Category Archives: Gene Medicine

HACKENSACK, N.J., April 8, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), awarded the University of Missouri School of Medicine a bridge grant for $31,500 to continue evaluating CRISPR therapy in a pre-clinical model of Duchenne. The project is led by Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.

Duchenne isthe most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the DMD gene that codes for the dystrophin protein. Currently, a number of strategies are in development to explore the possibility of using gene editing to restore production of a functional dystrophin protein.

Gene editing utilizing AAV-mediated CRISPR/Cas9 is an area of therapeutic development that has significant potential for treating individuals with Duchenne. The use of the CRISPR/Cas9 technology in Duchenne is still in early stages, with various strategies being investigated for how best to modify the DMD gene to restore production of the dystrophin protein. While there is excitement in the potential that gene editing holds, the Duchenne community recognizes that there are many questions that still remain unanswered.

One such question is around the topic of safety. Specifically, if the Cas9 enzyme, which is a bacterial protein, will cause an immune response when it is delivered throughout the body to reach the muscle. Dr. Duan will continue his ongoing investigation into this question by exploring immune responses from systemic delivery of AAV-mediated CRISPR/Cas9 in a large pre-clinical model.

"Duchenne research has progressed significantly over the past several years, with strategies such as gene editing holding incredible possibilities for our community," said Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation. "The work Dr. Duan is doing will contribute greatly to our understanding of how we can safely translate CRISPR/Cas9 gene editing strategies to patients with Duchenne."

"We greatly appreciate the support from PPMD and the Duchenne community," said Dr. Duan. "CRISPR/Cas9 editing therapy has the potential to permanently repair the mutated DMD gene. Studies from many groups, including us, have demonstrated efficient restoration of dystrophin in patient cells and rodent models. Yet, little is known about the immune response to the bacterial derived Cas9 protein. A better understanding on Cas9 immunity will pave the way to the translation of this promising therapeutic modality."

To learn more about PPMD's robust Research Strategy, funding initiatives and strategies for accelerating drug development,click here.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength.Parent Project Muscular Dystrophy (PPMD)fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne atEndDuchenne.org.Follow PPMD onFacebook,Twitter, Instagram, andYouTube.

SOURCE Parent Project Muscular Dystrophy (PPMD)

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Parent Project Muscular Dystrophy Awards $31,500 Grant to Understand Immune Response to CRISPR/Cas-9 Gene Editing Strategies - PRNewswire

FREMONT, Calif., April 6, 2021 /PRNewswire/ -- BIS Research has recently published nine premium market intelligence research studies under the precision medicinesegment. The nine titles published include an in-depth analysis on precision psychiatry, regenerative medicine, genomic data analysis, CRISPR gene-editing, rare disease diagnostics, single-cell multi-omics, and cell and gene therapy.

Besides, this year's World Health Day, which is to be celebrated on April 7, 2021, demands building a fairer and healthier world for all, especially in the light of the recent global COVID-19 pandemic. The study of emerging technologies under precision medicine is a step by BIS Research to understand how these advancements can help its partners in the healthcare industry toward making quality healthcare available for all.

The studies conducted under the precision medicine division of BIS Healthcare highlight that each market intelligence report compiled offers an update on micro segments that are influencing the market, existing products and the advancements, market share analysis of various key players along with their financials, end-user preference data, and detailed global and regional market study analysis, among other things.

Regenerative medicine is challenging the current healthcare practices by targeting the treatments for the root causes of diseases and disorders, thereby transforming it into an incredibly exciting space full of opportunities. As per the market research conducted by BIS Research, the regenerative medicines marketwas estimated at $21.59 billion in 2019 and is expected to grow at a CAGR of 13.99% during the forecast period 2020-2030. The growth can be attributed to factors, including increasing consolidation among healthcare juggernauts and rising number of clinical trials for regenerative therapies, among others.

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The precision psychiatry market is the next big thing under the precision medicine umbrella. The initiatives undertaken in advancing sequencing technologies tools have facilitated an increasing number of psychiatric genomic and proteomic studies to be performed in the field of neurology. As per BIS Research analysis, the global precision psychiatry marketis projected to grow at a CAGR of 17.26% during the forecast period 2021-2026. Growth in this market is expected to be driven by the decreasing cost of sequencing, increasing adoption of inorganic growth strategies in the market, and global increase in mental health burden.

