Category Archives: California Stem Cells

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

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Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

Image: Mesenchymal stromal cells (MSC) (Photo courtesy of UCSF)

There is currently no drug to treat ARDS, which has a mortality rate of 27% for mild cases and 45% for the most severe cases. An early study on COVID-19 patients with ARDS found that only about 25% survive, though that percentage may be as high as 40%. The clinical trial is testing the effects of infusions of stem cells drawn from bone marrow in the hopes of finding a therapy for the deadly syndrome. Patients in a randomized double blind clinical trial are being given mesenchymal stem cells (MSCs) or placebo to test the effectiveness of the therapy. The trial hopes to repair the severe and often fatal lung damage in people suffering from ARDS.

MSCs are stromal cells found in bone marrow, play a key role in how human bodies make and repair skeletal tissues - things like bone and cartilage and support the cells in the bone marrow that produce red and white blood cells. Once researchers discovered that MSCs could modulate inflammation when transplanted into other people, they began exploring them as potential therapies for a range of diseases and traumas.

ARDS can be caused by trauma, bacterial infection, or a viral infection like COVID-19. In an earlier trial, the team had showed that MSCs are safe to use, and that they have promising benefits in treating patients with ARDS. In the current Phase 2 double blind clinical trial, the researchers are enrolling 120 ARDS patients who are adult ICU patients on ventilators with excess fluid in their lungs, and no sign of heart failure as a primary cause of the respiratory failure. Based on these outcomes and further studies on MSCs, the researchers view them as a possible route for cell-based therapy. Much like cancer treatment is not a single approach but instead different therapies tailored to the type of cancer cells, the use of something like MSCs could be perhaps tailored exactly to whatever specific kind of syndrome of respiratory failure.

Its a very interesting possible therapy, said Michael Matthay, MD, professor of medicine at the UCSF School of Medicine. Were really in the early phases of understanding these cell-based therapies.

Related Links:University of California San Francisco

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Stem Cells Offer Hope of Treatment for COVID-19 Patients with Acute Respiratory Distress Syndrome (ARDS) - HospiMedica

While there is no cure for blindness and macular degeneration, scientists have accelerated the process to find a cure by visualizing the inner workings of the eye and its diseases at the cellular level.

In an effort led by UW Medicine, researchers successfully modified the standard process of optical coherence tomography (OCT) to detect minute changes in response to light in individual photoreceptors in the living eye.

The results were published Sept. 9 in Science Advances.

We have now accelerated the life cycle of vision restoration, said lead author Vimal Prabhu Pandiyan, a ophthalmology researcherat the University of Washington School of Medicine.

The study was fundedin partby the National Eye Institutes Audacious Goals Initiative, which embraces bold ideas in helping people to see better.

The OCT modifications outlined in the study will help researchers who want to test therapiessuch as stem cells or gene therapy to treat retinal disease. They now have the tools to zoom in on the retina to evaluate whether the therapy is working.

Corresponding author Ramkumar Sabesan, a UW assistant research professor of ophthalmology, said the only wayto objectively measure the eye currently is to look at a wide retinal area. Sabesan said researchers currently can attach electrodes on the cornea but it captures a large area with around 1 million cells. Now they are talking about nanometers, or one billionth of a meter a small fraction of the size of a cell, providing orders of magnitude improvement.

Since photoreceptors are the primary cells affected in retinal generation and the target cells of many treatments, noninvasive visualization of their physiology at high resolution is invaluable, the researchers wrote.

Cone photoreceptors are the building blocks of sight, capturinglight and funneling information to the other retinal neurons. They are a key ingredient in how we process images and patterns of light falling on the retina.

Optical coherence tomography has been around since the 1990s. In this study, researchers used OCT with adaptive optics, line-scanning and phase-resolved acquisition to deliver the concept of Thomas Youngs interference to the human eye. With the ability to zoom in on the retina at high speeds, they found that cone photoreceptors deform at the scale of nanometers when they first capture light and begin the process of seeing.

As Sabesan explained: You can imagine a picture that looks visually and structurally normal. But when we interrogate the inner working of the retina at a cellular scale, we may detect a dysfunction sooner than what other modalities can do. A doctor then can prescribe medication to intervene early or follow the time-course of its repair via gene therapy or stem cell therapy in the future.

We will now have a way to see if these therapies are acting in the way they should, Sabesan said.

The study also involved researchers at Stanford University, University of California,Berkeley, and University of California, Riverside.

The study was funded by NIH grants U01EY025501, EY027941, EY029710, EY025501, and P30EY001730; Research to Prevent Blindness Career Development Award; Foundation Fighting Blindness; Murdock Charitable Trust; Burroughs Welcome Fund Careers at the Scientific Interfaces; and Unrestricted grant from the Research to Prevent Blindness.

