Novartis acquires Vedere Bio, adding novel optogenetic gene therapy technology for treating blindness

• Acquisition expands the Novartis footprint in ophthalmology and enhances the company’s position as an AAV-based gene therapy powerhouse.
  
  
• Novartis gains two pre-clinical optogenetic AAV gene therapy programs and novel delivery technology for treating inherited retinal dystrophies and geographic atrophy.
  
  
• Inherited retinal dystrophies, including advanced retinitis pigmentosa, affect greater than 2 million patients worldwide, and geographic atrophy affects approximately 5 million patients worldwide.

Basel, October 29, 2020 — Novartis announced today that it has acquired Vedere Bio, adding a powerful new platform for AAV-based delivery of gene therapies and a best-in-class optogenetics program to help reimagine the treatment and prevention of vision loss and blindness. The acquisition builds on the company’s commitment in cell and gene therapy, and will enable Novartis to further advance its efforts to bring transformative therapies to a wide range of patients with blinding diseases.

The technologies acquired include light-sensing proteins that can be delivered to cells in the retina and unique, adeno-associated virus (AAV) delivery vectors that enable treatment via intravitreal injection. Together, these assets have the potential to vastly 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.”

Novartis is broadening its footprint in the gene therapy space, with a focus on three distinct platforms—AAVs, chimeric antigen receptor T-cells (CAR-Ts) and clustered regularly interspaced short palindromic repeats (CRISPR). The acquisition of Vedere Bio’s unique technology is the latest addition to the company’s expanding therapeutic arsenal.

Inherited retinal dystrophies (IRDs) include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss. The conditions, which impact more than 2 million people globally, often result in complete blindness. Yet existing treatments target only one of the more than 250 genes that can cause IRDs, limiting the population of patients who can benefit.

The optogenetics approach acquired from Vedere Bio acts directly on surviving cells in the retina, altering their behavior and making it possible for anyone suffering from vision loss due to photoreceptor cell death to potentially benefit. This approach also holds promise for treating other conditions that involve photoreceptor loss, including a “dry form” of age-related macular degeneration (AMD) called geographic atrophy, which affects more than 5 million people globally.

The acquired optogenetics technology is based on technology from the labs of Vedere’s scientific founders, Drs. Ehud Isacoff and John G. Flannery of UC Berkeley, as well as technology directed at enhanced ocular gene therapy delivery arising jointly from UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania. It is designed to work by delivering naturally occurring, light-sensing proteins to specific retinal cells, 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 eye. This could potentially be administered by an ophthalmologist in the clinic.

“We believe that gene therapy technologies have transformative potential for treating blinding diseases,” said Cynthia Grosskreutz, Global Head of Ophthalmology at the Novartis Institutes for BioMedical Research. “With the new tools that this acquisition brings to the table, we will no longer be limited to replacing single genetic mutations that are causing eye diseases. This introduces the potential to treat any patient with retinal degeneration leading to photoreceptor death.”

Vedere developed the assets with the support of the investor syndicate of Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund).

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