This company is about to grow new organs in a person for the first time – MIT Technology Review

And the lymph nodes near the liver are close enough to receive chemical distress signals sent out by the dying tissue of a diseased liver, says Lagasse. These signals are meant to encourage any remaining healthy liver tissue to regenerate, but this doesnt work in cases of severe disease. However, the signals do appear to help along the growth of liver tissue in neighboring lymph nodes.

Its incredible, says Gouon-Evans. Having this little incubator in the body [that can grow organs] is just amazing.

LYGENESIS

Around five years ago, Lagasse, along with entrepreneur and drug developer Michael Hufford and transplant surgeon Paulo Fontes, founded LyGenesis to take the technology further. The team are exploring the use of lymph nodes to grow new thymuses, kidneys, and pancreases.

But the companys priority is livers. Over the last 10 years, members of the team have collected promising evidence that suggests they can use their approach to grow new mini livers in mice, pigs, and dogs. The mini livers dont grow indefinitelythe body has an internal regulator that stops liver growth at a certain point, which is why healthy livers dont overshoot when they regenerate.

The teams research in mice with a genetic liver disorder has shown that most of the cells injected into a lymph node will stay there but some will migrate to the liver, providing there is enough healthy liver tissue remaining. These migrating cells can help the remaining liver tissue regenerate and heal. When this happens, the new mini liver in the lymph node will shrink, keeping the total amount of liver tissue in balance, says Lagasse.

Other studies have focused on pigs and dogs that have the blood supply to the liver diverted, causing the organ to die. Injecting liver cells into the animals lymph nodes will eventually rescue their liver function.

In the pig study, for example, the team first surgically diverted the blood supply away from the liver in six animals. Once the pigs had recovered from surgery, the team injected healthy liver cells into their lymph nodes. The doses ranged from 360 million cells injected across three lymph nodes to 1.8 billion cells across 18 lymph nodes.

Within a couple of months, all the animals appeared to show recovery from their liver damage. Tests suggested their liver function had improved. And when the team later performed autopsies on the animals, the new organs in the lymph nodes looked very much like miniature healthy livers, each up to around 2% of the size of a typical adult liver. Other studies suggest it takes around three months for the treatment to have significant benefits.

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This company is about to grow new organs in a person for the first time - MIT Technology Review

Global Cell Banking Outsourcing Market to Grow at a CAGR of ~18% during 2022-2031; Market to Expand Owing to the Development of Advanced Cell…

New York, Aug. 23, 2022 (GLOBE NEWSWIRE) -- Kenneth Research has published a detailed market report on Global Cell Banking Outsourcing Market for the forecast period, i.e., 2022 2031, which includes the following factors:

Global Cell Banking Outsourcing Market Size:

The global cell banking outsourcing market generated the revenue of approximately USD 7200.1 million in the year 2021 and is expected to garner a significant revenue by the end of 2031, growing at a CAGR of ~18% over the forecast period, i.e., 2022 2031. The growth of the market can primarily be attributed to the development of advanced preservation techniques for cells, and increasing adoption of regenerative cell therapies for the treatment of chronic diseases such as cancer. Additionally, factors such as growing demand for gene therapy, and increasing worldwide prevalence of cancer are expected to drive the market growth. According to the World Health Organization, nearly 10 million people died of cancer across the globe in 2020. The most recurrent cases of deaths because of cancer were lung cancer which caused 1.80 million deaths, colon, and rectum cancer which caused 916 000 deaths, liver cancer which caused 830 000 deaths, stomach cancer which caused 769 000 deaths, and breast cancer which caused 685 000 deaths. Furthermore, it was noticed that about 30% of cancer cases in low and lower-middle income nations are caused by cancer-causing diseases such the human papillomavirus (HPV) and hepatitis.

Get a Sample PDF of This Report @ https://www.kennethresearch.com/sample-request-10070777

Global Cell Banking Outsourcing Market: Key Takeaways

Increasing Geriatric Population across the Globe to Boost Market Growth

Increasing demand for stem cell therapy, and increasing biopharmaceutical production are estimated to fuel the growth of the global cell banking outsourcing market. Among the geriatric population around the world, the demand of stem cell therapy is at quite a high rate. Hence, growing geriatric population across the globe is also expected be an important factor to influence the market growth. According to the data by World Health Organisation (WHO), the number and proportion of geriatric population, meaning the people aged 60 years and older in the population is rising. The number of people aged 60 years and older was 1 billion in 2019. This number is estimated to increase to 1.4 billion by 2030 and 2.1 billion by 2050.

In addition to this, increasing prevalence of chronic diseases, supportive initiatives by governments around the world, and growing awareness about stem cell banking are predicted to be major factors to propel the growth of the market. The growth of the global cell banking outsourcing market, over the forecast period, can be further ascribed to the rising investments in the R&D activities to continuously bring up more feasible solutions for medical procedures. According to research reports, since 2000, global research and development expenditure has more than tripled in real terms, rising from approximately USD 680 billion to over USD 2.5 trillion in 2019.