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The BIS Research study on somatic genetic testing markethighlights that the market was estimated at $6.90 billion in 2020 and is projected to reach $16.79 billion by the end of 2030. The growth of the market is influenced by several factors, including ambiguous regulatory and reimbursement policies and lack of trained professionals in the oncology field.

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Rare disease diagnostics has grown significantly since the technology was first commercialized with whole genome and exome sequencing, but it is important to quantify that growth and describe future trends. Based on the study by BIS Research, the rare disease diagnostics marketwas estimated at $28.68 billion in 2019 and is projected to reach $70.69 billion by 2030. However, challenges including expensive sequencing procedures and their applications in medical treatments and high capital requirements hampering the expansion of global reach are in a way hindering the growth of the market.

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The development of genome engineering with potential applications proved to reflect a remarkable impact on the future of the healthcare and life science industry. The high efficiency of the CRISPR-Cas9 system has been demonstrated in various studies for genome editing, which resulted in significant investments in the field of genome engineering. The global CRISPR gene editing marketwas valued at $846.2 million in 2019 and is expected to grow at a CAGR of 26.86% during the forecast period 2020-2030.

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The genomic data analysis and interpretation industry is witnessing an upsurge due to the growing number of genomic research initiatives being undertaken by the developing and developed countries coupled with the decreasing cost of sequencing, resulting in the high demand for software solutions for genomic data analysis. According to the market analysis published by BIS Research, the global genomic data analysis and interpretation marketis predicted to grow at a CAGR of 10.21% during the forecast period 2020-2030.

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BIS Research healthcare experts have found the single-cell multi-omics marketto be one of the most rapidly evolving markets, which is predicted to grow at a CAGR of 21.16% during the forecast period 2020-2025. The market is driven by the need for the development of an advanced solution based on single-cell technology for clinical research in various applications such as cancer, rare disease, cell biology, and synthetic biology.

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Cell and gene therapy are overlapping fields of biomedical research having similar therapeutic goals, which target DNA or RNA inside or outside the body. Both therapies aim at modifying genetic material for the treatment of a disease. Gene therapy uses genetic material, or DNA, to manipulate a patient's cells for the treatment of an inherited or acquired disease. According to the market study by BIS Research, the global cell and gene therapy marketwas valued at $2.59 billion in 2020, and it is expected to grow at an impressive CAGR of 33.82% during the forecast period 2021-2027.

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Expert analysts at BIS Research have found the manufacturing QC for cell and gene therapy to be one of the most rapidly evolving and dynamic markets. The global market for cell and gene therapy manufacturing QCis predicted to grow at a CAGR of 22.80% over the forecast period 2020-2030. The market is driven by certain factors, which include the increasing prevalence of cancer and chronic diseases, rising number of clinical trials for cell and gene therapy, steady investments and consolidations in the cell and gene therapy market, and favorable regulatory environment.

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The studies conducted under BIS Healthcare offer strategic recommendations that can help organizations in tracking various products, trends, and technologies that are changing the dynamics of the market. BIS Research also offers bespoke research services to help organizations meet their objectives.

For Custom Research Services: https://bisresearch.com/our-offerings/custom-research

How can market intelligence published under BIS Healthcare add value to an organization's decision-making process?

About BIS Research:

BIS Research, a premium market intelligence company, offers in-depth insights and consulting to Fortune 500 companies. We are established and known for tracking the growth of deep technologies across key industry verticals and the subsequent challenges and opportunities associated with deep tech across markets, applications, and products.

With more than 500 clients, over 7000 plus primary interviews, and approximately 200 reports published in a year, BIS Research has set an interesting growth trajectory for itself. We provide market estimations, analysis on emerging high-growth applications, technology analysis, highly segmented granular country-level market data, and other important market parameters that come in handy for our clients during strategic decision-making.

We publish a wide gamut of syndicated market studies across industry verticals. In addition, bespoke market studies are another area where BIS Research excels at, along with consulting services.

