Category Archives: Transhuman News

Human diversity did not appear to matter to modern medicine. At the time, the state of medical practice ignored the differences between individuals and between men and women.

This practice was reflected in how doctors were trained. They took courses in basic biology, biochemistry, anatomy and physiology. But genetics, the science of variation, was not a required course until recently.

Advances in genetics research have slowly transformed the practice of medicine. There has been a slow accumulation of a long list of diseases caused by variations in a single gene. Since the disease-causing variants generally occurred with some exception in low frequency, these diseases did not occupy the mainstream concern of the medical profession.

All this changed with the Human Genome Project (HGP). Completed in 2003, the sequencing of human genome pushed us into a new era of how genetic diseases would be defined, and how future health services would be delivered.

Medical schools need to do a lot better preparing future physicians and health professionals if the dreams of personalized medicine are to be realized.

Personalized medicine means treating patients based on the individual characteristics of their DNA. The information can be used either in direct intervention, as in cancer treatment, or in predictive medicine.

Different specializations would require varying levels of proficiency: for example, family physicians would need a sufficient background in genetics, while oncologists would need in-depth education.

The HGP made two big promises. First, it promised personalized predictive medicine based on an individuals genome sequences. Disease-causing mutations at different locations on a gene would be identified, and an overall personalized risk score would be calculated that would tell the individual his or her chances of developing that disease.

The second promise was to develop a better and faster cures for complex diseases such as cancer.

The letdown came when genomic studies showed that genes affecting complex diseases were potentially large in number and individually of small effect, and worse still, only a small number of all potential genes affecting a given disease could be identified.

Even more problematic, it turned out that all individuals sharing the same risk factor for a given disease did not develop the disease. This creates a problem for predictive medicine if scientists cannot link a disease to a gene with any certainty.

The uncovered genomic complexity of diseases was contrary to expectations of the Mendelian model, which did not account for genetic variations beyond one gene one disease.

This is where the work my collaborators and I carried out in our labs comes in. Our work in population genetics and evolutionary genomics relates to how these characteristics are calculated and combined into an overall score used in predictive medicine.

My lab specializes in the evolution of molecular complexity and its impact on precision medicine. We also study variation and evolution of sex and reproduction related genes and their role in the evolution of sexual dimorphism in complex diseases and mental disorders. We reviewed three decades of relevant work in genetics, genomics and molecular evolution and drew the following conclusions.

First, we showed that because of the blind nature of evolutionary forces and the role of chance in evolution in humans, many combinations of genes can lead to the same disease. This implies the existence of a considerable amount of redundancy in the molecular machinery of the organism.

Second, we showed that genes do not work alone: gene-gene and gene-environment interactions are a major part of any organisms functional biology. This would explain, for example, why some women with breast cancer genes develop breast or ovarian cancer and some do not.

Third, we showed that since males fight for mates and early reproduction, this would lead to an evolution of male-benefitting mutations even at the cost of them being harmful later, making males vulnerable to diseases in their old age. Male-benefitting mutations harmful to females would trigger a female-driven response leading to the evolution of increased female immunity, and possibly evolution of higher thresholds for complex diseases and mental disorders.

This would explain why many diseases such as autism are more common in boys than girls. In addition, some differences in disease prevalence, such as depression in women, is theorized to be the result of interaction between hormone fluctuation and social stress factors.

If you have sought medical attention, its likely that your doctor may have asked you about your parents and your siblings. Your physician is interested in knowing if there are any health conditions, such as cardiovascular disease, diabetes or high blood pressure that run in the family and that might affect your health.

Future physicians will need to know a lot more than their patients family history.

The number of situations that involve relevant genetic contributions will continue to increase with advances in molecular insights and precision medication. The medical research establishment is becoming increasingly aware of the importance of individual genetic differences and of sex and gender when assessing diseases and health-care proposals. Health professionals must have sufficient expertise in diversity, genomics and gene-environment (gene-drug) interaction.

Future physicians will be part of health networks involving medical lab technicians, data analysts, disease specialists and the patients and their family members. The physician would need to be knowledgeable about the basic principles of genetics, genomics and evolution to be able to take part in the chain of communication, information sharing and decision-making process.

This would require a more in-depth knowledge of genomics than generally provided in basic genetics courses.

Much has changed in genetics since the discovery of DNA, but much less has changed how genetics and evolution are taught in medical schools.

In 2013-14 a survey of course curriculums in American and Canadian medical schools showed that while most medical schools taught genetics, most respondents felt the amount of time spent was insufficient preparation for clinical practice as it did not provide them with sufficient knowledge base. The survey showed that only 15 per cent of schools covered evolutionary genetics in their programs.

