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Pyrroloquinoline Quinone Market Is Set for a Rapid Growth and is Expected to Reach USD Billion by 2027| Haotian Pharm, Absorb Health, Life Extension,…

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Pyrroloquinoline Quinone Market Is Set for a Rapid Growth and is Expected to Reach USD Billion by 2027| Haotian Pharm, Absorb Health, Life Extension,...

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The Bronco Is Back! A Fleet Of OV-10s Will Help Train Air Force Forward Air Controllers – The Drive

It's not entirely clear where the seven aircraft are coming from. An OV-10D+, known by its old U.S. Navy Bureau Number (BuNo) 155493, and an OV-10G, also referred to by its BuNo, 155409, are among the Broncos that Blue Air Training is buying, according to Warbird News.

The Navy first acquired 155493 as an OV-10A in 1968 and it was among the fleet assigned to the service's famous Light Attack Squadron Four (VAL-4), also known as the "Black Ponies," during the Vietnam War. It was later transferred to the U.S. Marine Corps, where it was later converted into the OV-10D configuration with its distinctly longer nose, as well as uprated engines and other improvements. All of the Marine OV-10Ds were further upgraded into D+s in the 1990s with strengthed wings, updated wiring, and other improvements as part of a service-life extension program.

The Marine Corps, which was the last U.S. military service to fly the Bronco on a widespread basis, retired the last of its examples in 1995. The Air Force sent its remaining OV-10s to the bone yard four years earlier.

The U.S. State Department's Air Wing, which you can read about in more detail in this past War Zone story, eventually acquired 155493, among other OV-10s, and had Marsh Aviation put it through another upgrade and modification program to turn it into an herbicide sprayer aircraft to support counter-narcotics operations. Beyond the spray equipment, the most visible change was the addition of four-blade propellers to the plane's two engines.

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The Bronco Is Back! A Fleet Of OV-10s Will Help Train Air Force Forward Air Controllers - The Drive

Recommendation and review posted by Bethany Smith

This Startup’s Computer Chips Are Powered by Human Neurons – Futurism

Australian startup Cortical Labs is building computer chips that use biological neurons extracted from mice and humans, Fortune reports.

The goal is to dramatically lower the amount of power current artificial intelligence systems need to operate by mimicking the way the human brain.

According to Cortical Labs announcement, the company is planning to build technology that harnesses the power of synthetic biology and the full potential of the human brain in order to create a new class of AI that could solve societys greatest challenges.

The mouse neurons are extracted from embryos, according to Fortune, but the human ones are created by turning skin cells back into stem cells and then into neurons.

The idea of using biological neurons to power computers isnt new. Cortical Labs announcement comes one week after a group of European researchers managed to turn on a working neural network that allows biological and silicon-based brain cells to communicate with each other over the internet.

Researchers at MIT have also attempted to use bacteria, not neurons, to build a computing system in 2016.

As of right now, Corticals mini-brains have less processing power than a dragonfly brain. The company is looking to get its mouse-neuron-powered chips to be capable of playing a game of Pong, as CEO Hon Weng Chong told Fortune, following the footsteps of AI company DeepMind, which used the game to test the power of its AI algorithms back in 2013.

What we are trying to do is show we can shape the behavior of these neurons, Chong told Fortune.

READ MORE: A startup is building computer chips using human neurons [Fortune]

More on neurons: Artificial and Biological Neurons Just Talked Over the Internet

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This Startup's Computer Chips Are Powered by Human Neurons - Futurism

Recommendation and review posted by Bethany Smith

Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19…

CRANFORD, N.J., April 1, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today signed an exclusive six-month option agreement to in-license a stem-cell therapy for acute respiratory distress syndrome (ARDS) from a subsidiary of Novellus, Inc., a preclinical-stage biotechnology company based in Cambridge, MA.

Novellus's patented process uses its exclusive non-immunogenic synthetic messenger ribonucleic acid (mRNA) molecules to create induced pluripotent stem cells (iPSCs) that, in turn, generate mesenchymal stem cells (MSCs) with superior immunomodulatory properties. MSCs have been shown to be safe in over 900 clinical trials and to be safe and effective in treating a number of inflammatory diseases, including ARDS.

"ARDS is the most common cause of respiratory failure and mortality in COVID-19 patients. Currently, there is no proven treatment for ARDS. Literature supports the use of counter-inflammatory MSCs for ARDS, and papers published in China have shown that at least seven COVID-19 patients with ARDS responded to MSC therapy. Clearly this is an avenue that shows promise and should be pursued as a potential treatment for ARDS. We believe Novellus is at the forefront of creating allogeneic, iPSC-derived MSCs. These cells have the potential to overcome the limitations of MSCs derived from adult donors, which are telomere shortened and introduce variability into the manufacturing process," said Citius Chief Executive Officer Myron Holubiak.

Novellus Chief Science Officer Matt Angel, PhD, stated, "Using our mRNA-based cell-reprogramming technology, Novellus can provide a near-unlimited supply of MSCs for treating patients with ARDS, including those critically ill from COVID-19. These will be allogeneic ('off-the-shelf') cells that in vitro have demonstrated much greater expansion potential and much higher immunomodulatory protein expression than donor-derived MSCs. We are excited to employ our technology to such an urgent medical crisis and believe that our MSCs represent an ideal source of cells to be used in this extremely important development effort."

Holubiak added, "No effective pharmacotherapy for ARDS exists, and ARDS-related morbidity and mortality are high. MSCs have been studied in the treatment of lung injury, and we aim to build upon this work with Novellus's iPSC-derived MSCs to improve the immunomodulatory response in humans. We have assembled a team of experts who are dedicated to advancing this project to an Investigational New Drug (IND) application as quickly as possible."

About ARDSAcute respiratory distress syndrome (ARDS) is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain, particularly while inhaling, and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677ascott@citiuspharma.com

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Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19...

Recommendation and review posted by Bethany Smith

In a coronavirus crisis, Gairdner Awards honour eight explorers of how cells, genes and viruses work – The Globe and Mail

Guy Rouleau, shown at middle in 2010, is director of the Montreal Neurological Institute and Hospital and one of the recipients of this year's Gairdner Awards. The others are Mina Bissell, top left; Salim and Quarraisha Abdool Karim, middle left; Elaine Fuchs, bottom left; Rolf Kemier, top right; Masatoshi Takeichi, middle right; and Roeland Nusse, bottom right.

John Morstad/The Globe and Mail, handouts

A diverse group of eight scientists whose work has offered insight into how cells interact with each other and their environment, the genetic underpinnings of neurological disease and the transmission of the virus that causes AIDS, have been named this years winners of the Gairdner Awards the countrys most prestigious biomedical research prizes.

Coming in the midst of the COVID-19 pandemic, this years set of awards highlights the importance of basic science to understanding the fundamental processes of life and how those processes relate to human health around the world.

When you build up a scientific environment and a scientific community, you have people who are prepared to do whatever it takes [to address a global health crisis], said Janet Rossant, president and scientific director of the Toronto-based Gairdner Foundation, which announced the award winners on Tuesday. You never know whats going to give you insight into disease, Dr. Rossant said.

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Watch: Guy Rouleau explains the research that won him the Gairdner Wrightman Award.

Among the winners is Guy Rouleau, director of the Montreal Neurological Institute and Hospital, who is this years recipient of the Gairdner Wightman Award, which recognizes scientific leadership in Canada.

In addition to his work linking various rare genes that occur in the French Canadian population to disorders such as ALS (Amyotrophic Lateral Sclerosis), Dr. Rouleau is known for his efforts to make scientific research more accessible.

Starting in 2016, he placed his institute at the forefront of the open science movement by allowing the free flow of data, tools and research results without restrictions related to who will profit from the knowledge.

Reached at his home in Montreal, Dr. Rouleau said the initiative was spurred by a lack of new development in neurological disease, where few treatment options are available for those dealing with brain disorders including Alzheimers disease. We thought that by sharing openly and by breaking down barriers, this would accelerate things, Dr. Rouleau said.

A human T-cell, in blue, comes under attack by HIV, in yellow, the virus that causes AIDS.

Seth Pincus, Elizabeth Fischer, Austin Athman/National Institute of Allergy and Infectious Diseases/NIH via AP

The husband and wife team of Quarraisha and Salim Abdool Karim at the Centre for the AIDS Programme of Research in South Africa were named the joint winners of the John Dirks Gairdner Globe Health award for their work tackling HIV in Africa.

In 1990, the pair described the transmission of the virus that causes AIDS through the African population, which frequently involves the infection of teenage girls by older men. Their work laid the foundations for successful HIV prevention programs focused on women and womens health.

Previous winners of the global health award include Anthony Fauci of the National Institutes of Health in Bethesda, Md., who has lately become a prominent figure for helping to steer the U.S. response to COVID-19 and for repeatedly clarifying or correcting misleading statements by U.S. President Donald Trump.

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Past Gairdner recipient Dr. Anthony Fauci walks past U.S. President Donald Trump at a March 29 news conference at the White House.

Al Drago/Reuters

The five researchers named as recipients of this years Canada Gairdner International Award a prize that often portends a future Nobel win have all done groundbreaking work related to some aspect of the field known as cell signalling.

They include:

In previous years, Gairdner award winners have travelled across Canada giving lectures and meeting with students ahead of a fall symposium and award ceremony in Toronto.

Dr. Rossant said the Foundation is still assessing how this years activities will proceed in light of COVID-19.

Sign up for the Coronavirus Update newsletter to read the days essential coronavirus news, features and explainers written by Globe reporters.

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In a coronavirus crisis, Gairdner Awards honour eight explorers of how cells, genes and viruses work - The Globe and Mail

Recommendation and review posted by Bethany Smith

‘There was a growing weariness’: Rushing against a pandemic clock, Aspen Neurosciences secures $70M Series A – Endpoints News

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group he didnt give a name for the group to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinsons cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

I started thinking rather early onThere might be something on the horizon that we dont fully understand, Federoff told Endpoints News. We knew that if something did change, it could change rather quickly.

Operating with insight and knowledge other biotechs lacked access to, Federoff went into overdrive trying to close before Covid-19 hit the US, and he emerged today with $70 million in Series A funding led by OrbiMed. The other investors included Frazier Health Partners, Sam Altman and ARCH Venture Partners, the VC whose leader Robert Nelsen became one of the earliest and most prominent voices calling for change.

Weve had long conversations, Federoff said of him and Nelsen.

With the Series A, Federoff has convinced A-list investors to back one version of a long-sought solution to Parkinsons. Aspen will use stem cells grown from Parkinsons patients own skin tissue to grow dopamine neurons that can be implanted into the brain and hopefully replace the degenerating neurons. The idea has been around for decades, with the first transplant occurring in the 80s, but it was never scaleable. The technology to produce stem cells on demand didnt exist.

The company has a rival in BlueRock, which uses donor stem cells and which Bayer acquired in August at a valuation of $1 billion.

Over the winter, though, the investor hunt became less about pitching the science which Federoff says everyone agreed was promising than about beating the clock and investors rising worries about the economy. He prepared to work fast, turning an early meeting with Frazier at the JP Morgan Healthcare Conference into a pivotal one. As the months passed, he phoned investors multiple times a day to keep funding on track.

They were already in from the standpoint of the science, Federoff said. I could tell that there was a growing weariness about whether all that they had previously considered as part of their own respective portfolios outside of Aspen would all be possible.

