Autoimmune Diseases That Can Be Fatal – Yahoo News

Life and death with an autoimmune disease

There are now more than 100 autoimmune diseases, in which the immune system that would normally help a person fight off disease instead mistakenly attacks healthy tissue and does damage to the body.

According to the National Institutes of Health, up to 23.5 million Americans suffer from an autoimmune disease, and the incidence of many of the conditions is rising for reasons that aren't entirely clear. And the list of potentially fatal autoimmune diseases, or those that can shorten life span, is long. These conditions include everything from rheumatoid arthritis, which causes pain and swelling in the joints and raises the risk for life-threatening cardiovascular disease, to lupus, a chronic disease that can cause inflammation in any part of the body.

"Almost all autoimmune diseases decrease life expectancy," says Dr. Betty Diamond, director of the Institute of Molecular Medicine at the Feinstein Institutes for Medical Research at Northwell Health in Manhasset, New York. One notable exception is hypothyroidism, or underactive thyroid, she says.

Treatment advancements mean many with autoimmune diseases now live longer.

While disease severity varies, improvements in treatment have greatly bolstered life expectancy and improved quality of life for many with autoimmune conditions. Therapeutic advances include biologic drugs that suppress the immune system activity -- or overactivity -- that cause myriad health problems for those with autoimmune diseases such as lupus and multiple sclerosis.

Frequently, as with other disease prevention and management, a combination of lifestyle improvements -- like eating well, exercising regularly and getting adequate sleep -- and medical management are recommended. "The most important thing is to find a physician who's knowledgeable and experienced and with whom you work well, and together work out a treatment regimen that works for you," Diamond says.

Autoimmune myocarditis

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Autoimmune diseases may impact mortality in a couple ways: First, more common autoimmune diseases like rheumatoid arthritis and Type 1 diabetes can have an impact on the lives of a greater number of people. And second, "There are autoimmune diseases that have a very high rate of mortality but are very rare," says Dr. Virginia Pascual, director of the Gale and Ira Drukier Institute for Children's Health at Weill Cornell Medicine in New York City. One example of that, she notes, is autoimmune myocarditis, a rare autoimmune disease characterized by inflammation of the heart muscle.

Myocarditis is often diagnosed in people their 20s to 40s, and symptoms like abnormal heart beat, chest pain, shortness of breath, fatigue and fever can come on suddenly and without warning. The condition is underdiagnosed and may cause sudden death.

So with possible heart attack symptoms, it's important to seek medical attention immediately for possible symptoms of the disease. When it is diagnosed, drugs that suppress the immune system may be used to treat autoimmune myocardititis.

Multiple sclerosis

Nearly 1 million people in the U.S. are living with multiple sclerosis, according to the National Multiple Sclerosis Society. This autoimmune disorder affecting the brain and spinal cord results from the immune system attacking a protective layer around the nerves called the myelin sheath, which can cause a range of problems. These include difficulty with coordination and balance to thinking and memory issues.

Treatment improvements continue to inch life expectancy for those with MS closer to what's typical for people without the neurologic disorder. However, research published in the journal Neurology suggests people with MS live seven years less, on average; those with MS had a median age of survival of about age 76 compared to 83 for a matched general population, according to the 2015 study.

Among other treatment options, drugs therapies targeting B cells -- a type of white blood cells, cells used by body's immune system -- have shown promise. "B cell depletion therapy has been phenomenal for many patients with multiple sclerosis, and is now standard of care, which it certainly wasn't some years ago," Diamond says.


In addition to treating the autoimmune disorders, clinicians have to be mindful of treating other health problems caused by or related to the autoimmune disorders. For example, the most common type of lupus, systemic lupus erythematosus, causes widespread inflammation and impacts organs like the kidneys (a form called lupus nephritis). So improved treatment of kidney disease, including the availability of kidneys for transplant, has helped improve the outlook for many with lupus.

Therapies that suppress the immune system also put patients at a higher risk of infection, a cause of death in some patients with lupus. So improved treatment of infections -- including, as Diamond notes, having better antibiotics for some of infectious complications of treating lupus -- has also positively impacted survival rates for people with lupus.

As with other autoimmune conditions, various factors impact life expectancy for people with lupus, including, for reasons not fully understood, ethnicity. "Lupus mortality is increased in this country in African-Americans, in Hispanic populations, in Asians and is decreased in Caucasians," Pascual says.

Type 1 diabetes

Commonly diagnosed in kids, Type 1 diabetes is an autoimmune disease in which the immune system attacks the pancreas so that it can't make the insulin needed to properly control blood sugar.

Those with diabetes that's well-controlled can live long, full lives and have good quality of life. However, particularly if it isn't well-managed, Type 1 and other forms of diabetes (which aren't autoimmune diseases) can cause a variety of serious complications. Those can include kidney and heart disease, eye disease, or diabetic retinopathy, and nerve problems.

Type 1 diabetes is managed with insulin -- to keep blood sugar within a healthy range -- rather than suppressing the immune system. Exemplifying the impact of lifestyle, proper management of Type 1 diabetes means also carefully considering diet, as well as exercising regularly. That includes controlling carbohydrate consumption and evenly spreading carbs across the day with meals and snacks.


Vasculitis -- inflammation of the blood vessels, which is often caused by autoimmune disorders -- also decreases life expectancy, notes Dr. Ignacio Sanz, chief of the division of rheumatology at Emory University School of Medicine, and director of the Lowance Center for Human Immunology at Emory University and Children's Healthcare of Atlanta.

Treatment involves using medications like corticosteroids to control inflammation as well as addressing any underlying disease. Biologic therapies may be used to deal with immune system issues that contribute to the condition.

To prevent infections that can occur when immunosuppressive treatments are used for any autoimmune disorder, patients are encouraged to stay up to date on vaccinations. That includes getting vaccinated to prevent the flu, shingles and pneumonia, as recommended by a doctor. Vaccination should be part of the standard of care with autoimmune diseases, Sanz says.

Rheumatoid arthritis

The joint disease -- which affects up to 1% of the population, predominantly women -- can raise the risk for heart disease and lung disease. Based on research to date, having RA may decrease life expectancy by a decade or more.

RA is one of a number of autoimmune disorders called rheumatic diseases, or musculoskeletal conditions marked by inflammation. Most rheumatic diseases have a decreased life expectancy and increased mortality, Sanz says. "That is very well-documented for rheumatoid arthritis, it's well-documented for lupus (and) it's clear for vasculitis."

However, as with other autoimmune diseases, life span varies greatly with RA by the individual, and proper management of the disease can improve a person's outlook. Treatment often includes the use of immunosuppressive anti-rheumatic drugs (DMARDs) and self-care for arthritis symptoms such as using a heating pad and pain relievers to ease joint discomfort.


Just as rheumatoid arthritis can impact health well beyond inflaming joints, psoriasis is more than a skin disease. The autoimmune disorder is also associated with other serious issues, including an increased risk of diabetes, depression and heart disease.

As a result, depending on the severity of psoriasis, it may affect life expectancy and raise mortality risk. For that reason, experts say, patients and their doctors should discuss not only topical treatment to address scaly patches of skin, but if and when immunosuppressive therapy might be warranted.

Some autoimmune conditions that may affect life expectancy:

-- Autoimmune myocarditis.

-- Multiple sclerosis.

-- Lupus.

-- Type 1 diabetes.

-- Vasculitis.

-- Rheumatoid arthrtis.

-- Psoriasis.

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Autoimmune Diseases That Can Be Fatal - Yahoo News

Discovery Could Lead to New Breast Cancer Drugs – HSC Newsbeat

Eric Prossnitz, PhD, hopes to help many of the 12% of American women who are projected to be diagnosed with breast cancer in their lifetimes. He and his team have recently completed studies on a compound that they think could be made to attack breast cancer cells differently than current drugs. Their work is reported in the November online issue of Cell Chemical Biology.

Prossnitz understands firsthand the long path between discovery and U.S. Food and Drug Administration approval. A professor and cancer scientist at The University of New Mexico Comprehensive Cancer Center, he has studied breast cancer for the past 15 years. One of the compounds that he and his team discovered in 2006 has been licensed to Linnaeus Therapeutics for the treatment of melanoma and other cancers. Phase I clinical trials began at the UNM Comprehensive Cancer Center in 2019.

Prossnitz remains cautiously optimistic that the compound he and his team are now studying could help thousands of women. According to the National Cancer Institutes Surveillance, Epidemiology and End Results program, more than 80% of women with breast cancer have estrogen receptor-positive (ER+) breast cancer.

The anti-hormonal breast cancer drugs available drugs like tamoxifen and fulvestrant work very well, but they work in only about two-thirds of the women with ER+ breast cancers. And, some of these women initially respond but then their breast cancer comes back in a form that resists the drug.

Its a huge number of women, Prossnitz says. In 2019 alone, more than 60,000 women could face a new or recurrent ER+ breast cancer that wont respond to anti-hormonal therapy.

Prossnitz and his team discovered some years ago that another cell receptor responds to anti-hormonal drugs. They named it GPER, for G protein-coupled estrogen receptor. While the primary estrogen receptor, ER-alpha, resides primarily inside the nucleus of a cell, GPER sits within cell membranes.

Tamoxifen, and drugs like it, block the ER-alpha receptor, while fulvestrant and similar drugs induce the cell to degrade it. By blocking or degrading the ER-alpha receptor, these anti-hormonal drugs greatly reduce the signal for the cancer cell to grow and reproduce. But, as Prossnitz and his team have previously shown, they also activate GPER, and GPER signals the cell to keep growing and reproducing.

ER-alpha is the one [receptor] that plays an important role in ER+ breast cancer, Prossnitz says.

