Monthly Archives: January 2021

CRISPR and the Splice to Survive – The New Yorker

Posted: January 13, 2021 at 4:17 pm

Odin, in Norse mythology, is an extremely powerful god whos also a trickster. He has only one eye, having sacrificed the other for wisdom. Among his many talents, he can wake the dead, calm storms, cure the sick, and blind his enemies. Not infrequently, he transforms himself into an animal; as a snake, he acquires the gift of poetry, which he transfers to people, inadvertently.

The Odin, in Oakland, California, is a company that sells genetic-engineering kits. The companys founder, Josiah Zayner, sports a side-swept undercut, multiple piercings, and a tattoo that urges: Create Something Beautiful. He holds a Ph.D. in biophysics and is a well-known provocateur. Among his many stunts, he has coaxed his skin to produce a fluorescent protein, ingested a friends poop in a D.I.Y. fecal-matter transplant, and attempted to deactivate one of his genes so that he could grow bigger muscles. (This last effort, he acknowledges, failed.) Zayner calls himself a genetic designer and has said that his goal is to give people access to the resources they need to modify life in their spare time.

The Odins offerings range from a Biohack the Planet shot glass, which costs three bucks, to a genetic engineering home lab kit, which runs almost two thousand dollars and includes a centrifuge, a polymerase-chain-reaction machine, and an electrophoresis gel box. I opted for something in between: the bacterial CRISPR and fluorescent yeast combo kit, which set me back two hundred and nine dollars. It came in a cardboard box decorated with the companys logo, a twisting tree circled by a double helix. The tree, I believe, is supposed to represent Yggdrasil, whose trunk, in Norse mythology, rises through the center of the cosmos.

Inside the box, I found an assortment of lab toolspipette tips, petri dishes, disposable glovesas well as several vials containing E. coli and all Id need to rearrange its genome. The E. coli went into the fridge, next to the butter. The other vials went into a bin in the freezer, with the ice cream.

Genetic engineering is, by now, middle-aged. The first genetically engineered bacterium was produced in 1973. This was soon followed by a genetically engineered mouse, in 1974, and a genetically engineered tobacco plant, in 1983. The first genetically engineered food approved for human consumption, the Flavr Savr tomato, was introduced in 1994; it proved such a disappointment that it went out of production a few years later. Genetically engineered varieties of corn and soy were developed around the same time; these, by contrast, have become more or less ubiquitous.

In the past decade or so, genetic engineering has undergone its own transformation, thanks to CRISPRshorthand for a suite of techniques, mostly borrowed from bacteria, that make it vastly easier for biohackers and researchers to manipulate DNA. (The acronym stands for clustered regularly interspaced short palindromic repeats.) CRISPR allows its users to snip a stretch of DNA and then either disable the affected sequence or replace it with a new one.

The possibilities that follow are pretty much endless. Jennifer Doudna, a professor at the University of California, Berkeley, and one of the developers of CRISPR, has put it like this: we now have a way to rewrite the very molecules of life any way we wish. With CRISPR, biologists have already createdamong many, many other living thingsants that cant smell, beagles that put on superhero-like brawn, pigs that resist swine fever, macaques that suffer from sleep disorders, coffee beans that contain no caffeine, salmon that dont lay eggs, mice that dont get fat, and bacteria whose genes contain, in code, Eadweard Muybridges famous series of photographs showing a horse in motion. Two years ago, a Chinese scientist, He Jiankui, announced that he had produced the worlds first CRISPR-edited humans, twin baby girls. According to He, the girls genes had been tweaked to confer resistance to H.I.V., though whether this is actually the case remains unclear. Following his announcement, He was fired from his academic post, in Shenzhen, and sentenced to three years in prison.

I have almost no experience in genetics and have not done hands-on lab work since high school. Still, by following the instructions that came in the box from the Odin, in the course of a weekend I was able to create a novel organism. First I grew a colony of E. coli in one of the petri dishes. Then I doused it with the various proteins and bits of designer DNA Id stored in the freezer. The process swapped out one letter of the bacterias genome, replacing an A (adenine) with a C (cytosine). Thanks to this emendation, my new and improved E. coli could, in effect, thumb its nose at streptomycin, a powerful antibiotic. Although it felt a little creepy engineering a drug-resistant strain of E. coli in my kitchen, there was also a definite sense of achievement, so much so that I decided to move on to the second project in the kit: inserting a jellyfish gene into yeast in order to make it glow.

The Australian Centre for Disease Preparedness, in the city of Geelong, is one of the most advanced high-containment laboratories in the world. It sits behind two sets of gates, the second of which is intended to foil truck bombers, and its poured-concrete walls are thick enough, I was told, to withstand a plane crash. There are five hundred and twenty air-lock doors at the facility and four levels of security. Its where youd want to be in the zombie apocalypse, a staff member told me. Until recently, the center was known as the Australian Animal Health Laboratory, and at the highest biosecurity levelBSL-4there are vials of some of the nastiest animal-borne pathogens on the planet, including Ebola. (The laboratory gets a shout-out in the movie Contagion.) Staff members who work in BSL-4 units cant wear their own clothes into the lab and have to shower for at least three minutes before heading home. The animals at the facility, for their part, cant leave at all. Their only way out is through the incinerator is how one employee put it to me.

