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uniQure Announces Presentations at the Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT)
Oral Presentation to Highlight AMT-191 for Fabry Disease LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 19, 2021 (GLOBE NEWSWIRE) -- uniQure N.V.
About this update from Uniqure N.v.
[{"type":"text","content":"Oral Presentation to Highlight AMT-191 for Fabry Disease\nLEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 19, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that four data presentations, including one oral presentation, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) 28th Annual Meeting taking place virtually today through October 22, 2021. Featured in the oral presentation are preclinical data of AMT-191, uniQure’s gene-therapy candidate for the treatment of Fabry disease which is advancing into IND-enabling studies. AMT-191 is a one-time administered AAV5 gene therapy incorporating an α-galactosidase A (GLA) transgene. New preclinical data confirms high efficiency and cross correction of AMT-191 in a Fabry mouse model, with increased GLA-activity in the liver, kidney, heart, and brain and normalized (lyso-)Gb3 levels in main target organs. In addition, a single administration of AMT-191 led to phenotype improvement of nociception or a faster reaction time to physical stimulus. These new data build on earlier preclinical studies comparing multiple product candidates, including constructs incorporating a modified alpha-N-acetylgalactosaminidase transgene (modNAGA) where AMT-191 demonstrated robust and sustained increases in GLA activity and subsequent functional improvement. Moreover, pre-clinical studies in non-human primates with AMT-191 demonstrated robust and sustained increases in GLA activity in multiple organs. “We are enthusiastic about sharing these data on our Fabry program with the scientific community, and we plan to accelerate the preclinical work necessary to file an IND for AMT-191 by 2023,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “We believe that this preclinical data on AMT-191 showing high efficiency and cross correction in a mouse model differentiates this program from other current investigational gene therapy programs in Fabry disease. We look forward to accelerating this program closer to IND filing and the clinic.” Specific details on uniQure’s preclinical data presentations at ESGCT include: Oral Presentation Title: Pre-clinical proof of concept of an AAV5-GLA gene therapy for Fabry disease results in cr...