RARE Daily

CIRM Award $3.9 Million Translational Grant to Juvena for DM1

February 2, 2022

The California Institute for Regenerative Medicine awarded a $3.9 million translational grant to Juvena Therapeutics to advance its lead program, fusion protein therapeutic JUV-161 for the treatment of the rare muscle wasting disease myotonic dystrophy type 1 through initial IND-enabling studies.

Myotonic dystrophy type 1 (DM1) is an underrecognized, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, but patients can also suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no treatments for patients living with DM1.

Juvena’s technology mines the secretomes of human pluripotent stem cells through a machine learning- enhanced platform that integrates proteomics, transcriptomics, and images with phenotypic human in vitro disease model screening and extensive preclinical validation to build a map of secreted proteins in a compounding database linking therapeutic proteins to disease indications.

Juvena will also be positioned to secure a second CIRM grant to fund JUV-161, and subsequently an anticipated FDA approval, after completion of the designated criteria in TRAN I, the first grant.

Additionally, Juvena said it is applying for a phase 2 NIH SBIR grant in 2022 to support the preclinical translation of JUV-161. The application process will follow completion of the milestones in the phase I $254,262 grant that the company was awarded in August 2021. The company is also raising a Series A financing round to advance the platform and expand the pipeline of biologics to multiple therapeutic areas.

Author: Rare Daily Staff

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