MeiraGTx announces presentations at ESGCT

MeiraGTx announced the Company will exhibit eight posters and deliver one oral presentation at the European Society of Gene and Cell Therapy, ESGCT, 2023 Annual Congress, which is being held from October 24-27, 2023, in Brussels, Belgium. Poster #167: Potency assay cell line development for ocular gene therapy vectors. When producing GMP AAV drug products, the ability to assess their potency is an essential part of release and stability. the company assessed different methods to increase the transducibility of AAV5 and AAV8 vectors as well as trans-activate photoreceptor-specific and RPE-specific promoters. In dose-ranging experiments, it identified potent dCas9-mediated trans-activators for both promoters and established exogenous factor supplementation that increased the transducibility of both capsids multiple folds over baseline. Poster #324: Use of mechanistic modeling to design a platform process for the separation of full and empty AAV capsids. Adeno-associated viruses are a relative newcomer to the field of biopharmaceutical modalities and are used to deliver a therapeutic gene to a patient. During their upstream production, separate cellular processes are used to produce the viral capsid and the therapeutic transgene, and package the transgene within the capsid. This leads to the expression of empty capsids which must be removed during the downstream process as they may stimulate an immune response in the patient. Poster #334: Improving the elution step in capture chromatography: a mechanistic approach. Bulk recovery of adeno-associated viruses in the capture step is often performed using affinity chromatography. To understand this operation a DoE was performed, using an AAV5-based product, to determine the main factors affecting the elution behaviour. Poster #431: Synthetic neuronal promoters that surpass synapsin in the central nervous system. The promoter is an essential cis-regulatory element in any DNA-based gene therapy. Poster #464: Riboswitch-regulated chimeric antigen receptor enhances CAR-T cell anti-cancer efficacy. The company presents the development of RiboCAR, a mammalian synthetic riboswitch-regulated CAR expression via small molecule inducer.