Clinical-stage biopharmaceutical firm Aruvant Sciences and Lonza have entered a strategic long-term manufacturing agreement for the former’s one-time investigational gene therapy for sickle cell disease (SCD), ARU-1801.

The gene therapy will be developed for Aruvant’s upcoming pivotal trial.

Designed to tackle the limitations of present curative treatment options, ARU-1801 has unique characteristics that permit it to be given with reduced intensity conditioning (RIC).

The investigational lentiviral gene therapy is currently under analysis as a one-time potentially curative treatment for SCD in the Phase I / II MOMENTUM study. The trial is now enrolling subjects.

According to preliminary clinical data, engraftment of ARU-1801 and amelioration of SCD is achievable with a single dose of low-intensity chemotherapy.

Aruvant chief technology officer Palani Palaniappan said: “We are partnering with Lonza, one of the world’s leading cell and gene therapy manufacturing organisations, to help us manufacture our gene therapy ARU-1801, a potential cure for sickle cell disease that can be given with one low dose of chemotherapy.

“Our internal cell therapy process expertise combined with Lonza’s cell processing know-how provide the perfect combination to manufacture ARU-1801 for our pivotal study.”

The collaboration with Lonza is a major move in progressing ARU-1801’s clinical development for sickle cell patients.

As directed by Aruvant, Lonza has initiated process development and technology transfer activities from its Houston centre of excellence to aid in setting up robust manufacturing for ARU-1801.

On concluding this, Lonza will provide cGMP material needed for the pivotal clinical trial.

Lonza senior vice-president, cell and gene technology head Alberto Santagostino said: “Our track record in the cell and gene therapy space makes us the ideal partner to help manufacture ARU-1801, a differentiated gene therapy that is an important future option for patients with sickle cell disease.”