Daily Archives: February 5, 2022

Cytiva and NecstGen say they plan to accelerate the development of new cell and gene therapies globally by entering into a strategic collaboration. Cytiva will provide its technologies, services, and solutions to NecstGen, and both organizations will share their knowledge and expertise as research programs are translated into next generation therapies, according to officials at both companies.

Cell and gene therapies are transformative medicines and accelerating their development requires harnessing the power of the industry, says Catarina Flyborg, vice president, cell and gene therapy, Cytiva. By sharing our expertise and providing NecstGen with access to our team of specialists, Cytiva will play a critical role in taking translational research from the laboratory to the bedside.

NecstGen is a non-profit contract development and manufacturing organization (CDMO) specializing in cell and gene therapies in the Netherlands. It brings the development, production, QC, QA, and QP functions together in a new 4,000 m2 facility in Leiden Bio Science Park, the largest bio-cluster in the Netherlands, notes Paul Bilars, CEO, NecstGen.

The new facility is designed to serve all organizations worldwide, particularly academic and small/large industry enterprises that are looking to bring their research to the clinical stage. NecstGen will provide process development, cGMP manufacturing services up to 200L, and cleanroom rental.

Our partnership with Cytiva will provide us with the flexible and scalable solutions needed by pioneers in the field of cell and gene therapy, says Bilars. Working together, we will accelerate the development of future therapies, bringing these to patients faster.

During the first half of 2021, there were 1,328 regenerative medicine trials underway globally sponsored by non-industry groups such as academic centers and government entities, according to the Alliance for Regenerative Medicine. Small and mid-size enterprises and academic centers play an important role in the development of novel cell and gene therapies.

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Cytiva and NecstGen Collaborate on Development of Cell and Gene Therapies - Genetic Engineering & Biotechnology News

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Cross-disciplinary team identifies genetic cause of rare, undiagnosed lung disease

New research from Washington University School of Medicine in St. Louis has solved the medical mystery of why a 2-year-old child seemingly healthy at birth succumbed to an undiagnosed, rare illness. On the left is normal lung tissue showing air sacs with thin cell layers for the exchange of oxygen and carbon dioxide. On the right is the patient's lung tissue. Because of a mutation in the RAB5B gene, the walls of the air sacs are thick and unable to participate in gas transfer.

New research from Washington University School of Medicine in St. Louis has solved the medical mystery of why a 2-year-old child seemingly healthy at birth succumbed to an undiagnosed, rare illness. The research team identified a previously unknown genetic cause of interstitial lung disease, providing answers to the parents and doctors puzzled by the childs condition.

The research, conducted as part of the National Institutes of Healths (NIH) Undiagnosed Diseases Network, demonstrates, among other benefits, how an interdisciplinary team of researchers can work together to solve medical mysteries, helping patients understand a diagnosis, prognosis and what a genetic abnormality may mean for family members and family planning.

The study is published the week of Jan. 31 in the Proceedings of the National Academy of Sciences. The Undiagnosed Diseases Network is a national research network aimed at diagnosing rare and previously undescribed diseases in patients whose conditions present as medical mysteries. Washington University serves as a clinical site that evaluates patients, and a model organism screening site that develops models to study genes in zebrafish and roundworms.

Interstitial lung disease is a broad term for a disease in which the lungs gradually deteriorate, causing scarring that makes it increasingly difficult to breathe. Several gene abnormalities have been associated with interstitial lung disease in infants and children, but some patients have the disease despite harboring none of the known genetic abnormalities. In the new study, the researchers were presented with the case of a young child with interstitial lung disease of unknown cause. The child later died of the disease.

The researchers analyzed the childs DNA code as well as the DNA code of both parents. A team of bioinformatics specialists at Baylor College of Medicine then narrowed down the initial long list of DNA code changes or genetic variants they identified many of which are harmless to a smaller list of possible culprits. The lung tissue from the child had evidence of a problem with surfactant in the lungs. In the lungs air sacs, surfactant is a complex mixture of proteins and lipids that reduces surface tension in the air sacs and keeps them open, easing the exchange of oxygen and carbon dioxide during breathing. Many people with interstitial lung disease have abnormalities in the surfactant protein genes. But this child did not have any genetic variants in the code of the surfactant protein genes.

Rather, the researchers found a variant in a gene that makes a protein called RAB5B that turns out to be part of the cellular machinery that processes the surfactant proteins, the researchers later learned. They showed that the RAB5B protein plays a vital role in packaging the surfactants into tiny compartments called vesicles and moving them to their proper locations. In this case, the genetic variant did not simply prevent the protein from working the genetic variant caused the protein to be actively harmful.

When mutations happen that break a protein, usually the protein just doesnt work anymore its function is missing, said co-senior author Tim Schedl, PhD, a professor of genetics. But this is a case where the broken protein is not only not working, its actively poisoning other processes. This results in the loss of the surfactants in the lungs.

