Stem Cell Clinic

Umbilical cord stem cells have the potential to treat COVID-19 – Open Access Government

By Stem Cell Doctors

The year of 2020 has been unlike any other year. Since January, the global COVID-19 pandemic has impacted every aspect of our lives affecting our health, our economy and our livelihoods. With the world in lockdown, the race is on to find a solution to the fast spreading deadly virus.

Scientists and researchers are uniting in a global effort to develop cutting edge technologies and novel treatments for the virus. As a result, numerous early studies have emerged, proposing stem cells as an effective method in treating the disease, with several clinical trials now underway. In particular, these studies have focused on the therapeutic potential of stem cells from the umbilical cord in the treatment of COVID-19 patients.

Recently, mesenchymal stem cells (MSCs) harvested from umbilical cord blood have attracted significant attention in clinical trials for COVID-19 due to their immunomodulatory and regenerative properties.

When an individual contracts COVID-19, an immune overreaction is triggered in the body, producing large amounts of inflammatory factors whilst simultaneously destroying numerous essential cells. This results in in an overproduction of immune cells and cytokines, which researchers refer to as an inflammatory storm.

Here, the idea to use MSCs in the treatment of COVID-19 patients was coined, to prevent the storm release of cytokines by the immune system and promote repair by reparative properties of the stem cells.

These MSCs oppose viral infections due to the presence of specific cytokines improved qualities. Subsequently, they can prevent lung tissue damage by counteracting the cytokine storm through their immunomodulatory function and differentiation ability to regenerate and reconstruct damaged tissue. As a result, it has been demonstrated that MSCs significantly reduce the risk of COVID-19 pneumonia and respiratory failure.

The application of these stem cells seems to be particularly effective in cases of critically ill patients, as reported in the Aging and Disease journal. In this study, seven COVID-19 patients were administered with MSCs from the umbilical cord. After two days of receiving the MSC treatment, the pulmonary function and symptoms of the seven patients were significantly improved without any adverse effects.

Another study of a critically ill COVID-19 patient has shown remarkable reversal of symptoms using MSCs. The 65-year-old woman, whose condition had progressed despite intensive therapy, was on a ventilator and was showing evidence of liver damage. The patient was treated with three intravenous infusions of MSCs, three days apart. Within four days of the first dose of the MSC treatment, she was able to come off the ventilator and walk again.

The FDA have also recently authorised compassionate use of stem cells, including MSCs and cells taken from the placenta, for intravenous infusions in patients with a severe prognosis. This has come following the success of a placenta-based cell-therapy to treat COVID-19 patients, which has resulted in the coronavirus-recovered patients being discharged from hospital.

To date, there are 17 completed clinical trials which have investigated the application of MSCs in treating COVID-19 as well as more than 70 other trials in this regard currently ongoing. Whilst the clinical use of MSCs in treating COVID-19 is still in its early phases, the initial results are certainly promising.

The use of mesenchymal stem cells (MSCs) has gained global traction in recent years due to their vast regenerative potential and their role in immune disorders. In particular, the umbilical cord has been identified as the richest and purest source of these stem cells. These cells can be easily harvested, isolated, cultured and used in cell-based therapy, from basic research to clinical trials.

Stem cells from the umbilical cord are widely recognised by doctors as an invaluable resource for regenerative therapies as they can transform into a wide array of tissue types. They play a significant role in repair and renewal of damaged tissue, and clinical studies have even demonstrated its ability to regenerate entire organsUmbilical cord blood stem cells were first introduced in the 1980s, when it was used to successfully treat a young boy with Fanconi anaemia. Today, cord blood stem cells can treat more than 80 conditions, including many blood cancers and immune disorders such as leukaemia, lymphoma and severe combined immunodeficiency (SCID).

These stem cells are also now central to hundreds of promising clinical trials for common and often life-threatening conditions such as arthritis, heart disease and multiple sclerosis.

To find out more about umbilical cord stem cells and their role in regenerative medicine, visit cells4life.com

References

Campbell & MacDonald, (2020) Exploring the Utility of Stem Cell Therapy for COVID-19, BioPharma, available at:

https://www.technologynetworks.com/biopharma/articles/exploring-the-utility-of-stem-cell-therapy-for-covid-19-334068

Bing Liang et al., (2020) Clinical remission of a critically ill COVID-19 patient treated by human umbilical cord mesenchymal stem cells, ChinaXiv, available at:http://chinaxiv.org/abs/202002.00084

Golchin et al., (2020) Mesenchymal Stem Cell Therapy for COVID-19: Present or Future, Stem Cell Reviews and Reports, available at:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7152513/

Zikuan Leng et al., (2020) Transplantation of ACE2- Mesenchymal Stem Cells Improves the Outcome of Patients with COVID-19 Pneumonia, Aging and Disease, available at:http://www.aginganddisease.org/EN/10.14336/AD.2020.0228

Michael Hill (2020) Hospital uses placenta cells to try to improve two COVID-19 patients NJTV News, available at:

Hospital uses placenta cells to try to improve two COVID-19 patients

Pluristem (2020) Pluristem Reports Preliminary Data from its COVID-19 Compassionate Use Program, Treating Seven Patients with Acute Respiratory Failure, Pluristem, available at:

https://www.pluristem.com/wp-content/uploads/2020/04/PSTI-PR-Follow-up-on-Covid-19-treatments-FINAL-FOR-RELEASE.pdf

Ballen et al., (2013) Umbilical cord blood transplantation: the first 25 years and beyond, Blood, available at:

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3952633/

Parents Guide to Cord Blood Foundation, (2020) Diseases Treated, available at: https://parentsguidecordblood.org/en/diseases

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Umbilical cord stem cells have the potential to treat COVID-19 - Open Access Government

Growing Number of Patients Suffering From Neurological Disorder to Support the Growth of Global Autologous Cell Therapy Market, TMR – PRNewswire

By Stem Cell Doctors

ALBANY, New York, May 11, 2020 /PRNewswire/ -- As a result of growing pandemic of COVID-19, the pharmaceutical industry is investing a major amount in research and development of an effective vaccine that can help the doctors to save lives and help controlling the pandemic. As per a recent analysis by Transparency Market Research over pharmaceutical sector, it was found that the global autologous cell therapy market is witnessing a major push. The market is projected to grow exponentially during the tenure of 2019 to 2027 and is expected to witness a substantial 18.1% CAGR during this tenure. Moreover, the global autologous cell therapy market is expected to register a value of US$ 34.7 Bn by the end of 2027, says a recent report by Transparency Market Research.

