Category Archives: Genome

Published: Oct. 7, 2021 at 7:00 AM EDT|Updated: 9 hours ago

BRISBANE, Calif., Oct. 7, 2021 /PRNewswire/ -- Second Genome, a biotechnology company that leverages its proprietary platform sg-4sight to discover and develop precision therapies and biomarkers, presented preclinical data demonstrating that the Company's novel microbiome-derived peptide, SG-3-0020, can upregulate key co-stimulatory and checkpoint molecules on T cells. The data (poster P260) were presented at the 2021 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held virtually on October 7-10, 2021.

"Our findings that SG-3-0020 enhanced PD-1 expression and IFNg secretion by anti-CD3 activated human T cells in vitro provide a strong rationale for SG-3-0020's potential future use in cancer therapies. Additionally, we are pleased to see these results provided additional validation of the advanced capabilities of sg-4sight, Second Genome's proprietary platform, to identify targets for microbial peptides and proteins with potential therapeutic relevance in immuno-oncology," said Helena Kiefel, Ph.D., Head of Immuno-Oncology at Second Genome. "Given these promising preclinical data, we are looking to furtherdevelop SG-3-0020 with an experienced partner, as we continue to advance SG-3-00802, our lead immuno-oncologyprogramfor whichwe anticipate an IND submission in 2022 for SG-3-00802."

Using Second Genome's large and curated microbiome database coupled with its proprietary bioinformatics and machine learning tools, the Company analyzed the genome of Bifidobacterium (B.) breve and B. longum for proteins which were potentially secreted and had unknown functions. 50 peptides were chemically synthesized and then screened in cell-based assays for T cell activation and cytokine secretion.

SG-3-0020, a novel B. breve-derived 42-aa peptide, stimulated secretion of effector cytokines by in vitro-cultured T cells (IFNg, TNF-a, IL-10 and IL-2) and increased the expression of PD-1 on both CD4+ and CD8+ T cells when stimulated with low-dose anti-CD3 antibody. Mass spec analysis showed that SG-3-05308, a variant of SG-3-0020, binds to a transmembrane glycoprotein of the immunoglobulin superfamily. Silencing this gene via CRISPR-Cas significantly decreased PD-1 levels, cell proliferation and IFNg production in human pan T cells.

The potency of SG-3-0020 was further optimized for binding to activated T cells through protein engineering. The peptide with the highest potency, SG-3-05429, was selected for further investigation of the mechanism by which binding of its glycoprotein target results in activation of downstream T cell signaling pathways.

The poster presentation will be available for on-demand viewing on the AACR-NCI-EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics website and will also be made available on the Company's website at https://www.secondgenome.com/news/events.

About Second Genome

Second Genome is a biotechnology company that leverages its proprietary tech platform sg-4sight to discover and develop transformational precision therapies and biomarkers through clinical development and commercialization based on novel microbial genetic insights. We built a proprietary microbiome-based drug discovery and development platform with machine-learning analytics, customized protein engineering techniques, phage library screening, mass spec analysis and CRISPR, that we couple with traditional drug development approaches to progress the development of therapies and diagnostics for wide-ranging diseases. Second Genome is advancing a deep drug discovery and biomarker pipeline with precision therapeutics and biomarker programs in inflammatory bowel disease (IBD) and cancer, with the lead programs IBD and cancer expected to enter clinical development in 2022. We also collaborate with industry, academic and governmental partners to leverage our microbiome platform and data science. We hold a strategic collaboration with Gilead Sciences, Inc., utilizing our proprietary platform and comprehensive data sets to identify novel biomarkers associated with clinical response to Gilead's investigational medicines. We also hold a strategic collaboration with Arena Pharmaceuticals to identify microbiome biomarkers associated with clinical response for their lead program in gastroenterology, etrasimod. For more information, please visit http://www.secondgenome.com.

Investor Contact: Argot Partners212-600-1902secondgenome@argotpartners.com

Media Contact: Argot Partners212-600-1902secondgenome@argotpartners.com

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Second Genome Presents Preclinical Data at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics Demonstrating that...

