SubsidieMeestersFirst In class oliGo THerapy for Myotonic Dystrophy type 1 (DM1)
The project aims to develop ATX-01, a first-in-class miRNA therapy for myotonic dystrophy type 1, utilizing a patented delivery platform to improve treatment outcomes in clinical trials.
Projectdetails
Introduction
Myotonic dystrophy type 1 (DM1) is a clinically and genetically heterogeneous disorder with more than 1 million diagnosed patients worldwide, making it the most common adult muscular dystrophy. Available treatments only relieve symptoms.
Treatment Overview
Given this need, we offer ATX-01, a first-in-class miRNA therapy that inhibits miR-23b without changing the DNA, which rescues DM1 pathogenic mis-splicing.
Research and Development
We have demonstrated improved target engagement effects in skeletal and respiratory muscle in in-vitro and in-vivo studies, granting us an FDA pre-IND approval and orphan drug designation.
Delivery Platform
Our approach, antimiRs conjugated to fatty acids, constitutes the ENTRY platform, which guarantees efficient drug delivery to target muscular and extra-muscular tissue at low doses.
Future Applications
This patented technology will be validated through a Phase I/IIa clinical trial and chronic preclinical studies, to then be extended to applications for other myotonic dystrophies, Fuchs dystrophy, cachexia, among other opportunities.
Financiële details & Tijdlijn
Financiële details
| Subsidiebedrag | € 2.500.000 |
| Totale projectbegroting | € 20.074.282 |
Tijdlijn
| Startdatum | 1-1-2023 |
| Einddatum | 30-6-2025 |
| Subsidiejaar | 2023 |
Partners & Locaties
Projectpartners
- ARTHEX BIOTECH SLpenvoerder
Land(en)
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FIRST-IN-CLASS MYC INHIBITOR: THE MAKING OF A BREAKTHROUGH CANCER THERAPY
MYCureX aims to evaluate the safety and efficacy of OMO-103, a novel MYC inhibitor, in combination with standard care for PDAC, while refining companion diagnostics for improved patient outcomes.
Clinical validation of GLIX1: a small molecule that targets epigenetic changes in cancer cells to treat glioblastoma multiforme (GBM), the highest global unmet need in oncology.
GLIX1, a novel therapy targeting deregulated mechanisms in GBM, shows promising tumor regression in preclinical studies and aims for clinical validation to improve patient outcomes.
Dementia/Alzheimer Drug Attributed to Cystatin-C and other mutations – precision medicine drug development
ATI's AT-001 aims to stop and revert dementia by preventing protein aggregation, with promising Phase IIa results leading to upcoming registration trials and POC studies for commercialization.
Beta-cell recovery to counter diabetes
DiogenX aims to cure Type 1 Diabetes by regenerating pancreatic beta-cells for autonomous insulin release, with plans to out-license the drug following human clinical proof by 2026.
The i-Thymus: wielding the potential of gene therapy, cell therapy and induced pluripotent stem cells for the regeneration of the thymus and the adaptive immune system
i-Thymus aims to revolutionize treatment for babies born without a thymus by using gene therapy to create implantable thymus organoids, targeting clinical trials and market entry by 2028.
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