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Category Archives: Transhuman News

Psoriasis Treatment: The Role Of Detoxification, Nutrition And Oxygenation – TheHealthSite

Posted: October 30, 2021 at 2:34 pm

For treatment of psoriasis, it is always necessary to analyze whether the basic needs of the body are met, which include detoxification, nutrition, and oxygenation. Read on to know more

Written by Longjam Dineshwori | Updated : October 30, 2021 3:13 PM IST

Psoriasis is the most common skin disease, affecting more than 2.6 crore people in India, as per industry sources. Due to its physical manifestations, the condition often leads to social discrimination. The typical psoriasis scales on the skin, often itchy, can make everyday life very difficult for patients and lead to social isolation. Psoriasis is an autoimmune condition which is considered incurable. The main cause is inflammation, a natural reaction of the body to an external attacks such as bacteria, viruses, fungi. In psoriasis, the levels of auxiliary T lymphocytes increase significantly, contributing to the secretion of inflammatory cytokines and the increased proliferation of skin cells.

While typical treatment of psoriasis is limited to external preparations, immunosuppressive treatment is used in more serious cases. In complementary medicine, it is assumed that if the immune system is ill, it is worth considering the cause of this state of affairs and addressing it. For treatment of psoriasis, it is always necessary to analyze whether the basic needs of the body are met, which include detoxification, nutrition, and oxygenation.

On the occasion of World Psoriasis Day Friday (October 29th), Dr. Mili Shah, President, Ozone Forum of India & Dr. Renu Mathani, DM, FNMN, (USA) Founder of Autoimmunity Treatment Centre at Pune, highlighted the impact of oxygenation & vitamin D on this condition.

Dr. Mahtani said, "Megadose vitamin D therapy is the need of the hour which works as a defense against autoimmunity."

Vitamin D does more than regulation of calcium and bone health. Deficiency of vitamin D can drastically impact a person's physical and mental wellbeing. Vitamin D deficiency in the body has been linked to various health problems, including cognitive decline, depression, osteoporosis, cardiovascular disease, hypertension, diabetes, autoimmune diseases, and cancer.

Vitamin D in its active form is actually a master hormone that controls at least 200 genes in our cells. Following the discovery of vitamin D receptors throughout the body, its role in the prevention and treatment of chronic diseases is gaining momentum. It is being positioned as the critical aspect in immune system functioning and gut homeostasis too. Vitamin D empowers the immune system against viruses, bacteria, and other microorganisms. Vitamin D is not an optional supplement. It is a non-negotiable cellular necessity, Dr. Mathani noted.

According to Dr. Shah, Ozone therapy is a wonderful therapy for detoxification which enhances and modulates your immunity and circulation. Treatment results show that ozone therapy is effective in treating psoriasis. Ozone therapy works as a regulation of the overactive response of the immune system. It reduces the level of oxidative stress. Exerts an anti-inflammatory effect by acting on the NF-KB nuclear receptor.

The body is unable to function efficiently if the amount of toxins it struggles with exceeds its ability to be removed. This affects the condition of the immune system. The body overloaded with toxins will try to remove them through the skin, which may also result in psoriatic lesions.

Ozone rectal Insufflation works wonderfully on gut dysbiosis, she added.

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Psoriasis Treatment: The Role Of Detoxification, Nutrition And Oxygenation - TheHealthSite

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How to Manage Psoriatic Arthritis on Feet, Toes, and Ankles – Self

Posted: at 2:34 pm

Enthesitis can be asymptomatic or it can cause swelling and pain around the affected area.8 Your doctor may want to do an MRI to look for tendon or ligament enlargement and thickening (which are signs of enthesitis) when assessing whether you might have psoriatic arthritis.

Plantar fasciitis

When psoriatic arthritis involves the feet, the pain can seem like plantar fasciitis, which feels like a stabbing pain10 along the bottom of your foot near the heel. It can be easy to assume something like this is the cause of your foot pain, but if you have ongoing symptoms, exhibit other signs of psoriatic arthritis, and have a family history of psoriatic arthritis, then you may want to talk to your doctor about psoriatic arthritis screening.