The researchers were able to identify this abnormality by studying the genetic variant in roundworms that are called C. elegans. The child had only one abnormal copy of the gene, demonstrating that even having one normal copy did not compensate for the poisonous protein produced by the mutated copy. Worms with one abnormal copy required three normal copies to restore normal function, demonstrating the poisonous activity of the abnormal copy, according to experiments conducted by first author Huiyan Winnie Huang, PhD, an instructor in pediatrics. And consistent with these genetics, the researchers found that neither of the childs parents had the genetic abnormality, indicating that the variant was only present, by happenstance, in the childs genes and was therefore a new variant in the DNA that arose during embryonic development.

In so many cases, we dont know why a patient has a particular disease, said co-senior author Steven L. Brody, MD, the Dorothy R. and Hubert C. Moog Professor of Pulmonary Medicine. But we were able to solve this case, and theres a real satisfaction in that. Potentially, this could lead to finding answers for other people who have diseases similar to this.

Added co-author Jennifer A. Wambach, MD, an associate professor of pediatrics: This gene, RAB5B, is now associated with interstitial lung disease in children. There are patients with a clinical diagnosis of interstitial lung disease without a genetic explanation. For these patients, sequencing RAB5B may reveal changes in their DNA code that could account for their disease. Knowing the underlying genetic cause and identifying other patients with the same genetic problem can help us better predict the course of the disease, so we can better prepare patients and their families for what is to come, such as whether the patient may respond to treatments, or worsen to needing a lung transplant, or whether it may be appropriate to begin discussing compassionate care.

While the diagnosis was not able to help the patient in this case, knowledge of the underlying cause allowed the parents to know that the genetic variant was not inherited and there would be a very low chance of future children having the same disease.

Because these types of genetic diseases are so rare, theres very little information out there for patients or families, said co-senior author Stephen C. Pak, PhD, an associate professor of pediatrics. But collectively, there are millions of people who live with rare genetic diseases. Thats why the Undiagnosed Diseases Network was formed to bring together bioinformatics specialists, researchers, lung biologists, pediatricians and other experts into this type of unique collaboration to try and address this unmet need.

This work was supported by the National Institutes of Health (NIH) Common Fund, through the Office of Strategic Coordination/Office of the NIH Director, grant numbers U54 NS108251 and U01 HG007709. Funding also was provided by the NIH, grant number R01 GM100756; the NIH Office of Research Infrastructure Programs, grant number P40 OD010440; the National Heart, Lung, and Blood Institute (NHLBI) of the NIH through the LungMAP consortium, grant number U01HL122642, and the LungMAP Data Coordinating Center, grant number 1U01HL122638; the Childrens Discovery Institute; the St. Louis Childrens Hospital Foundation; and The Foundation for Barnes-Jewish Hospital.

Huang H, et al. A dominant negative variant of RAB5B disrupts maturation of surfactant protein B and surfactant protein C. Proceedings of the National Academy of Sciences. Jan. 31, 2022.

Washington University School of Medicines 1,700 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, and is among the top recipients of research funding from the National Institutes of Health (NIH). Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Researchers solve medical mystery of deadly illness in young child Washington University School of Medicine in St. Louis - Washington University...

JERUSALEM, Feb. 3, 2022 /PRNewswire/ --The Hadassah Cancer Research Institute at the Hadassah University Medical Center in Jerusalem, announced today that using artificial intelligence (AI), researchers have developed, an algorithm to identify, with an unprecedented 96.5% level of accuracy, all possible deleterious mutations of TP53 gene which are commonly found in 50% of tumors. This breakthrough can potentially lead to improved genetic screening and consultation and to improved precision medicine for cancer patients.

This groundbreaking algorithm was trained using AI, huge cancer and normal genomics databases coupled with computational and experimental parameters specific to TP53 gene. Prof. Thierry Soussi (Sorbonne Universit, Paris, France), a world leading researcher of TP53 gene, lent his expertise to an international research collaboration, led by Dr. Shai Rosenberg, aimed at increasing identification of variants posing risk of developing cancer from variants that are not, from 190 (in the ClinVar database) to all 2,314 possible missense variants.

This gene-specific, AI approach can be generalized to other cancer genes and thus, contribute to more accurate genetic screening and consultation. Additionally, it can lead to more effective precision medicine for oncological patients by the creation of customized cancer treatment decision support systems that are able to identify and discern the important mutations that require treatment from the total number of somatic mutations of the tumor.

"The research and technology behind this breakthrough not only provide life-saving screening for carriers of previously unknown cancerous mutations who may be at increased risk, but it is also critical for genomic analysis of somatic mutation profiles in all tumors," said Prof. Michal Lotem, MD, Head of the Center for Melanoma and Cancer Immunotherapy, Dept. of Oncology.

