Category Archives: Genetic Engineering

The UK Government announced on 10 May in the Queens Speech that it intends to pass the Genetic Technology (Precision Breeding) Billto permit the use of gene-editing technologies in the commercialisation of plants and animals to improve agricultural efficiency and food production in the UK. This comes on top of a recent change in legislationback in Aprilto permit research and development using gene-editing techniques.

Both changes come in a post-Brexit change to the regulatory landscape, bringing the contentious issue of GMOs and intellectual property to the fore once again. The ground-breaking news follows a decade of a largely EU-wide moratorium on the approval and commercialisation of gene-edited crops.

With the fast pace of change brought by genome editing as part of new breeding techniques (NBTs) to develop plant varieties, there is a real concern that breeders may seek to launch new crops with a biotech trait, by using existing plant material without authorisation or any compensation to the holder of plant breeders rights in that existing variety.

Meanwhile, some biotech companies argue that the uncertainty of the legal concept of essentially derived varieties (EDVs) is stifling innovation. Others cite the difficulty of policing use of material of their protected initial variety, bringing a claim for infringement and the risk of damage to their business from competition from a derived variety.

We expect to see increased regulatory challenges and disputes in this area and businesses must monitor the latest developments to ensure they are prepared. A revolution in genetic engineering has transformed plant breeding. Development of all new plant varieties typically involves access to, and use of, plant material of existing plant varieties. The traditional process of crossing and selection relies largely on unintended recombination of genes and random mutants occurring naturally, which are selected and developed further over many years. Using multiplication techniques, a new variety can take over ten years to get to market. However, new breeding techniques can reduce the development time for a new variety to only two to four years.

One of the most controversial legal areas of plant breeding is the concept of essentially derived varieties (EDVs). The concept first set out in the 1991 UPOV Convention is predicated upon an open model of innovation, which allows breeders to use plant material for the purpose of breeding a new variety, the so-called breeders exception (Art 15(1)(iii)).

Recognising the advent of biotechnology, UPOV introduced a compulsory limitation upon the breeders exception in respect of EDVs under Art 14(5)(b). The main driver for the limitation was to prevent plagiarism of traditionally-bred varieties using new technologies, but it was also in response to erosion of the minimum distance requirement in phenotypical characteristics, as part of the DUS (distinct, uniform and stability) requirements for protecting a new variety.

The complex EDV concept was intended to prevent the commercialisation of a bred derived variety, without the consent of the owner of the plant breeders rights in the protected initial variety. The idea was that, given the significant contribution that the work in breeding the initial variety had made to the derived variety, the owner of rights in the initial variety should be entitled to licence the material embodied in the derived variety and obtain a financial benefit. It would prevent the developer from simply patenting the biotech trait and then commercialising the trait by inserting it into plant material already protected by plant breeders rights, to produce an improved variety. It is not intended to prevent the developer of a derived variety from obtaining protection for that variety.

The complex definition of EDV raises many legal issues and uncertainties, including what amounts to predominantly derived, and whether it is relevant to the expression of the essential characteristics that the EDV has strong phenotypical similarity to, as well as genetic identity. The concept has polarised views and been applied differently around the world, making this an even more complex issue to grasp.

The lack of clarity on EDVs has already led to significant controversies and litigation, notably in the case of Danziger'Dan' Flower Farmv.AsteFlowers B.V. where the Dutch Court of Appeal, The Hague, held that the Gypsophila variety, Blancanieves, was not an EDV of Million Stars Dangypmini.

There is also a long-running dispute over the mandarin varieties Nadorcott and Tango, over whether Tango is an EDV of Nadorcott. The latest spin-off in 2021 pitted the King of Morocco (owner of Nadorcott) against a Spanish farming business over alleged unlicensed production of the variety, with the Advocate General giving his Opinion upon a preliminary reference to the CJEU on 14 October 2021 from a reference from the Supreme Court in Spain. Cases have been heard over whether Tango is an EDV in the US, Australia and South Africa and before the Community Plant Variety Office as to whether Tango is a distinct variety capable of protection.

So, what is the way forward? The debate continues but there have been some recent developments which businesses should be aware of. For example, a Working Group on EDVs was set by UPOV for the revision of the Guidance on EDVs. In addition, a draft text was last discussed on 19 October 2021, with a view to being adopted by the UPOV Council.

Some experts are keen that the new Guidance provides a more practical framework to distinguish between the cases which are clearer instances of EDV or non-EDV and how borderline cases may be determined. They argue that the inventive contribution of both the initial variety and the derived variety should be reviewed, in terms of the expression of their essential characteristics.

The legal presumption may be upon the owner of the protected initial variety to prove essential derivation and then claim dependency. However, some argue that the burden of proof should be reversed as the developer of the derived variety may have better data about the gene edit.

There is a role for the use of biochemical and molecular markers, though they need to be used cautiously, if used to determine whether a variety is an EDV.

There may also be a role for plant variety or patent offices to be able to provide an early declaratory decision, based upon receiving technical evidence. Alternative dispute resolution, such as expert determination or mediation facilitated by an industry expert, may also become more commonplace.

In addition, there may be value in developing industry-standard approaches to licensing and model form contractual clauses, perhaps drawing upon other licensing regimes (such as SEPs) or perhaps even under the Nagoya Protocol for plant genetic resources.

Biotech companies and plant breeders alike should closely monitor these developments and weigh up the risks of dispute, to avoid disruption to a smooth launch of a new crop into the desired market.

The UPOV Council needs to take a clear position soon on how to determine easily an EDV to give certainty to industry when using gene editing to develop new varieties, on situations when a licence may be required and royalties payable before launching commercially.

This article was first published in May 2022, on IAM media.com here.

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Legal confusion on essentially derived varieties must be cleared up for the sake of innovation - Green light to gene-editing plants risks uncertainty...

It was the election interview that gripped the nation, taking the then white-hot issue of genetically modified food and turning it into incredible political TV drama. Duncan Greive reflects on Corngate an epochal moment in our media history.

The first thing that strikes you is the staging the studio is pitch dark, with bright spotlights on Helen Clark and John Campbell. He is rounding into the early era of his cult status as a probing, fearless interviewer; she is at the height of her power and influence as prime minister.

Clark looks fierce, Campbell locked in. Hes holding his clipboard and unleashing a volley of very heated statements. Did you mislead the Royal Commission? he asks, and later, Its about whether or not we can trust you, repeating it for emphasis. Feel free to shoot the messenger, he says toward the end.

