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Novartis provides update on FDA review of ofatumumab, a self-administered, targeted B-cell therapy for patients with relapsing multiple sclerosis -…

Posted: June 4, 2020 at 8:59 am

Basel, June 2, 2020 Novartis today announced that it has received notice from the US Food and Drug Administration (FDA) that the agency has extended its review of the Supplemental Biologics License Application (sBLA) for ofatumumab (OMB 157), a self-administered, targeted B-cell therapy for patients with relapsing multiple sclerosis. Regulatory action is now expected in September 2020.

Novartis will continue to work with the FDA to complete the review as soon as possible, said Marie-France Tschudin, President, Novartis Pharmaceuticals. We are well prepared and ready to launch ofatumumab upon approval. We are committed to the MS community and look forward to bringing this important advancement to patients with MS.

Additional regulatory filings are currently underway and regulatory approval for ofatumumab in Europe is expected by Q2 2021.

About ofatumumabOfatumumab (OMB 157) is a fully human anti-CD20 monoclonal antibody (mAb) in development for RMS that is self-adminstered by a once-monthly injection, delivered subcutaneously1,2,7. As shown in preclinical studies, ofatumumab is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion3. The selective mechanism of action and subcutaneous administration of ofatumumab allows precise delivery to the lymph nodes, where B-cell depletion in MS is needed, and may preserve the B-cells in the spleen, as shown in preclinical studies4. Once-monthly dosing of ofatumumab also allows fast repletion of B-cells and offers more flexibility5. Ofatumumab was originated by Genmab and licensed to GlaxoSmithKline; Novartis obtained rights for ofatumumab from GlaxoSmithKline in all indications, including RMS, in December 20156.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, can, will, plan, may, could, would, expect, anticipate, seek, look forward, believe, committed, investigational, pipeline, launch, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact media.relations@novartis.com

References1. Hauser S.Efficacy and safety of ofatumumab versus teriflunomide in relapsing multiple sclerosis: results of the phase 3 ASCLEPIOS I and II trials. Presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Annual Conference; September 1113, 2019; Stockholm, Sweden.2. Bar-Or A, Fox E, Goodyear A, et al.Onset of B-cell Depletion with Subcutaneous Administration of Ofatumumab in Relapsing Multiple Sclerosis: Results from the APLIOS Bioequivalence Study. Presented at Americas Committee for Treatment and Research in Multiple Sclerosis Forum; February 2729, 2020.3. Smith P, Kakarieka A, Wallstroem E.Ofatumumab is a fully human anti-CD20 antibody achieving potent B-cell depletion through binding a distinct epitope. Poster presented at ECTRIMS; September 2016; London, UK.4. Smith P, Huck C, Wegert V, et al.Low-dose, subcutaneous anti-CD20 therapy effectively depletes B-cells and ameliorates CNS autoimmunity. Poster presented at ECTRIMS; September 1417, 2016; London, UK.5. Savelieva M, Kahn J, Bagger M, et al.Comparison of the B-Cell Recovery Time Following Discontinuation of Anti-CD20 Therapies. ePoster presented at ECTRIMS; October 2528, 2017; Paris, France.6. GSK press release.GSK completes divestment of rights to ofatumumab for auto-immune indications to Novartis. December 21, 2015. Available from:https://www.gsk.com/en-gb/media/press-releases/gsk-completes-divestment-of-rights-to-ofatumumab-for-auto-immune-indications-to-novartis/%5BLast accessed: June 2020].7. Efficacy and Safety of Ofatumumab Compared to Teriflunomide in Patients With Relapsing Multiple Sclerosis (ASCLEPIOS I & II clinical trials), data on file, Novartis. https://clinicaltrials.gov/ct2/show/NCT02792218; https://clinicaltrials.gov/ct2/show/NCT02792231.

# # #

Novartis Media RelationsE-mail: media.relations@novartis.com

Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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The COVID-19 Shuffle: Adoptive Cell Therapy For Cancer Treatment Market Size, Share, Applications, Regions, Top Companies, Trends, Market Drivers and…

Posted: June 4, 2020 at 8:59 am

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Lessons from the epicenter: How one N.J. hospital endured the worst of the pandemic and learned from it – nj.com

Posted: June 4, 2020 at 8:59 am

The dying patient just needed to hold on a little while longer.

Within a few hours, she would receive the new treatment. The placenta cells a therapy that showed promise in boosting the immune system were on their way to Holy Name Medical Center from another state.

It was April, and the coronavirus crisis was intensifying. The patient, an unidentified woman, lay dying of COVID-19 at the Teaneck facility, one of New Jerseys hardest-hit hospitals.

