WILMINGTON, Del.--(BUSINESS WIRE)--Incyte Corporation (Nasdaq:INCY) today announced positive results from the Novartis-sponsored pivotal Phase 3 REACH2 study evaluating ruxolitinib (Jakafi) in patients with steroid-refractory acute graft-versus-host disease (GVHD). The study met its primary endpoint of improving overall response rate (ORR) at Day 28 with ruxolitinib treatment compared to best available therapy. No new safety signals were observed, and the ruxolitinib safety profile in REACH2 was consistent with that seen in previously reported studies in steroid-refractory acute GVHD.

Further analysis of the safety and efficacy data is ongoing. Novartis expects to initiate discussions with ex-U.S. regulatory authorities in 2020, and to submit REACH2 results for presentation at an upcoming scientific meeting.

GVHD is a challenging and serious disease, and physicians around the world need access to therapies that can improve outcomes for patients, said Peter Langmuir, M.D., Group Vice President, Targeted Therapies, Incyte. This positive result of the REACH2 study is excellent news for patients as it further reinforces the potential of ruxolitinib as a treatment option that can provide meaningful results for patients with steroid-refractory acute GVHD.

GVHD is a condition that can occur after an allogeneic transplant (the transfer of stem cells from a donor) where the donated cells initiate an immune response and attack the transplant recipients organs, leading to significant morbidity and mortality. There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.

Earlier this year, Jakafi was approved by the U.S. Food and Drug Administration (FDA) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older based on results of the REACH1 trial. Jakafi is marketed by Incyte in the U.S.; ruxolitinib (Jakavi) is licensed to Novartis ex-U.S.

In addition, the pivotal REACH3 trial evaluating ruxolitinib in patients with steroid-refractory chronic GVHD is ongoing. A recent interim efficacy and safety analysis conducted by an Independent Data Monitoring Committee has recommended that REACH3, which is co-sponsored by Incyte and Novartis, should continue without modification. The results of the REACH3 trial are expected to be available in 2020.

About REACH2

REACH2 (NCT02913261) is a randomized, open-label, multicenter Phase 3 study sponsored by Novartis, evaluating safety and efficacy of ruxolitinib compared with best available therapy in patients with steroid-refractory acute GVHD.

The primary endpoint was overall response rate (ORR) at Day 28, defined as the proportion of patients demonstrating a best overall response (complete response or partial response). Secondary endpoints include durable ORR at Day 56, ORR at Day 14, duration of response, overall survival and event-free survival, among others. For more information about the study, please visit https://clinicaltrials.gov/ct2/show/NCT02913261.

About REACH

The REACH clinical trial program is evaluating Jakafi in patients with steroid-refractory GVHD and includes the collaborative Novartis-sponsored randomized pivotal Phase 3 trials: REACH2 and REACH3. The ongoing REACH3 trial is evaluating patients with steroid-refractory chronic GVHD with results expected next year. For more information about the REACH3 study, please visit https://clinicaltrials.gov/ct2/show/NCT03112603.

The REACH program was initiated with the Incyte-sponsored REACH1 trial, a prospective, open-label, single-cohort, multicenter, pivotal Phase 2 trial (NCT02953678) evaluating Jakafi in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. For more information about the study, including trial results, please visit https://clinicaltrials.gov/show/NCT02953678.

About Jakafi (ruxolitinib)

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is also indicated for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea as well as adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

Important Safety Information

Jakafi can cause serious side effects, including:

Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.

Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.

Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.

Increases in cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.

The most common side effects of Jakafi include: for certain types of MF and PV - low platelet count, low red blood cell count, bruising, dizziness, and headache; and for acute GVHD low red blood cell counts, low platelet counts, low white blood cell counts, infections and fluid retention.

These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, have a high level of fat in your blood (high blood cholesterol or triglycerides), had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider.

Women should not take Jakafi while pregnant or planning to become pregnant. Do not breast-feed during treatment with Jakafi and for 2 weeks after the final dose.

Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at http://www.jakafi.com.

About Incyte

Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Companys website at http://www.incyte.com.

Follow @Incyte on Twitter at https://twitter.com/Incyte.

Forward Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether and when the REACH2 data will be presented, when results from the REACH3 study will be available, and the effect of the REACH2 results on patients with GVHD, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA; the Companys dependence on its relationships with its collaboration partners; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission, including its Form 10-Q for the quarter ended June 30, 2019. The Company disclaims any intent or obligation to update these forward-looking statements.

Original post:
Incyte Announces that the REACH2 Pivotal Trial of Ruxolitinib (Jakafi) Meets Primary Endpoint in Patients with Steroid-Refractory Acute...

Recommendation and review posted by Bethany Smith

WHEN teacher Matt Meads started suffering with stomach pains, night sweats and tiredness - he quickly dismissed it as end of school year fatigue.

He was fit and healthy, and avoided drinking and smoking.

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But just three weeks after first falling ill, Matt died - aged just 33 - and after just three chemo sessions.

He and his wife Abi were devastated when they discovered his symptoms were actually a sign ofleukaemia.

Heartbroken Abi, 27, from Nottingham, is now sharing his story to raise awareness and to urge others to be vigilant for signs of acute lymphoblastic leukaemia, a rare and aggressive form of blood cancer.

"I've got so many people around me offering help and support but I still feel really lonely because I've lost my best mate, my husband, my soulmate," Abi said.

It comes as blood cancer charity Bloodwise warns thousands of people are dying of the disease because it's diagnosed too late.

Matt and Abi first suspected something was wrong on July 6, when he began feeling more and more fatigued.

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And when he started vomiting to the point of being unable to keep ice cubes down, Abi urged him to go to the doctor.

At first he was told by his GP he had gastroenteritis or constipation but he soon returned to the hospital for a second time where they did a blood test.

Abi, who works as a teacher too, said: "Obviously I wish it was spotted sooner.

"I don't feel any anger towards to the hospital.

"I genuinely believe they did everything they could for him.

"He would say he was feeling sick."He would send a text saying he wasn't feeling well so was going to bed. I was out with some friends.

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"He said something about feeling hot as well, but we didn't put that down to anything because it was the middle of summer and everyone was feeling hot.

"He was sleeping a lot, particularly at weekends which he would spend mostly asleep.

"But we're both teachers, it was coming up to the end of the school year and we had both got a lot of work on.

"We put it down to the job, and just tried to keep going because we had five weeks off soon.

"We thought it was the usual fatigue that we feel at the end of the year.

"There were sickness bugs going around at both of our schools so it wasn't anything out of the ordinary.

"He was referred to A&E for the second time thinking it was gallstones.

I've got so many people around me offering help and support but I still feel really lonely because I've lost my best mate, my husband, my soulmate

"They did some blood tests on him, sent him for a CT scan. The doctor came back and basically said that it was leukaemia."

Despite the devastating diagnosis, Abi said her brave husband remained positive as he came to terms with what was really happening.

She added: "Matt was a really positive person and was always somebody who believed what would be would be, it is what it is and all that stuff.

"So when the doctor told him he was quite composed.

"He didn't really give anything away about what he was feeling.

"It was obviously a massive shock for him but he didn't really respond in the way I would have done. He was listening to the doctor.

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"The doctor actually stopped at one point because he was explaining what would happen next.

"He actually stopped at one point to ask him if he was okay, it was really big news and is he taking it all in?

"Matt's response was, 'yes, but there's nothing I can do about it. It is what it is'.

"He was definitely really brave."

And Abi says the diagnosis came as even more of a shock given how healthy he was.

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She was forced to watch her husband go from happy and sporty to intensive care within days, before he passed away from a pulmonary embolism after just three chemotherapy sessions.

She said: "We knew he was poorly, but maybe not quite how poorly he was.

"I certainly wasn't expecting a phone call from the hospital.

"I don't know how I am now. It's hard. I don't think I really started to grieve until after the funeral.

"It's hard to think ahead for anything. It's a case of taking everything one day at a time.

"Some days are better than others. Some days are horrific and I don't want to get out of bed.

What is Acute Lymphoblastic Leukaemia?

Acute lymphoblastic leukaemia (ALL) is a type of blood cancer that starts from young white blood cells called lymphocytes in the bone marrow.

Adults and children can get it but it is most often diagnosed in younger people.

It'svery rare, with around 650 people diagnosed with the condition each year in the UK.

Many symptoms of ALL are vague and non specific. It may feel like the flu as symptoms are caused by too many abnormal white blood cells and not enough normal white cells, red cells and platelets.

Symptoms can include:

Recently blood cancer charity Bloodwise warned thousands of patients in England could be unnecessarily dying from blood cancer because they are diagnosed too late.

Experts analysed NHSdata and found 28 per cent of patients are told they have the disease after needing emergency treatment for their symptoms.

Figures show there are around 40,000 cases of blood cancer - a group of diseases including leukaemia, lymphoma and myeloma - each year in the UK.

Around 77 per cent of patients will survive for three years if they are diagnosed after visiting their GP, Bloodwise says.

In contrast, the same survival rate plummets to just 40 per cent for patients who are diagnosed as an emergency.

This is because symptoms develop over a few weeks and become more severe as the number of immature white blood cells increases.

"Matt was a really happy person. He was really positive.

"He was kind, caring, loving, wicked sense of humour. He would make a joke about anything and was very quick-witted.