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Seeing the eye like never before | Newsroom - UW Medicine Newsroom

The MemorialCare Heart & Vascular Institute at Long Beach Medical Centeris expanding its leadership team with accomplishedSouthern Californiacardiologist,David Shavelle, M.D., being named medical director of adult cardiology. Dr. Shavelle is bringing his extensive leadership experience in cardiology to this new role that will provide leadership and strategic direction for adult cardiology programs, as well as oversight for the interventional catheterization laboratories.

Dr. Shavelle, a Millikan High School (Long Beach, Calif.) graduate, is returning toLong Beachwith more than 20 years of cardiology practice, research leadership, and teaching experience. He joins Long Beach Medical Center from KeckMedical Center at the University of Southern California, where he served as the Director of Interventional Cardiology while leading a multitude of clinical research trials, including several focused on implanted devices for heart failure. He plans on increasing the availability ofclinical research trialsfor cardiology patients at Long Beach Medical Center.

The MemorialCare Heart & Vascular Institute has a rich history of research and pioneering new treatment techniques, says Ike Mmeje, chief operating officer, Long Beach Medical Center.

Dr. Shavelles passion for research makes him a perfect fit to continue that legacy and find the next cutting-edge treatment for our cardiology patients.

MemorialCare Heart & Vascular Institute facilities are among the most comprehensive centers for diagnosis, treatment and rehabilitation of cardiac disease, providing groundbreaking care for complex heart conditions, including myocardial infarction, heart failure, arrhythmias and peripheral vascular disease. In addition to his hopes to expand research opportunities, Dr. Shavelle plans on expanding the programs for heart failure and structural heart disease.

I am excited to join the MemorialCare Heart & Vascular Institute at Long Beach Medical Center, says Dr. Shavelle. My dad was a physician here, and many of my mentors and fellows are at Long Beach Medical Center. Im looking forward to creating more collaboration among cardiologists, surgeons, residents and the entire team to expand the already comprehensive cardiology care available to the community.

After earning his medical degree from theUniversity of California, Los Angeles(UCLA), Dr. Shavelle completed his internal medicine internship and residency at Harbor-UCLA Medical Center. He completed General Cardiology Fellowship at theUniversity of Washingtonand Interventional Cardiology Fellowship at Harbor-UCLA Medical Center/Good Samaritan Hospital. Dr. Shavelle served as Associate Professor at both the David Geffen School of Medicine atUCLAand the Keck School of Medicine at theUniversity of Southern California. He alsoserveson the editorial boards for theJournal of Cardiovascular Pharmacology and Therapeutics, Current Medical Research and Opinion and Cardiology Clinics.

The MemorialCare Heart & Vascular Institute delivering nearly 20,000 cardiovascular diagnostic tests and treatments last year continues to push the boundaries of discovery with many firsts. These began 70 years ago when world-renowned cardiologist, researcher and educator, the lateMervyn Ellestad, M.D., co-invented at Long Beach Medical Center the modern-day maximum stress test to detect heart disease. Today, millions of exercise stress tests performed annually save hundreds of thousands of lives globally.

It is amazing how the field of cardiology has grown and how many treatment options are available through minimally invasive techniques, says Dr. Shavelle. Many of these new treatment options have come from research trials, and Im looking forward to expanding the opportunities for patients in theLong Beacharea. The studies we have in the pipeline include trials with stem cells and heart failure devices.

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David Shavelle, MD, Named Medical Director of Adult Cardiology for the MemorialCare Heart & Vascular Institute at Long Beach Medical Center -...

The global war against the coronavirus pandemic continues to wage on while researchers and medical experts seek to find a cure for COVID-19 symptoms. While many believe this is here to stay for an indefinite period, others feel that this too, shall pass.

The number of confirmed cases and death rates only seem to darken our world with over 400,000 confirmed cases in New York, the death toll is now over 31,000, with over 233,000 confirmed cases in Florida, the death toll is now over 4,000, and with over 250,000 confirmed cases in Texas, the death toll is now over 3,000.

And here we are in July 2020 where you can simply add those statistics and drive to find a cure for COVID-19 to our to-do list in the war on cancer and other diseases that still have not seen a cure.

With Florida continuing to make headlines by the day, most recently with one family facing federal charges after allegedly marketing a toxic bleach solution as a cure for multiple ailments, including COVID-19, the timing for our society to come together to help find a cure is essential.

And no, were not kidding. The Florida family (Mark Grennon and his three sons) were charged Wednesday with conspiracy to defraud the United States, conspiracy to violate the Federal Food, Drug and Cosmetic Act, and criminal contempt, according to the Department of Justice.

As for the Florida community, one Tampa business has been conducting the first-in-human clinical study for cutaneous melanoma. Morphogenesis, a clinical-stage company developing novel cell and gene therapies has been making headlines after receiving FDA approval to expand its human clinical trials into two more types of cancer: Merkel cell carcinoma (MCC) and cutaneous squamous cell carcinoma (cSCC).

Using its ImmuneFx (IFx) cancer vaccine technology that initiates the power of the immune system on the destruction of tumor cells, Morphogenesis of Tampa will focus its newly FDA approved trials on understanding these two new cancers, which follows in the footsteps of successful human trials on cutaneous melanoma, conducted in cooperation with Moffitt Cancer Center in 2019.

