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Stanford researchers find signs of inflammation in brains of people who died of COVID-19 – Stanford Medical Center Report

Posted: June 23, 2021 at 1:55 am

The most comprehensive molecular study to date of the brains of people who died of COVID-19 turned up unmistakable signs of inflammation and impaired brain circuits.

Investigators at the Stanford School of Medicine and Saarland University in Germany report that what they saw looks a lot like whats observed in the brains of people who died of neurodegenerative conditions such as Alzheimers disease and Parkinsons disease.

The findings may help explain why many COVID-19 patients report neurological problems. These complaints increase with more severe cases of COVID-19. And they can persist as an aspect of long COVID, a long-lasting disorder that sometimes arises following infection with SARS-CoV-2, the virus that causes COVID-19. About one-third of individuals hospitalized for COVID-19 report symptoms of fuzzy thinking, forgetfulness, difficulty concentrating and depression, said Tony Wyss-Coray, PhD, professor of neurology and neurological sciences at Stanford.

Yet the researchers couldnt find any signs of SARS-CoV-2 in brain tissue they obtained from eight individuals who died of the disease. Brain samples from 14 people who died of other causes were used as controls for the study.

The brains of patients who died from severe COVID-19 showed profound molecular markers of inflammation, even though those patients didnt have any reported clinical signs of neurological impairment, said Wyss-Coray, who is the D. H. Chen Professor II.

Scientists disagree about whether SARS-CoV-2 is present in COVID-19 patients brains. We used the same tools theyve used as well as other, more definitive ones and really looked hard for the viruss presence, he said. And we couldnt find it.

A paper describing the study will be published June 21 in Nature. Wyss-Coray shares senior authorship with Andreas Keller, PhD, chair of clinical bioinformatics at Saarland University. The lead authors are Andrew Yang, PhD, a postdoctoral scholar in Wyss-Corays group, and Fabian Kern, a graduate student in Kellers group.

The blood-brain barrier, which consists in part of blood-vessel cells that are tightly stitched together and blob-like abutments created by brain cells projections squishing up against the vessels, has until recently been thought to be exquisitely selective in granting access to cells and molecules produced outside the brain.

But previous work by Wyss-Corays group and by others has shown that bloodborne factors outside the brain can signal through the blood-brain barrier to ignite inflammatory responses inside the brain. This could explain why, as Wyss-Coray and his colleagues have discovered, factors in young mices blood can rejuvenate older mices cognitive performance, whereas blood from old mice can detrimentally affect their younger peers mental ability.

On hearing reports of enduring neurological symptoms among some COVID-19 patients, Wyss-Coray became interested in how SARS-CoV-2 infection might cause such problems, which resemble those that occur due to aging as well as to various neurodegenerative diseases. Having also seen conflicting reports of the viruss presence in brain tissue in other studies, he wanted to know whether the virus does indeed penetrate the brain.

Brain tissue from COVID-19 patients is hard to find, Wyss-Coray said. Neuropathologists are reluctant to take the steps required to excise it because of potential exposure to SARS-CoV-2 and because regulations often prohibit such procedures to prevent viral transmission. But Keller, who has worked in the Wyss-Coray lab as a visiting professor at Stanford, was able to access COVID-19 brain-tissue samples from autopsies conducted at the hospital thats associated with Saarland University.

Using an approach called single-cell RNA sequencing, the scientists logged the activation levels of thousands of genes in each of 65,309 individual cells taken from brain-tissue samples from the COVID-19 patients and the controls.

Activation levels of hundreds of genes in all major cell types in the brain differed in the COVID-19 patients brains versus the control groups brains. Many of these genes are associated with inflammatory processes.

There also were signs of distress in neurons in the cerebral cortex, the brain region that plays a key role in decision-making, memory and mathematical reasoning. These neurons, which are mostly of two types excitatory and inhibitory form complex logic circuits that perform those higher brain functions.

The outermost layers of the cerebral cortex of patients who died of COVID-19 showed molecular changes suggesting suppressed signaling by excitatory neurons, along with heightened signaling by inhibitory neurons, which act like brakes on excitatory neurons. This kind of signaling imbalance has been associated with cognitive deficits and neurodegenerative conditions such as Alzheimers disease.

An additional finding was that peripheral immune cells called T cells, immune cells that prowl for pathogens, were significantly more abundant in brain tissue from dead COVID-19 patients. In healthy brains, these immune cells are few and far between.

Viral infection appears to trigger inflammatory responses throughout the body that may cause inflammatory signaling across the blood-brain barrier, which in turn could trip off neuroinflammation in the brain, Wyss-Coray said.

Its likely that many COVID-19 patients, especially those reporting or exhibiting neurological problems or those who are hospitalized, have these neuroinflammatory markers we saw in the people we looked at who had died from the disease, he added. It may be possible to find out by analyzing these patients cerebrospinal fluid, whose contents to some extent mirror those of the living brain.

Our findings may help explain the brain fog, fatigue, and other neurological and psychiatric symptoms of long COVID, he said.

Wyss-Coray is co-director of the Paul F. Glenn Center for Biology of Aging Research at Stanford, a member of Stanford Bio-X, Stanfords Maternal and Child Health Research Institute, and Wu Tsai Neurosciences Institute at Stanford, and a faculty fellow of ChEM-H.

Other Stanford co-authors of the study are postdoctoral scholars Patricia Losada, PhD, Nicholas Schaum, PhD, Ryan Vest, PhD, Nannan Lu, PhD, and Oliver Hahn, PhD; basic life research scientist Daniela Berdnik, PhD; life science research professionals Maayan Agam and Kruti Calcuttawala; former life science research associate Davis Lee; former visiting student researcher Christina Maat; life science research professional Divya Channappa; David Gate, PhD, instructor of neurology and neurological sciences; M. Windy McNerney, PhD, clinical assistant professor of psychiatry and behavioral sciences; and Imma Cobos, MD, PhD, assistant professor of pathology.

In addition to Keller and Kern, other researchers at Saarland University also contributed to the study.

The work was funded by the Nomis Foundation, the National Institutes of Health (grants T32-AG0047126, 1RF1AG059694 and P30AG066515), Nan Fung Life Sciences, the Wu Tsai Neurosciences Institute and the Stanford Alzheimers Disease Research Center.