"COVID-19 has impacted the economy at global level. With the death tolls still on the rise in developed and developing countries, the pandemic nowhere seems to get stop. However, the pharmaceutical sector is constantly investing in research and development. Nevertheless, the industry is majorly investing in autologous cell therapyto find an effective cure for CORONA Virus. Owing to these research and development, the global autologous cell therapy market is projected to grow exponentially during the tenure of 2019 to 2027" Transparency Market Research.

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Major Finding in Global Autologous Cell Therapy Market Study

Explore the latest study on global autologous cell therapy market under the title: Autologous Cell Therapy Market (Source - Bone Marrow and Epidermis; Application - Neurology, Orthopedics, Cancer, Wound Healing, CVD, and Autoimmune; End User - Hospitals, Ambulatory Centers, and Research Centers) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast 2019 2027 at https://www.transparencymarketresearch.com/report-toc/715

Major Drivers Propelling the Growth of Global Autologous Cell Therapy Market

Growing cases of Neurological Disorders to Propel the Growth

The world is witnessing a massive growth in the number of patients suffering from neurological diseases. The report by Transparency Market Research states that there are more than 50 Mn people suffering from autoimmune disorder which can cause a painful death. In order to develop an effective drug that can help the neurological patients, the pharmaceutical industry is investing massive in autologous cell therapywhich as a result is propelling the growth of global autologous cell therapy market during the tenure of 2019 to 2027.

Strong Government Support to Boost the Growth of the Autologous Cell Therapy Market

Governments across the globe are encouraging pharmaceutical industry to develop an effective cure for Parkinson's, nervous breakdowns, and most importantly COVID-19. This has significantly helped the players to develop new drugs. Owing to this support, the global autologous cell therapy market shall witness a substantial growth during the tenure of 2019 to 2027, states the report by Transparency Market Research.

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Major Challenges Mentioned in the Global Autologous Cell Therapy Market

The major challenge that might impede the growth of global autologous cell therapy market is the cost of the therapy. Since the cost of therapy is quite high, the some of the patient might not be able to afford it which might deplete the growth of global autologous cell therapy market during the tenure of 2019 to 2029. However, with ongoing research and development in the pharmaceutical industry, there are high chances that the world might see a cost effective therapy in the global autologous cell therapy market in future.

Global Autologous Cell Therapy: Regional Analysis

North America shall account for the largest share in the global autologous cell therapy market in future. It is noticeable that the regional front of the global autologous cell therapy market was dominated by this particular region in 2018. This dominance is the result of improving healthcare infrastructure, technological development and supportive government efforts. Owing to these factors the global autologous cell therapy market is expected to be dominated by North America during the tenure of 2019 to 2027.

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Competitive Analysis

The global autologous cell therapy market is highly progressive yet highly consolidated. The nature of the market is the result of presence of AA handful of prominent players. However, due to this the new players are unable to enter the global autologous cell therapy market. To overcome this situation, the new players are merging and collaborating with the established players of global disposable face mask market.

The global autologous cell therapy market is segmented in the basis of:

Global Autologous Cell Therapy Market, by Source

Global Autologous Cell Therapy Market, by Application

Global Autologous Cell Therapy Market, by End-user

Global Autologous Cell Therapy Market, Region

Explore Transparency Market Research's award-winning coverage of the global Healthcare industry:

Autoimmune Disease Diagnostics Market- the global autoimmune diseases diagnostics market is expected to exhibit a CAGR of 3.8%. The global autoimmune disease diagnostics market is likely to reach US$17.06 bn by 2023. The stiff price competition among players is expected to impact the growth of the market in a negative way.

Stem Cells Market- The global market for stem cells is projected to reach a value of US$270.5 bn by the end of 2025. The market is likely to exhibit a strong 13.80% CAGR between 2017 and 2025

Cell Therapy Market-The study reports an insight about the global cell therapy market, along with its current and the past market situation. This enables the customers to comprehend the situation before entering or investing in the market. The report gives driving components fueling the development of the market, restrictions which are probably going to limit the market development, and so forth.

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Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

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Growing Number of Patients Suffering From Neurological Disorder to Support the Growth of Global Autologous Cell Therapy Market, TMR - PRNewswire

Q&A: ‘Survivor’ champ Ethan Zohn’s deep Colorado connection, who he was shocked to see on the beach and his epic episode – Colorado Springs Gazette

By Stem Cell Doctors

The hit CBS reality series Survivor wraps up its 40th, and quite possibly best, season when Winners at War crowns a champion Wednesday. In preparation for the finale, I spoke with fan favorite and three-time contestant Ethan Zohn.

The winner of Survivor: Africa and, more importantly, a man who has defeated cancer twice, discussed this latest season, why he frequently comes to Colorado and who he was shocked to see on the beach when season 40 started.

Have you been to Colorado before?

Ive been to Colorado so many times! I guess my strongest connection is through a bunch of different cancer organizations that I've been involved with that are either based in Colorado or host events in Colorado. First Descents is one of them, the other one is called Epic Experience. They're basically outdoor adventure camps for young adult, cancer survivors. I work with them as an ambassador.