BIS healthcare vertical offers intelligence in the healthcare technology market for medical devices, digital health, life sciences, robotics and imaging, information technology, MRD testing, and other emerging healthcare technologies, covering the entire industry spectrum. In the past five years, BIS Healthcare has published more than 50 reports under the precision medicine banner. Additionally, BIS Research has been nominating 'Top 25 Voices' in precision medicine on its Insight Monk platform for the past two years successfully.

Contact:Bhavya BangaEmail: [emailprotected] BIS Research Inc.39111 PASEO PADRE PKWY STE 313,FREMONT CA 94538-1686Visit our Blog @ https://blog.bisresearch.com/Connect with us on LinkedIn @ https://www.linkedin.com/company/bis-research Connect with us on [emailprotected] https://twitter.com/BISResearch

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BIS Research Publishes Nine Market Intelligence Reports in Precision Medicine in the First Quarter of 2021 - PRNewswire

Women tend to be more greatly affected by chronic pain, which may be due to differences in the group of genes that influence the severity of the condition.

A team of researchers, led by Kiera Johnston of the University of Glasgow, conducted a genetic study investigating chronic pain in men and women. They found that there were 31 genes in women associated with chronic pain and 37 in men, with only one overlapping gene associated with chronic pain in both sexes.

The team conducted a Genome Wide Association Study (GWAS), which screens the genome for genes that are associated with disease. The study consisted of 209,000 women and 178,000 men.

They also compared the genes to see whether they interacted with each other and found that many of the genes would help others perform, suggesting that chronic pain could be influenced by multiple genes and gene interactions.

In addition, the team found that all of the genes in men and all but one of the genes in women were actively switched on in cells around the dorsal root ganglion, a nerve cluster in the spinal cord responsible for transmitting pain signals from around the body back up to the brain.

In their paper, published in PLOS Genetics, the authors say this builds on previous work that shows chronic pain originates largely in the brain, and sometimes in the sites where the pain is experienced.

They also suggest that the differences in chronic pain between sexes is at least somewhat genetically based and needs to be considered when developing treatments.

Overall, they argue that chronic pain research would benefit from taking individual experiences based on sex into account.

Our study highlights the importance of considering sex as a biological variable and showed subtle but interesting sex differences in the genetics of chronic pain, says Johnston.

Dr Deborah Devis is a science journalist at The Royal Institution of Australia.

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

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Chronic pain in women could be genetic - Cosmos

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its Executive Vice President and Chief Operating Officer, Michael Redman, will present at the annual Cell & Gene Meeting on the Mediterranean, which will take place virtually April 6-9, 2021.

Event: 2021 Virtual Cell & Gene Meeting on the MediterraneanDate: The conference will take place virtually Tuesday, April 6 - Friday, April 9Time: Company presentations will be available to view on-demand throughout the entirety of the conferenceRegistration Link: https://bit.ly/3wdVAd1

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mediterranean is a four-day virtual conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies. The meeting also includes more than 50 panelists and featured speakers taking part in 13 in-depth sessions covering all aspects of cell and gene therapy commercialization.

Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Laura Stringham at lstringham@alliancerm.org and interested media should contact Kaitlyn Dupont at kdupont@alliancerm.org.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for REQORSA for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone.

For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of REQORSA immunogene therapy drug, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining any FDA approvals of REQORSA and our other potential product candidates including whether we receive necessary approvals to commence clinical trials or benefit from fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex to Present at the 2021 Virtual Cell & Gene Meeting on the Mediterranean - Business Wire

LEXINGTON, Mass. and AMSTERDAM, April 08, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that three manuscripts on preclinical data from its gene therapy candidate AMT-130 in Huntingtons disease have been accepted for publication, in the journals Science Translational Medicine, Brain Science, and Brain Communications. The publications show the safety and efficacy of AMT-130 in the deep brain structures of a large animal model and outline a promising novel efficacy biomarker for AMT-130.