Most breast cancer cells follow the ER-alpha signal and die on schedule when ER-alpha is blocked or degraded. But a very small number of breast cancer cells may follow the GPER signal and survive. And those cells can grow into aggressive tumors that no longer respond to anti-hormonal drugs.

Prossnitz and his team discovered a compound some years ago called AB-1 that binds to ER-alpha but does not activate GPER, thereby avoiding the undesirable side effect of current anti-hormonal drugs. In their Cell Chemical Biology paper, they report their studies that describe AB-1s unique binding and activity behavior.

As theyve done previously, Prossnitz and his team are working to change the structure of AB-1 to control its properties more tightly, before they progress to preclinical studies. Prossnitz is familiar with the path, and again, he hopes to develop a drug that will benefit many women with breast cancer.

A Selective Ligand for Estrogen Receptor Proteins Discriminates Rapid and Genomic Signaling was published online on November 6, 2019, and will be published in the December 19 print edition of Cell Chemical Biology. Authors are: Chetana M. Revankar, Cristian G. Bologa, Richard A. Pepermans, Geetanjali Sharma, Whitney K. Petrie, Sara N. Alcon, Angela S. Field, Chinnasamy Ramesh, Matthew A. Parker, Nikolay P. Savchuk, Larry A. Sklar, Helen J. Hathaway, Jeffrey B. Arterburn, Tudor I. Oprea, and Eric R. Prossnitz.

Eric Prossnitz, PhD, is a Distinguished Professor and Chief of the Division of Molecular Medicine, in the Department of Internal Medicine, at The University of New Mexico School of Medicine. He co-leads the Cancer Therapeutics research program at the UNM Comprehensive Cancer Center.

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Discovery Could Lead to New Breast Cancer Drugs - HSC Newsbeat

LogicBio Therapeutics Announces Collaboration with Takeda to Develop New Genome Editing Candidate LB-301 for the Treatment of Crigler-Najjar Syndrome…

LB-301 is a recombinant AAV vector with a UGT1A1 gene leveraging GeneRidegenome editing platform for the treatment of Crigler-Najjar syndrome

The collaboration agreement grants Takeda an exclusive option to negotiate an exclusive, worldwide license to LogicBios LB-301 program

Crigler-Najjar syndrome is the second indication to be pursued using GeneRideplatform

CAMBRIDGE, Mass., Jan. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients today announced a research collaboration with Takeda Pharmaceutical Company Limited (Takeda) to further develop LB-301, an investigational therapy using LogicBios proprietary, promoterless, nuclease-free genome editing technology, GeneRide, for the treatment of Crigler-Najjar syndrome. LB-301 is a recombinant adeno-associated viral (AAV) vector with a uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene. The collaboration will bring together LogicBios propriety platform for genome editing and Takedas expertise in researching and developing gene therapies.

LogicBios innovative, site-specific, genome editing platform has the potential to overcome the limitations that make it challenging to apply conventional gene editing and gene transfer in pediatric patients, said Dan Curran, Head, Rare Diseases Therapeutic Area Unit at Takeda. We see GeneRide as a promising approach to explore as part of our aspiration to develop transformative or even potentially curative therapies to patients living with rare diseases.

We are thrilled to be working with Takeda to advance our GeneRide platform in a second indication, said Fred Chereau, CEO of LogicBio. Their insights and expertise in rare diseases drug development is expected to significantly accelerate the development of a much-needed therapy for this devastating pediatric disease. This collaboration recognizes GeneRide as a promising approach for bringing the transformational power of genome editing to children with an array of relentless, progressive pediatric diseases.

Under the agreement, LogicBio and Takeda will collaborate to further research and develop LB-301. Takeda will provide funding for the research program under the collaboration agreement and will have an exclusive option to negotiate an exclusive, worldwide license to LogicBios LB-301 program.

CriglerNajjar syndrome is a rare monogenic pediatric disease caused by a deficiency in the liverspecific UGT1A1 gene, resulting in severely high levels of unconjugated bilirubin in the blood starting at birth, with lifelong risk of permanent neurological damage and death. Current clinical practice consists of daily, intense phototherapy treatment for approximately 12 hours, but this treatment becomes less effective with age, ultimately leaving liver transplantation as the only therapeutic option for survival.

LogicBio has demonstrated that a murine GeneRide construct of LB-301 can correct the gene deficiency in an animal model of Crigler-Najjar syndrome. The introduction of a UGT1A1 gene into the albumin locus in mouse liver cells resulted in normalization of bilirubin levels and long-term survival of mice deficient in UGT1A1 from fewer than 20 days to at least one year. The results from this research were published in EMBO Molecular Medicine (Porro et al., 2017).

About LB-301LB-301 is an investigational pediatric genome editing therapy based on LogicBios GeneRide technology. GeneRide enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-301 is designed to incorporate a functioning version of the faulty uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene into the genome of Crigler-Najjar patients. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology.

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

For more information, please visitwww.logicbio.com.

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

LogicBio Contacts:

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

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

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LogicBio Therapeutics Announces Collaboration with Takeda to Develop New Genome Editing Candidate LB-301 for the Treatment of Crigler-Najjar Syndrome...

Antibiotics Could Be Promising Treatment for Form of Dementia – UKNow

LEXINGTON, Ky. (Jan. 10, 2020)Researchers at the University of Kentuckys College of Medicine have found that a class of antibiotics called aminoglycosides could be a promising treatment for frontotemporal dementia.

Results of their proof of concept study, which was a collaborative effort between UKs Department of Molecular and Cellular Biochemistry and the University of California San Franciscos Department of Pathology, were recently published in the journal,Human Molecular Genetics.

Frontotemporal dementia is the second-most common dementia after Alzheimers disease and the most common type of early onset dementia. It typically begins between ages 40 and 65 and affects the frontal and temporal lobes of the brain, which leads to behavior changes, difficulty speaking and writing, andmemory deterioration.

A subgroup of patients with frontotemporal dementia have a specific genetic mutation that prevents brain cells from making a protein called progranulin. Although progranulin is not widely understood, its absence is linked to the disease.

A group led by Haining Zhu, a professor in UKs Department of Molecular and Cellular Biochemistry, discovered that after aminoglycoside antibiotics were added to neuronal cells with this mutation, the cells started making the full-length progranulin protein by skipping the mutation.

These patients brain cells have a mutation that prevents progranulin from being made. The team found that by adding a small antibiotic molecule to the cells, they could trick the cellular machinery into making it, said Matthew Gentry, a co-author of the study and the Antonio S. Turco Endowed Professor in the Department of Molecular and Cellular Biochemistry.

The researchers found two specific aminoglycoside antibiotics - Gentamicin and G418 - were both effective in fixing the mutation and making the functional progranulin protein. After adding Gentamicin or G418 molecules to the affected cells, the progranulin protein level was recovered up to about 50 to 60%.

These results could be promising to drug development. Currently, there are no effective therapies for any type of dementia.

After this preclinical proof of concept study, the next step is to study the antibiotics effects on mice with the mutation that causes frontotemporal dementia, Zhu says. Another focus is to possibly develop new compounds from Gentamicin and G418 that could be safer and more effective. Although Gentamicin is an FDA-approved medication, its clinical usage is limited as it is associated with a number of adverse side effects.

If we can get the right resources and physician to work with, we could potentially repurpose this drug. This is an early stage of the study, but it provides an important proof of concept that these aminoglycoside antibiotics or their derivatives can be a therapeutic avenue for frontotemporal dementia, said Zhu.


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Antibiotics Could Be Promising Treatment for Form of Dementia - UKNow

Spotlight on Comprehensive Genomic Profiling – BSA bureau

Over 1.2 million mutations in 350 genes in a human body causing cancer make every cancer case a unique one

From the understanding of 250 types of cancers by the conventional diagnostic methods the medical field is moving fast forward to identification of mutated cell. The medical field has moved from histological diagnosis in 2000 to molecular diagnosis in 2018, said Dr Christian Rommel, Global Head, Oncology Pharma Research, Roche.

The new generation technology now knows the uniqueness of each cancer case by understanding what mutations in which of the 350 genes that contribute to cancer development cause it. Dr Rommel informed that scientists are targeting to find more genes that may contribute to cancer.

Advances in cancer biology are driving future and cancer care is becoming personalised, said Josh Jordon, Lifecycle Leader, Foundation Medicine, Roche. Advances in genomic testing are driving the personalisation of cancer care and helping physicians understand patients unique cancer types.

This uniqueness of each cancer case leads to precision and personalised medicine which is changing the treatment paradigm and offering a big promise to cancer patients, said Oliver Bleck, GM, Roche Pharma, Switzerland. He was addressing the media from different countries at a media event on The future of personalised healthcare: Advances in precision medicine and genomics held at the Roche Innovation Centre in Zurich.

The two-day event by Roche and its partner Foundation Medicine, a molecular insight company from the US, introduced to the international media the new emerging world of Comprehensive Genomic Profiling (CGP) leading to precision medicine and targeted & personalised healthcare. Several executives and researchers of Roche and Foundation Medicine, as well as academicians from universities made presentations on various aspects of genomic testing and its importance in understanding cancer and treating it.

Charlotte Colthorpe, Group Global Scientific Director, Medical Affairs Team Lead, Skin and Rare Cancers, Roche, said, Transition to precision medicine leads to paradigm shift in cancer care. But the main requirement of precision medicine is a massive data of patients. Researchers need large database so that even for the rarest type of cancer they have large sample size to understand the specific cancer.