The money he secured will help fund their Phase I trial on Parkinsons and a second program that uses a form of gene therapy to implant stem cells that have a genetic marker for Parkinsons edited out. The plan had been to start a trial in 2021, but Federoff knows there are no more guarantees.

At this time its not clear what Covid-19 will do to projections, he said.

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'There was a growing weariness': Rushing against a pandemic clock, Aspen Neurosciences secures $70M Series A - Endpoints News

Recommendation and review posted by Bethany Smith

Infrared Laser Treatment of TBI, PTSD, and Depression: An Expert Perspective – Psychiatry Advisor

Disclosure: Dr Henderson is the president and principal owner of The Synaptic Space, a neuroimaging consulting firm, and owner of Neuro-Luminance Corporation. Please see the listed studies for a full list of disclosures.

During the last 20 years, a large body of research has accumulated on the beneficial effects of infrared light in the range of 600 to 1000 nm. Infrared light can activate mitochondria, which in turn stimulate second messenger systems, DNA transcription, and growth factors.1,2 As a result, new synapses are formed, circuits regrow, and pluripotent stem cells differentiate into neurons.

Animal studies have shown that infrared photobiomodulation (PBM) may reduce the size and severity of brain injury and stroke, as well as diminish damage and physiological symptoms in depression, posttraumatic stress disorder (PTSD), Parkinson disease, and Alzheimer disease.1,3-6 Michael Hamblin, PhD, from the Wellman Center for Photomedicine at Massachusetts General Hospital in Boston, a leader in the field, describes PBM as the use of red or near-infrared light to stimulate, heal, regenerate, and protect tissue that has either been injured, is degenerating, or else is at risk of dying.1

Generally in medicine we shy away from the word heal when referring to the brain, and regenerate stirs vague recollections of Frankenstein. Nevertheless, early findings in mouse models of brain injury and disease have spawned a different sort of monster in the commercial world. The internet is now loaded with companies offering infrared LED helmets or pads for the treatment of traumatic brain injury (TBI) and other brain disorders, often based on exaggerated claims about healing the brain. Exorbitant prices in the thousands of dollars are charged for a device that can be made for less than $30. As a result, the public is misled and the potential scientific benefits of infrared light are sullied.

It is time to separate fact from fiction. Yes, infrared light can induce the cellular events described here, reduce the size of stroke injury or TBI in mouse models, and protect neurons from neurotoxins. But is treating a human with a 0.5-W LED the same as treating a mouse? Certainly not! When it comes to infrared light treatment, it is all a matter of getting there: the infrared light must be able to penetrate all the overlying tissue to reach the brain.

Can Infrared Light Reach the Brain?

Can 0.5-W LEDs penetrate human scalp and skull to reach the brain? The answer is No.2 My colleague, Larry Morries, DC, and I showed that these LEDs did not even penetrate 2 mm of human skin. In contrast, our laser device, which emits infrared light in the range of 10 to 15 W, was able to effectively penetrate human tissue. We found that 33% of our 10-W infrared laser energy penetrated 2 mm of human skin and delivered from 1.2% to 2.4% of the energy from our device 3 cm into the brain. These data were replicated in a study by Juanita Anders, PhD, and colleagues at the Uniformed Services University of Health Sciences.7

The human scalp and skull provide a significant barrier. Infrared light energy needs to be in the range of 0.9 to 15 J/cm2 at the target tissue to activate mitochondria and other cellular events.2-3,8-9 Even if a 0.5-W LED only had to penetrate the skull to reach the surface of the brain, it could only deliver 0.0064 J/cm2, or 1/140th of the minimum energy necessary to induce PBM.10 No energy would be expected to reach the depths of the brain needed to treat stroke, Parkinson disease, Alzheimer disease, or many brain injuries. Although more than 40% of the incident light from a light source may penetrate mouse skull, only 4.2% penetrates human skull.8,10

There is a hairier problem facing LED devices: human hair blocks infrared light. More than 98% of infrared light can be blocked by 2 mm of hair (ie, 9.764 W of a 10-W beam of 810 nm infrared light is absorbed by human hair).11 If 98% of the energy from a 0.5-W LED is absorbed by hair, 80% to 90% is absorbed by 2 mm of skin, and 96% of incident energy is attenuated by skull, then claims of neurophysiological benefits of LED-based devices become highly questionable.

Another misconception propagated by companies selling LED-based devices is that multiple LEDs somehow increase light penetration, even though each LED projects light on its own path. For example, 100 0.5-W LEDs do not generate 50 W on the brain, they generate 0.5 W on 100 spots.11 The argument that light scattering in the brain provides the cumulative value of multiple LEDs also falls apart if nothing can get through the overlying tissues.

Given that a small percentage (<1%) of incident infrared light gets through human scalp and skull, we must question the results of human trials of LEDs. Studies demonstrated small yet almost insignificant positive effects, and the benefits are generally transient.12 In contrast, our protocol yields persistent and robust clinical changes in patients with TBI, PTSD, and depression.

Treating TBI, PTSD, and Depression with Infrared Light

Our patented multi-Watt Neuro-Luminance approach involves transcranial infrared laser treatment (NILT), and in 2015 we published an initial open-label trial of 10 subjects with mild to moderate TBI.13 After a course of 10 NILT treatments (20 treatments in a subset of 4 patients), all patients experienced significant clinical improvement of symptoms, including headaches, cognitive problems, sleep disturbances, irritability, and depression. In telephone interviews every 6 months after treatment, patients report sustained improvements.12

An open-label clinical trial (n=39) of multi-Watt Neuro-Luminance demonstrated effectiveness for depression.4 Overall, 92% of patients responded and 82% remitted, which is notably better than the response rate for oral antidepressants. Patients saw benefits within 4 treatments, and some achieved resolution of depressive symptoms within 8 treatments. In follow-up telephone interviews, patients report sustained improvements. Similarly, in our unpublished data, using a protocol of 20 treatments, each lasting 24 minutes, over the course of 9 weeks, 20 patients with PTSD treated with multi-Watt NILT experienced reduced hyperarousal, anxiety, sleep disturbance, and nightmares.

LED Photobiomodulation in Comparison

Naeser and colleagues15 treated 2 patients with TBI daily for approximately 1 hour by applying 3 separate LED cluster heads (2 head; 1 foot). The first patient, who was 7 years post-TBI and had significant postconcussive symptoms, received weekly treatments over the course of 7 months and then daily treatments at home for more than 6 years. The patient experienced transient benefits, and if treatment was stopped, symptoms returned within 2 weeks.15 The second patient received daily treatments, and in 4 months, most symptoms improved, allowing her to return to work. This patient also noted that symptoms returned if treatments were stopped for more than 1 week.15

In an open-label study,16 11 patients with TBI and persistent cognitive dysfunction were treated for 18 sessions, each lasting 20 minutes, over the course of 6 weeks. At follow-up, there had been a significant effect on attention, inhibition, verbal learning and memory, and long-delay free recall.16 The LED treatment led to mild improvement in 3 of 5 cases of depression.

In 12 patients with TBI treated with 220 0.5-W LEDs for 18 sessions, each lasting 20 minutes, over the course of 6 weeks, there was significant improvement in psychological testing results (P =.45).17 However, the study did not correct for multiple comparisons, instead using parallel paired t-tests, which could exaggerate findings.18 PTSD has received considerably less attention.19,20

Cassano and colleagues21 described a 5-W laser treatment of 4 patients with depression. In a double-blind, sham-controlled extension of their initial findings, subjects in the treatment group received 16 treatments, each lasting 30 minutes, over the course of 8 weeks.22 In 13 completers, Hamilton-D-17 scores separated the treatment group from sham controls (mean score, 15.74.41 vs 6.17.86; P =.031). In contrast, in our open-label trial of a 13-W laser, the mean Hamilton-D-17 score decreased from baseline (mean score, 21.485.24 to 6.05.12; P =6.4510-13).23

Table. Case series, open-label, and double-blind studies of infrared light therapy for TBI, PTSD, and depression

Alternative Explanation for Clinical Response to LED Brain Treatments

Researchers, along with the human PBM field, need to reconsider the potential mechanisms underlying the meager improvements derived from LED-based devices. The light from LED devices may not penetrate beyond the skin, but could induce central nervous system benefits via a remote or systemic effect in irradiated skin, dubbed remote photobiomodulation.24

Infrared irradiation can have remote or indirect effects on tissue that has not been irradiated. For example, Braverman and colleagues25 demonstrated this indirect effect by creating matching skin lesions on the left and right dorsum of a rabbit, treating 1 side with infrared light. Both lesions showed accelerated healing relative to nonirradiated controls. Rochkind and colleagues26 demonstrated that remote PBM could occur in the peripheral nervous system and the central nervous system. After bilateral sciatic nerve crush, 1 side was irradiated with infrared light and the other side was not. Nerves on both sides showed enhanced recovery of function, and the number of anterior horn motor neurons was greater on both sides compared with nonirradiated controls.

Ganeshan and colleagues27 irradiated the dorsum and hind limbs of a rat with infrared light (670 nm) before injection of a neurotoxin (MPTP) and demonstrated reduced loss of dopaminergic neurons in rodents treated with indirect PBM to the skin compared with untreated controls. Given the overwhelming evidence that low-power LEDs do not penetrate the brain, it is more likely that the benefits of LED-based devices result from an effect mediated by the skin, where most, if not all, of the infrared energy is absorbed. In other words, LED-based devices may be working by remote PBM.

Conclusions

The excitement about the potential of infrared light therapy is not merely that it does not involve taking a pill. There is considerable enthusiasm about its potential to treat conditions such as TBI, dementia, and Parkinson disease. In our excitement, we must not overlook the unique physical limitations of light. Similarly, we must not imbue infrared light with magical powers. Infrared light can only work if it reaches target tissue.

Thus, a sharp divide can be drawn between LED-based treatment technologies, which offer minimal results and may not even reach the brain, and multi-Watt technologies that demonstrably reach the brain and offer lasting clinical benefit. Potentially, infrared light may prove to be effective for numerous neuropsychiatric conditions. However, for infrared light to work on the brain, it must be able to reach the brain.

References

1. Hamblin MR. Shining light on the head: Photobiomodulation for brain disorders. BBA Clin. 2016;6:113-124.

2. Henderson TA, Morries, LD. Near-infrared photonic energy penetration: can infrared phototherapy effectively reach the human brain? Neuropsychiatr Dis Treat. 2015;11:2191-2208.

3. Chung H, Dai T, Sharma SK, Huang YY, Carroll JD, Hamblin MR. The nuts and bolts of low-level laser (light) therapy. Ann Biomed Eng. 2012;40(2):516-533.

4. Henderson TA, Morries LD. Multi-Watt near-infrared phototherapy for the treatment of comorbid depression: an open-label single-arm study. Front Psychiatry. 2017;8:187.

5. Johnstone DM, Moro C, Stone J, Benabid AL, Mitrofanis J. Turning on lights to stop neurodegeneration: the potential of near infrared light therapy in Alzheimers and Parkinsons disease. Front Neurosci. 2016;11;9:500.

6. Hamblin MR. Photobiomodulation for Alzheimers disease: has the light dawned? Photonics. 2019;6(3):77.

7. Tedford CE, DeLapp S, Jacques S, Anders J. Quantitative analysis of transcranial and intraparenchymal light penetration in human cadaver brain tissue. Lasers Surg Med. 2015;47(4):312-322.

