PYC-002

PYC-002 is an investigational precision RNA therapeutic developed by PYC Therapeutics for the treatment of Phelan-McDermid Syndrome (PMS). It utilizes a proprietary Phosphorodiamidate Morpholino Oligomer (PPMO) platform to target the *SHANK3* gene. PMS is a severe neurodevelopmental disorder typically caused by haploinsufficiency of the *SHANK3* gene, which is critical for neuronal function and synaptic plasticity. PYC-002 is an antisense oligonucleotide designed to increase the expression of the SHANK3 protein from the remaining functional allele, thereby restoring normal protein levels and rescuing neuronal function. The drug is intended for intrathecal administration via lumbar puncture and is currently in the preclinical stage, with human clinical trials expected to commence in 2026.