Browse to access In-depth research report on Global Cell Banking Outsourcing Market with detailed charts and figures: https://www.kennethresearch.com/report-details/cell-banking-outsourcing-market/10070777

Global Cell Banking Outsourcing Market: Regional Overview

The global cell banking outsourcing market is segmented into five major regions including North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa region.

Advanced Healthcare Facilities Drove Market in the North America Region

The market in the North America region held the largest market share in terms of revenue in the year 2021. The growth of the market in this region is majorly associated with the increasing number of pharmaceutical companies & manufacturers in the region, and increasing awareness for the use of stem cells as therapeutics. Increasing number of bone marrow and cord blood transplants throughout the region is also estimated to positively influence the market growth. It was noted that, 4,864 unrelated and 4,160 related bone marrow and cord blood transplants were performed in the United States in 2020.

Increasing Prevalence of Chronic Diseases to Influence Market Growth in the Asia Pacific Region

On the other hand, market in the Asia Pacific region is estimated to grow with the highest CAGR during the forecast period. The market in this region is driven by the increasing investment in biotechnology sector by government and private companies specifically in countries such as China, India, and Japan. Moreover, the increasing pool of patient with chronic diseases, such as cancer, and the ongoing research & development activities for cancer treatment is expected to propel the growth of the market. Further, increasing percentage of regional health expenditure contributing to the GDP is also estimated to be a significant factor to influence the growth of the cell banking outsourcing market in the Asia Pacific region. As per The World Bank, in the year 2019, share of global health expenditure in East Asia & Pacific region accounted to 6.67% of GDP.

Get a Sample PDF of the Global Cell Banking Outsourcing Market @ https://www.kennethresearch.com/sample-request-10070777

The study further incorporates Y-O-Y growth, demand & supply and forecast future opportunity in:

Middle East and Africa (Israel, GCC [Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of Middle East and Africa).

Global Cell Banking Outsourcing Market, Segmentation by Bank Phase

The bank storage segment held the largest market share in the year 2021 and is expected to maintain its share by growing with a notable CAGR during the forecast period. The market growth is anticipated to be driven by the development of effective preservation technologies such as cryopreservation technique. Cryopreservation is a technique in which low temperature is used to preserve the living cells and tissue for a longer time. With the growing healthcare expenditure per capita across the world, demand for bank storage increasing notably. As sourced from The World Bank, in 2019, worldwide health expenditure per capita was USD 1121.97.

Access full Report Description, TOC, Table of Figure, Chart, etc. @ https://www.kennethresearch.com/sample-request-10070777

Global Cell Banking Outsourcing Market, Segmentation by Product

The adult cell banking segment is estimated to hold a substantial market share in the global cell banking outsourcing market during the forecast period. The growth of this segment can be attributed to the significant prevalence of chronic diseases among the adults around the globe. For instance, according to the National Library of Medicine 71.8% of adult population suffered from cardiovascular diseases, 56% had diabetes, and 14.7% adults had arthritis as of 2020.

Global Cell Banking Outsourcing Market, Segmentation by Cell Type

Global Cell Banking Outsourcing Market, Segmentation by Bank Type

Few of the well-known market leaders in the global cell banking outsourcing market that are profiled by Kenneth Research are SGS SA, WuXi AppTec, LifeCell International Pvt. Ltd., BSL Bioservice, LUMITOS AG, Cryo-Cell International, Inc., REPROCELL Inc, CORDLIFE GROUP LIMITED, Reliance Life Sciences, and Clean Biologics and others. Enquiry before Buying This Report @ https://www.kennethresearch.com/sample-request-10070777

Recent Developments in the Global Cell Banking Outsourcing Market

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About Kenneth Research

Kenneth Research is a leading service provider for strategic market research and consulting. We aim to provide unbiased, unparalleled market insights and industry analysis to help industries, conglomerates and executives to take wise decisions for their future marketing strategy, expansion and investment, etc. We believe every business can expand to its new horizon, provided a right guidance at a right time is available through strategic minds. Our out of box thinking helps our clients to take wise decision so as to avoid future uncertainties.

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Global Cell Banking Outsourcing Market to Grow at a CAGR of ~18% during 2022-2031; Market to Expand Owing to the Development of Advanced Cell...

Myeloid Therapeutics Strengthens Senior Team to Support the Expansion of its Manufacturing Capabilities – PR Newswire

CAMBRIDGE, Mass., Aug. 23, 2022 /PRNewswire/ -- Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage mRNA-immunotherapy company harnessing the power of myeloid and innate biology to engineer novel therapies that elicit a broad immune response for cancer and autoimmune diseases, today announced that it has expanded its manufacturing capabilities with the appointments of Jerome Chal, Ph.D., as Vice President, Process Development, and Eric Chapdelaine as Vice President, CMC Operations.