8. Ando T, Xuan W, Xu T, et al. Comparison of therapeutic effects between pulsed and continuous wave 810-nm wavelength laser irradiation for traumatic brain injury in mice. PLoS One. 2011;6(10):e26212.

9. Yip KK, Lo SC, Leung MC, So SK, Tang CY, Poon DM. The effect of low-energy laser irradiation on apoptotic factors following experimentally induced transient cerebral ischemia. Neuroscience. 2011;190:301-306.

10. Lapchak PA, Boitano PD, Butte PV, et al. Transcranial near-infrared laser transmission (NILT) profiles (800 nm): systematic comparison in four common research species. PLoS One. 2015;3;10(6):e0127580.

11. Henderson TA, Morries LD. Near-infrared photonic energy penetration principles and practice. In: Hamblin, MR and Huang YY, eds. Photobiomodulation and the Brain: Low-level Laser (Light) Therapy in Neurology and Neuroscience. London: Academic Press; 2019.

12. Morries LD, Henderson TA. Treatment of traumatic brain injury with near-infrared light. In: Hamblin, MR and Huang YY, eds. Photobiomodulation and the Brain: Low-level Laser (Light) Therapy in Neurology and Neuroscience. London: Academic Press; 2019.

13. Morries LD, Cassano P, Henderson TA. Treatments for traumatic brain injury with emphasis on transcranial near-infrared laser phototherapy. Neuropsychiatr Dis Treat. 2015;11:2159-75.

14. Connolly KR, Thase ME. If at first you dont succeed: a review of the evidence for antidepressant augmentation, combination and switching strategies. Drugs. 2011;71(1):43-64.

15. Naeser MA, Saltmarche A, Krengel MA, Hamblin MR, Knight JA. Improved cognitive function after transcranial, light-emitting diode treatments in chronic, traumatic brain injury: two case reports. Photomed Laser Surg. 2011;29(5):351-358.

16. Naeser MA, Zafonte R, Krengel MH, et al. Significant improvements in cognitive performance post-transcranial, red/near-infrared light-emitting diode treatments in chronic, mild traumatic brain injury: open-protocol study. J Neurotrauma. 2014;31(11):1008-1017.

17. Hipskind SG, Grover FL Jr, Fort TR, et al. Pulsed transcranial red/near-infrared light therapy using light-emitting diodes improves cerebral blood flow and cognitive function in veterans with chronic traumatic brain injury: a case series. Photobiomodul Photomed Laser Surg. 2019;37(2):77-84.

18. Henderson TA, Morries LD. Infrared light cannot be doing what you think it is doing (re: DOI: 10.1089/photob.2018.4489). Photobiomodul Photomed Laser Surg. 2019;37(2):124-125.

19. Schiffer F, Johnston AL, Ravichandran C, et al. Psychological benefits 2 and 4 weeks after a single treatment with near infrared light to the forehead: a pilot study of 10 patients with major depression and anxiety. Behav Brain Funct. 2009;5:46.

20. LED light therapy to improve cognitive & psychosocial function in TBI-PTSD veterans. ClinicalTrials.gov. NCT02356861. https://clinicaltrials.gov/ct2/show/NCT02356861. Accessed February 29, 2020.

21. Cassano P, Cusin C, Mischoulon D, et al. Near-infrared transcranial radiation for major depressive disorder: proof of concept study. Psychiatry J. 2015;2015:352979.

22. Cassano P, Petrie SR, Mischoulon D, et al. Transcranial photobiomodulation for the treatment of major depressive disorder. The ELATED-2 Pilot Trial. Photomed Laser Surg. 2018;36(12):634-646.

23. Henderson TA, Morries LD. Multi-Watt near-infrared phototherapy for the treatment of comorbid depression: an open-label single-arm study. Front Psychiatry. 2017;8:187.

24. Gordon LC, Johnstone DM. Remote photobiomodulation: an emerging strategy for neuroprotection. Neural Regen Res. 2019;14(12):2086-2087.

25. Braverman B, McCarthy RJ, Ivankovich AD, Forde DE, Overfield M, Bapna MS. Effect of helium-neon and infrared laser irradiation on wound healing in rabbits. Lasers Surg Med. 1989;9(1):50-58.

26. Rochkind S, Rousso M, Nissan M, Villarreal M, Barr-Nea L, Rees DG. Systemic effects of low-power laser irradiation on the peripheral and central nervous system, cutaneous wounds, and burns. Lasers Surg Med. 1989;9(2):174-182.

27. Ganeshan V, Skladnev NV, Kim JY, Mitrofanis J, Stone J, Johnstone DM. Pre-conditioning with remote photobiomodulation modulates the brain transcriptome and protects against MPTP insult in mice. Neuroscience. 2019;400:85-97.

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Seattle Genetics Announces Potential Accelerated Approval Pathway in the US for PADCEV (enfortumab vedotin-ejfv) in Combination with Immune Therapy…

BOTHELL, Wash.--(BUSINESS WIRE)-- Seattle Genetics, Inc.. (Nasdaq:SGEN) today provided an update on the phase 1b/2 multicohort EV-103 trial (also known as KEYNOTE-869) of PADCEVTM (enfortumab vedotin-ejfv) in combination with anti-PD-1 therapy pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting. Based on recent discussions with the U.S. Food and Drug Administration (FDA), data from the randomized cohort K, along with other data from the EV-103 trial evaluating PADCEV combined with pembrolizumab as first-line therapy for cisplatin-ineligible patients, could potentially support registration under accelerated approval regulations in the United States. PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.1

We are excited that EV-103 provides PADCEV with a potential pathway for U.S. accelerated approval in first-line metastatic urothelial cancer, said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. Our initial data on the combination of PADCEV and pembrolizumab in previously untreated patients who could not receive cisplatin are encouraging.

EV-103 is a multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive urothelial cancer, and in locally advanced or metastatic urothelial cancer in first- or second-line settings. Cohort K from EV-103 is intended to enroll 150 patients randomized 1:1 to PADCEV monotherapy or PADCEV in combination with pembrolizumab in locally advanced or metastatic urothelial cancer patients who are ineligible for cisplatin-based chemotherapy. The primary outcome measure is objective response rate (ORR) per blinded independent central review (BICR) using RECIST 1.1 and duration of response (DoR).

In addition to EV-103, the recently initiated EV-302 phase 3 randomized clinical trial is intended to support global registrations and potentially serve as a confirmatory trial if accelerated approval is granted based on EV-103. The EV-302 trial is evaluating the combination of PADCEV and pembrolizumab with or without chemotherapy versus chemotherapy alone in patients with previously untreated locally advanced or metastatic urothelial cancer. Importantly, EV-302 includes metastatic urothelial cancer patients that are either eligible or ineligible for cisplatin-based chemotherapy. The trial is expected to enroll 1,095 patients and has dual primary endpoints of progression-free survival and overall survival. Both the EV-103 and EV-302 trials are being conducted in collaboration with Astellas and Merck.

FDA recently granted Breakthrough Therapy designation for PADCEV in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting based on initial results from the EV-103 trial.

PADCEV (enfortumab vedotin-ejfv) was approved by the FDA in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDAs Accelerated Approval Program based on tumor response rate. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.2

About Bladder and Urothelial Cancer

It is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020.3 Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.4 Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide.5

About PADCEV

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.6,7 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).8 PADCEV is co-developed by Astellas and Seattle Genetics.

Important Safety Information

Warnings and Precautions

Adverse Reactions

Serious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab Abnormalities

In one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased, hemoglobin decreased, phosphate decreased, lipase increased, sodium decreased, glucose increased, urate increased, neutrophils decreased.

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Seattle Genetics

Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in peoples lives. The company is headquartered in Bothell, Washington, and has offices in California, Switzerland and the European Union. For more information on our robust pipeline, visit https://www.seattlegenetics.com and follow @SeattleGenetics on Twitter. For information on our response to the COVID-19 pandemic, please visit our website.

About the Astellas and Seattle Genetics Collaboration

Seattle Genetics and Astellas are co-developing PADCEV under a collaboration that was entered into in 2007 and expanded in 2009. Under the collaboration, the companies are sharing costs and profits on a 50:50 basis worldwide.

About the Seattle Genetics, Astellas and Merck Collaboration

Seattle Genetics and Astellas entered a clinical collaboration agreement with Merck to evaluate the combination of Seattle Genetics and Astellas PADCEV and Mercks KEYTRUDA (pembrolizumab), in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Seattle Genetics Forward-Looking Statements

Certain statements made in this press release are forward looking, such as those, among others, relating to the potential of data from the EV-103 clinical trial to support accelerated approval in the U.S. of PADCEV in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting; the possibility of using data from the EV-302 clinical trial to obtain global regulatory approval or confirm accelerated approval of PADCEV in the referenced first line setting; clinical development plans relating to PADCEV; the therapeutic potential of PADCEV; and its possible safety, efficacy, and therapeutic uses, including in the first-line setting. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the possibility that ongoing and subsequent clinical trials of PADCEV may fail to produce data sufficient to support regulatory approvals; the fact that FDA has not made a final determination regarding whether the data from the EV-103 clinical trial will be sufficient to support accelerated approval in the U.S.; the risk that the COVID-19 pandemic could delay our ability to conduct the EV-103 clinical trial and delay FDAs regulatory timelines, including with respect to any potential accelerated approval; the fact that adverse events or safety signals may occur and that adverse regulatory actions or other setbacks could occur as PADCEV advances in clinical trials even after promising results in earlier clinical trials. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption Risk Factors included in the companys Annual Report on Form 10-K for the year ended December 31, 2019 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

1Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.2 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.3 American Cancer Society. Cancer Facts & Figures 2020. https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2020/cancer-facts-and-figures-2020.pdf. Accessed 02-20-2020.4 American Society of Clinical Oncology. Bladder cancer: introduction (10-2017). https://www.cancer.net/cancer-types/bladder-cancer/introduction. Accessed 05-09-2019.5 International Agency for Research on Cancer. Cancer Tomorrow: Bladder. http://gco.iarc.fr/tomorrow.6 Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.7 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.8 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.

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Evotec Expands its iPSC-Based Cell Therapy Platform EVOcells Through Licensing Agreement with panCELLa – BioSpace

HAMBURG, Germany and TORONTO, April 2, 2020 /CNW/ - Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the innovative biotechnology company panCELLa Inc. announced today that the companies have entered into a licensing and investment agreement.

Under the terms of the agreement, Evotec will receive a non-exclusive licence to access panCELLa's proprietary iPS cell lines "iACT Stealth Cells", which are genetically modified to prevent immune rejection of derived cell therapy products ("cloaking"). Furthermore, Evotec will also have access to a new-generation cloaking technology known as hypoimmunogenic cells. In addition, the "FailSafe" mechanism effectively addresses a key challenge in iPSC-based cell therapy, potential tumour formation by residual undifferentiated cells.

Using the cell lines, Evotec will be able to develop iPSC-based, off-the-shelf cell therapies with long-lasting efficacy that can be safely administered to a broad population of patients without the use of medication to supress the patients' immune system. With a growing portfolio of iPSC-based cell therapy projects at Evotec, access to research as well as GMP-grade iPSC lines modified with one or both of the panCELLa technologies significantly accelerates Evotec's cell therapy discovery and development efforts. Modified iPSC lines will be available for the development of cell therapy approaches across a broad range of indications by Evotec and potential partners. Furthermore, Evotec has made an investment to take a minority stake in panCELLa and has nominated Dr Andreas Scheel to join panCELLa's supervisory board.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Cell therapies hold enormous potential as truly regenerative or curative approaches for a broad range of different diseases with significant medical need. Integrating panCELLa's technology and cell lines into our ongoing proprietary research and development efforts strengthens Evotec's position in cell therapy. It is our goal to provide safe highly-effective cell therapy products to as many patients as possible. In addition to small molecules and biologics, cell therapy will become yet another major pillar of Evotec's multimodality discovery and development platform."

