Who are the 10 Most Innovative Biopharma Companies? – BioSpace

As the annual JP Morgan Healthcare Conference draws to a close, lets celebrate the remarkable innovation that drives the biopharma industry. Heres a look at the top 10 companies noted in the BioSpace Ideal Employer 2019 survey for being the most innovative and what theyve been up to recently.

Regeneron Pharmaceuticals. Ranked as the top innovator in the survey, Regeneron has long had a reputation for innovation, and is often cited as Sanofis innovation engine because of its numerous collaborations with the French-based company. Regeneron has seven marketed products, including Arcalyst for rare autoinflammatory disease, Eylea for a common cause of blindness, Praluent for high cholesterol, and Dupixent for atopic dermatitis. It also has REGN-EB3, a three-antibody therapy used to treat Ebola.

On January 9, Regeneron announced results from LUMINA-1, a Phase II trial of garetosmab in patients with fibrodysplasia ossificans progressive (FOP), an ultra-rare genetic disorder that leads to abnormal bone formation. After 28 weeks, the drug decreased total lesion activity compared to placebo by 25%.

Verily Life Sciences. Formerly known as Google Life Sciences, Verily is Alphabets life science research company. It was originally a division of Google X. At this point, the company doesnt have any marketed products and its not completely clear on the companys overall focus. It has numerous partnerships, such as one with Sanofi to develop products for managing diabetes, a disease-detecting nanoparticle platform called project Tricorder, and a partnership with Johnson & Johnson on surgical robotics. It also has partnerships with Alon, 3M, Allergan, Biogen, Dexcon, GlaxoSmithKline, Mayo Clinic, Brigham and Womens Hospital and many others.

On December 23, 2019, Verily partnered with Emory Healthcare to deploy new solutions to help improve cost-effectiveness, operational efficiency and quality. This deal with utilize Emorys academic medical center and partner with Verilys expertise in data science, analytics, user experience and product development. The initial focus is a deep analysis of existing drugs and lab-ordering patterns at Emory.

Illumina. Illumina develops, manufactures, and markets laboratory devices, with particular emphasis on DNA sequencing, genotyping, gene expression and proteomics. On January 4, 2020, Illumina announced a 15-year, non-exclusive deal with Roche. Not only will that increase the availability of next-generation sequencing-based in vitro diagnostic (IVD) tests on Illuminas systems, but the two companies will collaborate to complement Illuminas pan-cancer assay TruSight Oncology 500 (TSO 500) with new companion diagnostic (CDx) claims.

That deal came only a short time after Illumina canceled a $1.2 billion merger with another next-generation sequencing company, Pacific Biosciences (PacBio). Illumina decided the deal was not likely to be approved by antitrust regulators in the U.S. and UK. Illumina holds about 80% of the global DNA sequencing market.

bluebird bio. Bluebird bio focuses on the nascent field of gene therapy. Currently its sole approved product is Zynteglo. It was approved by the European Commission (EC) on June 14, 2019 for patients 12 years or older with transfusion-dependent beta-thalassemia who did not have a 0/0 genotype and for patients where hematopoietic stem cell (HSC) transplantation wasnt appropriate, but a human leukocyte antigen (HLA-matched related HSC donor isnt available. Its a little difficult to mention bluebird without mentioning the price of Zynteglo, which is $1.8 million in Europe. In addition to its scientific innovation, bluebird bio is innovative in terms of pricing structure. Zynteglos price is spread out over five years, with an initial upfront price of 315,000 euros with the four additional yearly payments due only if the treatment continues to work.

The product launched in Germany on Jan. 13, 2020. Bluebird initiated the rolling Biologics Licensing Application for Zynteglo in the U.S. and is currently in discussions with the U.S. Food and Drug Administration (FDA) on the timing and various components of the submission. They hope to complete the BLA submission in the first half of this year.

Biogen. Biogen specializes in therapies for central nervous system disorders. Some of its most well-known products are Alprolix for hemophilia B, Avonex, Fampyra, Tecfidera and Tysabri for multiple sclerosis, and Spinraza for spinal muscular atrophy (SMA). However, most recently, the company has been in the headlines for its aducanumab for Alzheimers disease. The drug was declared a failure in March 2019, but was resurrected this year after some of the later trial data showed effectiveness at the highest dose. Still, its not a slam dunk to be approved by the FDA and there are plenty of skeptics.

Analysts expect Biogen to submit aducanumab to the FDA in a matter of weeks, although the company is being tight-lipped about the timetable.

Bayer. Based in Germany, Bayer is one of the largest pharma companies in the world. In 2018, Bayer acquired U.S.-based Monsanto, which no longer exists under the Monsanto name. The companys business units include Bayer Crop Science, Consumer Health, Pharmaceuticals, Animal Health and Business Services.

On Jan. 16, 2020, Bayer sold one of its last Germany-based manufacturing facilities to Shanghai, China-based WuXi Biologics. The plant will be run by WuXi Biologics and act as a backup site for the manufacture of Bayers Kovaltry (antihemophilic factor). The primary site for Kovaltry product is Bayers facility in Berkeley, California.

Novartis. Based in Switzerland, Novartis has a well-known portfolio of drugs, including Clozaril, Voltaren, Tegretol, Diovan, Gleevec, and Ritalin. Its Sandoz Division is a global leader in generic drugs and biosimilars.

Last year, the FDA approved Zolgensma, a gene therapy for SMA, which was developed by its subsidiary, AveXis. Although there was some controversy over data manipulation in preclinical studies, it was determined not to affect the safety or efficacy of the therapy.