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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities – Yahoo Finance

Posted: June 23, 2021 at 1:55 am

WASHINGTON, June 22, 2021 /PRNewswire/ -- We are in a new era of medicine where groundbreaking biopharmaceutical research and development is transforming medicine, but these innovations are meaningless if they don't reach patients, including those in underserved communities. Health disparities are not new, but the COVID-19 pandemic put a spotlight on long-standing health inequities that affect diverse racial and ethnic communities in America. Data shows these populations have been disproportionately impacted by COVID-19. In fact, American Indian/Alaskan Native, Hispanic, and Black populations are approximately twice as likely to die from COVID-19, as compared to non-Hispanic whites.

Medicines in Development for Underlying Medical Conditions Associated with Severe COVID-19 Infection Outcomes

Researchers have found that people with certain health conditions, including chronic conditions such as Alzheimer's disease, certain cancers, chronic kidney disease, chronic lung diseases, type 2 diabetes, heart conditions, HIV infection, liver disease, obesity, sickle cell disease and stroke, are at higher risk of severe illness or death from COVID-19. Many of these conditions are tied to health disparities that disproportionality affect racial and ethnic communities for genetic and environmental reasons, or due to inequities in social and economic conditions.

Today, PhRMA released a new report exploring the 829 medicines in development that aim to address the diseases and conditions that affect racial and ethnic communities at a higher rate and are also associated with worse COVID-19 outcomes.

Among the medicines in development to improve management of these diseases are:

An anti-retroviral treatment for HIV infections. The medicine inhibits HIV-1 replication in human peripheral blood cells by inhibiting capsid protein formation. It is being studied in both heavily treatment-experienced patients with multi-drug resistance and treatment-nave patients living with HIV.

A gene-edited cell therapy that could potentially be a one-time treatment for sickle cell disease, uses zinc finger nucleases (ZFNs), which consists of a protein with a DNA-cutting enzyme, to modify a patient's own hematopoietic stem cells to produce normal-shaped red blood cells using fetal hemoglobin.

A first-in-class medicine for asthma that blocks TSLP, an immune system messenger protein critical in the development and persistence of inflammation of the airways. By blocking TSLP, the release of pro-inflammatory proteins by immune cells will be stopped, preventing asthma exacerbations and improving asthma control.

A potential treatment for renal cell carcinoma, a type of kidney cancer, by stimulating cancer killing cells in the body in combination with an approved immune checkpoint inhibitor. It works by unleashing the body's own powerful immune system to target and kill cancer cells, while leaving normal cells alone.

A medicine for treating large hemispheric infarction, a severe form of ischemic stroke, where brain swelling leads to stroke-related deaths and disabilities. The medicine targets and blocks a receptor that mediates stoke-related brain swelling. In clinical trials, it has demonstrated a potential to reduce brain swelling, disability and the risk of death.

It is critical that all patients, including historically underserved racial and ethnic communities, have access to medicines. One way to reduce barriers to health care access and enable everyone to benefit from new medicines is to ensure that clinical trials are diverse and inclusive and include participants representative of the population the medicine intends (or aims) to treat. The biopharmaceutical industry has been working with patients, communities, regulatory authorities, health care practitioners, academics and policymakers to enhance diversity in clinical trials, so the clinical trial population testing medicines better reflect the patients that will use the new therapies and medicines should they are approved by the U.S. Food and Drug Administration.

Story continues

To this end, PhRMA and its member companies have voluntarily adopted first-ever industry-wide principles on clinical trials diversity, adding a new chapter to the already existing Principles on Conduct Clinical Trials & Communication of Clinical Trial Results. The new clinical trial diversity principles are designed to build trust, reduce barriers to clinical trial access, enhance an understanding of drug effects in diverse patient populations, and promote the sharing of information on policies and practices to increase clinical trial diversity.

Equity is critical to the health and well-being of diverse racial and ethnic communities, and it remains essential to a robust ecosystem of innovation. America's biopharmaceutical companies are pushing for necessary systemic and long-term change to better meet the needs of underserved communities in America.

To learn more about the PhRMA Equity Initiative and PhRMA's commitment to inclusion, visit https://phrma.org/Equity and tune in to The Atlantic's Health Equity Summit where PhRMA's Chief Operating Officer, Lori Reilly, and Genentech's Chief Diversity Officer, Quita Highsmith, will have a conversation about building trust in clinical trials.

Learn more about the medicines in development to address health equity here.

This post originally appeared on the Catalyst blog.

CONTACT: Andrew Powaleny, newsroom@phrma.org, 202-835-3460

Pharmaceutical Research and Manufacturers of America.(PRNewsFoto/PHARMACEUTICAL RESEARCH & MANUFACTURERS OF AMERICA)

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More than 800 medicines are in development for diseases that disproportionately affect racial and ethnic communities - Yahoo Finance

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Arab Health: Innovative therapy can kill cancer cells, viral infections – Khaleej Times

Posted: June 23, 2021 at 1:55 am

An innovative immunotherapy for cancer and viral infections, was jointly launched by the Ministry of Health and Prevention (MoHAP) and the Emirates Health Services (EHS), in cooperation with Japans Kyoto University.

The announcement was made on the second day of the Arab Health 2021 that is taking place at the Dubai World Trade Centre until June 24.

Explaining the new treatment, a ministry official said: The treatment is based on the clinical application of the therapy using T-cell preparation. T-cells are white blood cells in the immune system that fight infection. The goal of the T-cell immunotherapy is to reprogramme a persons own T-cells, so they can find and destroy cancer cells wherever they are hiding in the body.

It was found that such type of cells have the ability to fight cancer and viral infections. The T-cell medicine will be produced using the a unique cell technology. These cells can be produced in large numbers and stored in appropriate conditions so they can be administered to patients when needed, the ministry stated.

Dr Kalthoum Al Balushi, Director of Hospitals Department, said: The groundbreaking treatment technology for cancer and viral infections, in cooperation with the Kyoto University, represents a paradigm shift in health services provided by the Ministry and the EHS.

Al Balushi added that the treatment is based on stimulating immune cells to fight cancer cells, using pluripotent stem cells, which is a recent global trend that has begun to open great prospects for improving the quality of life of patients.

Dr. Youssef Mohamed Al Serkal, Director-General of EHS, noted: Although cancer prevalence in the UAE is considered lower than in other parts of the world, we work hard to make a qualitative shift in cancer and viral infection healthcare. This is part of our strategy to provide healthcare services in innovative and sustainable ways, and implement the national strategy to reduce cancer mortality rates.

Al Serkal pointed out that the ministry and EHS support the National Cancer Control Programme, and prepare a road-map to achieve the target indicator.

