Stem Cell Clinic

AgeX Therapeutics and Juvenescence Publish Paper on Engineering Strategies for Universal Cells and Provide in Vivo Observation on Immunotolerance…

ALAMEDA, Calif. & DOUGLAS, Isle of Man--(BUSINESS WIRE)--

AgeX Therapeutics, Inc. (AgeX; NYSE American: AGE), a biotechnology company developing therapeutics for human aging and regeneration, and Juvenescence, a life sciences company developing therapeutics and technologies to treat diseases of aging and to increase human longevity, announce the publication of a new paper in the peer-reviewed scientific journal Regenerative Medicine. The paper is on the engineering of allogeneic cells to be hypoimmunogenic (universal), so as not to produce an immune response. The strategies reviewed in the paper include deletion of human leukocyte antigen (HLA) class Ia/II proteins, expression of HLA class Ib molecules, and manipulation of immune checkpoints.

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In addition, the paper presents a previously unpublished in vivo observation on allogeneic human pluripotent stem cells (hPSCs) modified with AgeXs proprietary immunotolerance technology, UniverCyteTM. In humanized mice (those with a functional human immune system), UniverCyte-positive hPSCs formed larger and heavier tissue compared to controls. This observation provides support for the premise that UniverCyte-expressing tissue was potentially hypoimmunogenic and might have escaped recognition by a functional human immune system and continued to grow. Further work is required to substantiate this preliminary in vivo finding.

Hypoimmunogenic allogeneic cells are the Holy Grail in regenerative medicine, and a number of accomplished researchers have made great strides toward engineering them over the last few years, commented Dr. Nafees Malik, Chief Operating Officer at AgeX, Head of Cell & Gene Therapy at Juvenescence (a major investor in AgeX), and lead author on the paper. This is a huge area of focus for us at AgeX, via our UniverCyte technology platform. In support of our own research and as a service to the overall field, we decided to put together this paper, analyzing all the leading strategies to engineer universal cells and encapsulating them in one paper.

Dr. Maliks co-authors on the paper are Gregory Bailey, MD, Chairman of the Board of Directors of AgeX and CEO of Juvenescence; Annalisa Jenkins, MBBS, FRCP, who serves on the Board of Directors of AgeX; and Jim Mellon, Chairman of Juvenescence.

Mr. Mellon added, AgeXs UniverCyte technology platform will not only be important to the company in developing in-house therapies, it may also be transformative for the wider cell therapy industry via collaborations and licensing deals. It is quite conceivable that in the near future, allogeneic cell therapies may potentially need to be universal to be clinically and commercially competitive.

AgeX is developing its UniverCyte technology platform at its new 15,700-square-feet R&D facility, in the San Francisco Bay Area, which has current good manufacturing practices (cGMP)-capable manufacturing capacity.

Universal cells would help us and others to fulfill the original vision of cell therapy, said Dr. Bailey. Thus, I am pleased that my colleagues at AgeX and Juvenescence have put together this paper, as it should be of considerable benefit to researchers, possibly enabling them to accelerate their progress. He added, AgeXs UniverCyte technology uses a novel, modified form of the tolerogenic molecule HLA-G, which in nature plays a key role in preventing a mother from rejecting her semi-allogeneic baby.

The paper is being published online ahead of print on Wednesday, October 30, 2019. It may be found here.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a whole host of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform named induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies and slowly release iTR molecules in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to form licensing and partnership agreements around its broad IP estate and proprietary technology platforms for non-core clinical applications.

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For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Juvenescence

Juvenescence is a life sciences company developing therapies to increase healthy human longevity. It was founded by Jim Mellon, Dr. Greg Bailey and Dr. Declan Doogan. The Juvenescence team are highly experienced drug developers, entrepreneurs and investors with a significant history of success in the life sciences sector. Juvenescence will create, partner with or invest in new companies with longevity-related therapeutics, by in-licensing compounds from academia and industry, or forming joint ventures to develop therapeutics for longevity. Juvenescence believes that recent advances in science have greatly improved our understanding of the biology of aging and seeks to develop therapeutics with the possibility of slowing, halting or potentially reversing elements of aging.

