Studies show self-assembling scaffold promotes cartilage growth without growth factors.
Regenerative medicines firm Nanotope could receive milestone payments of up to $26.5 million from Smith & Nephew (S&N) as part of a deal focused on the development of a nanofiber gel-based cartilage regeneration product. Nanotope says the deal represents its first commercial transaction. Under terms of the partnership S&N will work with Nanotope to develop and optimize a product based on the latter’s technologies. S&N will also carry out all related preclinical development, and shoulder all costs relating to preclinical and clinical development.
“S&N is ideal for the commercialization of Nanotope’s cartilage repair technology given its global reach in orthopaedics and potential synergies of a new cartilage regeneration capability with its suite of existing orthopaedic products,” remarks Christopher Anzalone, Ph.D., president and CEO at nanotech firm Arrowhead Research, which holds a minority equity stake in Nanotope. “We view this agreement as a validation of Nanotope’s technology and business strategy. This deal also represents the achievement of a key milestone Arrowhead has articulated for the last quarter of calendar 2010.”
Nanotope is developing a suite of topical or injectable products for tissue repair, based on a customizable nanofiber matrix that assembles into a three-dimensional bioactive scaffolding in which cells and tissues are directed to grow and differentiate. The underlying technology was developed by Nanotope co-founder, Samuel Stupp, Ph.D., at Northwestern University.
Nanotope says key features of the technology are the customizable bioactivity and controllability of matrix gel formation. These features hinge on the properties of the individual engineered small molecules that self-assemble into nanofibers under physiological conditions, the firm explains. The molecules are entirely customizable so that every factor, from the rate of self-assembly to the type of bioactivity conferred by the fully formed nanofiber gel, can be controlled.
The gel molecules essentially consist of a hydrophobic tail and a hydrophilic head region that confers bioactivity to the gel, Nanotope continues. This head region is a short peptide sequence derived from a protein or peptide that exerts an influence of interest on target tissues. Nanotope claims the number of different bioactive regions that can be engineered to elicit specific cell responses is essentially limitless. This means the same core technology can be used to elicit the regeneration of different types of tissue.
A study published in February in the Proceedings of the National Academy of Sciences demonstrated the ability of Nanotope’s lead cartilage regeneration product to promote the growth of new cartilage in a rabbit model of cartilage injury. The nanofiber product was injected as a liquid into microfracture holes generated in the bone beneath the damaged cartilage. After injection, the product self-assembled into a bioactive scaffold that triggered new cartilage growth, even in the absence of growth factors.
Nanotope is separately developing a nanofiber gel-based product for spinal cord repair, which is designed to redirect the differentiation of immature neuroprogenitor cells into neurons. In vivo rodent studies are in progress, and have demonstrated what the firm claims is significant reversal of paralysis in a clinically relevant injury model. Longer term animal studies for spinal cord regeneration are under way, alongside toxicology studies in anticipation of future filing for approval to start clinical development in the U.S.
Nanotope’s third project, based on a similar nanofiber platform, is designed to promote the growth of vascular endothelium and angiogenesis. In vivo studies in small animals have already demonstrated that topical application of the product to a fresh wound accelerates healing rates. The firm suggests that the ability of this product candidate to promote vascular regeneration may lead to additional applications in the treatment of myocardial infarction and peripheral vascular disease.