Everyone was anxious and was excited that we would treat her, said Dr. Ravit Barkama, a clinical researcher at the hospital. And the cells were basically on the way to Holy Name from Maryland.

It was an example of the hope and heartache doctors and nurses faced in the early days of the unfolding medical crisis. They rushed to treat a crush of patients flooding the emergency room, while clinical researchers studied the latest data and searched for anything in their arsenals to keep the infected alive.

While the first phase of the COVID-19 crisis is winding down, the pandemic and the health care establishments work are far from over. Holy Name is taking a hard look at everything its medical personnel learned in enduring those treacherous first three months.

The clinical researchers recently shared a report with NJ Advance Media outlining a series of medical takeaways: Everything theyve learned after treating hundreds of coronavirus patients since the crisis began.

Its an orderly account with dense detail and precise explanations a dramatic contrast to March and April, when more than 200 patients were dying on ventilators, one after the other. When the intensive care units were filled.

When there seemed to be no clear answers.

The researchers learned the ways COVID-19 affects the body, the various stages in which it manifests and consumes patients, the medications and therapies that show promise and the treatments that proved ineffective.

Like every hospital in the state, Holy Name was desperately looking for ways to combat the disease after the pandemic hit New Jersey.

Treatment was trial and error. It was a scramble, often necessitating learning on the fly.

The first few weeks, basically, almost every night was ... working with a lot of frustration, Barkama said.

She and her counterpart, Dr. Thomas Birch, have been working as a research duo of sorts, investigating possible therapies for a deluge of COVID-19 patients.

"How are we going to treat the patients? What can we give them? How can we make this better? How can we keep people from dying? How can we keep people off of the ventilator?" Birch said.

It was an exhausting sprint of confusion and dashed hopes.

Barkama and Birch recalled late nights poring over research and data. Barkama called counterparts in Israel and Europe asking what was working.

They found insights, if not epiphanies.

One of the hard-won lessons was COVID-19 is a disease of stages.

It follows a largely defined progression in the human body, from exposure and incubation (phase 1), to the showing of symptoms (phase 2), to the virus impact on the lungs (phase 3) to the decrease of lung inflammation (phase 4).

We basically realized certain interventions might be effective in certain phases and not effective in others. And so thats the key, said Dr. Suraj Saggar, infectious disease specialist at Holy Name.

Early intervention is crucial. Antiviral drugs likely work best during the first phase when used as a pre-emptive therapy, the report said.

The antiviral drug, remdesivir, seems most effective during phase 2, the stage when the initial symptoms emerge. Convalescent plasma may also have some benefit during this stage.

The third phase is what makes COVID-19 different, the report said. This is when symptoms like shortness of breath, persistent fever and pneumonia develop. Severe inflammation begins to batter the lungs, blood vessels and organs.

The bodys overreactive immune system not the virus does most of the damage. It is why inflammatory drugs and immunosuppressive appear to help during this period.

The final stage, or the convalescent phase, is when the inflammation begins to subside and the lungs and other tissues clear the debris from inflammation, injured tissues and fluid, the report said. The stage can span three to six weeks, delaying the time when the patient can be weaned from the ventilator.

It is possible placenta-derived cells may further reduce inflammation, according to the report.

It was a tremendous amount of reading, a tremendous amount of reviewing patient cases and seeing what was going on with them clinically, their symptoms, their physical exam findings, the laboratory and imaging, said Birch, who is also an infectious disease specialist. How they responded to some of the therapies that were being used, and how we might use them more rationally.

When potential treatments emerged, such as placenta cell therapy or remdesivir, it offered hope after weeks of anguish at Holy Name.

It was no different in April as they tried to help the dying woman.

The doctors and researchers waited April 11.

The cells would arrive in just a few more hours, they were told.

The findings on placenta cell therapy were still preliminary, but the treatment championed by a company in Israel was showing promise. Six critically ill coronavirus patients in Israel had shown signs of improvement after taking it.

Some saw it as a possible dream treatment.

But then an alarm blared over Holy Names loudspeakers. It was a Code Blue, an emergency calling for the CPR team.

"It goes 'Code Blue ICU,' and then, of course, I knew that something was very wrong," Barkama said.

Her heart dropped. She rushed to see who it was.

It was the dying woman.

"A few hours before we were supposed to give the treatment, she desaturated and passed away," Barkama said.

The loss devastated her.

"When I saw it was her I must say that it was emotionally very difficult to lose a patient a few hours before the so-called 'dream treatment,'" Barkama said.

While the researchers remain hopeful about placenta-derived cell therapy, only four patients have been treated at the hospital thus far. Its too early to assess the results.