"Hadassah Medical Center has been at the forefront of promoting technological innovation in medicine in order to provide patients in our care with the most advanced treatment options," said Prof. Aron Popovtzer a Professor of Radiation Oncology and Head of the Sharett Institute of Oncology. "Following this important milestone, Dr. Rosenberg's research group will continue actively working to develop similar models for additional cancer genes."

Read the full articleby Dr. Rosenberg, published in the prestigious - Briefings in Bioinformatics Journal. Dr. Rosenberg is a physician-researcher at Hadassah Medical Center. He is a senior Neuro-Oncologist and heads the Laboratory for Computational Biology of Cancer. He is a graduate of the Rothschild Excellence Program and the Technion's MD-PhD Program. He heads the Sagol program for dual degree in Medicine and Computer Science in the Faculty of Medicine in the Hebrew University.

This research is funded by Israel Academy of Sciences and Humanities, Trudy Mandel Louis Charitable Trust, Y.M.H and Hadassah-France. The research of Professor Soussi by Hadassha France.

For more information on the Hadassah Cancer Research Institute, contact:Amalia HerszkowiczHadassah Research Institute (HCRI)[emailprotected]

SOURCE Hadassah Cancer Research Institute

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Predictive-AI Algorithm Used to Successfully Identify Common Cancerous Gene Mutations Demonstrated at Hadassah Cancer Research Institute - PRNewswire

DUBLIN, February 04, 2022--(BUSINESS WIRE)--The "Orthopedic Regenerative Medicine Market, by Treatment Type, by Disease Indication, by End User, and by Region - Size, Share, Outlook, and Opportunity Analysis, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

Regenerative medicines can be consider as manufacturing of pharmaceuticals for the regrowth of cells, replacing damaged cells, organs, or tissues .The developing methods for medicines includes the generation, tissue engineering, and therapeutic stem cells and production of artificial organs.

Regenerative orthopedic medicines can help in healing damaged tissues, and improve the discomfort and pain which occurred due to musculoskeletal diseases. These regenerative medicines can be developed by using stem cells, plasma-rich sources, biological tissues, and bone marrow. Orthopedic regenerative medicines help in healing injuries of ligament, tendon, bone, spinal disc, muscle, cartilage, and musculoskeletal tissues.

Market players are indulged in receiving approvals from regulatory authorities for the treatment of orthopedic diseases, which is expected to drive the growth of the global orthopedic regenerative medicine market over the forecast period. For instance, on June 28, 2021, Curasan AG, a company which produce regenerative medicines, has received approval from U.S. Food and Drug Administration (FDA) and The National Health Surveillance Agency (ANVISA) Brazilian regulatory agency for CERASORB Foam for use with antibiotics which is a ?-TCP collagen matrix which use for delivering antimicrobials to wound surface to inhibit microbes capable of healing. The major use of this foam is minimizing the risk of reinfection of the infected area by combining it with antibiotics.

Increasing inorganic activities by market players such as collaborations, partnerships, and agreements is expected to drive the growth of the global orthopedic regenerative medicine market over the forecast period. For instance, on January 14, 2021, Bone Therapeutics, which is a manufacturer of cell therapy products, signed partnership with Rigener and, a company which develops and produces cGMP products, used for cell-gene therapy. The aim of this partnership was to develop products based on bone therapeutics by expanding research for therapeutic portfolio. This partnership will be focused on augmented bone forming cells that are programmed for specific task and by studying the new mechanisms of action for gene modifications for orthopedic regenerative medicines.

Story continues

Company Profiles

Ortho Regenerative Technologies Inc.

Personalized stem cells Inc.

Anika Therapeutics Inc.

Arthrex Inc.

Baxter International Inc.

Conmed Corporation

Aziyo Biologics

Curasan Inc.

Swiss biomed Orthopedics AG

Octane Medical Inc.

Stryker Corporation

Carmell Therapeutics Corporation

Zimmer Holdings

Smith & Nephew plc.

NuVasive Inc.

Key features of the study:

This report provides an in-depth analysis of the global orthopedic regenerative medicine market, provides market size (US$ Million), and compound annual growth rate (CAGR %) for the forecast period (2021-2028), considering 2020 as the base year.

