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Category Archives: Gene Medicine

Gene Edited vs Genetically Modified | Science-Based Medicine

The public debate about genetically modified organisms (GMOs) seems to have quieted down a bit, but I suspect that is an artifact of the pandemic (all news is COVID news these days). Still, public concern about the health effects and overall safety of GMOs remains high, and is the single issue in which there is the greatest disconnect between public opinion and scientific consensus.

In a 2020 Pew Research survey 38% of Americans felt GMOs were unsafe, 27% felt they were safe, and 33% did not know. These numbers varied significantly by country, with the mean being 48/13/37 respectively. Australia was the only country to reach parity, with 31% saying they are safe and 31% unsafe, and no country had a plurality of the public believing GMOs are safe.

Meanwhile, there is a strong scientific consensus that GMO crops are indeed safe for human or animal consumption. According to a 2016 study by the National Academies of Sciences, Engineering and Medicine:

while recognizing the inherent difficulty of detecting subtle or long-term effects on health or the environment, the study committee found no substantiated evidence of a difference in risks to human health between current commercially available genetically engineered (GE) crops and conventionally bred crops, nor did it find conclusive cause-and-effect evidence of environmental problems from the GE crops.

Specifically regarding human health:

The committee carefully searched all available research studies for persuasive evidence of adverse health effects directly attributable to consumption of foods derived from GE crops but found none.

Those who wish to fearmonger about GMOs (as they do about vaccines, cell phones, or other alleged hazards) can always demand more evidence, or claim risks that have eluded existing evidence. This is always possible because it is impossible to prove zero risk for anything. And of course we legitimately need to be on the look out for evidence of subtle harm missed by prior research. But at some point the evidence showing a lack of risk is robust enough that we can be relatively confident any remaining possible risk is small enough to safely ignore.

We can also follow basic scientific principles there is no scientific reason to suspect that GMOs should be unsafe for consumption. There is no plausible mechanism, and public fears are mostly based on gross misunderstandings of the basic science, such as the false notions that only GMOs have DNA or that they can alter the genes of those who consume them. Often fears are based upon an overapplication of the precautionary principle, while ignoring a more proper risk vs benefit analysis, and further ignoring comparisons to the alternatives. It is reasonable to argue that not using genetic technology to improve agriculture is a greater risk than using it. Further, techniques for developing new cultivars that are not categorized as genetic modification may pose a higher risk of introducing harmful substances into the food chain.

There is also the point that GMO is a somewhat arbitrary category, targeting certain technologies but not others, based, it appears, on nothing but a vague ick factor. For example, mutation farming, in which radiation or chemicals are used to increase the rate of mutation in the hopes that a rare mutation will prove useful, is not considered GMO. Another example is forced hybridization, in which species that would not normally cross fertilize are forced to do so. There is also greater fear for transgenic GMOs (where a gene is inserted from a distant species) vs cisgenic (where the inserted gene is from a closely related species). Again, there is no scientific basis for this distinction, only an ick factors that derives from the idea that a fish gene is being inserted into a tomato. Such distinctions are not genetically important, however (apart from some technical aspects that dont affect the final product), as evidenced by the fact that humans and bananas share about 60% of their genes. In short there is no such thing as a fish gene.

The lines between different techniques for developing crops is getting further blurred over time, making the GMO category increasingly arbitrary and scientifically dubious. But this can have a good effect from a regulatory perspective, in that it can allow certain techniques to escape from the unnecessarily harsh regulatory environment imposed on GMOs. One such emerging category is gene-edited.

Gene-edited crops are those that are created by altering existing genes already present within the genome, rather than inserting an entirely new gene. This is an increasingly important category because of innovations like CRISPR, that makes gene editing relatively quick, cheap, and easy. In fact gene editing is becoming so powerful it may, to some extent, render the GMO category obsolete.

