Daily Archives: July 13, 2020

Part 2 of 3. Read Part 1.

Humanity has been on a very steep learning curve since, reacting to a 31 December 2019 report from the Wuhan Municipal Health Commission that they were seeing an atypical pneumonia of unknown cause, the World Health Organisation (WHO) published (5 January, 2020) the first Disease Outbreak Bulletin to alert the public health community and the global media to the disease we call COVID-19. From the outset, both the WHO and the local virologists were primed by the 2002/3 SARS-CoV-1 experience to think that a coronavirus might be involved.

That was confirmed when Chinese investigators isolated the SARS-CoV-2 virus that causes COVID-19 on 7 January and made the gene sequence available globally a couple of days later. Knowing the viral RNA sequence enabled those who already had appropriate platform technologies to start right away with the job of making vaccines. It also allowed Mike Catton and Julian Druce of The Royal Melbourne Hospitals Victorian Infectious Disease Reference Laboratory (VIDRL) that is part of our Institute to add a sensitive PCR (polymerase chain reaction) test specific for SARS-CoV-2 to the more broadly reactive assay established earlier to detect the SARS-CoV-1 and MERS viruses, neither of which caused disease in Australia. Variants of the SARS-CoV-2 PCR are the tests that, as we all understand, have allowed the identification of currently infected individuals that facilitates targeted control strategies.

Arriving in Melbourne from China on 19 January, a returned resident very responsibly alerted authorities several days later to the fact that he was symptomatic and could be infected with this new virus. The diagnosis was immediately confirmed by PCR and, by Australia Day (26 January), Mike, Julian and teams had recovered infectious SARS-CoV-2 from him and announced that that the virus would be made available globally to legitimate laboratories. That was important: having fully infectious virus meant that researchers and diagnosticians could, for example, do virus neutralisation tests to measure protective antibodies in serum, screen chemical compounds (small molecules) for possible antiviral effects in tissue culture and challenge immunised animals to test for vaccine efficacy.

So, here are a couple of very big lessons that those of us who work in science understood but some in government and many in the broader community may not have fully appreciated.

The international co-operation that is essential for all science, and especially for public health science, functioned for COVID-19 from the outset and has, in fact, continued to work. The WHO did its job of alerting responsible individuals and agencies to the fact that there could be a problem, the Chinese gave out the gene sequence as soon as they had it, and our Institute was the first to provide the infectious virus globally for key laboratory tests. Locally, the communication mechanisms between public health professionals and elected officials proved fit for purpose. The Australian government was immediately aware of the potential threat and the Minister for Health, Greg Hunt, made a public announcement on 25 January that we had recorded our index case.

Additional to that, the very big lesson we should all take on board here is that modern science protects and serves us. Though everyone understood that the catastrophic influenza pandemic of 1918/19 was caused by a virus, diagnosis back then was all symptomatic, no human influenza virus was isolated until 1933 and it was only during World War 2 (1939-45) that the first, primitive influenza vaccines were rolled out to protect the troops against the possibility of a repeat pandemic that, thankfully, did not occur. When it comes to SARS-CoV-2 and COVID-19 we had a specific diagnostic test within days and, I will personally be very surprised if large-scale human vaccination is not in full swing by the second half of 2021.

Even so, the big lesson for the public is that, no matter how wonderful the laboratory science, actually getting products out there to protect people is a much more cumbersome process. Ensuring that a novel drug or vaccine is safe and efficacious takes time. Even though regulatory authorities have been comfortable with the idea that preliminary trials in animals and small numbers of human volunteers (Phase 1) can be conducted simultaneously, all that information must be evaluated before any product can be given to substantial numbers of people. Every possible effort is being made to ensure that all participants in large, closely monitored Phase 2 then Phase 3 trials will be protected, or at least safe, following community exposure to SARS-CoV-2.