Taken together, these publications demonstrate widespread biodistribution and strong, durable efficiency of AMT-130 in disease-relevant regions in a large brain, stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. The data provide further support for the potential therapeutic value of AMT-130, and we remain enthusiastic about our Phase I/II clinical trial of AMT-130 in patients with Huntingtons disease.

Widespread and Sustained Target Engagement in Huntington Disease Minipigs

The paper published this week in Science Translational Medicine examines the translatability and long-term durability of AMT-130 in transgenic Huntingtons disease minipigs, which were used to assess the biodistribution and target engagement in a larger brain. The minipig model is the largest diseased animal model available, generally weighing up to 300 pounds.

AMT-130 was administered by MRI-guided convention-enhanced delivery (CED) at a single dose, bilaterally in the caudate and putamen. Vector DNA distribution and transgene expression in minipig brains demonstrated extensive brain coverage comparable at the interim sacrifice timepoints of 6- and 12-months post administration, leading to significant lowering of mutant huntingtin (mHTT) protein in the brain.

At 12 months, the most pronounced mHTT protein lowering was observed in the putamen (85%), caudate (80%) and amygdala (78%), followed by thalamus (56%) and cerebral cortex (44%).

The publication, Widespread and Sustained Target Engagement in Huntington Disease Minipigs upon Intrastriatal MicroRNA-based Gene Therapy, is available online in the journal Science Translational Medicine (DOI: 10.1126/scitranslmed.abb8920).

Well-tolerated in non-human primates and rats

In addition, a GLP toxicity study of AMT-130 in non-human primates and rats was published in January 2021 in the journal Brain Science. The study demonstrated an excellent safety profile and biodistribution after MRI-guided CED of AMT-130 in the treated animals. One-time bilateral administration in the caudate and putamen resulted in widespread vector DNA and miHTT transgene distribution in the brain, particularly in areas associated with HD pathology. Intrastriatal administration of AAV5-miHTT was well tolerated, with no clinically relevant changes in either species.

The publication, Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology, is available online in the journal Brain Science (DOI:10.3390/brainsci11020129).

Monitoring Durability of MicroRNA-based Therapies

A third manuscript was published last week in the journal Brain Communications, examining the potential use of measuring therapeutic HTT microRNA (miHTT) in extracellular vesicles in CSF as sources to monitor the expression and durability of gene therapies in the brain. After AAV treatment in non-human primates, the secretion of mature engineered microRNA molecules was confirmed, with extracellular microRNA levels correlating with viral dose and cellular microRNA expression in neurons. In investigating the detection of engineered microRNAs over time in the CSF of non-human primates after a single intrastriatal injection of AAV5-miHTT, quantifiable engineered microRNA levels enriched in extracellular vesicles were detected in the CSF up to two years after brain infusion.

The results confirm the long-term expression (up to two years) of AAV5-delivered microRNAs in non-human primates and provide further support for the potential use of extracellular vesicle-associated microRNAs as novel biomarkers in ongoing clinical trials of gene therapies for neurodegenerative diseases, including AMT-130.

The publication, Secreted therapeutics: Monitoring durability of microRNA-based gene therapies in the central nervous system, is available online in the journal Brain Communications (DOI:10.1093/braincomms/fcab054).

About AMT-130

AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that lowers Huntingtin protein in Huntingtons disease patients. AMT-130 is uniQures first clinical program incorporating its proprietary miQURE platform. miQURE is designed to degrade disease-causing genes without off-target toxicity and induce silencing of the entire target organ through secondary exosome-mediated delivery.

About Huntingtons Disease

Huntingtons disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no currently approved therapies to delay the onset or to slow the diseases progression.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to whether we will advance our Phase I/II gene therapy clinical trial of AMT-130 in Huntingtons disease. uniQures actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, the regulatory approval of that transaction, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures periodic securities filings, including its Annual Report on Form 10-K filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and uniQure assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

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uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington's Disease Showing Safety of Administration in NHPs and Widespread...

SAN DIEGO and CALGARY, AB, April 5, 2021 /PRNewswire/ -- Oncolytics BiotechInc. (NASDAQ: ONCY) (TSX: ONC) today announced that it will host a Key Opinion Leader (KOL) webinar discussing AWARE-1 data, the immunotherapeutic effects of pelareorep in breast cancer, and its synergistic activity with CAR T cells in solid tumors. The webinar will take place on Monday, April 12, 2021 at 2:00 pm ET.

