Stanford readies for CRISPR human testing after sickle cell success

Scientists from Stanford University School of Medicine have used CRISPR in early-stage work to patch up the gene that causes sickle cell disease as they eye human trials in 2018.

CRISPR’s preclinical activity has caused much hype given its gene-editing capabilities, with its core tech centering on a new method for reengineering DNA. The Stanford researchers, reporting in Nature, used CRISPR to “snip” the faulty gene and then used a virus to introduce the repair mechanism into cells.

Sickle cell disease, which affects around 100,000 Americans, is caused by a single mutation in a gene that makes a hemoglobin protein. They hope this could lead to a new gene therapy in the future, with Dr. Matthew Porteus, senior author of the study, telling Reuters that the Stanford team “plans to make an initial submission to the FDA in the next few months to map out the clinical trial, and hopes to treat the first patient in 2018.”

Proteus has an equity interest in CRISPR Therapeutics ($CRSP), one of several CRISPR-focused biotechs that have gone public this year, though he told the newswire service that the work will be carried out at Stanford and its cell manufacturing labs, and not with CRISPR Therapeutics.  

This comes a month after a team of researchers from UC Berkeley, UC San Francisco Benioff Children’s Hospital, Oakland Research Institute and the University of Utah School of Medicine also used CRISPR, but in a slightly different way.

They snipped out the diseased gene but then delivered a new stretch of DNA to correct the mutation in human stem cells.

There is however an ongoing patent battle over just who owns the rights to the cutting-edge CRISPR-Cas9 technology between Feng Zhang from the Broad Institute of Harvard and MIT and Jennifer Doudna, from the University of California, Berkeley and Emmanuelle Charpentier. This is set to run for the foreseeable future.

CRISPR also hasn’t yet entered into the lab for in-human testing--but American academic researchers are in a race with Chinese scientists to be the first to start--with the world’s first-ever test earmarked to begin before the end of the year, with an early focus on cancer targets.

Of the publicly traded gene editing biotechs, Editas ($EDIT) still appears to be in the lead for its first Phase I, which it has previously said is slated for 2017.