"We are thrilled to continue to attract the highest levels of talent to Myeloid, as we expand our manufacturing capabilities to support our clinical-stage cell therapy programs and the advancement of our in vivo cell programming programs into the clinic next year," said Daniel Getts, Ph.D., CEO of Myeloid. "Jerome and Eric bring a diverse set of skills with significant quality and CMC experience at leading companies like Vertex, Alnylam, and Genzyme. As senior leaders, they will provide invaluable stewardship as we advance and expand our pipeline and read out more clinical data. We are thrilled to welcome them to the Myeloid team, and we look forward to their contributions to our technology and the cell therapy field generally."

Jerome Chal, Ph.D., Vice President, Process Development

Dr. Chal brings to Myeloid over twenty years of experience in the biotechnology industry, with a focus on CMC leadership across multiple therapeutic modalities, including cell and gene therapies, with landmark achievements in the field. Prior to Myeloid, Jerome was Executive Director, Analytical Sciences at Vertex Pharmaceuticals, where he provided strategic leadership to a multi-site and multi-department CMC organization across the T1D, blood disorders and DMD programs, along with end-to-end implementation of all analytical CMC activities from preclinical to pivotal stage. Prior to Vertex, Dr. Chal was Associate Director, Cell and Analytical Development at Semma Therapeutics, where he directed cell, process and assay development programs for cell therapy products based on a proprietary preclinical-stage PSC-based pancreatic islets (SC-islets) platform. Earlier in his career, he held positions of increasing responsibility at Coyne Scientific, IGBMC in Strasbourg, France, and faculty scientist at Brigham and Women's Hospital, Harvard Medical School. Dr. Chal received his MS in Biology-Biochemistey from ENS Paris, Paris 7 University, an MS in Molecular and Cellular Developmental Biology from Sorbonne University, and a Ph.D. in Stem Cell and Developmental Biology from Sorbonne University/Stowers Institute, Kansas City, MO. Dr. Chal is a decorated scientist with multiple patents and over twenty peer-reviewed publications in the fields of cell and gene therapy.

Eric Chapdelaine , Vice President, CMC Operations

Mr. Chapdelaine has over twenty years of experience leading Manufacturing, CMC, Quality (QC+QA), Analytical Development, and Regulatory Affairs organizations in cell and gene therapy. Eric has overseen and led IND and NDA filings for multiple products that received approval in the US and EU. Prior to joining Myeloid, Eric was Senior Vice President of Operations at iVexSol, Inc., where he designed and optimized the manufacturing facility layout, flow and associated office space, as well as overseeing the buildout of the cGMP manufacturing facility. Earlier, he was Vice President of Manufacturing at Genprex, Inc., where he was responsible for translating the corporate strategy into supportive strategies for global pharmaceutical manufacturing, packaging, supply chain, logistics and quality control from early product development through global manufacturing for commercial products. He has also held senior positions at Cognate Bioservices, Inc., and Alnylam Pharmaceuticals, where he was responsible for authoring CMC sections for regulatory submissions, IMPD/IND/NDA, for Patisiran and Givosiran, which are the first two RNA interference therapeutics approved for commercialization in the US and EU. Eric started his career in positions within quality control, manufacturing and CMC at Genzyme Corporation, VaxInnate Corporation, and Pfizer, Inc, respectively. Mr. Chapdelaine holds a Bachelor's of Science from the University of Massachusetts at Amherst and a Masters of Science, Chemistry, from Vanderbilt University.

About Myeloid Therapeutics

Myeloid Therapeutics is a clinical stage mRNA-immunotherapy company harnessing the power of myeloid cell biology to engineer new therapeutic alternatives for patients with cancer and autoimmune diseases.Integrating the fields of RNA biology, immunology, and medicine, the Company's proprietary platform provides clinical solutions that match therapeutic modalities to disease conditions, including use of autologous cell therapies, in vivo cell programming using mRNA, RNA-based gene-editing using RetroT and multi-targeted biologics. Myeloid is advancing a broad portfolio of clinical and preclinical candidates designed to enable full immune system responses. Myeloid has entered into strategic partnerships with Prime Medicine and Acuitas and is supported by well-known biotechnology investors. Myeloid is headquartered in Boston, MA. For more information, visit https://www.myeloidtx.com/.

Investor and Media ContactAmy Conrad Juniper Point [emailprotected] 858-914-1962

SOURCE Myeloid Therapeutics

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Myeloid Therapeutics Strengthens Senior Team to Support the Expansion of its Manufacturing Capabilities - PR Newswire

Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location – Benzinga

Personalized Regenerative Medicine, the San Clemente office of Dr. Steenblock, announces its grand reopening celebration. This event will be held at 187 Av. La Pata, San Clemente, CA 92673 on Thursday, September 1st from 5:30 to 7:30 PM. The celebration produced by Rachel Dares PR includes a red carpet, a ribbon-cutting ceremony, a DJ, delectable food, refreshments, press coverage, VIP swag bags, and raffle prizes. Come enjoy this wonderful occasion with the staff of Dr. Steenblock, his patients, generous sponsors, the local community, San Clemente Chamber of Commerce members, and local officials.

