#Shares in gene-editing specialist #IntelliaTherapeutics came under renewed pressure today after a patient in one of its clinical trials who was hospitalised for #livertoxicity died.
pharmaphorum.com/news/intelli...
#Geneediting specialist #IntelliaTherapeutics lost more than 45% of its value in pre-market trading today as it revealed phase 3 trials of one of its two priority programmes had been suspended due to #safety concerns.
buff.ly/xO95WeI
What we've learned since #IntelliaTherapeutics liver enzyme disclosure 2 weeks ago:
1) late response (week 4-5)...unlikely LNP tox
2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis)
3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
Updated my #IntelliaTherapeutics blog to include recent disclosures on toxicity event: delayed liver enzyme elevations and prior paracetamol use.
rnaitherapeutics.blogspot.com/2025/05/grad...
#IntelliaTherapeutics #CRISPR it looks like tylenol caused gr4 liver enzymes, at least contributed.
FDA must pull Tylenol from market, I guess :).
x.com/GeneInvestin...
Wow, the American Heart Association specifically getting behind #IntelliaTherapeutics' genome editing for #ATTR-CM.
What did I say? Physicians/KOLs love #CRISPR:
newsroom.heart.org/news/advanci...
#IntelliaTherapeutics with impressive #ATTR-CM data showing that HEREDITARY ATTR-CM patients not just stabilized by #CRISPR NTLA-2001 nex-z, but actually IMPROVE on it.
Never seen before improvement e.g. in walk distance.
Cannot wait for longer-term data.
www.intelliatx.com/wp-content/u...
#MFN The hereditary angioedema (HAE) market is already an example where due to US pricing, access ex-US practically non-existent.
As a result, patients are queueing up ex-US for #IntelliaTherapeutics genome editing trial. Only way to access these medicines at moment --> run trials in Europe/NZ/AUS