Amyris' product development and formulation teamuses a proprietaryLab-to-Market operating system to develop and scale a growing portfolio of sustainable ingredients. The Onyx platform automates benchtop biofoundry activity and will bring greater genetic diversity and value to Amyris' ingredient development pipeline, complementing Amyris' existing Lab-to-Market operating systemwith the goal of improving efficiency and reducing timelines for the development of future molecules. To date, Amyris has successfully commercialized 13 sustainable ingredients, which are formulated in over 20,000 products and used by over 300 million consumers, demonstrating the growing demand for sustainable products with clean and effective ingredients.

Automated, high-throughput gene editing is revolutionizing the writing of genomes the way next-generation sequencing transformed the reading of genomes. Inscripta is the first company to deliver an integrated and intuitive benchtop platform that will expand access to scalable, robust genome engineering and help scientists develop solutions to some of today's most pressing challenges.

"Amyris has shown the world how new products can be made more sustainable through biology. Their team has high proficiency in utilizing cutting-edge technology, and we are excited they will be pioneering the use of our platform," said Sri Kosaraju, President and CEO of Inscripta. "We have great regard for Amyris' mission, and we are committed to seeing the Onyx platform become a substantial contributor to new clean chemistry products in the future."

"The Onyx platform offers significant potential for generating greater genetic diversity in our projects, which we expect to lead to more efficient product innovation," said Sunil Chandran, Senior Vice President of Research and Development at Amyris. "Inscripta's platform seamlessly integrates with our own and opens up new experimentation avenues for our scientists to continue bringing unique bio-based products to customers. We pride ourselves on continuous innovation and expect Onyx to help us expand our pipeline, while achieving lower costs and reducing time to market."

For more information about Amyris visit amyris.comand to learn about Onyx, visitwww.inscripta.com/products.

About InscriptaInscripta is a life science technology company enabling scientists to solve some of today's most pressing challenges with the first benchtop system for genome editing. The company's automatedOnyx platform,consisting of an instrument, consumables, assays, and software, makes CRISPR-based genome engineering accessible to any research lab. Inscripta supports its customers around the world from facilities in Boulder, Colorado; San Diego and Pleasanton, California; and Copenhagen, Denmark. To learn more, visitInscripta.comand follow@InscriptaInc.

About AmyrisAmyris (Nasdaq: AMRS) is a science and technology leader in the research, development and production of sustainable ingredients for the Clean Health & Beauty and Flavors & Fragrances markets. Amyris uses an impressive array of exclusive technologies, including state-of-the-art machine learning, robotics and artificial intelligence. Our ingredients are included in over 20,000 products from the world's top brands, reaching more than 300 million consumers. Amyris is proud to own and operate a family of consumer brands - all built around its No Compromise promise of clean ingredients: Biossanceclean beauty skincare, Pipetteclean baby skincare, Purecane, a zero-calorie sweetener naturally derived from sugarcane, Terasanaclean skincare treatment, Costa Brazil luxury skincare, OLIKA hygiene and wellness, Rose Inc. clean color cosmetics and JVN clean haircare. For more information, please visit http://www.amyris.com.

Amyris, the Amyris logo, No Compromise, Biossance, Pipette, Purecane, Terasana, Rose Inc. and Lab-to-Market are trademarks or registered trademarks of Amyris, Inc. in the U.S. and/or other countries.

Forward-Looking StatementsThis release contains forward-looking statements, and any statements other than statements of historical fact could be deemed to be forward-looking statements.These forward-looking statements include, among other things, statements regarding Amyris' expectation of exploring additional research and development opportunities with Inscripta in the future and its expectation that Onyx will help Amyris expand its pipeline while achieving lower costs and reducing time to market. These statements are based on management's current expectations and actual results and future events may differ materially due to risks and uncertainties, including risks related to any delays or failures in the successful launch of a clean skincare brand; potential delays or failures in development, production, regulatory approval and commercialization of products, risks related to Amyris' reliance on third parties; Amyris' liquidity and ability to fund operating and capital expenses; and other risks detailed from time to time in filings Amyris makes with the Securities and Exchange Commission, including Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Amyris disclaims any obligation to update information contained in these forward-looking statements, whether as a result of new information, future events, or otherwise.