Dr. David Steenblock is widely regarded as one of the country's foremost stem cell experts, specializing in the treatment of ALS, TBI, Cerebral Palsy, Osteoarthritis, and stroke. Dr. David Steenblock, D.O., is an interdisciplinary medical genius who is exploring uncharted territory to find the best treatment for his patients.

The San Clemente Chamber of Commerce will host a Ribbon Cutting Ceremony as part of the festivities. Author, humanitarian, superfood creator, TV show, and radio personality Dr. Tony O'Donnell will serve as the event's emcee. Delicious food and beverages will be provided by local businesses, including mouthwatering appetizers sponsored by BASANTI Indian Cuisine, signature cheese boards by Riviera, delicious chips & salsa from Chelas Mexican Restaurant, scrumptious fresh baked goods from Elisa Marie Baking, thirst-quenching bottled water from MOTUS Physical Therapy, appetizing deli platters from Primerica, and delightful salad trays from The Kitchen Lady. The party will also feature music by DJ George.

The first 25 early attendees will receive VIP Swag Bags from Orange Sculpting inclusive of Dr.Steenblocks break-through supplement, Stemgevity which utilizes encapsulated stem cell growth activators to help patients relieve pain, promote tissue regeneration, stimulate collagen, and see fast results in overall youth factors.

With the generous sponsorship fromHome Saver Realty, the event will also feature great photo opportunities with a red carpet and backdrop banner. Amazing raffle prizes will be given out to lucky winners throughout the evening provided by Laguna Aesthetics, The Kitchen Lady, Orange Sculpting, Radiant Greens, The Dorothea Clinic and Rachel Dares PR!

The office of Personalized Regenerative Medicine invites everyone to witness and experience the future of regenerative medicine and to see for yourselves what all the excitement is about! RSVP now on Event Brite!

Media Contact Company Name: Rachel Dares PR Contact Person: RACHEL B DARES Email: Send Email Phone: 7147189043 Address:16480 Bake Parkway #106 City: Irvine State: CA Country: United States Website: http://www.RachelDaresPR.com

Press Release Distributed by ABNewswire.com To view the original version on ABNewswire visit: Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location

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Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location - Benzinga

Is Regen BioPharma a Good Investment? – Value the Markets

Regen BioPharma recently filed a patent related to a mRNA cancer immunotherapeutic vaccine. Does the market growth potential make RGBP a good investment opportunity?

Oncology stock Regen BioPharma (OTCMKTS: RGBP) is a penny stock listed on the OTC pink market.

Regen BioPharma, Inc focuses on the development of translational medicine platforms for the commercialization of stem cell therapies. The company aims to obtain regulatory approval and execute clinical trials in cell therapy. Regen BioPharma develops medicines for diabetes, heart-related illness, circulatory issues, and Chronic Obstructive Pulmonary Disease (COPD).

The company actively identifies small molecules that inhibit or express NR2F6 leading to immune cell activation for oncology applications and immune cell suppression for autoimmune diseases.

Regen BioPharma is in the early stages of developing its products and therapies. The business was incorporated in 2012 and is based in La Mesa, California.

Regen has identified and filed patents on small molecules that activate and inhibit a novel gene (NR2F6) which controls how the immune system reacts to cancer cells and inflammatory responses.

Its progress is currently pre-clinical, so commercialization is a long way off.

The conditions Regen hopes its investigations will eventually be able to address include:

Initial indications (inhibitor): bladder cancer, myelodysplastic syndrome, lung cancer

Initial indications (activator): rheumatoid arthritis, inflammatory bowel disease, psoriasis

Additional indications include solid tumors, acute leukemia and GVHD

Regen currently has the following products in its pipeline:

HemaXellerate: Cleared to proceed to Phase I/II clinical trials (stimulates blood production in patients whose bone marrow is not correctly functioning)

dCellVax: a breast cancer therapy (a dendritic cell-based immunotherapy that stimulates the patient's immune system)

DiffronC: a novel form of therapy called differentiation therapy that is expected to have much milder toxicity than chemotherapy.

For the quarterly period ended June 30, 2022

Revenues: $31.2k (up 6.8% Y/Y)

Total Revenues: $58.7k (up 3.7% Y/Y)

Operating Loss: -$58.9k (from -$42.4k Y/Y)

Between September 2021 and June 2022, Regen saw its cash decline by 85%. This is primarily due to paying off debts and using cash to fund the business.

Regen BioPharma is expected to report its Fiscal Year 2022 results on December 22, 2022.

RGBP stock has five billion shares outstanding.

On July 19, 2022, Regen BioPharma issued 54.5 million shares to Coventry Enterprises LLC to settle $180.5k in convertible debt.