Clark was not told about the specifics of the interview, and is clearly furious as a result, responding to Campbells questions with relentless real-time media criticism: You may think this is a really smart way to set up the prime minister, she says at one point. The more this interview goes on, the more offended I am, she says later. Its simply preposterous to carry on.

Every moment of it is extraordinary. The original tape seems to have essentially vanished, with Three not responding to requests for the archive and only a grainy six-minute clip available on NZ On Screen (Update, July 9: Three has now responded and surfaced the full interview from their archives!). Still, you can feel the heat even after all these years.

Given the challenges we confront today, its head-spinning to think all this arose over some delicious corn. The core of the issue was whether, during a time of a major debate about the safety of genetically modified crops, GM corn was accidentally released into New Zealands food supply. Campbell had been given an advance copy of Nicky Hagers book Seeds of Distrust, which alleged that a field of GM corn was mistakenly grown and distributed to consumers here, and furthermore, that cabinet had known and conspired with officials from the Ministry for Agriculture to cover it up.

The interview became known as Corngate and was one of the defining flashpoints of the decade, accurately described by a contemporary report as a bomb, dropped right into late stages of the 2002 election period. It derailed Labours campaign, pitted them bitterly against the Greens, caused a lasting rift between TV3 and Clark, and played a role in making genetic engineering a politically untouchable subject to this day.

The idea that a television interview could have such impact speaks to the era in which it aired. Twenty years ago TV news presenters were figures of huge socio-cultural power, and sometimes major newsmakers in their own right.

John Hawkesby received a $5.2m payout after his ill-fated three-week stint presenting the 6pm news on One. A few years later, Paul Holmes resigned from TVNZ in a fury over what he perceived as an insulting contract offer, and for a brief, glorious moment we had an impossible bounty of current affairs in primetime: Close Up on One, Campbell Live on TV3 and Paul Holmes (the show) on Prime all competing with the juggernaut that was mid-2000s Shortland Street.

This was the absolute apex of television as the agenda-setting centre of our lives, and TV3, playing David to TVNZs Goliath, had a pair of white-hot young stars reading the 6pm news in Carol Hirschfeld and John Campbell. The channel had become a beloved challenger to state-owned monolith TVNZ, innovating on style and form. Hirschfeld and Campbell had come to embody the network young, sharp and fearless. Campbell was a brilliant interviewer, smart yet with a rare ability to emotionally connect with the audience.

He had been inserting live political interviews into bulletins for some years, yet Corngate represented a massive escalation. It was a major break from the schedule TV3 was still years away from elevating Campbell to his own show in Campbell Live, and had settled on syndicated airings of broad American sitcoms like Home Improvement as its best weapon to confront TVNZs Holmes and Shortland Street at 7pm. The very fact of the interview breaking that 7pm routine gave it a huge sense of occasion.

It came towards the end of an oddly discombobulated election campaign. National was at its lowest ebb, careening toward its worst-ever election result under the leadership of a baby-faced Bill English, who barely warranted the withering stare of Clark. She was a prime minister of immense force of will and personality, probably our most imposing since Rob Muldoon. But with the capitulation of the right, the chaos of a divided left bloc became the focal point of the election.

Labour had governed its first term in coalition with the Alliance, which became a cautionary tale for minor parties thereafter, collapsing to a less than 2% share of the party vote in 2002. In their stead came a thicket of smaller players that collectively amassed an MMP record 37% of the vote, with NZ First, Act, United Future and the Greens all attracting over 6.5% of the electorates support.

It was the latter party that became Clark and Labours biggest headache, resulting in some memorable lines, including the prime minister referring to the Greens as goths and anarcho-feminists in the days before the Corngate interview. That this has not become the Greens official slogan is one of the enduring mysteries of our politics.

The focal point of much of this rancour was genetic modification (GM), a relatively new form of science that relied on gene editing to produce novel or altered organisms. It has applications across medicine and industry, but its use in agriculture drew the most attention. Proponents saw the potential for higher-yielding or more drought-resistant crops, or livestock that was less prone to particular forms of disease. The science has subsequently essentially settled in favour of GM, but it was highly contentious in our politics at the time, and had been subject to a Royal Commission in 2000.

The commission came back with a cautious endorsement of the technology, saying, New Zealand should keep its options open. It would be unwise to turn our back on the potential advantages on offer. It did little to resolve divisions over the issue, which saw the country split into two camps. Broadly speaking, business and the agricultural sector saw GM as a science crucial to growth, while much of the environmentally minded left saw it as dangerous, unproven practice that risked our clean, green reputation. Following the commissions report, a two-year moratorium preventing applications for the release of genetically modified organisms was put in place in 2001.

As the country rounded into the 2002 election, investigative journalist Nicky Hager was working on his third book. Seeds of Distrust examined the accidental release of GM corn, the potential for contamination of other crops and the decision not to notify the public of the incident. He scheduled it for publication on July 10 less than three weeks before the general election on July 27.

Campbell had worked with Hager twice before, fronting major stories accompanying both Secret Power, which covered the Waihpai spy base and its links to international espionage networks, and Secrets and Lies, which exposed the infiltration of West Coast environmental groups. The pair had become close, and Hager sent Campbell the manuscript for Seeds of Distrust, which emerged on a gigantic roll of fax paper. Campbell pored over it for weeks, recalls Hirschfeld, with the support of news editors Mark Jennings and Mike Brockie. (Jennings did not respond to a request for comment for this story.)

The team knew that this was a big story, but had to approach it carefully. TV3 went to Clarks office with an innocuous cover, suggesting a general conversation about genetic engineering rather than a laser focus on the decision not to publicise the potential release of the GM corn in November 2000. The fateful interview took place in-studio, but 20 years on, Hirschfeld says the infamous spotlit aesthetic was not an attempt to dial up the drama. It instead had a much more banal explanation. What was the thinking? We had no money, she laughs.

They recorded enough footage for that electric half hour of television, cutting only around five minutes, which Campbell says was an attempt to give viewers the whole context. Afterwards, Clark left in a hurry, but not before telling Campbell exactly what she thought of him. She used the word treachery, Campbell recalls, while Hirschfeld remembers you traitor. Clark declined to be interviewed for this story, but has in the aftermath made her view of the interview abundantly clear.

