EMERYVILLE, Calif.--(BUSINESS WIRE)--Metagenomi, a gene editing company with a versatile portfolio of next-generation CRISPR gene editing tools, today announced its participation in the following investor and industry conferences:

Chardans 6th Annual Genetic Medicines Conference - New York CityCorporate presentation, 9:00 9:25 a.m. EDT, followed by a panel presentation on next-generation gene editing, 9:30 10:15 a.m. EDT, on October 311 meetings with institutional investorsParticipants: Brian C. Thomas, Ph.D., CEO and founder, and Simon Harnest, CIO, SVP Strategy

BMO BioPharma Spotlight Series - Gene Editing & TherapyPanel presentation titled Discovery Of Unique Gene Editing Systems And Application In Therapeutics on October 6, 12:00 p.m. EDTParticipant: Simon Harnest, CIO, SVP Strategy

Truist Securities Genetic Medicine SummitOctober 20Participant: Simon Harnest, CIO, SVP Strategy

5th International Conference on CRISPR TechnologiesTwo scientific presentations, October 31 November 2Participants: Chris Brown, Ph.D., Director of Discovery, and Cindy Castelle, Ph.D., Associate Director, Functional Assay Development & Screening

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.

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Metagenomi Announces Participation in October Investor and Industry Conferences - Business Wire

MADRID, Spain and BOSTON, Oct. 03, 2022 (GLOBE NEWSWIRE) -- ORYZON Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today that its management will give an update on corporate progress at several international events in October.

Oryzon will participate at the Chardan's 6th Annual Genetic Medicines Conference, which will be held on October 3-4 in New York (USA). The company will hold one-to-one meetings with pharmaceutical companies and global investors. Click on link for more info about the Chardan's 6th Annual Genetic Medicines Conference

Oryzon will participate at the MSD European Business Development Outreach, which will be held on October 5 in Zurich (Switzerland). The company will hold one-to-one meetings with pharmaceutical companies and global investors.

Oryzon will participate at the Iberian Digital Forum on October 6-7, where the company will hold one-to-one meetings with national investors.

Oryzon will participate at the AACR Special Conference: Cancer Epigenomics in Washington (USA) on October 6-8, where the company will present a poster communication entitled: ASCL1 and SOX2 expression levels predict sensitivity to LSD1 inhibition with iadademstat in small cell lung cancer on October 7 at 18:00 ET. Click on link for more info about the AACR Special Conference: Cancer Epigenomics

Oryzon has been invited to the European Brain Council (EBC)s annual European congress, Brain Innovation Days, which will be held on October 11-12 in Brussels (Belgium). The company will take part in a panel discussion entitled Innovative Funding Models, taking place on October 12 at 14:35 CEST. Click on link for more info about the Brain Innovation Days

Oryzon has been invited to the HealthTech Innovation Days, which will be held on October 13-14 in Paris (France). The company will participate at a round table devoted to age-related diseases on October 13 at 11:00 CEST. Click on link for more info about the HealthTech Innovation Days

Oryzon will attend BIO-Europe 2022, which will be held in Leipzig (Germany) and virtually on October 24-26, where the company will provide a corporate update and will also hold one-to-one meetings with pharmaceutical companies and global investors. Click on link for more info about BIO-Europe 2022

Finally, Oryzon will participate at the 34th EORTC-NCI-AACR Symposium, which will be held on October 26-28 in Barcelona (Spain). The company will present two poster communications entitled Iadademstat effects on neuroendocrine, inflamed and mesenchymal gene expression patterns in small cell lung cancer subtypesand Iadademstat and gilteritinib synergistically abrogate viability of both treatment-nave and drug-resistant AML cellson October 27. Click on link for more info about the 34th EORTC-NCI-AACR Symposium

About OryzonFounded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European leader in epigenetics. Oryzon has one of the strongest portfolios in the field, with two LSD1 inhibitors, iadademstat and vafidemstat, in Phase II clinical trials, and other pipeline assets directed against other epigenetic targets. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit http://www.oryzon.com

About Iadademstat Iadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). In an ongoing, fully-accrued Phase IIa trial in elder 1L-AML patients (ALICE trial), iadademstat has shown encouraging safety and efficacy data in combination with azacitidine (see Salamero et al., EHA 2022 poster). The company has obtained approval from the U.S. FDA for its IND for FRIDA, a Phase Ib trial of iadademstat plus gilteritinib in patients with relapsed/refractory AML with FLT3 mutations. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). New trials in combination in SCLC and NET are under preparation. Oryzon has recently entered into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI) to collaborate on potential further clinical development of iadademstat in different types of solid and hematological cancers. In total iadademstat has been dosed so far to more than 100 cancer patients in four clinical trials. Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU.

