Monthly Archives: April 2022

SOMERVILLE, Mass.--(BUSINESS WIRE)--Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as GENE WRITING technology, announced today that it has raised over $300 million in Series C financing. Investors included a wholly-owned subsidiary of the Abu Dhabi Investment Authority (ADIA); Alaska Permanent Fund Corporation; Altitude Life Science Ventures; ARTIS Ventures; Cormorant Asset Management; Tesseras founder, Flagship Pioneering; Hanwha Impact Partners; Longevity Vision Fund; March Capital; SALT Fund; SoftBank Vision Fund 2; funds and accounts advised by T. Rowe Price Associates, Inc., and others including all of Tesseras existing institutional shareholders.

We are thankful for the support from our new partners and existing investors alike in this latest funding round. It is our belief that genetic medicine will be the most important next epoch in medicineoffering the ability to cure genetic diseases and to someday even prevent disease from occurring, said Geoffrey von Maltzahn, Ph.D., Co-Founder, Chief Executive Officer, and Board Director of Tessera Therapeutics. Todays announcement will help us realize the promise of GENE WRITING technology and our mission of curing disease by writing in the code of life.

While there have been many advancements in the area of genetic medicine over the past decade, Tesseras GENE WRITING platform is charting an entirely new courseone that aims to revolutionize genetic medicine as we know it, said Noubar Afeyan, Ph.D., Co-Founder and Chairman of Tessera Therapeutics and Founder and CEO of Flagship Pioneering. This latest funding round is a testament to the immense potential of this bioplatform to provide new treatments and cures to previously untreatable genetic diseases.

Tesseras GENE WRITING technology is designed to cure disease by writing in the code of life. The GENE WRITING platform can change any base pair to any other, make small insertions or deletions, and write entire genes into the genome with delivery of only RNA. This unlocks the potential to cure nearly any genetic disease, create life-changing medicines for other serious conditions such as cancer, and prevent illnesses with curative, scalable, and easily administered genetic medicines that could become a new modality in human healthcare.

GENE WRITING technology is inspired by natures genome architectsMobile Genetic Elements (MGEs)to overcome the limitations of gene editing and gene therapies. MGEs evolved to write new sequences of DNA into the genome, in contrast to nucleases like CRISPR which evolved to destroy DNA. Therefore, MGEs offer the potential for immense impact in genetic medicine by writing short and long therapeutic sequences of DNA into human cells. Tessera has designed, built, and tested tens of thousands of engineered and synthetic MGEs to create programmable GENE WRITING systems that can write and rewrite the genome with high-efficiency, specificity, and fidelity.

The Tessera team has developed a remarkable GENE WRITING platform that confers unique advantages over alternative genetic medicine technologies, including base editing, CRISPR, and gene therapies, said Vali Barsan, M.D., of SoftBank Investment Advisers. Having worked closely with the company over the past year, its hugely exciting to observe GENE WRITING catalyze a new category of genetic medicine.

For more information on Tessera Therapeutics, including how GENE WRITING technology works, partnership opportunities, and job openings, please visit http://www.tesseratherapeutics.com.

About Tesseras GENE WRITER Technology

Tesseras GENE WRITER tools are based on natures genome architects, Mobile Genetic Elements (MGEs)the most abundant class of genes across the tree of life, representing approximately half of the human genome. Tessera has evaluated tens of thousands of natural and synthetic MGEs to create GENE WRITER candidates with the ability to write therapeutic messages into the human genome. Tesseras research engine further optimizes the discovered GENE WRITER candidates for efficiency, specificity, and fidelityessentially compressing eons of evolution into a few months.

About Tessera Therapeutics

Tessera Therapeutics is pioneering GENE WRITING technology, which consists of multiple technology platforms designed to offer scientists and clinicians the ability to write therapeutic messages into the human genome, thereby curing diseases at their source. The GENE WRITING platform allows the correction of single nucleotides, the deletion or insertion of short sequences of DNA, and the writing of entire genes into the genome, offering the potential for a new category of genetic medicines with broad applications both in vivo and ex vivo. Tessera Therapeutics was founded by Flagship Pioneering in 2018, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability. For more information about Tessera, please visit http://www.tesseratherapeutics.com.

