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AAV-CNTFR

Development stage: Preclinical
Lead developer: MeiraGTx
Modality: Gene Therapies
Administration: Injection

Overview

AAV-CNTFR is an adeno-associated virus (AAV) vector-based gene therapy being developed by MeiraGTx for the treatment of Amyotrophic Lateral Sclerosis (ALS). The therapy is designed to deliver the gene encoding the Ciliary Neurotrophic Factor Receptor (CNTFR) to motor neurons. CNTFR is a critical component of the signaling complex for ciliary neurotrophic factor (CNTF), which has been shown to promote the survival and maintenance of motor neurons. By increasing the expression of CNTFR, the therapy aims to enhance neuroprotective signaling and support motor neuron function in patients with ALS. The program is currently in the preclinical stage of development.

Other names: AAV-CNTFR gene therapy

Targets

CNTFR (Ciliary neurotrophic factor receptor alpha subunit-lif receptor beta Subunit-gp130 complex)

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