The two entities, he added, are also responsible for analysing the current status of cancer diseases, diagnostic and therapeutic pathways, do research and studies on the control of cancer diseases and viral infections, and support workshops and educational and training activities. awareness campaigns, and innovative initiatives.

saman@khaleejtimes.com

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Scailyte Announces Michael Brenner, of Harvard Medical School, as an Advisor to Its Board of Directo – PharmiWeb.com

Posted: June 23, 2021 at 1:55 am

BASEL, Switzerland-June 22, 2021- (Newswire.com)

Artificial Intelligence (AI)-driven biomarker discovery innovator Scailyte announced today that Michael Brenner MD, Elizabeth Fay Brigham Professor of Medicine at Harvard Medical School, has joined Scailyte as an advisor to the Board of Directors.

Prof. Brenner is the director of Brigham and Women's Hospital's Human Immunology Center, which creates and performs single-cell transcriptional analysis and functional investigations in order to decipher human autoimmune illnesses, stratify patient groups, and find prospective therapeutic target cells and pathways. He is a member of the National Academy of Sciences in the United States and has received numerous awards.

Prof Brenner's research focuses on T cell biology, antigen presentation, microbial pathogenesis, and autoimmunity. His research discovered the integrin molecule aEb7 (CD103) and its role in leukocyte homing, as well as a role for the CD1 antigen presentation system in the activation of T cells and NKT cells. His team has recently identified distinct fibroblast subpopulations that are pathologically expanded in disease, as well as a new T helper cell population, T peripheral helper (Tph) cells, that are implicated in antibody production in autoantibody-mediated disorders.

Michael Brenner, MD comments: "I am thrilled to support Scailyte in their pioneering efforts combining single-cell technologies and artificial intelligence for biomarker discovery. Single-cell technology is moving into the clinical space, and Scailyte is there at the right moment to capture the potential and create a new generation of clinical applications that will make a difference for patients."

About Scailyte

Scailyte AG is a spin-off of ETH Zurich and combines single-cell technologies, high-quality datasets and machine learning methods to identify disease-specific biomarkers, with a focus on oncology and immunology. Scailyte's proprietary best-in-class data analysis platform ScaiVision associates multimodal single-cell datasets (RNA-/TCR-/BCR-seq, proteomics, etc) with clinical endpoints, such as disease diagnosis, progression, severity, treatment response, in order to identify ultra-sensitive biomarker signatures. Thanks to the single-cell resolution and AI-driven approach, ScaiVision is the most powerful and cutting-edge approach to develop methods for precise diagnosis and prediction of therapy response. Scailyte leverages a global network of clinicians to identify disease indications with high unmet needs and collaborates with global pharmaceutical and biotech companies to translate the novel biomarkers into ultra-sensitive companion diagnostics (CDx) and IVD assays.

For more information, visit http://www.scailyte.com and connect on social media @LinkedIn and @Twitter.

Press Release ServicebyNewswire.com

Original Source:Scailyte Announces Michael Brenner, of Harvard Medical School, as an Advisor to Its Board of Directors

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Cost watchdog ICER will bless some high-priced gene and cell therapies, but only with solid proof of benefit: Analyst – FiercePharma

Posted: June 23, 2021 at 1:55 am

On the surface, it may seem that the higher the price of a new medicine, the harder it is for its developer to get a thumbs-up from drug-price watchdog Institute for Clinical and Economic Review (ICER). For companies in the rapidly growing gene and cell therapy field, that would make ICERs review a tough hurdle to overcome, particularly if the agencys opinions take on more weight in determining insurance coverage decisions in the future.

But analysts at Mizuho Securities dug into most of ICERs verdicts on gene and cell therapies to date and found a surprising trend: The institutes reviewers have deemed more than half the products they reviewed so far to be cost-effective. And the evidence they used to reach those positive conclusions offers important lessons on research, development and pricing to developers of the next generation of gene and cell therapies, said analyst Difei Yang, Ph.D., the lead author of two reports Mizuho sent to clients last week, in an interview.

Clearly, theyre looking at the amount of time the patient gainsbut also the quality of life, Yang said. Companies that put in the effort to provide robust data proving a gene or cell therapy could outperform the standard of care on both measureshead-to-head trials, solid evidence of long-term benefit and so forthhad a higher likelihood of a positive ICER verdict on cost-effectiveness, Mizuho found.

Makers of CAR-T cancer cell therapies have so far been much more successful at proving superiority over the standard of care than have manufacturers of gene therapies for rare diseases, Mizuho noted. ICER approved of the cost-effectiveness of the first two CAR-Ts on the market, Gileads Yescarta for lymphoma and Novartis Kymriah for lymphoma and leukemia, both approved in 2017 and launched at $373,000 and $475,000, respectively. And the agency has already said it will give the thumbs-up to the yet-to-be-approved multiple myeloma CAR-T cilta-cel from Janssen and Legend Biotechprovided that it is priced at $475,000 or less.

Only one of four CAR-Ts that ICER reviewed did not hit the cost-effectiveness threshold: Bristol Myers Squibbs ide-cel for multiple myeloma, which was launched this spring with the brand name Abecma at a price of $419,500. A spokesperson for BMS griped at the time that ICER relied on inappropriate modeling of ide-cels overall survival data and a misguided view of retreatment.

ICERs president Steve Pearson, M.D.,cited important evidence gaps in the data available to the agency while it was reviewing the cost-effectiveness of multiple myeloma treatments.

Pearson said in an interview that ICERs reviewers sometimes struggle to determine how data from what are often brief studies will translate to real-world outcomes. We need to see longer-term data that we can connect to what were seeing in the short term, Pearson said. What we really want to see are patient-relevant outcomes, like how well theyre functioning and their quality of life.

Often, companies fall short when it comes to providing data that provide a clear contrast between patients who receive gene or cell therapies and those who do not, Pearson said. They may say [non-treated] patients would only have six months to live, but sometimes the comparison is just not that clean, he said. [Or] the [standard-of-care] data may be 10 years old. We know that care changes over 10 years.

BMS took a step in that direction with another CAR-T in its portfolio, Breyanzi, which was approved in February for large B-cell lymphoma. Last week, it released trial data showing an improvement in event-free survival in patients who received the CAR-T treatment versus those who were treated with the gold standard, Roches Rituxan, high-dose chemotherapy and stem cell transplant.

It is too early to define Breyanzis ability to extend lives, but BMS hopes the data will ultimately move the CAR-T earlier in the treatment regimen and set the product apart from lymphoma rivals Yescarta and Kymriah. Breyanzi was introduced at a list price of $410,300. ICER has not yet reviewed the product.

Mizuhos Yang says being able to getthe green light for earlier treatment may be the motivation for such head-to-head trials, and, ultimately, these data would likely bring ICER on board with high-priced cell and gene therapies, too. The earlier you treat the patient, the better off they are. And the better off they are, the lower the net cost will be to the healthcare system, she says.

