- While CRISPR-based gene editing might garner the most headlines, Swiss pharma Novartis AG is betting another form of genetic reprogramming called homologous recombination could provide another approach to treat certain inherited diseases.
- Novartis will make an undisclosed upfront payment and equity investment in Homology Medicine, a gene editing start-up headed by former Shire plc executives, the biotech announced Monday.
- Under the research collaboration, the two companies are looking to develop treatments aimed at certain opthalmic and hemoglobinopathy diseases, tapping Homology's stem-cell derived adeno associated vector (AAV) technology.
Homology launched in May of 2016, raising nearly $44 million in a venture financing led by initial backer 5AM Ventures. The biotech added to that haul with another $83.5 million in Series B funding this past August, including an investment from Novartis.
Led by CEO Arthur Tzianabos, Homology aims to prove homologous recombination — a natural mechanism that in certain instances overwrites DNA sequences during cell division — could be a more efficient and targeted approach to modifying genes.
Homology proposes to deliver a correct DNA strand via an AAV vector in vivo in order to induce the process. The possibility of using AAVs and homologous recombination has been raised previously, but the frequency with which the process occurred appeared to be too low.
Now, Homology thinks it has hit on a better approach, using AAVs licensed from the City of Hope and Saswati Chatterjee. However, with little published evidence, the company's efforts have been met with some skepticism.
Per the deal with Novartis, Homology will grant the Swiss pharma exclusive rights to the biotech's technology for select ophthalmic targets and an unspecified hemoglobinopathy disease. Homology retains U.S. rights and would split any future U.S. profits with Novartis for in vivo applications of the hemoglobinopathy program.
For the pharma, the collaboration is meant to complement ongoing work into gene modification at the Novartis Institutes for BioMedical Research. A blog post published by Novartis Monday noted that AAV-mediated homologous recombination could be more effective than CRISPR in situations where correcting an existing gene is the main goal rather than deleting and replacing.
On its own, Homology notes it is currently in an IND-enabling study for potential candidates to treat an inborn error of metabolism disease.