Remdesivir has also shown promise, but research remains ongoing. The National Institutes of Health has found positive results, and Gilead, the maker of the drug, said in a statement earlier this week that it found encouraging data from a recent trial, especially if used as an intervention therapy.

Holy Names staff hope the lessons pay dividends for New Jersey hospitals in case they again encounter chaos like they did in March.

Looming in the back of everyones mind is the possible resurgence of the virus in the fall, after already claiming 11,880 lives and causing 162,068 infections in New Jersey.

There is, of course, a good chance that there will be [another phase], and were not looking at this as a historical episode, Barkama said.

The research continues. Hospitals were caught flat-footed, often forced to learn and sift out new techniques on the fly, revamp old ones and apply them in new ways.

We obviously hope, just like everyone else, that it will never hit us so hard again, Barkama said. But were going to be prepared in case it does.

Spencer Kent may be reached at skent@njadvancemedia.com. Follow him on Twitter @SpencerMKent. Find NJ.com on Facebook.

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Halo Labs Announces Partnership with Zkittlez Providing Award Winning Genetics in Oregon – Business Wire

Posted: June 4, 2020 at 8:58 am

TORONTO--(BUSINESS WIRE)--Halo Labs Inc. ("Halo" or the "Company") (NEO: HALO, OTCQX: AGEEF, Germany: A9KN) is pleased to announce that the Company has entered into an exclusive strategic partnership with Terphogz, LLC (Zkittlez) to develop and commercialize new and unique cannabis genetics in Oregon.

Background & Partnership Highlights

Terphogz, LLC owns a genetic library that is well-known for creating the famous Zkittlez cannabis variety. Zkittlez has a unique and distinct terpene profile unlike other genetics in the market, making it distinguishable from other strains. Halo has secured a 5-year exclusive partnership with Zkittlez in Oregon to cultivate the groups strains and provide this beloved brand to the Oregon marketplace and consumers for the first time.

Halo will license and have exclusive rights to a large repertoire of genetic strains, most notably Zkittlez, Zmoothi and Z3 Kush. Focusing on the whole plant, Zkittlez has proven that THC alone cant compete with robust and unique terpene profiles. Highly awarded and recognized, the strains will allow Halo develop more brand and retail focused products aligning with the Companys long term growth strategy. With over 200,000 followers on social media and a history of excellence in cannabis genetics, Zkittlez provides extensive value to Halos already strong portfolio of cannabis consumer-centric partnerships.

We are extremely excited to join forces with Halo. With our award-winning genetics and Halo's cultivation firepower, our collaboration will be a force to be reckoned with in Oregon. Stay tuned, we can't wait to get to work!" comments Green R. Fieldz, CEO of Terphogz, LLC.

Kiran Sidhu CEO and Co-Founder of Halo commented, We are pleased to be partnering with Terphogz, LLC in Oregon to build a new branded product line of flower, pre-rolls and concentrates. Aligning with a beloved and Emerald Cup award winning brand such as Zkittlez to cultivate existing and develop new cannabis strains demonstrates Halos commitment to offering cannabis consumers access to some of the best genetics. We expect to do more projects with the Terphogz, LLC team and expand the partnership into additional markets.

About Halo

Halo is a leading cannabis cultivation, manufacturing, and distribution company that grows and extracts and processes quality cannabis flower, oils, and concentrates and has sold over 5 million grams of oils and concentrates since inception. Additionally, Halo has continued to evolve its business through delivering value with its products and now via verticalization in key markets in the United States and Africa with planned expansion into European and Canadian markets. With a consumer-centric focus, Halo markets innovative, branded, and private label products across multiple product categories.

Recently, the Company entered into binding agreements to acquire a dispensary in Los Angeles, 3 KushBar branded dispensaries, 5 development permits in Alberta Canada, and Canmart Limited which holds wholesale distribution and special licenses allowing the import and distribution of cannabis based products for medicinal use (CBPMs) in the United Kingdom. Halo is led by a strong, diverse management team with deep industry knowledge and blue-chip experience. The Company is currently operating in the United States in California, Oregon, and Nevada while having an international presence in Lesotho within a planned 200-hectare cultivation zone via Bophelo Bioscience & Wellness (Pty) Ltd. as well as planned importation and distribution in the United Kingdom via Canmart.

For further information regarding Halo, see Halos disclosure documents on SEDAR at http://www.sedar.com.

