StemCell Therapy

MUNICH, Germany, Nov. 28, 2019 (GLOBE NEWSWIRE) -- ViGeneron GmbH, a gene therapy company, announced the closing of its series A financing round led by WuXi AppTec and Sequoia Capital China. The proceeds will enable ViGeneron to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy programs.

ViGenerons pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two programs in development for undisclosed indications where no approved treatment options are currently available.

The companys two novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The vgAAV vector platform, based on novel engineered AAV capsids, enables a superior transduction of target cells and is designed to efficiently cross biological barriers. These attributes allow vgAAV vectors to target a broad spectrum of cell types in the retina and potentially other tissues, such as central nervous system tissue; enabling intravitreal, less invasive treatment administration. For larger genes (>5Kb) the company has developed the innovative REVeRT vector platform. This platform uses an innovative vector approach to pack split genes into individual vgAAV vectors and generate a full-length protein via mRNA trans-splicing.

ViGeneron is a spin-off of the Ludwig-Maximilians-University (LMU) in Munich. The companys founding team includes highly experienced executives and internationally renowned experts with track records in developing retinal gene therapy programs from discovery to clinical stage: Dr. Caroline Man Xu (Co-founder and CEO), Prof. Dr. Martin Biel (Scientific Co-founder), and Prof. Dr. Stylianos Michalakis (Scientific Co-founder).

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said: The evolution of medicines from small molecules to proteins has driven increased therapeutic benefits in the past; the next generation of gene therapies holds tremendous promise for patients. We are passionate about bringing innovations to patients. This financing is an important validation of our next-generation gene therapy technology platforms and ophthalmic development programs. With these top-tier investors and a strong ophthalmologic network supporting us, we are now in an excellent position to accelerate our development programs.

Edward Hu, Co-CEO of WuXi AppTec, commented: We are impressed by ViGenerons vgAAV vector platform and the innovative REVeRT vector platform. These gene therapy platform technologies will potentially generate superior gene therapy products to treat a wide range of diseases that are traditionally difficult to treat. We look forward to supporting the company to deliver on its great promises for the patients in need.

About ViGeneronViGeneron is dedicated to developing innovative gene therapies to treat ophthalmic diseases with high unmet medical need, as well as partnering with leading biopharmaceutical players in other disease areas. The companys pipeline is built on two proprietary adeno-associated virus (AAV) technology platforms. The first, vgAAV gene therapy vector platform, allows superior transduction efficiency and intravitreal, less invasive treatment administration. The second, REVeRT vector platform, targets diseases caused by mutations in large genes. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit http://www.vigeneron.com.

About WuXi AppTecWuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries worldwide to advance discoveries and deliver groundbreaking treatments to patients. As an innovation-driven and customer-focused company, WuXi AppTec helps our partners improve the productivity of advancing healthcare products through cost-effective and efficient solutions. With industry-leading capabilities such as R&D and manufacturing for small molecule drugs, cell and gene therapies, and testing for medical devices, WuXi AppTecs open-access platform is enabling more than 3,700 collaborators from over 30 countries to improve the health of those in need and to realize our vision that "every drug can be made and every disease can be treated".

About Sequoia Capital ChinaThe Sequoia team helps daring founders build legendary companies. In partnering with Sequoia, companies benefit from our unmatched network and the lessons we've learned over 47 years. As The Entrepreneurs Behind The Entrepreneurs, leading venture capital firm Sequoia Capital China is renowed for investing early in many successful companies and focuses on four sectors: TMT, healthcare, consumer/serive, and industrial technology.

Contact:ViGeneron GmbH info@vigeneron.com

Media and Investor Relations:MC Services AGShaun Brown/ Julia von Hummelphone: +49 (0)89 21022880 vigeneron@mc-services.eu

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ViGeneron announces closing of series A financing to drive development of next generation gene therapy pipeline - GlobeNewswire

Certain levels of plasma protein adenosine deaminase 2 (ADA2) are sensitive and specific, and can be used as a novel biomarker of macrophage activation syndrome in patients with systemic juvenile idiopathic arthritis (JIA), according to research results published in the Annals of the Rheumatic Diseases.

Researchers sought to evaluate the role and function of ADA2 as a novel biomarker of macrophage activation syndrome because of the overlapping clinical features between macrophage activation syndrome and active systemic JIA.

A reference range for plasma ADA2 activity was first established using samples from 324 healthy individuals (174 children and 150 adults). Spectrophotometric assays showed that patients with biallelic ADA2 mutations had a near absence of ADA2 activity compared with carriers who had approximately half-normal plasma ADA2 activity.

After establishing the reference range, the researchers compared ADA2 levels in children with Kawasaki disease, pediatric systemic lupus erythematosus, and juvenile dermatomyositis with age-matched healthy controls. Investigators established that ADA2 levels did not correlate with markers of disease activity and were not a general marker of systemic inflammation in any of the 3 disorders.

Among the JIA population, most patients showed plasma ADA2 activity levels that were comparable with age-matched healthy controls; however, a small subset had levels well above the upper limit of normal. After stratification by JIA category, investigators found that ADA2 activity was present in children with oligoarticular and polyarticular JIA, enthesitis-related arthritis, and psoriatic arthritis.

Using multiple macrophage activation syndrome biomarkers, researchers compared ADA2 activity in patients with JIA and created a correlation matrix based on Spearman rank correlation r values. The comparisons demonstrated that ADA2 levels correlated with ferritin, interleukin-18, and C-X-C Motif Chemokine Ligand 9. ADA2 activity also correlated well with increased aspartate aminotransferase levels in macrophage activation syndrome, but not with conventional markers of inflammation.

Although the patterns displayed by these markers were different, all the markers were sensitive and specific in discriminating macrophage activation syndrome from systemic JIA using the upper limit of normal as a cutoff.

Seven patients with macrophage activation syndrome had available serum samples. Consistent with previous analyses, investigators found that ADA2 levels were generally higher during a confirmed episode of macrophage activation syndrome compared with other time points. Researchers noted that patients who experienced recurrent episodes of macrophage activation syndrome exhibited ADA2 levels near the upper limit of normal even when macrophage activation syndrome was absent.

Whether ADA2 contributes to the pathophysiology of [macrophage activation syndrome] is not clear, the researchers concluded. The [physiologic] function of ADA2 remains to be determined.

Reference

Lee PY, Schulert GS, Canna SW, et al. Adenosine deaminase 2 as a biomarker of macrophage activation syndrome in systemic juvenile idiopathic arthritis [published online November 9, 2019]. Ann Rheum Dis. doi: 10.1136/annrheumdis-2019-216030

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Novel Biomarker of Macrophage Activation Syndrome Identified in Juvenile Idiopathic Arthritis - Rheumatology Advisor

CHICAGO - About 54 million adults have doctor-diagnosed arthritis, according to The Arthritis Foundation. Almost 300,000 babies and children have arthritis or a rheumatic condition

For Chelsea May, those numbers hit too close to home. Her 14-year-old daughter, Jenna, suffers from Juvenile Psoriatic Arthritis and Uveitis. She was diagnosed four years ago.

