Novartis Expands Foothold in Gene Therapy With New Acquisition

By Eduardo Longoria

On October 29th, Swiss firm Novartis announced its acquisition of Vedere Bio with an upfront payment of $150 million and the possibility of another $130 million in milestone payments for a total price tag of $280 million.  By adding Vedere to its portfolio, Novartis now has a new platform for AAV (adeno-associated virus)-based delivery of gene therapies along with two pre-clinical optogenetic gene therapy programs for the treatment of IRDs (Inherited retinal dystrophies) and geographic atrophy (a type of dry age-related macular degeneration). 

Novartis is trying to cultivate itself in the gene therapy space by harnessing AAVs, chimeric antigen receptor T-cells (CAR-Ts), and clustered regularly interspaced short palindromic repeats (CRISPR).  Vedere’s technology that Novartis has acquired is designed to work by delivering light-sensing proteins to specific retinal cells via intravitreal injection of AAV delivery vectors, stimulating the targeted cells to sense and transmit information to the visual processing centers in the brain. This method bypasses photoreceptor cells that may have died in retinal degeneration. Additionally, the novel AAV capsids acquired in the deal allow the optogenetic therapies to be injected intravitreally (into the vitreous humor of the eye). An ophthalmologist can do this treatment with the tools they have in their clinic.

 

Vedere Bio

Vedere’s scientific founders, Drs. Ehud Isacoff and John G. Flannery developed the technology directed at enhanced ocular gene therapy delivery during their time in academia. The company itself was formed in June 2019 and launched with a $21 million Series A from the investor syndicate of Atlas Venture, Mission BioCapital, and Foundation Fighting Blindness (RD Fund). It began operations at LabCentral in Cambridge, Massachusetts.  

Just before Verdere was purchased, some of the company’s earlier-stage restoration and vision preservation assets were made into a new company, Vedere Bio II, Inc., with the full support of the original Vedere Series A investment syndicate. 

“Our sale to Novartis is an important milestone in advancing Vedere Bio’s most advanced programs to patients around the world. At the same time, I look forward to working with our experienced team to advance our highly innovative, earlier stage assets as part of the newly established Vedere Bio II,” said Cyrus Mozayeni, CEO and president of Vedere Bio and Atlas Venture Entrepreneur in Residence

Vedere Bio II will develop a pipeline of novel vision restoration and preservation drugs by focusing on underserved indications and will do so independently of both Verdere and Novartis. 

Together, the ophthalmologic gene therapy and AAV delivery assets can expand the number of patients who could be treated for vision loss due to photoreceptor death, including all inherited retinal dystrophies.  

“The next frontier in ophthalmology involves finding ways to bring potentially transformative gene therapies to a broader patient population,” says Jay Bradner, President of the Novartis Institutes for BioMedical Research. “The acquisition of Vedere Bio reflects our commitment to next-generation gene therapy and brings hope to patients with otherwise untreatable forms of vision loss.”

 

Financial Benefits of Acquiring Vedere

IRDs include a range of genetic disorders marked by progressive vision loss. This group of conditions impacts more than 2 million people worldwide and often results in complete blindness. Along with IRDs, this technology can benefit nearly 5 million sufferers of geographic atrophy effects. Despite the widespread nature of these disorders, their treatment is lacking. Existing methods target only 1 out of the greater than 250 genes that can cause IRDs and limit the healing potential of these treatments. 

Novartis’ spending of $280 million on acquiring this company is part of a multiyear pattern of getting into gene therapy and appears to be a good way to get over their investment in Alcon. While Novartis decided to hold onto Alcon’s eye drugs, the rest of the company was spun off in April of 2019. The pieces Novartis acquired between 2007 and 2011 totaled to a cost of $51.6 billion, unfortunately, meaning that Novartis lost nearly 50% of its investment in Alcon.

Novartis’ current eye products include Xiidra, for dry eye disease, Beovu, for the wet form of AMD, and Lucentis, which is approved for several conditions, including wet AMD. For the third quarter, the pharma giant recorded $665 million in sales for these three medications. With the acquisition of the Vedere and its research into treatments for geographic atrophy, Novartis will tap into the dry AMD market in North America.

Related Article: Novartis Inks Deal with Molecular Partners for a Novel Way to Take Down COVID-19

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

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