Category Archives: Gene Medicine

High blood pressure means the force of blood flowing through your arteries is greater than it should be. If not controlled, it could damage your blood vessels and cause other health problems.

High blood pressure (hypertension) tends to be a condition we associate with being too sedentary or getting older. But high blood pressure can also be a genetic condition, affecting people who are otherwise fit and healthy.

A parent with high blood pressure can pass along a gene to a child, raising that persons risk of developing hypertension one day. Familial hypertension may also result from a family lifestyle that includes high blood pressure risk factors, such as smoking or an unhealthy diet.

Blood pressure is the force of circulating blood against the inner wall of your arteries. Its measured in millimeters of mercury (mm Hg) and is presented as two numbers:

According to the American Heart Association, healthy blood pressure is a systolic pressure of less than 120 mm Hg and a diastolic pressure of less than 80 mm Hg. This is a blood pressure of less than 120/80 mm Hg.

If your blood pressure is higher than that, doctors consider you to have elevated blood pressure or stage 1 or 2 hypertension.

Risk factors for high blood pressure include a family history of hypertension, as well as:

What makes high blood pressure so dangerous is that it can exist for a long time without presenting any obvious symptoms. Measuring your blood pressure is the only way to know if you have hypertension.

In extreme cases, when blood pressure exceeds 180/120 mm Hg, you have a medical emergency known as a hypertensive crisis. Symptoms can include:

Research from 2017 suggests that high blood pressure results from a combination of factors, including genetic, environmental, and behavioral components.

Unlike some diseases with only one or a few genes as risk factors, familial hypertension can result from variations in hundreds of different genes, according to a 2019 study of more than 750,000 individuals. This makes it difficult to pinpoint specific genes that could be treatment targets.

The Centers for Disease Control and Prevention (CDC) also notes that families may affect a persons hypertension risk because of the home environment.

Smoking or even breathing in secondhand smoke can raise blood pressure risks. A diet high in sodium and saturated fat may also cause a blood pressure increase. If physical activity and good sleeping habits arent part of a family dynamic, blood pressure can also be negatively affected.

Monogenic hypertension refers to blood pressure caused by one genetic variant inherited from a parent. Monogenic hypertension accounts for about 30 percent of hypertension cases. Most of those are associated with imbalances of electrolytes, such as potassium.

There are several types of monogenic hypertension syndromes, each with a unique set of origins and symptoms. These include:

Knowing about your family medical history is important for many reasons. A history of certain cancers, for example, may determine when you get screened for those cancers. If high blood pressure runs in your family, its important to share this information with your doctor and regularly monitor your blood pressure.

One way to organize information about your family health history, as well as your own, is to use My Family Health Portrait, an online tool created by the National Institutes of Health. You can gather your family medical history, share it with other relatives, and learn about your risk levels for conditions that tend to run in families.

If your blood pressure is currently at a healthy level, you can make several key lifestyle adjustments to lower the odds of it rising too much. If your blood pressure is higher than usual, these steps, along with medications, may help you bring it back down to a healthy range:

The National Heart, Lung, and Blood Institute developed the Dietary Approaches to Stop Hypertension (DASH) eating plan as a heart-healthy eating strategy.

This plan focuses on managing blood pressure by emphasizing fruits, vegetables, whole grains, lean proteins, and sodium reduction. Its also flexible enough to let people enjoy many of their favorite foods.

Sufficient sleep is essential to good overall health, especially for brain and heart function. Blood pressure is especially susceptible to problems related to poor sleep.

A 2022 study suggests that frequent sleep disturbances and short sleep, or less than 5, 6, or 7 hours, can contribute to hypertension.

Taking steps to improve sleep duration and quality may improve more than just your cardiovascular health. It can also improve your mood, concentration, energy, metabolism, and more.

Hypertension is a major risk factor for cardiovascular disease, the leading cause of death in the United States. High blood pressure is also a leading cause of stroke and a risk factor for chronic kidney disease and other health problems.

If your family medical history includes high blood pressure, start taking steps to lower your risk through heart-healthy behaviors. Even if you dont know your family history or dont have a close relative with hypertension, its still important to take steps to keep your blood pressure under control.

Theres a variety of anti-hypertensive medications that can help. But these medications dont take the place of a healthy diet, exercise, and getting plenty of sleep to help maintain a healthy blood pressure.

Originally posted here:
Familial Hypertension: The Genetics of High Blood Pressure - Healthline

This document has been translated from the Japanese original for reference purposes only.

In the event of any discrepancy between this translated document and the Japanese original, the original shall prevail. The Company assumes no responsibility for this translation or for direct, indirect or any other forms of damages arising from the translation.

April 20, 2022

Company name: Modalis Therapeutics Corporation

Stock exchange listing: Tokyo Stock Exchange

Code number: 4883

URL:https://www.modalistx.com/en/

Representative: Haruhiko Morita

Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines for the Treatment of a Type of Muscular Dystrophy and the Other Genetic Disorders at the ASGCT Annual Meeting

MDL-101 preclinical data support durability and efficacy of a differentiated precision medicine approach for Congenital Muscular Dystrophy type 1a

Preclinical data demonstrating our proprietary CRISPR based epigenetic modulating technology regulates target gene expression, demonstrating its potential as a therapeutic approach for serious genetic disorders.

20-Apr-2022 TOKYO & Waltham, Mass - Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM epigenetic modulating technology, today announced that six scientific abstracts have been accepted for presentation at the 25th Annual Meeting of the The American Society of Gene & Cell Therapy (ASGCT), being held in Washington D.C. and virtually, May 16-19, 2022. The abstracts present preclinical data from the Company's Congenital Muscular Dystrophy type 1a (CMD1A) and the other rare disease programs in cardiovascular and neuroscience indications, as well as validation of our technology.

