CRISPR gene therapy or geneloop therapy has advanced to clinical trials for treating a diverse range of conditions and now the company that invented the technique is launching a cell therapy research team at Stanford Medicine.
The effort funded by the National Institutes of Health and its National Cancer Institute will take an approach that would be easily adopted in hundreds and trillions of people in under a decade said Ethan Garfin co-founder and senior scientific director of the Stanley Manne Disease Research Advisory Board Center for Genomic Therapy and director of the Stanford clinic where the CRISPR team will work.
Preliminary data from a study of the therapy in mice shows positive results said Garfin who co-founded Thermo Fisher. In a phase 1 clinical trial in which 48 prostrate leukemia patients were treated with the therapy led by assistant professor of genetics Joy Cannon there was no toxicity the company reported Wednesday.
A trial with more neurons injected as well as immunotherapy where the scientists treat the leukemia with antibodies that push the body to fight tumors is also on track for an assay President Trump has said could lead to an effective treatment by the end of 2020.
Hans Ming head of the companys clinical trials arm was optimistic that Thermo Fisher had success with its CRISPR-based treatment.
When we first got the results we expected the cells to start to die he told Reuters but did not expect that before 20 to 30 percent of the cells would actually die.
The company had started a pilot program in the U. S. last year in which mice engineered to develop leukemia were treated with CRISPR-Cas9 to kill off the cancer cells which are able to do this by recognizing the target document.
For its trial the company will enroll 8000 patients from the Eli Lilly-Ingber fast-tramp…
CRISPR-Cas9 is a revolutionary technique that allows scientists to cut out or edit DNA strands with a tiny needle using a tiny needle biopsy and other cutting tools.
It has been hailed as a game changer enabling genetic surgeons to cut out abnormal gene sequences that often cause inherited blood disorders as well as cancer Garfin said.
The therapy which is targeted at his patients uses CRISPRCas9 to disable for a year a biological glitch that can sometimes also be triggered by a toxin used by e-cigarette or vaping-related lung illnesses.
The technology allows for safe and effective cell replacement he said. They can still be delivered effectively but this allows them to achieve their goal for the therapy.