About Flagship Pioneering

Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in more than $140 billion in aggregate value. To date, Flagship has deployed over $2.6 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 42 transformative companies, including Axcella Therapeutics (NASDAQ: AXLA), Codiak Biosciences (NASDAQ: CDAK) Denali Therapeutics (NASDAQ: DNLI), Evelo Biosciences (NASDAQ: EVLO), Foghorn Therapeutics (NASDAQ: FHTX), Indigo Ag, Moderna (NASDAQ: MRNA), Omega Therapeutics (NASDAQ: OMGA), Rubius Therapeutics (NASDAQ: RUBY), Sana Biotechnology (NASDAQ: SANA), Seres Therapeutics (NASDAQ: MCRB), and Sigilon Therapeutics (NASDAQ: SGTX).

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Tessera Therapeutics Announces Over $300M Series C Financing to Advance its GENE WRITING Platform - Business Wire

LONDON, April 25, 2022 -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead gene therapy obecabatagene autoleucel (obe-cel), a CD19-directed autologous chimeric antigen receptor (CAR) T therapy that is being investigated in the ongoing FELIX Phase 2 study of adult relapsed / refractory B-Acute Lymphocytic Leukemia (ALL).

The FDA grants RMAT designation to drug candidates in recognition of the therapy's potential to address significant unmet medical needs in patients with serious or life-threatening conditions. Similar to Breakthrough Therapy designation, RMAT designation provides a number of important benefits in the drug development process, including early interactions with the FDA and other actions to expedite development and review.

"We are very proud to have received RMAT designation, which is an important regulatory milestone for obe-cel," said Dr. Christian Itin, Chief Executive Officer of Autolus. "This supports our belief that obe-cel has the potential to address a serious unmet medical need. It will enable us to further optimize our development and regulatory process and desire to bring this potentially curative therapy to patients as quickly as possible."

obe-cel has previously been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom.

# # #

About Autolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the Company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information, please visit http://www.autolus.com.

About obe-cel (AUTO1)

Obe-cel is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, obe-cel may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. In collaboration with Autolus' academic partner, UCL, obe-cel is currently being evaluated in a Phase 1 clinical trialsfor B-NHL. Autolus has progressed obe-cel to the FELIX trial, a potential pivotal trial for adult ALL.

About obe-celFELIX clinical trial

Autolus' FELIX Phase 1b/2 clinical trial of obe-cel is enrolling adult patients with relapsed / refractory B-precursor ALL. The trial had a Phase 1b component prior to proceeding to the single arm, Phase 2 clinical trial. The primary endpoint is overall response rate, and the secondary endpoints include duration of response, MRD negative CR rate and safety. The trial is designed to enroll approximately 140 patients across 34 of the leading academic and non-academic centers in the United States, United Kingdom and Europe. [NCT04404660]

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus' development of the obe-cel program; the future clinical development, efficacy, safety and therapeutic potential of its product candidates, including progress, expectations as to the reporting of data, conduct and timing and potential future clinical activity and milestones; expectations regarding the initiation, design and reporting of data from clinical trials; expectations regarding regulatory approval process for any product candidates; the collaboration between Autolus and Blackstone; the discovery, development and potential commercialization of potential product candidates including obe-cel using Autolus' technology and under the collaboration agreement; the therapeutic potential for Autolus in next generation product developments of obe-cel in B-cell malignancies; the potential and timing to receive milestone payments and pay royalties under the strategic collaboration; and the Company's anticipated cash runway. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, the risks that Autolus' preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results; the cost, timing and results of clinical trials; that many product candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; possible safety and efficacy concerns; and the impact of the ongoing COVID-19 pandemic on Autolus' business. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Autolus' actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 10, 2022, as well as discussions of potential risks, uncertainties, and other important factors in Autolus' subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Autolus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Contact:

Olivia Manser

+44 (0) 7780 471568

o.manser@autolus.com

Julia Wilson

+44 (0) 7818 430877

j.wilson@autolus.com

Susan A. Noonan

S.A. Noonan Communications

+1-917-513-5303

susan@sanoonan.com

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Syncona : FDA Grants Regenerative Medicine Advanced Therapy (RMAT) designation to Autolus' CAR T cell therapy, obe-cel, for the treatment of adult...