Mahendra Rao, MD, PhD, CEO at panCELLa, added: "We welcome the partnership with Evotec. Evotec's widely recognised expertise and existing portfolio of iPSC-related technology platforms will allow panCELLa to rapidly advance its own therapeutic interests in NK cell therapy, pancreatic islet production and iPSC-derived MSC platform, in addition to enabling panCELLa to make its platform technologies widely available. I believe that the investment by Evotec in our company is a strong validation of the leading role of panCELLa in the field of regenerative medicine and in the utility of its platform technologies. We welcome Dr Andreas Scheel to our Board."

No financial details of the agreement were disclosed.

About Evotec and iPSCInduced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from adult cells. The iPSC technology was pioneered by Shinya Yamanaka's lab in Kyoto, Japan, who showed in 2006 that the introduction of four specific genes encoding transcription factors could convert adult cells into pluripotent stem cells. He was awarded the 2012 Nobel Prize along with Sir John Gurdon "for the discovery that mature cells can be reprogrammed to become pluripotent". Pluripotent stem cells hold great promise in the field of regenerative medicine. Because they can propagate indefinitely, as well as give rise to every other cell type in the body (such as neurons, heart, pancreatic and liver cells), they represent a single source of cells that could be used to replace those lost to damage or disease.

Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated iPSC platforms in the industry. Evotec's iPSC platform has been developed over the last years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach the highest industrial standards, and to use iPSC-based cells in cell therapy approaches via the Company's proprietary EVOcells platform.

About cell therapy and panCELLa's FailSafe iPSC technologyCell therapy, one of the most promising regenerative medicine approaches, replaces a patient's missing or broken cells with functioning cells from a range of different sources, either from a donor, from the patient's own material, or from stem cells. The advent of induced pluripotent stem cells ("iPSC") has opened up stem cells as an almost unlimited source of consistent-quality material for such cell therapies. At the same time, differentiating cell therapies from a single validated source circumvents critical risks of contamination associated with administering both donor and patient cell material.

However, the patient's immune system will treat such iPSC-based transplant as "foreign" and use the body's immune system to counteract the therapy, thus undermining its long-term efficacy. While organ transplants require an often lifelong regimen of immunosuppressants, iPSC-derived cells used for cell therapies can be cloaked to make them undetectable by the patient's immune system, thus avoiding rejection and enabling effective long-term relief of the patient's symptoms.

To increase the safety of such iPSC-derived cell products, panCELLa's proprietary FailSafe technology is able to inactivate any iPSC-derived proliferating cell before and after transplantation through the use of a readily available anti-infective medication. FailSafe is the only quantifiable "safety switch" on the market which is expected to be critical for regulators, clinicians and patients to make informed decisions when evaluating treatment options.

About panCELLa Inc. Incorporated in August 2015, panCELLa (www.pancella.com) was founded by Dr Andras Nagy and Dr Armand Keating based on Dr Nagy's ground-breaking work in the area of stem cell research. Through panCELLa, Drs Keating and Nagy are seeking to create an effective cell therapy derived from stem cells, which are modified to provide a sufficient and very high level of safety before and after the cells are introduced to the patient. panCELLa serves those companies developing products from stem cells. panCELLa seeks to create universal "off the shelf" FailSafe Cells and to assist pharmaceutical and biotechnology sectors to achieve such with their own cell lines. Targeted medical applications include deadly, debilitating, or aggressive diseases requiring immediate treatment where there is no time to cultivate a customized stem cell treatment from the patient (i.e. cancer, cardiac infarct, diabetes, stroke and spinal cord injury).

About Evotec SEEvotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD LOOKING STATEMENTSInformation set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

SOURCE panCELLa Inc.

Company Codes: Frankfurt:EVT, OTC-PINK:EVTCY

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Stem Cell Therapy for Colon Cancer – Yahoo Finance

WASHINGTON, April 2, 2020 /PRNewswire/ -- An article published in Experimental Biology and Medicine (Volume 245, Issue 6, March 2020) (https://journals.sagepub.com/doi/pdf/10.1177/1535370220910690) examines the safety of stem cell therapy for the treatment of colon cancer.The study, led by Dr. J. Liu in the State Key Laboratory of Bioreactor Engineering and Shanghai Key Laboratory of New Drug Design at the East China University of Science and Technology in Shanghai (China), reports that mesenchymal stem cells from a variety of sources promote the growth and metastasis of colon cancer cells in an animal model.

Mesenchymal stem (MSCs), a category of adult stem cells, are being evaluated as therapy for numerous cancers.MSCs are excellent carriers for tumor treatment because they migrate to tumor tissues, can be genetically modified to secrete anticancer molecules and do not elicit immune responses.Clinical trials have shown that MSCs carrying modified genes can be used to treat colon cancer as well as ulcerative colitis. However, some studies have demonstrated MSCs can differentiate into cancer-associated fibroblasts and promote tumor growth.Therefore, additional studies are needed to evaluate the safety of MSCs for targeted treatment of colon cancer.

In the current study, Dr. Liu and colleagues examined the effects of mesenchymal stem cells (MSCs) from three sources (bone marrow, adipose and placenta) on colon cancer cells.MSCs from all three sources promoted tumor growth and metastasis in vivo. In vitro studies demonstrated that MSCs promote colon cancer cell stemness and epithelial to mesenchymal transition, which would enhance tumor growth and metastasis respectively.Finally, the detrimental effects of MSCs could be reversed by blocking IL-8 signaling pathways. Dr. Ma, co-author on the study, said that "Mesenchymal stem cells have a dual role: promoting and/or suppressing cancer. Which effect is dominant depends on the type of tumor cell, the tissue source of the MSC and the interaction between the MSC and the cancer cell. This is the major issue in the clinical application research of MSCs, and additional preclinical experimental data will be needed to evaluate the safety of MSCs for colon cancer treatment."

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said: "Lui and colleagues have performed elegant studies on the impact of mesenchymal stem cells (MSCs), from various sources, upon the proliferation, stemness and metastasis of colon cancer stem cells (CSCs) in vitro and in vivo. They further demonstrate that IL-8 stimulates the interaction between colon CSCs and MSCs, and activates the MAPK signaling pathway in colon CSCs.This provides a basis for the further study of MSCs as a biologic therapy for colon cancer."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit http://www.sebm.org. For anyone interested in publishing in the journal, please visit http://ebm.sagepub.com.

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Is There a New CAR T-Cell Treatment for Mantle Cell Lymphoma? – AJMC.com Managed Markets Network

Maggie L. Shaw

Mantle cell lymphoma is a type of B-cell non-Hodgkin lymphoma with a typically poor prognosis. Even with an allogeneic stem cell transplant, patients can become resistant to chemotherapy. Most do not survive 4 or 5 years after diagnosis, and the 10-year survival rate hovers between 5% and 10%.

Chimeric antigen receptor (CAR) T-cell therapy has been making great inroads as targeted treatment for many times of cancers highly resistant to other treatments, by prolonging patient survival and increasing their quality of life. Until now, similar results have not been seen in patients with MCL. However, with their successful phase 2 ZUMA-2 trial results just published in the New England Journal of Medicine, a group of researchers led by Michael Wang, MD, from The University of Texas MD Anderson Cancer Center, are able to show that these patients can benefit from the specialized therapy.

In this study conducted in the United States and Europe, the patient population had relapsed/refractory progressive disease despite receiving Brutons tyrosine kinase (BTK) inhibitor therapy and from 3 to 5 prior therapies.

BTK inhibitor therapy has greatly improved outcomes in patients with relapsed or refractory mantle cell lymphoma, yet patients who have disease progression after receiving the treatment are likely to have poor outcomes, with median overall survival of just 6 to 10 months, the authors said.

The median patient age was 65 years (range, 38-79). They were evaluated for response to a single infusion of KTE-X19, an anti-CD19 CAR T-cell therapy, that was dosed at 2106 CAR T cells/kg of body weight. Seventy-four patients were enrolled between October 24, 2016, and April 16, 2019; and the treatment was manufactured for 71 and ultimately administered to 68.

There was a follow-up after 60 patients were monitored for 7 months, at which time a primary efficacy analysis was conducted. The primary endpoint was objective response (complete [CR] or partial [PR]), which was confirmed via bone marrow evaluation and positron emission tomography-computed tomography.

Overall, 85% of the entire study cohort of 74 patients was able to reach an objective response to KTE-X19, 59% of whom had a CR. These numbers were even higher among the group of 60 patients. Ninety-three percent (95% CI, 84%-98%) achieved an objective response, which was evaluated by an independent radiologic review committee. And of this group, 67% (95% CI, 53%-78%) had a CR.

The median times to response were impressive, with there being 1 month (range, 0.8-3.1) to initial response and 3 months (range, 0.9-9.3) to CR. In addition, of the 42 patients who initially had a PR or stable disease, 24 (21 who had a PR, 3 who had SD) progressed to a CR in a median 2.2 months (range, 1.8-8.3).

Progression-free (PFS) and overall survival (OS) results also show promise to treatment with KTE-X19. As of the data cutoff date, there was evidence of remission in 78% patients who had a CR, with similar results seen in 57% of patients from the primary efficacy analysis. Overall, at 12 months, the PFS and OS were 61% and 83%, respectively.

Common adverse events to the treatment of grade 3 or higher included cytopenias (94%) and infections (32%). Ninety-one percent also experienced cytokine release syndrome, with a median time to onset of 2 days (range, 1-13) for any grade and 4 days (range, 1-9) for at least grade 3, but none died as a result. According to the study authors, most symptoms were reversible.

ZUMA-2 is the first multi-center, phase 2 study of CAR T-cell therapy for relapsed/refractory mantle cell lymphoma, and these efficacy and safety results are encouraging, stated Wang. Although this study continues, our reported results, including a manageable safety profile, point to this therapy as an effective and viable option for patients with relapsed or refractory mantle cell lymphoma.

Reference

Wang M, Munoz J, Goy A, et al. KTE-X19 CAR T-Cell therapy in relapsed or refractory mantle-cell lymphoma. N Engl J Med. 2020;382;1331-1342. doi: 10.1056/NEJM0a1914347.

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Stem Cells, Nerves Found to Interact in Cancer Progression – Pharmacy Times

Researchers at the Institute of Oral Biology of the University of Zurich have released 2 studies that examine how stem cells promote neuronal growth in tissue regeneration and in cancer progression.

Their findings demonstrate that different stem cell populations are innervated in distinct ways and that innervation may be crucial for proper tissue regeneration, according to the study. They also demonstrate that cancer steam cells likewise establish contacts with nerves.

Stem cells can generate a variety of specific tissues that are increasingly being used for clinical application, such as the replacement of bone or cartilage, but are present in cancerous tissues and are involved in cancer progression and metastasis. Nerves are therefore fundamental for regulating the physiological and regenerative processes involving stem cells.

Using organ-on-a-chip technology, which relies on small 3-dimensional devices mimicking the basic function of human organs and tissues, the researchers demonstrated that both types of stem cells promoted neuronal growth. The dental pulp stem cells, however, yielded better results compared with bone marrow stem cells. They induced more elongated neurons, formed dense neuronal networks, and established close contacts with nerves.

