Category Archives: Human Genetic Engineering

However, in both medicine and dentistry, there is an important role as well for ingenious, low-tech, less expensive approaches to improved health and increased longevity.

The FDA last year approved a high-tech gene therapy drug, Zolgensma, for a rare childhood genetic disease, spinal muscular atrophy, that costs justover $2 millionfor the single dose of the treatment.The illness, which is caused by a defect in a gene calledSMN1, affects about 400 babies in the United States annually and kills those with the most common form of the disease in the first few years of life.The new treatment uses non-pathogenic, genetically engineered viruses to deliver healthy copies of theSMN1gene to patients cells so they can synthesize a protein needed to develop normal muscle neurons.

Another remarkable genetic engineering feat wasreportedin the journalNaturein 2017.An experimental gene therapy procedureused to transform and grow sheets of healthy skin saved the life of a 7-year-old boywho suffered from a genetic disease,junctional epidermolysis bullosa, that had blistered and destroyed most of his skin.He was on the verge of death, but two years after the treatment with genetically engineered cells produced by a multi-national team, he had healthy skin and was leading a normal life.

Those high-tech interventions are spectacular, but there are many simpler and cheaper yet tremendously important innovations for the diagnosis and prevention of illness.Among the most cost-effective are checklists for personnel in operating rooms and ICUs. According to a Norwegian research group, Safety checklists appear to be effective tools for improving patient safety in various clinical settings by strengthening compliance with guidelines, improving human factors, reducing the incidence of adverse events, and decreasing mortality and morbidity.

Sometimes, a simple tool or device is important to clinical diagnosis. One example is the hand-held direct ophthalmoscope, which allows a medical practitioner to look intotheback oftheeye to ascertain the health oftheretina, optic nerve, vasculature, and vitreous humor (the liquid inside the eyeball). Invented in 1851, it costs less than $200.

Another example is the way a singleblood testcan ascertain that a patient in the emergency room is not having a heart attack and so can forego the inconvenience and expense of additional invasive tests or unnecessary hospitalization.The highly sensitive blood test measures levels of cardiac troponin, a protein involved in muscle contraction; if the level is undetectable that is, below the limit of detection of the test there is a greater than 99% likelihood that the patient isnotexperiencing a heart attack and is at very low risk of other cardiac adverse events for at least 30 days.

That innovative approach is advantageous to patients and helps to reduce the frequency of hospitalizations and, therefore, healthcare costs.

Falls are both a cause and effect of declining health in the elderly.They are the leading cause of injury-related visits to emergency rooms and the primary cause of accidental deaths in Americans over the age of 65.To measure the potential benefits of a low-tech approach to preventing injuries from them, a research group in New Zealandcompared rates of falling and injuriesfrom falls on low-impact flooring (LIF) compared with standard vinyl flooring on an older persons health ward.Falls were prospectively monitored with written reports of all incidents, noting the location and consequences of each fall.The frequency of falls and injuries on LIF and those occurring on standard vinyl flooring (controls) were compared.

The investigators found that over the 31months of the study, there were 278 falls (among 178 persons who fell).The rate of falls was indistinguishable in the two groups, but fall-related injuries were significantly less frequent when they occurred on LIFs (22% of falls versus 34% of falls on control flooring).And many of the injuries that were averted were serious: Fractures occurred in 0.7% of falls in the LIF cohort versus 2.3% in the control cohort.

Thus, the New Zealand study provides a compelling rationale for adding low-impact flooring to housing for seniors (along withother modifications).

Dentistry has also benefited from costly high-tech innovations such asdental implants, but low-tech prophylaxis can also provide much needed benefits for dental health.

Tooth decay remains one of the majorpublic health concerns for both developing and developed countriesaccording to the World Health Organization.It is one of the most commonchronic problemsin the United States, where most adults will have at least one cavity in their lifetime. Decay causes inflammation in surrounding gum tissue, abscesses, and eventually, tooth loss. In addition to taking a significant toll on quality of life, decay and periodontitis has been linked to an increased risk of cardiovascular events, systemic infections such as endocarditis, and complications in pregnancy.

