Category Archives: Gene Medicine

1. In this case study involving one generally well infant with coronavirus disease 2019 (COVID-19), various parts of the isolation room were found to be contaminated with PCR-detectable SARS-CoV-2 on day 2 of admission.

2. Despite close physical contact with the infant during feeding, all three items of personal protective equipment worn by the healthcare worker were found to be negative for the virus.

Evidence Rating Level: 4 (Below Average)

Study Rundown: Severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2) is suspected to spread primarily through droplets and direct contact, but it is unknown whether airborne and fomite transmission are also causes for concern. Additionally, there is no data available regarding the risk of transmission from asymptomatic or pauci-symptomatic infants and children. This case study of an asymptomatic 6-month-old infant with COVID-19 revealed that, by day 2 of admission, the bedding, cot rail, and table in the isolation room all carried detectable amounts of the virus. While the viral load of the bedding was higher than that of the railing, the table, situated one meter from the patient, was found to have nearly the same concentration of viral particles as the bedding. Because viral load in the environment ought to fall with increasing distance from the source for droplet transmission, this unusual finding supports the possibility of contamination via indirect contact when the healthcare worker transferred items such as baby formula and baby wipes between the patient and the table. While virus viability was not assessed, these results reaffirm the importance of hand hygiene and social distancing to prevent unwitting spread by asymptomatic or presymptomatic carriers.

Click here to read the study in Annals of Internal Medicine

Relevant Reading: A Well Infant With Coronavirus Disease 2019 With High Viral Load

In-Depth [case study]: In this case study, a 6-month-old male was admitted to KK Womens and Childrens Hospital in Singapore after both his parents developed fever and sore throat within three days of each other. Upon arrival, the infant was afebrile and in no respiratory distress, but real-time reverse transcription polymerase chain reaction (rRT-PCR) testing of a nasopharyngeal specimen confirmed COVID-19 infection with very high viral load and cycle threshold (Ct) values of 15.6 and 13.7 for the N gene and Orf1ab gene, respectively. Ct values of <36 were considered positive, with lower values corresponding to higher viral load. On day 2 of admission, nasopharynx Ct values for the N gene and Orf1ab gene were 18.8 and 18.6, respectively, while urine and stool samples continued to test negative. After the infant was carried and fed within a span of 15 minutes, synthetic fiber flocked swabs with Universal Transport Medium were run over nearly 100% of the infants bedding, the cot rail, and a table located 1 meter away from the bed as well as the healthcare workers face shield, N95 mask, and waterproof gown. All three PPE samples were found to be negative for SARS-nCov-2, but the RdRp gene Ct values for the bedding, rail, and table were 28.7, 33.3, and 29.7, respectively.

Image: PD

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SARS-CoV-2 detected on various surroundings of asymptomatic COVID-19 positive infant - 2 Minute Medicine

SARS-COV-2 has upended modern health care, leaving health systems struggling to cope. Addressing a fast-moving and uncontrolled disease requires an equally efficient method of discovery, development and administration. Artificial Intelligence (AI) and Machine Learning driven health care solutions provide such an answer. AI-enabled health care is not the medicine of the future, nor does it mean robot doctors rolling room to room in hospitals treating patients. Instead of a hospital from some future Jetsons-like fantasy, AI is poised to make impactful and urgent contributions to the current health care ecosystem. Already AI-based systems are helping to alleviate the strain on health care providers overwhelmed by a crushing patient load, accelerate diagnostic and reporting systems, and enable rapid development of new drugs and existing drug combinations that better match a patients unique genetic profile and specific symptoms.

For the thousands of patients fighting for their lives against this deadly disease and the health care providers who incur a constant risk of infection, AI provides an accelerated route to understand the biology of COVID-19. Leveraging AI to assist in prediction, correlation and reporting allow health care providers to make informed decisions quickly. With the current standard of PCR based testing requiring up to 48 hours to return a result, New York-based Envisagenics has developed an AI platform that analyzes 1,000 patient samples in parallel in just two hours. Time saves lives, and the company hopes to release the platform for commercial use in the coming weeks.