Dental stem cells produce specific molecules that are fundamental for the growth and attraction of neurons. Therefore, stem cells are abundantly innervated, according to the study authors. The formation of such extended networks and the establishment of numerous contacts suggest that dental stem cells create functional connections with nerves of the face. Therefore, these cells could represent an attractive choice for the regeneration of functional, properly innervated facial tissue.

In the second study, the researchers examined the interaction between nerves and cancer stem cells found in ameloblastoma, an aggressive tumor of the mouth. They first demonstrated that ameloblastomas have stem cell properties and are innervated by facial neurons. When ameloblastoma cells were isolated and placed in the organ-on-a-chip devices, they retained not only their stem cell properties, but also attracted nerves and established contact with them.

Nerves appear to be fundamental for the survival and function of cancer stem cells. These results create new possibilities for cancer treatment using drugs that modify the communication between neurons and cancer stem cells. The researchers hope this opens unforeseen paths toward effective therapies against cancer.

The combination of advanced molecular and imaging tools and organ-on-a-chip technology offers an opportunity to reveal the hidden functions of neurons and their interactions with various stem cell types, in both healthy and pathological conditions.

Reference

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Global Hematopoietic Stem Cells Transplantation (HSCT) Market 2020-2024 | Evolving Opportunities with AllCells Corp. and bluebird bio Inc. | Technavio…

LONDON--(BUSINESS WIRE)--The global hematopoietic stem cells transplantation (HSCT) market is poised to grow by USD 4.64 billion during 2020-2024, progressing at a CAGR of about 6% during the forecast period. Request free sample pages

Read the 120-page report with TOC on "Hematopoietic Stem Cells Transplantation (HSCT) Market Analysis Report by Type (Autologous HSCT and Allogeneic HSCT), Geographic segmentation (Asia, Europe, North America, and ROW), and the Segment Forecasts, 2020-2024".

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The market is driven by the availability of technologically advanced equipment. In addition, the growing demand for personalized medicine is anticipated to boost the growth of the hematopoietic stem cells transplantation (HSCT) market.

The increasing efforts on improving the success rates of HSCT procedure is encouraging the adoption of advanced technologies and sophisticated instruments. For instance, Marrow Cellution, a patented technology, was developed by Ranfac. This technology is gaining traction in the market as it addresses the issues associated with conventional transplant syringe. It prevents contamination of peripheral blood during transplantation. In addition, this technology allows high-quality stem and progenitor cells to harvest within a narrow space. Thus, the availability of technologically advanced equipment is expected to drive market growth during the forecast period.

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Major Five Hematopoietic Stem Cells Transplantation (HSCT) Market Companies:

AllCells Corp.

AllCells Corp. provides mobilized leukopak, bone marrow and cord blood, whole blood, and leukopak. They also offer Cord Blood, which are collected from a single umbilical cord. They are cryopreserved and offered in the format of isolated cells. Cord Blood products include progenitor cells and CD34 HSCs.

bluebird bio Inc.

bluebird bio Inc. focuses on research, development, and commercialization of potentially transformative gene therapies. The company provides Zynteglo, which is a medicine used to treat patients of 12 years and older who are affected by a blood disorder known as beta thalassaemia.

FUJIFILM Holdings Corp.

FUJIFILM Holdings Corp. operates under various business segments, namely Imaging solutions, Healthcare and material solutions, and Document solutions. The company offers iCell Hematopoietic Progenitor Cells 2.0.

Lineage Cell Therapeutics Inc.

Lineage Cell Therapeutics Inc. focuses on research and development of therapeutic products for blood plasma volume expansion, orthopedics, oncology, diagnostic products for the early detection of cancer, neurological diseases and disorders, and more. The company provides OpRegen, which is in Phase I/IIa clinical trial.

Lonza Group Ltd.

Lonza Group Ltd. offers products through the following business segments: Pharma Biotech and Nutrition and Specialty ingredients. The company offers a wide range of stem cells such as bone marrow stromal cells, human peripheral blood CD14+ monocytes, cryopreseverd human CD34+ cells isolated from single donor, osteoclast precusor, and more.

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Hematopoietic Stem Cells Transplantation (HSCT) Market Type Outlook (Revenue, USD Billion, 2020-2024)

Hematopoietic Stem Cells Transplantation (HSCT) Market Geographic Outlook (Revenue, USD Billion, 2020-2024)

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Related Reports on Health Care Include:

Allogeneic Stem Cells Market Global Allogeneic Stem Cells Market by geography (Asia, Europe, North America, and ROW) and application (regenerative therapy and drug discovery and development).

Cancer Stem Cell Therapeutics Market Global Cancer Stem Cell Therapeutics Market by type (allogeneic stem cell transplant and autologous stem cell transplant) and geography (Asia, Europe, North America, and ROW).

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Global Hematopoietic Stem Cells Transplantation (HSCT) Market 2020-2024 | Evolving Opportunities with AllCells Corp. and bluebird bio Inc. | Technavio...

Recommendation and review posted by Bethany Smith

Australians living with an incurable blood cancer set to receive new reimbursed treatment option – Mirage News

VIVA! Communications

Australians living with an incurable blood cancer set to receive new reimbursed treatment option

Australians living with the incurable blood cancer, multiple myeloma1 are set to gain access to a new treatment option, with the listing of REVLIMID(lenalidomide) for maintenance treatment on the Pharmaceutical Benefits Scheme (PBS) from Wednesday April 1, 2020.2

REVLIMID(lenalidomide) represents Australias first and only maintenance treatment specifically indicated and reimbursed for those newly diagnosed with multiple myeloma (NDMM) who have undergone an autologous stem cell transplant (ASCT). 2, 3

This announcement coincides with an article just published in Medical Journal of Australias MJA Insight, calling for timely access to effective treatments for those living with multiple myeloma.4

According to article author, Professor Miles Prince, Clinical Haematologist and Director of Cancer Immunology and Molecular Oncology (Epworth Healthcare, Melbourne), continuing to broaden access to multiple myeloma treatments is critical to improving patient quality of life.4

Currently, people living with myeloma have a median survival rate of more than seven years which is significant in comparison to the median survival rate of just three years in the early 2000s.4

For survival rates to continue to improve however, patients must receive timely access to the most effective treatments, said Prof. Prince.

The PBS listing of maintenance for multiple myeloma will provide newly diagnosed patients with an additional treatment option for their disease.

Not to be confused with the skin cancer, melanoma, multiple myeloma is an incurable blood cancer that develops from plasma cells, a type of white blood cell found in the bone marrow.5, 6

Representing Australias third most common blood cancer (after lymphoma and leukaemia),7, 8 approximately 18,000 Australians are living with multiple myeloma at any given time,1 only half of whom will survive five years post- diagnosis.9

Myeloma Australia CEO, Steve Roach, today welcomed the availability of a new treatment option for the incurable disease.

The multiple myeloma patient journey involves a pattern of Response, Remission and Relapse, with individuals responding differently to certain treatments due to the complex nature of the devastating disease.

Additional treatment options are required throughout the patient journey, for both the newly diagnosed, and those who have already commenced therapy. Although incurable, we hope that multiple myeloma will one day be treated as a chronic, rather than a terminal disease, Mr Roach said.

The incurable nature of the disease and the likelihood of relapse, may have a psychological impact on patients, who can continue to live in fear even during periods of remission.10, 11

With studies revealing more than half (52%) of those living with multiple myeloma experience symptoms of anxiety or depression,12 improving access to treatment, and extending time spent in remission, may help to improve psychological wellbeing.

Wife and mother-to-two, Maria (53) was diagnosed with multiple myeloma in December 2018 and found her initial diagnosis very overwhelming.

When my husband Danny and I first heard the diagnosis, we were completely overwhelmed. We didnt know what Myeloma was and we didnt know what this meant short or long term. I didnt know if I was going to die in a month, a year or 10 years. How on earth was I going to tell everyone I had cancer, said Maria.

During my journey I blogged about my experience with multiple myeloma and posted to my Facebook daily to keep the calls and fears of my family and friends at bay. I have since accepted that myeloma is now a part of my life. I have no anger or fear and instead just live in the moment and take one day at a time.

Its very exciting to see new treatment options for multiple myeloma being funded by the government, and I hope to keep raising awareness, to ensure the myeloma community continues to receive access to the best treatment options available, Maria said.

About multiple myeloma

Multiple myeloma is a cancer that develops from abnormal plasma cells. A plasma cell is a type of white blood cell found in the bone marrow, that forms part of the immune system and helps to protect against infection.4, 7 The abnormal plasma cells crowd the bone marrow and make it difficult to produce enough normal blood cells.7

Multiple myeloma can be challenging to diagnose due to its wide range of symptoms, including high blood calcium levels, anaemia, fatigue, kidney failure, recurrent infections and bone pain.6

Current treatment for multiple myeloma includes a continuous approach, often comprising initial therapy, consolidation maintenance, and salvage therapy.13 Treatment options for multiple myeloma include chemotherapy, corticosteroids, autologous stem cell transplant (ASCT), immunomodulating drugs (IMiDs) monoclonal antibodies therapy, and proteasome inhibitors.14, 15

About REVLIMID (lenalidomide)

Representing an oral medication approved for the treatment of relapsed myeloma over 10 years ago,16 REVLIMID is an immunomodulating agent (IMiD) that slows the growth of multiple myeloma plasma tumour cells and proteins know to play a key role multiple myeloma, delays the development of new blood vessels, and enhances immune function.3

The most commonly reported side-effects of REVLIMID include diarrhoea, constipation, nausea, vomiting, stomach pain, indigestion, dehydration, dry mouth, mouth ulcer, sore mouth, increase or decrease in weight, increase or decrease in appetite, loss of taste, itchiness, rash, redness of the skin, dry skin, bruising, excessive sweating, dizziness, fainting, headache, shaking or tremors, unusual weakness, night sweats, reduced sense of touch, difficulty sleeping, depression, anxiety, feeling of confusion, back pain, muscle spasms, muscle and/or joint pain, swollen joints, bone pain, muscular weakness, pain in the extremities, feeling tired, falling, swelling of hands, ankles or feet.17

Other possible side effects of REVLIMID include heart palpitations or fast heartbeat, chest pains, dizziness or fainting, shortness of breath, weakness or reduced ability to exercise, bleeding (including nose-bleeds), bruising more than usual, numbness, tingling, blurred vision or difficulty seeing, passing large amounts of urine, excessive thirst, and having a dry mouth and skin, abnormal eye movements, convulsions, mood changes, irregular heart rhythms or tender swollen lymph nodes. 17

All patients receiving REVLIMID must be registered on, and abide by the requirements of, i-access risk management program to avoid exposure to unborn babies, due to the potential for birth defects. It is important to note that a small number of patients with multiple myeloma may develop additional types of cancer, regardless of their type of therapy. At this stage, it cannot be excluded that this risk may be slightly increased with REVLIMID maintenance treatment.17

Disclosure

Celgene supports disclosure and transparency on interactions between the healthcare industry and healthcare professionals to ensure public trust and confidence. No expert spokespeople have been offered compensation for their involvement in this media campaign. All expert spokespeople have been briefed on the approved use of these products and their obligations with regard to promotion to the general public. Prof Miles Prince and Myeloma Australia have received funding from Celgene for projects unrelated to this announcement. All opinions expressed are their own.

