Skip to main content
Premium Trial:

Request an Annual Quote

Gene-Editing Startup Exonics Therapeutics Receives $5M From CureDuchenne Ventures

NEW YORK (GenomeWeb) – CureDuchenne Ventures has provided $5 million in seed financing to Exonics Therapeutics, a new firm exploring the use of CRISPR/Cas9 in the development of new treatments for Duchenne muscular dystrophy (DMD).

Exonics said in a statement that it will use the proceeds to advance preclinical research from the lab of founder Eric Olson using adeno-associated viruses to deliver CRISPR to cells in order to identify and correct exon mutations in DMD patients. Olson is also a professor and researcher at the University of Texas-Southwestern Medical Center whose work on correcting DMD mutations with CRISPR/Cas9 has led to improvement in a live animal model of the disease.

CureDuchenne Ventures is a subsidiary of the non-profit CureDuchenne, which seeks to improve the standard of care and living of boys with DMD, a genetic disease that hampers the production of the muscle fiber protein dystrophin. The group collaborates with biotech firms to develop therapies for DMD.

"Exonics' CRISPR/Cas9 technology has the potential to dramatically improve the lives of those who live with Duchenne," CureDuchenne CEO Debra Miller said in a statement. "We are hopeful that a one-time treatment with gene editing therapy could provide a lifelong benefit to Duchenne patients."

The Scan

Study Points to Tuberculosis Protection by Gaucher Disease Mutation

A mutation linked to Gaucher disease in the Ashkenazi Jewish population appears to boost Mycobacterium tuberculosis resistance in a zebrafish model of the lysosomal storage condition, a new PNAS study finds.

SpliceVault Portal Provides Look at RNA Splicing Changes Linked to Genetic Variants

The portal, described in Nature Genetics, houses variant-related messenger RNA splicing insights drawn from RNA sequencing data in nearly 335,700 samples — a set known as the 300K-RNA resource.

Automated Sequencing Pipeline Appears to Allow Rapid SARS-CoV-2 Lineage Detection in Nevada Study

Researchers in the Journal of Molecular Diagnostics describe and assess a Clear Labs Dx automated workflow, sequencing, and bioinformatic analysis method for quickly identifying SARS-CoV-2 lineages.

UK Team Presents Genetic, Epigenetic Sequencing Method

Using enzymatic DNA preparation steps, researchers in Nature Biotechnology develop a strategy for sequencing DNA, along with 5-methylcytosine and 5-hydroxymethylcytosine, on existing sequencers.