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https://amyris.com

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Amyris Partners with Inscripta to Enhance Development of Sustainable Ingredients Using the Onyx Genome Engineering Platform - PRNewswire

Monitoring changes in the viral genome is continuing to play a crucial role in shaping the response to the COVID-19 pandemic.The COVID-19 pandemic has caused over 4.5 million deaths worldwide, with more than 200 million people having been infected with the novel coronavirus, SARS-CoV-2. Since the virus first emerged in China in December 2019, the analysis of genetic sequencing data from millions of positive samples has revealed how the virus has evolved.Changes in the SARS-CoV-2 genome occur at a rate of around one or two mutations per month, says Dr. Alessandro Carabelli, Research Associate at COG-UK, University of Cambridge, UK.Most mutations have little or no effect on the biology of SARS-CoV-2 and persist in the descendants of that virus providing useful tags for tracking its spread.By understanding viral transmission pathways within a certain setting, such as a workplace or prison, this can help inform decisions on what actions are needed to contain an outbreak, says Patricia Brennan, director of Science Product Management at Chan Zuckerberg Initiative, San Francisco, USA.But some mutations can alter the biological behavior of SARS-CoV-2, such as improved transmissibility or ability to evade the immune system. Detecting and tracking the emergence of these changes is vital as they may threaten the performance of vaccines, therapies, diagnostic tools or other public health and social measures.

While coronaviruses, like SARS-CoV-2, have evolved some proofreading mechanisms to maintain their genomes, random errors can still occur.

As mutations accumulate, this leads to numerous differences in the virus population over time, explains Carabelli.But some mutations can change the behavior of the virus and these will persist if they confer an advantage, such as altering its ability to cause disease or transmit from one person to another. Changes that occur in the gene encoding the spike protein are of particular interest, as it is involved in the virus entering human cells and is the target for most COVID-19 vaccines.A concern is that mutations may arise that lead to lower vaccine efficacy, says Carabelli. The most numerically important mutation to date is the spike protein variant D614G, which was not present when the virus first emerged but is now almost ubiquitous.Accurate SARS-CoV-2 Surveillance With Resilience to Genomic Variation

As a result of persistent transmission, there are now several mutational variants of SARS-CoV-2. These variants have emerged and rapidly changed the viral dynamics, thus threatening ongoing public health efforts to limit the transmission of SARS-CoV-2. Download this app note to discover a surveillance assay that will remain effective as new variants emerge while offering high sensitivity and capture efficiency.

New variants will continue to emerge, and it is important we understand the phenotypes of emerging variants in terms of infectivity, transmissibility and antigenicity as soon as possible, says Carabelli.

Tracking mutations in the virus is an essential surveillance partner to vaccine roll-out and it likely to be needed for as long as the vaccine is required, states Carabelli.

COVID-19 vaccines might have to be periodically updated to reflect any changes in the virus.Whats great about many of the latest technologies used to develop COVID-19 vaccines such as mRNA vaccines is that they can be updated quickly to target those emerging variants, says Ayscue.Where Do We Go From Here: A Reflection of COVID-19

In less than two years, much has been discovered about SARS-CoV-2 and its variants, and to date several vaccines have been created. Capitalizing on several decades of progress on new vaccine technologies, viral immunology, structural biology and protein engineering research, along with clinical trial operations, seven vaccines have undergone the process of rapid development, evaluation, manufacturing and deployment. Download this app note to explore various solutions designed to improve COVID-19 detection and the development of vaccines and therapeutics.

Genomic data is great for understanding transmission pathways it can tease out whether a cluster of infections is associated with people getting infected within that setting or bringing it in from outside, explains Ayscue. Its essentially giving you breadcrumbs to follow those mutations and work out where viral transmission is happening providing the evidence to inform what measures are needed to disrupt it.

But the COVID-19 pandemic has exposed huge disparities in the current capacity of public health systems to perform genomic surveillance of circulating pathogens both at a national and international level.

Public health systems are extremely fragmented in many countries, including the US, says Brennan. This means that the frontline work is often being done by people who dont have the necessary skills and infrastructure to carry out these analyses.The development of an open-source free software tool, called Aspen, is one step towards democratizing the technology enabling individual public health laboratories to conduct genomic analyses and interpret the data independently.People can load their data and do the analysis so they can see where the mutations have occurred, says Brennan. They can look at those in terms of the population they came from and work out where they need to take action.

Genomic surveillance and the use of pathogen genomes on this scale to track the spread of the virus, study local outbreaks and inform public health policy signifies a new age in virus genomic investigations, says Carabelli.

The global community will need to work together to put the necessary infrastructure in place to be ready for future pandemics.