On August 9, 2022, KCL Therapeutics, Inc. (a wholly owned subsidiary of Regen BioPharma) filed a provisional patent application with the US Patent and Trademark Office covering Regen's novel approach for enabling chimeric antigen receptor (CAR)-T cell-based therapies to kill solid tumors through prevention of a process called "T cell exhaustion." T cells are part of the immune system and develop from stem cells in the bone marrow.

Regen BioPharma's patent application protects the use of its patented survivin mRNA both as a stand-alone vaccine and as an immunotherapy.

David Koos, Chairman and CEO of Regen BioPharma, commented:

The currently filed application discloses means of significantly increasing efficacy by combining modified-mRNA with unique cellular immunotherapy as well as adjuvant approaches,

CAR-T cells have been in the clinic for 6 years, but they have hardly made a dent in treating solid tumors,

We hope that through the diligent work of our scientists and collaborators that advancements such as what we announced today will pave the way to bring this incredible cellular immunotherapy to patients suffering from solid tumors such as lung, colon, prostate and others.

Regen BioPharma is not profitable but makes a small income from royalties and anniversary fees under license agreements.

The company holds shares in Oncology Pharma (OTCMKTS: ONPH) and Zander Therapeutics.

Zander Therapeutic has been granted an exclusive license to develop and commercialize IP controlled by Regen BioPharma for non-human veterinary therapeutic use.

Meanwhile, Regen has granted an exclusive license to Oncology Pharma, Inc. to develop and commercialize "Antigen specific mRNA cellular cancer vaccines" for treating pancreatic cancer.

And KCL Therapeutics, Inc. has granted an exclusive license to Oncology Pharma, Inc. to develop and commercialize certain intellectual property for the treatment of colon cancer.

David Koos, Chairman and CEO of Regen BioPharma, said:

We are very proud of our broad, deep and cutting-edge patent portfolio,

We are continuing to develop additional IP focused on immunotherapy of cancer and look forward to developing some of these technologies in-house and some of them via outlicensing.

RGBP stock has a price-to-earnings ratio (P/E) of 48.7. Its price-to-book-value (P/BV) is negative, which means it owes more than it owns. RGBP stock does not come with a shareholder dividend.

Over the past year, Regen BioPharma Inc (RGBP) has traded between $0 and $0.08. Today the stock trades at $0.0096 and has a market cap of $48m.

Year-to-date, the RGBP stock price is down by -50%, while the S&P 500 is down -13.73% over the same period.

Furthermore, on August 22, 2022, Regen BioPharma (RGBP-US) closed down -86.3% off its 52-week high vs. the subsector average change of -39.4%.

Regen BioPharma plans to rapidly advance novel technologies through pre-clinical and Phase I/ II clinical trials. If it can successfully advance its small molecule therapies for treating cancer and autoimmune disorders, the addressable market is significant, and it could potentially make money.

Immunotherapy of cancer represents a vast market currently being led by the class of drugs called "checkpoint inhibitors" and "CAR-T" cells. To date, there is no mRNA immunotherapy available for treating cancer.

A stock with a beta higher than 1.0 is expected to be more volatile than the S&P 500. RGBP stock has a beta of 3.75, which accurately reflects its volatile nature.

Its price-to-book-value (P/BV) is negative, meaning it owes more than it owns.

RGBP common stock is a "penny stock," and therefore comes with the associated risk in dealing in penny stocks, such as lack of liquidity.

The economy is experiencing a period of high inflation, which could lead to interest rate rises and/or a recession. This is not conducive to raising funds at favorable rates and could adversely affect the company's ability to raise the money needed to conduct its research and trials.

There is a lack of liquidity in trading a stock such as Regen BioPharma. Therefore, there will probably be a large spread between the bid and ask price of RGBP shares. This means if you want to sell your shares in a hurry, you may not get a reasonable price for them.

Furthermore, the company has a history of issuing additional stock, which dilutes existing shareholder value.

Regen BioPharma stock seems like a hugely speculative investment with a lot of shareholder risk in the current investing environment.

Author: Kirsteen Mackay

This article does not provide any financial advice and is not a recommendation to deal in any securities or product. Investments may fall in value and an investor may lose some or all of their investment. Past performance is not an indicator of future performance.

Kirsteen Mackay does not hold any position in the stock(s) and/or financial instrument(s) mentioned in the above article.

Kirsteen Mackay has not been paid to produce this piece by the company or companies mentioned above.

Digitonic Ltd, the owner of ValueTheMarkets.com, does not hold a position or positions in the stock(s) and/or financial instrument(s) mentioned in the above article.

Digitonic Ltd, the owner of ValueTheMarkets.com, has not been paid for the production of this piece by the company or companies mentioned above.

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Is Regen BioPharma a Good Investment? - Value the Markets

Scientists use stem cells to create synthetic mouse embryos – ABC News

Scientists have created synthetic mouse embryos from stem cells without a dad's sperm or a mom's egg and womb

ByLAURA UNGAR AP Science Writer

Scientists have created synthetic mouse embryos from stem cells without a dad's sperm or a mom's egg or womb.