About Vafidemstat Vafidemstat (ORY-2001) is an oral, CNS optimized LSD1 inhibitor. The molecule acts on several levels: it reduces cognitive impairment, including memory loss and neuroinflammation, and at the same time has neuroprotective effects. In animal studies vafidemstat not only restores memory but reduces the exacerbated aggressiveness of SAMP8 mice, a model for accelerated aging and Alzheimers disease (AD), to normal levels and also reduces social avoidance and enhances sociability in murine models. In addition, vafidemstat exhibits fast, strong and durable efficacy in several preclinical models of multiple sclerosis (MS). Oryzon has performed two Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders (REIMAGINE) and in aggressive/agitated patients with moderate or severe AD (REIMAGINE-AD), with positive clinical results reported in both. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL trial in patients with Mild to Moderate AD, where a significant reduction of the inflammatory biomarker YKL40 has been observed after 6 and 12 months of treatment, and the pilot, small scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS, where antiinflammatory activity has also been observed. Vafidemstat has also been tested in a Phase II in severe Covid-19 patients (ESCAPE) assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection, where it showed significant anti-inflammatory effects in severe Covid-19 patients. Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically-defined patient subpopulations of certain CNS disorders and is preparing a clinical trial in Kabuki Syndrome patients. The company is also exploring the clinical development of vafidemstat in other neurodevelopmental syndromes.

FORWARD-LOOKING STATEMENTSThis communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words expects, anticipates, believes, intends, estimates and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisin Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forwardlooking statements, whether as a result of new information, future events or otherwise. This press release is not an offer of securities for sale in the United States or any other jurisdiction. Oryzons securities may not be offered or sold in the United States absent registration or an exemption from registration. Any public offering of Oryzons securities to be made in the United States will be made by means of a prospectus that may be obtained from Oryzon or the selling security holder, as applicable, that will contain detailed information about Oryzon and management, as well as financial statements.

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ORYZON to Give Updates on Corporate Progress in October - BioSpace

The human genome consists of more than 6 billion letters. Each person has a unique configuration of A, C, G, and T the molecular building blocks that make up DNA. Determining the sequence of all these letters used to require a huge amount of money, time, and effort. Thousands of researchers worked on the Human Genome project for 13 years. Its value is $2.7 billion.

In 1990, this project ushered in the era of genomics helping scientists unravel the genetic factors of cancer and many hereditary diseases, as well as has stimulated the development of home DNA tests. Next, researchers began to sequence more genomes: animals, plants, bacteria, and viruses. Ten years ago, sequencing a human genome cost researchers about $10,000. A few years ago, that price dropped to $1,000. Today, its about $600. And from now on, sequencing will become even cheaper.

At the branch event in San Diego, a genomics giant Illumina company introduced what it calls its fastest and most efficient sequencing machines, the NovaSeq X series.

The company, which controls about 80% of the global DNA sequencing market, believes that its new technology will reduce the cost to $200 per human genome while providing twice the reading speed.

Francis deSouza, CEO of Illumina says that a more powerful model will be able to sequence 20,000 genomes per year; the companys current equipment can sequence about 7,500 genomes. Illumina will start selling the new instruments today and ship them next year.

As we look to the next decade, we believe were entering the era of genomic medicine going mainstream. To do that requires the next generation of sequencers, deSouza says. We need price points to keep coming down to make genomic medicine and genomic tests available much more broadly.

Sequencing has led to genetically targeted drugs, blood tests that can detect cancer in its early stages, and diagnostics for people with rare diseases. We can thank sequencing for vaccines against Covid-19, which scientists began developing in January 2020, as soon as the first sample of the viruss genome was created.

In research laboratories, this technology has become indispensable for a better understanding of pathogens and human evolution. But in medicine, it is still not used everywhere, which is partially connected to the price. While sequencing costs about $600 for scientists, clinical interpretation and genetic counseling can bring the price to several thousand dollars for patients.

Another reason is that for healthy people, there is insufficient evidence that genome sequencing will be worth the money spent. Now the test is mainly used by people with certain types of cancer or undiagnosed diseases. Although in two recent studies, about 12-15% of healthy people whose genomes were sequenced ended up having a genetic variation that put them at increased risk for a disease that could be cured or prevented, indicating that sequencing could provide early prevention of diseases.