Modalis presentations at ASGCT will include preclinical data demonstrating that:

Our single AAV vector system coding dCas9-trans activating domain fusion protein and gRNA targeting LAMA-1 gene (AAV9-CRISPR-GNDM-LAMA1) upregulate LAMA-1 protein to compensate LAMA-2 function in LAMA-2 knock out mice that improved survival, supporting continued development of MDL-101 for the treatment of CMD1A; and

"At ASGCT, we will present preclinical data on MDL-101 that have validate our differentiated therapeutic strategy leveraging our CRISPR-GNDM (guide nucleotide directed modulation) technology for CMD1A which has been an undruggable target. We believe that MDL-101 has the potential to be a life changing gene modulation therapy for CMD1A, and Modalis remains on track to file an IND by end 2023," said Tetsuya Yamagata, M.D. Ph.D., Chief Scientific Officer, Modalis Therapeutics. "We will also share exciting preclinical data showcasing use of our proprietary epigenetic technology to modulate genes to restore expression levels of disease-causing genes. These data reinforce our view that we have a unique epigenetic modulating approach with the potential to treat a wide range of serious genetic disorders that have been unapproachable with other platforms."

The complete list of Modalis Therapeutic presentations is below. Abstracts can be accessed on the ASGCT website and the presentations will be posted on the Modalis website during the conference.

Oral Presentations:

Title: NGS based evaluation of AAV genome integrity for improved production and function Date and Time: 5/16/2022 11:45AM

Session Name: Vector Manufacturing and Engineering 1

Title: Novel single AAV vector treatment for Congenital Muscular Dystrophy type 1A (MDC1A) using CRISPR-GNDMtechnology

Date and Time: 5/18/2022 10:30AM

Session Name: Musculo-skeletal Diseases

Poster Presentations:

Title: Robust suppression of Tau by CRISPR-GNDM system for treatment of Tauopathies Date and Time: 5/16/2022 5:30 PM

Session Name and poster board#: Neurologic Diseases I /M-141

Title: Utilizing CRISPR-GNDM mediated gene activation of the extra-large gene titin for the treatment of dilated cardiomyopathy and other titinopathies

Date and Time: 5/17/2022 5:30 PM

Session Name and poster board#: Cardiovascular and Pulmonary Diseases/Tu-124

Title: Blocking SNHG14/UBE3A-ATS lncRNA Transcription with Dead Cas9 (CRISPR-GNDM) Can Un-Silence Paternal UBE3A in an Angelman Syndrome Mouse Model Date and Time: 5/18/2022 5:30 PM

Session Name and poster board#: Neurologic Diseases III/W-153

Title: Evaluation of Cas9 mediated immune response effect on long term transgene expression in WT mice and NHPs without immune suppression

Date and Time: 5/18/2022 5:30 PM

Session Name and poster board#: Immunological Aspect of Gene Therapy and Vaccine II /W-252

About MDL-101

MDL-101 is an experimental, epigenetic modulation therapy under investigation for the treatment of Congenital Muscular Dystrophy type 1A (CMD1A). MDL-101. MDL-101 is comprised of guide nucleotide targeting LAMA-1 gene, a highly homologous sister gene of the disease-causing gene LAMA-2, enzyme-null Cas9 (dCas9) fused with trans-activating domain driven by a muscle specific promoter and coded in an AAV vector. MDL-101 upregulates LAMA-1 gene products in patient's muscle tissue to compensate loss of function caused by mutation of LAMA-2, and therefore has the potential to provide a one-time, durable treatment benefit for people living with CMD1A.

About Modalis:

Modalis Therapeutics develops precision genetic medicines using epigenetic gene modulation. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or epigenetic editing without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is initially focusing on genetic disorders caused by loss of gene regulation - resulting in excess or insufficient protein production - by targeting more than 660 genes that are thought to cause human disease as a result of haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Waltham Massachusetts, the company is listed on Tokyo Stock Exchange's Growth market. For additional information, visitwww.modalistx.com.

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Modalis Therapeutics : to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines at the ASGCT Annual Meeting -...

Pune, India, April 20, 2022 (GLOBE NEWSWIRE) -- The global gene therapy market size is set to gain momentum from the rising incidence of different types of cancer. The field of this therapy is undergoing several technological advancements that would help in treating cancer in those patients who are at high risks of getting affected by this disease through genetic mutations. The report further mentions that the market size was USD 3.61 billion in 2019 and is projected to reach USD 35.67 billion by 2027, exhibiting a CAGR of 33.6% during the forecast period.

Fortune Business Insights provided this information in a new report, titled, Gene Therapy Market Size, Share & COVID-19 Impact Analysis, By Application (Oncology, Neurology, and Others), By Vector Type (Viral and Non-viral), By Distribution Channel (Hospitals, Clinics, and Others), and Regional Forecast, 2020-2027.

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Industry Developments:

Report Scope & Segmentation

Drivers & Restraints-

Increasing Innovations & Research Activities to Boost Growth

The U.S Food and Drug Administration (FDA) stated that it is expecting to receive more than 200 applications of this therapy by the end of 2020. This showcases that the rising number of research studies and innovations in this field would affect the gene therapy market growth positively in the near future. In North America, almost 208 companies are currently operating in this market. In addition to this, the Alliance for Regenerative Medicine declared that as of 2018, approximately 259 potential drug candidates are under Phase I clinical trials across the globe.

For more information in the analysis of this report, visit: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

However, the outbreak of the COVID-19 pandemic is presently impacting the field of research. According to the director of the Office of Tissues and Advanced Therapy (FDA) named Wilson Brayan, nowadays the officials are prioritizing only those drugs that are associated with coronavirus. Hence, there hasnt been a surge in the application of potential drugs for gene therapy. This factor may hamper the gene therapy market growth in the forthcoming years.

