In the latest move to build up Novartis’ eye drug pipeline, the pharma giant is sinking fresh capital into a gene editing company to work on secret targets in ophthalmology.
Financial details weren’t disclosed, but we do know that Novartis made an upfront payment and an equity investment in the startup Homology Medicines. The deal gets Novartis access to Homology’s gene editing platform, which both companies hope will lead to new drug targets.
As part of the deal, Novartis will pay for further development of the platform, advancing new programs and leaving the R&D deal somewhat open-ended to explore other disease areas with the tech.
Homology’s gene editing tech differs from CRISPR, which uses an enzyme from bacteria that “chops” DNA to disrupt a damaged gene. Homology’s CEO Arthur Tzianabos said the CRISPR approach can be problematic when adding whole genes.
Instead, Homology is employing viral vectors to encourage what’s called “homologous recombination” — the body’s natural response to “correcting” genes that have been damaged. In short, the company packages a single strand of synthetic DNA inside a non-infectious virus, which then carries the DNA to the body’s cells.
“The DNA is designed to be a perfect mirror image of the DNA you want to take out,” Tzianabos said. Pairing the corrected DNA with its damaged twin inside the cell is what promotes homologous recombination, or the swapping of nucleotide sequences between two similar strands of DNA.
In theory, the healthy strand would swap or insert correct sequences where needed in patients with mutated or damaged genes. Out with the bad, damaged chunk of DNA, in with the new, correct one.
The idea isn’t new. LogicBio, based in Foster City, is doing something similar but with a different virus. Tzianabos said it’s Homology’s adeno-associated virus (AAV) that’s unique, and it allows them to correct “a lot more cells” than LogicBio can.
Novartis participated in Homology’s $83.5 million Series B round this August, and is now deepening its ties with the company with this new R&D agreement.
As part of the new deal with Novartis, Homology will be exploring undisclosed targets in ophthalmology (a key research area for Novartis), as well as targets in genetic blood disorders. Novartis has worldwide rights to Homology’s platform for select ophthalmic targets and an undisclosed hemoglobinopathy disease. Homology gets to retain US commercial rights (and a share of US profits) for in vivo applications related to the hemoglobinopathy program.
The collaboration makes sense for Novartis, as the company has a strong interest in ophthalmology. Its blockbuster Eylea rival RTH258 has been touted as a $1-billion-plus drug for wet age-related macular degeneration. Novartis plans to seek FDA approval for the drug by the end of next year.
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