Psoriatic arthritis ankle

Anyone with psoriatic arthritis can experience pain, tenderness, stiffness, and swelling in their ankles. Ankle pain is often related to inflammation of the enthesis11 (that area we mentioned above where the tendons and ligaments meet the bone and help us move our joints). Generally, psoriatic arthritis ankle stiffness and swelling can be worse in the mornings and improve when you are active. Some people may have a hard time walking at all during a particularly painful flare-up. If you can, doing some range of motion exercises first to warm up can help alleviate your pain and stiffness.

Psoriatic arthritis toes

About 50% of people with psoriatic arthritis experience dactylitis, a condition thats also referred to as sausage digits.12 As the nickname implies, your toes can become very swollen and painful due to inflammation in the toe joints and connective tissue around your toes and ligaments. Dactylitis can be present in all toes, and the swelling can be acute or chronic.13 It is important to treat and manage dactylitis because your pain and swelling can become worse. Having swollen toes doesnt automatically mean you have psoriatic arthritisdactylitis is also associated with a few other conditions, like gout, tuberculosis, and sarcoidosis (an inflammatory condition that is associated with granulomata, or inflammation that is caused by growths in your organs). However, dactylitis is a strong indicator of psoriatic arthritis, so if you experience toe swelling, be sure to bring it up to your doctor who can take this into consideration when diagnosing you.

Psoriatic arthritis nails

Psoriatic arthritis nail symptoms are common and roughly 80% of people with psoriatic arthritis14 have nail lesions, according to a 2017 paper published in Reumatologia. These changes arent the direct result of psoriatic arthritis but instead happen when you develop nail psoriasis. As a result, you may experience issues like pitting, deformity, nail plate thickening, and separation of the nail from the nail bed15 (just to name a few). If you have foot or ankle pain, toe swelling, and changes in your toenails, then you might want to talk to your doctor about whether you could have nail psoriasis and psoriatic arthritis. If you are already diagnosed with psoriatic arthritis and notice any nail changes as we described, then its a good idea to talk to your doctor about whether your condition is progressing.

Morning stiffness

There are various reasons that your feet may feel stiff in the morning, including going on a new hike. However, morning stiffness is a common symptom among people with psoriatic arthritis, especially during a flare. In the morning your feet may feel extra stiff and sore, which occurs because your joints can stiffen up from lack of movement16 when you sleep. Generally, light movement can help mobility, so going for a short walk if you are able to might alleviate some of your discomfort.

Gout

Gout is a painful type of inflammatory arthritis that initially affects just one joint17 (usually the big toe), but later it may affect other joints, even at the same time, such asthe ankles, knees, elbows, wrists, and fingers. However, it can also affect the ankles, knees, elbows, wrists, and fingers. Similar to psoriatic arthritis, gout symptoms include pain and swelling and may flare and subside. If your toes become very painful and swollen, then you may wonder whether its related to gout or psoriatic arthritis. There is no single test to confirm psoriatic arthritis or gout, but X-rays, lab tests, physical exams, and patient histories can help physicians differentiate between the two. And its important to get an accurate diagnosis because gout and psoriatic arthritis are two very different diseases with different treatments.

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How to Manage Psoriatic Arthritis on Feet, Toes, and Ankles - Self

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Gene Therapy Shows Early Promise as Angelman Syndrome Treatment | Newsroom – UNC Health and UNC School of Medicine

Posted: October 26, 2021 at 5:30 pm

Led by Ben Philpot, PhD, and Matt Judson, PhD, the new therapy was generally well-tolerated and prevented key signs of the condition in animal models.

CHAPEL HILL, NC Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities.

Angelman syndrome affects roughly one in every 20,000 children, and in the US alone it is thought that there are more than 15,000 people with the condition. There is no specific treatment, but scientists led by Ben Philpot, PhD, Kenan Distinguished Professor of Cell Biology and Physiology at UNC School of Medicine and Associate Director of the UNC Neuroscience Center, previously suggested that the best way to treat the disorder would be to restore function of the UBE3A gene in neurons, which has been lost in the brains of people with Angelman syndrome.

