Category Archives: Stem Cell Medical Center

Toxys Strengthens Leadership Team with Appointment of CBO and COO Positions – BioSpace

LEIDEN, The Netherlands, January 21, 2020 /B3C newswire/ --Today,Toxysannounces the appointment of a Chief Business Officer (CBO) and a Chief Operating Officer (COO). Toxys BV is a biotech company based in Leiden, The Netherlands, that provides innovative, high-fidelityin vitrotoxicity screening solutions to identify carcinogenic and other hazardous properties of compounds for the pharmaceutical, chemical, cosmetics and food industries. With this strengthened leadership, Toxys will further accelerate business growth to become an industry-leader in animal-free chemical safety testing.

Paula van Rossumis promoted toCBO. Paula joined Toxys in 2016 as Director Business Development & Sales and has been instrumental for the business growth of Toxys. Paula holds a MSc degree from Leiden University in Biomedical Sciences and Science-based Business.

Remco Derris promoted toCOO. Remco joined Toxys in 2014 as lab manager and product specialist. He has been instrumental for management of all commercial toxicity screening as well operational management of the R&D laboratory. Remco holds a MSc degree from Leiden University in Biomedical Sciences.

Paula and Remco are valuable additions to the senior Toxys Management Team. Their proven experience and expertise in business development, sales, customer relations and lab management will further boost the growth of Toxyssaid Giel Hendriks, CEO of Toxys BV.

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AboutToxysToxys is a Dutch biotech companythat offers a broad spectrum of innovativein vitrotoxicology solutions. Toxys was founded in 2014 as a spin-off from the Leiden University Medical Center and has its state-of-the-art laboratory facilities located at the Leiden BioScience Park. We are experts in the field of genetic and developmental toxicology with a focus on Mode-of-Action. We are dedicated to bringing relevant information to our clients on potential human health hazards of novel and existing drugs, chemicals and other substances. Toxys is currently working with 7 of the Top10 global Pharma companies and several major chemical, cosmetics and food multinationals.

Toxys has developed the unique ToxTracker and ReproTracker assays. ToxTracker is a high-throughput stem cell-based reporter suite of assays that allows reliable identification of genotoxic carcinogens. ToxTracker provides mechanistic insight into undesired properties of chemicals. ReproTracker is currently under development and its commercial launch (planned for early 2020) will allowin vitrodevelopmental toxicity hazard identification. These assays can be particularly useful in Mode-of-Action and Adverse Outcome Pathway (AOP) approaches for chemical safety assessment.

We offer ToxTracker and ReproTracker as tailored service to our customers, often in combination with standard toxicology assays or under license and as kits for use in partners own laboratories. Toxys is highly valued for its scientific expertise, high quality results and responsiveness.


Giel HendriksCEO+31 71

Media Contact

Paula van RossumCBO+31 71

Keywords: Humans; Carcinogens; Chemical Safety; Cosmetics; Carcinogenesis; Industry; Food Industry; DNA Damage; Drug-Related Side Effects and Adverse Reactions; Mutagenicity Tests; Biotechnology

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Toxys Strengthens Leadership Team with Appointment of CBO and COO Positions - BioSpace

The Center for Breakthrough Medicines is Building the World’s Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)…

KING OF PRUSSIA, Pa., Jan. 22, 2020 /PRNewswire/--The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO is occupying over 40 percent of The Discovery Labs' 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.

The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA,viral vectors, cell banking, cell processing, and support testing capabilities all under one roof. The immense $1.1 billion facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of lifesaving and life-changing therapies from the bench to the patient's bedside.

The Company has initiated a substantial hiring effort targeting the best and brightest of the life sciences community including, experts in CGMP manufacturing. The Company expects to hire over 2,000 team members within the next 30 months.

The CDMO has retained Nucleus Careers, a cloud-based specialty life sciences human capital recruiting and retention management expert, to buildout the entire team. Nucleus has proprietary recruiting and retention software designed for large scale human capital buildouts of high growth companies.

In addition to developing the world's largest single-point cell and gene therapy manufacturing facility, The Discovery Labs is establishing THE COLONY which will provide custom built discovery labs, breakthrough funding, sponsored research agreements, housing and relocation for the world's leading iconic experts in cell and gene therapy.

