How can an RNA be used to treat myotonic dystrophy?

Project specs

Format

2D/3D

Length

90 sec

Summary

“ARTHEx biotech is focused on the development of the next generation of RNA therapies for genetic diseases. Our first product in the pipeline is a first-in-class treatment against myotonic dystrophy type 1, an orphan and rare disease. This therapy is based on an antisense oligonucleotide that inhibits the action of microRNAs (also known as antimiR).

Links:
Website: www.arthexbiotech.com
Twitter: https://twitter.com/Arthexbiotech (@arthexbiotech)
LinkedIn: https://www.linkedin.com/company/arth…
Facebook: https://es-es.facebook.com/pages/cate…

Researcher Profile

"ARTHEX BIOTECH is a preclinical stage life sciences company focused on developing the next generation of RNA therapies for the treatment of Myotonic Dystrophy Type 1.
The organisation chart of ARTHEx follows a lean and virtual model, based on a reduced management team specialised in the pathology, project management and business development, with external agents that provide the specialisation: R&D, financial management and toxicology, CMC, clinical research and IP.
The core Team is composed by cofounders, Beatriz Llamusí (CEO) and Rubén Artero (full professor at the University of Valencia).

Dr. Beatriz Llamusi
Msc and PhD in Biochemistry, CEO of Arthex Biotech S.L
Dr.Llamusi focused her scientific career on studying the molecular basis of pathogenesis and
potential treatments by generating animal models of human diseases. As a postdoc she
moved her focus from neuro-regeneration in spinal cord, to rare diseases, specifically Myotonic Dystrophy disease (DM), working under the supervision of Prof. Dr. Artero, in the laboratory of Translational Genomics affiliated to the University of Valencia and the INCLIVA health research institute. She contributed more than 20 scientific articles in indexed journals, including original research and reviews on the state of the art in Myotonic Dystrophy. Her main research interest is to understand myotonic dystrophy neurodegeneration, heart dysfunction, and muscle atrophy, to investigate their molecular basis and to find drugs that suppress these phenotypes. Her motivation is the translation of her research to benefit society and, as such, she discovered five anti-DM1 compounds or repurposing drugs, which are protected by patents and licensed to biotech companies.
Currently, she founded the biotech company ARTHEX Biotech S.L, in September 2019, dedicated to the development of an antimiR-based drug against Myotonic dystrophy disease. The company raised seed funds from Invivo Capital and ADVENT France VCs and is actually working on the preclinical development of these molecules, planning to enter FIH on 2022.

Dr. Rubén Artero
Msc and PhD in Biochemistry, full professor of Genetics at the University of Valencia. Leader of the translational genomics lab in the UV. His laboratory is focused on the molecular basis of muscle atrophy in myotonic dystrophy and in identifying appropriate therapeutic targets and candidate drugs using cell, animal and bioinformatic approaches. During his doctoral degree, he discovered the muscleblind gene, the founding member of the MBNL family of proteins. He received six years of postdoctoral training at the Memorial Sloan-Kettering Cancer Center in New York studying Drosophila myogenesis and is leading the Translational genomics lab since 2005. He contributed more than 50 research articles related to Myotonic Dystrophy disease mechanisms, and pioneeres the research on MBNL function and regulation. He produced more than 10 patents with his research in the field and acted as advisor for different companies. Currently, he co-founded Arthex Biotech S.L and is a member of its scientific advisory board.
"