Category Archives: Molecular Genetics

Special to The Sentinel Published 12:02 a.m. ET Oct. 16, 2019

Denison University's Goodspeed Lecture Series welcomes Terence Keel, associate professor with a split appointment in the Department of African American Studies, and the UCLA Institute for Society and Genetics presenting, "Christianity, Race, and the Haunting of the Biomedical Sciences" at 7 p.m. on Tuesday, Oct. 22, in the Auditorium of Denison Universitys Slayter Hall (200 Ridge Road).

The event is free and open to the public. For more information, contact Jodi Weibel at 740-587-6241 or visit Denison.edu.

The idea that so-called races reflect inherent biological differences between social groups has been a prominent aspect of Western thought since at least the Enlightenment, according to a DU news release. While there have been moments of refuting this way of thinking most notably, the social constructionist thesis emerging as a dominant framework in the aftermath of WWII fixed biological conceptions of race haunt new genetic technologies, where race is thought to be measurable at the molecular level.

Keel argues that the resilience of this naturalized understanding of race may stem less from overtly political motives on the part of scientists and more from our inherited theological traditions that continue to shape and limit the intellectual horizon of scientific reasoning.

Keel is a critical race theorist, historian of science, and scholar of religion who has written widely about American biomedical science, religion, law, and modern thought.

His first book, "Divine Variations" explained how Christian thought made possible the development of the race concept in Euro-American science while also shaping the moral and epistemic commitments embedded in the study of human biology. Keel is an affiliate of the newly formed Center for the Study of Racism, Social Justice and Health at the UCLA Fielding School of Public Health.

He is also a senior advisor to the Goldin Institute, a Chicago based non-profit organization that advocates globally for grassroots leadership, conflict resolution, poverty alleviation and environmental sustainability.

Keel's visit is sponsored by the Department of Religion, Black Studies, and the Faculty Research Table on Race Science: Histories, Legacies, Continuities.

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Denison presents: 'Christianity, Race, and the Haunting of the Biomedical Sciences' - The Newark Advocate

Successful validation of Saphyr to replace gold standard method of southern blotting for molecular diagnosis of FSHD supports broad utility of Saphyr

SAN DIEGO, Oct. 16, 2019 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO), a life sciences instrumentation company that develops and markets the Saphyr system, a genome imaging platform for ultra-sensitive and ultra-specific genome-wide structural variation detection, today announced that leading organizations, including PerkinElmer Genomics and the University of Iowa, have adopted Saphyr for use in their clinical genomics laboratories. PerkinElmer Genomics and the University of Iowa have developed assays based on the Bionano optical mapping technology to expand their comprehensive suite of genetic tests assessing disease-associated chromosomal abnormalities. Their lead indication is Facioscapulohumeral Muscular Dystrophy (FSHD).

FSHD is one of the most prevalent forms of muscular dystrophy and affects approximately 1 in 10,000 individuals. It is caused by changes in the number of repeats in a section of chromosome 4. To correctly diagnose FSHD, an exact count of the repeat number is necessary. To date, molecular diagnoses for FSHD are generated using outdated Southern Blot techniques, which are imprecise, labor intensive and involve radioactive labeling methods which are being phased out of laboratory use for safety reasons. In contrast, the assays developed by PerkinElmer Genomics and the University of Iowawith the Bionano EnFocus FSHD Analysis tool are reproducible, safe, fast, and automated with minimal hands-on time. These assays provide an exact repeat number for the pathogenic and non-pathogenic variants, give a high-resolution view of the repeat regions and have a high sensitivity to mosaicism.

Jamshid Arjomand, Ph.D., CSO of the FSHD Society, the leading research-focused patient organization forFSHD, said, The FSHD community has been waiting years for an accessible and robust assay like this. The lack of timely and affordable genetic testing has been a major hurdle for the FSHD community. Thousands of patients have never received a molecular diagnosis, which limits successful recruitment into the increasing number of clinical research and clinical trial studies for this devastating disease. We are delighted that Bionanos Saphyr system enables a more precise and higher throughput method for FSHD genetic testing and are grateful to diagnostic groups and companies that are making genetic testing more accessible to our families.

We are pleased to be the first US laboratory to develop and validate an assay based on the Bionano Saphyr system in a clinical setting under CLIA/CAP guidelines" stated Madhuri Hegde, Ph.D., FACMG, Vice President and CSO of PerkinElmer Genomics. "We are committed to helping patients and families that need genetic testing and are excited about the strong clinical utility of this assay for the molecular assessment of FSHD patients."

Erik Holmlin, Ph.D., CEO of Bionano, commented, We have always believed that Bionanos unique ability to image long, intact DNA molecules could enable the Saphyr system users to develop assays in a clinical setting to modernize and streamline the practice of cytogenetics. Our teams have worked tirelessly to improve the speed, quality, throughput, and robustness of the optical mapping application of genome imaging while simultaneously reducing cost, assay complexity and data analysis. We believe Saphyr is ready to be adopted for assay development in a routine clinical workflow, and we are thrilled that PerkinElmer Genomics and the University of Iowa are taking the lead in making the Saphyr system a tool for next-generation cytogenomics, with many other academic, CRO and reference laboratories expected to follow. We believe that FSHD is just the start of a wide array of clinical genetics assays that labs will develop with our technology.

Results of the PerkinElmer Genomics FSHD evaluation study using the Saphyr system will be presented by Alka Chaubey, Ph.D., FACMG, Head of Cytogenomics and Laboratory Director at PerkinElmer Genomics at the Bionano Genomics ASHG exhibitor workshop on Thursday, Oct. 17, 2019 from 12:45 pm 2:00 pm at the Houston Marriott Marquis. More information about the workshop can be found online, and a recording will be made available on Bionanos website.

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Bionano will showcase the Bionano EnFocus FSHD Analysis tool for fast, streamlinedbioinformaticsassessment of theFSHD locusfrom genome-wide optical mapping data at booth #527 during the annualAmerican Society of Human Genetics Annual Meeting, Oct. 15-19, 2019.