RELATED: Bristol's new myeloma CAR-T needs a hefty discount to be cost-effective, watchdogs say while endorsing GSK's Blenrep

Gene therapies, on the other hand, have received mixed reviews from ICER on cost-effectiveness. Take Spark Therapeutics Luxturna, which is approved for a rareinherited disease that causes blindness for those aged one year and older. ICER found that the product, whichlaunched at $850,000, would be cost-effective if given to patients at the age of threebut not if patients were dosed at age 15 or later, because by that time they would have already lost a significant amount of eyesight.

Yang attributed the verdict to the cost of care. ICER tries to quantify how much a caregiver costs, so if the child is treated earlier, the idea would be that theyre more independent, they require less care, Yang says. If theyre treated at 15, a lot of that cost has already happened. And this message is consistent with gene therapy in general.

But Pearson said ICERs reviewers placed more weight on the overall value to society of preventing blindness. There really are no significant healthcare costs for being blind, he said. But society saves money to a certain extent, because educational costs are lower, patients have full careers and higher earnings potential if theyre fully sighted.

ICER initially balked at Novartis' $2.1 million gene therapy for spinal muscular atrophy (SMA), Zolgensma, but it didn't approve of Biogen's SMA drug Spinraza either. Spinraza launched at up to $750,000 for the first year and half that every year thereafter, prompting ICER to ultimatelydetermine Zolgensma is the lesser of two evils on cost.

BioMarins Roctavian, a hemophilia gene therapy, has hit some bumps on the road to approval, but ICER has already determined how it would be cost-effective. Using a placeholder price of $2.5 million, ICER estimated if the gene therapy is durable for a dozen years, it would save the healthcare system about $5 million per patient compared to chronically dosed factor VIII. Thats an example of where gene therapy can really bring down [costs], Yang said.

RELATED: ICER's blasted pharma pricing for years, but now drugmakers are 'rolling up their sleeves' to cooperate

After delving into ICERs methodology, Yang said she came away with one bone to pick with the agency. Right now, their judgments are based on clinical valuation, but there should be some sort of consideration for the value of innovation, she said. We learn something along the way, and there has to be long-lasting value in that. I dont think thats being captured.

Pearsons response? We are sensitive to the idea that theres value in having treatments that take different approaches, but we dont consider innovation as something thats separate from the benefits to patients and families, he said. Innovation matters to patients to the degree it helps them live better lives. If its not better for patients, Im not sure we should be paying more for it.

ICER has recently boosted its efforts to maintain an ongoing dialogue with biopharma developers and insurance companies. The agency adopted a formal 12-month checkup policy for each of its published reports, Pearson said. If any new data have been released during that time, reviewers may revisit their original verdict.

It also introduced ICER Analytics, a cloud-based portal that houses all of its economic models. Life sciences companies and insurers can subscribe to ICER Analytics. If [a drugmaker] has new evidence, or if they disagreed with our assumptions the first time around, they can put in their own information and create a different result on fair price, Pearson said. Well put that side by side with our original results so payers can see it. More than 50 companies have signed up for trial subscriptions since ICER Analytics launched in November, he said.

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Cost watchdog ICER will bless some high-priced gene and cell therapies, but only with solid proof of benefit: Analyst - FiercePharma

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Covid-19 mRNA Vaccines Are Not Gene Therapy, As Some Are …

Posted: June 23, 2021 at 1:54 am

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not designed to alter your genes. (Photo by: ... [+] Daniel Garzon/VW Pics/Universal Images Group via Getty Images)

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not genes in a bottle.

Yet, some on social media are now suggesting that such mRNA vaccines are actually gene therapy. For example, Alex Berenson, who has described himself as a former reporter for The New York Times and a current thriller novel author, mentioned the words gene therapy in a recent tweet. And presumably he wasnt referring to Gene Simmons therapy:

As you can see, Berensons tweet prompted a response and a correction from Seth Trueger, MD, MPH, an Assistant Professor of Emergency Medicine at Northwestern University.

Then theres the Its Gene Therapy, Not a Vaccine with Dr. David Martin podcast on the Weston A. Price Foundation website. This is the Foundation that once issued a press release entitled FDA and CDC Bias Against Raw Milk. Yes, the CDC has warned about the possibility of getting dangerous infections from raw milk, otherwise known as unpasteurized milk, as I covered previously from Forbes.

The Foundation website doesnt really describe Martins scientific background, simply saying that he received his Undergrad degree from Goshen College, Masters of Science from Ball State University and Doctorate from the University of Virginia, without specifying the actual subject matters of his degrees. So it isnt clear why they chose him to talk about Covid-19 versus someone else who happened to go to college and some kind of graduate school.

On the podcast, Martin said the following: The problem is that in the case of Moderna and Pfizer, this is not a vaccine. This is gene therapy. Its a chemotherapy agent that is gene therapy. It is not a vaccine. Chemotherapy? How did that word get in there? The National Cancer Institute (NCI) defines chemotherapy as treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Isnt mentioning chemotherapy when you are talking about a vaccine against a virus like mentioning spaghetti and hot dogs when talking about building a bear in a Build A Bear Workshop? Its mixing different things that dont seem to be related.

Martin then tried to explain, What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen. Its a toxin inside of human beings. This is not only not keeping you from getting sick, its making your body produce the thing that makes you sick.

No, no, no. Spike proteins line the surface of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV2), which looks like a spiky massage ball but shouldnt be used as one. The spike protein helps the virus enter your cells. But the spike protein itself is not supposed to make you sick. Claiming that the spike protein alone is responsible for illness would be like claiming that a bleached blonde wig with frosted tips alone can cook like celebrity chef Guy Fieri.

The rest of the podcast didnt exactly scream science. It included the statement by Martin that Covid 19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases. No. Covid-19 is not a giant umbrella. Where the ella, ella, eh, eh did that come from? Covid-19 is not the flu either. Repeat, having Covid-19 is not the same as having influenza.

Oh, and holy conspiracy theories, Batman, Martin asserted that Anthony Fauci, MD, Director of the National Institute of Allergy and Infectious Disease (NIAID) is manipulating this situation to force a vaccine on a population.

How much real evidence did Martin offer to support his claims? Well, the answer rhymes with not much. Its easy to say anything when the interviewer doesnt ask you to provide real concrete evidence behind what you are saying.