"As a teacher he would have done anything for his students. He would have done anything for his family.

"He was just a really positive person who would have done anything for anybody.

"He loved his sport. He would go to the gym, he loved being outside and walking. He liked cycling. He was careful about what he ate.

"Everything the doctors warn you about, he didn't do. He didn't drink, he didn't smoke, he had a good diet, he exercised.

If you've got any of the symptoms which are lasting or you can't explain why you've got them, you need to go to the doctor and get checked out and be persistent in asking for a blood test

"He always put sun-cream on because he was paranoid he might catch skin cancer or something.

"He did everything he could to try and prevent anything from happening to him.

"As the doctors said there was nothing he could have done to prevent this."

Abi is now speaking out to urge others to get checked out and insist for a blood test if they have persistent symptoms of blood cancer.

She said: "If you've got any of the symptoms which are lasting or you can't explain why you've got them, you need to go to the doctor and get checked out and be persistent in asking for a blood test.

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"You know your own body. It's as simple as having a blood test.

"If you've got it for days and it's not getting any better, if you're in any doubt, get it checked.

"We didn't know what the symptoms were.

"The only one I knew was bruising, but Matt didn't have any bruises until he was in hospital. So the one thing I knew wasn't relevant.

"I didn't realise about the night sweats, fatigue or heavy breathing.

"We never expected it would be that.

"We had thought worst case scenario it was gallstones or an impacted bowel, so when he came and said leukaemia it was just unexpected.

"When you're poorly you have all these possibilities going through your head but you never think it's going to be that.

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"I had a really good chat with Matt's consultant where I questioned whether I should have done more, if I had spotted things sooner, whether if I had been more pushy in getting him to hospital.

"But the symptoms are vague and it can come on within days. It doesn't have to be something that has been happening for weeks or months."

You can donate on Abi's fundraising page in memory of Matt here.

Visit link:
Fit and healthy teacher dies 20 days after discovering he had leukaemia at 33 - The Sun

Recommendation and review posted by Bethany Smith

The Duchess of York, affectionately known by British royal fans as Fergie, shared the cover of The Perfect World Magazine, where she can be seen giving Sir David Attenborough an honorary award. Fergie, who turns 60 today, revealed in an Instagram post that moment is one of the best of her life.

She wrote on Instagram: "Thank you all for the birthday messages! This is one of the highlights of my life."

In the cover, Fergie can be seen smiling as she hands the award, shaped like a rhino, toSir Attenborough, the world-famousEnglish broadcaster and natural historian.

Fergie andSir Attenborough attended together last September thePerfect World Foundation gala dinner, where she will present him the award.

The same award has been previously given toMark Shand, the late brother of the Duchess of Cornwall, and primatologist Dr Jane Goodall.

In the early afternoon, the Duchess of York published on Instagram another picture, showing her wearing a colourful sari.

She added in the caption: "Colourful moments"

Fergie's Instagram messages come after her former husband Prince Andrew celebrated her birthday on social media.

Earlier this morning, the Duke of Yorkpublished two identical messages dedicated to Fergie on both Instagram and Twitter.

READ MORE:How Fergie was 'forced to cancel birthday party due to crisis'

The messages read: "Wishing Sarah Ferguson a very Happy 60th Birthday!"

On Instagram, the message was accompanied by three pictures, one depicting Sarah surrounded by members of the York family, including Princess Eugenie's husband Jack Brooksbank and Princess Beatrice's fiancee Edoardo Mapelli Mozzi.

The second picture shows Fergie surrounded by children supported by the charity she is a patron of, Street Child UK.

And the third snap depicts Eugenie, Beatrice and Fergie together while carrying out an engagement.

DON'T MISS

Fergie has previously spoke with enthusiasm about her approaching 60th birthday.

Speaking at a charity event held onbehalf of Street Child and hosted by Hello, the Duchess said:"I think it's really important that my life is beginning at 60, I'm so excited by it."

Outspoken Fergie also revealed she has undertaken laser treatment to her skin to look her best for her milestone birthday.

Similarly, the Duchess had laser also before the wedding of her youngest daughter Princess Eugenie, which helped to make her skin glow.

But these haven't been the only beauty treatments Fergie has undertaken in the past years.

In March this year, Fergie travelled to the Bahamas toundergo stem cell therapy - an alternative to surgery which is yet to be proven fully safe and effective on humans and is therefore not widely available in the UK.

This therapy helped her solving a painful problem related to her feet, which eventually forced her to stop wearing high heels.

I went to the Bahamas in March to have the treatment.

I think my toes were ruined by all the riding I did when I was young.

Visit link:
Sarah Ferguson reveals highlight of my life as she celebrates 60th birthday - Express

Recommendation and review posted by Bethany Smith

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Anti-Aging Drugs Market Innovations, And Top Companies - Forecast To 2028| Allergan, Johnson and Johnson, Nu Skin - Healthcare News

Recommendation and review posted by Bethany Smith

LESS than a week away from her 60th birthday, Sarah Ferguson has revealed that she has had Botox, two types of facelift and organic fillers to wind back the clock.

Many royal fans marvelled when the Duchess of York stepped out looking surprisingly youthful for her daughter Princess Eugenies high profile royal wedding last year.

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Addressing rumours of cosmetic surgery, Fergie has confessed that her looks arent entirely natural and she has had professional tweaks.

Speaking to the Mail, she said: The happiness was shining out of me because my daughter was getting married. I was so glad. I love Jack. When Im passionate about anything, my eyes shine.

Above all, it was being joyful for Eugenie that made me look good. But Id had some laser treatment on my face which helped, too.

Although she previously had Botox to tackle facial lines, Sarah admitted she now prefers having laser cosmetic procedures, which are pain-free, non-invasive and take just 90 minutes.

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Mum-of-two Sarah goes to Harley Streets Dr Gabriela Mercik, who is the creator of the worlds first 6-Dimension Ultimate Laser Treatment facelift, which Fergie has now tried.

Sarah first met Dr Gabriela in 1992 when the royal was visiting young cancer victims in Upper Silesia, Poland.

At the time, Dr Gabriela was a medical student helping to treat the patients, and she ended up staying in touch with loyal Sarah.

Fergie first became one of her customers after Gabriele opened her own aesthetic clinic in the UK, and the royal felt her fair skin was too exposed to sunshine as a child.

Sarah said: I live on jingly-jangly nerves. Im very focused, earnestly intense, but I live at high speed. I dont sit down for long. Im always saying to Dr Gabriela, Come on, come on! Hurry up! Im the worst patient.

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She opts for the rejuvenating ultimate facelift, which can be done in a lunch hour and involves boosting the skins production of collagen.

Fergie admitted she had Botox a long time ago when alternative treatments werent on the market, but she now prefers having the facelifts.

The royal said shes not a fan of the frozen look as she is so animated, and also hates needles.

Despite her fears of the surgical tool, she opted for a mesotherapy beauty treatment at the clinic in 2013, which is when vitamins, minerals and amino acids are injected under the faces mesodermal layer to plump skin.

Now she has moved on to organic fillers, which are non-invasive injectable used to tackle lines and wrinkles.

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Five years ago, Dr Gabriela suggested Sarah had a thread lift also known as a puppet lift which is when threads are inserted in to the skin to make a mesh which pulls the face upwards.

Although results last for two years, the threads dissolve over six to eight months.

Sarah said: Before I had it done I thought, Oh this is going to be painful, but it wasnt bad. My skin responded well. I think if you look at photos of me after I had it done, I look much better.

She now hopes the 3,750 6-Dimension Ultimate Laser Treatment facelift will help her look her best for her 60th birthday milestone next Tuesday.

The doctor confirmed that Sarah does pay for treatments at her discretion as the pair have become close friends.

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Sarah admitted that the drive to have facial cosmetic surgery comes from skin damage that started from a young age, when her mum incorrectly thought Nivea moisturiser could be used as sunscreen.

The royal was also motivated to take care of her skin after her dad, Major Ronald Ferguson, passed away from skin cancer in 2003, and her best friend Carolyn Cotterell also died of a malignant melanoma when she was 43.

She now admits any tan she has is from a bottle and she hopes to rebuild her collagen by her birthday.

My skin responded well. I think if you look at photos of me after I had it done, I look much better.

In March she revealed she flew to the Bahamas to have a regenerative stem cell therapy on her feet, after feeling her toes were ruined by frequent horse riding when she was young.

Doctors shaved a spot near her big toe and implanted 20 million stem cells from her midriff into the cavity to make new cartilage.

Due to the surgery not yet being proven to be safe, the UK does not widely offer the treatment.

She may be turning 60, but Fergie insisted in July this year, at the launch of a British Heart Foundation campaign, that she feels sexy, sassy and super saucy.

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The proud mum has recently shared how excited she is for her eldest daughter, Princess Beatrice, to get married next year.

Taking to Instagram last month, Fergie shared six photos of the 31-year-old princess with her millionaire husband-to-be Edoardo Mapelli Mozzi.

She later shared a tweet, writing: "I know what a mother feels so I have tears of joy. I am so proud of this sensational news.

Andrew and I are just the luckiest people ever to have two great sons in law."

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Sarah recently confirmed that the "only place" Beatrice can get married is Britain.

Meanwhile its been claimed Beatrice is ready to start a family with multi-millionaire property tycoon Edoardo.