There have been significant advances in replacing and restoring teeth that need treatment due to dental caries, and these are often costly. However, preventing the problem in the first place is optimal, and an important advance was the introduction of water fluoridation, a simple low-tech intervention withproven efficacy, in 1945. The concept is that fluoride, a negatively charged ion, binds to calcium and phosphate on tooth surfaces to prevent the bacteria that cause cavities from even entering the tooth, thus protecting the teeth from dental decay and in some cases even reversing early decay. In a systematic review published in 2016, researchersfoundthat fluoridating drinking water in communities decreased overall decay and, thereby, the cost of more aggressive and costly dental interventions.

The Centers for Disease Control estimates that there are approximately100 million Americanswho are still without access to fluoridated water. Introducing this low-tech, high-impact measure more widely would substantially decrease the need for many dental procedures.

The high-tech miracles will continue to garner headlines, but to advance public health, simpler and relatively inexpensive innovations are also essential. That has policy implications: We need to put research dollars not only into potential big-ticket, high-tech blockbusters but also into ingenious, low-tech innovation.

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High-tech medical and dental innovation garner the headlines but the most impactful practices are mostly lower tech and prevention-focused - Genetic...

DetailsCategory: AntibodiesPublished on Tuesday, 01 December 2020 10:26Hits: 181

SIOUX FALLS, SD, USA I November 30, 2020 I SAB Biotherapeutics (SAB), a clinical stage biopharmaceutical company developing a novel immunotherapy platform to produce specifically targeted, high-potency, fully human polyclonal antibodies without the need for human serum, today announced that, as part of Operation Warp Speed, the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services, and the Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) have awarded SAB $57.5 million in expanded scope for its DiversitAb Rapid Response Antibody Program contract for the manufacturing of SAB-185, the companys clinical stage therapeutic candidate for COVID-19.

"We are pleased to be awarded this additional contract scope, which we believe is a reflection of the compelling science that supports SAB-185s potential in COVID-19, as well as the urgent need for treatment options amidst the global pandemic. Previous data has indicated that this human polyclonal antibody therapeutic has potent neutralizing activity against SARS-CoV-2, potentially driving more available doses, giving us the confidence to continue to progress our clinical development programs for SAB-185, said Eddie J. Sullivan, PhD, co-founder, president and CEO of SAB Biotherapeutics. This manufacturing agreement with BARDA and the Department of Defense supports our vision of bringing a novel, first-of-its-kind human polyclonal antibody therapeutic candidate for COVID-19 to patients, and I am proud of the work by our team and appreciate the continued support from BARDA and JPEO as we continue to rapidly advance SAB-185.

SAB-185 is currently being tested as a COVID-19 therapeutic in an ongoing Phase 1 trial in healthy volunteers and an ongoing Phase Ib trial in patients with mild or moderate COVID-19. SAB has leveraged its expertise to develop scalable manufacturing capabilities to support clinical activities, and continues to increase capacities in working with contract manufacturing organizations.

About SAB-185

SAB-185 is a fully-human, specifically-targeted and broadly neutralizing polyclonal antibody therapeutic candidate for COVID-19. The therapeutic was developed from SABs novel proprietary DiversitAb Rapid Response Antibody Program. SAB filed the Investigational New Drug (IND) application and produced the initial clinical doses in just 98 days from program initiation. The novel therapeutic has shown neutralization of both the Munich and Washington strains of mutated virus in preclinical studies. Preclinical data has also demonstrated SAB-185 to be significantly more potent than human-derived convalescent plasma.

About SAB Biotherapeutics, Inc.

SAB Biotherapeutics, Inc. (SAB) is a clinical-stage, biopharmaceutical company advancing a new class of immunotherapies leveraging fully human polyclonal antibodies. Utilizing some of the most complex genetic engineering and antibody science in the world, SAB has developed the only platform that can rapidly produce natural, specifically-targeted, high-potency, human polyclonal immunotherapies at commercial scale. SAB-185, a fully-human polyclonal antibody therapeutic candidate for COVID-19, is being developed with initial funding supported by the Biomedical Advanced Research Development Authority (BARDA), part of the Assistant Secretary for Preparedness and Response (ASPR) at the U.S. Department of Health and Human Services and the Department of Defense (DoD) Joint Program Executive Office for Chemical, Biological, Radiological, and Nuclear Defense (JPEO-CBRND) Joint Project Lead for Enabling Biotechnologies (JPL-EB). In addition to COVID-19, the companys pipeline also includes programs in Type 1 diabetes, organ transplant and influenza. For more information visit: http://www.sabbiotherapeutics.com or follow @SABBantibody on Twitter.

