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Roches Xofluza issued FDA approval to treat influenza in children aged five years and older – PMLiVE

Posted: August 14, 2022 at 2:38 am

Roche has received approval from the US Food and Drug Administration (FDA) for a supplemental New Drug Application (sNDA) for Xofluza (baloxavir marboxil). The approval is specifically for the treatment of acute, uncomplicated influenza in otherwise healthy children aged five to less than 12 years of age who have been symptomatic for no more than 48 hours.

In addition to this, the FDA has granted approval for Xofluza to be used as a preventative treatment of influenza in children aged five to less than 12 years old, following contact with someone who is infected with influenza.

The FDAs decision makes Xofluza the first single-dose oral influenza medicine to be approved in the US for children in this age group.

The approval is supported by phase 3 trial results taken from miniSTONE-2 which assessed the use of Xofluza in children and BLOCKSTONE which assessed Xofluza as a preventive treatment for households, in both adults and children. The results were published in The Pediatric Infectious Disease Journal and The New England Journal of Medicine, respectively.

There were more than six million illnesses, thousands of hospitalisations and over 100 deaths among children aged five to 17 caused by influenza in the US 2018-2019 influenza season.

miniSTONE-2 was a phase 3, multicentre, randomised, double-blind study that evaluated the safety, pharmacokinetics and effectiveness of a single-dose of Xofluza versus oseltamivir, in otherwise healthy children aged one to less than 12 years with influenza infection and displaying influenza symptoms for no more than 48 hours. The results showed that Xofluza was well tolerated with no new safety signals identified.

BLOCKSTONE was a phase 3, double-blind, multicentre, randomised, placebo-controlled, post-exposure prophylaxis study that evaluated single-dose Xofluza versus placebo in household members adults and children who were living with someone with influenza confirmed by a rapid influenza diagnostic test.

In the BLOCKSTONE trial, Xofluza showed a statistically significant preventative impact on influenza after a single dose, by reducing the risk of individuals aged 12 years and above from developing influenza after exposure to an infected household member by 90% versus placebo. The proportion of household members aged 12 years and above who developed laboratory-confirmed clinical influenza was 1.3% in participants treated with Xofluza and 13.2% in the placebo-treated group.

Levi Garraway, Roches chief medical officer and head of global product development, said: Xofluza has proven to be an important tool in fighting and preventing influenza in adults as well as adolescents, and we are pleased to now offer households and younger children our single-dose oral treatment.

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Why death matters – Big Think

Posted: August 14, 2022 at 2:35 am

Definitions of life are notoriously hard to pin down. Is a fire alive? It has a kind of metabolism, and in a sense it reproduces by spreading. Is a crystal alive? It certainly grows. What about a virus, which can reproduce and mutate, but only if it can find a living cell to use as a host?

Scientific definitions of life tend to focus on things like reproduction, metabolism, heredity, and evolution. But there is another, more basic property of life that has profound consequences for its study, and which I want to explore today: the capacity to die. While this may seem obvious, reframing life in terms of death reveals some of the biggest philosophical and scientific problems with the way we think about living systems.

Focusing on the biomolecular mechanisms of life has yielded remarkable insights into what happens inside cells. However, this emphasis over the last 70 years on molecules such as deoxyribonucleic acid has produced a kind of myopia that can lead researchers to blind themselves to a critical insight. Life is not just molecules. It cannot be reduced to the interactions of a set of molecular actors. Instead, life is really about organization. This is why, alongside the emphasis on biochemistry, there has always been a focus on life as an organism. An organism is a whole that is also wholly invested in its interactions with the environment. Biomolecules would never take on the activities they play in the cell were it not for the higher levels of organization the cell makes possible.

And this is where death comes in.

Biologists Humberto Maturana and Francisco Varela developed the concept of autopoiesis in the 1970s and 1980s to describe the essential character of life as an organism. Autopoiesis means self-producing. The term, which Maturana and Varela coined, refers to a kind of strange loop that occurs in living systems whereby the processes and products needed for an organism to survive must be created by the processes and products needed for the organism to survive. The classic example is the cell membrane, whose presence is required to create the very compounds that maintain it.

Over the next year I will be writing more about autopoiesis, as it forms part of a new research program on life and information funded by the Templeton Institute. The key point for today is to understand that one thing Maturana and Varela wanted to focus on with autopoiesis was its intrinsic capacity to end. To be an autopoietic system is to constantly face death.