About a year ago, not long before the pandemic began, I paid a visit to the center, which is an hour southwest of Melbourne. The draw was an experiment on a species of giant toad known familiarly as the cane toad. The toad was introduced to Australia as an agent of pest control, but it promptly got out of control itself, producing an ecological disaster. Researchers at the A.C.D.P. were hoping to put the toad back in the bottle, as it were, using CRISPR.

A molecular biologist named Mark Tizard, who was in charge of the project, had agreed to show me around. Tizard is a slight man with a fringe of white hair and twinkling blue eyes. Like many of the scientists I met in Australia, hes from somewhere elsein his case, England. Before getting into amphibians, Tizard worked mostly on poultry. Several years ago, he and some colleagues at the center inserted a jellyfish gene into a hen. This gene, similar to the one I was planning to plug into my yeast, encodes a fluorescent protein. A chicken in possession of it will, as a consequence, emit an eerie glow under UV light. Next, Tizard figured out a way to insert the fluorescence gene so that it would be passed down to male offspring only. The result is a hen whose chicks can be sexed while theyre still in their shells.

Tizard knows that many people are freaked out by genetically modified organisms. They find the idea of eating them repugnant, and of releasing them into the world anathema. Though hes no provocateur, he, like Zayner, believes that such people are looking at things all wrong. We have chickens that glow green, Tizard told me. And so we have school groups that come, and when they see the green chicken, you know, some of the kids go, Oh, thats really cool. Hey, if I eat that chicken, will I turn green? And Im, like, You eat chicken already, right? Have you grown feathers and a beak?

Anyway, according to Tizard, its too late to be worried about a few genes here and there. If you look at a native Australian environment, you see eucalyptus trees, koalas, kookaburras, whatever, he said. If I look at it, as a scientist, what Im seeing is multiple copies of the eucalyptus genome, multiple copies of the koala genome, and so on. And these genomes are interacting with each other. Then, all of a sudden, ploomph, you put an additional genome in therethe cane-toad genome. It was never there before, and its interaction with all these other genomes is catastrophic. It takes other genomes out completely. He went on, What people are not seeing is that this is already a genetically modified environment. Invasive species alter the environment by adding entire creatures that dont belong. Genetic engineers, by contrast, just alter a few stretches of DNA here and there.

What were doing is potentially adding maybe ten more genes onto the twenty thousand toad genes that shouldnt be there in the first place, and those ten will sabotage the rest and take them out of the system and so restore balance, Tizard said. The classic thing people say with molecular biology is: Are you playing God? Well, no. We are using our understanding of biological processes to see if we can benefit a system that is in trauma.

Formally known as Rhinella marina, cane toads are a splotchy brown, with thick limbs and bumpy skin. Descriptions inevitably emphasize their size. Rhinella marina is an enormous, warty bufonid (true toad), the U.S.Fish and Wildlife Service notes. The U.S.Geological Survey observes that large individuals sitting on roadways are easily mistaken for boulders. The biggest cane toad ever recorded was fifteen inches long and weighed six poundsas much as a chubby chihuahua. A toad named Big Bette, who lived at the Queensland Museum, in Brisbane, in the nineteen-eighties, was nine and a half inches long and almost as wideabout the size of a dinner plate. The toads will eat almost anything they can fit in their oversized mouths, including mice, dog food, and other cane toads.

Cane toads are native to South America, Central America, and the southernmost tip of Texas. In the mid-eighteen-hundreds, they were brought to the Caribbean. The idea was to enlist the toads in the battle against beetle grubs, which were plaguing the regions cash crop, sugar cane. (Sugar cane, too, is an import; it is native to New Guinea.) From the Caribbean, the toads were shipped to Hawaii. In 1935, a hundred and two toads were loaded onto a steamer in Honolulu, headed for Australia. A hundred and one survived the journey and ended up at a research station in sugar-cane country, in northeast Queensland. Within a year, theyd produced more than 1.5 million eggs. (A female cane toad can produce up to thirty thousand eggs at a go.) The resulting toadlets were intentionally released into the regions rivers and ponds.

Its doubtful that the toads ever did the sugar cane much good. Cane beetles perch too high off the ground for a boulder-size amphibian to reach. This didnt faze the toads. They found plenty else to eat, and continued to produce toadlets by the truckload. From a sliver of the Queensland coast, they pushed north, into the Cape York Peninsula, and south, into New South Wales. Sometime in the nineteen-eighties, they crossed into the Northern Territory. In 2005, they reached a spot known as Middle Point, in the western part of the Territory, not far from the city of Darwin.

Along the way, something curious happened. In the early phase of the invasion, the toads were advancing at the rate of about six miles a year. A few decades later, they were moving at the pace of twelve miles a year. By the time they hit Middle Point, theyd sped up to thirty miles a year. When researchers measured the individuals at the invasion front, they found out why. The toads had significantly longer legs than the toads back in Queensland, and this trait was heritable. The Northern Territory News played the story on its front page, under the headline SUPER TOAD. Accompanying the article was a doctored photo of a cane toad wearing a cape. It has invaded the Territory and now the hated cane toad is evolving, the newspaper gasped. Contra Darwin, it seemed, evolution could be observed in real time.

