Novartis Terminates Development of CRISPR-Based Sickle Cell Disease Therapy OTQ923/HIX763

On February 23, Intellia disclosed in its 2022 annual report that Novartis had terminated the development of the sickle cell disease (SCD) drug OTQ923/HIX763. It is a hematopoietic stem cell (HSCs) therapy developed by Intellia and Novartis based on CRISPR/Cas9 gene editing technology, currently in the Phase I/II clinical stage.

Novartis stated in an email to Fierce Biotech that the company decided to terminate the project's development mainly due to strategic considerations, without affecting its cooperative relationship with Intellia Therapeutics.

Intellia is a leading clinical-stage gene editing company primarily developing proprietary, potentially therapeutic treatments based on CRISPR/Cas9 technology. The company’s co-founders include Nobel Prize winner Jennifer Doudna.

The company has a deep-rooted history with Novartis, dating back 10 years. In November 2014, Intellia's Series A financing was led by Atlas Venture and Novartis. Additionally, in early 2015, Novartis entered into a five-year research and development collaboration with Intellia, focusing on leveraging CRISPR-Cas9 technology to develop chimeric antigen receptor T-cell (CAR-T) and hematopoietic stem cell (HSC) therapies. In 2018, the collaboration further expanded into the OSC (ocular stem cells) field.

In addition to Novartis, on the same day, Sangamo also announced the termination of the development of sickle cell disease drug BIVV003 (Phase I/II clinical trial), and Graphite Bio announced the termination of the development of sickle cell disease drug nulabeglogene autogedtemcel (Phase I/II clinical trial).

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