The webinar will feature presentations by Key Opinion Leaders Aleix Prat, M.D., Ph.D. (Clnic Barcelona) and Richard Vile, Ph.D., (Mayo Clinic). Dr. Prat's portion of the presentation will focus on data from the AWARE-1 window-of-opportunity clinical trial evaluating pelareorep with and without atezolizumab (Tecentriq) in early-stage breast cancer, which will be presented at this year's American Association for Cancer Research (AACR) Annual Meeting. Dr. Vile will then discuss the results of a preclinical study evaluating pelareorep and chimeric antigen receptor (CAR) T cell combination therapy in solid tumors (linkto PR, linkto poster).

During the event, Oncolytics' management team will also give a corporate update and discuss the company's upcoming milestones. Dr. Prat, Dr. Vile, and company management will be available to answer questions following the formal presentations.

To register for the event, please click here.

About the KOLs

Aleix Prat, M.D., Ph.D.is the Head of the Medical Oncology Department of the Hospital Clnic Barcelona (Spain), Associate Professor at the University of Barcelona, Head of the Translational Genomics and Targeted Therapeutics in Solid Tumors Lab at IDIBAPS, and President of the governing board of the Spanish Breast Cancer Cooperative Research Group (SOLTI), which performs clinical trials of excellence in oncology. He was also named a member of the Executive Board of The Breast International Group (BIG) in 2018. BIG is an international non-profit organization that is linked to more than 3,000 hospitals and includes more than 10,000 experts and more than 56 cooperative groups from around the world.

Over his career, Dr. Prat obtained worldwide prestige as a research scientist in the field of breast cancer genomics and biomarker development. In 2008, he became a postdoctoral research associate (2008-2012) at the Lineberger Comprehensive Cancer Center (University of North Carolina at Chapel Hill, USA) in the Laboratory of Prof. Charles M. Perou, a world-renowned translational researcher in breast cancer. During this postdoctoral experience, he discovered and characterized a new molecular subtype of breast cancer, known as a Claudin-low (Prat et al. Breast Cancer Research 2010; Citations: 1,518). In addition, he contributed to the breast cancer portion of The Cancer Genome Atlas (Nature 2012; Citations: 4,661), which was a landmark molecular characterization study in the field of cancer research.

Richard Vile, Ph.D.is a world-renowned scientist and member of the Oncolytics Scientific Advisory Board with extensive experience studying pelareorep. As a recognized KOL, his research focuses on several areas of immuno-oncology, including oncolytic viruses, adoptive cell therapies (ACTs) such as chimeric antigen receptor (CAR) T cells, and potential synergistic interactions between oncolytic viruses and ACTs. In addition to his role as a professor at the Mayo Clinic ("Mayo"), Dr. Vile is the Director of Mayo's Immuno-oncology and Gene and Virus Therapy programs and Co-Director of the Cancer Immunology and Immunotherapy program. He also serves on the editorial board of several prestigious scientific journals, including Molecular Therapy, Gene Therapy, The Journal of Gene Medicine, and OncoImmunology. Dr. Vile received his B.A. in Biochemistry from the University of Oxford and his Ph.D. in Viral Vectors from the University of London.

About AWARE-1AWARE-1 is an open label window-of-opportunity study in early-stage breast cancer enrolling 38 patients into five cohorts:

The study combines pelareorep, without or with atezolizumab, and the standard of care therapy according to breast cancer subtype. Tumor tissue is collected from patients as part of their initial breast cancer diagnosis, again on day three following initial treatment, and finally at three weeks following treatment, on the day of their mastectomy. Data generated from this study are intended to confirm that pelareorep is acting as a novel immunotherapy, to evaluate potential synergy between pelareorep and checkpoint blockade, and to provide comprehensive biomarker data by breast cancer subtype. The primary endpoint of the study is overall CelTIL score (a measurement of cellularity and tumor-infiltrating lymphocytes). Secondary endpoints for the study include CelTIL by breast cancer subtype, safety, and tumor and blood-based biomarkers.

