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Trial Shows Same High/Low Dose Aspirin Outcomes

White pills

(Heung Soon, Pixabay,

17 May 2021. Results from a large-scale clinical trial show almost the same health outcomes for patients prescribed high or low doses of aspirin to prevent heart disease. Findings from the six-year study appearing in the 15 May issue of New England Journal of Medicine (registration required) and given at a virtual meeting of the American College of Cardiology, also show the feasibility of conducting lower-cost clinical trials in real-world settings.

Heart disease continues to be a major health problem in the U.S., responsible for some 655,000 deaths, making it the leading cause of death in this country, according to Centers for Disease Control and Prevention. Common aspirin is often prescribed to people with coronary heart disease, where a build-up of cholesterol narrows arteries and interrupts blood flow, yet uncertainty still remains on the optimal aspirin dosage. While aspirin is inexpensive and usually safe, it can still cause bleeding episodes in some patients, thus the dose prescribed can have safety implications.

The Adaptable clinical trial — “Adaptable” is an acronym for a 10-word title — begun in 2015, seeks to produce firm guidance on the optimal aspirin dose for heart disease patients. Given the widespread use of aspirin, the study team designed the trial with individuals already prescribed the drug, rather than recruiting new patients for a separate explanatory trial. This type of study, called a pragmatic clinical trial, aims to capture data from real-world settings instead of a carefully controlled clinical environment.

In this case, more than 15,000 participants with coronary heart disease at 40 sites in the U.S. were randomly assigned by their physicians to continue or start taking either a lower 81 milligram dose or higher 325 milligram dose of aspirin each day; the vast majority of each group (85% to 96%) were already taking aspirin. Researchers led by cardiac disease researcher W. Schuyler Jones at Duke University in Durham, North Carolina tracked participants for four years. The study team looked primarily at combined rates of death from any cause and hospitalizations for heart attack or stroke, as well as other clinical indicators, and reports of major bleeding complications requiring hospitalization.

Several cost-saving measures

The results show combined rates for death or hospitalization for heart attack or stroke are almost identical in each group, with 7.3 percent for lower-dose and 7.5 percent for higher-dose aspirin. The safety results also show nearly identical outcomes with 0.6 percent of recipients in each group hospitalized for major bleeding episodes. The findings are somewhat muddled, however, by large numbers of participants switching doses during the trial, with 42 percent in the higher-dose group switching to the lower dose and seven percent of lower-dose recipients later taking the higher dose.

The Adaptable trial is also notable for its cost-saving measures. The study uses an existing network of clinical trial sites offered by Patient-Centered Outcomes Research Institute, or PCORI, a not-for-profit group studying comparative effectiveness in medical treatments. In August 2020, Science & Enterprise reported on a study using this same network for real-world mental health outcomes from Covid-19. And the study team reduced costs further by enrolling participants online, asking them to report data through a web portal, and extracting data from participants’ electronic health records.

“We learned so many incredible things from Adaptable about how to streamline processes and alleviate the burden of participating in research for both sites and patients,” says Jones in a Duke University statement. Jones also tells Medscape Medical News the trial cost from $18 million to $19 million, while “a typical trial with this many patients conducted in the traditional way would have cost at least five or 10 times more.”

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Infographic – Health Care A.I. Funds Jump in Q1

Health care AI investing

Click on image for full-size view (CB Insights)

15 May 2021. Venture funding for start-ups providing artificial intelligence services in health care climbed to a record high volume in the first quarter of 2021. In the first three months of 2021, venture financing for companies providing A.I. services for health care or using A.I. for product development reached nearly $2.5 billion worldwide, in 111 transactions, according to a report on health care venture capital by technology industry analysts at CB Insights (registration required).

The $2.494 billion raised in the first quarter of 2021 is the fifth consecutive quarterly increase in A.I. venture funding for health care, beginning in Q1 2020. The 111 deals is up slightly from the 102 transactions in the previous quarter. CB Insights says venture funding rounds of $100 million or more dominated the first quarter deals. These so-called mega-rounds accounted for $1.5 billion of the total.