Cautionary Note Regarding Forward-Looking Information and Statements

This press release contains certain "forward-looking information" within the meaning of applicable Canadian securities legislation and may also contain statements that may constitute "forward-looking statements" within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. Such forward-looking information and forward-looking statements are not representative of historical facts or information or current condition, but instead represent only Halos beliefs regarding future events, plans or objectives, many of which, by their nature, are inherently uncertain and outside of Halos control. Generally, such forward-looking information or forward-looking statements can be identified by the use of forward-looking terminology such as "plans", "expects" or "does not expect", "is expected", "budget", "scheduled", "estimates", "forecasts", "intends", "anticipates" or "does not anticipate", or "believes", or variations of such words and phrases or may contain statements that certain actions, events or results "may", "could", "would", "might" or "will be taken", "will continue", "will occur" or "will be achieved". The forward-looking information and forward-looking statements contained herein may include, but are not limited to, statements in respect of the Companys license arrangement with Terphogz, LLC and the cultivation, sale and distribution of Zkittlez branded and other products by the Company.

By identifying such information and statements in this manner, Halo is alerting the reader that such information and statements are subject to known and unknown risks, uncertainties and other factors that may cause the actual results to be materially different from those expressed or implied by such information and statements. In addition, in connection with the forward-looking information and forward-looking statements contained in this press release, Halo has made certain assumptions. Although Halo believes that the assumptions and factors used in preparing, and the expectations contained in, the forward-looking information and statements are reasonable, undue reliance should not be placed on such information and statements, and no assurance or guarantee can be given that such forward-looking information and statements will prove to be accurate, as actual results and future events could differ materially from those anticipated in such information and statements. Among others, the key factors that could cause actual results to differ materially from those projected in the forward-looking information and statements are the following: unexpected costs or delays in the completion of the Company's proposed dispensaries and other operation; negative results experienced by the Company as a result of general economic conditions or the ongoing COVID-19 pandemic; delays in the ability of the Company to obtain certain regulatory approvals; unforeseen delays or costs in the completion of the Company's construction projects; adverse changes to demand for cannabis products; ongoing projects by competitors that may impact the relative size of the Companys growing operation; adverse changes in applicable laws; adverse changes in the application or enforcement of current laws, including those related to taxation; increasing costs of compliance with extensive government regulation; changes in general economic, business and political conditions, including changes in the financial markets; risks related to licensing, including the ability to obtain the requisite licenses or renew existing licenses for the Company's proposed operations; dependence upon third party service providers, skilled labor and other key inputs; and the other risks disclosed in the Company's annual information form dated April 16, 2020 and available on the Companys profile at http://www.sedar.com. Should one or more of these risks, uncertainties or other factors materialize, or should assumptions underlying the forward-looking information or statements prove incorrect, actual results may vary materially from those described herein as intended, planned, anticipated, believed, estimated or expected.

The forward-looking information and forward-looking statements contained in this press release are made as of the date of this press release, and Halo does not undertake to update any forward-looking information and/or forward-looking statements that are contained or referenced herein, except in accordance with applicable securities laws. All subsequent written and oral forward-looking information and statements attributable to Halo or persons acting on its behalf is expressly qualified in its entirety by this notice.

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Myriad Genetics Announces Publication of a Prospective Clinical Study of the EndoPredict Test in Women with Early-Stage Breast Cancer – GlobeNewswire

Posted: June 4, 2020 at 8:58 am

SALT LAKE CITY, June 04, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN, Myriad or the Company), a global leader in molecular diagnostics and precision medicine, today announced the publication of a prospective study demonstrating that the EndoPredict test predicts which patients with ER+, HER2- early-stage breast cancer will benefit from neoadjuvant therapy. The article titled, The EndoPredict score predicts response to neoadjuvant chemotherapy and neoendocrine therapy in hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer patients from the ABCSG-34 trial, appeared online in theEuropean Journal of Cancer.

This study demonstrated that the EndoPredict (EP) test predicted response to neoadjuvant chemotherapy or neoadjuvant endocrine therapy in women with ER+, HER2 negative early-stage breast cancer, said Peter Dubsky, M.D., lead author, speaking on behalf of the Austrian Breast and Colorectal Cancer Study Group (ABCSG). Based on these findings and prior studies, we are confident the EndoPredict test can add valuable information to aid in personalized treatment selection in neoadjuvant therapy and provides an important basis for future design of neoadjuvant clinical trials.

The primary objective of this prospective study was to test the predictive value of the EndoPredict test regarding tumor response after neoadjuvant chemotherapy (NaCT) or neoadjuvant endocrine therapy (NET) within the ABCSG-34 trial. The analysis included data from 217 women with HR+ breast cancer. Of these, 134 patients were assigned to receive NaCT (eight cycles of anthracycline/taxane) according to aggressive clinico-pathologic tumor features. The remaining 83 patients were clinically identified as having luminal A-like types of breast cancer and were assigned to receive NET (six months of letrozole). The primary endpoint was residual cancer burden RCB0/I (i.e., good tumor response) vs. RCB II/III (i.e., poor tumor response) at time of surgery.

