Category Archives: Genetic Medicine

First-ever large-scale genetic study examining Hawaiian Polynesians and health risk is led by the Keck School of Medicine of USC

(Photo/iStock)

By Wayne Lewis

With advances in analyzing human DNA, some well-studied populations have benefited from insights about how their health is affected by their genetics. Others, however, have been left behind. Among them are people of Polynesian descent from Hawaii.

Although population studies of Native Hawaiians have shown a substantial increase in risk for obesity, type2 diabetes, heart disease and certain cancers compared to their neighbors of European and Asian descent, there has been little to no insight into how genetics contribute on top of environmental factors to influence these disparities.

In an attempt to begin rectifying that gap, a USC-led research team has conducted the first study to systematically investigate the genomes of Native Hawaiians and test the components for health risks associated with genetic ancestry. The findings, which appeared in the journal PLoS Genetics, show that, for example, Polynesian ancestry in Native Hawaiians is linked to increased risk of diabetes, heart failure and higher body-mass index, a measure of body fat.

(Charleston Chiang. USC Photo/Ricardo Carrasco III)

Native Hawaiians really have been understudied from a genetic perspective, said corresponding author Charleston Chiang, PhD, an assistant professor of preventive medicine at the Keck School of Medicine of USC and of quantitative and computational biology at USC Dornsife College. Health disparities are a major research emphasis at USC in general, and my team focuses on looking at the genetic component of health risk within geographically diverse populations.

Characterizing Hawaiian Polynesian genetics to understand health risk

Chiang and his colleagues correlated health data (from questionnaire, laboratory measurements, and hospital Medicare claims) and the genetics of 3,940 people who identify as Native Hawaiian from the Multiethnic Cohort Study, a joint project of USC and the University of Hawaii. The research team found that for each 10% increase in estimated Polynesian ancestry, there is on average an 11% increase in risk of heart failure, an 8.6% increase in risk of type2 diabetes and a 0.35 unit increase in body-mass index.

Further studies may be able to identify genetic variants and underlying biological factors specific to Polynesian populations, knowledge that could help reduce these health risks. Chiang also hopes to test a hypothesis outlining a combination of nature and nurture.

For example, its possible that Native Hawaiians had adapted to a traditional diet, and the introduction of the Western diet has led to all kinds of health problems, he said. Thats actually an interaction between their genetics and their environment.

There was a unique challenge for the studys authors to overcome: Researchers focusing on the genetics of people with roots in Europe, Africa and Asia are able to call upon publicly available genomic references for those populations. No such resource exists for Polynesian ancestry. Native Hawaiians are characterized by a mixture of Polynesian, Asian, European and African ancestry. Using the existing references from other populations to run two analyses, the scientists searched for known origins as reflected both across each participants entire genome and location by location along their chromosomes. The research team essentially constructed a genomic model for Polynesian ancestry among Native Hawaiians by identifying a subsample of roughly 150 participants with the least amount of external heritance.

Genomics cant define ethnicity, and biology is not destiny

As should be expected with research charting new territory in biomedical science, the studys authors urge that their findings be interpreted with care and clarity on a few fronts.

Chiang pointed out that race and ethnicity are socially constructed concepts, and distinct from the issues explored in this study that is how certain genes shared among a population contribute to specific health metrics and outcomes. Ethnicity instead is, and should be, defined by genealogical records or how a person self-identifies.

Geneticists should not try to quantize a persons ancestry and use that to define whether that person belongs to a particular ethnic group, he said. While we needed to quantify the proportion of Polynesian ancestry in order to perform our research, we do not want to give the impression that this is a way for people to define their membership in the community based on some arbitrary threshold.

Additionally, Chiang emphasized that the model for Polynesian heritance among Native Hawaiians does not necessarily apply perfectly to populations in other islands such as Samoa.

Perhaps most important, the links between genetics and health revealed in this study should not be construed to mean that being part of any particular population automatically relegates a person to poor health in and of itself.

