In a phase 1 trial, a gene-editing therapy that targets ANGPTL3 was associated with few serious adverse events and at higher ...

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...

A cutting-edge medical experiment in a small trial has demonstrated the effectiveness of a one-time CRISPR gene editing treatment in lowering cholesterol levels ...

A patient with transthyretin amyloidosis (ATTR) who received the investigational gene therapy nexiguran ziclumeran (nex-z) in ...

Intellia Therapeutics stock crashed Friday after a patient in its gene-editing study died following serious increases in liver enzymes.

Intellia Therapeutics has acknowledged the death of the elderly patient hospitalized last week in its Phase III trial of assessing nexiguran ziclumeran (nex-z) in transthyretin amyloidosis with ...

Intellia had already paused dosing in trials of nex-z in transthyretin amyloidosis after the patient was hospitalized due to a serious adverse event.

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex ...

Intellia Therapeutics (NTLA) stock plummets after the company's Q3 updates indicate a patient deeath in a Phase 3 for its nex ...

Lowering the transcription factor PU.1 reprograms microglia into a neuroprotective, lymphoid-like state that compacts amyloid ...

New diagnostic kits aim to revolutionize early screening of the disease, potentially allowing patients to receive ...