Foundation medicine has been building such a database for the past nine years and has now one of the largest database containing genomic profiles of over 300,000 cancer patients, said Dr Prasanth Reddy, VP, Medical Affairs, Foundation Medicine. The Foundation Medicine is providing services like FoundationOne CDx which is first FDA approved broad companion diagnostic designed to provide physicians with clinically actionable information based on the individual genomic profile of each patients cancer. Its FoundationOne Heme is a comprehensive genomic profiling test for hematologic malignancies and sarcomas and FoundationOne Liquid is next-generation liquid biopsy test for solid tumors utilizing circulating tumor DNA (ctDNA). All these services are available in India too.

Josh Lauer, Global Head, Personalised Healthcare Market Development, Roche, said that for driving value from data and analytics what was needed was meaningful data at scale and advanced analytics. With high resolution view of each patient we have increasing level of information and the challenge was that how we use it perfectly, he added.

Benjamin Szilagyi, Business Lead, Enhanced Data & Insights Sharing, Roche, said that meaningful medical data gives exquisite insights in the disease in the last 30 years. When asked about the requirement of facilities like artificial intelligence (AI) for analysis of such a huge data, he said, before analysis even data management is a problem that will have to be looked into. Even in some developed countries the data is stored on paper, which needs to be changed."

"The data of Swiss patients is kept in the lab here only and the patients have the choice to give data for research or not", clarified Prof Holger Moch, Medical Director, Department of Pathology and Molecular Pathology, University Hospital Zurich (USZ). He explained how the role of pathologist has changed in molecular pathology. It is two-fold and evolving, he said.

He explained physicians are now able to personalise treatment to the unique molecular profile of the patient and thus precision medicine was driving personalised healthcare. He informed that Roche, Foundation Medicine and the USZ have joined forces in an academic-industry collaboration to improve personalised cancer therapy using comprehensive tumor profiling and to enhance personalised and targeted therapy options.

USZ runs the lab to do genome profiling and generate reports of the cancer patients in Switzerland on the basis of the Foundation Medicine system. "When a patient is diagnosed with cancer, his genome profiling is done here in the lab", said Dr Martin Zoche, Director, Molecular Tumor Profiling, USZ while showing the media persons functioning of the lab. The profiling costs $ 4000 in the US and the process takes 14 to 20 days.

Dr Abdullah Kahraman, Head, Clinical Bioinformatics in Molecular Tumor Profiling, USZ, explained about the report generation. Besides the gene alternations, the report also carries potential therapy options and relevant clinical trials. It is a simplified clear report, said Dr Merlene Thomas, Global Medical Lead for Integrated Molecular Medicine, Roche.

The report is then matched with the existing database to find out matching profiles of earlier patients and then put before the Molecular Tumor Board. The board facilitates its interpretation, assign precise treatment and provide education, she said. "How to integrate ever-increasing amount of information into clinical decision is a challenge", she added.

Prof Alwin Kramer, Head, Clinical Cooperation Unit, University of Heidelberg, who discussed the cancer of unknown primary (CUP), said that CUP incidences have decreased, probably due to improved diagnostics, but mortality has not improved. He added that CGP has the potential to transform how we care for even CUP patients as it opens up the possibility to bring personalised therapies to patients with CUP. He described the details of CUPISCO, a Roche-initiated, multi-cohort trial in CUP patients based on genomic profiling for comparing the efficacy and safety of molecularly-guided therapy versus platinum-containing chemotherapy in patients with newly diagnosed, previously untreated CUP. "It is planned at 128 sites in 32 countries over 790 patients", he said adding 193 patients have been enrolled till now. Every patient in the CUPISCO study will be discussed in molecular tumor board, said Prof Moch.

Prof David Thomas, Head, Cancer Division, Garvan Institute, gave details of WINTHER, I-PREDICT, TARGET clinical trials launched to study various aspects of CGP. He predicted that molecular guided therapy will be routine in clinical practice.

Later in a panel discussion he said, We are measurably improving patient survival. He pointed out that molecular testing can be wrong some time and we are re-analysing the data. Kramar said that over 80 molecular track treatments were approved. It was extremely difficult to do randomised studies, he added.

Bogi Eliasen, Special Advisor on Future of Health, Copenhagen Institute of future studies, gave details of Faroe genome project, a holistic genomic health approach in Faroe in Denmark. He said, Genomics is going to be the most important baseline to be worked with. Prof Roger Moos, President, Swiss Group for Clinical Research, said that 32 per cent of cancer patients in Switzerland were treated outside of treatment guidelines leading to lack of data collection. He gave details of the Swiss Group of Clinical Cancer Research (SKSS).

The event also included narrat ions of experiences by the cancer survivors. Prostate cancer survivor Bryce Olson, Global Strategist, Health and Life sciences Group, Intel Corporation; breast cancer survivor journalist Susan McClure, Founder and CEO, Genome Creative; and sister of a cancer victim Ruth Knott, Co-founder and Head of Governance, the SJK Foundation, presented their experiences of passing through cancer and cancer care respectively.

The one-size-fits-all approach to healthcare no longer cuts it, and it is time for people to understand that in order to get targeted treatments tailored uniquely to them, they have to do some form of genomic testing, said McClure, who has devoted her life to better educating the public about genomics.

Milind Kokje

(Milind Kokje was invited to participate in the Media Event at Zurich and his trip was sponsored by Roche)


Spotlight on Comprehensive Genomic Profiling - BSA bureau

Novel Dementia Vaccine Could Be the "Breakthrough" of the Decade – Interesting Engineering

New research has developed a vaccine designed to prevent the neurodegeneration associated with Alzheimers disease, and researchers hope this will be the "breakthrough of the decade," according to ABC News Australia.The researchers believe that this treatment is now ready for human trials.


The vaccine is created to produce antibodies that both prevent and remove amyloid and tau proteins in the brain, the accumulation of which is believed to be the main cause of neurodegeneration in Alzheimers disease.

Many previously failed Alzheimers treatments have focused either on amyloid or tau protein reductions, but never both. However, research increasingly suggests it is a synergic relationship between the two toxic proteins that may be responsible for neurodegeneration.

The novel vaccine is actually a combination of two vaccines. AV-1959R targets amyloid aggregations, and AV-1980R targets the tau protein ones.

Nikolai Petrovsky, a scientist from Australias Flinders University and one of the team's researchers, told ABC News Australia the new treatment could be both preventative and curative.

"It's actuallydesigned to be both a prophylactic and a therapeutic," he said.

"In the animal models, we can both use it to prevent the development of memory loss by giving it before the animal starts to get these build-ups of proteins. But we can also show that even when we give it after the animals have proteins, we can actuallyget rid of the abnormal proteins," added the researcher.

Now,Petrovsky hopes human trials can startin the next 18-to-24 months."It's an exciting time to be starting the new decade hopefully this is the breakthrough of the next decade if we can get it to work in the human trials," he said.

The research is being led and funded by the Institute for Molecular Medicine and the University of California, in the US. And we can only hope it ends up being a total success.

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Novel Dementia Vaccine Could Be the "Breakthrough" of the Decade - Interesting Engineering

Bioinformatics Market: Overview with Detailed Analysis, Competitive landscape Forecast to 2020 – Voice of Reports

Prominent participants in the global market for bioinformatics include ID Business Solutions, Ltd., Illumina, Inc., GenoLogics Life Sciences Software, Inc., Affymetrix, Inc., Life Technologies Corporation, Agilent Technologies, Inc., CLC bio A/S, and Accelrys, Inc. In order to march ahead, they are seen investing heavily in research and development of more cutting-edge products.

A report by Transparency Market Research projects theglobal bioinformatics markets worth to be around US$9.1 bn by 2018-end.

The global market for bioinformatics has been segmented broadly into platforms, content management, and services by the report. They are all commercially available for sorting the data accumulating form research and development projects by leveraging bioinformatics. Among them, the bioinformatics platform comprising of sequence alignment platforms, sequence manipulation platforms, structural analysis platforms, and sequence analysis platforms is leading vis--vis consumption. This is because of their growing application in the process of research and development for drug discovery. Geographically, North America leads the market on account of the huge allocations towards cutting-edge research and development, swift uptake of latest technologies, and a well etched out regulatory framework in place.

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Pressing Need to Cure a Range of Ailments Boosts Adoption of Bioinformatics

Bioinformatics finds application in managing the data emanating from research and development in the domain of life science, biopharmaceutical, and biotechnology. Its market has been seeing a steady upswing owing to the increasing usage of IT applications in the domain of healthcare sector to store, process, access, and retrieve data. Besides, the pressing need to treat several diseases effectively alongside the demand for preventive solutions are also having a positive impact on the market. Growing thrust on research and development has been a major catalyst too.

Study of Molecular Medicine and Genetics Augurs Well for Market

Bioinformatics finds usage in preventive medicine, molecular medicine, drug development, and gene therapy. Currently, the molecular medicine is stoking their demand majorly. They also find usage in genetic research for antibiotic resistance, veterinary science, and microbes. All these are decidedly impacting their market positively.

Elaborates the lead analyst of our report, The growth in the worldwide market for bioinformatics market is being propped up by the solid progress in the study of genetics. As per estimates, the volume of genetic data entries would rise twofold every 15 years. And with the total amount of generated data being too large to be supported by the present data entry programs, savvy companies are seen resorting to more advanced models of bioinformatics.

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Key Players Train Sights on Asia Pacific

At present, developed geographies of Europe and North America are where most of the development in the bioinformatics has occurred. This is because of the huge amounts allocated for research and development and swifter deployment of newest technologies. However, key players have also set their gaze upon Asia Pacific with its fast developing infrastructure in scientific fields, the improvements in government and private funding, and the rising literacy and awareness rates.

I am Sheila Shipman and I have over 16 years experience in the financial services industry giving me a vast understanding of how news affects the financial markets.