The mega-round trend for A.I. enterprises in health care appears to be continuing in the second quarter of 2021. As reported by Science & Enterprise since the beginning of April, a company using machine learning to create engineered viruses to deliver gene therapies and a biotech company designing vaccines for viral diseases with A.I. are each raising $100 million in their first or second venture rounds.

Health care overall remains a prime target for global venture capital, according to CB Insights. In the first quarter, health care venture investors placed some $31.6 billion, a record high dollar volume, in more than 1,500 deals, the second largest number of transactions in 12 quarters.

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Medical Devices to Reduce Military Travel Disorders

75th Ranger Battalion

The 75th Ranger Battalion prepares to deploy from Fort Benning, Georgia. (

14 May 2021. Biomedical engineers at university labs are designing medical devices that reduce jet lag and intestinal problems faced by military service members. The devices are being developed under the ADvanced Acclimation and Protection Tool for Environmental Readiness, or Adapter program of DARPA, Defense Advanced Research Projects Agency, part of Department of Defense.

Adapter aims to make it easier for military service members to adjust to new environments when deployed quickly to distant regions. “The Adapter technology will alleviate operational limitations imposed by human physiology for two high priority military needs: sleep and safe sustenance,” says Paul Sheehan, Adapter program manager in a DARPA statement. DARPA expects the initiative to develop implanted devices and swallowed capsules that can sense physiological changes in a service member and dispense therapeutic molecules to help the individual adjust to new time zones and disrupted sleep or reduce intestinal distress from food borne pathogens.

Researchers at Northwestern University in Evanston, Illinois are leading design and development of the time-zone adjustment device. That device is envisioned as an implanted lab-on-a-chip system activated by light and dispensing synthesized peptides that the body produces to regulate sleep cycles. The device also responds to the wearer’s body chemistry to deliver the appropriate peptide dose.

“This control system allows us to deliver a peptide of interest on demand, directly into the blood stream,” says project principal investigator and biomedical engineering professor Jonathan Rivnay in a Northwestern statement. “No need to carry drugs, no need to inject therapeutics and, depending on how long we can make the device last, no need to refill the device. It’s like an implantable pharmacy on a chip that never runs out.”

Creating engineered cells to produce synthetic peptides

The Northwestern team includes specialists from the university’s Center for Sleep and Circadian Biology that studies mechanisms underlying sleep and circadian rhythms and their health impacts. Researchers at Rice University in Houston are designing the wireless implant housing the chip system and creating engineered cells that produce synthetic therapeutic peptides, work led respectively by biomedical engineering professors Jacob Robinson and Omid Veiseh.

“Sleep control is something we can track while we develop this implant,” says Veiseh in a Rice statement, “but the real innovation here is being able to produce drugs inside the patient,” and adding, “If we can bring all of that manufacturing right into the patient and produce high-quality compounds on an as-needed basis, the possibilities are infinite.”

Researchers from Carnegie Mellon University in Pittsburgh and neuroscience technology company Blackrock Microsystems in Salt Lake City, Utah are also taking part in design and development of the time-zone device. That project could bring the researchers as much as $33 million over 4.5 years, if all aspects of the work are completed.

The overall Adapter initiative has two other parts. A team from Stanford University in California is developing an implantable device to combat fatigue, dispensing the hormone melatonin that regulates sleep cycles, on demand for up to 30 days. And researchers from Massachusetts Institute of Technology are developing a capsule that service members can swallow, which travels to the gut and releases compounds to kill food borne pathogens and neutralize their toxins. Science & Enterprise reported in February 2019 on a similar capsule developed at MIT that releases a tiny needle delivering an insulin dose to the stomach lining, equivalent to conventional insulin injections.

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New Biotech Creating Precision Cancer Therapies

DNA analysis graphic

(Gerd Altmann, Pixabay)

13 May 2021. A biotechnology company began work in public to develop new treatments that target proteins responsible for gene expression, beginning with cancer. Flare Therapeutics in Cambridge, Massachusetts, formed by life science investor Third Rock Ventures and operating in stealth mode up to today, is also raising $82 million in its first venture funding round.