THE COLONY will seek to work hand in hand with scientists from both academic and pharmaceutical institutions to unlock and expedite groundbreaking therapies.

Marco A. Chacn, Ph.D., Founder of Paragon Bioservices and Chairman of The Discovery Labs states, "musicians, artists, members of religious communities and great thinkers throughout time have formed colonies where freedom of thought and expression combined with unlimited dreams and potential have resulted in the world's greatest accomplishments." Dr. Chacn went on to say, "the goal of THE COLONY is to unshackle the potential of the world's greatest scientific minds."

The ability for the industry's greatest scientists to cohabitate, collaborate, cooperate, and communicate via technology and in person will create an exponential therapeutic "X FACTOR." THE COLONY seeks to unlock institutional barriers prohibiting the world's greatest scientists from moving at a pace necessary in today's ever-changing therapeutic revolution. THE COLONY will partner with the institutions where the scientists currently work by providing equity, license fees, and revenue sharing.

"The Center for Breakthrough Medicines will be serving companies from the earliest stages through commercialization. Its exceptional scale and offering will quickly relieve the production bottleneck for advanced therapies by reducing the time, complexity, and cost of commercializing vitally needed gene and cell therapies," noted Audrey Greenberg, Board Member and Executive Managing Director for The Discovery Labs.

The addition of this end-to-end manufacturing capability is expected to significantly enhance the offerings of The Discovery Labs in an area that has become one of the largest life sciences hubs in the world. Renovations are underway to construct a total of 86 plasmid, viral vector production, universal cell processing, CGMP testing, process development and cell banking suites. The viral vector and cell processing suites will be fully compliant with both U.S. Food and Drug Administration and European Medicines Agency standards. All suites will offer the flexibility to meet client-specific workflows and will be able to adapt quickly to meet demand. The Company is in the process of reserving capacity now for late 2020.

"Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive," said Alex Karnal, Partner and Managing Director of Deerfield Management and a Board Member of the Discovery Labs. "With its visionary business model, it is hoped that The Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them."

The Discovery Labs provides a central campus where the world's greatest scientists can collaborate on new therapeutic discoveries to eradicate diseases affecting small and large segments of the global population. The Center for Breakthrough Medicines will work with these leaders, life sciences companies, large pharmaceutical companies, and academic and government institutions.

This new manufacturing capability is a transformational addition to The Discovery Labs market offering and dovetails with The Discovery Labs biotech incubator, Unite IQ. Unite IQ offers immediate space to emerging life sciences companies and scientists giving them the ability to grow from startup to enterprise company on one campus. The incubator and accelerator space at Unite IQ provides a comprehensive home for startups with every resource needed to initiate business operations. Unite IQ tenants are expected to utilize the discovery, development, testing, and manufacturing capabilities of the Center for Breakthrough Medicines with seamless forward integration of processes and analytics, and seamless tech transfer from research lab to large scale production

The Emerging Field of Cell and Gene Therapy in Pennsylvania

The demand for clinical and commercial manufacturing capacity is acute and expected to remain that way. The current shortfall in manufacturing for cell and gene therapies is severely underserved with few approved products. There are currently approximately 1,100 advanced therapies in the pipeline pending FDA approval. This will greatly increase highly skilled manufacturing demand. Dr. Peter Marks, Director of the FDA Center for Biologics Evaluation and Research, states, "what keeps me up at night is will we be able to manufacture these on a scale that will allow us to bring the benefit of these therapies to patients?"He further added that "if we can help see cost of goods and ability to manufacture reproducibly improve, I think that'll be a big thing."All of this adds up to a supply constrained market that The Center for Breakthrough Medicines aims to help address.

With the potential to treat and even cure disabling, and deadly diseases, gene and cell therapies are ushering in a new era of medicine. These therapies may eventually be able to cure genetic conditions, such as cystic fibrosis, hemophilia A, and a range of cancers. The Philadelphia area has become the epicenter for the flourishing field of gene and cell therapy. Research from CBRE currently ranks the market among the top biotech clusters for medical research and health services. The cluster has become known worldwide as "Cellicon Valley"for its leadership in research and development of this rapidly evolving field. The Discovery Lab's suburban Philadelphia location offers a talent rich environment due to the area's preponderance of large pharmaceutical companies and the Philadelphia region's position boasting the top 10 universities and primary school systems in nation.