Other MCC and cSCC clinical trial sites across the country include the University of Southern California, the University of Utah, the University of Colorado, and the Dana Farber / Harvard Cancer Center. So why so many trial sites?

Well, according to Morphogenesis CEO Dr. Patricia Lawman, clinical trials need patients, which to qualify, requires an individual having been diagnosed with advanced Merkel or cutaneous squamous cell carcinoma, and having failed or refused other therapies.

As a cell and gene company, the mission from the beginning was to learn from the body and use the bodys building blocks and communication systems to treat chronic disease. Morphogenesis, according to Lawman, was built to identify, isolate and proliferate stem cells and progenitor cells to treat diseases such as diabetes.

With the world united to change the way in which chronic diseases are treated by engaging the innate intelligence of the body, how do companies (on a local level) push for national change?

The ability to genetically modify stem cells to enhance functionality is one aspect of this, but in order to perform a biological function, the stem cells must differentiate into mature cells, e.g. hematopoietic stem cells differentiate into macrophages that perform phagocytic and antigen presentation functions and T cells that kill cancer cells or virally infected cells. Morphogenesis means the evolution of form, which connotes the change for stem cells to these functional cells capable of mitigating chronic disease.

And what this means for our bodies, according to Lawman, is that regardless of the species, our bodies have developed systems that maintain structure and function over a long period of time. When we need to control blood sugar, beta islet cells produce just enough insulin as needed.

Indeed with modern technology, you would think that this is a relatively easy process to control.

There is an exquisite feedback system that regulates this. When things go awry, our best solution has been to provide insulin through pumps that are controlled in part by constant glucose monitors. This one example of where modern technology has tried to solve a problem mimicking how the body works.

However, even providing insulin through a pump cant do what a pancreas can. When it comes to dealing with foreign invaders, the immune system is unequaled. No drug, small molecule or compound can eliminate an invader as well as a fully functional immune system. We can kill cancer cells (while not foreign, they are still invaders) with chemo and radiation, but given the proper assurance, the immune system can eliminate the invader and do it with fewer adverse effects. Almost always, the body performs healing functions better than a synthetic drug or compound.

The companys recent FDA approval to move forward with stage 2 of its clinical trials is a gigantic win for the companys mission. The ability to expand our proof of concept studies from a single skin cancer into other, quite different skin cancers under the same Investigational New Drug (IND) is the next step in the execution of our clinical development plan.

And that starts with Morphogenesis focus on easily accessible tumors.

Since our therapy can be used to treat virtually any type of cancer, we wanted to start out with easily accessible tumors that could be directly injected with our plasmid DNA. The safety data collected from the cutaneous melanoma, Merkel cell carcinoma and cutaneous squamous cell carcinoma Phase 1 trials is a Segway into a Phase 2 skin cancer basket trial testing IFx-Hu2.0 as a monotherapy and in combination with a checkpoint inhibitor.

But anytime there is discussion over stem cell research or breakthroughs in the war on cancer, of course comes naysayers and disbelievers.

One thing that Dr. Lawman has noticed is the bias within scientific circles.

In scientific circles, there has been a bias against simple solutions, including the assumption that to get efficient transfer of genetic material you need viral vectors for all applications. These vectors are complicated to manufacture and use and pose a certain amount of risk to the patients. Plasmid DNA or mRNA, on the other hand, are much safer and are a viable alternative to viruses.

As an example, we inject our plasmid DNA directly into a patients tumor. We get sufficient uptake and expression of our protein to initiate an immune cascade with the effect spreading to multiple tumor antigens. The use of a viral vector in this case would be an unnecessary complication and risk.

Now in todays landscape with COVID-19 putting more pressure on experts to find a cure, Morphogenesis, like any company, is similarly faced with logistical challenges and supply issues.

COVID-19 has certainly affected patient recruitment to our trials. Hopefully, ways will be developed for patients to receive treatment for their terminal diseases even if restrictions continue. Otherwise, the death rate for these patients will be much higher than COVIDs. The biggest challenge for us is the continual process of raising funds that all small biotechs face.

As for ImmuneFx, the companys newest vaccine, we got the exclusive.

Beyond the Phase 2 skin cancer trial, we will be opening trials for head and neck cancer, gastric cancers, cervical cancer and colorectal cancer. The value add here is that successful trial results in multiple types of cancer will substantiate the efficacy and expand our label claims.

But with new products and solutions, come criticism. Lawman added that the one thing that is not usually discussed in such conversations is the importance of the safety profile of a new product.

Some of the new cellular immunotherapies not only come with a hefty price tag, the cost of treating the adverse side effects caused by the therapies can be as much as double the cost of the therapy itself (up to $1.5M total). Some of the newer gene therapies can have a price tag of up to $2M per treatment.

Not only can our plasmid DNA be cost effectively manufactured, it is causing minimal side effects, i.e. what we saw in hundreds of companion animals with naturally occurring cancers is being born out in human patients. Both of these cost saving factors means that ultimately, millions of people will have access to cancer treatment who otherwise would have none.

