This section highlights FDA-related milestones and regulatory updates for drugs developed by Intellia Therapeutics (NTLA).
Over the past two years, Intellia Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
nex-z, NTLA-2001, NTLA-2002, and NTLA-3001. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
nex-z - FDA Regulatory Timeline and Events
nex-z is a drug developed by Intellia Therapeutics for the following indication: For Transthyretin (ATTR) Amyloidosis with Polyneuropathy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- nex-z
- Announced Date:
- May 18, 2025
- Indication:
- For Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Announcement
Intellia Therapeutics, Inc. announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
AI Summary
Intellia Therapeutics recently shared promising two-year follow-up data from its ongoing Phase 1 trial of nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The results show that a single dose of nex-z led to rapid, deep, and durable reductions in serum transthyretin (TTR) levels, maintaining these reductions consistently for at least 24 months. This sustained decrease in TTR is key because lower TTR levels are believed to improve patient outcomes.
Importantly, the study also noted clinically meaningful improvements in disease-related measures, even for patients who had previously worsened on patisiran. The safety profile remains generally favorable, with no new drug-related adverse events reported during the follow-up period. These encouraging findings support further development of nex-z and underline its potential as a one-time treatment option for ATTR amyloidosis.
Read Announcement- Drug:
- nex-z
- Announced Date:
- April 3, 2025
- Indication:
- For Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Announcement
Intellia Therapeutics announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
AI Summary
Intellia Therapeutics announced that the first patient has been dosed in the MAGNITUDE-2 trial, a global, pivotal Phase 3 study designed to test nexiguran ziclumeran (nex-z) for treating hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). This randomized, double-blind, placebo-controlled trial will include around 50 patients and is testing whether a single dose of nex-z can halt or reverse the progression of this debilitating condition.
Nex-z is a CRISPR-based in vivo therapy that works by inactivating the TTR gene, which is responsible for producing the abnormal protein that leads to the buildup causing nerve damage. Early Phase 1 data shows that a single dose dramatically reduces serum TTR levels. Intellia is optimistic that the MAGNITUDE-2 trial will demonstrate nex-z’s potential as a groundbreaking treatment for ATTRv-PN.
Read Announcement
NTLA-2001 - FDA Regulatory Timeline and Events
NTLA-2001 is a drug developed by Intellia Therapeutics for the following indication: Transthyretin (ATTR) Amyloidosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NTLA-2001
- Announced Date:
- March 26, 2025
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Intellia Therapeutics announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its CRISPR-based treatment, nexiguran ziclumeran (nex-z, also known as NTLA-2001). This important designation is for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a serious condition that affects heart function. The RMAT status is part of an effort under the 21st Century Cures Act to speed up the development and review of promising therapies aimed at life-threatening diseases. It offers benefits such as early interactions with the FDA, discussions on surrogate endpoints, and potentially faster review times. Intellia’s CEO, John Leonard, M.D., said this designation represents a significant step toward accelerating the treatment’s development so that patients might receive this transformative therapy sooner.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- November 25, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).
AI Summary
Intellia Therapeutics, Inc. announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational treatment nexiguran ziclumeran (nex-z, also known as NTLA-2001) for hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is a one-time, in vivo CRISPR-based therapy designed to inactivate the TTR gene, which stops the body from producing the TTR protein that forms harmful amyloid deposits. Early interim Phase 1 data showed that nex-z achieved rapid, deep, and lasting TTR reduction, suggesting the treatment could halt and possibly reverse disease progression. With RMAT designation, Intellia will benefit from early FDA interactions that may help speed up development and review. This status is expected to promote a faster path toward potential regulatory approval, providing hope for patients with this serious and life-threatening condition.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- November 16, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for ATTR amyloidosis. .
AI Summary
Intellia Therapeutics announced positive Phase 1 clinical data for nexiguran ziclumeran (nex-z, also known as NTLA-2001), an investigational in vivo CRISPR-based gene editing therapy. Nex-z is being developed as a one-time treatment for patients with transthyretin (ATTR) amyloidosis. The clinical trial data show that a single infusion of nex-z can produce rapid, deep, and durable reduction in serum TTR levels. This significant TTR reduction is linked with signs of disease stabilization and improvement, particularly in patients with ATTR cardiomyopathy and hereditary ATTR amyloidosis with polyneuropathy. The observed efficacy suggests that greater TTR reduction may lead to improved clinical benefit by potentially slowing disease progression. Moreover, the data support the potential of nex-z as a breakthrough approach in treating ATTR amyloidosis with a favorable safety and tolerability profile.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- October 1, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc announced that interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex-z, also known as NTLA-2001) will be presented in a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, taking place November 16 – 18 in Chicago, Illinois.