TV3 cut it together into the half-hour special and aired it the following evening, July 9, 2002. It began with an opening segment in which Campbell interviewed Hager and travelled to the location of the alleged leak of the corn; the final two segments were dominated by that extraordinary interview with Clark. It was an immediate sensation, deeply uncomfortable yet incredibly compelling TV.

It was almost unbearable to watch, says Hager now. Writing for the NZ Herald, Jeremy Rees described it as a study of outrage and anger. In the days that followed, media reporting of the special tended to side with Clark, with Russell Brown typifying the criticism on his Hard News segment on bFM, saying that the way it did emerge dropped like a bomb on the election campaign was simply wrong.

Clark certainly thought so. She labelled Campbell a sanctimonious little creep, and interrupted her campaign to respond with a fusillade delivered from the lectern at a hastily arranged press conference. There she made it clear she blamed the Greens, and leader Jeanette Fitzsimons. I am going to sing from the rooftops that this is a very dirty campaign where the Greens and their supporters have descended to the gutter of the National Party.

Fitzsimons did not deter that impression when she issued a press release saying she was deeply distressed that the prime minister apparently decided to let this contaminated crop be grown, harvested, eaten and possibly exported in 2000/2001, and that the government participated in efforts to keep the truth from the public. Similarly, the fact Seeds of Distrust was published by Craig Potton, a former Greens candidate, made it easy for Clark and Labour to frame it as an orchestrated hit though Hager is adamant there was no collusion, and says the Green Party was privately furious with him for distracting from its policy agenda.

The interview became the defining moment of the campaign, and while it didnt impact the result, the bitter taste lingered, and reared up again a year later.

The Broadcasting Standards Authority received numerous complaints about the episode, including one from Mike Munro, the prime ministers chief press secretary, on behalf of himself and Clark. The regulator ultimately released a highly publicised ruling in July 2003. It ran to 92 pages and broadly vindicated the complainants, saying that standards were breached on multiple counts around balance and fairness. It faulted the tenor of the interview with Hager versus that with Clark as neither impartial nor objective, and the fact that Clark was not advised of the source of the allegations.

By BSA standards it was damning, but not unequivocal, and allowed for TV3 to issue a press release that quoted Jennings as saying we knew the story was right, we knew we had done our homework and the BSA ruling largely validates that view.

Russell Brown wrote a reflective response for Public Address afterwards in which he acknowledged that many parties got consumed by the heat of the moment and overdid their reactions, himself included, but faulted TV3 for only preserving the raw footage of the Clark interview, and not that of Hager. Brown says this left the unavoidable impression that it had treated Hagers allegations far more credulously than it did Clarks response.

Campbell disputes that characterisation today, saying he asked for and received the primary materials on which the book was based, and Hirschfeld says Campbell spent weeks on the story. There were endless sessions going over the details, she says. Ive never seen him so prepared.

Clark herself was clearly completely blindsided, and spent much of the interview underlining that fact through gritted teeth. It is simply not acceptable to set up the prime minister on something which happened a long time back in the term of government, that she was not the minister responsible for, she says at one point. Campbell was unconvinced of this then I think you do remember what happened, he says at one point and remains so today. She had a forensic rigour about her she was across the detail of all the portfolios, he says.

While it ultimately had little obvious effect on the election outcome, it had a huge impact on the journalists involved, coming up against a popular PM at the height of her influence. Hirschfeld recalls Campbell so spaced out after it aired that he was nearly run down on Ponsonby Road while getting out of a car. Hager remains frustrated by what happened, and the way the furore completely overwhelmed the book upon which it was based. I couldnt bear to look at it for years afterwards, he says, also believing that Clark has never forgiven him.

Campbell got it most personally, though, with one incident still seared into his memory. One night, not long after the special aired, he was out walking his one-year-old daughter through Three Lamps in Ponsonby, near his home. A woman he describes as having a patrician bearing approached him, and bent down to peer into the buggy. I pity you, having him for a father, Campbell recalls her saying. His relationship with the prime minister was also seriously damaged by the incident. It took years to recover.

Clarks fury dimmed but did not pass, and Hirschfeld remembers TV3 being pointedly left until dead last for an interview as late as the 2005 election campaign. But by that stage their lives had changed, and in some ways Corngate, for all its complexity as an incident, had helped them grow.

Hirschfeld went on to become the producer of Campbell Live, a full-time 7pm current affairs show that cemented Campbell as a star. The fearlessness of that 2002 interview was present in his live interviews as he supplanted Holmes to become the emblematic broadcaster of the era. Hager moved on, too. During the next election he picked up the threads of what was to become The Hollow Men, his book about Nationals 2005 campaign, and the one he considers his best.

As for Clark, she would go on to win a third term, and leave the rancour of Corngate to bulldoze through future controversies, from anti-smacking legislation to the pain of the Foreshore and Seabed Bill. Perhaps the most lasting scar of the era is a political circumspection around genetic modification issues that lingers to this day, with Greens food policy embodying a tension in stressing affordability while confining GM to the lab.

Despite the BSA ruling, Campbell remains proud of the work. To me, it wasnt a GM story, it was a political story. At a time of huge public interest in and fear about genetic modification, did bureaucrats and politicians combine to cover up the release of GM material into the environment? Hager and Campbell both remain convinced that they did. Campbells only regret is that his team were not more candid about the topic of the interview, but isnt sure whether the prime minister would have fronted had they been more direct. Was it the best of all the shitty options? I dont know, he says.

Twenty years on, our politics and media have changed immeasurably, and Hirschfeld expresses a sadness that such an interview has no place in primetime today. Watching it now its obvious why it had such an impact. It was enormous, says Hirschfeld, and it was immensely compelling television that still retains its power to this day.

Follow Duncan Greives NZ media podcast The Fold on Apple Podcasts, Spotify or your favourite podcast provider.

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Helen Clark vs John Campbell: the Corngate interview, 20 years on - The Spinoff

The authors at a clinic in Palpite in Cuba. Photo: Odalys Miranda/Twitter

Palpite, Cuba, is just a few miles away from Playa Girn, along the Bay of Pigs, where the United States attempted to overthrow the Cuban Revolution in 1961. Down a modest street in a small building with a Cuban flag and a large picture of Fidel Castro near the front door, Dr. Dayamis Gmez La Rosa sees patients from 8 am to 5 pm. In fact, that is an inaccurate sentence. Dr. Dayamis, like most primary care doctors in Cuba, lives above the clinic that she runs. I became a doctor, she told us as we sat in the clinics waiting room, because I wanted to make the world a better place. Her father was a bartender, and her mother was a housecleaner, but thanks to the Revolution, she says, she is a primary care doctor, and her brother is a dentist. Patients come when they need care, even in the middle of the night.