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Why The FDA's Recent Approval Of New Vaccine Is A Gigantic Win In The War On Cancer - Innovation & Tech Today

ViaCyte, Inc. and Newark-based W. L. Gore & Associates, Inc. announced the two companies have signed an agreement covering the next phase of their collaboration focused on the development of ViaCytes Encaptra Cell Delivery System that uses Gore material technologies.

The technology is designed to produce insulin for patients with Type I diabetes, formerly known as juvenile diabetes.

The Encaptra System could be a transformative therapy for patients with type 1 diabetes, the companies noted.

Tapping into Gores world-class materials science and medical device expertise, new membrane technology was developed that appears to allow successful engraftment and beta cell proliferation while minimizing the host foreign body response, said Paul Laikind, CEO of ViaCyte. This agreement is an important next step on our path towards a commercial product as well as a template for future agreements for additional work ViaCyte and Gore may do together on cell delivery technology.

Our collaboration with ViaCyte has been very productive in enabling the clinical advancement of the PEC-Encap product candidate with proprietary Gore materials technology, said Erin Hutchinson, business leader, Gore PharmBIO Cell Encapsulation Products. We are delighted to further strengthen our collaboration on the path to the delivery of a potentially functional curative product for those currently suffering with type 1 diabetes and help pioneer the new field of cell replacement therapy.

ViaCyte is the only company with stem cell-derived islet replacement candidates undergoing clinical testing and the only group to show that the implanted cells are capable of producing insulin in people with type 1 diabetes.

The Companys promising candidates have the potential to deliver a functional cure for people with type 1 diabetes and could be an advancement for people with insulin-requiring type 2 diabetes.

Under the terms of the agreement, Gore will manufacture and supply a proprietary membrane and the device component for use with ViaCytes PEC-Encap product candidate.

The novel Gore membrane has been evaluated in preclinical studies. Gore will continue to collaborate with ViaCyte to optimize the device design and implant techniques in support of human clinical trials.

Early human clinical trials of PEC-Encap with the new membrane are underway and preliminary histological results are encouraging. Should the membrane prove successful in the ongoing trial, Gore will assume the responsibility for manufacturing the Encaptra System incorporating the novel membrane technology for late stage clinical development and commercial use.

ViaCyte has shown that once implanted and successfully engrafted PEC-01 cells mature into beta cells that secrete insulin in a regulated manner to control blood glucose levels. The Encaptra System serves to protect the PEC-01 cells from the host immune system thus eliminating the need for immune suppression drugs commonly used with other transplants.

A critical enabling component of the Encaptra System that contains and protects the cells is a specifically designed semi-permeable membrane that encapsulates the cells but allows for diffusion of oxygen and nutrients into the device and insulin and other hormones out of the device and into the circulation.

The Encaptra System membrane is composed of an expanded PTFE composite, similar to materials that are used in Gores implantable medical products..

Gore and ViaCyte began a collaboration in 2017.

ViaCyte is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. ViaCyte is headquartered in San Diego.

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Gore partners with San Diego company on cell delivery system with potential to treat Type 1 diabetes - delawarebusinessnow.com

FLORHAM PARK, N.J., Aug. 5, 2020 /PRNewswire/ --Celularity today announced that it has been awarded a $750,000 COVID-19 Project grant by the California Institute for Regenerative Medicine (CIRM), one of the three clinical awards targeting the coronavirus. This grant will support California Institutions participating in the Phase I/II clinical trial of human placental hematopoietic stem cell derived natural killer (NK) cells (CYNK-001) for the treatment of adults with COVID-19.The University of California Irvine is the first CA site to open for patient enrollment.

CIRM's COVID-19 Project supports promising discovery, preclinical and clinical trial stage projects that could quickly advance treatments or vaccines that utilize stem and/or progenitor cells. Celularity will use the CIRM grant to support the evaluation of the anti-viral activities of its cryopreserved investigational product, CYNK-001, in underserved and disproportionately affected populations with COVID-19, an infectious disease caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Celularity received clearance from the United States Food and Drug Administration to proceed with a Phase I/II study to evaluate the safety, tolerability, and efficacy of CYNK-001 in patients with COVID-19.

"Our investigational product CYNK-001 showed great promise in preclinical studies, and we are optimistic that it will prove effective against corona virus diseases, including COVID-19. As part of our rapidly-scalable placental-derived cellular medicine platform, CYNK-001 could play an important role in the public health response to outbreaks of COVID-19 or other coronaviruses," said Robert J. Hariri, MD, PhD, Founder, Chairman and CEO of Celularity. "We are very grateful to CIRM for supporting our effort to make novel cellular medicines available to medically underserved and disproportionately affected persons in California."

Xiaokui Zhang, PhD, Chief Scientific Officer and Principal Investigator under the CIRM grant says "CYNK-001 has a range of biological activities that not only recognize and destroy virus-infected cells, but also coordinate a robust immune response that may lead to an effective and durable defense against the viral infection."