And Fergie recently honoured the family of a teen who died from severe allergy reaction after eating Pret a Manger sandwich.

See the original post:
Sarah Ferguson admits shes had Botox, TWO types of facelift and vitamin injections in a bid to stay youthful - The Sun

Recommendation and review posted by Bethany Smith

The members of the British royal family are always in the public eye. They cant blow their noses without people hearing about it. But with so much attention, comes even more scrutiny.

The royals are under pressure to be perfect at all times. Every move they make is photographed by the paparazzi and the public isunforgiving. Even after they gave birth, people expected both Meghan Markle and Kate Middleton to be back to their normal thin frames in a matter of days.

When celebrities are feeling the pressure to keep up appearances, many of them seek out plastic surgery procedures. Does the royal family do the same?

For many people, the Duchess of Cambridge is the epitome of style and grace. Youd be hardpressed to find a photo of Middleton looking less than perfect. Because of this, people have often wondered if the future Queen Consort has had any work done.

Earlier this year, Dr. Munir Somji of Dr. Medi Spa Clinic in London claimed that Middleton was one of his clients.

He shared before and after pictures of the Duchess on his Instagram.

Our Kate loves a bit of baby Botox, he wrote under the photos, which have since been deleted.

View this post on Instagram

The Duchess of Cambridge, Patron of the @Natural_History_Museum, visited the Angela Marmont Centre for UK Biodiversity to hear how it is championing and helping to protect UK wildlife. The Angela Marmont Centre (AMC) is a unique scientific hub located in the Natural History Museum, and is dedicated to the study of the UKs natural world. The AMC helps public visitors identify their finds, provides vital training in wildlife identification, and leads research and citizen science projects that are helping to transform our understanding of over 80,000 species of wildlife known to exist in the UK today. With populations of many UK species declining, and a significant proportion threatened with local extinction, the AMC brings together a host of individuals and organisations all dedicated to building understanding of the UKs wildlife, so that it can be better protected for future generations. AMC staff work with colleagues from across the Natural History Museum to carry out a range of scientific research to study how and why the UKs wildlife is changing. This ranges from applying ground-breaking scientific techniques, including analysis of environmental DNA through to detecting and identify the miniscule insects and microorganisms that play an important role in healthy environments. At the AMC today The Duchess viewed some of the Museums specimens and saw DNA sequencing live in action.

A post shared by Kensington Palace (@kensingtonroyal) on Oct 9, 2019 at 5:20am PDT

Note the reduction of fine lines on the forehead, he continued. But also note the depression of the medial (middle part) brow but elevation of the lateral tail of the brow.

So many people started believing the rumor that Kensington Palace gave a statement to the New York Post.

A spokesperson for the palace said that the post by Dr. Somji was categorically not true and in addition, The Royal Family never endorse commercial activity.

One royal who has been open about the procedures shes had done is the Duchess of York, Sarah Ferguson.

Ive had a lot of help to look like this at 60! she said in an interview withDaily Mail.

Ive started the laser treatment, but its not finished yet, she continued. The collagen needs to rebuild. I hope it will all be done by my birthday.

She also had this treatment before her daughter, Princess Eugenies, wedding last year. The procedure was done by Dr. Gabriela Mercik in a facility in London.

I dont like the frozen look, Ferguson said of why she chooses more natural procedures. Im so animated and I like to be myself. I dont like the thought of needles and am very glad if I look well and happy Im really happy to be open about what Ive had done.

Originally, the Duchess tried botox but has since switched to less invasive procedures, like her most recent treatment which refines the skins texture.

Shes also had mesotherapy, which is a non-surgical cosmetic treatment.

I need to repair the damage that was done on the beach when I was a child, she continued. Its why I had the mesotherapy, the vitamin cocktail to hydrate and boost the skin.

Not only does Ferguson get work done on her face, but she makes sure her feet are perfect as well!

I think my toes were ruined by all the riding I did when I was young, she said. They shaved the bone here. And implanted stem cells, 20 million of them taken from my midriff, into my feet to make new cartilage. It takes about six months to heal but now I can walk in heels!

Read more:
Which Plastic Surgery Procedures Have Members of the Royal Family Had Done? - Showbiz Cheat Sheet

Recommendation and review posted by Bethany Smith

CRISPR Therapeutics to receive non-exclusive access to certain KSQ IP for its allogeneic CAR-T programs

KSQ Therapeutics to receive non-exclusive access to certain CRISPR IP for its autologous cell therapies, including its existing eTILTM cell franchise

ZUG, Switzerland & CAMBRIDGE, Mass.(BUSINESS WIRE)CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development, today announced a license agreement whereby CRISPR Therapeutics will gain access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ will gain access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The financial terms of the agreement are not being disclosed.

We are thrilled to gain access to CRISPR Therapeutics foundational IP estate through this agreement, said David Meeker, M.D., Chief Executive Officer at KSQ Therapeutics. Our eTILTM programs involve editing gene targets in human TILs that were discovered at KSQ by applying our proprietary CRISPRomics approach to immune cells in multiple in vivo models. This agreement clears an important path for us to be able to bring these programs through development and commercialization, leveraging CRISPR Therapeutics proprietary editing technology.

The gene targets within the scope of the license agreement were identified using KSQs proprietary CRISPRomics drug discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery. These specific targets were uncovered in screens to identify genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology.

KSQ has built an industry-leading platform to screen for novel gene targets using its technology, and has identified a group of targets that could help unlock the full potential of adoptive cell therapy in oncology, said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. As a result of this license agreement, CRISPR Therapeutics will have the opportunity to bring these novel targets into our leading allogeneic CAR-T development platform to further strengthen our future programs in this important therapeutic area.

About KSQ Therapeutics

KSQ Therapeutics is using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics drug discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SECs website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies' Respective Cell Therapy Programs in Oncology - SynBioBeta

Recommendation and review posted by Bethany Smith

Scientists have successfully grown tomatoes, leeks, potatoes and several other crops in simulated Lunar and Martian soil, meaning that humans could survive off a closed-loop agriculture ecosystem on Mars. They also emphasized that cellular agriculture and insect farming would be instrumental to feeding a future Martian population.Read more at Modern Farmer

CRISPR technology is a precise form of gene-editing that genetically modifies foods in a faster and cheaper way than ever beforeand its been subtly present in the dairy aisle for quite some time. But while this tool could transform agriculture as we know it, there is much scientists still dont know about the ramifications of using it on the environment and human health.Read more at The Atlantic

Emerging data shows that the boom in consumer desire for convenient, ready-to-cook frozen fare is sweeping the frozen fish and seafood section. Seafood marketers and producers should pay special attention to childless, single adults over 35 and senior couplestheyre driving the dollar growth in this category.Read more at Nielsen

In a statement published yesterday the Federal Drug Administration solidified its position on the use of cannabidiol (CBD) by pregnant women. While there is no comprehensive research on the effects of the popular ingredient on fetuses, the administration argues that the potential for contamination in CBD products is too high to ignore and cites a study wherein high doses of CBD in pregnant test animals adversely affected developing male fetuses. Read more at FDA.gov

Small farmers that use responsible and regenerative growing practices are few and far between now that industrialized, multinational corporations have a stronghold on the U.S. food system. Just 43% of farms are profitable according to USDA, and federal policies are in place that work to uphold the soil-depleting practices of larger corporations. Whats clear is that the government immediately must act to improve crop subsidies, increase biodiversity, promote soil health, use integrated pest management practices and manage energy use via regenerative actions to prevent an agricultural crisis.Read more at Quartz

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5@5: Crops on Mars? | The future of CRISPR - New Hope Network

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

Intellias data includes important updates about the companys programs and platform development activities:

Oral Presentation:

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellias gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellias similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.Abstract number: OR48Session 5b: New delivery systems and technologiesPresentation date/time: Friday, October 25, 2019, 11:30 a.m. 1:30 p.m. CETLocation: Room 113-115

Poster Presentations:

In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics

This poster presentation will highlight Intellias approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel OConnell, Ph.D.Poster ID Number: P655Date: Wednesday, October 23, 2019

Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia

This poster presentation focuses on Intellias ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San RaffaelePoster ID Number: P111Date: Wednesday, October 23, 2019

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout

This poster presentation focuses on the companys T cell engineering technology, which is being applied in its Wilms Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.Poster ID Number: P162Date: Thursday, October 24, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.Poster ID Number: P552Date: Thursday, October 24, 2019

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing 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 ofIntellia 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 IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

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 our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our 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 will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations 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 theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

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

Excerpt from:
Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - BioSpace

Recommendation and review posted by Bethany Smith

As word spread in 2018 about the birth of the world's first genetically altered babies, concerns over their future health mounted, with one study even raising the tragic possibility of shortened lives for the newborns. That risk now seems far less likely.

The alarming paper published in Nature last June has now been retracted by the authors themselves, who in the wake of criticism admit the way they searched for signs of a mutated gene in a data sample left too much room for doubt.

It's an important lesson not only in how science values self-correction, but how researchers need to tread lightly as they trawl through population-sized databanks in search of new discoveries.

"I feel I have a responsibility to put the record straight for the public," University of California population geneticist, Rasmus Nielsen, told Ewen Callaway at Nature.

The gene at the centre of the research serves as a template for a receptor on white blood cells.