It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrix for this market

This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategies adopted by the leading players

It profiles leading players in the global orthopedic regenerative medicine market based on the following parameters - company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies, and future plans

Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launch, technology up-gradation, market expansion, and marketing tactics

The global orthopedic regenerative medicine market report caters to various stakeholders in this industry including investors, suppliers, manufacturers, distributors, new entrants, and financial analysts

Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global orthopedic regenerative medicine market

Key Topics Covered:

1. Research Objectives and Assumptions

2. Market Overview

Report Description

Market Definition and Scope

Executive Summary

Market Snippet, By Treatment Type

Market Snippet, By Disease Indication

Market Snippet, By End User

Market Snippet, By Region

Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

4. COVID - 19 Impact Analysis

5. Global Orthopedic Regenerative Medicine Market, By Treatment Type, 2017 - 2028 (US$ Million)

6. Global Orthopedic Regenerative Medicine Market, By Disease Indication , 2017 - 2028 (US$ Million)

7. Global Orthopedic Regenerative Medicine Market, By End User, 2017 - 2028 (US$ Million)

8. Global Orthopedic Regenerative Medicine Market, By Region, 2017 - 2028 (US$ Million)

9. Competitive Landscape

10. Section

For more information about this report visit https://www.researchandmarkets.com/r/c8xqzo

View source version on businesswire.com: https://www.businesswire.com/news/home/20220204005208/en/

Contacts

ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.com For E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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Global Orthopedic Regenerative Medicine Market to 2028 - Featuring Anika Therapeutics, Baxter International and Stryker Among Others -...

The CGTs gaining marketing authorisation in Europe and the US each year can currently be counted in single digitals, but hundreds of potential new CGTs are in the pipeline. According to the AMR report, 136 of the 956 unique therapies under development are already in the phase three stage of clinical trials commonly the last stage of clinical trials where the safety and effectiveness of the proposed new treatments is compared to existing treatments.

Much of the research ongoing is focused on delivering new cancer treatments, but CGTs have potential utility across other areas of medicine. The AMR report highlights the efforts being undertaken by medical researchers across industry, academia and government to develop new CGTs to address problems such as heart failure, rare genetic diseases, and neuromuscular diseases, for example.

Research overseen by the Massachusetts Institute of Technologys Center for Biomedical Innovation, published in 2018, projects that around 500,000 patients in the US will have been treated with between 40 and 60 approved CGTs by 2030.

CGTs are often developed to treat small patient populations at high cost. This poses a problem for budget-constrained health systems in terms of fitting the therapies within existing reimbursement models in a way which enables the developers of the therapies to obtain a fair return on their investment. A lack of harmonisation over the way each country approaches reimbursement creates uncertainty for researchers and their financial backers.

In addition, small biotech companies behind CGTs must navigate a complex regulatory framework and find infrastructure solutions to scale-up the manufacturing of approved treatments.

The challenges facing the CGT sector are well-recognised by policy makers, and efforts to resolve them are underway across Europe at EU level, EU27 member state level and within the UK alone to address them. We have set out examples of the initiatives we think could be taken forward.

Covid-19 has shown how collaborative, concerted efforts can drive innovation forward. There is an opportunity for stakeholders across the CGT sector to come together differently, perhaps in a pre-competitive way, to help drive economies that reduce costs.

The BioPharmaceutical industry has historically turned to industry-wide collaboration through organisations such as the Innovative Medicines Initiative (IMI) and the Pistoria Alliance to address resource-wasting replication of pre-competitive research activities and solve bottlenecks in drug discovery and development across diseases. CGT should be no exception.

An international group of senior researchers is lobbying the G7 to reform the way they manage collaboration on emerging technologies. The proposed framework would produce model, multilateral agreements on sharing and exploiting emerging technologies, including CGTs, that companies, universities and governments could follow; reducing uncertainty about IP, standardisation, data sharing, researcher mobility and other contentious issues.

Standardisation will cut development time and costs of CGTs too. Companies are already working with regulators and standard setting bodies, such as the International Organization for Standardisation, to agree regulatory standards. Various groups globally are discussing harmonisation of best practices to eliminate or reduce costs. In time, standardisation will facilitate technology platforms that are able to target many different diseases; viral-vector platforms that target a range of diseases in gene therapy are already in development.

Innovative payment models are already being used by health systems across Europe for CGTs to address affordability. As the pipeline of CGTs approaching market is increasing, there is a growing awareness and acknowledgment that outcomes-based reimbursement (OBR) schemes offer a potentially effective mechanism to reduce the long-term data uncertainty and have the potential to ensure greater access and reward for efficacious therapeutic innovation.

One of the takeaways from NICEs regenerative medicines study in 2016 is that outcomes-based payments increased the probability of a therapy being cost-effective but without the risk of eroding product value, unlike upfront discounts. When evaluating an OBR scheme it will be important to consider the feasibility of patient follow-up and the availability of a suitable data collection infrastructure, such as a patient registry. Covid-19 sped the adoption of digitally-enabled processes and CGT companies can leverage new practices such as remote monitoring for long-term patient follow-up. Clearly different CGTs may call for different pricing models in different markets.

The ability to combine clinical trial data and real world data is seen by many as one of the most critical transformations occurring in CGT development. Looking to strike a careful balance between the benefits of early access and the potential, still-unknown, long-term safety risks, regulators including the European Medicines Agency (EMA) and Medicines & Healthcare products Regulatory Agency (MHRA) in the UK are already performing accelerated assessment and granting approvals for CGTs on the condition that real-world evidence (RWE), based on real-world data, is periodically submitted thereafter.

