Category Archives: Genetic Engineering

The COVID-19 pandemic highlighted the importance of health care and the role biopharma companies play in drug and vaccine development. As a result, the sector grew tremendously during the past 2 years.

However, with the easing of global coronavirus restrictions, along with a broad market selloff, the fortunes of these companies changed entirely in 2022.

For example, the is down about 9.4% year-to-datewhile still returning 3.5% over the past 12 months. On the other hand, the has dropped over 20.6% year-to-date (YTD) and 24.8% in the past 52 weeks, meaning it is now in the bear market territory.

In comparison, the and the have lost 13.6% and 22.7% so far in 2022.

Still, these declines potentially offer better entry points into many robust stocks and exchange-traded funds (ETFs) that hold them. So, today's article introduces two ETFs that deserve readers' attention during the rest of the year.

Health care stocks are typically excellent defensive plays in an environment of high and market . In addition, metrics suggest these companies could further benefit from changing demographics stateside.

For instance, the most recent census shows that America is aging. Research highlights:

"By 2030, more people in the United States will be older than age sixty-five than younger than age five. Our health care system is unprepared for the complexity of caring for a heterogeneous population of older adults."

Health care spending currently accounts for about a fifth of the national gross domestic product (GDP). As the needs of Americans change and possibly increase, we can expect health care stocks to capture part of that growth as well.

Therefore, the first fund on today's list is the Invesco S&P 500 Equal Weight Health Care ETF (NYSE:). It offers exposure to large- and mid-cap health care companies from the S&P 500 index.

RYH currently has 66 holdings, where the top 10 comprise almost 14% of $890.2 million in net assets. The fund was first listed in November 2006.

Over a quarter of the companies are in the health care equipment & supplies segment. Next come health care providers & services (24.6%), life sciences tools & services (18.2%), pharmaceuticals (15.4%), and biotechnology (13.1%).

Among the leading names on the roster are Viatris (NASDAQ:); Catalent (NYSE:), Merck & Company (NYSE:), Vertex Pharmaceuticals (NASDAQ:), Eli Lilly (NYSE:), McKesson (NYSE:) and Cigna (NYSE:).

RYH hit a 52-week on May 12 and is down close to 12.3% YTD. Trailing price-to-earnings (P/E) and price-to-book (P/B) ratios stand at 16.75x and 4.26x.

We like the diversity offered by this equal-weight ETF. Interested readers could find long-term value in RYH around these levels.

Academic research suggests:

"The biotechnology industry has been an engine of innovation for the U.S. healthcare system and, more generally, the U.S. economy. It is by far the most research-intensive industry in the U.S."

At present, the U.S. biotech industry is valued around $140 billion. A recent survey revealed a "strengthening investor appetite" for biotechs despite the recent decline in biotech shares. Among the investors surveyed, "66% think that biotech will outperform the broader market in 2022," while "58% plan to increase their exposure to the sector."

Next up on the list is the First Trust NYSE Arca Biotechnology Index Fund (NYSE:). It invests in global biotech firms that concentrate on life sciences tools, genetic engineering, molecular biology, and genomics. It started trading in June 2006, and has close to $1.3 billion in net assets.

This passively managed ETF tracks the . It is rebalanced quarterly and holds a basket of 30 stocks.

40% of the portfolio is the top 10 stocks. Among them are United Therapeutics (NASDAQ:), Grifols (NASDAQ:), Gilead Sciences (NASDAQ:), Mettler-Toledo International (NYSE:), Qiagen (NYSE:), Amgen (NASDAQ:), and Seagen (NASDAQ:).

FBT is down roughly 15.7% YTD and 19% over the past 12 months. The fund saw a multi-year low also on May 12.

Trailing P/E and P/B ratios stand at 15.11x and 3.61x. Contrarian readers ready to become "greedy when others are fearful" could consider buying the dips in FBT.

More:
2 Health Care And Biotech ETFs Poised For Robust Returns This Year - Investing.com

Genome editing technologies are important for drug development as well as the detection and treatment of human genetic disorders. Genome editing is used in NGS, DNA analysis and profiling, as well as plant and animal genetic engineering. Demand for genome editing technologies is predicted to rise significantly throughout the forecast period due to the rising application areas of genomics.

Increasing cancer and other genetic disorders, growing preference for personalized medicine, increase in R&D expenditure and growth of biotechnology and pharmaceutical industries, increase in private and public sector funding, rapid advancements in sequencing and genome editing technologies, non-labelling of gene-edited products such as Genetically Modified Organisms (GMOs), applications in various drug discovery processes are some of the factors driving this rise in global genome editing market .

Get Free [emailprotected]https://skyquestt.com/sample-request/genome-editing-market

Genome editing has already shown potential in the treatment of genetic disorders, infectious diseases, and cancer. Cancer and other genetic illnesses are becoming more common, and individualized medicine is becoming more popular. Increased R&D spending and the expansion of the biotechnology and pharmaceutical industries increased business and public sector investment, as appears to have done rapid advances in sequencing and genome editing industry. Non-labelling of gene-edited goods such as GMOs, as well as applications in various drug development procedures, are a few of the factors driving the global genome editing markets growth.

Drug discovery research activities increase the preference for gene-editing techniques, and thus extensive use of gene editing techniques will stimulate industry progression. Furthermore, recently developed therapies and drugs used in the treatment of genetic diseases have reduced mortality rates, which will have an impact on genome editing market revenue in the coming years. Because of the benefits of genome editing, various governments are funding public and commercial research, as well as academic institutes, to increase genome editing market revenue.

Read the market research report, Global Genome Editing Market Segmented By Technology (CRISPR, TALEN, ZFN, Antisense, Other Technologies) By Delivery Method (Ex-vivo, In-vivo) By Mode (Contract, In-house) By End-use (Biotechnology And Pharmaceutical Companies, Academic And Government Research Institutes, Contract Research Organizations) & By Region Forecast And Analysis 2022-2028 by SkyQuest

Increased funding for genetic research in developed regions, including North America, will drive global genome editing market growth during the forecast period. The United States government funds a variety of research programs that aid in the drug discovery process for several rare genetic diseases. The National Institutes of Health (NIH), a primary agency of the United States government, is in charge of biomedical and genetic research and awards billions of dollars in funding. For instance, a research team from Arizona State University and Baylor College of Medicine received approximately USD 1.6 million in NIH funding to evaluate advances in human genome editing. This funding also aided in the development of recommendations for technology governance and prospects.