AI Summary
Intellia Therapeutics, Inc. announced that interim data from its ongoing Phase 1 study of nexiguran ziclumeran (nex-z or NTLA-2001) will be shared during a late-breaking oral presentation at the 2024 American Heart Association Scientific Sessions in Chicago, Illinois from November 16 to 18. The study focuses on using this investigational in vivo CRISPR gene editing therapy as a potential one-time treatment for transthyretin (ATTR) amyloidosis, a disease that can cause heart complications. During the session led by Dr. Marianna Fontana, the presentation will reveal important information on safety, reductions in serum TTR levels, and biomarkers of disease progression. This data may provide key insights into how nex-z affects functional capacity in patients with ATTR amyloidosis with cardiomyopathy. The upcoming presentation is part of Intellia’s continued efforts to develop innovative therapies using CRISPR technology for life-changing treatments.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- June 25, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, presented new data demonstrating for the first time the potential for redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy.
AI Summary
Intellia Therapeutics has revealed promising new data showing for the first time that redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy is possible. In their ongoing Phase 1 study of NTLA-2001, an experimental treatment for transthyretin (ATTR) amyloidosis, patients who initially received a lower dose were later given a follow-on 55 mg dose. This second dose resulted in a 90% median reduction in the serum TTR protein by day 28, and a cumulative 95% reduction from baseline, demonstrating an additive effect when the redose was administered. The 55 mg dose was generally well tolerated, with only one patient experiencing a mild infusion-related reaction. Although redosing is not planned for the current ATTR amyloidosis program, these findings highlight the potential of Intellia’s non-viral, lipid nanoparticle (LNP)-based delivery platform for future therapies where multiple doses could be beneficial.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- June 17, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc. announced the acceptance of an abstract featuring redosing data from the Phase 1 study of NTLA-2001 has been selected for an oral presentation at the Peripheral Nerve Society Annual Meeting, taking place June 22 – 25 in Montreal, Canada. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis.
AI Summary
Intellia Therapeutics, Inc. announced that an abstract featuring redosing data from its Phase 1 study of NTLA-2001 has been accepted for oral presentation at the Peripheral Nerve Society Annual Meeting in Montreal, Canada, from June 22–25, 2024. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed as a one-time treatment for transthyretin (ATTR) amyloidosis. In the Phase 1 study, the first three patients were given a low initial dose of 0.1 mg/kg, followed by a higher follow-on dose of 55 mg. These findings provide the first clinical data on repeat dosing with an in vivo CRISPR candidate, shedding light on its safety profile and pharmacodynamic effects. While NTLA-2001 is intended as a single-dose treatment, the option to redose could be an important benefit for future therapies using Intellia’s non-viral lipid nanoparticle delivery platform.
Read Announcement
NTLA-2002 - FDA Regulatory Timeline and Events
NTLA-2002 is a drug developed by Intellia Therapeutics for the following indication: Hereditary Angioedema (HAE).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NTLA-2002
- Announced Date:
- June 15, 2025
- Indication:
- Hereditary Angioedema (HAE)
Announcement
Intellia Therapeutics, announced three-year follow-up data from the Phase 1 portion of the ongoing Phase 1/2 study in patients with HAE after receiving a single dose of lonvoguran ziclumeran (lonvo-z, also known as NTLA-2002). Results were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held June 13-16 in Glasgow, United Kingdom.
AI Summary
Intellia Therapeutics announced promising three-year follow-up data from the Phase 1 portion of its ongoing Phase 1/2 trial of lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE). In an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025 in Glasgow, the company shared that a single infusion of lonvo-z resulted in a 98% mean reduction in the monthly HAE attack rate over the study period. Additionally, all 10 patients remained attack-free and did not require additional treatment for a median of nearly two years.
The treatment showed deep, dose-dependent, and durable reductions in plasma kallikrein protein while demonstrating a favorable safety profile with mainly mild infusion-related reactions. These findings highlight lonvo-z’s potential as a one-time gene editing therapy to free HAE patients from both attacks and the challenges of chronic treatment.
Read Announcement- Drug:
- NTLA-2002
- Announced Date:
- February 27, 2025
- Indication:
- Hereditary Angioedema (HAE)
Announcement
Intellia Therapeutics, today reported operational highlights
AI Summary
Intellia Therapeutics recently announced key operational highlights focused on its NTLA-2002 program for hereditary angioedema (HAE). The company dosed the first patient in the global Phase 3 HAELO study, which is testing NTLA-2002, an investigational CRISPR-based therapy that targets the KLKB1 gene in the liver. This approach aims to provide lifelong control of HAE attacks with just a single dose. Intellia expects to complete patient enrollment for this study in the second half of 2025 and plans to submit a Biologics License Application in the latter half of 2026, supporting a planned U.S. launch in 2027. The update underscores strong operational progress and reflects the company’s focus on advancing innovative gene editing therapies, offering hope for a functional cure for HAE patients while setting the stage for future regulatory milestones.
Read Announcement- Drug:
- NTLA-2002
- Announced Date:
- January 22, 2025
- Indication:
- Hereditary Angioedema (HAE)
Announcement
Intellia Therapeutics, Inc. announced the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE).