Apart from the waiting room, the clinic only has three other rooms, all of them small and clean. The 1,970 people in Palpite come to see Dr. Dayamis, who emphasizes that she has in her care several pregnant women and infants. She wants to talk about pregnancy and children because she wants to let me know that over the past three years, not one infant has died in her town or in the municipality. The last time an infant died, she said, was in 2008 when a child was born prematurely and had great difficulty breathing. When we asked her how she remembered that death with such clarity, she said that for her as a doctor any death is terrible, but the death of a child must be avoided at all costs. I wish I did not have to experience that, she said.

The region of the Zapata Swamp, where the Bay of Pigs is located, before the Revolution, had an infant mortality rate of 59 per 1,000 live births. The population of the area, mostly engaged in subsistence fishing and in the charcoal trade, lived in great poverty. Fidel spent the first Christmas Eve after the Revolution of 1959 with the newly formed cooperative of charcoal producers, listening to them talk about their problems and working with them to find a way to exit the condition of hunger, illiteracy, and ill-health. A large-scale project of transformation had been set into motion a few months before, which drew in hundreds of very poor people into a process to lift themselves up from the wretched conditions that afflicted them. This is the reason why these people rose in large numbers to defend the Revolution against the attack by the US and its mercenaries in 1961.

To move from 59 infant deaths out of every 1,000 live births to no infant deaths in the matter of a few decades is an extraordinary feat. It was done, Dr. Dayamis says, because the Cuban Revolution pays an enormous attention to the health of the population. Pregnant mothers are given regular care from primary care doctors and gynecologists and their infants are tended by pediatriciansall of it paid from the social wealth of the country. Small towns such as Palpite do not have specialists such as gynecologists and pediatricians, but within a short ride a few miles away, they can access these doctors in Playa Larga.

Walking through the Playa Giron museum earlier that day, the museums director Dulce Mara Limonta del Pozo tells us that the many of the captured mercenaries were returned to the US in exchange for food and medicines for children; it is telling that this is what the Cuban Revolution demanded. From early into the Revolution, literacy campaigns and vaccination campaigns developed to address the facts of poverty. Now, Dr. Dayamis reports, each child gets between 12 and 16 vaccinations for such ailments as smallpox and hepatitis.

In Havanas Center for Genetic Engineering and Biotechnology (CIGB), Dr. Merardo Pujol Ferrer tells us that the country has almost eradicated hepatitis B using a vaccine developed by their Center. That vaccineHeberbiovac HBhas been administered to 70 million people around the world. We believe that this vaccine is safe and effective, he said. It could help to eradicate hepatitis around the world, particularly in poorer countries. All the children in her town are vaccinated against hepatitis, Dr. Dayamis says. The health care system ensures that not one person dies from diarrhea or malnutrition, and not one person dies from diseases of poverty.

What ails the people of Palpite, Dr. Dayamis says, are now the diseases that one sees in richer countries. It is one of the paradoxes of Cuba, which remains a country of limited meanslargely because of the US governments blockade of this island of 11 million peopleand yet has transcended the diseases of poverty. The new illnesses that she says are hypertension and cardiovascular diseases as well as prostate and breast cancer. These problems, she points out, must be dealt with by public education, which is why she has a radio show on Radio Victoria de Girn, the local community station, each Thursday, called Education for Health.

If we invest in sports, says Ral Forns Valenciano, the vice-president of the Institute of Physical Education and Recreation (INDER), then we will have less problems of health. Across the country, INDER focuses on getting the entire population active with a variety of sports and physical exercises. Over 70,000 sports health workers collaborate with the schools and the centers for the elderly to provide opportunities for leisure time to be spent in physical activity. This, along with the public education campaign that Dr. Dayamis told us about, are key mechanisms to prevent chronic diseases from harming the population.

If you take a boat out of the Bay of Pigs and land in other Caribbean countries, you will find yourself in a situation where healthcare is almost nonexistent. In the Dominican Republic, for example, infant mortality is at 34 per 1,000 live births. These countriesunlike Cubahave not been able to harness the commitment and ingenuity of people such as Dr. Dayamis and Dr. Merardo. In these other countries, children die in conditions where no doctor is present to mourn their loss decades later.

Excerpt from:
How Cuba is eradicating child mortality and banishing the diseases of the poor - Peoples Dispatch

Sickle cell disease (SCD) is a debilitating illness affecting up to 40% of the population in some African countries. Its caused by mutations in the gene that makes haemoglobin the protein that carries oxygen in red blood cells.

It might one day be possible to treat this disease using gene editing by switching back on the production of a healthy form of haemoglobin called foetal haemoglobin, which is usually only produced by the body when were in the womb.

But a new study testing this promising new treatment in mice has found that scientists still have a long way to go before it can be attempted in humans. The research has been published in Disease Models & Mechanisms.

Healthy red blood cells (RBCs) are shaped similar to a donut but with an indentation instead of a hole.

In sickle cell disease the abnormal haemoglobin distorts the RBCs shape when they arent carrying oxygen. Instead, sickled RBCs are C-shaped, like the farm tool called a sickle, and they become hard and sticky, and die earlier.

Because of their shape, sickled RBCs can become stuck and stop blood flow when travelling through small blood vessels. This causes patients to suffer from episodes of excruciating pain, organ damage and a reduced life-expectancy.

Although current treatments have reduced complications and extended the life expectancies of affected children, most still die prematurely.

Red blood cells are made from haematopoietic stem cells in our bone marrow. These stem cells are able to develop into more than one cell type, in a process called haematopoiesis.

Researchers hope to edit the genes of these stem cells so that they produce RBCs with foetal haemoglobin instead of the abnormal protein and can be reintroduced into the body to alleviate the symptoms of SCD.

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Unfortunately, they found that although two types of lab mice had the symptoms of sickle cell disease, their foetal haemoglobin gene and surrounding DNA were not properly configured, making the stem-cell treatment ineffective or even harmful.

These mice called Berkley and Townes mice were genetically engineered in different ways to carry several human haemoglobin genes (replacing the mice genes) so scientists could study sickle cell disease in an animal model.