To help fight spread awareness of the debilitating disease and raise money for research, Jenna's family will host their third annual bake sale on Dec. 6. The bake sale will take place at the Mt. Greenwood Holiday Stroll from 4 p.m. to 7 p.m.

"We are also looking for donations of baked goods as well," May said.

The donations and money received from the bake sale will be donated to the Arthritis Foundation's Jingle Bell Run which will take place on Dec. 14.

The Arthritis Foundation's Jingle Bell Run is the original festive race for charity, bringing people from all walks of life together to champion arthritis research and resources.

Less than a week after the bake sale, Jenna's family will gear up for the Jingle Bell Run in Chicago. If you would like to donate or join Jenna's run team, click here.

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Jingle Bake Sale To Benefit The Arthritis Foundation - Beverly, IL Patch

ATLANTA There is no significant difference in the rate of serious infections in patients with rheumatoid arthritis (RA) receiving upadacitinib 15 mg or adalimumab, according to study results presented at the 2019 American College of Rheumatology/Association of Rheumatology Professionals (ACR/ARP) Annual Meeting, held November 8 to 13, 2019, in Atlanta, Georgia.

However, the results indicated that patients receiving upadacitinib 15 mg and 30 mg had higher rates of herpes zoster compared with patients receiving adalimumab or methotrexate.

The study included data from 5 randomized double-blind, placebo- or active-controlled phase 3 trials of upadacitinib 15 mg (included in all 5 trials) or 30 mg daily (included in 4 trials) in patients with RA.

Researchers calculated the exposure adjusted event rates (EAERs; events/100 patientyears [PY]) of treatment-emergent adverse events (AEs) for the integrated placebo (3 trials; 12/14 weeks), the integrated methotrexate (2 trials; mean exposure, 36 weeks), the originator adalimumab (mean exposure, 42 weeks), upadacitinib 15 mg (mean exposure, 53 weeks), and upadacitinib 30 mg (mean exposure, 59 weeks) groups.

In all 5 phase 3 trials, 3834 patients received 1 dose of upadacitinib 15 mg (n=2630) or 30 mg (n=1204) with no option to switch doses, for a total of 4020.1 PY of upadacitinib exposure.

Similar to patients who received adalimumab, patients who received upadacitinib 15 mg had EAERs of overall serious adverse events (SAEs) and AEs that led to discontinuation. Compared with methotrexate, upadacitinib 15 mg and 30 mg had higher EAERs of both SAEs and AEs.

The most commonly reported AEs were upper respiratory tract infection, nasopharyngitis, and urinary tract infections, all of which occurred more frequently among patients in the upadacitinib groups compared with the placebo group.

Patients in the upadacitinib 15 mg and adalimumab groups had comparable rates of serious infection events (SIEs); however, rates of SIEs were higher among patients receiving upadacitinib compared with methotrexate. Compared with patients receiving methotrexate and adalimumab, patients receiving either dose of upadacitinib had higher rates of herpes zoster.

Among patients receiving upadacitinib, those in the 15-mg group had lower rates of SIEs and herpes zoster compared with the 30-mg group.

All treatment groups had similar rates of malignancies, excluding non-melanoma skin cancer, and adjudicated major adverse cardiovascular events and venous thromboembolic events. In addition, all treatment groups had comparable rates of death.

Compared with the other treatment groups, upadacitinib 30 mg had a higher rate of non-melanoma skin cancer; however, the rates of non-melanoma skin cancer for both the upadacitinib groups fell within the range reported for patients with RA treated with disease-modifying antirheumatic drugs.

Visit Rheumatology Advisor for live coverage and more news from the 2019 ACR/ARP Annual Meeting.

Reference

Cohen S, van Vollenhoven R, Winthrop K, et al. Safety profile of upadacitinib in rheumatoid arthritis: integrated analysis from the SELECT phase 3 clinical program. Presented at: 2019 ACR/ARP Annual Meeting; November 8-13, 2019; Atlanta, GA. Abstract 509.

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Rates of Serious Infection Events Similar With Upadacitinib, Adalimumab in Rheumatoid Arthritis - Rheumatology Advisor

In September, the world of entertainment news buzzed with word that Kim Kardashian West tested positive for lupus and rheumatoid arthritis. The star underwent further tests, however, resulting in a diagnosis of psoriatic arthritis instead. While all three autoimmune disorders share some signs and symptoms, psoriatic arthritis is generally considered to have a better prognosis than lupus. That said, the conditions can co-exist and lupus has gotten a reputation for being difficult to diagnose, especially in the absence of the butterfly-shaped rash on ones cheeks and nose.

Im so relieved. The pain is going to come and go sometimes, but I can manage it and this is not going to stop me, Kardashian said in an article in response to receiving her psoriatic arthritis diagnosis. Her relief at not having lupus is understandable, given that lupus can affect a greater number of organs and systems in the body and is considered to be life-threatening.

Lupus, rheumatoid arthritis and psoriatic arthritis are examples of some conditions that are often considered when an individual is undergoing diagnosis for certain autoimmune diseases, because they share several symptoms and can trigger positive results in the same diagnostic tests. Kim Kardashian received the initial news that she had lupus or rheumatoid arthritis likely due to positive antinuclear antibody (ANA) test results.

An ANA is a blood test ordered when a doctor, usually a rheumatologist, suspects that a patient has a particular kind of autoimmune disorder. This test checks for the existence of autoantibodies, which are produced when a persons body is, in effect, attacking itself and several areas of the body are affected. A positive ANA test usually indicates that the doctors suspicions are confirmed, and then other factors (like medical and family history) need to be considered and more tests done to arrive at a diagnosis.

Psoriatic arthritis is usually diagnosed between the ages of 20 and 50, and occurs in women and men equally. While there is no cure, appropriate and early treatment can help prevent major damage to affected parts of the body.

Psoriatic arthritis appears in a minority of individuals who have already been diagnosed with psoriasis, an autoimmune skin condition with which Kim Kardashian and her mother, Kris Jenner, had already been diagnosed. Psoriatic arthritis affects around 520,000 individuals in the United States alone.

The autoimmune condition is believed to be caused by a combination of genetic factors and environmental triggers. So while some people inherit psoriatic arthritis-related genes, only a subset of those individuals will go on to develop the condition. In these cases, the disease could be triggered by other illnesses or infections, various forms of extreme stress, poor diet, smoking, and so on.

Around 40 percent of psoriatic arthritis patients have one or more close family members with psoriasis or psoriatic arthritis diagnosis, which strongly indicates that the disease is hereditary. Interestingly, recent research has suggested that psoriasis patients who go on to develop psoriatic arthritis have a different genetic profile than those who do not. And the most well-studied of the psoriatic arthritis genes belong to a family of genes called the human leukocyte antigen (HLA) complex, which help the body tell the difference between its own proteins and viral or bacterial proteins.

According to Genetics Home Reference by the U.S. National Library of Medicine, Variations of several HLA genes seem to affect the risk of developing psoriatic arthritis, as well as the type, severity, and progression of the condition.