Highlights of This Report:

Segment-

Neurology Segment to Earn High Share Fueled by High Cost of Drugs

Based on product type, the market is divided into neurology, oncology, and others. Out of these, the neurology segment earned 78.2% in terms of gene therapy market share in 2019. This growth is attributable to the increasing usage of this therapy for treating patients living with spinal muscular atrophy.

Quick Buy - Gene Therapy Market Research Report: https://www.fortunebusinessinsights.com/checkout-page/100243

The U.S. to Dominate Owing to Presence of Favorable Policies

In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions. Besides, the presence of favorable reimbursement policies and guidelines would also help in propelling the market growth here. As this type of treatment is not legal in several developing nations, industry giants are emphasizing on the U.S. for launching their products.

Europe, on the other hand, is anticipated to grow significantly backed by the adoption of unique treatment options. Asia Pacific is set to hold a comparatively lower share on account of the decreasing usage of gene therapy because of its expensive nature.

Fortune Business Insights lists out the names of all the gene therapy providers present in the global market. They are as follows:

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Major Table of Contents:

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With 33.6% CAGR, Gene Therapy Market Size worth USD 35.67 Billion in 2027 - GlobeNewswire

The UAB Cardiogenomics Clinic provides genetic testing and counseling for a gene variant associated with a risk of heart failure and death.

Researchers believe that the presence of the Val122Ile genetic variant in African Americans is believed to predispose them to the development of transthyretin amyloidosis, which can lead to higher risk of heart failure.A new study published in the Journal of the American Medical Association led by researchers from the University of Alabama at Birmingham Marnix E. Heersink School of Medicine found that being a carrier of a genetic variation known as Val122Ile in the transthyretin, or TTR gene, was significantly associated with an increased risk of heart failure and death. Research shows that this Val122Ile variation is more commonly seen among individuals of African ancestry.

Transthyretin protein is produced by the liver and helps circulate vitamin A and thyroxine through the body. This genetic variation causes misfolding of the transthyretin protein leading to hereditary transthyretin amyloidosis, a condition characterized by the buildup of abnormal deposits of a protein in the bodys organs and tissues. As buildup increases over time, the heart may become stiff, leading to cardiomyopathy, a disease of the heart muscle that makes it difficult to pump blood through the heart.

For this study, UAB researchers Vibhu Parcha, M.D., and Pankaj Arora, M.D., looked at this genetic variation in a cohort of 7,500 Black individuals living in the United States.

The TTR Val122Ile genetic variant is unfortunately more common among those of African ancestry with nearly three out of 100 individuals carrying the genetic variation, said Parcha, a clinical research fellow in the UAB Cardiogenomics Clinic and the UAB Division of Cardiovascular Disease.

Parcha says the presence of the Val122Ile genetic variant in African Americans is believed to predispose them to the development of transthyretin amyloidosis.

We wanted to examine whether carrying this genetic variant will lead to a higher risk of new-onset heart failure, death due to heart failure, cardiovascular causes or any other causes, Parcha said.

(Left) Vibhu Parcha, M.D., clinical research fellow in the UAB Cardiogenomics Clinic and the UAB Division of Cardiovascular Disease. (Right) Pankaj Arora, M.D., an associate professor in the Division of Cardiovascular Disease and director of the UAB Cardiogenomics Clinic.In this study, researchers analyzed participants from the REasons for Geographic and Racial Differences in Stroke study living in the United States without baseline heart failure. Among 7,514 Black participants, the population frequency of the TTR Val122Ile variant was 3.1 percent. Over a median follow-up of 10.9 years, Val122Ile variant carriers had a higher risk of incident heart failure compared with non-carriers. Over a median follow-up of 11.6 years, Val122Ile variant carriers had a higher risk of mortality compared with non-carriers. Overall researchers found that those with the TTR Val122Ile variant had a 2.5-fold higher risk of heart failure and a 40 percent higher risk of death from any reason.

Among those with the pathogenic TTR Val122Ile genetic variation, the heart may gradually become unable to function correctly, which will lead to heart failure and ultimately death, said Arora, an associate professor in the Division of Cardiovascular Disease and director of the UAB Cardiogenomics Clinic. However, the true probability of genetic variation being expressed in all those with the variant is not known, and further work is needed to understand this. The good news is that there are several new treatments approved or awaiting approval for this hereditary heart disease.

Medical facilities like the UAB Cardiogenomics Clinic provide genetic testing for this variant. At the clinic, those who carry this variant will have access to comprehensive genetic counseling and assessment of their heart structure and function.

Those with the variant may be eligible for getting access to evidence-based therapies that improve their heart health and improve their long-term outcomes, Arora said. It is also important to identify any family members who may have the genetic variation as they will benefit from early diagnosis and access to medical therapies that improve their health.

Learn more about the UAB Cardiogenomics Clinic here.

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Genetic variation common among Black individuals is associated with higher risk of heart failure and death - University of Alabama at Birmingham

Discovering new genes that are linked to undiagnosed diseases not only has important scientific and clinical consequences, but it also carries important personal implications for patients.

For instance, a study by a team of researchers at Baylor College of Medicine of a novel neurodevelopmental disorder resulted in the discovery of associated mutations or deletions affecting a gene called PAX5.

Led by corresponding authorDr. Daryl Scott, the Baylor team connected with researchers around the globe who studied PAX5 using an online site calledGeneMatcher. Working together, the group described 16 patients who exhibited similar characteristics, including developmental delay, intellectual disability and autism spectrum disorder. Some patients also experienced seizures and hearing loss.

The researchers also examined data from theInternational Mouse Phenotyping Consortiumand discovered that defects in Pax5 caused similar symptoms in mouse models.

The data show that PAX5 is a gene that causes medical problems even if only one copy is mutated or deleted, said Scott, associate professor in theDepartment of Molecular and Human Geneticsat Baylor.

