For Dr Jogin Desai, CEO and co-founder of Eyestem, a leading Indian cell therapy company, a meeting in 2015 with ophthalmologist Dr Rajani Battu for a medical appointment changed everything. Following the appointment, she introduced him to patients diagnosed with degenerative diseases of the eye and the terrible suffering they have to endure.

It was an eye-opener for Desai, a native of Ahmedabad and an expert in the field of drug development. Desai, at the time, was CEO of Cenduit, an industry leader in the IRT (interactive response technology) market and the eClinical field.

From school students who cannot see their blackboards to families with children suffering from blindness because of genetic disorders and senior citizens who have lost all agency due to their inability to read, the conversation opened my eyes to a world that I didnt know existed. In the same week, I met Prof S. Ramaswamy at Instem (Institute for Stem Cell and Regenerative Medicine), again for something completely unrelated, and we discussed the possibilities of how the next two-three decades will evolve with the maturation of cell and gene therapy products worldwide, recalls Desai, in a conversation with The Better India.

Following the meeting, it dawned on Desai at the time that the world is on the cusp of a fundamental change in the healthcare landscape that will evolve over the next few decades.

Today, Dr Rajani Battu is the chief medical officer at Eyestem.

Diseases that were previously incurable will start becoming curable. As I dove deeper, I understood that most of these therapies developed in the West will cost upwards of $450,000 (about Rs 3.5 Crore) per injection. This, I believe, presented a once-in-a-lifetime opportunity for a platform that can help disrupt this paradigm and create an incredible impact on the lives of patients. I made the decision to establish Eyestem within two days of these meetings in late 2015, adds Desai.

Eyestems vision is to create a scalable cell therapy platform to treat incurable diseases and democratise access to these newer technologies.

Incorporated in late 2015, Eyestem started operations in early 2017.

As cell and gene therapies become available across the world, they will only be available to the top 0.01 % of the worlds population. Our purpose is simple: We aim to democratise access by creating a therapy that is available to a large part of the bottom 99.99% of the population in the world. To our knowledge, we are the only company in the world with such a mission. While it is hard to predict the price of the therapy at this stage we anticipate the therapy to cost 80-90% cheaper than such comparable therapies in the West. The only purpose to start Eyestem has been ensuring that the therapy reaches the people that need it the most, claims Desai.

Through its flagship product Eyecyte-RPE, the company replaces lost retinal pigment epithelium cells. It is designed to restore sight for patients in the early stages of Age-Related Macular Degeneration (AMD) and arrest losses for those in the later stages.

There is no cure for Dry Age-Related Macular Degeneration in the world. It is the largest cause of blindness for people over 50. It is estimated that over 170 million people (40 million of which are in India) suffer from this disease. Eyestem is looking to take Eyecyte-RPE for Dry AMD through clinical trials and is one of six companies worldwide pursuing this treatment, he says.

Underpinning this type of treatment are Induced-Pluripotent Stem Cell (iPSC)-based products. An iPSC is a cell that can be developed into any cell of the body. At present, there are no iPSC-based products approved anywhere in the world.

Over the next ten years, Desai explains that several such products will start becoming available in the market and diseases such as inherited blindness/pulmonary fibrosis/diabetes which were hitherto considered incurable will start becoming curable.

Subsequently, an explosion in iPSC based therapeutics combined with gene engineering will be the next wave of pharma innovation. A lot of diseases occur when the cells of our body die early or are malfunctioning. At its most basic detail, one can create tissue of any kind (be it brain, heart, lung, eye, liver, kidney) through iPSC and replace the lost/damaged cells of the body. For example, in Dry AMD, the RPE layer degenerates and our treatment would be to inject that layer (from a healthy donor) back into the body part to treat the disease, he explains.

Curing Incurable Blindness and Other Diseases

So, how does Eyestems flagship product, Eyecte-RPE, work as a mode of treatment?

The Retinal Pigment Epithelium (RPE) is one of ten layers of the retina and acts as a foundation on which the rest of the retina sits. In Dry AMD, the RPE layer disintegrates and the retinal layers disintegrate just as a building collapses when the foundation collapses.

We have grown the RPE layer in our lab and this is Eyecyte-RPE, our flagship product. This product has been injected into special models of blind rats. We have proven that the rats where our product is injected can retain their sight while the ones that are controlled rats go blind. This animal experiment has been done at Oregon Health and Science University which is a global centre of excellence for macular degeneration research. The DCGI (Drugs Controller General of India) has approved manufacturing only for clinical trials and we have not yet applied for commercial manufacturing, he explains.

In addition to this, Eyestem is also among one of five companies globally advancing the treatment of Retinitis Pigmentosa (RP) to the market called Eyecyte-PRP. Eyecyte-PRP replaces the photoreceptor cells that are lost as a consequence of this disease. RP is a group of rare, genetic disorders that involve loss of the light-sensing photoreceptor cells in the retina. It affects children and causes total blindness by the time they reach their 20s and 30s. It is estimated to affect 4 million children worldwide, of which 1.5 million are in India. Human trials for Eyecyte-PRP to treat retinitis pigmentosa is expected to begin in the second half of FY 2023.

Similarly, Eyestem is also looking to treat Idiopathic Pulmonary Fibrosis (IPF), a serious chronic disease that affects the tissue surrounding the alveoli (tiny air sacs in your lungs), with a pluripotent stem cell-based approach.

Our first human trials for our Aircyte-AEC treatment to treat idiopathic pulmonary fibrosis will begin in the second half of FY2024. Aircyte-AEC is a suspension of lung alveolar epithelial cells that are lost due to this disease. It is worth noting that pulmonary fibrosis occurs as an end-stage event in several other diseases like COVID-19, Tuberculosis and Chronic Obstructive Pulmonary Disease, notes Desai.

Meanwhile, for the sake of clarification, we asked Desai whether cell therapy necessarily means stem cell therapy? Yes, it means the same from a laymans perspective. The only difference is that there are clinics that advertise stem cell therapy for patients in India as well as the US. It is important to remember that none of these stem cell therapy products are approved by the DCGI in India or the FDA in the United States, he notes.

State of Cell Therapy in India, Funding and Moving Ahead

There are only a handful of companies in India that are pursuing cell therapy. According to Desai, the developed world, especially countries like Israel, Japan and the United States, are at least two decades ahead of India in that regard.

As more incurable diseases become curable (think diabetes or cancer) it is absolutely imperative for India to develop a base of such product development research or else we will find ourselves in a situation similar to the HIV epidemic (and cell therapy is not easy to reverse engineer unlike HIV medicines). Our patients will be at the mercy of predatory pricing of global pharma unless globally innovative products are manufactured and made available locally. Our nine-member team at Eyestem, a majority of whom are cell biology scientists specializing in this field, is looking to significantly address this issue in advance, says Desai.

Meanwhile, Desai claims that his venture has been very selective in raising funds so far since its purpose is not to raise rounds of money with higher valuations but to benefit end patients. They are laser-focused on creating affordable cell therapy and hence have set themselves a benchmark of not spending more than $4 million from idea to first in human trials. Most pharmaceutical companies, he claims, spend 10 times this amount to reach this stage.

Any drug development venture needs smart money. We were fortunate to have interest from a group of investors who have multi-decade experience in global drug development. Two institutional investors (Endiya Partners and Kotak Private Equity) joined in a subsequent pre-series A round. We are currently raising our series A and we are halfway there. About 30% has been committed by current shareholders and we have a soft commitment from a global venture fund for the other 20%. We anticipate closing this round in the next 8-12 weeks. Investors invest in ventures like ours due to the promise of immense market potential for these therapies. As soon as one obtains human data, startups like ourselves reach an inflexion point and become extremely valuable, claims Desai.

Please Note: The Better India does not verify for the future efficacy of any therapy or medical treatment mentioned in the article. Kindly consult your doctor for an informed medical opinion.

(Edited by Vinayak Hegde)

Read the original here:
Doctor Innovates Cell Therapy in India; Seeks to Make Vision Restoration 80% Cheaper - The Better India

Recommendation and review posted by Bethany Smith

The hemochromatosis gene, known as HFE, helps regulate the bodys absorption of iron. Some people can inherit a mutation to this gene that causes their bodies to absorb too much iron. Most cases occur due to C282Y and H63D mutations. When this happens, a person can develop hemochromatosis.

A person may develop symptoms related to too much iron in the body, such as joint pain, fatigue, and a loss of libido. Over time, they may also develop serious complications, such as liver damage.

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) states that rare forms of hemochromatosis, called non-HFE hemochromatosis, occur due to mutations in the HAMP and HJV genes. Healthcare professionals may also refer to the HJV gene as the HFE2 gene.

If these mutations occur, a person will develop complications and symptoms at a younger age. They may also develop complications by the time they are teenagers.

This article reviews what the hemochromatosis gene is, symptoms associated with hemochromatosis, and more on how a person inherits the gene.

Hereditary hemochromatosis is a genetic disease that alters how the body regulates iron absorption. The condition causes a persons iron levels to increase to dangerous levels.

According to the National Human Genome Research Institute, in the case of hereditary hemochromatosis, a person is likely to develop the condition if they inherit two copies of the mutated hemochromatosis gene, or HFE gene. However, it is important to note that not everyone who inherits two copies will experience symptoms.

A person who inherits one copy of the mutated gene will either not develop signs and symptoms or experience mild symptoms.

When functioning normally, this gene helps regulate and prevent the excessive absorption of iron. When too much iron circulates in the body, it can cause damage to tissues and organs.

Some affected areas include:

Hereditary hemochromatosis affects approximately 1 million people in the United States.

When too much iron consistently enters the body, a person may start to experience symptoms related to the condition.

The NIDDK states that common symptoms of too much iron include:

People may also experience a darkening of their skin color, which initially appears on sun-exposed areas such as their face. This is often known as bronzing.

Not everyone will develop symptoms. Additionally, symptoms usually occur after a person reaches 40 years old, and females may develop symptoms 10 years later compared to males.

Without treatment, a person may develop complications that could cause additional symptoms. Some potential complications include:

Both parents can carry the hereditary hemochromatosis gene.

According to the National Human Genome Research Institute, a child that inherits two copies of the mutated gene has a high risk of developing hemochromatosis. However, having two copies does not guarantee a child will develop the condition.

When a person inherits only one copy of the gene, they may not develop any symptoms of the condition at all, or they may only develop slight symptoms. In these cases, they may become silent carriers of the condition.

In other words, if they have a child with another silent carrier, their child has a chance of inheriting two mutated genes and a higher chance of developing hemochromatosis.

Parents pass the hemochromatosis gene to their children. A child inherits two copies of the gene, one from each parent.

The affected genes may be recessive or dominant, which affects how many copies of the genes a person needs to develop the condition.

According to the Genetic and Rare Diseases Information Center (GARD), a child of two recessive gene carriers has a:

On the other hand, a child with a dominant gene change has a:

Several potential mutations can occur within the genes that can cause hereditary hemochromatosis to develop.

The location of the mutation determines the type of hereditary hemochromatosis a person may develop. Healthcare professionals classify the type of hereditary hemochromatosis based on the age of onset, which genes are affected, and how it is inherited.

A person can inherit an autosomal recessive condition if they inherit one copy of a mutated gene from each parent.

Individuals can develop an autosomal dominant condition if they inherit a single copy of the mutated gene from one parent. A person has a 50% chance of inheriting the mutated gene and developing the condition.

GARD states that the mutations can occur on the following genes:

Type 5 hereditary hemochromatosis occurs due to changes with the FTH1 gene. Only one family in Japan has reported the condition.

According to a 2015 article, healthcare professionals perform gene testing for hereditary hemochromatosis if they suspect a person has an iron overload alongside high levels of ferritin, which is a blood protein that stores iron, and transferrin, which is a protein in that transports iron the blood. High levels can indicate that a person has hemochromatosis.

Gene testing can confirm a hereditary hemochromatosis diagnosis.

A person with hemochromatosis will need regular testing as part of their treatment. Testing can help check iron levels and look for complications related to too much iron.

A doctor may recommend some common tests, such as a complete blood count to check for iron and protein levels, a liver biopsy to check for liver damage, and an MRI.

A person with hemochromatosis has a good chance of living a normal, healthy lifespan provided they seek treatment for their condition.

Without diagnosis and treatment, a person has a higher chance of developing serious health complications, such as liver damage or heart disease, that can affect a persons overall outlook and life expectancy.

The most common treatment for reducing iron in the blood is via phlebotomy, or drawing blood. In addition, a doctor may recommend dietary changes, iron chelation therapy, and treating any complications of the disease.

At first, a person will likely need regular blood draws. Once their blood iron levels reach a normal level, the number of blood draws will vary based on how well their ferritin and transferrin saturation levels stay within the normal range.

Regular treatment can help prevent complications and reduce their impact on a persons life if they have already begun. As a result, a person should seek treatment even if complications have already started.

A person who has a sibling living with hereditary hemochromatosis should speak with a doctor about undergoing genetic testing due to an increased risk of also having the mutated genes.

Additionally, a person with a parent who either has the condition or is a known carrier should speak with a doctor about genetic testing.

Finally, a person who knows they may have the mutated genes should speak with a doctor if they develop any symptoms that could indicate a problem with iron absorption.

Hereditary hemochromatosis can occur when a person inherits one or more copies of a mutated HFE gene. Hemochromatosis results in the body absorbing too much iron, which can lead to complications, such as cirrhosis.

A person should seek treatment for the condition as soon as possible to help prevent complications. Treatment typically involves blood draws to help reduce iron levels.

A person should also contact a doctor if they experience symptoms of iron absorption issues and have a known family member who either is living with the condition or may be a carrier.

View post:
Hemochromatosis gene: Definition, signs, and is it hereditary? - Medical News Today

Recommendation and review posted by Bethany Smith

This week on Market Squared we look at the trucker convoys and protests that befuddle us, and why mocking them, or ignoring them, is not an option

I submit the following to GuelphToday for this weeks column knowing that Im about to nuke my email from orbit. It had a good run.

So lets talk about whats going on in Ottawa right this moment (at noon on Saturday), and what happened here in Guelph last weekend.

Though theres a difference in scale, the intention and desire of the ones organizing these two gatherings are more or less the same, but while the so-called 'Freedom Convoy' has gotten a lot of attention, whats happened in Guelph with these anti-mandate groups has gotten practically none.

Last weekend, a couple of hundred people who dont consider themselves anti-mask or anti-vaccine walked though Old Quebec Street making noise while maskless to say theyre fed up with the pandemic and pandemic restrictions. Im glad were finally doing this, one woman said. Another man asked the stunned people looking on in Old Quebec Street to take off their masks and show off their smiles.