Ive been feeling so tired, so nauseous, and my hands are really getting swollen. I feel like I literally am falling apart. My hands are numb, Kardashian said on a recent episode of Keeping Up with the Kardashians.

These kinds of descriptions are common in all three conditions lupus, rheumatoid arthritis, and psoriatic arthritis though each patient presents with a different array of symptoms, and all with varying degrees of severity. The main symptoms of psoriatic arthritis are pain, stiffness, and swelling in affected joints, along with chronic fatigue. Joints near the end of the fingertips and tips of the toes are often affected, as are bones in the spine.

The symptoms of psoriatic arthritis tend to worsen over time, though some patients experience periods of remission when symptoms temporarily improve. Compared to rheumatoid arthritis, psoriatic arthritis is more likely to cause swelling in the smallest joints of the fingers and toes, foot pain (in the heel and/or sole of the foot), and lower back pain caused by inflammation in vertebral joints. Patients with psoriatic arthritis are also more likely to experience symptoms on one side of the body or in different appendages on each side (in other words, it tends to be an asymmetric disease), whereas patients with rheumatoid arthritis are more likely to experience symptoms that affect both sides of the body equally (symmetric disease).

Most if not all patients with psoriatic arthritis also have psoriasis, an autoimmune condition that causes red, scaly patches of skin that can be itchy, painful and embarrassing. Psoriasis usually precedes the onset of psoriatic arthritis by several years. People with psoriatic arthritis commonly experience fingernail changes, too, such as the formation of a pitted or ridged nail surface, or the nails become separated from the nail beds.

There are several treatment options for psoriatic arthritis, which include nonsteroidal anti-inflammatory drugs (NSAIDs) to reduce inflammation and pain, immunosuppressants to suppress the immune system, disease-modifying antirheumatic drugs (DMARDs) to slow the progression of the disease, and newer medications that minimize the activity of certain enzymes involved in the inflammatory process. Treatment plans may also involve steroid injections administered directly into affected joints, or joint replacement surgery in cases where the disease has significantly progressed.

Kristen Hovet covers genetics, medical innovations and the intersection of sociology and culture. The North Dakota native is based in Vancouver, Canada, where she is working on a masters degree in health communication at Washington State University. Follow her on her website or Twitter @kristenhovet

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Kim Kardashian West's battle with psoriatic arthritis: Will understanding the genetics of the autoimmune disorder point to a cure? - Genetic Literacy...

AbbVie's (ABBV - Get Report) fledgling rheumatoid arthritis treatment Rinvoq is showing promise and appears to be tracking "meaningfully ahead of expectations," according to analysts at Piper Jaffray.

Analyst Christopher Raymond reiterated the firm's overweight rating on theNorth Chicago biopharma while increasing its price target to $92 from $90.

At last check Tuesday AbbVie was trading down 1.2% at $86.

Piper Jaffray's bullish outlook on Rinvoq is fueled by the results of a new survey of 100 rheumatologists, who provided mostly positive feedback about the drug. A rival treatment still enjoys success, but Rinvoq could soon be on its heels, the analyst wrote.

"While indications of Xeljanz (PFE - Get Report) entrenchment do exist, fully 33% of doctors indicate they would be early Rinvoq adopters, just because of AbbVie's involvement," Raymond wrote.

"And in terms of reported sales contact," AbbVie's rheumatology focus on Rinvoq appears to rival that "of its Humira effort, and then finally, when compared to the prior two rheumatoid arthritis launches (Olumiant and Kevzara), unaided awareness, brand familiarity and initial user base are all head-and-shoulders above."

"[Just] months post-launch, ... Rinvoq registers 1% patient share," Raymond wrote. But "asked to delineate most recent scripts, doctors give Rinvoq 8% share. When asked to projectshare in six months, rheumatologists indicate Rinvoq's share will grow to 6%."

"In that Rinvoq largely takes a back seat to [AbbVie treatment] Skyrizi as a key growth driver in the minds of most investors we speak with, we see this feedback as a welcome source of upside in coming quarters," Raymond wrote.

AbbVie is a holding in Jim Cramer's Action Alerts PLUS Charitable Trust Portfolio. Want to be alerted before Cramer buys or sells ABBV? Learn more now.

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AbbVie Affirmed Overweight at Piper Jaffray on Potential of Arthritis Drug - TheStreet.com

While theres no cure for knee osteoarthritis, a combination of strategies can help relieve your pain and keep you active.

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Although the cornerstones of treatment are exercise and physical therapy and pain medications and steroid injections are also options you can also try knee braces, shoe inserts or simply wearing more supportive shoes.

Knee braces can be helpful for managing your pain, says physical therapist Dawn Lorring, PT, MPT. The location and severity of your symptoms will drive which brace works best for you.

Osteoarthritis is caused by the breakdown of cartilage (thats the cushioning material that covers the ends of bones in joints.) This causes pain and stiffness.

In the knee joint, arthritis can occur at any of three points where the bones come in contact:

Sleeve braces. People who have mild pain or stiffness that limits their activities can try a sleeve-type brace. These provide compression, which can reduce swelling and warm the joint. This might relieve the stiffness.

These braces also provide added support. If your knee feels unsteady or wobbly, a compression-type brace can be helpful, Lorring says. Some of them have plastic stays or a hinge on the side, which provides a little more support. She recommends getting one that has an opening at the knee cap.

Sleeve braces arent covered by insurance, but they are relatively inexpensive, ranging from $10 to 100.

Web brace. A more advanced brace is a sleeve with silicone webbing over the front. As you bend and straighten your knee, the webbing tightens in certain areas. This provides extra support to the knee.

A regular sleeve brace provides compression all over. The brace with the webbing also provides guidance for how the knee cap moves, Lorring says.

This type of brace might be the most helpful for someone with osteoarthritis beneath the knee cap. A web brace costs about $100.

Unloader brace. When arthritic changes are between the femur and tibia, a device called an unloader knee brace may help, especially if one side is more arthritic than the other. These have a metal band that goes around the thigh and another one around the calf, connected by a hinged bar. This creates a frame that can be adjusted to shift pressure (unload) from one side of the knee to the other.

If the inside of your knee hurts, the brace can be adjusted to put more force on the outside of your knee, unloading weight off the inside, Lorring explains.

These are less beneficial if your arthritis symptoms are similar on both the inside and outside of the joint.

Unloader knee braces are expensive ($500 to $1,000), but they can be covered by insurance. Youll need a doctors prescription and documentation that it is medically necessary.

Shoes and inserts. Various foot problems (like high arches or flat feet) or just the particular way you walk can affect the alignment of your body. That might be putting more pressure on your knee joints. You may get some relief by choosing better shoes or wearing shoe inserts (also called orthotics).

Because everyone is different, theres no universal advice for shoes or inserts. Lorring recommends consulting a physical therapist or an expert in foot mechanics who can observe how you walk and help you pick out shoes or shoe inserts that match your needs. I encourage people to look at running shoes because there are more support options, she says.