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To be alive is always to live in a precarious condition, as Varela called it. You, me, a butterfly, a single-celled organism all life must constantly be at work to produce and maintain itself. Life can never take a rest from the internal activities it must carry out to do that. And this self-production and self-maintenance must work on a remarkable array of scales. At the molecular level, the ribosomes that drive lifes nano-machinery must never halt. At the cellular level, the membrane can never stop its work of monitoring and adjusting the flux of compounds into the cell. At the system level in more complex life, the various components of a plant or animal must always be synchronized and synchronizing.

Or else, what?

We know the answer to that question, for it drives so much of our higher animal psychology: or else, we die. The organism is always and forever bound to its state of precariousness, and eventually that precariousness must win. It always wins. To be alive is to be able to die.

This emphasis on death as the definition of life serves many roles and will be useful for many purposes. On a purely scientific level, it can help us understand which features of organisms and their organization to focus on. This is important for the Templeton project I am beginning, because it sharpens our focus on how information can serve to keep an organism viable, i.e. self-maintaining.

On a philosophical level, the focus on death reveals a key problem with reductionist descriptions of life that rely on what is called the machine metaphor. For reductionists, life is nothing but a set of molecular mechanisms. We are therefore nothing but biochemical machines. This is a fundamental mistake, because while a machine can be switched off, there can be no off button for life. Even seeds that remain dormant for years are not off like my blender is off when I am not using it. Life is not a machine.

Finally, understanding life as what can die has a personal or even spiritual valence. It gives the lie to the strange transhumanist, techno-religious fantasy about conquering death. While I am all for extending my life if I can, I would never think to avoid its end. Instead, what I long for is the fullest experience I can muster out of this strange trip. Then when death does come, I will greet it like the old friend it has always been.

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Why death matters - Big Think

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Ocugen CEO to Present at H.C. Wainwright 2nd Annual Ophthalmology Virtual Conference

Posted: August 14, 2022 at 2:33 am

MALVERN, Pa., Aug. 12, 2022 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals, and vaccines, announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen, will deliver a virtual presentation at the H.C. Wainwright 2nd Annual Ophthalmology Virtual Conference on August 17.

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Longeveron Inc. Provides Corporate Update and Reports Second Quarter 2022 Financial Results

Posted: August 14, 2022 at 2:33 am

—Longeveron’s Phase 2a trial of Lomecel-B for patients with mild Alzheimer’s Disease proceeding on schedule, currently at 50% enrollment

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Bespoke Extracts to Expand its Colorado Business and Capabilities with Acquisition of Best Day Ever

Posted: August 14, 2022 at 2:33 am

Acquisition will create a vertically integrated Colorado cannabis company, significantly accelerating Bespoke’s growth strategy

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Spectral Announces Second Quarter Results and Provides Corporate Update

Posted: August 14, 2022 at 2:33 am

TORONTO, Aug. 12, 2022 (GLOBE NEWSWIRE) -- Spectral Medical Inc. (“Spectral” or the “Company”) (TSX: EDT), a late stage theranostic company advancing therapeutic options for sepsis and septic shock, as well as commercializing a new proprietary platform targeting the renal replacement therapy market through its wholly-owned subsidiary Dialco Medical Inc. (“Dialco”), today announced its financial results for the second quarter ended June 30, 2022 and provided a corporate update.

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SciSparc Issued Israeli Patent for its Core Technology for Treating Central Nervous Systems Disorders

Posted: August 14, 2022 at 2:33 am

Patent extends protection for SciSparc’s novel compounds and methods already granted in the U.S., Australia and Japan

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PepGen Reports Second Quarter 2022 Financial Results and Recent Corporate Developments

Posted: August 14, 2022 at 2:33 am

BOSTON, Aug. 12, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (“PepGen”), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results for the second quarter ended June 30, 2022.

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Crinetics Pharmaceuticals Reports Second Quarter 2022 Financial Results and Provides Corporate Update

Posted: August 14, 2022 at 2:33 am

Paltusotine’s Phase 3 PATHFNDR Program in Acromegaly and Phase 2 Program in Carcinoid Syndrome Remain on Track for Top-Line Data in 2023

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VYNE Therapeutics Reports Second Quarter 2022 Financial Results

Posted: August 14, 2022 at 2:33 am

BRIDGEWATER, N.J., Aug. 12, 2022 (GLOBE NEWSWIRE) -- VYNE Therapeutics Inc. (Nasdaq: VYNE) (“VYNE” or the “Company”), a biopharmaceutical company developing proprietary, innovative, and differentiated therapies for the treatment of immuno-inflammatory conditions, today announced financial results for the three and six months ended June 30, 2022.

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