The Covid-19 mRNA vaccines are not gene therapy because they are not designed to alter or change your genes in any way. The U.S. National Library of Medicine (NLM) describes gene therapy as a technique that may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. For example, doctors may be able to either inactivate or replace a mutated gene that isnt functioning properly or place a new gene in your body that will do something to combat a disease.

A gene consists of DNA and serves as the basic physical and functional unit of heredity, according to the NLM. Messenger RNA, known as mRNA for short, is different from DNA. RNA stands for ribonucleic acid. DNA stands for deoxyribonucleic acid. DNA serves as the library for instructions to produce different proteins. When a cell wants to produce a protein, it uses the DNA to produce a copy of mRNA. That mRNA then serves as a blueprint for the protein that is built by the ribosomes in your cells. The DNA is in the nucleus of the cell. The ribosomes are not. Thus, the mRNA from a Covid-19 vaccine will not go into the nucleus but instead will simply go to the ribosomes, which in turn will manufacture the spike protein.

This PBS News Hour video shows this process and emphasizes that mRNA should not alter your DNA:

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not gene therapy. Repeat, these vaccines are not gene therapy. In fact, they dont really involve your genes, unless Gene Simmons or Gene Hackman happen to be getting the vaccines. Or you accidentally drops the vaccines on your jeans and then misspell the word jeans.

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COVID-19 ‘Vaccines’ Are Gene Therapy – Atlanta Business …

Posted: June 23, 2021 at 1:54 am

As calls for mandatory COVID-19 vaccination grow around the world, its becoming ever more crucial to understand what these injections actually are. The mRNA vaccines created by Moderna and Pfizer are in fact gene therapies.

Interestingly enough, mainstream media, fact checkers and various industry front groups insist the gene therapy claim is bogus, even though every single detail about the vaccines shouts otherwise. Why are they spreading this disinformation? Why do they not want you to know what these injections actually are?

To start, lets take a look at some basic definitions of words. According to the U.S. Centers for Disease Control and Prevention, a vaccine is:1

Immunity, in turn, is defined as:

Thats the medical definition. The legal definition, in the few cases where it has been detailed, is equally unequivocal:

These definitions, both medical and legal, present problems for mRNA vaccines, since:

We should not be fooled by attempts to condition the public to accept redefined terms. As of February 2019, Merriam-Webster defined5 vaccine as a preparation of killed microorganisms, living attenuated organisms, or living fully virulent organisms that is administered to produce or artificially increase immunity to a particular disease. By February 26, 2021, they had updated the definition of vaccine to:6

A preparation that is administered (as by injection) to stimulate the bodys immune response against a specific infectious disease:

a: an antigenic preparation of a typically inactivated or attenuated pathogenic agent (such as a bacterium or virus) or one of its components or products (such as a protein or toxin)

b: a preparation of genetic material (such as a strand of synthesized messenger RNA) that is used by the cells of the body to produce an antigenic substance (such as a fragment of virus spike protein)

Lets be clear. Merriam-Webster does not dictate medical terminology. It can be used, however, to confuse people. For now, all medical dictionaries still show the traditional definition of vaccine,7 as Merriam-Webster did up until this year. That said, I would not be surprised if changes are made there as well, eventually, if the misrepresentation of COVID-19 mRNA vaccines is allowed to stand.

Theres also the issue of whether a gene therapy can be mandated, and this may hinge on it being accepted as a vaccine. The 1905 Supreme Court ruling in Jacobson v. Massachusetts8 essentially established that collective benefit supersedes individual benefit.

Since mRNA therapies do not render the immunized person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk, and therefore cannot be mandated.

Put another way, the ruling argues (although legal experts diverge on some of the finer details of its interpretation) that its acceptable for some individuals to be harmed by a public health directive as long as it benefits the collective. However, if vaccination is a public health measure meant to protect and benefit the collective, then it would need to accomplish two things:

Were now back to the original problem that mRNA therapies for COVID-19 do not accomplish either of these things. Since these gene therapies do not render the person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk.

On the contrary, the only one benefiting from an mRNA vaccine is the individual receiving the gene therapy, since all they are designed to do is lessen clinical symptoms associated with the S-1 spike protein.

In other words, they wont keep you from getting sick with SARS-CoV-2; they are only supposed to lessen your infection symptoms if or when you do get infected. So, getting vaccinated protects no one but yourself. Since youre the only one who will reap a benefit (less severe COVID-19 symptoms upon infection), the justification to accept the risks of the therapy for the greater good of your community is blatantly irrational.

Since mRNA vaccines do not meet the medical and/or legal definition of a vaccine, referring to them as vaccines, and marketing them as such, is a deceptive practice that violates9 15 U.S. Code Section 41 of the Federal Trade Commission Act,10 the law that governs advertising of medical practices.

The lack of completed human trials also puts these mRNA products at odds with 15 U.S. Code Section 41. Per this law,11,12 it is unlawful to advertise that a product or service can prevent, treat, or cure human disease unless you possess competent and reliable scientific evidence, including, when appropriate, well-controlled human clinical studies, substantiating that the claims are true at the time they are made.

Heres the problem: The primary end point in the COVID-19 vaccine trials is not an actual vaccine trial end point because, again, vaccine trial end points have to do with immunity and transmission reduction. Neither of those was measured.

Whats more, key secondary end points in Modernas trial include prevention of severe COVID-19 disease (defined as need for hospitalization) and prevention of infection by SARS-CoV-2, regardless of symptoms.13,14 However, Moderna did not actually measure rate of infection, stating that it was too impractical to do so.

That means theres no evidence of this gene therapy having an impact on infection, for better or worse. And, if you have no evidence, you cannot fulfill the U.S. Code requirement that states you must have competent and reliable scientific evidence substantiating that the claims are true.

Making matters worse, both Pfizer and Moderna are now eliminating their control groups by offering the real vaccine to any and all placebo recipients who want it.15 The studies are supposed to go on for a full two years, but by eliminating the control group, determining effectiveness and risks is going to be near impossible.

Alright. Lets move on to the definition of gene therapy. As detailed on MedlinePlus.govs What Is Gene Therapy page:16

Gene therapy is an experimental technique that uses genes to treat or prevent disease Researchers are testing several approaches to gene therapy, including: Introducing a new gene into the body to help fight a disease

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

Here, its worth noting that there are many different treatments that have been shown to be very effective against COVID-19, so it certainly does not qualify as a disease that has no cure. It makes sense that gene therapy should be restricted to incurable diseases, as this is the only time that taking drastic risks might be warranted. That said, heres how the U.S. Food and Drug Administration defines gene therapy:17

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a persons genes to treat or cure disease. Gene therapies can work by several mechanisms:

Replacing a disease-causing gene with a healthy copy of the gene

Inactivating a disease-causing gene that is not functioning properly

Introducing a new or modified gene into the body to help treat a disease

November 17, 2020, the American Society of Gene + Cell Therapy (ASGCT) announced COVID-19 Vaccine Candidates Show Gene Therapy Is a Viable Strategy, noting that:18

Two COVID-19 vaccine trials, both of which use messenger RNA (or mRNA) technology to teach the body to fight the virus, have reported efficacy over 90 percent.