In late November 2019, Novartis acquired The Medicines Company for $9.7 billion, only a week after The Medicines Company announced positive data from its ORION-10 Phase III trial for inclisiran for lowering cholesterol.

GlaxoSmithKline. Headquartered in London, GSK markets drugs for numerous major diseases, such as asthma, cancer, infections, diabetes and mental health. Its best-known drugs include Advair, Augmentin, Flovent, Lamictal and others.

At the recent JP Morgan Healthcare Conference, Emma Walmsley, GSKs chief executive officer, predicted it will have six regulatory approvals in the U.S. this year.

I am pleased with the progress and the momentum that weve been able to make over the past couple years, she told CNBCs Jim Cramer.

Walmsley noted positive data for a number of programs, including ones gained from its $5.1 billion acquisition of Tesaro Oncology in 2018. She also noted a two-drug regimen for HIV that the companys subsidiary ViiV Healthcare has been developing.

Genentech. Generally viewed as the first modern biotechnology company, Genentech is a subsidiary of Swiss-based Roche, although Genentech is based in South San Francisco. It has a laundry list of successful drugs, often in the oncology market, such as Avastin, Tarceva, Zelboraf, Kadcyla, Alecansa, Venclexta and Tecentriq. It also has antivirals, such as Xofluza, Hemlibra for hemophilia A, and Esbriet for idiopathic pulmonary fibrosis.

On Dec. 12, 2019, Genentech announced that its Phase III IMspire150 trial in patients with previously untreated BRAF V600 mutation-positive advanced melanoma, hit its primary endpoint of progression-free survival (PFS). It showed adding Tecentriq to Cotellic and Zeleboraf decreased the risk of the disease getting worse or death, compared to placebo plus Cotellic and Zelboraf.

Amgen. Based in Thousand Oaks, California, Amgens best-selling products are Neulasta, an immunostimulatory for patients undergoing chemotherapy, and Enbrel, used to treat rheumatoid arthritis and other autoimmune diseases. Other products include Epogen, Aranesp, Prolia and XGeva.

On Nov. 1, 2019, the company expanded its presence in China by taking a 20.5% stake in China-based BeiGene Co. Amgen paid $2.7 billion in cash for the stake. As a result of the deal, BeiGene will commercialize Xgeva, Kyprolis and Blincyto in China.

On Jan. 13, 2020, the company inked strategic collaborations with Guardant Health and QIAGEN to develop blood- and tissue-based companion diagnostics for investigational cancer treatment AMG 510. AMG 510 is the first KRASG12C inhibitor to advance to the clinic for multiple cancer types.

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Who are the 10 Most Innovative Biopharma Companies? - BioSpace

Using RNA as a Delivery Vehicle Is the Magic Bullet to Cure Gene Mutation – Science Times

(Photo : cdn.the-scientist.com) One way to correct a cell that has undergone a mutation is by using RNA to transport proteins to cure ailing mitochondrion. This gene treatment has the promise to cure the most deadly diseases that are caused by mutations that harm it. If this cure is perfected then it will benefit everyone.

Mitochondria is the powerhouse of cells that fuels its functions, but sometimes there is a failure in this part of the cell which has deathly results. These specialists at the UCLA stem cell center and several departments have committed a study to unravel the mechanisms why it happens to the host cell. Clues to the working of the how and why of the mitochondrial disease will shed light on possible cures for it.

One of the causes of metabolic defects, aging, and especially neuromuscular diseases that are caused by mutations in the chromosomes and human mitochondria malfunctions. When this happens to the cell, it will be a slow march to death for the sufferer. Despite most advancements in medical technology, there is no cure to repair cellular deficiency. Finding a cure for it is committing experts like Dr. Michael Teitell, who is working along with others to find out ways to prevent it.

The tally of those afflicted with mitochondrial disease in the US is 1,000 to 4,000 alone per year. Other data indicates it will go full-blown by ten years old, these grim statistics are from the Mito Action Non-profit organization. They stress the need to commit more resources to seek more definitive cures for mitochondrial disease and other deadly diseases.

Stressing the discovery of using RNA as a possible corrector of bad genes, should give hope to a cure for this malady. Despite the use of RNA as a magic bullet, there is a need to complete the process to make the remedy viable. Human testing is not an alternative yet, with mitochondrial disease so other options are on the table. One is using test animals and later if proven successful, will be approve testing on human subjects.

The basis for the procedure is using a protein that is very essential for cellular processes. Teitel and Carla Koehler engineered the process, which will send more RNA molecules in the failing mitochondrion. As the RNA normalizes the mitochondrion, cellular processes are reawakened to cure the host cells. Properties of the RNA are made to repair the mitochondria, which will begin duplication of the right proteins in it. A drawback to knowing this is, more studies are needed to precisely target the cellular machinery.

For now, the study will derive a kind of gene therapy for restoring mitochondria to normal, lessening the chances of mutations to bog it down. Another benefit is more treatment to correct these cells based problems. As a first step, the insights gained from the study will make a big contribution to current knowledge.

Targeted RNA as the delivery system to send the regenerative proteins to fix, badly functioning mitochondrion has a good chance of treating similar diseases. Another specialist (Geng Wang) reaffirmed the process, with a similar way to deliver RNA molecules. When inserted inside the hosts' nucleus to unpack its cargo to do its work. These similar methods address what failures happen when genes mutate and multiply the wrong cell proteins.

Further refinement of the RNA delivery vehicle will give a chance for sufferers to be cured. Corrections to a mutation in human mitochondrion will allow fewer problems connected to it. If there is a magic bullet, then it might just pan out.