The lab-created embryos mirror a natural mouse embryo up to 8 days after fertilization, containing the same structures, including one like a beating heart.

In the near term, researchers hope to use these so-called embryoids to better understand early stages of development and study mechanisms behind disease without the need for as many lab animals. The feat could also lay the foundation for creating synthetic human embryos for research in the future.

We are undoubtedly facing a new technological revolution, still very inefficient but with enormous potential, said Llus Montoliu, a research professor at the National Biotechnology Centre in Spain who is not part of the research. It is reminiscent of such spectacular scientific advances as the birth of Dolly the sheep and others.

A study published Thursday in the journal Nature, by Magdalena Zernicka-Goetz at the California Institute of Technology and her colleagues, was the latest to describe the synthetic mouse embryos. A similar study, by Jacob Hanna at the Weizmann Institute of Science in Israel and his colleagues, was published earlier this month in the journal Cell. Hanna was also a coauthor on the Nature paper.

Zernicka-Goetz, an expert in stem cell biology, said one reason to study the early stages of development is to get more insight into why the majority of human pregnancies are lost at an early stage and embryos created for in vitro fertilization fail to implant and develop in up to 70% of cases. Studying natural development is difficult for many reasons, she said, including the fact that very few human embryos are donated for research and scientists face ethical constraints.

Building embryo models is an alternative way to study these issues.

To create the synthetic embryos, or embryoids, described in the Nature paper, scientists combined embryonic stem cells and two other types of stem cells all from mice. They did this in the lab, using a particular type of dish that allowed the three types of cells to come together. While the embryoids they created werent all perfect, Zernicka-Goetz said, the best ones were indistinguishable from natural mouse embryos. Besides the heart-like structure, they also develop head-like structures.

This is really the first model that allows you to study brain development in the context of the whole developing mouse embryo, she said.

The roots of this work go back decades, and both Zernicka-Goetz and Hanna said their groups were working on this line of research for many years. Zernicka-Goetz said her group submitted its study to Nature in November.

Scientists said next steps include trying to coax the synthetic mouse embryos to develop past 8 days with the eventual goal of getting them to term, which is 20 days for a mouse.

At this point, they struggle to go past the 8 1/2-day mark, said Gianluca Amadei, a coauthor on the Nature paper based at the University of Cambridge. We think that we will be able to get them over the hump, so to speak, so they can continue developing.

The scientists expect that after about 11 days of development the embryo will fail without a placenta, but they hope researchers can someday also find a way to create a synthetic placenta. At this point, they don't know if they will be able to get the synthetic embryos all the way to term without a mouse womb.

Researchers said they dont see creating human versions of these synthetic embryos soon but do see it happening in time. Hanna called it the next obvious thing.

Other scientists have already used human stem cells to create a blastoid, a structure mimicking a pre-embryo, that can serve as a research alternative to a real one.

Such work is subject to ethical concerns. For decades, a 14-day rule on growing human embryos in the lab has guided researchers. Last year, the International Society for Stem Cell Research recommended relaxing the rule under limited circumstances.

Scientists stress that growing a baby from a synthetic human embryo is neither possible nor under consideration.

Perspective on this report is important since, without it, the headline that a mammalian embryo has been built in vitro can lead to the thought that the same can be done with humans soon, said developmental biologist Alfonso Martinez Arias of the Universitat Pompeu Fabra in Spain, whose group has developed alternative stem cell based models of animal development.

"In the future, similar experiments will be done with human cells and that, at some point, will yield similar results," he said. "This should encourage considerations of the ethics and societal impact of these experiments before they happen.

The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institutes Department of Science Education. The AP is solely responsible for all content.

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Scientists use stem cells to create synthetic mouse embryos - ABC News

Dr. Lin on Patient Eligibility for CAR T-cell Therapy in Hematologic Caners – OncLive

Yi Lin, MD, PhD, discusses patient eligibility for CAR T-cell therapy in hematologic cancers.

Yi Lin, MD, PhD, hematologist, oncologist, Department of Hematology, CAR T-Cell Therapy Program, Mayo Clinic, discusses patient eligibility for CAR T-cell therapy in hematologic cancers.

Many oncologists have experience referring patients for stem cell transplant, and referring patients for CAR T-cell therapy can work in a similar fashion, though it may be more beneficial since CAR T-cell therapy can be effective in patients with active disease, Lin explains. When referring patients to CAR T-cell therapy, it is important to consider a patients number of prior lines of therapy, their comorbidities, and organ function, Lin adds.

Though clinical trials evaluating CAR T-cell therapies in hematologic malignancies have strict parameters, it can be difficult for many patients to fall inside all of those parameters in real-world practice, Lin continues. In reports on real-world data for patients with leukemia or lymphoma who received CAR T-cell therapy, approximately half of patients treated fall outside of at least 1 parameter that was used for CAR T-cell therapies in clinical trials, Lin notes.