The trial will evaluate the safety and the clinical efficacy of CYNK-001 in SARS-CoV-2 positive subjects as measured by clearance of the SARS-CoV-2 and improvement in clinical symptoms or improvement in radiological evaluation of disease related chest x-ray. The primary objective of the Phase I portion of the study is to evaluate the safety, tolerability, and efficacy of multiple CYNK-001 intravenous (IV) infusions in COVID-19 patients and will be administered to up to 14 patients in three doses over the course of seven days.

The Phase II portion of the study is a randomized, open-label, multi-site study measuring multiple doses of CYNK-001 against a control group experiencing a similar degree of infection with best supportive care, with two co-primary endpoints. The first co-primary endpoint is to determine the virologic efficacy of CYNK-001 in facilitating the clearance of SARS-CoV-2 from mucosal specimens and/or peripheral blood. The second co-primary endpoint is to assess the impact of treatment with CYNK-001 on clinical symptoms among patients with COVID-19 related lower respiratory tract infection.

Celularity has treated patients with severe COVID-19 and on ventilator support under compassionate use programs in U.S. CYNK-001 was well tolerated and may be associated with clinical benefit in selected cases.

About NK CellsNK cells are innate immune cells with an important role in early host response against various pathogens. Multiple NK cell receptors are involved in the recognition of infected cells. Studies in humans and mice have established that there is robust activation of NK cells during viral infection, regardless of the virus class, and that the depletion of NK cells aggravates viral pathogenesis.

About CYNK-001CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy developed from placental hematopoietic stem cells. CYNK-001 is being investigated as a potential treatment option in adults with COVID-19, as well as for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. CYNK-001 cells derived from the placenta are currently being investigated as a treatment for acute myeloid leukemia (AML), multiple myeloma (MM), and glioblastoma multiforme (GBM). On 1 April, the U.S. Food and Drug Administration cleared the Company's Investigational New Drug (IND) application for the use of CYNK-001 in adults with COVID-19.

About COVID-19The virus causing COVID-19 is called severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and is a novel coronavirus that has not been previously identified. COVID-19 has become a global pandemic, with over 4.2 million confirmed cases in the USA and over 650,000 deaths reported to date worldwide.

About CelularityCelularity, headquartered in Florham Park, N.J., is a next-generation Biotechnology company leading the next evolution in cellular medicine by delivering off-the-shelf allogeneic cellular therapies, at unparalleled scale, quality, and economics. Celularity's innovative approach to cell therapy harnesses the unique therapeutic potential locked within the cells of the post partum placenta. Through nature's immunotherapy engine the placenta Celularity is leading the next evolution of cellular medicine with placental-derived T cells, NK cells, and pluripotent stem cells to target unmet and underserved clinical needs in cancer, infectious and degenerative diseases. To learn more visit celularity.com

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Celularity announces the activation of first California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19 - PRNewswire

Today (August 11), Big Idea Ventures (BIV) announces the launch of its second food technology accelerator program in New York City and Singapore. This years cohort includes 12 early-stage startups working in the plant-based and cell-based space, innovating new technologies that will be vital if we are to successfully shift towards a more sustainable food system. Below we highlight the exciting companies in BIVs latest cohort.

Founding date: 2018

Founder: Vince Lu

Headquarters: Beijing, China

Mission: Zhenmeat is Chinas first and leading plant-based meat startup creating sustainable protein substitutes that are tailored to the Chinese palate. Its products include plant-based pork, mooncakes, sausages and meatballs, made from pea protein, fungus, cellulose, coconut oil and various natural flavour extracts and spices. Zhenmeat is also working on vegan crayfish and pork tenderloin. Their products are currently sold online and through various restaurant partners in China.

Founding date: 2019

Founders: Stephen Michael Co, Carlo Antonio Ng & Carissa Jane Lim

Headquarters: Manila, Philippines

Mission: WTH Foods (Worth The Health) is a plant-based meat company developing sustainable alternatives made using carefully-sourced local ingredients and inspired by regional flavours. Its products include pinoy pulled jackfruit pork, jackfruit nuggets and mung bean-based ground meat. WTH Foods products are currently available online via its website.

Founding date: 2019

Founder: Vinayaka Srinivas & Thanh Hung Nguyen

Headquarters: Singapore

Mission: GaiaFoods is Singapores first cell-based red meat company. Using stem cell biotechnology, the company cultivates meat that tastes and looks exactly like real meat but without the need to harm animals, making it a cruelty-free and sustainable alternative, and does not contain any hormones, antibiotics or the use of any toxic chemicals.

Founding date: 2015

Founders: Alejandro Cancino & Paola Moro

Headquarters: Brisbane, Australia

Mission: Fenn Foods is an Australian startup creating healthy, affordable and tasty plant-based protein alternatives made using ethically sourced ingredients such as textured vegetable protein derived from soybeans, yeast extract and pea protein. Its products include a beef alternative dubbed veef, schnitzel and chicken burger patties. The company does not use any ingredients that are GMO and source as many local and organic produce as possible.

