Gene therapy specialists Voyager Therapeutics, with a particular focus on central nervous system application, were in the news again last week. The company entered into a strategic collaboration with global pharma giants AbbVie to develop gene therapy mediated treatments for Alzheimer’s disease.
NASDAQ-listed Voyager has more than doubled its market value in the last 12-month period. The deal with AbbVie certainly contributed as Voyager received USD 69 million in cash as the deal was penned down and it also sees the Cambridge, Massachusetts-based biotech earn a potential USD 1 billion in stepwise milestone payments should the program be successfully developed. However, Voyager earned its fame last year as the company revealed initial data from their clinical trial in patients with Parkinson’s disease.
Although that data comprised of only a small number of patients with varying doses it was unique in many ways. First of course, the results were positive; neuronal cells in the patients started producing increasing levels of dopamine (the missing protein contributing the Parkinson’s symptoms), the patients experienced less troublesome involuntary movements (a hallmark for this disease), and they needed less dopamine replacement medicines. Secondly, the treatment is for a non-hereditary neurodegenerative disease which is a first. Other gene therapy approaches so far have focused on genetically caused disorders that patients are born with and often tend to be more acute, even at low ages. This therefore brings some hope to the development of an Alzheimer’s gene therapy, as all attempts so far to bring new drugs to patients with this disease have failed.
Besides Voyager, there was another high-profile gene technology deal that was announced last week. The Kite Pharma division of Gilead Sciences signed a collaborative agreement with Sangamo Therapeutics in order to further boost Kite’s T-cell capabilities to treat cancer. As a reminder, Kite was acquired last by Gilead year in a USD 12 billion deal.
Sangamo are gene editing specialists. Gene editing hold the promise of conducting more precise changes to the genome with the potential to treat a wider array of diseases than the gene therapies employed thus far. A group of companies using the much renowned CRISPR technology for gene editing has gotten most of the attention lately, but Sangamo are using an alternative proprietary approach called Zinc-finger, which led the company to be the first in gene editing to study the approach in humans with clinical trials ongoing in two hereditary metabolic disorders. Now Sangamo, will receive USD 150 million upfront potentially up to USD 3 billion in further milestone payments as Kite/Gilead will use Sangamo’s technology to improve their T-cell therapy. In other words, there will be ample opportunities to revisit the gene technology space in 2018 and beyond.
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