Called CCR5, its usual job is to detect chemical signals used in immune responses. Unfortunately the deadly human immunodeficiency virus (HIV) evolved to use it as a window to gain entry into the cells.

Ever since the receptor's role in HIV infection was discovered, researchers have wondered just how important this receptor really is. Would we really miss it if it was gone?

Luckily an answer might be found among a percentage of people of European descent with a naturally occurring 'broken' version of CCR5 called delta-32. Those who carry a single copy of the delta-32 variant seem to be less susceptible to HIV than the rest of the population.

In November 2018 a Chinese geneticist named He Jiankui claimed to have used the engineering technology CRISPR-Cas 9 to alter the CCR5 genes in human embryos to artificially give them resistance.

He Jiankui's initial announcement suggested at least one of the twins was carrying two altered CCR5 genes. While they don't appear to match the delta-32 variants, it was enough to invite speculation over what kind of lives the children might have.

HIV resistance is no doubt a good thing in a world where the disease it causes is still destroying too many lives. But those benefits to any one individual might not be so great if a low quality CCR5 receptor raises the risks of developing other health problems.

Nielsen and his colleagues intended to answer this question by looking for similarly altered versions of the CCR5 gene in the UK Biobank's giant genetics database.

They estimated about 1 percent of the records in the database came from individuals with two delta-32 variant copies of the gene. Importantly, they calculated that this tiny fraction was 21 percent more likely to die before their 76th birthday, compared with those at least one 'normal' copy of the gene.

Thankfully, as happens in science, big claims often attract sceptical inquiries. Others quickly dived into the statistics in search of similar correlations using both the UK Biobank and other nation's datasets, coming up empty handed.

So where did Nielsen and his colleagues go wrong?

The cause of the discrepancy could lie in how the data was collected in the first place.

One way to work out whether a person has a specific gene is to simply use a template that sticks to a target sequence. These probes don't always work perfectly, meaning some people will incorrectly appear as negative in the database.

By potentially undercounting the number of people with the CCR5 delta-32 receptor, Nielsen risked masking the true impact of the mutation, making it look like there is a difference in mortality statistics. Which is why he asked for the paper to be retracted.

For researchers, huge banks of genetic and medical data collected from across a population provide the necessary quantities of information needed to spot subtle patterns that demarcate healthy from unhealthy bodies.

Yet as potentially useful as those statistics are, there's dangers in forgetting they come with plenty of assumptions.

You can see the now-retracted paper here.

Read more:
Researchers Admit They Were Wrong to Predict Early Death of The Famed CRISPR Babies - ScienceAlert

Recommendation and review posted by Bethany Smith

Eli Lilly is closing down its U.K.-based Erl Wood neuroscience center in Surrey, leading to cuts and relocations.

According to a local report from the Basingstoke Gazette, and confimed to FierceBiotech, the research house will be closed by the end of 2020, seeing around 80 redundancies from the neuroscience unit.

In total, around 270 employees will be hit, with two-thirds of staff moving over to a new location within the local area while neuroscience research will move over to its home in the U.S., according to the company, which spoke to the Gazette.

Industry Insight Survey: Direct-to-Patient Distribution of Clinical Supplies

This industry survey seeks to gain insight on trial sponsors' perspective on offering a DTP option and their current level of awareness and understanding of any factors that may influence their ability to do so. The first 50 qualified respondents will receive a $5 Amazon gift card.

The company added that this comes as part of a global review of research operations and off the back of years of on and off cuts from the Big Pharma.

Lilly told FierceBiotech in a statement that over time, Lilly has made a number of strategic choices to consolidate research operations around the world into global hubs. Overall, this has resulted in there being fewer than 45 neuroscience researchers, supported by approximately 35 chemistry researchers, working at the companys UK research site today.

The company added: Today, Lilly is announcing a proposal to consolidate laboratory-based UK research efforts to other global centres to further enable transformation which aims to deliver medicines from the lab to patients faster. This would include: Relocating neuroscience research to Lillys expanding site in Cambridge, Massachusetts, where Lilly maintains a hub specialising in the application of novel technologies including siRNA and gene therapy; Relocating a small number of roles to Lillys research centre in Indianapolis; Rebalancing and consolidating chemistry capabilities around the world."

Lilly said it would retain a small number of bioinformatics and neuroscience roles in the UK to focus on expanding external R&D collaboration with the UK and EU science community, and continue to undertake research collaborations and partnership activity in the U.K. with academia, the NHS and biotechs.

Under the plans, non-laboratory based employees would move to a new site within the local area. Lilly will now launch a formal consultation with employees and will be focused on ensuringevery employee is treated with dignity and respect throughout this period of change.

There are no changes being announced to Lillys commercial infrastructure, based in Basingstoke, and the company will retain core clinical and regulatory functions in the U.K.

It also pointed out that this was not down to Brexit.

Tim Garnett, chief medical officer at Lilly, said: I know that this proposal will be difficult news for many working for Lilly in the UK. Our dedicated teams have delivered world-class research from Erl Wood for the last 50 years. They are some of the most talented people in the industry and this proposal is not a reflection on their hard work or skill.

He added: Our focus now is on supporting our people and treating them with the dignity and respect they deserve. We will now enter a formal consultation with all the employees potentially affected.

Continue reading here:
Eli Lilly to shutter neuroscience R&D center next year - FierceBiotech

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass. and RESEARCH TRIANGLE PARK, N.C., Oct. 15, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, and Asklepios BioPharmaceutical, Inc.(AskBio), a fully integrated adeno-associated virus (AAV) gene therapy company, today announced the companies have entered a strategic research collaboration to explore in vivo delivery of genome editing medicines to treat neurological diseases. This collaboration brings together AskBios leading capsid development, clinical stage AAV vector delivery system, and manufacturing expertise with Editas Medicines leading genome editing technologies to potentially develop novel medicines for patients with high unmet need.

We are excited to collaborate with AskBio, a team with unparalleled experience in AAV technology and clinical-stage manufacturing. We believe that together we can deliver innovative genome editing medicines to the nervous system and rapidly advance medicines to treat neurological diseases and help patients in great need, said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.

The team at Editas Medicine has one of the most innovative technology platforms dedicated to finding solutions to severe diseases where there are few or no treatment options a mission consistent with AskBios, said Jude Samulski, Ph.D., Co-Founder, AskBio. With this shared goal in mind, we will combine our technologies to create an innovative approach to treating neurological diseases.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical stage gene therapy platform company dedicated to improving the lives of children and adults with rare genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. The company has generated hundreds of proprietary third generation gene vectors, several of which have entered clinical testing. AskBio maintains a portfolio of clinical programs across a range of indications, including Pompe, Limb Girdle Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy, Huntingtons, Hemophilia (Chatham Therapeutic/Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information, visit http://www.askbio.com.

Editas Medicine Forward-Looking Statements This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Editas Medicine may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Editas Medicines product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for Editas Medicines foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in Editas Medicines most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that Editas Medicine may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Editas Medicine expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

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Editas Medicine and AskBio Enter Strategic Research Collaboration to Explore In Vivo Delivery of Genome Editing Medicines to Treat Neurological...

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced upcoming presentations at the European Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, held in Barcelona, Spain, October 22-25, 2019.

We are thrilled to be presenting positive data on our Next Generation Capsid Development Program on the anniversary of our collaboration with Childrens Medical Research Institute of Australia, a leader in gene therapy, childhood cancer, embryology and neurological diseases. The goal of the collaboration is to develop novel, synthetic adeno-associated virus (AAV) capsids which are highly tropic for human tissues and optimized for manufacturing. These data give us further confidence that we can improve the performance of current AAV vectors, expanding our pipeline and strengthening our GeneRide platform, said Fred Chereau, CEO of LogicBio. Further, we are pleased to present additional preclinical data further supporting the durability of expression, compared to canonical gene therapy, in one of our GeneRide platform programs and to have been invited to speak on AAV manufacturing.

Panel PresentationTitle: AAV manufacturing: critical parameters influencing vector quality attributesPresenter: Matthias Hebben, Ph.D., VP, Technology Development, LogicBio Therapeutics (INV36)Session: 1d ATMP manufacturingSession date/time: October 23, 2019, 8:30-10:30 a.m. CEST

Poster PresentationsTitle: AAV development program: towards next generation of livertropic AAV variants (P025)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Title: Durability of factor IX expression in mice treated neonatally with a nuclease-free, promoterless, AAV-based gene therapy, GeneRide (P423)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Additional information on the meeting can be found on the ESGCT website: https://www.esgct.eu/home.aspx

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visit http://www.logicbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene...

Recommendation and review posted by Bethany Smith

LONDON, Oct. 16, 2019 /PRNewswire/ -- The report "Protein Expression Global Market Report 2019" published By TheBusinessResearchCompany.com is about Protein Expression Market which is spread across 150+ pages and provides latest industry data, market trends, allowing you to identify the products and key players.