Forward-Looking Statements

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in AgeXs reports filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

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AgeX Therapeutics and Juvenescence Publish Paper on Engineering Strategies for Universal Cells and Provide in Vivo Observation on Immunotolerance...

Washington People: Benjamin D. Humphreys | The Source – Washington University Record

Benjamin D. Humphreys hobbies include experimenting with international cuisine in his kitchen, harvesting heirloom tomatoes in his backyard, and growing miniature kidneys in his laboratory.

He has been perfecting his first two hobbies for years. However, his interest in growing tiny kidneys specifically, using human stem cells to cultivate kidney organoids began in earnest in 2016, shortly after he was named director of theDivision of Nephrologyin theDepartment of Medicineat Washington University School of Medicine in St. Louis.

It still amazes me that we can do this, said Humphreys, MD, PhD, a notednephrologist whose labis funded by the National Institutes of Health (NIH). This is the sort of cutting-edge scientific innovation that made me want to come here. Organoids are great models of kidney development and, potentially, kidney disease and transplant. Theres tremendous potential.

Lab-grown organoids may one day help repair damaged kidneys or be used to test drugs aimed at fighting kidney failure, a growing problem in the United States, with more than 100,000 new cases annually. The potentially fatal condition affects 37 million Americans, most of whom dont realize they have chronic kidney disease. No cure exists, and current treatments for end-stage disease mostly are limited to costly kidney transplants and dialysis.

Ben Humphreys is an innovative and distinguished global leader in kidney research, said Victoria J. Fraser, MD, head of the Department of Medicine and the Adolphus Busch Professor of Medicine. Not only is he a brilliant physician-scientist, he is a generous collaborator and mentor.

Humphreys earned a bachelors degree in English and American literature in 1991 from Harvard College in Cambridge, Mass., and his medical degree and PhD in physiology and biophysics in 2000 from Case Western Reserve University in Cleveland. He completed an internal medicine residency at Massachusetts General Hospital in 2002 and a nephrology fellowship at Brigham and Womens Hospital in 2005. He was an associate professor at Harvard Medical School before joining the faculty at Washington University, where he is the Joseph Friedman Professor of Renal Diseases in Medicine.

What most interests you about the kidney?

The kidney is a fascinating, resilient organ. One of its characteristics that my lab studies is kidney regeneration. When a kidney suffers an acute injury, which might occur if you are ill and lose a lot of blood, the kidney has a powerful capacity to repair itself. Our hope is to harness this trait for therapeutic uses.

The kidney also is complex. The measure of its complexity is the number of different cell types that comprise it. There are more than 35 distinct cell types in a human kidney. Its the bodys second most complex organ, behind the brain. However, the kidneys function is simple. Its a highly tuned responsive filter that dictates whether sodium, potassium, water and other substances need to be reabsorbed or secreted to keep the body perfectly in balance.

Read Humphreys full profile on the School of Medicine site.

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Washington People: Benjamin D. Humphreys | The Source - Washington University Record

Ranking the Top 10 Biotech Clusters in Europe – BioSpace

Although Boston and San Francisco are the biggest biotech clusters in the United States, Europe boasts its own set of hot spots for biopharma development. Global management consulting firm McKinsey & Company recently published, Biotech in Europe: A strong foundation for growth and innovation.

The report notes, Biotech is entering its next S-curve of growth as companies work to transform an array of innovationsgene therapies, stem-cell treatments, antisense DNA, siRNA, CAR-Tinto powerful new therapeutic tools. Yet more scientific and technological breakthroughs are on the horizon. Europes role in this industry continues to grow, and the region leads in many ways. However, European companies have an opportunity to play an even stronger role in the growth of an important and dynamic industry.

One of the biggest differences between the U.S. biotech industry and Europe is the size of late-stage financing. Biotech companies in Europe tend to launch initial public offerings in the U.S. because they are three times bigger on the Nasdaq than on European exchanges. From 2012 through August 2019, when the report was published, about a third of European biotechs that filed for an IPO did so directly on U.S. exchanges, while 98% of follow-on launches by European biotech companies were in U.S. markets.

