After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
Clinical Trials Arena on MSN
Gene editing takes centre stage in FDA’s new rare disease approval pathway
The aim of the pathway is to remove red tape for bespoke therapies designed for patients with rare diseases.
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay ...
Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...
7d
Gene Editing Has Struggled To Go Commercial. This Nobel Laureate Has A $1 Billion Plan To Fix That.
Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer ...
Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to ...
Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...
Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix ...
The idea of self-amplifying gene editing is to get cells to pass on packages of CRISPR machinery to their neighbours, boosting the effect ...
Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results