Much of what had to be done over this first six months of the COVID-19 challenge was just plain hard work. An enormous effort was, for example, made within VIDRL to build testing capacity by helping other private and public laboratories get up to speed. And the Institute is still in the process of evaluating rapid person-side antibody tests that can be used for large-scale serological surveys. The obvious lesson here is that we are protected by having well-funded, high quality public laboratories and Institutions that can rapidly build capacity in the face of any pandemic threat. Next week, Ill finish my assessment of the lessons weve learned so far, to return to that six months from now.

This article is the latest in theSetting it Straightseries written by Laureate ProfessorPeter Dohertyfrom Australias University of Melbourne and Doherty Institute to explain aspects of the evolving COVID-19 pandemic. You can read them allhere.

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COVID-19: The lessons learned to date - Cosmos

NEW YORK and ROME, July 13, 2020 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID, hereinafter Ovid), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, and Angelini Pharma S.p.A. (hereinafter Angelini Pharma), an Italian family-owned pharmaceutical company committed to helping patients with a constant and prevalent focus on Mental Health, Rare Diseases and Consumer Health, announced an agreement in which Angelini Pharma will be responsible to develop, manufacture and commercialize OV101 (gaboxadol) for the potential treatment of Angelman syndrome in the European Union and other countries in the European Economic Area (Switzerland, Turkey and the United Kingdom) and Russia. Angelini Pharma will execute the agreement through its new affiliate Angelini Pharma Rare Diseases AG. OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and is currently being evaluated in the pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020.

Under the terms of the agreement, Ovid will receive an upfront payment of $20 million and is eligible to receive up to an additional $212.5 million in payments upon the achievement of development, manufacturing and sales milestones for the initial indication (Angelman syndrome), as well as double-digit royalties on net sales if OV101 is successfully commercialized. Ovid will retain all U.S. and rest-of-world commercial rights to OV101.

We are excited to enter into a strategic collaboration with Angelini Pharma with the goal of bringing OV101, if approved, to the Angelman community in Europe as quickly as possible. Angelini Pharma is an ideal partner for Europe as they have deep regional knowledge, an established infrastructure with a history of successful product launches, and a commitment to improving the quality of life of the patient communities they serve, said Jeremy Levin, DPhil, MB, BChir, Chairman and Chief Executive Officer of Ovid Therapeutics. Finding the right partners to bring OV101 to the Angelman community as rapidly as possible is a core part of our global strategy. We believe this partnership with Angelini will help to maximize the potential commercial value of OV101 and achieve our strategic objectives in this important geography."

Today is a day that we will remember. Through our collaboration with Ovid Therapeutics, we are laying the foundation to developing innovative health solutions for rare diseases, in line with Angelini Pharmas new strategy, said Pierluigi Antonelli, Angelini Pharma CEO. The new business unit Angelini Pharma Rare Diseases AG will contribute to the development, registration, production and, if approved, commercialization in Europe of OV101, Ovid Therapeutics very promising drug being evaluated in a Phase 3 clinical trial for the treatment of Angelman syndrome. As of now, there is no effective treatment for this rare genetic disease, characterized by severe psychomotor disability, which manifests itself from childhood. Delivering on our commitment makes us proud both from a scientific and social impact perspectives.

"As shareholders and executives of Angelini Holding we continue to invest in the pharma area, which today represents half of our Group's turnover, commented the executive vice president Thea Paola Angelini and the CEO Sergio Marullo di Condojanni. Our global development and internationalization strategy focuses on business areas with high growth potential. Particularly, we look closely at all the opportunities that can open up, not only in healthcare, but also in the consumer and machinery sector."

Rothschild & Co acted as an advisor to Ovid on the collaboration agreement.

About Angelman Syndrome Angelman syndrome is a rare genetic condition that is characterized by a variety of signs and symptoms. Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently. Therefore, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people globally.

There are no approved therapies by the U.S. Food and Drug Administration (FDA), European Medicines Agency or restof-world for Angelman syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta ()-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About OV101 (gaboxadol)OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has demonstrated in laboratory studies and animal models to selectively activate the -subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through modulation of tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the -subunit of GABAA receptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in more than 4,000 patients (more than 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE) and has completed a Phase 2 signal-finding clinical trial with OV101 in Fragile X syndrome (ROCKET).

