Monthly Archives: May 2021

SAN DIEGO, May 11, 2021 /PRNewswire/ -- Poseida Therapeutics, Inc.(Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today will give multiple oral and poster presentations at the American Society of Gene and Cell Therapy 2021 Virtual Annual Meetingbeing held May 11-14, 2021.

The Company's oral presentation will highlight new data demonstrating the potential of its proprietary piggyBac DNA Delivery System for the treatment of genetic liver disorders in children and infants. Two additional presentations will highlight preclinical data supporting Poseida's first allogeneic CAR-T product candidate, P-BCMA-ALLO1 for R/R multiple myeloma, as well as preclinical data supporting the Company's anti-c-kit CAR-T program as a potentially safer preconditioning regimen for hematopoietic stem cell transplantation in patients with AML.

"At Poseida, we are applying our proprietary technology platforms to develop the next wave of cell and gene therapies to not only treat severe cancers but to also unlock the potential of single treatment cures," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer. "The preclinical data being presented today further validate our CAR-T approach in multiple myeloma and in preconditioning for patients with AML, as well as demonstrate the exciting potential of our liver directed gene therapies, particularly in juvenile patients."

Presentation Highlights:

Oral Presentation: "Preclinical Evaluation of Combined Adeno-Associated Virus and Nanoparticle Delivery of piggyBac Transposon System for Durable Transgene Expression in the Growing Neonatal Murine Liver" Session Date/Time: Tuesday, May 11, 2021, 5:45pm - 6:00pm ETAbstract Number: 30

In a preclinical study, Poseida evaluated concomitant delivery of recombinant adeno-associated virus (rAAV) vectors and novel nanoparticle (NP) vectors using its piggyBac and "Super" piggyBac (SPB) technologies in order to deliver transposon and transposase in a growing neonatal mouse model. Data demonstrated that the piggyBac DNA Delivery System was effective in using both rAAV and NP vectors to introduce edited genes into targeted hepatocyte genomes. Poseida also found that SPB, a hyperactive form of the transposase, produced stable vector integration into the hepatocyte genome for more than three months, compared to transpose alone. Similarly, delivery of a novel NP formulation using SPB produced efficient delivery of mRNA to the liver hepatocytes, with similarly high levels of durability in the transgene expression. Taken together, these preclinical findings suggest the potential of piggyBac and SPB technology for gene therapies that treat congenital liver disease in infants and young children.

Poster Presentation: "P-BCMA-ALL01: A Fully Allogeneic Stem Cell Memory T Cell (TSCM) CAR-T Therapy Targeting BCMA for the Treatment of Multiple Myeloma Shows Potent Anti-Tumor Activity"Session Date/Time: Tuesday, May 11, 2021, 8:00am 10:00am ETAbstract Number: 789

P-BCMA-ALLO1 is Poseida's first fully allogenic product candidate targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. In in vitro and in vivo preclinicalstudies, P-BCMA-ALL01 showed effective, targeted cancer cell killing and cytokine secretion, with similar or superior performance in anti-tumor efficacy compared to an autologous CAR-T therapy. Inclusion of a proprietary "booster molecule" in the allogeneic manufacturing process further improved expansion of gene-edited cells and enabled production of hundreds of patient doses from a single manufacturing run, thereby reducing the manufacturing cost per dose into the same range as that of a monoclonal antibody.

Poster Presentation: "Anti-c-kit CAR-T Cells Afford Effective Eradication of Human AML and Normal Hematopoietic Cells in a Preclinical Model of Safer Non-Genotoxic Stem Cell Transplant Conditioning"Session Date/Time:Tuesday, May 11, 2021, 8:00am 10:00am ETAbstract Number:715