Genetics is a window into understanding the biology behind these diseases, Chiang said. Genetics does not determine everything, and it doesnt necessarily even amount to the majority of the disparity in risk. I want people to know there are modifiable components to your lifestyle, such as a healthy diet and regular hula dancing, that will absolutely help.

About the studies

The studys co-first authors are Hanxiao Sun, a former masters student in Chiangs research group, and Meng Lin, a former postdoctoral researcher in the group. Other authors are Tsz Fung Chan, Bryan Dinh and Christopher Haiman of USC; Emily Russell and Ryan Minster of the University of Pittsburgh; Take Naseri of the Government of Samoas Ministry of Health; Muagututia Sefuiva Reupena of Lutia i Puava ae Mapu i Fagalele, a nongovernmental organization based in Samoa; Annette Lum-Jones, Lynne Wilkens and Loc Le Marchand of the University of Hawaii; the Samoan Obesity, Lifestyle, and Genetic Adaptations Study Group; and Iona Cheng of the University of California, San Francisco.

The study was supported by the National Cancer Institute (U01CA164973, P01CA168530) and the National Human Genome Research Institute (U01HG007397).

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Genetic ancestry linked to diabetes, heart failure and obesity among Native Hawaiians | Keck School of Medicine of USC - USC News

Data Bridge Market Research recently added Global Precision Medicine Market by manufacturers, regions, type and application, forecast to 2028 in his database. This research report focus on complete assessment of market and contains future trend, growth factors, attentive opinions, facts, historical data, statistically supported and industry-validated market data. Environmental concerns & regulatory guidelines regarding release of effluents through different industries. Global Precision Medicine Market comprehensive coverage of underlying economic and technological factors under key trend analysis.

Global Precision Medicine Market to grow with a substantial CAGR in the forecast period of 2019-2026. Growing prevalence of cancer worldwide and accelerating demand of novel therapies to prevent of cancer related disorders are the key factors for lucrative growth of market

Global Precision Medicine Market By Application (Diagnostics, Therapeutics and Others), Technologies (Pharmacogenomics, Point-of-Care Testing, Stem Cell Therapy, Pharmacoproteomics and Others), Indication (Oncology, Central Nervous System (CNS) Disorders, Immunology Disorders, Respiratory Disorders, Others), Drugs (Alectinib, Osimertinib, Mepolizumab,Aripiprazole lauroxil and Others), Route of Administration (Oral,Injectable), End- Users (Hospitals, Homecare, Specialty Clinics, Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2026

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Competitive Analysis:

The precision medicine market is highly fragmented and is based on new product launches and clinical results of products. Hence the major players have used various strategies such as new product launches, clinical trials, market initiatives, high expense on research and development, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of mass spectrometry market for global, Europe, North America, Asia Pacific and South America.

Market Definition:

Precision medicines is also known as personalized medicines is an innovative approach to the patient care for disease treatment, diagnosis and prevention base on the persons individual genes. It allows doctors or physicians to select treatment option based on the patients genetic understanding of their disease.

According to the data published in PerMedCoalition, it was estimated that the USFDA has approved 25 novels personalized medicines in the year of 2018. These growing approvals annually by the regulatory authorities and rise in oncology and CNS disorders worldwide are the key factors for market growth.

Talk to The Author of Report @ http://databridgemarketresearch.com/speak-to-analyst/?dbmr=global-precision-medicine-market

Market Drivers

Market Restraints

Key Developments in the Market:

Competitive Analysis:

Global precision medicine market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global precision medicine market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key Market Players:

Few of the major competitors currently working in the global precision medicine market are Neon Therapeutics, Moderna, Inc, Merck & Co., Inc, Bayer AG, PERSONALIS INC, GENOCEA BIOSCIENCES, INC., F. Hoffmann-La Roche Ltd, CureVac AG, CELLDEX THERAPEUTICS, BIONTECH SE, Advaxis, Inc, GlaxoSmithKline plc, Bioven International Sdn Bhd, Agenus Inc., Immatics Biotechnologies GmbH, Immunovative Therapies, Bristol-Myers Squibb Company, Gritstone Oncology, NantKwest, Inc among others.

