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MeiraGTx has struck a deal with Hologen Limited, an artificial intelligence (AI) company specialising in multimodal generative AI models, to advance its Parkinson’s disease gene therapy programme.
Hologen has committed up to $430m in funding to support the development and commercialisation of AAV-GAD, MeiraGTx’s investigational gene therapy for Parkinson’s disease. The AI company will provide $200m upfront and allocate up to $230m in additional capital to fully finance the programme.
As part of the deal, the companies will set up a newly-formed joint company, Hologen Neuro AI Limited. Hologen Neuro AI will also oversee early-stage clinical programmes targeting the central nervous system, including an investigational gene therapy for genetic obesity dubbed AAV-BDNF.
MeiraGTx will retain a 30% ownership stake in Hologen Neuro AI while leading all clinical development and manufacturing activities, as per the 13 March announcement. In addition, MeiraGTx and Hologen will enter into clinical and commercial manufacturing supply agreements, with Hologen also acquiring a minority stake in MeiraGTx’s manufacturing subsidiary.
MeiraGTx’s AAV-GAD programme is currently preparing for a Phase III trial, which is expected to kick off in mid-2025. The gene therapy works by delivering a gene encoding the enzyme responsible for producing the neurotransmitter GABA, which plays a role in motor dysfunction associated with Parkinson’s disease.
In a Phase II trial (NCT05603312), AAV-GAD demonstrated statistically significant improvements in motor function and quality of life (QoL). The study showed an 18-point improvement on the Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 for patients receiving the high-dose treatment at 26 weeks, along with positive results on the Parkinson’s disease Questionnaire (PDQ-39), a quality-of-life assessment.
According to MeiraGTx’s CEO Alexandria Forbes, Hologen’s AI models have already been used to analyse data from the Phase II trial, identifying disease-modifying changes in brain physiology. “Our collaboration with Hologen has broad significance for MeiraGTx and for drug development for neurological disorders in general. Deploying Hologen’s LLMs [large language models] on MeiraGTx’s clinical data allows the characterisation of disease modification in the CNS with unprecedented fidelity,” Forbes added.
Beyond its Parkinson’s programme, MeiraGTx is advancing AAV-AIPL1, an investigational gene therapy for congenital blindness, toward potential regulatory approval in the UK. The New York-headquartered biotech hit the headlines in February 2025 after 11 children who were born blind from birth gained significant vision improvements following treatment with AAV-AIPL1. The company said it is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US.