Genetherapy

Ventilation Abnormalities Are Unlikely Random Among Patients With Asthma – Pulmonology Advisor

January 3rd, 2020

In contrast to previous in silico modeling studies, ventilation abnormalities do not appear randomly distributed among patients with asthma, and may persist in the same lung regions during a prolonged period. These findings were published in CHEST.

In a case study, researchers prospectively followed-up nonidentical female adult twins with lifelong asthma for 2 study visits between January 2010 and March 2017. Pulmonary magnetic resonance imaging, computed tomography imaging, and pulmonary function tests were used to prospectively evaluate the patients during this 7-year period.

Twins had parents who were heavy tobacco smokers in the home, and both parents had a history of airway disease. Different asthma specialists independently prescribed the twins 400 g daily budesonide combined with formoterol (patient 1: once-daily 200/6 g 2 puffs; patient 2: twice-daily 200/6 g 1 puff). Both patients reported weak to moderate controller medication adherence.

At baseline, each twin demonstrated spatially identical focal ventilation defects, and both twins showed left-sided upper lobe ventilation abnormalities at follow-up. Patients had a similar subsegmental airway wall area percentage at follow-up (71% in patient 1 and 75% in patient 2), which the researchers found substantially abnormal, based on the published literature.

Fewer airways were found in patient 2 vs patient 1 (166 vs 202, respectively), as demonstrated in airway number by airway tree generation distal to left-sided upper lobe apicoposterior bronchopulmonary segment and right-sided upper lobe apical bronchopulmonary segment.

Limitations of the study included using only 2 time points for evaluation and the lack of adjustment for shared genetics or in utero events.

If ventilation defects occur randomly in patients with asthma, the researchers wrote, the probability of this occurring in both patients in the same location, twice over 7 years, is approximately one in 130,000 people.

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors disclosures.

Reference

Eddy RL, Matheson AM, Svenningsen S, et al. Nonidentical twins with asthma: spatially matched CT airway and MRI ventilation abnormalities. CHEST. 2019;156(6):e111-e116.

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Ventilation Abnormalities Are Unlikely Random Among Patients With Asthma - Pulmonology Advisor

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‘I want to be real’: Ricki Lake debuts shaved head after years of struggling with ‘debilitating’ hair loss – Yahoo Style

January 3rd, 2020

Ricki Lake has opened up about about her private struggle with hair loss in a candid post to social media.

The 51-year-old actress and producer rang in the New Year feeling liberated and free after debuting a shaved head. In a lengthy post onFacebook, Lake detailed her nearly 30-year history of hair loss, in hopes that her story will help those who suffer in silence feel less alone.

I have been struggling with hair loss for most of my adult life, Lake wrote. It has been debilitating, embarrassing, painful, scary, depressing, lonely, all the things. There have been a few times where I have even felt suicidal over it. Almost no one in my life knew the level of deep pain and trauma I was experiencing. Not even my therapists over the years knew my truth.

Lake, who landed her first role in the 1988 film Hairspray by director John Waters, says her journey from Hairspray to hairless was likely caused by years of yo-yo-dieting, taking birth control, pregnancy, genetics and stress. The former Ricki Lake Show host relied on hair extensions and wigs to mask her hair loss.

(Photo by Amy Sussman/Getty Images)

It all felt fake and I was super self-conscious and uncomfortable, she said. Ive been to many doctors, gotten steroid shots in my head, [have taken] all the supplements and then some. My hair would recover and then shed again. It was maddening.

ALSO SEE: Model Iskra Lawrence shows off her curves in nude pregnancy shoot

Lake is not alone in her struggle with female hair loss. According to theCanadian Dermatology Association, 40 per cent of women will experience thinning hair and hair loss by the age of 50. In the United States,approximately30 million women experience noticeable hair loss including female pattern baldness and different forms of alopecia.

Ricki Lake. Image via Facebook/MsRickiLake. Photo by Amanda Demme.

Whilemost female hair loss occurs over the age of 40, women of any age can notice an increase of shedding and thinning hair. Genetics, tight or frequent hair styling, extreme stress, hormonal changes, chemotherapy and some medications all contribute to female hair loss.

An extreme diet that resulted in a rapid weight loss of 20 pounds in six weeks lead to a noticeable increase in hair loss, prompting Lake to take control of the situation and set herself free. She decided to forego hair extensions and hair colour, and shave her head.

Story continues

ALSO SEE: 'Sometimes we want to give up': Justin Bieber gets candid about mental health struggles

It is a New Year and a new decade and a new me...I buzzed my hair off and it feels so good! she wrote, giving thanks to her friends and partner Jeff Scult for their support.

(Photo by JB Lacroix/WireImage)

While she notes that she will continue to play with hair for fun, any changes to her look in the future will be done by choice, and not out of shame.

I am liberated. I am free. I am releasing and letting go. I am brave. I am beautiful. I am love, Lake told followers. For 2020 and beyond, I want to be real.

Let us know what you think by commenting below and tweeting @YahooStyleCA!Follow us onTwitterandInstagram.

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'I want to be real': Ricki Lake debuts shaved head after years of struggling with 'debilitating' hair loss - Yahoo Style

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Free Gene Therapy Available for Patients with Alzheimer’s – HealthITAnalytics.com

January 3rd, 2020

January 03, 2020 -Maximum Life Foundation (MaxLife), a non-profit organization focused on aging research, is providing a promising free gene therapy for ten patients with Alzheimers disease.

According to the Alzheimers Association, Alzheimers disease is the sixth leading cause of death in the US. Over five million Americans have the condition, leading to costs of $277 billion a year.

With this gene therapy, researchers have seen improvements in Alzheimers symptoms and the recovery of normal brain functions in experiments with mice. In human cell experiments, the therapy had the same effects through the rejuvenation of microglia, the brains first line of defense against infection, and neurons.

In August 2018, a patient received a low dose of the therapy with no adverse side effects. To date, the patients disease hasnt progressed.

MaxLife will grant 100 percent of the therapy costs to help bring pioneering gene therapy to cure this disease and make Alzheimers disease a thing of the past, said David Kekich, MaxLifes CEO.

Studies have proven that aging is the leading factor in many life-threatening diseases, including Alzheimers. This new gene therapy aims to treat the cellular degeneration caused by aging.

The new treatment is offered by Integrated Health Systems, a gene therapy facilitator that is seeking to treat other adult aging-related diseases with no known cure, including sarcopenia, chronic kidney disease, and atherosclerosis.

This technology could halt many of the big age-associated killers in industrialized countries, said Kekich. Compassionate care helps patients with no other option to get access to experimental therapies that may benefit both themselves and society as a whole.

Other healthcare organizations have stressed the need to leverage gene therapies and precision medicine to improve treatment for Alzheimers and other diseases. A recent study published in Frontiers in Aging Neuroscience discussed how precision medicine tactics will help improve cognitive disease treatment.

Taking a precision medicine approach, the question is no longer Does treatment work? but Who does treatment work for? Identifying the characteristics of non-responders becomes as important as responders in understanding the impact of a particular intervention, the team said.

Such an approach may result in considerable health benefits by allowing more effective selection of individuals for treatments based ona prioriknown profiles of disease risk and their potential response to treatment.

Researchers at Massachusetts General Hospital (MGH) also recently discovered that certain genetic variants may help protect individuals against Alzheimers disease, a finding that could hold important implications for precision medicine therapies.

The team studied a patient who carried a mutation in a gene known to cause early onset Alzheimers but didnt show signs of mild cognitive impairment until her seventies. This is nearly three decades after the typical age of onset. Evaluating this patient, and patients like her, could help researchers understand more about the progression of Alzheimers.

This single case opens a new door for treatments of Alzheimers disease, based more on the resistance to Alzheimers pathology rather than on the cause of the disease. In other words, not necessarily focusing on reduction of pathology, as it has been done traditionally in the field, but instead promoting resistance even in the face of significant brain pathology, said Yakeel T. Quiroz, PhD, clinical neuropsychologist and neuroimagingresearcher at MGH.

With the new gene therapy, MaxLife will add to the growing body of research exploring the use of precision medicine and genetics in chronic disease treatment.

If we can prove a benefit to patients that have no other option now, we can potentially treat Alzheimers disease in people in early to mid-stage Alzheimers, finally creating effective medicine at the cellular level, states Kekich. If successful, this treatment could potentially be used on other diseases such as Parkinsons and ALS.

To apply for a free therapy or for more information, click here.

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Free Gene Therapy Available for Patients with Alzheimer's - HealthITAnalytics.com

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Duke researchers land $6M in federal grants to advance gene editing – WRAL Tech Wire

January 3rd, 2020

DURHAM Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the source but significant hurdles must be overcome before genome-editing treatments are ready for use in humans.

Enter the National Institutes of Health Common FundsSomatic Cell Genome Editing (SCGE)program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.

Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.

In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.

Duke engineers improve CRISPR genome editing with biomedical tails

There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.

For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.

Duke researchers: Single CRISPR treatment provides long-term benefits in mice

Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.

Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.

Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing bothnew CRISPR systems for gene regulationandto regulate the epigenome rather than permanently delete DNA sequences.

Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in usingAAV and CRISPR to treat animal models of DMD.

We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.

Click through to learn more about theDuke Center for Advanced Genomic Technologies.

(C) Duke University

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Duke researchers land $6M in federal grants to advance gene editing - WRAL Tech Wire

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New Batten Research Key to Informing Potential Therapies, Review Says – Batten Disease News

January 3rd, 2020

New research has deepened the understanding of the underlying causes of Batten disease, including organs and cell types affected as well as crucial molecular mechanisms, which can help the design of novel therapies, a review study reports.

Researchers believe these new insights will be key to inform the targeting, timing, and strategies for future treatments.

The study, Pathomechanisms in the neuronal ceroid lipofuscinoses, was published in the journal Biochimica et Biophysica Acta (BBA) Molecular Basis of Disease.

Batten disease, also known as neuronal ceroid lipofuscinoses (NCLs), is a group of inherited neurodegenerative disorders that share certain clinical symptoms. The various forms of the disease are caused by different mutations and distinguished in part by the age at which symptoms appear.

All are lysosomal storage disorders (LSDs) characterized by the abnormal accumulation of fatty substances, known as ceroid and lipofuscin, inside cells in compartments called lysosomes, which are responsible for breaking down and recycling cell materials.

This buildup is particularly toxic to nerve cells (neurons) and leads to progressive deterioration of the brain, even though other tissues can also be affected.

Novel clinical and preclinical findings have deepened scientists understanding of what causes Batten disease and how these disorders progress over time.

The study reviews these insights, which could facilitate the development of new treatments that target each disease type.

Major advances have been made in therapies targeting the central nervous system, or CNS (comprising the brain and spinal cord), but NCLs should be considered diseases that can affect multiple organ systems, and not just the brain as has been the traditional view, the researchers wrote.

Evidence shows that multiple organs can be affected, with disease spreading to other body regions depending on disease type. Mapping all the tissues affected will be important to refine therapeutic delivery and timing, the researchers said.

Shrinkage, or atrophy, of the brain, accompanied by enlargement of the lateral ventricles (cavities within the brain filled with cerebrospinal fluid) is a common finding in Batten disease. But as the proteins affected in Batten disease are widely expressed in various tissues and cell types, it is likely that other organs are also affected by these disorders.

Observations in animal models and patients, for instance, suggest that the spine, as well as vision, the heart, and the bowel, are likely affected in multiple NCLs.

Identifying and targeting all organs and tissues involved, which so far have been overlooked, is important and could provide added benefit to treatment approaches for Batten disease, the scientists said.