SOURCE: SAB Biotherapeutics

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SAB Biotherapeutics Awarded $57.5M from BARDA and US Department of Defense for Manufacturing of SAB-185 for the Treatment of COVID-19 | Antibodies |...

A new study associates a gene that facilitates neuron communication in the nervous system with memory loss.

A recent study1 led by The Jackson Laboratory and University of Maine has discovered that the gene Dlgap2 is associated with Alzheimer disease, dementia, and cognitive decline. Researchers found post-mortem human brain tissues of individuals experiencing poorer cognitive health and faster cognitive decline had low levels of Dlgap2.

The reason why this is so important is because a lot of research around cognitive aging and Alzheimers has been hyper-focused on well-known risk genes like APOE and brain pathologies, Catherine Kaczorowski, associate professor and Evnin family chair in Alzheimer disease research at The Jackson Laboratory (JAX), and adjunct professor with the University of Maine Graduate School of Biomedical Science and Engineering (GSBSE), said to the press. We wanted to give ourselves the option of looking at new things people keep ignoring because they've never heard about a gene before.2

Located in the synapses of neurons, Dlgap2 anchors critical receptors for signals between learning and memory neurons. The research team examined the memory and brain tissue from a large group of genetically diverse mice, relying on diversity outbred mice. The population came from 8 parents created by JAX, as they thought a diversified group would better reflect genetic diversity in humans. About 437 mice, eacheither 6, 12, or 18 months old were used.

Its great because you can harness the best parts of a mouse study and human society, Andrew Ouellette, a PhD student at JAX and a GSBSE NIH T32 predoctoral awardee, said to the press. Historically, research has been done with inbred mice with similar genetic makeups; same, similar genetic models. But clinically, humans don't work like that because they're not genetically identical.2

Quantitative trait loci mapping was performed on the mouse population. Study of entire genome sequences allowed for identification of the genes responsible for varying cognitive function and where they occurred. Researchers pinpointed the connection between Dlgap2 and memory decline in mice, and were then able to evaluate its significance to humans.

They found Dlgap2 is associated with the degree of memory loss in mice and risk for Alzheimer dementia in humans. Further research will be needed to determine how the gene influences dementia and brain functioning.

References

1. Ouellette AR, Neuner SM, Dumitrescu L, Hadad N, et al. Cross-species analyses identify Dlgap2 as a regulator of age-related cognitive decline and alzheimers dementia. Cell Reports. 2020;32(9):108091.

2. University of Maine. New connection between Alzheimer's dementia and Dlgap2. News release. ScienceDaily. November 23, 2020. https://www.sciencedaily.com/releases/2020/11/201123161040.htm

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Dlgap2: The Gene Associated With Memory Loss - Psychiatric Times

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. What Klaus Schwab prophesized in his 2015 book, The Fourth Industrial Revolution, is playing out before our very eyes. Quantum computing power, a network architecture that is moving function closer to the edge of our interconnected devices, bandwidth speeds of 5G and beyond, natural language processing, artificial intelligence, and machine learning are all working together to accelerate innovation in fundamental ways. Given the global pandemic, in the biological sphere, government industrial policy drives the public sector to work hand-in-glove with private industry and academia to develop new therapies and vaccines to treat and prevent COVID-19 and other lethal diseases. This post will envision the future of gene editing technologies and the legal and ethical challenges that could imperil their mission of saving lives.

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. Emmanuelle Charpentier, a French professor of microbiology, genetics, and biochemistry, and Jennifer Doudna, an American professor of biochemistry, pioneered a revolutionary new gene-editing technology called CRISPR/Cas9. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and Cas9 stands for CRISPR-associated protein 9. In 2020, the revolutionary work of Drs. Charpentier and Doudna developing CRISPR/Cas9 were recognized with the Nobel Prize for Chemistry. The technology was also the source of a long-running and high-profile patent battle between two groups of scientsists.

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 cells genome at the desired location, allowing existing genes to be removed and new ones added to a living organisms 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.