I am an active day trader spending the majority of my time analyzing earnings reports and watching commodities and derivatives. I have a Masters Degree in Economics from Westminster University with previous roles counting Investment Banking.

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Bioinformatics Market: Overview with Detailed Analysis, Competitive landscape Forecast to 2020 - Voice of Reports

Prenatal Diagnostics Market Insight on the Important Factors and Trends Influencing the Industry – BulletintheNews

Global Prenatal Diagnostics Market: Overview

Prenatal diagnostics is a procedure for testing the fetus before birth to determine certain hereditary disorders such as Downs syndrome, genetic diseases, chromosome abnormalities, neural tube defects, and other conditions. In case of high risk related to the birth of a child, it can assist by conducting numerous procedures of genetic testing common testing, and screening.

The global prenatal diagnostics market is segmented on the basis of tests into Harmony PrenaTest, BambniTest, NIFTY (Non-invasive fetal trisomy test), Verifi, informaSeq, informaSeq Panorama, MaterniT21 PLUS, and VisibiliT.

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In the report, Transparency Market Report (TMR) presents a comprehensive analysis of drivers and restrictions in the global prenatal diagnostics market. It also provides insight into the major segments and regions of the market.

Global Prenatal Diagnostics Market: Trends and Prospects

The global prenatal diagnostics market is driven by numerous factors such as growing demand for non-invasive prenatal diagnostics, rise in detection rates of Downs syndrome, technology advancements, and the crucial one being increasing age of mothers which develops the possibility and risk of chromosomal abnormalities. However, strict regulatory framework and traditional ethical outlook towards prenatal testing can limit the growth of the prenatal diagnostics. In addition, non-invasive prenatal tests and alternative screening and testing methods can also create hurdles for the market growth.

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On basis of tests, MaterniT21 plus segment will largely contribute to the market share, and is expected to lead the market during the forecast period. It is preferred over the other tests in the category as it is the first of its kind diagnostic system. Due to higher accuracy and safety panorama and verifi tests are also high in demand and is projected to register a strong CAGR during the forecast period. Moreover, increased market penetration and aggressive marketing strategies by companies are further propelling the growth of these tests.

Global Prenatal Diagnostics Market: Regional Analysis

The global prenatal diagnostics market is segmented on the basis of geography into North America, Asia Pacific, Europe, Latin America, and the Middle East and Africa. Among the mentioned regions, North America is expected to lead the regional segment. The factors leading to the growth of this region can be attributed to growing efforts of coalitions between companies and hospitals and increasing penetration of the testing procedures. In addition, due to increase in the rate of high risk pregnancies and rising average age for child bearing leading to development of chromosomal disorders, the market is said to propel over the forecast period. With increased penetration of companies and rising approvals by the European commission organizations, the European market is anticipated to thrive in terms of revenue.

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The Middle East and Asia Pacific are untapped territories for many established market players. With better marketing strategies and positioning, major companies are planning to venture in this region, thereby increasing the market growth. Moreover, supportive government research initiatives, rising disposable income, increasing paranoia of to-be mothers for the health of their child are some of the factors supplementing the market growth of non-invasive prenatal diagnostics.

Companies Mentioned in the Report

Some of the leading players in the global prenatal diagnostics market are Hologic Inc., Sequenom, Inc., PerkinElmer, Inc., Ravgen, Inc., Sequenom Center for Molecular Medicine, LLC, Abbott Molecular, Angle Plc., and TrovaGene, Inc.

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Prenatal Diagnostics Market Insight on the Important Factors and Trends Influencing the Industry - BulletintheNews

Why don’t bears’ muscles atrophy during hibernation? – Mother Nature Network

Some bears have a brilliant strategy for getting through winter: staying in bed.

Not all bears hibernate, of course, and even those who do may technically be in a state called torpor, not true hibernation. Nonetheless, a bear's long winter nap can spare her from life-threatening cold and hunger until the weather warms up.

Bears fatten up before winter arrives, then reduce their heart rate and metabolism during hibernation, letting them sleep through the worst of winter without needing to worry about food. But since hibernation can involve barely moving for months, how do bears avoid muscle atrophy during such a sedentary period?

That's what a team of researchers sought to learn with a new study on hibernating grizzly bears, published in the journal Scientific Reports. Aside from shedding light on bears themselves, this research could also benefit our species, the researchers say, by helping us limit the muscle weakness that often occurs when people are bedridden or otherwise immobilized for stretches of time.

"Muscle atrophy is a real human problem that occurs in many circumstances. We are still not very good at preventing it," says lead author Douaa Mugahid, a postdoctoral researcher at Harvard Medical School, in a statement. "For me, the beauty of our work was to learn how nature has perfected a way to maintain muscle functions under the difficult conditions of hibernation. If we can better understand these strategies, we will be able to develop novel and non-intuitive methods to better prevent and treat muscle atrophy in patients."

A male brown bear emerges from hibernation at Ranua Zoo in Finland. (Photo: Kaisa Siren/AFP/Getty Images)

While curling up to sleep all winter may sound nice, a protracted slumber like this would wreak havoc with the human body, Mugahid and her co-authors point out. A person would probably suffer blood clots and psychological effects, they note, along with significant loss of muscle strength due to disuse, similar to what we experience after having a limb in a cast or having to stay in bed for extended periods.

Grizzly bears, however, seem to handle hibernation pretty well. They may be a little sluggish and hungry when they wake up in the spring, but that's about it. In hopes of understanding why, Mugahid and her colleagues studied muscle samples taken from grizzly bears during hibernation as well as more active times of year.

"By combining cutting-edge sequencing techniques with mass spectrometry, we wanted to determine which genes and proteins are upregulated or shut down both during and between the times of hibernation," says Michael Gotthardt, head of the Neuromuscular and Cardiovascular Cell Biology group at the Max Delbrck Center for Molecular Medicine (MDC) in Berlin.

A brown bear trudges through snow in Finland. (Photo: ArCaLu/Shutterstock)

The experiments revealed proteins that "strongly influence" a bear's amino acid metabolism during hibernation, the researchers report, resulting in higher levels of certain non-essential amino acids (NEAAs) within a bear's muscle cells. The team also compared their findings from bears with data from humans, mice and nematodes.

"In experiments with isolated muscle cells of humans and mice that exhibit muscle atrophy, cell growth could also be stimulated by NEAAs," Gotthardt says. That said, however, earlier clinical studies have shown "that the administration of amino acids in the form of pills or powders is not enough to prevent muscle atrophy in elderly or bedridden people," he adds.

This suggests it's important for the muscle to produce these amino acids itself, he explains, since just ingesting them might not deliver them where they're needed. So, rather than trying to mimic a bear's muscle-protecting technique in the form of pills, a better therapy for humans might involve trying to induce human muscle tissue to make NEAAs on its own. First, though, we need to know how to activate the right metabolic pathways in patients at risk for muscle atrophy.

To figure out which signaling pathways must be activated within the muscle, the researchers compared the activity of genes in grizzly bears with those of humans and mice. The human data came from elderly or bedridden patients, they report, while the mouse data came from mice experiencing muscle atrophy, caused by a plaster cast that reduced movement.

"We wanted to find out which genes are regulated differently between animals that hibernate and those that do not," Gotthardt says.

A grizzly bear sow leads her cubs through snow at Grand Teton National Park. (Photo: Chase Dekker/Shutterstock)

They found lots of genes matching that description, however, so they needed another plan to narrow down the list of candidates for muscle-atrophy therapy. They conducted more experiments, this time with tiny animals called nematodes. In nematodes, Gotthardt explains, "individual genes can be deactivated relatively easily and one can quickly see what effects this has on muscle growth."

Thanks to those nematodes, the researchers identified several intriguing genes that they now hope to study further. Those genes include Pdk4 and Serpinf1, which are involved in the metabolism of glucose and amino acids, as well as the gene Rora, which helps our bodies develop circadian rhythms.

This is a promising discovery, but as Gotthardt points out, we still need to fully grasp how this works before we can test it in humans. "We will now examine the effects of deactivating these genes," he says. "After all, they are only suitable as therapeutic targets if there are either limited side effects or none at all."

Why don't bears' muscles atrophy during hibernation?

Researchers hope to help humans borrow some secrets of bear biology.

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Why don't bears' muscles atrophy during hibernation? - Mother Nature Network

Winning Women: The scientist whose mission is to kill TB ‘stone dead’ – News24

Professor Valerie Mizrahi, a recent recipient of the Harry Oppenheimer Fellowship Award of R1.5million, fights the devastating disease with a sharp scientific mind that has made her a global leader in her field, writes Sue Grant-Marshall.

Tuberculosis (TB) kills more people in South Africa than any other infectious disease. According to the World Health Organisation (WHO), there were 22 000 TB-related deaths and 56 000 deaths of people with TB and HIV in 2017. Globally, 1.6 million lives are lost every year.

This bacterium has tricks up its sleeve that enables it to cause disease to the extent that it overtook [the number of] HIV/Aids [patients] two years ago, says Professor Valerie Mizrahi.

The renowned researcher into the science of TB is widely recognised as a global leader in the field. She recently received the Harry Oppenheimer Fellowship Award worth R1.5 million to further the valuable work done by her team at the Institute of Infectious Disease and Molecular Medicine at the University of Cape Town (UCT). As its director, she oversees hundreds of scientific, technical and administrative staff, among them 300 postgraduate students and 85 postdoctoral fellows working at the institute.

Although science is at the very core of my being, Mizrahi has a remarkable ability to convey her strong views about the astounding toll that TB takes on humanity.

TB is caused by a bacterium [mycobacterium tuberculosis] that causes enormous damage to the lungs. It can be expelled with a cough or a sneeze. Anybody in the vicinity can breathe in the airborne pathogen and become infected, she says.