Over the past three years, multiple Philadelphia companies have received approvals for major breakthroughs in cell and gene therapy. In 2017, the U.S. FDA approved the first-ever CAR-T cell therapy, Novartis's Kymriah, which originated at the University of Pennsylvania. Shortly thereafter, the FDA gave landmark approval for the first-ever gene therapy to treat a genetic blindness condition to Spark Therapeutics, a start-up founded by researchers at Children's Hospital of Philadelphia. These discoveries and others in the pipeline are attracting billions of dollars of venture capital. The Greater Philadelphia Region set a recent record in venture capital financing.

The Discovery Labs Center for Breakthrough Medicines joins more than 25 healthcare, life sciences and tech-enabled companies that already call The Discovery Labs King of Prussia home.

Brian O'Neill, Founder of The Discovery Labs Center for Breakthrough Medicines, and Tony Khoury, Board Member of The Discovery Labs and Engineer at Project Pharma, will be speaking at the 2020 PhacilitateWorld Stem Cell Summit discussing The Future of Gene Therapy Manufacturing at 4 p.m. today at the Hyatt Regency in Miami, Florida.

Contact Audrey Greenberg at for more information about development services, manufacturing capacity, incubator space or leasing information at the property.

About The Discovery LabsPart of MLP Ventures, The Discovery Labs is a global provider of world-class cGMP manufacturing, turnkey laboratory solutions, critical materials and office space that support therapeutic products and services to the biotechnology and pharmaceutical industry so that groundbreaking medicines get to the patients that need them. The location in eastern King of Prussia is a prototype for a global rollout of The Discovery Labs, providing Big Pharma, emerging life sciences, consumer and technology companies flexible, end-to-end technical real estate and business infrastructure for the customer's entire lifecycle from discovery to delivery, including manufacturing capacity. It is the first fully integrated environment that merges technology and life sciences under one roof to drive innovation.

About Deerfield Management

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Media Contact:Tony DeFazio, DeFazio Communications(o) 484-534-3306 (c)

SOURCE The Discovery Labs

The Discovery Labs

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The Center for Breakthrough Medicines is Building the World's Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)...

Share the Love on Valentine’s Day! Join the Cure 4 The Kids Foundation Blood Drive and Registry Event – Nevada Business Magazine

LAS VEGAS Regardless of your relationship status on Valentines Day, you can have an incredible impact on someones life in our communityand possibly save a life by donating blood or by becoming part of the bone marrow registry.

Cure 4 The Kids Foundation is well aware of the national shortage of blood and platelets, and how vital a reliable blood supply is to the on-going health of our community. Thats why Cure 4 The Kids Foundation is organizing the Valentines Day Blood Drive and Bone Marrow Registry Event on Friday, February 14, 2020.

The public is strongly encouraged to take part in either effort between 9:00 a.m. and 4:00 p.m. at One Breakthrough Way, Las Vegas, Nevada 89135.

Blood donors can ensure an easy and relatively quick donation process by making an appointment prior to Valentines Day by going towww.bloodhero.comand entering the code: cure4kids under sponsor. You can also schedule an appointment by calling Vitalant at 877-25-VITAL. The Vitalant bloodmobile will be parked in front of the One Breakthrough Way building.

Blood Donation Reminders:

Blood donors must be at least 16 years old (16 and 17-year olds must bring a signed permission form from a parent or guardian)Weigh at least 110 poundsBe in good general healthDrink plenty of water 24 to 48 hours prior to donationBring ID

Cure 4 The Kids Foundation is also partnering with the Be The Match Registry. The registry offers hope to those affected by cancer and other conditions by matching potential blood stem cell donors with those who are in need of a transplant.

The first step in joining the registry is to undergo a mouth swab by one of our volunteers. Those who are a possible match will be contacted at a later time. This service is free and available between 9:00 a.m. and 4:00 p.m. on February 14, 2020, in the lobby of the One Breakthrough Way building.