The following diagram from CRISPR Therapeutics AG, a Swiss company, illustrates how it functions:

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 The Business Research Company, the global CRISPR technology market reached a value of nearly $700 million in 2019, is expected to more than double in 2020, and reach $6.7 billion by 2030. Market applications target all forms of life, from animals to plants to humans.

Gene editings 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 $100 million or more according to the latest PwC/Moneytree report. Healthcare startups raised over $8 billion in the quarter in the United States alone. Gene-editing company Mammouth Biosciences raised a $45 million round of Series B capital in the second quarter of 2020. CRISPR Therapeutics AG raised more in the public markets in primary and secondary capital.

Bayer, Humboldt Fund and Leaps are co-leading a $65 million Series A round for Metagenomi, a biotech startup launched by UC Berkeley scientists. Metagenomi, which will be run by Berkeleys Brian Thomas, is developing a toolbox of CRISPR- and non-CRISPR-based gene-editing systems beyond the Cas9 protein. The goal is to apply machine learning to search through the genomes of these microorganisms, finding new nucleases that can be used in gene therapies. Other investors in the Series A include Sozo Ventures, Agent Capital, InCube Ventures and HOF Capital. Given the focus on new therapies and vaccines to treat the novel coronavirus, we expect continued wind in the sails for gene-editing companies, particularly those with strong product portfolios that leverage the technology.

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 China created the first human genetically edited babies, twin girls, causing public outcry (and triggering government sanctioning of the researcher). In response, the World Health Organization established a committee to advise on the creation of standards for gene editing oversight and governance standards on a global basis.

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 the United States, the National Institute of Health has stated that it will not fund gene-editing studies in human embryos. A U.S. statute called The Dickey-Wicker Amendment prohibits the use of federal funds for research projects that would create or destroy human life. Laws in the United Kingdom prohibit genetically modified embryos from being implanted into women. Still, embryos can be altered in research labs under license from the Human Fertilisation and Embryology Authority.

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. The U.S. National Academy of Sciences recommended seven principles for the governance of human genome editing, including promoting well-being, transparency, due care, responsible science, respect for persons, fairness, and transnational co-operation. In the United Kingdom, a non-governmental organization formed in 1991 called The Nuffield Council has proposed two principles for the ethical acceptability of genome editing in the context of reproduction. First, the intervention intends to secure the welfare of the individual born due to such technology. Second, social justice and solidarity principles are upheld, and the intervention should not result in an intensifying of social divides or marginalizing of disadvantaged groups in society. In 2016, in application of the same, the Crick Institute in London was approved to use CRISPR-Cas9 in human embryos to study early development. In response to a cacophony of conflicting national frameworks, the International Summit on Human Gene Editing was formed in 2015 by NGOs in the United States, the United Kingdom and China, and is working to harmonize regulations global from both the ethical and safety perspectives. As CRISPR co-inventor Jennifer Doudna has written in a now infamous editorial in SCIENCE, stakeholders must engage in thoughtfully crafting regulations of the technology without stifling it.

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.

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Future Visioning the Role of CRISPR Gene Editing: Navigating Law and Ethics to Regenerate Health and Cure Disease - IPWatchdog.com

"Despite the projected growth in market applications andabundant investment capital, there is a danger that legal andethical concerns related to genetic research could put the brakeson gene editing technologies and product programs emanatingtherefrom."

There are thousands of diseases occurring in humans, animals,and plants caused by aberrant DNA sequences. Traditional smallmolecule and biologic therapies have only had minimal success intreating many of these diseases because they mitigate symptomswhile failing to address the underlying genetic causes. While humanunderstanding of genetic diseases has increased tremendously sincethe mapping of the human genome in the late 1990s, our ability totreat them effectively has been limited by our historical inabilityto alter genetic sequences.

The science of gene editing was born in the 1990s, as scientistsdeveloped tools such as zinc-finger nucleases (ZFNs) and TALEnucleases (TALENs) to study the genome and attempt to altersequences that caused disease. While these systems were anessential first step to demonstrate the potential of gene editing,their development was challenging in practice due to the complexityof engineering protein-DNA interactions.