The political fight over how to regulate gene-edited crops is now underway. There has been partial success in some countries and regulatory zones, and failures in others. The landscape is complex, however. In the US, for example, regulating crops falls under the FDA, EPA, and USDA, and these agencies can have conflicting categories and regulations. Currently:

The three agencies regulate the characteristics of the products themselves and not the process to develop it. Gene edited crops lacking foreign genes (which trigger regulation as GMOs) and that do not pose a risk to other plants, and gene-edited food showing no food safety attributes different from those of traditionally bred crops, are not subject to pre-market regulatory evaluation. It remains the responsibility of the developer to assure that products placed on the market are safe for use and consumption.

Currently the EU and New Zealand are the only legislatures that regulate gene edited crops as if they were GMOs. Just recently the UK moved to relax regulations on gene edited crops, essentially allowing their development and use in the UK (thanks to Brexit). Canada, Japan, Brazil, Argentina, Colombia, Chile, Paraguay, and Uruguay also have relaxed regulations for gene edited crops. The rest of the world has not made a determination.

This is an important public debate to have. This is especially true because anti-GMO attitudes are one of the few topics where the knowledge deficit model of science communication is actually effective. In other words people who are anti-GMO are largely open to changing their minds if they are given more scientifically accurate information, because being anti-GMO is largely based on misinformation (rather than something harder to change like tribal affiliation or ideology). The stakes here are also very high. The experience of golden rice is a good example this is a GMO that can potentially save millions of poor children from blindness or death, and its deployment is being slowed by unscientific opposition and unreasonably burdensome regulations.

The fight for gene edited crops may be politically easier to win, and increasingly more important as technology shifts in this direction anyway.

Founder and currently Executive Editor of Science-Based Medicine Steven Novella, MD is an academic clinical neurologist at the Yale University School of Medicine. He is also the host and producer of the popular weekly science podcast, The Skeptics Guide to the Universe, and the author of the NeuroLogicaBlog, a daily blog that covers news and issues in neuroscience, but also general science, scientific skepticism, philosophy of science, critical thinking, and the intersection of science with the media and society. Dr. Novella also has produced two courses with The Great Courses, and published a book on critical thinking - also called The Skeptics Guide to the Universe.

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StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa – WITN

StemExpress to use utilize the Thermo Fisher Accula rapid PCR testing system to provide event attendees with accurate results in 30 minutes.

Published: Oct. 5, 2021 at 3:33 PM EDT|Updated: 2 hours ago

SACRAMENTO, Calif., Oct. 5, 2021 /PRNewswire/ --StemExpress is proud to announce that they will be the official COVID-19 testing provider for 2021's Meeting on the Mesa, a hybrid event bringing together great minds in the cell and gene biotech sphere. It has partnered with Alliance for Regenerative Medicine to comply with the newly implemented California state COVID-19 vaccination and testing policy regarding gatherings with 1,000 or more attendees. This partnership will allow the vital in-person networking aspect of the event to commence while protecting the health and safety of participants and attendees.

In-person networking commences at the 2021 Cell and Gene Meeting on the Mesa with COVID-19 testing options provided by StemExpress.

As a leading global provider of human biospecimen products, StemExpress understands the incredible impact that Meeting on the Mesa has on the industry and has been a proud participant for many years. For over a decade, StemExpress has provided the cell and gene industry with vital research products and holds valued partnerships with many of this year's participants. As such, it understands the immense value that in-person networking provides and is excited to help bring this element back to the meeting safely and responsibly.

StemExpress has been a trusted provider of widescale COVID-19 testing solutions since early 2020 - providing testing for government agencies, public health departments, private sector organizations, and the public nationwide. For Meeting on the Mesa, StemExpress is offering convenient testing options for unvaccinated attendees and those traveling from outside of the country. Options will include take-home RT-PCR COVID Self-Testing Kits and on-site, rapid PCR testing for the duration of the event. The self-testing kit option allows attendees to test for COVID in the days leading up to the event for a seamless admission and the days following the event to confirm they haven't been exposed. The on-site rapid testing option utilizes the new Thermo Fisher Accula, offering in-person testing at the event with results in around 30 minutes. StemExpress is excited to bring these state-of-the-art COVID testing solutions to the frontlines of the Cell & Gene industry to allow for safe in-person connections.