Bone Marrow Registry Reminders:

Donors between 18 and 44 years of age are especially neededYou cannot become a donor if you had have certain diseasesLearn more

About Cure 4 The Kids Foundation

Founded in Las Vegas in 2007, Cure 4 The Kids Foundation exists to provide high-quality, research-focused medical treatment to children battling cancer and many other life-threatening conditions. Cure 4 The Kids Foundation operates the only outpatient childhood cancer treatment center in the State of Nevada and is proudly accredited by The Joint Commission. This stringent medical accreditation and the required unannounced inspections ensure that patients are getting the best care possible. From the beginning, the mission of Cure 4 The Kids Foundation has been to bring increased community access to these specialized treatments that are leading the way to improved patient outcomes. Cure 4 The Kids Foundations Charity Care Program provides high-quality treatment to those who are uninsured, underinsured and those unable to pay for treatment. No patient is ever turned away for financial reasons. We are a 501(c)(3) nonprofit

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Share the Love on Valentine's Day! Join the Cure 4 The Kids Foundation Blood Drive and Registry Event - Nevada Business Magazine

Botox, medical drips and more offered at NVY Center –

STATN ISLAND, N.Y. -- As a Manhattan-based attorney, Chris Cardillo has overseen his share of medical management over the years.

Paperwork involved with being a doctor is so overwhelming these days, he said, noting that many physicians turn to attorneys to help manage their practices.

So with a wide knowledge of the medical industry, he sought to open a business that would house different physicians, as well as spa services, in a medical spa setting under one roof.

The result is NVY Center, a medical management company that opened in Richmond Valley more than one year ago.

As a Manhattan-based attorney, Chris Cardillo has completed his share of medical management over the years. Paperwork involved with being a doctor is so overwhelming these days, he said, noting that many physicians turn to attorneys to help manage their practices. (Staten Island Advance/Tracey Porpora)

I became very interested in the concept because I think its the wave of the future, said Cardillo, who has several partners in the business. Almost every medical office has a medical manager. ...When you go to a hospital, like New York University Medical Center, the hospital does all the management for doctors with affiliated practices. What hospitals do for their doctors, we do on a private level.


NVY Center rents a clean, white expansive space to various medical professionals, and aestheticians, who provide spa services.

We are hoping to be a national brand. What we do is solicit doctors to lease space for us and we manage them. That means when a doctor comes in here the only thing the doctor has to worry about is taking care of the patient, said Cardillo. We take care of the billing. They dont have to do any of the paperwork. Everything they need to do their job is here for them already.

Under the NVY Center roof are an array of spa services -- from stem cell therapy and IV drips to facials and aesthetic injections. (Staten Island Advance/Tracey Porpora)


Under the NVY Center roof are also an array of medical spa services, from stem cell therapy and IV drips, to facials and aesthetic injections.

Included in the facilitys design are individual medical offices, rooms where different procedures, such as facials, can take place, and an IV drip chair area. And the facility always has a medical professional on the premises, said Cardillo.

We do everything from anti-hangover to anti-flu drip. ... Soon, well be able to come to your house and do an IV drip, explained Cardillo. We want this to be a one-stop-shop for modern services."

He noted the IV drips often include vitamins and are used for various purposes, including by those people who desire to lose weight.

We have fast-drips that are 15 minutes long, where we can fill you with fluids if youre not feeling good, said Cardillo. Depending on what were trying to treat, we will mix vitamins and other medicines into the IV drip. But the fluid helps no matter what.

He said the company also hosts IV drip and Botox parties.

Other services include Trusculpting," a fat reduction procedure to lose weight, and laser hair removal, said Cardillo.


Address: 236 Richmond Valley Rd.



New Businesses in Focus is a weekly column that relates the stories of new Staten Island businesses owners.

If you have a new business on Staten Island, e-mail



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Walter named Edward P. Evans Endowed Professor – Washington University School of Medicine in St. Louis

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Oncologist honored for research, patient care in myelodysplastic syndromes

Medical oncologist Matthew Walter, MD, presents his research after being installed as the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes at Washington University School of Medicine in St. Louis.

Matthew J. Walter, MD, has been named the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes at Washington University School of Medicine in St. Louis. These syndromes make up a group of rare blood disorders that prevents the body from making sufficient healthy blood cells.