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Cell and gene therapies: our eyes to the future - Pinsent Masons

Researchers have discovered a mechanism that may explain why people with COVID-19 lose their sense of smell.

Published online February 1 in the journal Cell, the new study finds that infection with the pandemic virus, SARS-CoV-2, indirectly dials down the action of olfactory receptors, proteins on the surfaces of nerve cells in the nose that detect the molecules associated with odors.

Led by researchers from NYU Grossman School of Medicine and Columbia University, the new study may also shed light on the effects of COVID-19 on other types of brain cells and other lingering neurological effects of COVID-19 such as brain fog, headaches, and depression.

Experiments showed that the presence of the virus near nerve cells (neurons) in olfactory tissue brought an inrushing of immune cells, microglia, and T cells that sense and counter infection. Such cells release proteins called cytokines that changed the genetic activity of olfactory nerve cells, even though the virus cannot infect them, say the study authors. Where immune cell activity would dissipate quickly in other scenarios, in the brain, according to the teams theory, immune signaling persists in a way that reduces the activity of genes needed for the building of olfactory receptors.

Our findings provide the first mechanistic explanation of smell loss in COVID-19 and how this may underlie long COVID-19 biology, says co-corresponding author Benjamin tenOever, PhD, professor in the Departments of Medicine and Microbiology at NYU Langone Health. The work, in addition to another study from the tenOever group, also suggests how the pandemic virus, which infects less than 1 percent of cells in the human body, can cause such severe damage in so many organs.

One unique symptom of COVID-19 infection is loss of smell without the stuffy nose seen with other infections like the common cold, researchers say. In most cases, the smell loss lasts only a few weeks, but for more than 12 percent of people with COVID-19, olfactory dysfunction persists in the form of ongoing reduction in the ability to smell (hyposmia) or changes in how a person perceives the same smell (parosmia).

To gain insight into COVID-19induced smell loss, the current authors explored the molecular consequences of SARS-CoV-2 infection in golden hamsters and in olfactory tissue taken from 23 human autopsies. Hamsters represent a good model, being mammals that both depend more on the sense of smell than humans, and that are more susceptible to nasal cavity infection.

The study results build on the discovery over many years that the process that turns on genes involves complex 3D relationships, where DNA sections become more or less accessible to the cells gene-reading machinery based on key signals, and where some DNA chains loop around to form long-range interactions that enable the stable reading of genes. Some genes operate in chromatin compartmentsprotein complexes that house the genesthat are open and active, while others are compacted and closed, as part of the nuclear architecture.

In the current study, experiments confirmed that SARS-CoV-2 infection, and the immune reaction to it, decreases the ability of DNA chains in chromosomes that influence the formation of olfactory receptor building to be open and active, and to loop around to activate gene expression. In both hamster and human olfactory neuronal tissue, the research team detected persistent and widespread downregulation of olfactory receptor building. Other work posted by these authors suggests that olfactory neurons are wired into sensitive brain regions, and that ongoing immune cell reactions in the nasal cavity could influence emotions, and the ability to think clearly (cognition), consistent with long COVID.

Experiments in hamsters recorded over time revealed that downregulation of olfactory neuron receptors persisted after short-term changes that might affect the sense of smell had naturally recovered. The authors say this suggests that COVID-19 causes longer-lasting disruption in chromosomal regulation of gene expression, representing a form of nuclear memory that could prevent the restoration of olfactory receptor transcription even after SARS-CoV-2 is cleared.

The realization that the sense of smell relies on fragile genomic interactions between chromosomes has important implications, says Dr. tenOever. If olfactory gene expression ceases every time the immune system responds in certain ways that disrupts inter-chromosomal contacts, then the lost sense of smell may act as the canary in the coal mine, providing early signals that the COVID-19 virus is damaging brain tissue before other symptoms present, and suggesting new ways to treat it.

In a next step, the team is presently seeing whether treating hamsters with long COVID with steroids can restore restrain damaging immune reactions (inflammation) to protect nuclear architecture.

Along with Dr. tenOever, authors of the current study from the Department of Microbiology at NYU Langone Health were Justin Frere, Rasmus Moeller, Skyler Uhl, and Daisy Hoagland. Also leading the study were corresponding authors Jonathan Overdevest and Stavros Lomvardas from the Mortimer B. Zuckerman Mind Brain Behavior Institute at Columbia University. Additional contributors included Marianna Zazhytska, Albana Kodra, Hani Shayya, Stuart Firestein, Peter Canoll, and James Goldman. Also making important contributions were study authors John Fullard and Panos Roussos of the Icahn School of Medicine at Mt. Sinai; Arina Omer of Baylor Genetics in Houston; and Qizhi Gong of the Department of Cell Biology and Human Anatomy, School of Medicine, University of California at Davis.