SHORT Table of Contents

1.1. OVERVIEW OF THE GLOBAL PRECISION MEDICINE MARKET

1.2. CURRENCY AND PRICING

1.3. LIMITATION

1.4. MARKETS COVERED

2 MARKET SEGMENTATION

2.1. KEY TAKEAWAYS

2.2. ARRIVING AT THE GLOBAL PRECISION MEDICINE MARKET SIZE

2.3. GLOBAL PRECISION MEDICINE MARKET: RESEARCH SNAPSHOT

2.4. ASSUMPTIONS

3 MARKET OVERVIEW

3.1. DRIVERS

3.2. RESTRAINTS

3.3. OPPORTUNITIES

3.4. CHALLENGES

4 EXECUTIVE SUMMARY

5 PREMIUM INSIGHTS

6 GLOBAL PRECISION MEDICINE MARKET, BY TECHNOLOGY

6.1. OVERVIEW

6.2. BIG DATA ANALYTICS

6.3. BIOINFORMATICS

6.4. GENE SEQUENCING

6.5. DRUG DISCOVERY

6.6. COMPANION DIAGNOSTICS

6.7. OTHERS

7 GLOBAL PRECISION MEDICINE MARKET, BY APPLICATION

7.1. OVERVIEW

7.2. ONCOLGY

7.3. HEMATOLOGY

7.4. INFECTIOUS DISEASES

7.5. CARDIOLOGY

8 GLOBAL PRECISION MEDICINE MARKET, BY end-user

8.1. OVERVIEW

8.2. PHARMACEUTICALS

8.3. BIOTECHNOLOGY

9 GLOBAL PRECISION MEDICINE MARKET: COMPETITIVE LANDSCAPE

9.1. GLOBAL PRECISION MEDICINE MARKET: COMPANY SHARE ANALYSIS

9.2. MERGERS & ACQUISITIONS

9.3. NEW PRODUCT DEVELOPMENT & APPROVALS

9.4. EXPANSIONS

10 GLOBAL PRECISION MEDICINE MARKET, COMPANY PROFILES

10.1 Orion Health

10.2 ARIEL Precision Medicine, INC.

10.3 Metabolon, Inc.

10.4 Pfizer Inc.

10.5 Biocrates Life Sciences

10.6 Medtronic

10.7 NovartisAG

10.8 Quest Diagnostics Incorporated

10.9 F. Hoffmann-La Roche Ltd

10.10 Teva Pharmaceutical Industries Ltd

11 APPENDIX

12 ABOUT DATA BRIDGE MARKET RESEARCH

Get a Detail TOC @ http://databridgemarketresearch.com/toc/?dbmr=global-precision-medicine-market

Market Segmentation:

By technology:- big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics, and others.

By application:- oncology, hematology, infectious diseases, cardiology, neurology, endocrinology, pulmonary diseases, ophthalmology, metabolic diseases, pharmagenomics, and others.

On the basis of end-users:- pharmaceuticals, biotechnology, diagnostic companies, laboratories, and healthcare it specialist.

On the basis of geography:- North America & South America, Europe, Asia-Pacific, and Middle East & Africa. U.S., Canada, Germany, France, U.K., Netherlands, Switzerland, Turkey, Russia, China, India, South Korea, Japan, Australia, Singapore, Saudi Arabia, South Africa, and Brazil among others.

In 2017, North America is expected to dominate the market.

About Data Bridge Market Research:

Data Bridge Market Researchset forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Contact:

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475

Email:Corporatesales@databridgemarketresearch.com

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Global Precision Medicine Market Study including Growth Factors, New Top Players, Competitive Analysis by regions from 2021 to 2027 KSU | The...

Already containing health records, surveys, and measurements from more than 200,000 people from all walks of life, the All of Us Research Programs Researcher Workbench is a treasure trove waiting to be mined.