These findings, announced by Moderna on Nov. 16 and by Pfizer and its partner BioNTech on Nov. 9 demonstrate that gene therapy is a viable strategy for developing vaccines to combat COVID-19.

Both vaccine candidates use mRNA to program a persons cells to produce many copies of a fragment of the virus. The fragment then stimulates the immune system to attack if the real virus tries to invade the body.

As explained in the ASGCTs video above, mRNA are molecules that contain genetic instructions for making various proteins. mRNA vaccines deliver a synthetic version of mRNA into your cells that carry the instruction to produce the SARS-CoV-2 spike protein, the antigen, that then activates your immune system to produce antibodies. Then theres Modernas trial website,19 where they describe their technology thus:

Typical vaccines for viruses are made from a weakened or inactive virus, but mRNA-1273 is not made from the SARS-CoV-2 virus. It is made from messenger ribonucleic acid (mRNA), a genetic code that tells cells how to make protein, which help the bodys immune system make antibodies to fight the virus.

November 18, 2020, Wired magazine made a big deal about COVID-19 vaccines being genetic vaccines, noting:20

The active ingredient inside their shot is mRNA mobile strings of genetic code that contain the blueprints for proteins. Cells use mRNA to get those specs out of hard DNA storage and into their protein-making factories. The mRNA inside Pfizer and BioNTechs vaccine directs any cells it reaches to run a coronavirus spike-building program.

Importantly, as reported by David Martin, Ph.D.,21,22 Moderna describes its product not as a vaccine, but as gene therapy technology in SEC filings. This is because neither Moderna nor Pfizer make any claims about their products creating immunity or preventing transmission. Additionally, Modernas SEC filings specifically state that Currently, mRNA is considered a gene therapy product by the FDA, as well.23

In a February 2021 article, MIT Technology Review reviewed the history of mRNA technology in general, and Modernas in particular, stating:24

Vaccines were not their focus. At the companys founding in 2010, its leaders imagined they might be able to use RNA to replace the injected proteins that make up most of the biotech pharmacopoeia, essentially producing drugs inside the patients own cells from an RNA blueprint. We were asking, could we turn a human into a bioreactor? says Noubar Afeyan, the companys cofounder

Bloomberg, in August 2020, reported25 that the Moderna vaccine would seek to transform your body into a vaccine-making machine. The New York Times was more to the point. In May 2020, they reported26 that Researchers at two Harvard-affiliated hospitals are adapting a proven form of gene therapy to develop a coronavirus vaccine. Read it again A proven form of gene therapy.

So, to summarize: The definition of genetic is something relating to genes, and the definition of therapy is the medical treatment of a disease. The definition of gene therapy is the process of modifying or manipulating the expression of a gene, or altering the biological properties of living cells.

mRNA are snippets of genetic code that instructs cells to produce proteins. mRNA COVID-19 therapies deliver genetic instructions into your cells, thereby triggering your body to produce a fragment of the virus (the spike protein). So, mRNA vaccines ARE gene therapy. Theres simply no way around this. They fulfill all the definitions of gene therapy and none of the definitions for a vaccine.

Theres yet one more potential problem with the COVID-19 vaccine narrative as a whole, which Martin unpacked in a January 25, 2021, interview on the Wise Traditions podcast (above).27 In it, he explains:

COVID-19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases.

The problem that we have is that in February [2020], the World Health Organization was clear in stating that there should not be a conflation between [SARS-CoV-2 and COVID-19]. One is a virus, in their definition, and one is a set of clinical symptoms. The illusion in February was that SARS-CoV-2 caused COVID-19.

The problem with that definition, and with the expectation, is that the majority of people who test positive using the RT-PCR method for testing, for fragments of what is associated with SARS-CoV-2, are not ill at all. The illusion that the virus causes a disease fell apart. Thats the reason why they invented the term asymptomatic carrier.

In short, SARS-CoV-2 has yet to be definitively proven to be the actual cause of COVID-19. So, a gene therapy that instructs your body to produce a SARS-CoV-2 antigen the viral spike protein cannot even be touted as a preventative against COVID-19, as the two have not been shown to be causally linked.

They have been willfully lying since the inception of this, Martin says in the interview. There is not a causal link between these things It has never even been close to established.

We have a situation where the illusion of the problem is that people say, I dont want to get COVID-19. What they mean is they dont want to get infected with a virus. The problem is those two things are not related to each other. A viral infection hasnt been documented in the majority of what is called cases.

There is no basis for that conflation other than the manipulation of the public. Thats the first half of the problem. The second half of the problem is that what is being touted as a vaccination is not a vaccine. This is gene therapy

What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen A vaccine is supposed to trigger immunity. Its not supposed to trigger you to make a toxin

Its not somewhat different. Its not the same at all Its not a prohibiting infection. Its not a prohibiting transmission device. Its a means by which your body is conscripted to make the toxin that then, allegedly, your body somehow gets used to dealing with, but unlike a vaccine which is to trigger the immune response this is to trigger the creation of the toxin.

As for why drug companies are misrepresenting this technology, Martin suspects its done exclusively so that they can get themselves under the umbrella of public health laws that exploit vaccination.

Experimental gene therapies do not have financial liability shielding from the government, but pandemic vaccines do, even in the experimental stage, as long as the emergency use authorization is in effect. This is indeed a major incentive to make sure this technology is perceived as a vaccine and nothing else.

So, by maintaining the illusion that COVID-19 is a state of emergency, when in reality it is not, government leaders are providing cover for these gene therapy companies so that they are insulated from any liability.

Ive written many articles detailing the potential and expected side effects of these gene therapy vaccines. If all of this is new to you, consider reviewing How COVID-19 Vaccine Can Destroy Your Immune System, Seniors Dying After COVID Vaccine Labeled as Natural Causes and Side Effects and Data Gaps Raise Questions on COVID Vaccine.

The take-home message here is that these injections are not vaccines. They do not prevent infection, they do not render you immune and they do not prevent transmission of the disease. Instead, they alter your genetic coding, turning you into a viral protein factory that has no off-switch. Whats happening here is a medical fraud of unprecedented magnitude, and it really needs to be stopped before its too late for a majority of people.

