Impaired retinal synaptic transmission of neural signals causes loss of dark-adapted absolute visual perception. This is evident on clinical electroretinogram (ERG) testing as a characteristic ...

A new study shows that subretinal adeno-associated virus 8 (AAV8) injections cause proinflammatory T-cell immune response against the transgene product. According to the study, published in the ...

LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug ...

(RTTNews) - MeiraGTx Holdings Plc. (MGTX), Wednesday announced that the U.S. Food and Drug Administration has granted the Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the ...

Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 ...

Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, ...

Kanazawa, Japan – Malaria remains a deadly disease that affects people worldwide, particularly in Africa. Caused by a parasite that can enter the human bloodstream via mosquito bite, the parasites can ...

Peking University, July 23, 2025: On July 14, 2025, Professor Su Xiaodong’s team from the School of Life Sciences at Peking University, in collaboration with Professor John Rasko’s team from the ...

Recombinant adeno-associated virus (rAAV) is currently used as vector in about 250 clinical trials for gene therapy (Bulcha et al., 2021). Nonetheless, there are still several challenges concerning ...

MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Company’s proprietary ...