An immigrant from Peru, Dr. Ral Montaez-Valverde was surprised to encounter perplexing research showing that Latinos in the United States are at lower risk of heart disease than their white counterpartsdespite a generally lower socioeconomic status and access to health care.

It prompted me to think about why Hispanics could have better outcomes, given all the challenges, said Montaez-Valverde. I was very curious about this question.

So curious that, despite his intense schedule as a second-year internal medicine resident at Jackson Memorial Hospital, Montaez-Valverde jumped at the chance to use the All of Us Research Programs Researcher Workbench to investigate the long-debated validity of whats known as the Latino Epidemiological Paradox. Now open to investigators from all disciplines and career stages across the University of Miami, the Researcher Workbench contains the de-identified electronic health records of more than 200,000 people across the United Statesincluding 10,000 from Greater Miamiwho have enrolled in the most inclusive study ever undertaken by the National Institutes of Health.

Launched in 2018, the 10-year, $2 billion-plus All of Us Research Program (AoURP) is building one of the worlds largest and most diverse health datasets by collecting lifestyle, health, and genetic information from 1 million people of all races, ethnicities, backgrounds, and gender identities living in the U.S. The ultimate goal is to advance personalized medicine by helping researchers and physicians like Montaez-Valverde understand why different people are more vulnerable to different diseases and conditions and tailor prevention, treatment, and care approaches specifically for them.

But neither the AoURP nor the Universitys Miller School of Medicine, which is leading the AoURPs effort to recruit some 80,000 of the 1 million participants from Florida and Georgia, are waiting for the enrollment process to conclude before making the data available to researchers. The University has signed a data-use agreement with the NIH allowing any faculty members, research assistants, students, residents, or other trainees with an NIH eRA Commons account to begin mining the treasure trove, which will grow in both value and volume as more people discover its riches.

The Researcher Workbench is a major milestone in fulfilling the promise of the All of Us program, but for now it may be one of the best kept secrets in biomedical research, said Stephan Zchner, professor and chair of the Dr. John T. Macdonald Foundation Department of Human Genetics and the lead principal investigator for the AoURPs Southeast Enrollment Center (SEEC), which also includes the University of Florida, Emory University, and Morehouse School of Medicine.

At the end of the day, continued Zchner, who also co-directs the John P. Hussman Institute for Human Genomics, we want to work with data to create new knowledge and insights into medicine, and the workbench is a major tool for that. Whats exciting is that it opens biomedical data access to many qualified investigators, including people in the social sciences, basic sciences, sports, even the arts. The possibilities are endless, and there will be a lot more of them as the data get richer and larger over time.

For the time being, the cloud-based research platform, which requires proficiency with the R or Python programming languages, does not include the genetic information that most interests researchers like Zchner. But as he noted, the AoURP is currently sequencing the genomes of the first 100,000 participants and plans to do the same for all 1 million participantsall of which eventually will make its way into the database and the hands of those who shared their DNA.

Yet even in its infancy, the workbench already contains four types of data. In addition to the electronic health records of roughly 203,000 people, the database includes survey data from more than 315,000 people who answered questions about their medical history, lifestyle, access to care and, more recently, experiences with COVID-19, including the pandemics impact on their mental and financial health. It also contains physical measurementsincluding blood pressure, heart rate, and body mass indexfrom more than 260,000 people, and data collected by the Fitbit wearable devices of more than 8,000 people.

But for Dr. Olveen Carrasquillo, an expert in health disparities who serves as the SEECs participant engagement lead, the most exciting aspect of the AoURP is its success in recruiting minorities who have long been overlooked by medical research. According to the AoURP, about half of the participants whose data is in the Researcher Workbench are people of color.