The genetics of Angelman syndrome are more complicated than classic single-gene disorders such as cystic fibrosis and sickle-cell anemia. Humans inherit one maternal and one paternal copy of most genes. Angelman syndrome arises in children whose maternal UBE3A copy has somehow been mutated or deleted. For reasons that arent fully clear, mature neurons normally express only the maternal copy of UBE3A; the paternal copy is effectively silenced. Thus, when the maternal copy is lost, the genes function is absent in neurons. Because UBE3A encodes a protein that helps regulate the levels of other important proteins, its absence severely disrupts brain development.

Compounding the complexity, neurons express two different variants or isoforms of UBE3A that vary slightly in length a short form and a long form in a ratio of about three short forms for every one long form.

Philpots team was able to craft a version of UBE3A that, when expressed by neurons, yields short and long forms of the UBE3A protein at a near-normal ratio. The scientists inserted their therapeutic UBE3A gene into a virus-derived carrier, or vector, engineered for reliable delivery to neurons. They injected a solution of this vector into hollow spaces, called ventricles, in the brains of newborn Angelman syndrome model mice, which lack the maternal copy of the mouse Ube3a gene. Like humans with Angelman syndrome, these mice fail to express UBE3A protein in their neurons and develop motor deficits, seizures, and other neurological symptoms in the first months of life.

Philpot and colleagues verified that vector-borne UBE3A became active in neurons throughout the Angelman model mouse brain just days after injection, at a level similar to that of the normal gene. This treatment restored motor skill-learning and the essential mouse behaviors of digging, burrowing, and nest-building. Untreated mice developed the usual Angelman-like impairments. The treated mice also did not become as susceptible as their untreated counterparts to experimentally induced epileptic seizures, and importantly, did not suffer any obvious negative side effects.

This was a proof-of-concept study, but if these early results were translated to the clinic, they would represent big improvements in the quality of life for individuals with Angelman syndrome, said study lead author Matt Judson, PhD, a research associate in the Philpot Lab, who performed most of the experiments.

The researchers plan to further develop their strategy, first with more tests in mice and monkeys to optimize dose and delivery methods, and ultimately, pending promising safety results, human clinical trials. If such a therapy were available, the researchers expect it might be able to deliver benefits to individuals of any age, but perhaps with varying benefits.

The range from birth to four years is probably ideal, but we think that whenever we can reinstate this genes function in the brain, were likely to see some improvements, Philpot said.

Along with Judson and Philpot, who was recently ranked as the worlds leading Angelman syndrome researcher, the JCI Insight paper was co-authored by Charles Shyng, Jeremy Simon, Courtney Davis, Mattijs Punt, Mirabel Salmon, Noah Miller, Kimberly Ritola, Ype Elgersma, David Amaral, and Steven Gray.

The research was supported by the Angelman Syndrome Foundation, and the National Institutes of Health (R01HD093771, R01MH120229, R01NS114086).

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Gene Therapy Shows Early Promise as Angelman Syndrome Treatment | Newsroom - UNC Health and UNC School of Medicine

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Report by Mott Center researchers named NIEHS Extramural Paper of the Month – The South End

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The National Institutes of Environmental Health Sciences has selected a study published by Wayne State University School of Medicine researchers as an Extramural Paper of the Month.

The paper, Paternal preconception phthalate exposure alters sperm methylome and embryonic programming, published in in the October issue of the journal Environment International by J. Richard Pilsner, Ph.D., professor and Robert J. Sokol, M.D., Endowed Chair of Molecular Obstetrics and Gynecology, and director of Molecular Genetics and Infertility for the C.S. Mott Center for Human Growth and Development; and Stephen Krawetz, Ph.D., the Charlotte B. Failing Professor of Fetal Therapy and Diagnosis, and associate director of the Mott Center, was selected by the NIEHS as a paper of the month for September.

The Extramural Papers of the Month are selected based on their important findings and potential for public health impact.