North America dominated the genome editing market, accounting for 38.4% of the global market revenue in 2021. The presence of strong research as well as a commercial base for advanced therapy development, combined with a large number of clinical trials for gene and stem cell therapies in the region, has significantly contributed to the regions market dominance. Furthermore, the increasing number of patents granted to key players based in the United States has accelerated the adoption of these tools in the country, resulting in significant market growth of global genome editing market. For instance, Merck KGaA, recently announced that two of the companys patents for CRISPR gene-editing technologies had been approved in the United States. In the Asia Pacific, on the other hand, the market is expected to grow at the fastest rate during the forecast period. In November 2020, China announced its ten most significant agricultural advances, one of which was gene-editing technology, demonstrating the countrys high market penetration in this space.

The report published by SkyQuest Technology Consulting provides in-depth qualitative insights, historical data, and verifiable projections about genome editing market size. The projections featured in the report have been derived using proven research methodologies and assumptions.

What does this Report Deliver?

Speak to [emailprotected]https://skyquestt.com/speak-with-analyst/genome-editing-market

SkyQuest has segmented the Global Genome Editing Market based on Technology, Delivery Method, Mode, End-Use, Application, and Region:

Key Players of the Global Genome Editing Market

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The Global Genome Editing Market was valued at USD 5.25 Billion in 2021, and it is expected to reach a value of USD 17.02 Billion by 2028, at a CAGR...

The Director of International Centre for Genetic Engineering and Biotechnology, New Delhi, delivers convocation address at Karnatak University

The Director of International Centre for Genetic Engineering and Biotechnology, New Delhi, delivers convocation address at Karnatak University

Director of International Centre for Genetic Engineering and Biotechnology, New Delhi, Dinakar M. Salunke has said that India always had a strong foundation for science and technology partly due to traditions dating back to origins of Indian civilisation. And, a large part of modern scientific base in India has been due to the efforts of individuals who promoted science.

Delivering the convocation address at the 72nd annual convocation of Karnatak University in Dharwad on Tuesday, he said that modern India has resolved the path of economic, industrial and agricultural developments through scientific approaches.

India has emerged as a major player in the field of science and technology due to early emphasis on scientific temper and the responsibility of carrying forward the rich tradition lies with the younger generation, Mr. Salunke said.

Pointing out the countrys accomplishments in the field of biotechnology, pharmaceuticals, atomic energy, electronics, telecommunication, space technology, defence developments, agriculture and healthcare and other sectors have been globally acknowledged, he said that building a cadre of well trained scientists, technologists and field experts with scientific training will be a major driver of the countrys emergence as a knowledge superpower.

Mr. Salunke said that universities in the country have made a great contribution in this connection.

Governor and Chancellor of the university Thavarchand Gehlot conferred honorary doctorate degrees onthe seer of Murugha Mutt Sri Mallikarjun Swamy, the former vice-chancellor and scientist Srinivas Saidapur and Supreme Court advocate Manoj Gorkela.

Lauding the contribution of the university to the field of education in the last seven decades, the Governor called upon the fresh graduates to remember to serve society after accomplishing their dreams.

Mr. Gehlot also presented gold medals to meritorious students and doctoral degrees to research scholars. In all, 21,731 candidates were conferred various degrees during the convocation.

Registrar Yashpal Ksheersagar and Registrar (Evaluation) Krishnamurthy and deans of various streams were present.

Sujata Nagesh Jodalli (MA in Mass communication and Journalism), daughter of a waterman, emerged the golden girl at the convocation. A native of Sulikatti village in Kalghatgi taluk of Dharwad district, she aspires to be become a journalist.

D.V. Anuja (M.Sc Biochemistry), Lakshman Hanji (M.Sc Zoology) and Pavitra Gullannavar (Library and Information Science) bagged eight gold medals each. Akkamma Yadwad (MA Kannada) bagged seven gold medals. Megha Havanagi (MA Political Science), Swathi Joshi (M.Sc Maths) and Sahana Nagesh Shet (M.Sc Chemistry) secured six each.

Medini Nayak (MA Eng), Tejaswini Talawar (MA Eco), Namrata Uday Shetty (M.Sc Botany), Rahela Anjum (MBA) and Akhila B.S. (LL.B) secured five each followed by Jyoti Goravar (BA), Sudha Malladad (BA), Maria Simon (B.Ed), Ashiwni Hullur (M.Com), Sayeda Rubab Peerazade (M.Sc Microbiology),Sneha Vijay Jadhav (M.Sc Geology), Rajshekhar Benakanahalli (M.Sc Geography), Durrea Quazi (M.Ed), Gouramma Bammakkanavar (MA Sanskrit) and Chandrashekharayya Hiremath (MA Philosophy) won four gold medals each.

Original post:
India has strong foundation for science and technology: Salunke - The Hindu

Three scientists with exceptional promise and novel approaches to fighting cancer have been named the 2022 recipients of the Damon Runyon Physician-Scientist Training Award. The awardees were selected through a highly competitive and rigorous process by a scientific committee comprised of leading cancer researchers who are themselves physician-scientists.

Physician-scientists are uniquely positioned to translate scientific discoveries into therapies that improve and prolong the lives of their patients. However, this vital cadre of cancer researchers is declining at a time when cancer research holds the greatest promise of new discovery. To help increase the number of physician-scientists, the Foundation created the Damon Runyon Physician-Scientist Training Award, which provides physicians who have completed clinical specialty fellowship training the opportunity to gain the skills and experience needed to become leaders in translational and clinical research.

Damon Runyon seeks to address the financial disincentives that often deter physicians from pursuing a research career by providing considerably higher funding than most research fellowships$100,000 in the first year, with increases of $10,000 per year over the next three years. It will also retire up to $100,000 of any medical school debt still owed by an award recipient. (The average medical school debt now exceeds $200,000.)