One of my biggest concerns was that this project would be like everything else, and minorities would be left out, but weve seen really robust and good efforts at assuring they are included, said Carrasquillo, professor of public health sciences, chief of the Division of General Internal Medicine, and a co-principal investigator for the AoURP. And by minorities, I mean that in the full sense, not just race and ethnicity, but by income, education, gender identity. So, with lots of data on minorities, this humongous data source will be a very powerful tool for people who want to reduce health disparities and improve health equity.

To get an idea of its power, Carrasquillo enlisted Montaez-Valverde, the resident he happened to meet on the Metrorail after leaving Jackson Memorial one night, to become one of the Universitys first workbench users. At the time, Montaez-Valverde, who plans to specialize in cardiology, wasnt familiar with the AoURP, or the Latino paradox. But he shared Carrasquillos skepticism that, given their higher rates of diabetes and uncontrolled blood pressure, Latinos would have better cardiovascular health than their white counterparts, as other studies have shown.

Montaez-Valverde was amazed to learn he would have the electronic records of more than 200,000 people to analyze, a powerful tool that helped him conclude that Latinos in the AoURP dataset actually have a higher, not lower, or similar prevalence of cardiovascular disease, than whites. He was just invited to present those findings at theAmerican College of Cardiologys 70th annual scientific session in May.

What we saw clearly does not support the Latino paradox, Carrasquillo said. But thats only in this database, so for now were just throwing more fire on a debate thats been raging for 25 years.

But not subject to much debate will be the growing value of using the AoURP Research Workbench for research and discovery. As the data grow, the research is going to be a lot more powerful, meaningful, and useful, he said.

To learn more about the workbench, visit the All of Us Research Hub or listen to an overview by Drs. Zchner and Carrasquillo presented by the Clinical and Translational Science Institute. For more information about or to enroll in the study, visit the All of Us Research Program.

Original post:
National health database opens to University researchers - University of Miami

UC San Francisco scientists have formed a research alliance with pharmaceutical firm Eli Lilly and Company aimed at better understanding autoimmune diseases and fostering the development of new therapies. Based on the innovative Immunoprofiler model launched at UCSF in 2015, the new initiative, called AutoImmunoprofiler, inherits the most successful aspects of its predecessor and adapts them to the study of autoimmunity.

Autoimmune diseases such as lupus, inflammatory bowel disease, and type 1 diabetes occur when the immune system attacks the bodys own tissues. While there are some therapies to treat symptoms of these conditions, the underlying causes are largely unknown.

Matthew Max Krummel, PhD

What we have learned through the first Immunoprofiler consortiums work on cancer is that the immune system likely has a limited number of tricks, said AutoImmunoprofiler co-leader Matthew Max Krummel, PhD, professor of pathology. If we can find all the key circuits that control the immune system, we can understand how it will behave in response to different inflammatory diseases.

With foundational funding from Lilly, AutoImmunoprofiler launches with the goal of bringing up to four additional pharmaceutical companies into the consortium. Like Lilly, all these partner investors will have access to research data generated from biological samples through standardized procedures developed in the high-tech CoLabs facilities at UCSF. David Erle, MD, professor of medicine and director of the UCSF Colabs, said the hope is that this communal research environment will result in new breakthroughs in understanding autoimmune disease.

In addition to Krummel and Erle, UCSF scientific leaders of the AutoImmunoprofiler project include Jeroen Roose, PhD, professor of anatomy; Lindsey Criswell, MD, MPH, vice chancellor for research and Jean S. Engleman Distinguished Professor of Rheumatology; Jimmie Ye, PhD, associate professor of medicine; and Vincent Chan, PhD, assistant professor of microbiology and immunology.

David Erle, MD

The clinical programs for the first phase of the Autoimmunoprofiler consortium are led by Mark Anderson, MD, PhD, professor in the UCSF Diabetes Center (Type 1 diabetes); Maria DallEra, MD, professor of medicine (systemic lupus erythematosus); Caroline Shiboski, DDS, MPH, PhD, the Leland A. and Gladys K. Barber Distinguished Professor in Dentistry and chair of the Department of Orofacial Sciences in the UCSF School of Dentistry (Sjgrens syndrome); Michael Kattah, MD, PhD, assistant professor of medicine (ulcerative colitis and Crohns disease); Andrew Gross, MD, professor of medicine; and Sarah French, MD, professor of medicine (scleroderma).

