Photo credit: Homology Medicines

Novartis has revealed plans to collaborate with start-up, Homology Medicines to expand its programs in gene therapy and gene editing to develop therapies for genetic blood and eye diseases. 

Homology suggests that varieties of AAV recently discovered in healthy human blood stem cells could offer new capabilities, reports c&en, largely due to their large levels of uptake into the body compared with older versions, and their unique version of gene editing. 

When an AAV enters a cell it usually dumps its therapeutic DNA cargo into the cell’s nucleus, and then the DNA floats around until it is selected to produce a healthy protein to treat a disease. Homology on the other hand, believes it can also use its AAVs to do gene editing. In this case, the healthy DNA from the AAV precisely targets the mutant DNA and swaps places with it, allowing a permanent correction. 

Director in the chemical biology and therapeutics group at the Novartis Institutes for Biomedical Research (NIBR), Craig Mickanin, said, “This has huge potential to add to our toolbox.”

He continues to explain that Homology’s AAV system, doesn’t need to cut DNA strands to replace old or insert new DNA. Unlike CRISPR, it could avoid accidentally causing a new mutation during the repair process. 

Homology CEO, Arthur Tzianabos claims that the firm’s version of gene editing is based on spindly arms, hundreds of nucleotides long, that are complimentary to the surrounding stretches of DNA targeted for editing.

One primary focus of the Novartis-Homology partnership is genetic diseases affecting hemoglobin, of which sickle cell anemia is an example. Novartis has a collaboration with Intellia Therapeutics to use CRISPR gene editing to fix genetic mutations in blood stem cells. This approach requires the cells to be removed from a patient’s body, edited, and reinjected. 

Tzianbos added, “We believe the next iteration of this is our in vivo approach.” The therapy would be injected directly into the blood, without the complications of a bone marrow transplant. 

“It’s exciting times for cell and gene therapy,” said Mickanin, as he touches on the fact that the recent US Food and Drug Administration (FDA) approval of the Novartis cancer cell therapy Kymriah has “accelerated our interest in extending our leadership in cell and gene therapies.”