For more than two decades, I have been working to improve several staple food crops in Africa, including bananas, plantains, cassavas and yams. As principal scientist and a plant biotechnologist at the International Institute for Tropical Agriculture in Nairobi, I aim to develop varieties that are resistant to pests and diseases such as bacterial wilt, Fusarium wilt (caused by the fungusF. oxysporum) and banana streak virus.

[Editors note: Abdullahi Tsanni is a freelance science journalist based in Abuja, Nigeria.]

In 2011, my team and I created a set of tools, the only one of its kind in Africa, for changing DNA sequences so that we could develop genetically modified and genome-edited products in sub-Saharan Africa. In 2018, we pioneered the first application of CRISPR gene-editing technology to deactivate banana streak virus in plantains. This technology overcame a major hurdle in banana breeding on the continent, and is the first reported successful use of genome editing to improve bananas.

Kenya imposed a partial lockdown on 7 April, but I was allowed to continue some crucial laboratory work, and our research is not affected. Since mid-May, my team has been working in shifts. These plants are like our babies in the lab I cant leave them.

I am excited to see the resultant performance of the bananas in our greenhouse. By next year, we should have some plants ready for trials in the field.

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CRISPR-edited bananas immune to killer pathogens advance toward commercialization in Africa - Genetic Literacy Project

Recommendation and review posted by Bethany Smith

Recently published a detailed report on the CRISPR Technology Market. This market research report was prepared after considering the COVID-19 impacts and monitoring the market for a minimum of five years. The report provides you with growing market opportunities, revenue drivers, challenges, pricing trends & factors, and future market assessments. Our research team has implemented a robust research methodology that includes SWOT analysis, Porters 5 Force analysis, and real-time analysis. Furthermore, they have conducted interviews with the industry experts to offer a report that helps the clients to formulate strategies accordingly.

TheGlobal CRISPR Technology Markethas outlined the supply and demand scenario in the industry and provided a detailed analysis of the product developments, technology advancements, and competitive analysis in the market. It offers an in-depth analysis and all the information required by the new entrants and emerging players to stay ahead in the competition. This report includes information on the latest government policies, norms, and regulations that have and can affect the dynamics of the market.

The historical and forecast information provided in the report span between 2019 and 2026. The report provides detailed volume analysis and region-wise market size analysis of the market.

Get A Sample Copy Of The Report Including An Analysis Of COVID-19 Impact: https://www.researchmoz.us/enquiry.php?type=S&repid=2673637A dedicated section for the prominent companies in the market which provides information on their revenue drivers, product innovation, and challenges they are facing during in the industry. This company profiling section includes industry players mergers, acquisitions, and collaborations which have helped them to leverage or impacted their market position. Besides this, the report is fragmented on the basis of the products, applications, and region-based analysis which imparts a holistic view and scope of the market.

The market research report also offers information on potential investment opportunities, strategic growth market analysis, and probable threats that will adhere to the client to systematically and creatively plan out the business models and strategies. The critical data analysis in the CRISPR Technology market report is laid out in an upright way. This means that the information is represented in form of info graphics, statistics, and uncomplicated graphs to make it an effortless and time-saving task for the client.

Major Companies in the Market: Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT), Horizon Discovery Group, Agilent Technologies, Cellecta, GeneCopoeia, New England Biolabs, Origene Technologies, Synthego Corporation, Toolgen, etc.

Global CRISPR Technology Market Segmentation

The CRISPR Technology market report is fragmented into product types, applications, and regional analysis. In this report, the product flow, distribution, and possible future innovations are bestowed in a detailed manner. It also provides accurate calculations for product sales in terms of volume and value. The applications of the products are discussed in a coherent way which includes potential future applications.

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Major applications/end-users industry are as follows:

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Impact of Covid-19 in CRISPR Technology Market:The utility-owned segment is mainly being driven by increasing financial incentives and regulatory supports from the governments globally. The current utility-owned CRISPR Technology Market are affected primarily by the COVID-19 pandemic. Most of the projects in China, the US, Germany, and South Korea are delayed, and the companies are facing short-term operational issues due to supply chain constraints and lack of site access due to the COVID-19 outbreak. Asia-Pacific is anticipated to get highly affected by the spread of the COVID-19 due to the effect of the pandemic in China, Japan, and India.

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CRISPR Technology Market Forecast To 2026 And COVID-19 Impact Analysis - The Think Curiouser

Recommendation and review posted by Bethany Smith

UF engineers reach semi-finals in XPRIZE Contest for new COVID-19 test methods; their CRISPR-ENHANCE methodology published in Nature Communications journal

XPRIZE Rapid COVID Testing competition teams are located around the globe.

According to a survey conducted in July 2020 (pdf) by researchers from Northeastern, Harvard, Rutgers, and Northwestern universities, testing (and especially testing asymptomatic individuals) is a cornerstone of containing the spread of COVID-19. The speed in producing reliable results is of the essence in controlling the spread of the virus.

Piyush Jain, Ph.D., Assistant Professor in the Department of Chemical Engineering at UF Herbert Wertheim College of Engineering, has been improving existing methods for testing COVID-19 samples from nasal swabs and saliva since the deployment of the original qPCR-based test in February 2020. His research has gained recognition by XPRIZE Foundation, Inc., a non-profit corporation fostering and sponsoring competitions to create innovative breakthroughs for the benefit of humanity.

Dr. Jain and his colleagues were selected from more than 700 teams around the globe as one of the 219 semi-finalists from 35 countries in the XPRIZE Rapid COVID Testing competition. The goal of XPRIZE Rapid COVID Testing is to develop innovative, scalable COVID-19 testing solutions that will radically change the world by providing much needed insights to help society safely reopen and not in some distant future, but right now.

Jain and his doctoral student Long Nguyen have developed an enhanced CRISPR/Cas (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated proteins) system that amplifies the detection of the SARS-CoV-2 virus during the test process. This unique system, CRISPR-ENHANCE, shortens the time it takes to run the test by half and provides increased sensitivity for the results.

The gold-standard CDC-recommended qPCR-based tests currently in use are a fluorescence-based assay that requires an expensive thermocycler that cycles between temperatures and measures fluorescence using a camera. In contrast, Jain and Nguyen have been working on a lab-based fluorescence assay as well as a paper strip-based assay that can potentially be used for home-based tests that can significantly reduce cost and time.

A version of our lab-based test in development uses only common lab equipment including a heater, vortex mixer, micro-centrifuge, pipettes, and a fluorescence plate reader, Jain said.

This high-throughput lab-based test using an automated liquid handler robot is designed to analyze about 190 samples at once in just over two hours. The Jain lab is automating the entire workflow to reduce time for sample processing. With automation, pooling of samples and multiple cycles of tests in parallel, a small diagnostic lab can analyze thousands of samples daily.

If used for a single sample analysis, Jains entire lab-based assay can be performed in about 45-60 minutes, with the results being read in the last 5-10 minutes. In contrast, the current CDC-recommended qPCR-based tests take a total of 110-120 minutes, with the result-reading portion requiring approximately 80 minutes on the expensive thermocycler. This occupancy time becomes crucial when it comes to testing thousands of samples.

The CRISPR-ENHANCE method is also much more rapid and sensitive than two other CRISPR methods now on the market as it uses an engineered version of CRISPR that enhances the speed of CRISPR, shortening the time to receiving results by 3.2-fold. Also, if the CRISPR-ENHANCE assay runs longer, it enhances the sensitivity by several fold. The increase in cost of reagents using CRISPR-ENHANCE vs. conventional CRISPR is within $0.01 per sample, according to Jain.

Regarding the SARS-CoV-2 RNA extraction procedure, which has to occur before the reading phase, Jain said, The CDC recommended RNA extraction/purification steps take 30-40 minutes of laborious work and use an expensive kit with supply chain issues. However, our CRISPR-ENHANCE test can potentially work with a heat-based method that employs a simple 10- to 17-minute heating step without the need for further manipulations. The cost of the extraction reagents is 80-times lower than the current CDC-approved method, with the added benefit of helping to minimize supply chain issues. We are using this approach in the XPRIZE competition.

Jains CRISPR-ENHANCE team has tested 157 blind samples supplied by the XPRIZE Foundation and has sent those results to the judges, who will score them for specificity, sensitivity and limits of detection. From these scores, the XPRIZE Foundation will announce 20 teams on December 8, 2020, as finalists who will then have the opportunity to gain clinical validation from two world-class laboratories based in the United States, helping to accelerate the FDA approval process for their tests.

XPRIZE Competition Timeline

The five top testing protocols selected by the judges will be documented in a playbook for global dissemination in order to aid testing sites around the world in a broad effort to end the pandemic.

Proposed paper-strip COVID-19 Test

Nature Communications, doi:10.1038/s41467-020-18615-1, has recently published a paper by Jain, Nguyen and recent graduate Brianna Smith (B.S., MAE 19) on the results of their research. The paper features the development of the CRISPR-ENHANCE technology and its use in COVID-19 detection, using both the lab-based fluorescence assay and the paper strip-based assay. This paper focuses on the use of synthetic genomic RNA in the testing samples.

Jain and Nguyen hope to have their validation data on human samples completed soon and are preparing a filing with the Food and Drug Administration (FDA) to have their method approved for Emergency Use Authorization (EUA).

Following FDA approval of the enhanced fluorescence-based lab assay, Jain and Nguyen hope that, by fine-tuning their paper-strip-based assay and combining it with cell phone camera, it could potentially result in a point-of-contact and home-based tests.