Rowan Callahan, Elias Spiliotopoulos,and Thuy Ngo, Ph.D, inside their lab at OHSU Knight Cancer Research Building on Friday, April 22, 2022. (OHSU/Christine Torres Hicks)

Oregon Health & Science University scientists have devised an accurate and sensitive way to test blood to see if a pre-cancerous condition is escalating to outright cancer potentially enabling treatment early in tumor development when cancer is more likely to be curable.

The OHSU researchers focused on two common conditions that can precede highly lethal cancers: liver cirrhosis, which boosts the risk of liver cancer, and a precursor to the blood cancer multiple myeloma called MGUS, which stands for monoclonal gammopathy of undetermined significance.

If the findings hold up in larger clinical studies, they set the stage for a simple, affordable method to screen people at risk by taking a small blood sample a few times a year.

Our biomarker may recapitulate the transition to cancer, says Thuy Ngo, Ph.D., who led the research. Ngo is an assistant professor of molecular and medical genetics in the OHSU School of Medicine and a member of CEDAR, the OHSU Knight Cancer Institutes Cancer Early Detection Advanced Research Center.

Ngos team reported the findingstoday in the journal NPJ Precision Oncology.

Many competing groups are developing blood tests for cancer early detection, but most are focused on finding cancers in a general population. Ngo and coauthors say there is an unmet need for a blood test that can identify patients with pre-malignant conditions who require further intervention due to a higher likelihood of cancer incidence. In the case of cirrhosis and MGUS, some will progress to cancer, but many others will not. There currently is no reliable way to predict which cases will progress.

The blood test devised by Ngo and colleagues looks for molecules called messenger RNA, which convey instructions from genes. Each RNA molecule is built of chemical units arranged in a single strand like letters of an alphabet that spell out the message of a gene.

RNA routinely escape from cells and circulate in the blood, but unprotected, the free molecules quickly degrade; this makes it technically challenging to read the sequences of a mass of cell-free RNA collected from blood and make sense of it.

A few years ago, not many people believed cell-free messenger RNA could be reliably detected in the blood because it is prone to degradation, Ngo says. We found a way to handle it, and we are among the first to apply it in cancer and pre-cancer early detection.

In the new research, the team took blood samples from eight people with liver cancer, 10 with multiple myeloma, four with liver cirrhosis, eight with MGUS, and 20 non-cancer donors. They painstakingly sequenced the RNA from the samples to obtain a view of gene activity represented by the messenger molecules recovered from blood.

From this, they were able to identify patterns of gene activity in the different groups of people and build models to distinguish those with cancer from those with pre-malignant conditions, and from those without cancer.

The models were able to distinguish multiple myeloma blood samples from non-cancer samples with 90% accuracy, and multiple myeloma from its pre-malignant condition with 100% accuracy. They were able to distinguish liver cancer samples from non-cancer with 93 to 100% accuracy, and liver cancer from cirrhosis with 100% accuracy.

Experiments showed that the level of the cell-free RNA biomarkers displayed a gradual transition from non-cancerous states through pre-cancerous conditions and cancer. The researchers validated the biomarkers using a set of subjects different from the ones used to develop the models, and they proved highly accurate at distinguishing cancer from non-cancer.

This is promising, but the sample set is small, so we need to be modest here, Ngo cautions. Its still very early stage and further clinical validation will be needed.

The research lays the foundation for developing inexpensive assays that measure levels of cell-free RNA in blood for a small panel of genes that can differentiate cancer from pre-malignant conditions.You dont need any fancy equipment for doing this, Ngo says. Any central lab with standard equipment can do the work. Sophisticated sequencing was only required for the discovery work of identifying the telltale gene signatures.

Ngo and OHSU have filed a patent on their findings, and Ngo says she is talking with a company about a licensing deal to develop the technology. We have several directions to move it forward, she says.

Ngos priority is people at risk for liver cancer. One-quarter to one-third of adults in the U.S. have fatty liver disease, making them vulnerable to cirrhosis and liver cancer. Across the globe, infectious hepatitis puts enormous numbers of people in danger of developing liver cancer. This is why, Ngo says, the world needs a simple, affordable test to identify those at highest risk and monitor their health for the earliest signs of malignant growth.

This research was supported by grants from Oregon Health & Science University (CEDAR3250918), Cancer Research UK/OHSU (C63763/A27122), OCTRI (UL1TR000128), Kuni Foundation, Department of Defense (W81XWH2110853) and Susan G. Komen Foundation (CCR21663959).

