Translating Nobel Science Into Novel Drugs

10/12/12

This week, the Nobel Prize in Physiology or Medicine was awarded to Drs. Shinya Yamanaka and Dr. John Gurdon for their work in the stem cell field, including the discovery of induced pluripotent stem cells (iPSCs). iPSCs now rank among other fields that represent this pinnacle of profound discoveries that disrupt the fields of biology and medicine including: epigenetics, gene targeting using embryonic stem cells, oncogenes, and RNA interference. All of these discoveries offer us the promise of new ways to treat diseases and have spawned unique biotechnology companies. But, like any fundamental scientific observation, the translational path from novel biology to innovative therapeutics is not for the faint of heart.

The good news is that Nobel Prize-winning science gives rise to powerful drug discovery platforms. Nobel science equates to a profound breakthrough in biology that is transformative, foundational and platform-driven, allowing for broad applicability rather than the identification of a singular product. These are powerful domains, the ideal ‘hot beds’ for drug discovery scientists.

With this massive opportunity comes significant challenge. Biotech companies are generally founded on the premise of breakthrough science, nimbly applying new technologies to better understand mechanisms of disease to lead to first-in-class drug products. Marrying the application of Nobel-worthy science, with the requirement for a focused strategy with a high probability of success, is a tall order.

iPierian is now among a shortlist of biotech companies with the privilege of rising to the challenge of translating Nobel science into novel drugs. The sheer wealth of opportunity afforded by a Nobel Prize winning technology, such as iPSCs, is evidenced by the range of research being pursued across the continuum of academic, government and industry laboratories. Since iPSCs were first discovered in 2006, over 4,000 papers have cited the finding.

iPSCs were first discovered by Dr. Shinya Yamanaka. In his groundbreaking Cell paper in 2006, he developed the method for inducing skin cells from mice into becoming pluripotent stem cells. He repeated this same feat in 2007 with adult human skin cells. Although the Nobel Prize was just awarded this week for iPSCs, there is already extensive work to realize the seemingly limitless potential of human iPSCs – with real-world applications to disease modeling, drug discovery, tissue engineering and regenerative medicine.

iPierian was founded in 2008 based on the promise of Dr. Yamanaka’s iPSC technology to enable more enlightened drug discovery. The company maintains a strong link to the Nobel Prize winning science that launched the iPSC field, with Dr. Yamanaka as a member of iPierian’s Scientific Advisory Board. The Yamanaka technology offers an extraordinary opportunity to study human disease in a way never before possible, i.e. ‘disease-in-a-dish’. In the biotech world in general, and specifically in the neurodegenerative disease field, this provides a way to access human disease biology which was previously limited to engineered cell lines and mouse models.

Today, iPierian has made great strides by focusing the iPSC technology toward the development of disease-modifying antibody drugs for neurodegenerative diseases and inflammation. By taking skin cells from patients with diseases such as Alzheimer’s disease (AD), one can create patient-derived iPSCs, which are then differentiated into neurons or glial cells. Antibody programs against Tau and Complement Pathway are being evaluated now across numerous diseases, such as AD and other Tauopathies, Parkinson’s disease, and inflammation.

iPierian is one example of how Nobel Prize winning biology is being translated into biotech success, quite rapidly after the breakthrough discovery. While the broad potential of iPSC technology is accompanied by challenges in how to build an organization with a focused strategy, iPierian aims to move to and through the clinic in order to realize the ultimate goal – bringing disease-modifying therapies to patients.

Nancy Stagliano, Ph.D. is the CEO of South San Francisco-based iPierian. Follow @

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