Blogs

The development of risk-proportionate responses to emergent threats

In my view, the first step in developing a risk-proportionate response to an emergent threat is to assess the scale of risk and put the emergent risk in the context of other well understood and accepted risks. Clearly, humans react to emergent novel risks very differently from risks with which they are familiar. Consider our response to automobile accidents or the annual cold and flu … Continue reading The development of risk-proportionate responses to emergent threats »

Blogs

An alternative risk-proportionate, sustainable response to the next infectious threat

In a previous post, I asked several questions about the response to COVID-19. My purpose was to encourage a thoughtful discourse about how to deal with the recurrent infectious disease threats to which we are exposed. To hone the focus on creating a blueprint for a more effective response to future infectious threats – let’s call them New Risk Agent, in this post I propose … Continue reading An alternative risk-proportionate, sustainable response to the next infectious threat »

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What next? Questions to consider in assessing the response to COVID-19 and the risks of future pandemics

The study of risk, the perception of risk and the management of risk is an important area of scholarship that I believe all leaders in positions of authority should study. I am certainly not an expert, but over the years I have invested in some scholarship in this area. There are, of course, numerous ways to categorize risks. One simple exercise is to think about … Continue reading What next? Questions to consider in assessing the response to COVID-19 and the risks of future pandemics »

Blogs

Speaking of Books – March 2020

A few recent reads that I recommend: The Looming Tower: Al-Qaeda and the Road to 9/11, by Lawrence Wright. Both book and mini-series offer a well-documented retelling of the events leading to 9/11. Life on The Edge: The Coming Age of Quantum Biology, by Johnjoe McFadden and Jim Al-Khalili. If you are interested in the quantum world, this is a well-written book that makes the case … Continue reading Speaking of Books – March 2020 »

Blogs

Update on Core Antisense Research

In this paper, we continue our exploration of the molecular mechanisms by which PS ASOs enter and distribute within cells such that they can be pharmacologically active.  We show that M6PR plays a critical role in releasing PS ASOs from late endosomes. We now believe that we have identified most of the endosomal means of productive uptake and intracellular distribution of PS ASOs. We are assessing the … Continue reading Update on Core Antisense Research »

Blogs

The n-Lorem Foundation: up and running and making a difference in the lives of rare disease patients

Patients and families with genetic diseases that afflict one or very few patients in the world pose unique challenges to the healthcare industry. It is gratifying that the National Institutes of Health (NIH) and other institutions in the U.S. have begun to address identifying such patients and harnessing the power of modern genomics to diagnose and identify the molecular causes of these diseases. In fact, … Continue reading The n-Lorem Foundation: up and running and making a difference in the lives of rare disease patients »

Blogs

Mechanisms that improve therapeutic index

Mechanisms of ASO toxicities and the structure activity relationships that improve therapeutic index Last year, we published a landmark paper that described a general mechanism that explains how toxic ASOs produce toxicities and provided a straightforward medicinal chemistry solution to enhance safety with little effect on potency (Shen et.al., 2019). We have now published several papers that extend our observations and broaden the understanding of … Continue reading Mechanisms that improve therapeutic index »

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Ionis papers provide insight into the characteristics of antisense medicines

Not long ago, I wrote about an important paper that we published (Shen et. al, NBT, 2019) which, for the first time, provides a step-by-step molecular mechanism explaining most of the toxicities of ASOs and a straightforward medicinal chemical solution that dramatically enhances the therapeutic index of toxic ASOs. As you might imagine, we will be publishing a number of papers that provide more detail … Continue reading Ionis papers provide insight into the characteristics of antisense medicines »

Blogs

NEJM highlights Waylivra™ (volanesorsen)

The results of the Phase three study of WAYLIVRA (volanesorsen) in patients with familial chylomicronemia syndrome (FCS) were recently published in the New England Journal of Medicine (NEJM) (Witzum, et al., 2019). FCS patients have severely elevated triglycerides (often >2000 mg/dl). This drug is now approved in the EU and being considered for approval by the U.S. Food and Drug Administration (FDA). To date, it … Continue reading NEJM highlights Waylivra™ (volanesorsen) »