California's ballot measures often reveal much about the broader U.S. policy environment. This is particularly true of the approval by the state's voters in November of Proposition 14, The California Stem Cell Research, Treatments, and Cures Initiative of 2020. Proposition 14 extends the 2004 ballot Proposition 71, which established the California Institute for Regenerative Medicine (CIRM) and authorized $3 billion in state-issued bonds for CIRM to fund stem cell and regenerative research and medicine (restricted to California). Proposition 14, which authorizes $5.5 billion over the next 10 years to continue CIRM's work, succeeded in part by informing voters of CIRM's successes and that its conflict-of-interest provisions are extremely strong. This state-level action is critical because, contrary to opponents' opinions, the overall policy environment for human stem cell research in the United States is in some ways worse now than when Proposition 71 passed.

Since 2004, CIRM has funded groundbreaking work on immune disorders, cancer, spinal cord injury, diabetes, and more. The result has been more than 90 stem cellrelated clinical trials (directly or indirectly supported by CIRM), almost 3000 scientific papers, and contributions to two cancer therapies approved by the U.S. Food and Drug Administration. The lives of many patients have improved because of CIRM. Notably, many CIRM-funded clinical trials rely on human embryonic or fetal stem cells, whereas the federal government currently does not fund any clinical trials using these types of cells.

Proposition 71 was motivated largely in response to restrictions on human embryonic stem cell research in the United States in 2004. However, although research was limited to a small number of human embryonic stem cell lines, there was no formal ban on federal funding of research on such stem cells. In addition, in 2004 there were no restrictions on federal funding of human fetal stem cell and tissue research; however, there is now near-complete blockage of federal funding for such research. And federal funding for human embryonic stem cell research is again at risk. On 4 September 2020, 22 Republican senators and 72 Republican House members wrote to President Trump requesting an end to all federal funding of human embryonic stem cell research. Could President Trump impose a ban that would be difficult to revoke? Or, could Republican senators manufacture a ban by legislative maneuvering on a budget reconciliation vote, which requires 60% support? Such maneuvering created the effectively permanent 1995 Dickey-Wicker amendment, which prohibits federal funding of any research in which human embryos are created or destroyed. Dickey-Wicker has limited research on in vitro fertilization methods and stalled progress on understanding early human development. It has not solved the problem of the many, perhaps 1 million frozen embryos in the United States that will not be used for in vitro fertilization and will be destroyed without benefit if not used for research. Vital long-term research is greatly harmed by the U.S. policy environment, with the likely outcome that many young scientists will avoid research using human embryonic stem cells and human fetal tissue.

Restrictions on valuable, ethical research appear particularly fool-hardy during a deadly pandemic. Research on viruses such as HIV and SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2) can benefit greatly from work using mice that utilize human fetal stem cells and tissues to generate a human-like immune system. These mice allow evaluation of a human immune system in the contexts of infection mechanisms, generation of immunity, and drug response. These studies can be supported with Proposition 14 funds in California, but not with federal funds. It is crucial for the incoming Biden administration to evaluate the need for federal funding in these important areas with high-quality scientific input and evidence.

California's vote on Proposition 14 should also help the rest of the country appreciate the need to increase investments in biomedical research at the U.S. National Institutes of Health and other federal agencies. Current biomedical research expenditures amount to only a tiny fraction of the costs of disease, so an objective evaluation of appropriately increased research funding relative to disease costs is warranted. Once again, California is showing the way.

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Stem cells on the ballot - Science Magazine

Special delivery: Stem cells can be modified to produce a therapeutic protein in the brain.

Laguna Design / Science Photo Library

Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.

Researchers presented the work virtually on Monday and Tuesday at the 2021 Society for Neuroscience Global Connectome.

One of the biggest hurdles for targeted gene therapy is getting enough treatment to the right spot. In the first study, researchers overcame this obstacle by developing stem cells that produce a therapeutic protein inside the brain.

The team is using the approach to develop a treatment for Angelman syndrome, which is caused by mutation in or deletion of the maternal copy of the gene UBE3A. Because the paternal copy of the gene is typically silent, loss of the maternal copy results in an absence of UBE3A protein. People with Angelman syndrome usually have intellectual disability and motor impairments, and many are autistic.

The researchers had previously used modified stem cells to produce a protein that can activate the paternal copy of UBE3A. Transplanting the cells into the brains of Angelman syndrome model mice boosts levels of UBE3A protein, they found. However, the treatment required multiple direct injections into the animals brains.

