Initially developed for the treatment of inherited genetic diseases such as muscular dystrophy, AAV vectors have more recently taken a leading role on the clinical scene in neurological disorders and other muscle-related diseases. The field is deep, despite inherent payload and immunogenicity limitations that have given rise to a cottage industry of non-viral vector research.
Here’s a pretty cool, interactive graphic produced by DeciBio, illustrating direct, peripheral, and extended partnerships in the AAV therapy partner ecosystem. I’m not claiming it to be comprehensive, and neither is its creator, DeciBio Principal Carl Schoellhammer, Ph.D. But, it’s a pretty good in-the-moment snapshot, providing a representative glance at priorities in the space.
Cassidy Humphreys and Phillip Leclair, two analysts in DeciBio’s therapeutic practice, along with Schoellhammer, created the graphic from DeciBio’s AAV TheraTrack, which is an even more comprehensive database of the AAV landscape, its advances, and its in-process therapeutic applications. Ask him a question about who’s developing vectors for what indications and you’d do well to pull up a chair and get comfortable. Schoellhammer is to dissecting the cell and gene therapy ecosystems what Adam Schefter is to breaking down off-season moves in the NFL. That is to say, he has pretty impeccable vision from his perch.
Having said that, before you begin to manipulate this graphic and then quickly turn to the comments section to decry its completeness or accuracy, know this. Analysts are bold, brave, and thick-skinned. They have to be. They know that everything they create is at least slightly outdated by the time it’s published, and they know that nothing they create will ever capture 100 percent of the space they’re analyzing. So, please go ahead with those comments and criticisms—Carl invites and appreciates the dialog—but let’s keep it constructive.
This graphic from DeciBio’s TheraTrack defaults to the AAV ecosystem across multiple therapeutic areas, but allows you to drill down into specific therapeutic areas – or combinations of those areas – by selecting or deselecting the primary-colored therapeutic area menu at the top of the graph. What struck me in doing so was the depth of activity that remains in CNS disorders such as Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis (ALS), and Frontotemporal degeneration (FTD). This was an early target-rich environment for AAV-based gene therapy researchers a decade ago, and clearly that legacy continues to breed, despite the challenges associated with gene delivery to the brain (and in the shadow of the success of AAV approaches in hemophilia, muscular atrophy, ocular conditions, and other indications that are a long way from the wheelhouse of the central nervous system). Delivery challenges associated with targeting specific tissues or cells without causing toxicity or unwanted immune responses is challenging in its own right. When said cells and tissues are in and around the brain, the challenge reaches next-level status.
CNS, likely by virtue of its history, is also the most “networked” therapeutic area on the AAV chart, as illustrated by the connections drawn between and among players in the space. Use your mouse to grab those circles representing the leading players in the space and drag them for expanded views, and you’ll see Roche, Lilly, Biogen, Voyager, and Capsida anchoring multi-party partnerships in the space. Now layer in the other therapeutic areas in the graphic, and watch Roche’s influence expand like a spiderweb.
Like I said, the graphic doesn’t promise the totality of the AAV manufacturing space, but it offers some flavor, as well as a snapshot of DeciBio’s TheraTrack capability, which can present up-to-date asset development data and progress from the bulk of cell, viral gene, nucleic acid based, and antibody-drug conjugate therapies pretty much any way you want the data sliced. Learn more at decibio.com.