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Programmable genetics gets more cash as Tessera Therapeutics gets a $230 million infusion – TechCrunch

Posted: at 4:17 pm

Technologists are getting better at coding biology and venture firms are flooding a new generation of startups with cash so they can commercialize their technology bringing in the next wave of genetic innovation.

Tessera Therapeutics, the Boston-based spin-up from Flagship Pioneering, is the latest company to enter the mix with $230 million in new financing to build up its platform for better biological programming.

The round was led by Alaska Permanent Fund Corp., Altitude Life Science Ventures and the second SoftBank Vision Fund, with participation from the Qatari Investment Authority and other undisclosed investors.

Last year, the company took the covers off its gene-writing service, which combined an array of different gene editing, manufacturing and synthesizing technologies to provide more tailored therapeutic instructions to genetic code.

By providing more instructions to genetic material, the company aims to increase the precision of therapies while expanding the number of potential pathogens or mutations they can target, the company said in a statement.

The thesis is similar to the approach taken by companies like Senti Bio, another early-stage biotech company that raised $105 million earlier this month.

The ability to write in the code of life will be a defining technology of this century and drive a fundamental change in medicine. Todays support is a testament to Tesseras outstanding team of scientists and our focus on bringing the extraordinary promise of Gene Writing to patients, said Geoffrey von Maltzahn, CEO and co-founder of Tessera Therapeutics, and a partner at Flagship Pioneering. We look forward to turning this powerful technology into a new category of medicines.

Part of a number of companies focused on gene therapies and gene-editing technologies that have been developed under the Flagship Pioneering umbrella, Tessera Therapeutics focuses on the development of new therapies that will use messenger RNA, targeted fusogenic vectors and epigenetic controllers, according to Flagship Pioneering founder and chief executive Noubar Afeyan, who also serves as the chair and co-founder of Tessera.

While Senti Bio is adding more programming to existing genetic material, Tessera uses mobile genetic elements, the most abundant genetic material in the body to create new vectors for writing and rewriting the human genome.

The company asserts that this represents a breakthrough in genetic engineering, which can build better therapies. Thats because the technology can target very specific sites in the genome to make any substitutions, insertions or deletions in genetic code. Tessera also said that its tech allows for more efficient engineering of somatic cell genomes without double stranded-breaks and with very little reliance on DNA repair pathways.

Gene writing is inspired by and builds upon the shoulders of natures most prevalent class of genes: mobile genetic elements. Tesseras computational and high-throughput laboratory platform has enabled the team to design, build and test thousands of engineered and synthetic mobile genetic elements for writing and rewriting the human genome.

The company said it can also write entirely new sequences into the genome by delivering only RNA.

With the new round of funding, Tessera said it would look to further develop its tech, hire more staff and establish manufacturing and automation capabilities critical for its platform and programs.

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Last Call with Jenna Balestrini, the WPI grad treating cancer with cell therapy – Worcester Mag

Posted: at 4:16 pm

Sarah Connell Sanders| Correspondent

Jenna Balestrini is the Head of Strategy and Business Development for Precision Medicine and Cell Bioprocessing at Draper.

What is your connection to the city of Worcester?

Well, I love Worcester. I moved there to do my Ph.D. in 2003. I graduated with a Ph.D. from WPI in 2009. I have to say, those were six of the best years of my life. Worcester is such an amazing place and WPI is such a great school. I've always had a fondness for that city more than pretty much anywhere else I've lived. It's just a very special place filled with really great people.

I was hoping you could talk a bit about your career trajectory, particularly after you finished your Ph.D.

I went to WPI and worked for Kristen Billiar, who is the best advisor anyone could ever ask for. One of our focuses was to understand the environment in cells and around cells. Factors like breathing or stiffness can either stimulate cells or impact cells pathologically and create disease. Or, if you understand a cells environment, you can harness those signals and start to build therapies. You can get cells to make specific proteins and we looked at the fundamentals of that. From there, I did a postdoc in Toronto studying fibrosis with Boris Hinz. Then, I went to Yale and worked for Laura Niklason doing translational medicine work. All of this ties to understanding how we can direct regenerative medicine applications with cells by understanding the cues around them to make different therapies. In 2016, I was at the end of my postdoc and I was trying to think about what to do. I had wanted to be a professor for many years, but towards the end, I realized what I really wanted was to be more translational and be a little bit closer to where the patients and the action are. I had a friend that I met at WPI who recommended I speak to her uncle, Jeff Borenstein, at Draper. I'd never heard of Draper before and I didn't know much about the nonprofit world. He looked at my CV and said, "You know what, you'd actually make a really good fit here."

What can you tell me about Draper?