The researchers reported that male mice exposed to phthalates before conception had DNA methylation changes in sperm, which can be transferred to the next generation as altered gene expression in embryos. DNA methylation occurs when a chemical compound, called a methyl group, attaches to DNA, affecting whether a gene is turned on or off.

They exposed male mice to either a low or high level of di(2-ethylhexyl) phthalate for two sperm production cycles, or 67 days. Following exposure, they mated the mice with unexposed females. They then assessed genome-wide methylation in sperm, embryos and extra-embryonic tissues, which support the developing embryo.

Compared with unexposed controls, paternal preconception DEHP exposure altered methylation in 704 sperm gene regions, 1,716 embryo gene regions, and 3,181 extra-embryonic gene regions. Of these, 29 gene regions overlapped between sperm and embryonic tissues, suggesting methylation changes related to paternal DEHP exposure may be transmitted to the next generation. The researchers also identified changes in gene expression in embryos in both exposure groups compared with controls. Many of the altered genes were related to pathways important in development.

The researchers said their results indicate that preconception is a sensitive window in which phthalate exposure alters sperm methylation and embryo gene expression in ways that may influence offspring health and development.

Others involved in the research and subsequent publication include Oladele Oluwayiose, a doctoral student at the University of Massachusetts Amherst; Chelsea Marcho, Department of Environmental Health Sciences, University of Massachusetts Amherst; Haotian Wu, Department of Environmental Health Sciences, Mailman School of Public Health, Columbia University; Alexander Suvorov, Department of Environmental Health Sciences, University of Massachusetts Amherst; Emily Houle, Department of Environmental Health Sciences, University of Massachusetts Amherst; and Jesse Mager, Department of Veterinary and Animal Sciences, University of Massachusetts Amherst.

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Report by Mott Center researchers named NIEHS Extramural Paper of the Month - The South End

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Clinical Data from Editas Medicines Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10 to be Presented at the European Society of Gene…

Posted: at 5:30 pm

CAMBRIDGE,Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that an abstract featuring initial clinical data from the BRILLIANCE clinical trial of EDIT-101 has been selected for an oral presentation at the 2021 European Society of Gene and Cell Therapy (ESGCT) Annual Congress being held virtually October 19 22, 2021. EDIT-101 is under development for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder.

We are pleased to be working at the forefront of this research with Editas Medicine, evaluating genome editing for the treatment of CEP290-associated retinal disease in the BRILLIANCE trial, said Eric A. Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and the senior BRILLIANCE principal investigator. I am highly encouraged by the early efficacy signals in the mid-dose cohort, which suggest positive biological activity and potential early clinical benefits. I am also very pleased that the initial data from the BRILLIANCE trial demonstrate a favorable safety profile. I believe that the trial data support continued EDIT-101 development as well as the evaluation of gene editing approaches for other inherited retinal disorders.

Details of the Editas Medicine presentation can be accessed on the ESGCT website.

Oral Presentation:Title: BRILLIANCE: A Phase 1/2 single ascending dose study of EDIT-101, an in vivo CRISPR gene editing therapy, in CEP290-related retinal degenerationSession Title: Parallel 4b: Gene editing IIDate and Time: Thursday, October 21, 2021, 10:15 10:30 a.m. CETPresenter: Dr. Eric A. Pierce, M.D., Ph.D., Director of the Inherited Retinal Disorders Service, Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and a BRILLIANCE principal investigator.

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About EDIT-101 EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About BRILLIANCEThe BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. Clinical trial sites are enrolling up to five cohorts testing up to three dose levels in this open label, multi-center study. Both adult and pediatric patients (3 17 years old) with a range of baseline visual acuity assessments are eligible for enrollment. Patients receive a single administration of EDIT-101 via subretinal injection in one eye. Patients are monitored every three months for a year after dosing and less frequently for an additional two years thereafter. Additional details are available on http://www.clinicaltrials.gov (NCT#03872479).

About Leber Congenital AmaurosisLeber Congenital Amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of approximately three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10 or a CEP290-related retinal degenerative disorder, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20-30 percent of all LCA patients.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the completion of clinical trials, including the BRILLIANCE trial, and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Companys subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

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Clinical Data from Editas Medicines Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10 to be Presented at the European Society of Gene...