If I were to have stopped and asked those people what they just saw, they would probably be hard pressed to explain it to me. A lot of those people were too young to remember the 'freak outs'of the 1960s, and the one security guard I saw arrived on the scene several minutes late. It was over and done in less than eight minutes.

Consider what happened last Saturday as an experiment. It was an experiment for those participating to see how far they can push things, and it was an experiment for our city leadership: Can they ignore whats almost literally happening under their nose?

I know 90 per cent of the eligible population is fully vaccinated, but this is not just a matter of being vaccinated because Ive been told by a few of the people participating in these rallies that theyve gotten two jabs. Its also worth noting that over 80 per cent of Canadian truckers are fully vaccinated, but look at the attention wrought by the other 20.

To understand the inherent danger, you have to understand how people are indoctrinated. To use the word feels disturbing, but there is a continuum of information, or misinformation, that people go down and it starts with the simplest of ideas, an idea that youve already thought about in the course of the last 22 months: Arent you sick of this?

I talked to one man a few weeks ago who told me that he wont get vaccinated because he doesnt trust the government and he doesnt trust big pharma. Hey, fair enough. My sense is that a majority of Canadians, if asked, will enunciate some level of mistrust in most institutions, from the Government of Canada to the Loyal Order of Water Buffaloes. But this is how it begins.

The seeds of doubt, perhaps long sowed in the person, are then fertilized with a rapidly changing information landscape. With COVID-19 weve seen the scientific process happen in real time, and the best advice of March 2020 (disinfecting your groceries anyone?) has now been proven by researchers as pointless and ineffective in stopping the spread.

So now you dont know what to believe. Its possible you might have once trusted your family doctor, if you have a family doctor, but he or she is saying all the things you hear from the three levels of government and the World Health Organization. If you cant trust them, how can you trust your doctor? Are they in on it (whatever it is)?

Youre told to wear a mask, but some people you know on Facebook say that they dont work. Youre told to get a vaccine, but then youre told some vaccines have side effects, and those same online friends are now telling you that the other vaccines are bad, or that theyre gene therapy to change your DNA(theyre not).

And now you see the real plot and intentions. COVID-19, whether its real or fake, is a means to an end. That end could be Agenda 2021, or Agenda 2030, or the Great Reset, or some other international conspiracy to grind people under foot or de-populate the planet. All of this sounds crazy, but its also the best case scenario.

Whats more disturbing are the white supremacists and far-right figures who understand well how indoctrination works and have attached themselves to the anti-mandate movements, like the trucker convoy, to find new blood. The frightening thing is that the ones being recruited dont know or dont care that their perversion is happening, theyre just happy to have friends who believe as they believe.

And thats why we need to take these things seriously, the trucker rallies and the freak outs through local malls. To stand back and watch without saying anything is a kind of dereliction. To stand there and laugh, or to call those people names, is even worse.

Were rapidly approaching the second anniversary of the first lockdown because of COVID, and our public health leaders have started to pivot to the idea that COVID is something we must start learning to live with. Meanwhile, the pandemic of radicalization is just beginning, and living with it, like ignoring it, is not an option.

Read the rest here:
The pandemic of radicalization has begun, and we can't ignore it - GuelphToday

Recommendation and review posted by Bethany Smith

Vertex finished out 2021 strong, smashing analysts sales expectations for the full year. Chalk it up to the companys entrenched position in cystic fibrosis, where Vertex expects to maintain its lead for years to come, according to CEO Reshma Kewalramani, M.D.

Vertex snared $7.57 billion in product revenues for 2021, signaling a 22% increase year over year. For 2022, the company expects to pull in sales of $8.4 billion to $8.6 billion. For now, that growth is firmly tethered to Vertexs bread-and-butter CF franchises.

The companys 2022 guidance suggests penetration of additional CF patients will occur more rapidly than expected with age expansion and full-year impact of ex-U.S. reimbursements, RBC Capital Markets Brian Abrahams wrote in a note to clients Wednesday.

During its call Wednesday, Vertex repeated estimates that there are more than 25,000 patients who could benefit from CF newcomer Trikafta and arent yet on therapy. The company splits those patients into three groups: those whove yet to start on Trikafta in countries where the drug was recently reimbursed, patients in territories where the drug hasnt been reimbursed and younger patients, who Vertex aims to address with future Trikafta label expansions.

RELATED:JPM 2022: Vertex's Trikafta holds the line as company lays groundwork for gene editing launch

Meanwhile, the $8.4 billion to $8.6 billion range Vertex laid out seems notoriously conservative, Evercore ISIs Liisa Bayko wrote in a note to clients. [W]e expect VRTX to beat and raise throughout the year, she added.

Trikafta, for its part, carried the bulk of the sales weight last year, bringing in $1.69 billion for the last three months of 2021. The drug made about $1.09 billion during that same period in 2020. Trikafta reaped full-year 2022 sales of $5.69 billion, marking a blockbuster-worthy increase over the $3.86 billion it made in 2020.

Older CF med Kalydeco proved to be the companys next top sellerthough by a significantly smaller marginwith fourth quarter sales of $152 million. The drug made $684 million for the year. Orkambi made $147 million for the quarterslightly down from 2020s $215 millionand $772 for the year, while Symdeko pulled $80 million in the fourth quarter and $420 million for all of 2021.

Vertex is working to branch out from its CF base, and analysts seem to share the companys hopes for phase 1/2 Type 1 diabetes candidate, VX-880. That drug could potentially unlock a multibillion-dollar opportunity, which is comparable to CF and nearly all upside in our model, RBCs Abrahams said. CF forms the backbone of Vertexs pharma business, and investors have long pushed the company to diversify.

Vertex has also pinned its hopes on its gene editing program for sickle cell disease and beta thalassemia, CTX001, as its next commercial launch, Kewalramani said. The company sees tremendous potential for the treatment, and Vertex has already kicked off launch preparations ahead of planned global regulatory submissions toward the end of the year. The company is building out market access, patient support and its doctor-facing teams, as well as finalizing its manufacturing and supply chain network, the company said during its earnings call.

RELATED: Fierce Biotech's top 10 data readouts in 2022 | CRISPR Therapeutics and Vertex's CTX001amyotrophic lateral sclerosis

Still, when it comes to CF, Vertex is confident it can maintain its place at the head of the table, Kewalramani said.

More patients around the globe are treated with a Vertex CFTR modulator today than ever before, and the vast majority of that is with Trikafta, which boasts remarkable clinical trial data, the CEO said. If theres any medicine that will compete with Trikafta, it has to go head-to-head against Trikafta in clinical trials.

It has to have improved benefit, and you have to have the long-term data, she added.

The only company that has that right now is Vertex, and the most advanced competitor to Trikafta is the companys own triple combo of tezecaftor and the experimental VX-121 and VX-561, which is in phase 3 testing.

Kewalramani highlighted recent real-world Trikafta data, garnered from more than 16,000 U.S. patients, which showed the drug led to an 87% reduction in risks of lung transplant, 77% fewer pulmonary exacerbations and a 74% reduction in risk of death.

That said, the company is on the cusp of a critical external event that should settle the viability of VRTXs dominance in CF that is ABBVs triplet data, Evercores Bayko said. Consensus opinion is pointing toward lackluster data from AbbVie, but, It would be an unwelcomed surprise if the data were comparable to Trikafta, the analyst added.

AbbVie's combo, which isin phase 2 testing, comes from the company's purchase ofGalapagos' CF pipeline for$45 million in late 2018.

Read more here:
What competition? Vertex touts Trikafta's competitive edge on back of strong 2021 - FiercePharma

Recommendation and review posted by Bethany Smith

Matthew Hepburn, MD, Senior Advisor to the Director, Pandemic Prevention, U.S. Office of Science and Technology Policy (OSTP), Executive Office of the President

NORTH BETHESDA, Md. (PRWEB) January 27, 2022

The International Society for Pharmaceutical Engineering (ISPE) announced Matthew Hepburn, MD, Senior Advisor to the Director, Pandemic Prevention, U.S. Office of Science and Technology Policy (OSTP), Executive Office of the President, as a confirmed keynote for the 2022 ISPE Facilities of the Future Conference, taking place on 12 February in North Bethesda, Maryland and virtually.

The opening keynote session COVID's Impact on Pharma Facilities of the Future will take a high-level look ahead at global pharmaceutical manufacturing and related regulatory systems. Hepburn will discuss Lessons Learned from Operation Warp Speed for Rapid Vaccine Development at the opening plenary session and the executive forum dinner.

The mission of OSTP is to maximize the benefits of science and technology to advance health, prosperity, security, environmental quality, and justice for all Americans. At the OSTP, Hepburn works on preparing the country for future pandemics, with a spotlight on the acceleration of vaccines, therapies, and test development.

Previously, Hepburn was the Director of Vaccine Development at Countermeasures Acceleration Group, formerly known as Operation Warp Speed, which is an effort between the Defense Department and the Department of Health and Human Services. During the Obama administration, he was the Director of Medical Preparedness for the White House National Security Council. Additionally, he served as an infectious disease doctor for the U.S. Army for over two decades.

Representatives from Bristol-Myers Squibb, CRB, and Merck will round out a compelling line-up of expert speakers at the opening plenary session.

Featuring technical presentations from regulatory authorities and industry leaders already planning and building facilities of the future, the 2022 ISPE Facilities of the Future Conference will explore topics such as patient-specific cell and gene therapy facilities, new developments in the use of artificial intelligence, innovations in treatment and the transforming technologies that produce them, and moving forward from the COVID-19 era.

To ensure this experience is accessible to all, this conference will be in North Bethesda, Maryland, with virtual componentsdelivering thought-provoking learning and global networking opportunities whether attendees choose to join us in-person or virtually.

Explore the agenda and register at ISPE.org/FOF22.

About ISPEThe International Society for Pharmaceutical Engineering (ISPE) is a not-for-profit association serving its Members through leading scientific, technical, and regulatory advancement across the entire pharmaceutical lifecycle. The 18,000 Members of ISPE are building solutions in the development and manufacture of safe, effective pharmaceutical and biologic medicines, and medical delivery devices in more than 90 countries around the world. Founded in 1980, ISPE has its worldwide headquarters and training center in North Bethesda, Maryland, USA, and its operations center in Tampa, Florida, USA. Visit ISPE.org for more information.

Share article on social media or email:

Here is the original post:
US Office of Science and Technology Senior Advisor to Deliver Keynote Address at the 2022 ISPE Facilities of the Future Conference - PR Web

Recommendation and review posted by Bethany Smith

This article was originally published here

Mol Biotechnol. 2022 Jan 29. doi: 10.1007/s12033-021-00431-7. Online ahead of print.

ABSTRACT

Biotechnological approaches have always sought to utilize novel and efficient methods in the prevention, diagnosis, and treatment of diseases. This science has consistently tried to revolutionize medical science by employing state-of-the-art technologies in genomic and proteomic engineering. CRISPR-Cas system is one of the emerging techniques in the field of biotechnology. To date, the CRISPR-Cas system has been extensively applied in gene editing, targeting genomic sequences for diagnosis, treatment of diseases through genomic manipulation, and in creating animal models for preclinical researches. With the emergence of the COVID-19 pandemic in 2019, there is need for the development and modification of novel tools such as the CRISPR-Cas system for use in diagnostic emergencies. This system can compete with other existing biotechnological methods in accuracy, precision, and wide performance that could guarantee its future in these conditions. In this article, we review the various platforms of the CRISPR-Cas system meant for SARS-CoV-2 diagnosis, anti-viral therapeutic procedures, producing animal models for preclinical studies, and genome-wide screening studies toward drug and vaccine development.

PMID:35091986 | DOI:10.1007/s12033-021-00431-7

Visit link:
The Trend of CRISPR-Based Technologies in COVID-19 Disease: Beyond Genome Editing - DocWire News

Recommendation and review posted by Bethany Smith

A gene-editing treatment known as CRISPR appears to have positive results for people suffering from a rare visual disorder.

According to new reports, researchers injected seven volunteers with CRISPR to treat a rare vision disorder. Unlike other treatments, scientists designed CRISPR to fight diseases at a genetic level. Following the experiment, some of the volunteers noted a marked improvement in their eyesight. The affected individuals were all born with vision disorders, however, thanks to CRISPR, theyre now able to see better.

This is the first time scientists injected CRISPR directly into the human body. Previously, scientists had removed affected cells from the body and then conducted tests using the gene-editing procedure in the safety of a lab. From there, the scientists infused the modified cells back into the patients. With this experiment, though, the seven volunteers were injected directly with the treatment.

Scientists at the Casey Eye Institute conducted the study. The institute itself is part of the Oregon Health & Science University. Dr. Mark Pennesi shared the results of the CRISPR trial at the International Symposium on Retinal Degeneration late last year.

Following the injections, one of the patients shared that she was now able to safely navigate the area where she works. Another patient said that he could now see colors for the first time after the treatment. Both volunteers had suffered from LCA, or Leber congenital amaurosis, a severe vision impairment.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. Scientists engineered the treatment in 2012. It was intended to act as a biological tool for altering DNA. In the past ten years, though, scientists have found a multitude of other users for the gene-editing procedure.

Thats where the most recent study comes into play. By injecting the CRISPR directly into the body, scientists were able to see how it would more greatly affect it as a whole. Additionally, being able to inject it directly would allow them to treat disorders and diseases in areas where it isnt safe to remove cells, like the brain.

Because it focuses on editing things at a genetic level, scientists hope that CRISPR will open new doors to fighting cancer and other diseases. Once improved and thoroughly studied, it could become one of the most powerful treatments the medical community has available.

Its a really amazing technology and very powerful, Pennesi told NPR back in September.

Now that were seeing some additional pushes for the treatment, we could see some other studies appearing in the coming months. Of course, it is worth noting that not all the volunteers experienced any improvements. As such, theres no real timetable for when CRISPR could become a more widespread treatment.

See more here:
This remarkable tech can actually improve the eyesight of the visually impaired - BGR

Recommendation and review posted by Bethany Smith

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene-Editing Market report will be very beneficial to both of the sides in the market that is an established firm and a relatively new entrant. It helps the established firms to know about the moves which are being performed by their competitors whereas it helps the new entrants by educating them about the market situations and the industry trends. This market report includes market share appraisals for regional and global levels, detailed overview of parent market potential and niche segments/regions exhibiting promising growth, in-depth analysis of the global order management software market and current & future trends to elucidate imminent investment pockets.