The protein expression market (https://www.thebusinessresearchcompany.com/report/protein-expression-global-market-report) in the U.S. is governed by the Food and Drug Administration (FDA) that lays down a series of guidelines for the manufacturers and retailers of this industry. Within the FDA, the Center for Drug Evaluation and Research (CDER) regulates biological products under FDA 101 which includes gene therapy products and vaccines. These regulations ensure quality, safety and efficacy of biological therapeutics products, and speed up innovations that make these products safer and effective. In May 2014, the US's FDA announced a fast-track initiative to review its drugs and biologics policy to speed the availability of therapies to patients with serious conditions, and orphan drugs for rare diseases, while preserving the safety and efficacy standards. In 2016, the FDA also removed a rule (Section 610.21 of the FDA code) which specified minimal potency limits for certain antibodies and antigens. The European Medicines Agency has also introduced policies which include a provision to waive the scientific advice fee, which encourage more academic groups and small companies to propose candidates for biologics.

Stringent Regulations Will Hinder The Global Protein Expressions Market Growth

Government regulations related to protein therapeutics and production of biologics may hinder the protein expression market growth. Government regulations on biologics to undergo rigorous preclinical and clinical trials prior to regulatory approval, and time-consuming process for approval of biologics with regards to health and the safety of any individual are restraining the market growth. Marketing and distribution of biologics including insulin, hormones, therapeutic antibodies, and vaccines depends upon the successful completion of clinical trials, which is a long, expensive, and uncertain process. According to the FDA, for an approval of a new biologic, under the regulations (21 CFR 314.81(b)(2)(vii) and 601.70, a clinical trial approval usually takes 10-12 months where firms are required to submit a report annually on the status of clinical safety, clinical efficiency, clinical pharmacology, and nonclinical toxicology study.

Protein expression is a process in which proteins are synthesized, modified, regulated and controlled in living organisms according to the host cell. Protein expression includes yeast expression, insect expression, and bacterial expression, algal expression and mammalian cell expression. The global protein expression market was valued at about $1.74 billion in 2018 and is expected to grow to $2.63 billion at an annual growth rate of nearly 11% through 2022. Request for Sample Copy @ https://www.thebusinessresearchcompany.com/sample.aspx?id=2493&type=smp

Growing Demand For Biologics Will Drive The Protein Expression Market

Increase in demand for biologics to counter various genetic disorders and chronic diseases is a major factor driving the research and sales of protein expression. Biologics is a medicine produced from living organisms or contains components of living organisms such as protein, tissue, genes, allergens, cells, blood components, blood, and vaccines. The increasing use of biologics (therapeutic protein and others) to cure chronic diseases such as cancer, cardiovascular conditions and genetic disorders, is increasing the demand for protein expression devices and equipment. According to the World Health Organization, chronic disease prevalence is expected to rise by 57% globally, by the year 2020. According to an article published by Chemistry World in the year 2018, analysts expect the biologics market to hold a market share of more than a quarter of the entire pharmaceutical industry by 2020. The global biologics market is expected to grow at 9.9% during 2018-2024.

Microfluidics Technology- A Trend In The Protein Expression Market

Companies in the protein expression industry are increasingly adopting Microfluidics technology to enhance protein expression tests in order to reduce the time, cost, labor, and increase the accuracy and performance. The microfluidics technology effectively analyzes biological samples than the traditional (macroscale) instruments. Microfluidics technology is used to measure the expression of proteins on cells and optimizes the output to generate results regarding protein expression. Therapeutics-on-a-chip (TOC) uses microfluidic platform and is able to synthesize proteins in a point-of-care setting to reduce cost associated with storage and transportation of therapeutic proteins. For instance, companies such as MissionBio, NanoCellect Biomedical, RainDance Technologies and Sphere fluidics have implemented this technology in protein expression test. Place a Direct Purchase Order of Complete Report @ https://www.thebusinessresearchcompany.com/purchaseoptions.aspx?id=2493

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Therapeutic Proteins Market Report @ https://www.thebusinessresearchcompany.com/report/therapeutic-proteins-global-market-report

Biologics Market Report @ https://www.thebusinessresearchcompany.com/report/biologics-market

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Protein Expression Market Value is Expected to Reach $2.63 Billion by 2022 - PRNewswire

Recommendation and review posted by Bethany Smith

The Company plans to use this funding to progress development of its SVac platform towards a first in man clinical study to commence in two to three years from now.

In addition to raising new funds, Amarna has recruited a new Supervisory Board to help underpin this new clinical phase of its growth and development. Thomas Eldered has been appointed as Supervisory Board Chairman. Bernhard Kirschbaum, Maarten de Chateau, Ted Fjllman, Pim Berger and Guillaume Jetten have also joined the Supervisory Board (see biographies at the end of the release).

Ben van Leent, CEO of Amarna said: We are very happy to have attracted such strong investors. This significant new funding allows Amarna to accelerate the development of SVac and our lead product AMA001, and to help us achieve our ultimate goal: to become a leading global gene therapy player.We would like to extend a warm welcome to all of the members of our new Supervisory Board, led by the outstanding healthcare and biotech pioneer Thomas Eldered. They bring many years of in-depth life science knowledge and entrepreneurship to Amarna.

Thomas Eldered, new Chairman of Amarnas Supervisory Board, commented: Im very much looking forward to working at Amarna. Its viral gene delivery vector platform has the potential to make major medical breakthroughs possible, so that patients can be actually cured of significant diseases for which, to date, effective treatment have not become available. Together with my highly qualified and experienced colleagues in our new Supervisory Board, Im fully committed to help progress Amarna into the next important clinical stages of development of its groundbreaking technology.

SV40 Vectors are non-immunogenic in humans Viral gene delivery vectors that are currently used for in vivo gene therapy are ineffective because the particles are instable upon injection (in the case of lentiviral vectors) or because the particles are immunogenic in humans (in the case of AAV vectors). Gene delivery vectors derived from the macaque polyomavirus Simian Virus 40 (SV40) are an attractive alternative to lentiviral and AAV vectors for clinical gene therapy. Humans can be considered nave to SV40 since the virus only replicates in macaques, where it causes symptomless infections. Replication-defective SV40 vectors are non-immunogenic in humans and moreover, have the capacity to induce immune tolerance to the transgene products. SV40 vectors therefore hold a great potential for clinical applications treating genetic disorders, cancer, allergies and degenerative/inflammatory conditions such as neurodegenerative and psychiatric diseases, atherosclerotic cardiovascular disease, diabetes mellitus, arthritis, chronic obstructive pulmonary disease and many more.

Amarnas SVac platform: the benefits Amarna has genetically engineered the SV40 genome used for the production of vector particles and in parallel generated a novel Vero-based packaging cell line named SuperVero that produces similar numbers of vector particles to the currently used packaging cell lines but without contaminating wild type SV40 particles. Since SVac is safe, highly efficient, non-immunogenic in humans and vector particles can be cost effectively produced in SuperVero cells, Amarnas vector platform paves the way to clinically evaluate a whole new generation of SVac-based therapeutics for todays major diseases.

Notes to Editors

About Amarna Therapeutics Amarna Therapeutics is a privately held Biotech company founded in 2008 with its head office in Leiden, The Netherlands, and with a research facility in Seville, Spain. The company has developed the SVac viral gene delivery vector platform, the key to the success of gene replacement therapy and therapeutic reverse vaccines. SVac is safe, highly efficient and non-immunogenic in humans, creating opportunities to treat diseases with high unmet clinical need including genetic disorders, degenerative/inflammatory/autoimmune diseases, infectious diseases and cancer.

About Flerie Invest AB Flerie Invest AB is a Venture Capital firm located in Stockholm Sweden focused on life science investments globally.

About the Netherlands Enterprise Agency (RVO.nl) The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy and stimulates entrepreneurs, NGOs, knowledge institutions and organizations. The agency aims to improve opportunities, strengthen positions and help realize international ambitions with funding, networking, know-how and compliance with laws and regulations.

New Supervisory Board member biographies

Thomas Eldered received a graduate degree from Linkping Institute of Technology and founded Swedish listed pharmaceutical contract development and manufacturing company Recipharm AB. Presently, Thomas is Chief Executive Officer at Recipharm AB and at the Board of several companies including Kahr Medical Ltd, Provell Pharmaceuticals LLC, and Sixera Pharma AB.

Dr. Bernhard Kirschbaum studied biochemistry and physiological chemistry at the University of Konstanz and received his PhD in 1989. He moved on to work at the Rockefeller University and the Pasteur Institute before making a career in the pharmaceutical industry where he covered a broad range of technological and disease areas. In his most recent position, Bernhard served as head of Global Research and Early Development at Merck Serono where he was a member of the board. Currently he holds further Supervisory Board and SAB positions at Omeicos, Redx, FutuRx, Enlivex, KAHR and BioMedx.

Dr. Maarten de Chteau is Chief Executive Officer at Swedish biotech Sixera Pharma and Buzzard Pharmaceuticals, focusing on the development of drugs that restore the balance in diseased skin and cancer respectively. Maarten has extensive experience in pharmaceutical clinical development at Sanofi and Swedish Orphan Biovitrum (Sobi). Prior to that he worked as a financial analyst at two investment banks and conducted basic research at Lund University and Harvard Medical School. Maarten was the founder and CEO of Cormorant Pharmaceuticals, leading the development of an immune-oncology project. Cormorant was acquired by Bristol-Myers Squibb in 2016.