If you already got the vaccine and now regret it, you may be able to address your symptoms using the same strategies youd use to treat actual SARS-CoV-2 infection. I review these strategies at the end of Why COVID Vaccine Testing Is a Farce.

Last but not least, if you got the vaccine and are having side effects, please help raise public awareness by reporting it. The Childrens Health Defense is calling on all who have suffered a side effect from a COVID-19 vaccine to do these three things:28

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A year after getting UniQure’s gene therapy, hemophilia patients are still doing better – BioPharma Dive

Posted: June 23, 2021 at 1:54 am

One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have.

In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less or ideally, not at all on so-called replacement factor treatments.

UniQure said Tuesday that, a year after receiving the company's therapy, patients enrolled in the company's HOPE-B clinical trial were experiencing 80% fewer bleeding episodes that require treatment.

At the trial's start, these patients were having an average of three to four bleeding episodes annually. Now, according to UniQure, they're having less than one. The use of replacement factor therapy declined 96% during the 52-week period, as all but two of the 54 participants successfully discontinued their preventive infusions.

What's more, patients had almost 42% the amount of clotting protein typically observed in people who don't have hemophilia B. That's a slight improvement from six-month data presented in December, which showed protein levels at 39% of normal.

Yet, in spite of the positive data, UniQure shares dipped more than 5% in early afternoon trading Tuesday. CSL Behring, which spent $450 million to acquire the rights to UniQure's therapy, also saw its stock trade down.

Some of that stock market reaction may be related to an update Uniqure disclosed alongside the fresh data about the approval process for its therapy. According to UniQure, the Food and Drug Administration primarily wants to see evidence the therapy has a lasting effect, so the agency's focus will be on 52-week data. However, that time frame must start from when patients' protein levels stabilize, rather than from when they first received UniQure's therapy.

The company said all participants in its study achieved steady-state protein levels by week 26. As a result, UniQure is now measuring the study's success by annualized bleeding rates at week 78, with the expectation that patients to complete 78-week follow-up visits by the end of September. That should allow UniQure and CSL to file for approval in first quarter of 2022, months later than when the companies had estimated last year.

If approved, UniQure's gene therapy would become the first cleared to treat any kind of hemophilia. Another, for hemophilia A, was up for approval last year, but the FDA rejected it in in a major surprise. In issuing its rejection, the FDA asked for two years of follow-up data from the therapy's developer, BioMarin Pharmaceutical, a task that can't be completed until November 2021.

Estimates hold there are as many as 33,000 people living with hemophilia in the U.S., with the B form being about four times less common.

Though the FDA has approved around half a dozen drugs to treat hemophilia B, patients taking them can still experience bleeds. Additionally, they can develop a type of antibody called an inhibitor, which blocks the effects of replacement factor.

That's made gene therapy, with its potential as a one-time fix for the disease, an attractive option for some patients and physicians.

Still, concerns remain. UniQure's study was stopped late last year, for example, after researchers saw an unexpected case of liver carcinoma in one participant, reigniting long-held fears about gene therapy's potential to drive cancer.

UniQure investigated the case and recently concluded, along with an independent laboratory and outside experts, that its therapy was "highly unlikely" to have caused the cancer.

There are also worries that gene therapy won't work for everyone. Some people have pre-existing "neutralizing antibodies" that attack the viruses used to deliver functioning genetic material.

UniQure has found its therapy appears, for the most part, to bypass this issue of neutralizing antibodies. The company said Tuesday it hasn't seen any "significant correlation" in how patients who have these antibodies at least to a certain range are responding to its therapy.

UniQure said that range is high enough that it should encompass more than 93% of the general population. The company noted in December how one of the study participants who didn't respond its therapy had neutralizing antibody levels that were five times greater than anyone else in the group.

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A year after getting UniQure's gene therapy, hemophilia patients are still doing better - BioPharma Dive

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BioMarin’s gene therapy lead jumps ship to rival biotech Vedere Bio II – FierceBiotech

Posted: June 23, 2021 at 1:54 am

Vedere Bio II is fresh off a $77 million raise, and the follow-up act to the original Vedere Bio now has a big-name chief scientific officer in Gabor Veres, Ph.D.

The Cambridge, Massachusetts-based biotech is at work on next-gen eye disease gene therapies, aiming to restore sight, or help those keep it, with patients suffering from vision loss due to photoreceptor death.

To help in that mission, the company has poached Veres from rival biotech BioMarin, where he was vice president and head of gene therapy research focusing on AAV platform discovery and seeking out new indications. He also served stints at bluebird bio and Applied Genetic Technologies.

At VB II, he will provide strategic leadership to Vedere's research organization and will be responsible for driving and expanding the company's pipeline of ocular gene therapies, the biotech said in a statement.

The biotech has a history that while short, is very dense: Cyrus Mozayeni, M.D.s first go at vision-restoring gene therapies got snapped up as it was just getting started. Lots of suitors came knocking even when that company, Vedere Bio, was working under the radar, and Novartis eventually ponied up $150 million upfront to buy it last fall, with a promise of $130 million more.

Buoyed by that success but clearly still hungry for more, Mozayeni, its CEO and Atlas Venture entrepreneur in residence and his team last month nabbed $77 million to bankroll their second chapter: Vedere Bio II.

Although its technically a new entity, its working on gene therapies for vision loss with the same founders, management team and employees as its predecessor. Vederes focus is vision loss caused by the death of photoreceptors or light-sensing cells in the retina rather than blindness stemming from damage to the brain or optic nerve. With Veres, that team just got a little more diversified.

RELATED: Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price

"Gabor has over 15 years of experience leading preclinical research and development for international pharmaceutical and biotechnology companies and holds 20 years of experience in the development of cell and gene therapy products, said Mozayeni.

His extensive track record designing and executing gene therapy research programs for a range of diseases, including conditions of the eye, will be an incredible asset to our team as we continue our work to restore vision to patients with both genetic and non-genetic causes of vision loss.

Gene therapies have long held much promise, but safety issues and manufacturing complexities have always dampened companies capacity to deliver. Gene therapies for eye disorders have been among the few to see R&D success translate into the market, most recently with Roche/Sparks Luxturna, though sales forecasts are fairly modest, and it requires specialist centers for administration.

Vedere's approach to vision restoration holds great potential to treat vision loss regardless of underlying genetic cause or disease stage, and I'm looking forward to helping build out and progress the company's unique platform and product pipeline, added Veres.

