Novartis acquires Vedere Bio: ‘This enhances our position as an AAV-based gene therapy powerhouse'

By Rachel Arthur

- Last updated on GMT


Related tags Novartis Gene therapy AAV platform adeno-associated virus Eye Ophthalmology

Novartis has acquired Vedere Bio, giving the company a new platform for AAV-based delivery of gene therapies and an optogenetics program for the treatment and prevention of vision loss and blindness.

The Swiss healthcare giant says the acquisition builds on its commitments in cell and gene therapy.

The technologies acquired from Vedere Bio include light-sensing proteins that can be delivered to cells in the retina; and adeno-associated virus (AAV) delivery vectors that enable treatment via intravitreal injection.

These will be able to help patients with photoreceptor death, including inherited retinal dystrophies.

Novartis is widening its footprint in the gene therapy space across three areas: AAVs, chimeric antigen receptor T-cells (CAR-Ts) and clustered regularly interspaced short palindromic repeats (CRISPR).

Jay Bradner, President of the Novartis Institutes for BioMedical Research, said: “The next frontier in ophthalmology involves finding ways to bring potentially transformative gene therapies to a broader patient population.

“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.”

The technology

To date, gene therapy has been concentrated mainly in diseases that leave photoreceptors intact. However, inherited retinal dystrophies (IRDs) include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

Such conditions, which impact more than 2 million people globally, often result in complete blindness.

Founded just last year in Cambridge, Massachusetts, Vedere Bio’s optogenetics approach instead acts on surviving cells in the retina. The technology was developed in the labs of its scientific founders - Drs. Ehud Isacoff and John G. Flannery of UC Berkeley - as well as technology directed at enhanced ocular gene therapy delivery from UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania. Its development to date has been supported by the investor syndicate of Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund).

The technology delivers 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. In this way, signals can bypass photoreceptor cells that would usually have been used, but have died in retinal degeneration.

Its technology could also prove to have uses for treating other conditions that involve photoreceptor loss, including a “dry form” of age-related macular degeneration (AMD), geographic atrophy, which affects more than 5 million people globally.

In addition , the novel AAV capsids could allow optogenetic therapies – injected intravitreally into the eye – to be administered by an ophthalmologist in a clinic.

Existing treatments target only one of the more than 250 genes that can cause IRDs, limiting the population of patients who can benefit.

“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.”

In 2018 Novartis acquired AveXis Inc for $8.7bn, which has since become Novartis Gene Therapies and has six projects in its pipeline. AVXS-101 (onasemnogene abeparvovec) – a survival motor neuron gene replacement therapy for spinal muscular atrophy (IV formulation) was approved in the US and Europe earlier this year.

In August, Novartis launched a gene therapy partnership with Sangamo:​ with this technology also using AAV delivery. 

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