Since its launch in 2015, the program has funded 35 new physician-scientists from across a range of disciplines. Their research has not only brought forth insights into how cancer develops and spreads but also led to the development of new therapies, including several in clinical trials. Physician-scientists require protected time and funding for research, so that they can bring crucial insights from the clinic to the laboratory, and vice versa, said Yung S. Lie, PhD, President and Chief Executive Officer of Damon Runyon. Each of our physician-scientists is pursuing research with high potential for impact, and we are proud to enable this critical work.

The Physician-Scientist Training Award was established thanks to the generosity of Damon Runyon Board members Leon Cooperman and Michael Gordon.

2022 Damon Runyon Physician-Scientist Training Award Recipients:

Wallace A. Bourgeois, MD, with mentor Scott A. Armstrong, MD, PhD, at Dana-Farber Cancer Institute, Boston

Acute myeloid leukemia (AML) is an aggressive blood cancer that affects children and adults. One particularly difficult-to-treat subtype of AML that represents about 10% of all cases is characterized by a mutation in the KMT2A gene. Menin inhibitors (MI), a novel targeted therapy, have shown promise against this subtype in early clinical trials. Studies have also shown that compounds that degrade a protein called Ikaros can dramatically enhance the efficacy of MI. In seeking to uncover why MI and Ikaros protein degraders work well together, Dr. Bourgeois and his colleagues have found that both drugs target gene expression programs that are critical for the survival of KMT2A-mutant AML cells. Dr. Bourgeois is now working to better understand which genes can be targeted to further enhance the efficacy of Ikaros protein degraders in KMT2A-mutant AML. This work will shed light on the essential gene expression programs required for KMT2A-mutant AML cell survival, and ideally help guide drug development that specifically targets this subtype.

Mark B. Leick, MD, with mentor Marcela V. Maus, MD, PhD, at Massachusetts General Hospital, BostonThe Mark Foundation for Cancer Research Physician-Scientist

Acute myeloid leukemia (AML) is the most common acute leukemia in adults. Intensive chemotherapy cures only a subset of patients, and immunotherapy has had limited success in AML. One novel approach is chimeric antigen receptor (CAR) T cell therapy, which involves genetically engineering a patients own immune cells to target cancer cells. The difficulty with this approach is that the majority of available targets present on AML cells also reside on many normal cells. Based on emerging data demonstrating overexpression of the gene CD70 in AML cells compared to normal tissues, Dr. Leick and his colleagues have recently optimized a CD70-targeted CAR T therapy and demonstrated its efficacy in AML. Despite the superiority of this CAR over prior versions, however, it is less effective against AML cells that present a low amount of the antigen. Dr. Leick is now working to improve this CAR through genetic modification and/or a dual targeting approach. His work has the potential to generate a safe, highly potent, optimized strategy for treating this leukemia.

Mira A. Patel, MD, with mentor Sohail F. Tavazoie, MD, PhD, at The Rockefeller University, New York

One of the leading causes of death from cancer is metastasis, or when cancer spreads from its original tissue to other parts of the body. A gene that all humans carry, called Apolipoprotein E (APOE), plays a role in how our bodies respond to cancer, including risk of metastasis. The gene comes in one of three forms: APOE2, APOE3, or APOE4. Individuals who carry APOE2 tend to fare worse when diagnosed with melanoma, while those who carry APOE4 tend to have a much lower risk of melanoma metastasis and a much better chance of survival. (Those who carry APOE3 fall somewhere in between.) Dr. Patel is researching how APOE expression in immune cells either promotes cancer targeting, as in the case of APOE4, or cancer cell survival, as in the case of APOE2. With a better understanding of how the APOE gene affects the body's response to cancer, she hopes to improve cancer therapy by tailoring treatment to the form of APOE each patient carries.

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About the Damon Runyon Cancer Research Foundation

To accelerate breakthroughs, the Damon Runyon Cancer Research Foundation provides today's best young scientists with funding to pursue innovative research. The Foundation has gained worldwide prominence in cancer research by identifying outstanding researchers and physician-scientists. Thirteen scientists supported by the Foundation have received the Nobel Prize, and others are heads of cancer centers and leaders of renowned research programs. Each of its award programs is extremely competitive, with less than 10% of applications funded. Since our founding in 1946, in partnership with donors across the nation, the Damon Runyon Cancer Research Foundation has invested nearly $420 million and funded over 3,900 scientists. Last year, it committed over $18 million in new awards to brilliant young investigators.

100% of all donations to the Foundation are used to support scientific research. Administrative and fundraising costs are paid with revenue from theDamon Runyon Broadway TicketsService and our endowment.

For more information visitdamonrunyon.org.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

Excerpt from:
Damon Runyon Cancer Research Foundation announces three recipients of 2022 Physician-Scientist Training Award - EurekAlert

Respiratory syncytial virus (RSV) is a common and potentially dangerous virus for which no vaccine currently exists despite decades of effort from the scientific community. Associate Professor of Biomolecular Engineering at the Baskin School of Engineering Rebecca DuBois has set out to address this pressing need. To fund her innovative approach to the development of an RSV vaccine, DuBois has been awarded the prestigious and highly competitive National Institutes of Health Research Project Grant (RO1).

RSV causes contagious cold-like symptoms that can develop into serious lung problems and lead to hospitalizations, especially in young children and older populations each year, 3 million children under five years old are hospitalized from RSV and 64 million total people are affected worldwide.The five-year, $3.8 million grant will be shared with DuBoiss collaborator Ralph Tripp at the University of Georgia and will build on both researchers' years of work studying RSV. Their overall aim is to validate their RSV vaccine in pre-clinical trials.

Theres a huge need this is a really important gap in our vaccine schedule to protect children, said DuBois, whose experience with her childs severe RSV fueled her to take on this research. I think the NIH study section reviewers liked that it's a totally different strategy than what everyone else is taking.

DuBoiss lab focuses on bioengineering the commonly overlooked RSV G protein, used by the virus to attach to host cells, to expose its vulnerable points so the hosts body can fight back.

In previous work, they have found that a region, called the central conserved domain and just 40 amino acids long, can be engineered to evoke an protective immunogenic response from the host. Additionally, a recent paper from the DuBois lab determined that this altered protein is still recognized by the bodys immune system and therefore could be effective in a vaccine.