Dr. Ted Fjllman is the CEO of Prokarium, a London-based biotech, focusing on targeted oral vaccines and microbial immunotherapy. Ted has worked for clinical research and strategy consulting firms and built two early-stage companies. Ted holds a PhD from the University of Guelph, Canada, and has been recognized as a leader in biotechnology under the Sloan Foundation funded Synthetic Biology Leadership Accelerator Program (LEAP). He is a member of the international Bacterial Vaccines Network (BactiVac) management board, as well as an active member in the UK Bio-Industry Association (BIA).

Pim Berger is CEO and co-founder of Dutch IT company Schuberg Philis and one of Amarnas pioneering informal investors for over 10 years. He was managing director Netherlands of SiteSmith Inc., a US-based Internet infrastructure management services company. In 2000 SiteSmith Inc. was acquired by Metromedia Fiber Network Inc. (MFN) in a transaction valued at approximately $1.4 billion. Pim became Senior Vice President of MFNs European Region. In a management buyout by Pim and two partners in 2003 the Benelux subsidiary of MFN Inc. became Schuberg Philis.

Before this, Pim held several senior executive positions at Dutch IT companies Pink Elephant and PinkRoccade (now part of KPN). He holds a masters degree in computer engineering and economics from the Hogeschool IJsselland.

Guillaume Jetten is the CFO of Netherlands based MercachemSyncom, a leading European mid-sized contract research organization. Guillaume has been working in the healthcare industry for most of his career. He held positions at Merck, Sharpe & Dohme, and served as Chief Financial Officer (CFO) of Wolters Kluwer Health. He also worked as CFO for Galapagos between 2009 and 2014. Guillaume holds a masters degree in economics from the University of Maastricht and is a Certified Registered Accountant.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191016005612/en/

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Amarna Therapeutics Raises 10 million to Progress lead Development Candidate into Clinical Trials and Appoints a new Supervisory Board - BioSpace

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Global Longevity and Anti-Senescence Therapy Market" report has been added to ResearchAndMarkets.com's offering.

Global longevity and anti-senescence market will witness rapid growth over the forecast period (2018-2023) owing to an increasing emphasis on Stem Cell Research and increasing demand for cell-based assays in research and development.

An increasing geriatric population across the globe and rising awareness of antiaging products among generation Y and later generations are the major factors expected to promote the growth of global longevity and anti-senescence market. Factors such as a surging level of disposable income and increasing advancements in anti-senescence technologies are also providing traction to the global longevity and anti-senescence market growth over the forecast period (2018-2023).

Senolytics, placenta stem cells and blood transfusions are some of the hot technologies picking up pace in the longevity and anti-anti-senescence market. Companies and start-ups across the globe such as Unity Biotechnology, Human Longevity Inc., Calico Life Sciences, Acorda Therapeutics, etc. are working extensively in this field for the extension of human longevity by focusing on the study of genomics, microbiome, bioinformatics, and stem cell therapies, etc. These factors are poised to drive market growth over the forecast period.

The report provides analysis based on each market segment including therapies and application. The therapies segment is further sub-segmented into Senolytic drug therapy, Gene therapy, Immunotherapy, and Others. Senolytic drug therapy held the largest market revenue share in 2017. The fastest growth of the gene therapy segment is due to the Large investments in genomics.

Report Scope

The scope of this report is broad and covers various therapies currently under trials in the global longevity and anti-senescence therapy market. The market estimation has been performed with consideration for revenue generation in the forecast years 2018-2023 after the expected availability of products in the market by 2023.

The global longevity and anti-senescence therapy market has been segmented by the following therapies: Senolytic drug therapy, Gene therapy, Immunotherapy and Other therapies which includes stem cell-based therapies, etc.

Revenue forecasts from 2028 to 2023 are given for each therapy and application, with estimated values derived from the expected revenue generation in the first year of launch.

The report also includes a discussion of the major players performing research or the potential players across each regional longevity and anti-senescence therapy market. Further, it explains the major drivers and regional dynamics of the global longevity and anti-senescence therapy market and current trends within the industry.

The report concludes with a special focus on the vendor landscape and includes detailed profiles of the major vendors and potential entrants in the global longevity and anti-senescence therapy market.

The report includes:

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market Overview

Chapter 4 Global Longevity and Anti-senescence Market by Therapy

Chapter 5 Global Longevity and Anti-senescence Market by Application

Chapter 6 Global Longevity and Anti-senescence Market by Region

Chapter 7 Industry Structure in Longevity and Anti-senescence Market

Chapter 8 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/zy7jt

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Global Longevity & Anti-Senescence Therapy Market Review 2017-2018 and Forecast to 2023 - ResearchAndMarkets.com - Business Wire

Recommendation and review posted by Bethany Smith

A special advisor to the Charcot-Marie-Tooth Associations (CMTA) board of directors says he will match donations dollar-for-dollar up to $500,000 to support the organizations research strategy to fight the disease he has CMT2.

Bruce Chizen was diagnosed in 2016, after decades of experiencing symptoms. However, his physician discouraged him from undergoing genetic testing because he couldnt do anything about the results.

Unsatisfied, Chizen forged ahead with his quest for more disease information. The first place he turned to was CMTAs website. Since then, hes been grateful for the organizations help and encouraged by its efforts to find a cure that hes vowed to support it however he can.

His initial CMTA donation led to breakfast with his Silicon Valley neighbor and CMTA board chair, Gilles Bouchard. The meeting resulted in him taking on his current role.

A Brooklyn, New York native, Chizen graduated from Brooklyn College where he studied health sciences and planned to become a teacher. However, he learned that he had a head for business, and started out as a Mattel Toys salesman, and then became a Microsoft sales manager in the mid-80s. That was followed by stints at Apple and then Adobe, where he worked for 14 years, seven as CEO.

With his extensive business and nonprofit experience, Chizen appreciates how CMTA is run, calling the organization extremely focused and effective.

Ive seen a lot of waste, the semi-retiree states.

Chizen considers education and community services an essential part of the groups mission, and wants to fortify them. Hes also encouraged by the success of the organizations Strategy to Accelerate Research (STAR), which was created in 2008 with five elements: Assays (tests), animal models, stem cells, partners, and clinical trials. The goal is to find treatments to slow, stop, or reverse CMT progression.

The path to a cure is possible and probable, Chizen said.

Visit this site to double your donations to the organization. Contributions will go to implementing CMTAs new Type 2 strategy, a multi-pronged approach that includes projects on gene therapy, small molecule therapies, axon degeneration pathways, mitofusin activity regulation, and human stem cell studies. The strategy is outlined in the fall issue of The CMTA Report, which focuses exclusively on CMT2 and includes patient profiles. Its available to download here.

CMT2 is caused by genetic defects that disrupt the structure and function of axons of the peripheral nerves. Its less common than CMT1 and accounts for about one-third of all dominant CMT cases.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

Total Posts: 13

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Research Gets Boost With CMTA's Matching Donation Challenge - Charcot-Marie-Tooth News

Recommendation and review posted by Bethany Smith

ANI | Updated: Oct 14, 2019 14:31 IST

Washington D.C [USA], Oct 14 (ANI): Recently developed gene therapy seems to be a promising alternative for treating one of the most common causes of blindness, wet age-related macular degeneration (AMD), claims a study. Data presented at the 123rd Annual Meeting of the American Academy of Ophthalmology showed that six patients with wet age-related macular degeneration (AMD) went at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks.Researchers said the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential "one-and-done" treatment. It's not just about convenience; a more consistent treatment may also help people keep more of their vision."This is potentially paradigm-shifting," said lead researcher, Szilard Kiss, M.D., director of Clinical Research and chief of the Retina Service in the Department of Ophthalmology at Weill Cornell Medical College in New York City."It's the next evolutionary leap in treating AMD. When you think about what science fiction is and what science reality is; gene therapy for AMD is becoming a clinical reality," added Dr Kiss.AMD is a degenerative eye disease that happens when part of the retina is damaged. The damage happens when new, weak blood vessels form behind the retina at the back of the eye. These abnormal vessels leak, causing scarring and killing off the cells that allow us to see.One main reason why is that patients are undertreated. This is because most people with AMD must go to the ophthalmologist's office every four to eight weeks for an injection directly into their eye (oftentimes in both eyes).This can be a difficult schedule to maintain for many elderly patients struggling with other maladies and reliant on others to get them to their ophthalmologist visits. It's also unsustainable for the health care system.Last year alone, ophthalmologists performed more than 8 million anti-VEGF injections in the United States.Researchers have been searching for a better alternative to monthly injections almost from the moment anti-VEGFs were introduced. Gene therapy is emerging as one of the more promising alternatives to long-term anti-VEGF treatment.The goal of Dr Kiss' research is to develop a gene therapy that allows the eye to make its own anti-VEGF medicine. The ideal gene therapy would be administered not through a surgical procedure in an operating room, but through an injection into the eye that can be done in the doctor's office, just like routine anti-VEGF treatment is done today.To do this, Dr Kiss and colleagues have developed a next-generation vector that can insert into the cells of the eye, the genetic material that makes a molecule similar to a widely used anti-VEGF medicine called aflibercept. Once inside the cells, the DNA sequence begins making the aflibercept protein. (ANI)

Original post:
Gene therapy effective for treating wet age-related macular degeneration: Study - ANI News

Recommendation and review posted by Bethany Smith

Gene Therapy for Inherited Genetic Disorders Market research Report is a valuable supply of perceptive information for business strategists. This Gene Therapy for Inherited Genetic Disorders Market study provides comprehensive data which enhances the understanding, scope and application of this report.