An influential market report is quite fruitful in helping to understand the market definition and all the aspects of the market including CAGR values and key profiles. This report provides pin-point analysis for changing competitive dynamics. It provides a forward looking perspective on different factors driving or restraining market growth. It provides a six-year forecast assessed on the basis of how the market is predicted to grow. This market report helps in understanding the key product segments and their future. The credible report provides pin point analysis of changing competition dynamics and keeps business ahead of the competitors.

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market is expected to witness market growth at a rate of 26.88% in the forecast period of 2021 to 2028. Data Bridge Market Research report on clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market provides analysis and insights regarding the various factors are expected to be prevalent throughout the forecast period while providing their impacts on the markets growth. The rise in the demand in the food industry for better products with improved quality and nutrient enrichment is escalating the growth of clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market.

Download Exclusive Sample Report (350 Pages PDF with All Related Graphs & Charts) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-crispr-gene-editing-market

The major players covered in the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market report are Applied StemCell, ACEA BIO, Synthego, Thermo Fisher Scientific, GenScript, Addgene, Merck KGaA, Intellia Therapeutics, Inc, Cellectis, Precision Biosciences, Caribou Biosciences, Inc., Transposagen Biopharmaceuticals, Inc, OriGene Technologies, Inc., Novartis AG, New England Biolabs, Rockland Immunochemicals Inc., ToolGen, Inc., TAKARA BIO INC., Agilent Technologies, Inc., Abcam plc, and CRISPR Therapeutics AG among other domestic and global players.

Competitive Landscape and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene-Editing Market Share Analysis

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, clinical trials pipelines, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companies focus related to clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene is referred to as a genome editing technology that permits the genetic material to be added, altered and removed in an organisms DNA.

Major factors that are expected to boost the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the forecast period are the rise in the incidence ofgeneticdisorders and the utilization of genome editing. Furthermore, the private and government funding is further anticipated to propel the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market. Moreover, the rise in thetechnologydevelopment in the CRISPR gene editing is further estimated to propel the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market. On the other hand, the off target effects and delivery is further projected to impede thegrowthof the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the timeline period.

In addition, the growing of the gene and cell therapy area and the CRISPR gene editing scope in the agriculture sector will further provide potential opportunities for the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the coming years. However, the ethical issues and consequences regarding the human genome editing might further challenge the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the timeline period.

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene-Editing Market Scope and Market Size

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market is segmented on the basis of therapeutic application, application, technology, services, products and end users. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

For More Insights Get FREE Detailed TOC @ https://www.databridgemarketresearch.com/toc/?dbmr=global-crispr-gene-editing-market

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene-Editing Market Country Level Analysis

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market is analysed and market size information is provided by country, therapeutic application, application, technology, services, products and end users as referenced above.

The countries covered in the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market report are U.S., Canada and Mexico in North America, Peru, Brazil, Argentina and Rest of South America as part of South America, Germany, Italy, U.K., France, Spain, Netherlands, Belgium, Switzerland, Turkey, Russia, Hungary, Lithuania, Austria, Ireland, Norway, Poland, Rest of Europe in Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Vietnam, Rest of Asia-Pacific (APAC) in Asia-Pacific (APAC), South Africa, Saudi Arabia, U.A.E, Kuwait, Israel, Egypt, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA).

North America dominates the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market due to the considerable investments made by biotechnology and pharmaceutical companies. Furthermore, the rise in the healthcare infrastructure will further boost the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the region during the forecast period. Asia-Pacific is projected to observe significant amount of growth in the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market due to the increasing of the per capita income. Moreover, the early accessibility of authorized therapies is further anticipated to propel the growth of the clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market in the region in the coming years.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, disease epidemiology and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Read More @ https://www.databridgemarketresearch.com/reports/global-crispr-gene-editing-market

Patient Epidemiology Analysis

The clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing market also provides you with detailed market analysis for patient analysis, prognosis and cures. Prevalence, incidence, mortality, adherence rates are some of the data variables that are available in the report. Direct or indirect impact analysis of epidemiology to market growth are analysed to create a more robust and cohort multivariate statistical model for forecasting the market in the growth period.

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Contact:

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475

Email @Corporatesales@databridgemarketresearch.com

Go here to read the rest:
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene-Editing Market 2022 Business Outlook with Pandemic Scenario Analysis and...

Recommendation and review posted by Bethany Smith

AstraZeneca said the U.S. government agreed to purchase additional 500,000 doses of its Covid-19 preventive medicine Evusheld (tixagevimab/cilgavimab).

AZ had said that it would initially supply 1 million doses of Evusheld. However, if most of the doses go to the U.S., other countries will have limited access to AZs preventive therapy.

AZ welcomed the U.S. governments plan to buy 500,000 doses of Evusheld on Wednesday.

Evusheld is a combination drug mixing long-acting antibodies, tixagevimab and cilgavimab, derived from B cells donated by convalescent patients after Covid-19 infection.

The antibody treatment was discovered by Vanderbilt University Medical Center and licensed to AZ, which optimized the drug with half-life extension.

The medicine aims to prevent Covid-19 infection in immunocompromised people.

According to AZ, the half-life extension more than triples the durability of its action compared to conventional antibodies. In addition, the company said that the consecutive two doses of tixagevimab 150mg and cilgavimab 150 mg could afford up to 12 months of protection from Covid-19.

On Dec. 8, Evusheld received FDA approval for the pre-exposure prophylaxis of Covid-19 in people who experienced serious adverse reactions of Covid-19 vaccines or those with immune compromise. Earlier, the U.S. government signed a deal with AZ to purchase 700,000 doses of Evusheld.

The U.S. has confirmed the purchase of 1.2 million doses of Evusheld so far, and the additional 500,000 doses will be shipped to the U.S. within the first half of this year.

As the U.S. secured most of the initial supply of Evusheld, other countries will face a limited chance to purchase the preventive therapy.

Our headquarters basic stance is that we will apply for marketing license in countries that confirmed the pre-purchase of Evusheld, an official at AstraZeneca Korea said. As the quantity of Evusheld is insufficient, we are distributing the remaining stock of Evusheld, excluding those going to the U.S., to several developed countries in Europe.

This means that if the Korean government does not discuss a pre-purchase of Evusheld, AZ will not seek marketing approval pre-emptively.

The Ministry of Food and Drug Safety is doing a preliminary review on Covid-19 vaccines and treatments. But this happens only when a company submits related data.

The regulator is not reviewing Evusheld now.

However, the Korea Disease Control and Prevention Agency (KDCA) plans to discuss Evusheld purchase with AZ soon. The drug is expected to become an option for Korean immunocompromised people to prevent Covid-19.

View original post here:
US will buy additional doses of Covid-19 preventive drug Evusheld. What about Korea? - Korea Biomedical Review

Recommendation and review posted by Bethany Smith

Rory Moore (pictured) has worked at Ekati, the first diamond mine in Canadas Northwest Territories, on and off for years. He was part of the original crew that first developed the mine in the 1990s. In 2018, he rejoined what was then called Dominion Diamond. He then became its interim president in January 2020, and in November, he was appointed the full-time CEO of what is now called the Arctic Canadian Diamond Co.

Here, he talks with JCK about his view on the diamond market, how Arctic looks at Canadian cutting and Canadian-origin stones, and why Ekati might confound expectations and stay around for a while.

Can you bring us up to date on whats happening with Ekati and its current ownership structure?

With the onset of COVID, the diamond market closed. We had a partnership with Rio Tinto in the Diavik mine, which is a neighboring mine. After COVID, we [initially] elected to put Ekati on care and maintenance, because we had $200 million worth of diamonds in inventory, but we couldnt sell them. We didnt want to burn through our cash reserves quickly. However, when Diavik did continue to operate, we were obliged to pay our 40% interest in the operating costs, and that would have very quickly have chewed up our cash reserves. So, we [filed] for CCAA [insolvency protection].

That triggered its sale process and the former owners, Washington, participated in that and ultimately withdrew their bid to buy the assets, and then the debt holders stepped in, represented by the three principal organizations: DDJ Capital, Brigade Capital, and Western Asset Management. The three of them stepped in and bought the assets and [provided us] with some additional cash to reopen the mine and get going again.

We restarted the mine in January last year, which is in the dead of the Arctic winter, so that was challenging. We had lots of startup problems, but ultimately now we have got the mine running at a steady state. Fortunately, we had some nice tailwinds from a strong rough diamond market last year, so prices were very robust and that really helped us with our restart. We met our revenue targets last year despite all the challenges.

You have said you want to extend the mine life. How do you plan to do that?

The best days of Ekati are behind us in terms of quality of ore bodies. During the early years, when BHP owned the mine, the ore bodies were very rich and very close to the central infrastructure at the mine, which is the process plant. So operating costs were very low, and revenue was very high. It was a highly profitable mine.

As time has gone on, those bodies have been mined out, and we are now mining lower-value ore bodies further away from the plant. Our main open pit, Sable, is 17 kilometers north of the process plant. Its a lower-revenue ore body and theres additional transportation and logistics involved in getting the ore from the open pit to the process plant. Then, to the south, weve got our Misery underground mine, which is a high-revenue ore body that has now gone underground, so its mining at a lower production rate. And thats 25 kilometers south of the processing plant, so again, weve got to transport the ore to the plant.

What we faced going forward is, as these ore bodies come to their natural end, Stable in particular, they were not valuable enough to support conventional underground mining. And, so, what weve done is over the past four yearsand this is what Ive been working onis looking at alternative, more creative ways to mine these ore bodies so we can still mine them profitably.

Weve come up with a system, its called underwater remote mining. Essentially, we flood the pit, and after the open pit mining is completed, and we continue to mine it underwater, using specialized underwater surface mining machines that weve developed in conjunction with a company out of Holland called Royal IHC. We are very confident that its going to work, and the beauty of this method is that you only mine ore and not waste. So, with conventional mining, you have to mine a lot of waste. In fact, at Sable, theres seven times more waste [being mined] than ore. If you remove all that waste mining, suddenly your costs are dramatically lower, and youve got a profitable mining operation again. Thats the way we are going into the future.

We have designed all the equipment, and we are going to construct the first of that equipment this year. Then, were going to do a big production trial in 2024. Were building equipment this year, and then over 2023 and 2024, were gonna test that equipment and then implement it in 2024 for future mining at Sable, and then extend that to other ore bodies. The future of Ekati is underwater remote mining, which has not been done in this sort of setting at all in the past. We are spearheading some new innovative mining techniques.

Assuming that all comes to fruition, how long will that extend the mine life?

Initially, the first phase will take us to 2029. The other thing that were doing at the moment is developing a new conventional open-pit mine at the same time. That will take us to 2028. We also have the opportunity to extend mine life through underwater remote mining at Point Lake. That ore body has 50 million tons of kimberlite in it. So, if the underwater remote mining works, well get an additional 10 years of mining out of Point Lake. Then, we have another ore body that was mined by BHP called Fox, and that has 40 million tons of kimberlite left in it. Weve done studies to potentially develop it with underground conventional mining, and it doesnt make the grade. So, we would look to mine that underwater as well, which would add another 10 years of mine life. So, essentially, we have decades of mine life extension, but we have to prove the system first.

You said youre confident that will work.

Yes. Im very confident. Components of the technology have been routinely used in other applications, in conventional dredging operations throughout the world. We are putting it all together in a system that hasnt been used in this application. None of it is rocket science. Its all proven technology.

The owners have been very supportive, and they have been very open to funding our development of [the underwater remote mining] method. Weve got a stable operation now, and our immediate focus is to pay down the debt that we have, and weve already started that process this year. We had a good revenue year, and weve already paid down the debt very substantially.

There have been concerns raised about underwater mining from an environmental point of view. How do you respond to that?

In fact, this method is significantly more environmentally friendly than open-pit mining. Thats because, firstly, you dont have any big waste dumps. So, all that footprint of the waste dumps interfering with caribou migration or changing the landscape, that goes away. You have a closed water system, so all the water thats in the mine [goes to] a pond next door to the open-pit mine, where well settle out the sedimentary material. It will be a closed system. So, no water thats been disturbed or polluted in any way will get out into the secondary environment. Its very much contained and has an incredibly small footprint.

The machine has been devised so that all the hydraulic fluids in the machine are biodegradable. Id be lying to you if I said were not going to burst hydraulic hoses once in a while. We will have hydraulic oil spills, but [they will be] biodegradable hydraulic oils by design that will not contaminate the water in any meaningful way. So, the environmental impact is much lower, and thats a positive for the project as well.

What has been the reaction from the local community to Ekati restarting?

Our northern partners are very relieved that we are back in production. We have a lot of training and career opportunities for people of the north. There was big relief that we did come through the CCAA process, that weve hired a whole lot of new people, and our training programs, apprenticeship programs, leadership programs are back in place. Weve got partnerships with the local indigenous groups and communities. Theyve supported us very well, as has the government of Northwest Territories. Its all pretty positive, after a tough period of 10 months not being in production

You no longer have 40% ownership in the Diavik mine, as Rio Tinto took it over. How does that affect you?

On a practical basis, it doesnt impact us much, because we were essentially a passive partner in that. Rio Tinto operated that mine and we just participated in quarterly management committee meetings. It doesnt impact our workforce in any way, as we had nobody working at Diavik.

What it does impact is the product mix that we take to the market. We used to have a big production out of Ekati, supplemented by a fairly significant production out of Diavik, which gave us higher carats and higher volumes to sell into the rough market and gave us more options for product mix for our clients. Weve lost a bit of flexibility in accommodating our clients exact needs when it comes to the product mixes.

But the productions are fairly similar between Ekati and Diavik. So, the impact of that hasnt been too significant. Its more that weve just got a lower volume of diamonds to sell.

Your company has been renamed Arctic, which is probably a more potent name from a marketing point of view, in that it symbolizes Canada. Do you expect to get more involved in marketing your product?

We recognize that we are diamond producers, and thats what weve got to focus on. We are an active member of the Natural Diamond Council, and so we actively participate in that and contribute to that initiative through our membership fees. We think that the Natural Diamond Council is doing a great job of promoting natural diamonds. We recognize that if you wanted to do a good job of marketing a luxury product like diamonds, youve got to have a big budget for that, and thats just not where we fit in. All of us producers together are much more powerful than individuals trying to do that separately.

A lot of producers are looking into origin certification? Will you do that?

