Leniolislippertje schreef op 5 juli 2021 08:37:
Pharming loopt nu al vele jaren achter op de gentherapie van Intellia:
“ Intellia continues to build momentum as we progress our full-spectrum pipeline towards key upcoming milestones this year. We plan on submitting first-in- human regulatory filings for NTLA-2002 for HAE.”
NTLA-2002 aims to prevent attacks for people living with HAE after treatment consisting of a single administration. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.
Intellia expects to submit a regulatory application for NTLA-2002 in the second half of 2021. The first-in- human trial is expected to evaluate safety, tolerability and activity in patients with HAE.