Genetic Engineering & Biotechnology News ranked the European clusters on the basis of five criteria: patents, venture capital, public research funding, number of biotech companies, and jobs. Heres a look at the top 10.

#1. United Kingdom. Although currently in flux with yet another Brexit deadline on October 31, the UK ranks at the very top in public funding, with 7,981 Horizon 2020 grants and 2,153 biopharma companies according to Bioscience and Health Technology Statistics 2018, which was published in May 2019. It ranks second in biopharma jobs, with about 121,000, and fourth in patents, with 276 granted and 549 applications in 2018. In October, Oxford, UK-based Summit Therapeutics released new data to explain a link between the data from its Phase II trial of ridinilazole for C. difficile infection. The drug showed superior efficacy compared to vancomycin.

#2. Germany. Germanys tax structure and government bureaucracy are generally viewed as obstacles for biotech startups, but the Federal Ministry of Education and Research (BMBF) launched a stimulus program in 2005 dubbed GO-Bio. Some of the Germany biotech companies that started in the country include BioNTech, which launched its IPO on the Nasdaq on October 10, 2019 aimed at raising $150 million. And in September Hamburg-based Evotec partnered with Japans Takeda Pharmaceutical to develop at least five drug discovery programs across a broad range of indications.

Richard Mason, co-founder of the Foundation Institute for 21st Century Medicine, told Pharmaceutical Market Europe, While Germany has produced far fewer biotech companies than you would expect, its definitely the place to watch.

#3. France. On September 19, 2019, during France Digitale Day 2019, President Emmanuel Macron announced plans for a 5 billion-euro ($5.5 billion U.S.) development fund for early-stage digital health and other technology companies. This is part of Macrons pledge when taking the reins of the country in 2017 to make France a start-up nation. In terms of biotech, Paris-based Cellectis, which focuses on allogeneic off-the-shelf CAR-T cells for cancer treatment, on October 1 signed a manufacturing deal with Swiss-based Lonza for Cellectis UCART product candidates for hematological malignancies.

#4. Spain. Spain shows up ninth in patents, with 95 granted and 299 applications in 2018, but fourth in number of companies, 713, and jobs, 67,716. Its even stronger in public funding, with 6,154 grants. In September, the Spanish Association of Biotech Companies (ASEBIO) held an Investor Day event and investors from nine countries attended to discuss investing with representatives from 280 companies. Recently, Minoryx Therapeutics, based in Spain, completed recruitment for its Phase II clinical trial of its PPAR agonist leriglitazone (MIN-102) in Friedreichs Ataxia. The FRAMES trial will treat 39 patients from four European countries.

#5. The Netherlands. As the result of Brexit, the European Medicines Agency (EMA) relocated its headquarters from London to Amsterdam, the Netherlands, in January 2019. That was a pretty big deal by itself, but in August 2019, the Netherlands Foreign Investment Agency (NFIA) indicated that 98 companies were so concerned over Brexit that they were moving to the Netherlands. In early October, Netherlands-based Prepscan announced it had doubled its GMP production capacity, which will allow it to guarantee short production timelines. Its product line consists of a new synthesizer, a preparative high-pressure liquid chromatograph (HPLC) and lyophilizer in the cleanroom. A lyophilizer is basically a freeze dryer that removes water from perishable materials.

#6. Switzerland. It probably shouldnt be too big a surprise that the home of Roche and Novartis would have a good-sized presence in biopharma. It ranks highest in patents, third with 341 granted and 925 applications in 2018. It is second in venture capital. However, it ranked eighth in research funding with 2,544 grants and ninth in jobs, 14,319. In October, Swisss Haselmeier and Italy-based Stevanato Group inked an exclusive deal to license the Axis-D pen-injector technology and intellectual property (IP) for the development, manufacture and supply of the Axis-D pen-injector in the Therapeutic Area of Diabetes Care. Also in that period, Switzerland-based Galderma announced data from its Phase II clinical trial of its botulinum toxin, QM1114 for glabellar lines, which are frown lines. It is going forward with its plans for a Phase III trial.