Founding date: 2020

Founder: Nieves Martnez Marshall

Headquarters: Berkeley, California, U.S.

Mission: Novel Farms, Inc. uses synthetic biology to make cruelty-free, cost-efficient and functionalised 3D scaffolds for the production of structured cultured meats. Its technology helps to improve the marbling and texture of cell-based meats and can also reduce manufacturing costs. Its mission is to transform more sustainable and slaughter-free cell-based meats into a sought-after culinary experience.

Founding date: 2013

Founder: Aleem Ahmed

Headquarters: San Francisco, California, U.S.

Mission: Wild For Co. connects farmers to families by creating nutritious and delicious foods using the ancient Ethiopian superfood grain teff. Its star product is a vegan-friendly air-popped chip made with teff, which is naturally gluten-free, high-protein, high-fibre and is packed with nutrients such as calcium, iron and the essential amino acids.

Founding date: 2019

Founders: Kartik Dixit & Shraddha Bhansali

Headquarters: Mumbai, India

Mission: EVO Foods is Indias first plant-based egg startup using food science and plant biochemistry to develop a vegan egg alternative. Its first product is a 100% plant-based liquid egg made from protein derived from lentils, which contains no cholesterol and antibiotics. It recently received angel investment from Wild Earth founder Ryan Bethencourt, as well as VegInvest and Shiok Meats co-founder Sandhya Sriram.

Founding date: 2019

Founder: Patricia Bubner

Headquarters: Berkeley, California, U.S.

Mission: Orbillion Bio is a startup looking to develop premium cell-based meat products from heritage cell lines that are directly sourced from farmers. On a mission to accelerate the broad availability of a variety of nutritious cultivated meat products, the startups first product will be bison jerky that is low-fat, low-cholesterol and high-protein.

Founding date: 2020

Founders: Darko Mandich & Aaron Schaller

Headquarters: Berkeley, California, U.S.

Mission: MeliBio is using microbiology to cultivate sustainable and clean honey that has the same taste and nutritional profile as conventional honey. Because it is produced without traditional beekeeping techniques, its honey does not harm pollinator diversity, especially wild and native bee populations, which have been jeopardized over the years by the expansion of commercial beekeeping.

Founding date: 2020

Founders: Jason Rosenbaum & Hailey Swartz

Headquarters: New York City, New York, U.S.

Mission: Actual Veggies creates chef-crafted fresh and refrigerated burgers that puts vegetables front and centre all their burgers are colourful, showcasing their primary vegetable ingredient. They contain only fresh farm veggies and natural binding units, such as kale, sweet potatoes, mushrooms and beets. With added plant proteins such as legumes and seeds, each patty has around 10 grams of protein.

Founding date: 2018

Founders: Kerem Erikci & Can Akcali

Headquarters: Ankara, Turkey

Mission: Biftek.co is a startup producing cultivated medium that can replace fetal-bovine-serum (FBS) in the production of cell-based meats. Using its novel culture supplement formulation to grow muscle stem cells, its solution is 80% to 90% less expensive, which can help food techs lower the cost of producing cultivated proteins.

Founding date: 2016

Founder: Bonnie Lau

Headquarters: San Francisco, California, U.S.

Mission: Yoconut is a plant-based dairy brand creating delicious, healthy and allergy-friendly coconut-based yoghurts that contain live cultures and no added sugar. Currently, the brand has five vegan coconut yoghurt products in various flavours, and is available online as well as in a number of stores across California.

Lead image courtesy of Veef / Fenn Foods.

Original post:
Meet The 12 Next-Gen Food Techs Transforming The Future Of Protein - Green Queen Media

- NCCN Guideline Updates Highlight Need for Maximum CIN Prevention and Resource Allocation for COVID-19 Patients -

- First Patient Dosed in the U.S. Avoided Grade 4 Neutropenia in Cycle 2 with Plinabulin and Pegfilgrastim, Despite Experiencing Grade 4 Neutropenia in Cycle 1 with Pegfilgrastim Alone -

NEW YORK, Aug. 11, 2020 (GLOBE NEWSWIRE) -- BeyondSpring Inc.(the Company or BeyondSpring) (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative immuno-oncology cancer therapies, today announced that the Company has initiated an Expanded Access Program (EAP) to enable doctors across the U.S. to use BeyondSprings late-stage asset, Plinabulin, to prevent cancer patients chemotherapy-induced neutropenia (CIN), both alone and in combination with G-CSFs (the current standard of care), during the COVID-19 pandemic. Dr. Emad Ibrahim enrolled the first patient at Redlands Community Hospital in California on July 28, 2020.

