Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which could become the first in vivo gene-editing therapy to land FDA approval.

The post Intellia’s Data Reveal Tees Up FDA Filing for CRISPR-Based In Vivo Gene-Editing Med appeared first on MedCity News.

The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease.

The post Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease appeared first on MedCity News.

Intellia Therapeutics has encouraging early clinical data for a gene-editing therapy addressing the rare swelling disorder hereditary angioedema. This therapy, the biotech’s second CRISPR-editing based treatment that performs in vivo edits, offers potential for a one-time treatment of a disease that is currently addressed by chronic oral and injectable drugs.

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An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.

The post New data for Intellia’s CRISPR therapy shows potential for one-time treatment of a rare disease appeared first on MedCity News.

Gene-editing medicines developer Intellia Therapeutics is paying $45 million up front to acquire Rewrite Therapeutics. The startup’s technology brings Intellia another tool for its genetic medicines toolbox, one that could enable even more precise edits.

The post Intellia’s Rewrite Therapeutics buyout brings new precision to gene editing appeared first on MedCity News.

Intellia Therapeutics has early clinical trial data showing that its CRISPR-based therapy for a rare disease can edit genes inside the body safely and effectively. The trial is ongoing, but the preliminary results suggest that Intellia’s approach could offer a cure.

The post Intellia’s early CRISPR trial data validate a drug pipeline and the gene-editing field appeared first on MedCity News.

The Alliance for Regenerative Medicine and 13 biotech companies released a statement outlining bioethical principles on genome editing, including opposition to modifications that can be passed to future generations.

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The company, with ARIAD and Intellia veterans at the helm, aims to develop CTLA4-targeting monoclonal antibody that mitigates the immune toxicity of Bristol’s Yervoy.

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In a deal worth up to $25 million for ViaCyte, the companies will work together to create allogeneic stem cell therapies to treat the disease.

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