The goal with orthotics is to make sure your foot is moving in the best way it can so your knee isnt getting more force than it should, Lorring says. There are a wide variety of shoe inserts and heel wedges that you can buy in a drug store or online. You can also get them custom made or save some money and get semi-custom ones. Like with shoes, you need to get inserts and wedges that are specific to your needs.

You can have an insert that doesnt add much arch support but it adds cushion, which can be beneficial if you walk on the outside of your foot, Lorring says. However, if your foot rolls inward too much, you may need more arch support.

You can get heel wedges that are sloped in one direction or the other, which is similar to the action of an unloader brace. It shifts pressure from one side of the knee to the other.

Ultimately, you have to find what works for you, Lorring says.

This article originally appeared in Cleveland Clinic Arthritis Advisor.

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Could a Knee Brace Help Ease Your Osteoarthritis Pain? - Health Essentials from Cleveland Clinic

Study Assesses Risk of Infections, Cancer With Interleukin Inhibitors in Rheumatic Disease

According to the study, the risks may be comparable to those reported for other biologics approved for the treatment of rheumatic diseases.

A number of IL-1, IL-6, IL-12/23, and IL-17 inhibitors are used as therapy options in rheumatologic diseases. Although IL inhibitors have demonstrated efficacy, there are limited data regarding their safety profile and it is unknown to what extent IL inhibitors may increase the risk of serious infections and cancer.

To assess this risk, researchers conducted a systematic review and meta-analysis using data from clinical trials that evaluated IL inhibitor therapies and reported safety data. The meta-analysis included 74 studies including 29,214 patients. The studies included tocilizumab, secukinumab, anakinra, ixekizumab, rilonacept, sarilumab, sirukumab, ustekinumab, brodalumab, guselkumab, clazakizumab, canakizumab, and olokizumab.

Diseases in the study included rheumatoid arthritis (RA), psoriatic arthritis, ankylosing spondylitis, gout, juvenile idiopathic arthritis, giant call arteritis, systemic lupus erythematosus, primary Sjogren syndrome, systemic sclerosis, familial Mediterranean fever, and osteoarthritis.

Overall, 69 studies included data for serious infections across all rheumatic diseases, comprising a total of 24,236 patients. The pooled analysis showed an increased risk of serious infections with the use of IL inhibitors compared with placebo.

Fourteen of the studies reported the incidence of opportunistic infection, with 9998 patients in these trials. Oral candidiasis was the most commonly reported opportunistic infection, according to the analysis. Others included herpes zoster, esophageal candidiasis, Mycobacterium tuberculosis, atypical mycobacterial infections, and histoplasmosis. Overall, the results showed an increased risk of opportunistic infections with the use of IL inhibitors compared with placebo.

A total of 46 studies with approximately 21,000 patients had data on the incidence and type of cancers across all rheumatic diseases. A total of 141 cases of cancer reported in the treatment groups and 28 in the control groups. The analysis found that, overall, there was an increased risk for cancer with IL inhibitors compared with placebo.

The researchers noted that, although the review suggest that the risk of cancer may be increased with longer IL inhibitor therapy, the results are not conclusive and should be further investigated by long-term data.

This analysis provides estimates of toxic effects for infections and cancer associated with the use of IL inhibitors that can inform shared decision-making when patients and clinicians are contemplating the use of IL inhibitors for rheumatologic diseases, they concluded.

References

Bilal J, Berlinberg A, Riaz IB, et al. Risk of infections and cancer in patients with rheumatologic diseases receiving interleukin inhibitors: a systematic review and meta-analysis. JAMA Network Open. 2019. Doi: 10.1001/jamanetworkopen.2019.13102.

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Study Assesses Risk of Infections, Cancer With Interleukin Inhibitors in Rheumatic Disease - Pharmacy Times

BACKGROUNDS:

Rheumatoid arthritis (RA) is a systemic autoimmune disease that confers one of the strongest risks for cardiovascular disease (CVD) morbidity and mortality than in general population. Pulse wave velocity (PWV) and augmentation index (AIx) are composite measures of arterial stiffness (AS) and associated with CV risk.

The aim of this study was to systemically review the evidence regarding the relationship between PWV, AIx and RA, as well as underlying influential factors.

Eligible literatures were searched in PubMed, EMBASE and The Cochrane Library published up to February 28, 2019 in English. The pooled weight mean difference (WMD) with its 95% confidence interval (CI) was calculated using random-effect model analysis.

A total of 38 studies were finally incorporated in the meta-analysis. The results indicated that, compared to controls, RA patients had significantly increased levels of carotid-femoral (cf)-PWV (WMD=1.10m/s, 95% CI: 0.84-1.35), brachial-ankle (ba)-PWV (WMD=0.20m/s, 95% CI: 0.12-0.28), cartoid-radial (cr)-PWV (WMD=0.51m/s, 95% CI: 0.23-0.79), AIx (WMD=4.79%, 95% CI: 1.34-8.24) and AIx normalized to a 75 beats/minute heart rate ([emailprotected]) (WMD=5.78%, 95% CI: 3.82-7.74) (all p <0.001). Meta-regression and subgroup analysis demonstrated significant association of cf-PWV with age, disease duration and erythrocyte sedimentation rate (ESR) in RA.

Overall, there is increased PWV level in patients with RA, and this alteration is associated with age, disease duration and ESR.

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Assessment of Aortic Stiffness in Patients With Rheumatoid Arthritis Using Pulse Wave Velocity: An Update Meta-analysis - DocWire News

OBJECTIVE:

To determine the prevalence of comorbidities in rheumatoid arthritis (RA), discover which comorbidities might predispose to developing RA, and identify which comorbidities are more likely to develop after RA.

We performed a case-control study using a single-center biobank, identifying 821 cases of RA (143 incident RA) between January 1, 2009, and February 28, 2018, defined as 2 diagnosis codes plus a disease-modifying antirheumatic drug. We matched each case to 3 controls based on age and sex. Participants self-reported the presence and onset of 74 comorbidities. Logistic regression models adjusted for race, body mass index, education, smoking, and Charlson comorbidity index.

After adjustment for confounders and multiple comparisons, 11 comorbidities were associated with RA, including epilepsy (odds ratio [OR], 2.13; P=.009), obstructive sleep apnea (OR, 1.49; P=.001), and pulmonary fibrosis (OR, 4.63; P<.001), but cancer was not. Inflammatory bowel disease (OR, 3.82; P<.001), type 1 diabetes (OR, 3.07; P=.01), and venous thromboembolism (VTE; OR, 1.80; P<.001) occurred more often before RA diagnosis compared with controls. In contrast, myocardial infarction (OR, 3.09; P<.001) and VTE (OR, 1.84; P<.001) occurred more often after RA diagnosis compared with controls. Analyses restricted to incident RA cases and their matched controls mirrored these results.

Inflammatory bowel disease, type 1 diabetes, and VTE might predispose to RA development, whereas cardiovascular disease, VTE, and obstructive sleep apnea can result from RA. These findings have important implications for RA pathogenesis, early detection, and recommended screening.