A percentage of our diamonds are sold through the Canadamark program. They tend to be the higher-quality goods. We think its an important consideration, given that the new generations of customers are much more concerned about the origin of the products that they buy and that the products have been produced a sustainable way. I think Canadian-origin diamonds are highly prized by millennials because they know that Canada has high environmental guidelines and social economic guidelines and the way we run our mines in Canada is of the highest standard. Thats not to say that a diamond produced in Africa has not been ethically produced, because the big mines in Africa are run according to the highest international standards. There may be a perception that all these African [stones] are still blood diamonds, but the reality is that the absolute majority of diamonds coming out of Africa are produced in a very ethical way. But Canada does have brand appeal.

Theres been talk of new factories in Canada. Thats been tried before, and it didnt work. Do you think Canadian cutting has a future?

I think its always going to be a challenge to manufacture diamonds in Canada, particularly in the north. You are competing against India, and India has really done an incredible job of capturing that part of the supply chain of diamonds. You cant compete on costs, and they actually have become incredibly skilled at polishing diamonds.

Its admirable to see people try, because the more we can capture business opportunities in Canada, the better. But I think its a struggle.

Do you expect rough diamond prices to continue to remain strong?

At the moment, everybody in the business expects that rough diamond prices will remain strong in the short- to medium-term. Obviously, its very difficult to predict longer term. Supply has been impacted by COVID, so supply of rough diamonds is lower than it was forecast to be. Demand is incredibly strong, fueled mostly by the U.S. and China. Theres been very strong demand for our product, and the commentary over the holiday season was that sales were very strong on the retail side. In the short-term, I think the macro factors for our industry, and for rough diamond prices, are very good.

(Photos courtesy of Arctic Canadian Diamond Co.)

Read more:
Interview With Rory Moore, CEO Of Arctic Canadian Diamond Co. - JCK

Recommendation and review posted by Bethany Smith

Isoflavones Market Research Report by Product (Capsule, Liquid, and Powder), by Source (Chickpea, Red Clover, and Soybeans), by Component, by Application, by Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2026 - Cumulative Impact of COVID-19

New York, Jan. 13, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Isoflavones Market Research Report by Product, by Source, by Component, by Application, by Region - Global Forecast to 2026 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06202161/?utm_source=GNW

The Global Isoflavones Market size was estimated at USD 1,333.21 million in 2020 and expected to reach USD 1,412.48 million in 2021, at a CAGR 6.05% to reach USD 1,896.77 million by 2026.

Market Statistics:The report provides market sizing and forecast across five major currencies - USD, EUR GBP, JPY, and AUD. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2019 are considered historical years, 2020 as the base year, 2021 as the estimated year, and years from 2022 to 2026 are considered the forecast period.

Market Segmentation & Coverage:This research report categorizes the Isoflavones to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Product, the market was studied across Capsule, Liquid, Powder, and Tablet.

Based on Source, the market was studied across Chickpea, Red Clover, and Soybeans.

Based on Component, the market was studied across Daidzein, Formononetin, and Genistein.

Based on Application, the market was studied across Cosmetics & Personal Care, Food & Beverages, and Nutraceutical.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, and Thailand. The Europe, Middle East & Africa is further studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Isoflavones Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Isoflavones Market, including Archer Daniels Midland Company, BASF SE, Bio-Gen Extracts Pvt. Ltd., Cargill, Incorporated, Fujicco Co., Ltd., Futureceuticals Inc., GNC Holdings Inc., International Flavors & Fragrances, Inc., InVite Health, Inc., Koninklijke DSM N.V., Lactonova Nutripharm Pvt Ltd, Life Extension Foundation, Nexira Inc., NOW Foods, Nutra Green Biotechnology Co. Ltd., NutraScience Labs, Shaanxi Hongda Phytochemistry Co., Ltd., Shanghai Freemen, SK Bioland, and Xena Bio Herbals Pvt Ltd..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Isoflavones Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Isoflavones Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Isoflavones Market?4. What is the competitive strategic window for opportunities in the Global Isoflavones Market?5. What are the technology trends and regulatory frameworks in the Global Isoflavones Market?6. What is the market share of the leading vendors in the Global Isoflavones Market?7. What modes and strategic moves are considered suitable for entering the Global Isoflavones Market?Read the full report: https://www.reportlinker.com/p06202161/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Story continues

See the rest here:
Isoflavones Market Research Report by Product, by Source, by Component, by Application, by Region - Global Forecast to 2026 - Cumulative Impact of...

Recommendation and review posted by Bethany Smith

Although hot flashes are the quintessential symptom, there are several other menopause-related symptoms, such as brain fog or memory trouble, that women may not realize are connected to their changing hormones.

A new study published January 12 in the journalMenopause found a link between menopausal symptoms, especially depression and sexual dysfunction, and cognitive performance in areas such as memory, attention, and language skills.

Often women dont recognize these symptoms as part of menopause, says Margaret Nachtigall, MD, a gynecologist at NYU Langone Health and a clinical assistant professor at the NYU School of Medicine, both in New York City. Because cognitive issues can develop slowly, they might not see it as related to a loss of estrogen, she says.

According to theOffice on Womens Health, part of the Department of Health and Human Services, up to two-thirds of women in perimenopause report cognitive problems.

In the women that I see in clinic, that can manifest as difficulty remembering, the inability to find the right word for what they are trying to say, putting dishes in the cabinet instead of the dishwasher, not remembering everything on their list that they wanted at the store, or being in a meeting and forgetting what they were going to say, says Dr. Nachtigall.

RELATED: Predicting How Long the Menopausal Transition Will Last, and When Youll Reach Menopause: 10 Questions and Answers

To examine whether the cognitive performance of women is related to the severity of menopausal symptoms, investigators looked at 404 women in a rural area in north India. The subjects were between ages 40 and 65 and were not using hormone therapy (HT).

The women were divided in four groups so that researchers could look at the frequency and severity of different symptoms according to the stage in the menopause transition.

Researchers used standardized tools to record the presence and intensity of 21 different menopause symptoms, including sexual dysfunction, vasomotor symptoms (hot flashes), depression, and anxiety.

A Hindi version of the Mini-Mental State Examination (MMSE) was used to gauge cognitive performance and evaluate the effect of the severity of menopause symptoms on overall cognitive performance and its five domains, including

Investigators found that the kind of menopausal symptoms the women experienced were different depending on their life stage.

Researchers found that the cognitive performance of the women was linked to the severity of certain menopause symptoms, especially depression and sexual dysfunction.

Sexual dysfunction can manifest in different ways during menopause and people are often reluctant to talk about it, says Nachtigall. Some women feel as though they have vaginal dryness, itchiness, or a feeling like sandpaper in the vagina, she says.

A decrease in arousal or in sexual response and pleasure are also symptoms of sexual dysfunction, according to theNorth American Menopause Society.

The authors found no association between the severity of vasomotor symptoms and cognitive performance, although other studies have suggested that such an association exists.

In menopause, the ovaries arent producing any estrogen, notes Nachtigall. We have estrogen receptors almost everywhere brain, eyes, teeth, bones and so its not surprising that when we have a lack of estrogen that all these different symptoms can occur. It makes sense that a person with more menopause symptoms and more severe symptoms would also have a lower cognitive function, but interesting that they were able to show that in this study, she says.

These findings reflect what Nachtigall sees in her own practice. Often, people who have more hot flashes, more depression, and more symptoms of menopause in general are also the same people who experience difficulties with cognitive functioning.

This study provides further support that women should seek care when you are going through menopause or perimenopause, says Nachtigall. When you see a provider, talk about the symptoms you are experiencing, even if you dont think they are related to menopause, she says.

This can lead to an important discussion around different treatment options, including whether or not hormone therapy (HT) may help with your symptoms, says Nachtigall. Its been shown in multiple studies that estrogen does improve symptoms of cognitive functioning. Its also been shown in many studies that estrogen will reverse hot flashes, will improve mood, will improve sexual functioning and vaginal dryness, and therefore may be a good option for many people, she says.

Research published in Menopause in December 2019 found that there could be a cognitive benefit later in life for some women who had more exposure to estrogen, both through a longer reproductive window and the use of longer-term HT.

The risks and benefits of hormone therapy should be discussed with your doctor, as it is not appropriate for everyone, including women who have unexplained bleeding, estrogen receptor positive cancer or severe liver disease or a serious clotting disorder, says Nachtigall.

The findings of this study point to the fact that not all symptoms related to the menopause transition are hot flashes; there are other symptoms, says Stephanie S. Faubion, MD, the director of the Center for Womens Health at the Mayo Clinic in Rochester, Minnesota, and the medical director of theNorth American Menopause Society (NAMS).

Included in that are these cognitive type changes that women commonly describe, as well as mood issues that need to be addressed, says Dr. Faubion. That doesnt mean that all these symptoms can be treated with hormone therapy there could be other issues or conditions contributing to these symptoms that need to be addressed and evaluated, she adds.

RELATED: What Experts Want Women of Color to Know About Menopause

Its important to note that we dont know if giving hormone therapy during menopause helps with memory, short or long term. But we do know if youre going to take hormone therapy for other menopause symptoms hot flashes, night sweats it does tend to help with mood, she says. The relationship between depression and memory issues is well-established, adds Faubion.

If not through hormone therapy, is there a way to treat brain fog or memory issues? Solutions to memory issues might be related to sleep issues or an underlying sleep disorder. It may also be improved by managing stress or treating any underlying mood disorders, such as anxiety or depression, says Faubion.

Nachtigall echoes that sleep is an important issue and often a problem for women during the menopause transition. Many people that I see complain of insomnia, and thats one of the early signs of lack of estrogen that patients in menopause will experience, she says.

View original post here:
Menopausal Symptoms and Cognitive Problems Connected, Study Says - Everyday Health

Recommendation and review posted by Bethany Smith

Even 10-step skin care routine devotees get pimples. If youre suffering from red, irritated acne blemishes, there are ways you can minimize redness through skin care efforts.

However, if youve arrived to this page with little time to spare, there are also some tips for concealing blemishes until you can get the redness to go away for good.

The keys to reducing redness from acne are to apply anti-inflammatory compounds while being gentle to your skin. Thats right gentle. This means dont try to use every scrub or irritating product to treat your acne. Instead, try using a keep it simple approach.

If you apply too many oil-removing products, your skin could react the opposite way: It could start to overproduce oil, which will worsen your acne. The goal is to keep your skin clean and apply targeted products to reduce redness while your blemishes heal.

Here are some tips for reducing redness:

Sometimes, youll need to call in the professionals for consistent, painful, and inflamed pimples. A dermatologist can evaluate your skin and prescribe professional-level treatments, such as topical applications, chemical peels, or laser therapy.

Acne can also closely resemble rosacea, a condition that causes significant skin redness. Because the treatments for rosacea can be different from traditional acne-fighting approaches, its a good idea to see a dermatologist if youre not sure.

You can also apply a mild cortisone cream to an inflamed pimple to decrease redness and swelling. This should be done sparingly and only as a spot treatment, as topical steroids themselves can actually cause acne, as well other potential side effects such as skin thinning.

Sometimes, even your best efforts at reducing redness dont show enough immediate results. When this is the case, you can use makeup to cover up the redness. Heres how to start:

Youll want to use products that are all oil-free and noncomedogenic when concealing your pimple. Otherwise, youll run the risk of making it worse.

Consistent skin care is beneficial for reducing redness, and so is adopting an acne prevention plan. Your acne prevention skin care routine should include the following:

If this plan doesnt help your acne subside, see a dermatologist.

Acne redness can indicate underlying irritation and inflammation in your skin. A lot of this can be hormone-related, as hormones can determine how much oil your skin produces. However, you can use a consistent skin care routine with topical agents intended to reduce blemishes.

Keeping redness fighters (plus a good concealer) on hand isnt a bad idea for when the occasional blemish pops up. But remember to call your dermatologist if your blemish wont go away after a few days of at-home care.

Link:
Quick and Easy Ways to Get Rid of Redness from Pimples and Acne - Healthline

Recommendation and review posted by Bethany Smith

Results of the search

We retrieved 139 records from the search of the electronic database and no other records from other sources (Fig.1). A total of 94 records were screened after duplicates were removed. We reviewed full copies of 13 and assessed them for eligibility. We identified four articles as possibly meeting the review inclusion criteria, and nine of them were ineligible for inclusion. One article was a non-randomized controlled trial that evaluated the clinical response of elagolix-treated women who did not achieve the primary outcome [22]. Two reviews, one on predictors of response to elagolix with add-back therapy and the other on medical treatment of uterine leiomyoma, were relevant to our research query [4, 23]. There was no outcome of interest in the four papers as two papers [3, 11] on elagolix pharmacotherapy and pharmacodynamics and another two more papers [14, 24] on drug-drug interactions were written. Adenomyosis was the topic of two more publications [25, 26]. We attempted to contact the trial authors for the full article but received no response. Therefore, we included four trials.

Four randomized controlled trials with 1949 participants were included in the study [27,28,29,30]. All four trials reported the primary outcome. All trials were sponsored by AbbVie [27,28,29,30].

All four studies were carried out in 323 locations across the United States, Puerto Rico, and Canada. One trial recruited participants from clinic settings [27]. The other three trials did not mention the location from which participants were recruited [28,29,30]. Three studies included premenopausal women aged 18 to 51 at the screening time [28,29,30], while one study recruited participants aged 2049 [27]. They underwent ultrasonography-confirmed diagnosis of uterine fibroids and heavy menstrual bleeding, as characterized by more than 80ml of menstrual blood loss per menstrual cycle for at least two cycles. The trials excluded participants due to a complex ovarian cyst, cancer, pelvic inflammatory disorder, osteoporosis history, or metabolic bone disease. Participants who had a myomectomy or hysterectomy for symptomatic uterine fibroid were exempted from the study [27,28,29,30].

Participants in the trials were randomized to the intervention and comparison groups. Two identical, double-blind, randomized, placebo-controlled, six-month phase 3 trials (Elaris Uterine Fibroids 1 and Elaris Uterine Fibroid 2) have been reported in one trial [29]. Elaris Uterine Fibroid-1 and Elaris Uterine Fibroid-2 participants were later randomized or pooled into a new study [30] to look at the long-term twelve-month safety and efficacy of elagolix with or without estradiol/norethindrone acetate. The meta-analysis included four trials that evaluated the primary outcomes. Three trials compared elagolix with placebo [27,28,29], and four trials compared to elagolix with estradiol/norethindrone acetate [27,28,29,30]. Only one trial compared elagolix to placebo at different doses of 100mg bd, 200mg bd, 300mg bd, 400mg qd, and 600mg qd [27]. One study was compared to placebo at doses of 300mg bd and 600mg qd [28]. Another trial was compared elagolix to placebo at a dose of 300mg bd [29].