UCSF has a collaborative group of investigators who are innovating new genomics and single-cell technologies, as well as clinicians who have extensive expertise in specific autoimmune diseases, said Ye.This combination creates a unique opportunity to study autoimmune diseases at unprecedented precision and resolution.

The team will collect tissue and blood samples from patients with various forms of autoimmune disease and analyze these samples using techniques like single-cell RNA sequencing. With the help of the Organoid D2B unit, theyll also create a tissue bank of organoids 3D, tissue-like balls of cells grown in the lab from samples donated by patients with inflammatory bowel disease. One of the key goals of these analyses will be to identify what the team calls autoimmune archetypes.

Immune system archetypes, Krummel said, offer a way to classify the behavior of the immune system and how it reacts to disease. One of the key findings of the original Immunoprofiler initiative was that despite all the genetic diversity in the population, there are a limited number of ways the immune system reacts to cancer. The same is likely true for autoimmune disease, and AutoImmunoprofiler aims to identify similar archetypes for these diseases.

If we can find all the key circuits that control the immune system, we can understand how it will behave in response to different inflammatory diseases.

Matthew Max Krummel, PhD,AutoImmunoprofiler co-leader

Its kind of like asking how many different kinds of people there are it depends on how you look at it, said Krummel. In terms of the way the immune system responds to cancer we see about a dozen archetypes and we expect to find a similar number for autoimmune disease.

Identifying autoimmune archetypes will help the researchers understand the central operating mechanisms underlying autoimmune disease, potentially revealing hidden connections between seemingly unrelated illnesses. Understanding these connections may help speed the development of new treatments and identify existing drugs that may have the potential to treat several different autoimmune disorders.

The original Immunoprofiler initiative produced a similar breakthrough in understanding the importance of immune system cells known as dendritic cells. The data-sharing model of the consortium allowed researchers to discover that the behavior of dendritic cells largely determines the effectiveness of T cell therapies against cancer.

The discovery of the importance of dendritic cells in cancer treatments is an example of the research success of the Immunoprofiler program, but Krummel and Roose believe that the true legacy of that program is in the model itself what they call a pre-competitive space. This model provides several unique advantages for pharmaceutical company partners and academic scientists alike.

This unique publicprivate structure fosters a collaborative culture and allows us to tackle important biological questions. We know that pre-competitive consortia work when partners come together that are equally motivated to make a difference to patients, said Peter Kotsonis, PhD, executive director of the UCSF Office of Strategic Alliances (Innovation Ventures).

Pharmaceutical companies that join the consortium will enrich the program by funding more studies, which benefits UCSF by streamlining the funding process.

Our Immunology Team at Lilly is deeply honored to be the foundational partner for the AutoImmunoprofiler initiative. We look forward to collaborating with the world class scientists and clinicians at UCSF to better understand autoimmune disease at the cellular and molecular level, said Ajay Nirula, MD, PhD, vice president of immunology at Eli Lilly and Company.

This kind of collaboration is also better for patients because the data-sharing model encourages basic researchers to develop practices that facilitate rapid practical application it speeds the creation of new drugs and identifies old drugs that may be utilized in new ways.

Were setting ourselves up at the Parnassus Heights Campus to be the clinicaltranslational pipeline to basic research, said Roose. Were allowing industry and academia to work together and come up with solutions faster.

The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care.UCSF Health, which serves as UCSFs primary academic medical center, includes top-ranked specialty hospitals and other clinical programs, and has affiliations throughout the Bay Area.