Oregon Health & Science University has filed patent applications based on this work. The authors report no other competing interests.

Link:
Blood test could reveal transition to cancer in people at risk - OHSU News

Scientists from the United Kingdom may have identified several more environmental causes for cancer after evaluating the genetic data of over 12,000 cancer patients from National Health Servicefiles.

In arguably thelargest studyof its kind, researchers at Cambridge University Hospitals and the University of Cambridgelooked into whole-genome sequencing data of 12,222 patients to detect possible patterns in the DNA of cancer patients and succeeded. They found 58 new "mutational signatures," or patterns that offer clues on whether a particular patient had been exposed to certain environmental elements which led to cancer. These included cellular malfunctions, smoking and exposure to UV light.

Data were taken from the NHS's 100,000 Genomes Project, Genomics England's massivegenome sequencing projectbased on data from more than 85,000 patients diagnosed with cancer or rare diseases.

The research,publishedinScience, featured a new method the Signature Fit Multi-Step (FitMS) algorithm to enhance discrimination of common mutational processes from the rare, lower-frequency mutagenic processes. FitMS looks at signatures from new samples and compares these with existing findings by monitoring commonalities and identifying additional rare signatures.

The set of reference signatures used was identified after comparing and contrasting independent tissue-specific signatures and clustering mutational signatures from various tissues that may be due to similar processes. Other mutation bases were also linked to past clinical and treatment histories, where applicable.

"Whole genome sequencing gives us a total picture of all the mutations that have contributed to each person's cancer. With thousands of mutations per cancer, we have unprecedented power to look for commonalities and differences across NHS patients, and in doing so we uncovered 58 new mutational signatures and broadened our knowledge of cancer," said Dr. Andrea Degasperi, the study's first author and a research associate at the University of Cambridge, in a statement.

"The reason it is important to identify mutational signatures is because they are like fingerprints at a crime scene - they help to pinpoint cancer culprits. Some mutational signatures have clinical or treatment implications - they can highlight abnormalities that may be targeted with specific drugs or may indicate a potential 'Achilles heel' in individual cancers," added Serena Nik-Zainal, leader of the study and a professor of genomic medicine and bioinformatics at the University of Cambridge.

Cancer is a complex beast and severalstudies on mutationsalso came to light at the American Association for Cancer Research's annual meeting in mid-April 2022. Aadi Biosciencegot the U.S. Food and Drug Administration's green light for Fyarro, a new drug designed to treat PEComa, a very rare type of sarcoma. Fyarro has demonstrated the ability to treat patients with TSC1 or TSC2 mutations.

Ikena Oncologyalso presented data on an ongoing trial for IK-930. This TEAD inhibitor targets the Hippo signaling pathway and binds to TEAD transcription factors with the goal of preventing the transcription of genes that drive the progression of cancer.

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UK Researchers Uncover 58 New Mutational Signatures of Cancer - BioSpace

A team of US researchers have found a way to diagnose skin cancer using blood tests.

The researchers have shown in a lab-based study that melanoma cells can be detected in blood and plasma. If the test makes it through clinical trials, the researchers hope that it could one day be used to sidestep the invasive biopsies that are currently required to diagnose melanoma.

The test uses melanoma-specific antibodies, and a device designed specifically to react them with blood. The device is called MelanoBean, and it works with microfluidics: manipulating tiny amounts of fluid to do interesting things that they wouldnt do in larger volumes.

The test is described in a paper in Advanced NanoBiomed Research.

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This is the first comprehensive study of circulating tumour cells or CTCs to evaluate the efficacy of surgery using microfluidic systems in melanoma, including changes in the number of CTCs, CTC cluster configuration, and gene expression profiling, says first author Dr Yoon-Tae Kang, a researcher at the University of Michigan, US.

Read more: New cancer detection technique using old oranges

The researchers found that with their test, melanoma cells (CTCs) could be found in the blood of cancer patients at all stages of the disease I through to IV.

It could also identify whether any CTCs were hanging around in the blood of patients whod had skin cancer surgery to get their cells removed.

CTCs have the potential to pinpoint treatment resistance and recurrence, and can be a valuable biomarker to non-invasively monitor for disease progression, says corresponding author Dr Sunitha Nagrath also from the University of Michican.