Read more here:
Faster, Cheaper, Easier COVID-19 Testing - Powering the New Engineer - University of Florida

Recommendation and review posted by Bethany Smith

Michael Heltzen, CEO and co-founder of Cardea Bio, a San Diego startup integrating tiny bits of biology into modern electronics, believes that the way researchers have been observing biological signals is about to change.

Our ability to see detail in space and time is limited by resolution. Take, for example, the noisiness of a persons image on a digital video camera. At a low signal-to-noise ratio, an image is indecipherable, but with increasing sensor quality the image becomes progressively recognizable as a face and then a specific individual. If we only have one frame to look at, the image will be static, but with consecutive images appearing on a display we can see the person blink or make a change in facial expression.

This rings as true for human sight as does for any analytical measurement, especially in the life sciences. For example, when we want to examine a 3D genome, we amplify the genome to have enough of it to get a reflection out of it by illuminating it with a laser. But this genome is not in its dynamic, natural stateit is fragmented and static. This genome has typically been run through a molecular meat grinder, ripped up into small fragments, boiled to make it fall apart, and amplified with artificial nucleotides. Its almost like using a hammer to probe a computer and smashing it up to study the pieces.

But creating these genomic snapshots does little to provide richer information of real-time biological interactions. Using optics to observe live biological signals means that we only have one narrow perspective on a whole orchestra of different biological signals, says Heltzen. We can listen into one vertical, either genomics or proteomics, and it will often limit the signals that we get instead of being able to interact with them.

A new gate to natures signals

In Roman mythology, Cardea was the goddess of door hinges and handles who prevented evil spirits from crossing thresholds. So, it is not surprising that at the core of Cardea Bios technology is their graphene-based, biology-gated transistorscalled Cardean Transistors. These transistors leverage graphene, a nanomaterial that, in contrast to the common semiconductor material silicon, is biocompatible and a near perfect conductor due to only being one atom thick. Graphenes conductive qualities can therefore provide interactive live-streams of biological signal analysis, replacing optical static observations.

Its a different way of thinking about life science research because were basically saying that all signals of biology are binding interactions, says Heltzen. Thats how information is transferred from one molecule to another. When we take transistors and make one site of that binding interaction as the gate, if the binding interaction is there, there will be a change of the gate. This basically flips life science thinking upside down because theres no optics. Theres no analyte type of conversation. Theres no laser. Theres no heat. Theres no data sets to be converted from optical signals.

Since Cardea Bios system is based on binding interactions, it can probe any combination of biologically based binding interactions.

For example, if you want to probe for a protein biomarker, you would exploit the unique capturing mechanism between an antibody to an antigen. In this scenario, the antibody would be linked to the gate in the biological transistorif the antigen and antibody bind, the gate closes, and, if not, the gate remains open. This ability yields a computer chip that, when introduced to a sample containing the biomarker, will change gate states the same way that the transistors in computers change gates for ones and zeros. On top of that, you now have a live signal for that one biomarker.

The Cardean Transistor technology opens up the potential for new insights into biomarkers and molecular signals, says Tad Weems, managing director, Early Stage Partnerships at Agilent Technologies. The technology reads dynamic molecular signals as a function of time which means researchers will be able to interact directly with biology and observe changes in the biomarker signals. Additionally, the technology has multi-omic applications in that it will allow researchers to observe DNA, RNA, and protein biomarkers simultaneously. We believe there is not only a large market potential for these multi-omic devices, but the technology also has the potential to change patient lives.

Heltzen believes this will be the start of a paradigm-shift in many areas of life science and biotech, due to the multi-omics data streaming of DNA, RNA, and protein signals from biology fed to computers in near real time. We were very adamant about the need of a multi-omics system where the analytes on their own can keep doing what they do because otherwise this integration up against computers is not going to work, says Heltzen. If weve destroyed the signal in the process of detecting it, it doesnt make sense. With this, we are getting to the next generation of systems biology.

A digital CRISPR world

Cardea launched its breakthrough chipset called CRISPR-Chip built with its proprietary transistors. The chipset uses CRISPR as the transistor gate, and thereby harvests CRISPRs powerful natural ability to search through genomes for genetic sequences of interest, enabling the user to observe the CRISPR search activity and results in real-time on a computer screen. CRISPR has this feature of being able to run through a genome in record speeds and find specific areas, says Heltzen. So, we have made CRISPR the gate, and when it finds its targetfor example, a gene with a certain mutationit changes the gate signal.

In addition, Cardea Bio is working with partners to create a new generation of applications. Recently, it announced the global launch of a CRISPR quality control toolits first proprietary Powered by Cardea product in partnership. This idea was born out of CRISPR researchers wanting to take a guide RNA and, live on the screen, measure the kinetic strength of the guide RNAs binding to the Cas protein. The result was CRISPR-BIND, the first of a series of quality control applications that will come out of a partnership with CRISPR QC. This tool is a fast and sensitive high-throughput liquid handler that performs quality control of CRISPR-Cas complexes and gRNA. It can also be used to optimize CRISPR designs and identify the most optimal conditions for each CRISPR experiment.

Transistor diagnostics for pandemics and COVID-19

With pandemics like COVID-19, the diagnostic bottlenecks are PCR and DNA amplification. The world right now is basically doing a PCR test that is a reverse-transcription of the RNA into DNA that is just a super artificial step that then goes through the whole amplification phase, Heltzen says. He believes PCR doesnt reveal anything about virus load, virus versions, or even if the virus is alive or deadthe readout is simply that those nucleotides were in the sample.

When it comes to COVID-19, Cardea Bio takes multi-omics approach. So, you have this RNA virus itself that comes and looks at an ACE2 receptor that is an entrance into a cell, says Heltzen. We can test for the virus RNA. We can test for the use of the ACE2 receptor by using it as a gate to say that the virus is basically here now. We can look at the spike proteins or even the different sub domains of the spike protein.

To leverage their unique technology, Heltzen says Cardea Bio is creating a future pandemic solution based on handheld devices that can provide deeper insight into not just whether a person has the virus, but how their immune system is reacting to it. He believes that getting these answers, in real time, at scale will save lives. We know how to use the semiconductor industry infrastructure to make tests, and theres no other industry that knows how to make billions of units at the same time, says Heltzen. That is really the numbers we needtens of millionsand over time billions.

A plug and play toolbox to query life

Although the long-term goal for Cardea Bio is medical impact, it has applied its technology first in agriculture.

Were not going to mess around with things as important as human life until we have all technical bugs out of the platform, says Heltzen.

The medical impact Heltzen sees is a future where doctors query all the biological systems of a patient during an office visit. We already have the best security systems and surveillance systemsits called the immune system, he says.

The cells are communicating to each other, systems are communicating to each other, but the doctor standing there doesnt have a way of listening to that, Heltzen continues. We need the doctors to have live streams coming from the patients, and we already have the wildest technology stack, the patient himself, sitting there, ready to give the signals.

Heltzen says that Cardea Bios long game is to build chipsets that query all the relevant biological channels, no matter what the binding interaction is. He calls them chipsets because he envisions eventually providing ready-made modules with digitized biology at its core and infrastructure built around it.

Think of it like Legos (sic), where you can get a catalog of all the different LEGO blocks and you can be the creator, says Heltzen. We want to open up a developer community of people that understand the diseases, the opportunities, the problems, much better than us because were not going to tell people what to build.

Heltzen believes that such a community will take all the components that have been developed by biology over the past few billion years and start using them as a technology to release a new generation of natural resources on this planet.

Visit link:
Seeing Real-Time Biological Interactions with Graphene Transistors - Clinical OMICs News

Recommendation and review posted by Bethany Smith

The research study of the global Crispr Technology market provides the market size information and market trends along with the factors and parameters impacting it in both the short and long term. The report ensures a 360-degree assessment, bringing out the complete key insights of the industry. These insights help the business decision-makers to make better business plans and informed decisions for the future business. In addition, the study helps the venture capitalist in understanding the companies better and take informed decisions.

The Crispr Technology market research report provides essential statistics on the market position of the Crispr Technology manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. The report provides a basic summary of theCrispr Technology industry including its definition, applications and manufacturing technology. The report presents the company profile, product specifications, capacity, production value, and market shares for key vendors.

The overall market is split by the company, by country, and by application/type for the competitive landscape analysis. The report estimates market development trends of Crispr Technology industry. Analysis of upstream raw materials, downstream demand and current market dynamics is also carried out. The Crispr Technology market report makes some important proposals for a new project of Crispr Technology Industry before evaluating its feasibility.

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Key segments covered in Crispr Technology market report: Major key companies, product type segment, end use/application segment and geography segment.

The information for each competitor includes:

Company segment, the report includes global key players of Crispr Technology as well as some small players:

For product type segment, this report listed the main product type of Crispr Technology market

For end use/application segment, this report focuses on the status and outlook for key applications. End users are also listed.

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Crispr Technology Market to 2026: Report on Top Company Players, Industry Insights and Overview - PRnews Leader

Recommendation and review posted by Bethany Smith

The Clinical Grade Viral Vector & Plasmid DNA Manufacturing Market research report recently presentedby Prophecy Market Insights which provides reliable and sincere insights related to the various segments and sub-segments of the market. The market study throws light on the various factors that are projected to impact the overall dynamics of the Clinical Grade Viral Vector & Plasmid DNA Manufacturing market over the forecast period (2019-2029).