I think since RSV has been such a difficult virus to create a vaccine for, we're innovative in that we are using structural biology to learn more about this protein and make changes to it using protein engineering to improve its immunogenicity, said Maria Juarez, a third-year Ph.D. student in the DuBois lab. Thats something that our lab has really spearheaded.

Targeting this specific region of the G protein, which remains unchanged as the virus mutates, is a cutting-edge technique in vaccine development that may allow a vaccine to continue its effectiveness as the virus mutates.

By whittling down our vaccine to this important and conserved part, and designing it so [the antigen] is exposed to our immune system in a better way, we can refocus the antibody response it wont get distracted by all the parts that arent conserved, DuBois said. Its a more strategic way to do vaccine design, instead of just targeting the whole protein and choosing one strain to target.

Juarez and the others in the DuBois lab will continue to experiment with ways to ensure that the surface of their engineered protein is structured in a way to provoke the strongest immune response. Juarez also noted that the techniques she is using to engineer the protein are cheaper and less time intensive than other methods, making future production of the vaccine scalable so it can eventually be used commercially around the world.

Once DuBoiss group has developed their vaccine, they will send it to Tripps lab to test if it creates a strong antibody response in pre-clinical models. The group expects the first vaccines to be tested in pre-clinical models by the end of 2022.

This project differs from other RSV vaccine efforts, some of which are in phase III clinical trials, in the method it uses to evoke a protective immune response. The large majority of other researchers focus on the RSV F protein, which fuses the virus and host cell membranes together to get the viruss genetic information into cells.

Eventually, the researchers anticipate that their vaccine could be combined with one that uses the F protein in order to create an even more robust immune response.

See the rest here:
Prestigious $3.8M NIH grant awarded to biomolecular engineering professor to develop an RSV vaccine - University of California, Santa Cruz

Gene therapy deals

As a quick reminder, gene therapy involves the transfer of genetic material, usually in a carrier or vector, into the appropriate cells for uptake of the genetic material. The vector can either be delivered outside the body (ex vivo treatment) where the targeted cells are removed from the patient and administered to cells before they are returned to the patient, or the vectors can be injected into the body (in vivo treatment).

In March this year, Novartis entered into an agreement for Voyager Therapeutics TRACER adeno-associated virus (AAV) capsids to use in potential gene therapies for three neurological diseases. Under the agreement, Novartis will have the right to evaluate novel capsids from the RNA-driven TRACER platform and to exercise options to license capsids for exclusive use in its development of AAV gene therapies directed to three targets. Novartis also has the right to license capsids for two additional disease targets under the same terms. Voyager Therapeutics TRACER screening platform is identifying improved vectors selected to target desired cells and tissues with greater specificity, at lower doses, and with reduced risk of off-target effects compared to conventional AAVs. Initial data demonstrates TRACER-derived capsids have enhanced tropism for the central nervous system.

In February 2022, Takeda signed a deal with Code Biotherapeutics, potentially worth up to US$2 billion, to develop gene therapies for rare diseases. The partnership will make use of Code Biotherapeutics 3DNA non-viral, synthetic, genetic medicine delivery platform for a liver-directed programme, and also involves conducting additional studies for programmes directed to the central nervous system. 3DNA leverages targeting molecules (such as peptides, antibodies, and small molecules) which bind to cell surface proteins expressed on target cells. After binding to the target cell, the 3DNA is internalised into the cell via a receptor-mediated process. When utilised to deliver gene therapy, the gene construct then localises to the cell nucleus where a tissue specific promoter drives the expression of the desired protein.

PerkinElmer, Inc. announced that its SIRION Biotech business, experienced in gene and cell therapy and vaccine development, and the Centre for Genomic Regulation, an international biomedical research centre located in Barcelona, entered into an agreement to jointly develop new generation AAV vectors for type 1 and type 2 diabetes gene therapy in the pancreas. The end goal is to develop AAV vectors that target specific pancreatic cell types and contain payloads that express therapeutic genes under control of cell-specific regulatory elements. This new approach aims to increase the precision, safety, and efficacy of future AAV-based gene therapies for diabetes.

Novartis has entered into a definitive agreement to acquire all of the outstanding share capital of Gyroscope Therapeutics for US$800 million upfront, gaining the experimental AAV2-based gene therapy GT005. This one-time treatment, which is delivered under the retina, is currently in mid-stage development for patients with geographic atrophy (GA). GA is a chronic, progressive degeneration of the macula and leads to permanent loss of vision.

BioMarin Pharmaceutical Inc. and Skyline Therapeutics announced a multi-year global strategic collaboration for the discovery, development, and commercialisation of AAV gene therapies to treat genetic cardiovascular diseases. The partnership will leverage Skyline Therapeutics AAV gene therapy platform to develop innovative gene therapies, with a focus on genetic dilated cardiomyopathies, a group of progressively advancing, devastating diseases where the ventricles of the heart dilate and cannot efficiently pump blood. There are currently no targeted treatment options.

Astellas Pharma Inc is teaming up with Dyno Therapeutics to develop next-generation AAV vectors for gene therapy directed to skeletal and cardiac muscle. The partnership will rely on Dyno Therapeutics AI-based CapsidMap platform to create AAV capsids with optimised tissue targeting and immune-evading properties, while also improving packaging capacity and manufacturability. Under the agreed terms, Dyno Therapeutics will design novel AAV capsids with improved functional properties, while Astellas Pharma Inc will oversee preclinical, clinical, and commercialisation activities, including manufacturing, of gene therapy candidates using the capsids.

Spark Therapeutics, a member of the Roche Group, and CombiGene announced the signing of an exclusive collaboration and licensing agreement for CombiGenes CG01 project, a gene therapy which aims to treat drug resistant focal epilepsy.

The agreement provides Spark Therapeutics with an exclusive, worldwide licence to develop, manufacture, and commercialise CG01. CombiGene will continue to execute certain aspects of the preclinical programme in collaboration with Spark Therapeutics.

Epilepsy is a major global medical problem and CG01 is a unique gene therapy candidate aimed at this large patient population.