In response to COVID-19, the National Comprehensive Cancer Network (NCCN) recently updated its treatment guidelines for the prophylaxis of CIN, with the objective of preserving hospital and ER resources for COVID-19 patients and maximizing protection for cancer patients against CIN development. This is designed to help necessitate healthcare interactions, and avoidance of hospital / ER visits will also minimize cancer patients risk of contracting COVID-19. In light of these NCCN guideline updates, BeyondSpring initiated an Expanded Access Program to enable the use of Plinabulin by oncologists to better protect cancer patients against CIN with the use of myelosuppressive chemotherapies under the current COVID-19 challenges.

Dr. Emad Ibrahim enrolled the first patient under this EAP at Redlands Community Hospital in California:

The recent updates to the NCCN guidelines aim to protect cancer patients from developing CIN in the most effective way possible and enable the healthcare system to reserve precious resources for COVID-19 patients, said Ramon Mohanlal, BeyondSprings Chief Medical Officer and Executive Vice President, Research and Development. In our CIN studies, Plinabulin, in combination with Pegfilgrastim, provided superior protection against CIN, compared to the standard of care alone. The observation in this first EAP patient who completely avoided Grade 4 CIN when given Plinabulin and Pegfilgrastim is a significant achievement for us. At BeyondSpring, we strive to play our part in serving patients and healthcare providers to the highest degree while working through the many challenges imposed by COVID-19.

Preventing CIN during chemotherapy is extremely important, as this will enable cancer patients to receive the full regimen of chemotherapy and achieve treatment goals. The onset of CIN is the No. 1 reason for treatment modifications, such as downgrading the strength of chemotherapy or stopping chemotherapy altogether. When a patient develops CIN, the treating physician is required to delay the next round of chemotherapy until a patients white blood cell count recovers. These changes can have a profoundly negative impact on patient outcomes.

For more information on BeyondSprings Plinabulin Expanded Access Program, please visit http://www.beyondspringpharma.com/EAP/. Supplies may be limited.

If you are a physician in the U.S. who would like to request Plinabulin EAP access for your patient, please email expandedaccess@beyondspringpharma.com.

About BeyondSpringHeadquartered in New York, BeyondSpring is a global, clinical-stage biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients with high unmet medical needs. BeyondSprings first-in-class lead immune asset, Plinabulin, is a potent antigen-presenting cell (APC) inducer. It is currently in two Phase 3 clinical trials for two severely unmet medical needs indications: one is for the prevention of chemotherapy-induced neutropenia (CIN), the most frequent cause for a chemotherapy regimen doses decrease, delay, downgrade or discontinuation, which can lead to suboptimal clinical outcomes. The other is for non-small cell lung cancer (NSCLC) treatment in EGFR wild-type patients. As a pipeline drug, Plinabulin is in various I/O combination studies to boost PD-1 / PD-L1 antibody anti-cancer effects. In addition to Plinabulin, BeyondSprings extensive pipeline includes three pre-clinical immuno-oncology assets and a drug discovery platform dubbed molecular glue that uses the protein degradation pathway.

About PlinabulinPlinabulin, BeyondSprings lead asset, is a differentiated immune and stem cell modulator. Plinabulin is currently in late-stage clinical development to increase overall survival in cancer patients, as well as to alleviate chemotherapy-induced neutropenia (CIN). The durable anticancer benefits of Plinabulin have been associated with its effect as a potent antigen-presenting cell (APC) inducer (through dendritic cell maturation) and T-cell activation (Chem andCell Reports, 2019). Plinabulins CIN data highlights the ability to boost the number of hematopoietic stem / progenitor cells (HSPCs), or lineage-/cKit+/Sca1+ (LSK) cells in mice. Effects on HSPCs could explain the ability of Plinabulin to not only treat CIN but also to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34+ cells in patients.

Cautionary Note Regarding Forward-Looking StatementsThis press release includes forward-looking statements that are not historical facts. Words such as "will," "expect," "anticipate," "plan," "believe," "design," "may," "future," "estimate," "predict," "objective," "goal," or variations thereof and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are based on BeyondSpring's current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to, difficulties raising the anticipated amount needed to finance the Company's future operations on terms acceptable to the Company, if at all, unexpected results of clinical trials, delays or denial in regulatory approval process, results that do not meet our expectations regarding the potential safety, the ultimate efficacy or clinical utility of our product candidates, increased competition in the market, and other risks described in BeyondSprings most recent Form 20-F on file with the U.S. Securities and Exchange Commission. All forward-looking statements made herein speak only as of the date of this release and BeyondSpring undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.