Related Article: Using RNA Import to Repair Mutations in Human Mitochondria

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Using RNA as a Delivery Vehicle Is the Magic Bullet to Cure Gene Mutation - Science Times

The F.D.A. Is in Trouble. Heres How to Fix It. – The New York Times

Fortunately, options for fortifying the F.D.A. abound. For instance, laws that would make it easier for regulators to police the cosmetics industry and to hold medical device companies to account have been floating through Congress for years. A group of former F.D.A. commissioners last year proposed an even bolder fix: Restore the agencys autonomy by extracting it from the Department of Health and Human Services. The F.D.A.s decisions used to be final, but for decades now they have been subject to layers of political interference. Making the agency independent, as the Federal Reserve and the Social Security Administration are, could help reverse that trend.

But for these or other worthy ideas to get a fair hearing, Congress will have to step in, and the president and the electorate will need to come to terms with the essential role of regulations in protecting the nations food and drug supply.

In the meantime, the challenge of steering the F.D.A. will fall to Dr. Hahn. He will not have nearly enough resources to carry out the agencys stated mission no commissioner ever does. But he will not be completely powerless, either. Here are four things Dr. Hahn would do well to keep in mind as he takes the reins.

Stay vocal. Dr. Hahns predecessor, Dr. Gottlieb, managed to keep a spotlight on his chosen priorities namely e-cigarette regulations and generic drug development with a relentless and multifaceted public messaging campaign. He tweeted, he blogged, he gave speeches and he communicated openly and regularly with the press. Dr. Gottlieb did not achieve all of his goals in fact his e-cigarette strategy backfired, badly. But he made the F.D.A. less opaque, and he gave the agency an urgently needed voice. Dr. Hahn will have an easier time defending the agency, and keeping it relevant, if he fosters the same transparency.

Slow down on drug and device approvals. The F.D.A. has made several compromises in recent years such as accepting real world or surrogate evidence in lieu of traditional clinical trial data that have enabled increasingly dubious medical products to seep into the marketplace. Dr. Hahn ought to take a fresh look at some of these shifting standards and commit to abandoning the ones that dont work. That will almost certainly mean that the approval process slows down and thats O.K.

Stand up for science. As reporting from the medical news website Stat and other outlets suggests, the F.D.A. has become too susceptible to outside pressure. Regulators approved a powerful new opioid at the Department of Defenses urging, fast-tracked a dubious antidepressant after President Trump praised it, and reversed its decision to reject a muscular dystrophy drug after patient groups complained loudly. Such kowtowing hardly inspires confidence. Scientific evidence (or the lack thereof) needs to be the deciding factor in any final regulations from the F.D.A. That means saying no to politicians and drug and device makers as well as patients groups when their demands are not supported by the agencys own findings. It also means holding companies to account when they fail to complete postmarket studies, or when their products prove faulty or dangerous.

Follow through on existing commitments. The F.D.A. has yet to issue guidelines for the regulation of increasingly popular CBD products after promising to do so by the end of 2019. E-cigarette makers are supposed to submit their applications for market approval to the agency by May. And a regulatory grace period that the agency granted to so-called stem cell clinics back in 2017 is set to expire this year; when it does, regulators will need to figure out how to police nearly 1,000 businesses selling injections and other treatments that have not proved to work and that have already caused some patients serious harm. Dr. Hahn would build a lot of good will if he showed the F.D.A.s critics and the public at large that he takes all of these deadlines seriously.

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The F.D.A. Is in Trouble. Heres How to Fix It. - The New York Times

Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami – Yahoo Finance

2020 Honorees include Cystic Fibrosis Foundation, Emily Whitehead Foundation, Gift of Life Marrow Registry and Ret. Major General Bernard Burn Loeffke (US Military)

Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

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To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

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Joseph DawsonRegenerative Medicine Foundation561-906-4755joseph@regmedfoundation.org

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami - Yahoo Finance

John Theurer Cancer Center Announces Appointment of Five New Physicians – Newswise

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John Theurer Cancer Center Announces Appointment of Five New Physicians

Cancer doctors bolster center's stem cell transplantation, blood cancer, and thoracic cancer programs.

Newswise HACKENSACK, N.J.,January 9, 2019 Five new physicians have joined the medical staff at John Theurer Cancer Center at Hackensack University Cancer Center in New Jersey:

Hyung C. Suh, MD, PhD, is a hematologist-oncologist who joined the Stem Cell Transplantation and Cellular Therapy program. Dr. Suh specializes in the care of people with blood cancers (such as leukemia, lymphoma, and multiple myeloma) and in the use of stem cell transplantation, cellular therapy, and immunotherapy to treat these cancers. He is also an experienced biomedical scientist with expertise in hematology-oncology research and the translation of research findings from the laboratory to the patients they may help. A native of South Korea, Dr. Suh graduated from Yonsei University College of Medicine in Seoul, where he earned his MD and PhD degrees. He completed internal medicine residencies at Yonsei University College of Medicine and Cleveland Clinic Foundation and hematology-oncology fellowships at Yonsei University College of Medicine and UCLA School of Medicine.

Gurbakhash Kaur, MD, is a hematologist-oncologist who specializes in hematology-oncology, especially the treatment of multiple myeloma. She is especially interested in the application of novel immunotherapies to treat cancer, including multiple myeloma. Dr. Kaur received her medical degree from Drexel University College of Medicine and completed her internal medicine residency at Tufts Medical Center. She then went on to complete a fellowship in hematology-oncology at Montefiore Medical Center.