The researchers removed stem cells from the mice and used CRISPIR-Cas9 to try to turn on the healthy foetal haemoglobin gene. They then put the reprogrammed stem cells back into the mice and monitored the animals for 18 weeks to find out how the treatment affected them.

Surprisingly, 70% of Berkley mice died from the therapy and production of foetal haemoglobin was activated in only 3.1% of the stem cells. On the other hand, treatment did not affect the survival of Townes mice and even activated the foetal haemoglobin gene in 57% of RBCs.

Even then, the levels of foetal haemoglobin produced were seven to 10 times lower than seen when this approach was used in human cells grown in the laboratory and were not high enough to reduce clinical signs of sickle cell disease.

We realised that we did not know enough about the genetic configurations of these mice, says senior author Dr Mitchell Weiss, chair of the haematology department at St Jude Childrens Research Hospital, US.

The researchers sequenced the mices haemoglobin genes and surrounding DNA, and discovered that Berkley mice instead of having a single copy of the mutated human gene had 22 randomly arranged, broken-up copies of the mutated human sickle cell disease gene and 27 copies of the human foetal haemoglobin.

This caused the fatal effects seen and meant that the mice cannot be used to test this treatment in the future.

Our findings will help scientists using the Berkeley and Townes mice decide which to use to address their specific research question relating to sickle cell disease or haemoglobin, concludes Weiss.

Additionally, this work provides a reminder for scientists to carefully consider the genetics of the mice that they are using to study human diseases and find the right mouse for the job.

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Sickle cell disease gene therapy study set back by the mice - Cosmos

GAITHERSBURG, Md., July 5, 2022 /PRNewswire/ -- Novavax,Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced that the European Commission (EC) has approved the expanded conditional marketing authorization (CMA) of Nuvaxovid (NVX-CoV2373) COVID-19 vaccine in the European Union (EU) for adolescents aged 12 through 17. The approval follows the positive recommendation made by the European Medicines Agency's Committee for Medicinal Products for Human Use on June 23, 2022.

"With this authorization, we are extremely pleased to be able to offer our Nuvaxovid COVID-19 vaccine to adolescents in the EU," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "Our protein-based vaccine was developed using an innovative approach to traditional technology and has demonstrated efficacy and safety in both adolescents and adults."

The authorization was based on data from the ongoing pediatric expansionof PREVENT-19, a pivotal Phase 3 trial of 2,247 adolescents aged 12 through 17 years across 73 sites in the U.S., to evaluate the safety, effectiveness (immunogenicity), and efficacy of Nuvaxovid. In the trial, Nuvaxovid achieved its primary effectiveness endpoint and demonstrated 80% clinical efficacy overall at a time when the Delta variant was the predominant circulating SARS-CoV-2 strain in the U.S.

Preliminary safety data from the trial showed the vaccine to be generally well-tolerated. Serious and severe adverse events were low in number and balanced between vaccine and placebo groups, and not considered related to the vaccine. Local and systemic reactogenicity was generally lower than or similar to adults, after the first and second dose. The most common adverse reactions observed were injection site tenderness/pain, headache, myalgia, fatigue, and malaise. There was no increase in reactogenicity in younger (12 to <15 years old) adolescents compared to older (15 to <18 years old) adolescents. No new safety signal was observed through the placebo-controlled portion of the study.

The EC granted CMA for Nuvaxovid to prevent COVID-19 in individuals aged 18 and over in December 2021. In addition to the EC's expanded CMA, Indiahas granted emergency use authorization in the 12 through 17 year-old population.

Authorization in the U.S.

NVX-CoV2373 has not yet been authorized for use in the U.S. and the trade name Nuvaxovid has not yet been approved by the U.S. Food and Drug Administration.

Important Safety Information

For additional information on Nuvaxovid, please visit the following websites:

About NVX-CoV2373NVX-CoV2373 is a protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. The vaccine was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19.

The Novavax COVID-19 vaccine is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2- 8 Celsius, enabling the use of existing vaccine supply and cold chain channels. Use of the vaccine should be in accordance with official recommendations.

Novavax has established partnerships for the manufacture, commercialization and distribution of NVX-CoV2373 worldwide. Existing authorizations leverage Novavax' manufacturing partnership with Serum Institute of India, the world's largest vaccine manufacturer by volume. They will later be supplemented with data from additional manufacturing sites throughout Novavax' global supply chain.

About the NVX-CoV2373 Phase 3 TrialsNVX-CoV2373 continues being evaluated in two pivotal Phase 3 trials.

PREVENT-19 (thePRE-fusion protein subunitVaccineEfficacyNovavaxTrial | COVID-19) is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the efficacy, safety and immunogenicity of NVX-CoV2373 with Matrix-M adjuvant in 29,960 participants 18 years of age and over in 119 locations inthe U.S.andMexico. The primary endpoint for PREVENT-19 was the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second dose in serologically negative (to SARS-CoV-2) adult participants at baseline. The statistical success criterion included a lower bound of 95% CI >30%. A secondary endpoint was the prevention of PCR-confirmed, symptomatic moderate or severe COVID-19. Both endpoints were assessed at least seven days after the second study vaccination in volunteers who had not been previously infected with SARS-CoV-2. In the trial, NVX-CoV2373 achieved 90.4% efficacy overall. It was generally well-tolerated and elicited a robust antibody response after the second dose in both studies. Full results of the trial were published in theNew England Journal of Medicine(NEJM).

The pediatric expansion of PREVENT-19 is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the safety, effectiveness, and efficacy of NVX-CoV2373 with Matrix-M adjuvant in 2,247 adolescent participants 12 to 17 years of age in 73 locations in the United States, compared with placebo. In the pediatric trial, NVX-CoV2373 achieved its primary effectiveness endpoint (non-inferiority of the neutralizing antibody response compared to young adult participants 18 through 25 years of age from PREVENT-19) and demonstrated 80% efficacy overall at a time when the Delta variant of concern was the predominant circulating strain in the U.S.Additionally, immune responses were about two-to-three-fold higher in adolescents than in adults against all variants studied.

PREVENT-19 is being conducted with support from the U.S. government, including the Department of Defense, the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS), and the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health at HHS. BARDA is providing up to$1.75 billionunder a Department of Defense agreement (# MCDC2011-001).

Additionally, a trial conducted in the U.K. with 14,039 participants aged 18 years and over was designed as a randomized, placebo-controlled, observer-blinded study and achieved overall efficacy of 89.7%. The primary endpoint was based on the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second study vaccination in serologically negative (to SARS-CoV-2) adult participants at baseline. Full results of the trial were published inNEJM.