In a comparison of elagolix to elagolix with estradiol/norethindrone acetate, one trial compared it at a dose of 0.5mg estradiol/0.1mg norethindrone acetate [27], while two trials compared it at a dose of 1.0mg estradiol/0.5mg norethindrone acetate [29, 30]. In one trial, elagolix was compared to elagolix with estradiol/norethindrone acetate at two doses: 0.5mg estradiol/0.1mg norethindrone acetate and 1.0mg estradiol/0.5mg norethindrone acetate [28]. All medications are taken orally as tablets or capsules. The duration of treatment differed between trials compared to elagolix versus placebo, as only one trial was three months [27], and the other two trials were six months [28, 29]. In contrast, the length of treatment differed between trials when comparing elagolix to elagolix with estradiol/norethindrone acetate, with a three-month [27], a six-month [28, 29], and a twelve-month [30] period.

The validated alkaline hematin method was used to quantify and evaluate the primary outcome in all four trials [27,28,29,30]. Any spotting or bleeding episodes on a sanitary pad were reported at the time of screening or during treatment. Participants kept an electronic daily bleeding diary (eDiary) and assessed bleeding patterns using the validated Mansfield-Voda-Jorgenson Menstrual Bleeding Scale [31]. All studies were followed up to at least three-months duration. The primary outcome was measured during the last month of the treatment period.

All four trials reported all secondary outcomes except for one study [27], which did not record bone mineral density due to a limited study time and a small sample size per group. Reduction of uterine and fibroid volume was calculated using trans abdominal or transvaginal ultrasound. The mean percentage change from baseline to the end of the treatment month was recorded.

The Uterine Fibroid Symptom and Quality of Life questionnaires cumulative score were used to measure symptom severity reduction and change in health-related quality of life in women with symptomatic uterine fibroids. It was a disease-specific, self-administered, validated questionnaire. There were 37 questions in all, split into two parts. The first part consisted of an 8-item symptom severity scale. The second part consisted of a 29-item health-related quality of life subscale with six domains (concern, behaviors, energy/mood, power, self-consciousness, and sexual function). All items are rated on a 5-point scale, with symptom intensity items ranging from not at all to a great deal, and health-related quality of life items ranging from none of the time to all of the time. The cumulative score for each of the two components was determined by adding the symptom intensity and health-related quality of life subscale scores and translating them to a 0-to-100-point scale. Higher overall health-related quality of life scores indicated better quality of life, while lower symptom severity scores indicate better quality of life.

The percentage of increase in hemoglobin concentration from baseline to the last month of treatment was reported in all trials. Loss of bone mineral density was assessed by dual-energy x-ray absorptiometry scans of the lumbar spine, total hip, and femoral neck. The mean percentage change in bone mineral density from baseline to the last month of treatment was recorded in three studies [28,29,30]. Any adverse events were recorded beginning with the first dose of the study drug and continuing for up to 30days after completing the last dose of the study drug. All four trials identified common adverse events such as hot flushes, headaches, nausea, and fatigue. In this review, only two trials documented adverse events such as abdominal pain, dizziness, and hypertension [27, 28]. Other non-significant adverse events identified in clinical trials will not be addressed in this review.

The assessment risk of bias is shown in Figs.2 and 3. Figure2 shows the proportion of studies assessed as low, high or unclear risk of bias for each risk of bias indicator. Figure3 shows the risk of bias indicators for individual studies. The details of these trials are found in the table of characteristics of included studies (Table 1).

Risk of bias graph: review authors judgements about each risk of bias item presented as percentages across all included studies

Risk of bias summary: review authors judgements about each risk of bias item for each included study

Only one trial, with 271 participants, was reported to have been recruited in a clinic setting, while the other three were not [27]. The method of randomization was not reported in all four trials [27,28,29,30]. Thus, we judged random sequence generation as having an unclear risk of bias. Allocation concealment was not mentioned and regarded as unclear in four trials [27,28,29,30].

Participants, care provider, investigator and outcome assessor were masked in all four trials. The details on blinding were not recorded in all four trials, but the outcomes were unlikely to be influenced as it was objectively collected and measured using standardized methods [27,28,29,30]. Therefore, they are judged as having a low risk of bias.

More than 80% of participants completed the studies in two trials [27, 30]. Meanwhile, 74.4% of participants in one trial completed the study [28]. Approximately 129 of the 571 participants failing to complete the analysis due to hypoestrogenism side effects (n=39), withdrawal (n=38), loss of follow up (n=25), noncompliance (n=11), lack of efficacy (n=3), surgery (n=4) and other (n=9) [28]. About 78% of 791 participants completed studies in Elaris Uterine Fibroid-1 and Elaris Uterine Fibroid-2 [29]. The study drug was discontinued by similar proportions of women in both treatment groups (16.5% for elagolix with estradiol/norethindrone acetateand 19.4% for elagolix alone), with the most common primary reason being lost to follow-up (5.0%and 5.1%,respectively) in one trial [29]. Missing data were evenly balanced across groups, and the reasons were similar. The most common reasons for missing outcome data included withdrawal, noncompliance, loss to follow up, hypoestrogenism side effects, pregnancy, and surgery, which led to discontinuation.

All four trials reported the outcomes as specified in their methods section[27,28,29,30]. The outcomes listed in the registered protocol were those reported. Although changes in bone mineral density were assessed as an exploratory parameter, one trial did not report due to the short duration of the study and the relatively small sample size per group [27]. We graded it as having a low risk of bias.

We discovered that women with asymptomatic anemia and a hemoglobin level of less than 12g/dl at screening or during the study period were advised to take iron supplements in two trials [27, 30]. This could have an influence on the hemoglobin level at the end of the treatment period. Thus, we judged it as having a high risk of bias. We detected no other potential source of bias in the other two trials [28, 29].

There would be two comparisons evaluated in this review, i.e., comparing elagolix versus placebo and comparing elagolix versus estradiol/norethindrone acetate.

Elagolix has increased the number of patients with a reduction of menstrual blood loss of less than 80ml (RR 4.81, 95% CI 2.45 to 9.45; I2 statistic=89%; P<0.001; four trials, 869 participants; moderate quality evidence) (Fig.4, Table 2) [27,28,29] or more than 50% from baseline (RR 4.87, 95% CI 2.55 to 9.31; I2 statistic=87%; P<0.001; four trials, 869 participants; moderate quality evidence) (Fig.5, Table 2) [27,28,29] compared to placebo. The sensitivity analysis did not change the cumulative effect estimate. Table 3 showed the subgroup analysis for reduction of menstrual blood loss of less than 80ml or more than 50% reduction from baseline stratified by frequency of drug administration, uterine and fibroid volume (Additional file 1).

Comparison between elagolix and placebo for the outcome reduction of menstrual blood loss of less than 80ml

Comparison between elagolix and placebo for the outcome reduction of menstrual blood loss of more than 50%

For the secondary outcomes, elagolix has increased the number of patients with improved hemoglobin level (RR 2.46, 95% CI 1.93 to 3.13; I2 statistic=0%; P<0.001; four trials, 554 participants; moderate quality evidence) [27,28,29], reduced the mean percentage change in uterine volume (MD 34.50, 95% CI 43.48 to 25.53; I2 statistic=63%; P<0.001; four trials, 783 participants; moderate quality evidence) [27,28,29], fibroid volume (MD 31.39, 95% CI 44.69 to 18.09; I2 statistic=65%; P<0.001; four trials, 750 participants; moderate quality evidence) [27,28,29], severity of symptoms (MD 31.54, 95% CI 41.85 to 21.22; I2 statistic=96%; P<0.001; four trials, 814 participants; low quality evidence) [27,28,29], and improved health-related quality of life (MD 30.64, 95% CI 20.14 to 41.15; I2 statistic=95%; P<0.001; four trials, 812 participants; low quality evidence) [27,28,29] (Additional file 1, Table 2) compared to placebo.

Elagolix has reduced bone mineral density in lumbar spine (MD 2.82, 95% CI 3.30 to 2.35; I2 statistic=0%; P<0.001; three trials, 574 participants; moderate quality evidence) [28, 29], total hip (MD 1.97, 95% CI 2.37 to 1.57; I2 statistic=46%; P<0.001; three trials, 574 participants; moderate quality evidence) [28, 29] and femoral neck (MD 1.92, 95% CI 2.61 to 1.23; I2 statistic=34%; P<0.001; three trials, 574 participants; moderate quality evidence) [28, 29] (Fig.6, Table 2) compared to placebo.

Comparison between elagolix and placebo for the outcome of bone mineral density (A: lumbar spine, B: total hip, C: femoral neck)

There was no significant of severe, serious or adverse event led to discontinuation of elagolix treatment. Elagolix has increased the number of patients with side effect of hot flush (RR 7.47, 95% CI 4.99 to 11.18; I2 statistic=8%; P<0.001; four trials, 890 participants; moderate quality evidence) [27,28,29] and headache (RR 1.88, 95% CI 1.25 to 2.83; I2 statistic=0%; P<0.001; four trials, 890 participants; low quality evidence) [27,28,29] (Fig.7, Table 4) compared to placebo.

Comparison between elagolix and placebo for the outcome of adverse events (A: hot flush, B: headache)

B) Comparison between elagolix and elagolix with estradiol/norethindrone acetate.

There was no difference in menstrual blood loss of less than 80ml (RR 1.08, 95% CI 1.00 to 1.16; I2 statistic=56%; P=0.070; five trials, 1365 participants; moderate quality evidence) (Fig.8, Table 5) [27,28,29,30] or more than 50% reduction from baseline between the elagolix (RR 1.08, 95% CI 1.01 to 1.15; I2 statistic=43%; P=0.020; five trials, 1365 participants; high quality evidence) (Fig.9, Table 5) [27,28,29,30] and elagolix with estradiol/norethindrone acetate. The sensitivity analysis did not change the cumulative effect estimate. Table 6 showed the subgroup analysis for reduction of menstrual blood loss of less than 80ml or more than 50% reduction from baseline stratified by dosage and uterine volume (Additional file 1).

Comparison between elagolix and elagolix with estradiol/norethindrone acetate for the outcome reduction of menstrual blood loss of less than 80ml

Comparison between elagolix and elagolix with estradiol/norethindrone acetate for the outcome reduction of more than 50% menstrual blood loss

Foe secondary outcomes, there was no difference improvement in hemoglobin level between elagolix (RR 0.99, 95% CI 0.80 to 1.22; I2 statistic=68%; P=0.930; five trials, 899 participants; low quality evidence) [27,28,29,30] and elagolix with estradiol/norethindrone acetate. However, elagolix has reduced mean percentage change in uterine volume (MD 17.47, 95% CI 27.54 to 7.40; I2 statistic=58%; P<0.001; five trials, 1250 participants; moderate quality evidence) [27,28,29,30], fibroid volume (MD 23.18, 95% CI 28.98 to 17.38; I2 statistic=0%; P<0.001; five trials, 1208 participants; high quality evidence) [27,28,29,30], symptoms severity (MD 9.05, 95% CI 9.68 to 8.43; I2 statistic=0%; P<0.001; five trials, 1288 participants; high quality evidence) [27,28,29,30], and increased health-related quality of life (MD 9.94, 95% CI 5.82 to 14.06; I2 statistic=76%; P<0.001; five trials, 1287 participants; low quality evidence) [27,28,29,30] (Additional file 1, Table 5) compared to elagolix with estradiol/norethindrone acetate.

Elagolix has reduced bone mineral density in the lumbar spine (MD 2.63, 95% CI 3.12 to 2.14; I2 statistic=49%; P<0.001; four trials, 1126 participants; moderate quality evidence [28,29,30], and total hip (MD 1.93, 95% CI 2.56 to 1.31; I2 statistic=75%; P<0.001; four trials, 1126 participants; very low quality evidence) [28,29,30] except femoral neck (MD 0.77, 95% CI 1.84 to 0.30; I2 statistic=78%; P=0.160; four trials, 1126 participants; very low quality evidence) [28,29,30] (Fig.10, Table 5) compared to elagolix with estradiol/norethindrone acetate.

Comparison between elagolix and elagolix with estradiol/norethindrone acetate for the outcome of bone mineral density (A: lumbar spine, B: total hip, C: femoral neck)

There was no difference of severe, serious or adverse event led to discontinuation between elagolix treatment and elagolix with estradiol/norethindrone acetate. Elagolix has increased the number of patients with side effect of hot flush (RR 2.67, 95% CI 2.30 to 3.10; I2 statistic=0%; P<0.001; five trials, 1403 participants; moderate quality evidence) [27,28,29,30], reduced the number of patients with risk of nausea (RR 0.63, 95% CI 0.43 to 0.91; I2 statistic=0%; P=0.010; five trials, 1403 participants; low quality evidence) [27,28,29,30] and fatigue (RR 0.43, 95% CI 0.23 to 0.80; I2 statistic=0%; P=0.008; five trials, 1403 participants; low quality evidence) [27,28,29,30] (Fig.11, Table 7) compared to elagolix with estradiol/norethindrone acetate.

Comparison between elagolix and elagolix with estradiol/norethidrone acetate for the outcome adverse event (A: hot flush, B: nausea, C: fatigue)

Read the original:
Elagolix treatment in women with heavy menstrual bleeding associated with uterine fibroid: a systematic review and meta-analysis - BMC Women's Health...

Recommendation and review posted by Bethany Smith

Excess weight gain is never great news. But one form of weight gain is more dangerous than others. Putting on belly fatalso known as visceral fat or abdominal fatis a major hazard to your health. That's because this type of fat lies deep within the abdomen, near vital organs like the liver, pancreas, and intestines. It's metabolically active, meaning it actively releases toxic substances into those organs and bloodstream that raise the risk of cancer, heart disease, and diabetes. The good news: If you've packed on abdominal fat, you can take specific steps to burn it off. Here's what science says are some of the most effective ways to reverse that potentially deadly weight gain. Read on to find out moreand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had COVID.

The easiest way to reduce visceral fat is to lose weight. According to W. Scott Butsch, MD, an obesity medicine specialist with the Cleveland Clinic, weight loss alone can effectively reduce visceral fat. And just sending the scale slightly in the right direction can have a major effect on deadly belly fat gain. By losing 10% of your body weight, you may lose up to 30% of your body fat, he says.

Visceral fat lives for sugar. "Fructose, or sugar, causes fat cells to mature faster, specifically in the visceral fat," says the Cleveland Clinic. Reduce the amount of sugar in your dietlike sugar-sweetened drinks, simple carbs, baked goods, processed foods, and fast foodsand you'll likely see your waistline shrink.