About Celgene

Celgene, a Bristol-Myers Squibb Company, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialisation of innovative therapies for the treatment of cancer and inflammatory diseases, through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation.

/Public Release.

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Australians living with an incurable blood cancer set to receive new reimbursed treatment option - Mirage News

Recommendation and review posted by Bethany Smith

Aromatase excess syndrome – Genetics Home Reference – NIH

Fukami M, Shozu M, Ogata T. Molecular bases and phenotypic determinants of aromatase excess syndrome. Int J Endocrinol. 2012;2012:584807. doi: 10.1155/2012/584807. Epub 2012 Jan 26.

Fukami M, Shozu M, Soneda S, Kato F, Inagaki A, Takagi H, Hanaki K, Kanzaki S, Ohyama K, Sano T, Nishigaki T, Yokoya S, Binder G, Horikawa R, Ogata T. Aromatase excess syndrome: identification of cryptic duplications and deletions leading to gain of function of CYP19A1 and assessment of phenotypic determinants. J Clin Endocrinol Metab. 2011 Jun;96(6):E1035-43. doi: 10.1210/jc.2011-0145. Epub 2011 Apr 6.

Fukami M, Tsuchiya T, Vollbach H, Brown KA, Abe S, Ohtsu S, Wabitsch M, Burger H, Simpson ER, Umezawa A, Shihara D, Nakabayashi K, Bulun SE, Shozu M, Ogata T. Genomic basis of aromatase excess syndrome: recombination- and replication-mediated rearrangements leading to CYP19A1 overexpression. J Clin Endocrinol Metab. 2013 Dec;98(12):E2013-21. doi: 10.1210/jc.2013-2520. Epub 2013 Sep 24.

Shihara D, Miyado M, Nakabayashi K, Shozu M, Ogata T, Nagasaki K, Fukami M. Aromatase excess syndrome in a family with upstream deletion of CYP19A1. Clin Endocrinol (Oxf). 2014 Aug;81(2):314-6. doi: 10.1111/cen.12329. Epub 2013 Oct 18.

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Aromatase excess syndrome - Genetics Home Reference - NIH

Recommendation and review posted by Bethany Smith

Juvenile whio ducks take to the water for first time on a South Taranaki river – Stuff.co.nz

SUPPLIED

In being released into a South Taranaki river, whio Tipunakore, Trev and Tui made history.

It was a low key event for three juvenile whio blue ducks released to the Kapuni Stream recently - the first release of whio ona South Taranaki river in Egmont National Park.

A previous event with Te Korowai o Ngruahine, Supporting Today's At Risk Teenagers (START) Taranaki, and whio support groupswas cancelled due to Covid-19 risk.

Two male and one female whio were bred at Ng Manu Nature Reserve in Waikanae and taken to a 'hardening' facility at the Tongariro Trout Centre where thebirds provided new genetics to the current population.

The birds join a record number of whio ducklings found by Department of Conservation rangers on eight regularly surveyed rivers.

READ MORE:Whio population boosted by release of three more ducks on Mt Taranaki

The upcoming five yearly whio census scheduled in 2020would determine if overall numbers have increased since 2015.

There are now estimated to be more than 80 whio in the national park.

The released whio - Tipunakore, Trev and Tui - are named after two Taranaki START students now working with the Taranaki Mounga Project, and a supporter of the programme.

Taranaki Mounga project manager Sean Zieltjes saidTrev, one of the students, hadworked with the project for nearly a year.

He wasstokeda whio is named after him, Zieltjes said.

"Trev worked incredibly hard to help protect these taonga.

"Hebuilt over 300 DOC200 stoat traps in his neighbour's carport, and also helped to cut the tracks were these traps are all active on the southern side of the maunga.

"Thanks to Trevhe's protecting whio and giving them the best chance to thrive."

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Juvenile whio ducks take to the water for first time on a South Taranaki river - Stuff.co.nz

Recommendation and review posted by Bethany Smith

CRISPR Therapeutics braces for ‘severe impact’ from COVID-19 crisis – FierceBiotech

It was supposed to be an exciting time of next-gen science, where CRISPR gene editing was being used in the first series of human trials in the hope this tech could help cure a range of diseases.

But one of its proponents, CRISPR Therapeutics, has, alongside a growing number of biopharmas, admitted the spreading COVID-19 pandemic is starting to see it be adversely affected.

In a Securities and Exchange Commission filing Tuesday, the biotech said: We are conducting a number of clinical trials for product candidates in the fields of severe hemoglobinopathies and immuno-oncology in geographies which are affected by the coronavirus pandemic.

We believe that the coronavirus pandemic has had, and will likely continue to have, an impact on various aspects of our clinical trials. For example, with respect to our CTX001 clinical trials for severe hemoglobinopathies (specifically, transfusion-dependent beta thalassemia and severe sickle cell disease), since[intensive care unit] beds and related healthcare resources are anticipated to become significantly constrained in light of the coronavirus pandemic, no additional patients are currently scheduled to begin dosing in either study at this time.

CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from beta thalassemia and sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. This is currently in a long-term follow-up trial after posting some encouraging, though early, results in several patients late last year.

Looking at its cancer trials, it also added that investigators will likely not want to expose cancer patients to COVID-19 since the dosing of patients is conducted within an in-patient setting, which could also hit it tests.

RELATED: Pandemic sees one-quarter of investigators halting trial enrollment: poll

Its leading immuno-oncology cell therapy programs, CTX110 and CTX120, are using an allogenic approach to tumor targets. CTX110 targets CD19, an antigen expressed in various B-cell malignancies, while CTX120 targets BCMA, an antigen expressed in multiple myeloma.

Its third candidate, CTX130, is focused on solid tumorsand targets CD70, an antigen expressed on both hematologic cancers, including certain lymphomas, as well as solid tumors including renal cell carcinoma.

CTX110 started an early safety trial last summer, and the biotech said in its financial report back in February that it was still enrolling patients to assess the safety and efficacy of CTX120. Meanwhile, CTX130 is yet to enter the clinic.

The biotech has already closed its offices and asked that most of its staffers work remotely. It said it has restricted on-site staff to only those personnel and contractors who must perform essential activities that must be completed on-site and limited the number of staff in any given research and development laboratory.

But this comes with its own issues: Our increased reliance on personnel working from home may negatively impact productivity, or disrupt, delay, or otherwise adversely impact our business. In addition, this could increase our cyber security risk, create data accessibility concerns, and make us more susceptible to communication disruptions, any of which could adversely impact our business operations or delay necessary interactions with local and federal regulators, ethics committees, manufacturing sites, research or clinical trial sites and other important agencies and contractors.

It also said that, given that its labs are not easily accessible and talking to regulators is getting tough, there could be a delay for the timely completion of preclinical activities, including IND-enabling studies as well as its ability to select future development candidates and initiation of additional clinical trials for otherdevelopment programs.

Finances for the biotech may be hit, too.The trading prices for our common shares and other biopharmaceutical companies have been highly volatile as a result of the coronavirus pandemic, the company said. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms.

The biotech has seen its shares yo-yo over the past month as COVID-19 ramped updown from more than $53 a share at the start of Marchto less than $43 just two weeks laterand ended the month at around $42.

In short, CRISPR is braced for the worst but does not know how deeply this could cut into its trial and business. We do not yet know the full extent of potential delays or impacts on our business, our clinical trials, our research programs, healthcare systems or the global economy. We will continue to monitor the situation closely.

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CRISPR Therapeutics braces for 'severe impact' from COVID-19 crisis - FierceBiotech

Recommendation and review posted by Bethany Smith

Scientists expanded the Capabilities of CRISPR gene editing technique – Tech Explorist

CRISPR-Cas9, which is short for clustered, regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The technique is faster, cheaper, more accurate, and more efficient than other existing genome editing methods.

For the CRISPR-Cas9 system to work, a bacterial defense protein got Cas9 seeks out an adjacent protospacer motif (PAM) that is present in the viral DNA yet not in the bacterial DNA. CRISPR-Cas9 has been harnessed for editing the human genome because such PAM sequences are also quite common in our DNA; however, genes that are not near a PAM cannot be targeted.

To conquer this problem, a team led by Benjamin P. Kleinstiver, a biochemist at MGHs Center for Genomic Medicine, engineered variations of a Cas9 protein that dont require a particular PAM to bind and cut DNA. The two new Cas9 variations, named SpG and SpRY, allow editing of DNA sequences at efficiencies not achievable with conventional CRISPR-Cas9 enzymes.

As engineered proteins target independently, they enable targeting of previously inaccessible regions of the genome.

Benjamin P. Kleinstiver, a biochemist at MGHs Center for Genomic Medicine, said,By nearly completely relaxing the requirement for the enzymes to recognize a PAM, many genome editing applications are now possible. And since almost the entire genome is targetable, one of the most exciting implications is that that the entire genome is druggable from a DNA-editing perspective.

Scientists are further planning to comprehend the function of these proteins. They also want to explore their unique capabilities for a variety of different applications.

Lead author Russell T. Walton, also of MGHs Center for Genomic Medicine, said,We have demonstrated that these new enzymes will allow researchers to generate biologically and clinically relevant genetic modifications that were previously unfeasible.

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Scientists expanded the Capabilities of CRISPR gene editing technique - Tech Explorist

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Modalis Obtains Access to Foundational CRISPR IP – Business Wire

TOKYO & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Modalis Therapeutics Corporation (Modalis) today announced that the company has entered into a license agreement with Editas Medicine, Inc., under which Modalis has obtained a license to certain intellectual property that is controlled by Editas Medicine. Modalis is utilizing its proprietary epigenetic gene modulation technology, CRISPR-GNDM (Guide Nucleotide Directed Modulation), to treat patients with serious genetic disorders. Additional details including financial terms of the agreement were not disclosed.

"Our goal is to create CRISPR based gene therapies for genetic disorders, most of which fall into the orphan disease category. There should be no disease that is ignored because of its small patient population, and our mission to develop disease modifying treatments for these diseases reflects our belief that Every Life Deserves Attention. We are proud to be the pioneer in CRISPR based gene modulation therapy, said Haru Morita, Chief Executive Officer of Modalis.

We are pleased to establish this license agreement with Modalis Therapeutics as their mission is aligned with our mission to make transformative medicines for people living with serious diseases of unmet clinical need. CRISPR technology has many uses and applications, and we are pleased to include Modalis in our expanding portfolio of licensees so the greatest number of patients may benefit in the future from transformative medicines, said Cynthia Collins, president and chief executive officer, Editas Medicine.

About Modalis

Modalis Therapeutics is developing precision genetic medicines through epigenetic gene modulation. Founded by Osamu Nureki and leading scientists in CRISPR gene editing from University of Tokyo, Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is focusing initially on genetic disorders caused by loss of gene regulation resulting in excess or insufficient protein production which includes more than 660 genes that are currently estimated to cause human disease due to haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Cambridge, Massachusetts, the company is backed by leading Japanese investors including Fast Track Initiative, SBI Investment, UTokyo-IPC, SMBC Venture Capital, and Mizuho Capital. For additional information, visit http://www.modalistx.com.

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Modalis Obtains Access to Foundational CRISPR IP - Business Wire

Recommendation and review posted by Bethany Smith

Movie review: Doc Human Nature strikes a nerve in the age of coronavirus – The Patriot Ledger

Documentary "Human Nature" examines how gene editing can help - and hurt - humanity.