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Comorbidities As Risk Factors for Rheumatoid Arthritis and Their Accrual After Diagnosis - DocWire News

As we begin the final month of 2019, the U.S. Food and Drug Administration (FDA) has several PDUFA dates to approve drug applications. Heres a look at those scheduled for the first two weeks of the month.

Celgenes Luspatercept for Beta-thalassemia-associated Anemia

Celgene had a target action date of December 4, 2019 for its Biologics License Application (BLA) for luspatercept. Luspatercept is an investigational erythroid maturation agent for adults with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions, and for the treatment of adults with beta-thalassemia-associated anemia who require RBC transfusionsit is this second indication for which the company had the December 4 PDUFA date.

Luspatercept is also being reviewed by the European Medicines Agency (EMA). It is a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell maturation. The therapy was jointly developed by Celgene and Acceleron. The application was built on the results from the Phase III COMMANDS trial in ESA-nave, lower-risk MDS patients and the Phase II BEYOND trial in non-transfusion-dependent beta-thalassemia.

The FDA approved luspatercept-aamt under the brand name of Reblozyl on November 8 for anemia in adults with beta-thalassemia. Beta-thalassemia is a rare, inherited blood disorder caused by a genetic defect in hemoglobin. The target action date for luspatercept for erythroid maturation agent for adults with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions is April 4, 2020.

Amgens Biosimilar to Janssens Remicade

Amgen has a target action date of December 14 for its BLA of ABP 710, a biosimilar candidate to Janssen Pharmaceuticals Remicade (infliximab), a drug for various inflammatory diseases such as Crohns disease, ulcerative colitis, rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis and plaque psoriasis.

ABP 710 (and infliximab) are anti-tumor necrosis factor alpha (anti-TNF) monoclonal antibodies. The BLA submission includes analytical, pharmacokinetic and clinical data in addition to pharmacology and toxicology data. A Phase III study comparing efficacy, safety and immunogenicity was run in patients with moderate-to-severe rheumatoid arthritis and confirmed no clinically meaningful differences between ABP 710 and infliximab.

Avadels FT218 for Narcolepsy

Avadel Pharma has a target action date of December 15 for AV001. The New Drug Application (NDA) was originally accepted in May 2019 under the FDAs Priority Review program, which gives it a statutory six-month review. It was then extended by three months as the result of FDA requests for additional analytical information and Avadels resultant additional submissions.

FT218 is an investigational drug designed to be administered in one single dose, before bedtime, to treat excessive daytime sleepiness (EDS) and cataplexy in patients suffering from narcolepsy. It was granted orphan-drug designation by the FDA on January 8, 2018 on the theory that is may be clinically superior to the currently marketed, twice-nightly sodium oxybate product.

On November 25, 2019, Avadel announced it had completed patient enrollment of 205 patients in the REST-ON Phase III clinical trial for FT218. The enrollment target was 205 patients. Additional patients may be enrolled if they meet eligibility criteria. Topline data is expected in the second quarter of 2020.

The company says that if the drug is approved, it has the potential to capture a significant share of the twice-nightly sodium oxybate market, which is currently valued at $1.7 billion per year.

The REST-ON trial is a double-blind, randomized, placebo-controlled Phase III study to evaluate the efficacy and safety of once-nightly FT218. It is under a Special Protocol Assessment agreement with FDA.

Read the original here:
FDA Action Alert: Celgene, Amgen and Avadel - BioSpace

First metastatic triple-negative breast cancer patient showed no detectable circulating tumor cells (CTC) or putative metastatic tumor cells (EMTs) in the peripheral blood. Further, a significant reduction in CCR5 expression was demonstrated on cancer-associated cells after eight weeks of treatment with leronlimab

A second patient with metastatic breast cancer has been enrolled under an emergency use investigational new drug

VANCOUVER, Washington, Dec. 03, 2019 (GLOBE NEWSWIRE) CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today additional early Phase 1b/2 clinical trial results evaluating leronlimab (PRO 140) patients with CCR5+ metastatic triple-negative breast cancer (mTNBC). Data from the first patient enrolled show no detectable circulating tumor cells (CTC) or EMTs in the peripheral blood and additional reductions in CCR5 expression on cancer-associated cells at eight weeks. A second patient with metastatic breast cancer has been enrolled in the trial under an emergency use investigational new drug (IND).

The first patient in the open label study was given a weekly injection of leronlimab at 700mg along with carboplatin. The patient was enrolled in the trial with CCR5-positive, mTNBC and nave to chemotherapy in metastatic setting. The patient was previously exposed to anthracyclines and taxane in neoadjuvant and adjuvant settings.

The fourth blood sample from the mTNBC patient, drawn following eight weeks of treatment, demonstrates a notably sustained response to leronlimab, said Bruce Patterson, M.D., chief executive officer of IncellDx. Other cancer-associated macrophage-like (CAML) cells in the blood sample were found at the lower limits of detection and were also decreased in size. Most importantly, the CAML cells had reduced CCR5 staining compared to samples taken from the patient three weeks earlier, reflecting an ongoing blockade of the CCR5 receptor by leronlimab.

Nader Pourhassan, Ph.D., president and chief executive officer of CytoDyn, stated: It is very exciting to see additional preliminary results that demonstrate leronlimabs potential as a therapeutic option to treat mTNBC. We are also pleased to have enrolled a second patient with metastatic breast cancer under an emergency IND. If the results of the second patient are similarly impressive, we plan to file for Breakthrough Therapy designation before the end of January 2020. We have had many patients contact us for treatment under our expanded access protocol and another hospital has opened enrollment for our mTNBC trial. We look forward continuing our research in furtherance of this clinical development plan.

About Triple-Negative Breast CancerTriple-negative breast cancer (TNBC) is a type of breast cancer characterized by the absence of the three most common types of receptors in the cancer tumor known to fuel most breast cancer growthestrogen receptors (ER), progesterone receptors (PR) and the hormone epidermal growth factor receptor 2 (HER-2) gene.1 TNBC cancer occurs in about 10 to 20 percent of diagnosed breast cancers and can be more aggressive and more likely to spread and recur.2,3 Since the triple negative tumor cells lack these receptors, common treatments for breast cancer such as hormone therapy and drugs that target estrogen, progesterone, and HER-2 are ineffective.4 Currently, there are no targeted therapies approved to treat triple negative breast cancer.5

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients, and the second is for metastatic triple-negative breast cancer (mTNBC). Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98 percent in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additional clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.

CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of graft-versus-host disease (GvHD).

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as graft-vs-host disease (GvHD) and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab (PRO 140) as a once-weekly monotherapy for HIV-infected patients and, plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab (PRO 140) can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties, and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companys cash position, (ii) the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv) the Companys ability to enter into partnership or licensing arrangements with third parties, (v) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companys ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companys clinical trials, (viii) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSFor more information, visitwww.cytodyn.comor contact:

Media:Grace FotiadesLifeSci Public Relationsgfotiades@lifescipublicrelations.com(646) 876-5026

Investors:Nader Pourhassan, Ph.D.President & CEOnpourhassan@cytodyn.com

Source: CytoDyn, Inc.