Flare Therapeutics creates therapies addressing transcription factors, some 1,600 proteins in the human genome. Transcription factors bind to DNA and activate or limit gene expression, the process of translating genetic codes in DNA to RNA and proteins. Transcription factors are encoded by about 10 percent of human genes and implicated in one-third of cancer-causing genes, as well as one in five inherited diseases from haploinsufficient genes, where a functional copy of the gene is lost. Because transcription factors operate under complex conditions, with multiple proteins and chemical interconnections, they’re difficult to target directly for therapies.

The company’s technology is based on research by its scientific founders, particularly Fraydoon Rastinejad, professor of biochemistry and structural biology at University of Oxford in the U.K. Rastinejad work with one transcription factor revealed a biochemical process and structure in all transcription factors, particularly key pockets with amino acid residues critical to the protein’s structure and integration. These pockets, called switch sites, are targets for small molecule drugs for turning transcription factors on or off.

“Over the past decade,” says Rastinejad in a Flare Therapeutics statement, “there has been a constant flow of scientific discoveries showing evermore pointedly how transcription factors play a central role in diseases, notably cancer. Yet, transcription factors have continued to be elusive for finding targetable sites for drug discovery, with less than one percent of transcription factors successfully targeted for medicines.”

“Clear role” of transcription factors in cancer

The company says it will first apply its switch site technology to precision cancer treatments that address specific cancer-causing mutations rather than the organs where tumors reside. “Our early focus,” says Flare Therapeutics’ chief scientist and co-founder Robert Sims, “is on precision oncology based on the clear role that transcription factors play in cancer, and we look forward to expanding our future drug discovery in other areas such as neurology, rare genetic disorders, immunology, and inflammation.”

Some neurological disorders are associated with haploinsufficient genes. Another of Flare Therapeutics’ co-founders is Steven McKnight, biochemistry professor at University of Texas Southwestern Medical Center in Dallas. McKnight’s work includes studies of transcription factors, including those involving neurodegenerative diseases. As reported by Science & Enterprise in July 2020, McKnight is a scientific co-founder of Nura Bio Inc., a biotechnology company developing therapies for neurodegenerative disorders.

Also among Flare’s scientific co-founders are Mitchell Lazar, who studies transcriptional regulation of metabolism and genetics behind metabolic disorders at University of Pennsylvania, and Stephen Frye, co-director of the Center for Integrative Chemical Biology and Drug Discovery at the University of North Carolina at Chapel Hill.

Flare Therapeutics is raising $82 million in its first venture funding round led by Third Rock Ventures in Boston. Joining the round are Boxer Capital, Nextech Invest, Casdin Capital, Invus Financial Advisors, and Eventide Asset Management. Third Rock partners are filling in as the company’s interim CEO, chief operating officer, and chief people officer.

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Early-Stage Life Science Investment Fund Raises $515M

Growing money

(Nattanan Kanchanaprat, Pixabay)

12 May 2021. A new venture investment fund raised $515 million for life science start-ups, and its business incubator partner added five biotechs to its seed round portfolio. In addition, the current Perceptive Xontogeny Venture fund is leading the first venture round for Juno Diagnostics Inc., a company developing non-invasive prenatal genetic tests.

Perceptive Xontogeny Venture Funds are a collaboration between Perceptive Advisors, an investment management company in New York and Xontogeny LLC, a new-business incubator for life science start-ups in Boston. Perceptive Advisors invests mainly in life science companies throughout their life cycles, with investments in biotechnology companies as well as developers of pharmaceuticals, medical devices, diagnostics, and digital health. The company says its staff includes trained technologists, molecular biologists, and physicians as well as financial analysts.

Perceptive Xontogeny Venture Funds invest mainly in early-stage life science companies, primarily as lead investor in their first venture rounds known as series A, or as seed investors in companies incubated at Xontogeny. The new Perceptive Xontogeny Venture Fund 2 collected $515 million, which the partners say brings the total assets managed by the first and second funds to $725 million, in less than two years. As reported by Science & Enterprise in July 2020, the first Perceptive Xontogeny Venture Fund invested $40 million in Forge Biologics in Columbus, Ohio, a developer and manufacturer of gene therapies.

