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BioCentury

Gene editing

Part 1

Part 2

Crispr Tx

Part 3

Editas

Part 4

Intellia

Part 5

Caribou

ARTICLE | Product Development

Intellia: Balancing in vivo risk with validated targets

Sticking with genetic targets validated by Alnylam and Ionis gave Intellia confidence to take CRISPR in vivo

By Lauren Martz, Executive Director, Biopharma Intelligence
February 28, 2025 12:15 AM UTC

Intellia’s decision in 2014 to develop systemically delivered, in vivo gene editing therapies meant embracing some of the highest levels of technology risk ever encountered by a new technology platform. The biotech offset that risk by selecting targets already validated by other genetic medicine modalities in humans.

The company now appears to be on a path to clinical success, but the tradeoff of its target and indication selection strategy may be modest commercial opportunity...

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BCIQ Company Profiles

Alnylam Pharmaceuticals Inc.

Crispr Therapeutics AG

Intellia Therapeutics Inc.

Ionis Pharmaceuticals Inc.