Novartis, a Swiss pharmaceutical company, has reportedly acquired Vedere Bio, a gene therapy product developer. The deal has led to the addition of a new platform for adeno-associated virus (AAV)-based gene therapy delivery as well as a best-in-class optogenetics program to sharpen its focus on vision-restoring therapy.

The recent takeover deal is in line with Novartis’ strong commitment to enhance gene and cell therapy. It will enable the company to further drive efforts for the efficient development of transformative therapies and treat a range of patients suffering from blinding diseases.

Through the agreement, Novartis has acquired technologies such as light-sensing proteins, which can be delivered to the retina cells. Moreover, the company will gain access to the AAV delivery vector that will enable patients’ treatment through intravitreal injection. This significant addition can potentially expand the treatment of patients with vision loss due to photoreceptor death.

The company has been broadening its stance in the gene therapy industry and has 3 distinct platforms, namely AAVs, CAR-Ts (chimeric antigen receptor T-cells), and CRISPR (clustered regularly interspaced short palindromic repeats). The latest acquisition of Vedere’s unique technology will increase its therapeutic arsenal.

IRDs (inherited retinal dystrophies) include a range of genetic retinal disorders, leading to photoreceptor cell loss and progressive vision loss. This eye disorder has impacted above 2 million people across the globe, often resulting in complete blindness. However, existing treatments could target only one of more than 250 genes that cause IRDs.

The recently acquired optogenetics approach will act directly on the surviving retina cells. This approach can also be used to treat other conditions involving photoreceptor loss, such as the dry form of AMD (age-related macular degeneration) called geographic atrophy, affecting above 5 million people globally.

The optogenetics technology is directed towards an enhanced ocular gene therapy delivery. It was designed to deliver naturally occurring, light-sensing proteins to the retinal cells. This approach stimulates the sensing and information transmission capabilities of the targeted cells to the brain’s visual processing centers.

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https://www.novartis.com/news/media-releases/novartis-acquires-vedere-bio-adding-novel-optogenetic-gene-therapy-technology-treating-blindness