A simple viable solution may require that all medical applicants entering medical schools have completed rigorous courses in genetics and genomics.

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Medical schools need to prepare doctors for revolutionary advances in genetics - The Conversation CA

With the COVID-19 pandemic drawing more attention to America's obesity problem, a growing body of research indicates that our genetics should be used to determine what we eat.

Decades of research shows that, at least for most people, the secret to staving off disease is getting plenty of exercise and eating diet high in vegetables and with a healthy mix of fats, protein and carbs. But now a budding field called "nutrigenomics" aims to offer people personalized lifestyle advice based on each person's DNA.

Though still a new area of scientific study, researchers hope food plans based on genetic makeups will be more effective than traditional one-size-fits-all recommendations.

"Given the greater concern for high blood pressure, high blood sugar and obesity, and their association with severe COVID-19, I foresee a great emphasis on personalized nutrition, with the use of data from genetic tests and monitoring blood glucose, to help people make positive choices and decrease their risk," said Brigid Titgemeier, a functional medicine dietitian and founder of beingbrigid.com.

It's not the supplements or the food that we eat, it's what the food does to our body to make it heal itself.

Decades after the Human Genome Project mapped the genes of humans, scientists now are using this information to better understand how food can modify predispositions to disease and immune functions.

Nutrigenomics is described as a genetic approach to personalized nutrition, including not just diet but sleep patterns and one's overall lifestyle.

A doctor consults with a patient regarding diet nutrition in an undated stock image.

"It embraces this idea that despite all of us being 99.9% the same, there is that 0.1% that truly determines how you respond to the world around you," said Dr. Yael Joffe, founder and chief science officer of 3X4 Genetics.

"Following a diet that is restrictive or one seen on social media may result in some improvement, but they aren't sustainable and aren't data driven," said Dr. Marvin Singh, an integrative gastroenterologist and founder of Precisione Clinic. "Nutrigenomics provides an understanding of your predispositions and deficiencies. In terms of weight loss, it can provide data on particular gene mutations you have that might favor you acting or eating a certain way -- or even exercise patterns that may be more helpful."

Accessing one's genetic makeup can be done with saliva sampled from a cheek swab and sent to a lab. Using the data a subject gets back, Joffe said, can help inform that individual which foods can be eaten to turn on or off certain genes.

"We are all going to respond a bit differently when we eat a salad," said Kristin Kirkpatrick, a nutritionist and the president of KAK Nutrition consulting, "since there is no diet that is one-size-fits-all. We need to look at our DNA if we want to lose weight."

Diet and exercise is the first recommended treatment for the majority of the chronic diseases in the U.S. -- hypertension, obesity, diabetes and high cholesterol. But personalized nutrition based on genetics, research has shown, is more effective in reaching long-term weight-loss goals.

"Genetics is an extremely powerful behavioral tool to implement long-standing changes," Joffe added. "It's about you. It's your story. Not something you read on social media or the internet."

In his clinic, Singh finds that patients are more likely to stick to treatment plans tailored to their own genetics, so having access to that data helps him provide a framework for better treatments.

"A low-salt diet is recommended if someone has high blood pressure," Singh said, "but everyone's blood pressure may not respond to this. Using genetic information, I can see if a person's blood pressure would respond favorably to this dietary change and if there is something else that is driving their disease."

A doctor checks the weight of a patient in an undated stock image.

By changing variables such as sleep patterns, diet and exercise, it is ultimately difficult to measure the impact of a genetic test, explained Joffe.

Nutrigenomics is new and constantly evolving, and experts told ABC News there's much left to learn.

"More research needs to be done so we can have even more specific dietary guidance," Titgemeier said. "Right now, certain mutations in our genes can tell us to have a diet low in saturated fat, however, what we don't know is the percentage."

Health care consumers also need to be careful their genetic information doesn't end up in the wrong hands -- some companies have been found to collect and sell data to third parties. One of the best ways to avoid being scammed? Talk to your doctor.

"The best way to start is with your primary care [physician] and asking if they know someone who does nutrigenomics or if they can get some information on this," Kirkpatrick said.

Eventually, experts said, using food as medicine may help reduce the risk of other serious diseases such as Alzheimer's dementia or heart disease.

"It's not the supplements or the food that we eat, it's what the food does to our body to make it heal itself," Joffe said. "This area of gene expression is really the extraordinary power of where nutrition lies."

L. Nedda Dastmalchi, D.O., M.A., an internal medicine resident physician at The George Washington University, is a contributor to the ABC News Medical Unit.

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With obesity on the rise, the best diet may be tailored to our genes, experts say - ABC News

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.

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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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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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About BIS Research:

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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.

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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.

Read more at Penn Medicine News.

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