CRISPR

CRISPR Therapeutics last week announced the submission of a Clinical Trial Application (CTA) for CTX001 in β-thalassemia. CTX001 is an investigational CRISPR gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease.

“CRISPR Therapeutics is pioneering a new class of medicines with the CTA submission for CTX001 to conduct the first company-sponsored clinical trial of a CRISPR gene-edited therapy,” commented Samarth Kulkarni, Chief Executive Officer of CRISPR Therapeutics. “We are committed to translating the ground-breaking science of the CRISPR platform into therapies that can fundamentally change the lives of patients suffering from serious diseases such as β-thalassemia and sickle cell disease.”

The Phase 1/2 trial of CTX001 is designed to assess its safety and efficacy in adult transfusion dependent β-thalassemia patients and is expected to begin in Europe in 2018, reports Wired. CRISPR also plans to file an Investigational New Drug Application for CTX001 to treat sickle cell disease with the United States Food and Drug Administration in 2018.

“β-thalassemia is a devastating disease that requires serious and chronic medical intervention,” said Tony Ho, Head of Research and Development at CRISPR. “The efficient and precise editing in a patient’s own blood cells using CRISPR provides the possibility of a one-time treatment for those suffering from β-thalassemia and sickle cell disease.”

According to company data from human cell and animal studies presented at the American Society of Hematology Annual Meeting in Atlanta on Sunday, the treatment results in high editing efficiency, with more than 80 percent of the stem cells carrying at least one edited copy of the gene that turns on fetal hemoglobin production; enough to boost expression levels to 40 percent. Newly minted Crispr Therapeutics CEO Sam Kulkarni says that’s more than enough to ameliorate symptoms and reduce or even eliminate the need for transfusions for beta-thalassemia and sickle cell patients. Previous research has shown that even a small change in the percentage of stem cells that produce healthy red blood cells can have a positive effect on a person with sickle cell diseases.

“I think it’s a momentous occasion for us, but also for the field in general,” Kulkarni told Wired. “Just three years ago we were talking about Crispr-based treatments as sci-fi fantasy, but here we are.”

It was around this time last year that Chinese scientists first used Crispr in humans—to treat an aggressive lung cancer as part of a clinical trial in Chengdu, in Sichuan province. Since then, immunologists at the University of Pennsylvania have begun enrolling terminal cancer patients in the first US Crispr trial—an attempt to turbo-charge T cells so they can better target tumours. But no one has yet used Crispr to fix a genetic disease.

Therapeutic applications of CRISPR have the potential drive the potential for positive impact and commercial potential skywards.  Last month we reported Citi GPS’ new report that CRISPR, will grow into a $10 billion market by 2025

CRISPR Technology is on Track to be a $10 Billion Market by 2025

“Currently the CRISPR market is small, with its main offerings dedicated to lab work and scientific research via research toolkits. However, the real economic potential of CRISPR lies with human therapeutics. With CRISPR-based therapeutics having already entered human trials last year in China, the first CRISPR-based medicine could reach the market in ~6 years or less,” Citi biotech analyst Yigal Nochomovitz wrote in the report.

“If CRISPR gene editing works in early test cases of human disease, the long-term upside for the technology could be much, much greater,” Nochomovitz added.

We are now able to marvel (in low res) for the first time at CRISP in action via high-speed atomic-force microscopy

A new paper, published in Nature Communications, has provided us with real-time, molecule-scale footage of CRISPR chopping a strand of DNA right in two.

First Video of CRISPR Editing DNA in Real-Time

While the footage may be fuzzy many CRISP itself is can be very precise with so call calleded off-target effects, whereby edits are made in unintended place are minimal, though the extent of that problem is still up for debate. Just on Monday, a new study published in the Proceedings of the National Academy of Sciences suggested that genetic variation between patients may affect the efficacy and safety of Crispr-based treatments enough to warrant custom treatments. All of that means Crispr companies will have to work that much harder to prove to regulators that their treatments are safe enough to put in real people—and to prove to patients that participating in trials is worth the risk. Kulkarni says they looked at 6,000 sites in the genome and saw zero off-target effects. But it’ll be up to the FDA and the European Medicines Agency to say whether that’s good enough to send Crispr to the clinic.