In the neoadjuvant chemotherapy group, 125 patients had high EP scores and nine had a low EP score. The results show that 26.4 percent of those with a high score showed a good tumor response (RCB0/I) to neoadjuvant chemotherapy, while all patients with a low score showed only a poor tumor response (Table 1). In the luminal A group receiving neoendocrine therapy, 39 patients had a high EP score and 44 had a low EP score. The results show that 27.3 percent of those with a low EndoPredict score and 7.7 percent with a high score achieved excellent tumor response (RCB0/I) to neoendocrine therapy (Table 1).

EndoPredict Low Score

EndoPredict High Score

0.0

%

26.4

%

p=0.0001

In this prospective study, we demonstrated that the EndoPredict test is a useful tool pre-operatively, said Ralf Kronenwett, M.D., director of International Medical Affairs at Myriad. In two distinct ER-positive, HER2-negative cohorts selected by clinicians to receive neoadjuvant chemotherapy or neoadjuvant endocrine therapy, EndoPredict identified patients with poor neoadjuvant treatment response. Clinicians can use information to determine who might forgo these therapies prior to surgery.

About EndoPredictEndoPredict is a second-generation, 12-gene molecular prognostic test for patients diagnosed with breast cancer. The test provides vital information that helps clinicians devise personalized treatment plans for their patients. EndoPredict has been validated in more than 4,000 patients with node-negative and node-positive cancer and has been used clinically in more than 20,000 patients. In contrast to first-generation multigene prognostic tests, EndoPredict detects the likelihood of late metastases (i.e., metastasis formation after more than five years) and, therefore, can guide treatment decisions regarding the need for chemotherapy, as well as extended anti-hormonal therapy. Accordingly, therapy decisions backed by EndoPredict confer a high level of diagnostic safety. For more information, please visit: http://www.endopredict.com.

About Myriad GeneticsMyriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, riskScore and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the EndoPredict test adding valuable information to aid in personalized treatment selection in neoadjuvant therapy and providing an important basis for future design of neoadjuvant clinical trials; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Myriad Genetics Announces Publication of a Prospective Clinical Study of the EndoPredict Test in Women with Early-Stage Breast Cancer - GlobeNewswire

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Earliest Genetic Glimpses of the Movement and Mingling of Peoples in West Asia 8,500 Years Ago – India New England

Posted: June 4, 2020 at 8:57 am

Historically, Western Asia, which includes todays Middle East, is one of civilizations most important geographical locations. Not only did it create some of humanitys earliest cities, but its early trade routes laid the foundation for what would become the Silk Road, a route that commercially linked Asia, Africa, and Europe.

Even before they connected with other regions, however, populations across Western Asia had already developed their own distinct traditions and systems of social organization. The areas studied in this paper played major roles in the evolution from farming to pastoral communities to early state-level societies.

With the study, the researchers wanted to fill in some of the anthropological gaps between the origins of agriculture and of cities to get a better grip on how these different communities came together, a dynamic that is still not understood well.

What we see in archaeology is that the interconnectivity within Western Asia increased and areas such as Anatolia, the Northern Levant, and the Caucasus became a hub for [the] exchange of ideas and material culture, said Eirini Skourtanioti, a Ph.D. student at the Max Planck Institute and the lead author of the study, in avideoaccompanying the release of the paper. The goal of our study was to understand the role of human mobility throughout this process.

The authors came from many disciplines and countries, including Australia, Azerbaijan, France, Italy, Germany, South Korea, Turkey, and the U.S. They gathered 110 ancient remains from museums and labs around the world, and took samples from teeth and part of the temporal bone called the petrous, which houses the inner ear. The genetic analysis was conducted by scientists at the Max Planck Institute, including Warinner.

The paper outlines how populations across Anatolia and the Southern Caucasus began mixing approximately 8,500 years ago. That resulted in a gradual change in genetic profile that over a millennium slowly spread across both areas and entered into what is now Northern Iraq. Known as a cline in genetics, this mixture indicated to the researchers ongoing human mobility in the area and the development of a regional genetic melting pot in and surrounding Anatolia.

We see the past through artifacts, through the evidence people leave behind. But sometimes events are happening that dont leave traces in conventional ways.

Christina Warinner

The other shift researchers detected wasnt as gradual. They looked at samples from the ancient cities of Alalakh and Ebla in what is today Southern Turkey and Northern Syria, and saw that around 4,000 years ago the Northern Levant experienced a relatively sudden introduction of new people.

