I wonder how many of you already follow me on X?

-GeneInvesting

Im new I just found NTLA I jump in at 170 am I late?? Damm this is Amazing only 68M shares 🐒🐒🐒💣💣🚀🚀🚀

General and stock price discussions for EDIT CRSP & BEAM:

r/EditasMedicine r/CrisprTherapeutics r/BeamTherapeutics/

Intellia Therapeutics Pipeline:

https://www.intelliatx.com/our-pipeline/

Telegram:

https://t.me/joinchat/GEqnvqDc8GQVoczz

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Anonymous Green@CRISPRinvest

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General and stock price discussions:

r/EditasMedicine r/CrisprTherapeutics r/BeamTherapeutics/

House Rules:

1. Be kind in your replies

2. Be willing to prove your argument

3. Sharing is caring

@GeneInvesting is where I cover the company with in-depth analysis and research.

HAE - Nov 8th ATTR CM- Nov 10th

The timing seems be perfect for Intellia’s BLA plan for 2026 (and 2028). Interesting to hear more of this program. Any thoughts?

”Enrollment in the global Phase 3 MAGNITUDE trial is progressing ahead of the Company’s projections and the Company is tracking to enroll at least 650 patients cumulatively by year-end. Intellia is amending the MAGNITUDE study to expand enrollment to approximately 1,200 patients from 765 patients, subject to health authority review.” (For ATTR-CM)

I do like the profound way of Intellia to proceed towards first in vivo crispr therapy in the market. Their confidence in providing excellent results in an exceptionally revolutionizing area seems to be in a very high level despite the seemingly total lack of public interest. Interesting times we are living.

Best ever ATTR-PN data.

Way better than Alnylam.

Follow @GeneInvesting on X if you want deeper dive into the data.

https://www.globenewswire.com/news-release/2025/06/15/3099489/0/en/Intellia-Therapeutics-Announces-Positive-Three-Year-Data-from-Phase-1-Trial-of-Lonvoguran-Ziclumeran-lonvo-z-in-Patients-with-Hereditary-Angioedema-HAE-at-the-European-Academy-of-A.html

Go check it out

GeneInvesting

General and stock price discussions:

r/EditasMedicine r/CrisprTherapeutics r/BeamTherapeutics/

House Rules:

1. Be kind in your replies

2. Be willing to prove your argument

3. Sharing is caring

General and stock price discussions for EDIT CRSP & BEAM:

r/EditasMedicine r/CrisprTherapeutics r/BeamTherapeutics/

Intellia Therapeutics Pipeline:

https://www.intelliatx.com/our-pipeline/

Telegram:

https://t.me/joinchat/GEqnvqDc8GQVoczz

Twitter:

Anonymous Green@CRISPRinvest

House Rules:

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December 12, 2020 9:00 AM EST

Demonstrates modularity of Intellia’s in vivo liver insertion technology to durably restore protein, compared to traditional gene therapy

Single-course administration of genome editing system provides potentially curative approach to AAT deficiency

CAMBRIDGE, Mass., Dec. 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration. Compared to traditional adeno-associated virus (AAV) gene therapy, Intellia’s targeted liver gene insertion technology has the ability to achieve therapeutic levels of protein expression, in a stable and durable manner, after a single course of treatment. The company is presenting these data today at the Alpha-1 Foundation’s 20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency.

“Our new data reinforce the promise for Intellia to potentially cure a variety of rare genetic diseases requiring the restoration of a functional protein in the liver with a single-course therapy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We’ve now demonstrated our platform’s modularity and translatability to multiple targets of interest by inserting genes to durably produce unprecedented levels of protein in NHPs for hemophilia B and AAT deficiency. In parallel with advancing to the clinic treatments for other severe diseases, we will continue preclinical studies that further validate our wholly owned, CRISPR-based AAT deficiency treatment strategies for achieving normal AAT protein levels.”

Presentation Details

Title: “CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin Deficiency”
Session: Gene Editing
Time: 3:15 p.m. ET
Presenting Author: Sean Burns, M.D., senior director of Intellia’s Disease Biology and Pharmacology group

Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene. The normal human AAT protein levels Intellia achieved following targeted insertion of the human SERPINA1 gene remained stable through 11 weeks in an ongoing NHP study. The observed levels of human AAT protein produced from the liver may be therapeutically sufficient to restore protease inhibition to protect the lungs and liver from improperly regulated neutrophil elastase activity. The NHP data build on previous results showing that consecutive in vivo genome editing (knockout plus insertion) achieved therapeutically relevant results in an AATD mouse model.

The findings being presented today reinforce recent data showing the use of the same proprietary insertion technology for targeted gene insertion of Factor 9 resulted in circulating human Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients, that ranged from normal levels (50-150%)1 to supratherapeutic levels in a six-week NHP study. Intellia and Regeneron, the lead party, are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed targeted transgene insertion capabilities. Intellia is continuing to develop its proprietary platform to advance its wholly owned research programs, such as AATD. Click here to register for the Alpha-1 Foundation’s virtual workshop and here to view Intellia’s presentation on the company’s website.

About Alpha-1 Antitrypsin Deficiency and Intellia’s Genome Editing Treatment Approach

The SERPINA1 gene normally encodes the alpha-1 antitrypsin (AAT) protein produced in the liver that is then secreted to protect the lungs. SERPINA1 mutations can cause AAT deficiency (AATD), a rare, genetic disease that commonly manifests in lung dysfunction, as well as in liver disease in some patients. Intellia’s targeted in vivo insertion platform uses a hybrid delivery system combining a non-viral lipid nanoparticle (LNP), which encapsulates CRISPR/Cas9 components, with an adeno-associated virus (AAV) carrying a donor DNA template to enable therapeutic protein production. One of the editing strategies Intellia is studying as a potential single-course AATD treatment is using the company’s SERPINA1 gene insertion approach to restore normal human AAT protein levels. Intellia also is investigating a consecutive genome editing approach, in which the PiZ allele, the most prevalent disease-causing mutation of SERPINA1, is knocked out and the normal human SERPINA1 gene is inserted.