Biotechnology News

Novartis Acquires Gene Therapy Company to Expand Portfolio

The acquisition adds a pre-clinical optogenetics-based AAV gene therapy program to Novartis’ portfolio, a promising therapeutic approach that could help patients with vision loss.

Gene Therapy

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By Samantha McGrail

- Novartis recently acquired Arctos Medical to add a pre-clinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology to its ophthalmology portfolio.

Optogenetics has significant potential as the basis of successful therapeutics. The acquisition furthers Novartis’ commitment to finding treatments for patients with vision loss.

“Optogenetics is emerging as a promising therapeutic approach that might restore sight to patients who are legally blind,” Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in the announcement. 

“The Arctos technology builds on our conviction that optogenetic gene therapies may meaningfully help patients battling devastating eye diseases,” Bradner continued. 

Arctos developed its technology to treat inherited retinal dystrophies (IRDs) and other diseases that involve photoreceptor loss, including age-related macular degeneration (AMD). 

Arctos’ optogene is delivered to specific retinal cells using gene therapy, turning the targeted cells into replacement photoreceptor-like cells. But the technology has the potential to address various forms of IRDs regardless of the underlying mutation, a Novartis spokesperson explained. 

A therapeutic based on this technology could treat any disease that causes blindness due to photoreceptor death.

“We’ve watched this technology develop and mature into a therapeutic program that complements our existing portfolio and gives us new optogenetics technology to wield in our efforts to bring desperately needed therapeutic options to patients for these blinding diseases,” said Cynthia Grosskreutz, global head of ophthalmology at the Novartis Institutes for BioMedical Research.

IRDs affect over two million people globally and often result in complete blindness. And AMD is the leading cause of visual disability, affecting an estimated 170 million people globally. 

Currently, there are no approved curative therapies available for AMD.

Beovu is a Novartis’ anti-vascular endothelial growth factor (anti-VEGF) drug indicated to treat neovascular (wet) AMD.

In August, Novartis announced that Beovu elicited more significant reductions than aflibercept in central subfield thickness (CSFT) and the number of eyes with intraretinal fluid and/or subretinal fluid (IRF/SRF) in two Phase 2 clinical trials. 

Year two results were consistent with those seen at year one.