Theres never been a more important time to explain the facts, cherish evidence-based knowledge and to showcase the latest scientific, technological and engineering breakthroughs. Cosmos is published by The Royal Institution of Australia, a charity dedicated to connecting people with the world of science. Financial contributions, however big or small, help us provide access to trusted science information at a time when the world needs it most. Please support us by making a donation or purchasing a subscription today.

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Detecting skin cancer with a blood test - Cosmos

Las Vegas, USA, April 25, 2022 (GLOBE NEWSWIRE) -- HER2 negative Breast Cancer Pipeline Insights | Research Report 2022 by DelveInsight

HER2 negative Breast Cancer Pipeline involves 85+ key companies continuously working towards developing 85+ HER2 negative Breast Cancer treatment therapies, as per DelveInsight

DelveInsights HER2 negative Breast Cancer Pipeline Insight 2021 report offers exhaustive global coverage of available, marketed, and pipeline therapies in different phases of clinical development, major pharmaceutical companies working to advance the pipeline space, and future growth potential of the HER2 negative Breast Cancer pipeline domain.

Some of the essential takeaways from theHER2 negative Breast Cancer Pipelinereport:

Request a sample and discover more about the report offerings @ HER2 negative Breast Cancer Emerging Therapies

The HER2 negative Breast Cancer pipeline report lays down detailed profiles of the pipeline assets, comparative analysis of clinical and non-clinical stage HER2 negative Breast Cancer products, inactive and dormant assets, comprehensive assessment of driving and restraining factors, as well as the opportunities and risks in the HER2 negative Breast Cancer pipeline landscape.

HER2 negative Breast Cancer Overview

Most Breast Cancers express the Estrogen Receptor (ER) receptor and are negative for the human epidermal growth factor receptor 2 (HER2) receptor. ER+/ HER2 ve Breast Cancer includes tumors that are ER-positive and PR positive, but negative for HER2. ER+/ HER2 ve Breast Cancer is heterogeneous and accounts for about 70% of all breast cancers. HER2 stands for human epidermal growth factor receptor 2. There are various types of breast cancer, some have hormone receptors like estrogen or progesterone (some have both) and are called ER+ or PR+ breast cancer respectively.

Find out more about the disease and recent developments @ HER2 negative Breast Cancer Pipeline Assessment

HER2 negative Breast Cancer Pipeline Drugs

Learn more about the novel and emerging HER2 negative Breast Cancer pipeline therapies @ HER2 negative Breast Cancer Pipeline Analysis

HER2 negative Breast Cancer Therapeutics Assessment

TheHER2 negative Breast Cancer Pipelinereport proffers an integral view of the HER2 negative Breast Cancer emerging novel therapies segmented by Stage, Product Type, Molecule Type, Mechanism of Action, and Route of Administration.

Scope of the HER2 negative Breast Cancer Pipeline Report

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Table of Contents

For further information on the HER2 negative Breast Cancer current pipeline therapeutics, reach out @ HER2 negative Breast Cancer Ongoing Clinical Trials

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Interested to know more about the breakthrough happenings? Take a look at the posts below

AboutDelveInsight

DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platformPharmDelve.

For more insights, visit Pharma, Healthcare, and Biotech News

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HER2 negative Breast Cancer Pipeline Insights | Research Report 2022 by DelveInsight - GlobeNewswire

NEW YORK, April 25, 2022 (GLOBE NEWSWIRE) -- Xalud Therapeutics, a clinical-stage biotechnology company developing DNA-delivered therapeutics, today announced the appointment of Amy Chappell, M.D., as an independent strategic advisor. Dr. Chappell will work with Xalud senior management and other advisors to provide strategic input and direction for Xaluds R&D programs, including XT-150, a nonviral DNA-based delivery platform with potential for a broad number of indications.

Amys experience as a physician-scientist, especially with respect to her expertise in pain, will be invaluable to Xalud as we continue to develop XT-150 and advance our novel therapeutic platform for the treatment of chronic inflammatory disorders, which are often accompanied by pain, said Diem Nguyen, Ph.D., MBA, chief executive officer of Xalud.

