Pipeline | Kylix Bio

Our Pipeline

Kylix Bio is advancing two intrathecally delivered AAV-mediated gene replacement therapies to treat two sub-variants of Charcot-Marie-Tooth Disease.

CMT is an inherited peripheral neuropathy with more than 160 subtypes that impact three million patients worldwide. CMT symptoms vary but typically include impaired mobility, muscle weakness, balance challenges, sensory loss, and chronic pain.

There is currently no approved treatment for any type of CMT.

CMT 4C

IND-ENABLING STUDIES

CMT4C is an autosomal recessive, demyelinating form of CMT. We are advancing an AAV9-mediated gene replacement therapy that delivers a functional copy of SH3TC2 to Schwann cells.

CMT 1X

IND-Enabling Studies

CMT1X is the second most common CMT subtype, accounting for approximately 17% of all CMT cases. Kylix is advancing an AAV-mediated delivery of GJB1 that restores Connexin 32 function in Schwann cells.