Through coordination between clinicians and the CAR T-cell therapy team, patients who fall outside the parameters used in clinical trials can still have comparable outcomes from CAR T-cell therapy, Lin concludes.

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Dr. Lin on Patient Eligibility for CAR T-cell Therapy in Hematologic Caners - OncLive

Higher Doses of CAR T-Cell Therapy May Improve Survival for Young Patients With B-Cell ALL – The ASCO Post

By The ASCO Post Staff Posted: 8/16/2022 10:52:00 AM Last Updated: 8/16/2022 3:13:09 PM

Young people with B-cell acute lymphoblastic leukemia (ALL) who received doses of tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy, at the higher end of the approved dosing range had significantly better survival rates at 1 year compared with those who received lower doses within this range, according to research published by Stefanski et al inBlood Advances.

Since its approval as the first gene therapy available in the United States in 2017, tisagenlecleucel has offered a welcome treatment option for pediatric patients with B-cell ALL whose cancer does not respond to chemotherapy or recurs after prior response. However, the wide dosing range approved for the therapy can pose a conundrum for doctors who sometimes must choose whether to use a higher or lower dose with little evidence to guide these decisions. This new study offers the first insights into optimal dosing based on real-world data.

In the past, we did not have data to guide clinical decisions around commercial CAR T-cell dosing and didnt know if higher doses would affect toxicity and compromise outcomes, or support enhanced antileukemia effect, said Liora Schultz, MD, a pediatric oncologist at the Stanford Children's HealthLucile Packard Children's Hospital and the studys lead author. [These data have] direct clinical applicability, as it supports use of higher dosing, as available, within the approved tisagenlecleucel dose range.

More About B-Cell ALL and Tisagenlecleucel

ALL is the most common type of cancer in children, and B-cell ALL is its most common subtype. It is often treatable with chemotherapy, but about 20% of patients either do not respond to chemotherapy or subsequently relapse. CAR T-cell therapy is becoming an integral part of standard care as an alternative or supplement to stem cell transplantation for pediatric patients with relapsed or refractory B-cell ALL.

Tisagenlecleucel is approved at a dosing range of 0.2 to 5 million CAR T cells/kg for patients weighing 50 kg or less, or 10 to 250 million CAR T cells for patients weighing over 50 kg. In practice, the number of CAR T cells that are manufactured for each patient varies substantially based on the number of T cells that are obtained initially and the rate at which the modified cells grow in the laboratory.

Any amount of CAR T cells within the approved range is considered an acceptable dose, but if more than the minimum number of cells are available within the approved range, doctors can decide whether to use a higher amount or a lower amount. Clinical trials for tisagenlecleucel provided guidance on dosing leading to the therapys approval, but real-world data are useful to fine-tune dosing and inform decision-making when there are multiple options available.

Study Details

For the study, researchers analyzed rates of overall survival, event-free survival, and relapse-free survival at 1 year among 185 patients aged 26 years or younger who received tisagenlecleucel for relapsed or refractory B-cell ALL. They found that patients who received a dose at the higher end of the approved range (between 2.4 and 5.1 million cells/kg) had significantly higher survival rates according to all three measures compared with patients who received a dose at the lower end (between 0 and 1.3 million cells/kg). In the highest-dose group, 86% of patients were alive at 1 year, compared to 59% in the lowest-dose group. Researchers did not observe any signs of increased toxicity or safety concerns with higher doses.

The findings suggest that administering doses of tisagenlecleucel at the higher end of the approved range could help to achieve a more effective and long-term responsewithout raising the toxicity risk.

A lot of effort is focused on complex engineering and development of next-generation CAR T-cell therapies, said Dr. Schultz. This study aims to explore if clinical manipulations using our current approved construct, tisagenlecleucel, can achieve even incremental advances in the field.

The researchers plan to further examine the data to determine how additional clinical variables might influence outcomes following CAR T-cell therapy.

Disclosure: For full disclosures of the study authors, visit ashpublications.org/bloodadvances.

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Higher Doses of CAR T-Cell Therapy May Improve Survival for Young Patients With B-Cell ALL - The ASCO Post

Haematopoietic Stem Cell Gene Therapy in IEM – Physician’s Weekly

Due to donor-derived cells capacity to deliver enzymes to enzyme-deficient tissues and organs for the rest of ones life, allogeneic hematopoietic stem cell transplantation (allo-HSCT) has become widely used as a therapeutic approach for many inborn errors of metabolism (IEM) over the past 30 years. However, patients who have IEM were the only ones who could clinically benefit from allo-HSCT, patients still had a large burden from their residual illness, and allo-HSCT was still linked to severe short- and long-term toxicities as well as transplant-related death.