The first Perceptive Xontogeny Venture Fund is also leading a $25 million first venture financing round for Juno Diagnostics in San Diego. Juno Diagnostics is developing what it calls the next generation of non-invasive prenatal tests for genetic disorders such as Down syndrome. The company says its technology performs the tests without a blood draw, and a lower cost and faster turnaround that conventional cell-free DNA tests. The round’s proceeds are expected to support Juno Diagnostics’ clinical validation studies, product development, and commercial launch of its non-invasive prenatal test.

Xontogeny portfolio additions

Xontogeny is adding five life science start-ups to its business accelerator program. As participants in Xontogeny’s incubator, the companies receive seed capital and strategic support to guide their growth through preclinical stages, and help them qualify for further financing by Perceptive Advisors and others. The new participants are:

Nephraegis Therapeutics Inc. in Lake Forest, Illinois. Nephraegis Therapeutics is developing a compound code-named NPH-022 to prevent acute kidney injury, a condition with 8 million cases per year in the U.S. leading to chronic kidney disease and dialysis for some 80,000 individuals.

NephroDI Therapeutics in Philadelphia. NephroDI Therapeutics develops treatments for kidney disorders, beginning with nephrogenic diabetes insipidus, a genetic disorder that prevents proper kidney functioning in children, requiring them to drink large amounts of water to prevent dehydration. The company is based on research at Emory University.

Peroxitech LLC in Philadelphia. Peroxitech is developing a treatment for acute lung injuries, including those resulting from Covid-19 infections. The company’s technology, based on research at University of Pennsylvania, is producing a peptide that eliminates harmful reactive oxygen molecules building up in the lung that lead to respiratory failure.

Shifa Biomedical Corp. in Malvern, Pennsylvania. Shifa Biomedical is developer of small-molecule drugs to treat dyslipidemia, or excessive cholesterol, that can lead to clogged arteries, heart attacks, and stroke. The company’s lead product, code-named P-21, blocks PCSK9 proteins that regulate low-density lipoprotein cholesterol levels that now often require monoclonal antibodies.

Tellus Therapeutics in Durham, North Carolina. Tellus Therapeutics is a developer of therapies for newborns, starting with compounds derived from breast milk for producing myelin that protects nerve cells in the brain. The company’s lead product, code-named TT-20, repairs white matter injury, a brain condition affecting some newborn infants. Tellus Therapeutics is spun-off from labs at Duke University.

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Synthetic Bio Company Goes Public in $2.5B Merger

Investment graphic

(Gerd Altmann, Pixabay)

11 May 2021. A company developing synthetic biology and engineering tools for biotechnology is becoming a public company through a special-purpose acquisition. Soaring Eagle Acquisition Corp. is merging with Ginkgo Bioworks Inc. in Boston, to take Ginkgo Bioworks public, and raising $2.5 billion in proceeds for the merged company.

Soaring Eagle is a publicly-traded special purpose acquisition company or SPAC, a shell company created to merge with a private business to take that enterprise public, in this case Ginkgo Bioworks. While technically a public company, a SPAC raises funds from private investors to make the acquisition, with Arie Belldegrun, an early cell- and gene-therapy entrepreneur, among the leading investors in this transaction. The merger, in effect, turns the acquired enterprise into a public company, usually in less time and fewer steps than a conventional IPO, or initial public stock offering.

Ginkgo Bioworks is a 13 year-old company developing synthetic biology and engineering tools for biotechnology in medicines, agriculture, and bio-based materials. The company offers synthesized nucleic acids, including DNA, to design microorganisms for producing special-purpose enzymes and other bio-chemicals. Gingo Bioworks says its codebase — a library of cells, enzymes, and genetic programs — helps shortcut the discovery process for generating new bio-engineered products. In addition, Ginkgo Bioworks provides robotics and software, including artificial intelligence, to support its synthetic biology and engineering work.

“The magic of biology is that cells run on digital code similar to a computer, except that instead of 0s and 1s it’s As, Ts, Cs, and Gs,” says Jason Kelly, co-founder and CEO of Ginkgo Bioworks in a company statement released through Cision. “Ginkgo’s platform makes it easier to program this code, and we are making this platform available to organizations working to solve our most pressing problems.”