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CytoDyn Reports Early Results from First Patient in its Phase 1b/2 CCR5+ Metastatic Triple-Negative Breast Cancer Trial - Stock Day Media

Recently, a new study that appears in the journal Nature, focuses on stem cell therapy and shows unexpected ways in which it may be helpful in recovering the health of the heart. Stem cell therapy has become popular in the past few years due to its benefits for a big number of health conditions.

Currently, there is major ongoing research on stem cells since they are responsible for the regeneration of new cells and may play a fundamental role in understanding the development of a variety of different diseases as well as their potential treatments.

Some of the recent discoveries of medical science include using stem cells as regenerative medicine as they can be turned into particular types of cells that may be able to replace tissues damaged as a result of health issues and thereby control the disease.

Read also:New Study Reveals Hydromethylthionine Slows Cognitive Decline and Brain Atrophy

The therapy can be specifically useful for people with conditions such as type 1 diabetes, spinal cord injuries, Alzheimers disease, Parkinsons disease, stroke, cancer, burns, amyotrophic lateral sclerosis, heart disease, and osteoarthritis.

At the moment, the most successful procedure that involves stem cell therapy is performing a bone marrow transplant. This surgical operation replaces the cells which have been damaged during chemotherapy by programmed stem cells. People are usually able to maintain and live a normal life after recovery from the surgery.

Furthermore, stem cell usage in clinical trials designed for testing the effectiveness, safety, and potential negative impact of new drugs. To do so, the stem cells can be programmed into becoming the type of cells that the drug aims to target.

The new study, which was led by Jeffery Molkentin who is a professor of the Howard Hughes Medical Institute (HHMI) and the director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center, takes data from a study from the same journal, Nature, from the years 2014 which was conducted by the same medical team.

In the new paper, the team with Molkentin as the principal investigator found some unexpected results. There were two types of stem cells in the clinical trial cardiac progenitor cells and bone marrow mononuclear cells.

The main objective of the new trial was to re-evaluate the results of the 2014 study, which showed that injecting c-kit positive heart stem in the heart does not help in the regeneration of cardiomyocytes, to see how the cell therapy can be made to be effective.

It was instead discovered that injecting an inert chemical called zymosan, which is designed particularly for inducing an innate immune response, or dead stem cells can also be beneficial for the recovery of heart as they may speed up the healing procedure.

Injecting either dead stem cells or zymosan led to a reduction in the development of cellular matrix connective tissue in the areas which had been damaged in the heart. In addition, the mechanical properties of the targeted scar also improved.

Another important finding was that chemical substances such as zymosan are required to be injected directly into the heart for optimum results. In previous clinical trials, direct injections were avoided for safety reasons.

Molkentin and the team state that follow-up studies and trials on this new discovery are imminent as they may be important for developing therapies in the future.

Read more here:
Stem Cell Therapy May Improve Heart Health In New Ways - TheHealthMania

Latest Study on Industrial Growth of Transfection Technologies 2026 By-Data Bridge Market Research

A detailed study accumulated to offer Latest insights about acute features of Transfection Technologies . The report contains different market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary and SWOT analysis. Some are the key players taken under coverage for this study are Lonza., Promega Corporation., Sigma-Aldrich Co. , Thermo Fisher Scientific Inc., Bio-Rad Laboratories, Inc. , Roche Molecular Systems, Inc., QIAGEN, Inovio Pharmaceuticals, Inc., POLYPLUS TRANSFECTION, Mirus Bio LLC, Takara Bio Inc., SignaGen Laboratories, MaxCyte, Inc., Genlantis Inc., Techulon, BioAstrum Corporation., Altogen Biosystems, OZ Biosciences, Boca Scientific, Inc., Biontex Laboratories GmbH. , and others.

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Market Affecting Factors:

This section involves the list of various factors which have huge impact on the overall Transfection Technologies growth.

Global transfection technologies market is registering a substantial CAGR of 9.74% in the forecast period of 2019-2026.

Key Assessments:

There are various types of assessments carried out in Transfection Technologies report to analyze the crucial market details and evaluate market opportunities. These assessments are-

Primary and Secondary assessment- These are collected through industry journals, government bodies and stakeholders. And for secondary research, industry experts are consulted.

Qualitative and quantitative assessment

Feasibility analysis, Porters Five Forces analysis

SWOT Analysis which highlights strength, weakness, opportunities and threats of Transfection Technologies .

Market Drivers

Market Restraints

Crucial Market Segment Details:

Global Transfection Technologies Market By Transfection Method (Cotransfection, Electroporation, Cationic Lipid Transfection, In Vivo Transfection), By Applications (Virus production, Protein production, Gene silencing, Stem cell reprogramming & differentiation, Stable cell line generation)

Inquiry Before Buying at: https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-transfection-technologies-market

Each point covered in the Transfection Technologies report is examined to get clear thought regarding every variable and factor that is affecting the market development. Transfection Technologies report comprises of various segments linked to industry and market with comprehensive research and analysis. The Transfection Technologies report includes all the company profiles of the major players and brands.

Table of Content

1 Introduction

2 Research Methodology

3 Executive Summary

4 Premium Insights

5 Market Overview and Industry Trends

6 Product Stewardship Market, By Type

7 Product Stewardship Market, By Organization Size

8 Product Stewardship Market Analysis, By Region

9 Competitive Landscape

10 Company Profiles

New Business Strategies, Challenges & Policies are mentioned in Table of Content, Request Detailed TOC: https://www.databridgemarketresearch.com/toc/?dbmr=global-transfection-technologies-market

Significant Point Mentioned in the Research report

Purpose of This Report:

The purpose of Transfection Technologies report is to give organized market solutions to market players for smart decision marking. The report incorporates market size, patterns, details of business research and significantly more. It likewise offers investigation of worldwide and local insight, a 360-degree perspective available that incorporates factual figures, focused scene, comprehensive division, key patterns and key proposals.

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Demand Side: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

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

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Transfection Technologies Market Survey by Industry Trends, Growth Rate with CAGR Analysis 2026 By Top Key Players Lonza., Promega Corporation.,...

In a analysts note revealed to clients and investors on Friday, 29 November, equity research analysts at BidaskScores research division upgraded Gamida Cell (NASDAQ:GMDA)s stock to a Hold.