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BioMarin's gene therapy lead jumps ship to rival biotech Vedere Bio II - FierceBiotech

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Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -…

Posted: June 23, 2021 at 1:54 am

When you have retinitis pigmentosa, the world slowly goes dark. Most patients with RP lose most of their sight by young adulthood and are often legally blind by age 40. The genetic disorder affects around one in 4,000 people, causing the retinas photoreceptive cells to degrade over time. But a new gene therapy is giving hope and producing life-changing resultsand it could lead to treatments for far more common retinal diseases.

Bedford-based Nanoscope Therapeutics is trying to turn the light back on for RP patientsby developing gene therapies using light-sensitive molecules that could re-sensitize the retina to detect low light levels. That could restore vision in millions of visually impaired people who suffer from RP and other retinal degenerative diseases, like Stargardt disease and dry age-related macular degeneration (AMD). The startups gene therapies, called optogenetics, aim to bring sight to the blind. Nanoscope is one of a number of teams and companies exploring optogenetics as a treatment for RP.

There is no treatment for the diseases that were working on, Nanoscopesco-founder, president, and chief scientific officer, SamarendraMohanty, told Dallas Innovates. So theres a real unmet need that we are trying to fill.

Samarendra Mohanty [Photo: Nanoscope Therapeutics]

Nanoscope, a clinical-stage biotech company, announced earlier this month that its Phase 1/2a clinical study using Multi-Characteristic Opsin (MCO) on RP patients had restored clinically meaningful vision. Significant dose-dependent improvement of visual acuity was demonstrated at 16 weeks, and continued through one year in patients suffering from severe RP, the company said.

The study included 11 patients with advanced RP who had either no light perception or just perception of light in the study eye and no better ability than counting fingers in their other eye. The studys initial positive results were reported at the American Academy of Ophthalmologys 2020 annual meeting last November.

After MCO treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities, saidthe principal investigator, Dr. Santosh Mahapatra, an ophthalmologist and eye surgeon, in a statement. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.

Another benefit of the treatment: Vision restoration was produced without the need of stimulating retinal implants or explants (goggles).

Nanoscope co-founder and CEO Sulagna Bhattacharya said the trial studys impact on patients lives has been powerful.

Their quality of life improves significantly, she told us. This is a relief to patients family members, healthcare systems, and society as a whole.

Sulagna Bhattacharya [Photo: Nanoscope Therapeutics]

We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the U.S. this summer to further validate our gene therapys ability to improve clinically meaningful vision in RP patients, Bhattacharya said in the statement. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide.

The 2b trial will involve eight to 10 centers in the U.S., from Beverly Hills to Florida, to other locations on the east coast.

[Image: Ivan-balvan/istockphoto]

Nanoscopes RP gene therapy has received orphan drug designation from the FDA. It uses a proprietary AAV2 vector to deliver MCO genes into the retina. This mutation-independent gene therapy is delivered via a single injection through the eye administered in a doctors office.

All 11 subjects participating in the trial had objective and subjective improvement in functional vision, Nanoscope reports. Shape discrimination accuracy improved more than 90% in all the subjects compared to baseline. Further, the performance in two different mobility tests improved by 50%, including the reduction in time to touch a lighted panel. Nanoscope says the test outcomes were highly correlated with improved patient-reported outcomes.

Nanoscopes co-founder, Mohanty, is the inventor of the technology used in the trial.

Optogenetics is a powerful research tool, he said in a statement, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.

Nanoscope Therapeutics got a $2 million grant from the National Eye Institute in June 2020 and closed anoversubscribed Series A funding roundin July 2020 to help fund its clinical trial.The startup, a TechFW client, is a spinout from Nanoscope Technologies, serving as a commercialization partner for the R&D enginesMCO vision restoration work. It has 12 employees and a group of consultants and advisors.

Nanoscope Technologies, meanwhile, has received around$10 million in grants from theNational Institutes of Health to help fund its R&D,Bhattacharya said.

The CEO added that the RP study could lead to pivotal results.

Were very excited about our Phase 1/2a results, Bhattacharya said. This trial has the potential to become pivotal, which will allow our product to be available in the clinics to treat millions of blind individuals for whom there is no treatment so far.

Beyond the RP trials, Mohanty says his company placing a big focus on dry age-related macular degeneration (AMD),

Thats a big indication that we are targeting, Mohanty said of AMD, since unlike RP, AMD is super prevalent. According to the National Eye Institute, 11 million Americans have AMD.

Mohanty said that AMD is very rapidly progressing as we are aging and worldwide there are major concerns.

Nanoscope plans to initiatemultiple trials to treat both dry AMD and Stargardt disease, another inherited retina disorder.

Nanoscope Technologies was founded in 2009 by Mohanty. Bhattacharya joined the company as co-founder in 2013. Mohanty and CFO Anthony Togba told Dallas Innovates they are open-minded to the option of going public with their company, and doing due diligence to prepare for that potentiality.

CFO Anthony Togba [Photo: Nanoscope Therapeutics]

Togba says the company is performing readiness activities, undertaking internal processes, and putting in place the structures to be ready to go public.

The timeline? By late Q4 or before, well have a better idea about where were headed, Togba said. The upcoming clinical trial is the preoccupation now to make sure that we have a flawless execution to obtain those results that we expect.

Quincy Preston contributed to this report.

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The investment was led by Advantech Capital, a PE fund based in China that focuses on TMT, pharmaceuticals, and healthcare. This combined with the support from the Cancer Prevention and Research Institute of Texas (CPRIT), which granted OncoNano $9.97 million last year, will support Phase 3 clinical trials for the biotech's technology that can diagnose and treat cancer with high specificity.

Taysha Gene Therapies, which rocketed from a UTSW spinout to a $157 million IPO in under six months last year, has gone global with rights to TSHA-120, apromising AAV9 clinical-stage genetherapy. It's a historic announcement. There are no current treatments for giant axonal neuropathy, or GAN,a severe, progressive disease that affects the central and peripheral nervous systems.TSHA-120 is the first-ever successful in-human intrathecal (spinal) gene transfer.

In this weekly roundup of executive moves in North Texas, you'll also find news from Liberty Capital Bank, Krista Software, Tuesday Morning, Trive Capital, Cantey Hanger, UNT, JUNO, NuZee, Jaunt Air Mobility, Korbyt, and ID90 Travel.

The Series C funding brings Allied BioSciences total to more than $80 million, the team told us. It will be used to grow the biotech's flagship product, SurfaceWise2, which is an active surface coating that can continuously destroy 99.9 percent of viruses on surfaces. Last year, SurfaceWise2 was the first and only surface coating that the EPA approved for continuous protection against COVID-19 with a single application.

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Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -...

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