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Application and Product Portfolio Analysis During the forecasted period of CRISPR Technology Market - PRnews Leader

Recommendation and review posted by Bethany Smith

Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.

Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.

Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.

Start using real-world data to advance your clinical research

Much has been written about the promise of real-world data (RWD) in life sciences, but how does it work in practice? We address this question in a new whitepaper that demonstrates the potential benefits of new RWD technologies with a proof of concept study to show how RWD can be incorporated into clinical research.

The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.

AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.

Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.

The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.

Link:
Avrobio tracks improvements in first patient treated with Gaucher gene therapy - FierceBiotech

Recommendation and review posted by Bethany Smith

Vivet Therapeutics and Pfizer are one step closer to bringing a gene therapy for a rare liver disorder into the clinic.

The companies announced Wednesday morning that the FDA has accepted its IND application for a Phase I/II study in the treatment of Wilsons disease. The study, evaluating a program dubbed VTX-801, is expected to launch early next year.

VTX-801 is an rAAV-based gene therapy vector designed to deliver a protein called ATP7B in the hopes of restoring copper homeostasis, reversing liver pathology and reducing copper accumulation in the brain, as it was shown to do in mouse models.

The study will be open label and not be randomized. Researchers will give a one-time IV infusion of the gene therapy in up to 16 adult patients, with the goal of evaluating three different dosage levels. Ultimately, the companies set a primary endpoint for safety and tolerability after 52 weeks.

In March 2019, Pfizer acquired a minority stake in the company, and in September, the big pharma agreed to manufacture the VTX-801 vector for this Phase I/II study. Max Gelman

Florida-based biotech Generex has inked the biggest deal (it) could even imagine, bagging $50 million from a consortium of Chinese institutions that licensed its Ii-Key vaccine tech for infectious diseases and cancer.

Comprising hybrid peptides and a suppression, the platform has spawned a vaccine candidate against SARS-CoV-2 in addition to a pipeline of immuno-oncology therapies.

We are able to generate a detailed immune activation profile of our Ii-Key vaccine candidates by screening blood samples from COVID-19 recovered patients, explained Richard Purcell, EVP of R&D.

In addition to the upfront fee for the overall deal, the unnamed partners have handed over $5 million to license the Covid-19 vaccine candidate and promised a $20 million success fee if its approved in China. Separate contracts for the other indications are being finalized. Amber Tong

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News briefing: Pfizer and Vivet get the OK to start gene therapy trial for rare liver disorder; Florida biotech inks $50M China deal - Endpoints News

Recommendation and review posted by Bethany Smith

COVID-19 Coronavirus molecule, March 24, 2020.

CDC | API | Gamma-Rapho via Getty Images

A team of CRISPR scientists at the New York Genome Center, New York University and Icahn School of Medicine at Mount Sinai say they have identified the genes that can protect human cells against Covid-19, a disease that has infected over 40 million and led to 1 million deaths worldwide. The discovery comes after an eight-month screen of all 20,000 genes in the human genome led by Dr. Neville Sanjana at the New York Genome Center. Leading virologist at Mount Sinai, Dr. Benjamin tenOever, developed a series of human lung cell models for the coronavirus screening to better understand immune responses to the disease and co-authored the study.

Their study, published online last month by Cell, will appear in the scientific peer-reviewed journal's Jan. 7 print issue.

The goal was two-fold: to identify the genes that make human cells more resistant to SARS-CoV-2 virus; and test existing drugs on the market that may help stop the spread of the disease.

The breakthrough comes at a time when drug makers such as Pfizer, Oxford-AstraZeneca and Moderna are fast-forwarding vaccine and therapeutics to treat Covid-19. On Friday, Pfizer and BioNTech requested emergency authorization from the FDA for their Covid vaccine that contains genetic material called messenger RNA, which scientists expect provokes the immune system to fight the virus.

In order to better understand the complex relationships between host and virus genetic dependencies, the team used a broad range of analytical and experimental methods to validate their results. This integrative approach included genome editing, single-cell sequencing, confocal imaging and computational analyses of gene expression and proteomic datasets.

After intensive research the scientists and doctors claim they have found 30 genes that block the virus from infecting human cells including RAB7A, a gene that seems to regulate the ACE-2 receptor that the virus binds to and uses to enter the cell. The spike protein's first contact with a human cell is through ACE-2 receptor.

"Our findings confirmed what scientists believe to be true about ACE-2 receptor's role in infection; it holds the key to unlocking the virus," said Dr. tenOever. "It also revealed the virus needs a toolbox of components to infect human cells. Everything must be in alignment for the virus to enter human cells."

The team discovered that the top-ranked genes those whose loss reduces viral infection substantially clustered into a handful of protein complexes, including vacuolar ATPases, Retromer, Commander, Arp2/3, and PI3K. Many of these protein complexes are involved in trafficking proteins to and from the cell membrane.

"We were very pleased to see multiple genes within the same family as top-ranked hits in our genome-wide screen. This gave us a high degree of confidence that these protein families were crucial to the virus lifecycle, either for getting into human cells or successful viral replication," said Dr. Zharko Daniloski, a postdoctoral fellow in the Sanjana Lab and co-first author of the study.

Using proteomic data, they found that several of the top-ranked host genes directly interact with the virus's own proteins, highlighting their central role in the viral lifecycle. The team also analyzed common host genes required for other viral pathogens, such as Zika or H1N1 pandemic influenza.

The research team also identified drugs that are currently on the market for different diseases that they claim block the entry of Covid-19 into human cells by increasing cellular cholesterol. In particular, they found three drugs currently on the market were more than 100-fold more effective in stopping viral entry in human lung cells:

The other five drugs that were tested called PIK-111, Compound 19, SAR 405, Autophinib, ALLN -- are used in research but are not yet branded and used in clinical trials for existing diseases.

Our findings confirmed what scientists believe to be true about ACE-2 receptor's role in infection; it holds the key to unlocking the virus.

Their findings offer insight into novel therapies that may be effective in treating Covid-19 and reveal the underlying molecular targets of those therapies.

The bioengineers in New York were working on other projects with gene-editing technology from CRISPR but quickly pivoted to studying the coronavirus when it swept through the metropolitan area last March. "Seeing the tragic impact of Covid-19 here in New York and across the world, we felt that we could use the high-throughput CRISPR gene editing tools that we have applied to other diseases to understand what are the key human genes required by the SARS-CoV-2 virus," said Dr. Sanjana.

Dr. Neville Sanjana and his team at the New York Genome Center used CRISPR to identify the genes that can protect human cells against Covid-19.

New York Genome Center

As he explained, "current treatments for SARS-CoV-2 infection currently go after the virus itself, but this study offers a better understanding of how host genes influence viral entry and will enable new avenues for therapeutic discovery."

Previously, Dr. Sanjana has applied genome-wide CRISPR screens to identify the genetic drivers of diverse diseases, including drug resistance in melanoma, immunotherapy failure, lung cancer metastasis, innate immunity, inborn metabolic disorders and muscular dystrophy.

"The hope is that the data from this study which pinpoints required genes for SARS-CoV-2 infection could in the future work be combined with human genome sequencing data to identify individuals that might be either more susceptible or more resistant to COVID-19," Dr. Sanjana said.

The New York team is not the first to use CRISPR gene editing techniques to fight Covid-19. Other bioengineering groups at MIT and Stanford have been using CRISPR to develop ways to fight the SARS-CoV-2 and develop diagnostic tools for Covid-19.

The potential for using CRISPR to eliminate viruses has already generated some enthusiasm in the research community. Last year, for example, Excision BioTherapeuticslicenseda technology from Temple University that uses CRISPR, combined with antiretroviral therapy, to eliminate HIV, the virus that causes AIDS.

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Gene experts claim they identified human genes that can protect against Covid-19 - CNBC

Recommendation and review posted by Bethany Smith

Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivets Investigational Gene Therapy for Wilson Disease

PARIS, France and NEW YORK, N.Y.November 18, 2020 Vivet Therapeutics (Vivet), a privately held gene therapy biotech company dedicated to developing treatments for inherited liver disorders with high unmet medical need, and Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has cleared Vivets Investigational New Drug (IND) application for the GATEWAY study, a Phase 1/2 clinical trial evaluating Vivets proprietary, investigational gene therapy vector, VTX-801, for the potential treatment of Wilson disease (WD), a rare and potentially life-threatening liver disorder. The trial is expected to commence in early 2021.

We are pleased to announce Vivets first IND clearance by the FDA, which is for our GATEWAY Phase 1/2 study for VTX-801, said Jean-Philippe Combal, CEO and Co-Founder of Vivet. This is a very important milestone for the Wilson disease community for whom VTX-801 could bring significant potential therapeutic benefit. VTX-801 aims to restore copper homeostasis and the GATEWAY trial will measure relevant biomarkers to evaluate physiological restoration of copper elimination and transport in patients. We look forward to advancing VTX-801 into the clinic in early 2021.

VTX-801 is a novel, investigational rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson disease. VTX-801s rAAV serotype was selected based on its demonstrated tropism for transducing human liver cells.

In March 2019, the companies announced that Pfizer had acquired a minority equity interest in Vivet and secured an exclusive option to acquire all outstanding shares. In September 2020, Vivet and Pfizer announced the signing of an agreement for the manufacture by Pfizer of the VTX-801 vector for the GATEWAY study.

The FDA clearance of Vivets IND marks an important milestone for the VTX-801 program, which we believe has the potential to become a transformational therapy for people with Wilson disease, said Seng Cheng, Chief Scientific Officer, Rare Disease Research Unit, Pfizer. Pfizer has begun manufacturing clinical material for the GATEWAY study and look forward to the studys commencement.