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Delic Partners With GT Research for Genetic Analysis of Cannabis, Psychedelic Mushrooms – GenomeWeb

Posted: at 5:29 pm

NEW YORK Delic Labs has entered into a comarketing partnership with GT Research to provide detailed genomic analyses related to commercially interesting traits found in cannabis and psychedelic mushrooms to Canadian producers, its parent company Delic said on Tuesday.

Services provided under the agreement include sample preparation, DNA extraction, whole-genome sequencing, and computational analyses.

Delic Labs, a subsidiary of Delic, focuses on identifying scalable legal psychedelic medicine opportunities. As one of Canada's few licensed psilocybin labs, it applies chemical analytics, metabolomic identification, and process optimization to the psychedelics industry.

GTR performs gene profiling and trait optimization services related to the production of cannabis and psychedelics.

"As the cannabis and psychedelic sectors grow, interest in genomic analysis of the underlying organisms is increasing. GTR is excited to offer its cutting-edge suite of capabilities in partnership with Delic, a pioneer in this space," Sam Proctor, cofounder and CEO of GTR, said in a statement.

"Delic is committed to researching and identifying the safest, highest quality psychedelic compounds for commercial use," said Matt Stang, cofounder and CEO of Delic.

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Delic Partners With GT Research for Genetic Analysis of Cannabis, Psychedelic Mushrooms - GenomeWeb

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Alcyone Therapeutics Strengthens Executive Team with New Senior Leadership Appointments – WABI

Posted: at 5:29 pm

Published: Oct. 25, 2021 at 7:00 AM EDT

LOWELL, Mass., Oct. 25, 2021 /PRNewswire/ -- Alcyone Therapeutics ("Alcyone"), a biotechnology company developing precision therapies for neurological disorders with high unmet medical needs, today announced four key appointments to its executive leadership team, bringing significant expertise in neuroscience and genetic medicines development. Alcyone Therapeutics appointed Ottavio Vitolo, M.D., M.M.Sc., Chief Medical Officer and Global Head of R&D; Ravi Mehrotra, Ph.D., Chief Financial Officer and Head of Strategy; Susan D'Costa, Ph.D., Executive Vice President & Global Head of Technology; and Rachel Salzman, D.V.M., Executive Vice President of Portfolio, External Affairs & Development.

"I am thrilled to welcome Ottavio, Ravi, Susan and Rachel to Alcyone's executive management team. They are all accomplished leaders who bring diverse and vast expertise in the development of novel treatments for neurological disorders, in particular precision genetic therapies. They also all share the mission to build Alcyone into a premier organization that will bring transformative medicines to patients in need of better treatment options," said PJ Anand, Founder and Chief Executive Officer of Alcyone Therapeutics. "They will be integral to building our future as a leader in the development of central nervous system (CNS) therapies through a uniquely integrated, multi-disciplinary approach, as we leverage our FalconTM precision dosing technology and our multiple genetic therapy platforms."

About Alcyone TherapeuticsAlcyone Therapeutics is a biotechnology company developing precision therapies for neurological disorders with high unmet medical needs. The company integrates innovation in neuroscience, precision dosing platforms and in-house manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the company's proprietary intrathecal precising dosing platform that incorporates deep knowledge of CSF fluid dynamics, computational modeling and bioengineering, and multiple novel genetic therapy platforms developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological disease areas. Alcyone's lead programs target the treatment of Rett syndrome and IGHMBP2-Related Diseases (IRD). For more information, visit https://alcyonetx.com/, and follow Alcyone on LinkedIn and Twitter.

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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Alcyone Therapeutics Strengthens Executive Team with New Senior Leadership Appointments - WABI

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The Genomic Revolution: Why Investors Are Paying Attention – Visual Capitalist

Posted: at 5:29 pm

The Genomic Revolution: Why Investors Are Paying Attention

At the center of the genomic revolution is big data and DNA.