AI-powered wearables, such as a smart shirt developed by Montreal-based Hexoskin to continuously measure biometrics including respiration effort, cardiac activity, and a host of other metrics, provide options for hospital staff to minimize exposure by limiting the required visits to infected patients. This real-time data provides an opportunity for remote monitoring and creates a unique dataset to inform our understanding of disease progression to fuel innovation and enable the creation of predictive metrics, alleviating strain on clinical staff. Hexoskin has already begun to assist hospitals in New York City with monitoring programs for their COVID-19 patients, and they are developing an AI/ML platform to better assess the risk profile of COVID-19 patients recovering at home. Such novel platforms would offer a chance for providers and researchers to get ahead of the disease and develop more effective treatment plans.

AI also accelerates discovery and enables efficient and effective interrogation of, the necessary chemistry to address COVID-19. An increasing number of companies are leveraging AI/ML to identify new treatment paths, whether from a list of existing molecules or de novo discovery. San Francisco-based Auransa is using AI to map the gene sequence of SARS-COV-2 to its effect on the host to generate a short-list of already approved drugs that have a high likelihood to alleviate symptoms of COVID-19. Similarly, UK-based Healx has set its AI platform to discover combination therapies, identifying multi-drug approaches to simultaneously treat different aspects of the disease pathology to improve patient outcomes. The company analyzed a library of 4,000 approved drugs to map eight million possible pairs and 10.5 billion triplets to generate combination therapy candidates. Preclinical testing will begin in May 2020.

Developers cannot always act alone - realizing the potential of AI often requires the resources of a collaboration to succeed. Generally, the best data sets and the most advanced algorithms do not exist within the same organization, and it is often the case that multiple data sources and algorithms need to be combined for maximum efficacy. Over the last month, we have seen the rise of several collaborations to encourage information sharing and hasten potential outcomes to patients.

Medopad, a UK-based AI developer, has partnered with Johns Hopkins University to mine existing datasets on COVID-19 and relevant respiratory diseases captured by the UK Biobank and similar databases to identify a biomarker associated with a higher risk for COVID-19. A biomarker database is essential in executing long-term population health measures, and can most effectively be generated by an AI system. In the U.S., over 500 leading companies and organizations, including Mayo Clinic, Amazon Web Services and Microsoft, have formed the COVID-19 Healthcare Coalition to assist in coordinating on all COVID-19 related matters. As part of this effort, LabCorp and HD1, among others, have come together to use AI to make testing and diagnostic data available to researchers to help build disease models including predictions of future hotspots and at-risk populations. On the international stage, the recently launched COAI, a consortium of AI-companies being assembled by French-US OWKIN, aims to increase collaborative research, to accelerate the development of effective treatments, and to share COVID-19 findings with the global medical and scientific community.

Leveraging the potential of AI and machine learning capabilities provides a potent tool to the global community in tackling the pandemic. AI presents novel ways to address old problems and opens doors to solving newly developing population health concerns. The work of our health care system, from the research scientists to the nurses and physicians, should be celebrated, and we should embrace the new tools which are already providing tremendous value. With the rapid deployment and integration of AI solutions into the COVID-19 response, the health care of tomorrow is already addressing the challenges we face today.

Brandon Allgood, PhD, is vice chair of the Alliance for Artificial Intelligence in Healthcare, a global advocacy organization dedicated to the discovery, development and delivery of better solutions to improve patient lives. Allgood is a SVP of DS&AI at Integral Health, a computationally driven biotechnology company in Boston.

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Health care of tomorrow, today: How artificial intelligence is fighting the current, and future, COVID-19 pandemic | TheHill - The Hill

The coronavirus is running rampant through senior residential facilities, especially nursing homes. One source: Hospitals.