The genetic shifts point to a mass migration. The timing corresponds with a severe drought in Northern Mesopotamia, which likely resulted in an exodus to the Northern Levant. The scientists cant be sure, because they have no well-preserved genomes for people who lived in Mesopotamia.

Along with findings on interconnectivity in the region, the paper presents new information about long-distance migration during the late Bronze Age, roughly 4,000 years ago. A lone corpse, found buried in a well, was genetically linked to people who then lived in Central Asia, not in part of present-day Turkey.

We cant exactly know her story, but we can piece together a lot of information that suggests that either she or her ancestors were fairly recent migrants from Central Asia, said Warinner, who is also a group leader in the Department of Archaeogenetics at the Max Planck Institute. We dont know the context in which they arrived in the Eastern Mediterranean, but this is a period of increasing connectivity in this part of the world.

The corpse had many injuries and the way she was buried indicated a violent death. Warinner hopes more genomic analysis can help unravel the ancient womans story.

For Warinner, who earned her masters in 2008 and her Ph.D. in 2010 from theGraduate School of Arts and Sciences, such studies are proof of the insights DNA analysis can provide when traditional clues dont tell the full story.

Whats really interesting is that we see these populations are mixing genetically long before we see clear material culture evidence of this so long before we see direct evidence in pottery or tools or any of these more conventional archaeological evidence artifacts, Warinner said. Thats important because sometimes were limited in how we see the past. We see the past through artifacts, through the evidence people leave behind. But sometimes events are happening that dont leave traces in conventional ways, so by using genetics, we were able to access this much earlier mixing of populations that wasnt apparent before.

(Reprinted with permission from the Harvard Gazette.)

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Earliest Genetic Glimpses of the Movement and Mingling of Peoples in West Asia 8,500 Years Ago - India New England

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Stem Cell Therapy Market Analysis and Demand 2017 2025 – Cole of Duty

Posted: June 4, 2020 at 8:55 am

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Know the Growth Opportunities in Emerging Markets

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner…

Posted: June 4, 2020 at 8:55 am

The current coronavirus pandemic clearly illustrates how dangerous viral infections can become for us. Independent of the present situation, there are people whose bodies are defenseless against infections because their immune systems are unable to combat them - they suffer from immunodeficiency diseases such as ADA-SCID (adenosine deaminase severe combined immunodeficiency) or Wiskott-Aldrich syndrome. Prof. Dr. Alessandro Aiuti, a physician and research scientist based in Milan who works at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and at the Vita Salute San Raffaele University, is now being honored with the Else Krner Fresenius Prize for Medical Research 2020 for his groundbreaking successes in the development of gene therapies. The award is coupled to 2.5 million euros in prize money.

In the case of the rare immune disorder ADA-SCID, which exclusively afflicts young children and occurs about 15 times a year in Europe, a defective ADA gene within the genome disrupts lymphocyte development, leaving the young patient's body defenseless against infections. "Without effective therapy, the children rarely survive for more than 2 years because any infection can become perilous for them," Aiuti explains. Standard for this therapy is a bone marrow transplantation from a fully matched sibling. However, a suitable donor is available only for a minority of patients. "Meanwhile children with such a condition benefit from the advances we have made in the field of gene therapy. So far we have treated 36 children from 19 countries using the therapy we developed. In more than 80 percent of the cases, the treatment has had such an impact that no enzyme replacement therapy or transplantation is needed. This achievement has been made possible by the extraordinary effort and dedication of SR-Tiget researchers and clinical team throughout 25 years," Aiuti adds. All of the patients are still alive.

For these successes and his other work in the field of gene therapy, Alessandro Aiuti has now been honored with the Else Krner Fresenius Prize for Medical Research 2020 awarded by the Else Krner-Fresenius-Stiftung (EKFS) foundation. At 2.5 million euros, this award is one of the highest endowed prizes for medical research in the world. "Still young by comparison, this year the prize is being awarded for the third time. It honors research scientists for pioneering contributions in the areas of biomedical science. A major percentage of the prize money flows into the prizewinner's research and is supposed to contribute toward achieving further groundbreaking findings and medical breakthroughs in the future as well," emphasizes Prof. Dr. Michael Madeja, scientific director and member of the management board at EKFS.

The decision regarding the prize recipient was made by a ten-member international jury composed of renowned research scientists in the fields of genome editing and gene therapy along with delegates from the Scientific Commission at EKFS. Prof. Dr. Hildegard Bning, chairwoman of the jury and president of the European Society for Gene and Cell Therapy (ESGCT), substantiates the jury's decision: "Alessandro Aiuti is a truly outstanding physician and scientist. His work has decisively contributed to the development and successful treatment of rare, genetically caused disorders such as SCID. Thanks not least of all to the contributions he has made, even patients with other inheritable illnesses can presumably be treated successfully in the future."

