Category Archives: Genetic medicine

Very sick babies and children will be diagnosed and start treatment more quickly thanks to a revolutionary new genetic testing service being launched by the NHS.

Doctors will gain vital insights within as little as two days into what illnesses more than 1,000 newborns and infants a year in England have from the rapid analysis of blood tests.

Until now, when doctors suspected a genetic disorder, such tests have sometimes taken weeks as they had to be done in a sequential order to rule out other possible diagnoses, delaying treatment.

NHS England bosses say the service could save the lives of thousands of seriously ill children over time and will usher in a new era of genomic medicine.

The clinical scientists, genetic technologists and bioinformaticians will carry out much faster processing of DNA samples, including saliva and other tissue samples as well as blood. They will share their findings with medical teams and patients families.

They will undertake whole genome sequencing in a quest to identify changes in the childs DNA and so diagnose conditions such as cancer and rare genetic disorders.

While such testing is available in parts of other countries such as the US and Australia, NHS England said that its new service will be the first in the world to cover an entire country. Wales also has a similar service but it is more limited in its scope than the new service in England.

This global first is an incredible moment for the NHS and will be revolutionary in helping us to rapidly diagnose the illnesses of thousands of seriously ill children and babies, saving countless lives in the years to come, said Amanda Pritchard, NHS Englands chief executive.

The new national rapid whole genome sequencing service will be based in Exeter as part of the NHSs existing Genomic Medicine Service which is based there. It follows a successful trial in some parts of England.

Dr Emma Baple, who is running the new service, said it will transform how rare genetic conditions are diagnosed. It is a new national test being offered with results delivered inside seven days as compared to a much longer turnaround time.

It is the only test in the NHS that looks at all 22,000 genes in the human genome and all the parts in between the genes. Eighty-five per cent of all changes that lead to disease are in the genes themselves, whilst the rest is caused by the bits of DNA in between.

Test results should be available in anything between two and seven days, depending on the complexity of the childs condition, though that should get faster as technology improves, Baple added.

We know that with prompt and accurate diagnosis conditions could be cured or better managed with the right clinical care, which would be life-altering and potentially life-saving for so many seriously unwell babies and children.

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New NHS genetic testing service could save thousands of children in England - The Guardian

Vertex Pharmaceuticals is trying again at developing a drug for the rare, genetic disease alpha-1 antitrypsin deficiency after two earlier efforts fell short.

The biotech announced Tuesday that it has received clearance from the Food and Drug Administration to begin a clinical trial testing a new treatment for the condition, which is caused by the misfoldingof a protein called AAT and damages the lungs and liver of affected individuals.

The study, which will enroll healthy volunteers, is a Phase 1 trial designed to measure the drugs safety and find an appropriate dose for further testing.

In addition, Vertex said it will also begin a nearly year-long Phase 2 study of an earlier drug that wasnt potent enoughto advance into late-stage testing.While technically positive, the magnitude of treatment benefit observed in a mid-stage trial last year wasnt as large as Vertex was looking for. Further analysis of the study showed the drug reduced levels of a liver polymer in the blood of patients, however.

The new study will assess the effect of longer-term treatment on polymer clearance and whether it also yields greater increases in functional AAT protein.

By simultaneously advancing new, more potent molecules into the clinic and assessing the impact of longer-duration treatment, we expect to have both the assets and the data necessary to progress our AAT corrector program in 2023, said David Altshuler, Vertexs head of research and chief scientific officer, in the companys Tuesday statement.

Alpha-1 antitrypsin deficiency is one of several conditions Vertex has targeted in its efforts to build a pipeline of medicines that go beyond its historical focus on cystic fibrosis. While the drugs Vertex has made for the lung disease are clinically and commercially successful, pressure has increased for the company to prove it can do the same elsewhere.

It has some recent successes in that quest, reporting positive results for drugs to treat pain, kidney disease and Type 1 diabetes. Those findings have helped Vertex shares climb to all-time highs this year. Alpha-1 antitrypsin deficiency has been harder, though. Vertex stopped testingits first compound for the disease after reports of an early safety signal.

"Given the performance of the previous-gen AAT molecules, we think this is still a show-me story where we need to see strong efficacy data before making any conclusions," wrote Michael Yee, an analyst at Jefferies, in a Tuesday note to clients.

Vertex is also nearing submission of approval applications for a gene editing medicine its developing with CRISPR Therapeutics for sickle cell disease and beta thalassemia.

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Vertex, after setbacks, moves forward with second-generation rare disease drug - BioPharma Dive

Passage Bio

PHILADELPHIA, Oct. 10, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, today announced the appointment of William Chou, M.D. as chief executive officer (CEO) and a member of the board, effective immediately. Edgar B. (Chip) Cale will resign his position as the companys interim CEO and will continue in his role as general counsel and corporate secretary. Maxine Gowen, Ph.D., will step down as interim executive chairwoman following a brief transition period and will then continue to serve as chairwoman.

The Board and I are delighted to welcome Will to Passage Bio to lead the company through an exciting phase of development, said Dr. Gowen. Wills depth of experience and success in developing and commercializing advanced therapeutics will be instrumental in establishing and solidifying the company as a leader in genetic medicines.

Dr. Chou is an accomplished executive with nearly twenty years of healthcare experience across a range of development and commercialization roles. Most recently, Dr. Chou served as CEO of Aruvant Sciences, a clinical-stage biopharmaceutical company focused on developing gene therapies for rare diseases.