The last one, which is a pretty neat thing called CancerCon, is basically the largest collection of young adult cancer survivors in the world. They come to Denver each year for a big conference of class speakers and parties. ... I also visited Colorado for the research on hemp and CBD and THC and cannabis.

I've been to the Air Force Academy a couple of times. I gave a speech at the University of Colorado-Colorado Springs within the last two years. Beautiful area. Oh my god, I loved it.

Ive read that while you were out on Survivor this last go round that the CBD oil was something that you missed.

I definitely was missing that. I'm living a nice cushy life here in New Hampshire and I got my wife and my cats and my piles of CBD and then all of a sudden it's now taken away from you and now you're in the middle of this game of Survivor with all the added stress on that and they wouldn't even let me take my CBD. They actually took my consideration to the top and asked if I could use it because they let you bring prescription medicine there if you need it, but they wouldn't let me take CBD or THC because it's illegal in Fiji.

It was 15 years between Survivor: All Stars and Winners at War. What was the motivation behind wanting to come back?

Well, I mean, one, it is a season everyone has been waiting for, for both players and both fans, I think. They're taking the 20 most popular winners of all time, bringing them back and letting us battle for not $1 million, but $2 million. We didn't know it was $2 million when we signed up for it. They told us that on the beach. But one, it's an honor to be asked back. Two, I've always wanted to play again, but because of some of the health challenges that I've been through, I haven't been able to.

I think it was 2010, I was literally locked away getting my second stem cell transplant in a hospital in New York City, watching I think season 20, which is Heroes Versus Villains, and thinking, "Man, if I stay alive long enough or if I get out of this thing and I'm OK, I want to be able to play Survivor again." It was just a dream of mine and it took me 10 years to get ready mentally, physically, spiritually to go play this game of Survivor. When they called, I was like, "Yeah, you bet t I want to play." For me just to be healthy enough to do anything like that is a blessing, so I jumped at the chance.

Were there any winners that you expected to see but that weren't there?

I expected to see Richard Hatch, I expected to see Tina, but other than that I would've liked to have seen a few more old school winners, I think it was a little bit skewed. There was like 13 new schoolers and seven old schoolers and so it just threw the dynamic off a little bit and I don't think anyone really suspected that it was going to be such a landslide in terms of a new schoolers just picking off the old schoolers one by one. But I would like to have seen a more even split.

When did you know who everybody was on the cast? When did that happen?

When you get to Fiji to film, there's a couple of days where you've got to do stuff like medical checks and wardrobe and just sign papers and do interviews and stuff like that. So they split up men and women. At that time I knew the guys going into it. But you're not allowed to talk. They keep everyone separate, pretty strict in that sense, but you could see who they were. I originally thought it would be guy versus girls. I said, "Oh all right, cool." Then we started the game and I see Amber there and I was like, "Holy crap. Rob and Amber are both here?" That's what I realized after that, that's why I thought they probably separated us that way early on. But I was completely shocked to see Amber there. I think everyone was shocked to see Amber there.

We have to talk about probably what's going to be your defining moment from the season. This is where you had to go get 20 logs one at a time and bring them back to camp. It was such an iconic Survivor moment seeing someone struggling, yet never giving up. Tell me what was going through your mind during that particular challenge?

I thought at first, I'd be fine. I thought that 20 times up the hill, I went up once before and I thought it wouldn't be a problem. I definitely went out a little bit too fast. I tried to keep up with Natalie, who is an absolute Olympic champion at everything out there right now. So, yeah, I just think basically, it was probably one of the most difficult things I've ever had to put my body through. You're completely depleted, you're hungry, you're tired, you're thirsty. We didn't eat that day. The challenge got the best of me, I was walking, I think it was around log 15 or 16 and I completely lost my depth perception.

I tried to step up over a rock and I would always be like, 3, 5 inches short and I kept tripping and stumbling and I got around the corner and I slipped and kind of fell. When I fell then the doctors stepped in and they're like, "Maybe you should take a little bit of a rest." Then I passed out. Luckily the doctors are there when that happened. But what was interesting about this, and where I'm going with it is, I thought: Here you are in the middle of this game of Survivor, this cutthroat game, where I was competing forn $2 million dollars and I'm competing with three other women to get a fire token. And I thought, when I was down, they'd be beat up or kick you while I was down or spit on me as they walk by or whatnot.

But what ended up happening is they basically helped me up that last leg of the race. No one wants to go up that mountain once a day, nevertheless, 20 times a day, and here they're going off one more extra time, 21 times for me just to give me the energy and the power to get up there. So it was really nice to see how compassion and empathy and just human to human relationships transcended the game for that moment. I think it was just a really beautiful moment and I don't think I would have been able to make it up that hill if they weren't there with me.

What would you say is the hardest thing to adjust to going from being a viewer to an actual participant?

The days are really long. You guys see all the options and all the fun stuff, but there's 23 hours left in the day of stuff that you guys don't see. There's a lot of boredom and there's a lot of just chatting, you don't understand how little sleep we get, how little food we get. I don't think that's really clearly described. Sleeping conditions are absolutely horrible. It's freezing at night, it's warmer during the day. So just kind of like that stuff I think has been over the years has minimized. You don't necessarily see survival even being something you need to be good at in order to do well on the show Survivor.

Back in the day, and I don't want to be that guy, but survival mattered and the entire show is based on survival and location. And the challenges were big and huge and epic and it held a lot of weight in terms of if you did well in a challenge or not. Now the challenges don't even really matter as much in terms of strategy. You obviously want to win, but if you do bad in a challenge, you're not going to get voted off because of it. But to answer your question, I guess it's just the uncomfortableness, lack of sleep, lack of food, dealing with everyone else's bull--- all the time, that's the stuff you don't see because it's just minimized.

I'm assuming you think this will be your last season?

Oh yeah. Dude, I feel they should lose my number, Ill say during the finale, "Don't ever call me again." Unless I do a random talker and show up for the night to be some sort of, I don't know, mentor type situation. I would do that, but there's no way I'd play again.