Specific populations of neurons are more vulnerable to Batten disease. Early in the disease, interneurons neurons that connect sensory and motor neurons within the CNS are lost in several regions in the brain. Moreover, a type of neuron involved in controlling motor movement, called Purkinje cells, also seem to be particularly vulnerable.

While the reasons for this are still unclear, the unique biological and electrical properties of these neurons and their greater dependence on lysosomes could explain why they are more vulnerable to Batten disease.

Researchers have also been reconsidering the role played by glial cells, which are cells of the nervous system that provide protection and support to neurons.

Although Batten disease has been considered a disease of neurons, the abnormal accumulation of fatty and proteic substances that mark these disorders occurs in various cell types across the body, in addition to the nervous system.

A growing body of evidence suggests that the activation of astrocytes and microglia two types of glial cells precedes and more accurately predicts where neuronal loss is going to occur, when compared to the actual measurement of fatty material accumulation.

In cell models of CLN1 (known as infantile Batten disease) and CLN3 (known as juvenile Batten disease), astrocytes and microglia were seen to cause neuronal loss, which suggests that they have an important role in the development of Batten disease.

There is also evidence for an antibody-mediated immune response in Batten disease, with a possible autoimmune component a harmful immune response that attacks the bodys own tissues especially in CLN3.

In addition to their role in degrading cell waste, lysosomes are involved in other processes such as sensing nutrients and balancing the levels of calcium and metals, as well as the transport and communication between nerve cells.

Likely related to that is the fact that various Batten disease models are characterized by synaptic dysfunction a malfunction of the synapse, or the junctions between two nerve cells that allow them to communicate.

Other cellular pathways linked to lysosomes including autophagy (the self-eating waste disposal system of cells) and gene activation routes may also be abnormal in Batten disease and contribute to its development.

This information sheds light on potential mechanisms by which NCL mutations may lead to disease, beyond the role of lysosomes.

Various investigational therapies have gone through preclinical tests, including immunomodulatory agents, modulators of lysosomal function, agents that mimic the deficient enzyme in a particular NCL, and inhibitors of glutamate receptor (cell receptors important for transmitting signals between neurons). All these approaches have had varying degrees of success, the review stated.

Some medicines have been tested in patients, such as the immunosuppressive agent mycophenolate mofetil, sold under the brand name CellCept, which was evaluated in a Phase 2 study for CLN3 (NCT01399047).

Cystagon, a molecule that mimics PPT1 (the protein deficient in CLN1), has also been clinically tested in a Phase 4 clinical trial (NCT00028262). However, the benefits of both treatments have been only modest in patients.

This has further highlighted the importance of targeting [disease mechanisms] that are specific to each form of the disease, the researchers wrote.

They believe that targeting the known common defects in neuroinflammation and autophagy may help to develop add-on therapies that could greatly improve the therapeutic efficacy as compared to single-therapy strategies.

Moreover, the discovery of disease manifestations at unexpected sites within or outside the CNS will necessitate the development of therapies that can be targeted to these tissues successfully, the researchers wrote.

Defining the timing of disease in these different tissues, in relation to events in the CNS, will provide important information about effective therapeutic windows and is currently informing the design of various gene therapy clinical trials.

These include ongoing Phase 1/2 clinical trials at the Nationwide Childrens Hospital, in Ohio, testing Amicus Therapeutics gene therapies: AAV-CLN6 for CLN6 disease (NCT02725580) and AAV9-CLN3 for CLN3 (NCT03770572).

To evaluate a gene therapy for CLN2, safety and efficacy studies (NCT00151216, NCT01414985, and NCT01161576) are being conducted at Weill Cornell Medical College in New York.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 14

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Batten Research Key to Informing Potential Therapies, Review Says - Batten Disease News

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What Does 2020 Have in Store for the Life Sciences? – BioSpace

January 3rd, 2020

With the books being closed on 2019, its time to prognosticate about what the next year will hold in the biotech and pharmaceutical industry. And, to paraphrase a famous song from the 1980s, grab a set of shades, because the future looks bright.

BioSpace spoke to leaders from various corners of the industry who provided their insights into what the coming year is likely to hold for their particular sphere. Below are predictions for three spheres within the industry for the coming year.

Oncology

Immuno-oncology has been one of the research cornerstones in cancer research and that will continue. However, in 2020, traditional targeted therapies will continue to be of importance. Stephen Gately, chief executive officer of TD2 (Translational Drug Development), an oncology-focused contract research organization, said there are still roles for traditional targeted therapeutics for specific indications. Following Mercks acquisition of ArQule, Inc. and its kinase inhibitor discovery and development programs for cancer treatment, predicted the industry might see a refocus on understanding cancer and how it can respond to different drugs. He said Merck would not have spent $2.7 billion for ArQules Phase II Brutons tyrosine kinase inhibitor ARQ 531 if there was not a potential upside for the therapy, particularly as a treatment for B-cell cancers. If trial protocols are well-established and there are stricter guidelines for patient selections, Gately said the use of certain inhibitors are vastly superior to immunotherapies in development.

When it comes to clinical trials, Gately predicted there could be a change in how those are organized. He said the goal for companies is to attempt to get their assets into trials as quickly as possible but noted there is a heated and competitive battle for trial sites. As companies attempt to elbow their way to the front of the line, Gately said it is possible that the industry will begin to see more opportunities for economic incentives used to convince the clinical trial sites to take up trials sooner. If that is established, Gately said it will become a game-changer.

Cell and Gene Therapies

This past year saw the approval of gene therapies that can address devastating diseases. And that is likely to continue in 2020. But, safety will still be paramount. Ena Cratsenburg, chief business officer of Ginkgo Bioworks, said over the course of the next year, we will see drug developers use synthetic biology to improve their ability to develop therapeutics with unprecedented levels of activity and control. In 2020, synbio companies will solidify their positions as key enablers in bringing the most innovative medicines to consumers, Cratsenburg said.

Mark Sawicki, chief commercial officer at Cryoport, developer of the first shippers certified for the delivery of human advanced therapies, said that as the cell and gene therapies continue to show their importance for treating new diseases, 2020 will usher in a record year for biologics license applications and marketing authorization applications. Sawicki suggested that over the course of the coming year, eight or nine companies will file such applications to gain regulatory approval of their products. In addition to the high number of filing companies, he suggested that there will be multiple new products launching with three or four generating revenues in 2020. By 2023, Sawicki predicts there will be at least 22 commercial launches in the space.

Jason Steiner, vice president of business development and strategy at gene editing company Synthego, said in 2020, the gap between the pace of innovation in the cell and gene therapy landscape and the infrastructure required for commercialization and deployment will continue to widen before it converges again.Steiner said the development of engineered cell therapies will continue to move toward non-viral approaches in order to boost engineering sophistication while decreasing the time and cost of traditional engineering. He added that manufacturing consideration of cell and gene therapies will continue to move farther upstream. They will be more tightly integrated with early development to avoid commercialization bottlenecks that are being caused by a rapid increase in the pace of development and regulatory approval cycles.

Real World Evidence

The importance of Real World Evidence (RWE) will continue to grow in 2020. Jane Reed, director of life sciences at U.K.-based Linguamatics, said the industry will continue to harness RWE in its drug development programs. RWE was not a topic of conversation in the industry 10 years ago, Reed said, but now there is significant value seen from what RWE brings to the table. RWE relates to the collection of information about a drugs safety and efficacy outside the structure of a clinical trial. The data can be collected from a variety of sources, including electronic health records, wearable devices, lab tests and more. The pharma industry will need to not only continue to collect the data, but learn to use it in a way that improves the outcomes of the patients it serves. Reed said the industry has to show they are listening to the patient community and be flexible. From there, the industry will need to feed that patient-driven data into drug discovery efforts. To boost the collection of RWE, Reed said the use of targeted social media research will also increase the amount of data that companies have at their fingertips. As companies within the pharmaceutical industry continue to dig into the depths of disease states, particularly those of rare diseases, Reed said the use of RWE will prove to be increasingly important.

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What Does 2020 Have in Store for the Life Sciences? - BioSpace

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Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 – GlobeNewswire

January 3rd, 2020

Dublin, Jan. 03, 2020 (GLOBE NEWSWIRE) -- The "Cell and Advanced Therapies Supply Chain Management Market, 2019-2030: Focus on Technological Solutions" report has been added to ResearchAndMarkets.com's offering.

Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030 report features an extensive study of the growing supply chain management software solutions market.

The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030.

In addition, we have provided the likely distribution of the current and forecasted opportunity across:

Advanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions.

In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies.

In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers.

This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed.

The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

Amongst other elements, the report features:

In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

The opinions and insights presented in this study were influenced by discussions conducted with several stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. An Introduction to Cell and Advanced Therapies3.2.1. Classification of Advanced Therapy Medicinal Products3.2.2. Current Market Landscape3.3. Cell and Advanced Therapies Supply Chain3.3.1. Key Processes3.3.2. Challenges Associated with the Cell and Advanced Therapies Supply Chain3.4. Software Solutions for Cell and Advanced Therapies Supply Chain Management3.4.1. Cell Orchestration Platform3.4.2. Enterprise Manufacturing System3.4.3. Inventory Management System3.4.4. Laboratory Information Management System3.4.5. Logistics Management System3.4.6. Patient Management System3.4.7. Quality Management System3.4.8. Tracking and Tracing System3.5. Growth Drivers and Roadblocks3.6. Emergence of Digital Technologies in Supply Chain Management3.6.1. Blockchain Technology3.6.2. Internet of Things3.6.3. Augmented Reality3.6.4. Big Data Analytics3.6.5. Artificial Intelligence

4. CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. Cell and Advanced Therapies Supply Chain Management: Overall Market Landscape4.2.1. Analysis by Type of Software Solution4.2.2. Analysis by Key Specification and Benefit4.3.3. Analysis by Application4.3.4. Analysis by End User4.3.5. Analysis by Mode of Deployment4.3.6. Analysis by Scale of Management4.3.7. Analysis by Regulatory Certifications / Accreditations4.3. Cell and Advanced Therapies Supply Chain Management: Developer Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Location of Headquarters4.2.3. Analysis by Size of Company4.3.4. Analysis by Support Services Offered4.3.5. Leading Developers: Analysis by Number of Software Solutions

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. Competitiveness Analysis: Overview of Supply Chain Management Software Solution Providers5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. CORE SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS: COMPANY PROFILES6.1. Chapter Overview6.2. Brooks Life Sciences6.2.1. Company Overview6.2.2. Financial Information6.2.3. BiobankPro: Software Description6.2.4. Recent Developments and Future Outlook6.3. Cryoport6.3.1. Company Overview6.3.2. Financial Information6.3.3. Cryoportal: Software Description6.3.4. Recent Developments and Future Outlook6.4. MasterControl6.4.1. Company Overview6.4.2. MasterControl Platform: Software Description6.4.3. Recent Developments and Future Outlook6.5. SAP6.5.1. Company Overview6.5.2. Financial Information6.5.3. SAP S/4HANA: Software Description6.5.4. Recent Development and Future Outlook6.6. Savsu Technologies6.6.1. Company Overview6.6.2. Financial Information6.6.3. evo Cold Chain 2.0: Software Description6.6.4. Recent Development and Future Outlook6.7. TraceLink6.7.1. Company Overview6.7.2. Financial Information6.7.3. Digital Supply Chain Platform: Software Description6.7.4. Recent Developments and Future Outlook