Sukhdeep Kaur, MD, is a hematologist-oncologist who specializes in stem cell transplantation including allogeneic transplants (from a donor) and autologous ("self") transplants for blood cancers and blood diseases. She also oversees the use of cellular therapies in patients, including CAR T-cell therapy an innovative treatment which trains a patient's white blood cells (called T cells) to find and destroy cancer cells. Dr. Kaur received her medical degree from Ross University and completed her internal medicine residency at Drexel University College of Medicine, serving as Chief Resident in her final year. She then went on to complete a fellowship in hematology-oncology at Robert Wood Johnson University Hospital/Cancer Institute of New Jersey, where she was Chief Fellow.

Andrew Ip, MD, MS, is a hematologist-oncologist who specializes in the care of patients with lymphoma and multiple myeloma. In his research, Dr. Ip is part of John Theurer Cancer Center's Outcomes Division. He is interested in utilizing population science to see the "big picture" of cancer care in order to enhance the lives of patients and their families. He also has an interest in using digital health technology to promote improved cancer outcomes, having piloted an Apple Watch physical activity intervention for people with cancer. Dr. Ip received his medical degree from Jefferson Medical College (now Sidney Kimmel Medical College at Thomas Jefferson University). He completed his internal medicine residency at Emory University School of Medicine, where he was Chief Medical Resident in his last year. He then went on to complete a fellowship in hematology and medical oncology at the Winship Cancer Institute at Emory, where he was Chief Fellow for his final year.

Kaushal Parikh, MBBS, is a medical oncologist who specializes in the treatment of patients with thoracic cancers, such as lung cancer, thymoma, and mesothelioma. He is also involved in drug development and early-phase clinical trials, particularly through John Theurer Cancer Center's robust Phase I clinical trials program. He and his colleagues are working to expand the translational research program so that more findings from the laboratory can be translated to the clinic to help patients. Dr. Parikh received his medical degree from Topiwala National Medical College in Mumbai, India. He completed his internal medicine residency and hematology-oncology fellowship at New York Medical College, where he was Chief Hematology and Oncology Fellow for his final year. He then completed a fellowship in thoracic oncology at the Mayo Clinic School of Medicine in Minnesota.

For more information, contact Mary McGeever, Hackensack University Medical Center Communications and Public Relations Department, at 551-996-1730 (office), 551-795-1675 (cell) or Mary.McGeever@HackensackMeridian.org

ABOUTJOHN THEURER CANCER CENTERATHACKENSACKUNIVERSITY MEDICAL CENTER

John Theurer Cancer CenteratHackensack University Medical CenterisNew Jersey'slargest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in theNew Jersey/New York metropolitan area turn toJohn Theurer Cancer Centerfor cancer care than to any other facility inNew Jersey. Housed withinHackensack University Medical Center, a 775-bed not-for-profit teaching, tertiary care, and research hospital, John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguishJohn Theurer Cancer Centerfrom other facilities.For additional information, please visitwww.jtcancercenter.org.

About Hackensack Meridian Hackensack University Medical Center

Hackensack Meridian Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care, which is comprised of 35,000 team members and more than 7,000 physicians. Hackensack University Medical Center is ranked #2 in New Jersey and #59 in the country in U.S. News & World Reports 2019-20 Best Hospital rankings and is ranked high-performing in the U.S. in colon cancer surgery,lung cancersurgery,COPD, heart failure, heart bypass surgery, aortic valve surgery,abdominal aortic aneurysm repair, knee replacement and hip replacement. Out of 4,500 hospitals evaluated, Hackensack is one of only 57 that received a top rating in all nine procedures and conditions. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 26 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its sixth consecutive designation in 2019. Hackensack University Medical Center has created an entire campus of award-winning care, including: John Theurer Cancer Center, a consortium member of the NCI-designated Georgetown Lombardi Comprehensive Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

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John Theurer Cancer Center Announces Appointment of Five New Physicians - Newswise

Orgenesis Announces Addition of University of California, Davis, to its Point of Care Network; UC Davis Health to Utilize Orgenesis Point of Care…

First collaboration project focused on developing and commercializing lentiviral manufacturing system

GERMANTOWN, Md., Jan. 10, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (ORGS), a leading cell and gene therapy enabling company providing centralized CDMO manufacturing and development services, as well as localized point-of-care development and processing centers through its subsidiary Orgenesis Maryland, Inc., today announced the addition of the University of California, Davis (UC Davis) to its Point of Care (POCare) Network. Under the collaboration agreement, UC Davis Health will utilize Orgenesis POCare platform to develop, commercialize and supply cell and gene products and therapies. Orgenesis POCare Network enables hospitals to design and manage localized clean rooms, implementing Orgenesis proprietary automated, closed systems and know-how to process select cell therapies at each point-of-care site for the treatment of patients.

The first collaboration under the agreement involves scaling up and integrating UC Davis lentiviral vector process as part of the Orgenesis POCare platform for localized, development and processing of cell and gene therapies for treating patients. The UC Davis GMP facility has developed a small-intermediate scale, high quality vector process that has been successfully utilized to manufacture lentiviral vectors in several clinical trials, including manufacturing of CAR T cell therapies. Orgenesis POCare platform, which combines processing and therapeutic technologies, is designed to allow for the efficient production of high quality, affordable cell and gene based products. Upon successful completion of the collaboration, Orgenesis and UC Davis plan to pursue further commercialization of the technology and expand the processing and supply of their products under development at the UC Davis site. Lentivirus is a family of viruses that insert their DNA into the host cells' genome. Lentiviral vectors are increasingly utilized in cell and gene therapy as a method for inserting, modifying, or deleting specific genes within cells.

