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Arthex Biotech: Pioneering RNA Therapies for Rare Diseases

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Arthex Biotech, a vibrant spin-off from the University of Valencia in Spain, is on a mission to revolutionise treatment options for patients living with rare and currently untreatable diseases.

With a strong scientific foundation and an innovative approach to RNA-based therapies, Arthex is leading the way in targeting microRNAs tiny but powerful molecules that help regulate how genes function. EIT Health played a crucial role in Arthex's growth by supporting its development and scaling as a startup. 

At the heart of Arthex’s work is its proprietary platform designed to develop new types of genetic therapies (known as oligonucleotide therapies) that modulate microRNAs. RNA is a molecule that helps carry out the instructions encoded in DNA, playing a key role in regulating cell functions. This platform allows the company to create flexible and targeted drug candidates capable of reaching the right tissues in meaningful concentrations an essential step in treating complex diseases. 

Their lead candidate, ATX-01, is a first-in-class therapy in development for Myotonic Dystrophy Type 1 (DM1), a rare genetic disorder that affects approximately 1 in 8,000 people worldwide. ATX-01 works by targeting microRNA-23b, which plays a critical role in the disease, offering hope for a treatment that addresses the underlying cause.

How EIT Health Helped Arthex Accelerate 

Support from EIT Health came at a crucial moment in Arthex’s journey. Through the EIT Health Fast Track pilot programme, developed in collaboration with the European Innovation Council (EIC), Arthex received tailored mentoring and expert guidance.  

Thanks to this targeted training and preparation, Arthex secured a €2.5 million grant from the EIC Accelerator, a major milestone that helped further the development of ATX-01 and validated the company’s innovative science. 

Scaling Up: A €42 Million Boost 

In 2023, Arthex Biotech achieved another significant leap forward by closing a €42 million Series B financing round. The round was led by Columbus Venture Partners, with participation from the EIC Fund, Hadean Ventures, Sound Bioventures, and existing backers including Invivo Capital, AdBio Partners, and CDTI through its Innvierte programme. 

Looking Ahead 

With a groundbreaking therapeutic approach, strong scientific roots, and the backing of both public and private partners, Arthex Biotech is well positioned to make a lasting impact in the field of rare diseases. Their work offers new hope for people living with DM1 and exemplifies how the EIT Community empowers life-changing biotech solutions.