NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD) NTLA-3001 is Intellia's first wholly owned CRISPR-based in vivo targeted gene insertion candidate to advance into the clinic On track to dose the first patient in 2H 2024 CAMBRIDGE, Mass., July 30, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on ...

NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD) NTLA-3001 is Intellia's first wholly owned CRISPR-based in vivo targeted gene insertion candidate to advance into the clinic On track to dose the first patient in 2H 2024 "NTLA-3001 is a groundbreaking in vivo CRISPR-based gene insertion candidate designed to durably produce functional AAT protein at normal levels after ...

Intellia Therapeutics, Inc. (NTLA) could be a solid choice for investors given its recent upgrade to a Zacks Rank #1 (Strong Buy). This upgrade is essentially a reflection of an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices. The power of a changing earnings picture in determining near-term stock price movements makes the Zacks rating system highly useful for individual investors, since it can be difficult to make decisions based on rating upgrades by Wall Stree ...

文章核心观点 - Intellia Therapeutics公司开发的基因编辑疗法NTLA-2001有望成为治疗罕见疾病ATTR淀粉样变性的功能性治愈方案 [1][2][3][4] - 根据临床试验数据,NTLA-2001可以大幅降低患者体内致病蛋白的浓度,但仍有少量细胞未被编辑 [3] - 公司发现可以安全地对患者进行二次给药,从而进一步降低致病蛋白浓度至95%,这为公司未来其他基因编辑疗法的重复给药提供了可能 [7][8][9] - 这一发现有利于降低公司基因编辑疗法需要重复给药的潜在风险,并为公司创造了新的收入来源 [9] 行业分析 - 预计到2029年,ATTR淀粉样变性药物市场规模可达到每年110亿美元,这对于Intellia这样的生物科技公司来说意味着巨大的增长潜力 [5] - 但如果NTLA-2001真的能够彻底治愈ATTR淀粉样变性,那么公司可能会因此而失去市场 [6] - 目前Intellia的竞争对手还未能复制出类似的重复给药证据 [9] 公司现状 - Intellia尚未商业化任何药物,目前仍处于起步阶段 [10][11] - 公司目前拥有9.53亿美元的现金和等价物,但每12个月的运营费用为5.699亿美元,意味着不到2年的运营资金 [12] - 公司首席财务官最近离职,可能会影响公司的现金管理和融资活动 [13] - 对于风险偏好较低的投资者来说,Intellia可能还是一家过于风险的公司 [14]

Let's get down to it: why should investors consider pharma stocks? Primarily, it comes down to the permanence of the underlying narrative. So long as humans are afflicted by various diseases, there will be a need for advanced pharmaceutical solutions. Therefore, money has been flowing into the broader biotechnology space and that will probably not fade anytime soon. Plus, as an American investor, you're in luck. Just the U.S. sector itself reached a valuation of $246.18 billion last year. By 2033, the domes ...

NEW YORK, July 2, 2024 /PRNewswire/ -- Kuehn Law, PLLC, a shareholder litigation law firm, is investigating whether certain officers and directors of Intellia Therapeutics, Inc. (NASDAQ: NTLA) breached their fiduciary duties to shareholders. The investigation concerns potential self-dealing. Shareholders may be entitled to damages and corporate governance reforms. If you own NTLA please contact Justin Kuehn, Esq. by email at [email protected] or call (833) 672-0814. The consultation and case are free with n ...

The company has appointed Edward Dulac as its new CFO, executive vice president and treasurer. He will assume his new role with effect from Jul 22, 2024. Year to date, shares of Intellia have plunged 24.8% compared with the industry's decline of 4.6%. Intellia is a genome editing company focused on developing CRISPR/Cas9-based therapeutics. The company is developing its lead in vivo genome-editing candidate, NTLA-2001, for the treatment of transthyretin (ATTR) amyloidosis. NTLA-2001 is part of the company's ...

CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the appointment of Edward Dulac as Executive Vice President, Chief Financial Officer (CFO), and Treasurer, effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. "Ed's deep financial and business development experience at cli ...

CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the appointment of Edward Dulac as Executive Vice President, Chief Financial Officer (CFO), and Treasurer, effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. Mr. Dulac is a highly accomplished biotechnology business leade ...

"Today's data showcase an exciting new platform advancement for Intellia and the field of gene editing. For the first time ever, we demonstrated that redosing with CRISPR, utilizing our proprietary, non-viral LNP-based delivery platform, enabled an additive pharmacodynamic effect on the target protein," said Intellia President and Chief Executive Officer John Leonard, M.D. "While redosing is not planned for the NTLA-2001 program for the treatment of transthyretin amyloidosis, part of our commitment to patie ...