In the new work, they instead tried injecting the cells into a pocket of cerebrospinal fluid at the base of the skull an approach that is less invasive and can be performed multiple times. They compared the results with direct injection into the animals hippocampus. In both cases, the mice had UBE3A expression in the brain for up to three weeks.

Mice that received direct injection of the stem cells had fewer Angelman syndrome traits than controls, as measured by their motor skills.

This suggests that though the new route is effective, it may not provide a high enough dosage, says Peter Deng, a postdoctoral researcher inKyle Finkslab at the University of California, Davis, who presented the work. And because the transplanted cells produce protein for only a limited period of time, the effects are temporary a limitation the team is addressing.

Deng and his colleagues also found that monkeys treated with the stem cells had the therapeutic protein throughout their brain and spinal cord three weeks after injection, which suggests the approach has potential for treating people.

The second approach presented at the conference improves the delivery of a more permanent form of gene therapy that uses adeno-associated viruses (AAVs).

Researchers typically inject these viruses directly into the brain, and the viruses usually only affect cells immediately surrounding the injection site.

Youre required to use a ton of the virus to penetrate the whole brain, says Jerzy Szablowski, assistant professor of neuroengineering at Rice University in Houston, Texas, who presented the work.

One potential workaround is to inject the AAV into the blood and use focused ultrasound to temporarily open up the blood-brain barrier, allowing the AAV to cross into the brain. Sometimes with this approach, however, the virus also inserts itself into other organs.

In their new work, the team developed AAVs that more easily cross the blood-brain barrier and more selectively target neurons than previous versions do. As a result, the new AAVs can be given in lower doses, reducing the amount of tissue affected outside the brain, Szablowski says.

To identify the most efficient AAV, Szablowski and his colleagues designed 2,100 new viruses, injected them all into the bloodstream of mice and applied focused ultrasound to the animals skulls. The mice had been engineered so that AAVs that successfully inserted themselves into a neuron got tagged with a marker. The team performed genomic sequencing on the mouse brains a few weeks later and read out the levels of viruses.

Compared with the previously most effective AAV, the top five newly identified AAVs targeted twice as many cells in the brain (including more neurons), and nearly half as many cells outside the brain, the researchers found.

The approach could be used to more efficiently deliver treatments for conditions such as Angelman syndrome or Parkinsons disease, the team says.

Read more reports from the 2021 Society for Neuroscience Global Connectome.

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New gene therapy methods deliver promise - Spectrum

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Better Choice Company Announces Strategic Investment of $2.7 Million, Co-Lead by Insiders and Management

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WOBURN, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Yield10 Bioscience, Inc. (Nasdaq:YTEN), an agricultural bioscience company, today announced successful field testing of prototype lines of the oilseed Camelina sativa that have been programed to produce PHA bioplastics directly in seed.

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Yield10 Bioscience Announces Achievement of Proof-of-Concept Milestone for Producing PHA Bioplastic in Field Grown Camelina Plants

PHILADELPHIA, PA, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Vallon Pharmaceuticals Inc., (“Vallon” or the “Company”), a biopharmaceutical company focused on the development of novel drugs for CNS disorders, today announced the presentation of pharmacokinetics (PK) and pharmacodynamic (PD) data from two studies evaluating its investigational new drug, Abuse Deterrent Amphetamine Immediate Release (ADAIR), at the 2021 American Professional Society of ADHD and Related Disorders (APSARD) Annual Meeting which was held virtually January 15-17, 2021.

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Vallon Pharmaceuticals Announces Presentation of ADAIR Pharmacokinetics and Pharmacodynamic Data at the Virtual 2021 APSARD Annual Meeting

FLORHAM PARK, N.J., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a late clinical-stage biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases, today announced that it has completed patient enrollment in PHALCON-HP, a pivotal Phase 3 clinical trial of vonoprazan in combination with amoxicillin (vonoprazan dual therapy) and vonoprazan in combination with amoxicillin and clarithromycin (vonoprazan triple therapy) for the eradication of H. pylori infection. Phathom continues to expect topline results from the study in the second quarter of 2021.

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Phathom Pharmaceuticals Announces Completion of Patient Enrollment in Pivotal Phase 3 Helicobacter pylori (H. pylori) Trial

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Tarsus Pharmaceuticals, Inc. Appoints Bryan Wahl, MD, JD as General Counsel

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GAITHERSBURG, Md., Jan. 19, 2021 (GLOBE NEWSWIRE) -- NexImmune, a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to employ the body’s own T cells to generate a specific, potent, and durable immune response that mimics natural biology, today announced the appointment of Grant Verstandig to its Board of Directors.