About Bionano Genomics

Bionano is a life sciences instrumentation company in the genome analysis space. Bionano develops and markets the Saphyr system, a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets andto establish digital cytogenetics, which is designed to be a more systematic, streamlined and industrialized form of traditional cytogenetics. The Saphyr system comprises an instrument, chip consumables, reagents and a suite of data analysis tools. More information about Bionano Genomics is available at http://www.bionanogenomics.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, including, among other things: conclusions as to Saphyrs potential as a powerful new tool in cytogenetics; Saphyrs potential contribution to improvements in traditional cytogenetics; the University of Iowas or PerkinElmer Genomics plans to develop additional assays using our technology; our beliefs regarding the Saphyr systems readiness for clinical adoption andour expectations regarding adoption by other academic, CRO and reference laboratories using our technology; PerkinElmer Genomics commercial plans; plans of other Saphyr system users to implement their own assays for FSHD and other genetic disorders; and certain planned presentations by PerkinElmer Genomics and us. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks that our sales, revenue, expense and other financial guidance may not be as expected, as well as risks and uncertainties associated with general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of key clinical studies to demonstrate the effectiveness of our products; the loss of key members of management and our commercial team; and the risks and uncertainties associated with our business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2018 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

Contacts

Company Contact:Mike Ward, CFOBionano Genomics, Inc.+1 (858) 888-7600mward@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617)775-5956arr@lifesciadvisors.com

Media Contact:Kirsten ThomasThe Ruth Group+1 (508) 280-6592kthomas@theruthgroup.com

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Bionano Genomics Announces Adoption of Its Saphyr System by Clinical Cytogenetics Groups in Academia and Industry to Replace Traditional Methods for...

Findings to be presented cover broad range of scientifically and clinically relevant areas including schizophrenia, sex development, cancer and muscular dystrophy

SAN DIEGO, Oct. 16, 2019 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO) today announced that disease researchers using Bionanos Saphyr system for whole genome imaging will present their results at the American Society of Human Genetics (ASHG) Annual Meeting, between October 15-19 in Austin, Texas.

The impact of analysis using the Saphyr system for ultra-sensitive and ultra-specific genome-wide detection of structural variation will be presented at ASHG with 22 oral and poster presentations and an Educational Event hosted by Bionano.

ASHG 2019 represents a milestone for Bionano, with a record number of presentations demonstrating novel discoveries through our genome mapping technology, said Erik Holmlin, Ph.D., CEO of Bionano. The growing use of the Saphyr system in disease research illustrates the value in identifying genomic variations for deep understanding of disease origin and diagnostic development.

Optical mapping through Saphyr enables the direct observation of large genomic variations through imaging of fluorescently labeled, megabase-size native DNA molecules. Next-generation sequencing (NGS), in contrast, relies on short-reads that piece together sequence fragments in an attempt to rebuild the actual structure of the genome. NGS often misses large DNA variations, such as deletions, insertions, duplications, and translocations and inversions. Genome mapping resolves these structural variations for more insight into the genetic variations that cause disease.

Below is a summary of key presentations to be given at ASHG 2019 featuring the use of optical genome mapping:

Genetic diagnosis of sex development disorders through optical mappingHalf of disorders of sex development (DSD) patients lack a firm diagnosis. Prof. Eric Vilain, from George Washington University and Childrens National Medical Center, will present research validating the diagnostic and gene discovery use of Bionano genome mapping to identify structural variants in patients with DSD. The talk, entitled Integration of optical genome mapping and sequencing technologies for identification of structural variants in DSD, will be presented on Wed. Oct. 16 at 5:15 - 5:30 pm in the convention center Level 3, Room 361D.

Genomic mapping has the potential to replace a combination of current cytogenetic techniquesCurrently, a comprehensive clinical analysis of genomic aberrations requires a combination of various assays such as CNV-microarrays, karyotyping and fluorescence in situ hybridization (FISH). Dr. Tuomo Mantere, from Radboud University Medical Center, will present data directly comparing traditional cytogenetic assays with Bionano mapping in leukemia patient samples to illustrate that genome mapping can identify all aberrations found by the three conventional technologies combined, and additional variants as well. The poster, entitled Next-generation cytogenetics: High-resolution optical mapping to replace FISH, karyotyping and CNV-microarrays will be presented on Thurs. Oct. 17, between 2 - 3pm, PgmNr 2533/T.

Genomic architecture reveals critical factors that may contribute to schizophrenia-associated 3q29 chromosomal deletionDeletions at the 3q29 chromosomal locus are associated with a 40-fold increase in risk for schizophrenia. Knowing the features that contribute to genomic instability is critical for identifying risk factors of chromosomal deletions. Trenel Mosley, from Emory University, will present the discovery of novel genomic structural characteristics found in 12 patients with 3q29 deletion and their parents using Saphyr. The poster entitled, Optical mapping of the schizophrenia-associated 3q29 deletion reveals new features of genomic architecture, will be presented on Wed. Oct. 16, between 2 - 3pm, PgmNr 1389/W.

Bionano and NGS resolve complex rearrangements in extrachromosomal, circular DNA in glioblastoma The rapid growth of aggressive tumors such as glioblastoma is partially caused by the rapid amplification of oncogenes in circular structures outside of native chromosomes. Because these structures do not occur in the reference genome, standard analysis methods fail to correctly assemble them. Jens Luebeck, from the University of California, San Diego, demonstrates that a combination of Bionano genome mapping and NGS resolves important breakpoints and gene amplifications in extrachromosomal DNA. The talk, entitled Integrated Analysis of NGS and Optical Mapping Resolves the Complex Structure of Highly Rearranged Focal Amplifications in Cancer, will be presented on Sat. Oct. 19, from 10:15 - 10:30am PgmNr: 323

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Bionano Educational Event will feature research on muscular dystrophy, prenatal development & neurodegenerative disordersAt Bionanos educational event, Dr. Alka Chaubey from Perkin Elmer Genomics, Dr. Frances High from Mass General Hospital for Children, and Dr. Mark Ebbert from the Mayo Clinic will present findings from their work using the Saphyr system for structural genomic resolution. Analysis of chromosomal repeats, complex genomic haplotypes, and risk loci found in genetic disease will be highlighted by the speakers. Entitled Resolving Structural Variants Across the Whole Genome to Power Your Next Discovery in Human Genetics, the event will take place on Thurs. Oct 17, from 12:45 - 2:00pm at the Marriott Marquis, Houston, River Oaks, Level 3, and include a complimentary lunch.