Vered Caplan, CEO of Orgenesis, stated, We are delighted to add UC Davis to our POCare Network, which will allow us to collaborate with the university to develop and supply therapeutics within the point-of-care setting in general and specifically in our need for virus supply. Additionally, we look forward to leveraging our POCare platform to assist UC Davis in expanding their ability to address the worldwide shortages of lentiviral vectors. Their new system is designed to address the global need for a more efficient, large scale vector manufacturing processes in an efficient manner. We believe this partnership further validates the significant value proposition of our POCare platform.

Adjunct Professor Gerhard Bauer, Director of the GMP Facility at UC Davis, commented, We, at the UC Davis Health, in the Stem Cell Program and in the GMP Facility, are committed to bringing these novel cell and gene therapy based treatments to patients in need and making them affordable.

Professor Jan A. Nolta, Director of the Stem Cell Program and the Gene Therapy Center at UC Davis Health, added, We look forward to leveraging Orgenesis expertise to accelerate the development and commercialization of our lentiviral vector manufacturing system, which addresses a significant unmet need in the market for an efficient and scalable manufacturing process.

About Orgenesis

Orgenesis is a biopharmaceutical company specializing in the development, manufacturing and processing of technologies and services in the cell and gene therapy industry. The Company operates through two platforms: (i) a point-of-care (POCare) cell therapy platform (PT) and (ii) a Contract Development and Manufacturing Organization (CDMO) platform conducted through its subsidiary, Masthercell Global. Through its PT business, the Companys aim is to further the development of Advanced Therapy Medicinal Products (ATMPs) through collaborations and in-licensing with other pre-clinical and clinical-stage biopharmaceutical companies and research and healthcare institutes to bring such ATMPs to patients. The Company out-licenses these ATMPs through regional partners to whom it also provides regulatory, pre-clinical and training services to support their activity in order to reach patients in a point-of-care hospital setting. Through the Companys CDMO platform, it is focused on providing contract manufacturing and development services for biopharmaceutical companies. Additional information is available at: http://www.orgenesis.com.

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About UC Davis Stem Cell Program and Gene Therapy Center

UC Davis Stem Cell Program brings together physicians, research scientists, biomedical engineers and a range of other experts and collaborative partners at its Institute for Regenerative Cures, which is located on the universitys Sacramento campus. The $62 million facility, which was supported by the California Institute for Regenerative Medicine (CIRM), is the hub for collaborative, team-oriented science that is advancing breakthrough discoveries designed to bring stem cell therapies and cures to patients everywhere.

The UC Davis Gene Therapy Center brings together a uniquely comprehensive and established interdisciplinary network of experts and resources to lead the field of gene therapy through research, manufacturing, training and policy. The Gene Therapy Center offers expertise and state-of-the-art facilities and equipment including one of the largest university-based Good Manufacturing Practice (GMP) facilities and a Viral Vector Core.

About UC Davis

UC Davis is one of the top public universities in the United States. Since opening in 1908, it has been known for standout academics, sustainability and Aggie Pride as well as valuing the Northern California lifestyle. These themes are woven into its 100-plus-year history and its reputation for solving problems related to food, health, the environment and society. The universitys health system is based in Sacramento and provides the region's only academic health center. UC Davis Health is focused on discovering and sharing knowledge and providing the highest quality of care. It is a hub of innovation that encompasses UC Davis Medical Center, UC Davis School of Medicine, The Betty Irene Moore School of Nursing at UC Davis and UC Davis Medical Group.

Notice Regarding Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, the success of our reorganized CDMO operations, the success of our partnership with Great Point Partners, our ability to achieve and maintain overall profitability, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans-differentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended November 30, 2018, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:Crescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

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Orgenesis Announces Addition of University of California, Davis, to its Point of Care Network; UC Davis Health to Utilize Orgenesis Point of Care...

Aspen Neuroscience Announces Board of Directors and Scientific Advisory Board – PRNewswire

SAN DIEGO, Jan. 9, 2020 /PRNewswire/ -- Aspen Neuroscience, Inc.,a private biotechnology company developing the first autologous neuron replacement therapy to treat Parkinson disease, has announced the members of its Board of Directors and Scientific Advisory Board. These boards bring together many of the world's most distinguished industry and academic minds to deliver Aspen's personalized cell therapy to persons suffering with Parkinson disease.

"We are proud of the talented groups we have assembled," said Aspen CEO Howard Federoff, MD, PhD. "They bring a wealth of insights and experience in biomedical research and therapeutic development that will be essential for our success. Together, we are motivated to bring best-in-class treatments to Parkinson patients as rapidly as possible. They have waited long enough."

Last month, Aspen announced it has raised $6.5 million in seed fundingand unveiled its plans to use neurons derived from induced pluripotent stem cells (iPSCs) to combat the debilitating motor symptoms of Parkinson disease. These iPSCs will be created from each patient's own cells and then differentiated into dopamine-producing neurons, replacing the critical cells lost in Parkinson disease. Because the therapy is created from each patient's own tissue, no immunosuppression will be necessary.

"The creation of our esteemed Board of Directors and Scientific Advisory Board is an important step for Aspen's future development as it will help guide the company's progress toward regulatory approval," said Kim P. Kamdar, PhD, Aspen Board Chair and Partner at Domain Associates. "The members of each board bring significant expertise, complementary skills and an external perspective, which will be very valuable in informing Aspen's strategy."

Aspen's Board of Directors includes well-known industry veterans and financial minds from leading venture capital firms that are poised todirect the growth of Aspen through clinical trials and regulatory approval.

Aspen Neuroscience Board of Directors

The Scientific Advisory Board consists of leaders in the fields of bioethics, regenerative medicine clinical trials, autologous iPSC-based therapy, and Parkinson disease neuron replacement and neuroimaging.