In February of this year, it was reported that the NHS had struck a confidential deal for what is thought to be the most expensive drug developed to date. Orchard Therapeutics gene therapy Libmeldy is used to treat an extremely rare condition, metachromatic leukodystrophy (MLD), which causes severe damage to a childs nervous system and organs. Around five babies are born with the disorder in England every year.

MLD is a rare inherited disorder in which there is a mutation in a gene needed to make an enzyme called arylsulfatase A (ARSA), which breaks down substances called sulfatides. As a result, sulfatides build up and damage the nervous system and other organs, causing symptoms such as walking difficulties, gradual mental deterioration, and eventual death.

The active substance in Libmeldy is stem cells (CD34+ cells), derived from the patients own bone marrow or blood, that have been modified via a lentiviral vector to contain a copy of the gene to make ARSA and can then divide to produce other sorts of blood cells. The one-off treatment has a list price of 2.8 million but can be offered on the NHS after the health service negotiated a confidential discount.

LEXEO Therapeutics, a clinical-stage gene therapy company advancing a diverse pipeline of AAV-based gene therapy candidates for genetically defined cardiovascular and central nervous system diseases, announced that the United States Food and Drug Administration (FDA) cleared its Investigational New Drug (IND) application for LX2006. LX2006 is an AAV-based gene therapy candidate designed to intravenously deliver a functional frataxin gene for the treatment of Friedreichs ataxia cardiomyopathy via restoration of mitochondrial function in myocardial cells.

uniQure announced the dosing of the first two patients in its European clinical trial of AMT-130, a potential one-time gene therapy approach for the treatment of Huntingtons disease. The clinical trial is taking place at several sites in Poland, the UK, and Germany. AMT-130 is uniQures first central nervous system focused gene therapy product consisting of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating devastating Huntingtons disease.

SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of genetically defined neurological conditions, announced that its IND application for its lead candidate, SBT101 for the treatment of adrenomyeloneuropathy (AMN), was cleared by the FDA. SBT101 is the first AAV-based gene therapy in development specifically designed for people living with AMN, an adult-onset degenerative spinal cord disease caused by mutations in the ABCD1 gene.

Chimeric antigen receptor (CAR) T-cell therapy works by genetically engineering an individuals T-cells to recognise and destroy cancer cells.

In February 2022, it was reported in Nature that two of the first patients to be treated with such therapy still possess cancer-killing cells a decade later with no sign of their illness returning. Both patients were suffering from chronic lymphocytic leukaemia when they had CAR T-cell therapy in 2010, and achieved complete remission within months of treatment, meaning all signs and symptoms of their leukaemia disappeared. When the researchers examined the patients blood, they could still identify CAR T-cells that were capable of proliferating and killing cancer cells. This promising finding suggests CAR T-cell therapy may constitute a cure for certain blood cancers.

Visit link:
Venner Shipley - cell and gene therapy industry news update - Cambridge Network

By Codrin Paul OneciJun. 9, 2022

Im an MIT AeroAstro student, and I look at the sky daily. This is relaxing, bringing optimism and motivation: I know that I can predict various phenomena in the universe and put the forces of nature to humanitys use, to the best of my ability. I like to observe the physical interactions in our surrounding environment, because careful consideration of the processes within it allows one to clearly foresee world states and trajectories and make higher-value decisions in most situations.

Our world has many interesting and observable patterns and tendencies. Among them I consider the evolution of life-forms and their habitats to be of paramount importance, especially in the context of global warming. One essential aspect that apparently eludes the reasoning of many is the physical space that the biosphere exists in: this is the main constraint dictating the resources available for life to persist, evolve, and expand, of which matter composition, thermodynamic conditions, and energy fluctuations are the most important.

When addressing global warming, in recent years, many decision-makers have tended to focus only on bandaging the Earth. While the development of new technologies that would allow us to pollute less is definitely economically feasible and beneficial for the planet, instead, politicians often support anti-industrial social movements, allowing these movements to gain momentum and popularity. Think of the anti-nuclear energy movement in Germany, or movements to reduce industrial production or extractive industry activity in the U.S. (hurting businesses and workers). Think of public opinion on space exploration and colonization, that they are too expensive and too far in the realm of science fiction to be even remotely feasible. These are naive perspectives not supported by scientific fact and are usually promoted by those with a narrow vision of the world.

Obviously I consider the idea of reducing our industrial activities overall without starting new ones to be absurd. Considering economical aspects, even social tension phenomena would be generated through taking uncalculated decisions. From my perspective, the global development of fission and fusion plants in addition to research in the field of nuclear energy are essential to our sustainable evolution as a highly technological civilization. High energy levels and rates are requirements for traveling throughout the solar system, processing matter, and ultimately either terraforming celestial objects or creating purely antropic environments (artificial bodies). These energy levels and rates are attainable with the strong nuclear force.

At the level of the social conscience, by following lines of thought without making use of reason, we are physically limiting not only our horizons, but that of other living beings. We also have to take care of a biosphere often neglected by most members of the human species. The reality is that we are the highest on the trophic chain and have the most developed conscience. Science gathers knowledge much faster than humanity gains wisdom.

Polluting an entire planet, causing a mass extinction, and not taking serious action towards finding a new home in space dont seem reasonable. Actions are being taken in many highly developed countries to protect the planet through technological modernization of industry. However, in order to maintain an equilibrium between the development of our different fields of knowledge as a species, we must also take action in other scientific fields including, but not limited to, space exploration, human habitat extension onto the moon and Mars, and genetic engineering for accelerated evolution and disease prevention.

Moreover, without guidance at a high level of society, there is plenty of room after the pandemic for the youth to find escape through libertinage, hedonism, political violence, and many other things. Values in each nation are projected by prominent individuals, so when the dominant classes are not setting reasonable ideals for society, the social organism may end up evolving slowly, chaotically, or even backwards.

If you are a true leader, you must provide a sense of purpose and meaning to others. Otherwise, you are just a demagogue using cheap rhetoric and immoral acts for the purpose of maintaining your position of power.

I prefer societal lines of evolution in which we are looking at and reaching for the sky with the purpose of expanding our habitat and that of Earths biosphere while understanding what in the past would have been deemed secrets of the universe. This idea is well-documented at a hypothetical level in science fiction books such as those by Issac Asimov or Frank Herbert.