OV101 has received Rare Pediatric Disease Designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, the European Commission (EC) has granted orphan drug designation to OV101 for the treatment of Angelman syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions.

Ovid Conference Call and Webcast InformationOvid Therapeutics will host a live conference call and webcast today at 8:15 a.m. Eastern Time. The live webcast can be accessed by visiting the Investors section of the Companys website at https://investors.ovidrx.com/news-events/presentations-events. Alternatively, please call 866-830-1640 (U.S.) or 210-874-7820 (international) to listen to the live conference call. The conference ID number for the live call is 5579257. A replay of the webcast will be available on the Companys website following the live conference call.

About Ovid TherapeuticsOvid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The Companys most advanced investigational medicine, OV101 (gaboxadol), is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE). For more information on Ovid, please visit http://www.ovidrx.com.

About Angelini PharmaAngelini Pharma, owned by Angelini Holding, is a pharmaceutical Company committed to helping patients with a constant and prevalent focus on Mental Health, including Pain, Rare Diseases and Consumer Health. Angelini Pharma has an extensive and recognized R&D programs, "World Class" production plants and international commercialization activities of active ingredients and market-leading drugs. For further information, please visit http://www.angelinipharma.com

About Angelini HoldingAngelini Holding is the parent company of an international group operating in the pharmaceutical and consumer goods sectors. Founded in Italy in 1919, today Angelini group operates in 17 countries with a staff of 5,600 and a turnover of 1,7 billion. In addition to the Pharmaceutical sector, Angelini group operates in Personal and Home Care business area through Fater, a joint venture with Procter & Gamble, in the Machinery field, again in joint venture with P&G, with the group operating in automation and robotics for the consumer goods industry Fameccanica, in Perfumery and Skincare and Suncare with Angelini Beauty and in the Wine sector through Bertani Domains. Angelini Holding has recently entered the Baby food market as well through MadreNatura, a joint venture with Hero Group, which offers 100% organic baby food products.

Ovid Therapeutics Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding: advancing development of and commercializing OV101, the potential benefits and value of OV101; the anticipated reporting schedule of clinical data for OV101; and the potential benefits and outcome from this collaboration. You can identify forward-looking statements because they contain words such as will, appears, believes and expects. Forward-looking statements are based on Ovids current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in the development and regulatory approval processes, and the fact that initial data from clinical trials may not be indicative, and are not guarantees, of the final results of the clinical trials and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovids filings with the Securities and Exchange Commission under the caption Risk Factors. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovids business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Ovid Therapeutics Contacts

Investors and Media:Ovid Therapeutics Inc.Investor Relations & Public Relationsirpr@ovidrx.com

Or

Investors: Steve KlassBurns McClellan, Inc.sklass@burnsmc.com(212) 213-0006

Media:Katie Engleman1ABkatie@1abmedia.com

Angelini Pharma Contact:Daniela PoggioHead of Global Communications Angelini Pharma+39 348 6558882daniela.poggio@angelinipharma.com

Angelini Holding Contact:Institutional & External Relations Director Angelini Holding+39 348 6707240alessandra.favilli@angeliniholding.com

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Ovid Therapeutics and Angelini Pharma Enter into Exclusive License Agreement to Develop, Manufacture and Commercialize OV101 for the Treatment of...

The global market for DNA read, write and edit applications should grow from $17.0 billion in 2019 to $43.1 billion by 2024 with a compound annual growth rate (CAGR) of 20.5% during the period of 2019-2024.

Report Scope:

The scope of the report includes DNA read, write and edit technologies, applications, industries, initiatives, patents and companies. The markets for read, write and edit products and services are given for 2017, 2018, 2019 (estimated) and 2024 (forecast).

This report reviews the main read, write and edit technologies and explains why genetic variation is important in clinical testing and disease. It then discusses significant large-scale research initiatives that impact read, write and edit applications. Of particular interest is a discussion of population-scale sequencing projects throughout the world, and their likely impact. The main market driving forces for read, write and edit products and services are listed and discussed.