Walter, an international leader in the study and treatment of myelodysplastic syndromes (MDS), is a professor of medicine and director of the Edward P. Evans Center for Myelodysplastic Syndromes at the School of Medicine. The endowed professorship and the center are supported by funding from the Edward P. Evans Foundation, established in 1984 by Edward Parker Evans, a businessman and philanthropist whose foundation supports MDS research nationwide. He died in 2010 from a blood cancer related to MDS.

Walter treats patients with MDS and related blood disorders at Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine. His new professorship was announced by Chancellor Andrew D. Martin and David H. Perlmutter, MD, executive vice chancellor for medical affairs, the George and Carol Bauer Dean of the School of Medicine, and the Spencer T. and Ann W. Olin Distinguished Professor.

Matthew Walter has devoted his career to the study and treatment of myelodysplastic syndromes, and it is a great pleasure to be able to honor his work and commitment to his patients in this way, Martin said. There is no one better suited to lead this center. This endowed professorship will help support his work to understand the genetic and environmental underpinnings of this disease so that researchers can understand how it develops and work toward developing improved precision therapies to prevent or treat MDS.

Some 40,000 Americans are diagnosed with MDS each year. The symptoms can be vague, including shortness of breath and fatigue, which makes it difficult to diagnose. Low blood cell counts are among the first signs of the disease, and patients are diagnosed based on the appearance of abnormal blood cells. MDS can be fatal, but some patients with low-risk MDS live for many years with the disease and never know they have it. About one-third of patients with MDS develop an aggressive form of the disease that progresses to acute myeloid leukemia (AML), which is a fast-growing blood cancer. Without a successful stem cell transplant, AML is fatal.

Walter is interested in studying patients at risk of developing MDS but who dont yet have the disease. Such patients have mutations in a few important genes linked to MDS but dont yet have symptoms. This condition is called age-related clonal hematopoiesis. Even though relatively few of these patients go on to develop MDS, studying this group of patients could help doctors understand the differences between those who develop MDS and those who dont.

The work that Matthew Walter is leading at the new Edward P. Evans MDS Center, including his work with patients, is the epitome of precision medicine, Perlmutter said. This research will help illuminate what leads patients to progress to MDS or more aggressive blood disorders. His vision includes developing ways to prevent this progression, as well as novel therapies, including immunotherapy strategies. It is a great pleasure to recognize the innovative work Matthew Walter is doing to better care for these patients.

Said Victoria J. Fraser, MD, the Adolphus Busch Professor of Medicine and head of the Department of Medicine: Matthew Walter is a leader in the field in harnessing his labs research discoveries for the benefit of his patients. This professorship will help him continue his important research into the origins of this disease and potential ways to prevent and treat it.

Walter earned his bachelors degree in 1990 from American University in Washington and his medical degree in 1995 from Saint Louis University School of Medicine. He was a research scholar at the Howard Hughes Medical Institute and later completed his internship and residency at Johns Hopkins Hospital. He came to Washington University as a hematology-oncology fellow and joined the faculty in 2004.

Matthew Walter, MD, (center) shakes hands with Washington University Chancellor Andrew D. Martin as Victoria J. Fraser, MD, head of the Department of Medicine, applauds Walters installation as the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes.

He has been honored with numerous awards, including the Leukemia and Lymphoma Society Scholar Award, the MDS Foundation Young Investigator Award, and the American Society of Hematology Scholar Award. He is an elected member of the American Society for Clinical Investigation, and he serves on the National MDS Steering Committee of the National Institutes of Health (NIH), and on the American Society of Hematology Task Force on Precision Medicine. He is the 2019 chair of the American Society of Hematology Scientific Committee on Myeloid Neoplasia.

Edward Parker Evans ran Spring Hill Farm, the largest thoroughbred breeding farm in Virginia. Since his death, the Edward P. Evans Foundation has supported cutting-edge translational research on MDS at leading institutions across the country, including Washington University, which is noted for its innovative research and leadership in developing new therapies for patients with cancerous disorders of the blood.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Walter named Edward P. Evans Endowed Professor - Washington University School of Medicine in St. Louis

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 : 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,

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:

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:


Joseph DawsonRegenerative Medicine

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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


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

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

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