For more information about the AWARE-1 study, refer tohttps://clinicaltrials.gov/ct2/show/NCT04102618.

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

About Oncolytics Biotech Inc.

Oncolytics is a biotechnology company developing pelareorep, an intravenously delivered immuno-oncolytic virus. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype -- turning "cold" tumors "hot" -- through innate and adaptive immune responses to treat a variety of cancers.

Pelareorep has demonstrated synergies with immune checkpoint inhibitors and may also be synergistic with other approved immuno-oncology agents. Oncolytics is currently conducting and planning additional studies of pelareorep in combination with checkpoint inhibitors and targeted therapies in solid and hematological malignancies, as it prepares for a phase 3 registration study in metastatic breast cancer. For further information, please visit:www.oncolyticsbiotech.com.

This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended and forward-looking information under applicable Canadian securities laws (such forward-looking statements and forward-looking information are collectively referred to herein as "forward-looking statements"). Forward-looking statements contained in this press release include statements regarding Oncolytics' belief as to the potential and benefits of pelareorep as a cancer therapeutic; Oncolytics' expectations as to the purpose, design, outcomes and benefits of its current or pending clinical trials involving pelareorep; and other statements related to anticipated developments in Oncolytics' business and technologies. In any forward-looking statement in which Oncolytics expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause Oncolytics' actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of pelareorep as a cancer treatment, the success and timely completion of clinical studies and trials, Oncolytics' ability to successfully commercialize pelareorep, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. In particular, we may be impacted by business interruptions resulting from COVID-19 coronavirus, including operating, manufacturing supply chain, clinical trial and project development delays and disruptions, labour shortages, travel and shipping disruption, and shutdowns (including as a result of government regulation and prevention measures). It is unknown whether and how Oncolytics may be affected if the COVID-19 pandemic persists for an extended period of time. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results and financial condition. Investors should consult Oncolytics' quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements, except as required by applicable laws.

Company Contact

Kirk Look

Chief Financial Officer

+1-403-670-7658

[emailprotected]

Investor Relations for Oncolytics

Timothy McCarthy

LifeSci Advisors

+1-917-679-9282

[emailprotected]

SOURCE Oncolytics Biotech Inc.

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Oncolytics Biotech to Host Key Opinion Leader Webinar to Discuss AWARE-1 Data, the Immunotherapeutic Effects of Pelareorep in Breast Cancer, and its...

A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye. The clinical trial was conducted by researchers at the Scheie Eye Institute in the Perelman School of Medicine. Results of the case, detailed in a paper published in Nature Medicine, show that the treatment led to marked changes at the fovea, the most important locus of human central vision.

The treatment was designed for patients diagnosed with Leber congenital amaurosis (LCA)an eye disorder that primarily affects the retinawho have a CEP290 mutation, which is one of the more commonly implicated genes in patients with the disease. Patients with this form of LCA suffer from severe visual impairment, typically beginning in infancy.

Our results set a new standard of what biological improvements are possible with antisense oligonucleotide therapy in LCA caused by CEP290 mutations, says co-lead author Artur V. Cideciyan, a research professor of ophthalmology. Importantly, we established a comparator for currently-ongoing gene editing therapies for the same disease, which will allow comparison of the relative merits of two different interventions.

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A single injection reverses blindness in patient with rare genetic disorder | Penn Today - Penn Today

PITTSBURGH, April 08, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (NeuBase or the Company), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, announced today that it will host a virtual R&D day for investors and analysts on Tuesday, June 8, 2021, from 12:30 p.m. to 2:30 p.m. EDT.

During the event, NeuBase will present new data, along with an in-depth review of the Companys pipeline of drug candidates including in Huntingtons disease, myotonic dystrophy type 1, and a new oncology program for a target that has previously been thought of as undruggable as well as an introduction to the expanded management team.

Additional details will be made available prior to the event. The event will be webcast live on NeuBases website at https://ir.neubasetherapeutics.com/news-events/ir-calendar. Following the live webcast, a replay will be available on the Companys website and archived for approximately 90 days.