Funding for the study was provided by National Institutes of Health grants NIDCD 3R01DC018744-01S1 and U01DA052783, as well as a Howard Hughes Medical Institute Faculty Scholars award and the Zegar Family Foundation.

Greg WilliamsPhone: 212-404-3500gregory.williams@nyulangone.org

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Mechanism Revealed Behind Loss of Smell with COVID-19 - NYU Langone Health

DUBLIN, Feb. 3, 2022 /PRNewswire/ -- The "Precision Medicine Software Market - Global Outlook & Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

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The precision medicine software market size was valued at USD 1,344.28 million in 2021 and is expected to reach USD 2,657.21 million by 2027, growing at a CAGR of 12.03% during the forecast period.

Favorable government initiatives and the adoption of big data analytics and related software continue to drive precision medicine software industry growth. Precision medicine software is one of the fast-growing healthcare systems IT industry segments, driven predominantly by genomics, drug discovery & development, clinical research, and big data analytics.

Start-ups are leveraging many software and machine learning algorithms to help solve major and complex problems such as reducing R&D activities timeline and billion dollars of expenditure during drug development processes.

PRECISION MEDICINE SOFTWARE MARKET SEGMENTATION

The on-cloud segment will witness an absolute growth of more than 100% in the forecast period. Cloud technology supports the industry with an agile and mountable provider engagement model. This provides better outcomes by pushing crucial information to clinicians while pulling vital, real-world insight back from key experts in the field.

Precision oncology has the highest share in Precision medicine practices by application. Oncology is the leading and fastest-growing therapeutic area in the life sciences industry. New treatments are being established at a remarkable pace, with more than 1100 oncology therapeutics in clinical development in the US alone.

GEOGRAPHICAL OUTLOOK

North America: North America made remarkable progress post the Human Genome Project in genome sequencing and precision medicine. The region is actively engaged in developing and commercializing cell and gene therapies with ICT and genome sequencing. This will drive demand in the precision medicine software industry.

Europe: The European Commission has been a driver for developing PM approaches to be readily implemented in healthcare practice. Its efforts started in 2010 with a series of workshops exploring different research areas that can contribute to developing precision medicine.

APAC: The region will likely witness a dramatic rise and innovation in precision medicine. China has already begun to make significant progress in genomics research, announcing its precision medicine initiative in 2016 with an investment of around USD 9 billion by 2030.

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VENDOR LANDSCAPE

The key players in the precision medicine software market are Syapse, AccessDx Laboratory, Fabric Genomics, Foundation Medicine, Intel, and International Business Machines (IBM).

Companies are resolving to inorganic growth approaches. AccessDx Holdings acquired 2bPrecise to create the industry's most advanced precision medicine enablement solution.

KEY HIGHLIGHTS

Blockchain technology, which works on shared ledgers and distributed networks, can ensure the data is secured and used ethically while prohibiting mishandling. Thus, blockchain technology has a huge scope in the precision medicine market.

Start-ups and scaleups are developing research platforms and techniques to better understand the underlying causes of cancer. For instance, US-America start-up OncXerna creates an RNA expression biomarker panel that permits clinical researchers to develop algorithms for effective treatment using RNA signature derived from biomarker panels.

AI leverages sophisticated computation and deep learning to overcome the obstacles involved in sizeable disparate data sets and generate insights to enable the system to learn and reason. Over the last few years, AI approaches have been used in neurodevelopmental disorders, specifically autism spectrum disorder, epileptic encephalopathy, intellectual disability, attention deficit hyperactivity disorder (ADHD), and rare genetic disorders.

KEY GROWTH FACTORS

Technological Advancements for Improvement of Precision Medicine Delivery

Increased Adoption of Cloud-Based Platform

The emergence of Local & Regional Start-Ups

Prevalence of Cancer, Genetic and Rare Diseases

Increased Partnership Among Software and Pharmaceutical Companies

Key Vendors

AccessDx Laboratory

Fabric Genomics

Foundation Medicine

Intel

IBM

Syapse

Other Prominent Vendors

GenomOncology

Koninklijke Philips

LifeOmic

NantHealth

PhenoTips

PierianDx

Qiagen

Roper Technologies

SOPHiA GENETICS

Translational Software

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage4.1 Market Definition4.2 Base Year4.3 Scope Of The Study

5 Report Assumptions & Caveats5.1 Key Caveats5.2 Currency Conversion5.3 Market Derivation

6 Market at a Glance

7 Introduction7.1 Overview

8 Market Opportunities & Trends8.1 Technological Advances In Precision Medicine Delivery8.2 Increased Adoption Of Cloud-Based Platforms8.3 Emergence Of Local & Regional Start-UPS8.4 Artificial Intelligence In Precision Medicine