Request For Report sample @ https://www.trendsmarketresearch.com/report/sample/12558

The report quantifies each of the main market segments. The read (sequencing) market is quantified by delivered format, including sequencing workflow products (sample preparation kits and reagents, sequencing instruments and consumables, and informatics) and sequencing services (clinical diagnostics and sequencing services to applied market customers).

The sequencing workflow products market is quantified by type, that is, DNA isolation and extraction; target enrichment; library preparation; and informatics/ecosystems. The sequencing instruments and consumables market is given by platform (Sanger, NGS and 3GS).

The sequencing services market is analyzed by end user application (applied, clinical, and R&D). Within sequencing services, the applied market is analyzed by end-user application (agriculture, biopharma, consumer, microbiology, population-scale genomics, synthetic biology and other).

Also within sequencing services, the clinical market is analyzed and quantified by disease category (cardiovascular, clinical microbiology and infectious diseases, Mendelian disorders, metabolic/immune disorders, neurology, oncology, reproductive health and transplant medicine).

The DNA write (synthesis) market is quantified by product type (oligonucleotides, synthetic biology parts, genes and RNA therapeutics). The oligonucleotide market is analyzed by application (gene editing, sequencing, PCR, FISH, microarray, gene synthesis and other). The gene market is quantified by gene type (standardized, value-added). Finally, the RNA therapeutics market is quantified by platform (RNA interference, antisense oligos, micro RNA modulation and mRNA) and by disease category (cancer, hematology, musculoskeletal, neurology and rare diseases).

The DNA edit (gene editing) market is quantified by application (agriculture, biopharma, diagnostics and therapeutics); editing platform (CRISPR, meganuclease, TALEN, ZFN). The gene editing agriculture market is analyzed by product type (crop/seeds, livestock). The gene editing biotechnology market is analyzed by product type (kits and reagents, cell line engineering, animal models and services). The gene editing therapeutics market is analyzed by disease category (eye and rare diseases).

Specific geographic markets discussed include North America, Europe, Asia-Pacific, and the rest of the world (ROW).

Industry sectors analyzed include next-generation sequencing; long read sequencing; DNA synthesis; RNA therapies; and gene editing.

More than 320 companies in the read, write and edit industry are profiled in this report.

BCC Research also provides a summary of more than 180 of the main industry acquisitions and strategic alliances that took place from January 2018 through June 2019, including key alliance trends.

Report Includes:

28 data tables and 77 additional tables An overview of the global market for DNA read, write and edit technologies, applications, and industries Analyses of global market trends, with market data from 2017, 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 Discussion on sequencing technologies, market applications, industry structure, and important clinical sequencing initiatives Information pertaining to several significant large-scale research initiatives that are contributing to sequencing services, write synthesis and gene editing technologies market development A look at the innovations in pharmaceutical and biotechnology companies and research & development programs in stem cell-based therapies and gene therapies Coverage of significant patents and their allotments in each category, as well as major industry acquisitions and strategic alliances data Company profiles of over 320 major global players within the industry, including 3Billion Inc., 23Andme Inc., Bayer AG, Becton, Dickinson and Co. (BD), Bio-Rad Laboratories, Pacific Biosciences, Qiagen NV, Roche Holding AG and Thermo Fisher Scientific Inc.

Summary:

DNA (and RNA) read, write and editing includes the primary methods in which nucleic acids are analyzed (sequencing), created (synthesis) and modified (gene editing). It is becoming increasingly important in the life sciences industry to be expert in all aspects of nucleic acids in order to exploit significant opportunities within this industry. The end users for these technologies include any industry that works with biological systems, and even some that dont (e.g., DNA data storage).

The DNA read, write and edit industry is at the beginning stages of its growth story; penetration of the key markets is still at an early stage. For example, the cumulative number of human genomes sequenced reached REDACTED as of January 2019 (less than REDACTED of the global human population); we estimate that population-scale projects alone will increase that figure to more than REDACTED genomes sequenced during the next five years. The percentage of non-human species sequenced as of January 2019 was less than REDACTED of all species. These data indicate that there is significant future upside for sequencing across research, metagenomics, agriculture, synthetic biology and clinical applications,among others.

