MeiraGTx: MeiraGTx and Hologen launch AI-backed gene therapy venture

Hologen's AI models have already been used to analyse data from MeiraGTx's clinical trials. Credit: innni via Getty Images. · Pharmaceutical Technology · innni via Getty Images. In This Article: 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. In October 2023, Sanofi invested $30m into MeiraGTx through a pricing offering of ordinary shares. MeiraGTx sold 4 million ordinary shares for $7.50 each, granting Sanofi access to data from some of its programmes, including in immunology and inflammation, central nervous disorders and glucagon-like peptide-1 (GLP-1) and other gut peptides for metabolic diseases. "MeiraGTx and Hologen launch AI-backed gene therapy venture" was originally created and published by Pharmaceutical Technology , a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.