9 Market Growth Enablers9.1 High Prevalence Of Cancer, Genetic, & Rare Diseases9.2 Increased Partnerships Among Software & Pharmaceutical Companies9.3 Paradigm Shift Toward Tailored Disease Treatment

10 Market Growth Restraints10.1 High R&D & Implementation Cost Of Precision Medicine10.2 Dearth Of Skilled Professionals10.3 Lack Of Security & Storage For Large Volumes Of Sequenced Data

11 Market Landscape11.1 Market Overview11.2 Market Size & Forecast11.2.1 Geography Insights11.2.2 Deployment Insights11.2.3 Application Insights11.2.4 End-User Insights11.3 Five Forces Analysis

12 Deployment12.1 Market Snapshot & Growth Engine12.2 Market Overview12.3 On-Premise12.4 Cloud

13 Application13.1 Market Snapshot & Growth Engine13.2 Market Overview13.3 Precision Oncology13.4 Pharmacogenomics13.5 Rare Disease

14 End-User14.1 Market Snapshot & Growth Engine14.2 Market Overview14.3 Healthcare Providers14.4 Research Labs14.5 Pharma & Biotech Companies

15 Geography15.1 Market Snapshot & Growth Engine15.2 Geographic Overview

For more information about this report visit https://www.researchandmarkets.com/r/udylyk

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Global Precision Medicine Software Market Report 2022: Market was Valued at $1,344.28 Million in 2021 and is Expected to Reach $2,657.21 Million by...

A new study confirms that G207, a genetically engineered virus developed at UAB, may be a beneficial therapy for brain tumors.

A new study confirms that G207, a genetically engineered virus developed at UAB, may be a beneficial therapy for brain tumors.A new study provides additional evidence of the efficacy of virotherapy for glioblastoma, the most deadly type of brain tumor. The research findings, published Feb. 1, 2022, inClinical Cancer Research, indicate that an oncolytic herpes simplex virus, G207, appears to boost immune response and that this is associated with better overall survival for patients with glioblastoma.

The study, from investigators at theUniversity of Alabama at BirminghamandNationwide Childrens Hospital, builds on previous trials of G207. The current study further examined findings from an earlier Phase 1b study of six patients with glioblastoma recurrence. The earlier study indicated G207 was well tolerated by the patients. Overall survival, a secondary outcome, was generally but not uniformly improved in the study.

In this study, we sought to identify biological differences that could describe the variable survival duration associated with G207 virotherapy, said James Markert, M.D., Ph.D., chair of theDepartment of Neurosurgeryat UABsHeersink School of Medicineand a study co-author.

We performed RNA sequencing from pre- and post-G207 treatment tumor biopsies to investigate changes in gene expression during the treatment interval. We hypothesized that gene expression indicative of an active immune response would be characteristic of patients with improved survival.

The research team found that the adaptive immune response differed between patients and indicated there was an association with this immune response and increased length of survival in patients with recurrent glioblastoma after treatment with G207.

Glioblastomas are central nervous system tumors that are uniformly fatal with median survival of 12 to 15 months from initial diagnosis and four to six months after recurrence. The standard medical care including surgical removal of the tumor, chemotherapy and radiation currently offers limited survival benefit.

Markert, who is a senior scientist in the O'Neal Comprehensive Cancer Center, says previous research has confirmed that glioblastomas have an immunosuppressive quality, meaning they inhibit the bodys immune system from attacking and destroying the tumor. This allows the tumor to evade the immune system and continue to grow and spread. Chemotherapy and radiation therapy, necessary treatments for the tumor, unfortunately exacerbate suppression of the immune response.

Immunotherapies are an evolving strategy for cancer treatment. They are designed to stimulate the immune system to attack tumor cells and have led to remarkable responses in many cancers.

Oncolytic virotherapy, in particular, involves genetically engineered viruses designed to selectively replicate in tumor cells, relieving immunosuppression in the tumor microenvironment and enhancing antitumor immune responses.

Markert first became interested in genetically engineered viruses while a neurosurgical resident in a Boston laboratory more than 30 years ago. The lab was involved in testing the first genetically engineered herpes virus as an anti-cancer drug, based on a suitable candidate initially constructed to serve as a vaccine against herpes. The realization followed that the virus might be effective as a means to destroy brain tumors.

In 2001, Markert and his colleagues published initial results of G207, indicating it was safe to use as a sole therapy as well as in combination with radiation for malignant gliomas.

For the current study, Katherine Miller, Ph.D., a research assistant professor in the Institute for Genomic Medicine at Nationwide Childrens and the Ohio State University College of Medicine, looked at RNA extracted from the patients before and after treatment with G207. Analysis of 770 immune response genes representing 24 different immune cell types revealed that G207 treatment, more than any other component, had the greatest effect on immune gene expression changes.