About Matrix-M AdjuvantNovavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About NovavaxNovavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development, and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. NVX-CoV2373, the company's COVID-19 vaccine, has received conditional authorization from multiple regulatory authorities globally, including the European Commission and the World Health Organization. The vaccine is currently under review by multiple regulatory agencies worldwide and will soon be under review in the U.S. for use in adults, adolescents and as a booster. In addition to its COVID-19 vaccine, Novavax is also currently evaluating a COVID-seasonal influenza combination vaccine candidate in a Phase 1/2 clinical trial, which combines NVX-CoV2373 and NanoFlu*, its quadrivalent influenza investigational vaccine candidate, and is also evaluating an Omicron strain-based vaccine (NVX-CoV2515) as well as a bivalent Omicron-based / original strain-based vaccine. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visitwww.novavax.comand connect with us on LinkedIn.

*NanoFlu identifies a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax. This investigational candidate was evaluated during a controlled phase 3 trial conducted during the 2019-2020 influenza season.

Forward-Looking StatementsStatements herein relating to the future of Novavax, its operating plans and prospects, its partnerships, the timing of clinical trial results, the ongoing development of NVX-CoV2373, a COVID-seasonal influenza investigational vaccine candidate, the scope, timing and outcome of future regulatory filings and actions, including Novavax' plans to supplement existing authorizations with data from the additional manufacturing sites in Novavax' global supply chain, additional worldwide authorizations of NVX-CoV2373 for adolescents, the potential impact and reach of Novavax and NVX-CoV2373 in addressing vaccine access, controlling the pandemic and protecting populations, and the efficacy, safety and intended utilization of NVX-CoV2373 are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, challenges satisfying, alone or together with partners, various safety, efficacy, and product characterization requirements, including those related to process qualification and assay validation, necessary to satisfy applicable regulatory authorities; difficulty obtaining scarce raw materials and supplies; resource constraints, including human capital and manufacturing capacity, on the ability of Novavax to pursue planned regulatory pathways; challenges meeting contractual requirements under agreements with multiple commercial, governmental, and other entities; and those other risk factors identified in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Novavax' Annual Report on Form 10-K for the year ended December 31, 2021 and subsequent Quarterly Reports on Form 10-Q, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at http://www.sec.gov and http://www.novavax.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Contacts:InvestorsAlex Delacroix | 240-268-2022[emailprotected]

MediaAli Chartan | 240-720-7804[emailprotected]

SOURCE Novavax, Inc.

Read the original:
Novavax Nuvaxovid COVID-19 Vaccine Conditionally Authorized in the European Union for Adolescents Aged 12 Through 17 - PR Newswire

This article is excerpted from Tom Yeungs Moonshot Investor newsletter. To make sure you dont miss any of Toms potential 100x picks,subscribe to his mailing list here.

On Tuesday, the price of West Texas Intermediate briefly dipped below $100 for the first time since May.

Wall Street says a recession is coming, Philip Aldrick at Bloomberg News reported. Consumers say its already here.

Its hard to miss the warning signs. This year, my neighbors July Fourth celebrations involved no secret trip to New Hampshire for fireworks. Instead, the pyrotechnics were limited to Roman candles, the tiniest of pencil-sized firecrackers and a sigh of relief that their car will soon be paid off.

The numbers dont lie either. In May, the University of Michigan Index of Consumer Sentiment hit 58.4, one of its lowest points since the depths of the 2008 financial crisis. When the average lease payments for a new vehicle swallow up nearly 20% of a median households income, it doesnt take an economist to know that somethings wrong.

Thats the reason why the core Profit & Protection buy list contains no oil or commodity-based stocks. With the U.S. facing the prospect of a drawn-out slowdown, demand for cyclical, low-value materials will be volatile at best.

Instead, regular readers will notice a growing focus on healthcare and other non-cyclical companies that can thrive even during slowdowns. And today, well consider the three healthcare stocks investors need to put on their radar.

On Tuesday, I introduced nine biotech stocks with outstanding growth potential. After decades of development, the first CRISPR gene editing technologies could hit markets by 2023.

Of the group, my top picks are Crispr Therapeutics (CRSP) and Beam Therapeutics (BEAM).

The largest pure-play gene-editing firm is an obvious pick for the Profit & Protection watchlist. It has the most high-quality shots-on-goal among gene-editing startups, and is the blue-chip pick for the watchlist. The firm now has five drugs in clinical trials, while nearest rival Intelliaonly has two.

Crispr also has greater upside potential than Vertex Pharmaceuticals, a diversified biopharma company and drug marketing firm. Vertexs $74 billion market capitalization gives little room for anything better than a 50% gain over the next several years. Meanwhile, Crisprs $5.8 billion market cap gives the smaller startup a 100% upside if just one of its five drug candidates reaches FDA approval in the coming years. Multiple wins could send shares into the $30 billion range, given the average value of a blockbuster drug.

Finally, Crispr has some of the best proof of a working CRISPR-Cas9 platform so far. In 2021, the company revealed positive trial results for CTX001, its treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). All 15 patients with TDT ended the trial transfusion-free, while all seven SDC patients were free of vaso-occlusive crises, a common complication of sickle cell. The drug could launch as soon as 2023, according to industry watchers.

The smaller gene-editing firm is a less obvious choice for the Profit & Protection watchlist. The firm only has three candidates in IND-enabling studies to secure in-human clinical trials and is about 3 to 5 years behind Crispr when it comes to approvals.

Yet Beam makes the Profit & Protection watchlist because it is a leader in base editing the fiercest competitor to CRISPR-Cas9 systems. Rather than acting as genetic scissors the way CRISPR does, Beams base editing chemically transforms individual base pairs from A-T to G-C.

Its a far more precise (and valuable) method of gene editing.

More than 31,000 such mutations in the human genome are known to be associated with human genetic diseases. But CRISPR is not particularly efficient at correcting them, notes Sandeep Ravindran at Nature. Instead, its base editors that excel at single-site mutations.

Base editors also cause fewer genetic errors. Cutting genetic code with CRISPR creates the risk that random bases can be inserted or deleted (indels), altering the gene sequence, Mr. Ravindran continues. By altering just a specific nucleotide without making double-stranded breaks, base editors cause fewer unwanted mistakes. For in-vivo treatments, that gives a far better chance of curing a patient instead of causing uncontrolled genetic mutations.