Poseida is investigating its anti-c-kit CAR-T program, which leverages its proprietary piggyBac DNA Delivery System in preclinical studies as a potentially safer precursor conditioning therapy to the transplantation of hematopoietic stem cells (HSC) for patients suffering from AML. The piggyBac delivery vectors under investigation include a transposon that generates pure CAR+ product as well as a safety switch that allows rapid clearance of the reactive CAR-T cells prior to donor transplant of hematopoietic stem cells. Preclinical data to be presented in the poster showed that the lead CAR-T cells that express the anti-c-kit binder (CAR 1) deplete up to 92% of human CD34+ stem and progenitor cells in bone marrow within 48 hours. Additionally, an enhanced anti-c-kit CAR-T product, CAR 2, killed an estimated >99% of leukemia cells, exceeding the killing ability of a single dose of 30 mg/kg dose of busulfan. These encouraging data suggest that stem cell-directed CAR-T cells may be a safer preconditioning regimen compared to the current standard of care and may expand access to treatment for acute myeloid leukemia patients needing HSC transplant.

AboutPoseida Therapeutics, Inc.Poseida Therapeuticsis a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary geneticengineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBacDNA DeliverySystem, Cas-CLOVER Site-specific Gene Editing System and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.To learn more, visitwww.poseida.comand connect with us onTwitterandLinkedIn.

Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regardingthe clinical data presented, the potential benefits ofPoseida'stechnology platforms and product candidatesandPoseida'splans and strategy with respect to developing its technologies and product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based uponPoseida'scurrent expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry, the fact that future clinical results could be inconsistent with results observed to dateand the other risks described inPoseida'sfilings with theSecurities and Exchange Commission. All forward-looking statementscontained in this press release speak only as of the date on which they were made.Poseidaundertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

SOURCE Poseida Therapeutics, Inc.

http://www.poseida.com

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Poseida Therapeutics Presents Encouraging Preclinical Data Across its CAR-T and Gene Therapy Programs at the American Society of Gene and Cell Therapy...

DUBLIN, May 10, 2021 /PRNewswire/ -- Irish based cell engineering company, Avectas today announces the publication of 'A novel non-viral delivery method that enables efficient engineering of primary human T cells forex vivocell therapy applications' in leading international cell and gene therapy publication, Cytotherapy.1

Cytotherapy is the official journal of the International Cell & Gene Therapy Society (ISCT).

Dr Shirley O'Dea et al. examined the suitability of Avectas' SOLUPORE non-viral delivery system for engineering primary human T cells for cell therapy applications.This publication describes how the next generation of immune cell therapy products will require complex modifications using engineering technologies that can maintain high levels of cell functionality and how non-viral engineering methods such as SOLUPORE have the potential to address limitations associated with viral vectors.

Data demonstrates efficient transfection of human primary T Cells with mRNA and CRISPR CAS9 RNP cargos while consistently maintaining high levels of cell viability. Gene expression profiling revealed minimal up or down regulation of genes, demonstrating the low level of perturbation experienced by the cells during this transfection process in contrast to electroporation which resulted in substantial changes in immune gene expression. CAR T cells engineered using the SOLUPOREsystem exhibited high cytotoxicity against target cancer cellsin vitro and in vivo.

Avectas has previously published on the SOLUPORE Technology in PLOS ONE2 with citations in both Nature Biomedical Engineering3 and Chemical Reviews4.

Commenting on the publication, Dr Michael Maguire, CEO of Avectas, said: "It is impressive to see further published evidence showing the advantages of the SOLUPOREplatform in preserving cellular functionality essential for cell therapies. Congratulations to Avectas CSO, Dr Shirley O'Dea and the Avectas R&D team on this additional publication of important work to support the efficacy and potential ofSOLUPORE technology to the cell and gene therapy industry."

Using its patented SOLUPORE technology, Avectas partners with leading cell and gene therapy companies and research institutions to enable the next-generation of gene-modified cell therapies.

1. https://www.sciencedirect.com/science/article/pii/S1465324921001845

2. https://journals.plos.org/plosone/article/authors?id=10.1371/journal.pone.0174779

3. https://www.nature.com/articles/s41551-018-0246-6

4. https://pubs.acs.org/doi/abs/10.1021/acs.chemrev.7b00678

About Avectas

Avectas is a cell engineering company that has pioneered - SOLUPORE- a proprietary, simple, highly effective automated non-viral cell engineering system to enable efficient and safe genetic modification, accelerating the manufacture of cells for the next generation of immuno-oncology therapies.