If youre familiar with the Replicates from Blade Runner, the velociraptors from Jurassic Park or the genetic engineering so chillingly laid out in Aldous Huxleys novel Brave New World, youll be fascinated by how much science fiction has become science fact in Adam Bolts Human Nature. And its all due to CRISPR (pronounced crisper), a gene-altering technology that not only could facilitate designer babies, but possibly play a central role in putting the clamps on another acronym, COVID-19.

That timeliness is obviously on the side of Human Nature, a snazzy-looking documentary using sparkling graphics and top geneticists, journalists and one very adorable sickle-cell anemic to spell out a complicated subject in compelling, easy-to-grasp terms. But that same timeliness also works against it, given how now is not an advantageous moment for the films commercial aspects amid a landscape of shuttered theaters and a frightened populace whod like to avoid anything to do with medicine and science as sources of entertainment.

Yet, that double-edged sword fits snuggly in the wheelhouse of CRISPR (short for clustered regularly interspaced short palindromic repeats), a microorganism able to locate and repair defective DNA, as well as fend off invading viruses like COVID-19 by acting as a defense shield mimicking the offenders own DNA. But like the Internet, a revolutionary breakthrough for which CRISPR is often compared, theres a serious downside involving the morality of whether humans should have the right to, as the film calls it, play God. Namely, should parents be allowed to treat an embryo the same way theyd approach ordering a pizza? Well have the regular with blue eyes, blonde hair and an IQ of Einstein. Oh, and could you throw in some immense athletic ability, too?

Clearly, CRISPR has the potential to put us at the mercy of the type of mad scientists weve become accustomed to in just about every Bond film ever made. One geneticist, whose very name, Jennifer Doudna, includes DNA, admits having had a nightmare in which she comes face-to-face with Adolf Hitler! Are we willing to toy with the very real prospect of creating a master race?

Thats just one of the troubling questions Bolt confronts you with while weighing the pros and cons of a new frontier brimming in possibilities and danger. Personally, I come down on the side of CRISPRs benefits, particularly after meeting David Sanchez, a teen with sickle cell thats spent about half of his young life in hospitals receiving precious blood transfusions. Hes smart, personable and amazingly brave, so much so, you cant help but be all in when CRISPR offers him a chance at a more normal life. Yet, hes just as quick to recall to how hes learned to embrace -- even appreciate -- his illness because its made him a better, more resourceful kid, insights he would not have acquired had CRISPR been available when he was in utero. See? Hes torn, too.

Do we embrace a discovery wielding the promise of curing and preventing cancers and birth defects, or shun it for its ability to rob us of our unique individuality? Its a compelling argument I frankly wish Bolt had expanded more upon in his movies all-too-brief 90 minutes. But whats here is more than enough to spark a multitude of kitchen-table conversations about where we should set the limits on science, and more importantly, who should be making those decisions.

Given the disarray COVID-19 has put the world in, now probably isnt the time for us to evaluate, especially when CRISPR could well determine our fate by ridding our planet of a crippling plague. But what about after? Will, as Trump is fond to say, the cure be worse than the disease? Its a question for which Human Nature holds no answers, only utopian and despotic possibilities well be forced to uneasily choose between when and if the time comes.

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Movie review: Doc Human Nature strikes a nerve in the age of coronavirus - The Patriot Ledger

Recommendation and review posted by Bethany Smith

Hedge Funds Have Never Been This Bullish On CRISPR Therapeutics AG (CRSP) – Yahoo Finance

We hate to say this but, we told you so. On February 27th we published an article with the title Recession is Imminent: We Need A Travel Ban NOW and predicted a US recession when the S&P 500 Index was trading at the 3150 level. We also told you to short the market and buy long-term Treasury bonds. Our article also called for a total international travel ban. While we were warning you, President Trump minimized the threat and failed to act promptly. As a result of his inaction, we will now experience a deeper recession (see why hell is coming).

In these volatile markets we scrutinize hedge fund filings to get a reading on which direction each stock might be going. The latest 13F reporting period has come and gone, and Insider Monkey is again at the forefront when it comes to making use of this gold mine of data. Insider Monkey finished processing 835 13F filings submitted by hedge funds and prominent investors. These filings show these funds' portfolio positions as of December 31st, 2019. In this article we are going to take a look at smart money sentiment towards CRISPR Therapeutics AG (NASDAQ:CRSP).

Is CRISPR Therapeutics AG (NASDAQ:CRSP) the right investment to pursue these days? The best stock pickers are becoming hopeful. The number of long hedge fund bets improved by 13 recently. Our calculations also showed that CRSP isn't among the 30 most popular stocks among hedge funds (click for Q4 rankings and see the video at the end of this article for Q3 rankings).

In the financial world there are a large number of tools investors have at their disposal to grade stocks. A pair of the most under-the-radar tools are hedge fund and insider trading indicators. We have shown that, historically, those who follow the top picks of the best fund managers can outperform the broader indices by a solid amount. Insider Monkey's monthly stock picks returned 72.9% since March 2017 and outperformed the S&P 500 ETFs by more than 41 percentage points. Our short strategy outperformed the S&P 500 short ETFs by 20 percentage points annually (see the details here). That's why we believe hedge fund sentiment is a useful indicator that investors should pay attention to.

Samuel Isaly of OrbiMed Advisors

Samuel Isaly Orbimed Advisors

We leave no stone unturned when looking for the next great investment idea. For example we recently identified a stock that trades 25% below the net cash on its balance sheet. We read hedge fund investor letters and listen to stock pitches at hedge fund conferences, and go through short-term trade recommendations likethis one. We even check out the recommendations of services with hard to believe track records. Our best call in 2020 was shorting the market when S&P 500 was trading at 3150 after realizing the coronavirus pandemic's significance before most investors. Now let's take a gander at the new hedge fund action encompassing CRISPR Therapeutics AG (NASDAQ:CRSP).

At Q4's end, a total of 30 of the hedge funds tracked by Insider Monkey held long positions in this stock, a change of 76% from the third quarter of 2019. By comparison, 11 hedge funds held shares or bullish call options in CRSP a year ago. With hedgies' capital changing hands, there exists an "upper tier" of noteworthy hedge fund managers who were upping their stakes significantly (or already accumulated large positions).

Is CRSP A Good Stock To Buy?

Story continues

Of the funds tracked by Insider Monkey, Cormorant Asset Management, managed by Bihua Chen, holds the number one position in CRISPR Therapeutics AG (NASDAQ:CRSP). Cormorant Asset Management has a $60.9 million position in the stock, comprising 2.4% of its 13F portfolio. Coming in second is OrbiMed Advisors, managed by Samuel Isaly, which holds a $46.7 million position; the fund has 0.7% of its 13F portfolio invested in the stock. Some other hedge funds and institutional investors that hold long positions contain Charles Clough's Clough Capital Partners, Renaissance Technologies and Farallon Capital. In terms of the portfolio weights assigned to each position Cormorant Asset Management allocated the biggest weight to CRISPR Therapeutics AG (NASDAQ:CRSP), around 2.43% of its 13F portfolio. Clough Capital Partners is also relatively very bullish on the stock, earmarking 2.31 percent of its 13F equity portfolio to CRSP.

As aggregate interest increased, some big names have jumped into CRISPR Therapeutics AG (NASDAQ:CRSP) headfirst. Citadel Investment Group, managed by Ken Griffin, established the largest position in CRISPR Therapeutics AG (NASDAQ:CRSP). Citadel Investment Group had $12.6 million invested in the company at the end of the quarter. Peter Rathjens, Bruce Clarke and John Campbell's Arrowstreet Capital also initiated a $6.3 million position during the quarter. The other funds with new positions in the stock are Benjamin A. Smith's Laurion Capital Management, Principal Global Investors's Columbus Circle Investors, and David Harding's Winton Capital Management.

Let's check out hedge fund activity in other stocks - not necessarily in the same industry as CRISPR Therapeutics AG (NASDAQ:CRSP) but similarly valued. These stocks are Lithia Motors Inc (NYSE:LAD), Cimpress NV (NASDAQ:CMPR), Equitrans Midstream Corporation (NYSE:ETRN), and Graham Holdings Co (NYSE:GHC). This group of stocks' market values are similar to CRSP's market value.

[table] Ticker, No of HFs with positions, Total Value of HF Positions (x1000), Change in HF Position LAD,29,661944,3 CMPR,18,707978,6 ETRN,15,379764,-2 GHC,18,510146,0 Average,20,564958,1.75 [/table]

View table hereif you experience formatting issues.

As you can see these stocks had an average of 20 hedge funds with bullish positions and the average amount invested in these stocks was $565 million. That figure was $298 million in CRSP's case. Lithia Motors Inc (NYSE:LAD) is the most popular stock in this table. On the other hand Equitrans Midstream Corporation (NYSE:ETRN) is the least popular one with only 15 bullish hedge fund positions. Compared to these stocks CRISPR Therapeutics AG (NASDAQ:CRSP) is more popular among hedge funds. Our calculations showed that top 20 most popular stocks among hedge funds returned 41.3% in 2019 and outperformed the S&P 500 ETF (SPY) by 10.1 percentage points. These stocks lost 17.4% in 2020 through March 25th and still beat the market by 5.5 percentage points. Unfortunately CRSP wasn't nearly as popular as these 20 stocks and hedge funds that were betting on CRSP were disappointed as the stock returned -31.4% during the first two and a half months of 2020 (through March 25th) and underperformed the market. If you are interested in investing in large cap stocks with huge upside potential, you should check out the top 20 most popular stocks among hedge funds as most of these stocks already outperformed the market in Q1. Video: Click the image to watch our video about the top 5 most popular hedge fund stocks.

5 Most Popular Stocks Among Hedge Funds

Disclosure: None. This article was originally published at Insider Monkey.

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Hedge Funds Have Never Been This Bullish On CRISPR Therapeutics AG (CRSP) - Yahoo Finance

Recommendation and review posted by Bethany Smith

Oppenheimer Maintains Their Buy Rating on Crispr Therapeutics AG (CRSP) – Smarter Analyst

Oppenheimer analyst Silvan Tuerkcan maintained a Buy rating on Crispr Therapeutics AG (CRSP) yesterday and set a price target of $80.00. The companys shares closed last Tuesday at $42.41.

According to TipRanks.com, Tuerkcan is a 4-star analyst with an average return of 15.6% and a 37.5% success rate. Tuerkcan covers the Healthcare sector, focusing on stocks such as Constellation Pharmaceuticals, Intellia Therapeutics, and Seattle Genetics.

The word on The Street in general, suggests a Moderate Buy analyst consensus rating for Crispr Therapeutics AG with a $71.68 average price target.

See todays analyst top recommended stocks >>

Crispr Therapeutics AGs market cap is currently $2.69B and has a P/E ratio of 42.70. The company has a Price to Book ratio of 2.71.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

CRISPR Therapeutics AG engages in the development and commercialization of therapies derived from genome-editing technology. Its proprietary platform CRISPR/Cas9-based therapeutics allows for precise and directed changes to genomic DNA. The company was founded by Rodger Novak, Emmanuelle Charpentier, Shaun Patrick Foy, Matthew Porteus, Daniel Anderson, Chad Cowan and Craig Mellow in 2014 and is headquartered in Zug, Switzerland.

