The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

producing no functional dystrophin protein. DMD mutations are particularly common in “hotspot” areas of the gene (exons 45–55 and 2–10). Because the genetic underpinnings of Duchenne are known, ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...

Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD Date and Time ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...

"The amount of expression of near-full length dystrophin induced by DYNE-251 has ... and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy ...

The diagnosis of Duchenne muscular dystrophy was made in all patients on the basis of the clinical history and the absence or near-absence of the dystrophin protein on muscle biopsy. In addition ...

Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...