NTLA-2001

Intellia Therapeutics' shares dropped pre-market after announcing a strategic reorganization to focus on late-stage pipeline candidates, including Nexiguran ziclumeran for ATTR amyloidosis and NTLA-2002 for hereditary angioedema. The company plans a 27% workforce reduction by 2025, incurring $8M in charges, aiming for a cash runway into 2027. Collaborations with Regeneron for Nexiguran ziclumeran's development continue, with ongoing phase III studies for ATTR amyloidosis and hereditary angioedema treatments.

2024 was challenging for healthcare, with patent expirations, cost scrutiny, and declining reimbursement rates. 2025 faces policy uncertainty under Trump, impacting Medicaid, ACA subsidies, and biopharma. Despite this, therapeutic innovation thrives, with advancements in obesity drugs, cancer treatments, HIV prevention, and ATTR-CM therapies.

In 2024, significant progress was made in cellular and genetic treatments, focusing on cardiology. Key developments included the first US trial of Nexcella’s CAR-T therapy for AL Amyloidosis, Intellia’s successful redosing of CRISPR therapy, Sardocor’s gene therapy for HFpEF receiving FDA fast track, Intellia’s phase 3 trial for ATTR-CM, and preserved cardiac function in DMD patients treated with SGT-001.

In 2024, FAP News Today highlighted advancements in FAP treatment, including disease-modifying drugs, gene-editing therapies, and liver transplants. Key findings showed treatments like Onpattro, Tafamidis, and Wainua effectively slow disease progression, with some therapies showing benefits in early stages or reducing heart damage.

Intellia Therapeutics announces FDA's RMAT designation for nexiguran ziclumeran (NTLA-2001) to treat hereditary ATTR amyloidosis with polyneuropathy, based on interim Phase 1 data showing rapid, deep, and durable TTR reduction.

Intellia Therapeutics reports early-stage data indicating its gene editing treatment, NTLA-2001, may stabilize or improve outcomes for ATTR-CM patients, showing a median improvement in the six-minute walk test and 92% maintaining or improving NYHA class. The treatment uses CRISPR to reduce TTR production, with a 90% mean reduction observed. Intellia is now in Phase III trials for NTLA-2001, with potential revenue of $1.2bn by 2030.

Intellia Therapeutics announced positive Phase 1 trial data for nex-z in ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction, disease stabilization or improvement, and favorable safety profile. Data presented at the 2024 AHA Scientific Sessions and published in the New England Journal of Medicine.

Intellia Therapeutics announces positive Phase 1 data for nex-z in ATTR amyloidosis, showing rapid, deep, and durable serum TTR reduction, disease stabilization or improvement, and favorable safety profile. Data presented at 2024 AHA Scientific Sessions and published in NEJM.

Intellia Therapeutics initiates Phase 3 HAELO trial for NTLA-2002, a CRISPR gene editing treatment targeting hereditary angioedema, aiming to reduce attacks and kallikrein protein levels. The trial will involve 60 adults, with a 2-to-1 randomization to NTLA-2002 or placebo, followed by a crossover phase. Intellia seeks to compete with Pfizer, BridgeBio Pharma, and Alnylam in rare disease treatments.