CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001 - Healing Genes

CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001

Share on facebook
Share on google
Share on twitter
Share on linkedin

CRISPR Therapeutics and Vertex Pharmaceuticals today reported preliminary, mostly-positive safety and efficacy data from the first two patients enrolled in two Phase I/II trials assessing their CRISPR/Cas9 gene-edited therapy CTX001 for a pair of blood disorders—the first clinical trial of a gene-editing candidate sponsored by U.S. companies.

“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,” CRISPR Therapeutics CEO Samarth Kulkarni, PhD, said in a statement. “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.”

Added Vertex chairman, president, and CEO Jeffrey Leiden, MD, PhD: “The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy.”

Investors appeared to agree with Leiden and Kulkarni, snapping up enough shares of CRISPR Therapeutics to boost its stock price 21%, to $70.89 in early trading as of 10:02 a.m. Vertex shares barely budged, rising 0.46% (97 cents a share) to $210.97.

Read the full story here:

More to explorer

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader

CRISPR treatment inserted directly into the body for first time

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that

No Comments

Sorry, the comment form is closed at this time.