In the 1990s, hematopoietic stem/progenitor cell gene therapy (HSPC-GT) was developed for the treatment of a few monogenic primary immunodeficiencies, and in recent years, its use has expanded to include a number of IEM. Gene-corrected hematopoietic progenitors could provide supra-physiological enzyme levels to hard-to-reach regions, such as the brain and skeleton, with a possible improved therapeutic effect. This made HSPC-GT particularly appealing in neurodegenerative IEM.

Furthermore, HSPC-GT had lower rates of morbidity and death than allo-HSCT, albeit it must be weighed against the chance of insertional mutagenesis. Clinical trials in the IEM sector were multiplying quickly, and certain HSPC-GT items had just lately been given the go-ahead. In contrast to tried-and-true treatment approaches like allo-HSCT, the review discussed the development of ex vivo HSPC-GT in a variety of IEM with an emphasis on current findings from GT clinical trials and risks vs. benefits considerations.

Reference: onlinelibrary.wiley.com/doi/10.1111/bjh.18179

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Reprogramming the Brain’s Cleaning Crew to Mop Up Alzheimer’s Disease – Weill Cornell Medicine Newsroom

The discovery of how to shift damaged brain cells from a diseased state into a healthy one poses a new potential path to treating Alzheimers and other forms of dementia, according to a new collaborative study from researchers at UC San Francisco and Weill Cornell Medicine.

The research focuses on microglia, cells that stabilize the brain by clearing out damaged neurons and the protein plaques often associated with dementia and other brain diseases.

These cells are understudied, despite the fact that changes in them are known to play a significant role Alzheimer's and other brains diseases, said Dr. Martin Kampmann, associate professor at the Institute for Neurodegenerative Diseases in the UCSF Weill Institute for Neurosciences and co-senior author on the study, which appears Aug. 11 in Nature Neuroscience.

We were able to overcome limitations in microglia research by building a new platform to generate microglia in the lab using inducible stem cells, said co-senior author Dr. Li Gan, director of the Helen and Robert Appel Alzheimers Disease Research Institute and the Burton P. and Judith B. Resnick Distinguished Professor in Neurodegenerative Diseases in the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine.

Now, using a new CRISPR method we developed, we can uncover how to actually control these microglia, to get them to stop doing toxic things and go back to carrying out their vitally important cleaning jobs, Dr. Kampmann said. This capability presents the opportunity for an entirely new type of therapeutic approach.

Most of the genes known to increase risk for Alzheimers disease act through microglial cells, making it clear that they can have a big effect on how such neurodegenerative diseases play out, said Dr. Kampmann.

This makes sense because microglia act as the brains immune system. Ordinary immune cells cant cross the blood-brain barrier, so its the task of healthy microglia to clear out waste and toxins, keeping neurons functioning at their best. When microglia start losing their way, the result can be brain inflammation and damage to neurons and the networks they form.

Under some conditions, for example, microglia will start removing synapses between neurons. While this is a normal part of brain development in a persons childhood and adolescent years, it can have disastrous effects in the adult brain.

Over the past five years or so, many studies have observed and profiled these varying microglial states but havent been able to characterize the genetics behind them.

Dr. Kampmann and his team wanted to identify exactly which genes are involved in specific states of microglial activity, and how each of those states are regulated. With that knowledge, they could then flip genes on and off, setting wayward cells back on the right track.

Accomplishing that task required getting around some fundamental obstacles that have prevented researchers from being able to control gene expression in these cells. For example, microglia are very resistant to the most common CRISPR technique, which involves getting the desired genetic material into the cell by using a virus to deliver it.

To overcome this obstacle, Dr. Gan and Dr. Kampmann teamed up to coax stem cells donated by human volunteers to become microglia and confirmed that these cells function like their ordinary human counterparts.

By providing the stem cells with a road map, we gave them a fast track to become microglia in a week, a process that usually takes over a month, said Dr. Gan.

The team then developed a new platform that combines a form of CRISPR that enables researchers to turn individual genes on and off (which Kampmann had a significant hand in developing), with readouts of data that indicate functions and states of individual microglia cells.

Through this analysis, the team was able to pinpoint genes that effect the cells ability to survive and proliferate, how actively a cell produces inflammatory substances, and how aggressively a cell prunes synapses.

Beyond that, because the scientists had determined which genes control those activities, they were able to reset genes, flipping the cell from a diseased state to a healthy one.

Armed with this new technique, the next step is to investigate how to control the relevant states of microglia, targeting the cells with existing pharmaceutical molecules and testing them in preclinical models. The ultimate goal is to identify specific molecules that act on the genes necessary to nudge diseased cells back to healthy states.

Many Weill Cornell Medicine physicians and scientists maintain relationships and collaborate with external organizations to foster scientific innovation and provide expert guidance. The institution makes these disclosurespublic to ensure transparency. For this information, see profile for Dr. Li Gan.

The original version of this story appeared in the UCSF newsroom.

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Reprogramming the Brain's Cleaning Crew to Mop Up Alzheimer's Disease - Weill Cornell Medicine Newsroom