Ten years ago, as the conveyor belt of medicine geared up, profiteering hospitals learned to discharge patients as rapidly as possible. Some patients went home, but to expedite the transition, the path of least resistance was sending them to nursing homes.

To make them more palatable, nursing homes were rebranded as skilled nursing facilities, and many further enhanced their name to post-acute rehab. Yet, the care and reputation did not change.

Fast-forward to our present crisis. The glitch: Hospitals do not have to reveal whether medical staff members have tested positive for COVID-19 and continually hide behind the guise of confidentiality and HIPAA, shunning voluntary self-reporting. (Legally true in California. Legislators, are you listening?)

The existing hospital administrative attitude of get em in and get em out could therefore have created a vicious cycle of discharged patients contaminating residents at nursing facilities.

Moms, dads, aunts, uncles, sisters, brothers, veterans, retired teachers and first responders have been some of those vulnerable victims.

The roots of the present problem lie in the past, but we must dig in the future to connect the dots.

Gene Uzawa Dorio, M.D., is a geriatric house-call physician who serves as president of the Los Angeles County Commission for Older Adults and Assemblyman to the California Senior Legislature. He has practiced in the Santa Clarita Valley for 32 years.

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Connecting the Hospital-Nursing Home Dots | Doctor's Diary with Dr. Gene Dorio - SCVNEWS.com

GERMANTOWN, Md., April 20, 2020 /PRNewswire/ --Precigen, Inc.(Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to initiate a Phase I/II trial for Precigen's PRGN-2009, a first-in-class,off-the-shelf (OTS) investigational immunotherapy utilizing the AdenoVerse platform designed to activate the immune system to recognize and target HPV+ solid tumors. HPV+ cancers represent a significant health burden in indications such as head and neck, cervical, vaginal and anal cancer.

ThePhase I portion of the study will follow 3+3 dose escalation to evaluate the safety of PRGN-2009 administered as a monotherapy and to determine the recommended Phase II dose (R2PD) followed by an evaluation of the safety of the combination of PRGN-2009 at the R2PD and an investigational bifunctional fusion protein in patients with recurrent or metastatic HPV-associated cancers. The Phase II portion of the study will evaluate PRGN-2009 as a monotherapy or in combination with the bifunctional fusion protein in patients with newly-diagnosed stage II/III HPV16-positive oropharyngeal cancer.

PRGN-2009 leverages Precigen's UltraVector and AdenoVerse platforms to optimize HPV antigen design in combination with its gorilla adenovector with a large payload capacity and the ability for repeat administration due to very low to non-existent seroprevalence in the human population.

PRGN-2009 is under development through a Cooperative Research and Development Agreement, or CRADA, within the laboratory of Dr. Jeffrey Schlom, Chief oftheLaboratory of Tumor Immunology and Biology (LTIB), Center for Cancer Research (CCR),National Cancer Institute (NCI). This CRADA has allowed Precigen to rapidly and cost-effectively advance PRGN-2009 to the clinic.The Phase I/II clinical trial of PRGN-2009 will be conducted at the NIH Clinical Center and will be led by Dr. Julius Strauss, Co-Director of the LTIB's Clinical Trials Group, and Dr. James Gulley, Chief of the Genitourinary Malignancies Branch, CCR, NCI.

"Globally, high-risk HPVs cause nearly 5% of all cancers, with about 570,000 women and 60,000 men diagnosed with HPV-related cancers each year," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are incredibly proud of our continued relationship with NCI and the tremendous progress in bringing forward this novel asset class in such a short period of time. Advancements are critically needed to better target HPV+ tumors across multiple patient groups, and we have been encouraged by the promising preclinical data for PRGN-2009 in potentially targeting this patient population."