7. CELL ORCHESTRATION PLATFORMS: EMERGING TRENDS AND PROFILES OF KEY PLAYERS7.1. Chapter Overview7.2. Supply Chain Orchestration Platforms7.2.1. Key Functions of Supply Chain Orchestration Platforms7.2.2. Advantages of Supply Chain Orchestration Platforms7.2.3. Supply Chain Orchestration Platform Implementation Strategies7.3. Supply Chain Orchestration Platform: Trends on Twitter7.3.1. Scope and Methodology7.3.2. Historical Trends in Volume of Tweets7.3.3. Popular Keywords7.4. Key Industry Players7.4.1. Be The Match BioTherapies7.4.2. Clarkston Consulting7.4.3. Haemonetics7.4.4. Hypertrust Patient Data Care7.4.5. Lykan Bioscience7.4.6. MAK-SYSTEM7.4.7. sedApta Group7.4.8. Stafa Cellular Therapy7.4.9. Title 21 Health Solutions7.4.10. TrakCel7.4.11. Vineti

8. FUNDING AND INVESTMENT ANALYSIS8.1. Chapter Overview8.2. Types of Funding8.3. Cell and Advanced Therapies Supply Chain Management: Recent Funding Instances8.3.1. Analysis by Number of Funding Instances8.3.2. Analysis by Amount Invested8.3.3. Analysis by Type of Funding8.3.4. Analysis by Number of Funding Instances and Amount Invested across Different Software Solutions8.3.5. Most Active Players: Analysis by Amount Invested8.3.6. Most Active Investors: Analysis by Participation8.3.7. Geographical Analysis by Amount Invested8.4. Concluding Remarks

9. PARTNERSHIPS AND COLLABORATIONS9.1. Chapter Overview9.2. Partnership Models9.3. Cell and Advanced Therapies Supply Chain Management: Recent Collaborations and Partnerships9.3.1. Analysis by Year of Partnership9.3.2. Analysis by Type of Partnership9.3.3. Analysis by Partner's Focus Area9.3.4. Analysis by Type of Software Solution9.3.5. Most Active Players: Analysis by Number of Partnerships9.3.6. Analysis by Regions

10. PLATFORM UTILIZATION USE CASES10.1. Chapter Overview10.2. Cell and Advanced Therapies Supply Chain Management: Recent Platform Utilization Use Cases10.2.1. Analysis by Year of Utilization10.2.2. Analysis by User's Focus Area10.2.3. Analysis by Type of Software Solution10.2.4. Most Active Players: Analysis by Number of Utilization Instances10.2.5. Most Active Players: Regional Analysis by Number of Utilization Instances

11. VALUE CHAIN ANALYSIS11.1. Chapter Overview11.2. Cell and Advanced Therapies Value Chain11.2. Cell and Advanced Therapies Value Chain: Cost Distribution11.3.1. Donor Eligibility Assessment11.3.2. Sample Collection11.3.3. Manufacturing11.3.4. Logistics11.3.5. Patient Verification and Treatment Follow-up

12. STAKEHOLDER NEEDS ANALYSIS12.1. Chapter Overview12.2. Cell and Advanced Therapies Supply Chain Management: Needs of Different Stakeholders12.2.1. Comparison of Stakeholder Needs

13. COST SAVINGS ANALYSIS13.1. Chapter Overview13.2. Key Assumptions and Methodology13.3. Overall Cost Saving Potential of Supply Chain Management Software Solutions, 2019-203013.3.1. Cost Saving Potential in Donor Eligibility Assessment, 2019-203013.3.2. Cost Saving Potential in Sample Collection, 2019-203013.3.3. Cost Saving Potential in Manufacturing, 2019-203013.3.4. Cost Saving Potential in Logistics, 2019-203013.3.5. Cost Saving Potential in Patient Verification and Treatment Follow-up, 2019-2030

14. MARKET FORECAST14.1. Chapter Overview14.2. Key Assumptions and Forecast Methodology14.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market, 2019-203014.3.1. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application14.3.2. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by End User14.3.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Type of Software Solution14.3.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Mode of Deployment14.3.5. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Geography14.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application, Type of Software Solution and Mode of Deployment14.4.1. Cell and Advanced Therapies Supply Chain Management Solutions Market for Donor Eligibility Assessment, 2019-203014.4.2. Cell and Advanced Therapies Supply Chain Management Solutions Market for Sample Collection, 2019-203014.4.3. Cell and Advanced Therapies Supply Chain Management Solutions Market for Manufacturing, 2019-203014.4.4. Cell and Advanced Therapies Supply Chain Management Solutions Market for Logistics, 2019-203014.4.5. Cell and Advanced Therapies Supply Chain Management Solutions Market for Patient Verification and Treatment Follow-up, 2019-2030

15. EXECUTIVE INSIGHTS15.1. Chapter Overview15.2. Thermo Fisher Scientific15.2.1. Company Snapshot15.2.2. Interview Transcript: Bryan Poltilove, Vice President and General Manager15.3. Cell and Gene Therapy Catapult15.3.1. Company Snapshot15.3.2. Interview Transcript: Jacqueline Barry, Chief Clinical Officer15.4. McKesson15.4.1. Company Snapshot15.4.2. Interview Transcript: Jill Maddux, Director, Cell and Gene Therapy Product Strategy, and Divya Iyer, Senior Director, Corporate Strategy and Business Development15.5. TrakCel15.5.1. Company Snapshot15.5.2. Interview Transcript: Martin Lamb, Chief Business Officer

16. CONCLUDING REMARKS16.1. Chapter Overview16.2. Key Takeaways

17. APPENDIX 1: LIST OF ADDITIONAL SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS

18. APPENDIX 2: TABULATED DATA

19. APPENDIX 3: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/kw1hkc

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BLA Submitted for Gene Therapy to Treat Hemophilia A – Hematology Advisor

January 3rd, 2020

Home Topics Bleeding Disorders

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec.

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A, said Hank Fuchs, MD, President, Global Research and Development at BioMarin. Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.

For more information visit biomarin.com.

This article originally appeared on MPR

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The Face of Science – Clemson World magazine

January 3rd, 2020

That next day, Drake preferred sleeping over eating. But then, thats common with newborns. Tarah and Eric would wake him for feeding, careful to make sure he got plenty of nourishment.

By Saturday, these experienced parents became uneasy. Drake was just too lethargic. It was harder to wake him for feedings. The OSullivans called Drakes doctor and were assured there was nothing to be concerned about; Drake had been healthy when he left the hospital two days ago. And, the doctors office assured them, they would be checking him again on Monday at a scheduled office visit.

But the OSullivans disquiet grew by the hour. By Sunday evening, Drake would not open his eyes or respond to them. He was growing limp and struggling to breathe. The OSullivans rushed Drake to the hospital where the staff flew into emergency mode. Too sick for care at the local hospital, Drake was stabilized for transport to the pediatric intensive care unit (PICU) at Greenville Memorial Hospital. Just 72 hours after birth, Drake lapsed into a coma. And no one knew why.

That unforgettable night was the beginning of a long journey of test after test and a diagnosis by elimination.

Drake continued to decline as each negative test pushed aside another horrible possibility. You would think that eliminating terrible diseases would be a good thing, says Eric. But that just meant we were looking at something very rare.

Finally, blood tests revealed an ever-elevating level of glycine in Drakes blood, a symptom of an extremely rare, genetic metabolic disease called nonketotic hyperglycinemia or NKH.

The words nonketotic hyperglycinemia meant nothing to Tarah and Eric. But the next words were clear: Drake had a less than 10 percent chance of survival.

The diagnosis was like a starters pistol for the OSullivans. From that moment, everything would be a race against time to save Drake.

After 28 days of tests, monitors, tubes and wires, Drake was released to go home. There, as Tarah explains, Our house became a sort of lab. There were blood tests, feedings, medications and monitoring day and night, 24/7. Glycine became the OSullivans obsession as they tried desperately through medication and diet to moderate Drakes levels. They began to search for information, research, treatment, medical advice anything to save his life.

The OSullivans contacted anyone who might know about NKH, have a related research project or could tell them more. They learned that NKH affects fewer than 500 people worldwide and has no cure. There was no research underway, and no funding for research. And because there is no medically recognized cure for NKH, all treatments are considered experimental and not covered by medical insurance. Period.

So Tarah became a lay scientist. She read everything, called and emailed medical researchers and established the Drake Rayden Foundation to raise awareness for NKH, fight for better treatment and support research. She entered a world of genetics and vectors, glycine and metabolic pathways. Tarah had quit college just shy of completing her business degree. Now she desperately needed the scientific expertise that would help her understand the disease and find the cure.

Tarah decided to return to college.

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The Face of Science - Clemson World magazine

Recommendation and review posted by Bethany Smith

U.S. R&D investment ‘not tracking with disease burden’: report – DOTmed HealthCare Business News

January 3rd, 2020

The good news is that total U.S. medical and health R&D grew 6.4 percent from 2017 to 2018, topping a total of $194 billion and outpacing overall healthcare spending growth for the third year in a row.

The bad news is that research investment is just 5 cents of every health dollar spent, according to a new report from Research!America.

This growth in R&D investment is positive and welcome, certainly, said Research!Americas chair, the honorable Michael N. Castle. However, our nations total investment is not tracking with disease burden. Increased investment in medical and health R&D is essential to ending diseases that are taking time and quality of life from Americans and people across the globe.

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Academic and research institutions, including colleges and universities, independent research institutes, and independent hospital medical research centers invested $15.7 billion, or 8.1 percent, while foundations put in $2.3 billion or 1.2 percent.

State and local government put in $2.1 billion, or 1.1 percent, and voluntary health and professional groups invested $1.5 billion or 0.8 percent.

Increased investment across all sectors contributing to R&D is the right path for our nation, but relative to unmet medical needs, we are walking, rather than running down that path, Mary Woolley, president and CEO, Research!America, said in a statement. Federal funding and policies need to be aligned behind the objective of empowering both public sector and private sector-driven research, because research saves lives.

Illness that more research investment could combat kills almost 130,000 under age 45 each year, and chronic disease cost the U.S. $1.1 trillion in 2018 almost six times the amount all sectors spend on R&D, according to the report.

In other December research news, Harvard, MIT, GE and Fujifilm formed a Massachusetts-based, $50 million biomed consortium that hopes to smooth the path for cutting-edge gene therapy and cancer immunotherapy from research lab to hospital clinic.

The center will include a board of directors from Harvard University, Massachusetts Institute of Technology, Fujifilm Diosynth Biotechnologies, GE Healthcare Life Sciences, Alexandria Real Estate Equities Inc., and have contributing members from Beth Israel Deaconess Medical Center, Boston Childrens Hospital, Brigham and Womens Hospital, Dana-Farber Cancer Institute and Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.

"This is a momentous opportunity, noted Martin Meeson, president and COO of Fujifilm Diosynth Biotechnologies, U.S., said at the time. "Our participation as one of the founding members is to enable these very important therapies to be accessible to patients. We seek to bring very much needed expertise and capacity to the one of the leading biotechnology ecosystem in the world."

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Repurposing FDA-approved medicines and gene therapy to combat diabetic retinopathy – Open Access Government

January 3rd, 2020

Diabetic retinopathy (DR) is a sight-threatening complication of diabetes and the main cause of blindness in the United States among working adults. Initially, DR progresses as a non-proliferative DR (NPDR) which leads to blinding proliferative DR (PDR). Nearly all people with Type 1 Diabetes (T1D) undergo gradual vision loss over a 20-year period of diabetes, and about 2030% of them progress to the advanced blinding stage of the disease, PDR.

Tight glycaemic management lowers the risk of complications, yet, many diabetic patients develop DR, despite having good glycaemic control. Furthermore, there is no cure or a preventive measure to block PDR. Only recently, over the past decade, with the advent of medicines that block the actions of the vascular endothelial growth factor (VEGF), has considerable progress been made in therapeutic options for PDR.

