Drug intelligence / Profile preview

diacylglycerol kinase kappa gene therapy

Development stage
Preclinical
Lead developer
Lysogene
Modality
Gene Therapies
Administration
Intracerebral
01

Overview

Diacylglycerol kinase kappa gene therapy is an **experimental AAV-based CNS gene therapy** being investigated preclinically for **Fragile X syndrome**. The approach delivers a **modified, FMRP-independent form of diacylglycerol kinase kappa** to brain neurons, typically using an **AAV Rh10 vector**, with the goal of restoring DGKk enzymatic activity that is deficient downstream of FMRP loss in Fragile X biology. DGKk converts **diacylglycerol to phosphatidic acid**, thereby helping normalize abnormal neuronal lipid signaling and synaptic homeostasis. In **Fmr1 knockout mouse** models, AAV-mediated DGKk expression reportedly corrected brain lipid dysregulation and improved behavioral phenotypes. Based on the available context, this appears to be an **academic preclinical research program** rather than a named commercial drug asset, with no identified brand name, sponsor company, manufacturing partner, or registered clinical trial.

Other names
DGKk gene therapyAAV Rh10 DGKk gene therapydiacylglycerol kinase kappa AAV gene therapy
02

Targets

DGKK (Diacylglycerol kinase kappa)

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