Axt Inc (NASDAQ:AXTI) had a decrease of 0.34% in short interest. AXTIs SI was 1.63M shares in December as released by FINRA. Its down 0.34% from 1.64M shares previously. With 349,800 avg volume, 5 days are for Axt Inc (NASDAQ:AXTI)s short sellers to cover AXTIs short positions. The SI to Axt Incs float is 4.63%. It closed at $3.16 lastly. It is up 43.44% since December 2, 2018 and is downtrending. It has underperformed by 43.44% the S&P500. Some Historical AXTI News: 14/05/2018 Eam Investors LLC Exits Position in AXT; 25/04/2018 AXT 1Q EPS 7c; 24/04/2018 AXT Short-Interest Ratio Rises 33% to 7 Days; 21/04/2018 DJ AXT Inc, Inst Holders, 1Q 2018 (AXTI); 11/04/2018 AXT: Demand Remains Soli; 14/03/2018 AXT Closes Below 200-Day Moving Average: Technicals; 24/05/2018 AXT Short-Interest Ratio Rises 63% to 10 Days; 11/04/2018 AXT INC COMPLETED FIRST PHASE OF FACILITIZATION OF ITS NEW MANUFACTURING FACILITY IN DINGXING, CHINA; 11/04/2018 AXT Inc. Lowers 1Q Guidance; 11/04/2018 AXT Completes First Phase of New Factory in Dingxing, China

Gamida Cell Ltd., a clinical stage biopharmaceutical company, focuses on developing cell therapies to cure cancer, and rare and serious hematologic diseases in the United States, the European Union, and internationally. The company has market cap of $182.65 million. The company's lead product candidate is NiCord, a nicotinamide -expanded cord blood cell therapy that is in Phase 3 clinical trials for use as a curative stem cell graft for patients in hematopoietic stem cell transplant. It currently has negative earnings. It is also developing NAM-NK, an innate immunotherapy of expanded natural killer cells, which is in Phase 1 clinical trials for the treatment of refractory non-Hodgkin lymphoma and multiple myeloma.

Analysts await Gamida Cell Ltd. (NASDAQ:GMDA) to report earnings on February, 24. After $-0.30 actual earnings per share reported by Gamida Cell Ltd. for the previous quarter, Wall Street now forecasts 30.00% negative EPS growth.

The stock increased 5.96% or $0.31 during the last trading session, reaching $5.43. About 92,358 shares traded or 204.50% up from the average. Gamida Cell Ltd. (NASDAQ:GMDA) has 0.00% since December 2, 2018 and is . It has by 0.00% the S&P500.

More notable recent Gamida Cell Ltd. (NASDAQ:GMDA) news were published by: Finance.Yahoo.com which released: Will Gamida Cell (NASDAQ:GMDA) Spend Its Cash Wisely? Yahoo Finance on November 19, 2019, also Seekingalpha.com with their article: Gamida Cell Ltd. (GMDA) CEO Julian Adams on Q3 2019 Results Earnings Call Transcript Seeking Alpha published on November 13, 2019, Businesswire.com published: Gamida Cell Announces the Date of Its Third Quarter 2019 Financial Results and Webcast Business Wire on November 05, 2019. More interesting news about Gamida Cell Ltd. (NASDAQ:GMDA) were released by: Businesswire.com and their article: Gamida Cell to Present at the 31st Annual Piper Jaffray Healthcare Conference Business Wire published on November 21, 2019 as well as Businesswire.coms news article titled: Gamida Cell Announces Data to be Presented at ASH 2019 Annual Meeting Business Wire with publication date: November 06, 2019.

AXT, Inc. designs, develops, manufactures, and distributes compound and single element semiconductor substrates. The company has market cap of $127.10 million. The firm makes its semiconductor substrates using its proprietary vertical gradient freeze technology. It currently has negative earnings. It offers semi-insulating gallium with arsenic substrates, which are used for applications in power amplifiers for wireless devices, and transistors and solar cells for drones.

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BidaskScore Ups Gamida Cell (GMDA); Shorts at Axt (AXTI) Lowered By 0.34% - The Broch Herald

Outcome means a special Thanksgiving

Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

They still need to travel to Charleston once a week to see the transplant doctor. In between, Maci is seen in Greenville.

She's doing well, but recovery from a transplant can take months to years, Bryant said.

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Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Read or Share this story: https://www.greenvilleonline.com/story/news/health/2019/11/27/upstate-sc-toddler-survives-rare-cancer-and-risky-procedure-treat/4158606002/

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Upstate SC toddler survives rare cancer and the risky procedure used to treat it - Greenville News

Dr Sister Ltd continues to offer effective skin treatments to both men and women, and they have been doing this for over 45 years now. Doctor Sister provides a comprehensive portfolio of treatments while at the same time delivering natural-looking, regenerative and enhancing results for the face and body. They are big believers in helping slow down the ageing process for each client, as of course, they are unable to stop time for you.

At Dr Sister Ltd, you can be treated by a professional doctor that trains other practitioners. Dr Sisterhimself has introduced over ten ground-breaking treatments to the UK market, along with eight published books, many articles in international peer reviews medical journals and general press, as well as being one of UKs and Europes leading lecturer and trainer in the field of Aesthetic Medicine.

The treatments offered by Dr Sister Ltd are non-invasive so there is no surgery and no downtime. Some of the skin treatments on offer at Dr Sister Ltd include the following; mini face lift, non-surgical face lift, vampire facial, PRP treatment and PRP injection. That is not an exhaustive list and he is also a renowned hormonal expert.

Dr. Sister has perfected safe, effective, natural-looking treatments, which has made him a worldwide expert and teacher in regenerative and innovative procedures such as Dracula PRP, Mint Lift including the new Stem Cell Facelift.

The PRP treatment (Dr. Sister has his own superior trademarked version called Dracula Therapy) may be unfamiliar to some clients. Dr Sister explains the procedure in great detail on their site. APRP treatment is a powerful anti-ageing treatment that involves using your blood as an injectable treatment (PRP Injection). Dr Daniel Sister was the first to introduce the treatment into the UK, and now he calls it Dracula Therapy.

With the Dracula Therapy or vampire facial, you will notice results within 3-4 weeks, and often only one PRP injection is required. However, the treatment may need to be repeated every 2-6 months because of the on-going ageing process.

The PRP injections generally appeal to patients looking for a more natural approach to facial rejuvenation, which is the rejuvenation process of using their cells. This treatment does not use synthetic fillers or animal products and has no risks or side effects.

At Dr Sister Ltd, they are well known for their aesthetic treatments, in particular, the MINT lift and Dr Sister is the training partner for the MINT lift. It is a PDO thread lift that offers exceptional results. Dr. Sister has been particularly impressed by the results as it provides an immediate and obvious lift, which many of his patients are looking for.

Dr Sister Ltd also mentions that local anaesthetic is used making the procedure pain free, and patients generally return to work and usual activities the following day. There are many benefits such as soft tissue lifting, instant lift, results lasting around 18 months.

If you would like to find out more about the treatments on offer at Dr Sister Ltd, there are many ways to get in touch. You can email press@drdanielsister.com your query, and they will get back to you as soon as possible, or you can go online to their website at https://drdanielsister.com. On their site, you will find all the information about the top treatments, fees, testimonials, and Dr Sister Ltd.

Source:https://thenewsfront.com/dr-sister-ltd-offers-effective-skin-treatments-to-both-men-and-women/

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Dr Sister Ltd Offers Effective Skin Treatments to Both Men and Women - The News Front

A research report on Transplant Diagnostics Market by Persistence Market Research features an in-depth analysis on the latest market trends. The report also includes detailed abstracts about statistics, revenue forecasts and market valuation. The report also offers a detailed analysis on the competitive landscape of the market.

