Intellia Therapeutics (NTLA) said Wednesday that the US Food and Drug Administration granted the Regenerative Medicine Advanced Therapy designation to its nex-z treatment for transthyretin amyloidosis with cardiomyopathy or ATTR-CM.
The company said the CRISPR-based therapy has shown it can sharply reduce serum TTR levels with a single dose, potentially slowing disease progression in patients with cardiomyopathy and polyneuropathy.
Intellia added that nex-z has also received RMAT designation for polyneuropathy and Orphan Drug Designation from US and European regulators.
The company's shares were up over 1.9% in recent Wednesday premarket activity.
免責聲明：投資有風險，本文並非投資建議，以上內容不應被視為任何金融產品的購買或出售要約、建議或邀請，作者或其他用戶的任何相關討論、評論或帖子也不應被視為此類內容。本文僅供一般參考，不考慮您的個人投資目標、財務狀況或需求。TTM對信息的準確性和完整性不承擔任何責任或保證，投資者應自行研究並在投資前尋求專業建議。