Several Covid-19 applications

Ginkgo Bioworks collaborates with other companies to produce a variety of synthetic biology products. The company says vaccine developers use Gingo’s technology to create a production process for mRNA vaccines, including Covid-19 vaccines. Ginkgo also began a separate project called Concentric to develop simple Covid-19 tests for schools to use with their students. As reported by Science & Enterprise in September 2020, Ginkgo Bioworks is partnering with Totient, a new company developing synthetic antibodies for Covid-19 treatments.

Ginkgo Bioworks expects to raise $2.5 billion in the deal. Soaring Eagle is providing $1.725 billion in cash, with another $775 million raised from a consortium of investors through a private investment in public equity or PIPE transaction. The consortium is led by Eagle Equity Partners, the private equity company behind Soaring Eagle, and Bellco Capital, a venture investor led by Arie and Rebecka Belldegrun. Arie Belldegrun is an academic medical researcher turned entrepreneur, who founded cell therapy pioneer Kite Pharma, acquired by Gilead Sciences in October 2017, and gene therapy company Allogene.

Joining the consortium are Baillie Gifford, Putnam Investments, and funds and accounts managed by Counterpoint Global, part of Morgan Stanley, accounts advised by ARK Investment Management LLC, ArrowMark Partners, Bain Capital Public Equity, Berkshire Partners, and Franklin Advisers. Also joining are current investors Cascade Investment, Casdin Capital, General Atlantic, Senator Investment Group, funds and accounts advised by T. Rowe Price Associates, Inc., and Viking Global Investors.

Soaring Eagle says Ginkgo Bioworks’ market value now is $15 billion, and expects to generate $150 million in revenues this year, nearly doubling its income from 2020. And from the merger, Soaring Eagle expects to soon change its name to Ginkgo Bioworks Holdings Inc., and trade under a new ticker symbol.

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MD Anderson, Broad Institute Partner on Rare Cancers

Cancer on clipboard

(Nick Youngson,

11 May 2021. MD Anderson Cancer Center and the Broad Institute are developing a new research platform for better understanding rare types of cancer. The partnership aims to create a research system based on models of rare cancers with detailed data about those models to guide design of new therapies to treat rare types of tumors.

The collaboration joins Houston-based MD Anderson’s research on rare cancers with two programs underway at Broad Institute, a genetics research center in Cambridge, Mass. affiliated with Harvard University and MIT. MD Anderson, part of the University of Texas system, studies rare cancers in its Therapeutics Discovery division. That division establishes research platforms, organized systems for studying particular types or aspects of cancer.

In this project, MD Anderson and Broad Institute are establishing a research platform for rare cancers, defined by National Cancer Institute as cancers that occur in fewer than 15 in 100,000 people each year. MD Anderson says rare cancers account for a quarter of all cancer cases and deaths, but because each of these cancer types has a low rate of occurrence, they’re difficult to study and understand. More than 5,000 patients with rare types of cancer, says MD Anderson, seek treatment each year.

Produce more than 100 rare cancer models a year

In the partnership, Broad Institute is offering its Cancer Cell Line Factory, or CCLF, a library of tumor tissue samples converted into cell lines, organoids, or spherical models. Broad Institute says CCLF has 409 cancer models derived from more than 2,300 tissue samples, representing 37 types of cancer. More than one-third (36%) of the models represent rare or pediatric cancers. One of the goals of CCLF is to produce a diverse set of cancer models, particularly from underrepresented groups, which Broad Institute shares with the scientific community.

Models produced by CCLF will then be analyzed by Broad Institute’s Cancer Dependency Map. DepMap, as it’s called, catalogs genetic and pharmacological dependencies for each type of cancer, indicating their vulnerabilities and target biomarkers. DepMap offers analytics for more than 2,000 cancer models, and provides visualization tools. Downloads from Broad Institute’s DepMap portal are available to the public.

“Treatments for rare cancers have lagged behind common tumors,” says William Sellers, director of Broad Institute’s cancer program in a statement, “in large part because we as a community lack the tools to study and understand their unique biology in the laboratory. This initiative represents a significant opportunity to close that gap and to start identifying new treatment options for patients with rare cancers.”