If COVID-19 has taught us anything, its that borders and boundaries dont exist as long as people are moving around, says Brennan. We need to get the tools that researchers have developed to places where they can be applied and used more readily as a society, I hope we can tend to that more diligently than we have in the last many decades.

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Leveraging the Power of Genomics To Detect and Track SARS-CoV-2 Variants - Technology Networks

This story has been updated to include a response from Agilent Technologies.

NEW YORK Gene editing firm Synthego has sued Agilent Technologies in US federal court after Agilent allegedly tried to get the firm to license patents related to CRISPR synthetic guide RNAs.

In a complaint filed Tuesday in the US District Court for the Northern District of California, Synthego asked for a judge to declare that its CRISPRevolution products do not infringe US Patent Nos. 10,900,034 and 10,337,001, both titled "Guide RNA with chemical modifications" and held by Agilent.

Such a declaration is necessary, Synthego said, because Agilent tried to get the company to license patents related to synthetic guide RNAs and "create[d] a reasonable apprehension it would pursue legal action against Synthego were Synthego not to acquire a license."

"We are confident that the patents in question lack innovation," Synthego said in a statement. "We will continue to provide uninterrupted support for the essential work of CRISPR researchers and therapeutic developers with the highest quality products and services that our customers have come to expect."

Agilent declined tocomment.

Santa Clara, California-based Agilent has developed chemically modified guide RNAs for use in CRISPR genome editing in T cells and other cells. Synthego, based in Menlo Park, California, offers a variety of genome editing products, including synthetic guide RNAs, screening libraries, and even engineered cells and cell lines.

According to the complaint, Agilent Associate VP Thomas Redder sent Synthego a letter in June offering to license patents covering guide RNAs. Synthego alleged that in a subsequent email Redder said that if the firm did not respond by a certain deadline, the "process will likely be escalated to other, more formal, legalistic processes for managing [Agilent's] out-licensing programs and opportunities."

Synthego said that it asked for more time to conduct due diligence and alleged that Redder called the request "disappointing" and ultimately demanded that the company respond to a term sheet proposed by Agilent by Oct. 6.

Synthego's complaint said that because its products are used for purposes related to the development and submission of information to the US Food and Drug Administration, they do not infringe the patents under the safe harbor provisions of 35 U.S.C. 271(e)(1).

Synthego also asked the court to prevent Agilent from alleging infringement in the future and for attorneys' fees and expenses related to the case.

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Synthego Preemptively Sues Agilent Technologies to Ward Off CRISPR Patent Infringement Allegations - GenomeWeb

CAMBRIDGE, Mass., Oct. 07, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the Company will present preclinical data on its progress in the development of cell therapy medicines for the treatment of cancer at the Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting being held in Washington, D.C., and virtually, November 10-14, 2021.

The Company will present a poster on its new method to drive high-level constitutive CD16 expression on the surface of iPSC-derived natural killer (iNK) cells through transgene knock-in (KI) at the GAPDH locus using an Editas-engineered AsCas12a. New preclinical data demonstrated that CD16 KI confers significantly increased cytotoxic activity in iNK cells against tumor cells compared with wild type iNK cells.

Full details of the Editas Medicine presentation can be accessed on the SITC website at https://sitc.sitcancer.org/2021/abstracts/titles/.

Poster Presentation:Title: GAPDH Knock-in of High Affinity CD16 in iPSC Derived NK Cells Drives High-level Expression and Increased Anti-tumor FunctionDate and Time: Friday, November 12, 2021, 7:00 a.m. 8:30 p.m. ETAbstract #: 191 Session Title: Cellular Therapies

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Companys subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

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Editas Medicine to Present Preclinical Data on Novel Engineered iPSC Derived NK Cells for the Treatment of Cancer at the Society for Immunotherapy of...

NEW YORK--(BUSINESS WIRE)--Colossal and the Vertebrate Genomes Project (VGP) embark on the worlds first effort to obtain the genetic code of the existing elephant lineage, of which all three species are endangered. As part of the collaboration, Colossal will fund the VGP efforts to sequence, assemble, and annotate the Asian elephant (Elephas maximus), African elephant (Loxodonta africana), and Forest elephant (Loxodonta cyclotis) genomes to genetically preserve the species for future generations. These will be high-quality genome assemblies, complete and error-free as much as possible, so that all valuable parts of the genomes are captured for de-extinction efforts. Following the VGP principles, these genomes will be made publicly available to be used for research without use restrictions.