The implications are vast. With recent advancements, faster cancer detection is within reach, potentially saving thousands of lives each year. An initial research study shows this technology could save 66,000 live annually in the U.S. alone.

Whats more, genomic innovation goes beyond just cancer detection. Today it spans a variety of innovations, from gene editing to anti-cancer drugs.

In this graphic from MSCI, we look at four reasons why the genomics sector is positioned for growth thanks to powerful applications in medicine.

To start, the genomic revolution focuses on the study of the human genome, a human (or organisms) complete set of DNA.

A human consists of 23 pairs of chromosomes and 24,000 genes. Taken together, the human genetic code equals three billion DNA letters. Since most ailments have a link to our genetic condition, genomics involves the editing, mapping, and function of a genome.

With genomic innovation, large-scale applications of diagnostics and decision-making tools are made possible for a wide range of diseases.

Over the last century, the field of genomics has advanced faster than any other life sciences discipline.

The hallmark achievement is the Human Genome Project completed in 2001. Since then, scientists have analyzed thousands of peoples genes to identify the cause of heart disease, cancer, and other fatal afflictions.

Here are four areas where genomic innovation is making a big difference in the medical field.

Gene editing enables scientists to alter someones DNA, such as eye color. Broadly speaking, gene editing involves cutting DNA at a certain point and adding to, removing, or replacing this DNA.

For instance, gene editing enables living drugs. As the name suggests, living drugs are made from living organisms that harness a bodys immune system or other bodily process, and uses them to fight disease.

Based on analysis from ARK Invest, living drugs have a potential $200 billion addressable market.

Multi-cancer screening, supported by genomic sequencing and liquid biopsies, is projected to prevent more deaths from cancer than any other medical innovation.

Through a single blood test, multiple types of cancer can be detected early through synthetic biology advancements. Scientists use genomic sequencing (also referred to as DNA sequencing) to identify the genetic makeup of an organism, or a change in a gene which may lead to cancer.

Critically, screening costs are dropping rapidly, from $30,000 in 2015 to $1,500 in 2021. The combination of these factors is spurring a potential $150 billion market. This could be revolutionary for healthcare by shifting from a treatment-based model to a more preventative one in the future.

One modern form of DNA sequencing is long-read DNA sequencing. With long-read DNA sequencing, scientists can identify genetic sequences faster and more affordably.

For these reasons, long-read DNA sequencing is projected to grow to a $5 billion market, growing at a 82% annual rate.

Finally, the genomic revolution is making strides in agricultural biology. Here, research is looking at how to reduce the cost of producing crops, improving plant breeding, and enhancing quality.

One study shows that genomic advances in agriculture have led to six-fold increases in income for some farmers.

A number of genomic-focused companies have shown promising returns.

This can be illustrated by the MSCI ACWI Genomic Innovation Index, which has outperformed the benchmark by nearly 50% since 2013. The index, which was developed with ARK Invest, comprises roughly 250 companies who are working in the field of genomic innovation. In 2020 alone, the index returned over 43%.

From diagnostics to prevention, the genomic revolution is breaking ground in scalable solutions for global health. Investment opportunities are expected to follow.

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The Genomic Revolution: Why Investors Are Paying Attention - Visual Capitalist

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Precigen Announces Clearance of IND to Initiate Phase 1/1b Study for PRGN-3007 UltraCAR-T in Advanced ROR1+ Hematological and Solid Tumors – WAGM

Posted: at 5:29 pm

- PRGN-3007 is the first of the next generation UltraCAR-T, incorporating intrinsic PD-1 checkpoint inhibition in addition to the three effector genes used in the first generation UltraCAR-T technology -

Published: Oct. 26, 2021 at 8:05 AM EDT|Updated: 9 hours ago

GERMANTOWN,Md., Oct. 26, 2021 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to initiate the Phase 1/1b clinical trial of PRGN-3007 in advanced receptor tyrosine kinase-like orphan receptor 1-positive (ROR1+) hematological and solid tumors. PRGN-3007 is a first-in-class investigational therapy based on the next generation of Precigen's UltraCAR-T platform and incorporates intrinsic programmed cell death protein 1 (PD-1) blockade. This first-in-human investigator-initiated study of PRGN3007 will be conducted in collaboration with the H. Lee Moffitt Cancer Center & Research Institute.

