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AAV-UPF1 + AAV-CNTFR
- Development stage
- Preclinical
- Lead developer
- MeiraGTx Holdings
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathecal
01
Overview
MeiraGTx Holdings is developing a combination AAV-based gene therapy for the treatment of Amyotrophic Lateral Sclerosis (ALS). The therapy consists of two viral vectors: AAV-UPF1 (also referred to as AAV-hUPF1) and AAV-CNTFR. AAV-UPF1 delivers the gene for UPF1, a master regulator of the nonsense-mediated decay (NMD) pathway, which is intended to restore RNA metabolism and protect motor neurons from TDP-43-mediated toxicity. AAV-CNTFR delivers the gene for the ciliary neurotrophic factor receptor (CNTFR) to provide neurotrophic support and enhance neuronal survival. The program utilizes the AAV2Retro capsid for targeted delivery to the central nervous system and is currently in preclinical development.
- Other names
- UPF1-CNTFR-AAV gene therapyUPF-1-CNTFR-AAV gene therapyUPF 1-CNTFR-AAV gene therapy
02
Targets
UPF1CNTFR (Ciliary neurotrophic factor receptor alpha subunit-lif receptor beta Subunit-gp130 complex)
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