Lipid nanoparticles have been around for decades, but they took a spotlight in the delivery of the COVID19 mRNA-based vaccines. This chemical cage can fuse with cell membranes and deliver a specific payload to a cell. While the delivery to specific cells is routine, are there ways to improve the system to have more specific therapeutic benefit. Dr. John Lewis of Entos discusses lipid nanoparticle chemistry and new formulations that exhibit enhanced capacity to target specific locations and deliver payloads more effectively. We also discuss the current therapies that may benefit from the new technologies. 

There are many rare diseases that originate with a single letter change in DNA, and they can cause tremendous physical and financial hardship for families affected. Viral-based gene therapies have been a promise for decades, and modern technology is now bringing these concepts to fruition. Dr. Gaurav Shah, CEO of Rocket Pharma, describes the power of the current gene therapy approaches, along with the pipeline of potential therapeutics that could represent cures for rare disease. 

This week's podcast has two parts.  Dr. Leen Kawas , Managing General Partner at Propel Biopartners, speaks about how biotech startups can benefit from skilled oversight and capital provided from experts that understand the business/science ecosystem. In the second part we discuss the latest therapies for Alzheimer's Disease, and how trials and regulation can't follow traditional trajectories. Alzheimer's has variation in etiology, patients start treatments after a diagnosis which may be years after the disease is well established, and progression is dependent on many genetic factors. All of these variables make any assessments problematic. At the same time several new antibody-based therapies show promise. 

As the amount of plant genomic DNA sequence increases, scientists have detected DNA sequence information that simply does not conveniently fit into genome assemblies, and clearly appears to be from somewhere else. Dr. Lara Pereira from the University of Sheffield explains the surprising frequency of lateral gene transfer between grass species. The mechanism of crossing the species barrier is discussed, along with the frequency of horizontal transfer. We discuss the ramifications of unregulated integration of DNA into plant genomes and the perception of transgenic plants in the EU and other locations where crop genetic engineering is not viewed favorably. The results also suggest additional care when monitoring gene escape in transgenic grasses like maize.  Follow Dr. Pereira on Twitter at @LarrLarrr

Salmon are outstanding table fare, featuring flavor and nutrition. The AquAdvantage salmon is a genetically engineered salmon that is resistant to changes in growth due to seasonal fluctuation or the stresses encountered in traditional ocean-net salmon farms. Despite the advantages, the salmon has been caught in deregulation for many years, limiting consumer access. Silvia Wulf of AquAdvantage talks about the current status of availability, the technology, the criticisms and the future of the AquAdvantage salmon. 

The mythical Fountain of Youth may be an actual component of our physiology. Aging is not simply the body breaking down against the calendar, it is a coordinated series of molecular/biochemical changes that occur with time. There is an active hypothesis that later-life medical issues are not just happening as we age, but are actually caused by aging, meaning there are potential drug targets that may slow the process. BioAge performed -omics level analysis on populations of aging individuals, revealing patterns of proteins and metabolites that may be causal components of the aging process. New therapeutics seek to target aspects of human senescence, potentially delaying the onset of late-life disease. Dr. Eric Morgen of BioAge describes how his company has used a novel approach to identify new compounds that may play eventual roles in slowing the aging process. 

What are the risks associated with being able to create custom DNA?  Today's podcast discusses the potential weaponization of synthesized nucleic acid sequences. Dr. Kevin Esvelt discusses synthetic DNA and synthetic viruses, along with the possibility that they may drive the next pandemics or become the basis for future biological weapons. The dark realities are discussed, along with surveillance and mitigation strategies, as well as the broken social trust that could fuel a human-made pandemic. 

If a genetic disorder can be detected in the developing fetus, it provides families and physicians vital information that can guide important decisions. From family planning to medical intervention, new molecular techniques are being employed to detect potential anomalies as early as 10 weeks after conception. The modern genetic counselor is an expert at communicating the results of these tests, building trust to advise decisions that benefit the parents and the child.  

While there are many views on Brexit, one certain benefit is the UK's self-governing role in scientific regulatory affairs. The EU's position on genetic engineering has perplexed its scientists, as they create solutions for EU farmers that may never be implemented. Now out from the EU's onerous non-scientific regulatory oversight, the UK may choose to deregulate gene editing. Cameron English talks about the proposed regulatory changes and how they may benefit the UK's farmers and scientists.  You can read his report here:  https://iea.org.uk/publications/harvest-time-why-the-uk-should-unleash-the-power-of-gene-editing/

The American chestnut was a dominant tree in many forests in the eastern United States. In the late 1800's an imported fungus spread rapidly throughout the range, destroying most of the trees in a matter of decades. Scientists at the State University of New York have engineered trees to host a gene from wheat that disarms the fungal invader, breaking the production of an acid required for infection. This strategy allows the tree and the fungus to live together without infection. Erik Carlson discusses the problem, the project, the push back and the current deregulation efforts to release the tree in the interest of repatriating its native range.  Read the release docket and provide your comments here -- https://www.regulations.gov/document/APHIS-2020-0030-8291/comment