Additional presentations featuring optical genome mapping:

High Throughput Analysis of Tandem Repeat Contraction Associated with Facioscapulohumeral Muscular Dystrophy (FSHD) by Optical MappingPresented by Jian Wang, Bionano GenomicsWed. Oct. 16, 2 - 3pm PgmNr: 2535/W

Full Genome Analysis for Identification of Single Nucleotide and Structural Variants in Genes that Cause Developmental DelayPresented by Hsiao-Jung Kao, Academia SINICAWed. Oct. 16, 2 - 3pm PgmNr: 2547/W

A Robust Benchmark for Germline Structural Variant DetectionPresented by Justin Zook, National Institute of Standards and TechnologyWed. Oct. 16, 2 - 3pm PgmNr: 1695/W

De Novo Genome Assembly and Phasing for Undiagnosed ConditionsPresented by Joseph Shieh, University of California, San FranciscoWed. Oct. 16, 2 -3 pm PgmNr: 2529/W

Bionano Prep SP Isolates High Quality Ultra-high Molecular Weight (UHMW) Genomic DNA to Improve Research of Cancer and Undiagnosed DisordersPresented by Henry Sadowski, Bionano GenomicsWed. Oct. 16, 3 - 4pm PgmNr: 2598/W

nanotatoR: An Annotation Tool for Genomic Structural VariantsPresented by Surajit Bhattacharya, Childrens National Medical CenterWed. Oct. 16, 3 - 4pm PgmNr: 1506/W

Detection, Characterization, and Breakpoint Refinement of Balanced Rearrangements by Optical Mapping in Clinical CasesPresented by Alex Hastie, Bionano Genomics + LabCorpThurs. Oct. 17, 2 - 3pm PgmNr: 2569/T

Genetic/epigenetic Diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD) via Optical MappingPresented by Yi-Wen Chen, Childrens National Medical CenterThurs. Oct. 17, 2 - 3pm PgmNr: 2533/T

Comprehensive Analysis of Structural Variants in Clinical Cancer SamplesPresented by Ernest Lam, Bionano GenomicsThurs. Oct. 17, 3 - 4pm PgmNr: 1060/T

Advanced Structural Analysis of CDH Risk Loci with Optical Genome Mapping TechnologyPresented by Mauro Longoni, Massachusetts General HospitalThurs. Oct. 17, 3 - 4pm PgmNr: 2578/T

Structural Variants Associated with GWAS SNPs Provide Mechanistic Explanation of Phenotypic AssociationsPresented by Seth Berger, Childrens National Medical CenterThurs. Oct. 17, 3 - 4pm PgmNr: 2254/T

The Complete Linear Assembly and Methylation Map of Human Chromosome 8Presented by Glennis Logsdon, University of WashingtonFri. Oct. 18, 1 - 2pm PgmNr: 1703/F

High Throughput High Molecular Weight DNA Extraction from Human Tissues for Long-read SequencingPresented by Kelvin Liu, CirculomicsFri. Oct. 18, 1 - 2pm PgmNr: 1769/F

Optical Mapping for Chromosomal Abnormalities: A Pilot Feasibility Study for Clinical UsePresented by Gokce Toruner, UT MD Anderson Cancer CenterFri. Oct. 18, 1 - 2pm PgmNr: 2447/F

Comprehensive Detection of Germline and Somatic Structural Mutation in Cancer Genomes by Bionano Genomics Optical MappingPresented by Mark Ebbert, Mayo ClinicFri. Oct. 18, 2 - 3pm PgmNr: 1760/F

Dark and Camouflaged Genes May Harbor Disease-relevant Variants that Long-read Sequencing Can ResolvePresented by Andy Pang, Bionano GenomicsFri. Oct. 18, 2 - 3pm PgmNr: 1814/F

Bionano Genomics Sample to Answer Workflow for Single Molecule Analysis of Variation in Genome StructurePresented by Sven Bocklandt, Bionano GenomicsFri. Oct. 18, 2 - 3pm PgmNr: 1838/F

Draft Assembly of an Armenian GenomePresented by Hayk Barseghyan, Childrens National Medical CenterFri. Oct. 18, 2 - 3pm PgmNr: 2342/F

About Bionano GenomicsBionano is a life sciences instrumentation company in the genome analysis space. Bionano develops and markets the Saphyr system, a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline digital cytogenetics, which is designed to be a more systematic, streamlined and industrialized form of traditional cytogenetics. The Saphyr system comprises an instrument, chip consumables, reagents and a suite of data analysis tools. For more information, visit http://www.bionanogenomics.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, including among other things: the timing and content of the presentations identified in this press release; and the ability of genome mapping to perform comprehensive clinical analysis as well as conventional technologies. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks that our sales, revenue, expense and other financial guidance may not be as expected, as well as risks and uncertainties associated with general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of key clinical studies to demonstrate the effectiveness of our products; the loss of key members of management and our commercial team; and the risks and uncertainties associated with our business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2018 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

ContactsCompany Contact:Mike Ward, CFOBionano Genomics, Inc.+1 (858) 888-7600mward@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617) 775-5956arr@lifesciadvisors.com

Media Contact:Kirsten ThomasThe Ruth Group+1 (508) 280-6592kthomas@theruthgroup.com

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Top Researchers to Present Discoveries Made Possible by Bionanos Saphyr System for Genome Imaging Technology at the ASHG 2019 Annual Meeting - Yahoo...

