Company Reports

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Intellia reported $7.7 million in second-quarter collaboration revenue and submitted its US filing for lonvo-z in hereditary angioedema. Intellia ended the quarter with a net loss of $106 million and $628 million in cash and cash equivalents.

Intellia posted $15 million in first‑quarter collaboration revenue, and the pipeline is progressing, as highlighted by positive phase 3 top-line data for lonvo‑z in hereditary angioedema. Net loss narrowed to $96 million in the quarter, from a loss of $114 million in the prior-year period.

The US Food and Drug Administration lifted Intellia's clinical hold on its gene therapy trial for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) treated with nex-z after previous reports of serious liver toxicity led to a patient death in the trial.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Intellia’s shares plummeted more than 20% on Nov. 7 after news that a patient treated with its pipeline candidate Nex-Z, who had been hospitalized on Oct. 24 with elevated liver enzymes, has died. The company is investigating the liver-related events and developing a risk mitigation plan.

Intellia's shares tanked over 40% on Oct. 27 after it paused two phase 3 trials for Nex-z in transthyretin amyloidosis with cardiomyopathy and polyneuropathy, respectively. One patient receiving the treatment for ATTR-CM was hospitalized due to severe signs of liver damage.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Intellia reported $14.2 million in collaboration revenue in the second quarter, and the company's phase 3 pipeline continues to enroll patients in trials for hereditary angioedema and ATTR with cardiomyopathy and polyneuropathy.

No-moat Intellia’s shares dropped 23% after the company disclosed one patient exhibited signs of severe liver stress in its ongoing nex-z phase 3 trial for transthyretin amyloidosis with cardiomyopathy, or ATTR-CM. Management stated that the patient’s elevated liver enzymes are resolving and have fallen to less dangerous levels. However, investors’ response underscores the heightened risk Intellia faces from clinical trial setbacks, given its lack of approved drugs and reliance on its pipeline candidates.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

No-moat Intellia Therapeutics reported first-quarter results in line with our expectations, as its gene editing pipeline is continuing to make progress. The company is on track to complete enrollment of its global phase 3 study evaluating NTLA-2002 in hereditary angioedema in the third quarter. We assign a 35% probability of approval to this candidate, and it could reach the market as early as 2028. Intellia is also making progress with its pipeline candidate, Nex-z in ATTR amyloidosis with polyneuropathy, which dosed it first patient in the global phase 3 trial. We assign this pipeline candidate a 40% probability of approval, and we anticipate it could reach the market as early as 2027. Additionally, enrollment in the phase 3 trial for Nex-z in patients with ATTR amyloidosis with cardiomyopathy is tracking ahead of management’s expectations. Investors reacted favorably to Intellia’s progress and sent the stock up 15%.

No-moat Intellia Therapeutics’ gene editing pipeline is continuing to make progress as it dosed the first patient in its global phase 3 study evaluating NTLA-2002 in hereditary angioedema. Management has made the strategic decision to prioritize late-stage programs—NTLA-2002 and Nex-z (formerly NTLA-2001 in ATTR amyloidosis) to focus on near-term value creation. As a result, management discontinued NTLA-3001 (a gene editing treatment for lung disease) and it is reducing its workforce by approximately 27% in 2025. Intellia expects to incur charges of approximately $8 million for severance and other employee termination-related costs in the first quarter of 2025.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

No-moat Intellia Therapeutics' early-stage gene editing pipeline is continuing to make progress. We maintain our positive long-term outlook and fair value estimate of $85 per share. We view the stock as very undervalued, currently trading in 5-star territory. Intellia provides pure-play exposure to novel gene editing technology for long-term investors with a very high tolerance for risk. Pipeline candidate, NTLA-2002, is designed to inactivate a gene involved in potentially life-threatening inflammatory attacks for people suffering from hereditary angioedema. We like that Intellia is actively screening patients for its phase 3 study evaluating NTLA-2002, and we assign this pipeline candidate a 35% probability of approval in our base case. The clinical data looks positive, and we expect the phase 3 data could be released in 2026. Given time to review the data, submit regulatory filings, and prepare for a launch, we think it could reach the market in 2028, pending approvals.

No-moat Intellia Therapeutics released detailed phase 2 results from a study of a gene-editing therapy for hereditary angioedema, which drew mixed reactions from investors and medical experts and sent the stock down 20% on Oct. 24. The pipeline candidate, NTLA-2002, is designed to inactivate a gene involved in potentially life-threatening inflammatory attacks for people suffering from this rare disorder. In the ongoing phase 2 study of 27 patients, researchers found either a 75% or 77% reduction in attacks in the 16-week period after receiving therapy, depending on whether patients received the 25 mg or 50 mg dosages. Some of the data looks very promising, as 8 of the 11 patients in the higher-dose arm were completely free of attacks after a one-time infusion, and all these patients continued to be attack-free through the latest follow-up, which was a median of eight months.

Intellia Therapeutics' early-stage gene editing pipeline is continuing to make progress. We like that Intellia is on track to initiate the phase 3 study of NTLA-2001 for the treatment of hereditary ATTR amyloidosis with polyneuropathy by year-end. We assign this pipeline candidate a 40% probability of approval in our base case, and we think it could reach the market as early as 2026. We maintain our positive long-term outlook and fair value estimate of $85 per share. We view the stock as very undervalued, currently trading in 5-star territory. No-moat Intellia provides pure-play exposure to novel gene editing technology for long-term investors with a very high-risk tolerance.

Intellia Therapeutics’ relatively early-stage gene editing pipeline continued to make progress in the first quarter. We have raised our estimated probability of approval for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy and polyneuropathy to 40% from 30% as the company has quickly enrolled patients in its phase 3 trial for ATTR amyloidosis with cardiomyopathy, and it is tracking ahead of projections. We anticipate research and development costs as a percentage of sales will remain elevated over the next few years as Intellia focuses on developing its pipeline candidates.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Intellia Therapeutics’ gene editing pipeline continues to make progress. We like that Regeneron extended its technology collaboration with the company to April 2026; Intellia will receive a $30 million payment in April 2024 as part of that extension. Intellia and Regeneron are developing in vivo Crispr-based gene editing therapies focused on neurological and muscular diseases, a collaboration that will leverage Regeneron’s proprietary antibody-targeted adeno-associated virus vectors and delivery systems and Intellia’s proprietary Crispr/Cas9 systems adapted for viral vector delivery and designed to precisely modify a target gene.