Normal TB that responds to drugs is curable, provided the patient takes the medication regularly for six months. But drug-resistant TB is much harder to treat as doctors have to resort to medications that are less effective, have more severe side effects and are much more expensive.

Because TB is a notifiable disease, it is the states responsibility to treat it. Although drug-resistant TB only makes up about 5% of the 10 million new cases worldwide annually, its burden and cost to society is disproportionately high, says Mizrahi.

This is the driver behind her research into new therapies.

However, we need a combination of new tools for controlling the disease such as vaccines, biomarkers for diagnostics and new drugs, she says.

The advent of HIV worsened the TB epidemic considerably. Between 50% and 70% of all TB cases in South Africa occur in people who are also infected with HIV.

TB is the leading opportunistic infection associated with HIV. With the widespread roll-out of antiretroviral therapy, we have begun to turn the tide and are seeing a slow decline in the incidence of TB.

We now have two new antibiotics that are showing great promise for the management of drug-resistant TB. But we need to do more, which will require adequate resourcing, she says.

Little black book

Workplace tip:Ive raced through life and achieved a lot at a young age. Sometimes its better to be less rushed.

Life lesson: If I was advising my younger self, I would say: slow down.

Mizrahi says that TB research is underfunded when measured against the number of people who contract TB and the many lives lost because of the disease.

Mizrahi emphasises the challenges involved in TB research: We need a vaccine it would be transformative if we could prevent people [from] getting this disease. There are also major diagnostic challenges.

We really have a poor understanding of the whole TB transmission process, she says. It is a major research focus for people working in this institute.

Mizrahis particular interest is in developing antibiotics that can kill this bacterium stone dead.

Were all working with the WHO to end TB. The WHO is pushing for a major reduction in the incidence of the disease by 2035 thats only 16 years away.

The energetic and approachable professor obtained her PhD in chemistry at UCT in 1983 and worked in the US for six years before returning home. She moved into TB research in 1993, the year the WHO declared TB a global health emergency.

She came back to South Africa because she wanted to have children and bring them up surrounded by my family in Johannesburg. I do not know how I would have coped without my mother and extended family support structure.

Mizrahi, who has received numerous national and international awards for her work, says that, when she joined the world of science, there were only a few women in leadership positions.

Research requires obsession, dogged perseverance and long working hours. Its also hard to switch off when you get home. My two daughters now in their twenties would ask: Mom, are you listening?

The professor says she might make a significant shift in her career in the next few years.

But it will always involve youngsters, because the world of science belongs to them.

Id like to continue to train and develop young people, and will do so for as long as I can walk up these steps to my office.

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Winning Women: The scientist whose mission is to kill TB 'stone dead' - News24

MPM backs new biotech in the hunt for non-opioid painkiller – Endpoints News

A new MPM Capital-backed company is joining the search for a better way of treating chronic pain.

Six years after its quiet birth out of a St Louis lab, BioIntervene unveiled an MPM-led $30 million Series A funding round to take its lead drug, BIO-205, into human proof-of-concept studies. They announced a new CSO too: Charles Cohen, a neuroscience veteran of Merck, Bayer, Vertex and most recently Xenon.

BIO-205 demonstrates profound activity in multiple models of neuropathic and inflammatory pain as both a single agent and in combination with morphine and gabapentin, Cohen said in a statement. If we can reproduce these data in our clinical trials, BIO-205 has the potential to ameliorate tremendous suffering while also playing a key role in addressing the opioid epidemic.

You can add BioIntervene to the growing number of biotechs searching for new methods of pain relief a long-dormant hunt thats picked up in the wake of the opioid abuse epidemic. Some of that research has focused on repurposing NSAIDs such as acetaminophen. More commonly, though, researchers have looked for new neural pathways.

That includes groups like Xenon, who have focused on inhibiting the NaV1.7 sodium receptor although Roche and Biogen both abandoned their NaV1.7 programs in 2018 amid clinical failure. Eli Lilly-partnered Centrexion is using a compound found in chili peppers that targets the TRPV1 receptor. Still,others have tried to find better opioids based on a concept called biased agonism. Most of the work is early-stage.

BioIntervene comes out of Saint Louis University professor Daniela Salveminis work on the A3 adenosine receptor (A3AR). The four different adenosine receptors had long been investigated for the roles they may play in cancer, inflammation and immune-related diseases.

But in 2012 Salvemini began publishing work suggesting that an A3AR agonist could turn off pain signals from the spinal cord. In 2014, she formed BioIntervene to develop that work into treatments of chronic pain.

The agonists are theorized to work by restoring a form of neurotransmission called GABA signaling that is disrupted in certain forms of pain. Over the 6 years leading up to the Series A, Salvemini published animal studies showing the agonists potential to treat the pain from cancer drugs and other causes.

Neuropathic pain has been a particularly thorny area for drug developers, with the few existing drugs coming with significant side effects. Salvemini has insisted their receptor can curb pain without the side effects.

The protective effect of A3AR agonists in several models of chronic neuropathic pain including that caused by widely used chemotherapeutic agents does not desensitize over time with constant drug exposure, and there is no risk of addiction, she wrote in a 2015 note in Expert Opinion on Therapeutic Patents.

BioIntervene will focus first on pain, but theyre exploring one other therapeutic area: neurodegeneration. There are few details yet but Salvemini published a patent in November for using A3 agonists to ameliorate mitochondrial injury.

Thatll make two areas of high unmet medical need for the young biotech two that have ensnared a long list of biotechs before them.

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MPM backs new biotech in the hunt for non-opioid painkiller - Endpoints News

God Willing, He Will Cure Cancer, and Shrimp – CTech

Avi Schroeders lab at the Technion Israel Institute of Technology is tiny but mighty. Thirty researchers crowd it daily, covering areas such as biology, chemistry, medicine, pharmaceuticals, and even mechanical engineering. The lab is part of the chemical engineering faculty, but the researchers here were handpicked from different departments to serve the labs interdisciplinary mission. The content of the multitude of test tubes here could one day result in life-saving medication for a variety of conditions. Until then, the lab has already birthed several innovations that matured into business ventures: Schroeder and his team are involved in four such companies based on the labs scientific breakthroughs, ranging from using elephant protein to cure cancer to personalizing chemotherapy.

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God Willing, He Will Cure Cancer, and Shrimp - CTech

Theragnostics Expands Team with Significant Appointments – PRNewswire

LONDON, Jan. 10, 2020 /PRNewswire/ -- Theragnostics, which is developing molecular radiotherapy for imaging and treating a broad range of cancers, announces the appointments of Dr. Alex Jackson as Head of Research and Development and of Archie Hale as Director of Clinical Operations. This announcement follows the recent appointment of radiopharmaceutical drug development expert Dr. Daniel Stevens as Chief Medical Officer.

Dr. Jackson has over 20 years' experience of conducting and overseeing research activities in both academia and industry. He joins Theragnostics from GE Healthcare where he was responsible for the discovery phase of immune-oncology PET projects and for a multi-centre late stage clinical PET tracer development programme. Alex holds a PhD in Synthetic Chemistry from the University of Warwick and he is the author of 15 publications on molecular imaging and radiochemistry.

Archie Hale is a seasoned pharmaceutical industry professional with extensive experience across all stages of drug development for several leading companies including Biogen, BTG and Roche as well as smaller biotech companies and she will be conducting Theragnostics' variousclinical programmes.

Greg Mullen, Chief Executive Officer of Theragnostics, said: "I'm delighted to welcome Alex and Archie to the team as we continue to grow with the aim of bringing novel targeted therapies to cancer patients. This year, we have made great progress in strengthening our platform and expanded our team. As we enter 2020 and prepare for the new year ahead, we are well placed to submitting an NDA for Tc-99m-DMSA, initiating a PhI/II I-123 PARPi therapeutic trial and commencement of a F-18 PARP PhII and Ga-68 PSMA PhIII diagnostic clinical studies."

Theragnostics' technology platform enables the development of molecular radiotherapy based on a PARPi for imaging and treating cancer. Theragnostics modifies a PARPi drug molecule with a radioactive atom to create a radionuclide PARPi (rPARPi). This can either be used to image PARP in a cancer patient for diagnostic use or the radioactive isotope can be used to deliver a therapeutic dose of radiation into tumour cells, which offers the potential to molecularly target the radiation in order to hit and kill tumour cells whilst avoiding damage to healthy cells and associated side effects.

Theragnostics' management team will be attending the upcoming 38th Annual JP Morgan Healthcare Conference in San Francisco from 13-16 January 2020 to meet investors and pharmaceutical partners.

About Theragnostics

Theragnostics, a private biotech company based in UK and US, is developing innovative radiopharmaceuticals to improve cancer diagnosis and treatment. It has developed a radiopharmaceutical technology platform that allows for diagnostic tools as well as therapeutic solutions to inform clinical management, guide care and treat cancer patients in areas of unmet medical need. Three diagnostic imaging agents have been successfully developed. The first radiopharmaceutical therapy, rPARP inhibitor THG-009 (I-123 labelled PARPi) is expected to enter clinical development in 2020. Theragnostics is led by recognized experts in the clinical development and commercialization of innovative diagnostic and therapeutic nuclear medicine products.

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

For media and investor enquiries please contact:

Optimum Strategic CommunicationsMary Clark, Supriya Mathur, Manel Mateus theragnostics@optimumcomms.comTel: +44(0)20-950-9144

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Theragnostics Expands Team with Significant Appointments - PRNewswire

FedEx for cancer medicine, Cyca OncoSolutions wants to reduce the side effects of treatment – Economic Times

The problem with the current line of cancer treatment is that it is akin to carpet bombing it does not differentiate between healthy cells and the cancerous one. As a result, chemotherapy sometimes has side effects that are more debilitating than cancer itself.