That gives Beam Therapeutics an unusually high degree of downside protection. Though its drugs might be years away from commercialization, the company could realistically get acquired for its intellectual property if share prices drop another 50%. And if the firms drugs reach viability in 5 to 10 years, investors can expect BEAM shares to rise 5x in short order.

Finally, Luke Lango has one final biotech pick thats upending the world of gene-editing. Rather than focus on specific therapies, this picks and shovels play wraps software and automation around cell engineering workflows.

In other words, its a synthetic biology firm that designs, writes and inserts DNA directly into target cells.

Looking for cells that can produce vaccines? Or yeast that can create food-grade protein through fermentation? These futuristic goals are surprisingly close, thanks to this biotech marvel.

Additionally, the firm currently trades around $3, giving investors a potential 5x to 10x upside. Lukes found plenty of 1,000%+ tech winners before. And this one is a blue-chip gene-editing one that will interest you.

To learn more, check out Lukes Innovation Investor presentation by clicking here.

For all of biotechs potential, every firm has the same problem:

Crispr, Beam and all other research-based firms are long duration stocks that suffer during rate hikes.

These drug research firms typically have zero revenue; their entire business involves testing therapies and selling successful candidates to larger companies. Its how BioNTech (NASDAQ:BNTX) managed to trounce rival Novavax (NASDAQ:NVAX) in Covid-19 vaccine development; the former partnered with Pfizer (NYSE:PFE) to help with production and distribution, while the latter struggled to go it alone.

This research-only business model, however, has a significant downside. Because a biotechs profits are all in the future, a more significant share of its value must be discounted back compared to companies with profits today.

Rising interest rates have biotech investors on the back foot. Discount rates tend to move together with interest rates, so tightening cycles tend to torpedo the value of long duration stocks. Shares of Crispr Therapeutics have already fallen 50% over the past year. And Beam as an earlier-stage startup has naturally fallen even further.

Though Im adding CRSP shares to the Profit & Protection shortlist of stocks to buy, it will take signals of Fed easing before the company earns a place on the core buy list. But when that happens, investors would be wise to scoop up shares of these high-potential stocks with downside protection to match.

P.S. Do you want to hear more about cryptocurrencies? Penny stocks? Options? Leave me a note atfeedback@investorplace.comor connect with me onLinkedInand let me know what youd like to see.

On the date of publication, Tom Yeung did not have (either directly or indirectly) any positions in the securities mentioned in this article.

Tom Yeung, CFA, is a registered investment advisor on a mission to bring simplicity to the world of investing.

Original post:
The 3 Leaders of the Gene Editing Revolution - InvestorPlace

The government is twisting science and mangling the concept of natural to force gene-edited foods, unlabelled, onto our plates.

Environment secretary George Eustice promotes gene editing as a precision scienceone that is all benefits, no downside. Much of the media has fallen for this reassuring but thoroughly fallacious sales pitch, when in fact genome editing is just another form of risky genetic engineering.

Gene editing isnt just a single snip or tweak. As well as any intended genetic modification, it invariably involves large numbers of unintended alterationsto the plant or animal in question.It can also entail multiple cuts into an organisms DNA and the insertion of foreign genes.

For instance, Professor Cathie Martin, who led the development of the gene-edited tomato that could be the first crop of its kind to go on sale in the UK, told The National thatthe process did insert foreign genes that were removed before the final plant was put to market.

And be in no doubt that, as the European Court of Justice has ruled, gene editing does indeed create genetically modified organisms (GMOs).

Yet if the UK government gets away with its new legislation, it would allow plants created using foreign genes to be classified as natural and they will be exempt from regulation and labelling.

This should alarm us. A recent experiment in the US serves as a timely warning of how this volatile technology produces unpredictable, negative and irreversible results.

Researchers at Georgia State University just reported on how their gene editing experiment to reduce aggression in hamsters misfired when their precision alterations instead bred ultra-vicious rodents.In other words, the scientists anticipated a favourable outcome, but got the opposite result.

In the realm of food, the most obvious risks of gene editing are the production of toxins and allergens that could wreak havoc with human and animal health. Who has an appetite for that?

Last years government consultation found 88% of citizens dont want gene-edited foods treated differently to other genetically modified products.

Is it any wonder that, very sensibly, no UK supermarket is willing to say it will stock gene-edited food?

Read more from the original source:
Gene-edited foods shouldn't be enabled in the UK while risks are ignored - The Grocer

The emergence of CRISPR technology, which can be used for gene modification and editing, has been revolutionary for the science community and research field due to its applications for disease and health. Additionally, combining nanotechnology and CRISPR can hold tremendous potential for widening the opportunities for versatile applications, from diseases to agriculture.

Image Credit:Yurchanka Siarhei/Shutterstock.com

CRISPR is an acronym for clustered regularly interspaced short palindromic repeats and is a critical tool that can be used to find specific DNA within cells for the purpose of gene editing; this can also include turning a gene on or off without modifying the sequence.

Conventional gene therapy methods have taken researchers years to gain traction and can be quite expensive; however, the emergence of CRISPR has revolutionized this process into a cheaper and relatively simpler strategy for gene editing.

CRISPR Cas9 proteins found in bacteria can be programmed to find and bind to targeted DNA sequences by providing a complimentary piece of RNA, which can guide the tool for its purpose. Once found, the Cas9 protein can cut the target DNA, which during repair, can lead to mutations and the disablement and overall dysfunction of the gene.

Other types of applications for CRISPR can also include replacing dysfunctional genes, turning genes on and off, instead of modifying the DNA, and even modifying single nucleotide bases within the DNA sequence from one letter to another. However, these can have varying levels of difficulties in practice.

CRISPR/Cas9 delivery can be used for innovative cancer therapy; however, this would require the gene-editing tool to be delivered directly into target cancer cells, requiring the passing of physical barriers and maintaining functionality.

The strategies for delivery of the CRISPR system can include physical and viral vectors.

The physical vectors can include (i) microinjection, (ii) electroporation, and (iii) hydrodynamic delivery. These delivery methods have demonstrated efficiency within in vitro application; however, in vivo results have been more challenging.

Viral vectors have also been subject to challenges within in vivo applications, with limitations comprising immunogenic responses, high production expense, limited capacity with cargo packaging, as well as having possible effects outside of the target area.