With that in mind, let me ask you a couple of questions in regards to your overall experience. What is your favorite "Survivor" memory?

My favorite memory is definitely visiting the village, in Survivor: Africa. I got to play hacky sack with my luxury item with all these children in the parking lot of a hospital and I later found out that all these kids were HIV positive. It was at that moment I decided that I wanted to do something with my money if I did win Survivor: Africa. I got back and I started this organization, Grassroots Soccer. Since Survivor: Africa" and Survivor: All Stars, we're now in 60 countries and we've graduated over 2.9 million kids from the program, all from a little reality show. I think it's pretty neat.

That's fantastic. What is your least favorite "Survivor" memory?

I would say every day on the Edge of Extinction. It's not a pleasant place to be. It's purgatory, you are between life and death, but I would say it's more leaning towards hell than any other direction.

Who would you put on your "Survivor" Mount Rushmore?

Oh man, that's tough. It's going to be all old school because Im completely biased. Myself, Boston Rob, who already has his own freaking statue, and Parvarti. The last one is between Hatch just because he's the original gangster. But I also feel like Tyson just because he's like a good friend of mine and he deserves to be etched in stone.

What can we expect in the finale Wednesday night?

As you know it's the epic second play-in challenge and everyone is incredibly jazzed up. Some people have idols, some people have some advantages and it's just going to be a really exciting and epic play-in challenge. The finale is going to be fireworks, these last three hours are going to be absolutely insane.

Gazette TV critic Terry Terrones is a member of the Television Critics Association and the Broadcast Television Journalists Association. You can follow him on Twitter at @terryterrones.

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Q&A: 'Survivor' champ Ethan Zohn's deep Colorado connection, who he was shocked to see on the beach and his epic episode - Colorado Springs Gazette

EDITORIAL: Excessive haste in approving COVID-19 drugs is a no-no : The Asahi Shimbun – Asahi Shimbun

By Stem Cell Doctors

A number of trials and studies are under way on several drugs that have emerged as candidates in the search for treatments for new coronavirus infections.

Remdesivir, one of those drugs, has been approved with exceptional speed by the health ministry after the United States permitted emergency use on patients in severe conditions.

The ministry took the unusual procedure under a special framework that allows domestic screening processes to be simplified.

There are, of course, concerns about possible side effects. There should be a follow-up study on all patients to whom the drug is administered, and its effectiveness should be carefully reviewed.

Many people have expressed hopes for the early development of COVID-19 therapeutic drugs. Concerned parties should do their best to meet those expectations.

That does not mean, however, that procedures for verifying drug efficacy and safety can be slighted.

Doctors on the front lines have set their sights on Avigan. The drug, which was developed by a Japanese manufacturer, has been approved as a treatment for new types of influenza expected in the future.

Clinical tests are continuing for having Avigan also approved as a COVID-19 treatment. At the same time, patients are taking the medicine in medical research (clinical studies) in which many health care institutions are participating.

Prime Minister Shinzo Abe said during a news conference on May 4 that he hopes to have Avigan approved as a COVID-19 drug by the end of May.

It has been administered in nearly 3,000 cases, he said. I have been told the drug is turning out to be effective.

During a talk aired May 6 on internet TV, Kyoto University professor Shinya Yamanaka, a Nobel Prize-winning stem cell biologist, asked Abe to help speed the timeline by adding a word from the prime minister to the project.

However, excessive haste is a no-no.

Many COVID-19 patients have recovered without using Avigan. It will be difficult to determine how effective the medicine is simply based on reports of cases where conditions have improved under the use of Avigan.

Efficacy should not be evaluated under the influence of foregone anticipation. It should also be kept in mind that there were reports of serious side effects in experiments on animals.

We are reminded of Iressa, a cancer agent that Japan approved in 2002 ahead of the rest of the world.

It was initially touted as a new dream medicine with few side effects, but no sooner was it released for sale than there were cases of deaths that were likely caused by its side effects.

The serious damage arose because Iressa was put to widespread use under insufficient information, including on the types of patients for whom the medicine is effective.

Abe has reiterated that anyone will be entitled to take Avigan on request if approval is obtained from the ethics committee of the patient's hospital. Those remarks, however, could be a source of confusion.

Not every hospital will be able to prescribe Avigan. In addition, if patients who are deemed not eligible were to ask for the drug, pinning hopes on Abes remarks, that could increase the workload of health care workers who have to deal with such requests.

The government has said it will provide Avigan to more than 40 countries free of charge.

Its approval process should go through the proper steps without omission because the medicine must not only benefit patients in Japan but also contribute to international cooperation.

We should deal appropriately with the coronavirus crisis without forgetting the history of suffering induced by drugs.

--The Asahi Shimbun, May 10

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EDITORIAL: Excessive haste in approving COVID-19 drugs is a no-no : The Asahi Shimbun - Asahi Shimbun

Tens of thousands of cancer patients STILL havent received NHS coronavirus shielding letters – The Sun

By Stem Cell Doctors

TENS of thousands of cancer patients still havent received NHS coronavirus shielding letters, a top doctor is warning.

Countless individuals across the country should have been contacted to tell them they are clinically extremely vulnerable and need to take stringent measures to protect themselves from the coronavirus threat.

1

The NHS started writing to high-risk people seven weeks ago urging them to remain at home at all times.

However, Peter Johnson, national clinical director for cancer, has revealed that droves of cancer patients have not yet received their letters - and he is now urging charities to assist in tracing the missing patients.

His appeal comes after Government yesterday increased its estimate of the number of people at greatest risk of severe illness should they become infected with Covid-19 from 1.5million to 2.5million.

In the new a 50-page lockdown plan, it statedthat it had identified 2.5 million people who were clinically extremely vulnerable and advised to shield.