This gene also stood out as being really important for normal brain function in mouse models, which mirrored what we saw in our patients.

Patients in the cohort did not have a recurrent pattern of abnormal brain MRI findings, structural birth defects or dysmorphic features. Scott stresses that the lack of these findings should not deter doctors from performing genetic testing for their patients with neurodevelopmental symptoms.

Children who dont look like they have a genetic syndrome, but have these neurodevelopmental issues, could carry changes in this gene, Scott said. Thats why we should be doing genetic testing for all children who have developmental delay and intellectual disability.

Documenting characteristics of disease genes like PAX5 not only can help expedite basic science and clinical research. According to Scott, the findings in this report provided long-awaited answers to the families in the study and will have an immediate impact for undiagnosed patients in genetics clinics.

As we discover new genes like PAX5, our diagnostic tests get better, Scott said.

From this point forward, diagnostic labs will start to check for changes in this gene. As geneticists, we are constantly reviewing older patient data to see if we can come up with new diagnoses thanks to discoveries like this one.

Thanks to the discovery of genes like PAX5, scientists and physicians have a better understanding of a novel neurological condition, can provide answers to some undiagnosed patients and have an improved diagnostic tool box when presented with a patient with undiagnosed neurodevelopmental issues.

Find all the details about this study in the journal Human Mutation.

Dr. Yoel Gofin, second-year medical genetics fellow in the Department of Molecular and Human Genetics at Baylor, is first author of the paper. Other authors at Baylor include Dr. Aliska M. Berry, Dr. Mahshid S. Azamian, Dr. Carlos A. Bacino, Dr. Seema R. Lalani and Jill A. Rosenfeld.

For a full list of authors and funding information, see the publication.

By Molly Chiu

See the article here:
Discovery of genes like PAX5 improves diagnostic testing for neurodevelopmental disorders, and more - Baylor College of Medicine

COPENHAGEN, Denmark, April 20, 2022 /PRNewswire/ --SNIPR BIOME ApS, a leading CRISPR and microbiome gene therapy biotechnology company, today announced dosing of the first human subjects in its phase 1 clinical trial with SNIPR001, an orally administered CRISPR-based therapeutic.

The purpose of the study is to investigate safety and tolerability of SNIPR001 in healthy volunteers and to evaluate the effect of SNIPR001 on reducing E. coli colonization in the gut. The study plans to enroll 36 healthy volunteers for multiple ascending dosing of SNIPR001 (NCT05277350). SNIPR001 has been granted Fast-Track designation by the FDA and is being developed in collaboration with the US non-profit organization CARB-X.

With the initiation of the First-in-Human study SNIPR BIOME becomes a clinical stage company. The experimental CRISPR therapeutic, SNIPR001, is designed to selectively target and eradicate E. coli in the gut, thus preventing translocation of these bacteria to the bloodstream, in a high-risk population of hematological cancer patients at risk for neutropenia. This precision approach could transform the wayE. coliinfections are prevented and treated, especially in the cancer ward. Today, there are no approved therapies for prophylactic therapy in this setting.

"Today, is a very special moment for SNIPR BIOME. For the first time ever, we are dosing a CRISPR-drug candidate in humans. Getting to this point is a major achievement and I am extremely proud of the whole SNIPR BIOME team, our collaborators, and advisors and especially our skilled CMC partner, Jafral, for their relentless effort in successfully bringing our first CRISPR-medicine into humans. However, this is only the beginning, and we truly believe that SNIPR001 could have the potential to help hematological cancer patients at increased risk of life-threatening bloodstream infections caused by multidrug resistant E. coli", says Dr. Christian Grndahl, Co-founder & CEO.

Dr. Milan Zdravkovic, Chief Medical Officer and Head of R&D at SNIPR Biome, comments: "We are excited about having brought our first asset into humans and expect top line results around year-end. We are in parallel pursuing our pipeline of CRISPR-medicines of exciting targets within oncology, immunology and cardio-metabolism, and have an ambition of selecting the next molecule from our pipeline to move into IND enabling studies also by the end of this year"

For more information, please contact:

Christian Grndahl, Dr.Med, Co-founder & CEOE-mail: [emailprotected] Mobile: +45 20202747www.sniprbiome.com

Please follow us on LinkedIn & Twitter: @sniprbiome

About SNIPR BIOME

SNIPR BIOME is a leadingCRISPR,and microbiome biotech company incorporated in Copenhagen, Denmark. SNIPR BIOME is engaged in the discovery and development of CRISPR/Cas-based medicines deploying its proprietary and patent-protected CRISPR/Casplatform. The company applies its CRISPR technologies to selectively target microbial pathogens and remodel the microbiome to address important unmet medical needs. SNIPR BIOME is pioneering a novel use of CRISPR/Castechnology to selectively and precisely eradicate target bacteria, while leaving the rest of the patient's microbial community intact. SNIPR BIOME was recently awarded a several million-dollar grant by CARB-X for CRISPR-based treatment ofhaematologicalcancer patients at risk of neutropenic fever and life-threating infections (SNIPR001). In addition, SNIPR BIOME and The University of Texas MD Anderson CancerCenterhas a strategic collaboration agreement to advance new CRISPR-based microbiome therapeutics to reduce immune-related adverse events (irAE) in patients being treated with combined immune checkpoint inhibitors. The company also develops proprietary technologies forin situproduction of therapeutics in the human microbiome. SNIPR BIOME and Novo Nordisk recently entered into a research agreement on an undisclosed target to evaluate this technology for gene therapy of the microbiome i.e.,in situproduction of therapeutics in the human microbiome. SNIPR BIOME holds an extensive portfolio of granted patents protecting CRISPR modification of microbiota as an adjunct to cancer therapy, vaccine therapy and other immunotherapies. In March 2019, SNIPR BIOME closed a $50 million Series A financing byLundbeckfondenEmerge (Copenhagen), Life Sciences Partners (Amsterdam), North-East Family Office (Copenhagen) and Wellington Partners (Munich).