Then, in 2011, Dr. Emmanuelle Charpentier, a French professor ofmicrobiology, genetics, and biochemistry, and Jennifer Doudna, anAmerican professor of biochemistry, pioneered a revolutionary newgene-editing technology called CRISPR/Cas9. Clustered Regularly InterspacedShort Palindromic Repeats (CRISPR) and Cas9 stands forCRISPR-associated protein 9. In 2020, the revolutionary work ofDrs. Charpentier and Doudna developing CRISPR/Cas9 were recognizedwith the Nobel Prize for Chemistry. The technology was also thesource of a long-running and high-profile patent battle between two groups ofscientsists.

CRISPR/Cas9 for gene editing came about from a naturallyoccurring viral defense mechanism in bacteria. The system ischeaper and easier to use than previous technologies. It deliversthe Cas9 nuclease complexed with a synthetic guide RNA (gRNA) intoa cell, cutting the 'cell's genome at the desired location,allowing existing genes to be removed and new ones added to aliving organism's genome. The technique is essential inbiotechnology and medicine as it provides for the genomes to beedited in vivo with extremely high precision, efficiently, and withcomparative ease. It can create new drugs, agricultural products,and genetically modified organisms or control pathogens and pests.More possibilities include the treatment of inherited geneticdiseases and diseases arising from somatic mutations such ascancer. However, its use in human germline genetic modification ishighly controversial.

The following diagram from CRISPR Therapeutics AG, a Swisscompany, illustrates how it functions:

In the 1990s, nanotechnology and gene editing were necessaryplot points for science fiction films. In 2020, developments likenano-sensors and CRISPR gene editing technology have moved thesetechnologies directly into the mainstream, opening a new frontierof novel market applications. According to The Business ResearchCompany, the global CRISPR technology market reached a value ofnearly $700 million in 2019, is expected to more than double in2020, and reach $6.7 billion by 2030. Market applications targetall forms of life, from animals to plants to humans.

Gene editing's primary market applications are for thetreatment of genetically-defined diseases. CRISPR/Cas9 gene editingpromises to enable the engineering of genomes of cell-basedtherapies and make them safer and available to a broader group ofpatients. Cell therapies have already begun to make a meaningfulimpact on specific diseases, and gene editing helps to acceleratethat progress across diverse disease areas, including oncology anddiabetes.

In the area of human therapy, millions of people worldwidesuffer from genetic conditions. Gene-editing technologies likeCRISPR-Cas9 have introduced a way to address the cause ofdebilitating illnesses like cystic fibrosis and create betterinterventions and therapies. They also have promising marketapplications for agriculture, food safety, supply, anddistribution. For example, grocery retailers are even looking athow gene editing could impact the products they sell. Scientistshave created gene-edited crops like non-browning mushrooms andmildew-resistant grapes - experiments that are part of an effort toprevent spoilage, which could ultimately change the way food issold.

Despite the inability to travel and conduct face-to-facemeetings, attend industry conferences or conduct business otherthan remotely or with social distance, the investment markets forventure, growth, and private equity capital, as well as corporateR&D budgets, have remained buoyant through 2020 to date.Indeed, the third quarter of 2020 was the second strongest quarterever for VC-backed companies, with 88 companies raising roundsworth $100 million or more according to the latest PwC/Moneytreereport. Healthcare startups raised over $8 billion in the quarterin the United States alone. Gene-editing company MammouthBiosciences raised a $45 million round of Series B capital in thesecond quarter of 2020. CRISPR Therapeutics AG raised more in thepublic markets in primary and secondary capital.

Bayer, Humboldt Fund and Leaps are co-leading a $65 million Series A round for Metagenomi, abiotech startup launched by UC Berkeley scientists. Metagenomi,which will be run by Berkeley's Brian Thomas, is developing atoolbox of CRISPR- and non-CRISPR-based gene-editing systems beyondthe Cas9 protein. The goal is to apply machine learning to searchthrough the genomes of these microorganisms, finding new nucleasesthat can be used in gene therapies. Other investors in the Series Ainclude Sozo Ventures, Agent Capital, InCube Ventures and HOFCapital. Given the focus on new therapies and vaccines to treat thenovel coronavirus, we expect continued wind in the sails forgene-editing companies, particularly those with strong productportfolios that leverage the technology.