About HPV+ CancersHPV infects the squamous cells that line the inner surfaces of certain organs and, consequently, most HPV-related cancers are a type of cancer called squamous cell carcinoma. Some cervical cancers come from HPV infection of gland cells in the cervix and are referred to as adenocarcinomas.1 HPV-related cancers include cervical, oropharyngeal, anal, penile, vaginal, and vulvar.1 Nearly 44,000 HPV-associated cancers occur in the United States each year. Of these, approximately 25,000 occur in women and 19,000 occur in men.2HPV is considered responsible for more than 90% of analand cervicalcancers, about 70% of vaginal and vulvar cancers, and more than 60% of penile cancers.2 Recent studies indicate that about 70% of cancers of the oropharynxalso may be related to HPV.2

Precigen: Advancing Medicine with PrecisionPrecigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization.

For more information about Precigen, visit http://www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

References1HPV and Cancer, National Institutes of Health. Accessed in April 20202HPV-Associated Cancer Statistics, Centers for Disease Control and Prevention. Accessed in April 2020

TrademarksPrecigen, AdenoVerse, UltraVector, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

Safe Harbor StatementSome of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical and clinical trials and discovery programs, the promise of the Company's portfolio of therapies, the Company's refocus to a healthcare-oriented business, and its continuing evaluation of options for the Company's non-healthcare businesses. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties, including the possibility that the timeline for the Company's clinical trial might be impacted by the COVID-19 pandemic, and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission.

Investor Contact:

Steven Harasym

Vice President, Investor Relations

Tel: +1 (301) 556-9850

investors@precigen.com

Media Contact:

Marie Rossi, PhD

Vice President, Communications

Tel: +1 (301) 556-9850

press@precigen.com

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SOURCE Precigen, Inc.

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Precigen Announces Clearance of IND to Initiate Phase I/II Study for First-in-Class PRGN-2009 AdenoVerse Immunotherapy to Treat HPV-positive (HPV+)...

Emotional, social, and psychiatric problems in children and adolescents have been linked to higher levels of genetic vulnerability for adult depression, according to University of Queensland scientists. They made the finding Genetic Associations Between Childhood Psychopathology and Adult Depression and Associated Traits in 42998 Individuals: A Meta-Analysis, which appears inJAMA Psychiatry, while analyzing the genetic data of more than 42,000 children and adolescents from seven cohorts across five European countries.

Christel Middeldorp, MD, PhD, a child and adolescent psychiatrist at the Child Health Research Centre at the University of Queensland, said that researchers have also found a link with a higher genetic vulnerability for insomnia, neuroticism, and body mass index.

By contrast, study participants with higher genetic scores for educational attainment and emotional wellbeing were found to have reduced childhood problems, she pointed out.

We calculated a persons level of genetic vulnerability by adding up the number of risk genes they had for a specific disorder or trait, and then made adjustments based on the level of importance of each gene. We found the relationship was mostly similar across ages.

Adult mood disorders are often preceded by behavioral and emotional problems in childhood. It is yet unclear what explains the associations between childhood psychopathology and adult traits. To investigate whether genetic risk for adult mood disorders and associated traits is associated with childhood disorders, write the investigators.

This meta-analysis examined data from 7 ongoing longitudinal birth and childhood cohorts from the U.K., the Netherlands, Sweden, Norway, and Finland. Starting points of data collection ranged from July 1985 to April 2002. Participants were repeatedly assessed for childhood psychopathology from ages 6 to 17 years. Data analysis occurred from September 2017 to May 2019.

Individual polygenic scores (PGS) were constructed in children based on genome-wide association studies of adult major depression, bipolar disorder, subjective well-being, neuroticism, insomnia, educational attainment, and body mass index (BMI).

Results from this study suggest the existence of a set of genetic factors influencing a range of traits across the life span with stable associations present throughout childhood. Knowledge of underlying mechanisms may affect treatment and long-term outcomes of individuals with psychopathology.

The results indicate there are shared genetic factors that affect a range of psychiatric and related traits across a persons lifespan. Around 50 percent of children and adolescents with psychiatric problems, such as attention deficit hyper-activity disorder (ADHD), continue to experience mental disorders as adults, and are at risk of disengaging with their school community among other social and emotional problems, added Middeldorp.