I am thrilled to join the talented team at Passage Bio and build upon the companys many accomplishments and impressive capabilities, said Dr. Chou. With three ongoing clinical programs, we are poised to deliver multiple meaningful milestones over the coming quarters. As a clinician, it is my privilege to lead a company with tremendous potential to bring transformative therapies to patients with CNS disorders for which there are limited or no approved treatment options today.

Prior to joining Aruvant, Dr. Chou served in a variety of leadership roles at Novartis, including vice president, global disease lead for Novartis Cell and Gene Therapy unit where he oversaw the global commercial launch of Kymriah, the first CAR-T cell therapy. Prior to that role, Dr. Chou led the Kymriah lymphoma clinical development program to approvals in the United States, Europe, Australia, Canada and Japan. Before joining Novartis, Dr. Chou worked at the Boston Consulting Group where he focused on commercial and clinical pharmaceutical strategy.

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Dr. Chou holds an M.B.A. from the Yale School of Management, an M.D. from the University of Pittsburgh School of Medicine, and an A.B. in politics and economics from Princeton University. Dr. Chou completed his residency in internal medicine at Yale New Haven Hospital and his fellowship in geriatrics at Yale University.

About Passage Bio

Passage Bio (Nasdaq: PASG) is a clinical-stage genetic medicines company on a mission to provide life-transforming therapies for patients with CNS diseases with limited or no approved treatment options. Our portfolio spans pediatric and adult CNS indications, and we are currently advancing three clinical programs in GM1 gangliosidosis, Krabbe disease, and frontotemporal dementia with several additional programs in preclinical development. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. Through this collaboration, we have enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Passage Bio Investors:Stuart HendersonPassage Bio267-866-0114shenderson@passagebio.com

Passage Bio Media:Mike BeyerSam Brown Inc. Healthcare Communications312-961-2502MikeBeyer@sambrown.com

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Passage Bio Announces Appointment of William Chou, M.D. as Chief Executive Officer - Yahoo Finance

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

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

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

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

Curriculum overview

Our programs curricular goals are to teach our students to:

Our curriculum achieves these goals by providing our students with an integrated student-centered curriculum that incorporates evolving technology in the learning environment. In recognition of the varied opportunities for genetic counselors in todays medical climate, friends of our program include genetic counselors and other health care providers working in industry, practicing telehealth, serving in community and academic settings and making an impact on public policy.

Our course work, thesis component and clinical experiences provide students with a variety of methods to develop the core skills necessary to become highly competent genetic counselors wholl go on to practice in diverse settings. To ensure our graduate program continues to provide the training necessary to meet the demands required of practicing genetic counselors, our faculty, clinical supervisors and advisory board regularly assess our rapidly changing profession and adjust the curriculum accordingly.

Students who enroll in the Ohio State Genetic Counseling Graduate Program are taught by world-renowned professionals from The Ohio State University College of Medicine and Nationwide Childrens Hospital, as well as by other regionally and nationally known clinicians and collaborators. Our fieldwork supervisors and faculty include national leaders in professional organizations. Our students work alongside the very individuals who are helping to shape our field, so you learn the importance of leadership by example.

The Ohio State Genetic Counseling Graduate Program is accredited by the Accreditation Council for Genetic Counseling (ACGC), located at 7918 Jones Branch Drive, Suite 300, McLean, VA 22102. ACGC can be reached at 703-506-3266 or via its website. In 2018, our program underwent re-accreditation and received full accreditation through 2024.

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MS Genetic Counseling | Ohio State College of Medicine

What does a genetic counselor do?

Genetic counselors are health care professionals who have specialized education and training in the field of medical genetics. Using family history, a genetic counselor will assess individual or family risk of an inherited condition, such as a genetic disorder or a birth defect.

Genetic counselors educate patients and professionals about genetic diseases and genetic testing options. They also advise patients on the social and ethical issues associated with a genetic disorder or genetic test result, and help patients cope with a diagnosis of a genetic disease.

As members of the health care team, genetic counselors serve as educators to patients, physicians, other health care providers, and society. In a typical day, genetic counselors:

Genetic counselors might choose to specialize in a particular area or they may provide general care. Some areas they might specialize in include:

Employers of genetic counselors include hospitals, universities, private practices, labs, and a variety of clinical settings. Forty-hour work weeks are typical for genetic counselors and they generally are not required to work evenings or weekends.

Genetic counseling is a great career path for someone that is interested in a rewarding career with a high degree of patient interaction. Due to the limited number of accredited genetic counseling programs, it is recommended that individuals interested in genetic counseling prepare for a highly selective admission process with high school and undergraduate classes in chemistry, biology, genetics, and psychology. Prior experience either through paid work or volunteer experience is recommended (and may be required) when enrolling in the graduate program.

Common highereducation requirements for a genetic counselor include:

To become certified as a genetic counselor, you must complete an accredited masters program in genetic counseling. This program is typically two years in length and includes courses such as molecular genetics, counseling ethics, and research methods; as well as clinical training experience and a research project. After completing the program, students must take and pass a certification exam in order to become a certified genetic counselor.

Genetic counselors typically earn around $80,150 a year. The annual salary depends on their position, level of expertise, and area of the U.S. or world where they practice.

Job growth for genetic counselors in the U.S. is expected to grow much faster than average, according to the Bureau of Labor Statistics. The emphasis on personalized medicine and ongoing technological innovations will increase the demand for genetic counselors who can translate complex medical and scientific information for families and other health professionals.

In terms of career advancement, some genetic counselors become professors, and others find opportunities to conduct and publish research.

Mayo Clinic offers three internships to prepare students for a career as a genetic counselor:

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Genetic Counselor - Mayo Clinic College of Medicine & Science