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Oppenheimer Maintains Their Buy Rating on Crispr Therapeutics AG (CRSP) - Smarter Analyst

Recommendation and review posted by Bethany Smith

Crispr Therapeutics AG (CRSP) Gets a Buy Rating from Canaccord Genuity – Smarter Analyst

Canaccord Genuity analyst Arlinda Lee reiterated a Buy rating on Crispr Therapeutics AG (CRSP) today and set a price target of $80.00. The companys shares closed last Tuesday at $42.41.

According to TipRanks.com, Lee is a 3-star analyst with an average return of 0.1% and a 43.0% success rate. Lee covers the Healthcare sector, focusing on stocks such as Turning Point Therapeutics, Black Diamond Therapeutics, and Karyopharm Therapeutics.

Crispr Therapeutics AG has an analyst consensus of Moderate Buy, with a price target consensus of $71.68, representing a 62.2% upside. In a report released yesterday, Oppenheimer also maintained a Buy rating on the stock with a $80.00 price target.

See todays analyst top recommended stocks >>

Based on Crispr Therapeutics AGs latest earnings release for the quarter ending December 31, the company reported a quarterly revenue of $77.02 million and net profit of $30.54 million. In comparison, last year the company earned revenue of $115K and had a GAAP net loss of $47.59 million.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

CRISPR Therapeutics AG engages in the development and commercialization of therapies derived from genome-editing technology. Its proprietary platform CRISPR/Cas9-based therapeutics allows for precise and directed changes to genomic DNA. The company was founded by Rodger Novak, Emmanuelle Charpentier, Shaun Patrick Foy, Matthew Porteus, Daniel Anderson, Chad Cowan and Craig Mellow in 2014 and is headquartered in Zug, Switzerland.

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Crispr Therapeutics AG (CRSP) Gets a Buy Rating from Canaccord Genuity - Smarter Analyst

Recommendation and review posted by Bethany Smith

First Week of May 8th Options Trading For CRISPR Therapeutics (CRSP) – Nasdaq

Investors in CRISPR Therapeutics AG (Symbol: CRSP) saw new options become available this week, for the May 8th expiration. At Stock Options Channel, our YieldBoost formula has looked up and down the CRSP options chain for the new May 8th contracts and identified one put and one call contract of particular interest.

The put contract at the $41.00 strike price has a current bid of $3.30. If an investor was to sell-to-open that put contract, they are committing to purchase the stock at $41.00, but will also collect the premium, putting the cost basis of the shares at $37.70 (before broker commissions). To an investor already interested in purchasing shares of CRSP, that could represent an attractive alternative to paying $42.60/share today.

Because the $41.00 strike represents an approximate 4% discount to the current trading price of the stock (in other words it is out-of-the-money by that percentage), there is also the possibility that the put contract would expire worthless. The current analytical data (including greeks and implied greeks) suggest the current odds of that happening are 62%. Stock Options Channel will track those odds over time to see how they change, publishing a chart of those numbers on our website under the contract detail page for this contract. Should the contract expire worthless, the premium would represent a 8.05% return on the cash commitment, or 75.33% annualized at Stock Options Channel we call this the YieldBoost.

Below is a chart showing the trailing twelve month trading history for CRISPR Therapeutics AG, and highlighting in green where the $41.00 strike is located relative to that history:

Turning to the calls side of the option chain, the call contract at the $43.00 strike price has a current bid of $3.30. If an investor was to purchase shares of CRSP stock at the current price level of $42.60/share, and then sell-to-open that call contract as a "covered call," they are committing to sell the stock at $43.00. Considering the call seller will also collect the premium, that would drive a total return (excluding dividends, if any) of 8.69% if the stock gets called away at the May 8th expiration (before broker commissions). Of course, a lot of upside could potentially be left on the table if CRSP shares really soar, which is why looking at the trailing twelve month trading history for CRISPR Therapeutics AG, as well as studying the business fundamentals becomes important. Below is a chart showing CRSP's trailing twelve month trading history, with the $43.00 strike highlighted in red:

Considering the fact that the $43.00 strike represents an approximate 1% premium to the current trading price of the stock (in other words it is out-of-the-money by that percentage), there is also the possibility that the covered call contract would expire worthless, in which case the investor would keep both their shares of stock and the premium collected. The current analytical data (including greeks and implied greeks) suggest the current odds of that happening are 48%. On our website under the contract detail page for this contract, Stock Options Channel will track those odds over time to see how they change and publish a chart of those numbers (the trading history of the option contract will also be charted). Should the covered call contract expire worthless, the premium would represent a 7.75% boost of extra return to the investor, or 72.50% annualized, which we refer to as the YieldBoost.

The implied volatility in the put contract example is 141%, while the implied volatility in the call contract example is 135%.

Meanwhile, we calculate the actual trailing twelve month volatility (considering the last 251 trading day closing values as well as today's price of $42.60) to be 61%. For more put and call options contract ideas worth looking at, visit StockOptionsChannel.com.

Top YieldBoost Calls of the S&P 500

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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First Week of May 8th Options Trading For CRISPR Therapeutics (CRSP) - Nasdaq

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A Point-of-Care CRISPR-based COVID-19 Diagnostic on the Horizon – Technology Networks

As part of ongoing efforts to try to reduce the spread of COVID-19, researchers around the world are working hard to develop novel diagnostic technologies. Rapid and simple identification of people infected with SARS-CoV-2 plays a crucial role in reducing transmission of the virus, by enabling appropriate isolation measures and contact tracing to take place.The lab of Dr Changchun Liu at the University of Connecticut Health Center has recently reported progress in this area, with the development of a simple, low-cost, CRISPR-based method which can detect infectious diseases such as COVID-19.1 Technology Networks spoke to Dr Liu to learn more about the method, the advantages it offers over PCR-based methods of detection, and the next steps to be taken before a point-of-care diagnostic test using this method could become widely available.Anna MacDonald (AM): Many of the current diagnostic tests for infectious diseases such as COVID-19 are PCR-based. Why is this method currently the gold standard and what are some of the limitations associated with it?

Changchun Liu (CL): PCR/RT-PCR, particularly real-time PCR/RT-PCR, provides a highly sensitive and specific method for detection of infectious diseases (e.g., COVID-19). In addition, PCR/RT-PCR-based methods can enable early diagnosis of COVID-19 because it detects nucleic acid (e.g., RNA) of novel coronavirus SARS-CoV-2. PCR/RT-PCR-based methods are typically restricted in a centralized clinical laboratory due to the requirement for sophisticated equipment and well-trained personnel, which are not suitable for simple, rapid, point of care diagnostic applications. AM: What advantages could CRISPR-based detectionoffer? What is limiting greater use of these methods for the detection of infectious diseases so far? CL: CRISPR-based nucleic acid detection provides a highly sensitive, specific and reliable testing approach for nucleic acid-based molecular diagnostics. Unlike PCR-based methods, CRISPR-based nucleic acid detection methods can work at a constant temperature (e.g. 37oC) without the need for an expensive thermal cycler (e.g. PCR machine). However, currently available CRISPR-based nucleic acid detection methods typically require: i) separate nucleic acid amplification in different reaction systems, and ii) multiple manual operations, which undoubtedly complicates the testing procedures and potentially increases the risk of carry-over contaminations due to amplification products transferring.

AM: Can you tell us more about the CRISPR-based method you have developed and give us an overview of your studys results? CL: We developed an All-In-One Dual CRISPR-Cas12a (termed "AIOD-CRISPR") assay method for rapid, ultrasensitive, specific and visual detection of nucleic acid. To improve detection sensitivity, we have proposed a dual CRISPR-Cas12a detection strategy. In addition, unlike previous CRISPR-based nucleic acid detections, all reagents for nucleic acid detection in our AIOD-CRISPR assay can be incubated in one-pot, enabling simple, rapid, sensitive and specific nucleic acid detection. Our AIOD-CRISPR assay method has successfully been utilized to detect nucleic acids (DNA and RNA) of the SARS-CoV-2 and HIV with a sensitivity of a few copies. Also, it was evaluated by detecting HIV-1 RNA extracted from human plasma samples, achieving a comparable sensitivity with real-time RT-PCR, but within a shorter time (less than 20 minutes). AM: How does AIOD-CRISPR compare to other CRISPR-based detection methods?

CL: As mentioned above, we developed a dual CRISPR-Cas12a detection strategy to improve detection sensitivity in our AIOD-CRISPR assay. In addition, unlike previously reported CRISPR-based nucleic acid detections, all reagents of our AIOD-CRISPR assay can be incubated in one-pot, eliminating need for multiple manual operations and enabling simple, rapid point of care diagnostics. In summary, our AIOD-CRISPR assay provides a simple, rapid (typically 5-20 minutes), ultrasensitive (few copies) and highly specific method for nucleic acid-based molecular diagnostics at the point-of-care. AM: Why is a one-pot reaction system so important? CL: As mentioned above, previously reported CRISPR-based nucleic acid detections typically require separate nucleic acid amplification and multiple manual operations, which undoubtedly complicates the testing procedures and is not ideal for point of care diagnostics. In our AIOD-CRISPR assay, all components for isothermal amplification and CRISPR-based detection are prepared in a one-pot format, which greatly simplifies the detection procedures and eliminates the risk of carry-over contaminations. Thus, our AIOD-CRISPR assay method has a great potential for developing next-generation point-of-care molecular diagnostics. AM: What are the next steps before a point-of-care diagnostic test using this method could become widely available? CL: We are integrating our AIOD-CRISPR assay into our microfluidic diagnostic chip to develop a simple, rapid, affordable, point-of-care diagnostic platform for SARS-CoV-2 detection at home or small clinics. We have long focused on developing simple, low-cost, point-of-care diagnostic technologies for rapid detection of infectious diseases. For instance, during the 2015-2016 Zika outbreak, we developed an instrument-free point-of-care molecular diagnostic technology for Zika virus detection.2 AM: What difference could a rapid, affordable, point-of-care test such as the one you are developing make to the global response to the COVID-19 pandemic?

CL: Rapid and early detection of the SARS-CoV-2 virus plays a crucial role in facilitating early intervention and treatment (e.g. home isolation, social distancing) and preventing COVID-19 disease spread. We envision that such a simple, rapid, affordable, and point-of-care diagnostics technology can be widely used for detection of the SARS-CoV-2 at home or in small clinics, preventing or slowing the rapid spread of COVID-19. AM: Your method was used to detect SARs-CoV-2 and HIV could it be easily adapted in the future for other possible infectious disease outbreaks? CL: Yes, as a platform technology, our AIOD-CRISPR assay method can be easily adapted to detect other infectious disease in the future.

Changchun Liu, Associate Professor, Department of Biomedical Engineering, University of Connecticut Health Center, was speaking to Anna MacDonald, Science Writer, Technology Networks. References:

1: Ding, X., Yin, K., Li, Z., & Liu, C. (2020). All-in-One Dual CRISPR-Cas12a (AIOD-CRISPR) Assay: A Case for Rapid, Ultrasensitive and Visual Detection of Novel Coronavirus SARS-CoV-2 and HIV virus. https://doi.org/10.1101/2020.03.19.998724

2: Song, J., Mauk, M. G., Hackett, B. A., Cherry, S., Bau, H. H., & Liu, C. (2016). Instrument-Free Point-of-Care Molecular Detection of Zika Virus. Analytical Chemistry, 88(14), 72897294. https://doi.org/10.1021/acs.analchem.6b01632

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A Point-of-Care CRISPR-based COVID-19 Diagnostic on the Horizon - Technology Networks

Recommendation and review posted by Bethany Smith


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