Aspen Neuroscience Scientific Advisory Board

The newly established boards will join Aspen's impressive leadership team: Howard J. Federoff, MD, PhD, Chief Executive Officer; Jeanne Loring, PhD, Chief Scientific Officer; Edward Wirth, MD, PhD, Chief Medical Officer; Jay Sial, Chief Financial Officer; Andres Bratt-Leal, PhD, Vice President of Research and Development; Thorsten Gorba, PhD, Senior Director of Manufacturing; and Naveen M. Krishnan, MD, MPhil, Senior Director of Corporate Development.

About Aspen NeuroscienceAspen Neuroscience, Inc., is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson disease. Aspen's therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visithttp://www.aspenneuroscience.com.

SOURCE Aspen Neuroscience

https://www.aspenneuroscience.com

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Aspen Neuroscience Announces Board of Directors and Scientific Advisory Board - PRNewswire

How Kyoto Is Rebuilding Itself As A Nanotech And Regenerative Medicine Powerhouse – Forbes

As humans continue to pump more and more carbon dioxide into the atmosphere, concerns about global warming and climate change continue to grow. But what if that CO2 could be turned into a source of energy? One startup in Kyoto has developed cutting-edge nano-materials that could trap atmospheric CO2 and harness it as a power source. Its one way that Japans ancient capital is harnessing its large scientific and biomedical potential to address environmental and social problems.

Panning for invisible gold

Porous coordination polymers can be a form of carbon-capture technology, says discoverer Susumu Kitagawa, second from left, with (left to right) Atomis CTO Masakazu Higuchi, CEO Daisuke Asari, R&D officer Kenji Sumida, and COO Dai Kataoka.

Atomis is a new materials company that was spun off from Kyoto University. Founded in 2015 following government-supported research, its business is based on studies led by Susumu Kitagawa, a professor in the universitys Institute for Advanced Study. Its core technology is the production of materials comprising extremely small void spaces that can trap gases, including CO2. A breakthrough discovery in 1997 by Kitagawa, who has been considered a contender for the Nobel Prize in Chemistry, these porous coordination polymers (PCPs, aka metal-organic frameworks) have enormous potential as tools to precisely control gases.

Humans have used the principle behind PCPs for thousands of years. They work the same way that a hunk of charcoal traps ambient odor molecules in its large surface area, but PCPs are many times more powerful. To the naked eye, PCPs look like powders, pellets or granules of various colors, shapes and sizes. But if you were to zoom in, you would see that PCPs are sponge-like materials with pores the size of a nanometer, or one billionth of a meter. They can be designed as scaffoldlike 3D structures from metals and organic ligands, and can be used for storage, separation and conversion of molecules.

These materials are unique in that we can design the shapes and chemical properties of the pores to suit specific applications, and some of the materials have flexible structures, which can potentially provide them with even more advanced features, says Daisuke Asari, president and CEO of Atomis. The company is basically the only business in Japan working with these materials in an industrial context. Collaborating with Kitagawa is a big advantage over foreign rivals, adds Kenji Sumida, executive officer for R&D.

One challenge related to these nanomaterials is that its difficult and costly to produce more than a few kilograms per day. Massively scaling production so that PCPs can be used to fight climate change is one reason that Atomis was founded, says Atomis founder and CTO Masakazu Higuchi, one of Kitagawas collaborators. The firm is developing solid-state techniques and making capital investments to increase PCP production capacity. Meanwhile, Atomis has developed products that harness the groundbreaking potential of PCPs, including Cubitan, a compact and lightweight gas cylinder for industrial and consumer use packed with smart features, such as the ability to notify users when the amount of reserve gas becomes low.

When viewed without special equipment, PCPs look like powders, pellets or granules of various colors, shapes and sizes, but they are sponge-like materials with countless pores the size of a nanometer.

Kitagawa has his sights on the bigger picture. He believes PCPs can be used as a form of carbon-capture technology, allowing the synthesis of methanol, an energy source. Thats why he calls CO2 invisible gold.

In ancient China, Taoist mystics were said to live in the mountains and survive simply on mist, which consists of water, oxygen and CO2, says Kitagawa. They were taking something valueless and using it for energy. Similarly, PCPs can control gases that humans cannot use and turn them into something beneficial, for instance absorbing CO2 in the air and turning into methanol and other hydrocarbon materials.

Building a regenerative medicine Silicon Valley

Atomis is one of many science startups in Kyoto that have benefitted from collaborative research between industry and government. Its part of a growing startup industry in Japan, where total funding for new companies reached a record high of 388 billion yen in 2018, up from 64.5 billion yen in 2012, according to Japan Venture Research. One driver for this expansion is science and technology discoveries.

While it may be known for its traditional culture, Kyoto has a strong pedigree in scientific research. It is home to 38 universities and about 150,000 students, which form a large pool of institutional knowledge, experience and talent. Many recent Nobel laureates either graduated from or taught at Kyoto University, including professors Tasuku Honjo and Shinya Yamanaka, who won the Nobel Prize for Physiology or Medicine in 2018 and 2012, respectively. Working on discoveries by Yamanaka, Megakaryon has become a world leader in creating artificial blood platelets made from synthetic stem cells.Theres also a large group of high-tech companies that have carved out niches for themselves internationally.

Kyoto is a unique city in that it has an independent spirit that is similar to the U.S. West Coast, says Eiichi Yamaguchi, a professor at Kyoto University who has founded four companies.