I came to Draper in 2016. As a nonprofit, Draper reinvests its profits into research. One of the manifestations of that are large internal awards called IRaD, which stands for Internal Research and Development. Within six months, I got an IRaD to build technologies to make the next generation of cell therapy. That project went from concept to commercial pretty quickly. I transitioned into being a business development lead and then the portfolio grew even more, mainly because I work with some really talented engineers, some of whom went to WPI. Ultimately, we partnered with Kite Therapeutics. So what that means is my career in cell therapy literally went from an idea scribbled on a napkin with a colleague, to overseeing a partnership with, in my opinion, one of the best cell therapy companies in the world in just a few years. I am in a completely different space than I ever would have imagined. I had no idea I'd ever go into business or cell therapy and I'm really pleased.

I suspect a lot of great ideas have started on napkins. I'm curious about just the term cell bioprocessing. Can you explain it for someone lacking a science background?

Basically, what we're trying to do is take cells from a patient and modify them to make those cells into therapies themselves. It's a really interesting way to enable a patient to heal themselves. We take your immune system cells and then we genetically modify them with equipment that we've made. The equipment separates the cells from your blood, and then we introduce genes that serve as a set of instructions for your immune system to attack something like cancer. Think of it like taking those cells from the patient, giving them some extra tools to make them "super-powered," to make them better at hunting down and identifying things like cancer, and then putting them back into the patient.

Sounds very futuristic.

So here's the thing, I don't know why this is, but most people don't realize that it's not 10 years from now. It's happening right now. Cell therapy is FDA approved. If you have certain types of leukemia or lymphoma, you can get cell therapy made from your cells to target and kill your cancer. And this is a curative solution. You can get a dose of these cells that have been modified to hunt the leukemia down, or lymphoma down, and then you are cured from that disease.

That's amazing.

We're living in an era where cancer is curable. But now, the thing is, can we take it further? You can identify a unique combination that separates out the thing you're trying to hunt down HIV, hepatitis. You can also use the same tools to rectify genetic diseases like sickle cell anemia or cystic fibrosis. It's just a faulty gene that you can replace, right? Those are the next steps.

What are your hopes for the future?

You know what? I would like people to get excited. Everything I just described is what's called autologous cell therapy we take cells from a patient and do all of this work and it's really expensive to do but the future is something called allogeneic cell therapy, where we can take the same tool to do genetic engineering or modification of cells and knock out all those individual components that make yourself uniquely identifiable to you. From that, you create a universal donor. And you can use that as a starting material to make therapies for everyone. So what that means for you as a patient is that you could come into your doctor's office and find out you have something, let's just say a cancer, and you'll have an off-the-shelf ready-to-go therapy that day, frozen and ready to go for you. I don't think that chemotherapy and radiation are going to last much longer in terms of what the first line of defense is going to be. And they're terrible. The truth is, we are still behind the times with cancer therapy. If you look at the cause of death over the last hundred years, pretty much everything but cancer has gone down. Heart disease, influenza, strokes, but not cancer. This is for a variety of reasons, one of which is that we're living longer. But the thing is, our tools are terrible. We kill people with our drugs. Weve arrived at a moment when we can finally imagine a world where cancer is no big deal.

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Last Call with Jenna Balestrini, the WPI grad treating cancer with cell therapy - Worcester Mag

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Juggernaut Just Discovered the New Weapon X | Screen Rant – Screen Rant

Posted: at 4:16 pm

Juggernaut and D-Cel learn the secret, for-profit superhuman prison called the Dungeon is essentially becoming the latest Weapon X program.

Warning! Spoilers to Juggernaut #5ahead!

The Juggernaut and his mutant sidekick D-Cel thought they were going to take down a secret, for-profit superhuman prison called the Dungeon but what they discovered was actually worse. While the original facility was a decoy, the real Dungeon is an airborne prison full of genetically modified guards who are protected by the government to experiment on their seemingly permanent residents in the pursuit of using their powers to better the world struggling to survive everyday superhuman conflict. In essence, the Dungeon is becoming the latest Weapon X program.

The Weapon X program was a clandestine government project that sought to create superhumans that could be used for military purposes. Weapon X was the tenth attempt of the Weapon Plus program which attempted to recreate the success of Project: Rebirth but by using genetic engineering to create mutations within normal humans or manipulate pre-existing mutations within mutants. Weapon X is famously known for capturing Logan Howlett and grafting adamantium to his skeleton, implanting false memories in hopes of molding him into the perfect military operative. Although Wolverine killed many when he originally escaped, the Weapon X program or similar programs have resurfaced occasionally throughout the years, trying but inevitably failing to create the perfect soldier or an army of loyal superhumans.

RELATED:Weapon X Kept Wolverine Prisoner In The Most Humiliating Way

In Juggernaut #5 by Fabian Nicieza and Ron Garney, Juggernaut and D-Cel hit the Dungeon hard but discover the facility is empty, a decoy with a teleporter to the real Dungeon. Although Cain initially has little trouble with the guards, the Dungeon's Warden explains that the guards have been spliced with DNA taken from their residents as he exhibits enough control to temporarily stop Juggernaut in his tracks. Although the guards fail to strip Cain of his powers, they learn that the flying superhuman prison is not only contracted by the USA Justice Department but they've been experimenting on their prisoners, hoping to understand and control certain abilities so they can be properly weaponized.