This IND is a recognition of the expertise of Vivets research team led by our CSO and Co-Founder, Dr. Gloria Gonzlez-Aseguinolaza, research collaborations, notably with la Fundacin para la Investigacin Mdica Aplicada (FIMA), and experienced development team. We believe that our global development expertise, together with our collaboration with Pfizer, places us in a strong position to rapidly execute and bring this potentially transformational therapy to patients with high unmet medical needs, added Jean-Philippe Combal.

About GATEWAY - Phase 1/2 Clinical Trial of VTX-801 in Wilson disease

The GATEWAY trial is a multi-center, non-randomized, open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background WD therapy withdrawal.

Six leading centers in the United States and Europe are expected to participate in the GATEWAY Phase 1/2 trial. The trial is expected to enroll up to sixteen adult patients with Wilson disease and will evaluate up to three doses of VTX-801. Patients will participate in a pre-dosing observational period and will be administered a prophylactic steroid regimen.

The primary endpoint of the GATEWAY trial is to assess the safety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal.

Vivet Therapeutics expects to enroll the first patient in early 2021.

More details on:

https://clinicaltrials.gov/ct2/show/NCT04537377?term=VIVET&draw=2&rank=1

About Vivet Therapeutics

Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundacin para la Investigacin Mdica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigacin Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

Vivets lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

Vivets second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

Please visit us on http://www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice

The information contained in this release is as of November 18, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about Vivet Therapeutics (Vivet) investigational gene therapy, VTX-801, and Pfizers collaboration with Vivet on the development of VTX-801, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, risks related to the ability to realize the anticipated benefits of the collaboration, including the possibility that the expected benefits from the collaboration will not be realized or will not be realized in the expected time; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities;whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when any applications may be filed in any jurisdiction for VTX-801; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the products benefits outweigh its known risks and determination of the products efficacy and, if approved, whether VTX-801 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of VTX-801; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results;and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available atwww.sec.govandwww.pfizer.com.

Continued here:
Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet's Investigational Gene...

Recommendation and review posted by Bethany Smith

Nov. 16, 2020 12:30 UTC

- Highly innovative genome editing technology platform accelerates and broadens ElevateBios cell and gene therapy enabling technologies

- LifeEDIT Therapeutics to develop its own pipeline of potentially life-transforming therapeutics

- LifeEDIT Therapeutics to leverage its novel gene editing platform with strategic partners including ElevateBio portfolio companies

CAMBRIDGE, Mass. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- ElevateBio, a Cambridge-based cell and gene therapy holding company, and AgBiome, a leader in developing innovative products from the Earth's microbial communities, today announced that LifeEDIT Therapeutics has joined ElevateBios growing portfolio of therapeutic, technology and manufacturing companies. LifeEDIT Therapeutics combines a highly innovative genome editing platform, derived from AgBiomes massive proprietary microbial library, with ElevateBios proven expertise in the discovery and development of new cell and gene therapies. LifeEDIT will continue to develop internal cell and gene therapies while further strengthening its platform of diverse genome-editing enzymes and provide gene editing expertise to strategic partners including ElevateBio portfolio companies. AgBiome retains rights for gene editing in agriculture, animal health, and diagnostics.

Genome editing technologies have revolutionized the way we develop cell and gene therapies and regenerative medicines, said Mitchell Finer, Ph.D., President, ElevateBio BaseCamp, and newly appointed Chief Executive Officer, LifeEDIT Therapeutics. However, in order to realize the promise of, and democratize, these highly innovative therapeutic approaches, the field needs to access novel RNA-guided nucleases and base editors that offer greater specificity and broader genome coverage, which LifeEDIT can provide. LifeEDITs genome editing platform is one of the most versatile in the field and was the natural fit as we continue to build a world leading cell and gene therapy offering.

Members of ElevateBio will join the newly formed LifeEDIT Therapeutics management team, which will continue to benefit from its existing visionary scientific leadership and research team. The combined executive team will include:

Over the last 18 months we have built a truly unique platform of numerous RNA-guided nucleases with diverse PAM requirements, for which weve been able to show functional activity, said Tedd Elich, Ph.D., Chief Scientific Officer, LifeEDIT Therapeutics. The wide range of gene editing enzymes across our platform increases our ability to target any genomic sequence of interest and will allow us to tackle some of the most challenging diseases, bringing desperately needed, potentially curative, therapies to patients in need.

"AgBiome's GENESISTM platform is built on our microbial collection, their complete genome sequences, and our industry-best data science platform to identify new useful functions, said Eric Ward Co-Chief Executive Officer, AgBiome. This unique resource formed the basis for the many genome editing technologies that are now part of LifeEDIT Therapeutics. We look forward to continuing to collaborate with the LifeEDIT team as they build a world-class pipeline of clinical candidates and bring a broad array of genome editing technologies to innovators across the biotechnology industry."

About Genome Editing and LifeEDIT Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, LifeEDIT Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. LifeEDIT Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. LifeEDIT Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The LifeEDIT Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

LifeEDIT Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, LifeEDIT Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating and life-threatening diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBios team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBios vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and translational research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners, are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.

ElevateBios investors include F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, Samsara BioCapital, The Invus Group, Emerson Collective, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, please visit http://www.elevate.bio.

About AgBiome

AgBiome partners with the microbial world to improve our planet. AgBiome discovers and develops innovative biological and trait products for crop protection. The proprietary GENESIS discovery platform efficiently captures diverse, unique microbes for agriculturally relevant applications, and screens them with industry-best assays for insect, disease, and nematode control. Through its commercial subsidiary, AgBiome develops and sells proprietary crop protection solutions. The first of these, Howler, is a revolutionary fungicide for disease control in a broad variety of crops. AgBiome and Genective recently formed a strategic partnership to establish a new leader in insect traits, a market with over $5 billion in annual opportunities. AgBiome has a global R&D collaboration with Elanco Animal Health Incorporated (NYSE: ELAN), to develop nutritional health products for swine. AgBiomes investors include Polaris Partners, ARCH Venture Partners, Fidelity Investments Inc., UTIMCO, Pontifax AgTech, Innotech Advisors, Syngenta Ventures, Leaps by Bayer, and Novozymes. For more information, visit http://agbiome.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201116005105/en/

Original post:
ElevateBio and AgBiome Announce LifeEDIT Will Join ElevateBio's Portfolio of Innovative Cell and Gene Therapy Companies - BioSpace

Recommendation and review posted by Bethany Smith

MarketsandResearch.biz presents Global Cell and Gene Therapy Market 2020 by Company, Regions, Type and Application, Forecast to 2025 which shows the most recent industry chain structure and complete analysis of the market. The report offers detailed information on enterprises on a global and regional level through an all-inclusive analysis and insights into developments affecting businesses. The report covers the market landscape and its growth prospects over the coming years. Leading companies effective in this market are discussed further. The report calculates the market size and considers the revenue generated from the sales of Cell and Gene Therapy globally. The research accommodates an entire overview of the market, including the market share, and the projection of this global market, within a specific interval of time.

The report contains critical information on the market which will assist the industry players in making informed business move. The report is segmented by application/ end-user, product type, and geologies. This report will help you to make informed decisions, understand opportunities, plan new projects, analyze drivers and restraints, and give you a vision of the industry forecast (2020-2025). The report further explains the competitive backdrop of key players in the market as well as their product portfolio and business strategies. It offers an investigation of the global Cell and Gene Therapy industry in terms of consumption and production.

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The market report provides the major growth factors and limitations that notably affect market growth. The report studies the data about the past and present status of the global Cell and Gene Therapy market globally. The upcoming economic fluctuations related to the current market growth pattern of the market are investigated. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

The market competition by top manufacturers/key player profiled: Amgen Inc., Novartis AG, Fibrocell Science, Inc., bluebird bio, Inc., Kolon TissueGene, Inc., Dendreon Pharmaceuticals LLC., Organogenesis Holdings Inc., Kite Pharma, Inc., Human Stem Cells Institute, Orchard Therapeutics plc., Spark Therapeutics, Inc., Pfizer, Inc., Vericel Corporation, Sibiono GeneTech Co. Ltd.,, RENOVA THERAPEUTICS, ViroMed Co., Ltd., Shanghai Sunway Biotech Co., Ltd.,

The report conjointly categorizes the market into main product kind: Rare Diseases, Oncology, Hematology, Cardiovascular, Ophthalmology, Neurology, Other Therapeutic Classes

The report splits the market into main applications: Pharmaceutical and Biotechnology Companies, Research and Academic Institutions, Contract Research Organizations (CROs), Hospital, Others

The report offers an in-depth assessment of the growth and other aspects of the market in important regions, including: North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

The conclusion contains the evaluation of the global Cell and Gene Therapy market through multiple classifications and provides thorough information about the upstream raw materials, downstream buyers, and distribution channels established by the various competitors. More importantly, a study of recent market trends, drivers, challenges, and opportunities shaping the profitability graph of the industry are presented further in the report.