A specified study of the competitive landscape of the global Gene Therapy for Inherited Genetic Disorders Market has alloted, offering insights into the company profiles, financial status, recent developments, mergers and acquisitions, and the SWOT analysis. This research report can provides a clear plan to readers concerning about the overall market scenario to further decide on this market projects.

The Gene Therapy for Inherited Genetic Disorders Market report profiles the following companies, which includes: BioMarin Pharmaceutical Inc., bluebird bio Inc., Orchard Therapeutics Plc, Spark Therapeutics Inc., Novartis AG

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This report studies the global Gene Therapy for Inherited Genetic Disorders Market status and forecast, categorizes the global Gene Therapy for Inherited Genetic Disorders Market size (value & volume), revenue (Million USD), product price by manufacturers, type, application, and region. Gene Therapy for Inherited Genetic Disorders Market Report by Material, Application, and Geography-Global Forecast to 2024 is an expert and far-reaching research provide details regarding the worlds major provincial economic situations, Concentrating on the principle districts (North America, Europe, and Asia-Pacific) and the elementary nations (United States, Germany, UK, Japan, Asian country, and China).

By Types: Eye Disorders, Hematological Disorders, Central Nervous System Disorders, Muscular Disorders, Others

By Applications: Hospital, Clinic, Research Institute, Others

Market Segment by Regions, regional analysis coversNorth AmericaEuropeAsia-PacificSouth AmericaMiddle East and Africa

Table of Contents

Global Gene Therapy for Inherited Genetic Disorders Market Size, Status and Forecast 20241 Market Overview2 Manufacturers Profiles3 Global Gene Therapy for Inherited Genetic Disorders Sales, Revenue, Market Share andCompetitionby Manufacturer4 Global Gene Therapy for Inherited Genetic Disorders Market Analysis by Regions5 North America Gene Therapy for Inherited Genetic Disorders by Countries6 Europe Gene Therapy for Inherited Genetic Disorders by Countries7 Asia-Pacific Gene Therapy for Inherited Genetic Disorders by Countries8 South America Gene Therapy for Inherited Genetic Disorders by Countries9 Middle East and Africa Gene Therapy for Inherited Genetic Disorders by Countries10 Global Gene Therapy for Inherited Genetic Disorders Market Segment by Type11 Global Gene Therapy for Inherited Genetic Disorders Market Segment by Application12 Gene Therapy for Inherited Genetic Disorders Market Forecast13 Sales Channel, Distributors, Traders and Dealers14 Research Findings and Conclusion15 Appendix

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Overview of the chapters analysing the global Gene Therapy for Inherited Genetic Disorders Market in detail:

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Gene Therapy for Inherited Genetic Disorders Market Global Competitive Analysis and Growth Forecast till 2024 | Key Players: BioMarin Pharmaceutica,...

Recommendation and review posted by Bethany Smith

October 10, 2019

Difei Yang, Ph.D., is Managing Director of Equity Research at Mizuho Americas, Research Division. Dr. Yang has been covering the biotech and pharma sector for nearly a decade, most recently as Managing Director at Aegis Capital.

She also worked in senior equity analyst roles at Brean Capital, R.F. Lafferty, WallachBeth Capital and Auriga Global Investors. In addition, she has held senior science, program management and business development roles within the pharmaceutical industry and has authored many granted U.S. patents and peer-reviewed scientific publications.

She holds a Ph.D. in chemistry from UCLA, as well as an MBA from Georgia State University and a B.S. degree in physics from Peking University, China.

In this exclusive 2,716 word interview in the Wall Street Transcript, Dr. Yang explores the valuation of certain key biotech stocks in her coverage.

In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders.

Its actually across multiple therapeutic areas. A few common themes were observing in that space is that, number one, we tend to see the highest probability of success for those companies where they target a single gene mutation, and secondarily, when these companies are vertically integrated, theyre able to control the manufacturing and basically make products to support clinical development as well as commercialization.

In gene therapy, we continue to see developments for treating neurological disorders, blood disorders and liver-mediated disorders

One example is curing a dreaded genetic disease in infants:

We cover a company called Audentes Therapeutics(NASDAQ:BOLD). They have a therapy that is currently in late-stage development treating X-linked myotubular myopathy XLMTM an inherited form of a neuromuscular disorder.

These patients are typically diagnosed when they are babies.

The disease involves severe muscle weakness, so not only do these babies not reach developmental milestones, they probably never acquired the skills to walk.

Muscle weakness also impacts their lung function. And the result usually leads to babies not being able to breathe on their own and require ventilators for support, in some cases 24 hours per day.

Audentes Therapeutics has a gene therapy. And based on early clinical data, what were seeing is that not only the babies are alive, they started getting off ventilator support

Get more detail on this and other biotech winners from this exclusive 2,716 word interview in the Wall Street Transcript.

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Gene Therapy Stocks Drug Candidates Gain Value as Therapies Reach Market - The Wall Street Transcript

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--

The "Gene Therapy - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy market worldwide is projected to grow by US$3.3 Billion, driven by a compounded growth of 32.7%. Lentivirus, one of the segments analyzed and sized in this study, displays the potential to grow at over 25.3%.

The shifting dynamics supporting this growth makes it critical for businesses in this space to keep abreast of the changing pulse of the market. Poised to reach over US$125.3 Million by the year 2025, Lentivirus will bring in healthy gains adding significant momentum to global growth.

Representing the developed world, the United States will maintain a 30% growth momentum. Within Europe, which continues to remain an important element in the world economy, Germany will add over US$133.3 Million to the region's size and clout in the next 5 to 6 years. Over US$117.2 Million worth of projected demand in the region will come from Rest of Europe markets. In Japan, Lentivirus will reach a market size of US$6.5 Million by the close of the analysis period.

As the world's second largest economy and the new game changer in global markets, China exhibits the potential to grow at 39.2% over the next couple of years and add approximately US$797 Million in terms of addressable opportunity for the picking by aspiring businesses and their astute leaders.

Presented in visually rich graphics are these and many more need-to-know quantitative data important in ensuring quality of strategy decisions, be it entry into new markets or allocation of resources within a portfolio.

Several macroeconomic factors and internal market forces will shape growth and development of demand patterns in emerging countries in Asia-Pacific, Latin America and the Middle East. All research viewpoints presented are based on validated engagements from influencers in the market, whose opinions supersede all other research methodologies.

Competitors identified in this market include:

For more information about this report visit https://www.researchandmarkets.com/r/lj1g7w

View source version on businesswire.com: https://www.businesswire.com/news/home/20191015005833/en/

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Gene Therapy Market Analysis, Trends, and Forecasts, 2025 - ResearchAndMarkets.com - Yahoo Finance

Recommendation and review posted by Bethany Smith

GlobalGene TherapyMarketdisplays the research on the revenue figures, stock details, and purchases of the major firms. The report delivers analytical data of trading aspects such as local consumption, global consumption, import, and exports. The report offers a breakdown of the revenue for the globalGene Therapymarket. A forecast for the estimated timeframe from2018 to2025 has been further claimed in this research. The global market is classified based on top manufacturers, region, type, and end-user. Fundamental weaknesses and strengths of the main contenders along with the risks encountered by them have been analyzed.

DOWNLOAD FREE SAMPLE REPORT:https://www.fiormarkets.com/report-detail/376052/request-sample

Outline of The Market:

The research report offers analytical data on trading aspects such as local consumption, global consumption, import, and exports. In terms of the competition analysis, value, sales volumes, SWOT analysis, and detailed company profiles are provided. The later section of the report serves a comprehensive overview of product specification, product type, product scope, and production analysis with key factors such as capacity, production, revenue, price, and gross margin. In additionally, charts, tables, and numbers included in the report help to offer a transparent view of the market. A development component of the overallGene Therapymarket dependent on end-clients is covered in this study. The growth rate each geography is estimated to attain during the forecast years has also been stated in the research report. Based on the application landscape, the report lists details concerning the market share, procured by each application segment.

On a regional basis, the globalGene Therapymarket has been segmented intoNorth America, Europe, Asia Pacific, South America, and the Middle East and Africa.

The worldwide market is an enlarging domain for the top market players:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

The Market Research/Analysis Report Contains Answers To Your Following Questions:

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Entry strategies, countermeasures to economic impact, marketing channels for the industry are further covered in the report. Market chain analysis by upstream raw materials and downstream industry has been provided. The report guides you in all problems such as which companies are offering similar products and services and who are their competitors.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team (sales@fiormarkets.com), who will ensure that you get a report that suits your needs.

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Global Gene Therapy Market 2019 Industry Emerging Trend, Market Players, Revenue Insights to 2025 - The Ukiah Post

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The PharmaSphere: Emerging Biotechnologies-Gene Therapy Market report intends to provide cutting-edge market intelligence and help decision makers take sound investment evaluation. Additionally, the report also highlights market entry strategies for various companies across the globe along with pipeline and product analysis. Besides, the report also identifies and analyses the emerging trends along with major drivers, challenges and opportunities in the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. It has also covered and analyzed the potential of PharmaSphere: Emerging Biotechnologies-Gene Therapy market and provides statistics and information on market size, shares and growth factors.