The guard's experimenting on the prisoners, giving themselves new superpowers, and being protected by the government are all similar behavior that sounds within Weapon X's wheelhouse but what really confirmed the Dungeon's direction was something their warden said. After Juggernaut and D-Cel declare her mutant status and desire to seek political asylum on Krakoa, the Warden is bound by law to let them go but states that the Dungeon is a symbol that the corporate and political world are done with mutants, superhumans, and all the trouble they bring.

Weapon X followed in Project: Rebirth's footsteps but didn't want an army of Super Soldiers to help win a World War but instead desired brainwashed covert super-assassins to handle black ops missions and other secret directives. The Dungeon is not just keeping powerful metahuman criminals incarcerated, it wants to control and manipulate superhumans similar to the villain Syndrome in Disney's The Incredibles. By giving everyone impressive abilities via technology, Syndrome would negate the need for superheroes but the Dungeon is prepared to take that one step further, they seek to manipulate superhumans to destroy other superhumans instead of putting ordinary civilians at risk.

Supercharged and feeling protected, the Warden indirectly challenges Juggernaut by stating that people like him cannot stop what they are doing. No stranger to being manipulated by others, Juggernaut meets with his previous opponents such as Arnim Zola, his android Primus, and the reassembled Quicksand and proposes that they join forces against their common enemy, someone who seeks to abuse people like them for their own purposes. Weapon X's problem was they often tried to control people or forces beyond their capabilities which often ended in death and destruction. By putting themselves in Juggernaut's peripheral, the Dungeon may have set themselves down a similar fate, if they're not careful.

NEXT:Juggernaut Is Forming His Own Team of Reformed Supervillains

The Dark X-Men Just Discovered the Easiest Way to Beat a Villain

Drew is a reader, writer, artist, and creative professional based in Westchester, New York. He dabbles into cosplay, movie references, comics, and some anime while also being a Ghostbuster. He has a Bachelors in History, a Masters in Publishing and is excited to be working with Screen Rant. Previously his articles have been featured on Comic Book Resources and Iron Age Comics and he's excited to see what happens next!

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Jasper Therapeutics and Graphite Bio Announce Collaboration to Evaluate JSP191 as Conditioning Regimen for Novel Gene Replacement Therapy in Patients…

Posted: at 4:16 pm

Jan. 13, 2021 13:35 UTC

REDWOOD CITY, Calif. & SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, and Graphite Bio, Inc., a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced a research and clinical collaboration agreement to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen for Graphite Bios investigational GPH201 gene replacement therapy for severe combined immune deficiency (SCID) in patients with IL2RG deficiency, known as x-linked SCID (XSCID).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210113005538/en/

XSCID is a severe, inherited disorder of the immune system with symptoms often presenting in early infancy, including persistent infections and failure to thrive. Without treatment, XSCID is typically fatal to patients in the first two years of life.

Graphite Bio is focused on the development of potentially curative therapies for patients suffering from serious diseases, using its targeted gene integration platform to harness the natural cellular process of homology directed repair (HDR) in order to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Jasper Therapeutics JSP191 is a first-in-class humanized monoclonal antibody that depletes hematopoietic stem cells from bone marrow and acts as a conditioning agent in patients prior to receiving a hematopoietic stem cell transplant. JSP191 is currently being evaluated in multiple trials as a stem cell depleting conditioning agent, including a Phase 1/2 trial to achieve donor stem cell engraftment in SCID patients undergoing hematopoietic cell transplant and a separate Phase 1/2 trial in AML/MDS patients undergoing hematopoietic cell transplant.

This collaboration with Jasper demonstrates our shared commitment to pioneering novel therapeutic approaches with the potential to significantly improve the treatment experiences of individuals with devastating conditions who stand to benefit from gene replacement therapies, initially for patients with XSCID, said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. GPH201 harnesses our targeted gene integration platform to precisely target the defective gene that causes XSCID and replace it with a normal copy. We are impressed by the initial positive clinical results demonstrated by JSP191 when used as a conditioning regimen, and look forward to collaborating with the Jasper team to explore how our novel technologies can be brought to more patients with XSCID and other indications.

Our collaboration with Graphite Bio is an exciting opportunity to further advance the field of curative gene correction by combining a targeted gene integration platform with our first-in-class targeted CD117 antibody, JSP191, that has already demonstrated preliminary clinical efficacy and safety as a conditioning agent in XSCID patients and those with blood cancers undergoing allogeneic hematopoietic stem cell transplant, said Bill Lis, executive chairman and CEO, Jasper Therapeutics.

Graphite Bio and Jasper will collaborate on research, and potentially a clinical study, evaluating JSP191 as a conditioning agent for GPH201. Each company will retain commercial rights to their respective technologies.

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About GPH201

GPH201 is a first-in-human investigational hematopoietic stem cell treatment that will be evaluated as a potentially curative therapy for patients suffering from XSCID. GPH201 is generated using Graphite Bios precise and efficient targeted gene integration platform technology to directly replace the defective IL2RG gene, maintain normal IL2RG regulation and expression, and ultimately lead to the production of fully functional adaptive immune cells.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

About Graphite Bio, Inc.

Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The companys targeted gene integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit graphitebio.com.

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House of El: The Shadow Threat is The Perfect Man of Steel Prequel – CBR – Comic Book Resources

Posted: at 4:16 pm

Given how House of El: The Shadow Threat's depicts Superman's parents, this really could have been a prequel to Zack Snyder's Man of Steel.

WARNING: The following contains spoilers for House of El: The Shadow Threat#1 by Claudia Gray, Eric Zawadski, Dee Cunniffe, and Deron Bennett, on sale now.

We've seen quite a few takes from DC on Krypton's society before it was destroyed, showing what life was like for Jor-El and Lara before they shot Kal-El off to Earth to save him. Outside of the mainstream continuity where Jor-El eventually returned to inform his son of their history, it was addressed in Elseworld stories such as All-Star Superman.These stories add layers of sympathy to the planet and show how the various clans suffered through their civil war--with bad choices made rampantly.

Now, the young-adult graphic novel, House of El: The Shadow Threat adds its own spin, but after dissecting what Superman's parents are up to here, this is actually the perfect prequel to Zack Snyder's Man of Steel.

RELATED:How House of El: The Shadow Threat Sets Up a Sequel

In the opening sequence to the DCEU movie, we witnessedRussell Crowe's Jor-El wanting more for his son, especially after the elites lost the plot on how to deal with the quakes that were occurring. General Zod and his military had also become disillusioned with their terraforming missions.Following an attempted coup it became clear Krypton was imploding from the inside long before the big bang.

But what stood out in Snyder's vision was how Krypton created its houses, with genetic engineering leading to predetermined fates for clans. This is how Krypton locked in artists, scientists, soldiers and politicians -- with people having fixed destiniesas their genes were assembled in chambers. Jor-El hated it because it meant no one could evolve and dream to be something bigger, thus there wouldn't be as many leaders and revolutionists. More so, natural birth for Kryptonianswas taboo, which is how Kal-El was made.

RELATED:Superboy: Conner Kent Gets a New Role in the Superman Family

The Shadow Threat walks a very similar path as Lara's created a machine that can scramble people's genetic code to make them complete Kryptonians. It's a re-sequencing to give them emotions such as compassion and empathy. This is her way of bettering society because the tribunes have created a nasty caste system. They're younger here so this would have beenthe perfect precursor to what Snyder laid down as it's adetailed look into how this was affecting the youth on Krypton, especially the military.

Granted, Lara's pregnant in this story through natural means, but had House of El simply not had her conceiving, it'd inform how Jor-El and Lara were on the big-screen and why they acted the way they did. Here, they'rerevolting outcasts that the tribunes don't want to deal with. As Lara's cousin, Zahn-Re, triesto figure out his destiny, his perspective reveals just how crazy Lara and Jor-El were considered. Even Sera-Ur, the soldier Lara reformats, thinks that while the couple's revered, they're best left on the fringes of Krypton.Thestory adds nuance as Lara mopes around temples, gathering intel on the old ways and who might still be interested.

There's also Midnight, an insurrectionist group that feels like they're made to follow General Zod in overthrowing the guilds. They want freedom and an equal Krypton, much like Zod's legion in Man of Steel. In the film, we learned he and Jor-El were close, and thisbook follows suit. In fact, Zod's the one who sends Sera to be reformatted secretly in Lara's lab.

While the filmversion of Zod is dedicated to order, his military duty and the Genesis Chamber for genetic coding, House of El has him toeing the line. He wants to liberate people, buthe also wants peopleto fall in line when he says so. It's easy to see him actually realizing genetic coding and the chamber workin his terrorist mission to produce brainwashed soldiers, which can be where he and the Els fell apart. Ultimately, there are quite a few parallels that just can't be ignored which could have beefed up what Snyder did.

KEEP READING:Superman: Who's REALLY Running the Daily Planet Now

Valkyrie: Everyone Who's Been Marvel's OTHER Asgardian Avenger

I'm a former Chemical Engineer. It was boring so I decided to write about things I love. On the geek side of things, I write about comics, cartoons, video games, television, movies and basically, all things nerdy. I also write about music in terms of punk, indie, hardcore and emo because well, they rock! If you're bored by now, then you also don't want to hear that I write for ESPN on the PR side of things. And yes, I've written sports for them too! Not bad for someone from the Caribbean, eh? To top all this off, I've scribed short films and documentaries, conceptualizing stories and scripts from a human interest and social justice perspective. Business-wise, I make big cheddar (not really) as a copywriter and digital strategist working with some of the top brands in the Latin America region. In closing, let me remind you that the geek shall inherit the Earth. Oh, FYI, I'd love to write the Gargoyles movie for Disney. YOLO.