RELATED: I'm a Doctor and Here's How to Lose Visceral Fat

Researchers at Wake Forest University found that dieters who slept five hours or less every night put on 2.5 times more belly fat than people who got an adequate amount of sleep. Not sleeping enough can increase the production of cortisol, a stress hormone that tells the body to hold onto fat around the abdomen. Poor sleep alters the production of leptin and ghrelin, two hormones that regulate appetite, and that can increase feelings of hunger. And just plain being tired can cause you to overeat to try and boost your energy. How much sleep is ideal? Experts say seven to nine hours a night.

RELATED: The #1 Place to Not Walk Into Now, Say Virus Experts

According to a 2020 study published in the journal Nutrients, exercise reduces visceral fat even if you don't lose weight. That's because it lowers circulating insulin (which tells the body to hang on to fat) and tells the liver to burn nearby belly fat deposits. The best kind of exercise for belly-fat reduction is moderate-intensity activity combined with strength training, a 2021 review of studies found.

RELATED: Diabetes Warning Signs You Need to Know, Say Experts

Reducing stress can provide a one-two punch in the battle of the bulge. Chronic feelings of stress cause the brain to produce more cortisol, which makes belly fat hang around. Stressing out can also lead to comfort-eating fatty and sugary foods. The combination is a shortcut to belly fat, says a study published in The Annals of the New York Academy of Sciences.

RELATED: 7 Reasons to Use Marijuana, Say Doctors

Several studies have found that a high-protein diet can burn belly fat and help keep it off. One of the latest was published this summer in the journal Scientific Reports: Researchers found that a test group that took a protein supplement along with a mildly calorie-restricted diet lost more visceral fat than a group that took a placebo. Protein fills you up faster and for longer, and studies have found that it reduces levels of ghrelin, the hormone that tells the body it's hungry.And to get through this pandemic at your healthiest, don't miss these 35 Places You're Most Likely to Catch COVID.

More here:
Best Ways to Reverse Deadly Weight Gain Eat This Not That - Eat This, Not That

Recommendation and review posted by Bethany Smith

(THE CONVERSATION) One of my patients who had been struggling with obesity, uncontrolled diabetes and the cost of her medications agreed in June 2019 to adopt a more whole-food plant-based diet.

Excited by the challenge, she did a remarkable job. She increased her fresh fruit and vegetable intake, stopped eating candy, cookies and cakes and cut down on foods from animal sources. Over six months, she lost 19 pounds and her HbA1c a measure of her average blood sugar dropped from 11.5% to 7.6%.

She was doing so well, I expected that her HbA1c would continue to drop and she would be one of our plant-based successes who had reversed diabetes.

Her three-month follow-up visit in March 2020 was canceled because of COVID-19 lockdowns. When I eventually saw her again in May 2021, shed regained some of the weight and her HbA1c had climbed to 10.4%. She explained that her diabetes doctor and a diabetes nurse educator had told her that she was eating too much sugar on the plant-based diet.

Shed been advised to limit carbohydrates by cutting back on fruits and starchy vegetables and eating more fish and chicken. Sugar-free candy, cakes, cookies and artificial sweeteners were encouraged. In the face of conflicting medical advice, she fell back on conventional wisdom that sugar is bad and should be avoided whenever possible, especially if you have diabetes.

Im a physician, board certified in preventive medicine with a lifestyle medicine clinic at Morehouse Healthcare in Atlanta. This emerging medical specialty focuses on helping patients make healthy lifestyle behavior modifications. Patients who adopt whole-food plant-based diets increase carbohydrate intake and often see reversal of chronic diseases including diabetes and hypertension. In my clinical experience, myths about sugar and carbohydrates are common among patients and health professionals.

Fruit vs. sugar

Your body runs on glucose. It is the simple sugar that cells use for energy.

Glucose is a molecular building block of carbohydrates, one of the three essential macronutrients. The other two are fat and protein. Starches are long, branching chains of glucose.

Naturally occurring carbohydrates travel in nutrient-dense packages such as fruits, vegetables, whole grains, nuts and seeds.

Humans evolved to crave sweet tastes to get the nutrients needed to survive. A daily supply of vitamins, minerals and fiber is needed because our bodies cannot make them. The best source of these substances for our ancient ancestors was sweet, ripe, delicious fruit. In addition, fruits contain phytonutrients and antioxidants, chemicals produced only by plants. Phytonutrients such ellagic acid in strawberries have cancer-fighting properties and promote heart health.

Refined sugars, on the other hand, are highly processed and stripped of all nutrients except calories. Theyre a concentrated form of carbohydrates. The food industry produces refined sugars in many forms. The most common are sucrose crystals, which youd recognize as table sugar, and high-fructose corn syrup, which is found in many processed foods and sweetened beverages.

If you continually satisfy your taste for sweet with foods that contain refined sugar rather than the nutrient-rich fruits at the core of this craving passed on by evolution you may not get all the nutrients you need.

Over time, this deficit may create a vicious cycle of overeating that leads to obesity and obesity-related health problems. Women who eat the most fruit tend to have lower rates of obesity.

Sugar toxicity

Refined sugars are not directly toxic to cells, but they can combine with proteins and fats in food and in the bloodstream to produce toxic substances such as advanced glycation end products (AGEs). High blood glucose levels may produce glycated low-density lipoproteins. High levels of these and other glucose-related toxic substances are associated with an increased risk of a wide range of chronic health problems, including cardiovascular disease and diabetes.

The disease most commonly associated with sugar is Type 2 diabetes. A surprising number of people, including health professionals, incorrectly believe that eating sugar causes Type 2 diabetes. This myth leads to a focus on lowering blood sugar and counting carbs while ignoring the real cause: progressive loss of pancreatic beta cell function. At diagnosis, a patient may have lost between 40% and 60% of their beta cells, which are responsible for producing insulin.

Insulin is a hormone that controls how much glucose is in the bloodstream by blocking glucose production in the liver and driving it into fat and muscle cells. Loss of beta cell function means not enough insulin gets produced, resulting in the high blood glucose levels characteristic of Type 2 diabetes.

Beta cells have low levels of antioxidants and are susceptible to attack by metabolic and dietary oxidized free radicals and AGEs. Antioxidants in fruit can protect beta cells. Researchers have found that eating whole fruit decreases the risk of Type 2 diabetes, with those who eat the most fruit having the lowest risk.

Detoxing from sugar

People interested in losing weight and improving health often ask if they should do a sugar detox. In my opinion this is a waste of time, because it is not possible to eliminate sugar from the body. For instance, if you ate only baked chicken breasts, your liver would convert protein to glucose in a process called gluconeogenesis.

Low-carb diets may lead to weight loss, but at the expense of health. Diets that significantly reduce carbohydrates are associated with nutrient deficiencies and higher risk of death from any cause. On low-carbohydrate ketogenic diets the body will break down muscles and turn their protein into glucose. The lack of fiber causes constipation.

Eliminating foods sweetened with refined sugar is a worthy goal. But dont think of it as a detox it should be a permanent lifestyle change. The safest way to go on a refined sugar detox is to increase your intake of nutrient-dense fruits and vegetables. Once you eliminate refined sugar, youll likely find that your taste buds become more sensitive to and appreciative of the natural sweetness of fruits.

Article written by Jennifer Rooke,Morehouse School of Medicine via Associated Press.

Continued here:
Sugar detox? Cutting carbs? A doctor explains why you should keep fruit on the menu - WJW FOX 8 News Cleveland

Recommendation and review posted by Bethany Smith

How do you diagnose hormonal acne?

While it's best to see a trusted doctor to determine if you have hormonal acne, there are a few things you can look out for to self-diagnose it. Here's what Dr Joshi said:

Now onto the part that anyone with hormonal acne wants to know about: how TF do you fix it?

"Treatment is similar to that in adolescence," Dr Joshi explained. "However, due to persistence, more aggressive treatments and a combination of treatments may be needed to get it under control even for relatively mild symptoms."

"By the time I see patients with persistent acne, they have usually tried various over-the-counter products, they may have been to salons for facials and other treatments, some of which may have made the acne worse, and may contribute to scarring andpigmentation."

Dr Joshi explained that because acne is a medical disease, treatment options need to be medical.

She suggests a combination of topical prescription medication, oral medication (for a period of time), and a personalised, usually basic, skincare regimen.

Listen to Mamamia's podcast for your face, You Beauty. Post continues after audio.

"You can speed up the process by adding a series of in-clinic treatments if you wish but it will take on average three to six months minimum to begin tosee results," she said.

With that being said, Dr Joshi suggests saving your time and money and going straight for a skin consultation with a trusted doctor to obtain personalised advice and prescription medication, if appropriate.

"Treatment is time-consuming and depending on what is involved, may be costly, but the alternative means that acne continues to persist and cause unwanted side effects such as scarring and pigmentation that will also need to be addressed at some point,with added time and cost," she said.

"See a doctor who understands acne and save yourself time, money and the heartache of complications, and have realistic expectations," Dr Joshi said.

"The longer people leave it, thinking it will go away, or trying a variety of different over-the-counter things, the longer the disease is percolating under the surface and the longer it takes to rein it in and the chances of complications such as scarringand pigmentation.

"Treatment takes time, which is the single biggest thing I tell patients. And relapses are common if life gets in the way and they drop the ball."

Feature Image: Getty/Canva/Mamamia.

Excerpt from:
Struggling with hormonal acne and how to fix it? A skin expert shares her best advice. - Mamamia

Recommendation and review posted by Bethany Smith

There are pros and cons to letting Fluffy or Fido snuggle up with you at night.

Image Credit: LIVESTRONG.com Creative

How Bad Is It Really? sets the record straight on all the habits and behaviors youve heard might be unhealthy.

If you crave peak cozy, welcoming your cat or dog or both onto your bed delivers. After all, it's just so companionable to have a pet nearby thanks to the comfort of touching fur and the soft sounds of purrs and snuffles. But should you let your dog sleep with you?

And after a day spent together cats twining through your legs while you make a morning cup of coffee, or your dog curled up nearby while you watch TV cuddling in bed with your pet seems only natural. Indeed, a slim majority (56 percent) of pet owners invite cats and dogs into their bedroom, according to a December 2015 article in Mayo Clinic Proceedings.

But there are some health implications to this practice. Here's what experts have to say about the pros and cons of bringing pets in the bedroom, and whether your dog (or other furry friend) should sleep with you.

1. Your Allergies and Asthma Might Get Worse

About a third of people in the U.S. are allergic to cats and dogs (with cat allergies more common), according to the Asthma and Allergy Foundation of America.

If you're one of that group, allowing your pet in the bedroom means about eight hours of exposure to allergens, says immunologist Heather Moday, MD, author of The Immunotype Breakthrough.

"Breathing in this level of allergens all night long can cause nasal congestion, sinus inflammation and airway irritation if you have asthma," Dr. Moday says. That makes it harder to get restful sleep, and means you'll wake up stuffy and tired in the morning, she says.

And it's not only pet allergies that you need to consider. Cats and dogs carry pollen and dust on their fur, Dr. Moday says. That means allowing pets in bed puts you in close contact with two more common allergens.

This can also exacerbate asthma, as allergies and asthma often go hand-in-hand. Common triggers (like dander, dust and pollen) that lead to allergic reactions like sniffles or itchy eyes can also set off asthma symptoms, according to the Mayo Clinic.

Regularly washing yourself and your pet is one allergy remedy that can help prevent excess dander, dust and pollen.

2. You Might Get Flea Bites

Some parasites like to live on cats and dogs (think: fleas, ticks and lice). And allowing your cat or dog in the bedroom is placing yourself in very close contact with these itch-causing creatures.

Fleas, for instance, bite humans, which can cause itching and irritation, per the Cleveland Clinic.

3. Your Sleep Could Suffer

When it comes to sleep hygiene, there aren't any benefits to having pets in bed, says sleep psychologist Samina Ahmed Jauregui, PsyD, a Pluto Pillow advisor.

But there are some drawbacks: Pets like to move at night, says Abhinav Singh, MD, facility director of the Indiana Sleep Center and member of the National Sleep Foundation medical advisory board. "That can wake people up," he says.

And it's hard to go back asleep after waking up, especially as we age, Dr. Singh says which could reduce the quantity of sleep you get each night.

You'll know it if your pet wakes you up at 3 a.m. and you can't fall back to sleep right away. But what might be more insidious is when a pet's movement causes you to readjust more frequently than you would otherwise.

"Micro-awakenings are not recallable," Dr. Singh says. In other words, if you briefly wake up to toss and turn due to your cat or dog stirring, it's not long enough for your brain to register as a moment of wakefulness. But those brief bursts of sleeplessness still have an effect.

Dr. Singh compares it to flying: Would you rather fly nonstop or have seven connecting flights en route to your destination? These small, routine disruptions diminish the quality of your shut-eye. That'll leave you feeling unrested in the morning, even if you diligently clocked the Centers for Disease Control and Prevention-recommended seven-plus hours of sleep.

Jauregui sums it up: "Even if you are able to return to sleep quickly, having woken up in the first place is a disturbance in quality of sleep."

4. It Could Improve Your Mood

It's not all doom and gloom. Bringing a pet into your bedroom is associated with some significant mental health benefits.

"Many people claim to really enjoy sleeping with their animals because they feel less lonely and happier with their pets," Dr. Moday says. And there are studies to back that up.

For instance, in a survey of just under 1,000 pet owners assigned female at birth, dogs were "associated with stronger feelings of comfort and security" compared to human bed partners, according to November 2018 research in Anthrozos.

Also among the research-backed benefits of time with pets are an increase in oxytocin (a feel-good hormone) and a decrease in cortisol (a stress hormone), according to Johns Hopkins Medicine.

While Dr. Singh generally thinks bringing a pet to bed is best avoided, he makes an exception for someone who is overcoming loss or trauma and finds comfort in a pet's presence. "The dog or cat can be there to support you [and] help you recover emotionally," he says.

7 Healthy Tips for Sleeping With Your Pet in the Bed

Though there are some solid reasons why your dog shouldn't sleep with you, the joys of companionship likely outweigh these potential downsides if you and your pet are in the habit of sharing a bed.

We get it. So if you're disregarding the experts' take and persist in welcoming Fluffy or Fido onto the mattress, follow these strategies to reduce potential pitfalls:

So, How Bad Is It Really to Let Your Pet Sleep in Bed With You?

When it comes to deciding if you should let your dog sleep with you, truly, it depends.

Dr. Singh notes he has cats, and is an animal-lover but if forced to give a bottom line, he notes it's "generally not advisable" to sleep with your pet. It's also best avoided if you have allergies or allergy-trigged asthma. Instead, he recommends having a cat or dog bed or blanket in your bedroom, beside your bed, but not on it.