Nevertheless, not all patients achieve a satisfactory response and many of responders experience frequent invasive intravitreal injections and off-target effects, including increased risk of neuronal toxicity and geographic atrophy. While additional molecular targets, including the plasma kallikrein pathway, lipoprotein-associated phospholipase A2 (Lp-PLA2) and Tie-2, have been identified, with some being clinically evaluated, a critical gap still remains ineffective treatments.

Evidence suggests that targeting DR in the earlier stages, such as mild to moderate NPDR, before permanent damage occurs, would provide long-term benefit. In recent years, our research has focused on the molecular mechanisms of early DR including retinal oxidative stress, mitochondrial dysfunction, mitophagy, inflammation, and premature cell death in diabetes using both in vitro retinal cell cultures and in vivo diabetic rodent models.

Intriguingly, we have discovered a protein called thioredoxin-interacting protein (TXNIP) is strongly induced by diabetes in early DR where it is responsible for mediating cellular oxidative stress, mitophagy, inflammation and premature cell death. Knockdown of TXNIP by intravitreal injection of TXNIP shRNA prevents early molecular defects seen in DR. TXNIP binds to and inhibits the anti-oxidant and thiol-reducing capacity of thioredoxins (Trx) causing cellular redox imbalance and oxidative stress. Trx1 is present in the cytosol and nucleus while Trx2 is located in the mitochondrion. TXNIP is observed in all cellular compartments. Therefore, targeting TXNIP itself or downstream pathways could prevent or slow down the progression of early DR (NPDR) and hence PDR.

Currently, no clinically used, professional TXNIP inhibitors exist; however, several FDA-approved medicines have been reported to interfere with TXNIP pathway, but none of them has been used to treat DR. These drugs include amlexanox, tranilast, and romidepsin and they have been extensively studied for their efficacy, toxicity and safety. This consequently leads to saving time and money and may accelerate their entry to experimental clinical trials centred on targeting an over-activated TXNIP system to arrest DR away from their initial use.

Amlexanox is an inhibitor of TANK-Binding Kinase 1 (TBK1), which phosphorylates mitophagy adaptor optineurin and regulates mitophagic flux to lysosomes. In addition, TBK1 also phosphorylates interferon responsive factor 3 (IRF3) and mediates Type 1 interferon expression and inflammation. Tranilast has been shown to inhibit TXNIP and Nod-like NLRP3 inflammasome; therefore, it may help in preventing cellular oxidative stress and innate immune responses.

Therefore, a combination therapy using amlexanox and tranilast may prove to be effective in preventing or slowing down the progression of DR. Recently, we also demonstrated that a combination therapy of SS-31 (a mitochondria-targeted anti-oxidant), amlexanox and tranilast prevents auranofin-induced redox stress, mitochondrial-lysosomal axis dysregulation and proinflammatory pyroptotic cell death in retinal pigment epithelial cells, suggesting that combination therapies may be more effective than a single drug therapy.

In addition to drug treatment, gene therapy using a TXNIP promoter linked with a neuroprotective factor or an anti-oxidant gene may also be a potential approach for DR treatment. This is because the TXNIP promoter is strongly induced by hyperglycaemia in retinal cells in culture and in diabetic rodent retinas, but not under physiological glucose levels. In addition, the TXNIP promoter is also activated significantly by histone deacetylase inhibitors (HDACi), including suberoylanilide hydroxamic acid (SAHA) or romidepsin.

Therefore, HDACi and TXNIP-promoter gene therapy may also be incorporated in DR therapy for retinal neuroprotective gene expression, which could include pigment epithelium-derived factor (PEDF), glia-derived neurotrophic factor (GDNF), thioredoxin encoded Rod-Derived Cone Viability Factor (RdCVF) and others. These factors are known to be downregulated in DR.

It is currently accepted that neurodegeneration (particularly photoreceptor dysfunction) occurs early in DR before microvascular pathology develops. Therefore, early neuroprotective efforts may constitute a meaningful therapeutical approach to prevent or slow down the progression of late microvascular complications and PDR. Diabetes is a chronic and complex metabolic disease in which PDR develops only after prolonged hyperglycaemic exposure.

Therefore, a window of diabetic duration may exist early to prevent retinal neurovascular dysfunction and progression of PDR; within this time frame, we may devise preventive interventions activating endogenous genes or factors with neuroprotective drugs (preferably orally active drugs) and gene therapy including those mentioned above.

Such a hypothesis is supported by a recent observation, which showed that the expression of retinol binding protein 3 (RBP3), a protein secreted by photoreceptors in the retina, may play a protective role against PDR. RBP3 interacts with glucose transporter Glut1 and reduces excess cellular glucose uptake under hyperglycaemia in diabetics, thus, preventing glucotoxicity in retinal cells including Muller glia and capillary endothelial cells. Those individuals expressing RBP3 do not develop PDR although they have had 50 years of T1D.

In conclusion, new preventive therapies for PDR may be successfully developed by activating endogenous cellular survival mechanisms using drug and gene therapies once a clinical sign of NPDR is observed but before PDR. Such treatments may prevent blindness in diabetic patients.

References

1 Perrone L, Devi TS, Hosoya KI, Terasaki T, Singh LP. Inhibition of TXNIP expression in vivo blocks early pathologies of diabetic retinopathy. Cell Death Dis. 2010 Aug 19;1:e65. PMID: 21364670.

2 Devi TS, Somayajulu M, Kowluru RA, Singh LP. TXNIP regulates mitophagy in retinal Mller cells under high-glucose conditions: implications for diabetic retinopathy. Cell Death Dis. 2017 May 11;8(5):e2777. PMID: 28492550.

3 Lalit PS, Thangal Y, Fayi Y, Takhellambam SD. Potentials of Gene Therapy for Diabetic Retinopathy: The Use of Nucleic Acid Constructs Containing a TXNIP Promoter. Open Access J Ophthalmol. 2018;3(2). PMID: 31106306.

4 Devi TS, Yumnamcha T, Yao F, Somayajulu M, Kowluru RA, Singh LP. TXNIP mediates high glucose-induced mitophagic flux and lysosome enlargement in human retinal pigment epithelial cells. Biol Open. 2019 Apr 25;8(4). PMID: 31023645.

5 Yumnamcha T, Devi TS, Singh LP. Auranofin Mediates Mitochondrial Dysregulation and Inflammatory Cell Death in Human Retinal Pigment Epithelial Cells: Implications of Retinal Neurodegenerative Diseases. Front Neurosci. 2019 Oct 10;13:1065. PMID: 31649499.

6 Yokomizo H, Maeda Y, Park K, Clermont AC, Hernandez SL, et. al., Retinol binding protein 3 is increased in the retina of patients with diabetes resistant to diabetic retinopathy. Sci Transl Med. 2019 Jul 3;11(499). PMID: 31270273.

7 Ibrahim AS, Saleh H, El-Shafey M, Hussein KA, et. al., Targeting of 12/15-Lipoxygenase in retinal endothelial cells, but not in monocytes/macrophages, attenuates high glucose-induced retinal leukostasis. BBA: Mol Cell Biol Lipids. 2017 Jun;1862(6):636-645. PMID: 28351645.

8 Ibrahim AS, Elshafey S, Sellak H, Hussein KA, El-Sherbiny M, et. al., A lipidomic screen of hyperglycemia-treated HRECs links 12/15-Lipoxygenase to microvascular dysfunction during diabetic retinopathy via NADPH oxidase. J Lipid Res. 2015 Mar;56(3):599-611. PMID: 25598081.

Funding

NIH/NEI R01 EY023992 (LSP, OVAS).

NIH/NEI core grant P30EY004068 (LDH, OVAS).

Research to Prevent Blindness (MSJ, OVAS).

American Heart Association Grant 18CDA34080403 to ASI.

Please note: This is a commercial profile

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Repurposing FDA-approved medicines and gene therapy to combat diabetic retinopathy - Open Access Government

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Cancer Gene Therapy Market to grow massively by 2020 with profiling players Novartis AG, Gilead Sciences, UniQure NV, Spark Therapeutics LLC, Bluebird…

January 3rd, 2020

Cancer Gene Therapy Market is expected to grow at a Compound Annual Growth Rate (CAGR) of xx%. The base year considered for the study is 2018 and the forecast period considered is 2020 To 2026

Cancer Gene Therapy Market, projects a standardized and in-depth study on the ongoing state of Market, providing basic industry insights such as definitions, classifications, supply chain, applications and industry cost structure. The report precisely delivers productive information about development policies and plans as well as manufacturing processes and techniques.

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Dominant Companies Profiled in this report includes:

Novartis AG, Gilead Sciences,,UniQure N.V. ,Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline PLC, Celgene Corporation,Shire PLC, Sangamo Biosciences,Dimension Therapeutic,Voyager Therapeutics

Key questions answered in the report include:

What will the market size and the growth rate be in 2026?

What are the key factors driving the Cancer Gene Therapy Market?

What are the key market trends impacting the growth of the Cancer Gene Therapy Market?

What are the challenges to market growth?

Who are the key vendors in the Cancer Gene Therapy Market?

What are the market opportunities and threats faced by the vendors in the Cancer Gene Therapy Market?

What are the key outcomes of the five forces analysis of the Cancer Gene Therapy Market?

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For the better regional outlook, analysts examine different global regions such as North America, Latin America, Europe, Asia-Pacific, and India on the basis of different economic attributes like profit margin, shares and pricing structures. Leading key players in the global Cancer Gene Therapy market have been examined by considering different parameters such as productivity, manufacturing base, applications, and raw material. It explores different approaches to augment client base in the domestic and international market.

Major Points Covered in Table of Contents:

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Keysight, Analog Devices, Alexion, Amarin and Cellectis highlighted as Zacks Bull and Bear of the Day – Yahoo Finance

January 3rd, 2020

For Immediate Release

Chicago, IL January 2, 2019 Zacks Equity Research Keysight Technologies KEYS as the Bull of the Day, Analog Devices ADI as the Bear of the Day. In addition, Zacks Equity Research provides analysis on Alexion Pharmaceuticals, Inc. ALXN, Amarin Corporation plc. AMRN and Cellectis CLLS.

Here is a synopsis of all five stocks:

Bull of the Day:

Keysight Technologiesis a $19 billion provider of electronic design, measurement and test instrumentation systems. Keysight emerged as a public company from the 2014 move by Agilent Technologies to split apart divisions.

KEYS provides specialized electronics solutions to dozens of industries including aerospace and defense, automotive and energy, telecommunications, government and education, and, of course, semiconductors.

Keysight calls the top 25 technology enterprises in the world customers, and 78 of the Fortune 100, including Alphabet, Amazon, AT&T, Broadcom, Boeing, Cisco, Microsoft, NVIDIA, Samsung, Tesla and Toyota.

In fiscal 2019 (ended October), the company generated revenues of $4.312 billion, up 10% over the 2018 tally. In its Q4 reported on November 26, KEYS delivered sales of $1.122 billion, up 7% year over year.

Keysight generated 40.5% of non-GAAP revenues from Americas in fourth-quarter fiscal 2019. Meanwhile, revenues from Europe and Asia Pacific came in at 15.4% and 44.1%, respectively.

Evolving for the Hyper-Speed IoT/5G/AI World

As Keysight has prepared itself for advanced capabilities in wireless and mobile data environments, management sees a transformation from a hardware-centric product company to a software-centric solutions one.

In the company's most recent investor presentation in May, they outlined the evolution of their new "go-to-market" strategy thus...

Old model of scattered selling across separate channels to multiple divisions:

Individual Products with hardware bias

Slower, complex decision-making due to multiple owners interfacing with customers

Incentivized and compensated on parts of a solution

New model of total system designs:

Complete Solutions: Hardware + Software + Services

Faster customer commitments and solution development; one decision owner

Incentivized and compensated on total customer solutions by industry organization

Keysight management also announced their goal to be a top 5G solutions provider and have several first-to-market 5G design wins.