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House of El: The Shadow Threat is The Perfect Man of Steel Prequel - CBR - Comic Book Resources

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Genetic Engineering Market Size 2021 By Analysis, Manufacturers, Regions, Type and Application, and Forecasts to 2027 – Jumbo News

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The objectives of the Genetic Engineering Global Market Study are:

Split the breakdown data by region, type, manufacturer, and application. Identify trends, drivers, and key influencing factors around the world and in the regions Analysis and study of global Genetic Engineering status and future forecast, including production, sales, consumption, history, and forecast. Analysis of the potential and advantage, opportunities and challenges, limitations, and risks of the global market and key regions. Analyze competitive developments such as expansions, agreements, product launches, and acquisitions in the market. Introducing the major Genetic Engineering manufacturers, production, sales, market share, and recent developments.

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Moss-based expression firm receives 60m in funding – BioProcess Insider – BioProcess Insider

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German biotech Eleva has received funding to advance its candidate CPV-101, produced using a moss-based expression system.

Venture capitalist firm Zukunftshonds Heilbronn has invested up to 60 million ($73 million) in funding to progress drug candidate CPV-101 through the clinic. A spokesperson for Eleva told us we are developing CPV-101 for kidney-associated complement diseases such as aHUS, IgA Nephropathy, PNH, C3G.

The firm has developed a manufacturing process that produces biopharmaceuticals using moss called BryoTechnology.

Image: iStock/Svetlana Monyakova

Eleva told BioProcess Insider fermentation is done in Sartorius STR single use fermenters with unmodified cell culture bags and is done using established routines and equipment used commonly in mammalian cell-based production.

The company claims that BryoTechnology benefits from the absence of animal derived components and human viruses [] and batch to batch stability. Eleva added the manufacturing process is very robust and stable (unsensitive to change in pH, temp and salt). The glycosylation pattern is very stable, also upon scale up and tech transfer we do not see any changes in the glycosylation pattern.

Bjrn Voldborg, director of CHO cell line development at the Technical University of Denmark, previously discussed the problems that surround glycosylation at BPI Europe, telling delegates if you have the wrong glycans the protein may actually trigger immune responses.

Eleva said where glycosylation is crucial for the mode of action or for the efficacy of the molecule [it is] especially suited for the production in moss.

We have previously reported the limitations mammalian and bacterial cell lines have alongside documenting the growing interest in plant-derived alternatives.

Whatever system used, cells are engineered to produce the desired biologic drug substance in the highest yield and purity possible. Yet, with mammalian cell culture being notoriously expensive, plant-cells have become an alternative choice of platform due to their cost-effective expression system, free of animal proteins.

Eleva is not alone in its quest for plant-based substitutes, Sanofis deal with Dyadic showcased the demand for CHO alternatives. However, Eleva claims to be the only company using moss as an expression platform to make biologics.

Moss produces complex molecules (proteins, enzymes, antibodies, metabolites) with human-like glycosylation the spokesperson told us, adding antibodies produced in moss show >40-fold ADCC enhancement compared to antibodies produced in mammalian systems.

When asked what advantages a moss-based system has over mammalian, microbial and other plant-based systems, Eleva said moss combines the best of two worlds: it is a higher eukaryote same as mammalian cells and is haploid organism same as microbials.

The firm added: Contrary to other plant-based systems such as tobacco, moss has a haploid genome, making genetic engineering as easy as in microbials.

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UK feed and pig industries welcome UK consultation on gene editing – FeedNavigator.com

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Robert Sheasby, CEO of the AIC, the voice of the UK feed and the agri-supply sector, said:

The AIC warmly welcomes the launch of this government consultation on gene editing in crops and livestock. We have long sought to support sustainable modern commercial agriculture in the UK, and this is the opportunity for our members to put forward their views on this development in technology. We would encourage the industry at large to respond.

EU legislation controlling the use of GMOs was retained in the UK at the end of the transition period, after December 31, 2020. This retained legislation requires that all GE organisms are classified as GMOs irrespective of whether they could be produced by traditional breeding methods.

The UK's Department of Food, Rural Affairs and the Environment (Defra) said it is its view that organisms produced by GE or by other genetic technologies should not be regulated as GMOs if they could have been produced by traditional breeding methods.

Leaving the EU provides an opportunity to consult on the implications of addressing this issue. We recognize there is a spectrum of opinions on these topics, and we are consulting to provide an opportunity for all views to be shared."

Speaking at the Oxford Farming Conference, where the consultation was launched, UK environment secretary, George Eustice, said:

Gene editing has the ability to harness the genetic resources that mother nature has provided, in order to tackle the challenges of our age. This includes breeding crops that perform better, reducing costs to farmers and impacts on the environment, and helping us all adapt to the challenges of climate change.Its potential was blocked by a European Court of Justice ruling in 2018, which is flawed and stifling to scientific progress. Now that we have left the EU, we are free to make coherent policy decisions based on science and evidence. That begins with this consultation.

Consulting with academia, environmental groups, the food and farming sectors and the public is the beginning of this process that, depending on the outcome, will require primary legislation scrutinized and approved by the UK parliament, stressed Defra.

Professor Robin May, the chief scientific officer of the UKs Food Standards Agency (FSA), also welcomed the review, saying:

The UK prides itself in having the very highest standards of food safety, and there are strict controls on GM crops, seeds and food which the FSA will continue to apply moving forward. As with all novel foods, GE foods will only be permitted to be marketed if they are judged to not present a risk to health, not to mislead consumers, and not have lower nutritional value than existing equivalent foods. We will continue to put the consumer first and be transparent and open in our decision-making. Any possible change would be based on an appropriate risk assessment that looks at the best available science.