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NovaSeq X machines will make genetic analysis even faster and cheaper - Mezha.Media

LONDON, Sept. 30, 2022 /PRNewswire/ --e-Therapeutics plc(AIM: ETX) (OTC-QX: ETXPF), a company integrating computational power and biological data to discover life-transforming RNAi medicines, is pleased to announce a fundraise of 13.5 million before expenses by way of a subscription for new ordinary shares of 0.1p each ("Ordinary Shares") in the Company (the "Subscription") at a price of 20p per Ordinary Share by funds managed by M&G Investment Management Limited ("M&G"), an institutional investor and an existing shareholder of the Company.

The fundraise provides ETX with the opportunity to generate value and accelerate the next stage of its growth, advancing the Company's position in creating an entirely new template for drug discovery using computation to capture and model disease complexity, identify novel targets and design RNAi drugs against those targets that can be rapidly progressed to the clinic.

The net proceeds of the Subscription will be used to facilitate a number of initiatives to accelerate growth, with a focus on expanding the Company's in-house pipeline of first-in-class RNAi candidatesderived from ETX's computational platform; further developing cell type-specific computational tools and datasets; and general working capital including additional headcount.

Ali Mortazavi, Chief Executive Officer of e-therapeutics, commented:

"I am pleased to announce the fundraise of 13.5 million and excited by the prospect of being able to accelerate the development of our in-house RNAi pipeline through enhanced investment in our therapeutic programmes, hepatocyte datasets and computational capabilities. This successful fundraise underlines ETX's position at the intersection of computational approaches to drug discovery and genetic medicine, using RNA interference as our drug modality of choice. We are grateful for the support of our shareholders and look forward to delivering value from our platform technologies."

Michael Stiasny, Head of UK Equities at M&G Investments, commented:

"With the potential to re-shape the conventional drug discovery model, siRNA based therapies represent an extremely exciting new modality in medicine. We believe that e-therapeutics has a unique platform and strategically attractive IP in this space, combined with a strong computational edge, and are delighted to be increasing our long-term support for the Company."

The issue of new Ordinary Shares pursuant to the Subscription will be conditional on (i) the 67,500,000 shares ("Subscription Shares") being admitted to trading on AIM by not later than 8.00 a.m. on 6 October 2022, or such later time and/or date as the Company may agree (being not later than 8.00 a.m. on 14 October 2022) and (ii) the representations and warranties of the Company under the Subscription being true and accurate.

Highlights of the Fundraise

Company Overview

The latest information on the Company and its recent progress is included in its Interim Report for the 6 months to 31 July 2022 which is also being announced today.

ETX is a UK-based company integrating computational power and biology information to discover life-transforming RNAi medicines.The Company's technology uses computation tocapture and model human biology, identify novel targets and design RNAi medicines against those targets that can be rapidly progressed to the clinic.

ETX's proprietary Computational Biology Platform enables the generation and analysis of biological network models, providinga novel and mechanistic approach to drug discovery that explicitly considers the true complexity of biology and makes more reliable predictions from large complex data sets and ETX's proprietary hepatocyte knowledge base, - the world's most comprehensive and integrated hepatocyte-centric data and information resource. The Company generates, prioritises and tests millions of hypotheses in silico to identify better therapeutic targets with higher confidence.

ETX's proprietary RNAi Platform enables the targeted delivery to hepatocytes in the liver and the specific silencing of novel disease-associated genes, identified by ETX's Computational Biology Platform. The focus on hepatocytes offers the opportunity to work across a wide variety of diseases. The liver is a highly metabolically active organ which performs a key role in many biological processes and vital functions crucial for human health. ETX's GalNAc-siRNA constructs have demonstratedcompelling in vivo performance in terms of depth of gene silencing and duration of action.

ETXis progressing apipeline of first-in-class pre-clinical RNAi candidates in several therapeutic areas including haematology, cardiovascular disease andnon-alcoholic steatohepatitis ("NASH"). ETX has also partnered with biopharma companies such asNovo Nordisk, Galapagos NV and iTeos Therapeuticsusing its computational network biology approach across a diverse range of drug discovery projects.

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e-therapeutics: Fundraise of 13.5 million - BioSpace

Max Jarmey, 27, took part in our latest Live & Ticking event alongside his cardiologist, BHF Professor Hugh Watkins. Professor Watkins spoke about the 30 million research project hes leading, which aims to find a cure for genetic cardiomyopathies. Max talked poignantly about the loss of his dad and the hope this research has injected into his future.

Max Jarmey inherited his dads calm demeanour and reflective nature. He also inherited from him a serious heart condition. But the full extent of this less visible inheritance a type of genetic cardiomyopathy would remain hidden to Max until his dad tragically died aged just 53.