As many as 90 people die every hour due to cancer in India, research statistics suggest. Moreover, the estimated number of people living with the disease stands at around 2.25 million in the country, with the top five cancers in men and women alone accounting for 47.2% of all cancers.

What compounds the suffering of patients is also the high toxicity of the cancer drugs taken during treatment. Ironically, for Nusrat Sanghamitra, the journey into the world of entrepreneurship started on a deeply personal note. But it is one which motivated her enough to carry forward the journey in a way that could help those suffering from the damaging effects of the disease.

In 2001, Sanghamitra, founder of pharma startup CyCa OncoSolutions, was pursuing her PhD when her father was diagnosed with prostate cancer. This prompted her to find a better way that could make cancer drugs safer and better. In my perspective, we can make cancer more manageable, for instance, just like fever by making cancer drugs non-toxic, yet effective like Paracetamol, she says.

Sanghamitra deviated from her initial plan when she was solely finding a better drug for cancer by choosing chemistry as a subject in BSc. Later on, during her PhD research, she saw that the lead molecule as seen in the results was extremely toxic in vivo. I was disheartened by that and felt the need to understand the mechanism of action of the drugs. So I shifted my research area from chemistry to protein engineering to biophysics and cell biology to find a method for reducing the toxicity of the existing drugs, she recalls.

What doesnt kill you makes you weakerHer eureka moment eventually followed. It came by with the serendipitous discovery of a novel molecule that could help in reducing the side effects of the existing cancer drugs. This happened during my tenure at Kyoto University. Based on that discovery, I developed a hypothesis and came back to India. I got a Biotechnology Ignition Grant to prove my hypothesis, she says exuberantly.

Aligning chemotherapys effects to being as toxic as that of carpet bombing, she explains her rationale for holding this belief. It kills cancer cells as well as normal, healthy cells and the toxic side effects make 15 million cancer patients worldwide really sick during the process of healing. This causes reduced quality of life (often high mortality) leading to emotional and financial trauma of not just the patient, but for the whole family, she says, reflecting on the nature of the treatment.

The side effects, she explains, are due to the usage of massive doses of drugs because every human cell has a protective cell membrane that does not allow foreign molecules to enter into the cell. And crossing this barrier is vital for drugs to show efficacy. Though the startup initially began by reducing the dose of cancer chemotherapy, they found that crossing the living cell membrane is as crucial for improving the effects of other drugs. Hence, they expanded their product line.

The core technology of the startup is Universal Sliding Gateway (USG). As USB is for your computer, USG is for living cells to transfer biological information, Sanghamitra quips.

USG, in essence, is a high speed, high precision molecular nanomachine that can carry drugs directly into living cells like a delivery vehicle. It is like FedEx for molecules. USG delivers drugs right into the target cells. It can transform drugs from poison to remedy and give new life to the old drugs, she explains.

The technology claims to have no toxic side effects and adverse immune response so far (as observed in mice) and healthy human blood cells.

Skeptics galoreSanghamitra feels that on a journey like hers, skeptics have often raised eyebrows about the work that is being done. Many also questioned the premise on which the startup was founded, including scientists working in the field of cancer research. At times, I am indirectly told that this is not going to work. Once a scientist from a premier research institute of India and, ironically, also researching on the subject questioned me about why I think the side effects of cancer therapy are such a problem. I was taken aback by his callous response, she says.

Moreover, she found that success in her field is ascertained more by aspects such as a faculty position in a premier research institute as well as the number of publications in high impact journals. When I started off with a big grant, my contemporaries did not really understand what I was doing. Many assumed that I did not get a faculty position and so I took up grant to pay my bills, she recalls.

Besides the naysayers, there have been other blocks impeding her growth as an early stage startup too. Building a team and retaining skilled employees, finding access to high-end equipment and infrastructure needed by her project have been other challenges that still continue to play up.

The tough get goingAll this has hardly deterred Sanghamitra from pulling all the stops in making her dream come to fruition. Besides winning a lot of awards and accolades as a technology innovator and a Biotechnology Ignition Grant of $100,000 by Biotechnology Industry Research Assistance Council (BIRAC), she has her plan chalked out to go forward.

In 2018, the startup also received a grant of $140,000 under Bharat Petroleum Corporation Limited's (BPCL) Project Ankur scheme.

Their commercialisation strategy is to have strategic co-development partnership with oncology driven pharma companies as well as Contract Development and Manufacturing Organisations (CDMOs) for product development. This will lead to out-licensing of USG to the oncology driven pharma companies.

Sanghamitra says that being a pharma product in India mandates the approval of Central Drugs Standard Control Organisation (CDSCO) and internationally, United States Food and Drug Administration (USFDA) to be launched in the market. Both CDSCO and USFDA need the five stages of Preclinical, Clinical Phase I, Clinical Phase II, Clinical Phase III and Phase IV to be successfully passed for approval. We aim to complete our preclinical trials by mid 2021, she avers.

In terms of expansion plans, the startup has moved from oncology to antibiotics and gene therapy in its product verticals. There has been movement geographically as well. We have an Irish company in Cork, Ireland. We will complete our preclinical trials with a seed round of $1 million. But we will move to the golden triangle area in London or in Boston to get access to the series A round in which we expect to raise $10 million. We envision going for a pre-revenue IPO, she says, exuding an air of confidence.

Sanghamitras vision to make it all a reality comes through in her words. Cancer is not a single disease because its therapy and related side effects cause quite a few diseases that reduce the quality of life of cancer patients. It is scary. Many patients discontinue the therapy during this time. So I cant emphasise enough the need to reduce the dose and side effects. We are on a mission to do that, she states emphatically.

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FedEx for cancer medicine, Cyca OncoSolutions wants to reduce the side effects of treatment - Economic Times

Nuclear Medicine Market 2020 Research Report: Global Industry Trend Analysis, Top Manufacturers, Product Types, Applications, Growing Demand, Regions…

Global Nuclear Medicine Market 2019-2025 Overview

The nuclear medicine market is projected to reach USD 18.10 billion by 2025 growing with CAGR of 10.1% during the forecast period. The growing demand for radioisotopes in cancer imaging is a major driver for the nuclear medicine market.

The market research report on nuclear medicines evaluates the market demand, trends and opportunities for the period 2015 to 2025. The report highlights the historic trends from 2015 to 2017 with base estimates for 2018 and market forecast from 2019 to 2025. The report also studies the current status and future aspects the market at global as well as country level along with vendor landscape analysis and value chain analysis. The global nuclear medicine market has been segmented based on type, diagnostics and application. Furthermore, the report gives in-depth analysis of the market competitors along with company profiles of key manufacturers.

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Cancer is the most dreaded and leading cause of death worldwide. With the development of various radioisotopes for diagnostics in oncology, nuclear medicines have found new source of growth. Nuclear medicines are widely used for tumor localization, tumor staging, identifying metastatic sites and judging response to therapy.

The most common isotope used for diagnosis is Technetium-99 with around 40 million procedures performed worldwide. Technetium-99 is so adaptive that when it is taken up by a biologically active substance, its traces settle accurately into the tissue or organ of interest.

In diagnostic procedures, nuclear imaging serves a vital purpose in patient care. PET is more precise and sophisticated technique used for nuclear imaging as compared to SPECT. Moreover, hybrid imaging techniques such as PET-CT and PET-MRI are gaining traction these days due to improved functional information and reduced radiation exposure.

Read more details of Global Nuclear Medicine Market at:https://www.adroitmarketresearch.com/industry-reports/nuclear-medicine-market

North America will dominate the global nuclear medicine market in 2025 with more than 35% market share. Growth in this region is attributed to perpetual improvement and development of molecular imaging based diagnostics and treatment. However, Europe is expected to show lucrative growth in the foreseeable years as it is hub for isotope suppliers.

The major consumers of nuclear medicines are spread across North America, Europe and Asia Pacific. In 2018, North America was a major market for nuclear medicines however, the Asian countries are expected to outpace other regions by 2025 growing with a CAGR of 13.3%. Japan, in particularly is witnessing tremendous growth in the use of nuclear medicines. In China, the players are concerned about lack of intellectual property protection for their formulations. Also, the rapidly ageing population in China is seen as an opportunity for growth of nuclear medicine in this region due to the rising number of people with age related cancer. However, in less developed countries like Indonesia, the modality is seriously underutilized.

The major players operating in the global nuclear medicine market include: Curium, Nordion Inc., Cardinal Health Inc., Bracco Imaging S.p.A, Eczacibasi-Monrol Nkleer rnler San. ve Tic. A.S., Lantheus Medical Imaging, Inc., Jubilant Pharma. LLC, Ire-Elit SA, GE Healthcare Company, NTP and others. Due to the growing demand of nuclear medicines in health sciences, the players are forming strategic alliances and outsourcing the production of few radioisotopes to gain momentum in the industry. In line with this, Curium was founded in 2017, when IBA Molecular and Mallinckrodt Nuclear Medicine underwent partnership. This alliance was specifically formed to fulfill the growing demand of Technetium-99.

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1. Blogs on Advantages And Disadvantages Of Nuclear Medicine

We all know how beneficial nuclear energy is. It is a good source of electricity and it is also used in weaponry. But, another field that is using nuclear power and transforming lives is the field of medicine. The branch of medical science that uses nuclear energy is called nuclear medicine. Nuclear medicine has given new hope to the illness and ailments difficult to treat. Thanks to technological advancements, it is being used in the right way and the right amount.

Advantages of Nuclear Medicine:

Nuclear medicine has digitally and technically enhanced the treatment options for various medical conditions. One such ailment is cancer. Cancer treatment involves radiation and chemotherapy. In severe cases, when patients have lost hope of survival, nuclear medicine has been a blessing to them.

