Regeneron Pharmaceuticals has entered an expanded research collaboration with Intellia Therapeutics to develop clustered, regularly interspaced, short palindromic repeats (CRISPR)-based therapies.

The companies will develop additional in vivo CRISPR-based gene editing therapies to treat neurological and muscular diseases.

The partnership will utilise the antibody-targeted adeno-associated virus (AAV) vectors and delivery systems of Regeneron along with Intellia’s Nme2 CRISPR/Cas9 systems for viral vector delivery and for precisely modifying a target gene.

The companies will initially assess two in vivo non-liver targets.

Intellia will be responsible for designing the editing methodology, while Regeneron will lead the design of the targeted viral vector delivery method.

Both will have the chance to lead on the potential development and commercialisation of product candidates for a single target.

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The company not leading these activities can sign a co-development and co-commercialisation agreement for the target.

Regeneron genetic medicines co-head and research senior vice-president Christos Kyratsous stated: “Regeneron has invented and preclinically validated a proprietary antibody-directed AAV approach that builds on our decades of experience in antibodies and newly developed AAV capsid engineering technologies to deliver innovative payloads across many targeted tissue types and disease settings.”

In April 2016, the companies signed a licensing and collaboration agreement to discover and develop CRISPR/Cas gene-editing technology for in vivo therapeutics.

In June 2020, they expanded their collaboration to jointly develop products for the treatment of haemophilia A and B.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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