Intellia Therape Stock Price, News & Analysis

Intellia Therapeutics (NTLA) announced promising interim results from its Phase 1 trial of NTLA-2001, the first in vivo CRISPR genome editing therapy for hereditary transthyretin amyloidosis with polyneuropathy. A 0.3 mg/kg dose led to an 87% mean reduction in serum TTR levels, peaking at 96% by day 28. The treatment exhibited a favorable safety profile with no serious adverse events in the first six patients. These findings, published in The New England Journal of Medicine, highlight NTLA-2001's potential as a single-dose therapy that could transform treatment outcomes for this serious condition.

Intellia Therapeutics (NASDAQ: NTLA) has appointed James Basta as Executive Vice President, General Counsel, and Corporate Secretary. With over 25 years of legal experience, Basta previously served as Chief Legal Officer at Kura Oncology and held significant roles at Biogen. He succeeds José Rivera, a founding employee, who will assist during the transition. Intellia's leadership believes Basta's expertise will bolster their mission to develop curative genome editing therapies. Forward-looking statements address potential risks in advancing their CRISPR/Cas9 technology and pipeline.

Blackstone (NYSE: BX) has announced a $250 million investment to establish a new CAR T-cell therapy company alongside Intellia Therapeutics (NASDAQ: NTLA) and Cellex. This venture will feature a blend of GEMoaB's advanced CAR-T technology and Intellia's CRISPR genomic engineering. Aimed at improving efficacy and safety across various cancers, the new company will be based in Cambridge, Massachusetts, with operations in Germany. Andrew Schiermeier will lead this initiative, focusing on providing innovative treatments for tough-to-treat cancers and autoimmune conditions.

Intellia Therapeutics (NASDAQ:NTLA) announced that interim Phase 1 data from its lead CRISPR therapy, NTLA-2001, will be presented on June 26. This therapy aims to treat transthyretin amyloidosis (ATTR) with a single, systemically administered dose. The ongoing trial targets hereditary ATTR amyloidosis with polyneuropathy. Initial results will assess safety and pharmacodynamics, presenting a potential breakthrough in treatment compared to the traditional lifelong therapies. Following the Phase 1 trial, further studies will evaluate NTLA-2001's effectiveness in a broader ATTR population.

Intellia Therapeutics (NASDAQ:NTLA) reported first-quarter 2021 results, ending March 31 with $600.8 million in cash. The company plans to announce initial safety data for NTLA-2001, a CRISPR-based therapy for transthyretin amyloidosis (ATTR), in mid-2021 and to submit IND applications for NTLA-5001 (acute myeloid leukemia) and NTLA-2002 (hereditary angioedema) in 2021. Research and development expenses increased to $39.3 million due to advancements in key programs. The company recorded a net loss of $46.2 million, up from $31.8 million year-over-year.

Intellia Therapeutics (NASDAQ:NTLA) announced it will present its first quarter 2021 financial results and operational highlights on May 6, 2021, at 8 a.m. ET. The conference call will allow investors to understand the company's progress in developing proprietary therapeutics using CRISPR/Cas9 technology. U.S. callers can dial 1-833-316-0545; international callers should use 1-412-317-5726. A replay will be available on the company's website later that day.

Intellia Therapeutics (NASDAQ:NTLA) announced updated preclinical data on CRISPR/Cas9-mediated gene insertion for treating alpha-1 antitrypsin deficiency (AATD) at the ASGCT Annual Meeting, held virtually from May 11-14, 2021. The findings suggest a potential to restore normal AAT protein levels after a single dose, showcasing Intellia's commitment to innovative treatments. Presentations from key executives will cover topics such as genome editing strategies and on-target safety assessments. Further information is expected to be released during the meeting.

Intellia Therapeutics (NASDAQ: NTLA) has appointed Dr. Georgia Keresty to its Board of Directors, enhancing its leadership team. Dr. Keresty brings over 35 years of pharmaceutical experience, having held senior roles in leading companies like Takeda, Johnson & Johnson, and Bristol-Myers Squibb. Her expertise encompasses transitioning therapeutic programs to clinical development and improving manufacturing processes. Intellia aims to leverage her knowledge as it advances its CRISPR/Cas9 technology towards curative therapies.

Intellia Therapeutics (NASDAQ:NTLA) has received orphan drug designation from the European Commission for NTLA-2001, aimed at treating transthyretin amyloidosis (ATTR). This investigational CRISPR therapy is the first of its kind to be delivered systemically via intravenous dosing, targeting the TTR gene responsible for producing misfolded proteins. This milestone is significant for the ATTR community, where no existing cure is available. The therapy shows potential to halt and reverse ATTR progression and is part of a co-development agreement with Regeneron.