HOUSTON--(BUSINESS WIRE)--Quantabio, a leading provider of robust DNA and RNA amplification reagents for the most demanding molecular testing and life science research applications, today announced its lineup of podium and poster presentations at the American Society of Human Genetics (ASHG) Annual Meeting in Houston this week. During the meeting, Quantabio will showcase the ultra-fast data acquisition capabilities and unrivaled performance of the Q cycler, along with its entire line of sample prep, PCR, RT-PCR, qPCR, and NGS reagents at booth #1626.

The Quantabio team will demonstrate how the companys technology is continuing to accelerate PCR, qPCR and next-generation sequencing (NGS) run times, performance and affordability. The company will showcase the following new products:

Co-Lab Scientific PresentationSpeeding up Genomic Discoveries through Innovation: New Fast Library Quant (NGS) and High Fidelity and Long-Range Amplification (PCR)

WHEN: Friday, Oct. 18th at 12:45 1:30 pmWHERE: CoLab #3; Booth 1209FOR WHOM: All ASHG attendees are welcome to join. Seating is limitedFEATURED PRESENTER: David Schuster, Senior Director of Research and Development at Quantabio

Scientific PosterRapid and accurate quantification of Illumina NGS libraries using sparQ Fast Library Quant Kit on the Q real-time qPCR instrumentWHEN: Thursday, Oct. 17th at 3:00 4:00 pmWHERE: 1840T

About QuantabioQuantabio is a leading provider of advanced DNA and RNA amplification reagents for the most demanding molecular testing applications in applied, translational and life science research. The Quantabio team leverages decades of experience in developing pioneering amplification technologies to deliver cutting-edge products to researchers focused on critical PCR, qPCR and Next-Generation Sequencing (NGS) based applications. Based in Beverly, Mass., Quantabio offers a growing portfolio of products through its international sales operations, as well as a global network of distributors and commercial service providers. For more information, please visit http://www.quantabio.com.

The Quantabio products are for Research Use Only; not for use in diagnostic procedures.

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Quantabio to Showcase Industry's Fastest qPCR-based NGS Library Quantification Kit and HiFi PCR Mix at American Society of Human Genetics Annual...

The Global Veterinary Molecular Diagnostics Market is expected to rise from its initial estimated value of USD 453.83 million in 2018 to an estimated value of USD 870.34 million by 2026 registering a CAGR of 8.48% in the forecast period of 2019-2026. Molecular diagnostic market is driven by Increasing incidence of numerous animal diseases and by increasing expenditure on pet animal.

Global Veterinary Molecular Diagnostics Market By Technology (PCR kits, INAAT kits, Microarray kits, DNA sequencing kits), Product (Instruments & Software, Kits & Reagents, Services), , Animal Type (Companion Animals, Livestock Animals), Disease Indication (Vector-borne Diseases, Respiratory Pathogens Detection, Diarrhea Pathogens Detection, Others), Application (Infectious Diseases, Oncology, Genetics, Microbiology), End-User ( Veterinary Hospitals, Clinical Laboratories, Research Institutes),Geography (Europe, North America, Asia Pacific, South America and Middle East & Africa) Industry Trends & Forecast to 2026

Get Sample Copy of this Report @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-veterinary-molecular-diagnostics-market

Some of the major market competitors currently working in the veterinary molecular diagnostics market are IDEXX Laboratories, Inc., VCA, Inc., Abaxis, Heska Corporation , Zoetis , NEOGEN CORPORATION , Thermo Fisher Scientific, Inc., Virbac, General Electric Company, Agfa-Gevaert Group., Veterinary Molecular Diagnostics, Inc., Novacyt Group, Qiagen , Biomedica Medizinprodukte GmbH & Co KG, Veterinary Laboratory HealthGene Corp. , Bioneer, Abaxis, Neogen Corporatio.

Market Definition: Global Veterinary Molecular Diagnostics Market

The molecular diagnostic is a laboratory method for examining the RNA or DNA or other proteins in humans and animals which is used to identify diseases or the predisposition stage . The scope of this diagnostic comprises of the various clinical testing devices, supplies and reagents and which are used in hospitals, commercial laboratories, clinics, reference laboratories and research institutes for identifying disease indications for identifying and monitoring. Veterinary molecular diagnostics provides a number of benefits than the other traditional diagnostic techniques like fast shifting time (within 2.53 hours), high test specificity and sensitivity, and better distinction among the birulent and avirulant strains.

Market Drivers:

Market Restraints:

Download Detailed TOC @ https://www.databridgemarketresearch.com/toc/?dbmr=global-veterinary-molecular-diagnostics-market

Segmentation: Global Veterinary Molecular Diagnostics Market

By Technology

By Product

By Animal Type

By Disease Indication

By Application

By End-User

By Geography

Key Developments in the Market:

In Jan 2018, Mars Petcare(U.S.) acquired Genoscoper Laboratories(Finland), a specialist in molecular diagnostics for companion animals. This acquisition will help in accelerate discovery of genetic health markers for companion animals.

Competitive Analysis: Global Veterinary Molecular Diagnostics Market

The global veterinary molecular diagnostics market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of veterinary molecular diagnostics market for global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Customization of the Report:

Inquiry Before Buying @ https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-veterinary-molecular-diagnostics-market

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Veterinary Molecular Diagnostics Market to Reach at a CAGR of 8.48% by 2026 With NEOGEN CORPORATION , Thermo Fisher Scientific, Inc., Virbac, General...

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Researchers have found three new-to-science viruses in chinook and sockeye salmon in British Columbia. The discovery, led by Gideon Mordecai, a University of British Columbia molecular biologist who studies the ecology of viruses, is part of a larger investigation into whether viruses are contributing to the steep declines in wild British Columbia salmon populations over the past 30 years. The researchers now aim to find out if these infectious agents are being transmitted from farmed to wild fish. Whether farmed fish sicken wild fish is a key concern of fishers; local First Nations, for whom salmon is a critical part of their livelihoods and cultures; and people who worry about the fate of the struggling, chinook-eating southern resident killer whales.