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About PharmaSphere: Emerging Biotechnologies-Gene Therapy Industry

The overviews, SWOT analysis and strategies of each vendor in the PharmaSphere: Emerging Biotechnologies-Gene Therapy market provide understanding about the market forces and how those can be exploited to create future opportunities.

Key Players in this PharmaSphere: Emerging Biotechnologies-Gene Therapy market are:

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Important application areas of PharmaSphere: Emerging Biotechnologies-Gene Therapy are also assessed on the basis of their performance. Market predictions along with the statistical nuances presented in the report render an insightful view of the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. The market study on Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market 2018 report studies present as well as future aspects of the PharmaSphere: Emerging Biotechnologies-Gene Therapy Market primarily based upon factors on which the companies participate in the market growth, key trends and segmentation analysis.

Product Segment Analysis of the PharmaSphere: Emerging Biotechnologies-Gene Therapy Market is:

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Regions Covered in PharmaSphere: Emerging Biotechnologies-Gene Therapy Market are:-

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Also it analyses, roadways and provides the global market size of the main players in each region. Moreover, the report provides knowledge of the leading market players within the PharmaSphere: Emerging Biotechnologies-Gene Therapy market. The industry changing factors for the market segments are explored in this report. This analysis report covers the growth factors of the worldwide market based on end-users.

In this study, the years considered to estimate the market size ofPharmaSphere: Emerging Biotechnologies-Gene TherapyMarket are as follows:-

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Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market - Porter's Five Forces Strategy Analysis and Forecast 2024 - The Charterian

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Gene Therapy Market Snapshot

The global gene therapy market is expanding at an exponential pace due to promising therapeutic outcomes of gene therapy, high prevalence and rise in incidence of cancer, and large number of clinical research pipeline products. In terms of revenue, the market was valued at US$ 17.0 Mn in 2017. It is projected to reach a value of US$ 5164.03 Mn by 2026, expanding at a CAGR of 40.0% from 2018 to 2026. The global gene therapy market is driven by new product approvals and commercialization, increasing demand and number of gene therapy treatment centers, and large number of patient population with unmet medical needs.

Gene therapy is a way of fixing genetic disorders by introducing a normal and healthy gene in place of defective genes in a cell in order to prevent or cure different types of genetic and chronic disorders for which no final cure has been developed. Gene therapy is considered as an important means of treatment, as it helps eliminate the usage of drugs, surgery, or other procedures that can have side-effects on the health of individuals. Moreover, gene therapy results in the formation of beneficial proteins that can help the body perform normal functions to its full potential. Cell-based technologies have been developing at a rapid pace, which in turn has driven the demand for gene therapy. Till date, only five gene therapy products have been approved and are currently in the infancy stage of commercialization. Gendicine was the first commercialized gene therapy product developed by the Shenzhen, China-based SiBiono GeneTech, in 2003. Gendicine was commercialized in China, in 2004.

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The global market for gene therapy has been expanding due to the intensive research that has offset in the domain of genetics. Demand for gene therapy across the globe has increased significantly due to the rise in awareness about the ability of gene therapy to cure diseases. Gene therapy has considerable potential to eliminate and prevent several genetic disorders and numerous life-threatening disorders, especially cancer, heart diseases, AIDS, cystic fibrosis, and age-related disorders. Gene therapy provides a complete cure to patients affected with genetic disorders, rather than ease symptoms with other therapeutic treatments. Promising therapeutic outcomes and increasing competition among leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs, in order to gain the first mover advantage, are projected to boost the gene therapy market during the forecast period. Moreover, high prevalence and rise in incidence rates of different types of cancers for which complete cure has not been developed is a key factor that is expected to drive the gene therapy market.

Large number of gene therapy candidates are in late stage clinical studies and are anticipated to commercialize during the forecast period. Significant investments in the field of gene-related research and development by various biopharmaceutical companies, governments, as well as research institutes offers potential opportunity to gain the first mover advantage in the gene therapy market. According to Alliance for Regenerative Medicine, in 2015, more than US$ 10 Bn has been invested by both public as well as private companies in gene therapy-related research. Companies, after initial success rates of gene therapy, are striving to increase the number of treatment centers in order to increase the access to a large number of patient pool requiring gene therapy treatments. Thus, increasing number of gene therapy treatment centers in developed countries offers significant opportunity to global players operating in the gene therapy market.

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The global gene therapy market has been segmented based on product, application, and region. In terms of product, there are only five gene therapy products that have been approved and commercialized globally. These include Yescarta, Kymriah, Luxturna, Strimvelis, and Gendicine. The Yescarta segment dominated the global market in 2017. It is projected to gain market share by the end of 2026. Yescarta bagged the first mover advantage in 2017 in the U.S, by launching the first CAR T therapy approved by the US FDA for the treatment of certain relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL). Furthermore, high prevalence of DLBCL and anticipated commercialization of Yescarta in Europe and other developed countries are projected to drive the demand for Yescarta during the forecast period. The Luxturna segment is projected to expand at a significant growth rate during the forecast period.

Based on application, the global gene therapy market has been divided into ophthalmology, oncology, and adenosine deaminase ?deficient severe combined immunodeficiency (ADA-SCID). The oncology segment accounted for a prominent share of the market in 2017. Of the five gene therapy products approved, three products are directed toward the treatment of certain forms of cancer. Moreover, increased application of gene therapy products in treatment of other forms of cancers is poised to drive the oncology segment during the forecast period.

Based on region, the global gene therapy market has been segmented into three major regions: U.S., Europe, and Rest of World. Europe is projected to account for a dominant share of more than 40% of the global market by 2026. Increasing number of gene therapy treatment centers in the region and high prevalence and rising incidence rates of non-Hodgkin lymphoma are projected to fuel the gene therapy market in the region during the forecast period. The U.S. is projected to follow Europe, in terms of share of global gene therapy market, by the end of 2026. It is estimated that in the U.S., every year, around 7,500 patients with refractory DLBCL are eligible for CART therapy. The gene therapy market in Rest of World is projected to expand at a notable CAGR during the forecast period. In 2003, Gendicine was approved in China and was commercialized in 2004. Since then, more than 30,000 patients with head and neck cancers have been treated with Gendicine, in China. The anticipated approval and launch of gene therapy products in developed countries, such as Japan, Australia & New Zealand, and GCC is projected to drive the gene therapy market in this region.

Majority of biopharmaceutical companies have made significant investments in the clinical research and development of gene therapy products. According to a report by the Alliance for Regenerative Medicine, there were around 34 gene therapy candidates in phase III clinical trials as of June 2017. Key players operating in the global market include Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited.

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Gene Therapy Market is Projected to Reach a Value of US$ 5164.03 Mn by 2026 - Online News Guru

Recommendation and review posted by Bethany Smith

The worldwide Gene Therapy Market report consists of the current evolution in the global industry and crucial elements that affect the overall growth of the Gene Therapy market. In addition, it analyses the worlds major region Gene Therapy business conditions, including product cost, profit, production capacity, supply-demand, and market growth rate and forecast, etc. It also provides an analysis on market-size, shares supply-demand analysis, sales value and volume study of different industries combined with Gene Therapy division study, with respect to important topographical regions. Finally, the report offers a new project SWOT analysis, investment feasibility analysis, and investment return estimates.

The overview part of the report consists Gene Therapy market dynamics which include market growth drivers, controlling elements, opportunities and Gene Therapy current trends together with the value chain analysis and pricing structure study. The main goal of the Global Gene Therapy the Market report is to supply a piece of up-to-date information on the market and also discover all the chances for Gene Therapy market growth. The report starts with a market perspective and provides markets fundamental initiation and the explanation of the international Gene Therapy industry. Global Gene Therapy Market Analyzed by segments by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Applications (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases)

By region

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Key Companies Analyzed in Report:

Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc.

COMPETITIVE LANDSCAPE:

Furthermore, Several companies are showing a keen interest in the Global Gene Therapy Market. They have realized the growth possibility and are eager in implementing strategic moves to improve their market stance and take the market advantage. While it studied a few and recorded their latest moves to get a grasp on trends that might shape the market in the coming years.

KEY INSIGHTS IN THE REPORT:

Similarly, the Gene Therapy research also details several characteristics related to the market, including standardization, major trends, deployment designs, ecosystem player profiles, operator case studies, potential roadmap, regulatory landscape, methods, possibilities, technologies, value chain, challenges, and drivers. Also, it provides a layout of the Gene Therapy markets dynamics, by pinpointing several aspects comprising limitations, value chain, expenditure milieu, client acceptance, and drivers.

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KEY QUESTIONS ANSWERED:

1. What is the total Gene Therapy market size in 2019 and what would be the expected size in 2025?

2. What will the market growth rate of the Aquaponics market in 2025?

3. Also, What is the key factor motivating the global Gene Therapy market?

4. What are the Gene Therapy market opportunities for the existing and entry-level players?

5. What are the recent developments and business strategies of the key players?

This report presents an overview of the competitive situation of the Global Gene Therapy market. The further a piece of research report explores the size and valuation of the global market in the forthcoming forecast period 2019-2025. The report also presents a detailed qualitative and quantitative data helps to improve evaluation and affecting the projected impact of these factors on the markets future growth prospects.

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Global Gene Therapy Market Briefing with Major Drivers, Trends and Company Profiles : Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark...

Recommendation and review posted by Bethany Smith