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Global Cell and Gene Therapy Market 2020 Key Drivers and Challenges, Opportunities and Forecast Insights by 2025 - KYT24

Recommendation and review posted by Bethany Smith

FNF Research (fnfresearch.com) offering a comprehensive analysis on the Updated Research on Gene Therapy Market Analysis 2020 Size & Share Report Including COVID-19 Impact and Forecast to 2026 where users can benefit from the complete market research report with all the required useful information about this market. This is the latest report, covering the current COVID-19 impact on the market. The rapidly changing market scenario and initial and future assessment of the impact are covered in the report. The report discusses all major market aspects with an expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

TheGene Therapy Marketreport entails a comprehensive database on Upcoming market estimation based on historical SWOT data analysis. It enables clients with quantified data for current market perusal. It is a professional and detailed report focusing on primary and secondary drivers, market share, leading segments, and regional analysis. Listed out are key players, major collaborations, merger & acquisitions along with upcoming and trending innovation. Business policies are reviewed from the techno-commercial perspective demonstrating better results. The report contains granular information & analysis pertaining to the Gene Therapy Market size, share, growth, trends, segment, and forecasts from 2020-2026.

According to the research report, [185+ Pages PDF Report]Global gene therapy market was valued at approximately 1.4 billion in 2019 and is expected to reach a value of around USD 3.4 billion, at a CAGR of around 14.7% between 2019 and 2027. Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells.

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Gene Therapy Market Historic Data (2019-2026):

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Gene Therapy Market 2020 Size, Share, Demand and Detailed Competitive Outlook by 2026 - Illadel Graff Supply

Recommendation and review posted by Bethany Smith

Precise treatment leads to resolution of patient's debilitating symptoms and complete remodeling of her lymphatic system

PHILADELPHIA, Nov. 20, 2020 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a girl with Noonan Syndrome that had led to upper gastrointestinal bleeding, fluid collection around the lungs, and numerous surgeries that had been unable to resolve her symptoms. By identifying a genetic mutation along a pathway related to lymphatic vessel development and function, the research team was able to target the pathway using an existing drug they had used in a previous case to remodel a patient's lymphatic system.

The case study, which was published today in Pediatrics, describes a resolution of the patient's symptoms within three months while on the medication.

"This study is quite significant," said first author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. "Inhibiting this pathway seems to have sweeping, widespread effects on the lymphatic system. How this process occurs is not fully understood, but is remarkable in its speed and breadth. This gives us a lot of hope for treating other patients with genetic mutations along this same pathway in the future."

The patient described in the paper, Maria, first came to CHOP when she was 14, after experiencing severe anemia due to upper gastrointestinal bleeding, as well as other symptoms including fluid build-up in the cavity around her lungs, chronic fatigue, delayed puberty, and difficulty gaining weight. Maria had been born with Noonan Syndrome, a genetic disorder that prevents normal development in various parts of the body and often results in short stature, heart defects and other physical problems, including an abnormal lymphatic system. Despite aggressive medical therapy elsewhere, Maria continued to bleed internally, and she underwent multiple blood transfusions to try to stabilize her health.

Within two days of transferring to CHOP, the lymphatics team, led by Dori, determined Maria had many lymphatic irregularities, which were leading to internal bleeding and lung problems, so they scheduled Maria's first intervention, a lymphatic embolization procedure that would seal the leaky vessels in her gut.

However, within two months of the procedure, Maria's gastrointestinal bleeding recurred. Over the following 8 months, she underwent two additional procedures, as well as a cauterization procedure to close off some of the blood vessels in her gut, but the benefits of each procedure lasted only about three months before the bleeding and her symptoms returned.

Based on whole exome sequencing done at CHOP's Center for Applied Genomics, the research team learned that Maria had a genetic mutation in the SOS1 gene, which operates along the RAS-MAPK pathway. This pathway involves mitogen-activated protein kinase (MEK), and Maria's mutation caused an overproduction of MEK, which resulted in the uncontrolled proliferation of her lymphatic vessels.

The research team had previously used a MEK inhibitor in another patient with a severe lymphatic disorder with great success. That patient had a mutation in the ARAF gene, which is also on the RAS-MAPK pathway. Within months of beginning treatment with trametinib, a MEK inhibitor, the patient saw a resolution of his symptoms and a complete remodeling of his lymphatic system.

Given that SOS1 operates on the same pathway as ARAF, Jean Belasco, MD, an oncologist in CHOP's Cancer Center who co-led the study, applied for compassionate use of the drug in Maria's case, given the lack of other treatment options.

"The success of trametinib in another patient with a mutation on the RAS-MAPK pathway encouraged us to try this approach, since other procedures and therapies continued to be unsuccessful," Belasco said. "Although we are in the early days of this type of personalized medicine, the hope is that by looking at patients' mutations, we can find more drugs and better care for patients with genetic diseases."

Within three months of starting the drug, Maria's vital signs stabilized. The bleeding stopped, her electrolyte, hemoglobin, and albumin levels returned to normal, and she began to gain weight. Maria's mother noticed that Maria wasn't going through periods of exhaustion anymore, and her pallor improved.

"She looks better than she's ever looked," her mother said. "She looks like a normal teenager. It's like night and day. She's also a lot happier. I think she knew deep down she was dying. The medicine gave her hope."

Hakon Hakonarson, Director of the Center for Applied Genomics and co-author of the paper, said that although Maria's SOS1 mutation is distinctly different than the ARAF mutation seen in the other patient, the drug was equally effective because it targets and blocks the function of MEK. He likened the scenario to a pathway where 15 events need to occur for a cell to function. Maria's SOS1 mutation might occur at step nine, whereas the ARAF mutation might occur at step three, but both genes are on a chain that ultimately passes through a tunnel that leads to phosphorylation and overactivity of MEK. Since both mutations were so-called gain of function mutations, MEK and thus lymphatic activity was overexpressed in both patients. The MEK inhibitor put the brakes on a system in overdrive.

"Remarkable advances in genetics have allowed us to uncover these mutations and cluster them into selective pathways and determine effective therapies based on genetic mutations with very high precision," said Hakonarson. "No one could have guessed that this drug would have worked for Maria without knowing the underlying genetics. This discovery is extremely important because Noonan Syndrome has the biggest patient population with alterations in MEK signaling. Not all Noonan patients will have mutations that respond to this therapy, but a very good number of them will."

He added that the treatment could also benefit patients with other genetic defects, though he noted the ongoing use of the drug treats the symptoms caused by these mutations, but does not fix the gene or cure the underlying condition.

"MEK inhibition has the potential to have significant effects on other organ systems affected by RAS-MAPK gene defects, such as the heart, eyes, skin and the coagulation system," Hakonarson said.

Hakonarson is also part of CHOP's Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program that uses state-of-the-art genomics and personalized research strategies to determine the causes of complex vascular conditions and identify targeted therapies. The program works closely with the Lymphatic Imaging and Interventions Frontier Program, which is led by Dori. CHOP's Frontier Programs conduct cutting-edge research that translates into advanced clinical care. The CVAP, in particular, draws on the extensive clinical and genomic research capacity within the Cancer Center and Center for Applied Genomics.

Even with the success of the breakthrough treatment pioneered by these programs, it is not entirely clear why MEK inhibitors not only resolve patients' symptoms but also completely remodel their lymphatic systems. Hakonarson said one possibility is that when mutated genes cause uncontrolled growth of the lymphatic system, the body's vessels leak fluid everywhere in the body. When you shut down the unregulated growth, other homeostatic mechanisms that are balancing the system come into effect, so the overreactive cells that were growing out of control die and are replaced by normal cells that gradually build up the lymphatic system.

Whatever the mechanism, Maria's mother said her daughter had no hesitation at being the first patient with Noonan Syndrome to try this treatment to resolve a lymphatic issue.

"Maria saw the value from the beginning," she said. "She saw the value for herself, but she was also thinking of other Noonan kids, some of whom have passed away from lymphatic issues. She was willing and eager."

Dori et al. "Severe Lymphatic Disorder Resolved with MEK Inhibition in a Noonan Patient with SOS1 Mutation," Pediatrics, published online November 20, 2020, doi: 10.1542/2020-000123

About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Contact: Natalie SolimeoChildren's Hospital of Philadelphia267-426-6246solimeon@chop.edu

View original content:http://www.prnewswire.com/news-releases/chop-researchers-reverse-severe-lymphatic-disorder-in-patient-with-noonan-syndrome-by-targeting-genetic-pathway-301177697.html

SOURCE Children's Hospital of Philadelphia

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CHOP Researchers Reverse Severe Lymphatic Disorder in Patient with Noonan Syndrome by Targeting Genetic Pathway - BioSpace

Recommendation and review posted by Bethany Smith

Helixmith said Thursday the first enrolled patient has received its new gene therapy, Engensis (VM202), in phase 3-2 study of diabetic peripheral neuropathy (DPN).

The company plans to continue administering the drug to other participants, as it is screening many additional patients at five clinical trial centers. It is conducting DPN phase 3-2 trials, REGAIN-1A, at 15 clinical centers with 152 enrolled patients in the U.S.

Helixmith aims to meet two endpoints in the study.

The trial's primary efficacy endpoint is to compare changes in average daily pain scores between VM202 and placebo. The secondary endpoint is to reduce pain by 50 percent or more.

DPN is one of the most common complications of diabetes as 30 million U.S. adults have the disorder. Around 28.5 percent of diabetic patients develop DPN, and up to half of them may advance to painful DPN (PDPN).

Dr. Miguel Trevino, head of clinical trials at Innovative Research in which the first patient received VM202, noted that the existing treatments for DPN only relieve pain and show various adverse events with limited efficacy, inevitably leaving half of the patients in painful life.

"The delay of this phase 3 study was due to the preparation of clinical operation system and examining numerous items during the patient's screening process," Helixmith CEO Kim Sun-young said. "Our latest study will add considerable knowledge and evidence to support the optimal use of VM202 in patients needing therapeutic options."