The company is partnering its cGMP aligned SOLUPOREclinical-grade system with cell therapy companies to address emerging cell delivery challenges associated with multiple cell modifications and engineering of limited or fragile cells. Additionally, it will provide regulatory support to partners. Avectas is a private, international company with research facilities inDublin, IrelandandToronto, Canada, and an office inCambridge, MA, USA. It has over 30 employees and is led by a team of highly experienced pharmaceutical executives supported by a world-class Scientific Advisory Board of cancer cell therapy experts.

For further information, seewww.avectas.com.Follow us onLinkedInandTwitter.

SOURCE Avectas

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Avectas announces publication in leading cell therapy journal Cytotherapy of 'A novel non-viral delivery method that enables efficient engineering of...

Climate change, growing populations and less arable land means advanced farming practices are more important than ever before.

While many in agriculture showed collective outrage over the so-called Impossible Burger, science supporters missed a critical benefit hiding in the controversy. The Impossible Burger is one of the few consumer-facing companies that proudly boasts GMO ingredients and customers still flock to it.

The team at Impossible Burger showcases the climate-saving benefits biotechnology can provide by showcasing the value of soy leghemoglobin the key protein element that gives the Impossible Burger its flavor and texture is a genetically engineer protein. Soy leghemoglobin is made by splicing soybean DNA into yeast, which is then produced in large-scale fermentation.

Seeing this market success, why arent GMOs widely accepted?

Frankly, because agriculturalists from the farm gate, to the lab, to the desks of CEOs have done a bad job telling the story.

I think its everybodys responsibility who is involved in agriculture to be involved in communicating, says Robb Fraley, who commercialized the first genetically modified soybean and now acts as a communicator for the industry. When I was growing up on a small farm in Illinois, more than half of the people who lived in the state were involved in agriculture. Today its less than 1%.

Its the responsibility of everyone involved in food and agriculture to reach out to the other 99% regarding the importance of new scientific advances to increase food production and improve the sustainability of farming, he continues.

The Challenge

Think back to the early 1990s when the FLAVR SAVR tomato was commercialized albeit short lived. It carried promise to reduce waste and improve flavor, a promise that never came to fruition.

When the first GMO product, the FLAVR SAVR tomato, came out people had no idea what GMO even meant, says Kurtis Baute, climate change activist who produces frequent videos promoting science. It was something that really came out of the dark for them, and it was surprising and scary for many. Because of that, we ended up shelving the idea.

The long and short of it, if you need a reminder, is public backlash led to pulling FLAVR SAVR tomatoes from the shelf despite the scientific reality there was no risk in consuming them. People didnt know what they were, and ignorance bred fear, which manifested itself into nixing the product.

Fear lives on. Activist groups around the world are still fighting against GMOs, despite what has now been more than 20 years of research indicating no adverse human or environmental effects.

And the stakes are high. GMO products could be the key to reducing the impact of climate change and tackling hunger and food insecurity around the world. If science isnt trusted, GMOs and other modern practices are at risk.

Start the Conversation

For many scientists and experts in their fields, its easy to overload people with facts. But the fact is, most people who dont have scientific backgrounds dont care about the nitty-gritty details of scientific testing as an expert would. It takes a different approach.

Anytime you are promoting scientific ideas, youre going to have people who are not excited about them, says Kevin Folta, professor at the University of Florida. It applies to all science, whether youre talking about genetic engineering or climate change.

Before starting the conversation, think about how to be a better communicator. It starts by figuring out what neighbors, friends, relatives and, really, anyone cares about. Here are a few tips to get the conversation started the right way:

Its tough to convert someone to 100% stand behind science, but its possible to sway them on several aspects. Understand their backgrounds and why they think the way they do. Employing these three tactics helps get the conversation off on the right foot.