Kyoto companies like Murata Manufacturing, Horiba, Shimadzu, and Kyocera have a global market and theyre competing with China, says Eiichi Yamaguchi, a professor at Kyoto University who has founded four companies. Thats the difference with companies in Tokyo, which are more domestically oriented.

Yamaguchi has authored several books on innovation, and says there is a growing awareness of the importance of collaborative research and entrepreneurship in Kyoto. He cites a recently formed cooperative group of seven university chairpersons and presidents from leading materials and biosciences companies that meets to discuss issues such as fostering new technologies, for instance building high-speed hydrogen fueling systems.

Kyoto is a unique city in that it has an independent spirit that is similar to the U.S. West Coast, says Yamaguchi. Kyoto is only a fraction of the size of Tokyo, but if you take a stand here, people will pay attention.

Another group that is promoting local high-tech business is Innovation Hub Kyoto. Its an open innovation facility based in the Kyoto University Graduate School of Medicine aimed at commercializing research from the university. Steps away from Kyotos historic Kamo River, its geared to researchers, investors, startups, and established companies working in the field of medical innovation including device development and drug discovery. This is where Japanese researchers are trying to build a Silicon Valley of regenerative medicine.

Tenants at Innovation Hub Kyoto can use this wet lab for research.

Part of the Kyoto University Medical Science and Business Liaison Organization, the hub was established about 15 years ago and opened a new building in 2017 with the support of the Ministry of Education, Culture, Sports, Science and Technology. The structure has a variety of labs, including ones meeting biosafety level P2 and for animal experiments.

Its tough for startups in Japan to access to animal laboratories like the one we have, says hub leader Yutaka Teranishi, a professor in the Graduate School of Medicine who estimates that some 50% of university researchers want to work with industry, up from 10% a few years ago. Were focused on university startups because its very difficult for them to develop drugs from just an alliance between companies and universities.

About 28 companies are tenants at Innovation Hub Kyoto. They include major brands such as Shimadzu and Nippon Boehringer Ingelheim as well as younger businesses. One is AFI, founded in 2013 and focused on fluid, electric filtering and sorting (FES) technology that can be used for applications ranging from food safety inspections to rapid diagnosis of disease to regenerative medicine.

Tomoko Bylund heads the Japan office of CELLINK, a Swedish bioprinting and bioink company that is a tenant at Innovation Hub Kyoto.

Another tenant is CELLINK, a Swedish bioprinting and bioink company headed in the Japan by Tomoko Bylund. Using its products, researchers can print body parts with human cells for drug and cosmetics testing. In 2019, the first 3D print of a human cornea in the U.S. was accomplished with the companys BIO X Bioprinter.

iHeart Japan is also a tenant. It was established in 2013 as a regenerative medicine business and is aiming to address a major shortage in the Japanese medical system: only about 40 out of 200,000 people on national waiting lists can receive donor hearts every year. The company is developing innovative medical products such as multi-layered cardiac cell sheets derived from synthetic stem cells. The Hub basis its success in fostering companies on its diversity and the business environment in Kyoto.

We have people from different backgrounds here who are exchanging cultures and experimental results, and this diversity is powering innovation here, says Teranishi. There are many traditional industries in Kyoto, and though people say its a conservative city, these companies have survived because theyre open to new technologies and have taken the time to choose which ones can help them. Thats how this city and its businesses have lasted for more than 1,000 years.

Diversity is powering innovation here, says Yutaka Teranishi, center, head of Innovation Hub Kyoto, with Kyoto University professor Hirokazu Yamamoto, left, and Graduate School of Medicine lecturer Taro Yamaguchi, right.

To learn more about Atomis, click here.

To learn more about Innovation Hub Kyoto, click here.

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How Kyoto Is Rebuilding Itself As A Nanotech And Regenerative Medicine Powerhouse - Forbes

ASH 2019 | The impact of donor clonal hematopoiesis on aGvHD and patient outcomes – AML Global Portal

The presence of preleukemic mutations in peripheral blood (PB) samples is termed clonal hematopoiesis of indeterminate potential (CHIP). CHIP is defined as the absence of definitive morphologic evidence of hematologic neoplasms, with the presence of a somatic mutation with a variate allele frequency (VAF) of > 2%. The incidence of CHIP increases with age and comes with an increased risk of developing myeloid malignancies and cardiovascular complications. Therefore, using older donors with CHIP may impact the outcomes of patients undergoing transplantation.

During the 61st meeting of the American Society of Hematology (ASH), Betul Oran, MD Anderson Cancer Center, Houston, US, presented the results from a study which evaluated the impact of donor clonal hematopoiesis on the risk of acute graft-versus-host disease (GvHD, aGvHD) and patient outcomes. The trial was conducted in patients with AML or MDS who received a transplant from a matched-related donor (MRD) aged 55 years or older.

Table 1. Impact of CHIP on transplant outcomes

CHIP positive

CHIP negative

HR

95% CI

p value

N

57

245

-

-

-

Relapse incidence (RI) at 5-years

Not reported (NR)

NR

0.9

0.51.5

0.7

Age-adjusted RI at 5-years

NR

NR

0.9

0.61.4

0.7

Progression incidence at 5-years, %

40

44

0.9

0.51.4

0.5

TRM at 6 months, %

12

9

1.6

0.54.9

0.4

Age adjusted RM at 6 months

NR

NR

1.3

0.62.9

0.5

PFS at 5-years, %

38

36

0.97

0.71.4

0.9

Age adjusted PFS at 5-years

NR

NR

0.96

0.71.4

0.8

OS at 5-years, %

43

41

1.05

0.71.5

0.8

Table 2. Impact of donor CHIP on rates of GvHD

CHIP positive

CHIP negative

HR

95% CI

p value

N

57

245

-

-

-

Grade II-IV aGvHD at 6 months

Total, %

51

27

2.1

1.43.3

0.001

Donor > 65 years, %

54

27

2.1

1.054.4

0.04

Donor 65 years %

48

28

2

1.13.5

0.001

Grade III-IV aGvHD at 6 months

Total, %

16

5

3.2

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ASH 2019 | The impact of donor clonal hematopoiesis on aGvHD and patient outcomes - AML Global Portal