For more details, please visit:www.sniprbiome.comand follow us on LinkedIn & Twitter: @sniprbiome

About CARB-X

CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) is a global non-profit partnership dedicated to supporting early development antibacterial R&D to address the rising threat of drug-resistant bacteria. CARB-X is led by Boston University and funding is provided by the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) in the US Department of Health and Human Services; the Wellcome Trust, a global charity based in the UK working to improve health globally; Germany's Federal Ministry of Education and Research (BMBF); the UK Department of Health and Social Care's Global Antimicrobial Resistance Innovation Fund (GAMRIF) funded by the UK Government Department of Health and Social Care (DHSC); the Bill & Melinda Gates Foundation, and with in-kind support from National Institute of Allergy and Infectious Diseases (NIAID), part of the US National Institutes of Health (NIH) within the US Department of Health and Human Services. CARB-X is investing up to US$480 million from 2016-2022 to support innovative therapeutics, preventatives and rapid diagnostics. CARB-X funds only projects that target drug-resistant bacteria highlighted on the CDC's Antibiotic Resistant Threats list, or the Priority Bacterial Pathogens list published by the WHO, with a priority on those pathogens deemed Serious or Urgent on the CDC list or Critical or High on the WHO list. CARB-X is headquartered at Boston University School of Law. https://carb-x.org/. Follow us on Twitter @CARB_X

Disclaimer

Research reported in this press release is supported by CARB-X. CARB-X's funding for this project is sponsored by the Cooperative Agreement Number IDSEP160030 from ASPR/BARDA and by an award from the Wellcome Trust. The content is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

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SNIPR BIOME Initiates First-in-Human Clinical Trial with SNIPR001 - PR Newswire

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., announced that data from a Phase 2a trial of the investigational, novel gene therapy, URO-902, will feature as a late-breaker at the 2022 annual meeting of the American Urological Association (AUA2022), May 13-16, in New Orleans, Louisiana. The plenary presentation will include interim efficacy and safety data on URO-902 from the ongoing Phase 2a trial.

In addition, two podium presentations at AUA2022 will feature new analyses of data from the EMPOWUR 40-week extension trial of GEMTESA (vibegron) 75 mg, a Phase 3, randomized, double blind, active-comparator controlled multicenter study to evaluate long-term safety and efficacy in patients with symptoms of OAB. GEMTESA is approved by the U.S. Food and Drug Administration (FDA) for the treatment of OAB in adults with symptoms of UUI, urgency, and urinary frequency.

Overactive bladder remains a condition in need of additional treatment options. We look forward to sharing new data related to the use of GEMTESA in the OAB patient population as well as providing an initial read-out on the progress of our investigational gene therapy, URO-902, said Sef Kurstjens, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Urovant Sciences. We believe that URO-902 could potentially offer a new treatment option for patients with overactive bladder who have been inadequately managed by oral pharmacologic therapy, if approved by the FDA. The two podium presentations on GEMTESA will also add to the scientific and medical communitys understanding of this important therapy.

Data on the potential novel gene therapy, URO-902, will be presented during Friday mornings plenary session:

Late-Breaking Abstract PLLBA-03, presented by Kenneth M. Peters, M.D., principal investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Womens Urology and Pelvic Health Center and professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Michigan., titled, Efficacy and Safety of a Novel Gene Therapy (URO-902; pVAX/hSlo) in Female Patients with Overactive Bladder and Urge Urinary Incontinence: Results from a Phase 2a Trial. This presentation will take place on Friday, May 13, at 11:21 to 11:29 a.m. CDT during the plenary session in the Ernest N. Morial Convention Center, Great Hall A.

Data on GEMTESA will also be featured in two podium presentations at the conference on May 15, 2022:

Abstracts are available in the Journal of Urology at the following links:

URO-902: https://www.auajournals.org/doi/10.1097/JU.0000000000002671.03

EMPOWUR-EXT older adults: https://www.auajournals.org/doi/10.1097/JU.0000000000002596.11

EMPOWUR-EXT PRO: https://www.auajournals.org/doi/10.1097/JU.0000000000002596.12

About the Phase 2a Study of URO-902

This 48-week multicenter, randomized, double blind, placebo-controlled, dose-escalation study will evaluate the efficacy, safety, and tolerability of a single administration of URO-902, a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. URO-902 is administered via direct intradetrusor injections via cystoscopy into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and UUI.

The Phase 2a trial includes 80 female patients in two cohorts. The first cohort received either a single administration of 24 mg of URO-902 or matching placebo into the bladder wall, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Patients will be followed for up to 48 weeks after initial administration. Exploratory endpoints included change from baseline to week 12 in mean daily micturitions, urgency episodes, UUI episodes, and quality of life measures, as well as assessing the safety and tolerability of this investigational gene therapy for OAB.

About URO-902

URO-902 has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment has the potential to address an unmet need for patients who have failed oral pharmacologic therapies.

About the EMPOWUR Trial

The EMPOWUR trial was an international, randomized, double-blind, placebo and active comparator-controlled Phase 3 clinical trial evaluating the safety and efficacy of investigational vibegron in men and women with symptoms of overactive bladder, including frequent micturition, urgency, and UUI. A total of 1,518 patients were randomized across 215 study sites into one of three groups for a 12-week treatment period with a four-week safety follow-up period: vibegron 75 mg administered orally once daily; placebo administered orally once daily; or tolterodine ER 4 mg administered orally once daily.