Dr. Chappell has more than 30 years of experience planning, designing and implementing all stages of clinical trials in neurosciences with expertise in pain, migraine and epilepsy. Dr. Chappell serves on the scientific advisory board of Pinteon Therapeutics, is an adjunct clinical associate professor at the Indiana University School of Medicine and is a fellow of the American Academy of Neurology (FAAN). She spent over 25 years at Eli Lilly and Company, where she started as an associated clinical research physician and took on roles of increasing responsibility until her retirement in 2014 as a senior medical fellow. Dr. Chappell earned her B.S. in biology from Antioch College and her M.D. from Indiana University School of Medicine.

I believe that Xaluds DNA-delivered therapeutic approach holds significant promise for treating a range of inflammatory disorders, Dr. Chappell noted. Im grateful for the opportunity to influence the clinical development of the companys innovative therapies.

About Xalud TherapeuticsXalud Therapeutics is a biotechnology company developing a DNA-delivered therapy platform to treat pathologic inflammation through immune modulation. The company is harnessing the power of interleukin-10 (IL-10), a potent cytokine that acts as a master regulator for multiple inflammatory pathways, to address the root cause of inflammation and subsequently restore homeostasis in the immune system. Xaluds lead product candidate, XT-150, is a locally injectable plasmid DNA gene therapy expressing IL-10v, a proprietary modified variant of IL-10, that addresses pathologic inflammation and pain.

Media Contact

Kara Stephens-WeaverLifeSci CommunicationsKstephens-weaver@lifescicomms.com407-765-1185

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Xalud Therapeutics Adds Amy Chappell, MD, as a Strategic Advisor - BioSpace

Newswise Experts from the University of Nottingham have developed a ground-breaking software, which combines DNA sequencing and machine learning to help them find where, and to what extent, antibiotic resistant bacteria is being transmitted between humans, animals and the environment.

The study, which is published inPLOS Computational Biology, was led by Dr Tania Dottorini from the School of Veterinary Medicine and Science at the University.

Anthropogenic environments (spaces created by humans), such as areas of intensive livestock farming, are seen as ideal breeding grounds for antimicrobial-resistant bacteria and antimicrobial resistant genes, which are capable of infecting humans and carrying resistance to drugs used in human medicine. This can have huge implications for how certain illnesses and infections can be treated effectively.

China has a large intensive livestock farming industry, poultry being the second most important source of meat in the country, and is the largest user of antibiotics for food production in the world.

In this new study, a team of experts looked at a large scale commercial poultry farm in China, and collected 154 samples from animals, carcasses, workers and their households and environments. From the samples, they isolated a specific bacteria calledEscherichia coli (E. coli).These bacteria can live quite harmlessly in a persons gut, but can also be pathogenic, and genome carry resistance genes against certain drugs, which can result in illness including severe stomach cramps, diarrhoea and vomiting.

Researchers used a computational approach that integrates machine learning, whole genome sequencing, gene sharing networks and mobile genetic elements, to characterise the different types of pathogens found in the farm. They found that antimicrobial genes (genes conferring resistance to the antibiotics) were present in both pathogenic and non-pathogenic bacteria.

The new approach, using machine learning, enabled the team to uncover an entire network of genes associated with antimicrobial resistance, shared across animals, farm workers and the environment around them. Notably, this network included genes known to cause antibiotic resistance as well as yet unknown genes associated to antibiotic resistance.

Dr Dottorini said: We cannot say at this stage where the bacteria originated from, we can only say we found it and it has been shared between animals and humans. As we already know there has been sharing, this is worrying, because people can acquire resistances to drugs from two different ways - from direct contact with an animal, or indirectly by eating contaminated meat. This could be a particular problem in poultry farming, as it is the most widely used meat in the world.

The computational tools that we have developed will enable us to analyse large complex data from different sources, at the same time as identifying where hotspots for certain bacteria may be. They are fast, they are precise and they can be applied on large environments for instance multiple farms at the same time.

There are many antimicrobial resistant genes we already know about, but how do we go beyond these and unravel new targets to design new drugs?

Our approach, using machine learning, opens up new possibilities for the development of fast, affordable and effective computational methods that can provide new insights into the epidemiology of antimicrobial resistance in livestock farming.

The research was done in collaboration with Professor Junshi Chen, Professor Fengqin Li and Professor Zixin Peng from China National Center for Food Safety Risk Assessment (CFSA).

The full study can be foundhere.