Research into non-viral vectors and the possible use of nanotechnology to advance delivery has been explored as a result.

Non-viral nanotechnology-based delivery methods may include the use of nanocarriers, such as polymers, lipids, silica nanoparticles and metallic nanoparticles.

These have been revered for various applications in different fields due to their versatility and have also been used within cancer therapy due to advantages such as having low immunogenicity and a high cargo delivery capacity.

The high potential of the CRISPR/Cas9 system within clinical applications has been recognized globally by researchers, evident from 18 CRISPR-based clinical trials that have been launched since 2016, with almost half being a type of immunotherapy.

Additionally, the use of nanotechnology for furthering this gene-editing tool for diverse applications such as cancer, HPV treatment, as well as agriculture may advance the field of science and healthcare on a global scale.

A previous clinical trial has demonstrated the use of CRISPR/Cas9 on the human papillomavirus (HPV) that is associated with cervical cancer, where the correlated genes, E6 and E7, were destroyed to inhibit cancer cell proliferation and overall survival.

However, while trials have shown effective results, human safety remains a priority, with off-target effects and delivery being a concern for the CRISPR/Cas9 system.

The use of a lipid nanoparticle-based CRISPR/Cas9 system has recently been applied to treat transthyretin amyloidosis (ATTR), becoming the first clinical data of therapeutic gene editing within patients. This nanotechnology incorporation into CRISPR gene editing has demonstrated the potential of using nanotechnology to advance the treatment of genetic disorders through innovative delivery.

With the global CRISPR market being valued at $846.2 million in 2019 and estimations of $10,825.1 million by the year 2030, at a compound annual growth rate of 26.86%, this gene-editing tool is predicted to have a significant impact worldwide.

The potential of editing the genome to modify human disease and disorders may be a tangible reality in the near future, with clinical trials already launched and set in motion.

Additionally, the use of nanotechnology for this advanced gene-editing tool may aid in satisfying concerns over have off-target effects due to the precise targetability characteristic for which this innovative field is known.

The versatility of nanotechnology enables the possibility of aiding the field of medicine as well as other fields, such as agriculture. This is critical as CRISPR systems also face delivery challenges within plants and with the use of nanomaterials, these may be solved.

With the growth of the global population, the essential survival of plants has become even more imperative to sustain the global demand for food, therapeutics, and bioenergy.

Genetic engineering methods are not only significant for medicine, but also for one of the most critical elements of life, namely, plants, to ensure species survival, higher yield, and nutrient density.

Nanotechnology uses for interdisciplinary applications have become a common theme and the opportunities for advancing the lives and health of humans are a critical component for research. The use of nanotechnology-based CRISPR can only advance the field further, providing promising solutions for current challenges.

Demirer, G., Silva, T., Jackson, C., Thomas, J., W. Ehrhardt, D., Rhee, S., Mortimer, J. and Landry, M., 2021. Nanotechnology to advance CRISPRCas genetic engineering of plants.Nature Nanotechnology, 16(3), pp.243-250. Available at: https://doi.org/10.1038/s41565-021-00854-y

Markets, R., 2022.Outlook on the CRISPR Gene Editing Global Market to 2030 - Analysis and Forecasts. [online] GlobeNewswire News Room. Available at: https://www.globenewswire.com/news-release/2021/02/08/2171285/0/en/Outlook-on-the-CRISPR-Gene-Editing-Global-Market-to-2030-Analysis-and-Forecasts.html

New Scientist. 2022.CRISPR. [online] Available at: https://www.newscientist.com/definition/what-is-crispr/

Uddin, F., Rudin, C. and Sen, T., 2020. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.Frontiers in Oncology, 10. Available at: 10.3389/fonc.2020.01387

Xu, X., Liu, C., Wang, Y., Koivisto, O., Zhou, J., Shu, Y. and Zhang, H., 2021. Nanotechnology-based delivery of CRISPR/Cas9 for cancer treatment.Advanced Drug Delivery Reviews, 176, p.113891. Available at: https://doi.org/10.1016/j.addr.2021.113891

Disclaimer: The views expressed here are those of the author expressed in their private capacity and do not necessarily represent the views of AZoM.com Limited T/A AZoNetwork the owner and operator of this website. This disclaimer forms part of the Terms and conditions of use of this website.

Continue reading here:
Nanotechnology and CRISPR; Opportunities and Outlook - AZoNano

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Insights of OPs and PYR cytotoxic potential Invitro and genotoxic impact on PON1 genetic variant among exposed workers in Pakistan | Scientific...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Ambys Medicines, a company pioneering cell-replacement therapies for patients with liver disease, today announced that data from its universal hepatocyte program will be presented at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, which will be held June 15-18, 2022, in San Francisco and virtually. Ambys will present proof-of-concept data on its genetically engineered hypoimmunogenic human hepatocytes during an oral session.

We look forward to presenting data on our universal hepatocyte program that demonstrate the efficient ex vivo genetic engineering of primary human hepatocytes in a metabolic liver disease model using a range of gene editing and delivery technologies, said Markus Grompe, M.D., Founder and Chief Scientific Officer of Ambys Medicines. Were progressing multiple liver cell replacement therapies in development, including our universal human hepatocyte therapy which eliminates the need for immune suppression. These preclinical findings show proof of concept for our engineered hepatocyte replacement approach for severe liver diseases.

Presentation details follow:

Title: Rescue of a Metabolic Liver Disease Model by Genetically Engineered Hypoimmunogenic Human HepatocytesSession: Biotech, Pharma and Academia Bringing Stem Cells to PatientsDate & Time: Thursday, June 16, 2022, at 6:10 p.m. PTRoom: 2004, Level 2

About Ambys Medicines

Ambys Medicines is focused on pioneering cell replacement therapies for patients with liver failure. Ambyss proprietary platform enables the company to be the first and only company able to develop and manufacture functional human hepatocytes at scale. Our scientific approach has the potential to fundamentally transform the treatment paradigm for patients with acute and chronic liver failure and genetic diseases of the liver. Our lead program, AMI-918, is a hepatocyte replacement cell therapy in development to restore lost hepatic function. Beyond AMI-918, we are building a pipeline of next-generation modified hepatocytes that will rapidly expand the range of treatable patient populations. Learn more at ambys.com and follow us on Twitter, LinkedIn, and Instagram.

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Ambys Medicines to Present Data from Universal Human Hepatocyte Program at the 2022 ISSCR Annual Meeting - Business Wire