About the 40-Week EMPOWUR Extension

The EMPOWUR 40-week extension trial was a Phase 3, randomized, double blind, active-comparator controlled multicenter study to evaluate the long-term safety and efficacy of vibegron in patients with symptoms of overactive bladder. The extension study enrolled approximately 500 EMPOWUR completers. The primary endpoint was safety, measured by incidence of adverse events. Secondary endpoints were changes from EMPOWUR baseline at week 52 in average daily micturitions, UUI, urgency, and total urinary incontinence.

About Overactive Bladder

Overactive bladder (OAB) is a clinical condition that occurs when the bladder muscle contracts involuntarily. Symptoms may include urinary urgency (the sudden urge to urinate that is difficult to control), urgency incontinence (unintentional loss of urine immediately after an urgent need to urinate), frequent urination (usually eight or more times in 24 hours), and nocturia (waking up more than two times in the night to urinate).1

Approximately 30 million Americans suffer from bothersome symptoms of OAB, which can have a significant impairment on a patients day-to-day activities.1, 2

About GEMTESA

GEMTESA is a prescription medicine for adults used to treat the following symptoms due to a condition called overactive bladder:

It is not known if GEMTESA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take GEMTESA if you are allergic to vibegron or any of the ingredients in GEMTESA.

Before you take GEMTESA, tell your doctor about all your medical conditions, including if you have liver problems; have kidney problems; have trouble emptying your bladder or you have a weak urine stream; take medicines that contain digoxin; are pregnant or plan to become pregnant (it is not known if GEMTESA will harm your unborn baby; talk to your doctor if you are pregnant or plan to become pregnant); are breastfeeding or plan to breastfeed (it is not known if GEMTESA passes into your breast milk; talk to your doctor about the best way to feed your baby if you take GEMTESA).

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What are the possible side effects of GEMTESA?

GEMTESA may cause serious side effects including the inability to empty your bladder (urinary retention). GEMTESA may increase your chances of not being able to empty your bladder, especially if you have bladder outlet obstruction or take other medicines for treatment of overactive bladder. Tell your doctor right away if you are unable to empty your bladder.

The most common side effects of GEMTESA include headache, urinary tract infection, nasal congestion, sore throat or runny nose, diarrhea, nausea, and upper respiratory tract infection. These are not all the possible side effects of GEMTESA. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please click here for full Product Information for GEMTESA.

About Urovant Sciences

Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys lead product, GEMTESA(vibegron), is an oral, once-daily (75 mg) small molecule beta-3 agonist for the treatment of adult patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency. GEMTESA was approved by the U.S. FDA in December 2020 and launched in the U.S. in April 2021. GEMTESA is also being evaluated for the treatment of OAB in men with benign prostatic hyperplasia. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need. Learn more about us at http://www.urovant.com or follow us on Twitter or LinkedIn.

About Sumitovant Biopharma

Sumitovant is a global biopharmaceutical company leveraging data-driven insights to rapidly accelerate development of new potential therapies for unmet patient conditions. Through our unique portfolio of wholly-owned Vant subsidiariesUrovant, Enzyvant, Spirovant, Altavantand use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported the development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovant, a wholly-owned subsidiary of Sumitomo Pharma, is also the majority-shareholder of Myovant (NYSE: MYOV). For more information, please visit our website at http://www.sumitovant.com

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Urovant Sciences to Present Interim Data from Phase 2a Study of Potential Novel Gene Therapy, URO-902, and New Analyses of Data from Phase 3 EMPOWUR...

Data Highlights Clinical Utility of CareDx Solutions, Including Findings from Surveillance HeartCare Outcomes Registry (SHORE) Demonstrating Power of Multimodality Assessment for More Precise Interventions

CareDx Symposia Feature Latest Advancements Including XenoSure and XenoMap

SOUTH SAN FRANCISCO, Calif., April 20, 2022 (GLOBE NEWSWIRE) -- CareDx, Inc. (Nasdaq: CDNA) The Transplant Company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers today announced a leading presence at the International Society for Heart and Lung Transplantation (ISHLT) Annual Meeting and Scientific Sessions, with over 25 oral presentations and posters covering the latest innovations in heart and lung transplant surveillance and two symposia. ISHLT 2022 takes place on April 27-30 in Boston, Massachusetts.

CareDx showcases its leadership in heart and lung transplant patient care with a significant body of evidence highlighting the value of its diagnostic tools for heart and lung transplant recipients. Notably, oral presentations covering interim findings from the SHORE (Surveillance HeartCare Outcomes Registry) observational study will be presented, highlighting the value of multimodality assessment of heart transplant health for assessing the risk of rejection and de novo donor-specific antibodies (dnDSA). Additionally, CareDx symposia will spotlight leaders in the field delivering cutting-edge content using multimodality solutions, HeartCare, AlloMap gene-expression profiling, and AlloSure donor-derived cell-free DNA (dd-cfDNA).

CareDx is proud of its history of innovation in cardiothoracic transplantation, with many firsts, from multimodality molecular assessment of allograft health, to donor-derived cell-free DNA for lung transplantation, to recently introducing, XenoSure and XenoMap, for investigational use in xenotransplantation research, said Reg Seeto, CEO and President of CareDx. Its extremely rewarding to have another remarkable presence at this years ISHLT meeting with extensive data elucidating the clinical value of integrating CareDx solutions and multimodality assessments into clinical practice.

The initial findings from the SHORE study build upon a growing literature that supports non-invasive surveillance of heart transplant patients with AlloSure donor-derived cell-free DNA and AlloMap gene-expression profiling. These studies provide a more comprehensive assessment of the health of the transplanted heart, which will hopefully lead to better outcomes for our patients through more tailored management strategies and earlier detection of graft injury, said Dr. Jeffrey Teuteberg, MD, Cardiologist, and Associate Professor of Cardiovascular Medicine, Stanford Health Care. Multimodality surveillance using both dd-cfDNA and gene-expression profiling may prove to be a better marker of immune quiescence or activation than endomyocardial biopsy (EMB) and questioning the place of the EMB as the gold standard for cardiac allograft monitoring.