ROR1 is overexpressed in various cancers with minimal expression in healthy adult tissues. ROR1 is aberrantly expressed in multiple hematological tumors, including chronic lymphocytic leukemia (CLL), mantle cell leukemia (MCL), acute lymphoblastic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL) and solid tumors, including breast adenocarcinomas encompassing triple negative breast cancer (TNBC), pancreatic cancer, ovarian cancer, and lung adenocarcinoma.

PRGN-3007 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy utilizing Precigen's clinically validated advanced non-viral gene delivery system and the well-established overnight, decentralized manufacturing process. Precigen has further advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment by incorporating intrinsic checkpoint blockade without the need for complex and costly gene editing techniques. PRGN-3007 is engineered using a single multicistronic transposon plasmid to simultaneously express a chimeric antigen receptor (CAR) targeting ROR1, membrane-bound interleukin15 (mbIL15), a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.

The PD-1/programmed death ligand 1 (PD-L1) pathway plays a vital role in how tumor cells evade immune response. While the blockade of the PD-1/PD-L1 pathway has demonstrated considerable benefit for treating various cancers, the use of systemic checkpoint inhibitors can lead to side effects associated with autoimmune response. The innovative design of PRGN-3007, where the blockade of PD-1 expression is intrinsic and localized to UltraCAR-T cells, is aimed at avoiding systemic toxicity and the high cost of checkpoint inhibitors by eliminating the need for combination treatment.

The Phase 1/1b clinical trial is an open-label study designed to evaluate the safety and efficacy of PRGN-3007 in patients with advanced ROR1+ hematological (Arm 1) and solid (Arm 2) tumors. The target patient population for Arm 1 includes relapsed or refractory CLL, relapsed or refractory MCL, relapsed or refractory ALL, and relapsed or refractory DLBCL. The target patient population for Arm 2 includes locally advanced unresectable or metastatic histologically confirmed TNBC. The study will enroll in two parts: an initial 3+3 dose escalation in each arm followed by a dose expansion at the maximum tolerated dose (MTD). Arm 1 and Arm 2 will enroll in parallel.

"ROR1 is an attractive target for treatment of multiple hematological and solid tumors due to its high expression in cancer and minimal expression in healthy adult tissues," said Javier Pinilla-Ibarz, MD, PhD, Senior Member, Lymphoma Section Head and Director of Immunotherapy, Malignant Hematology Department, H. Lee Moffitt Cancer Center & Research Institute, and Principal Investigator for the PRGN-3007 clinical study. "Preclinical studies of PRGN-3007 UltraCAR-T indicate the potential for improved efficacy by specific targeting of ROR1 combined with intrinsic blockade of PD-1 expression and we look forward to investigating the potential in this first-in-human clinical study."

"ROR1 expression is thought to be a potential adverse prognostic factor in TNBC patients," said Hatem Soliman, MD, Medical Director of the Clinical Trials Office, H. Lee Moffitt Cancer Center & Research Institute, and Principal Investigator for the TNBC cohort of PRGN-3007 clinical study. "Given the aggressive nature of TNBC and the need for additional treatment options, we are eager to investigate PRGN-3007 in this setting."

"This is the first study of our next generation UltraCAR-T, which adds checkpoint blockade to our non-viral, multigenic UltraCAR-T platform," said Helen Sabzevari, PhD, President and CEO of Precigen. "PRGN-3007 eliminates the need to combine an antigen-specific CAR-T with a separate checkpoint inhibitor, which has the potential to avoid systemic toxicity and reduce cost. This new study is a big step toward our UltraCAR-T library approach, which aims to deliver personalized autologous UltraCAR-T therapies based on apatient's cancer indication and biomarker profile usingovernight manufacturingat the patient's medical center."