Professor Johnsons letter, seen by The Times, reads: "We are still receiving reports of cancer patients who believe that they should have received a shielding letter but have not yet received one or have not been added to the national list.

It is crucial that those who are clinically extremely vulnerable receive a letter advising them to shield

"It is crucial that those who are clinically extremely vulnerable receive a letter advising them to shield.

"As well as ensuring their safety through shielding, the letter provides evidence to employers and enables these patients to access government support to the shielding cohort."

Those deemed "high-risk" include those with suppressed immune systems, such as cancer and transplant patients.

These individuals are strongly advised to stay at home at all times and avoid any face-to-face contact -with couples being forced to sleep in separate bedrooms and parents advised to keep away from their children.

Those who are shielding are also not allowed to attend any gatherings at all, with very limited exceptions.

Who is clinically extremely vulnerable?

Expert doctors in England have identified specific medical conditions that, based on what we know about the virus so far, place someone at greatest risk of severe illness from Covid-19.

Clinically extremely vulnerable people may include the following people. Disease severity, history or treatment levels will also affect who is in the group.

1. Solid organ transplant recipients.

2. People with specific cancers:

3. People with severe respiratory conditions including all cystic fibrosis, severe asthma and severe chronic obstructive pulmonary (COPD).

4. People with rare diseases that significantly increase the risk of infections (such asSCID, homozygous sickle cell).

5. People on immunosuppression therapies sufficient to significantly increase risk of infection.

6. Women who are pregnant with significant heart disease, congenital or acquired.

People who fall in this group should have been contacted to tell them they are clinically extremely vulnerable.

If youre still concerned, you should discuss your concerns with your GP or hospital clinician.Source: GOV.UK

However, the messaging system has been plagued with problems - with 107,000 being wrongly told they were high risk, according to the Health Service Journal.

And GPs have only now started sending out letters saying they can carry out essential activities outdoors - more than a month after they were locked down.

On top of this, 10,000 dead people were also sent letters from NHS England.

Macmillan Cancer Support are now urging the Government to get on top of the missing letters.

Lynda Thomas, from the chairty, said: We must urgently see a plan from the government that sets out how every vulnerable person will be identified and supported to stay safe as they face further months of isolation in total lockdown.

The Government is currently advising people to shield until the end of June and is regularly monitoring this position.

Those in the shielding cohortare eligible for help, such as food parcels and prescription deliveries, from the NHS volunteer army.

An NHS spokesman said: Hospitals and family doctors have helped identify and contact more than 1.28 million people that have been asked to take particular care staying at home, but if anyone with cancer is concerned that they should also be added they can contact their GP or hospital clinician.

Matt Hancock is considering extending the 12-week shielding period for the most vulnerable Brits.

The Health Secretary told Sky News: We've asked those being shielded to avoid as much contact as possible for 12 weeks."

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"We may have to extend that 12 weeks but we hope not to.

Mr Hancock said he hoped to release some measures before a vaccine is found if cases continue to fall.

He added: "If we succeed in doing that we will be able to take clinical advice on whether it is safe for those who are shielded to, for instance, hug their grandchildren. That is right at the front of my mind."

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Tens of thousands of cancer patients STILL havent received NHS coronavirus shielding letters - The Sun

2020 Insights on the Worldwide Induced Pluripotent Stem Cell Industry – ResearchAndMarkets.com – Business Wire

By Induced Pluripotent Stem Cells

DUBLIN--(BUSINESS WIRE)--The "Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report" report has been added to ResearchAndMarkets.com's offering.

Since the discovery of induced pluripotent stem cells (iPSCs) a large and thriving research product market has grown into existence, largely because the cells are non-controversial and can be generated directly from adult cells. It is clear that iPSCs represent a lucrative market segment because methods for commercializing this cell type are expanding every year and clinical studies investigating iPSCs are swelling in number.

Therapeutic applications of iPSCs have surged in recent years. 2013 was a landmark year in Japan because it saw the first cellular therapy involving the transplant of iPSCs into humans initiated at the RIKEN Center in Kobe, Japan. Led by Masayo Takahashi of the RIKEN Center for Developmental Biology (CDB), it investigated the safety of iPSC-derived cell sheets in patients with macular degeneration.

In another world-first, Cynata Therapeutics received approval in 2016 to launch the world's first formal clinical trial of an allogeneic iPSC-derived cell product (CYP-001) for the treatment of GvHD. Riding the momentum within the CAR-T field, Fate Therapeutics is developing FT819, its off-the-shelf iPSC-derived CAR-T cell product candidate. Numerous physician-led studies using iPSCs are also underway in Japan, a leading country for basic and applied iPSC applications.

Key Topics Covered:

1. Report Overview

2. Introduction

3. History of Induced Pluripotent Stem Cells (IPSCS)

3.1 First iPSC generation from Mouse Fibroblasts, 2006

3.2 First Human iPSC Generation, 2007

3.3 Creation of CiRA, 2010

3.4 First High-Throughput screening using iPSCs, 2012

3.5 First iPSCs Clinical Trial Approved in Japan, 2013

3.6 The First iPSC-RPE Cell Sheet Transplantation for AMD, 2014

3.7 EBiSC Founded, 2014

3.8 First Clinical Trial using Allogeneic iPSCs for AMD, 2017

3.9 Clinical Trials for Parkinson's disease using Allogeneic iPSCs, 2018

3.10 Commercial iPSC Plant SMaRT Established, 2018

3.11 First iPSC Therapy Center in Japan, 2019

4. Research Publications on IPSCS

4.1 Categories of Research Publications

4.2 Percent Share of Published Articles by Disease Type

4.3 Number of Articles by Country

5. IPSCS: Patent Landscape

5.1 Timeline and Status of Patents

5.2 Patent Filing Destinations

5.3 Patent Application Trends iPSC Preparation Technologies

5.4 Patent Application Trends in iPSC Differentiation Technologies

5.5 Patent Application Trends in Disease-Specific Cell Technologies

6. Clinical Trials Involving IPSCS

6.1 Current Clinical Trials Landscape

6.1.1 Clinical Trials Involving iPSCs by Major Diseases

6.1.2 Clinical Trials Involving iPSCs by Country

7. Funding for IPSC

7.1 Value of NIH Funding for iPSCs

7.1.1 NHI's Intended Funding Through its Component Organizations in 2020

7.1.2 NIH Funding for Select Universities for iPSC Studies

7.2 CIRM Funding for iPSCs

8. Generation of Induced Pluripotent Stem Cells: An Overview

8.1 Reprogramming Factors

8.2 Overview of Four Key Methods of Gene Delivery

8.3 Comparative Effectiveness of Different Vector Types

8.4 Genome Editing Technologies in iPSCs Generation

9. Human IPSC Banking

9.1 Cell Sources for iPSCs Banking

9.2 Reprogramming methods used in iPSC Banking

9.3 Workflow in iPSC Banks

9.4 Existing iPSC Banks

10. Biomedical Applications of IPSCS

10.1 iPSCs in Basic Research

10.2 iPSCs in Drug Discovery

10.3 iPSCs in Toxicology Studies

10.4 iPSCs in Disease Modeling

10.5 iPSCs in Cell-Based Therapies

11. Other Novel Applications of IPSCS

11.1 iPSCs in Tissue Engineering

11.2 iPSCs in Animal Conservation

11.3 iPSCs and Cultured Meat

12. Deals in the IPSCS Sector

12.1 $250 million Raised by Century Therapeutics

12.2 BlueRock Therapeutics Launched with $225 Million

12.3 Collaboration between Allogene Therapeutics and Notch Therapeutics

12.4 Acquisition of Semma Therapeutics by Vertex Therapeutics

12.5 Evotec's Extended Collaboration with BMS

12.6 Licensing Agreement between Allele Biotechnology and Astellas Pharma

12.7 Co-development Agreement between Allele & SCM Lifesciences

12.8 Fate Therapeutics Signs $100 Million Deal with Janssen

12.9 Allele's Deal with Alpine Biotherapeutics

12.10 Editas and BlueRock's Development Agreement

13. Market Overview

13.1 Global Market for iPSCs by Geography

13.2 Global Market for iPSCs by Technology

13.3 Global Market for iPSCs by Biomedical Application

13.4 Global Market for iPSCs by Cell Types

13.5 Market Drivers

13.6 Market Restraints

13.6.1 Economic Issues

13.6.2 Genomic Instability

13.6.3 Immunogenicity

13.6.4 Biobanking of iPSCs

14. Company Profiles

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/t6jcqp

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2020 Insights on the Worldwide Induced Pluripotent Stem Cell Industry - ResearchAndMarkets.com - Business Wire

Cell and Gene Therapy Catapult links with Japan’s CiRA Foundation – PharmaTimes

By Induced Pluripotent Stem Cells

The Cell and Gene Therapy Catapult (CGT Catapult) and Kyoto, Japan-based CiRA Foundation are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation.

With the move, the groups are hoping to further the application of iPS cell technologies for the manufacture of regenerative medicine products.

The potential of distinct iPS cell lines for differentiation into specific cell types is usually biased towards some cell line-specificity which, the parties note, is very difficult to predict. As such, in order to select an appropriate iPS cell line for clinical trials it is necessary to differentiate several candidate cell lines, which is time-consuming.

CGT Catapult and CiRA plan to explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.

We are honoured to collaborate with CiRA Foundation, an organisation with world-leading capabilities in iPS cell technology, and to be the first group to utilise CiRAs innovative iPS cell lines outside of Japan, said CGT's chief executive Matthew Durdy

This is a truly exciting project to help further the application and manufacture of iPS cells into cell therapies. We look forward to progressing this promising research together, which has potential benefits for the global advanced therapies industry.

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Cell and Gene Therapy Catapult links with Japan's CiRA Foundation - PharmaTimes

Covid 19 Pandemic: Parkinsons disease treatment Market Size, Share & Trends Analysis Report By Product, By Technology, By Application, By End Use,…

By Gene Therapy Clinics

Global Parkinsons disease treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Parkinsons disease treatment Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

>>Need a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=71&RequestType=Sample

Global Parkinsons disease treatment Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2018-2024Scope of Global Parkinsons disease treatment Market Reports

Parkinsons disease is a neuro degenerative disorder in which parts of the brain become progressively damaged over many years. It is a type of movement disorder. It occurs when nerve tissue unable to produce a chemical named Dopamine which is used to send signals that help you move your body. Parkinsons disease usually developed for aged people i.e. above 60 years, but it can also start earlier. It is more common in men than in women. The cause of Parkinsons disease is unknown, but researchers think that both genetic and environmental factors are involved. Primary symptoms include tremors, Stiffness, Slowness, impaired balance, mild memory & Thinking problem and shuffling gait. Secondary symptoms include Anxiety, Depression, Fatigue, Low blood pressure, muscle cramp, Speech & communication problem, skin & Dental Problem and Dizziness, and Dementia. Risk of Parkinsons disease increases with age and the disease mainly affects person over 50 years of age. The development of Parkinsons disease and the degree of destruction differ from person to person. Many people with Parkinsons disease live long productive lives, whereas others become disabled much more quickly. As symptoms worsen, it may become difficult to walk, talk, and complete simple tasks. Malnutrition and weight maintenance is common problem for people with Parkinsons disease. There is no test to diagnose Parkinsons disease with certainty. Treatment options include medication or surgical treatment. Combination of Levodopa with carbidopa is considered as a gold standard for symptomatic treatment of Parkinsons disease. Surgical treatment options include deep brain stimulation and Carbidopa/levodopa enteral suspension therapy but surgical therapy is effective only for patients who have previously responded to Levodopa therapy. Commercially available drugs for treatment of Parkinsons disease are Sinemet, Safinamide, Rotigotine, Ropinirole, Pramiprexol, Amantidine, Cycloset, Parlodel (Bromocriptine Mesylate), Rytary, Azilect, Northera, Stalevo, Comtess/Comtan and others. More than past decade, huge improvement has been made in the treatment of Parkinsons disease such as identification of new therapeutic targets through genetic research, understanding the common mutations that contribute to Parkinsons disease etc.