The StemExpress partnership with Alliance for Regenerative Medicine seeks to empower the entire cell and gene industry with a long-awaited opportunity to return to traditional networking practices. It is well known that innovation doesn't exist in a vacuum - allowing great minds to come together is a sure way to spur scientific growth and advance cutting-edge research, giving hope for future cures.

Cell and Gene Meeting on the Mesa will take place October 12th, 2021, through October 14th, 2021, at Park Hyatt Aviara,7100 Aviara Resort Drive Carlsbad, CA 92011. To learn more about the event, please visit MeetingOnTheMesa.com.

For more information about COVID testing solutions for businesses and events, visit https://www.stemexpress.com/covid-19-testing/.

About StemExpress:

Founded in 2010 and headquartered in Sacramento, California, StemExpress is a leading global biospecimen provider of human primary cells, stem cells, bone marrow, cord blood, peripheral blood, and disease-state products. Its products are used for research and development, clinical trials, and commercial production of cell and gene therapies by academic, biotech, diagnostic, pharmaceutical, and contract research organizations (CRO's).

StemExpress has over a dozen global distribution partners and seven (7) brick-and-mortar cellular clinics in the United States, outfitted with GMP certified laboratories. StemExpress runs its own non-profit supporting STEM initiatives, college and high school internships, and women-led organizations. It is registered with the U.S. Food and Drug Administration (FDA) and is continuously expanding its network of healthcare partnerships, which currently includes over 50 hospitals in Europe and 3 US healthcare systems - encompassing 31 hospitals, 35 outpatient facilities, and over 200 individual practices and clinics.

StemExpress has been ranked by Inc. 500 as one of the fastest-growing companies in the U.S.

About the Alliance for Regenerative Medicine:

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact: Anthony Tucker, atucker@stemexpress.com

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Nucleic Acid Based Gene Therapy Market Analysis Of Industry Trends And Market Growth Opportunities As Per The Business Research Company’s Nucleic Acid…

This report describes and evaluates the global nucleic acid-based gene therapy market. It covers three five-year periods, including 2015 to -2020, termed the historic period, 2020-2025 forecast period and 2025-2030 a further forecast period.

LONDON, Oct. 05, 2021 (GLOBE NEWSWIRE) -- According to The Business Research Companys research report on the nucleic acid-based gene therapy market, companies in the nucleic acid-based gene therapy market and research institutes are increasing the number of pipeline studies to develop gene therapy to treat various diseases. Companies have also started investing in startups and other early-stage companies to develop pipelines for gene therapies. Cell and gene therapies (CGT) have transformed not only how humans treat intractable and genetic diseases, but also reformed the entire pharmaceutical ecosystem. As of 2019, more than 27 CGT products were launched across the globe and nearly 990 companies are engaged in the commercialization, and research & development of next-generation therapies. Additionally, there are more than 1,000 regenerative medicine trials taking place across the globe.

Such global nucleic acid based gene therapy market trends are obtainable with nucleic acid-based gene therapy manufacturers progressively investing in the launch of new manufacturing facilities and product portfolio expansion to meet the increasing demand for gene therapy and related products. Players operating in the nucleic acid-based gene therapy market are gradually investing in the developing regions to capitalize on untapped market opportunities. For example, in September 2021, Viralgen, a Bayer-owned CDMO, spent upwards of 50 million (US$ 55 million) to expand its capacity for gene therapy manufacturing services at its Miramon Technology Park site in San Sebastian, Spain. The commercial facility will have nine cleanrooms, each with a batch capacity of up to 2,000 L. Viralgen claims that this has expanded its existing viral vector capacity 15-fold, helping to meet the demand for gene therapy production. In addition, in May 2021, AGC Biologics, a global biopharmaceutical contract development and manufacturing organization (CDMO), announced plans to expand their Gene Therapy Center of Excellence in Milan, Italy.

Story continues

Major players in the nucleic acid gene therapy market include Copernicus Therapeutics, Moderna Inc., Wave Life Sciences, Protagonist Therapeutics and Transgene.

