The 3 Small CRISPR Biotechs That Could Cure 10,000 Diseases
By Mark Terry
CRISPR is likely to revolutionize medicine. The technology, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a fast and simple way of editing DNA. In theory, it makes it possible to quickly excise the damaged part of a gene that causes a disease and replace it with one that works better. Although there are concerns about it causing mistakes, as well as concerns over eugenics applications, the technology shows amazing potential. It was first used in the U.S. in 2009 to engineer immune cells outside the body in HIV patients, and inside the body in November 2017 to treat Hunter syndrome. And it was recently reported that 86 people have been treated in China since 2015 using CRISPR genome-editing technology.
Investor’s Business Daily recently looked at three small biotech companies that appear to be leading the CRISPR race.
With headquarters in Zug, Switzerland and Cambridge, Massachusetts, CRISPR Therapeutics announced that it and Vertex Pharmaceuticals were co-developing and co-commercializing CTX001, an investigational gene editing treatment to treat beta-thalassemia and sickle cell disease.
“Over the past two years, we’ve made significant progress with CRISPR Therapeutics on the discovery and preclinical development of multiple CRISPR/Cas9-based treatments, and we’re pleased to select CTX001 as the first of these treatments to move into clinical development as part of our collaboration,” said David Altshuler, Vertex’s executive vice president, Global Research and chief scientific officer, in a statement. “The addition of CTX001 to our clinical development pipeline provides us with a near-term opportunity to generate the first proof-of-concept clinical data for a CRISPR/Cas9-based medicine in two genetic diseases that are highly aligned with our research strategy.”
The clinical trial is to launch in Europe sometime this year for beta-thalassemia, and in the U.S. in sickle cell disease this year as well.
Based in Cambridge, Mass., Intellia is using CRISPR to work on therapies for cancer, genetic disorders, viral infections, inflammatory disorders and others. On Dec. 11, 2017, the company presented, along with its collaborator, Novartis, initial data on genome-edited human hematopoietic stem cells at the American Society of Hematology Annual Meeting.
John Leonard, Intellia’s executive vice president, Research & Development, said in a statement, “These results are significant as we have shown high levels of editing as well as increased production of fetal hemoglobin to clinically relevant levels, which could potentially ameliorate sickle cell disease in affected patients. We are very encouraged to present this progress given that sickle cell disease is a serious condition that currently has limited treatment options.”
Investor’s Business Daily writes, “Intellia also has in vivo and ex vivo programs in gene editing, and also is working in sickle cell disease. It’s furthest along in a partnership with Regeneron Pharmaceuticals for a therapy to treat what’s known as transthyretin amyloidosis, a condition characterized by the buildup of abnormal protein deposits throughout the body.”
Also headquartered in Cambridge, Massachusetts, Editas is also using CRISPR to focus on cancer, liver diseases, lung diseases, blood diseases, muscle diseases and eye diseases. At the J.P. Morgan Healthcare Conference in January, the company’s president and chief executive officer, Katrine Bosley, presented the company’s long-range plans for the year 2022, dubbed EM22. The goal is to have three experimental therapeutics in early-stage clinical trials, two in or ready for late-stage clinical trials, and a best-in-class platform and pipeline for developing genomic medicines.
The company’s lead program is for Leber Congenital Amaurosis type 10 (LCA10), with the company expecting to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for EDIT-101 in mid-2018. Two more programs are in early stages of development to treat recurrent ocular herpes simplex virus type 1 (HSV-1) infection and Usher Syndrome type 2a (USH2a). It has shown proof-of-concept in rabbit models for HSV-1. It has a strategic research deal with Massachusetts Eye and Ear to validate its approach in USH2a.
CRISPR is more of a tool, than anything else, and numerous companies are using it, although these three are using it as their predominant methodology. Nessan Bermingham, a former chief executive officer of Intellia, estimates that 5,000 diseases could be cured by changing a single targeted gene using CRISPR. The World Health Organization (WHO) has estimated that figure at 10,000.