Of the three new viruses, one, an arenavirus, was found in farmed, hatchery, and wild chinook and sockeye salmon. A nidovirus was found in farmed, hatchery, and wild chinook. And a reovirus was found only in farmed salmon.

Arenaviruses are known to primarily infect mammals, so the researchers were surprised to find one in salmon. Farmed fish with the arenavirus had anemia and damage to their gills, kidneys, spleens, swim bladders, and livers. The nidovirus is from a group that includes the viruses behind severe acute respiratory syndrome (SARS) and Middle East respiratory syndrome (MERS), which affect mammals lungs. This nidovirus was mostly found in the salmons gills. The reovirus is related to viruses that cause hemorrhagic diseases that have killed many fish in Chinese aquaculture.

The discoveries contribute to a growing understanding of the threats facing British Columbias wild salmon.

A decade ago, people thought the main causes of salmon population declines were development of land around rivers, which makes streams warmer and pushes sediment into rivers, and climate change, which warms the ocean and causes currents and prey species to move. But in 2011, Kristi Miller, head of molecular genetics at the Pacific Biological Station in Nanaimo, British Columbia, showed that infectious diseases may be contributing as well.

In 2013, Miller became the head of genomic research for the Strategic Salmon Health Initiative, a collaboration among Fisheries and Oceans Canada, and the nonprofits Pacific Salmon Foundation and Genome BC.* This new study is one of more than 30 the initiative has done to investigate the population declines.

In the project, Mordecai, Miller, and their colleagues first studied dead and dying farmed chinook and used DNA analyses to identify the previously unknown viruses.

They looked at the farmed fish first because it is difficult to find diseased fish in the wild. On fish farms, diseases are much more obvious because fish are routinely found dead or dying with abnormalities on their bodies. As with any type of factory farming, the unnatural, crowded conditions of fish farms foster disease. Sick wild fish dont usually die from their diseases, says Miller. As soon as their swim performance or visual acuity is affected, theyre eaten. This is a big part of why salmon viruses are understudied, says Mordecai.

But thanks to new tools, Mordecais team was able to find the viruses in wild salmon, which they had collected and tested in the lab. Although the wild fish were not yet showing evidence of disease, the researchers used molecular analyses to see if their immune responses were triggered by a virus. If researchers could not detect a known virus, they looked for new viruses using next-generation DNA sequencing that allowed them to map genomes quickly, searching much more broadly than earlier methods. This is so incredibly powerful; it essentially allows fish to talk to us, says scientist turned activist Alexandra Morton, who was not involved with this study.

Although the scientists found that the viruses were infecting the salmons cells, they have not proved that they cause diseases, says Mordecai. Although it might seem obvious to assume that dead farmed salmon died from disease, their deaths could be caused by something other than the viruses in question. Linking the new viruses to diseases is the next research step.

The scientists are also unsure if the viruses are being passed between farmed and wild fish. They tested chinook and sockeye salmon from different locations as a first step to understanding possible transmission. Then they will use genomic sequencing to see whether viruses found in different populations are the same. Tracking transmission is relatively easy to do with viruses because they mutate rapidly so you can follow the transmission pathway, says Miller.

As Mordecai puts it: If you infected me with a cold virus, we could look at the sequence of the virus in me and the virus in you and see theyre closely related. Whereas a virus someone picked up a six-hour drive away or in the UK might be different.

First Nations activists who are fighting fish farms in their territories feel certain that farmed fish are spreading disease to wild fish. Chief Ernest Alfred of the Namgis First Nation in Alert Bay occupied the Swanson Bay fish farm in protest for 280 days. He and other occupiers took daily photos of sick fish, including some that were deformed or yellowing, a sign of jaundice. There were also all sorts of skin disorders, there was blindness. Hes also seen wild salmon in British Columbias rivers and streams with similar symptoms, many of which die before spawning.

Despite recent studies highlighting the likelihood of transmission of another virusPiscine orthoreovirusfrom farmed to wild salmon, neither government nor industry has taken preventative action, such as testing farmed fish prior to moving them to ocean pens. In the current federal election campaign, the Liberal and Green parties announced a commitment to phase out ocean fish farms by 2025. But Alfred and Morton, who are following political developments closely, say they remain skeptical that government will get tough on industry to protect wild salmon.

Miller also questions whether election promises will translate into real change, but she is encouraged that public pressure is having an effect. As for whether that results in shifts in policies and regulations, we have to wait and see, she says.

Correction: Genome BC is a nonprofit, not a private company.

Original post:
New Viruses Found in Farmed and Wild Salmon - Hakai Magazine

Name: Sue Jinks-Robertson

Position: Professor, Vice Chair and Director of the Cell and Molecular Biology Program

Years at Duke: 13

What she does at Duke: Jinks-Robertson has many duties in the Department of Molecular Genetics and Microbiology. She oversees the Cell and Molecular Biology Training Program which features around 100 graduate students and involves around 130 faculty members. She also co-directs the Cancer Genetics and Genomics program at Duke Cancer Institute.

But Jinks-Robertson is most at home in her lab, where she studies the genetic makeup of yeast.

Her team examines yeast DNA, looking for the factors behind mutations or changes in sequence. This research is important is because the DNA of yeast is essentially the same that found in many other organisms, including humans.

If we understand how this works in yeast, we can get information about what can go wrong in humans, Jinks-Robertson said.

The research is of great value in the fight against cancer, since it can occur when cells with genetic flaws multiply. Therapies that help identify and repair these flaws can be critical in battling the disease.

The basis for the therapies comes from the very basic work done in the trenches with an organism like yeast, Jinks-Robertson said.

What she loves about Duke: When she arrived at Duke after two decades on the faculty at Emory University, Jinks-Robertson was struck by the affection and loyalty her new colleagues both staff and faculty had for the university.

Soon, she too came to have a similar relationship with the university. She said its hard to pin down one specific reason for her connection with Duke, but she knows its there.

I dont know if its some of the physical structures around, like the Gardens, the Chapel, theres a central focus, of course theres basketball, its hard to put your finger on what it is, Jinks-Robertson said. Its just a very nice place to work. You feel connected to something bigger than yourself.