Our findings are important as they suggest this continuity between childhood and adult traits is partly explained by genetic risk, she continued. Individuals at risk of being affected should be the focus of attention and targeted treatment. Although genetic vulnerability is not accurate enough at this stage to make individual predictions about how a persons symptoms will develop over time, it may become so in the future, in combination with other risk factors.

Middeldorp believes that this study and others may support precision medicine by providing targeted treatments to children at the highest risk of persistent emotional and social problems.

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Childhood Psychopathology Linked to Higher Levels of Genetic Vulnerability of Adult Depression - Clinical OMICs News

CARLSBAD, Calif., April 20, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, today announced that its partner Roche, also known as Genentech in the United States, has completed enrollment for GENERATION HD1, a global Phase 3 study evaluating the efficacy and safety of tominersen (previously IONIS-HTTRx or RG6042), an investigational antisense therapy for people living with Huntington's disease (HD).

"Completion of the enrollment of this Phase 3 study is an important landmark for the clinical development of tominersen and for families affected by Huntington's disease. While there is much work ahead of us, we are now closer to potentially providing a treatment for people living with this devastating disease. We are grateful to Huntington's disease patients, their families and healthcare providers for their courage and resilience, particularly in the current challenging environment," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "At Ionis, knowing that sick people depend on us fuels our passion for discovering and delivering novel antisense medicines like tominersen, the first and only therapy in pivotal trials targeting the underlying cause of HD."

GENERATION HD1 is evaluating the efficacy and safety of tominersen treatment administered once every two months (eight weeks) or every four months (16 weeks) over a period of 25 months, compared to placebo. The study has completed enrollment with 791 patients across approximately 100 sites around the world.

HD is a devastating, and ultimately fatal, hereditary disease resulting in deterioration in mental abilities and physical control. Currently, there is no approved disease-modifying treatment for HD. There are approximately 3 to 10 per 100,000 people worldwide affected by HD. In the U.S. alone, there are approximately 40,000 people with symptomatic HD and more than 200,000 people at risk of having inherited the gene that causes HD.

About tominersenTominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. Tominersen is the first therapy in pivotal trials targeting the underlying cause of HD. In December 2017, Roche licensed the investigational molecule from Ionis.

In the Phase 1/2 study, 46 people with early stage HD were treated with tominersen or placebo for 13 weeks. The data demonstrated significant, dose-dependent reductions in mHTT in the cerebrospinal fluid (CSF) of treated participants with a favorable safety and tolerability profile.

Tominersen is being investigated in a Phase 3 study (GENERATION HD1), an open label extension study in HD patients and a Phase I pharmacokinetics and pharmacodynamics study (GEN-PEAK). These studies, in addition to the non-interventional HD Natural History Study, are important elements of the clinical program to thoroughly evaluate the potential of tominersen to be the first disease-modifying medicine for the treatment of HD. The Phase 3 GENERATION HD1 study is expected to complete in 2022. The timing for this study's completion remains unchanged.

Additional information about tominersen clinical trials may be found at https://clinicaltrials.gov/ct2/show/NCT03761849.

About Ionis Pharmaceuticals, Inc.As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardiovascular, infectious, and pulmonary diseases.

To learn more about Ionis visit http://www.ionispharma.com or follow us on twitter @ionispharma.

Ionis' Forward-looking StatementThis press release includes forward-looking statements regarding Ionis' alliance with Roche and the development, activity, therapeutic potential, commercial potential and safety of tominersen (IONIS-HTTRx or RG6042). Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2019, which is on file with the SEC. Copies of this and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals is a trademark of Ionis Pharmaceuticals, Inc.

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SOURCE Ionis Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:IONS

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Ionis and partner announce enrollment completion of global Phase 3 GENERATION HD1 study for Huntington's disease - BioSpace