hospital for the treatment of telomere biology disorders (TBDs). TBDs are rare, inherited conditions characterized by critically short telomeres, which lead to cellular senescence and organ failure, most commonly presenting as bone marrow failure. The therapy is based on research identifying thymidine nucleotide metabolism as a limiting factor in human telomere maintenance. By supplementing these nucleosides orally, the treatment aims to increase the available nucleotide pool to support telomere elongation and cellular replication. It is currently being evaluated in a Phase 1 clinical trial (NCT06817590) to assess safety and tolerability.

Other names

dC + dTNucleoside Therapy (dC + dT)