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Mark Curtis & Richard Philipson
Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.
Organova has been busy for some time now developing 3D bio-printed tissues for use in screening drug candidates. The thesis being that Pharma can save dollars by adopting screens that provide greater ‘predictiveness’. It’s a tough business model. Cellular Dynamics International, now owned by FujiFilm, burnt through a great deal of money in its day trying to persuade Pharma to screen compounds with its cell lines. Organovo upped the ante, though, with its printing platform, creating 3D, multi-cellular aggregates, or ‘organoids’, which more closely resemble real tissue. The company announced a collaboration with Merck in 2015, giving Merck access to its human liver tissue platform for drug development. Now, Organovo has plans to transcend tools into the realm of therapeutics, and will bring its bio-printed tissue into the clinic, initially for liver disease. Ambitious? Absolutely, but it’s a small step towards realizing the big vision of artificial organ replacement.
This month illustrates the mixed fortunes of companies working in cell and gene therapy. Whilst Spark Therapeutics continues to roll out positive clinical data in the rare eye disease Leber’s congenital amaurosis-2, and Agilis Biotherapeutics announced authorization of a Phase IIb trial in the rare CNS disease aromatic-l-amino acid decarbolylase deficiency, there is less good news from Adverum, which announced a 1-year delay to its alpha-1-antitrypsin deficiency program due to manufacturing issues. Adverum uses a baculovirus-based AAV production system; whilst these systems are scalable, they can present problems with low yields and baculovirus stability. R&D in gene therapy is as healthy as ever, but manufacturing can often prove to be the Achilles heel for the field.
Citation: Cell Gene Therapy Insights 2016; 2(4), 407-422