Where to Reach People

The world of communications has changed dramatically since the early 90s when the first GMOs were commercialized. This presents immense opportunity to reach a wide array of people, but that opportunity is not without challenge.

When I first joined Monsanto, you published papers, did newsprint and that kind of thing, Fraley says. Today most of the information people get is on social media its digital. If youre not on social media, basically, your voice doesnt count.

Social media takes the audience from one or two people, to potentially millions. However, its not just any post that will capture the attention of the masses communication looks different online.

Ive basically made a career by building over-the-top projects, explains Baute. For example, I set up 13,799 dominoes so I could show a timeline of the history of the universe. It was a spectacle that engaged people in personal storytelling so I could talk about real science.

There are amazing stories to be told we shouldnt miss that opportunity, he continues. For example, if theres a new product engage people in personal storytelling about how these products came to be. For some technology, it could be 100 years in the making.

Not everyone will want to set up nearly 14,000 dominos or live in a bubble like Baute did to talk about climate change, but theres opportunities to find a fun way to talk about science all the same. Perhaps it makes more sense to tell someone about a personal connection to agriculture and science. Find the story and tell it.

I once baked a cake in 418 steps to explain genetics, Baute says. Sometimes it helps to have a little fun when explaining complex topics.

Protect Science for Tomorrow

Its never been more critical than today to advocate for science literacy. While social media, television and other platforms represent opportunity to talk about science, it also dispels a tremendous amount of misinformation.

We need to be champions and advocates across the biological, the digital and the agronomic space to amplify the accurate messages, Fraley says. Because its just 1% of the population in agriculture reaching out to the 99%.

Europes Science Desert

Scientists looking for new opportunities to apply the latest and greatest technologies in agriculture: steer clear of the European Union. Policies in the region are virtually devoid of scientific reason and instead are political decisions based on pressure from activist groups, along with misunderstanding and emotions.

For example, the 27-member countries are facing a new proposal called the Farm to Fork Strategy that threatens producer access to technologies and could force them to abandon common farming techniques.

Im not sure there was much science involved [in creating the Farm to Fork Strategy], says David Zaruk, EU risk and science communications specialist. The proposed strategy, in a nutshell, would reduce pesticide use by 50%, fertilizers by 20% and convert 25% of all agricultural land to organic production under the assumption these moves would reduce the impact of climate change.

This isnt the first-time agricultural practices have come under fire in the EU either. The countries previously passed regulations that require all products made with gene editing technology to be labeled as GMO regardless of whether or not new genes were introduced in the process.

Researchers in the EU who oppose such rules are between a rock and a hard place, Zaruk explains. Many scientists who support GMOs, gene editing, pesticide use and other common agricultural technologies find themselves without jobs or under attack personally from activist groups.

Most of the scientist that do speak out are retired, he continues. Regardless of the risk, Zaruk still speaks out about the benefits of changing policies to enable farmers to use the latest agricultural technologies available and encourages other scientists to do the same.

The EU can, and should, serve as a warning to other countries about what happens when science is ignored in the policy arena and what happens when agriculturalists arent consulted when planning policy.

Canadas Gene Editing Conundrum

The clock is ticking in Canada. At the end of May, the comment period regarding Canadas Novel Food Regulations will be over and a decision that could make or break the future of plant breeding will be rendered.

This decision will directly impact how gene editing, through technologies such as CRISPR/Cas9, is used and identified to consumers. The question? Should there be additional regulations for crops created by gene editing like the regulations applied to GMO crops?

Cas9 [based gene editing] is a great example of something thats happened naturally for billions of years and now were applying a process to our benefit, says Kurtis Baute, a Canadian native who advocates for science literacy and improving the climate on various channels. Its important to communicate the benefit. This technique will be more precise, and well know exactly what were breeding for, compared to older breeding techniques.

CRISPR/Cas9 and other gene editing technologies could revolutionize the breeding industry if given the latitude to be used. The precision associated with the technique results in better products, faster production and the ability to cater breeding to the needs of todays farmer and consumer.

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Want GMOs, Gene Editing and Access to New Science? Communication is Key - Seed World