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Scientists use machine learning to identify antibiotic resistant bacteria that can spread between animals, humans and the environment - Newswise

-Approximately 500 Canadians ages 6-11 are now eligible for PrTRIKAFTA-

-Vertex has submitted this indication to CADTH & INESSS for Health Technology Assessments-

TORONTO, April 20, 2022 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Marketing Authorization for the expanded use of PrTRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With this announcement, approximately 500 Canadians with CF ages 6-11 are now eligible for TRIKAFTA. As a result of this approval, an additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg).

Vertex Pharmaceuticals (Canada) Inc. Logo (CNW Group/Vertex Pharmaceuticals (Canada) Inc.)

"We are delighted that TRIKAFTA is now available for these young patients in Canada. It provides a new treatment option for those with CF ages 6-11 with at least one F508del mutation and a first-in-class treatment option for the approximately 500 6-11-year-olds who are newly eligible for a medicine that treats the underlying cause of their disease," said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. "This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF. We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible."

Vertex completed a 24-week Phase 3 open-label, multicenter study which enrolled 66 children ages 6 through 11 years old with CF who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA. The regimen was generally well tolerated, and safety data were consistent with those observed in previous studies in patients ages 12 years and older.

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"As a trial investigator, I have seen firsthand the demonstrated efficacy of TRIKAFTA in people ages 6-11 living with cystic fibrosis," said Larry C. Lands, M.D., Ph.D., Director, Pediatric Respiratory Medicine, Pediatric Cystic Fibrosis Clinic, and Pediatric Pulmonary Function Laboratory, Montreal Children's Hospital, McGill University Health Center, and Professor, Department of Pediatrics, Faculty of Medicine, McGill University. "This is an exciting next step that will allow eligible patients to begin treatment earlier."

Vertex has also submitted this indication to both the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en sant et en services sociaux (INESSS) in Qubec for Health Technology Assessments.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

About PrTRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. PrTRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways. TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Dr. Kewalramani and Dr. Lands in this press release, and statements regarding the estimated number of children eligible for TRIKAFTA for the first time, our beliefs regarding the benefits of our medicines, and anticipated patient access to TRIKAFTA. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

SOURCE Vertex Pharmaceuticals (Canada) Inc.

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Health Canada Grants Marketing Authorization for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6...

Cell culture, virus, and plasmids

The human kidney cell line (HEK293) used in this study was obtained and cultured as previously described14,15. The swine kidney line L (SK-L) cells were propagated in Eagles Minimum Essential Medium (Nissui Pharmaceutical, Tokyo, Japan) supplemented with 0.3mg/mL l-glutamine (Nacalai Tesque, Kyoto, Japan), 100 U/mg penicillin G (Meiji Seika Pharma, Tokyo, Japan), 8mg/mL gentamycin (TAKATA Pharmaceutical, Saitama, Japan), sodium bicarbonate (Nacalai Tesque), 0.1mg/mL streptomycin (Meiji Seika Pharma), 0.295% tryptose phosphate broth (Becton Dickinson and Company, Franklin Lakes, NJ, USA), 10mMN,N-bis-(2-hydroxyethyl)-2-aminoethanesulfonic acid (BSE; MilliporeSigma, St. Louis, MO, USA), and 10% horse serum (Thermo Fisher Scientific, Waltham, MA, USA).

The vCSFV GPE-/HiBiT recombinant classical swine fever virus encoding the HiBit luciferase gene16 was derived from the recombinant full-length cDNA of the CSFV GPE-strain17. SK-L cells were infected with tenfold serially diluted vCSFV GPE-HiBiT in 96-well plates and incubated at 37C for 3 days. Virus growth was analyzed using luciferase activity as an indicator. Viral titers were calculated and expressed as the tissue culture infectious dose (TCID50) per mL. The luciferase assay was performed according to a previously described protocol18. The luciferase activity of the culture supernatants was measured using a Nano-Glo HiBiT lytic detection system (Promega, Madison, WI, USA) according to the manufacturers protocol. Twenty L of culture medium was mixed with an equal volume of Nano-Glo HiBiT lytic buffer. Luciferase activity was measured in a 96-well LumiNunc plate (Thermo Fisher Scientific) using the microtiter plate reader POWERSCAN 4 (DS Pharma Biomedical, Osaka, Japan). The average number of mock-infected 96-well plates plus five times the standard deviation of this population (i.e., luciferase activity=70) was set as the cutoff value.