A memorable day at work: This spring, Jinks-Robertson was preparing for a major grant application when she got a call from colleague Thomas Petes with exciting news.

Petes, the Minnie Gellar Professor of Molecular Genetics and Microbiology, told her that shed been elected to the National Academy of Sciences, a 156-year old organization comprised of the nations top scientific minds.

It was a big surprise, Jinks-Robertson said. If youre a scientist, at least in this country, its a great recognition.

Jinks-Robertson was one of two Duke scientists elected to the 2019 class. Susan Alberts, the Robert F. Durden Professor of Biology, also earned election to the academy.

The nicest part of it was that I was hit with a flood of emails and phone calls, it was really wonderful, Jinks-Robertson said.

Special object/memorabilia in her workspace: On a shelf in her office, Jinks-Robertson has a collection of gifts given to her by former students who came to Duke from other countries. Theres a statue of Saraswati, the Hindu goddess of learning, which was given to her by a student from India. Theres also a vase from Russia, a screen from China and small house from the Philippines.

I like to think it shows I was successful in training the next generation, Jinks-Robertson said.

First ever job: A native of Panama City, Florida, Jinks-Robertson grew up around the water. As a child, she swam and water skied often. After she graduated from high school, she spent the next two summers working as a mermaid at Gulf World Marine Park, a popular attraction in Panama City.

We didnt have tails, but we had on scuba tanks and dove in saltwater tanks and fed the fish as people watched, Jinks-Robertson said of the mermaid role, which also had her swimming with dolphins and sea lions. It was fun.

Best advice received: In 1986, when she was finishing up her time as a post-doctoral researcher at the University of Chicago working with Thomas Petes, who many years later helped bring her to Duke, Jinks-Robertson began looking for faculty positions.

I was pregnant with my first child and I was concerned about that, Jinks-Robinson said. His advice was, If its a problem, its not a place you want to be. He really put me at ease and told me I shouldnt worry about that. Hes always been very supportive of women in science.

Something most people dont know about her: Much of Jinks-Robertsons work is done with a sleeping labradoodle at her feet. With soft, curly light brown hair, Gracie is Jinks-Robertsons constant companion, often accompanying her to work.

Its calming, Jinks-Robertson said. I walk her every day, so it gets me moving and out of my chair. Shes good company.

Is there a colleague at Duke who has an intriguing job or goes above and beyond to make a difference? Nominate that person for Blue Devil of the Week.

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Blue Devil of the Week: Searching for Answers in the Genetic Code - Duke Today

Chances are, youve personally used or benefitted from bioproducts products made from renewable biologic materials. Alternative protein sources such as the Impossible Burger and Pivot Bios nitrogen-fixing PROVEN fertilizer are just two of many successful bioproducts. In December, North Face Japan will offer the Moon Parka a high-performance ski parka manufactured with Spiber, Inc.s bioengineered spider silk. Geltors HumaColl21TM , a biocompatible collagen for human skin cells, is a key component of Kolam Koreas anti-aging face cream. Once bioproducts like these reach the market, its easy to overlook the fact that the work needed to adapt a biological process for use in the manufacture of a marketable product is just one step in a complex series of steps necessary for success.

The process of bringing a bioproduct to market is a challenge for a number of reasons. At the start of the process, its often the case that entrepreneurs with a technological capability struggle to find a real-world use for that technology. Once a use is identified, there must be an associated product, process, or service that compels consumers to become customers. As with any product, positioning in the marketplace with an eye toward positive consumer reaction is essential. When each of these stages has been accomplished, bioproducts generally face an additional hitch in the fact that, despite their environmental friendliness and sustainability, the public and regulatory agencies tend to be wary at best.

Advice on how to navigate such challenges is essential for many young startups in the biotech and synthetic biology space. Cambridge Consultants, a UK-based firm, assists synthetic biology companies around the world at all stages of the product life cycle. Over the years, James Hallinan, Business Development Manager of Synthetic Biology at Cambridge Consultants, has studied the success of a wide range of companies.

Its not just about having a great idea, he says. Its about having a great idea that is executed well. He identifies three traits that are especially important in a successful company: perseverance, flexibility, and execution. Here are three companies that exemplify how each of these challenges can be successfully met.

The first lesson, says Hallinan, is about perseverance as a really critical component. AquaBounty is a prime example. In 1989, Garth Fletcher co-inventor of GM salmon and his team found they could use advanced molecular genetics to increase the growth rate of Atlantic salmon. The increase in growth rate was enough to shorten the time required to bring salmon to marketable size. With a shorter growing time, less food for the fish was required. It was also possible to grow the fish inland, rather than in fish farms in the ocean. These factors led to lower costs and a reduced environmental impact.

Photo byCaroline AttwoodonUnsplash

With all of the benefits, it would seem that AquaBountys AquaAdvantage Atlantic salmon would be a sure hit. But consumers and the FDA were not ready for GM salmon in 1995 when the first application was submitted. For that matter, they were not ready in the early 2000s. One of the concerns was that the genetic modification would reach the native salmon population despite the fact that the fish grown from AquaBounty eggs are sterile females. It was not until nineteen years later that the salmon received approval for consumption in the U.S. AquaAdvantage Atlantic Salmon is expected to hit grocers shelves in 2020.

What I really admire about [AquaBounty], says Hallinan, is that they had the persistence to just keep going even when the public wasnt ready for their product and they had to continue on despite that. I think that on a larger scale, aquaculture is going to become increasingly important as we start to reduce the use of protein sources with significant greenhouse gas impacts like beef and pork.

Its entirely possible to have a great idea and set out to pursue it to what seems to you to be the logical conclusion. Frequently, this does not lead to success because the position you envision for your company is not the position the market will support. In the case of Oxford Biomedica, the original plan was to produce treatments for neurodegenerative disease and spinal cord repair, says Hallinan.

They were a classic biotech developing biological drugs. But the challenge to entering the market was that its a high-risk endeavor. Testing the product and coming up with a viable product requires a lot of time and capital.

