"Despite the projected growth in market applications and abundant investment capital, there is a danger that legal and ethical concerns related to genetic research could put the brakes on gene editing technologies and product programs emanating therefrom."
As society adjusts to a new world of social distance and remote everything, rapid advancements in the digital, physical, and biological spheres are accelerating fundamental changes to the way we live, work, and relate to one another. WhatWhat is Genome Editing?
There are thousands of diseases occurring in humans, animals, and plants caused by aberrant DNA sequences. Traditional small molecule and biologic therapies have only had minimal success in treating many of these diseases because they mitigate symptoms while failing to address the underlying genetic causes. While human understanding of genetic diseases has increased tremendously since the mapping of the human genome in the late 1990s, our ability to treat them effectively has been limited by our historical inability to alter genetic sequences.
The science of gene editing was born in the 1990s, as scientists developed tools such as zinc-finger nucleases (ZFNs) and TALE nucleases (TALENs) to study the genome and attempt to alter sequences that caused disease. While these systems were an essential first step to demonstrate the potential of gene editing, their development was challenging in practice due to the complexity of engineering protein-DNA interactions.
Then, in 2011, Dr.
CRISPR/Cas9 for gene editing came about from a naturally occurring viral defense mechanism in bacteria. The system is cheaper and easier to use than previous technologies. It delivers the Cas9 nuclease complexed with a synthetic guide RNA (gRNA) into a cell, cutting the 'cell's genome at the desired location, allowing existing genes to be removed and new ones added to a living organism's genome. The technique is essential in biotechnology and medicine as it provides for the genomes to be edited in vivo with extremely high precision, efficiently, and with comparative ease. It can create new drugs, agricultural products, and genetically modified organisms or control pathogens and pests. More possibilities include the treatment of inherited genetic diseases and diseases arising from somatic mutations such as cancer. However, its use in human germline genetic modification is highly controversial.
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Market Applications
In the 1990s, nanotechnology and gene editing were necessary plot points for science fiction films. In 2020, developments like nano-sensors and CRISPR gene editing technology have moved these technologies directly into the mainstream, opening a new frontier of novel market applications. According to
Gene editing's primary market applications are for the treatment of genetically-defined diseases. CRISPR/Cas9 gene editing promises to enable the engineering of genomes of cell-based therapies and make them safer and available to a broader group of patients. Cell therapies have already begun to make a meaningful impact on specific diseases, and gene editing helps to accelerate that progress across diverse disease areas, including oncology and diabetes.
In the area of human therapy, millions of people worldwide suffer from genetic conditions. Gene-editing technologies like CRISPR-Cas9 have introduced a way to address the cause of debilitating illnesses like cystic fibrosis and create better interventions and therapies. They also have promising market applications for agriculture, food safety, supply, and distribution. For example, grocery retailers are even looking at how gene editing could impact the products they sell. Scientists have created gene-edited crops like non-browning mushrooms and mildew-resistant grapes - experiments that are part of an effort to prevent spoilage, which could ultimately change the way food is sold.
Despite the inability to travel and conduct face-to-face meetings, attend industry conferences or conduct business other than remotely or with social distance, the investment markets for venture, growth, and private equity capital, as well as corporate R&D budgets, have remained buoyant through 2020 to date. Indeed, the third quarter of 2020 was the second strongest quarter ever for VC-backed companies, with 88 companies raising rounds worth
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Legal and Ethical Considerations
Despite the projected growth in market applications and abundant investment capital, there is a danger that legal and ethical concerns related to genetic research could put the brakes on gene-editing technologies and product programs emanating therefrom. The possibility of off-target effects, lack of informed consent for germline therapy, and other ethical concerns could cause government regulators to put a stop on important research and development required to cure disease and regenerate human health.
Gene-editing companies can only make money by developing products that involve editing the human genome. The clinical and commercial success of these product candidates depends on public acceptance of gene-editing therapies for the treatment of human diseases. Public attitudes could be influenced by claims that gene editing is unsafe, unethical, or immoral. Consequently, products created through gene editing may not gain the acceptance of the government, the public, or the medical community. Adverse public reaction to gene therapy, in general, could result in greater government regulation and stricter labeling requirements of gene-editing products. Stakeholders in government, third-party payors, the medical community, and private industry must work to create standards that are both safe and comply with prevailing ethical norms.
The most significant danger to growth in gene-editing technologies lies in ethical concerns about their application to human embryos or the human germline. In 2016, a group of scientists edited the genome of human embryos to modify the gene for hemoglobin beta, the gene in which a mutation occurs in patients with the inherited blood disorder beta thalassemia. Although conducted in non-viable embryos, it shocked the public that scientists could be experimenting with human eggs, sperm, and embryos to alter human life at creation. Then, in 2018, a biophysics researcher in
Some influential non-governmental agencies have called for a moratorium on gene editing, particularly as applied to altering the creation or editing of human life. Other have set forth guidelines on how to use gene-editing technologies in therapeutic applications. In
Regulations must keep pace with the change that CRISPR-Cas9 has brought to research labs worldwide. Developing international guidelines could be a step towards establishing cohesive national frameworks.
Where Do We Go from Here?
The COVID-19 pandemic has forced us to rely more on new technologies to keep us healthy, adapt to working from home, and more. The pandemic makes us more reliant on innovative digital, biological, and physical solutions. It has created a united sense of urgency among the public and private industry (together with government and academia) to be more creative about using technology to regenerate health. With continued advances in computing power, network architecture, communications bandwidths, artificial intelligence, machine learning, and gene editing, society will undoubtedly find more cures for debilitating disease and succeed in regenerating human health. As science advances, it inevitably intersects with legal and ethical norms, both for individuals and civil society, and there are new externalities to consider. Legal and ethical norms will adapt, rebalancing the interests of each. The fourth industrial revolution is accelerating, and hopefully towards curing disease.
Originally published by IPWatchdog.com,
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