Sir David Baulcombe, professor of botany in the Department of Plant Sciences at the University of Cambridge, said the overwhelming view of public sector scientists is that the Nobel Prize winning methods for gene editing can accelerate the availability of crops and livestock for sustainable, productive and profitable agriculture.

The UK National Pig Association (NPA) said that gene editing technology could potentially deliver long-term benefits for pig production.

In the NPA's response to the Nuffield Council of Bioethics call for evidence on genome editing in September 2019, its senior policy adviser, Rebecca Veale, identified the potential value of gene editing tools in improving the efficiency of pig production.

"The opportunities for application are long. We might be in a better place to tackle diseases such as ASF and PRRS and we might be able to reduce emissions in pig production or exploit nutritional availability in feed better.

"Afew countries have made small steps to utilizing this technology, but these have been limited. Our industry cannot be disadvantaged by a lack of access to such a tool and any future policy must be clear not to breach ethical boundaries, but to have flexibility to allow the use of the technology to be exploited to its full potential. Any future developments are reliant on support for the research required to explore the opportunities available, she added.

Responding to the consultation, the director of anti-GM campaign group, GM Freeze, Liz ONeill said:

"People have many concerns about the use of genetic engineering in food and farming so public engagement is vital but it has to be done well. Unfortunately this consultation has started very badly. Its been launched in the midst of an unprecedented health crisis; it has a clear bias in favour of removing vital safeguards; and the text of the consultation grossly misrepresents the nature of highly experimental new GM techniques.

"Instead of working with people to understand their concerns, Defra is pushing the high-tech, quick-fix agenda favoured by industrial farming corporations. GM Freeze will, of course, be submitting evidence and we encourage everyone who wants to know what they are eating to do the same, but the government should be doing much more to protect our food, our farms and the natural environment."

Aside from gene editing, the consultation will also begin a longer-term project to gather evidence on updating the UK approach to genetic modification by gathering information on what controls are needed and how best to deliver them, said Defra.

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Kombucha Inspires Creation of a Microbial Living Material – Technology Networks

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Scientists have created a living material made from microbes that can respond to stimuli from their surrounding environment. It is hoped the material could find diverse applications in contaminant detection, highlighting damage, for example, to packaging, delivering nutrients or therapeutics and even in creating living photographs.Biological systems are able to assemble and arrange tissues and structures, building in changes that reflect external stimuli, in ways beyond the capability of man-made materials. Consequently, the development of materials with these capabilities is highly desirable for monitoring purposes. Engineered living materials (ELMs) such as these have been created previously, however, the stringent growth conditions required for the microbes used have restricted their use to trained personnel. Scaling up production to facilitate their use as a technology has also consequently proved challenging. This latest research, published today in Nature Materials, however, overcomes these problems by taking its inspiration from the fermented drink kombucha.

Kombucha is a fermented culture of yeast and bacteria used to make a tea drink thought to have beneficial health properties. The kombucha mother culture from which the tea is made normally contains one or two strains of bacteria and at least two strains of yeast that live in symbiosis.

Scientists from Imperial College London and Massachusetts Institute of Technology combined genetically engineered bakers yeast - the yeast typically found in kombucha is hard to engineer - with cellulose-producing bacteria to create a mutually beneficial symbiotic culture. The Komagataeibacter rhaeticus bacteria used was isolated from a kombucha mother culture and produces large quantities of cellulose that acts as a scaffold to support multi-functional enzymes produced by the yeast.

The researchers were able to engineer the yeast according to the needs of their system, for example to produce enzymes that fluoresced or that broke down target molecules. The systems for genetically engineering bakers yeast are well established and fast, meaning that new strains with particular desired characteristics can be created quickly. The simple growth conditions required by the kombucha-style culture also mean that any new combination can quickly be established and bulked up ready for use. Both of these features make this a favorable ELM system.

In a press release, senior author Professor Tom Ellis from Imperial College London said, The genetic toolbox for engineering these bacteria is underdeveloped compared to the number of tools available for manipulating yeast DNA. That is why we chose to use this division-of-labor strategy so we could first focus on engineering the yeast cells and explore the possibilities of various living functional materials."

The malleable nature of the yeast engineering system gives it utility in many areas opens doors to adapting the system to many purposes. In the study, the team incorporated yeast capable of sensing estradiol, a hormone found as an environmental pollutant, but this is just one example of the many possible applications. The team foresee that it could even be used to deliver essential nutrients or release therapeutics in response to stimuli.

Although we are still far from a future in which people can cheaply grow their own biological sensors, our new system moves us forward by creating materials that are scalable and therefore more likely to be useful in the real world, commented Dr Charlie Gilbert, one of the studys authors.

ReferenceGilbert C et al. Living materials with programmable functionalities grown from engineered microbial co-cultures. Nat. Mater. (2021). https://doi.org/10.1038/s41563-020-00857-5

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Kombucha Inspires Creation of a Microbial Living Material - Technology Networks

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