Lack of initial success left Oxford Biomedica to rethink their goal. They decided to pivot from being a company where the therapy was the aim to one where a product to help others to create therapies was the aim. Their LentiVector delivery platform is the basis of their partnerships with companies like Novartis. The partnerships bring income to Oxford Biomedica while making it possible for Novartis and others to have a reliable, stable source of material.

By Stan Zurek Own work, CC BY-SA 3.0

Oxford Biomedica is now the worlds largest provider of viruses for selling gene therapy, says Hallinan. They are so successful, he says, because even when you get your product and youre starting to target the market you may find that you need to pivot, and you need to pivot effectively. Oxford Biomedica was not a tiny startup when they pivoted. They were an established company with a couple of hundred people. It was a big thing for them to change direction, but theyve done that, and theyve done that successfully. Now theyre in a position where theyre selling their product at significant scale.

You sometimes need to do something really different in order to make an impact, says Hallinan. Using the example of taking a collection of cells and isolating some population from that group of cells, Hallinan emphasized that people have been using technology that hasnt really advanced significantly for the best part of three decades.

Until now, that is. A company called Berkeley Lights, in recognizing the bottleneck created by the massive amount of data created during antibody discovery, has successfully made an impact by doing things really differently. As Hallinan puts it, Berkeley lights set out to create an entirely new architecture.

Their desired architecture optimized throughput and permitted greater precision and control down to the individual cell level. The company developed an optofluidic-based technology to achieve this goal, and their platform now enables real-time, non-destructive, and manufacturing-relevant analysis with optimized multiple serial assays across thousands of clonal populations in a single experiment. Berkeley Lights ability to deliver in a big way disrupted an entire industry.

AquaBounty, Oxford Biomedica, and Berkeley Lights are just three examples of how companies in this space can successfully address and overcome the challenges unique to bringing a bioproduct to market. With more companies following their lead, its only a matter of time until the power of synthetic biology changes our world forever.

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Leading by example: how AquaBounty, Oxford Biomedica, and Berkeley Lights have successfully brought products to market - SynBioBeta

Its an age-old question that many social scientists would love to have a definitive answer for are entrepreneurs born or made?

While there have been many studies of the genetics of entrepreneurs, on the face of it, there are several shared mental attributes among those who flourish. We dont need to delve too deeply into the psychological side to recognise the more obvious qualities: being highly motivated, confident, persistent, intuitive and impulsive all behaviours which can be learned by people who dont possess a special gift.

So, could any business owner adopt a similar winning mindset and have a positive impact on their companys outcome?

37pc-48pc of the tendency to be an entrepreneur is genetic

One study carried out by Kings College London into the behavioural and molecular genetics of entrepreneurship found that 37pc-48pc of the tendency to be an entrepreneur is genetic. The study assessed a general pool of 3,000 people, testing the number of businesses a person had started, how long they were self-employed for, and factors such as the desire to run a business. Even if those figures do paint an accurate picture, that still leaves us with 52pc-63pc who doesnt fall into the genetically gifted category.

Whether entrepreneurship is in the blood or not, there are several well-known executives who clearly had a natural entrepreneurial gift from an early age. Take Sir Richard Branson who started selling records from a church, or Lord Sugar, who sold car radio aerials and other electrical goods out of a van when he was a teenager. Humble beginnings and now billionaire business owners.

You cant determine where you start in life, but you can determine where you end up

On the other hand, we have those who had careers in different fields but were driven by entrepreneurial foresight, ambition and a determination to succeed. For example, Karren Brady began her career in advertising sales straight after leaving school and, aged just 23, was appointed managing director of Birmingham City Football Club. Now shes one of the most high-profile, respected business leaders in the UK. Also, Lady Michelle Mone, who was made redundant from a sales and marketing role at a brewing company before having a lightbulb moment which took her from designing bras to founder of the nations leading lingerie brand.

Ask those women the secret to success and you can bet your bottom dollar they wouldnt say it was just down to genetics. As Karren Brady says on her website: You cant determine where you start in life, but you can determine where you end up.

In other words, if people want to start their own business and really make it work, taking certain behavioural steps could help them on their journey. By adopting a different mindset, it will allow people to think and act like the successful entrepreneur they aspire to be.

It would be interesting to see from genetic studies whether a persons gender impacts their probability of being entrepreneurs. As champions of Women in Business, we aim to address the different challenges and barriers facing women when they rise to the top of their profession at our forthcoming Women in Business event, to be held in Farnborough October 16-17. Only one third of UK entrepreneurs are women, so there is a pool of untapped female potential out there who just need the right support and guidance to embrace their entrepreneurial spirit.

Genetics and gender aside, there are skills and behaviours which could be learned to get people into the entrepreneurial mindset.

See also: Everyone knows JK Rowling, but what about other women in business?

Here, we explore some of the most common traits of todays successful business leaders:

An entrepreneurial mindset involves having a steadfast commitment to a defined vision. This unwavering focus remains throughout the up and downs and daily demands of running the business. The end goal should always be front of mind and that will drive you to carry out the necessary steps to accomplish your vision. Strategic planning and thinking are critical for every business owner. Keeping eyes fixed on the big picture means you can see what direction the industry is going in, identify challenges for the company and devise the right solutions to meet your overall initiatives.

A fundamental characteristic of all successful entrepreneurs is their level of confidence in both their ideas and their ability. Buyers and investors will only believe in ideas if the business owner truly believes in them themselves. Be open to constructive feedback or critique but stay completely convinced that your business idea will yield positive results and that you have the capacity to make it happen. Carrying out in-depth research into your business idea will help to dispel any doubts. Know your market, your USP and your key personal strengths to bolster your self-belief.

Being willing to take risks and move outside of your comfort zone is all part of the journey. Entrepreneurs have amazing resilience and thrive off turning around negativity. Risks are inherent in any new venture and we are frequently told by those who have made it that failure is an inevitable part of success. Its crucial that you frame failure as an opportunity to learn. The path to success will hopefully have an upwards trajectory but it is rarely a straight line. Any failure should drive you forward. Observe and absorb what you have learned and use it to progress.