Colossal announced last month it will be the first to implement a working model of de-extinction to apply advanced gene-editing techniques to restore the woolly mammoth, which shares 99.6 percent of its DNA with the Asian elephant, to the Arctic tundra. To achieve this goal, in addition to the high-quality reference genomes, Colossal aims to produce population sequence data on 100 African and Asian elephants to record and understand extant elephantid population genomes. The Asian and African elephants are classified as Endangered, and the Forest elephant is classified as Critically Endangered by the IUCN.

The VGP has already been successful in genetically backing up species and has demonstrated how vital this process is. By sequencing these three elephant species, we will genetically preserve the entire order. Together we can safeguard the genetic lineage of elephants to protect biodiversity and restore healthy ecosystems, which is part of Colossals mission of thoughtful disruptive conservation, said Ben Lamm, Colossal Co-Founder and CEO.

There is not enough focus on genomic data in conservation efforts , including with high-quality reference genomes. It is critical that we act with urgency as we aim to genetically preserve and protect endangered species, before we lose them forever. With Colossals help, we can rapidly advance efforts starting with elephants, said Erich Jarvis, PhD, a Howard Hughes Medical Institute Investigator and Professor at The Rockefeller University. Dr. Jarvis is Chair of the Vertebrate Genomes Project.

This is the first of many species preservation and extension efforts for Colossal. In addition to funding previously non-sequenced species, in collaboration with the VGP and others, Colossal will pursue and fund the sequencing and resequencing of additional individuals from select species categorized as Endangered or Critically Endangered by the IUCN, with the goal of preserving the genetic biodiversity found in wild populations.

ABOUT COLOSSAL

Colossal is a breakthrough bioscience and genetic engineering company that builds radical new technologies to advance the field of genomics. Colossal creates disruptive technologies for extinct species restoration, critically endangered species protection and the repopulation of critical ecosystems that support the continuation of life on Earth. The company is the first to apply CRISPR technology for the purposes of species de-extinction, beginning with the woolly mammoth. Colossal is accepting humanitys duty to restore Earth to a healthier state, while also solving for the future economies and biological necessities of the human condition through cutting-edge science and technologies. To follow along, please visit: http://www.colossal.com.

ABOUT THE VERTEBRATE GENOMES PROJECT (VGP)

The Vertebrate Genomes Project (VGP) aims to sequence high-quality reference genomes for all living 70,000+ vertebrates (i.e., mammals, amphibians, birds, reptiles, and fishes), and to use those genomes to address fundamental questions in biology, disease, and conservation. This includes using to the genomes to identify species most genetically at risk of extinction, and to preserve the genetic information of life. The VGP produces near error-free and complete chromosomal-level genome assemblies, and has become a model for the broader Earth Biogenome Project, to produce high-quality assemblies of all eukaryotic species. To date, the current VGP pipelines have led to high-quality reference genomes for over 135 species deposited in the public databases, representing the most complete and accurate versions of those species to date, including multiple species at risk of extinction. The VGP 2021 flagship paper and associated publications demonstrates feasibility in quality standardization and scale for the field of genomics. To find our more information, visit https://vertebrategenomesproject.org/.

WEBSITE & SOCIALS

Websites: http://www.colossal.com, http://www.vertebrategenomesproject.org Twitter: https://twitter.com/ItIsColossal, https://twitter.com/genomeark Instagram: https://www.instagram.com/itiscolossal/ LinkedIn: https://www.linkedin.com/company/itiscolossal/ Facebook: https://www.facebook.com/ItIsColossal Hashtag: #ItIsColossal, #VGP, #GenomicsForAll

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Colossal Biosciences and the Vertebrate Genomes Project Will Preserve the Genetic Code of all Endangered Elephant Species Through Genomic Sequencing -...

NTLA-2002 is the first single-dose genome editing therapeutic candidate designed to prevent attacks in people living with HAE to enter clinical study

NTLA-2002 is Intellias second in vivo CRISPR genome editing therapeutic candidate; program to leverage platform insights gained from ongoing development of NTLA-2001 for transthyretin (ATTR) amyloidosis

On track to initiate patient enrollment by year-end

CAMBRIDGE, Mass., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced the authorization of its Clinical Trial Application (CTA) by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with hereditary angioedema (HAE). HAE is a genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. NTLA-2002 is a systemically administered single-dose CRISPR/Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to permanently reduce plasma kallikrein activity and thus prevent HAE attacks.