CareDx Lunch Symposia:

For more detailed agendas for the lunch symposia, please follow this link.

The following heart and lung transplant data will be presented:

About CareDx The Transplant CompanyCareDx, Inc., headquartered in South San Francisco, California, is a leading precision medicine solutions company focused on the discovery, development and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers. CareDx offers testing services, products, and digital healthcare solutions along the pre- and post-transplant patient journey and is the leading provider of genomics-based information for transplant patients. For more information, please visit: http://www.CareDx.com.

Forward Looking StatementsThis press release includes forward-looking statements related to CareDx, Inc., including statements regarding the potential benefits and results that may be achieved with CareDxs leading presence at the ISHLT Annual Meeting and Scientific Sessions, including oral presentations covering interim findings from the SHORE observational study and the CareDx symposia (the CareDx ISHLT Leadership). These forward-looking statements are based upon information that is currently available to CareDx and its current expectations, speak only as of the date hereof, and are subject to risks and uncertainties that could cause actual results to differ materially from those projected, including risks that CareDx does not realize the expected benefits of the CareDx ISHLT Leadership; risks that the findings of SHORE study may not be accurate; risks that ISHLT 2022 and the agendas of CareDx symposia fail to take place as planned; general economic and market factors; and other risks discussed in CareDxs filings with the SEC, including the Annual Report on Form 10-K for the fiscal year ended December 31, 2021 filed by CareDx with the SEC on February 24, 2022, and other reports that CareDx has filed with the SEC. Any of these may cause CareDxs actual results, performance or achievements to differ materially and adversely from those anticipated or implied by CareDxs forward-looking statements. CareDx expressly disclaims any obligation, except as required by law, or undertaking to update or revise any such forward-looking statements.

CONTACTS:CareDx, Inc.Sasha KingChief Marketing Officer415-287-2393sking@caredx.com

Investor RelationsIan Cooney(415) 722-4563investor@CareDx.com

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CareDx Showcases Leadership with Over 25 Oral Presentations and Posters at the International Society for Heart and Lung Transplantation Meeting -...

HOPEWELL, N.J.--(BUSINESS WIRE)--Gennao Bio, a privately held genetic medicines company developing first-in-class, targeted nucleic acid therapeutics, today announced promising preclinical results of its proprietary, non-viral gene monoclonal antibody (GMAB) platform in multiple solid tumor models at the American Association for Cancer Research (AACR) 2022 Annual Meeting. The findings from these studies were reported and discussed in an oral presentation by Elias Quijano, M.D./Ph.D. candidate in the laboratory of Dr. Peter Glazer at the Yale School of Medicine, and co-founder of Gennao Bio, entitled, Systemic targeting of therapeutic RNA to cancer via a novel, cell-penetrating and nucleic acid binding, monoclonal antibody.

The preclinical results demonstrated GMABs ability to form non-covalent complexes with and systemically target and deliver 3p-hpRNA, a potent activator of the immune signaling RIG-I pathway, to solid tumors, including orthotopic mouse models of human pancreatic cancer (KPC) and medulloblastoma (DAOY). GMABs highly specific delivery into tumors is independent of the endocytic pathway and is uniquely enabled by targeting ENT2, a nucleoside transporter that is overexpressed in many tumors. In vitro studies of GMAB/3p-hpRNA demonstrated that delivery of a RIG-I agonist to tumor cells triggers an immune stimulating type-1 interferon response and triggers direct tumor cell death.

These positive results further reinforce our strong belief in the broad therapeutic potential and diverse application of our GMAB platform in treating cancers with substantial unmet need, said Stephen Squinto, Ph.D., chief executive officer and chair of the board of Gennao Bio. We expect to advance the humanized version of GMAB/3p-hpRNA, GMAB-7001, into Investigational New Drug (IND)-enabling studies in the second half of 2022 and will continue to assess additional oncology pipeline programs.

In the KPC pancreatic cancer model, multiple doses of GMAB/3p-hpRNA resulted in a significant survival benefit, driven in part by long-term increases in tumor-infiltrating lymphocytes, including CD45+, CD8+, CD4+, and CD19+ cells. GMAB/3p-hpRNA treatment also showed a statistically significant increase in tumor cell necrosis compared to the control group. Previous studies of a single dose administration of GMAB/3p-hpRNA in an orthotopic model of medulloblastoma demonstrated its ability to penetrate the central nervous system, reduce intracranial tumor burden by 50%, and prevent spinal metastases.

The GMAB platform has the potential to address the challenges faced by alternative methods of delivery of immunostimulatory nucleic acids to tumors, which have been associated with systemic toxicities or rely on suboptimal intra-tumoral injections. Studies of single and multiple intravenous doses of GMAB/3p-hpRNA have shown targeted payload tumor delivery and resultant tumor growth suppression in several preclinical models of difficult-to-treat forms of cancer, said Mr. Quijano. These promising monotherapy results, and the new data generated in a difficult-to-treat pancreatic cancer model, warrant continued research of the GMAB platform and development of this new class of targeted nucleic acid therapeutics for cancer.

A copy of the AACR presentation can be found under the News section on the Companys website, http://www.gennao.com.

About Gennao Bio

Gennao Bio is a privately held genetic medicines company developing first-in-class targeted nucleic acid therapeutics utilizing a proprietary gene monoclonal antibody (GMAB) platform technology. GMAB is an adaptive technology that uses a novel, cell-penetrating antibody to non-covalently bind to, and deliver therapeutic levels of a wide variety of nucleic acid payloads, to select cells. This non-viral delivery platform is differentiated from traditional gene delivery systems as it can deliver multiple types of nucleic acids, allows for repeat dosing, and employs well-established manufacturing processes. Gennao Bio is developing this delivery system with an initial focus on addressing significant unmet needs in oncology and rare monogenic skeletal muscle diseases.