Wasn't it always our dream to fly? If you look into the past, the gods from our mythologies were navigating the seas of the skies and heavens. Whether youre religious or atheist, looking at the sun, moon, stars, and beyond gives you a sense of power and intimacy with nature itself. This was felt even by our prehistoric ancestors who used the suns position relative to stars to calculate agricultural cycles. We should especially feel this intimacy today as humans prepare to occupy the near solar system. Only nations capable of highly organized and scientifically founded planning of their collective efforts will be true leaders at global level: they will become the most influential at the interplanetary level, silently leaving others somewhere behind in history.

Preparation for space exploration is motivating for some individuals, including myself. We are lucky: technologically, we already have the resources and ability to colonize space. We have the technology to transport materials for colonies on the moon and to bring equipment to Mars for a self-sustaining city, with enough people eager to be pioneers and explore these new frontiers. We have the materials, the software and hardware. It is mostly a lack of will that separates us from the vision described by Elon Musk as comparable to the fleet of one thousand ships sent to the shores of Ilion, under the leadership of Agamemnon. If no action is taken to fix the lack of willpower, especially from political leaders, we will likely enter a stagnant phase in our evolution as a civilization. Insufficient resources on an overpopulated planet will lead to economic tension and conflicts.

We are not confined to a single planet. We are the generation of the space age, and we have great chances to make life multiplanetary.

What I and others ask our leaders to do is to support high-caliber projects that will mobilize all of humanity in a collaborative effort. We think of individuals like President John F. Kennedy, who started the most demanding space program on the planet and said clearly that we choose to go to the Moon in this decade and do the other things, not because they are easy, but because they are hard, because that goal will serve to organize and measure the best of our energies and skills, because that challenge is one that we are willing to accept, one we are unwilling to postpone, and one which we intend to win, and the others, too. Sadly enough, these visionary leaders often have fates similar to that of Prometheus. Regardless, we must look into the future with hope and be determined in using our strength to win.

We already have gifted people across the planet willing to help scatter humanity across the universe, we have much of the technology developed, and we definitely have the economic basis to support this endeavor. It will be just a matter of ratios of various means and forces across the planet whether we achieve a higher level of social conscience and global collaboration. Knowledge and reason are the foundation of our strength.

Codrin Paul Oneci 21 is a first-year Master of Science student in AeroAstro.

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Expanding our horizons through nuclear energy and space exploration - The Tech

In the few years that Lalitpurs Windhorse Gallery has been operational, it has earned a reputation for providing little-known artists from Himalayan communities its space to showcase their works. The most recent artist to exhibit works at the gallery is Tenzin Gyurmey Dorjee, a second-generation Tibetan refugee based in India. Since May 27, the gallery has been exhibiting Dorjees 'I Shouldn't Be Here', which features intimate personal portraits of the Tibetan diaspora.

Some of the factors that make Dorjee's artworks unique are the material used and his art-making process. In many of the exhibitions artworks, Dorjee uses Drochak-bhureh (tarp sacks) as a canvas. The decision to use sacks echoes the Tibetan diasporas experience of resettlement. In the early years of life in exile, relief materials to the Tibetan communities in India and Nepal came packed in tarp sacks. His art-making process is meticulous and meditative and is different from how artists usually fill colours in their works. In many of his works, Dorjee transfers colours to his canvas instead of applying paint directly.

The first painting that greets visitors is the Silent Playground, which unfolds on top of a collage of notebook papers belonging to a student from a Tibetan school. As the name suggests, the artwork depicts a scene of children playing in a playground. If one observes the artwork carefully, one can almost hear crackles of laughter ringing in the air. The painting also has a lot of small details that lend the artwork a touch of Tibetan-ness. In the middle of the painting is a playful little girl wearing a photo of the Dalai Lama around her neck, a common practice among Tibetan Buddhists to show their devotion to their spiritual leader. A little above the playful girl is a childish drawing of an oddly-shaped bus with the word Tibet written across it, perhaps symbolising that the bus is heading to Tibet. At the bottom end of the artwork are answers written by a Tibetan kid to questions about his country, origin, love, and religion. In his childish handwriting, the boy responds as honestly as he canhe identifies Tibet as his country of origin but his birth country as India. For love, he writes down a long list that includes pala (which translates to father in Tibetan), amala (mother in Tibetan), world, country, and Payal, his Indian friend. At the bottom left corner of the painting are childish drawings of what is supposed to be a gold aeroplane and a turquoise car, both of which are mentioned in a popular Tibetan song that pays tribute to the Dalai Lama.

The exhibition features several such artworks that allow viewers to go on a heartwarming (oftentimes heartaching) journey to see what life is like for Tibetan refugees and the nuances of their struggles in attempting to find some semblance of normal life far from their country Tibet. But Dorjees works are not fishing for sympathy for the Tibetans. Instead, it wants viewers to see the Tibetan communitys resilience and humility and the great lengths they have gone to adapt to a life in exile and keep their cultures and traditions alive.

While India may be home to the largest Tibetan diaspora, Nepals Tibetan refugees make up the countrys largest refugee community. Nepal has been hosting Tibetan refugees since the late 1950s, and it is estimated more than 15,000 Tibetan refugees continue to live in the country. This is why exhibiting I Shouldnt Be Here in Nepal makes sense because it serves as a window for Nepalis to better understand what life is like for Nepals Tibetan diaspora.

Most of these works are part of my memories. I wanted to celebrate how the Tibetan diaspora has held intact our resilience and humility even after all these years since leaving Tibet, says Dorjee on a recent afternoon at the gallery.

Even though the artworks might be deeply personal to Dorjee, some of the subject matters they explore are familiar and universal.

One such universal artwork is Mood Swing. The work is set inside a bathroom and features Dorjees sister wearing costumes of DC and Disney characters. The painting shows his sister making comical facial expressions and just being herself and confronting her moods. The artwork delves into the identity we all try to make for ourselves in a world that keeps demanding that we be something else. Dorjee says that the idea for the artwork came to him while trying to cheer his sister after she lost her job.