AI Summary
Intellia Therapeutics, Inc. has taken a major step forward by dosing the first patient in its global Phase 3 study of NTLA-2002, a CRISPR-based gene editing therapy developed as a one-time treatment for hereditary angioedema (HAE). This groundbreaking study aims to evaluate the safety and effectiveness of NTLA-2002 in reducing and potentially eliminating HAE attacks, offering hope for patients to gain independence from the constant need for maintenance medications. The trial, which will enroll 60 adults with Type I or Type II HAE, uses a double-blind, randomized design and compares a single 50 mg infusion of NTLA-2002 against a placebo, with patients in the placebo group eligible for crossover at week 28. This advancement in gene editing research marks a milestone in the clinical development of NTLA-2002, with the company planning to complete enrollment in the second half of 2025 and submit regulatory applications in the coming years.
Read Announcement- Drug:
- NTLA-2002
- Announced Date:
- October 7, 2024
- Indication:
- Hereditary Angioedema (HAE)
Announcement
Intellia Therapeutics, Inc
AI Summary
Intellia Therapeutics, Inc. has advanced NTLA-2002—a single-dose, in vivo CRISPR-based gene editing therapy—into a global Phase 3 study called HAELO to treat hereditary angioedema (HAE). NTLA-2002 is designed to inactivate the KLKB1 gene, thereby preventing the severe and potentially life-threatening swelling attacks that characterize HAE. The therapy emerged from positive results in earlier Phase 1/2 studies, where significant reductions in attack frequency and kallikrein levels were noted.
Following a successful end-of-Phase 2 meeting, patient screening began after Intellia submitted an Investigational New Drug Application amendment to the U.S. Food and Drug Administration. The Phase 3 trial will enroll 60 adults with HAE and compare the effects of a single 50 mg infusion of NTLA-2002 to a placebo, with patients on placebo eligible for crossover. Intellia hopes that this one-time treatment approach will ultimately eliminate ongoing therapy for HAE patients.
Read Announcement- Drug:
- NTLA-2002
- Announced Date:
- September 12, 2024
- Indication:
- Hereditary Angioedema (HAE)
Announcement
Intellia Therapeutics announced that data from the Phase 2 study of NTLA-2002 will be presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.
AI Summary
Intellia Therapeutics announced that data from its Phase 2 trial of NTLA-2002, an investigational CRISPR-based gene editing therapy for hereditary angioedema, will be presented at the 2024 American College of Allergy, Asthma & Immunology Annual Scientific Meeting. The event is scheduled to take place in Boston, Massachusetts, from October 24 to 28, 2024. The detailed results will be shared in a session on Saturday, October 26, from 4:30 to 6:30 p.m. ET, where Dr. Danny Cohn from Amsterdam University Medical Center will present the findings under the title “Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema.”
In addition, Intellia will host an investor webcast on Monday, October 28, at 8:00 a.m. ET to review the new study data in detail, with a replay available on the company’s website for at least 30 days after the event.
Read Announcement
NTLA-3001 - FDA Regulatory Timeline and Events
NTLA-3001 is a drug developed by Intellia Therapeutics for the following indication: For the Treatment of Alpha-1 Antitrypsin Deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NTLA-3001
- Announced Date:
- July 30, 2024
- Indication:
- For the Treatment of Alpha-1 Antitrypsin Deficiency
Announcement
Intellia Therapeutics, Inc. announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom's Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease.
AI Summary
Intellia Therapeutics, Inc. recently received authorization from the United Kingdom’s MHRA for its Clinical Trial Application (CTA) to launch a Phase 1/2 study of NTLA-3001. This groundbreaking study will evaluate the gene editing candidate for treating lung disease linked to alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder that leads to low levels of the AAT protein and lung damage.
NTLA-3001 is the company’s first entirely in vivo CRISPR-based targeted gene insertion candidate. The therapy aims to insert a healthy copy of the SERPINA1 gene into the body, potentially normalizing AAT protein levels after just one dose. The trial, designed as a multicenter, open-label study involving up to 30 patients, will assess the treatment’s safety, tolerability, pharmacokinetics, and pharmacodynamics. This promising approach could offer a more durable solution than current treatments for AATD-associated lung disease.
Read Announcement- Drug:
- NTLA-3001
- Announced Date:
- July 30, 2024
- Estimated Event Date Range:
- July 1, 2024 - December 31, 2024
- Target Action Date:
- 2024-H2
- Indication:
- For the Treatment of Alpha-1 Antitrypsin Deficiency
Announcement
Intellia Therapeutics, Inc. announced that On track to dose the first patient in 2H 2024
AI Summary
Intellia Therapeutics announced that its potential one-time gene editing treatment, NTLA-3001, is on track to dose its first patient in the second half of 2024. This CRISPR-based therapy is designed to treat lung disease caused by alpha-1 antitrypsin deficiency (AATD) by inserting a healthy copy of the SERPINA1 gene into patients’ cells. The goal is to restore normal levels of AAT protein after a single treatment, which might eliminate the need for ongoing weekly infusions or even a lung transplant. The upcoming Phase 1/2 clinical trial, which has received regulatory approval in the United Kingdom, will evaluate the safety, effectiveness, and proper dosing of the therapy. Success in these early studies could support further development of Intellia’s gene insertion platform to address other diseases linked to defective or missing proteins.
Read Announcement
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