The Business Research Companys report titled Nucleic Acid Based Gene Therapy Global Market Report 2021 - By Technology (Anti-Sence and Anti-Gene, Short Inhibitory Sequences, Gene Transfer Therapy, Nucleoside Analogs, Ribozymes, Aptamers), By Application (Oncology, Muscular Dystrophy/ Muscular Disorders, Rare Diseases), By End User (Hospitals And Clinics, Academic And Research Institutes), COVID-19 Growth And Change covers major nucleic acid based gene therapy companies, nucleic acid based gene therapy market share by company, nucleic acid based gene therapy manufacturers, nucleic acid based gene therapy market size, and nucleic acid based gene therapy market forecasts. The report also covers the global nucleic acid based gene therapy market and its segments.

Request For A Sample Of The Global Nucleic Acid Based Gene Therapy Market Report:

https://www.thebusinessresearchcompany.com/sample.aspx?id=2820&type=smp

The global nucleic acid based gene therapy market size is expected to grow from $0.56 billion in 2020 to $0.61 billion in 2021 at a compound annual growth rate (CAGR) of 8.9%. The growth is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges. The nucleic acid-based gene therapy market is expected to reach $0.85 billion in 2025 at a CAGR of 9%.

North America is the largest region in the global nucleic acid-based gene therapy market, accounting for 46.2% of the total in 2020. It is followed by the Western Europe, Asia Pacific and then the other regions. Going forward, the fastest-growing regions in the nucleic acid-based gene therapy market will be the Middle East and Eastern Europe where growth will be at CAGRs of 33.7% and 26.0% respectively. These will be followed by South America and Asia Pacific, where the markets are expected to register CAGRs of 21.0% and 20.4% respectively.

The nucleic acid-based gene therapy market covered in this report is segmented by technology into anti-sense and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, others. It is also segmented by application into oncology, muscular dystrophy/ muscular disorders, rare diseases and by end user into hospitals and clinics, academic and research institutes.

The top opportunities in the nucleic acid-based gene therapy market segmented by technology will arise in the anti-sense and anti-gene oligonucleotides segment, which will gain $1,290.7 million of global annual sales by 2025. The top opportunities segmented by application will arise in the muscular dystrophy/muscular disorders segment, which will gain $1,000.2 million of global annual sales by 2025, segmented by end-user will arise in the hospitals and clinics segment, which will gain $2,133.7 million of global annual sales by 2025. The nucleic acid-based gene therapy market size will gain the most in the USA at $915.0 million.

Nucleic Acid Based Gene Therapy Global Market Report 2021 COVID-19 Growth And Change is one of a series of new reports from The Business Research Company that provide nucleic acid-based gene therapy market overviews, nucleic acid-based gene therapy market analyze and forecast market size and growth for the whole market, nucleic acid-based gene therapy market segments and geographies, nucleic acid-based gene therapy market trends, nucleic acid-based gene therapy market drivers, nucleic acid-based gene therapy market restraints, nucleic acid-based gene therapy market leading competitors revenues, profiles and market shares in over 1,000 industry reports, covering over 2,500 market segments and 60 geographies.

The report also gives in-depth analysis of the impact of COVID-19 on the market. The reports draw on 150,000 datasets, extensive secondary research, and exclusive insights from interviews with industry leaders. A highly experienced and expert team of analysts and modelers provides market analysis and forecasts. The reports identify top countries and segments for opportunities and strategies based on market trends and leading competitors approaches.

Here Is A List Of Similar Reports By The Business Research Company:

Gene Editing Global Market Report 2021 - By Technology (CRISPR, TALEN, ZFN), By End Users (Biotechnology, Pharmaceutical, Contract Research Organization), By Application (Animal Genetic Engineering, Plant Genetic Engineering, Cell Line Engineering), COVID-19 Growth And Change

CRISPR Technology Global Market Report 2021 - By Product Type (Design Tools, Plasmid And Vector, Cas9 And G-RNA, Delivery System Products), By Application (Genome Editing/ Genetic Engineering, Genetically Modified Organisms, Agricultural Biotechnology), By End-User (Industrial Biotech, Biological Research, Agricultural Research, Therapeutics And Drug Discovery), COVID-19 Growth And Change

Stem Cell Therapy Global Market Report 2021 - By Type (Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy), By Cell Source (Adult Stem Cells, Induced Pluripotent Stem Cells, Embryonic Stem Cells), By Application (Musculoskeletal Disorders, Wounds And Injuries, Cancer, Autoimmune Disorders), By End-User (Hospitals, Clinics), COVID-19 Growth And Change

Interested to know more about The Business Research Company?

