In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease ... a lung biologist and research director at Cystic Fibrosis Trust, who was not involved in ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

The cystic fibrosis treatment leader exceeded revenue expectations, but missed slightly on earnings per share.

The effect of cystic fibrosis on many organs and the frequent lung infections means that it is mostly diagnosed in childhood. Somehow, for Verônica Stasiak Bednarczuk, that diagnosis did not arrive ...

In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform. "ReCode to secure funds for cystic fibrosis gene ...

Stuart Arbuckle, who’s been with Vertex since 2012, plans to retire from his role in July. His departure comes as Vertex ...

Learn about our Editorial Policies. In a study published in Nature Communications, scientists reported a novel strategy to deliver the CRISPR-Cas9 gene-editing system into the lungs of a cystic ...

Viral and nonviral clinical trials have established proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether gene transfer efficiency is sufficient ...

Cystic fibrosis is caused by hereditary genetic mutations that impair or halt the production of the CFTR protein. The respiratory tract is most severely affected. There, the mucus becomes so ...

The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...

Gene therapy and gene editing are on the cutting ... company that specializes in developing therapies to treat cystic fibrosis. Vertex also owns the rights to 60% of the profits from sales of ...

Compare that with a disease like cystic fibrosis, which is caused by more ... creating hundreds or thousands of different gene editing drugs for every single mutation is very impractical, right?” ...