Attacking Facioscapulohumeral muscular dystrophy (FSHD) at the source 

Epigenetic gene regulation

ABOUT RENOGENYX

RENOGENYX is a biotechnology company developing therapeutics for FSHD, which affects over 1 million people worldwide.

 

We are developing epigenetic tools to treat the root cause of disease. 

Our most advanced preclinical program is a CRISPR-based approach to treat the underlying cause of FSHD.

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Our Mission

Our vision is to bring new treatments and therapies to patients with FSHD.

There are over 50,000 people affected by FSHD in the USA and Canada, and another 1 million around the world.  

Currently, there are no approved treatment options for FSHD, and we aim to change that.   

By bringing together the best scientists, key opinion leaders and disease experts, and working directly with patient communities, clinicians, payors and other health stakeholders, we hope to make a transformational difference and change lives. 

 
Male Scientist
 

FSHD (facioscapulohumeral muscular dystrophy)

FSHD is an epigenetic gain-of-function neuromuscular disorder caused by the misexpression of DUX4 protein in skeletal muscle.

CRISPRi therapy for FSHD

CRISPR inhibition restores a more normal state of epigenetic repression at the DUX4 gene, making it an ideal candidate for FSHD therapy.

     

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