We look forward to initiating this year our first-in-human study of NTLA-2002 for people living with HAE, a debilitating disorder that causes frequent, potentially life-threatening attacks, said Intellia President and Chief Executive Officer John Leonard, M.D. We believe NTLA-2002 has the potential to be a curative therapy for patients with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and eliminating the significant treatment burden associated with currently available HAE therapies. This study of NTLA-2002 leverages early insights from our ATTR amyloidosis program, where we established proof-of-concept for our modular in vivo genome editing platform with interim Phase 1 data earlier this year. The NTLA-2002 program represents the second systemic in vivo CRISPR genome editing therapy candidate to enter human clinical trials.

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The Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies. More information about the study will be available at clinicaltrials.gov.

Beyond New Zealand, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development approach for NTLA-2002.

About Hereditary Angioedema

Hereditary Angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.

About NTLA-2002

Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is potentially the first single-dose treatment to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly-owned investigational CRISPR therapeutic candidate designed to inactivate the Kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellias second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. Intellias proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which together carry out the precision editing.

About Intellia Therapeutics

Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The companys in vivo programs use intravenously administered CRISPR as the therapy, leveraging proprietary delivery technology to enable highly precise editing of disease-causing genes directly within specific target tissues. Intellias ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellias deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: being able to initiate clinical studies for NTLA-2002 for the treatment of hereditary angioedema (HAE) pursuant to its clinical trial applications (CTA), including enrollment of a first patient by the end of 2021, and submitting similar regulatory applications in other countries; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its transthyretin amyloidosis and HAE programs, in any future studies, including human clinical trials; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; statements regarding the timing of regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other 2021 financial results or in the future; and ability to fund operations beyond the next 24 months.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Regeneron or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Ian KarpSenior Vice President, Investor Relations and Corporate Communications+1-857-449-4175ian.karp@intelliatx.com

Lina LiDirector, Investor Relations+1-857-706-1612lina.li@intelliatx.com

Media:

Lisa QuTen Bridge Communications+1-678-662-9166media@intelliatx.comlqu@tenbridgecommunications.com

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Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema - Yahoo...

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Circ Genom Precis Med. 2021 Oct 4:CIRCGEN119002862. doi: 10.1161/CIRCGEN.119.002862. Online ahead of print.

ABSTRACT

BACKGROUND: Peripheral artery disease (PAD) affects >200 million people worldwide and is associated with high mortality and morbidity. We sought to identify genomic variants associated with PAD overall and in the contexts of diabetes and smoking status.

METHODS: We identified genetic variants associated with PAD and then meta-analyzed with published summary statistics from the Million Veterans Program and UK Biobank to replicate their findings. Next, we ran stratified genome-wide association analysis in ever smokers, never smokers, individuals with diabetes, and individuals with no history of diabetes and corresponding interaction analyses, to identify variants that modify the risk of PAD dependent on diabetic or smoking status.

RESULTS: We identified 5 genome-wide significant (Passociation 510-8) associations with PAD in 449 548 (Ncases=12 086) individuals of European ancestry near LPA, CDKN2BAS1, SH2B3-PTPN11, HDAC9, and CHRNA5 loci (which overlapped previously reported associations). Meta-analysis with variants previously association with PAD showed that 18 of 19 published variants remained genome-wide significant. In individuals with diabetes, rs116405693 at the CCSER1 locus was associated with PAD (odds ratio [95% CI], 1.51 [1.32-1.74], Pdiabetes=2.510-9, Pinteractionwithdiabetes=5.310-7). Furthermore, in smokers, rs12910984 at the CHRNA3 locus was associated with PAD (odds ratio [95% CI], 1.15 [1.11-1.19], Psmokers=9.310-10, Pinteractionwithsmoking=3.910-5).

CONCLUSIONS: Our analyses confirm the published genetic associations with PAD and identify novel variants that may influence susceptibility to PAD in the context of diabetes or smoking status.