Global Parkinsons diseases Treatment Market report is segmented on the basis of Medication, Medical devices, End user, Distribution Channels and Geography. Based on Medication Global Parkinsons disease Treatment Market is classified into Dopamine Precursors, Peripheral Decarboxylase Inhibitors, COMT (Catechol-O-methyltransferase) Inhibitors, MAO (Monoamine Oxidase), Inhibitors and Others (anticholinergics, antihistaminics etc.)

On the basis of Medical Devices Global Parkinsons disease Treatment Market is classified into Deep Brain Stimulation (DBS) Devices, Carbidopa/Levodopa Enteral Suspension (Duopa) delivery devices and others. On the basis of End User Global Parkinsons disease Treatment Market is classified into Hospitals, Homecare setting, Clinics and Others. On the basis of Distribution Channel Global Parkinsons disease Drug Market is classified into Retail Pharmacies, Online Pharmacies, Hospital Pharmacies and Drug Stores.

The regions covered in Global Parkinsons diseases Treatment Market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, Global Melanoma Drug Market sub divided in to U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Parkinsons disease treatment Market Reports

Global Parkinsons disease Treatment Market reports cover prominent players like Teva Pharmaceuticals, AstraZeneca, Novartis AG, Boehringer Ingelheim, GlaxoSmithKline Plc., Salix Pharmaceuticals, mpax Laboratories, Orion Corporation, Mylan N.V., Cipla Inc., Daiichi Sankyo, Apotex Inc, Impax Laboratories, Lundbeck, Sun Pharma, Wockhardt ,UCB, Valeant Pharmaceuticals Acadia, Abbvie, Zydus Cadilla, Strides, 1 A Pharma, Upsher-Smith, Intas, US World Meds, Dr. Reddys, Medtronic plc, Biotie Therapies Corp., Adamas Pharmaceuticals Inc., Akorn Inc., Astellas Pharma Inc., Desitin Arzneimittel GmbH, Endo International plc, Kyowa Hakko Kirin, Newron Pharmaceuticals and Merck.

Global Parkinsons disease treatment Market Dynamics

Global Parkinsons disease Treatment Market is mainly driven by growing prevalence of Parkinsons disease and government funding for research on Parkinsons disease. The increase in number of geriatric population and technological advancements such as combination therapies for prolong action of continuous dopaminergic stimulation drugs, gene therapy, neural transplantation are few other causes which are possible to boost the Parkinsons disease Treatment market. However some restraints like availability of alternative treatments is expected to hold back market and expiry of patents for several drugs such as Stalevo, Azilect, Rytary, Comtan etc. can cause the termination of Parkinsons disease Treatment market. High cost of treatment such as 14.4 Billion USD and lack of expertise for early diagnosis is likely to restrict the market growth.

Also lack of novel and successful therapies in the market to reduce the risk of mortality restraints for this market. The opportunities for Global Parkinsons disease Treatment market includes upcoming technologies like surface EMG (Electromyography) sensors, Pulse oximetry sensors, development in personalized medicine along with huge investment in anti-Parkinsons drugs research will boost the global Parkinsons disease Treatment market. Also development of novel drugs and combination therapy with less side effects and better survival rates is expected to increase the global Parkinsons disease Treatment market.

Global Parkinsons disease treatment Market Regional Analysis

Europe is likely to achieve the largest share of the global Parkinsons disease treatment market, which is expected to be followed by North America with a sizeable market share. High knowledge about the treatment, increase in expenditure for Research & Development, favorable medicinal reimbursement regulations and policies, the high occurrence of Parkinsons disease, the rapidly growing geriatric population and potential medical pipeline products are likely to contribute to the major share of Europe in Global Parkinsons disease treatment market. The global Parkinsons disease treatment market in the Asia Pacific is anticipated to develop at a profitable rate. The market share of the global Parkinsons disease treatment market is to be expected to grasp by China and Japan in the Asia Pacific, due to the large population of elderly people who are affected with Parkinsons disease.

Furthermore, increase in awareness of disease and government initiatives for improving health care facilities are expected to boost the regional market to a certain extent.

Key Benefits for Global Parkinsons disease treatment Market Reports

Global Parkinsons disease Treatment Market report covers in depth historical and forecast analysis.Global Parkinsons disease Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Global market sale (K Units), Global market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.Global Parkinsons disease Treatment Market report helps to identify opportunities in market place.Global Parkinsons disease Treatment Market report covers extensive analysis of emerging trends and competitive landscape.Global Parkinsons disease treatment Market Segmentation

Global Parkinsons disease Treatment Market: By Medication Analysis

Dopamine PrecursorPeripheral Decarboxylase InhibitorsCOMT (Catechol-O-methyltransferase) InhibitorsMAO(monoamine oxidase) InhibitorsOthers (anticholinergics, antihistaminics etc.)Global Parkinsons disease Treatment Market: By Medical Devices Analysis

Deep Brain Stimulation (DBS) DevicesCarbidopa/Levodopa Enteral Suspension (Duopa) delivery devicesOthersGlobal Parkinsons disease Treatment Market: By End User Analysis

HospitalsClinicsHomecare settingOthersGlobal Parkinsons disease Treatment Market: By Distribution Channel

Retail PharmaciesOnline PharmaciesHospital PharmaciesDrug StoreGlobal Parkinsons disease Treatment Market: By Regional & Country Analysis

North AmericaU.S.MexicoCanadaEuropeUKFranceGermanyItalyAsia PacificChinaJapanIndiaSoutheast AsiaLatin AmericaBrazilThe Middle East and AfricaGCCAfricaRest of Middle East and AfricaNeed a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=71&RequestType=Methodology

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Parkinsons disease treatment Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Parkinsons disease treatment Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Parkinsons disease treatment Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Parkinsons disease treatment Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Parkinsons disease treatment Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Parkinsons disease treatment Market.