The partnership aims to produce more than 100 rare cancer models each year. “Through this initiative, we hope to overcome some of the challenges that have prevented effective translational research in rare cancers,” adds Timothy Heffernan, head of oncology research in MD Anderson’s Therapeutics Discovery division. “By collaborating with the Broad Institute, we have a tremendous opportunity to create a valuable resource for the entire scientific community that will inspire and catalyze a wave of innovative research to advance impactful new therapies to patients in need.”

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New Biotech Developing Synthetic Long-Term RNA

Avak Kahvejian

Avak Kahvejian (Flagship Pioneering)

10 May 2021. A new enterprise, created by a life science venture capital company, began work on developing therapies with long-acting synthetic RNA. The company Laronde is spun-off from venture capital investor Flagship Pioneering in Cambridge, Massachusetts, staked to $50 million in initial funding.

Laronde designs therapies with a synthetic form of RNA that overcomes drawbacks of messenger RNA, the underlying technology in some of the vaccines to prevent Covid-19 infections. In its natural form, the end of a messenger RNA strand interacts with ribosomes, particles in cells that translate RNA sequences one time from the original genetic code into amino acids for proteins. Another natural type of RNA, called long non-coding RNAs or lncRNAs, form into circular strands in cells that do not interact with ribosomes, and thus do not convert into amino acids.

A Flagship Pioneering team led by its general partner Avak Kahvejian began researching the eRNA idea in 2017. “We asked,” says Kahvejian in a Flagship Pioneering statement, “‘What if the circular nature of certain lncRNAs makes them ultra-stable in the body? Could we benefit from that stability to make a new class of therapeutic by making an RNA that has no free ends, but is translatable?’”

Laronde plans to create treatments for disease with synthetic circular RNA forms designed to interact with ribosomes, but operate continuously to provide therapeutic effects over longer periods of time. The company calls this concept endless RNA or eRNA, with programmable modules plugged into the basic RNA structure for producing amino acid chemistries for specific peptides, antibodies, enzymes, or receptors. The circular design, says the company, also prevents the immune system from reacting to eRNAs, making them more stable and allowing for longer-term therapeutic effects.

Create 100 new drugs in 10 years

Kahvejian adds, “eRNA therapeutics have the potential to be an essential and widespread class of medicines, expanding beyond small molecules and antibodies in their therapeutic applicability and utility. We can program eRNA medicines to code for a wide variety of therapeutic modalities.”

Diego Miralles, a Flagship Pioneering partner and CEO of Laronde notes, “Because the programmable platform is so scalable, we have the potential to parallel process the development of multiple programs at the same time that, if successful, could help millions of people around the world.”

The company expects to create 100 new treatments in the next 10 years, from its ability to scale up the process and produce therapies in parallel. Laronde says it plans to build a manufacturing plant for its treatments and hire some 200 people in the next two years.

“This is a once-in-a-lifetime opportunity to join a company like Laronde, which will advance such a groundbreaking therapeutic platform capable of biological applications we could only dream of a few years ago,” says Miralles, adding “We look forward to recruiting the best talent in the industry to build a great company that will fully realize the potential of eRNA.”

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Infographic – Public Money Funds Covid-19 Vaccines

Chart: Covid-19 vaccine funding

Click on image for full-size view (Statista)

8 May 2021. As the race to produce and distribute Covid-19 vaccines intensifies, financing of this global public health project is starting to emerge. As part of this picture, the business research company Statista published a chart on Thursday showing the source of funds for the world’s vaccines.

Statista reported data compiled by the Knowledge Portal on Innovation and Access to Medicines. That group gathered data on research and development of Covid-19 vaccines through March 2021. The data show public expenditures, particularly in the U.S. and Germany, are the largest R&D funders of Covid-19 vaccines, with Moderna Inc., Janssen, and Pfizer/BioNTech the largest recipients. Moderna and Janssen, a division of drug maker Johnson & Johnson, each secured more than $900 million and Pfizer/BioNTech gained $800 million. These numbers do not include funds for manufacturing scale-up or bulk purchases.