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Gennao Bio Presents Promising New Results from its Gene Monoclonal Antibody (GMAB) Platform at the American Association for Cancer Research (AACR)...

Janssens Catherine Taylor, vice-president, EMEA medical affairs, therapy area strategy, discusses the importance of systemic innovation across the healthcare system to realise the full value of medicines.

Medicines and vaccines are among the most powerful interventions that can help improve quality of life for people across the world. As an oncology trained medical doctor who practiced in the UK NHS for 10 years, I have seen the benefits first-hand, as patients live longer lives, free from symptoms, and are able to return to work.

Beyond that, there are positive impacts for wider society, healthcare systems and economies. It is essential, therefore, that we continue to streamline the development, regulatory and access process, so that patients have faster access to better and safer medicines.

Cultivating a pro-innovation environment

To achieve this, it will be critical to maintain a pro-innovation environment. Systemic innovation isnt limited to discovering new molecules; its a mindset we must continue to apply across the board. We need to be able to think outside the box and find new interventions and new delivery methods that lead to better and more sustainable standards of medical care.

Because, despite the great improvements made across the industry in recent years to accelerate innovative development, we still face many challenges. Innovative next-generation medicines are met with a series of hurdles such as increased cost of development, regulatory challenges and delays, and longer and more complex clinical trials. And it all fuels the debate: are medicines a cost or an investment?

The answer to this question comes back to our definition of value. Value-based healthcare is a delivery model in which providers are rewarded based on the value and difference they bring to patients lives, rather than volume of treatments provided.

A few months ago, I spoke with Professor Joaquin Mateo, chair of the ESMO Translational Research and Precision Medicine Working Group. We agreed that one of the key future foci of innovation in medicines lay in the treatment of patients with medicines targeted specifically towards the molecular signature of their disease. The ideal state is that one day patients only receive (and the healthcare system only provides) medicines that will improve their condition. While we have made great progress with some diseases, we are not there yet, and we need to ensure that focused investment into research continues to bring the value that patients truly deserve.

The role of innovation in value-based healthcare

Delivering value for patients doesnt start and end in our R&D labs; actions can be implemented across all stages of the drug development lifecycle to encourage and embrace a pro-innovation environment.

For example, alongside stable, transparent and long-term policies or research plans, incentives can encourage innovation from those willing to take risks and tackle areas of unmet patient need. Effective intellectual property systems are also essential in stimulating research and sustainable innovation for the future.

And strong collaboration from all involved in health research is another key component of a value-based healthcare approach. From patients, patient bodies, pharmaceutical companies, and academia, to regulatory bodies, public institutions, and small and medium-sized enterprises (SMEs), all key stakeholders must come together to help the patients who most need new medicines to access them as quickly as possible.

The European Commissions Pharmaceutical Strategy for Europe, adopted in November 2020, outlines a series of concrete actions to ensure accessibility, availability, and affordability of medicines. The Strategy also highlights the importance of drawing lessons from the COVID-19 pandemic to enhance crisis preparedness and response mechanisms, and so help make the healthcare industry more prepared and resilient.

How can innovation within Medical Affairs drive value?

Medical Affairs is at the forefront of driving innovation across our industry, making it an exciting and ever-evolving area to work in. From innovative evidence generation to accelerating medical treatment adoption and transforming medical engagement, our teams play a huge role in helping to deliver much-needed treatments to patients across the world.

According to EFPIA, there are over 7,000 medicines currently in development globally. As part of this, innovative treatments such as CAR-T therapy and retinal gene therapy are emerging as ways to target rare diseases, multiple indications and underserved populations. Such therapies present significant challenges to our current regulatory and access processes, but these are challenges we must overcome if patients are to benefit.

The key, of course, is to always start with the end goal in mind what change do we want to bring to patients? So, alongside developing and providing the treatment itself, its also important to provide comprehensive medical education, and to explore new patient pathways and delivery systems. That way, everyone understands the value of the treatment, and the route to getting it to the right patients at the right time is clear.

Data will always be central to everything we do, as it has the power to communicate the potential of a new treatment to change a persons life. If we are to move towards a value-based healthcare system, then we must utilise the power of both clinical trials data and real-world evidence, to produce the best possible data package for regulatory submissions. Effective data analysis can lead to accelerated access and improved outcomes for patients. And building the voice of the patient into our business innovation is crucial we need patients unique perspectives and experiences to keep us focused on solutions that will have the largest impact on their lives.

At Janssen, we strive to improve access to our innovative medicines and vaccines and achieve the best possible outcomes for patients across the world. Where I feel Medical Affairs can move the needle is as the scientific bridge, engaging with key stakeholders like clinicians, researchers, payers, policymakers, and regulators. Our role is to help translate the data and impactfully articulate the demonstrated medical value of a medicine, and to collaboratively explore how the value of that medicine could be improved. Ultimately, we want to ensure that those patients who could benefit most from our treatments are able to receive them thats what drives our Medical Affairs purpose.

About the author

Between 2016-2019, she was the medical affairs therapeutic area lead in haematology for Janssen Oncology across the EMEA region. Dr Taylor holds an MD from Kings College London and is a member of the UK Royal College of Physicians. Before joining the pharmaceutical industry, she trained and practiced in Clinical Oncology in London for 7 years. She also holds the Diploma in Pharmaceutical Medicine and has completed her UK specialty training in Pharmaceutical Medicine. Prior to joining Janssen in 2015, she held positions in UK, EMEA regional and global medical affairs at Pfizer and Astellas.

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Innovation across the healthcare system is needed to realise the value of medicines - - pharmaphorum