After successful clinical trials, the gene therapy developed for ADA-SCID patients was approved as a pharmaceutical remedy in Europe. It is considered to be one of the key findings in the development of gene therapies worldwide. With this treatment certain blood stem cells (CD34+) are taken from the patient, then the cell DNA is modified. The cells are treated outside the body using a viral vector to accomplish this. The correct version of the gene for the ADA enzyme is introduced into the genome of the cells that were collected. The genetically modified cells are returned to the patient's bloodstream via intravenous infusion. A portion of the modified cells subsequently establish themselves in bone marrow again. The patient now has blood stem cells that function properly and produce lymphocytes to defend against infections - presumably on a life-long basis.

Alessandro Aiuti wants to utilize the prize money from EKFS to set the success story forth, to optimize the therapies further and map out the healing mechanisms involved in a better fashion. The scientist sees another major challenge in conveying the acquired knowledge beyond the successful gene therapies from Milan to as many other genetic disorders as possible. Alongside the therapy for ADA-SCID, the San Raffaele Telethon Institute for Gene Therapy has also developed gene therapies for four more hereditary diseases, among them the Wiskott-Aldrich syndrome and metachromatic leukodystrophy (MLD). To this day a total of more than 100 patients from 35 different countries have been treated.

Biography of Alessandro Aiuti

Alessandro Aiuti was born in Rome in 1966 and studied medicine there at Sapienza University. Following a stay at Harvard Medical School in Boston, Massachusetts in the USA, he received his doctorate in Human Biology in 1996 from Sapienza University. Since 1997 he has been active at the San Raffaele Scientific Institute in Milan, where he meanwhile also teaches as a professor at the Vita Salute San Raffaele University. He is furthermore Deputy Director of Clinical Research at the San Raffaele Telethon Institute for Gene Therapy and Head of the Pediatric Immunohematology Unit at San Raffaele Hospital.

Aiuti is the author of numerous and highly acclaimed publications. Over the course of his career he has received a number of prizes from national and international institutions. Aiuti is a member of the board of the European Society of Gene and Cell Therapy, and a member of the EMA Committee for Advanced Therapies since 2019.

The Else Krner Fresenius Prize for Medical Research

The international Else Krner Fresenius Prize for Medical Research came into existence in 2013 on the occasion of the 25th anniversary of Else Krner's death and is awarded in alternating fields of biomedical science. Endowed with 2.5 million euros, the prize is one of the most highly endowed medical research awards in the world. It honors and supports research scientists who have made significant scientific contributions in their fields and whose work can be expected to yield groundbreaking findings and medical breakthroughs in the future as well.

The Else Krner-Fresenius-Stiftung (EKFS) foundation - Advancing research. Helping people.

The Else Krner-Fresenius-Stiftung, a non-profit foundation, is dedicated to the funding of medical research and supports medical/humanitarian projects. The foundation was established in 1983 by entrepreneur Else Krner and appointed as her sole heir. EKFS receives virtually all of its income in dividends from the Fresenius healthcare group, in which the foundation is the majority shareholder. To date, the foundation has funded around 2,000 projects. With a current annual funding volume around 60 million euros the EKFS is one of the largest foundations for medicine in Germany. More information:www.ekfs.de.

The San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than EUR 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

Issued by news aktuell/ots on behalf of Else Krner-Fresenius-Stiftung

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner...

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ReNeuron encouraged by progress in stroke and RP treatments – Proactive Investors Australia

Posted: June 4, 2020 at 8:55 am

What the company does

Human retinal progenitor cells (hRPC)

Human retinal progenitor cells differentiate into components of the retina.

Reneuron has developed the ability to scale up the manufacturing of hRPCs using a patented low-oxygen cell expansion technology.

The hRPC cell therapy candidate is being evaluated in an ongoing phase I/IIa clinical trial in the US in subjects with a blindness-causing inherited retinal disease, retinitis pigmentosa (RP).

CTX Cells

CTX cell therapy candidate is a treatment for patients left disabled by the effects of a stroke.

Reneurons product is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments.

Data from the Phase II PISCES trial indicated CTX therapy was safe and well-tolerated and produced clinically meaningful and sustained improvement in the level of disability and dependence as well as motor function.

Exosome platform

Exosomes are nanoparticles, released by cells, and contain a number of active proteins and micro RNAs, which are short non-coding RNAs capable of regulating gene expression, that arebelieved to play a key role in cell-to-cell communication.

ExoPr0, Reneurons first CTX-derived exosome therapeutic candidate, has demonstrated potential as both a novel therapeutic candidate as well as a drug delivery vehicle

hRPC

's ()human retinal progenitor cells (hRPC) have scored some early success.