They also examined which immune cell types were enriched in the post-G207 samples. Looking at 109 marker genes specific to 24 major immune-cell populations to assign cell type scores, they found that nearly all post-G207 samples had a higher abundance of immune cells relative to pre-G207 samples.

Specifically, G207 treatment significantly increased the cytotoxic, T-cell, natural killer, macrophage, neutrophil and dendritic cell scores, said study co-author Kevin Cassady, M.D., professor of pediatrics at theOhio State University College of Medicineand aninfectious diseases physicianat Nationwide Childrens Hospital. We also found that G207 reduced the number of dysfunctional T cells, known as exhausted T cells in that they become tolerant of the tumor they are supposed to attack. Exhausted T cells are one contributor to a poor immune response.

The investigators also looked to see whether there was a direct correlation between the post-G207 treatment gene expression levels and survival.

We detected approximately 500 genes that significantly correlated with patient survival and demonstrated that approximately 50 percent of these genes were related to immune response pathways and functions, Markert said. Our analysis of immune-cell populations after treatment with G207 revealed associations with patient survival and identified important mechanistic events related to cellular immune infiltrate changes including an increase in the myeloid, cytotoxic and T-cell populations, suggesting a relationship between immune gene response and survival duration.

The authors suggest that the gene sets identified permit further examination of gene expression response predictors when evaluating the impact of different recombinant oncolytic viruses. Currently, there are numerous active oncolytic viral clinical trials ongoing, five of which involve the treatment of central nervous system tumors using recombinant herpes virus vectors of various designs.

Malignant glioma is one of the most devastating forms of cancer, Cassady said. Survival after diagnosis is often no more than a few years and is frequently measured in months. Our hope is that these genetically engineered viruses will ultimately extend lifespan and improve quality of life for patients with these malignant brain tumors.

This research was supported by grants from the National Cancer Institute, part of the National Institutes of Health.

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Study supports virotherapy as a potential treatment for brain tumors - The Mix

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REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, announced today that it has appointed Maan Muhsin, M.D., as President and Chief Medical Officer of Kriya Oncology, the companys oncology therapeutic area division. Dr. Muhsin will lead overall strategic, development, and partnership activities to accelerate and expand Kriyas portfolio of transformative gene therapies for cancers of high unmet need.

Maan brings a wealth of experience in the development of novel oncology therapies across a number of modalities, said Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya. He is uniquely positioned to accelerate the expansion and clinical translation of our growing pipeline of gene therapies that can be combined with existing and emerging standards of care in oncology. We are keen to bring forward in vivo gene therapy as a new modality to treat cancer, and Maan is a leader with an exceptional track record that will help us achieve this goal.

Dr. Muhsin previously served as Chief Medical Officer at Medicenna Therapeutics where he designed and executed clinical trials of the companys solid tumor programs. Prior to joining Medicenna, he served as Medical Lead, Oncology Clinical Development for Nektar Therapeutics where he oversaw the progression of the PIVOT-12 and REVEAL clinical studies for metastatic melanoma and advanced and local solid tumors, respectively. Dr. Muhsin has held roles of increasing responsibility at HUYA Bioscience International where he served as the Senior Vice President, Oncology Clinical Development, and at Halozyme Therapeutics where he served as Senior Medical Director, Oncology Clinical Development. He also worked in the U.S. Army Combat Support Hospitals (CSH) and held other positions within the Medical Brigade and the Medical Command under the United States Department of Defense (DoD). Dr. Muhsin completed his medical education at the Baghdad University School of Medicine and completed postgraduate education in oncology drug development at Tufts University Center for the Study of Drug Development (CSDD).

I am excited to join Kriya and look forward to advancing its promising portfolio of gene therapies in oncology, said Dr. Muhsin. While the management of cancer has come a long way in the last decade, I believe in the potential to further enhance the treatment of patients with the incorporation of rationally engineered gene therapies that can transform treatment paradigms for a wide array of cancers. I look forward to leveraging Kriyas fully-integrated gene therapy engine to deliver a pipeline of novel medicines with the potential to significantly impact the lives of cancer patients.

About Kriya

Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market and delivering significant reductions in cost. Kriya is organized into four principal business units: Kriya Technologies, Kriya Therapeutics, Kriya R&D, and Kriya Manufacturing. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in ophthalmology, oncology, rare disease, and chronic disease. Kriya was founded by pioneers in the biopharmaceutical industry and is backed by leading life sciences and technology investors. The company has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. For more information, please visit http://www.kriyatx.com and follow on LinkedIn.

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Danielle CanteyCanale Communicationsdanielle.cantey@canalecomm.com(619) 826-4657

Source: Kriya Therapeutics, Inc.

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Kriya Announces the Appointment of Ma'an Muhsin, MD, as President and Chief Medical Officer of Its Oncology Therapeutic Area Division -...