And this strategic focus on "complete solutions" also keeps them at the heart of other bleeding edge innovations in machine learning and artificial intelligence where the requirements for hardware to be embedded with specialized software stacks is only increasing.

Segment Breakdown

Keysight reports under three operating segments namely Communications Solutions Group or CSG, Electronic Industrial Solutions Group or EISG and Ixia Solutions Group (a 2017 acquisition) or ISG. In a bid to remove ambiguity in the reporting processes, Keysight removed Services Solutions Group or SSG as a discrete reportable segment, from first-quarter fiscal 2019.

Under CSG segment (62.9% of the non-GAAP revenues in fourth-quarter fiscal 2019), the company offers radio frequency (RF) and microwave test instruments and allied software, and electronic design automation (EDA) software instruments, laser source products, optical amplifiers, and other software solutions.

EIS Group (25.3%) accounts for design verification devices; general purpose test and measurement equipments; end-to-end manufacturing systems, and material analysis devices.

ISG (11.8%) was formed after conclusion of Ixia buyout in Apr 18, 2017. Under the segment, Keysight offers test and visibility solutions, and software maintenance services.

Quiet, Steady Growth that Investors Favor

Keysight's fortunes and stock price certainly benefited from the tailwinds for the semiconductor industries that were able to shake off supply chain disruptions from the tariff battles.

Even before the hype over 5G, smart technology investors were tuning out the trade war and focusing on the major trends as I described in my 2018 report and video The Technology Super Cycle.

Story continues

But you may be surprised to learn that the 70% advance of KEYS shares in 2019 -- to trade at 4.5X sales and 20X EPS -- is about to be challenged by revenue growth rates for the current fiscal year and next that are expected to slow to around 6.5%, with EPS growth down to just under 10%.

Yet this growth has been enough for Wall Street because its exceeding the plan that KEYS management laid out in 2015 where they expected 4-5% CAGR.

And management is also delivering on its promise to turn 17-18% operating margins into 20%-plus. They certainly keep giving positive surprises with the past 4 quarters averaging a +19% EPS beat.

With global leadership positions in their key markets, the company currently maintains 24% market share in a $16-17 billion addressable market.

That market will keep expanding as new technologies in 5G, datacenters, aerospace and autos evolve. Keysight's strategic "complete solutions" approach to serving the world's biggest tech, hardware, communications and transportation companies will keep them growing at a steady 4-5% right along with those markets -- especially as their customers seek more specialized and custom electronics solutions that KEYS has the platform and vision for.

Bear of the Day:

Analog Devicesis the $44 billion manufacturer of analog, mixed signal and digital signal processing (DSP) integrated circuits, and other semiconductor devices found in cars, planes, factories and home appliances around the globe.

ADI moved to the cellar of the Zacks Rank after reporting a disappointing Q4 fiscal 2019 (ended October) and outlook that caused analysts to take down estimates for their FY20. Adjusted earnings of $1.19 per share missed the Zacks Consensus Estimate of $1.21, as the bottom line decreased 19.6% year over year and 12.5% sequentially.

Revenues of $1.44 billion in the quarter also missed the Zacks Consensus Estimate by 0.6%. And the top line declined 6% year over year and 5.5% from the fiscal third quarter.

This downside can be attributed to weak performance of the company in all end-markets served. Moreover, macroeconomic headwinds negatively impacted the topline.

Analysts responded to the company outlook by dropping full-year 2020 revenue projections to $5.66 billion, representing a 5.46% decline from FY19.

And the 2020 EPS consensus fell 10.3% to $4.78 from $5.33, for a projected 7.2% drop on the bottom line.

Revenues by End Markets

Industrial generated revenue of $744.1 million (accounting for 52% of total revenues), which was flat year over year.

Communications revenue came in at $260.1 million (18% of revenues), decreasing 19% year over year.

Automotive revenue fell to $226.1 million (16% of revenues), down 8% from the year-ago quarter.

Consumer generated revenue of $212.8 million (15% of revenues), reflecting a 7% decline on a year-over-year basis.

Guidance Lowered

For the first quarter of fiscal 2020, Analog Devices expects revenues to be $1.30 billion (+/- $50 million), representing a 15.6% decline from the year ago quarter. Prior to this disappointing guidance, the Zacks Consensus Estimate for Q1 was pegged at $1.41 billion.

Non-GAAP earnings are expected to be $1 (+/- $0.07) per share, reflecting a 24% y-o-y drop. The consensus mark for the same was $1.16 per share.

The company anticipates non-GAAP operating margins to be approximately 36.7% (+/- 100 bps).

Clearly ADI is not going out of business despite being a leader of analog solutions in a digital world.

But until the estimates stop going down and start heading back up, it may be best to stand aside. The Zacks Rank will let you know.

3 Biotechs Likely to Maintain Solid Momentum in 2020

It has been a roller-coaster ride for the volatile biotech sector in 2019 after a disappointing run in 2018. The year started with a bang for this sector, with the announcement of the mega-merger of majors Bristol-Myers and Celgene. This significantly perked up the prices of quite a few stocks. However, these were partially offset by the overall weakness in the global market. Nevertheless, the sector has again picked up in the past couple of months, primarily owing to the recent spree of mergers and acquisitions, and positive pipeline readouts.

Overall, the Nasdaq Biotechnology Index has seen 23.2% growth in the past year.

Relatively, the biotech sector continued being riskier than the more stable large cap pharmaceuticals industry or the overall medical sector, as investors are mostly banking on companies with very few approved drugs in the portfolios.

A slowdown in mature products due to increasing competition and the rise of biosimilars forced most pharma/biotech behemoths to target lucrative buyouts in the biotech space to bolster their portfolios. In particular, biotechs (both small and big), which have a dominant position in the lesser competitive arena of rare diseases, gene therapy and NASH, and are well-equipped with path-breaking technologies, are significant acquisition targets.

A slew of licensing and buyout deals was struck by most companies eyeing smaller entities with impressive pipelines. Novartis recently announced that it will acquire The Medicines Company and add a potentially transformational investigational cholesterol-lowering therapy to its portfolio.

Roche is finally set to acquire Spark Therapeutics after a prolonged delay, while Japanese company Astellas Pharma is taking over gene therapy company Audentes Therapeutics, Inc and has already acquired privately-held, development-stage biotechnology company Xyphos Biosciences, Inc to boost its immuno-oncology pipeline.

Meanwhile, new drug approvals and label expansions of blockbuster drugs boosted investor sentiment. Key approvals include Vyondys 53, Oxbryta, Givlaari, Reblozyl, Trikafta, Inrebic, Vyleesi and Evenity, among others.

Choosing a biotech stock for investment can be tricky as smaller biotechs carry a risk with their product pipelines being several years away from commercialization. Nevertheless, here we zero in a few biotech companies, which have a market capitalization of more than $500 million.

With a favorable Zacks Rank #2 (Buy), these companies are likely to perform well in 2020. You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

Moreover, the Zacks Biomedical and Genetics industry is placed within the top 22% of the 252 Zacks-ranked industries.

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company, focused on developing and commercializing life-transforming drugs for the treatment of patients with ultra-rare disorders. Its blockbuster drug Soliris approved for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) continues to perform well.

The drugs label expansion for the generalized myasthenia gravis indication has boosted sales further. The company received a significant boost with the FDA approval of its long-acting C5 complement inhibitor Ultomiris for the treatment of adult patients with PNH.

The approval has strengthened Alexion's PNH franchise and reduced its dependence on Soliris for growth. The company is working on the label expansion of Ultomiris and taking steps to further diversify its pipeline, which should reap returns in the long run.

Shares of Alexion have gained 10.2% in the past year.

Amarin Corporation plc. focuses on developing and commercializing therapeutics to cost-effectively improve cardiovascular health. The company recently obtained FDA approval for the label expansion of its key drug Vascepa. The drug is now approved as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease.

The label expansion should significantly boost Vascepa sales as, per estimates, millions of high-risk patients in the United States could benefit from this one-of-a-kind prescription therapy. We expect the initial uptake of the drug to be strong and boost the top line, given the market potential.

Amarins shares have surged 54.5% in the past year.

Cellectisis a clinical-stage biopharmaceutical company, focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART). The company is developing life-changing product candidates utilizing TALEN, its proprietary gene-editing technology, and PulseAgile, its pioneering electroporation system, to harness the power of the immune system to target and eradicate cancer cells. The company is striving hard to develop life-saving UCART product candidates to address unmet needs for multiple cancers, including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL), multiple myeloma (MM), Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL).

Gene therapy is set to become one of the most vital spaces with high prospects within the volatile biotech sector.

Cellectis shares have gained 3.7% in a year.

Zacks Top 10 Stocks for 2020

In addition to the stocks discussed above, would you like to know about our 10 top tickers for the entirety of 2020?

These 10 are painstakingly hand-picked from over 4,000 companies covered by the Zacks Rank. They are our primary picks to buy and hold. Start Your Access to the New Zacks Top 10 Stocks >>

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportKeysight Technologies Inc. (KEYS) : Free Stock Analysis ReportAlexion Pharmaceuticals, Inc. (ALXN) : Free Stock Analysis ReportAmarin Corporation PLC (AMRN) : Free Stock Analysis ReportCellectis S.A. (CLLS) : Free Stock Analysis ReportAnalog Devices, Inc. (ADI) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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Keysight, Analog Devices, Alexion, Amarin and Cellectis highlighted as Zacks Bull and Bear of the Day - Yahoo Finance

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Global Gene Therapy Market- Technology, New Innovations, Opportunities, Future Guidelines, Key Players, Trends and Forecast 2019-2024 -…

January 3rd, 2020

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Novel discovery in gene therapy to treat kidney diseases – WeForNews

January 3rd, 2020

San Francisco:In a ray of hope for those who have to go for breast cancer screening and even for healthy women who get false alarms during digital mammography, an Artificial Intelligence (AI)-based Google model has left radiologists behind in spotting breast cancer by just scanning the X-ray results.

Reading mammograms is a difficult task, even for experts, and can often result in both false positives and false negatives.

In turn, these inaccuracies can lead to delays in detection and treatment, unnecessary stress for patients and a higher workload for radiologists who are already in short supply, Google said in a blog post on Wednesday.

Googles AI model spotted breast cancer in de-identified screening mammograms (where identifiable information has been removed) with greater accuracy, fewer false positives and fewer false negatives than experts.

This sets the stage for future applications where the model could potentially support radiologists performing breast cancer screenings, said Shravya Shetty, Technical Lead, Google Health.

Digital mammography or X-ray imaging of the breast, is the most common method to screen for breast cancer, with over 42 million exams performed each year in the US and the UK combined.

But despite the wide usage of digital mammography, spotting and diagnosing breast cancer early remains a challenge, said Daniel Tse, Product Manager, Google Health.

Together with colleagues at DeepMind, Cancer Research UK Imperial Centre, Northwestern University and Royal Surrey County Hospital, Google set out to see if AI could support radiologists to spot the signs of breast cancer more accurately.

The findings, published in the journal Nature, showed that AI could improve the detection of breast cancer.

Google AI model was trained and tuned on a representative data set comprised of de-identified mammograms from more than 76,000 women in the UK and more than 15,000 women in the US, to see if it could learn to spot signs of breast cancer in the scans.

The model was then evaluated on a separate de-identified data set of more than 25,000 women in the UK and over 3,000 women in the US.