I made this painting to make my sister smile and convince her not to worry too much about what the world might think about her. I wanted her to keep her child-like nature intact and not be too hard on herself, he says.

In his artwork titled The Sky is Falling, the central character is his father, and the work is Dorjees attempt to explore the importance his father places on compassion. The painting shows Dorjees father chasing away a cat that is only doing what is natural to him: trying to kill a bird.

My father is a very compassionate person. He cant bear the sight of any living being suffering and is always trying to save everyone, which I dont think is humanly possible, says Dorjee.

There are a few other paintings in the exhibition that explore such universal themes, but the subject of the majority of the artworks in the exhibition revolves around the experiences of the Tibetan diaspora. His artwork titled Champions wanders into that very subject and does so in a way that makes it the most moving piece in the exhibition.

The artwork features a Tibetan-Indian athlete standing on a podium and raising the Indian flag to mark her victory, perhaps in a national competition. Instead of being surrounded by audiences cheering for her victory, she is alone and enclosed by a wall, and you can almost hear the heart-aching silence lingering in the air.

Dorjee explains that the artwork was inspired by the experiences of Tibetan-Indian wushu and a mixed martial arts athlete who has represented India in international martial arts competitions.

Circumstances have forced many Tibetans in India to take up Indian citizenship. A few of them have even represented India on international platforms and made their adopted country proud, but despite their achievements, they are still considered outsiders and do not get the same recognition, says Dorjee.

Born and raised in Indias Himachal Pradesh, Dorjee, like many Tibetan refugee children in India, attended a Tibetan community school. Dorjee says that he developed an appreciation for art very early on in life.

My father often makes free-hand sketches, and he is very good at it. As a young boy, I would make sketches as well. As far as I can remember, I have always been this person who enjoyed creating art and collecting them, says Dorjee.

But it was much later in life that Dorjee decided to become a full-time artist. After graduating from high school, he had his mind set on becoming a genetic engineer. When he failed to get into his desired genetic engineering college, his sister suggested that he take a gap year and try getting into the college the following year.

During this gap year, Dorjee started drawing again and fell in love with the process. The following year, he joined College of Art, Delhi and graduated in 2012. For the next few years, the artworks Dorjee made dealt with darker stylistics.

It took me a few years to realise that people dont want to spend their hard-earned money on artworks that dealt with dark subject matters, says Dorjee. To sustain myself financially, I started making digital art.

But the journey to becoming the artist that Dorjee has become today started when he met Nepali contemporary artist Ang Tsherin Sherpa in 2017. His interaction with Sherpa, says Dorjee, changed how he looked at art.

When I met Ang Tsherin-la for the first time, I had just graduated from art school. When I met him again in 2017, I was on the verge of giving up art for good. During that meeting, he bought one of my paintings, and something within me shifted, says Dorjee. You see, it was the first time someone had truly valued my work. And that was enough for me to rethink my decision to give up art. I will forever be grateful to him for having faith in me.

Sherpa still remembers the reserved Dorjee who assisted him during a residency programme in 2012.

To be honest, I didnt really like his works at first. They felt dark, and maybe it was because he was in that space at the time, shares Sherpa in a phone interview.

But through the years, Sherpa says that Dorjee has grown as an artist and become more committed to the idea of becoming an artist.

With this exhibition, he brings to the audience stories from the Tibetan diaspora that we rarely see represented in art. And his work feels universal as we all are moving around so much. We all can relate to the diaspora experience, Sherpa tells the Post.

The exhibition, says Dorjee, has made him realise the important role art can play in telling stories and highlighting issues.

I have interacted with a few local Tibetans here at the gallery, and they have thanked me for making them feel seen and heard. It also surprised me that many Nepali art lovers that I have met here at the gallery told me that they had no idea that Tibetan refugees even exist in Nepal, says Dorjee. It is beginning to dawn on me my responsibility as an artist to serve my community, and I am really looking forward to the journey ahead.

I Shouldnt Be Here is on display until June 30 at Wind Horse Gallery, Bhanimandal, Lalitpur.

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Portraits from the Tibetan diaspora - The Kathmandu Post

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Credit: Mary Ann Liebert, Inc., publishers

In response to the historical devastation of mass shootings in the United States, including the recent mass shooting of 19 innocent children in Uvalde, TX, Violence and GenderEditor-in-ChiefMary Ellen O'Toole, PhDhas issued an immediate call to action for superior gun control laws and legislation.

Weve waited long enough, says Dr. OToole, Editor-in-Chief. This political football must stop. Our children are being killed and the laws must be changed now. We can no longer normalize these behaviors or expect our children to be the victims on the front lines. The research is clear and we must continue to stay educated, relentless, and vigilant in our quest for the future of our country.

In support of Dr. OTooles mission, Violence and Gender is providing free access to the following special issues spanning themes including gun ownership, beliefs about gun control and gun ownership, gun policy, among others.

Special Issue on Gun Violence: Part I

Special Issue on Gun Violence: Part II

Gun violence against our families and our children must be addressed aggressively, echoes Mary Ann Liebert, Publisher of Violence and Gender and president and CEO of the company that bears her name. The current recommendations for gun control reformation are pitiful given the extent of the loss of lives weve seen in schools. Legislators, policy makers, educators, and civilians who refuse to acknowledge the extent of the problem of gun violence should subscribe to Violence and Gender and educate themselves by reading the published, academic research on systematic gun violence in our country. The research is clear and journals such as Violence and Gender couldnt be more important to support real and lasting change. We must remain educated and informed to collectively make the best decisions for our families and our future.

Emphasizing this call to action, Dr. Anna Satterfield, Deputy Editor adds: The US Centers for Disease Control and Prevention (CDC) reported that in the United States in 2020, there were 45,222 firearm-related deaths of which 54 percent were suicides and 43 percent were homicides.Despite various versions of enacted laws to reduce gun violence, laws and regulations continue to miss the mark of what we understand about those that use firearms as weapons.Until we actively enforce a balance between effective gun control laws and effective gun violence prevention/intervention and risk-reduction programs, the United States will remain an outlier in firearm-related deaths compared to other countries.

