Direct editing of the human genome in vivo

News this week of results from Intellia Therapeutic’s first-in-human clinical trial captured the world’s imagination. The phase 1 trial demonstrated for the first time that direct CRISPR/Cas9 genome editing works in humans. Intellia are far from the only company working on this technology.

Below are three other companies with actively recruiting clinical trials working on CRISPR/Cas9 therapeutics, organized by the condition targeted. There are many projects that are working on editing cells in vitro followed by infusion of the edited cells - these projects were excluded. Only groups that are working on directly editing the genome in vivo are included. Data are based on clinicaltrials.gov.

Blindness (Viral Keratitis, Herpes Simplex Virus)

  • Candidate: BD111
    Mechanism: Targeted editing of HSV-1 viral genome that has integrated into the genome of cells in the cornea
    Sponsor: Shanghai BDgene Co
    Phase: 1

Blindness (Leber congenital amaurosis)

  • Candidate: EDIT-101
    Mechanism: Edit and repair the genome of retinal cells to fix a mutation in the CEP290 gene that causes blindness
    Sponsor: Editas Medicine
    Phase: 1/2

HPV-driven cervical cancer

  • Candidate: CRISPR/Cas9-HPV E6/E7
    Mechanism: Target HPV+ cancers by editing E6/E7 oncogenes in cancer cells
    Sponsor: Sun Yat-Sen University
    Phase: 1