Sanofi and Scribe Therapeutics have announced an expanded collaboration agreement worth over $1.2bn to advance in vivo genetic medicines for sickle cell and other genomic diseases.

The agreement follows the launch of the companies’ existing partnership focused on developing ex vivo editing of natural killer cell therapies for cancer.

Under the latest deal, Sanofi will gain an exclusive licence to use Scribe’s CRISPR X-Editing genome editing technologies to develop in vivo therapies.

In exchange, Scribe will receive a $40m upfront payment and will also be eligible for more than $1.2bn based on the successful completion of certain development and sales milestones.

Sanofi will largely be responsible for any resulting candidates, but Scribe will have the opportunity to opt into sharing the development costs and profits on one future programme in the US.

Christian Mueller, global head of Genomic Medicine Unit at Sanofi, said: “We’re encouraged by what we’ve accomplished to date with Scribe in creating ex vivo natural killer cell therapies and now look forward to accelerating our ability to effectively leverage genome editing in vivo through Sanofi’s innovative research work in targeted lipid nanoparticles, for in vivo therapies with the potential to dramatically improve treatment outcomes and ultimately to change patients’ lives.”

By directly modifying genes within the body, Scribe believes that its tools for in vivo genome editing offer “several key benefits” over existing treatment options and late-stage investigational therapies.

The approach being explored under the collaboration could streamline the treatment process for sickle cell disease and minimise complications associated with investigational ex vivo autologous treatments, Scribe said, such as conditioning regimen toxicities and a long, complex cell manufacturing process.

In vivo genome editing also has the potential to improve access for patients by reducing both the cost and time required for treatment.

“Scribe’s platform holds tremendous potential to address an unprecedented number of therapeutically relevant targets across the human genome for many serious diseases,” said Svetlana Lucas, chief business officer at Scribe.

She continued: “We look forward to our continued collaboration with Sanofi to expand the application of Scribe’s best-in-class genome editing tools and fully realise the promise of CRISPR for patients as broadly and rapidly as possible.”

Source: pmlive.com