About the Journal

Violence and Genderis the only peer-reviewed journal focusing on the understanding, prediction, and prevention of acts of violence. Through research papers, roundtable discussions, case studies, and other original content, the Journal critically examines biological, genetic, behavioral, psychological, racial, ethnic, and cultural factors as they relate to the gender of perpetrators of violence. Led by Editor-in-Chief Mary Ellen O'Toole, PhD, Forensic Behavioral Consultant and Senior FBI Profiler/Criminal Investigative Analyst (ret.), Violence and Gender explores the difficult issues that are vital to threat assessment and prevention of the epidemic of violence. Violence and Gender is published quarterly online with Open Access options and in print. Complete tables of content and a sample issue may be viewed on theViolence and Genderwebsite.

About the Publisher

Mary Ann Liebert, Inc., publishersis a privately held, fully integrated media company known for establishing authoritative medical and biomedical peer-reviewed journals, including Cyberpsychology, Behavior, and Social Networking, Journal of Child and Adolescent Psychopharmacology, and Journal of Women's Health. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms more than 100 journals, newsmagazines, and books is available on theMary Ann Liebert, Inc., publisherswebsite.

Violence and Gender

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An immediate call to action from the editorial leadership of Violence and Gender - EurekAlert

Elizabeth II has been on the throne for 70 years, as I am sure you have heard. She is 96 years old. Theres no delicate way of putting this, so let us be blunt: she will not reign over us for much longer. She will be succeeded by a man who has proudly announced his readiness to break the conventions controlling the behaviour of the head of state. Because there is no prospect of parliament jumping a generation and passing the crown to his son, no one can stop the forward march of Charles III taking the throne.

The design flaw in all systems of hereditary power is that they eventually throw up a duffer. Monarchy indiscriminately admits every species of character to the same authority Thomas Paine wrote in 1791. The reign of Charles III will be such a neurotic experience because we will have a monarch who doesnt accept that his authority has nothing to do with his ability and everything to do with an accident of birth.

Elizabeth IIs modesty has made many support what Helen Mirren called queenism rather than monarchism, and wish we could have a queen without the rest of the royal family.

She does her job and stays out of politics. In the 20th century, there were good reasons to behave with restraint. Elizabeth II only came to the throne because parliament had, in effect, deposed her uncle, Edward VIII. The House of Windsor survived, but all around it war and revolution had destroyed the Habsburgs, Romanovs and Hohenzollerns. Caution, as much as personal preference, demanded that she be careful.

Times change and aristocrats are no longer frightened. There will be no sense of caution about Charles III: only a sense of entitlement. Without self-consciousness, he denounced young people with ideas above their station in 2003. What is wrong with everyone nowadays? Why do they all seem to think they are qualified to do things far beyond their technical capabilities? People seem to think they can all be pop stars, high court judges, brilliant TV personalities or infinitely more competent heads of state without ever putting in the necessary work or having natural ability. This is the result of social utopianism which believes humanity can be genetically and socially engineered to contradict the lessons of history.

He showed no awareness that he was the beneficiary of, if not genetic engineering, for any half-competent engineer could produce a better product, a genetic fluke. In his mind he will be a self-made monarch who will succeed to the throne on merit rather than by luck.

The first decade of the 21st century saw what we used to call the establishment begin to realise that Charles was a hard prince to house train. Mark Bolland, a former courtier, said he routinely meddled in political issues and wrote sometimes in extreme terms to ministers, MPs and others in positions of political power. Aides to the then Labour administration said that if he carried on opposing government policy sooner or later there will be real constitutional trouble.

Heirs to the throne are often in conflict with monarchs because there is little else for them to do than hang around waiting for the king or queen to die. The Queen doesnt moan. Her son does. The Queen doesnt politick. He cant help himself. You could, if not forgive, then at least understand Prince Charless behaviour when he was decades away from getting a proper job. He had to pass the time, after all. The excuse doesnt wash today, as there is no evidence that he has calmed down now that his coronation is in sight.

Like their counterparts in politics, the courtier journalists who surround royalty have picked a degraded way to earn a living. I read their books out of duty rather than pleasure because I know there will be nuggets of truth in the slurry. To maintain access, they must be faithful transcribers of their masters unintentionally revealing musings. The story they bring from Clarence House is of a presumptuous prince, whose conviction that the rules dont apply to him leaves him closer to Boris Johnson than his mother.

Robert Jobsons all but officially endorsed biography from 2018 describes a future king who expects to lead as monarch, not just follow. One close source said that Charles III will want a seat at the table, not just to be briefed or rubber-stamping the decisions after they are taken. A raucously divided country, with a border in the Irish Sea and a separatist government in Scotland, will soon have a puffed-up monarch adding his demands to the unstable mix. Will elected politicians put him in his place? Can they? As Johnson has shown, the old conventions of public life are flimsy protections. Once narcissists are in power, they blow around like bin bags in the wind.

Greenish readers who believe that interventions from an ecological King Charles would be welcome should look at where his environmentalism comes from and where it leads. Charless widely unread Harmony: A New Way of Looking at the World is another book worth forcing yourself to plod through. It sets out an obscurantist vision that is so reactionary it opposes all aspects of modernity from the scientific revolution on. Hence his fondness for the dictatorial petro-monarchies of the Gulf. They may cause devastating environmental damage but at least they are free from the democratic constraints the Enlightenment put on European royals. Hence the belief in quack alternative medicines, the damage to health they bring notwithstanding.

His wide-eyed mysticism takes him far from the Anglicanism of his mother. One can only pity the archbishop of Canterbury when the next supreme governor of the Church of England explains how he has found the sacred geometry of the orbit of Mercury sits within the orbit of the Earth in such a proportion that it fits exactly over the pentagon at the heart of the five-pointed star.

When dominant prime ministers or CEOs retire after only a decade of achievement, their successors struggle to repeat their success. How much harder will it be to follow 70 years of a reign that even republicans concede has been an accomplished performance? The more so when an accident of birth has thrown up a silly, vain, zealous and fatally unself-conscious monarch, who, to use his own anti-meritocratic notions against him, doesnt know his place. In other words, the UK is heading for a smash-up. Aprs maam, le dluge.

Nick Cohen is an Observer columnist

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Unlike the Queen, King Charles will have no sense of caution, only of entitlement - The Guardian