Media ContactsCaitlin Kasunich / Raquel ConaKCSA Strategic Communications212.896.1241 / 212.896.1276ckasunich@kcsa.com / rcona@kcsa.com

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BeyondSpring Initiates Expanded Access Program with Plinabulin for Patients Suffering from CIN in the US - BioSpace

By David Bautz, PhD

NASDAQ:BCLI

READ THE FULL BCLI RESEARCH REPORT

Business Update

Topline Data from Phase 3 ALS Trial Before End of November 2020

BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) is currently conducting a Phase 3 clinical trial of NurOwn in patients with amyotrophic lateral sclerosis (ALS) (NCT03280056). A total of 200 patients were randomized 1:1 to receive NurOwn or placebo in the randomized, double blind, placebo controlled, multi-dose trial. Cells were extracted once from each patient prior to treatment, with all administrations of NurOwn derived from the same extraction of cells due to a cryopreservation process the company developed for long-term storage of mesenchymal stem cells (MSC). Just as with the companys prior studies, there was a 3-month run-in period prior to the first treatment with two additional NurOwn treatments occurring two and four months following the first treatment. The company is focusing the trial on faster-progressing ALS patients since those patients demonstrated superior outcomes in the Phase 2 trial of NurOwn. The primary outcome of the trial is the ALSFRS-R score responder analysis and we now anticipate topline results before the end of November 2020.

Update on Phase 2 Progressive Multiple Sclerosis Trial

BrainStorm is currently conducting a Phase 2 clinical trial of NurOwn in patients with progressive multiple sclerosis (MS) (NCT03799718). The trial is an open label, single arm study that is enrolling patients with progressive MS with Expanded Disability Status Scale (EDSS) scores of 3.0 6.5. The primary endpoint of the study is the safety and tolerability of three doses of NurOwn with secondary endpoints examining the timed 25-foot walking speed or 9-hole peg test (both validated MS clinical outcome assessments) along with paired cerebrospinal fluid (CSF) and blood biomarker analysis. The National Multiple Sclerosis Society awarded the company a $0.5 million grant to help fund the study.

The trial is now fully enrolled and we anticipate dosing to be completed for all patients by the end of 2020. While the company had previously considered performing an interim analysis, since topline data would be available soon after an interim analysis could be performed the company has decided against performing an interim analysis and will instead report topline data for all 20 patients when it becomes available.

NurOwn Derived Exosomes Show Promise in Preclinical ARDS Study

On July 23, 2020, BrainStorm announced the successful completion of the first milestone in developing an exosome-based platform for the treatment of severe acute respiratory distress syndrome (ARDS) caused by COVID-19. Exosomes are small vesicles (30-150 nm) that are secreted by all cell types. Exosomes from mesenchymal stem cells (MSCs), from which NurOwn is derived, can penetrate into deep tissues and deliver various bioactive molecules. In addition, they can be delivered both intravenously or intratracheally.

BrainStorm conducted a preclinical study of MSC-derived exosomes in a mouse model of lipopolysaccharide (LPS)-induced ARDS. Treatment with intratracheally administered exosomes resulted in a statistically significant improvement in various lung functions, including functional lung recovery and decreased lung damage, as judged by the lung disease severity score (P=0.03). In addition, they led to a reduction in a number of pro-inflammatory cytokines. Lastly, exosomes derived from MSC-NTF cells were superior to exosomes derived from nave MSC from the same donor.

We anticipate the results being submitted to a peer reviewed journal for publication and the company is currently deciding whether to initiate a clinical trial program in COVID-19 patients with ARDS.

Financial Update

On August 5, 2020, BrainStorm announced financial results for the second quarter of 2020. As anticipated, the company did not report any revenues during the second quarter of 2020. Net R&D expenses for the second quarter of 2020 were $5.7 million, compared to $3.6 million for the second quarter of 2019. The increase was primarily due to an increase in payroll and stock-based compensation and a decrease in support from the Israel Innovation Authority (IIA) and California Institute for Regenerative Medicine (CIRM) along with a decrease in costs related to the Phase 3 and Phase 2 clinical trials. Excluding participation from the IIA and CIRM, R&D expenses were $6.0 million in the second quarter of 2020, compared to $6.5 million in the second quarter of 2019. G&A expenses for the second quarter of 2020 were $1.7 million, compared to $1.3 million in the second quarter of 2019. The increase was primarily due to increased payroll and stock-based compensation.

Story continues

The company exited the second quarter of 2020 with approximately $16.2 million in cash, cash equivalents, and short-term investments. Subsequent to the end of the quarter, the company raised gross proceeds of approximately $13.7 million from the $50 million ATM facility entered into in March 2020 through the sale of 945,082 shares at an average price of $14.48 per share. In addition, the company raised gross proceeds of approximately $6.3 million from the exercise of warrants. We estimate that the company currently has approximately $35 million in cash, cash equivalents, and short-term investments.

As of July 31, 2020, the company had approximately 31.5 million shares outstanding and, when factoring in warrants and stock options, a fully diluted share count of approximately 37.0 million.

Conclusion

The countdown is on to the topline data release for the Phase 3 ALS trial, with those results expected before the end of November 2020. Even with the great run the stock has had since its recent lows in March 2020, we continue to view the shares as undervalued as we currently forecast peak sales for NurOwn of >$1 billion in ALS, >$500 million in MS, and >$2 billion in Alzheimers disease. We recently made a slight change to our model, in which we decreased the discount rate from 17% to 13%, and combined with the recent financing activity has resulted in an increase to our valuation to $33 per share.

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BCLI: Phase 3 ALS Data Expected by the End of November 2020 - Yahoo Finance