Intellia gene editing technology shows promise in pre-clinical study

 

• Intellia Therapeutics (NASDAQ:NTLA) announces that its targeted liver gene insertion technology has achieved the physiological protein levels of human alpha-1 antitrypsin in non-human primates (NHPs) following a single administration.
• Compared to traditional adeno-associated virus gene therapy, the therapeutic levels of protein expression thus achieved after a single course of treatment will be stable and durable, the company noted.
• “Our new data reinforce the promise for Intellia to potentially cure a variety of rare genetic diseases requiring the restoration of a functional protein in the liver with a single-course therapy,” said Intellia President and Chief Executive Officer John Leonard, M.D., adding that the company has now demonstrated “platform’s modularity and translatability to multiple targets of interest by inserting genes to durably produce unprecedented levels of protein in NHPs for hemophilia B and AAT deficiency.”
• https://seekingalpha.com/news/3644051-intellia-therapeutics-gene-editing-technology-shows-promise-in-pre-clinical-study