Market Forecast: Here, the report offers a complete forecast of the Global Parkinsons disease treatment Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

About Us:

We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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Hofseth Biocare ASA: Two-year research collaboration contract signed with Stanford on the use of Salmon Protein Hydrolysate peptides for the treatment…

By Gene Therapy Clinics

Hofseth BioCare ASA (HBC) has signed a two-year research agreement with Stanford University School of Medicine, CA, USA to further investigate the gastro-intestinal (GI)-protective properties of HBCs Salmon Protein Hydrolysate Peptides (SPH) in children and adults with GI tract disorders. The research agreement follows recent findings of SPHs reduction of GI injury in both in vitro and in vivo pre-clinical models that reflect the underlying disease process of necrotizing enterocolitis (NEC) and ulcerative colitis / inflammatory bowel disease (IBD).

The focus of the research collaboration is to further elucidate how SPH reduces or prevents intestinal inflammation in preclinical models of NEC and IBD and thereby helps to restore GI barrier function. In the second year of the project, it is anticipated that this work may form the basis for proceeding to a pilot study to assess SPH as an adjunctive treatment for the maintenance of remission in human subjects with mild to moderate ulcerative colitis.

Prior research undertaken by HBC and Stanford has demonstrated that gene systems that protect the GI tract against oxidative stress and inflammation are upregulated in human GI cells exposed to SPH. In proprietary animal models of GI inflammation, Stanfords work has shown SPH to markedly reduce gut inflammation. Oxidative stress is implicated as an important underlying driver of many digestive diseases, including NEC and IBD.

IBD is a chronic disease that can affect both physical health and quality of life (QoL). Symptoms include crampy abdominal pain, diarrhoea and weight loss. Ongoing blood loss from the bowel can result in anaemia further exacerbating the impact on QoL with anaemia symptoms including fatigue and reduced exercise capacity. Long term effects of bowel inflammation include an increased risk of colon cancer. There are more than 70,000 new cases of IBD diagnosed each year in the US alone. Steroids and biologic therapies are commonly used treatments. Whilst these can provide significant benefit and are generally well tolerated, they are associated with side effects such as osteoporosis and diabetes with long term steroid use and serious infection with biologic therapy. Furthermore, a significant number of patients do not attain full remission and continue to suffer from the effects of IBD. There is therefore a clear need for further, well tolerated treatments which can help to attain and maintain remission for these patients.

For further information, please contact:James Berger, Head of Investor Relations & Strategy of Hofseth BioCare ASAPhone: +41 79 950 1034E-mail: jb@hofsethbiocare.no

Dr. Bomi Framroze, Chief Science Officer at Hofseth BioCare ASAPhone: +1 650 283 1196E-mail: bf@hofsethbiocare.no

About Hofseth BioCare ASA:HBC is a Norwegian biotech company that develops high-value ingredients and finished products. Ingredients are further developed into discovery and pre-clinical studies in multiple clinics and university research labs in several countries. Lead clinical and pre-clinical candidates are development towards a Gastro-Intestinal (GI) Protective Medical Food, Medical Food to help treat age-related Sarcopenia, and future phase 1 studies on treatment of Anemia and NEC-IBS with Salmon Protein Hydrolysate fractions.The company is founded on the core values of sustainability, traceability and optimal utilization of natural resources. Through an innovative hydrolysis technology, HBC can preserve the quality of lipids, proteins and calcium from fresh salmon off-cuts. Hofseth BioCare's headquarters are in lesund, Norway with branches in Oslo, London, Zrich, Chicago, Mumbai, Menlo Park and Tokyo.HBC is listed on Oslo Stock Exchange Axess list with ticker "HBC". More information about Hofseth BioCare at hofsethbiocare.com and facebook.com/hofsethbiocare

This information is subject to the disclosure requirements pursuant to Section 5-12 the Norwegian Securities Trading Act

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Hofseth Biocare ASA: Two-year research collaboration contract signed with Stanford on the use of Salmon Protein Hydrolysate peptides for the treatment...

Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the…

By Stem Cell Clinic

CAMBRIDGE, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo,is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. Intellia researchers are presenting new data in support of NTLA-5001, the companys engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellias approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year, said Intellia President and CEO John Leonard, M.D. We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellias second systemic therapy employing our in vivo knockout approach and modular delivery platform.

Data Presentations on Intellias First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellias first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Boninis team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patients leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults(Source:NIH SEER Cancer Stat Facts: Leukemia AML).

Intellias proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellias process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellias novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patients healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellias unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from todays presentation include the following:

Intellias cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering, will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellias website. These data were a follow-on to the study presented at Keystone Symposias Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellias Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellias TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellias engineered TCR T cell approach.

The presentation titled, Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia, will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT meeting organizers selected this presentation as one of six to receive the ASGCT Excellence in Research Award this year.

Continued Progress on Intellias Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the companys systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates(Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema, will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellias website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: planned submission of an investigational new drug (IND) application or similar clinical trial application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020 and its planned dosing of first patients in the second half of 2020; plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; plans to submit an IND or similar clinical trial application for its hereditary angioedema (HAE) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program and HAE programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML, and HAE programs, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron Pharmaceuticals, Inc., and Regenerons ability to enter into a co-development and co-promotion agreement for the HAE program; statements regarding the timing of regulatory filings regarding its development programs.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Novartis or Regeneron or its other ex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917 lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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