{"id":11201,"date":"2025-04-11T09:59:44","date_gmt":"2025-04-11T07:59:44","guid":{"rendered":"https:\/\/www.uni.lu\/lcsb-en\/?post_type=news&p=11201"},"modified":"2025-07-04T12:28:45","modified_gmt":"2025-07-04T10:28:45","slug":"public-private-partnership-for-novel-drug-candidate-for-parkinson","status":"publish","type":"news","link":"https:\/\/www.uni.lu\/lcsb-en\/news\/public-private-partnership-for-novel-drug-candidate-for-parkinson\/","title":{"rendered":"A public-private partnership to assess a novel drug candidate for Parkinson\u2019s disease"},"content":{"rendered":"\n
\n

Through its BRIDGES programme, the Luxembourg National Research Fund<\/a> (FNR) is supporting Transcend-PD1<\/a><\/sup>, a collaborative project between the Luxembourg Centre for Systems Biomedicine<\/a> (LCSB) and Accure Therapeutics<\/a>, a translational R&D biotech in neuroscience based in Barcelona. Successfully launched in 2024, Transcend-PD aims to assess the efficacy of ACT-02, a drug candidate from Accure Therapeutics\u2019 portfolio, in models developed by the Translational Neuroscience<\/a> group at the LCSB. The 480.000\u20ac grant, including monetary and in kind contributions, awarded to this public-private partnership will help evaluate the potential of this new compound in addressing critical unmet needs in Parkinson\u2019s disease.<\/p>\n\n\n\n

ACT-02 is a novel compound with disease-modifying potential. It inhibits the enzyme Prolyl Endopeptidase (PREP), a promising but previously overlooked target in Parkinson\u2019s disease. This drug candidate has demonstrated properties to improve cognition while reducing motor symptoms, neuroinflammation and alpha-synuclein aggregation in preclinical models of brain disorders. The ACT-02 program is currently at an advanced preclinical stage, supported by comprehensive positive efficacy data in in vivo<\/em> Parkinson\u2019s models and by toxicology and safety pharmacology data sets.<\/p>\n\n\n\n

\u201cThis collaboration will allow us to get further insights into Parkinson\u2019s pathophysiology by understanding the mechanisms of action of ACT-02, which targets pathways involved in mitochondrial dysfunction and alpha-synuclein accumulation, two cardinal features of the disease,\u201d details Dr Giuseppe Arena<\/a>, who was a senior research scientist in the Translational Neuroscience group and has now taken on a scientific advisor role in Transcend-PD.<\/p>\n\n\n\n

The LCSB offers an ideal setting to assess the efficacy of ACT-02, thanks to a range of different cellular models of Parkinson\u2019s disease developed for translational studies. These serve as a robust, clinically-relevant platform for drug testing in patient-derived cells. Using neuronal models from carefully selected Parkinson\u2019s patients will strengthen patient selection and stratification, enhancing the translational potential of ACT-02. \u201cThese cellular models will allow us to narrow down the subset of patients most likely to respond positively to the treatment in future clinical trials,\u201d continues Dr Arena. A hurdle that many previous treatments have failed to overcome due to the heterogeneity of the disease.<\/p>\n\n\n\n

\u201cThe goal of our research is ultimately to help develop new disease-modifying therapeutic strategies for Parkinson’s disease, so it is great to see our expertise with human induced pluripotent stem cells (iPSC)-derived neurons being used to test a new orally active drug candidate,\u201d says Prof. Rejko Kr\u00fcger<\/a>, head of the Translational Neuroscience group. \u201cWe are looking forward to extending our highly translational iPSC work to guide the next clinical trial phases for ACT-02.\u201d

\u201cWe are grateful to the FNR for their support of our ACT-02 asset. This grant is a significant step forward in the development of our first-in-class drug candidate,\u201d concludes Dr Laurent Nguyen<\/a>, co-founder and CEO at Accure Therapeutics. \u201cWith ACT-02, we aim to develop a game changer drug to treat patients suffering from Parkinson\u2019s disease.\u201d
<\/p>\n\n\n\n

—

1 <\/sup>Pharmacological targeting of Prolyl Endopeptidase in human induced pluripotent stem cells-derived neurons for the development of precision medicine therapeutic strategies in Parkinson’s disease (Transcend-PD)<\/p>\n\n\n

\n \n

\nMeet the researcher<\/h2>\n
    \n
  • \n
    \n
    \n
    \n \n\"Prof\n <\/figure>\n <\/div>\n
    \n

    Prof Rejko KR\u00dcGER<\/h3>\n

    Full professor in Neurosciences \u2013 FNR PEARL Chair<\/p>\n

    \n \n \n Learn more<\/span>\n <\/span>\n <\/use><\/svg> <\/a>\n<\/div>\n <\/div>\n <\/div>\n<\/div>\n<\/div><\/li><\/ul>\n\n<\/div>\n\n<\/div><\/section>\n\n\n
    \n
    \n \n

    \nDiscover more<\/h2>\n\n
      \n
    • \n \n \n \n Translational Neuroscience <\/span>\n <\/use><\/svg> <\/span>\n <\/a>\n<\/li>\n\n\n\n
    • \n \n \n \n Accure Therapeutics <\/span>\n <\/use><\/svg> <\/span>\n <\/a>\n<\/li>\n\n\n\n
    • \n \n \n \n FNR BRIDGES programme <\/span>\n <\/use><\/svg> <\/span>\n <\/a>\n<\/li>\n<\/ul>\n <\/div>\n<\/section>","protected":false},"excerpt":{"rendered":"

      ACT-02 is a novel compound with disease-modifying potential. It inhibits the enzyme Prolyl Endopeptidase (PREP), a promising but previously overlooked target in Parkinson\u2019s disease. This drug candidate has demonstrated properties to improve cognition while reducing motor symptoms, neuroinflammation and alpha-synuclein aggregation in preclinical models of brain disorders. The ACT-02 program is currently at an advanced preclinical stage, supported by comprehensive positive efficacy data in in vivo Parkinson\u2019s models and by toxicology and safety pharmacology data sets.<\/p>\n","protected":false},"author":55,"featured_media":11202,"template":"","format":"standard","meta":{"featured_image_focal_point":[],"show_featured_caption":false,"ulux_newsletter_groups":"","uluxPostTitle":"","uluxPrePostTitle":"","_trash_the_other_posts":false,"_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"0","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false},"news-category":[4],"news-topic":[19],"organisation":[202,220],"authorship":[55],"acf":[],"yoast_head":"\nPublic-private partnership for novel drug candidate for Parkinson\u2019s - University of Luxembourg I Uni.lu<\/title>\n<meta name=\"description\" content=\"The FNR supports Transcend-PD, a collaborative project between the LCSB and Accure Therapeutics aiming to assess the efficacy of ACT-02, a drug candidate for Parkinson\u2019s disease.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.uni.lu\/lcsb-en\/news\/public-private-partnership-for-novel-drug-candidate-for-parkinson\/\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"A public-private partnership to assess a novel drug candidate for Parkinson\u2019s disease\" \/>\n<meta property=\"og:description\" content=\"ACT-02 is a novel compound with disease-modifying potential. 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