About Receptor Tyrosine Kinase-like Orphan Receptor 1 (ROR1)ROR1 is a type I orphan-receptor that is expressed during embryogenesis and by certain hematological and solid tumors but is undetectable on normal adult tissues.1-3 ROR1 plays an important role in oncogenesis by activating cell survival signaling events, particularly the non-canonical WNT signaling pathway.4 Aberrant expression of ROR1 is detected in multiple hematological malignancies including CLL5, MCL6, ALL7, and DLBCL.8 Elevated ROR1 expression is detected in various solid tumors, including breast adenocarcinoma encompassing TNBC, pancreatic cancer, ovarian cancer, Ewing's sarcoma and lung adenocarcinoma.9-14 Many human breast adenocarcinomas express high levels of ROR1, which is not expressed by normal breast tissue.15

Precigen: Advancing Medicine with PrecisionPrecigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit http://www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

TrademarksPrecigen, UltraCAR-T and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

Cautionary Statement Regarding Forward-Looking Statements Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical studies, clinical trials, discovery programs and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties, including the possibility that the timeline for the Company's clinical trials might be impacted by the COVID-19 pandemic, and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission.

References

1Balakrishnan, A., et al., Analysis of ROR1 Protein Expression in Human Cancer and Normal Tissues. Clin Cancer Res, 2017. 23(12): p. 3061-3071.

2Green, J.L., et al., ROR receptor tyrosine kinases: orphans no more. Trends in Cell Biology, 2008. 18(11): p. 536-544.

3Rebagay, G., et al., ROR1 and ROR2 in Human Malignancies: Potentials for Targeted Therapy. Front Oncol, 2012. 2(34).

4Zhao Y, et al., Tyrosine Kinase ROR1 as a Target for Anti-Cancer Therapies. Front. Oncol, 2021.

5Baskar, S., et al., Unique Cell Surface Expression of Receptor Tyrosine Kinase ROR1 in Human B-Cell Chronic Lymphocytic Leukemia. Clin Cancer Res, 2008. 14(2): p. 396-404.

6Hudecek, M., et al., The B-cell tumorassociated antigen ROR1 can be targeted with T cells modified to express a ROR1-specific chimeric antigen receptor. Blood, 2010. 116(22): p. 4532-4541.

7Enayati H, et al., Expression of ROR1 Gene in Patients with Acute Lymphoblastic Leukemia. IJBC 2019; 11(2): 57-62.

8Ghaderi, A., et al., ROR1 Is Expressed in Diffuse Large B-Cell Lymphoma (DLBCL) and a Small Molecule Inhibitor of ROR1 (KAN0441571C) Induced Apoptosis of Lymphoma Cells. Biomedicines, 2020. 8(6).

9Zhang, S., et al., The onco-embryonic antigen ROR1 is expressed by a variety of human cancers. Am J Pathol, 2012. 181(6): p. 1903-10.

10Zhang, S., et al., ROR1 is expressed in human breast cancer and associated with enhanced tumor-cell growth. PLoS One, 2012.7(3): p. e31127.

11Potratz, J., et al., Receptor tyrosine kinase gene expression profiles of Ewing sarcomas reveal ROR1 as a potential therapeutic target in metastatic disease. Mol Oncol, 2016. 10(5): p. 677-92.

12Zheng, Y.Z., et al., ROR1 is a novel prognostic biomarker in patients with lung adenocarcinoma. Sci Rep, 2016. 6: p. 36447.

13Choi, M.Y., et al., Pre-clinical Specificity and Safety of UC-961, a First-In-Class Monoclonal Antibody Targeting ROR1. Clin Lymphoma Myeloma Leuk, 2015. 15 Suppl: p. S167-9.

14Balakrishnan, A., et al., Analysis of ROR1 Protein Expression in Human Cancer and Normal Tissues. Clin Cancer Res, 2017. 23(12): p. 3061-3071.

15Zhang S. et al., ROR1 is expressed in human breast cancer and associated with enhanced tumor-cell growth. PLoS One, 2012, 7:e31127.

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Precigen Announces Clearance of IND to Initiate Phase 1/1b Study for PRGN-3007 UltraCAR-T in Advanced ROR1+ Hematological and Solid Tumors - WAGM

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