Jauregui agrees that it's best to keep the bedroom pet-free. But, she adds: "If you struggle with sleep and are not comfortable sleeping in the bed by yourself, then having a pet in the bed with you (that is large enough for both of you) can be OK." Some sleep even if it's potentially disrupted would be better than none, she says.

And if you just can't quit those nighttime snuggles (guilty!), follow the best practices described above when it comes to laundry, pet care and adjusting your sleep routine.

Read this article:
How Bad Is It Really to Let Your Pet Sleep in Your Bed? - Livestrong

Recommendation and review posted by Bethany Smith

Four years ago, a small Philadelphia biotech company won U.S. approval for the first gene therapy to treat an inherited disease, a landmark after decades of research aimed at finding ways to correct errors in DNA.

Since then, most of the world's largest pharmaceutical companies have invested in gene therapy, as well as cell therapies that rely on genetic modification. Dozens of new biotech companies have launched, while scientists have taken forward breakthroughs in gene editing science to open up new treatment possibilities.

But the confidence brought on by such advances has also been tempered by safety setbacks and clinical trial results that fell short of expectations. In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients. Here are five:

Food and Drug Administration approval of Spark Therapeutics' blindness treatment Luxturna a first in the U.S. came in 2017. A year and a half later, Novartis' spinal muscular atrophy therapy Zolgensma won a landmark OK.

But none have reached market since, with treatments from BioMarin Pharmaceutical and Bluebird bio unexpectedly derailed or delayed.

That could change in 2022. Two of Bluebird's treatments, for the blood disease beta thalassemia and a rare brain disorder, are now under review by the FDA, with target decision dates in May and June. BioMarin, after obtaining more data for its hemophilia A gene therapy, plans to soon approach the FDA about resubmitting an application for approval.

Others, such as CSL Behring and PTC Therapeutics, are also currently planning to file their experimental gene therapies with the FDA in 2022.

Approvals, should they come, could provide important validation for their makers and expand the number of patients for whom genetic medicines are an option. In biotech, though, approvals aren't the end of the road, but rather the mark of a sometimes challenging transition from research to commercial operations. With price tags expected to be high, and still outstanding questions around safety and long-term benefit, new gene therapies may prove difficult to sell.

A record $20 billion flowed into gene and cell therapy developers in 2020, significantly eclipsing the previous high-water mark set in 2018.

Last year, the bar was set higher still, with a total of $23 billion invested in the sector, according to figures compiled by the Alliance for Regenerative Medicine. About half of that funding went toward gene therapy developers specifically, with a similar share going to cell-based immunotherapy makers.

Driving the jump was a sharp increase in the amount of venture funding, which rose 73% to total nearly $10 billion, per ARM. Initial public offerings also helped, with sixteen new startups raising at least $50 million on U.S. markets.

Entering 2022, the question facing the field is whether those record numbers will continue. Biotech as a whole slumped into the end of last year, with shares of many companies falling amid a broader investment pullback. Gene therapy developers, a number of which had notable safety concerns crop up over 2021, were hit particularly hard.

Moreover, many startups that jumped to public markets hadn't yet begun clinical trials roughly half of the 29 gene and cell therapy companies that IPO'd over the past two years were preclinical, according to data compiled by BioPharma Dive. That can set high expectations companies will be hard pressed to meet.

Generation Bio, for example, raised $200 million in June 2020 with a pipeline of preclinical gene therapies for rare diseases of the liver and eye. Unexpected findings in animal studies, however, sank company shares by nearly 60% last December.

Still, the pace of progress in gene and cell therapy is fast. The potential is vast, too, which could continue to support high levels of investment.

"I think fundamentally, investment in this sector is driven by scientific advances, and clinical events and milestones," said Janet Lambert, ARM's CEO, in an interview. "And I think we see those in 2022."

The potential of replacing or editing faulty genes has been clear for decades. How to do so safely has been much less certain, and concerns on that front have set back the field several times.

"Safety, safety and safety are the first three top-of-mind risks," said Luca Issi, an analyst at RBC Capital Markets, in an interview.

Researchers have spent years making the technology that underpins gene therapy safer and now have a much better understanding of the tools at their disposal. But as dozens of companies push into clinical trials, a number of them have run into safety problems that raise crucial questions for investigators.

In trials run by Audentes Therapeutics and by Pfizer (in separate diseases), study volunteers have tragically died for reasons that aren't fully understood. UniQure, Bluebird bio and, most recently, Allogene Therapeutics have reported cases of cancer or worrisome genetic abnormalities that triggered study halts and investigations.

While the treatments being tested were later cleared in the three latter cases, the FDA was sufficiently alarmed to convene a panel of outside experts to review potential safety risks last fall. (Bluebird recently disclosed a new hold in a study of its sickle cell gene therapy due to a patient developing chronic anemia.)

The meeting was welcomed by some in the industry, who hope to work with the FDA to better detail known risks and how to avoid them in testing.

"[There's] nothing better than getting people together and talking about your struggles, and having FDA participate in that," said Ken Mills, CEO of gene therapy developer Regenxbio, in an interview. "The biggest benefit probably is for the new and emerging teams and people and companies that are coming into this space."

Safety scares and setbacks are likely to happen again, as more companies launch additional clinical trials. The FDA, as the recent meeting and clinical holds have shown, appears to be carefully weighing the potential risks to patients.

But, notably, there hasn't been a pullback from pursuing further research, as has happened in the past. Different technologies and diseases present different risks, which regulators, companies and the patient community are recognizing.

"We're by definition pushing the scientific envelope, and patients that we seek to treat often have few or no other treatment options," said ARM's Lambert.

Last June, Intellia Therapeutics disclosed early results from a study that offered the first clinical evidence CRISPR gene editing could be done safely and effectively inside the body.

The data were a major milestone for a technology that's dramatically expanded the possibility for editing DNA to treat disease. But the first glimpse left many important questions unanswered, not least of which are how long the reported effects might last and whether they'll drive the kind of dramatic clinical benefit gene editing promises.

Intellia is set to give an update on the study this quarter, which will start to give a better sense of how patients are faring. Later in the year the company is expecting to have preliminary data from an early study of another "in vivo" gene editing treatment.

In vivo gene editing is seen as a simpler approach that could work in more diseases than treatments that rely on stem cells extracted from each patient. But it's also potentially riskier, with the editing of DNA taking place inside the body rather than in a laboratory.

Areas like the eye, which is protected from some of the body's immune responses, have been a common first in vivo target by companies like Editas Medicine. But Intellia and others are targeting other tissues like the liver, muscle and lungs.

Later this year, Verve Therapeutics, a company that uses a more precise form of gene editing called base editing, plans to treat the first patient with an in vivo treatment for heart disease (which targets a gene expressed in the liver.)

"The future of gene editing is in vivo," said RBC's Issi. His view seems to be shared by Pfizer, which on Monday announced a $300 million research deal with Beam Therapeutics to pursue in vivo gene editing targets in the liver, muscle and central nervous system.

With more and more cell and gene therapy companies launching, the pipeline of would-be therapies has grown rapidly, as has the number of clinical trials being launched.

Yet, many companies are exploring similar approaches for the same diseases, resulting in drug pipelines that mirror each other. A September 2021 report from investment bank Piper Sandler found 21 gene therapy programs aimed at hemophilia A, 19 targeting Duchenne muscular dystrophy and 18 going after sickle cell disease.

In gene editing, Intellia, Editas, Beam and CRISPR Therapeutics are all developing treatments for sickle cell disease, with CRISPR the furthest along.

As programs advance and begin to deliver more clinical data, companies may be forced into making hard choices.

"[W]e think investors will place greater scrutiny as programs enter the clinic and certain rare diseases are disproportionately pursued," analysts at Stifel wrote in a recent note to investors, citing Fabry disease and hemophilia in particular.

This January, for example, Cambridge, Massachusetts-based Avrobio stopped work on a treatment for Fabry that was, until that point, the company's lead candidate. The decision was triggered by unexpected findings that looked different than earlier study results, but Avrobio also cited "multiple challenging regulatory and market dynamics."

See the rest here:
5 questions facing gene therapy in 2022 - BioPharma Dive

Recommendation and review posted by Bethany Smith

DUBLIN, Jan. 14, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Business Outlook" report has been added to ResearchAndMarkets.com's offering.

Research and Markets Logo

The Twice-Monthly Publication Cell and Gene Therapy Business Outlook will offer the following:

Market Sizing and Forecasting of CAGT Markets: Each issue sizes up the market opportunity and projects the future revenues for a given therapeutic segment.

Keeping an Eye on Financing: With billions of investment dollars announced each year, Cell and Gene Therapy Business Outlook tracks who is getting financed (and the companies behind the financing) each issue. On a regular basis we will analyze trends in that financing.

News Briefs and Analysis of the Science That will Shape Tomorrow's Business: Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won't miss an important development in cell and gene therapy.

Deals Between CAGT Companies Tracked: Each issue's "Recent Deals Table" tracks the important deals between stem cell companies as well as the deals they engage in (tech transfers, partnerships, mergers, distribution and other activities) with companies outside the industry.

Cell and Gene Therapy Tools: This newsletter will also report on developments, product launches and deals relating to the makers of cell and gene therapy manufacturing equipment and supplies.

Target Audience Includes:

Director, Vice President or Manager of Market Research

Director, Vice President or Manager of Marketing

Director, Vice President or Manager of Research and Development

Director of Business Insights

Director of Business Development

CEO

Reviews

"Great product overall...and external comprehensive analysis"- A Major Pharmaceutical Company

"GT and CT have been a hot topic! We are tasked to know this area and need a resource like this"-Consulting Firm

Story continues

"The charts and tables are a good idea to keep up with the amount of new announcements in CGT. We appreciated the scientific discussion in this issue and your recent webinar." -Cell and Gene Therapy Startup

For more information about this report visit https://www.researchandmarkets.com/r/upanl3

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

Cision

View original content:https://www.prnewswire.com/news-releases/cell-and-gene-therapy-business-outlook-service-301461330.html

SOURCE Research and Markets

See the original post:
Cell and Gene Therapy Business Outlook Service - Yahoo Finance

Recommendation and review posted by Bethany Smith

BioMarin Pharmaceutical plans to return to the Food and Drug Administration later this year with clinical trial results it says prove its gene therapy for hemophilia can prevent bleeding for years after treatment.

The data from BioMarin's study, disclosed Sunday ahead of the J.P. Morgan Healthcare Conference, are meant to meet requirements laid out by the agency when it rejected the company's previous approval application a year and a half ago.

They show the gene therapy restored blood clotting protein levels to a range consistent with mild hemophilia and, while those levels waned over time, that trial participants experienced very few, if any, bleeds across the two years most were studied. A handful of volunteers in the Phase 3 trial, the largest to date of a hemophilia gene therapy, were followed for three years and had similar results.

"I believe that these results will answer, quantitatively, quite a lot of the questions that agencies have had," said Hank Fuchs, BioMarin's head of research and development, on a conference call Sunday.

Regulators in Europe have already begun evaluating an application from BioMarin and are expected to make a decision in the first half of this year. In the U.S., BioMarin aims to quickly review the results with the FDA and, should the agency agree, potentially resubmit the therapy in the second quarter.

BioMarin's gene therapy, called Roctavian, is the product of years of research by the California biotech and builds on more than a decade of work by other scientists to develop a treatment for hemophilia's genetic cause. It is designed to deliver into the body a functional copy of the gene that's mutated in people with the "A" form of hemophilia, who are left with little or no clotting protein to stem bleeding.

People with severe hemophilia A, who make up about half of all those with disease, must take regular, preventive infusions of "replacement" clotting protein, also known as Factor VIII. Roctavian, which is meant for these individuals, would in theory allow them to stop, freeing them from chronic treatment while more effectively preventing bleeding.

Results from an earlier, much smaller trial showed such promise and, by mid-2020, BioMarin had come close to replicating those findings in the first group of volunteers enrolled in its Phase 3 study. But the FDA unexpectedly, according to the company sought more information to prove that benefit could last two years.

Last January, BioMarin revealed one-year results from all participants in the trial and, on Sunday, disclosed data from its two-year analysis. Treatment decreased the number of bleeds per year by 85%, from an average of nearly five among the 112 volunteers who were studied for at least six months before infusion to less than one at year two.

Among 17 participants who were given Roctavian three years before the analysis was conducted, the average annual bleeding rate remained below one as well.

"Our clinical outcome here is unassailably great," said Fuchs in a separate interview. "It almost makes the application, honestly, bulletproof."

But levels of Factor VIII activity, which had risen sharply to an average of 43 international units per deciliter of blood at one year, declined to 23 IU/dL by year two and, for those 17 participants, 17 IU/dL by year three. BioMarin reported these values using a lab test known as a chromogenic assay, which it says is more conservative than another one also used.

People with severe hemophilia typically have less than one IU/dL of Factor VIII in their blood, while mild hemophilia is typically considered to be between 5 IU/dL and 40 IU/dL.

The decline has been a source of doubt, causing concerns that Roctavian's ability to prevent bleeds might wane over time as well. At least for the first few years, Sunday's results show that isn't happening yet. BioMarin also points to data from an earlier study, in which annualized bleeding rates remained below one through five years, despite reduced Factor VIII activity.

"A small amount of Factor VIII is going to go a long way towards hemostatic efficacy," said Fuchs on Sunday's call, "and it gives us confidence that what we've seen so far in the Phase 2 study is gonna read through to the Phase 3 study when we get there."

Extrapolating efficacy puts BioMarin on somewhat uncertain ground, however, as it is the first company to advance this far with a gene therapy for hemophilia A. Jean-Jacques Bienaime, BioMarin's CEO, argues the data so far for Roctavian indicate treatment should result in at least five years of bleeding control and perhaps even eight or longer.

"With the Phase 2, we have demonstrated at least five years already. Predicting eight years, I don't think, is a big stretch," he said in an interview.

How the FDA will view BioMarin's data is unclear, although analysts on Wall Street predicted the latest results would be enough to merit an approval. The agency could convene a panel of outside experts to review a resubmitted application from the company, a possibility Fuchs acknowledged on the conference call.

Also uncertain is how Roctavian would be perceived by hemophilia patients and by insurers, should it eventually secure an approval. BioMarin has previously suggested a price as high as between $2 million and $3 million, but that might be viewed as high if Roctavian's benefit isn't lifelong. (ICER, a looked-to drug cost watchdog, previously found Roctavian could be cost effective at a price of even $2.5 million.)