The Business Research Company is a market intelligence firm that excels in company, market, and consumer research. Located globally it has specialist consultants in a wide range of industries including manufacturing, healthcare, financial services, chemicals, and technology.

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The Business Research Companys flagship product, Global Market Model, is a market intelligence platform covering various macroeconomic indicators and metrics across 60 geographies and 27 industries. The Global Market Model covers multi-layered datasets which help its users assess supply-demand gaps.

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Nobel prize in medicine won by US scientists who unlocked the secrets of our sense of touch – Livescience.com

The 2021 Nobel prize in physiology or medicine has been awarded to two U.S. scientists who discovered the microscopic secrets behind the human sense of touch.

David Julius, of the University of California San Francisco, received half of the prize for using "capsaicin, a pungent compound from chili peppers that induces a burning sensation, to identify a sensor in the nerve endings of the skin that responds to heat," while Ardem Patapoutian, of the Scripps Research Institute in La Jolla, California, received the other half for using "pressure-sensitive cells to discover a novel class of sensors that respond to mechanical stimuli in the skin and internal organs," the Royal Swedish Academy of Sciences announced Monday (Oct. 4).

Their discoveries "have allowed us to understand how heat, cold and mechanical force can initiate the nerve impulses that allow us to perceive and adapt to the world around us," the Nobel Committee said in a statement. "This knowledge is being used to develop treatments for a wide range of disease conditions, including chronic pain."

Related: 7 revolutionary Nobel Prizes in medicine

The award comes with a prize of 10 million Swedish kronor ($1.15 million) to be shared equally between the two winners.

Beginning in the 1990s, the scientists pieced together the molecular pathways that translate heat and pressure detected on the skin into nerve impulses perceived by the brain. Julius and his colleagues started the work by creating a library of millions of DNA segments containing genes found in sensory nerve cells. By adding the genes one by one to cells that did not normally react to capsaicin, they eventually found that a single gene was responsible for the burning sensation associated with capsaicin. The gene they had discovered gave cells the ability to build a protein called TRPV1, which was activated at temperatures hot enough to be considered painful.

Both Julius and Patapoutian independently went on to use menthol to discover another protein, TPRM8, which was activated by cold temperatures, as well as a number of other proteins that detected a range of different temperatures.

Building on this work, Patapoutian and his colleagues created a library of 72 genes that they suspected encoded blueprints to make receptors for mechanical pressure. By painstakingly deactivating these genes one by one in cells, they discovered that one of the genes produced a protein that spurred cells to produce a tiny electrical signal each time they were prodded. The receptor they had discovered was not only vital for sensing mechanical force, but was also used in various ways to maintain blood vessels, alongside having a proposed role in adjusting the bodys blood pressure.

Soon after that, they found a second protein receptor that was vital in sensing body position and motion, a sense known as proprioception. They named the two receptors Piezo1 and Piezo2, after the Greek word for pressure.

Not only did the discoveries help explain the mechanisms behind sensory experiences like temperature and pressure, but they also opened up a world of possibilities for new drugs targeting the receptors from painkillers to drugs that could alleviate blood pressure across blood vessels and organs.

"While we understood the physiology of the senses, what we didn't understand was how we sensed differences in temperature or pressure," Oscar Marin, director of the MRC Centre for Neurodevelopmental Disorders at Kings College London told The Associated Press. "Knowing how our body senses these changes is fundamental because once we know those molecules, they can be targeted. It's like finding a lock and now we know the precise keys that will be necessary to unlock it."

Joseph Erlanger and Herbert Gasser, who shared the Nobel prize in physiology or medicine in 1944, first discovered specialized nerve cells responsive to both painful and non-painful touch.

Last year's prize went to three scientists for their discovery of hepatitis C, a blood-borne virus that causes chronic liver inflammation. The deadly disease's discovery was a breakthrough that enabled doctors to identify the virus in patients' blood and develop a cure, Live Science previously reported.