Moderna developed its Covid-19 vaccine with National Institute of Allergy and Infectious Diseases, part of National Institutes of Health, and took part in the U.S. government’s vaccine development initiative, along with Janssen. While the Pfizer/BioNTech vaccine was not part of the U.S. government’s project, the German company BioNTech received support from its home government.

The next tier of vaccine developers — Curevac, Novavax, AstraZeneca/Oxford, and Sichuan Clover — received from a small slice to a majority of funds from Coalition for Epidemic Preparedness Innovations, or CEPI, an organization in Oslo, Norway. The amount of funding provided by CEPI may be less important than its early recognition and support for Covid-19 vaccines that helped get these efforts started. Science & Enterprise reported on CEPI’s early funding for vaccines against the novel coronavirus, as it was called at the time, in January 2020.

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Disclosure: The author owns shares in Pfizer and Johnson & Johnson

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Biotechs Partner on Gene-Edited Cancer Cell Therapies

Natural killer cell

Natural killer cell (NIAID, Flickr)

7 May 2021. Two biotechnology companies are collaborating on using the gene editing technique Crispr to create engineered immune-system cells to treat cancer. Nkarta Inc. in South San Francisco, California and Crispr Therapeutics in Zug, Switzerland aim to co-develop and commercialize gene-edited natural killer cells as cancer therapies.

Nkarta develops engineered natural killer cells from the immune system as off-the-shelf cancer therapies. Like their cousins B- and T-cells, natural killer cells are white blood cells, which act against cells infected by viruses and in tumors. They’re called “natural killers” for their innate ability to seek out and attack pathogens and tumors without priming or prior activation. Nkarta says it further engineers natural killer cells with receptors designed to identify and bind to specific molecular targets, the release cancer-killing cytokine enzymes. The company also adds interleukin-15, a cytokine that induces proliferation of natural killer cells.

To produce its cancer treatments, Nkarta takes natural killer cells from healthy donors, The donated natural killer cells are expanded and engineered to express chimeric antigen receptors or CARs and interleukin-15 on the cell membranes. The cells are then expanded further with interleukin-15 and frozen to preserve for future administration to patients. The company’s lead product, code-named NKX101, is in an early-stage clinical trial with acute myeloid leukemia and myelodysplastic syndrome patients, two forms of blood-related cancer. Another product code-named NKX019 is completing preclinical development as a treatment for B-cell malignancies.

Gene-edited natural killer cells

Crispr — short for clustered, regularly interspaced short palindromic repeats — makes it possible to edit genomes of organisms harnessing bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. At Crispr Therapeutics and in most other cases, the actual editing is done by Crispr-associated protein 9, or Cas9, enzyme that programs RNA to cut DNA at precise points in genomes, making it possible to delete, insert, or correct defects in human genomes.

For cell therapies, Crispr Therapeutics edits genes that improve the safety or efficacy of the treatments, either as immune system cells outside the body then re-infused to the patient, or packaged in nanoscale lipid particles and delivered systemically or into target organs. As reported in Science & Enterprise, Crispr Therapeutics and Vertex Pharmaceuticals are collaborating on a gene-edited treatment for the blood disorders sickle cell disease and beta thalassemia, now in mid-stage clinical trials.

In their project, Nkarta and Crispr Therapeutics will jointly develop two cancer treatments with natural killer cells engineered with CARs. One of those programs targets the CD70 tumor antigen, part of a pathway for regulating T-cells in the immune system and implicated in non-small cell lung cancer. The second program, for an undisclosed target, engineers both natural killer and T-cells to harness both innate and adaptive immunity. In the collaboration, Nkarta and Crispr Therapeutics are equally sharing all research and development costs, and any subsequent profits.

Nkarta is also licensing Crispr Therapeutics’ technology to edit five gene targets, applicable to any number of Nkarta’s natural killer cell products. Nkarta retains all worldwide rights for these targets, with Crispr Therapeutics eligible for undisclosed milestone payments and royalties on sales. The agreement has a three-year exclusivity period between the two companies for R&D and commercialization of off-the-shelf, gene-edited, donor-derived natural killer and combination natural killer/T-cells. Dollar amounts in the agreement were not disclosed.

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