Despite the projected growth in market applications and abundantinvestment capital, there is a danger that legal and ethicalconcerns related to genetic research could put the brakes ongene-editing technologies and product programs emanating therefrom.The possibility of off-target effects, lack of informed consent forgermline therapy, and other ethical concerns could cause governmentregulators to put a stop on important research and developmentrequired to cure disease and regenerate human health.

Gene-editing companies can only make money by developingproducts that involve editing the human genome. The clinical andcommercial success of these product candidates depends on publicacceptance of gene-editing therapies for the treatment of humandiseases. Public attitudes could be influenced by claims that geneediting is unsafe, unethical, or immoral. Consequently, productscreated through gene editing may not gain the acceptance of thegovernment, the public, or the medical community. Adverse publicreaction to gene therapy, in general, could result in greatergovernment regulation and stricter labeling requirements ofgene-editing products. Stakeholders in government, third-partypayors, the medical community, and private industry must work tocreate standards that are both safe and comply with prevailingethical norms.

The most significant danger to growth in gene-editingtechnologies lies in ethical concerns about their application tohuman embryos or the human germline. In 2016, a group of scientistsedited the genome of human embryos to modify the gene forhemoglobin beta, the gene in which a mutation occurs in patientswith the inherited blood disorder beta thalassemia. Althoughconducted in non-viable embryos, it shocked the public thatscientists could be experimenting with human eggs, sperm, andembryos to alter human life at creation. Then, in 2018, abiophysics researcher in China created the first human geneticallyedited babies, twin girls, causing public outcry (and triggeringgovernment sanctioning of the researcher). In response, the WorldHealth Organization established a committee to advise on thecreation of standards for gene editing oversight and governancestandards on a global basis.

Some influential non-governmental agencies have called for amoratorium on gene editing, particularly as applied to altering thecreation or editing of human life. Other have set forth guidelineson how to use gene-editing technologies in therapeuticapplications. In the United States, the National Institute ofHealth has stated that it will not fund gene-editing studies inhuman embryos. A U.S. statute called "The Dickey-WickerAmendment" prohibits the use of federal funds for researchprojects that would create or destroy human life. Laws in theUnited Kingdom prohibit genetically modified embryos from beingimplanted into women. Still, embryos can be altered in researchlabs under license from the Human Fertilisation and EmbryologyAuthority.

Regulations must keep pace with the change that CRISPR-Cas9 hasbrought to research labs worldwide. Developing international guidelines could be a steptowards establishing cohesive national frameworks. The U.S.National Academy of Sciences recommended seven principles for thegovernance of human genome editing, including promoting well-being,transparency, due care, responsible science, respect for persons,fairness, and transnational co-operation. In the United Kingdom, anon-governmental organization formed in 1991 called The NuffieldCouncil has proposed two principles for the ethical acceptabilityof genome editing in the context of reproduction. First, theintervention intends to secure the welfare of the individual borndue to such technology. Second, social justice and solidarityprinciples are upheld, and the intervention should not result in anintensifying of social divides or marginalizing of disadvantagedgroups in society. In 2016, in application of the same, the CrickInstitute in London was approved to use CRISPR-Cas9 in humanembryos to study early development. In response to a cacophony ofconflicting national frameworks, the International Summit on HumanGene Editing was formed in 2015 by NGOs in the United States, theUnited Kingdom and China, and is working to harmonize regulationsglobal from both the ethical and safety perspectives. As CRISPRco-inventor Jennifer Doudna has written in a now infamous editorialin SCIENCE, "stakeholders must engage in thoughtfullycrafting regulations of the technology without stiflingit."

The COVID-19 pandemic has forced us to rely more on newtechnologies to keep us healthy, adapt to working from home, andmore. The pandemic makes us more reliant on innovative digital,biological, and physical solutions. It has created a united senseof urgency among the public and private industry (together withgovernment and academia) to be more creative about using technologyto regenerate health. With continued advances in computing power, networkarchitecture, communications bandwidths, artificial intelligence,machine learning, and gene editing, society will undoubtedly findmore cures for debilitating disease and succeed in regeneratinghuman health. As science advances, it inevitably intersects withlegal and ethical norms, both for individuals and civil society,and there are new externalities to consider. Legal and ethicalnorms will adapt, rebalancing the interests of each. The fourthindustrial revolution is accelerating, and hopefully towards curingdisease.