About NeuBase Therapeutics, Inc.NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines which can be designed to increase, decrease, or change gene function, as appropriate, to resolve genetic defects that drive disease. NeuBase's targeted PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. Forward-looking statements include, among others, those related to our expectations regarding virtual R&D day and the information to be presented during the event. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the timing of the availability of data from the Company's clinical trials; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.com OP: (617)430-7576

NeuBase Media Contact:Jessica Yingling, Ph.D.Little Dog Communications Inc.jessica@litldog.com+1 (858) 344-8091

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NeuBase Therapeutics to Host a Virtual R&D Day on June 8th to Provide Updates on the Drug Development Pipeline Targeting Genetic Diseases -...

Tao Guo,1 Xue Dong,2 Shanli Xie,3 Ling Zhang,4 Peibin Zeng,5 Lin Zhang6

1Department of Gynecology and Obstetrics, West China Second Hospital, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of China; 2Department of Gynecology, Cheng Du Shang Jin Nan Fu Hospital, West China Hospital, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of China; 3First Peoples Hospital of Guangyuan, Guangyuan, Sichuan, 628000, Peoples Republic of China; 4Department of Gynecology and Obstetrics, Guangyuan Central Hospital, Guangyuan, Sichuan, 628000, Peoples Republic of China; 5West China School of Public Health and West China Fourth Hospital, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of China; 6Department of Forensic Biology, West China School of Preclinical and Forensic Medicine, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of China

Correspondence: Peibin ZengWest China School of Public Health and West China Fourth Hospital, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of ChinaEmail [emailprotected]Lin ZhangDepartment of Forensic Biology, West China School of Preclinical and Forensic Medicine, Sichuan University, Chengdu, Sichuan, 610041, Peoples Republic of ChinaEmail [emailprotected]

Abstract: Ovarian cancer is a common and complex malignancy with poor prognostic outcome. Most women with ovarian cancer are diagnosed with advanced stage disease due to a lack of effective detection strategies in the early stage. Traditional treatment with cytoreductive surgery and platinum-based combination chemotherapy has not significantly improved prognosis and 5-year survival rates are still extremely poor. Therefore, novel treatment strategies are needed to improve the treatment of ovarian cancer patients. Recent advances of next generation sequencing technologies have both confirmed previous known mutated genes and discovered novel candidate genes in ovarian cancer. In this review, we illustrate recent advances in identifying ovarian cancer gene mutations, including those of TP53, BRCA1/2, PIK3CA, and KRAS genes. In addition, we discuss advances in targeting therapies for ovarian cancer based on these mutated genes in ovarian cancer. Further, we associate between detection of mutation genes by liquid biopsy and the potential early diagnostic value in ovarian cancer.

Keywords: ovarian cancer, gene mutation, TP53, PIK3CA, BRCA1/2, KRAS, targeted therapy, liquids biopsy

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Ovarian Cancer: Mechanism, Mutations and Therapeutic Targets | CMAR - Dove Medical Press

Larner College of Medicine faculty Gary and Janet Stein are principal investigators on a new $9 million grant from the National Cancer Institute to explore the epigenetics of breast cancer with the goal of finding new drug targets. (Photos: Sally McCay, Larner College of Medicine)

Vermont Business Magazine A team of University of Vermont scientists and physician investigators at the UVM Cancer Center and the Northern New England Clinical and Translational Research Network has been awarded a $9 million, five-year grant from the National Cancer Institute (NCI) to investigate the underlying causes of breast cancer with the goal of identifying new cellular-level targets that could be treated with drugs to prevent the disease or halt its progression.

The principal investigators for the project are Gary Stein, PhD, Perelman Professor and chair of the UVM Larner College of Medicine Department of Biochemistry and professor of surgery, and Janet Stein, PhD, a professor of biochemistry at the Larner College of Medicine. They lead a large, collaborative team representing scientific expertise across three colleges at the University of Vermont.

The competitive grantabout 5 percent of similar NCI grant proposals are fundedcame to UVM, in part, because of the research teams track record.