In this evaluation, our system produced a 5.7 per cent reduction of false positives in the US, and a 1.2 per cent reduction in the UK. It produced a 9.4 per cent reduction in false negatives in the US, and a 2.7 per cent reduction in the UK, informed Google.

The researchers then trained the AI model only on the data from the women in the UK and then evaluated it on the data set from women in the US.

In this separate experiment, there was a 3.5 per cent reduction in false positives and an 8.1 per cent reduction in false negatives, showing the models potential to generalize to new clinical settings while still performing at a higher level than experts.

Notably, when making its decisions, the model received less information than human experts did.

The human experts (in line with routine practice) had access to patient histories and prior mammograms, while the model only processed the most recent anonymized mammogram with no extra information.

Despite working from these X-ray images alone, the model surpassed individual experts in accurately identifying breast cancer.

This work, said Google, is the latest strand of its research looking into detection and diagnosis of breast cancer, not just within the scope of radiology, but also pathology.

Were looking forward to working with our partners in the coming years to translate our machine learning research into tools that benefit clinicians and patients, said the tech giant.

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Novel discovery in gene therapy to treat kidney diseases - WeForNews

Recommendation and review posted by Bethany Smith

BrainStorm Cell Therapeutics Wins 2020 ‘Buzz of BIO’ Award for ALS Investigational Therapy – ALS News Today

January 3rd, 2020

For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories ofBuzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration (FDA).

As a developer of autologous cellular therapies treatments that use a patients own cells and tissues for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. The treatment involves extracting, from human bone, marrow-derived mesenchymal stem cells (MSCs), which are capable of differentiating into other cell types. The MSCs are then matured into a specific cell type that produces neurotrophic factors compounds that promote nervous tissue growth and survival. They are then reintroduced to the body via injection into muscles and/or the spinal canal.

Backed by a California Institute for Regenerative Medicine grant, Brainstorm has fully enrolledits randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03280056) at six U.S. sites in California, Massachusetts, and Minnesota. Some 200 ALS patients are participating. A secondary safety analysis by the trials independent Data Safety Monitoring Board (DSMB) revealed no new concerns. Every two months, study subjects will be given three injections into the spinal canal of either NurOwn or placebo.

The trial is expected to conclude late this year. Results will be announced shortly afterward.

In a Phase 2 study (NCT02017912), which included individuals with rapidly progressing ALS, NurOwn demonstrated a positive safety profile as well as prospective efficacy.

The use of autologous MSC cells to potentially treat ALS was given orphan drug status by both the FDA and the European Medicines Agency.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release. The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

As a contest winner, BrainStorm is invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City, along with exposure to multiple industry elites and potential investors.

NurOwn cells also are being tested in a Phase 2 clinical study (NCT03799718) in patients with progressive multiple sclerosis.

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

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award for ALS Investigational Therapy - ALS News Today

Recommendation and review posted by Bethany Smith

Innovative therapies: Novel targets in allergic inflammation – SelectScience

January 3rd, 2020

Meet the inflammation and immunity researcher studying the fundamental cellular mechanisms behind uncontrolled inflammatory responses to allergens

As the prevalence of allergic disease continues to rise worldwide, the work of immunologist Dr. Adam MacNeil has never been more important. By identifying novel targets in allergic inflammation to enable the development of innovative therapies, MacNeil and his team are pushing toward a healthier future. Were interested in allergic inflammation from two different branches, firstly, how the cells that contribute to inflammation emerge from the bone marrow, and secondly, how mature mast cells contribute to inflammatory mechanisms at the site of exposure, explains MacNeil, associate professor in the interdisciplinary Health Sciences department at Brock University, Canada.

Dr. Adam J. MacNeil, Associate Professor of Immunologyat Brock University's Department of Health Sciences.Pictured from left to rightare;Melissa Rouillard, Aindriu Maguire, Rob Crozier, Adam MacNeil, Jeremia Coish, Katie Hunter, Colton Watson, and Natalie Hicks. Image courtesy of theMacNeil Lab.

The MacNeil Lab investigates mechanisms in hematopoietic stem cells directing the maturation of the most well-known allergic mediator cellsmature mast cellsthat drive allergic inflammation. A key research goal for the team is to identify how an allergen activates a mast cell to create an inflammatory response.

Seeking to understand the signals that stimulate a progenitor cell to become a mast cell in different tissues, this research looks to determine the signaling pathways directing the epigenetic, and ultimately proteomic, profile of these cells1-3. To do this, cells are isolated and matured from bone marrow to create functional, phenotypical mast cells, which are primed with allergen-specific IgE molecules before addition of the allergen to activate the cells. The inflammatory response to the allergen, and the cell signaling processes that contribute to the inflammatory mechanisms, can then be measured through the secretion of histamines in degranulation mechanisms, or release of pro-inflammatory mediators such as cytokines, chemokines, and lipid metabolites.

Brock University

Being able to identify and sort cells with a specific immune profile requires tools capable of precision sorting of heterogeneous populations of cells. MacNeil expands: Were working with a heterogeneous population of cells in the bone marrow and trying to take only the stem cells out. So, it's a very small population within the total population of cells. Many of the assays that we want to do with that small population of cells are very well-suited to being sorted directly onto a 96-well plate where we can then actually conduct the experiment directly, knowing exactly how many cells are in each well and what the particular profile of those cells is. That makes the Sony SH800S a really strong tool for our lab.

When it comes to optimizing and streamlining the lab's work, Sony technology offers advantages over traditional methods. The traditional flow cytometer or cell sorter in any core lab is operated by a technician, and they're the only one allowed to touch it. That doesn't make for great learning opportunities for graduate students, and it's much better if they can actually interface with the instrument themselves, says MacNeil. The software and automation really allow for that to happen, but also adds to the robustness of the instrument. The way in which it has been designed means that it's pretty difficult to break it.

With an epigenetic approach to understanding how mast cells differentiate, and the effect of inhibiting specific signaling pathways in those cells, the MacNeil Lab uses sorted cells in functional assays such as immune cell profiling and cytokine secretion. Also, the cells can be sorted into plate-based assays for ChIP or RNA-Seq to assess their genetic profile. We're not only interested in sorting. We bought the device because it's robustly dynamic, explains MacNeil, referring to the Sony SH800S. You can look at data acquisition and not have to even use the sorting function at all in certain scenarios. There are many times that were simply interested in looking at the phenotype of our cells and not worried about sorting necessarily. Weve found this instrument to be very easy to use and to give us robust data in terms of the immune profile of our cells.

In addition, the SH800S microfluidic sorting chip helps to automate key stages of instrument setup and demonstrates versatility with a wide range of chip sizes, ranging from 70130 m, for sorting a variety of cells. The chip ultimately gets to the robustness of the instrument, explains MacNeil. Because of the chip, we have such peace of mind about how the instrument functions that we don't even worry about clogging of the instrument and all of the problems that the chip ultimately solves. If we do run into a problem, we can just change the chip. I certainly find the chip technology to be really well suited to our type of lab environment.

For MacNeil, the Sony SH800S Cell Sorter is a great fit for the lab, with a seamless software interface and great overall instrument design and modularity for easy plate-based sorting.MacNeil lab logocourtesy of the MacNeil Lab.

Working within the diverse multidisciplinary department at Brock University opens unique and fascinating research avenues not available to all immunologists and has led MacNeil to interesting collaborations and knowledge exchange on transdisciplinary projects.

As part of these broader research avenues, working with sociologist Prof. Terrance Wade and cardiovascular biologist Prof. Deborah OLeary, MacNeil also studies adverse experiences in childhood. The team is investigating whether such events may set the immunological stage for dysregulated inflammation in later life, through mechanisms involving stress-stimulated cortisol release that can shape how the immune system is responding4.

In another stream of collaborative immunological research, MacNeil collaborates with psychologist Prof. Anthony Bogaert to look at the role of the immune system in shaping sexual orientation as part of the fraternal birth order effect. This research looks at how early pregnancies stimulate the immune system to make antibodies against brain proteins in fetal males that may then affect their social behaviors in later life5. Its something I may not have expected to ever work on, says MacNeil. But when you come to a diverse department with a wide lens on health, these kinds of opportunities emerge. Were now interested in using the SH800S to test hypotheses for particular mechanisms underlying this phenomenon.

Looking ahead, MacNeil expects tissue heterogeneity to be a key issue to tackle in the field of immunology. Cell populations simply aren't uniform, he says. Mast cells in different locations in the body don't have exactly the same phenotype, and so, as our research proceeds and we continue to probe the role of the mast cell in allergic inflammation, we're very conscious that tissue heterogeneity is going to be a factor. But with such challenges come opportunities. Were ultimately interested in going into those tissues and trying to pull mast cells out. To do this, we would require an instrument like a cell sorter. Once the cells are sorted, we can interrogate their functional phenotype, including how they degranulate, secrete cytokines and metabolize lipids etc. toward one day potentially modulating their phenotype for the hundreds of millions affected by this inappropriate immune response, MacNeil concludes.

Originally posted here:
Innovative therapies: Novel targets in allergic inflammation - SelectScience

Recommendation and review posted by Bethany Smith

Will Prime Editors be the New CRISPR? – Discover Magazine

January 3rd, 2020

CRISPR may have generated a lot of buzz this year, but some researchers are already looking beyond it to the next new gene-editing technique. Say hello to prime editing.

If CRISPR is like scissors then you can think of prime editors like word processors, said chemist David Liu in an October press briefing. He spoke days ahead of the first-ever prime editing study, published in Nature and authored by Liu and his team at the Broad Institute of MIT and Harvard University. Liu explained that, while CRISPR cuts through DNAs double helix to snip out genes, prime editing searches for and replaces targeted genes without such slicing and dicing, reducing the risk of unintended changes to the genetic code.

The team was able to correct mutations associated with both sickle cell and Tay-Sachs diseases. Liu believes the technique ultimately might be able to correct almost 90 percent of such mutations, but stressed additional studies are needed to gauge prime editings full potential.

This is the beginning, rather than the end, said Liu.

[This story appeared in print as "It's Prime Time for a New Gene Editor."]

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Will Prime Editors be the New CRISPR? - Discover Magazine

Recommendation and review posted by Bethany Smith

These 6 Incredible Discoveries From The Past Decade Have Changed Science Forever – ScienceAlert

January 3rd, 2020

From finding the building blocks for life on Mars to breakthroughs in gene editing and the rise of artificial intelligence, here are six major scientific discoveries that shaped the 2010s - and what leading experts say could come next

We don't yet know whether there was ever life on Mars - but thanks to a small, six-wheeled robot, we do know the Red Planet was habitable.

Shortly after landing on 6 August 2012, NASA's Curiosity rover discovered rounded pebbles - new evidence that rivers flowed there billions of years ago.

The proof has since multiplied, showing there was in fact a lot of water on Mars - the surface was covered in hot springs, lakes, and maybe even oceans.

A crater on the Red Planet filled with water ice. (ESA/DLR/FU Berlin, CC BY-SA 3.0 IGO)

Curiosity also discovered what NASA calls the building blocks of life, complex organic molecules, in 2014.

And so the hunt continues for signs that Earth-based life is not (or wasn't always) alone.

Two new rovers will be launched next year - America's Mars 2020 and Europe's Rosalind Franklin rovers, looking for ancient microbes.

"Going into the coming decade, Mars research will shift from the question 'Was Mars habitable?' to 'Did (or does) Mars support life?'" said Emily Lakdawalla, a geologist at The Planetary Society.

We had long thought of the little corner of the Universe that we call home as unique, but observations made thanks to the Kepler space telescope blew apart those pretensions.

Launched in 2009, the Kepler mission helped identify more than 2,600 planets outside of our Solar System, also known as exoplanets - and astronomers believe each star has a planet, meaning there are billions out there.

