Novartis International AG (SIX: NOVN; NYSE: NVS) has announced the acquisition of Vedere Bio, a venture based optogenetics company in Cambridge, Massachusetts. Vedere had developed a platform for AAV-based delivery of gene therapies optogenetic technologies applying for the treatment and prevention of ophthalmic disorders. According to BioCentury, Novartis has paid $150M upfront for Vedere, with shareholders eligible to receive further milestone payments of $130M.
Novartis Institutes for BioMedical Research (NIBR) identified light-sensing proteins that can be delivered to cells in the retina using adeno-associated virus (AAV) delivery vectors via intravitreal injection. Commenting on the acquisition, Jay Bradner, President of NIBR stated that, “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.”
According to Novartis, the acquired optogenetics technology was 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. The optogenetics approach acquired from Vedere Bio acts directly on surviving cells in the retina, altering their behaviour and potentially benefit a further treatment for a range of retinal disorders including a “dry form” of age-related macular degeneration (AMD).
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. At the announcement of the acquisition of Vedere, Cynthia Grosskreutz, Global Head of Ophthalmology at the Novartis Institutes for BioMedical Research commented that, “we believe that gene therapy technologies have transformative potential for treating blinding diseases. 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.”