The U.S. Food and Drug Administration recognized the scientific and clinical results of VM202 and designated it as RMAT (regenerative medicine advanced therapy) in 2018.

Read this article:
Helixmith injects DPN therapy into 1st patient of P3 study - Korea Biomedical Review

Recommendation and review posted by Bethany Smith

Gene Therapy Market Growth, Trends, and Forecasts (2020 2025)

The Gene Therapy Market research report 2020 provides a basic overview of the industry including definitions, classifications, applications, and industry chain structure. The Gene Therapy market report provides information regarding market size, share, trends, growth, cost structure, Gene Therapy market competition landscape, market drivers, challenges and opportunity, capacity, revenue, and forecast 2025.

The report delivers a comprehensive overview of the crucial elements of the market and elements such as drivers, current trends of the past and present times, supervisory scenario & technological growth. This report also includes the overall and comprehensive study of the Gene Therapy market with all its aspects influencing the growth of the market. This report is exhaustive quantitative analyses of the Gene Therapy industry and provides data for making strategies to increase market growth and effectiveness.

The global Gene Therapy Market is expected to witness a 28.32% of CAGR during the forecast period.

Scope of the report:

The report evaluates the growth rate and the Market value based on Market dynamics, growth inducing factors. The complete knowledge is based on the latest industry news, opportunities, and trends. The report contains a comprehensive Market analysis and vendor landscape in addition to a SWOT analysis of the key vendors.

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The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market. Top Companies in the Global Gene Therapy Market: Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd., Spark Therapeutics LLC, UniQure N.V., and others.

Key Market Trends

Cancer is Expected to Hold Significant Market Share in the Indication Segment

The ever-rising prevalence of cancer all across the globe is majorly driving the growth of this segment. Getting genes into cancer cells has been one of the most difficult aspects of present-day gene therapy. Researchers have been working on finding new as well as better ways of doing this. Usually, the gene is taken into the cancer cell by a carrier known as a vector. The most common types of a carrier that are used in gene therapy are viruses owing to the fact that they can enter cells and eventually deliver genetic material. The viruses are generally changed so that they cannot result in any serious disease. However, they may still cause mild, flu-like symptoms. Some viruses have been modified in the laboratory so that they can target cancer cells only and not the healthy cells. Therefore, they only carry the gene into cancer cells.

North America Dominates the Market and Expected to do Same in the Forecasted Period

For gene therapy, in the United States, the DHHS (Department of Health and Human Services) has been appointed to oversee the clinical trials. Two other organizations within DHHS, the OHRP (Office for Human Research Protections) and the U.S. FDA (Food and Drug Administration), have a specific authority that is described in the CFR (Code of Federal Regulations). All investigators must obey these regulations while conducting clinical gene therapy trials. The increasing approval of various gene therapies by the afore-mentioned organizations is driving the market growth in the region. For instance, in May 2019, AveXis, a Novartis company, announced that the US FDA had approved Zolgensma (onasemnogene abeparvovec-xioi) for treating pediatric patients of less than 2 years of age having spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Influence of the Gene Therapy market report

Comprehensive assessment of all opportunities and risks in the Gene Therapy market.

Gene Therapy market recent innovations and major events.

A Detailed study of business strategies for the growth of the Gene Therapy market-leading players.

Conclusive study about the growth plot of the Gene Therapy market for forthcoming years.

In-depth understanding of Gene Therapy market-particular drivers, constraints, and major micro markets.

Favorable impression inside vital technological and market latest trends striking the market report.

What are the market factors that are explained in the report?

Key Strategic Developments: The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

Analytical Tools: The Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

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Gene Therapy Market 2020 After COVID-19 Developments Analysis of Best Key Players Amgen Inc., Bluebird Bio, Gilead Sciences, Inc. - The Daily...

Recommendation and review posted by Bethany Smith

The Gene Therapy Market research report recently presentedby Prophecy Market Insights which provides reliable and sincere insights related to the various segments and sub-segments of the market. The market study throws light on the various factors that are projected to impact the overall dynamics of the Gene Therapy market over the forecast period (2019-2029).

Influences of the market report:

An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Gene Therapy market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Get Free Sample Copy of This Report @ https://www.prophecymarketinsights.com/market_insight/Insight/request-sample/39

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

Australia, New Zealand, Rest of Asia-Pacific

Stakeholders Benefit:

Segmentation Overview:

The Gene Therapy research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Gene Therapy market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Highlights of the Report

Complete access to COVID-19 Impact on the Gene Therapy market dynamics, key regions, market size, growth rate and forecast to 2029

The report on the Gene Therapy market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have been included in the report.

Some important Questions Answered in Gene Therapy Market Report are:

Gene TherapyMarket by Top Manufacturers:

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

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Product Portfolio Analysis and Technological Development of Gene Therapy Market during the forecasted period - PRnews Leader

Recommendation and review posted by Bethany Smith

Every time a cell inside your body replicates, a slither of your youth crumbles to dust. This occurs via the shortening oftelomeres, structures that 'cap' the tips of our chromosomes.

Now, scientists in Israel say they've been able to reverse this process and extend the length of telomeres in a small study involving 26 patients.

The participants sat in a hyperbaric oxygen chamber for five 90 minutes sessions per week over three months, and as a result, some of their cell's telomeres were extended by up to 20 percent.

It's an impressive claim - and something many other researchers have attempted in the past without success. But of course it's worth flagging that this is a small sample size, and the results will need to be replicated before we can get too excited.

However, the fact that hyperbaric oxygen therapy appears to affect telomere length is a compelling link worth investigating further.

Lead researcher Shair Efrati, a physician from the Faculty of Medicine and Sagol School of Neuroscience at Tel Aviv University, explained to ScienceAlert how the inspiration behind their experiment was somewhat out of this world.

"After the twin experiment done by NASA, where one of the twins was sent out to the outer space and the other stayed on Earth, demonstrated a significant difference in their telomere length we have realised that changes in the outside environment may affect the core cellular changes that happens along ageing," said Efrati.

Telomeres are repeating chunks of code that act as the DNA equivalent of the plastic or metal aglet capping the end of a shoelace.

They copy themselves along with the rest of the chromosomes whenever a cell divides. Yet with every replication, tiny fragments of code from the very tip of the sequence fail to make it into the new copy, leaving the freshly minted chromosome a touch shorter than its predecessor.

As anybody who has lost the cap of their shoelace knows, it doesn't take long for the shoelace to lose its integrity. Similarly, shorter telomeres put sequences further down the chromosome at higher risk of hazardous mutations.

These mutations coincide with changes that predispose us to a bunch of age-related conditions, not least of all diseases such as cancer.

That's not necessarily to say that we age because our telomeres shrink, but there is a connection between telomere length and health that researchers are keen to investigate further.

"Longer telomeres correlates with better cellular performance," Efriti explained.

There are plenty of ways to accelerate the erosion of our telomeres. Failing to get adequate sleepcould do it, as might chowing down on too much processed food, and maybe even having kids.

Slowing down the loss takes a bit more effort, but engaging in regular exercise and eating well are sound bets if you want your chromosomes to remain as long as possible.

A real achievement would be to flip our chromosomal hourglass completely and return lost sections of telomere. The fact that high-turnover tissues lining our gut do this naturally using an enzyme called telomerase has fuelled research over the years.

There have been plenty of milestones in attempts to achieve this task. Gene therapy in mice has shown it could one day be feasible in humans. More recently, stem cells from a supercentenarian woman had their telomeres completely reset outside of her body.

Some studies have found potential for tiny increases of maybe a few percent with provision ofnutritional supplementssuch asvitamin D.

But while there are plenty of hyped promises of reversing aging in living humans already on the market, the reality of science-backed therapies we can use to give us the telomeres of a 20-year-old has been underwhelming.

Which is why the latest study is attracting so much attention. Far from a measly two or three percent, this latest study found telomeres in white blood cells taken from 26 subjects had regained around a fifth of their lost length.

The key, it seems, is hyperbaric oxygen therapy (HBOT) the absorbing of pure oxygen while sitting in a pressurised chamber for extensive periods; in this case, five 90 minute sessions per week over three months.

HBOT has attracted controversy in the past for claims it could treat a range of conditions.It's usually the kind of therapy you'd give a diver who came up too fast from the ocean depths, or to kill off oxygen-sensitive microbes in a wound that just won't heal any other way.

But oxygen-rich environments are also behind a weird paradox, one where the body desperately stirs up a host of genetic and molecular changes that typically occur in a low oxygen one.

In this study, the researchers were able to show that the genetic changes provoked by the HBOT has extended telomeres, and also had a potentially positive effect on the health of the tissues themselves.

A slightly smaller sample of volunteers also showed a significant decrease in the number of senescentT cells, tissues that form a vital part of our immune system's targeted response against invaders.

Whether you'd sit in a small tank every day for a quarter of a year is a matter of preference, but future research could help make the whole process a touch more efficient, at least for some.

"Once we have demonstrated the reverse ageing effect on the study cohort using predefined HBOT protocol, further studies are needed in order to optimise the specific protocol per individual," Efrati told ScienceAlert.

In a press release from The Sagol Center for Hyperbaric Medicine and Research, Efrati says understanding telomere shortening is "considered the 'Holy Grail' of the biology of aging".

As significant as telomere shrinking seems to be, the failure of our biology as we age is no doubt a complicated matter involving far more than lost pieces of chromosomes.