Organ transplantation is the need to relocate organ in order to treat organ failure such as liver, pancreas, lungs, kidney, and heart. Human leukocyte antigens are the antigens found on the surface of the cell that regulates the body recognition and rejects foreign tissue transplant. It is the major histocompatibility complex in the human beings, which is controlled by the genes located on the chromosome six. Human leukocyte antigen (HLA) diagnostic testing is performed to determine the tissue compatibility between the donor and recipient in organ and bone marrow transplant.

In addition, a close match between a donor and recipient HLA marker is essential. It increases the probability of graft survival and minimizes severe immunologic transplant complications. It is performed with the help of non-molecular assay and molecular assay. The non-molecular assay includes serological assay and mixed lymphocyte culture (MLC) assay. In addition, molecular assay comprises PCR-based assay (sequence -pecific primer PCR, and sequence-specific oligonucleotide PCR), and sequencing-based assay (Sanger sequencing and next-generation sequencing). Hospitals, transplant centers, research laboratories, academic institutes, and commercial service providers are the major end-users of transplant diagnostics.

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North America dominates the global market for transplant diagnostics due to continuous reduction in the average cost of gene sequencing, private-public funding for the development of HLA typing technology and rising number of stem cells, and solid organ-based transplantation in the region. Asia is expected to show a high growth rate in the next five years in the global transplant diagnostics market with China and India being the fastest growing markets in the Asia Pacific region. The key driving forces for the transplant diagnostics market in developing countries are the large pool of patients, improving healthcare infrastructure, rising public and private support to develop human leukocyte antigen typing technologies, and growing focus of life science companies in the region.

Growing geriatric population, rising number of soft tissue, solid and stem cell based transplantation, rising prevalence of chronic diseases, growth in robot-assisted laboratory automation of diagnostic procedures, and technological advancement in the field of human leukocyte antigen (HLA) typing are some of the key factors driving the growth of the global transplant diagnostics market. In addition, increase in public-private investment to develop innovative human leukocyte antigen (HLA) testing products, rising application of HLA typing products in clinical diagnostics, improving healthcare infrastructure in developing countries, continuous reduction in average cost of gene sequencing, and increasing installation base of PCR and NGS instruments are driving the growth of the global transplant diagnostic market. However, factors such as high cost of PCR and NGS instruments, limited medical reimbursement for transplantation procedure, and a huge gap between organ donation and transplantation annually act as major restraints for the growth of the global transplant diagnostics market.

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Increasing shift of preference from serological assay method to genome-based HLA profiling, and rising market penetration in developing countries to develop transplant diagnostic products would pose further opportunities for the growth of the transplant diagnostic market. New product launches and product development are among the major trends for the global transplant diagnostics market.

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Transplant Diagnostics Market to Witness Heightened Revenue Growth During the Forecast Period (2015-2021) - Statsflash

New Report on Absorbable and Non-Absorbable Sutures Market size | Industry Segment by Applications (Cardiovascular, Orthopedic, Gynecology, Opthalmology, General Surgery and Others), by Type (Absorbable Sutures and Non-Absorbable Sutures), Regional Outlook, Market Demand, Latest Trends, Absorbable and Non-Absorbable Sutures Industry Share & Revenue by Manufacturers, Company Profiles, Growth Forecasts 2025. Analyzes current market size and upcoming 5 years growth of this industry.

A comprehensive analysis of the Absorbable and Non-Absorbable Sutures market is presented in this document, along with a brief overview of the segments in the industry. The study presents a feasible estimate of the current market scenario, including the Absorbable and Non-Absorbable Sutures market size with regards to the volume and renumeration. The report is a collection of significant data related to the competitive landscape of the industry. It also contains data with regards to several regions that have successfully established its position in the Absorbable and Non-Absorbable Suturesmarket.

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Repot Scope:

Absorbable and Non-Absorbable Sutures Market Outlook by Applications:

Absorbable and Non-Absorbable Sutures Market Statistics by Types:

Absorbable and Non-Absorbable Sutures market competition by top Manufacturers:

A Glimpse over the highlights of the report:

Providing a thorough outline of the competitive and regional spheres of the Absorbable and Non-Absorbable Sutures market:

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Absorbable and Non-Absorbable Sutures Market Research, Recent Trends and Growth - News by aeresearch

Credit: ACS Appl. Mater. Interfaces

An array of platinum nanostraws can be used to deliver molecules to cells or sample their contents.

Hollow nanosized needles, or nanostraws, are a promising tool for opening up tiny, temporary holes in cell membranes to deliver molecules or sample a cells contents. Nanostraws could also deliver gene editors into cells for immunotherapy, cutting the need to use costly viruses for the job. But making nanostraws requires expensive manufacturing equipment in a clean room facility, and using nanostraws often requires applying a high voltage in order to open up the cell membrane. Now, researchers have developed a more affordable fabrication approach that can be done in an ordinary lab. Whats more, the new nanostraws are conductive, thus lowering the amount of voltage needed to levels less likely to damage cells (ACS Appl. Mater. Interfaces 2019, DOI: 10.1021/acsami.9b15619).

Researchers made earlier iterations of nanostraws with atomic layer deposition (ALD), which grows thin films of materials such as metal oxides one layer of atoms at a time. In their new approach, Xi Xie of Sun Yat-Sen University and colleagues replaced ALD with electroplating, a simple process which uses an electrical potential to deposit ions in a solution onto a surface.

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They first sputtered a thin layer of gold on the bottom surface of a polycarbonate template containing an array of pores in order to make a conductive base layer. Then they electroplated platinum, gold, or the conductive polymer poly(3,4-ethylenedioxythiophene)three common materials used in electrophysiology studiesfrom the top. The materials lined the pores of the template, creating the hollow nanostraws. The team then used mechanical polishing and oxygen plasma etching to remove the polycarbonate template, revealing an array of vertical nanostraws, each a few hundred nanometers in diameter. According to Xie, their method can work with templates of various pore sizes or pore densities, or with other plating materials.

Ciro Chiappini, a nanomedicine researcher at Kings College London, says this study is a needed and significant step toward developing affordable nanostraws.

Using a representative platinum nanostraw array, Xie and colleagues demonstrated that they could deliver a fluorescent dye into cultured human cells and extract intracellular materials to examine how the levels of an enzyme changed over time.

The conductivity of the new nanostraws allowed the researchers to open tiny pores in the cell membrane by applying a voltage of only 35 V, a safer range for cells compared with 1020 V needed when using nonconductive nanostraws.

These straws could make cellular treatments such as CAR-T therapy faster, safer, and cheaper, says Nicholas A. Melosh, a materials scientist at Stanford University who has done nanostraw research. Typical immunotherapy delivers therapy to a patients immune cells using viruses, which is costly and carries the risk of dangerous immune responses once the cells are put back into the patient, he says. Nanostraws could potentially deliver the necessary therapies to cells without the need for viruses.

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Electroplating method makes conductive nanostraws for injecting into and sampling from cells - Chemical & Engineering News