PMID:34601942 | DOI:10.1161/CIRCGEN.119.002862

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Genome-Wide Association Study of Peripheral Artery Disease - DocWire News

SAN DIEGO, Sept. 29, 2021 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO) today announced the publication of the largest clinical research study to date evaluating optical genome mapping (OGM) for facioscapulohumeral muscular dystrophy (FSHD), which concluded that OGM can be performed more quickly, accurately, and reproducibly than the current gold standard method of Southern blot analysis. This study, now available online before print in The Journal of Molecular Diagnostics, describes how OGM can be adopted as an alternative to Southern blot analysis for the identification of chromosomal abnormalities associated with FSHD, a neuromuscular disorder resulting in progressive weakness and atrophy of muscles.

FSHD is typically caused by a contraction of a D4Z4 repeat array near the telomere of chromosome 4 (chr 4) at 4q35, which activates a dormant toxic gene, DUX4, to be overexpressed in skeletal muscle. Because of its complex genotype, FSHD can often be challenging to detect using next-generation sequencing (NGS) and Southern blot analysis, which can be slow, laborious and require radioactive labeling. In this robust study of 351 participants, the authors Stence et al., validated the use of OGM with Bionanos Saphyr system to determine the size and haplotype of D4Z4 alleles to confirm a diagnosis of FSHD. The studys authors found that OGM had much higher sizing accuracy and was >99% concordant with Southern blot analysis.

We are pleased to be using optical genome mapping for FSHD mutation analysis because it more reliably identifies the patients mutation and simplifies the initial analysis for the laboratory, said Aaron D. Bossler, MD, PhD, FCAP, Clinical Professor and Director, Molecular Pathology Laboratory at University of Iowa Hospitals and Clinics. The extensive validation that we conducted with optical genome mapping demonstrates the power of this new technology to decrease time to result, improve standardization, and help tease out complex cases.

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OGM is well-suited for analyzing inherited genetic disorders like FSHD that require the measurement of long, intact DNA molecules for accurate sizing. The higher resolution of OGM relative to traditional techniques can enable improved detection and discrimination ability for clinically relevant variants, as was observed in this study.

The study completed by Dr. Stence and team demonstrated the superior performance of OGM over traditional molecular genetic testing for FSHD, commented Erik Holmlin, PhD, CEO of Bionano Genomics. As the largest OGM clinical study to date, the study demonstrated that using the Saphyr system to perform FSHD analysis resulted in a significantly faster turnaround time, as the OGM turnaround time was only 5 days as compared to Southern blot at 11 days. The ability to obtain accurate, reliable data more quickly allowed for the timely application of this information for the management of study participants. We believe that studies like this one could pave the way for broad adoption of OGM for a wide variety of applications in genetic disorders and cancer.

This publication is available at https://pubmed.ncbi.nlm.nih.gov/34384893/

About Bionano Genomics

Bionano is a genome analysis company providing tools and services based on its Saphyr system to scientists and clinicians conducting genetic research and patient testing; it also provides diagnostic testing for those with autism spectrum disorder (ASD) and other neurodevelopmental disabilities through its Lineagen business. Bionanos Saphyr system is a research use only platform for ultra-sensitive and ultra-specific structural variation detection that enables scientists and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline the study of changes in chromosomes, which is known as cytogenetics. The Saphyr system is comprised of an instrument, chip consumables, reagents and a suite of data analysis tools. Bionano offers genome analysis services to provide access to data generated by the Saphyr system for researchers who prefer not to adopt the Saphyr system in their labs. Lineagen has been providing genetic testing services to families and their healthcare providers for more than nine years and has performed more than 65,000 tests for those with neurodevelopmental concerns. For more information, visit http://www.bionanogenomics.com or http://www.lineagen.com.

Forward-Looking Statements of Bionano Genomics

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, among other things: the impact of the study results discussed in this press release, including our expectations regarding the studys potential to increase adoption of OGM for analysis of FSHD or other genetic disorders; OGMs superiority in genomic analysis as compared to traditional techniques; the potential benefits resulting from a combination of OGM and NGS technologies; and the advancement of our business strategy. 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 and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive products; incompatibility between our and NGS technologies; future study results that may contradict the results of the study identified in this press release; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; 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, 2020 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 managements 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:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com

Investor Relations:Amy ConradJuniper Point+1 (858) 366-3243amy@juniper-point.com

Media Relations:Michael SullivanSeismic+1 (503) 799-7520michael@teamseismic.com

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Largest Clinical Research Study to Date Evaluating Optical Genome Mapping for Analysis of Facioscapulohumeral Muscular Dystrophy Showed Concordance...

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How the GOAST in the machine takes the fear out of genomics computing - The Register