Collaboration will explore potential of small viruses in cell and gene therapy

Novartis has announced a collaboration with biotech company, Homology Medicines, to adapt and refine the technology for the treatment of a blood disorder and certain eye diseases.

Homology Medicines has a proprietary adeno-associated viruses (AAV) platform that, through this collaboration, will be used to accelerate an initiative at the Novartis Institutes for BioMedical Research (NIBR), engaging researchers involved in the genetic reprogramming of cells.

“AAV biology is one of the most febrile areas of basic research, and we’re planning to explore its therapeutic potential through a new collaboration,” explained Craig Mickanin, who focuses on new tools and technologies as a director at NIBR.

“It is our hope that this collaboration will help advance our Cell and Gene Therapy initiative,” added Susan Stevenson, an executive director at NIBR who leads the initiative.

AAVs are small viruses that are attractive for genome editing as, unlike larger viruses, they do not seem to cause illness. These viruses can be engineered to carry a specific genetic sequence and programmed to home in on a target site in the genome. Once they arrive at the target site, they trigger homologous recombination — a process that overwrites a particular portion of a gene or completely replaces the gene. Through this process, AAVs may have the potential to correct genetic defects.

“We aim to select the right tool for the right project,” continued Mickanin. “In some cases, that will mean using AAVs to correct a genetic defect rather than disabling a gene.”

This collaboration will involve three work streams, the first of which will focus on a blood disorder. It is hoped that by working together a single AAV reagent that can be injected into the bloodstream of a patient with a defective gene to cure the disease can be designed. “We want to figure out if these AAVs are safe enough to inject directly into the bloodstream — and if we can use them to fix a defective gene once and for all,” stated Stevenson.

Secondly, the collaborative team will look at diseases of the eye. This is an important area for gene editing therapies as they can be delivered locally to the eye. For example, gene editing agents can be injected directly under the retina, where it is hoped they will work without affecting the rest of the body.

“The fact that we can directly observe the treatment and its effects in the eye gives us an important opportunity for assessing gene editing efficacy and helping patients with eye disease,” said Cynthia Grosskreutz, global head of Ophthalmology at NIBR.

Finally, the third work stream, exploratory, will enable researchers across NIBR to nominate projects that they believe could benefit from Homology’s AAV technology. “This technology could be applied to many different diseases,” Mickanin summarised. “We’re excited to work with the Homology team to explore the possibilities.”

In addition to this collaboration, Novartis has made an equity investment in Homology Medicines.