Imagine a genetic glitch that makes muscles stiff and hard to relax, like a brake that won't release. This project creates a molecular 'key' that unlocks these muscles by blocking a specific protein that causes the problem. It doesn't change your DNA; it just fixes the instructions the cell is reading to help muscles work normally again.
Myotonic Dystrophy Type 1 affects over 1 million people and has no approved treatments to slow disease progression, leading to high disability and shortened lifespans.
ATX-01, an antimiR oligonucleotide therapy, and the ENTRY delivery platform using fatty acid conjugation.
If you are a drug developer dealing with the lack of disease-modifying treatments for muscular dystrophies — this project developed ATX-01 that targets miR-23b to rescue pathogenic mis-splicing. It provides a path to treat over 1 million diagnosed patients worldwide.
If you are a biotech firm dealing with the difficulty of getting drugs into muscle tissue — this project developed the ENTRY platform. This patented fatty acid conjugation ensures efficient delivery to target tissues at low doses.
If you are a healthcare provider dealing with the high long-term costs of managing disabling muscle diseases — this project developed a therapy that slows disease progression. This aims to create significant economic savings for healthcare systems.
Based on available project data, the specific price per dose or treatment cost is not disclosed.
The project is currently validating the product through a Phase I/IIa clinical trial and preclinical studies, indicating it is in the clinical validation stage rather than full industrial scale.
The project utilizes the patented ENTRY platform for drug delivery and has received Orphan Drug Designation from US and European authorities.
The project has secured FDA pre-IND approval and Orphan Drug Designation in both the US and Europe.
The project period runs from 2023-01-01 to 2025-06-30, focusing on Phase I/IIa trials and chronic preclinical studies.
The project is led by a single Spanish SME, ARTHEX BIOTECH SL, with a 100% industry ratio. This lean structure suggests a highly focused commercial drive, utilizing an in-house discovery engine to move from target identification to Phase I/IIa clinical trials.
Contact ARTHEX BIOTECH SL regarding the ENTRY platform and ATX-01 clinical data.
Contact us to explore licensing opportunities for the ENTRY delivery platform.
