Intellia to stop work on rare disease therapy, lay off staff

Jan 10 , 2025
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Dive Brief:

  • Intellia Therapeutics will stop work on one of its principal drug research programs and lay off more than one-quarter of its staff in a restructuring meant to prioritize resources around its two most advanced experimental medicines.
  • The discontinued program, dubbed NTLA-3001, targets a rare lung disease known as alpha-1 antitrypsin deficiency. Intellia had begun a Phase 1/2 clinical trial testing the therapy in August. Alongside NTLA-3001, the company is also ending “select research-stage programs.”
  • Intellia will additionally prepare for a change in its scientific leadership as Laura Sepp-Lorenzino, the company’s chief scientific officer since May 2019, plans to retire at the end of the year. Birgit Schultes, who’s led Intellia’s immunology and cell therapy work since 2017, will take Sepp-Lorenzino’s place as CSO.

 

Dive Insight:

For the second time in a row, Intellia is starting the new year with layoffs and research cuts.

In January 2024, the CRISPR gene editing company said it would eliminate 15% of its workforce and pause some exploratory research. A few months prior, Intellia had nixed an earlier program for alpha-1 antitrypsin deficiency, also known as AATD.

At the time, Intellia said it intended to focus on enrolling pivotal studies of its two lead programs, launch new clinical trials and experiment with new gene editing and delivery tools.

This time around, Intellia’s primary concerns look a bit different. The company will prioritize clinical execution for those two programs, targeting the rare diseases hereditary angioedema and transthyretin amyloidosis, and “advance commercial readiness” to prepare for launching those medicines.

Both of those programs, dubbed NTLA-2002 and nex-z respectively, are now in Phase 3, and Intellia hopes to complete or speed enrollment into those trials.

“Our early clinical data for both NTLA-2002 and nex-z support novel, highly differentiated product profiles that directly address the significant unmet needs of patients and prescribers in HAE and ATTR,” said Intellia CEO John Leonard in a Thursday statement. “We understand the significant potential of our late-stage programs, and within a challenging market environment, have made a difficult decision to focus our resources predominantly on NTLA-2002 and nex-z where we have the greatest opportunity to create significant, near-term value.”

Intellia was one of a handful of companies targeting AATD with new drugmaking technologies like CRISPR. Wave Life Sciences, Korro Bio and ProQr are all developing RNA medicines for the disease, which can damage the liver as well as lungs.

Beam Therapeutics, meanwhile, expects to soon share initial trial data for an AATD therapy that uses what’s known as base editing to correct the disease-causing mutation.

The environment for gene editing companies has darkened over the past year or so. Several others have been forced to lay off staff and pare back their research plans in response to funding constraints or shifting market expectations.

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