Although the idea of editing one’s specific genes might sound something out of a sci-fi novel, this could easily become a reality in the upcoming years. Gene-editing is a highly lucrative market, and a number of companies are making headway in this space. One of these is CRISPR Therapeutics, which announced today alongside another company, Vertex Pharmaceuticals, positive results in a recent gene-editing test.
Specifically, the companies announced that after testing its first two patients with its CRISPR/Cas9 gene-editing drug CTX001 meant to target patients with blood cell disorders, both patients had seen no adverse effects. Safety is a major concern for gene-editing drugs, as due to the complex and possibly irreversible nature of editing a humans’ genome, making sure that there aren’t any unexpected side-effects is very important.
“We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.”
Several more patients have enrolled to receive this treatment, and the first two patients will be monitored for an additional two years to make sure no adverse developments occur following the treatment. If successful, it would help push CRISPR’s gene editing drugs that much closer to seeking approval from the FDA.
There are a number of genetic disorders that can be potentially treated with gene-editing technology, but most companies in this space seem to focus on blood disorders, such as thalassemia. This condition causes problems such as a lack of iron in the blood, slow bone growth, heart defects, and other problems. For the most part, patients have had few treatment options, with many children who are diagnosed with the disease are likely to end up dying due to medical complications. As such, treating thalassemia as well as blood disorders, in general, appears to be a highly lucrative market.
CRISPR shot up by 17% in response to the news, making it one of the best days of the year for the company. In the highly promising gene-editing sector, CRISPR is one of the forerunners in the field, having developed an iconic gene-editing technology that bears its own name. While there are a few other noteworthy competitors in this space, including companies like Editas Medicines, Sangomo Therapeutics, and Vertex Pharmaceuticals, currently CRISPR seems to have a mild lead over its rivals in the gene-editing space.
CRISPR Therapeutics Company Profile
CRISPR Therapeutics AG is a gene-editing company. It is engaged in the development of CRISPR/Cas9-based therapeutics. CRISPR/Cas9 is a technology that allows for precise, directed changes to genomic DNA. The company advanced programs target beta-thalassemia and sickle cell disease, two hemoglobinopathies that have a high unmet medical need. – Warrior Trading News