Nuclear medicine is extremely capable of detection of highly severe medical conditions. Their precision is what makes people believe in its power. It has helped many physicians and other medical professionals detect difficult possibilities in early stage of treatments.

Read More Details of[emailprotected]https://www.adroitmarketresearch.com/blogs/advantages-and-disadvantages-of-nuclear-medicine2. White Paper on Nuclear Medicine Advanced Associate: What It Needs Right Now?

A team to study the existing program for Nuclear Medicine Advanced Associate was assigned by the president of the Society of Nuclear Medicine and Molecular Imaging, Technologist Section July 2013.

The task force included a range of stakeholders across profession, these included practicing technologists, research technologists, NMAAs, corporate representatives, educators, and physicians. Post the study various programs were identified which included business administration and masters degree. However, there is a lack of programs for technologists that are interested in careers across clinical research. This was mainly in field of nuclear medicine and molecular imaging.

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Key Segments of the Global Nuclear Medicine Market

Type Overview, 2015-2025 (USD Million)

Diagnostics Overview, 2015-2025 (USD Million)

Application Overview, 2015-2025 (USD Million)

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Adroit Market Research is an India-based business analytics and consulting company incorporated in 2018. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable market insights to help create opportunities that increase their revenues. We follow a code Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

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Ionis finds new partner in a young biotech and its hopeful founder – Endpoints News

Omri Gottesman came to the US from the UK 10 years ago, wide-eyed like many others.

The human genome project was long-complete and, with great fanfare, researchers were beginning to try and leverage those insights into treatments. Mount Sinai School of Medicine offered him a fellowship focused on just that: genomic medicine.

At the time, there was a lot of hope and hype that we had solved health, and would be able to discover and prevent everything and treat everyone, Gottesman told Endpoints News. It was the reason I came to Mount Sinai.

Over the ensuing decade, human biology proved much more elusive than they imagined. Genetics was more complicated than A-T and C-G. There were no easy hacks. Still, the field advanced and after 4 years at Mount Sinai and a stint at Regeneron, Gottesman decided he wanted to advance it on his own and build a new platform around the original mission: Analyze genetic information to home in on ideal drug targets. He called the new company Empirico and soon raised $30 million.

Its not a unique concept, but its one Gottesman pulled off well enough to today score a three-year partnership with Ionis Pharmaceuticals, one of the oldest and more successful genetics-based drug developers.

Ionis will make a $10 million equity investment into Empirico and offer $30 million in near-term operational and preclinical milestones, with another $620 million promised for clinical, regulatory and sales goals. In exchange, Empirico will make up-to 10 platform-discovered drug targets available to Ionis.

We spent about a year building the discover-first platform, Gottesman said. Part of the motivation for the Ionis collaboration and exploring collaborations more broadly is that we now have more potential targets than our capacity to develop therapies for them.

On the Ionis side, this is the latest collaboration for a biotech that has sought many since its founding and is trying to keep early-stage strong while some late-stage programs come to fruition. The deal also includes an option for Empirico to license a drug back from Ionis as it strives to become a target-to-market company.

Youve heard of platforms like Empirico before; its the mixed machine learning and wet lab approach taken by a growing number of biotechs. Gottesman and his team use their computer platform to interrogate genetic data for the roots of diseases. Should the computer find a mispelling or other variant in genetic code that appears to cause the malfunction, they then produce cell lines with that mispelling in the lab. Should those cell lines show the same malfunction as the disease, they then use their platform to identify the best form for a therapy.

Theyve used that method to build 5 preclinical targets, leading to a small molecule program for an upper airway disease and an siRNA candidate for glaucoma.

The targets for the Ionis collaboration will be those Empirico finds can be best-treated with their partners signature antisense technology. The biotech has already identified a couple, although they didnt disclosed them.

A 40-year-old concept, antisense therapies have recently brought some of the genetics-based health gains Gottesman and others sought. Along with a handful of approved drugs, last year a neurologist in Boston created a personalized therapy for a young girl with a never-before-seen genetic error. And yesterday, Ionis founder Stanley and Rosanne Crooke launched a charity to make those kinds of genetics-based ultra-personalized medicines broadly accessible.

Those bespoke therapies are arguably the apex of the vision Gottesman and others had 10 years ago. Its a vision he thinks has made more progress than some think, even if it hasnt brought any utopian dream.

Over the last decade, weve realized its really harder than it seems, he said. But actually, if you work at it, theres real value.

Social image credit: Ionis via YouTube

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FDA rushed to approve new drugs in 2019, sometimes way ahead of schedule. Could that be dangerous? – Endpoints News

While the roster of 48 drugs (plus 4 notable biologics) the FDA approved in 2019 didnt break any records with the sheer number it was remarkable in one aspect: The agency reached deep into a group of therapies they didnt need to make a decision on until later this year, in some cases speeding drugs up by months ahead of even priority review deadlines.

On the surface, thats great news for drugmakers and the patients they hope to help. But according to a recent study on rushed approvals, it could also spell safety troubles down the road.

Back in July, a trio of economists at NBER crunched the data on drugs OKd before supposed deadlines such as the end of months, years or before holidays and found that treatments approved under these apparent desk-clearing efforts in which regulators felt the pressure of informal performance benchmarks are more likely to be associated with postmarket safety issues.

We see about twice as many adverse effects, Lauren Cohen, a professor of finance and entrepreneurial management at Harvard Business School and one of the authors, told the Wall Street Journal.

Cohen and his colleagues, Umit Gurun of the University of Texas at Dallas and Danielle Li of MIT, considered only the surge of approvals in December, at the end of each month and before holidays such as Thanksgiving.

In 2019 seven out of 48 novel chemical entities were approved in December, largely in line with the 15% proportion that the researchers noted in previous years. But notably, 21 OKs came through in Q4, accounting for 43% of the whole crop.

FDA spokesperson Nathan Arnold acknowledged the December jump pattern but told the Journal that its share has actually diminished from the 1980s.

While we cannot speak directly to the results on informal deadlines highlighted in this study, FDA hason multiple occasionsinvestigated a closely related issue, which is the relationship between formal PDUFA deadlines and postmarket safety of drugs, Arnold added. We have not found evidence of such a relationship.

Drugs that appeared to have been approved with little heed of the PDUFA deadlines might be another issue. Vertexs Trikafta (for cystic fibrosis), BeiGenes Brukinsa (for mantle cell lymphoma), Novartis Adakveo and Global Blood Therapeutics Oxbryta (for sickle cell disease), as well as Alnylams Givlaari (for acute hepatic porphyria ) and AstraZeneca/Daiichi Sankyos Enhertu (for HER+ breast cancer) all belonged to that group.

Cohen, Gurun and Li prescribed a solution for the safety concerns they spotted:

A potential policy response to the patterns we document is to more carefully scrutinize drugs that are approved during such output surges. Regulators could, for instance, review end-of-year decisions in the following year before providing an ultimate approval (or lack thereof).

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FDA rushed to approve new drugs in 2019, sometimes way ahead of schedule. Could that be dangerous? - Endpoints News

Molecular mechanism suggests new ways to bolster immunity to deadly rotavirus: U of T researchers – News@UofT

Researchers at the University of Toronto have discovered how a brief disruption to a molecular pathway in the guts of mice before they are born can compromise immunity in adulthood to a common and often deadly intestinal virus.

The researchers found that in utero inhibition of molecular signalling in the lymphotoxin pathway, long known as important in the development of the immune system, prevented a robust antibody response in adult mice to rotavirus. In humans, rotavirus causes an estimated 215,000 deaths annually, mostly in the developing world.

That early disruption limits the ability of the immune system to later trigger and generate production of Immunoglobulin A (IgA) antibodies, the researchers showed. It also interferes with the nature and function of cells in the gut that support the antibody response, called mesenteric lymph node stromal cells.The research was recently published in the journal Science Immunology.

It was surprising that these non-immune stromal cells were so important to the immune response, saysJennifer Gommerman, a professor ofimmunologyin U of Ts Faculty of Medicine and principal investigator on the study.

It turns out that stromal cells affect the ability of immune B cells to produce IgA that neutralizes rotavirus. Were just beginning to understand the influence these stromal cells can have.

Gommerman says the findings highlight the growing importance of research on the environment in which immune cells function. We typically think of a lymph node as just a bag of lymphocytes, but there is also this supporting structure that clearly has an active role in shaping immunity.

The studys first author, post-doctoral researcherConglei Li, identified a broad subset of stromal cells that affect the immune response to rotavirus. But the key players are likely a subset of that subset, Gommerman says. New technology known as single-cell RNA sequencing should soon enable researchers to identify many more of those cells, she adds.

That work could, in turn, lead to a better understanding of the genetic and environmental factors that may undermine immunity to rotavirus in the developing world, where rotavirus vaccines are much less effective than in high-resource settings.

Gommerman says that while several dysfunctions in the immune system likely contribute to reduced immunity to rotavirus in low-income countries, the current study offers a hint that prevention may be possible.

The thinking would be that if youre pregnant in a resource-depleted area, you may take a dietary supplement at a specific point to ensure proper development of tissues that support immunity, and which enable a vaccine to be more effective, she says.

That kind of intervention is likely a long way off, adds Gommerman, and replicating her results in human pregnancy presents obvious ethical problems. A more immediate next step for her lab is a collaborative study on IgA immune responses to other pathogens such as norovirus, another highly contagious disease.

A focus on single pathogens is useful in studies of IgA, according to Gommerman, because so many factors can influence IgA response. If you simplify the system of study, you get more predictable kinetics and can ask more discrete questions, she says. Weve made a contribution with that approachon a question that has been percolating in several labs for years. That feels good.

