Leading the Way on Gene Editing

Jun 12, 2020

Leading the Way on Gene Editing

At Vertex, our strategy is to invest in scientific innovation to make transformative medicines for serious diseases. We are advancing that strategy with cell and genetic therapies that now make it possible to discover and develop one-time, potentially curative treatments. At the new Vertex Cell and Genetic Therapies (VCGT) site that we’re building in Boston, we’re assembling the tools and team needed to bring transformative genetic and cell therapies to patients quickly and at high quality.

Today marks a big step along that journey for both VCGT and the field of gene editing. At the virtual European Hematology Association (EHA),we and our colleagues at CRISPR Therapeutics presented the first peer-reviewed clinical evidence to demonstrate CRISPR/Cas9-based gene editing has curative potential for genetic disorders like sickle cell disease and beta thalassemia. These results provide proof-of-concept for beta thalassemia and are extremely encouraging not only for our hemoglobinopathies program, but for all the diseases we are pursuing with CRISPR-based gene editing. We take confidence in the specificity afforded by CRISPR/Cas9 technology, and the potential that the specific changes made will be lasting and address all signs and symptoms of disease.

We are particularly grateful to and inspired by the spirit and resilience of the patients and patient community that has stepped forward to participate in clinical trials and make breakthroughs possible. We are excited to continue to work with physicians, patients, caregivers, and families over the coming months and years to bring forward the best possible therapy for sickle cell disease and beta thalassemia, but also in our other focus areas with pioneering approaches like Duchenne muscular dystrophy, type 1 diabetes, and others.

In addition, we are excited to work with partners and collaborators outside our walls. While VCGT represents one of the broadest and most diverse set of capabilities to deliver on the promise and potential of cell and genetic therapies, we’re leveraging this expertise by working with colleagues at Molecular Templates to research and develop more gentle conditioning regimens for patients with the potential to ease the burden of having a stem cell transplant. We’re continually thinking about how to out-innovate ourselves in genetic and cell therapies so we are not only first-in-class, but also best-in-class.

Learn more about Vertex Cell and Genetic Therapies here.

It’s incredibly exciting to be part of the team pioneering the first CRISPR gene-editing therapy for serious inherited diseases like sickle cell disease and beta thalassemia. We look forward to continuing to share our progress in these trials, and our progress as we expand into new areas with VCGT.


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