The pRF vector containing an FMDV-IRES (serotype C; 5-UTR sequence: nucleotides (nt) 5691038 in FMDV serotype C, AF274010.1)19 was kindly donated by Dr. Hirasawa of the Memorial University of Newfoundland, and those containing a CSFV-IRES20 were gifts from Professor Graham J. Belsham of the University of Copenhagen. The pCAGGS-Neo vector was constructed using the pCAG Neo (Fujifilm Wako, Tokyo, Japan) and pCAGGS vectors (Cat. No. RDB08938; Riken Bank, Ibaraki, Japan). The CSFV-IRES cDNA (nt. 124401) was excised from a reporter plasmid20 using the EcoRI and NcoI restriction sites, and the excised DNA was inserted between the Renilla and firefly luciferase genes. Reporter genes were cut using the restriction endonucleases EcoRV (Toyobo, Osaka, Japan) and BamHI (New England Biolabs, Ipswich, MA, USA). A pCAGGS-Neo/CSFV-IRES vector was generated by inserting a reporter gene into pCAGGS-Neo, which was subsequently treated with EcoRV (Toyobo), BamHI (New England Biolabs), and rAPid alkaline phosphatase (Roche, Basel, Switzerland) using a ligation mixture (Mighty Mix, Takara, Shiga, Japan).

Cells expressing pCAGGS-Neo-CSFV-IRES (clones pCI5 and pCI5-1) and pCAGGS-Neo-FMDV-IRES (clones B5 and B10) were established as described previously15.

DNA sequencing was performed by FASMAC Co. (Kanagawa, Japan), and DNA sequence characterization was performed using the GENETYX-Mac software (GENETYX Co., Tokyo, Japan) and GENBANK.

Cell viability was evaluated using WST assays (Dojindo, Kumamoto, Japan) by determining the optical density at 450nm (OD450) according to the manufacturers instructions. Luciferase assays were performed using a dual-luciferase reporter assay system (Promega, Madison, WI, USA). Luminescence was measured using a GloMax 96 Microplate Luminometer (Promega) for 10s, as described previously14.

Total RNA was extracted from PYC-treated (10g/mL, 72h) and untreated B10 cells using ISOGEN (Nippon Gene Co. Tokyo, Japan) from cells growing in the linear phase of PYC treatment. RNA quality was measured using an Agilent 2100 bioanalyzer and showed an RNA integrity number (RIN) of 9.8 (7.0

The amounts of PKD1L3 and USP31 mRNA in cells were quantified using the SYBR Green real-time PCR master mix (Thermo Fisher, Waltham, MA, USA) and the primers pKD1L3-544S: 5-CATCTTCCAACCACATGTCACTATCC-3, pKD1L3-903AS: 5-CTGTAGTTTGTTAAGAGCTTGCAAACC-3; USP31-700S: 5- TGTGGCTTTTGGACCGAGTTGC-3, and USP31-900AS: 5- TGCAGTGAGAACATTTGCCTGC-3. The data was evaluated using the 2Ct method.

The siRNAs targeting host factors (summarized in Table 2) were designed using the BLOCK-iT RNAi Designer (Thermo Fisher Scientific, Waltham, MA, USA). For the control siRNA, an ON-target plus siRNA control (Horizon/Dharmacon, Lafayette, CO, USA) was used. Then, siRNA (5nM) reverse transfection was performed using the Lipofectamine RNAiMAX reagent (Invitrogen) according to the manufacturers specifications. The effect of siRNA was evaluated by immunoblot analysis as described previously14 using anti-polycystic kidney disease 1-like 3 (PKD1L3) (OSP00014W, Invitrogen) and anti-ubiquitin-specific peptidase 31 (USP31) (Santa Cruz Biotechnology Co.) antibodies. Original blots presented in the supplementary original gel image_Fig. 5 which shows fuller-length of both sides and bottom, but top stacking part gel was removed.

All data are presented as meanstandard deviation (S.D.) from three independent experiments, and figures were generated using GraphPad Prism (version 9) software. Statistical analysis was performed using Students t-test to evaluate significant differences. Statistical significance was set at P<0.05.

This study was performed in accordance with institutional committee protocols of Kagoshima University.

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Characterization of host factors associated with the internal ribosomal entry sites of foot-and-mouth disease and classical swine fever viruses |...