#7. Italy. Italy shows up fifth in research funding with 5,434 grants as well as fifth in venture capital, with about $172 million in funding. It runs seventh in patents, with 177 granted and 256 applications in 2018. In addition to the previously mentioned deal between Stevanato Group and Haselmeier, Milan-based Genenta Science Thermo recently raked in $14.4 million in private funding to support two ongoing Phase I/II clinical trials for Temferon, its oncology stem cell therapy. One trial will focus on solid tumor glioblastoma multiforme while the other will test the drug on multiple myeloma patients.

#8. Belgium. In July, Gilead Sciences indicated it planned to invest $5.1 billion in Belgiums Galapagos, almost double its minority stake in the company. The investment is part of a 10-year global research-and-development deal to develop Gileads arthritis drug filgotinib and co-develop the rest of Galapagos pipeline. In May, Promethera Biosciences, which has offices in Mont-Saint-Guibert, Belgium, dosed the first patient in a Phase IIa trial of its HepaStem in patients with late-stage nonalcoholic steatohepatitis (NASH). The PANASH trial will evaluate the safety and preliminary efficacy of the drug, which is an allogeneic cell-based therapy for severe liver diseases.

#9. Denmark. When it comes to Denmark and drug development, most would think of Novo Nordisk, one of the dominant players in the diabetes market. However, the country has 536 biopharma companies, placing fifth, and ranks sixth in patents, 156 granted and 465 applications in 2018. On October 9, Novo Nordisk entered a collaboration partnership with Cambridge, Massachusetts-based bluebird bio to develop next-generation genome editing therapies for genetic diseases, including hemophilia A. On September 25, Denmark-based Bavarian Nordic A/S became the first company to win approval by the FDA for a vaccine to prevent smallpox and monkeypox disease for adults at high risk who are viewed as bioterrorism threats.

#10. Sweden. Rounding out the list, Sweden launched an effort to increase life sciences in the country by creating a government Office of Life Sciences. A November 2018 report called the Life Sciences Road Map urged the country to broaden its efforts into digital healthcare and data and precision medicine. The country ranks sixth in number of companies, with 566. It hits number six in jobs, with 40,000, and ninth in public research funding with 2,380 grants. In October, in addition to Swiss-based Galdermas news about its botulinum toxin trial, the company received approval from the Swedish Medical Products Agency to build a new state-of-the-art manufacturing plant at the Center of Excellence located in Uppsala, Sweden. It is designed to manufacture QM1114 and to meet what it believes will be increasing worldwide demand.

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Ranking the Top 10 Biotech Clusters in Europe - BioSpace

Second Fetal Surgeon Joins The Chicago Institute for Fetal Health – Newswise

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Newswise Amir Alhajjat, MD, a specialist in fetal surgical intervention, recently joined The Chicago Institute for Fetal Health (CIFH) and the Division of Pediatric Surgery at Ann & Robert H. Lurie Childrens Hospital of Chicago. He has been appointed as an Assistant Professor of Surgery at Northwestern University Feinberg School of Medicine.

Dr. Alhajjat recently completed a two-year fellowship in Pediatric Surgery at Phoenix Childrens Hospital/Mayo Clinic in Arizona and a one-year fellowship in Fetal Surgery at Cincinnati Childrens Hospital. He completed his residency in General Surgery at the University of Iowa.

Dr. Alhajjats addition will be a real boost to The Chicago Institute for Fetal Healths clinical team, said Aimen Shaaban, MD, Director of the Institute. The expertise and skills that he brings will allow us to advance several innovative new programs to care for an increasingly broader group of patients. Dr. Alhajjat also has a strong interest in minimally invasive newborn surgery.

In his research laboratory, Dr. Alhajjat will study the immune response to in-utero stem cell transplantation and maternal-fetal immune tolerance during normal and complicated pregnancy.