A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment.

Six months after treatment there was a mean improvement of 18.5per treated eye, with a mean improvement of 12 letters per treated eye after nine months, whereasinexorable disease progression is the norm for this disease.

With a total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye threemonths after treatment.

In February, clinicaldata from the PISCES II clinical trial were published in peer-reviewedJournal of Neurology, Neurosurgery, and Psychiatry.

CTX

A peer journal review published in May indicated a CTX human neural stem cell line can rescue deficits associated with an accepted animal model of Huntington's disease, a progressive genetic brain disorder.

ReNeuron has previously presented data demonstrating that its CTX stem cell line, currently undergoing clinical evaluation for the treatment of stroke disability, can cause functional and behavioural recovery in animal models of ischemic (restriction of blood supply) injury.

The new data showed that implantation of CTX cells into a model of Huntington's disease can reduce inflammation, glial scar formation and induce host neurogenesis (the generation of new brain cells) leading to a recovery in behavioural deficits.

Coronavirus

In April, Reneuron said it haddeveloped a line of the human exosomes that can deliver a medically relevant payload: Viral vaccines thatmight help in the fight against coronavirus.

The stem cell specialist added that the disruption from lockdowns would inevitably lead to delays in the recruitment of patients for trials of its treatments for stroke disability and retinitis pigmentosa (RP).

It said it will update on how this will affect the release of top-line data from the two studies once it knows the full impact of the restrictions.

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ReNeuron encouraged by progress in stroke and RP treatments - Proactive Investors Australia

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Tiny Human Livers Grown in The Lab Have Been Successfully Transplanted Into Rats – ScienceAlert

Posted: June 4, 2020 at 8:55 am

Scientists have successfully transplanted functional miniature livers into rats, after growing the bioengineered organs in the lab from reprogrammed human skin cells.

The experiment, which gave the animals working liver organs, could lay the groundwork for future treatments to address terminal liver failure a disease that claims the lives of over 40,000 people in the US every year.

While there's still a lot of work to be done before the technique can directly aid human patients, the researchers say their proof of concept may help underpin a future alternative to liver transplants, which are often incredibly expensive procedures to perform, in addition to being strictly limited by donor supply.

Another positive outcome would be using the approach to temporarily augment failing liver function in patients, lengthening people's lives while they're on the waiting list for these vital organs: a situation facing about 14,000 Americans at any given moment, most of whom won't ever receive a transplant.

"The long-term goal is to create organs that can replace organ donation, but in the near future, I see this as a bridge to transplant," explains pathologist Alejandro Soto-Gutirrez from the University of Pittsburgh.

"For instance, in acute liver failure, you might just need hepatic boost for a while instead of a whole new liver."

To grow their mini livers, the researchers took human skin cells donated by volunteers and reverted them to a stem cell state, known as induced pluripotent stem cells, from which other kinds of cell types can be derived.

The researchers then induced differentiation in the cells with the aid of hormones and other chemicals, prompting them to become liver cells, which were cultured in the lab.

While it ordinarily takes two years for a human's liver to mature from the moment of their birth, the researchers were able to grow their miniature analogues in only a matter of weeks, seeding the grown cells on a rat liver scaffold that had been stripped of its rat cells.

While previous experimental liver graft research has incorporated rodent cells onto the scaffold, here the researchers used the human stem cells to populate the liver's functional tissue, along with its vascular system and bile duct network.

When transplanted into five rats, the mini livers appeared to be functional. After four days - at which point the animals were sacrificed and dissected - tests revealed that the bioengineered livers secreted bile acids and urea; human liver proteins in the animals' blood were another sign that the organs were working.

Not that the transplants functioned perfectly. Evidence of poor blood flow into the graft, in addition to thrombosis and ischemia, reveals there are still serious difficulties in properly connecting grafts like these to an animal's vascular network.

Nonetheless, it's still a remarkable achievement. For a short time, five rats lived their lives with miniature human livers, which isn't something that's ever been demonstrated before, and it could bring us closer to employing the same techniques for the benefit of human patients one day.

That day might be a long time away (perhaps a decade off, the researchers suggest), although it depends on a huge range of future experiments succeeding, including demonstrating that these kinds of engineered transplants are safe for humans, which remains to be seen.

In the meantime, methods like this including work pioneered by the same laboratory last year could enable the use of such mini organs to study simulated diseases and test different treatment options.

"I believe it's a very important step because we know it can be done," Soto-Gutirrez explained to Inverse. "You can make a whole organ that can be functional from one cell of the skin."

The findings are reported in Cell Reports.

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