Fuchs said the company plans to present more data at a medical meeting, likely this year, that should help clarify the relationship between Factor VIII activity and expected durability of benefit.

Importantly for Roctavian's future, Sunday's data, while relatively sparse, indicated no new safety issues had emerged in testing. There were no cases of "inhibitors," or antibodies that work against clotting protein, developing following treatment, nor were there any cases of cancer or blood clot blockages.

The former two are both newly of interest following reports of cancers developing in other gene therapy trials, and data showing higher-than-normal levels of clotting factor in a trial of another hemophilia gene therapy being developed by Pfizer and Sangamo Therapeutics.

Note: This story has been updated to include mention of the assay used by BioMarin to measure Factor VIII activity, and of ICER's analysis.

Read more:
BioMarin plans return to FDA with updated data on hemophilia gene therapy - BioPharma Dive

Recommendation and review posted by Bethany Smith

EXTON, Pa., Jan. 10, 2022 /PRNewswire/ -- Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for rare genetic diseases, today announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek's technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek's development pipeline beyond skin and connective tissue disorders to rare liver diseases.

"This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB)."

With the acquisition of Novavita Thera, formerly aCytotheryx, Inc., company, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (HT1),a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.

Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. The therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.Castle Creek plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.

In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children's Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Dr. Lillegard and Dr. Kaiser will be the company leads for Castle Creek's recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

"It is an exciting time to join Castle Creek, a company that has already established an impressive research and development program in cell and gene therapies with proven clinical development and in-house manufacturing capabilities," said Dr. Lillegard. "I look forward to collaborating with the company's dedicated team on development of novel gene therapies. We believe our work to evaluate the safety of in vivo lentiviral vector administration in HT1 has the potential to be a precedent setting approach that can be applied to a range of new therapeutic areas for underserved patient populations."

About Castle Creek Biosciences, Inc.

Castle Creek Biosciences, Inc. is a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for patients living with rare genetic diseases. Castle Creek's most advanced product candidate, dabocemagene autoficel (FCX-007, D-Fi), an ex vivo, autologous gene therapy, is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also evaluating FCX-013, an ex vivo, autologous gene therapy, in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma.In addition, LV-FAH, an in vivo, investigational gene therapy candidate, is being assessed in preclinical studies for the treatment of hereditary tyrosinemia type 1 (HT1).Castle Creek is pursuing discovery and development of early-stage novel product candidates utilizing its dual platform of ex vivo and in vivo technologies to expand its robust pipeline. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences, LLC

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contacts

Adam DaleyBerry & Company Public Relations212.253.8881[emailprotected]

Karen CaseyCastle Creek Biosciences302.750.4675[emailprotected]

SOURCE Castle Creek Biosciences, Inc.

Visit link:
Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform - PRNewswire

Recommendation and review posted by Bethany Smith

NEW YORK, Jan. 12, 2022 /PRNewswire/ -- Polaris Market Research recently published a research report on "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" in its online research storage.

According to [127+ Pages] research report published by Polaris Market Research, the global Gene Delivery Technologies Market size & share expected to reach to USD 7.86 Billion by 2028 from USD 2.64 Billion in 2020, at a compound annual growth rate (CAGR) of 15.0% during forecast period 2021 to 2028.

What is Gene Delivery Technology? How big is Gene Delivery Technology Industry?

Gene delivery technology is widely used in gene therapies, which involves transferring of genetic and hereditary disorders. These therapies have also performed an important role in shaping the entire pharma landscape. Around 27 gene therapies were revealed in the marketplace and over 990 companies emphasized the research & development, and commercialization of innovative therapies by 2020. The constantly changing market environment for advanced therapies is reportedly driving the market for gene delivery technologies.

The operating market players are building various business strategies to boost the market for gene delivery technologies, while the developing gene delivery technologies are creating openings for several new players in the market. Different research settings offer market applications for various gene delivery technologies. However, due to technical challenges related to each modal type, the clinical settings produce very few applications. Within the clinical settings, physical technologies require a breakthrough in their use.

Request a Sample Report of Gene Delivery Technologies Market: https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market/request-for-sample

Key Aspects Covered By Report:

Top Market Companies Profiles Covered:

Directly Purchase a copy of report with TOC @ https://www.polarismarketresearch.com/checkouts/8241

Basic Questions Answered By Report:

Industrial Gene Delivery Technology Market: Growth Factors

The adoption of gene therapies and subsequent increase in clinical research activities around the globe has fueled the market growth. Also, the growing acceptance of gene therapy products and services has supported the gene delivery technologies market growth prospects. Other key driving factors of the market involve technological advancements in viral vectors, a rising pipeline of advanced therapies, and a growing number of regulatory approvals for advanced therapy products.

Inquire more about this report before purchase @ https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market/inquire-before-buying

Global Gene Delivery Technology Market: Key Segmentation

Insight by Mode

In 2020, the biological vectors market segment secured the largest revenue share of the gene delivery technologies industry due to the high success rate of Kymriah and Yescarta. Following the acceptance of vectors-based therapy products, the above-mentioned vectors have experienced greater attention.

The chemical delivery method market segment is expected to secure a lucrative growth rate over the study period. Clinical challenges are observed in viral systems which propelled the chemical methods' use. Chemical delivery systems have replaced viral delivery systems because of their capability in combating challenges.

The physical delivery methods market segment has lower transfection efficiency than biological or chemical modes. One other drawback called low cell viability in electroporation-based physical methods enables other market players to gain more share. It helps them to address a high focus on transfection and cell viability issues.

Also Read, Global Gene Therapy Market Report, 2021-2028

Insight by Method

The ex-vivo market segment of the gene delivery technologies market witnessed the largest share in 2020. Its transduction efficiency is the major factor behind the high share achievement, making it an ideal candidate to be used in research settings.

In this market, the in-vivo delivery method market segment is expected to obtain a lucrative growth rate over the estimation period as it features a high preference for highly targeted gene deliveries. Researchers are extending research & development for the market segment. For example, Oregon Health and Science University built the gene-editing tool "Crispr-Cas9" in 2020, which enables genetic code editing for blind people.

Request Customized Copy of Report @ https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market/request-for-customization

Gene Delivery Technologies Market: Report Scope

Report Attribute

Details

Market Size 2020 Value

USD 2.64 Billion

Market Outlook for 2028

USD 7.86Billion

Expected CAGR Growth

CAGR 150% from 2021 - 2028

Base Year

2020

Forecast Year

2021 - 2028

Top Market Players

Horizon Discovery Group Co., QIAGEN, Oxford Biomedica, SignaGen Laboratories, Hoffmann-La Roche AG, Vectalys, Sirion-Biotech GmbH and Others

Segments Covered

By Mode, By Application, By Method, By Region

Geographies Covered

North America, Europe, Asia Pacific, Latin America and Middle East & Africa

Customization Options

Customized purchase options are available to meet your research needs. Explore customized purchase options

Geographic Overview: Gene Delivery Technology Market

The North American region registered a significant share of the global gene delivery technology market. Various clinical trials are used to access the efficacy of gene therapies to treat hereditary, cancer, genetic mutations, and rare disorders in the U.S. This factor is the key driver of the gene delivery technologies demand growth in North America.

In addition, the availability of better clinical infrastructure also contributes to market growth. Many companies are marketing gene delivery products and accessories, which will boost the gene delivery technologies industry growth prospects. The U.S. has already announced many research projects combined with other leaders under its Horizon 2020 plans. This project will also cover other vector-based gene delivery trials for rare diseases.

Moreover, the Asia Pacific gene delivery technologies industry is anticipated to account for a profitable gene delivery technology market growth rate over the assessment period. The region is well known for the developed pharmaceutical industry even with its large population size, and low labor costs.

Browse the [127+ Pages] Detail Report "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" with in-depth TOC: https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market

For Additional Information OR Media Enquiry, Please Mail Us At: [emailprotected]

The market is primarily segmented on the basis of mode, application, method, and geographic region.

Gene Delivery Technology Market: By Mode Outlook

Gene Delivery Technology Market: By Application Outlook

Gene Delivery Technology Market: By Method Outlook

Explore the Latest Market Research Reports by PMR:

Genomic Biomarkers Market Share, Size, Trends, Industry Analysis Report, By Disease Indication (Oncology, Cardiovascular Diseases, Neurological Diseases, Renal Disorders, Others); By Validation; By Type; By End-Use; By Region; Segment Forecast, 2021 2029

Nucleic Acid Isolation and Purification Market Share, Size, Trends, Industry Analysis Report, By Product (Kits & Reagents, Instruments); By Type; By Method (Column-based, Magnetic Beads, Reagent-based, Others); By Application (Precision Medicine, Diagnostics, Drug Discovery & Development, Agriculture and Animal Research, Others); By Region; Segment Forecast, 2021 2028

Drug Testing Market Share, Size, Trends, Industry Analysis Report, By Drug Type; By Sample Type; By Product (Consumables, Instruments, Rapid Testing Devices, Services); By End-Use; By Region; Segment Forecast, 2021 - 2028

About Polaris Market Research

Polaris Market Research is a global market research and consulting company. The company specializes in providing exceptional market intelligence and in-depth business research services for our clientele spread across different enterprises. We at Polaris are obliged to serve our diverse customer base present across the industries of healthcare, technology, semi-conductors and chemicals among various other industries present around the world. We strive to provide our customers with updated information on innovative technologies, high growth markets, emerging business environments and latest business-centric applications, thereby helping them always to make informed decisions and leverage new opportunities. Adept with a highly competent, experienced and extremely qualified team of experts comprising SMEs, analysts and consultants, we at Polaris endeavor to deliver value-added business solutions to our customers.

Contact Us:Likhil G30 Wall Street8th Floor,New York City, NY 10005,United StatesPhone:+1-917-985-9017Email:[emailprotected]Web:https://www.polarismarketresearch.com/Follow US:LinkedIn|twitter

SOURCE Polaris Market Research

Read this article:
At 15.0% CAGR, Global Gene Delivery Technologies Market Size Will Reach USD 7.86 Billion By 2028: Polaris Market Research - PRNewswire

Recommendation and review posted by Bethany Smith

Risky biotechnology start-ups that soared in recent years can't catch a break in 2022. Beam Therapeutics (NASDAQ:BEAM) recently signed a major research deal with Pfizer (NYSE:PFE) that could be worth up to $1.35 billion, and hardly anyone seemed to notice.

This was clearly great news for Beam Therapeutics, but a stock market scorned for clinical-stage biotech stocks didn't respond the way anyone familiar with the company would have expected. Instead of surging higher in response to the Pfizer deal, Beam Therapeutics stock actually fell nearly 2% on the day of the announcement.

Nearly all biotech stocks are in the doghouse lately and it looks like the market may have missed something here. Let's look closer to see if Beam Therapeutics is a smart buy at the moment.

Image source: Getty Images.

Over the past few years, Pfizer has watched its peers experiment with CRISPR-based gene editing techniques without making any significant investments. Beam Therapeutics' base-editing technology, though, really got the big pharma company's attention.

Instead of removing and replacing entire sections of genetic material like Intellia, andCRISPR Therapeutics (NASDAQ:CRSP), Beam Therapeutics is pioneering a more precise method called base editing. This involves altering just one letter of genetic material at a time, which is a lot more useful than it might seem. Around half of all known genetic variations associated with diseases are caused by single-point mutations.

Pfizer will give Beam Therapeutics a $300 million payment up front to discover new drug candidates aimed at three undisclosed targets that won't compete with Beam's existing programs. Beam's eligible for up to $1.05 billion in milestone payments if all three go on to become a commercial success.

Beam Therapeutics is eligible to receive royalties at an undisclosed percentage of global sales for each future program. Beam Therapeutics even has an option to co-develop and co-commercialize one of the future candidates for a larger cut of sales.

Beam Therapeutics finished September with $934 million in cash after operations burned through $329 million during the first nine months of the year. Pfizer's cash injection should raise the company's cash balance high enough to get through 2023 before it needs to tap investors for more.

There's no telling whether Pfizer will decide to license a candidate from Beam Therapeutics. If Pfizer drags its feet, the gene-editing start-up has some preclinical-stage programs of its own that might have a chance to impress investors before it's time to raise capital again.

The most advanced candidate in Beam's pipeline at the moment, BEAM-101 is an experimental gene therapy for the treatment of sickle cell disease. The company doesn't expect to begin enrolling patients into the first clinical trial with BEAM-101 until the second half of 2022.

With a recent market cap of $4.5 billion, there's already a lot of success for Beam's pipeline priced into the stock. Unfortunately, the road ahead could be a lot more challenging than investors are anticipating. Last year, Vertex Pharmaceuticals (NASDAQ:VRTX) and collaboration partner CRISPR Therapeutics reported highly encouraging results from a clinical trial with CTX001 that started way back in 2018.

CTX001 is an experimental gene therapy for sickle cell disease that's similar to BEAM-101 in that it encourages the production of fetal hemoglobin. If early interim data that Beam Therapeutics posts a couple of years from now doesn't appear competitive with CTX001, the stock could take a tumble. While this is a top gene-editing stock to watch, it's still a little too risky to buy right now.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

See the rest here:
Is Beam Therapeutics a Good Stock to Buy Now? - The Motley Fool

Recommendation and review posted by Bethany Smith

Amicus Therapeutics is moving on from an early-phase Batten disease program after follow-up data showed the therapy didn't stop the fatal nervous system disease from progressing long-term.

The company disclosed the pipeline trim in a preliminary revenue guidance announcement ahead of its Wednesday presentation at the J.P. Morgan Healthcare Conference.

Amicus was advancing a gene therapy program for CLN6 Batten disease, a type of the rare genetic disorder that causes development regression and typically begins in childhood. Back in 2019, Amicus shared data on AAV-CLN6 that showed the gene therapy stabilized children's motor and language functions.

But the CLN6 program will now be discontinued after the company got a look at long-term extension data from a phase 1/2 trial.

RELATED:Amicus shares early look at Batten disease gene therapy

The company found the disease stabilization that had occurred during the earlier portion of the trial was not sustained at the two-year mark. Amicus plans to review the data with the CLN6 Batten disease community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed, according to the guidance announcement.

During its last earnings report in November 2021, Amicus had said it would be ramping up manufacturing activities and regulatory discussions for the program.

Meanwhile, the company will advance its CLN3 Batten disease program, which is currently in a phase 1/2 trial. A readout from the trial and additional preclinical data are expected in 2022, Amicus said. Once the data are released, the company can begin work on a pivotal trial.

Continued here:
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another - FierceBiotech

Recommendation and review posted by Bethany Smith