Originally published by IPWatchdog.com, November 24,2020.

The content of this article is intended to provide a generalguide to the subject matter. Specialist advice should be soughtabout your specific circumstances.

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Future Visioning The Role Of CRISPR Gene Editing: Navigating Law And Ethics To Regenerate Health And Cure Disease - Technology - United States -...

Over centuries of human existence, we have made significant inventions that have modified the kind of lives we lead today. From the invention of the wheel to the invention of the computer that we carry inside our pockets- we, as humans, have evolved tremendously on account of technological and scientific advancements. Many such innovations have also been made in the field of biology or human reproduction. From birth control to IVF procedures- we have come a long way.

But what if there was a technology that allowed modification of the DNA present in our genes to create a designer baby? Apart from being the feature of a famous movie- youll be amazed to know that the technology to create genetically modified DNA exists in the world today. How does the technology work? Is it safe? Are there any ethical concerns revolving around the issue, AND will the future of humanity have genetically designed babies? Lets find out!

When you alter a babys genetic makeup to remove a particular gene(s) associated with a disease, you successfully create a designer baby. How is it done? One way is to use a process is called preimplantation genetic diagnosis. This process analyses a wide range of human embryos associated with a particular disease and selecting seeds that have the desired genetic makeup.

Source News-medical

Another less popular method is called Germline engineering, which enables altering a babys genetic information before birth. The desired genetic material is introduced into the embryo itself or the sperm and egg cells, either by delivering the selected genes directly into the cell or using the gene-editing technology.

The latter is an illegal procedure in various nations, and the only instance of the process being used was in the case of Lulu and Nana, a pair of Chinese twins in 2019. Subsequently, the development attracted widespread criticism for the same.

Genetic editing is done either by removing small sections of the existing genome or by introducing new segments of DNA into the genome. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a genome editing technology introduced by researchers Emmanuelle Charpentier and Jennifer Doudna. The technology allows scientists to cheaply and very rapidly alter the genome of almost any organism. CRISPR makes use of an enzyme called Cas-9, which is used to cut out selected sections of DNA or add new units to the existing DNA.

The technology introduced by CRISPR can be used on various organisms, plants, and animals to modify their DNA since all living species contain genes. This provides much scope for altering a bodies immune system to destroy cancer cells or battle sickle cell anemia by introducing non-sickle cell genes. It can be used to make better crops, enhance drugs, and to treat severe diseases. However, the debate regarding the ethical standing of gene editing comes in when deciding what do we want to use it for.

The gene-editing technology can be used on somatic cells and germ cells, which offers varied results. While any changes made to somatic cells, which are particular cells present in, say, our liver or lungs- will not be passed onto the future generation. However, changes made to germ cells will be transferred into the subsequent generations. Hence, posing a threat to civilization.

Credits: Netflix: explained

Using the gene-editing technology for cosmetic purposes such as changing the eye color, introducing freckles, etc., further questions the moral and ethical standards of the procedure. It is no surprise that biological processes such as these are not very economical and can only be accessed by a particular section of society. This is arguably one of the most controversial arguments against designer DNA. This would enable parents or doctors will to dictate traits such as gender, height, and even the intellect of their baby. This would give an advantage to people who can afford gene-editing, potentially leading to a genetic class systemenabling science to enable evolution instead of nature.

The authors of the news study have not dismissed the ethical concerns surrounding the same and have actively advocated the use of technology only for preventing serious diseases and conditions. Despite the concerns regarding the possibility of a genetic class system, the fact remains that Talents and traits are genetically complexabout93,000 genetic variations influence even more superficial features like height. Gene editing will not and cannot guarantee superior traits and wont be capable of curing several illnesses.

Source abc.net

While I appreciate the fear, I think we need to realize that with every technology we have had these fears, and they havent been realized, said R. Alta Charo, a bioethicist at University of Wisconsin-Madison, who co-led the national committee on human embryo editing

Designer babies or designer DNA can hence, be something that can find a middle ground between advancement and ethics in the future.

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Are Designer Babies Going to be the Future of Mankind? - Sambad English