Successful entrepreneurs leverage their strengths and understand that they cant do everything. All business people are naturally better at some things and where there are areas of weakness or lack of knowledge that is the time to outsource and seek external help. Knowing that you cant tackle every obstacle on your own is a strength. If bringing people in house is ruled out on financial grounds, hire consultants to look at the more complex, labour-intensive parts of the business. That will ensure you can focus on the overarching business strategy and not get bogged down by minutiae.

The ability to adapt to change quickly is a key entrepreneurial attribute. Whether theres a new competitor springing on to the scene or dip in demand in the target market. Being flexible means having the courage and conviction to rethink a situation, keeping track of feedback about pricing, products and services and making tweaks when necessary. The path of an entrepreneur will occasionally go off course and flexibility is an important skill to keep you on track.

Christie Day is event director for Women in Business Expo, which takes place October 16-17 at Farnborough International Conference & Exhibition Centre

Start young, sleep less: New study reveals leadership formula

Excerpt from:
5 habits of highly successful entrepreneurs revealed - GrowthBusiness.co.uk

The promise of precision medicine is focused on providing the right drug to the right patient at the right time. Usually that means matching patients, based on their molecular profiles to specific approved drugs. But precision medicine has never created a unique drug intended to treat oneand only onepatient.

Until now.

In what is likely the first instance of a drug being specifically designed for a single patient, researchers at Boston Childrens Hospital (BCH) have reported in the New England Journal of Medicinethe one-year development process of a drug custom-designedand approved by the FDAto bypass a unique mutation and treat an 8-year-old girls CLN7 Batten disease.

The team at BCH was led by Timothy Yu, M.D., Ph.D., a neurologist and genetics researcher.

One of the unique things about this trial is that, due to clinical urgency, we were starting a first-in-human trial with a drug that had only been tested in our patients cells in a dish, said Yu in a press release. What was also unique is that we have a drug that is targeted to a mutation seen to this date only in one patient.

Mila, the patient, was diagnosed in late 2016 with Batten disease, a rare neurodegenerative disease that is marked by rapid progression and is ultimately fatal. Milas form of the disease is called CLN7 Batten disease as those suffering from this have inherited a bad copy of the CLN7 gene. CLN7 Batten disease has no known cure, and Milas form of the disease is caused by a mutation in the gene not previously observed.

Milas family first became concerned about her health around the age of 3 when her right foot began to turn inward. Her disease continued to progress over the next few years with additional signs including pulling books close to her face as a 4-year-old, and beginning to stumble when she was 5.

By the time Mila was first seen at BCH two years ago, Mila was blind, was experiencing seizures, was barely speaking and showed signs of both developmental and neurologic regression. Despite her difficulties, the BCH team reported, she remained alert, happy and able to respond to favorite activities and people.

Finding the mutation in the dark

CLN7 Batten disease is recessive, and while the mutation Mila inherited from her father was readily identified in her CLN7 gene, the mutation from her mother could not be found. To try to find an answer for her ill daughter Milas mother posted on Facebook, looking for a lab that could quickly do whole-genome sequencing. Yus wife saw the ad in January 2017, brought the appeal to his attention since his lab specializes in WGS and finding unusual mutations.

Within three months Yu and his colleagues found the mutation, residing in the dark matter of the genomeaffecting a regulatory piece of DNA that controls splicing of the CLN7 gene. Even more unusual, it consisted of a jumping genea rogue piece of DNA known as a retrotransposon that inserted itself into the genome, altering how the splicing machinery read CLN7 resulting in the production of an abnormal, truncated protein.

Designing Milas drug

As Yu and colleagues wrestled with how to use this information to treat Mila, they decided a good approach was to design a number of antisense oligonucleotide drugs, one of which they hoped would help bypass her unique mutations. The oligonucleotides were designed to find the CLN7 gene and act as a bandage to cover the malfunctioning gene so that the CLN7 gene could be read normally.

By September, 2017, Yus team had showed that several of their candidate oligonucleotide drugs could repair the splicing defect in Milas CLN7 gene. One month later, they showed that the drugs also corrected the lysosomal abnormalities in Milas cells. They named their best candidate milasen and sent it to contract research organizations for animal testing and manufacturing.

n of 1 trial

In January 2018, the FDA granted permission to test milasen as a Single-Patient Compassionate-Use Investigational New Drug and by the end of the month, Mila began her single-patient clinical trial, a short eight months after her diagnosis.

Every two weeks Mila received a total of nine escalating doses of milasen, which was given via spinal injection to target it to the brain. Since August 2018, she has received maintenance treatments every two to three months.

Since beginning treatment on milasen, Milas seizure frequency has decreased from roughly 30 seizures per day to 5-10 per day. Further, the duration of her seizures decreased significantly from one to two minutes to only a few second each.

Looking ahead

As the first instance of a team creating a customized drug for a single patient, the development of milasen, over such a short time span, could give new hope to how rare genetic diseases are treated.

Everyone knows that the future of precision medicine in pediatrics is developing treatments for kids one at a time, the way this work has done, said Christopher A. Walsh, M.D., Ph.D., chief of the Division of Genetics at Boston Childrens Hospital in a prepared statement, but until now no one had a real-life case where everything lined up in a way to allow them to do it.

Based on his teams work, Yu believes other cases could also be treatable with oligonucleotides. He and Milas mother recently met with the FDA to discuss a new regulatory model for offering custom antisense oligonucleotide treatments for patients with rare or seemingly one-of-a-kind conditions. However, many questions and obstacles remain to be addressed before widespread adoption of this approach.

There are whole categories of diseases with populations too small to attract industry effort, Yu said. But in the hospital where research is a major focus, we can go one step, one patient at a time. I think that some of the most exciting parts in science are when you try to do something new, when there isnt a recipe.

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BCH N of 1 Trial Yields Approved Therapy for Single Rare Disease Patient - Clinical OMICs News