The Chicago Institute for Fetal Health is a leader in the research and care of pregnant women with fetal complications. As one of only a few comprehensive fetal centers in the country, the Institute offers prenatal counseling, care and intervention.

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Second Fetal Surgeon Joins The Chicago Institute for Fetal Health - Newswise

Vertex Reports Third-Quarter 2019 Financial Results – Business Wire

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the third quarter ended September 30, 2019 and reiterated its full-year 2019 total product revenue guidance.

"2019 has been a year of significant progress for Vertex across all parts of our business. With the historic approval of TRIKAFTA, we are now one step closer to providing treatment for up to 90% of all people with CF. We've also had tremendous success bringing our CF medicines to more patients globally with reimbursement agreements recently reached in England, Spain, Australia, and Scotland, and through label expansions to younger patients," said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. "The company also continues to successfully execute on our strategy of creating transformative medicines for serious diseases through serial innovation. The rapid progress of our pipeline is expected to yield proof-of-concept data in multiple diseases in 2020, which will position Vertex for continued growth in the years ahead."

Third-Quarter 2019 Financial Highlights

Three Months Ended September 30,

%

2019

2018

Change

(in millions, except per share amounts)

Total product revenues, net

$

950

$

783

21%

KALYDECO

$

249

$

246

ORKAMBI

$

297

$

282

SYMDEKO/SYMKEVI

$

404

$

255

GAAP Operating income

$

99

$

206

(52)%

Non-GAAP Operating income

$

403

$

295

37%

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Vertex Reports Third-Quarter 2019 Financial Results - Business Wire

Meet University of Arizona Arthritis Center researchers – Jewish Post

Conquering Arthritis Meet the University of Arizona Arthritis Center Researchers will be presented Wednesday, Nov. 6, 6-7:15 p.m., at the Health Sciences Innovation Building on the UA Health Science campus, 1670 E. Drachman St., Tucson.

This event features a look into the future of care, prevention, and ultimately a cure, for this debilitating disease. A panel discussion with UArizona Arthritis Center Director C. Kent Kwoh, MD, pain management specialist Mohab Ibrahim, PhD, MD, and mind-body medicine pioneer Esther Sternberg, MD, will follow the researcher open house and poster displays.

The U.S. Centers for Disease Control and Prevention estimate nearly 55 million Americans have some form of arthritis, including almost half of those over age 65. Arthritis affects more women than men and can affect children as young as 6 months old. It is the leading cause of disability in the United States.

The UArizona Arthritis Center is Arizonas only multi-disciplinary center of excellence dedicated to research and education into the causes, treatments and eventually a cure for arthritis. The center conducts basic, translational and epidemiological research to understand why patients get arthritis, the risk factors for who gets arthritis and analyzes the outcomes to understand how arthritis impacts the patients quality of life.

Featured UArizona Arthritis Center researchers who will present at the event include:

Research topics will include:

Seating for the lecture is limited and prior registration is requested. For more information or to register, visit the UArizona Arthritis Center website, arthritis.arizona.edu, or call 520-626-5040 or email [emailprotected]

Free parking is available after 5 p.m. in the Lot Specific 2012 parking lot next to the Health Sciences Innovation Building and the Lot Specific 2147 parking lot across the street on Cherry Avenue between Helen and Mabel Streets, as well as in all Lot Specific parking lots on the UArizona Health Sciences campus and the Health Sciences Garage (formerly the Banner University Medical Center Tucson Visitor/Patient Parking Garage) at 1501 N. Campbell Ave. For disabled parking, or drop off location next to the Health Sciences Innovation Building, please email [emailprotected], or call 520-626-5040.

If you have questions concerning access, wish to request a Sign Language interpreter or disability-related accommodations, contact Tracy Shake, 520-626-5040, email: [emailprotected]

The lecture is part of the Living Healthy with Arthritis series of free monthly talks presented by the UArizona Arthritis Center at the UArizona College of Medicine Tucson and supported through the Susan and Saul Tobin Endowment for Research and Education in Rheumatology.

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Meet University of Arizona Arthritis Center researchers - Jewish Post