The NCI is investing in research groups that have made major, proven contributions, said Gary Stein. There is a high expectation that the progress wont be incremental but will represent a paradigm shift.

Stein and his collaborators have published their earlier findings on the underlying mechanisms of breast cancer in publications ranging from Nature to Cancer Research. The new work will build on the previous research contributions to expand both general understanding of breast cancer dynamics and how that molecular-level knowledge could reveal new potential drug targets leading to novel, more effective treatments.

The research will focus on three interrelated projects to determine what goes awry when cell division in healthy breast tissue becomes defective and produces cancer cells. Its focus will be on epigenetics, the instructions that proteins and nucleic acids within a cells nucleus give to its genetic codeits DNAthat tell genes to turn on or off. When a cell reproduces, these epigenetic instructions are passed on to newly formed cells, along with the parental cell DNA, its genetic blueprint. If the epigenetic instructions dont function precisely, cells can become cancerous.

The goal is understanding cancer-compromised epigenetic control of genes in breast tumors, Janet Stein said.

The first project will examine in detail how instructions for the epigenetic regulation of geneswhether they are turned on or off, or poised to turn onare passed on from a parent cell to its two offspring cells accurately, so there are no defects in the control of its specialized function or its ability to go through cell division in a normal way, Janet Stein said.Were trying to understand, precisely, what are the components, what are the epigenetic factors that remain with the gene during cell division, in order to ensure that you are not going to compromise normal function in the progeny cells.

The second project focuses on the proteins, called histones, that organize the two-and-a-half yards of DNA within a cells nucleus into intricate folds inside a chromosome. If these proteins bind a section of the folded DNA tightly, genes wont be expressed; if the bond is loose, the genes will be expressed or poised to express. In cancer cells, the ability to modify these proteins is undermined. The project will seek both to chart how the DNA organizing process works and identify drug targets that will restore function of cancer-compromised proteins.

In the third project, researchers will examine the functioning of a type of long non-coding RNA found in a cells nucleus that the research team discovered in an earlier project. It is present only when cells divide, the team found, and is associated with breast cancer, especially in its most aggressive forms, and is not responsive to conventional cancer treatments. The earlier work showed that, if the activity of this particular type of RNA is blocked, the cancer cell is unable to reproduce and dies. It is a prime drug target, Gary Stein said.

Technology and Team-based Approach Helped Win the Day

UVM has two other capabilities that helped the Cancer Center team win the grantaccess to advanced technology and the strength of its collaborative scientific team. The Larner College of Medicines advanced genome sequencing, microscopy and bioinformatics core facilities allow researchers to peer into cells and chromosomes and view genes and proteins visually in three dimensions.

We have a saying: Seeing is believing, Gary Stein said. The work would be impossible without this highly advanced instrumentation and the world-class expertise behind that technology, which allow us to identify and visualize modified genes and gene expression in cancer cells. It was definitely a credential that the NCI looked favorably on.

In addition, the NCI places priority on what it calls collaborative team science, an interdisciplinary research approach that is the fabric of the UVM Cancer Center.

Collaboration is the guiding principle for the UVM Cancer Center, Gary Stein said.We engage the complementary perspectives, expertise and experience of clinicians, oncologists, molecular biologists, biochemists, bioinformaticists, pathologists and others. UVMs breadth and culture incentivize partnerships that synergize to accelerate the translation of laboratory discovery to advances in cancer prevention, early detection, treatment and survivorship, he said.

Members of the research team come from the University of Vermonts Larner College of Medicine, College of Nursing and Health Sciences, and College of Engineering and Mathematical Sciences and cover both basic science and clinical departments.Members of the team, in addition to Gary and Janet Stein, are Mark Evans, Seth Frietze, Karen Glass, Jonathan Gordon, Peter Kaufman, Jane Lian, Jason Stumpff, Coralee Tye, Pamela Vacek, Jos Van der Velden, Donald Weaver and Marie Wood. Andrew Fritz and Kirsten Tracy are NCI postdoctoral fellows participating in the project.

Source: University of Vermont 4.6.2021

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UVM gets $9 million to explore epigenetics of breast cancer - Vermont Biz