The situation is similar for DNA writing and editing technologies, with clinical therapeutic applications in particular providing an enormous total available future market that is yet to be significantly penetrated. Major successes in this industry include the adoption of next-generation sequencing (NGS) for noninvasive prenatal testing; enabling the roles of synthetic DNA oligonucleotides and genes in the rise of the synthetic biology industry; and rapid adoption of CRISPR gene editing by research institutions and biopharma industries.

There is increasing interplay among the three DNA technology platforms, giving rise to innovative corporate strategies. For example, Arbor Biotechnologies employs sequencing, gene synthesis and artificial intelligence to perform high-throughput discovery of biomolecules, including new CRISPR proteins.

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DNA Read, Write And Edit Market Sales Volume, Status, Growth, Opportunities and World the COVID-19 - 3rd Watch News

Researchers in the Jacobs School of Medicine and Biomedical Sciencescontinue to spearhead a number of projects related to the COVID-19 global health pandemic.

Peter L. Elkin, professor and chair of biomedical informatics, says several current studies are focused on data collection that can be used to better understand how to combat COVID-19.

Much of the work is being completed through the Clinical and Translational Science Awards (CTSA) consortium, of which UB is a member. It is one of more than 50 medical research institutions across the nation currently receiving CTSA program funding from the National Institutes of Health.

One such project is the launch of the National COVID Cohort Collaborative (N3C), a joint program between the National Center for Data to Health and the National Center for Advancing Translational Sciences.

Elkin says the projects aim is to build a warehouse of COVID-19 data for the entire CTSA consortium and for otherinterested contributing health care organizations.

This is intended to hold all patient data (inpatient and outpatient) on COVID-tested patients from all of the CTSA hubs, he says. It entails a cloud-based method for data collection on the COVID-19 pandemic.

We are working closely with N3C to see how this can be designed and implemented in astandardized and timely fashion.

The goal of developing a national-level COVID-19 database is to facilitate research and improve recruitment to clinical trials, he says.

N3C is looking to address the many difficult questions raised by the COVID-19 global emergency, such as:

UB is also a member of COMBATCOVID, a New York State initiative to save case report formson all hospital admissions for upper respiratory infections,including all patients tested for COVID-19 or patients who are suspected to have COVID-19.

The statewide consortium will collect and analyze the results from all the CTSA institutions in the state.

It is being run out of New York University, and I am participating from our site as our CTSA informatics core director, Elkin says. I am working on the design and data governance.

The data use agreements are being signed, and the database design and data definitions are being built, he adds. This larger row-level dataset will allow us to ask questions that would notbe possible at any one institution.

In UBs Department of Biomedical Informatics, Elkin and Frank D. LeHouillier, senior programmer and analyst, are involved in the project.

Clinical researchers in the Jacobs School who are involved include:

Researchers in the Department of Biomedical Informatics have also developed a validated microbiome platform that finds infected persons with COVID-19 whether symptomatic or not using deep sequencing of stool microbiome samples.

Elkin is working with postdoctoral associate Sapan Mandloi in using a National Center for Biotechnology Information (NCBI) Sequence Read Archive (SRA) database to collect and process metagenomics data for the organism classified as human gut metagenome.

The more than 300,000 samples are divided into 3,464 projects, according to Mandloi.

We are performing comparison of all samples raw sequences with SARS-Cov-2 genome using a NCBI SRA Taxonomy Analysis Tool (STAT), which utilizes precomputed k-mer dictionary databases and gene-specific profiling, Mandloi says. This allows us to perform geographic mapping of samples identified across the world.

Some 9,720 samples were identified as potential cases of colonization for COVID-19, which were mostly from the U.S., China, Australia and the U.K., he adds.

The ability to identify and track this trafficking of genetic material is vital as a public health topic, he says. As of now, this large pool of genetic data remains largely untapped for clinical surveillance using the combined strategy of gene-based profiling and k-mer-based classification on raw genomic data.

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Jacobs School researchers collecting COVID-19 data - UB Now: News and views for UB faculty and staff - University at Buffalo Reporter