New protocol could signal shift in bone regenerative medicine – PR Web

DURHAM, N.C. (PRWEB) January 06, 2020

A new, safe and efficient way to coax stem cells into bone cells is reported in a recently published article from STEM CELLS Translational Medicine (SCTM). The protocol, developed by researchers at the University of Sydney, Australian Research Centre (ARC) for Innovative BioEngineering, could lead to a shift in the treatment of bone regenerative medicine.

Large bone defects and loss due to cancer or trauma can result in scar tissue that impairs the bones ability to repair and regenerate. The current gold standard therapy, autografting, has inherent drawbacks, including limited availability and donor site morbidity. This leaves researchers seeking an alternative source of bone cells and makes bone tissue engineering a growing field with considerable translational potential.

The success of induced pluripotent stem cell (iPSC) technology to reprogram fibroblasts into progenitor cells of various lineages offers an exciting route for tissue repair and regeneration, said Zufu Lu, Ph.D., a member of the University of Sydneys Biomaterials and Tissue Engineering Research Unit and a research associate at the ARC for Innovative BioEngineering. He is a co-lead investigator of the SCTM study, along with Professor Hala Zreiqat, Ph.D., head of the research unit and director of the ARC Training Centre for Innovative BioEngineering.

However, while iPSC technology represents a potentially unlimited source of progenitor cells and allows patients to use their own cells for tissue repair and regeneration thus posing little or no risk of immune rejection the technology has several constraints. Among them are the requirement for complex reprogramming using the Yamanaka factors (Oct3/4, Sox2, Klf4, c-Myc). To add to the complexity, specific stimuli are required to direct iPSCs to re-differentiate to progenitor cells of the lineage of interest.

In addition, Dr. Lu said, any remaining iPSCs pose the risk of tumors following implantation.

One potential way around this, as demonstrated by recent studies, is through the direct reprogramming of fibroblasts into bone cells. Fibroblasts are morphologically similar to osteoblasts. Their similar transcriptomic profiles led us to hypothesize that distinct factors produced by osteoblasts may be capable of coaxing fibroblasts to become osteoblast-like cells, Prof. Zreiqat said.

Previous studies aimed at using fibroblasts to produce various cell types relied on the genetic manipulation of one or more transcription regulators. But just as with iPSCs, reprogramming fibroblasts in this manner has its own inherent technical and safety issues. The Lu-Zreiqat team, however, surmised that an approach employing natural factors might just allow better control over reprogramming and improve the safety.

Unlike genetic reprogramming, chemical induction of cell reprogramming is generally rapid and reversible, and is also more amenable to control through factor dosage and/or combinations with other molecules, Dr. Lu explained.

The team initially determined that media conditioned by human osteoblasts can induce reprogramming of human fibroblasts to functional osteoblasts. Next, said Prof. Zreiqat, our proteomic analysis identified a single naturally bioactive protein, insulin growth factor binding protein-7 (IGFBP7), as being significantly elevated in media conditioned with osteoblasts, compared to those with fibroblasts.

This led them to test IGFBP7s ability as a transcription factor. They found it, indeed, successfully induced a switch from fibroblasts to osteoblasts in vitro. They next tested it in a mouse model and once again experienced success when the fibroblasts produced mineralized tissue. The switch was associated with senescence and dependent on autocrine IL-6 signaling.

The approach we describe in our study has significant advantages over other commonly used cell sources including iPSCs and adult mesenchymal stem cells, Dr. Lu and Prof Zreiqat concluded.

Bone tissue engineering is a growing field where cell therapies have considerable translational potential, but current cell-based approaches face limitations, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. The novel observation described in this study could potentially lead to a shift in the current paradigm of bone regenerative medicine.

This study was conducted in collaboration with the Charles Perkins Centre and the Childrens Hospital at Westmead, University of Sydney.

The full article, Reprogramming of human fibroblasts into osteoblasts by insulin-like growth factor binding protein 7, can be accessed at https://stemcellsjournals.onlinelibrary.wiley.com/doi/abs/10.1002/sctm.19-0281.

About STEM CELLS Translational Medicine: STEM CELLS Translational Medicine (SCTM), co-published by AlphaMed Press and Wiley, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices. SCTM is the official journal partner of Regenerative Medicine Foundation.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS (http://www.StemCells.com), celebrating its 38th year, is the world's first journal devoted to this fast paced field of research. The Oncologist (http://www.TheOncologist.com), also a monthly peer-reviewed publication, entering its 25th year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

About Wiley: Wiley, a global company, helps people and organizations develop the skills and knowledge they need to succeed. Our online scientific, technical, medical and scholarly journals, combined with our digital learning, assessment and certification solutions, help universities, learned societies, businesses, governments and individuals increase the academic and professional impact of their work. For more than 200 years, we have delivered consistent performance to our stakeholders. The company's website can be accessed at http://www.wiley.com.

About Regenerative Medicine Foundation (RMF): The non-profit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF pursues its mission by producing its flagship World Stem Cell Summit, honouring leaders through the Stem Cell and Regenerative Medicine Action Awards, and promoting educational initiatives.

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New protocol could signal shift in bone regenerative medicine - PR Web