Growing up we knew dad had some heart problems, says Max, but he never told us exactly what it was.

Max has since learnt that his dad, Chris, realised something was wrong when he experienced an arrhythmia a potentially dangerous abnormal heart rhythm when he was 40. Max was a baby at the time.

This scare led to Chris being diagnosed with arrhythmogenic right ventricular cardiomyopathy, or ARVC. If one of your parents has a faulty gene linked to this heart muscle disease, theres a 50 per cent chance it will pass down to you.

Around this time, Chris, who was always very health and diet conscious, stopped running and playing competitive sports. But Max says he never really noticed anything different about his dad.

He was always such a calm and level-headed person, and I understand now that after his diagnosis he focused on what he could do, like yoga and lots of walking, rather than what he couldnt.

Chris also continued doing what he loved for a living. He taught Shiatsu, a type of massage based on traditional Chinese medicine, as well as a meditative kind of Tai Chi, a form of shadowboxing.

He wrote lots of books on these techniques and was very knowledgeable about Chinese medicine, says Max. He also set up the European Shiatsu School that brought the practice to the west.

It all changed, though, on a Sunday afternoon in 2008 when Chris suffered a sudden cardiac arrest, brought on by his ARVC. Max was 13.

Dad had always been a big influence on me, and it was really tough because suddenly I didnt have that father figure to draw on their experience and learn from.

Chriss death set in motion a series of medical tests and screenings over the next five years that ultimately led to Maxs own diagnosis of ARVC.

At 18, his life was upended again.

Max was now one of the 260,000 people in the UK living with an inherited heart muscle disease. Globally, its estimated one in 250 people live with such a condition.

He recalls leaving the hospital after getting the news, sitting in the car with his mum in the carpark when he broke down.

It was dawning on him that, at this age when most of us are finding our place in the world, if he was to try to slow the progression of the disease he would have to give up so many things he loved.

By this stage Max had become an accomplished mountain biker and hockey player. But strenuous sports and activity can often exacerbate the condition or, in some cases, lead to a sudden cardiac arrest.

He also started googling and researching the condition after he was diagnosed. This terrified him.

However, his cardiologist Hugh Watkins, also a British Heart Foundation professor, gently conveyed to Max the precautionary measures needed in order to manage the condition. Professor Watkins words and guidance offered Max some peace of mind amidst his inner turmoil.

Shortly after his diagnosis Max had an ICD fitted a mini defibrillator placed under the skin that shocks your heart back into a normal rhythm if it detects a dangerous abnormal heart rhythm.

His younger brother Tom was also found to have the condition. But his older brother and sister, from their screenings, dont show any signs of it. Genetic testing confirmed that only Max and Tom had inherited the genetic spelling mistake from their dad.

Over the years, Max and Tom have both been shocked by their ICDs many times. Its a painful reminder of how serious their condition is and, indeed, how crucial and lifesaving the little device under their skin is.

Recently, Max heard about another possible lifesaver CureHeart. Its the most ambitious BHF research project in the history of the charity.

Professor Hugh Watkins and his renowned international team have been awarded 30 million their aim is to find a cure for inherited heart muscle diseases such as the condition Max lives with, and the condition that stole his dads life.

The CureHeart team won the award after a rigorous global competition, the BHFs Big Beat Challenge. The best scientists and researchers from across the globe were invited to lay out what they believed to be the next transformational leap in cardiovascular research. And CureHeart won the day.

Its pioneering approach will use ultra-precise gene therapy technologies that could edit or silence the faulty genes that cause these deadly conditions. Put simply, the team endeavour to correct the spelling mistakes found in these faulty genes.

This is our once-in-generation opportunity to relieve families of the constant worry of sudden death, heart failure and potential need for a heart transplant, says Professor Watkins.

The 30 million from the BHF will give us the platform to turbo-charge our progress in finding a cure so the next generation of children diagnosed with genetic cardiomyopathies can live long, happy and productive lives.

Within years, its possible that a simple injection given to someone living with ARVC or another type of inherited heart muscle disease will cure them of the condition.

For Max who now works as a product manager for a financial services firm and is in a loving relationship worry and fear has given way to a sense of optimism.

When I think about my future, the decision to have children and their future, CureHeart could make that decision easier. My children might never have to suffer like I have with this condition. This project gives me hope. Thats what the BHF is funding hope.

Beyond everything else, though, May says: All I really want to do is live a normal life and just go for a run.

LEARN MORE FROM PROFESSOR HUGH WATKINS AND MAX JARMEY

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The inheritance of hope curing genetic heart disease now within grasp - British Heart Foundation