Reactivation of telomerase is also a trick used by cancers to remain ahead of the growth-curve, making this holy grail a potentially poisoned chalice we need to understand better before drinking too heavily from.

Excitingly, research like this will help us develop a better picture of the aging process.

This research was published in Aging.

Read more:
In a First, Scientists Say They've Partially Reversed a Cellular Aging Process in Humans - ScienceAlert

Recommendation and review posted by Bethany Smith

Embargoed by the Journal of Clinical Investigation until

Thursday, Nov. 19, 2020 at 12 p.m. EST

Newswise November 19, 2020 - Canadian scientists looking at a rare genetic mutation think they may have discovered the proverbial fountain of youth. But its not for everyone: so far, the mutation has only been found in a handful of French-Canadian families.

Called PCSK9Q152H, the mutation of the PCSK9 gene was initially thought to protect against cardiovascular diseases. Recent studies reveal that it may protect against other human illnesses, mainly liver diseases. It may allow the lucky PCSK9Q152H mutant subjects to stay in good health and live longer, the researchers say in a study published today in the prestigious Journal of Clinical Investigation.

The work was led by vascular biologists Richard Austin and Paul Lebeau of McMaster University and by Montreal Clinical Research Institute endocrinologist Michel Chrtien, an emeritus professor at the University of Montreal.

Gene therapy next?

These are exciting findings what weve found may represent a kind of fountain of youth, said Austin, a medical professor and one of the senior authors of the study. Now we want to see whether we can come up with a gene therapy approach to overexpress this specific mutant gene variant in the liver, and thereby offer an innovative treatment for a number of diseases that normally lead to early death.

News of the PCSK9Q152H mutation was first published in 2011, after Chrtien, the current studys lead clinical investigator, discovered it in a French-Canadian family. Mainly expressed in the liver, the mutated gene lowers a persons plasma LDL-cholesterol (bad) and prevents cardiovascular diseases. Chrtien and his IRCM colleague Hanny Wassef found it in two other large French-Canadian families, as well.

People carrying this gene mutation are surprisingly healthy well into their late 80s and mid-90s. In addition to their low plasma LDL-cholesterol and their low risk of cardiovascular diseases, their liver function is completely normal when measured through imaging and a complete medical evaluation, the Montreal scientists found.

Until now, however, the underlying mechanism by which the mutation represented a health benefit other than cardiovascular was a mystery.

A surprising protective effect

In the new study, the McMaster researchers show that overexpression of this gene variant in the livers of mice who dont carry PCSK9Q152H had a surprising protective effect against injury and dysfunction of their liver. As well, overexpression led to a large reduction in their circulating PCSK9 levels just as it does in people, decreasing individuals bad cholesterol and keeping them in good cardiovascular health.

When we initiated these studies, we had speculated that introducing the mutant PCSK9Q152H protein into the liver of mice would cause liver injury or dysfunction, Austin said. But to our amazement, added Lebeau, overexpression of the mutant gene variant in the livers of mice failed to cause stress in the cellular manufacturing and packaging system called the endoplasmic reticulum, or ER, and actually protected against ER stress-induced liver injury.

In their laboratory, the McMaster scientists went on to show that the mutant gene variant acts as a unique co-chaperone protein to stabilize several well-known ER chaperones, namely GRP78 and GRP94, and to increase their protective activity against liver damage.

Particularly gratifying results

These results from Dr. Austins group are particularly gratifying since they experimentally explain that this gene mutation, known to lower cardiovascular accidents, also protects against liver injury and dysfunction, even in individuals who are in their late 80s and mid-90s, said Chrtien, also an emeritus scientist at the Ottawa Hospital Research Institute.

Furthermore, these findings should allow us to determine whether this unique mutation provides additional protection against liver diseases such as cancer, over and above its protective effect against cardiovascular accidents.

About this study

The article The loss-of-function PCSK9Q152H variant increases ER chaperones GRP78 and GRP94 and protects against liver injury , by Paul Lebeau and his collaborators will be published in the Journal of Clinical Investigation, on January 4, 2021.

The study was funded in part by the Canadian Institutes of Health Research and the Heart and Stroke Foundation of Canada, the Fondation J-Louis Lvesque, the Richard and Edith Strauss Foundation, the Fondation Notre-Dame de Zeitoun, and the Aclon Foundation.

-30-

Original post:
A gene mutation that protects against disease - Newswise

Recommendation and review posted by Bethany Smith

The Personalized Gene Therapy Treatment Market research report recently presentedby Prophecy Market Insights which provides reliable and sincere insights related to the various segments and sub-segments of the market. The market study throws light on the various factors that are projected to impact the overall dynamics of the Personalized Gene Therapy Treatment market over the forecast period (2019-2029).

Influences of the market report:

An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Personalized Gene Therapy Treatment market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

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Australia, New Zealand, Rest of Asia-Pacific

Stakeholders Benefit:

Segmentation Overview:

The Personalized Gene Therapy Treatment research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Personalized Gene Therapy Treatment market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Highlights of the Report

Complete access to COVID-19 Impact on the Personalized Gene Therapy Treatment market dynamics, key regions, market size, growth rate and forecast to 2029

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Some important Questions Answered in Personalized Gene Therapy Treatment Market Report are:

Personalized Gene Therapy TreatmentMarket by Top Manufacturers:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

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Personalized Gene Therapy Treatment Market Dynamicas and Opportunity Analysis till 2030 - TechnoWeekly

Recommendation and review posted by Bethany Smith

Industrial Growth Forecast Report Hemophilia Gene Therapy Market 2020-2027: The Global Hemophilia Gene Therapy Market Report provides Insightful information to the clients enhancing their basic leadership capacity identified with the global Hemophilia Gene Therapy Market business, including market dynamics, segmentation, competition, and regional growth. The strategy of expansion has been adopted by key players who are increasing their production capacities to cater to the increasing demand for various application.

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Read more here:
Hemophilia Gene Therapy Market Industry Outlines, Future Trends, Insight And Quality Analysis And Sustainable Growth Strategy Over 2026 - PRnews...

Recommendation and review posted by Bethany Smith

Just a few months after withdrawing its IPO filing, 4D Molecular Therapeutics is seeking to go public once again.

The Emeryville, CA-based company submitted a new S-1 on Tuesday, detailing plans for a $75 million raise as it aims for the second time to hit the Nasdaq. 4DMT had previously sought a $100 million IPO back in September 2019, but withdrew the filing in July of this year after completing a $75 million Series C in June.

Should 4DMT complete the transition to a public company this time around, theyll join a crowded IPO party thats lasted nearly the entire year.

Nasdaq head of healthcare listings Jordan Saxe provided the most recent tally for biotech IPOs in late October, counting 72 companies going public at the time. Combined, those outlets have raised roughly $13.2 billion. The debuts have slowed since the summer, but Saxe pegged a fair estimate of 75 IPOs and just under $14 billion in proceeds to round out 2020.

Several factors have contributed to this years wave, Saxe previously told Endpoints News, as the Covid-19 pandemic has highlighted innovation and crossover investors have steadily increased biotech investments in the second half of the 2010s. The pandemic economy has also made biotech companies more appealing given that theyre less reliant on quarter-to-quarter sales numbers.

In the last four years, only 2018 comes close in terms of the sheer amount of biotechs shooting for Wall Street. That years tab totaled 56 IPOs, according to independent analyst Brad Loncar.

Within the new S-1, 4DMT didnt provide too much detail about how much money theyd spend on each of their programs. The company did list, however, that ongoing clinical trials for their leading programs 4D-310 and 4D-125 would be their top priority. Both of those candidates are currently in Phase I/II with data likely coming next year.

The main research driving the company has been building out a base of more than a billion vector capsid sequences, which CEO David Kirn said in June needed years to take place. 4DMT needed that time to run the sequences through non-human primates to see which shells were the least toxic and most likely to prevent antibody resistance.

By doing so, the biotech hopes this screening model can help find the capsids most suitable for the vector delivery of gene therapies.

4DMTs lead candidate, 4D-310, is intended to treat Fabry disease, with the goal of initially treating early onset versions before expanding into severe, late-onset patients. 4D-125, meanwhile, has the goal of treating an inherited vision loss disorder called XLRP. Roche has partnered with 4DMT to in-license the program before it begins a pivotal trial.

The biotech is also conducting a Phase I study in 4D-110, which is targeted at patients with choroideremia related to mutations in the CHM gene.

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4DMT shoots for a $75M IPO, its second attempt to go public with its gene therapy vector programs - Endpoints News

Recommendation and review posted by Bethany Smith

BASEL, Switzerland & MINNEAPOLIS--(BUSINESS WIRE)--Be The Match BioTherapies and Lonza:

Quote from Alberto Santagostino, SVP, Head of Cell and Gene Therapy, Lonza:

"Be The Match BioTherapies brings deep experience in overcoming supply chain and logistical challenges impacting developers of cell and gene therapies, including factors that delay patients' ability to access the treatments they need. We're pleased to add their team to our growing network of strategic partners and look forward to working together to cultivate a more seamless, streamlined ecosystem for cell and gene therapy development."

Quote from Chris McClain, MBA, SVP, Sales and New Business Development, Be The Match BioTherapies:

"Lonza plays a central role in Be The Match BioTherapies' pursuit of accelerating patient access to high-quality manufactured cell therapies. This collaboration will allow our teams to combine our substantial expertise and resources across the cell therapy supply chain to ease the logistical burden for cell and gene therapy developers globally. By offering the ability to leverage a fully integrated cell and gene therapy supply chain, we can ultimately provide a brighter future for patients."

Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, and Lonza today announced a strategic partnership to integrate industry-leading solutions in CGT. The goal of the collaboration is to improve efficiency across the CGT supply chain, inclusive of apheresis network management, healthy donor tissue supply, manufacturing and, where appropriate, supply chain management and logistics.

The partnership establishes Be The Match BioTherapies and Lonza as preferred partners and is aimed at supporting the companies' shared goal of providing end-to-end solutions that streamline the development of cell and gene therapies across the CGT supply chain. Lonza and Be The Match BioTherapies will integrate each other's services in their respective offerings to provide a seamless offering to customers.

This collaboration builds on current partnerships announced by Lonza and Be The Match BioTherapies to build a seamless, vein-to-vein network for customers and their patients. Partners of Lonza include Cryoport, a leading temperature-controlled supply chain company, and Vineti, a company developing the first commercial, configurable, cloud-based digital platform to orchestrate advanced therapy supply chains. Be The Match BioTherapies brings expertise in successfully delivering more than 100,000 cell therapies, expertise in logistics, managing apheresis networks, and the ability to provide high-quality cellular source material from the Be The Match Registry which offers the world's largest, most ethnically diverse listing of more than 22 million potential blood and marrow donors, with Lonza's industry-leading capabilities in cell and gene therapy manufacturing

About Lonza

At Lonza, we combine technological innovation with world class manufacturing and process excellence. Together, these enable our customers to deliver their discoveries in the healthcare, preservation, and protection sectors.

We are a preferred global partner to the pharmaceutical, biotech and specialty ingredients markets. We work to prevent illness and promote a healthier world by enabling our customers to deliver innovative medicines that help treat or even cure a wide range of diseases. We also offer a broad range of microbial control solutions, which help to create and maintain a healthy environment.

Founded in 1897 in the Swiss Alps, Lonza today operates in 120 sites and offices in more than 35 countries. With approximately 15,500 full-time employees, we are built from high-performing teams and of individual employees who make a meaningful difference to our own business, as well as the communities in which we operate. The company generated sales of CHF 5.9 billion in 2019 with a CORE EBITDA of CHF 1.6 billion. Find out more at http://www.lonza.com and follow us on Twitter @LonzaGroup or Facebook @LonzaGroupAG.

About Be The Match BioTherapies

Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program (NMDP)/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the world's largest and most diverse registry of more than 22 million potential blood stem cell donors and more than 300,000 umbilical cord blood units. Through established relationships with apheresis, marrow collection and transplant centers worldwide, the organization develops, onboards, trains and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven integrated model of both cell therapy supply chain and logistics managers, complimented by regulatory compliance experts to successfully transport and deliver life-saving therapies across the globe. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter at @BTMBioTherapies.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

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Lonza and Be The Match BioTherapies Partner to Expand Vein-to-Vein Cell and Gene Therapy Supply Chain Network - Business Wire

Recommendation and review posted by Bethany Smith

Its PODCAST Thursday! New episode of The Breast of Everything out now!

On this episode, Comprehensive Breast Care surgeon Ashely Richardson, DO, talks with patient Shana Carter about her experience with genetic testing.

Through genetic testing, Shana, a Young Fives teacher and mother of two, learned she had a mutation in her ATM gene, placing her in the high-risk category for developing breast cancer. On this episode, she chats about her cancer risk, and why she feels a prophylactic mastectomy was the best option for her. The decision to take my breasts was an easy one for me, says Shana. I wanted to live as long as humanly possible.

Many of my patients who were in a similar situation like Shanas ask me, What would you do? says Ashley. I cant answer that question, everyones personal experience and situation is different. Ashley goes on to point out that, Genetic testing covers more than 50 different genes that can be related to breast cancer and other cancers. Testing is much more accessible and affordable, and we know so much more now that we did 11 years ago.

At Comprehensive Breast Care, we recommend genetic testing to all breast cancer patients, all high-risk patients and all first-degree relatives who have a gene mutation. says Ashley. And for Sarah, that testing is potentially life changing, I now have less than a 1 percent chance of developing breast cancer.

To hear the full episode, TUNE IN at https://bit.ly/3fc4Fey, orwww.compbreastcare.comApplePodcasts, GooglePlay, or listen wherever you get your podcasts.

If you are concerned you may have breast cancer, or want to know more about genetic testing, go to https://www.compbreastcare.com/ or call, 248-687-7300.

More:
Local Announcement: Why I did Genetic Testing - NEW Breast of Everything Podcast - Patch.com

Recommendation and review posted by Bethany Smith

-- Study finds nearly 1 in 10 show genetic changes with established clinical management recommendations, potential eligibility for precision therapies and/or clinical treatment trials --

-- Data presented at the National Society of Genetic Counselors Annual Conference, which will also include ceremony honoring excellence in genetic counseling patient care --

SAN FRANCISCO, Nov. 19, 2020 /PRNewswire/ -- Invitae (NYSE: NVTA), a leading medical genetics company, today presented study findings that show nine percent of patients with pancreatic cancer had genetic changes in DNA damage repair (DDR) genes that would make them eligible for PARP inhibitor therapy or clinical treatment trials. Despite professional guidelines that recommend testing for all pancreatic cancer patients, it remains underutilized in routine care. The study was presented at the National Society of Genetic Counselors 39th Annual Conference.

"New therapeutics have recently become available to treat pancreatic cancer for patients with certain changes in genes such as BRCA1 and BRCA2. Yet despite the availability of these treatments and professional guidelines recommending testing, utilization is still lagging," said Robert Nussbaum, M.D., chief medical officer of Invitae and study author. "Pathogenic variants in these genes are associated with an increased risk of other cancers as well, such as breast, ovarian and prostate cancer, which means that a failure to test patients with pancreatic cancer impacts not only their treatment, but also the health of their families."

Importantly, the study of over 2,000 patients found that 15% of patients with actionable genetic changes reported no family history of cancer, which underscores the limitations of using testing criteria based on reported family history. National Comprehensive Cancer Network (NCCN) guidelines recommend genetic counseling and germline genetic testing for everyone diagnosed with pancreatic cancer as well as their first degree relatives -- approximately 3.5 million individuals in the United States.

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In addition to evaluating the clinical relevance of genetic testing results, the study offered sponsored, no-charge testing to patients to evaluate the role of cost as a barrier to testing. Researchers found a small but significant increase (2%) in testing among African-American patients compared to typical rates among patients using health insurance, suggesting reducing cost may increase access to testing among this population.

The research was presented at the virtual annual meeting of the National Society of Genetic Counselors. The full research presentation from Invitae included:

Oral platform presentations:

Increasing access for patients with pancreatic cancer to germline genetic testing: Clinical impacts across disease stage and ethnicity. Presented by Ed Esplin, MD, PhD, FACMG, FACP

Evaluating variant reclassification in reproductive carrier screening. Presented by Julia Wilkinson, MS, LCGC

Poster presentations:

Utility of adding phenotypic criteria refinement to ACMG guidelines. Presented by Lauren Frank, MS, CGC

Comprehensive germline multigene panels change clinical care and inform treatment strategies for breast cancer patients. Presented by Sarah Nielsen, MS, LCGC

Carrier screening for X-linked conditions is common practice. Presented by Dana Neitzel, MS, CGC

In addition to its scientific presence, Invitae will again partner with NSGC to present the Heart of Genetic Counseling award, which honors excellence in genetic counseling and patient care as recognized by patients. Nominations include stories from patients that highlight both the clinical and personal impact a genetic counselor had on their lives and the lives of their families. This year's award will be presented during a virtual ceremony on Thursday, November 17th. The finalists include:

Rachelle Manookian, MS, LCGC, City of Hope in Duarte, CA, was nominated by a man who had been searching for answers about the origin of his cancer for more than 10 years. He hoped that any identified mutations would lead to a newly developed drug or clinical trial that could attack his cancer with precision. Manookian left no stone unturned and remained in touch until she found a clinical trial. Thanks to Manookian's persistence, he finally got answers and learned that his sons did not carry the same increased genetic risk of prostate cancer.

Donna McDonald-McGinn, MS, LCGC, Children's Hospital of Philadelphia in Philadelphia, was nominated by the mother of a son with a rare genetic condition called 22q11.2 deletion syndrome. McDonald-McGinn has been his care coordinator and navigator for 21 years, explaining and managing the care that he has needed through his life. Always acting as his advocate, McDonald-McGinn has been there for each of his 21 surgeries and is a staunch advocate for those with 22q11.2 deletion syndrome.

Gretchen MacCarrick, MS, CGC, Johns Hopkins Medicine in Baltimore, was nominated by a mother whose daughter has Loeys-Dietz syndrome (LDS), which was discovered in 2005. For nearly 12 years, MacCarrick has been by their side guiding them. She explains what tests are needed, what the results mean and navigates their daughter's care during their regular visits to Baltimore, including a major heart surgery. Importantly, MacCarrick has even developed a strong relationship directly with the patient who is now 16 and getting more engaged in her care.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the implications of the company's study results; and the importance and potential benefits of genetic testing for pancreatic cancer patients. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:

Laura D'Angelopr@invitae.com(628) 213-3283

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SOURCE Invitae Corporation

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New Study Highlights the Importance of Genetic Testing for Pancreatic Cancer Patients - Yahoo Finance

Recommendation and review posted by Bethany Smith