The research received support from the Canadian Institutes of Health Research, Princess Margaret Cancer Foundation and the Swiss National Science Foundation.

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Molecular mechanism suggests new ways to bolster immunity to deadly rotavirus: U of T researchers - News@UofT

Hoth Therapeutics Announces the Appointment of Dr. William Weglicki, M.D. to Scientific Advisory Board – P&T Community

NEW YORK, Dec. 17, 2019 /PRNewswire/ -- Hoth Therapeutics,Inc. (NASDAQ: HOTH), ("HOTH" or the "Company"), a biopharmaceutical company focused on developing new generation therapies for dermatological disorders such as atopic dermatitis, chronic wounds, psoriasis and acne, today announcedthe addition of Dr.William Weglicki, M.D. to the Company's Scientific Advisory Board. While serving on the Board, Dr, Weglicki will oversee Hoth's Aprepitant Program under its Scientific Research Agreement with the George Washington University ("GW").

Mr.Robb Knie, CEO of Hoth Therapeutics, commented, "I am pleased to welcome Dr. Weglicki to our Scientific Advisory Board.Dr. Weglicki will be instrumental towards the advancement of our Aprepitant program.Hoth is fortunate to have such well-respected leaders providing their invaluable expertise and insight to each of our significant research and development initiatives."

Dr. Weglicki isProfessor of Biochemistry and Molecular Medicine at the George Washington University School of Medicine.Dr Weglicki is a recipient of the Lifetime Achievement Award in Cardiovascular Science, Medicine and Surgery, The International Academy of Cardiovascular Sciences. After completing a medical residency at Georgetown University Hospital in 1964 he completed a Cardiology fellowship in 1966 at Duke University where he trained in clinical cardiac catheterization and pharmacology basic research. He was then appointed as a research associate at NIH's NICHHD and the McCollum Pratt Institute of the Johns Hopkins University where he studied the cardiac pathobiology of alpha tocopherol deficiency. In 1968 He joined Harvard's Peter Bent Brigham Hospital Department of Medicine's Cardiovascular Research unit, androse from Instructor to Associate Professor in 1975. There he concentrated his research on the role of phospholipases of the heart in the ischemic perturbation of cardiac membrane phospholipids and organelles, which led to several RO1 NIH grant awards. With his research team he moved to the Medical College of Virginia/VCU in 1975 as chairman of the Department of Biophysics and served as a professor of Medicine (Cardiology); there he was active in teaching medical and mentoring graduate students, while publishing a series of manuscripts on cardiac lysosomal activity in myocardial ischemia and the distribution of phospholipases in cardiac mitochondria and sarcolemmal membranes, and overseeing a new NHLBI Training Grant on molecular mechanisms of cardiovascular injury. At the Oklahoma Medical Research Foundation he led their Cardiovascular Research Program. He joined the George Washington University Department of Medicine in 1985 and formed the Division of Experimental Medicine; in 1987 this core group of investigators was awarded a Program Project grant on Molecular Mechanisms of Cardiovascular Injury from the National Heart, Lung, and Blood Institute. This research included identifying free-radical peroxidative injury during myocardial ischemia and the protective antioxidant properties of cardiac beta blocking drugs such as propranolol and carvedilol. At present he is a Professor of Biochemistry and Molecular Medicine and Medicine (Cardiology). He has been a principal investigator of NIH grants for more than thirty years. His ongoing research focuses on preventing neurogenic inflammation and hyomagnesemia effects associated with some anticancer drugs.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, and acne. HOTH has the exclusive worldwide rights to BioLexa, the company's proprietary lead drug candidate topical platform that uniquely combines two FDA approved compounds to fight bacterial infections across multiple indications. HOTH is preparing to launch its clinical trial for the treatment of adolescent subjects, 2-17 years of age, with mild to moderate atopic dermatitis during 2020. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2018, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto / Daniela Guerrero(212) 896-1254 / (212) 682-6300Hoth@kcsa.com

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Hoth Therapeutics Announces the Appointment of Dr. William Weglicki, M.D. to Scientific Advisory Board - P&T Community

Ribon Therapeutics Strengthens Scientific Advisory Board with Appointment of Neal Rosen, M.D., Ph.D. – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ribon Therapeutics, a clinical stage biotechnology company developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions, today announced the appointment of Neal Rosen, M.D., Ph.D., to its scientific advisory board (SAB). Dr. Rosen is a Member of the Molecular Pharmacology Program and the Department of Medicine at Memorial Sloan Kettering Cancer Center (MSK).

Neals experience identifying and elucidating new molecular signaling pathways and understanding how to transform these early discoveries into effective drug development strategies fits perfectly with the work were undertaking at Ribon, said Victoria Richon, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. We see great untapped potential for the development of novel, first-in-class therapeutics that target the novel stress response pathways that many cancers rely on for survival, and working with experts such as Neal, who have successfully forged new paths in drug development will be invaluable.

Targeting stress response pathways to treat cancer has great potential, and shares similarities with the discovery and therapeutic targeting of kinase pathways, an area I have spent much of my career working to better understand and advance, commented Dr. Rosen. It is now understood that stress response pathways play a wide range of roles aiding cancer development and growth. I look forward to working with this truly pioneering team.

Ribon has done tremendous work taking its foundational discoveries and translating them into development programs, as evidenced by how quickly the Company was able to advance their lead program, RBN-2397, into the clinic, said Jim Audia, Ph.D., Chairman of the Ribon SAB. At this point in the Company's development, Neal is a fantastic addition to Ribons already stellar SAB, bringing his vast experience in the development and understanding of novel cancer targets and therapeutics to Ribon's developing pipeline.

Dr. Rosen's major interests involve identification and study of the key molecular events and growth signaling pathways responsible for the development of human cancers, and the use of this information for the development of mechanism-based therapeutic strategies. Dr. Rosen has pioneered the concepts that feedback inhibition of physiologic signaling is an important consequence of oncogene activation that shapes the phenotype of cancer cells and that relief of this feedback in tumors treated with inhibitors of oncoprotein-activated signaling causes adaptive resistance to these drugs. Recent work from the Rosen laboratory includes the elucidation of the underlying mechanisms whereby mutated BRAF genes cause cancer and the discovery that these mutations may be divided into three different classes that determine the effective strategies for their treatment. These studies predicted several of the cellular mechanisms whereby tumors develop acquired resistance and adaptive resistance to standard therapy and the discovery and development of new drugs that will reverse this resistance. Recently, the Rosen laboratory has also focused on the development of the first direct inhibitor of RAS, a gene involved in the development of 25% of human cancers. This work, in addition to other recent studies by the Rosen laboratory on the consequences of relief of negative feedback by oncoprotein inhibitors, has led to multiple clinical trials of combination therapies at Memorial Sloan Kettering and other cancer centers in the United States and internationally that have shown promising early results. He is the incumbent of the Enid A. Haupt Chair in Medical Oncology at MSK and the recipient of the Lifetime Achievement Award from the Society for Melanoma Research.

Dr. Rosen received his undergraduate degree in chemistry from Columbia College and an M.D. and Ph.D. in Molecular Biology from the Albert Einstein College of Medicine. He completed a residency in Internal Medicine at the Brigham and Womens Hospital, and postdoctoral training and a fellowship in Medical Oncology at the National Cancer Institute. He was on the senior staff of the Medicine Branch at the NCI prior to joining the faculty of MSK.

RBN-2397 Inhibiting PARP7, a Key MonoPARP Cancer Dependency

Ribons lead program, RBN-2397, is focused on inhibiting overactive PARP7 in tumors, which has been shown to play a key role in cancer survival. Ribons research has discovered that many cancer cells rely on PARP7 for intrinsic cell survival, and that PARP7 allows cancer cells to hide from the immune system. Ribon has demonstrated that inhibition of PARP7 with RBN-2397 can potently inhibit the growth of cancer cells and restore interferon signaling, effectively releasing the brake cancer uses to hide from the immune system and suppress both innate and adaptive immune mechanisms. In several cancer models, RBN-2397 demonstrated durable tumor growth inhibition, potent antiproliferative activity and restoration of interferon signaling. Ribon plans to initially develop RBN-2397 in squamous cell carcinoma of the lung, where research has shown PARP7 to be genetically amplified. The company also plans to explore RBN-2397 for the treatment of additional cancers, including cancers of the aerodigestive tract, pancreatic cancer and ovarian cancer.

PARP7 is a member of the monoPARP family of proteins, which are key regulators of stress responses that enable cancer cells to survive and also evade immune detection, and emerging science has linked their activity with disease development. MonoPARPs are a family of 12 enzymes that are functionally and structurally distinct from the more well-known polyPARPs, such as PARP1/2. MonoPARPs function across a variety of stress responses relevant to disease development in cancer, inflammatory conditions and neurodegenerative diseases. Ribon has built an integrated technology platform to interrogate monoPARPs to develop first-in-class, small molecule therapeutics.

About Ribon Therapeutics

Ribon Therapeutics is a biotechnology company developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions that contribute to disease. We are exploring novel areas of biology to develop effective treatments for patients with limited therapeutic options. Leveraging a chemical biology approach and our proprietary discovery platform, we are building a pipeline of selective, small molecule inhibitors to numerous NAD+ utilizing enzymes, beginning with monoPARPs, which have applications across multiple therapeutic areas. Our lead program is RBN-2397, a first-in-class PARP7 inhibitor in development for the treatment of cancer. Ribon is located in Cambridge, Massachusetts. For more information, please visit http://www.ribontx.com.

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Ribon Therapeutics Strengthens Scientific Advisory Board with Appointment of Neal Rosen, M.D., Ph.D. - Business Wire