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NexImmune Appoints Healthcare Innovator Grant Verstandig as New Board Member

SAN CARLOS, Calif., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, today announced that senior leadership plans to present at the following virtual healthcare conferences:

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Iovance Biotherapeutics to Present at Upcoming Healthcare Conferences

– B-SIMPLE4 pivotal Phase 3 study in molluscum contagiosum reaches 90% enrollment with topline data targeted for Q2 2021 –

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Novan Provides Enrollment Update and Announces New Corporate Headquarters

DOYLESTOWN, Pa., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Neuropathix, Inc. (“Neuropathix” or the “Company”) (OTCQB: NPTX), a socially responsible pain management life sciences company, announced the publication of its global WIPO/PCT Patent WO 2020/ 264324 – “Use of Certain Phosphatidylcholines Containing Long Chain Polyunsaturated Fatty Acids (“PUFAs”) as Neuroprotective Agents” (the “PUFA Patent”).

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Neuropathix, Inc. Announces Publication of Its Global PCT Patent for NPTX-204, a Novel Hops-Inspired Neuroprotectant

SUNNY ISLES, Fla., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Bespoke Extracts, Inc. (OTC Pink: BSPK), producer of high quality, hemp-derived CBD products, today announced that it has entered into an amendment with the holders of the Company’s original issue discount convertible debentures with an original issuance date of December 24, 2019 and subsequently amended on May 28, 2020, August 21, 2020 and December 10, 2020, in the aggregate outstanding principal amount of $500,000. Pursuant to the latest amendment (No. 4), the conversion price of the debentures was increased from $0.001 to $0.05 per share, subject to adjustment for stock splits, stock dividends and similar transactions.

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Bespoke Extracts Increases Conversion Price of Outstanding Discount Convertible Debentures

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Capricor Therapeutics Announces Publication Demonstrating Methods for Enhanced Potency of Cardiosphere-Derived Exosomes

EAST SETAUKET, NY, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Lixte Biotechnology Holdings, Inc. (NASDAQ: LIXT), a clinical-stage drug discovery company developing pharmacologically active drugs for use in cancer treatment, today announced an agreement on a Phase 1b clinical trial with City of Hope, a world-renowned independent cancer research and treatment center. The trial will assess the combination of Lixte’s first-in-class protein phosphatase inhibitor LB-100 with a standard regimen for untreated, extensive stage-disease small cell lung cancer (ED-SCLC).

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Lixte Biotechnology and City of Hope to Initiate Phase 1b Clinical Trial of Lixte’s Lead Compound LB-100 to Treat Small Cell Lung Cancer

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Theratechnologies Announces Closing of US$46 Million Bought-Deal Public Offering, Including Full Exercise of the Over-Allotment Option

NEW YORK, NY, Jan. 19, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- Tauriga Sciences, Inc. (OTCQB: TAUG) (“Tauriga” or the “Company”), a revenue generating, diversified life sciences company, with a proprietary line of CBD & CBG infused Supplement chewing gums (Flavors: Pomegranate, Blood Orange, Peach-Lemon, Pear Bellini, Mint, Black Currant) as well as an ongoing Pharmaceutical Development initiative, today announced that it is constructing a Product Showroom (“Showroom”) inside its new corporate headquarters.  The Company has made this decision in anticipation of and in preparation for important future Agreement(s) & Partnership(s).  The Company expects to host a broad array of prospective new customers, once this Showroom is functional.  These potential new customers include, but are not limited to: Individual Customers, Mom & Pop Stores, Gas Station Convenience Stores, Large Regional & National Retailers, Major Distributors, and College Campus Stores.

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Tauriga Sciences Inc. to Construct Product Showroom Within its New Corporate Headquarters

President and CEO Sanjeev Luther and Co-Chief Medical Officer Tim Pardee, M.D., Ph.D., will discuss clinical trial launches, FDA-granted Fast Track designations and other recent accomplishments on Wednesday, Jan. 20 President and CEO Sanjeev Luther and Co-Chief Medical Officer Tim Pardee, M.D., Ph.D., will discuss clinical trial launches, FDA-granted Fast Track designations and other recent accomplishments on Wednesday, Jan. 20

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Rafael Pharmaceuticals to Present at B. Riley Securities Virtual Oncology Investor Conference