Originally published on Live Science.

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NIH awards UT Southwestern researchers $4.4 million to study the genetic basis of vocal learning – UT Southwestern

DALLAS Oct. 5, 2021 A UT Southwestern research team has received the National Institutes of Healths prestigious Transformative Research Award to further their study of zebra finches to investigate the genetic basis of vocal imitation abilities.

Todd Roberts, Ph.D.

The award grants $4.4 million over five years to Todd Roberts, Ph.D., Associate Professor of Neuroscience, Joseph Takahashi, Ph.D., Professor and Chair of Neuroscience, and Kent Hamra, Ph.D., a Senior Research Associate in Obstetrics and Gynecology. Drs. Roberts and Takahashi are members of the Peter ODonnell Jr. Brain Institute.

TheTransformative Research Award is part of nearly$9 millionin prestigious NIH Directors Awards received by UT Southwestern researchers today from the NIH Common Funds High-Risk, High-Reward Program, which supports scientists pursuing highly innovative research with the potential to have a broad impact on biomedical, behavioral, or social sciences.

Joseph Takahashi, Ph.D.

Zebra finches are a vocal learning species that provide the only practical platform for systematically identifying the genes involved in this important social behavior. Like speech, zebra finch song is a culturally transmitted behavior learned via imitation, said Dr. Roberts, the principal investigator on this award. We think a forward genetic screen for mutations that affect vocal imitation, followed by the detailed genetic mapping and manipulations developed through this proposal, will identify genetic signatures for this polygenic trait.

The scientists are seeking to establish the first mutagenesis screen in a vocal learning species and the genetic tools to independently test the function of identified genes by developing novel transgenic zebra finches using germline gene targeting technologies. The research may shed new light on speech and language deficits associated with autism spectrum disorder.

Kent Hamra, Ph.D.

Previous research by Dr. Roberts, published in Science Advances, found that inactivating a gene closely associated with autism prevents songbirds from replicating their fathers songs.

UTSouthwestern ratesNo. 1 among global institutions in the health care sectorin the2021NatureIndexfor its published research, as well as among thetop 20 U.S. institutions overall for published research in life sciences journals.

Dr. Roberts is the Thomas O. Hicks Scholar in Medical Research. Dr. Takahashi holds the Loyd B. Sands Distinguished Chair in Neuroscience.

About UTSouthwestern Medical Center

UT Southwestern, one of the nations premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 25 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,800 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UTSouthwestern physicians provide care in about 80 specialties to more than 117,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 3 million outpatient visits a year.

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BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa – WWNY

Published: Oct. 4, 2021 at 6:00 AM EDT

NEW YORK, Oct. 4, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced today that Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the2021 Cell & Gene Meeting on the Mesa, being held as a hybrid conferenceOctober 12-14, and October 19-20, 2021.

Dr. Lindborg's presentation highlights the expansion of Brainstorm's technology portfolio to include autologous and allogeneic product candidates, covering multiple neurological diseases. The most progressed clinical development program, which includes a completed phase 3 trial of NurOwn in ALS patients, remains the highest priority for Brainstorm. Brainstorm is committed to pursuing the best and most expeditious path forward to enable patients to access NurOwn.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning October 12. Those who wish to listen to the presentation are required to registerhere. At the conclusion of the 2021 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website underEvents and Presentations.

About the 2021 Cell & Gene Meeting on the Mesa

The meeting will feature sessions and workshops covering a mix of commercialization topics related to the cell and gene therapy sector including the latest updates on market access and reimbursement schemes, international regulation harmonization, manufacturing and CMC challenges, investment opportunities for the sector, among others. There will be over 135 presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

The conference will be delivered in a hybrid format to allow for an in-person experience as well as a virtual participation option. The in-person conference will take place October 12-14 in Carlsbad, CA. Virtual registrants will have access to all content via livestream during program dates. Additionally, all content will be available on-demand within 24 hours of the live program time. Virtual partnering meetings will take place October 19-20 via Zoom.

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

ContactsInvestor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 646.791.9729egoldstein@lifesciadvisors.com

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768Paul.tyahla@smithsolve.com

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