Kepler's successor TESS was launched by NASA in 2018, as we scope out the potential for extraterrestrial life.

Expect more detailed analysis of the chemical composition of these planets' atmospheres in the 2020s, said Tim Swindle, an astrophysicist at the University of Arizona.

We also got our first glimpse of a black hole this year thanks to the groundbreaking work of the Event Horizon Telescope collaboration.

(Event Horizon Telescope Collaboration)

"What I predict is that by the end of the next decade, we will be making high quality real-time movies of black holes that reveal not just how they look, but how they act on the cosmic stage," Shep Doeleman, the project's director, told AFP.

But one event from the decade undoubtedly stood above the rest: the detection for the first time on September 14, 2015 of gravitational waves, ripples in the fabric of the universe.

The collision of two black holes 1.3 billion years earlier was so powerful it spread waves throughout the cosmos that bend space and travel at the speed of light. That morning, they finally reached Earth.

The phenomenon had been predicted by Albert Einstein in his theory of relativity, and here was proof he was right all along.

Three Americans won the Nobel prize in physics in 2017 for their work on the project, and there have been many more gravitational waves detected since.

Cosmologists meanwhile continue to debate the origin and composition of the universe. The invisible dark matter that makes up its vast majority remains one of the greatest puzzles to solve.

"We're dying to know what it might be," said cosmologist James Peebles, who won this year's Nobel prize in physics.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) - a family of DNA sequences - is a phrase that doesn't exactly roll off the tongue.

(Meletios Verras/iStock)

But the field of biomedicine can now be divided into two eras, one defined during the past decade: before and after CRISPR-Cas9 (or CRISPR for short), the basis for a gene editing technology.

"CRISPR-based gene editing stands above all the others," William Kaelin, a 2019 Nobel prize winner for medicine, told AFP.

In 2012, Emmanuelle Charpentier and Jennifer Doudna reported that they had developed the new tool that exploits the immune defense system of bacteria to edit the genes of other organisms.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

Charpentier and Doudna were showered in awards. but the technique is also far from perfect and can create unintended mutations.

Experts believe this may have happened to Chinese twins born in 2018 as a result of edits performed by a researcher who was widely criticized for ignoring scientific and ethical norms.

Still, CRISPR remains one of the biggest science stories of recent years, with Kaelin predicting an "explosion" in its use to combat human disease.

For decades, doctors had three main weapons to fight cancer: surgery, chemotherapy drugs, and radiation.

The 2010s saw the rise of a fourth, one that was long doubted: immunotherapy, or leveraging the body's own immune system to target tumor cells.

(Design Cells/iStock)

One of the most advanced techniques is known as CAR T-cell therapy, in which a patient's T-cells - part of their immune system - are collected from their blood, modified and reinfused into the body.

A wave of drugs have hit the market since the mid-2010s for more and more types of cancer including melanomas, lymphomas, leukemias and lung cancers - heralding what some oncologists hope could be a golden era.

For William Cance, scientific director of the American Cancer Society, the next decade could bring new immunotherapies that are "better and cheaper" than what we have now.

The decade began with a major new addition to the human family tree: Denisovans, named after the Denisova Cave in the Altai Mountains of Siberia.

Scientists sequenced the DNA of a female juvenile's finger bone in 2010, finding it was distinct both from genetically modern humans and Neanderthals, our most famous ancient cousins who lived alongside us until around 40,000 years ago.

The mysterious hominin species is thought to have ranged from Siberia to Indonesia, but the only remains have been found in the Altai region and Tibet.

We also learned that, unlike previously assumed, Homo sapiens bred extensively with Neanderthals - and our relatives were not the brutish simpletons previously assumed but were responsible for artworks, such as the handprints in a Spanish cave they were credited for crafting in 2018.

They also wore jewelry, and buried their dead with flowers - just like we do.

Next came Homo naledi, remains of which were discovered in South Africa in 2015, while this year, paleontologists classified yet another species found in the Philippines: a small-sized hominin called Homo luzonensis.

Advances in DNA testing have led to a revolution in our ability to sequence genetic material tens of thousands of years old, helping unravel ancient migrations, like that of the Bronze Age herders who left the steppes 5,000 years ago, spreading Indo-European languages to Europe and Asia.

"This discovery has led to a revolution in our ability to study human evolution and how we came to be in a way never possible before," said Vagheesh Narasimhan, a geneticist at Harvard Medical School.

One exciting new avenue for the next decade is paleoproteomics, which allows scientists to analyze bones millions of years old.

"Using this technique, it will be possible to sort out many fossils whose evolutionary position is unclear," said Aida Gomez-Robles, an anthropologist at University College London.

"Neo" skull of Homo naledi from the Lesedi Chamber. (John Hawks/University of the Witwatersrand)

Machine learning - what we most commonly mean when talking about "artificial intelligence" - came into its own in the 2010s.

Using statistics to identify patterns in vast datasets, machine learning today powers everything from voice assistants to recommendations on Netflix and Facebook.

So-called "deep learning" takes this process even further and begins to mimic some of the complexity of a human brain.

It is the technology behind some of the most eye-catching breakthroughs of the decade: from Google's AlphaGo, which beat the world champion of the fiendishly difficult game Go in 2017, to the advent of real-time voice translations and advanced facial recognition on Facebook.

In 2016, for example, Google Translate - launched a decade earlier - transformed from a service that provided results that were stilted at best, nonsensical at worst, to one that offered translations that were far more natural and accurate.

At times, the results even seemed polished.

"Certainly the biggest breakthrough in the 2010s was deep learning - the discovery that artificial neural networks could be scaled up to many real-world tasks," said Henry Kautz, a computer science professor at the University of Rochester.

"In applied research, I think AI has the potential to power new methods for scientific discovery," from enhancing the strength of materials to discovering new drugs and even making breakthroughs in physics, Kautz said.

For Max Jaderberg, a research scientist at DeepMind, owned by Google's parent company Alphabet, the next big leap will come via "algorithms that can learn to discover information, and rapidly adapt and internalize and act on this new knowledge," as opposed to depending on humans to feed them the correct data.

That could eventually pave the way to "artificial general intelligence", or a machine capable of performing any tasks humans can, rather than excelling at a single function.

Agence France-Presse

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These 6 Incredible Discoveries From The Past Decade Have Changed Science Forever - ScienceAlert

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What to Analyze About the CRISPR Therapeutics AG (CRSP)? – News Welcome

January 3rd, 2020

First we will be looking for the boiling points and excitability of CRISPR Therapeutics AG (CRSP) stock, it purposes common trait for traders and value investors.

Volatility Indicators for CRISPR Therapeutics AG:

Volatility of the CRISPR Therapeutics AG remained at 5.00% over last week and shows 4.74% volatility in last month. In addition to number of shares traded in last few trading sessions volatility also tells about the fluctuation level of the stock price, commonly a high volatility is the friend of day traders. Volatility is also measured by ATR an exponential moving average (14-days) of the True Ranges. Currently, the ATR value of companys stock is situated at 3.33.

CRISPR Therapeutics AG (CRSP) traded 1718001 shares in most recent trading session as compared to an average volume of 1.06M shares. It shows that the shares were traded in the recent trading session and traders shown interest in CRSP stock. Listed Shares of the CRISPR Therapeutics AG (CRSP) moved down -2.60% to trade at $59.32 on Thursday (01/02/2020) trading session. It has a market capitalization of $3.41B. Knowing about the market capitalization of a company helps investor to determine the company size, market value and the risk. The stock EPS is $-0.46 against its recent stock value of $59.32 per share.

Now entering into the performance part of the article on CRISPR Therapeutics AG stock we should check the stocks actual performance in the past.

Performance of the CRSP Stock:

CRISPR Therapeutics AG revealed performance of -11.77% during the period of last 5 trading days and shown last 12 months performance of 98.59%. The stock moved to 23.84% in last six months and it maintained for the month at -13.55%. The stock noted year to date 2019 performance at -2.60% and changed about 51.75% over the last three months. The stock is now standing at -19.84% from 52 week-high and is situated at 118.46% above from 52-week low price.

Technical Indicators of CRISPR Therapeutics AG Stock:

RSI momentum oscillator is the most common technical indicator of a stock to determine about the momentum of the shares price and whether the stock trading at normal range or its becoming oversold or overbought. It also helps to measure Speed and change of stock price movement. RSI reading varies between 0 and 100. Commonly when RSI goes below 30 then stock is oversold and stock is overbought when it goes above 70. So as currently the Relative Strength Index (RSI-14) reading of CRISPR Therapeutics AG stock is 39.32.

Although it is important to look for trades in a direction of bigger trends when stocks are indicating an opposite short-term movement. Like looking for overbought conditions when bigger trend remained down and oversold conditions when bigger trend is up. In order to check a bigger trend for CRSP a 14-day RSI can fell short and considered as a short-term indicator. So in that situation a Simple moving average of a stock can also be an important element to look in addition to RSI.

The share price of CRSP is currently down -11.59% from its 20 days moving average and trading 0.16% above the 50 days moving average. The stock price has been seen performing along overhead drift from its 200 days moving average with 26.32%. Moving averages are an important analytical tool used to identify current price trends and the potential for a change in an established trend. The simplest form of using a simple moving average in analysis is using it to quickly identify if a security is in an uptrend or downtrend.

Profitability Spotlight for CRISPR Therapeutics AG:

Operating Margin which tells about what proportion of a companys revenue is left over after paying for variable costs of production such as wages & raw materials is noted at -4.50%. Net profit margin of the company is -5.30% that shows how much the company is actually earning by every dollar of sales.

Return on Investment (ROI) of stock is -40.70%. ROI ratio tells about the efficiency of a number of investments in a company. Return on Assets (ROA) which shows how much the company is profitable as compared to its total assets is observed at -2.00%. Return on Equity (ROE), which tells about the profitability of the corporation by evaluating the profit it generates in ratio to the money shareholders have invested, is noted at -2.60%.

Analysts Estimation on Stock:

The current analyst consensus rating stood at 2.1 on shares (where according to data provided by FINVIZ, 1.0 Strong Buy, 2.0 Buy, 3.0 Hold, 4.0 Sell, 5.0 Strong Sell). Analysts opinion is also an important factor to conclude a stocks trend. Many individual analysts and firms give their ratings on a stock. While Looking ahead of 52-week period, the mean Target Price set by analysts is $77.5.

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Tenn. bill would require trans athletes to play on teams based on birth gender – KPAX-TV

January 3rd, 2020

NASHVILLE, Tenn A state lawmaker wants to require transgender students to play on school sports teams based on their sex at birth.

State Representative Bruce Griffey (R-Paris) introduced House Bill 1572, which would require Tennessee transgender students to participate in the sports categories based on the sex on their birth certificate.

Griffey says he introduced the bill to ensure that there's fairness in sports competitions throughout the state.

"There's no ill will intended toward anyone regarding this legislation," Griffey said. "We all know that traditionally males generally have bigger hearts, bigger upper body strength, and that can give them a genetic advantage when competing against women in a number of sports."

However, advocates for LGBT rights say this bill is an attack on the transgender community.

"Some members of the General Assembly have not made an effort to understand that trans youth are a part of our school population and we need to serve and protect them like all students," said Chris Sanders, executive director with the Tennessee Equality Project.

Sanders says the bill part of a "2020 slate of hate bills," which he claims are an attack on the LGBT community. Sanders called the purpose of proposal "ignorance, hate and discrimination."

"It is insulting to trans youth. It is an attack on them. Their state government should be serving them and not seeking ways to marginalize them further," Sanders said.

Griffey says there's no intention to punish or discriminate when it comes down to the bones of the bill. He said it's about fair competitions.

"We've split up sports into male and female competitions to begin with for a sense of fairness; and if we're going to begin blurring the lines we're really defeating the purpose of having fair competitions to begin with," Griffey said.

Under the bill, if any elementary or secondary school willfully or intentionally violates the guidelines, the schools would be "immediately ineligible to continue to receive public funds of any type from this state or a local government."

If the bill becomes law, it would impose a fine of up to $10,000 on any school or state official who knowingly violates the ban, and the official accused of violating it would have to leave their office.

They would also be ineligible to hold public office, school administration positions, or principal positions for five years after.

This story was originally published by Kelsey Gibbs on WTVF in Nashville.

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Tenn. bill would require trans athletes to play on teams based on birth gender - KPAX-TV

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There’s no such thing as a male or female brain – and why that matters – Management Today

January 3rd, 2020

Men are more confident, naturally dominant and prefer things. Women are kinder, more hesitant and prefer people. These differences, as received wisdom would have it, are biological. The female and male brain are different.

Except theyre not, says Gina Rippon, professor of cognitive neuroimaging at the Aston University Brain Centre and author of The Gendered Brain. Over the last 200 years weve just been encouraged to think they are.

The lifelong "plasticity" of the human brain means that it can change and adapt as a result of experience, she says. And that means whats going on outside the head is as important as whats going on inside.

Rippon spoke to Management Today about the consequences of the male and female brain myth, why gender doesnt determine skill and the usefulness of IQ tests for recruitment.

"Mens brains are on average bigger than womens, but thats because on average men are bigger than women. Brain size is pretty meaningless in terms of functional significance."

"It links to the way in which our brain determines our behaviour. The brain responds very well to how the world expects people to behave.

"Theres a great phrase by Reshma Saujani, who founded an organisation called Girls Who Code: 'We raise our boys to be brave and our girls to be perfect.' Thats a fantastic summary of the different pressures on boys and girls from an early age."

"Im horrified by how much research is being misrepresented and misused. Scientists aresometimes less than responsible in how they explain what they find and the implications of those findings.

"The arguments they make are plausible because they "confirm" what peoplealready think beliefs that continue to shape the environment within which people grow up, are educated and employed.

"Just recently I was reading about a firm whose business manual explained how women should act in certain situations, how they should dress and what tone of voice they should use. I think its fed by a sort of accessible, but ill-informed self-help manual.

"Language is really important in terms of what people hear. Next time youre confronted with a claim such as at last, the truth that mens and womens brains are different, just look at what kind of arguments are being made: are we harking back to evolutionary biology as the reason?"

"One of the downsides of gender initiatives, even if theyre trying to solve a pay gap, is that you sustain the belief that there is a difference between men and women. If you measure their behavioural and neurological information, youll get classic bell-shaped curves and a huge amount of variability within each group. But if you put that data on the same axes, they overlap enormously.

"There are issues associated with saying that women have a particular set of skills that are missing from the boardroom. Just appointing people because theyre men or because theyre women is not actually going to change that. But clearly people with exactly the same potential are not achieving that potential in equal numbers.

"Thats why you should be looking at the longer-term issues that affect how people arrived at a particular choice point."

"Businesses are quite fond of their questionnaires and personality profiles. But one of the issues we need to look at is how good are the measurement tools that were using to gather the data? Theres a lot of promotion of quick and dirty tests without anybody asking whether they are actually useful.

"IQ tests are a classic example. They dont really measure anything useful with respect to individual skills. Research came out recently that suggests a link between particular genetic profiles and IQ, but the idea that there is a single common factor that will discriminate one individual from another is flawed. It ignores variability within groups."

"One of the more optimistic views that I hope comes out of this is that were not necessarily driven through life by the brains were born with, or the brains were stuck with because of how weve been treated at school.

"We know now that there are dramatic changes we can bring about in our brain and the skills we can learn. If there are particular skills your business needs, for instance, you dont necessarily need to look outside the organisation, there are things that your existing workforce can learn."

Image credits:John Greim/Contributor via Getty Images

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There's no such thing as a male or female brain - and why that matters - Management Today

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Anne Dagg, pioneering giraffe biologist and feminist critic of "evolutionary psychology" receives the Order of Canada – Boing Boing

January 3rd, 2020

Anne Innis Dagg was the first female biologist to study giraffes; while all the men who preceded her had observed firsthand that male giraffes are super queer (their primary form of play is a game dubbed "penis fencing," which is exactly what it sounds like), only Dagg was willing to write it down and publish it.

Dagg's work on giraffes -- several of the seminal books on the animals -- was initially mocked or ignored, partly because of her pioneering approach of living among the animals (as opposed to observing them at a distance) offended the establishment; partly because of her gender.

Though Dagg earned a PhD and taught for decades, she was denied tenure. She continued to produce challenging, brave, brilliant work at the intersection of biology and gender politics, ranging over both scholarly and popular works. In particular, she specialized in pointing out the lack of rigor in her male colleagues' work when discussing sex and gender among animals, and how that spilled over into the way the field was organized, and gender bias within research institutions and in research publishing.

Her 2004 book, Love of Shopping is Not a Gene, is an absolute must-read book on the subject, addressing the total absence of rigor and falsifiability in hypotheses from male biologists to explain human gender and power roles with reference to animal behavior and/or the imaginary lives of early hominids -- howlers like "Rape is genetic" or "Black people are genetically destined to have lower IQ scores than white people."

These comforting fairy tales (I always think of them as being reducible to, "But honey, it's not my fault I'm fucking my undergrads, it's because of the chimps!") are especially in vogue today, as white nationalists, plutocrats (and their bootlickers), and other advocates for gross inequality and population-scale subjugation seek to justify their ideology by claiming that it is biologically determined, and any attempt to change it is literally unnatural. Exhibit A for this is Jordan Peterson, whose obsession with a single species of lobsters is the founding myth of a transphobic, misogynist cult.

Dagg anticipated this debate decades in advance and repeatedly demolished its arguments for anyone who would listen, wielding science to slice through the self-serving bullshit of mediocre thinkers who want so desperately for their privilege to be the result of a biological process and not their own sociopathy.

Despite organized campaigns to marginalize Dagg and her work, she never gave up and was hugely influential on all kinds of scholars and thinkers. She was my own undergrad advisor at the University of Waterloo's Independent Studies program, and was an excellent mentor to me there. More broadly, she inspired generations of largely female giraffe biologists (I just met a giraffe keeper at Walt Disney World's Animal Kingdom who was a fan!), serving as a mentor and inspiration.

Dagg just received the Order of Canada, the second-highest honor awarded to Canadians (after the Order of Merit). The honor comes on the heels of The Woman Who Loves Giraffes, a documentary on Dagg's life and work.

This is wonderful news, seriously. Dagg is such a clear, uncompromising advocate for a rigorous approach to biology both as a means of understanding other animals and as a means for understand humans -- and is such a strong tonic against those who would abuse this tool for making sense of human behavior and social organization -- and she has accepted her marginalization as the price for her commitment to the truth.

Anne Dagg, Queen of Giraffes, appointed to Order of Canada among recipients with global influence [Stephanie Levitz/The National Post]

Wanda Diaz Merced is an astronomer at the International Astronomical Union (IAU) Office for Astronomy Outreach in Mitaka, Japan. Diaz Merced is blind and uses a technique to transform data from astronomical surveys into sounds for analysis. Over at Nature, Elizabeth Gibney interviewed Merced about how converting astronomical data into sound could bring discoveries that []

In the Galapagos Islands, a shoreside crane toppled over while loading a shipping container onto a barge, capsizing the boat and causing a terrible oil spill of hundreds of gallons of diesel fuel. It was Charles Darwins 1835 studies of the Galapagos Islandss biodiversity that sparked his theory of evolution by natural selection. From ABC []

Photographer Eric Brummel created this magnificent time-lapse video of the Milky Way in which the sky is stabilized so you can experience the Earths rotation. He captured the footage at Fonts Point, Anza-Borrego Desert State Park, California. From Universe Today: Eric created this time-lapse by using a star-tracker with his camera. A star-tracker rotates the []

One of the first things marketers learn in todays wired world is that theres no direct path to potential clients. Big companies are learning to laser-target their pitches to customers on the platforms they like best. Theres no reason start-ups of any size cant do the same, and the Become a Social Media Manager Certification []

A good website should be able to do a lot of things, and that means a good web designer needs to be just as versatile. If youre looking to break into this in-demand field but arent sure where to start, give The Complete 2020 Learn to Design Certification Bundlea look. Its actually a package of []

If youve got more than one Apple device, chances are your nightstand is a cluttered mess of charging cables; and if you take them out with you on the daily, your bag probably also has a tangled mass of chargers for your iPhone, AirPods, Apple Watch and so on. Its time to de-clutter your charging []

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Anne Dagg, pioneering giraffe biologist and feminist critic of "evolutionary psychology" receives the Order of Canada - Boing Boing

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DNA Of Headless Corpse In Idaho Reveals Story Of Ax Murders And Outlaws – IFLScience

January 3rd, 2020

The identity of a decapitated torso found in a dusty cave in Idaho has finally been revealed after 40 years of mystery. However, many questions still hang over this strange tale of murder, outlaws, and jailbreaks.

Investigators have confirmedthe headless body once belonged to Joseph Henry Loveless, a bootlegging outlaw who died in 1916 shortly after escaping from prison where he was serving time for murdering his second wife with an ax.

Speaking at a press conferenceon December 31, 2019, theteam explained how they cracked the case, but warned they are still unsure how his headless body ended up in the remote cave. Nevertheless, they were able to find Loveless 87-year-old grandson and tell him about his grandfathers wild story.

The mystery first came to light on August 26, 1979, when a family hunting for arrowheads in Buffalo Cave in rural Clark Country discovered a headless corpse wrapped in burlap.In 1991, a girl exploring the same cave system found a hand, leading to the discovery of a number of odd limbs also wrapped in burlap.

The remains were handed to anthropologists from Idaho State University and the Smithsonian Institution, as well as investigators from the FBI. Although they were able to deduce he was a male around 40 years old at the time of death and had reddish-brown hair, they were unable to make much progress with the identification.

Then, in 2019, came the help of the DNA Doe Project, a non-profit organization that uses genetic evidence to identify John and Jane Does using DNA obtained from people who have opted into law enforcement matching.

Using DNA obtained from the man's bones, they were able to find his grandson and hundreds of relatives (after all, hundreds of first cousins can descend from a single grandparent). Dealing with outlaws in the early 20th century who had numerous aliases, numerous wives, and few written records is no easy task, but further investigative work was able to pinpoint Joseph Henry Loveless out of a number of possible candidates. One major clue was that Loveless grave was empty with no date, as if he had just gone missing without a trace.

Further digging through news reports and records revealed that Loveless was born December 3, 1870, in the Utah Territory. Throughout his life of crime, he switched between a number of aliases, including Walter Cairns and Charles Smith. He married his second wife in 1905, with whom he had four kids, and was arrested for liquor bootlegging in 1914. He managed to break out of jail two years later and murdered his wife just a couple of months after his escape.

While serving another term in prison in 1916, he escaped by sawing through the bars using a tool he hid in his shoes. The next part remains a mystery, but shortly after the jailbreak, he was somehow killed, decapitated, and ended up in the Buffalo Cave system. The case remains open.

Its blown everyones minds, Lee Bingham Redgrave, a forensic genealogist with the DNA Doe Project, told theAssociated Press. The really cool thing, though, is that his wanted poster from his last escape is described as wearing the same clothing that he was found in, so that leads us to put his death date at likely 1916.

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DNA Of Headless Corpse In Idaho Reveals Story Of Ax Murders And Outlaws - IFLScience

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