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'AutoImmunoprofiler' Builds on Success in Cancer Research to Advance Treatments for Autoimmune Diseases - UCSF News Services

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced today that the company has placed its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) studies of LentiGlobin gene therapy for sickle cell disease (SCD) (bb1111) on a temporary suspension due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

In line with the clinical study protocols for HGB-206 and HGB-210, bluebird bio placed the studies on temporary suspension following a report received last week that a patient who was treated more than five years ago in Group A of HGB-206 was diagnosed with AML. The company is investigating the cause of this patients AML in order to determine if there is any relationship to the use of BB305 lentiviral vector in the manufacture of LentiGlobin gene therapy for SCD. In addition, a second SUSAR of myelodysplastic syndrome (MDS) in a patient from Group C of HGB-206 was reported last week to the company and is currently being investigated.

No cases of hematologic malignancy have been reported in any patient who has received treatment with betibeglogene autotemcel for transfusion-dependent -thalassemia (licensed as ZYNTEGLOTM in the European Union and the United Kingdom), however because it is also manufactured using the same BB305 lentiviral vector used in LentiGlobin gene therapy for SCD, the company has decided to temporarily suspend marketing of ZYNTEGLO while the AML case is assessed.

The safety of every patient who has participated in our studies or is treated with our gene therapies is the utmost priority for us, said Nick Leschly, chief bluebird. We are committed to fully assessing these cases in partnership with the healthcare providers supporting our clinical studies and appropriate regulatory agencies. Our thoughts are with these patients and their families during this time.

The independent safety review board monitoring the companys studies as well as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have been advised of these cases and bluebird bio will continue to work with regulatory agencies to complete its investigation.

Investor Conference Call Information

bluebird bio will hold a conference call to discuss this update on Tuesday, February 16 at 8:00 a.m. ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 880-6406.

To access the live webcast of bluebird bios presentation, please visit the Events & Presentations page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com. A replay of the webcast will be available on the bluebird bio website for 90 days following the event.

About HGB-206 and HGB-210

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD) that includes three treatment cohorts: Groups A, B and C. A refined manufacturing process designed to increase vector copy number (VCN) and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

HGB-210 is an ongoing Phase 3 single-arm open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD in patients between two years and 50 years of age with sickle cell disease.

About LentiGlobin for SCD (bb1111)

LentiGlobin gene therapy for sickle cell disease (bb1111) is an investigational treatment being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study, and the ongoing Phase 3 HGB-210 study.

The U.S. Food and Drug Administration granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT04628585 for LTF-307.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About ZYNTEGLO (betibeglogene autotemcel)

Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived adult Hb, at levels that may eliminate or significantly reduce the need for transfusions. In studies of beti-cel, transfusion independence (TI) is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

For details, please see the Summary of Product Characteristics (SmPC).

On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway.

The U.S. Food and Drug Administration granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the U.S. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study, LTF-303 for people who have participated in bluebird bio-sponsored clinical studies of ZYNTEGLO.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, betibeglogene autotemcel, beti-cel, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys timing and expectations regarding its investigation of the relationship of the AML and MDS events to the use of lentiviral vector BB305 in LentiGlobin gene therapy for SCD, and any myeloablation regimen used in connection with treatment. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements, many of which are beyond the Companys control. These risks and uncertainties include, but are not limited to: the risk that the Company may not be able to definitively determine whether the lentiviral vector BB305 used in LentiGlobin gene therapy for SCD and in betibeglogene autotemcel is related to the patients AML in a timely manner, or at all; the risk that the lentiviral vector BB305 has caused insertional oncogenic events, including AML; the risk that insertional oncogenic events associated with lentiviral vector or additional MDS events associated with myeloablation will be discovered or reported over time; the risk that regulatory authorities may impose a clinical hold, in addition to our temporary clinical hold on the HGB-206 and HGB-210 studies, or on additional programs; the risk that we may not be able to address regulatory authorities concerns quickly or at all; the risk that we may not resume patient treatment with ZYNTEGLO in the commercial context in a timely manner or at all; the risk that our lentiviral vector platform across our severe genetic disease programs may be implicated, affecting the development and potential approval of elivaldogene autotemcel; the risk that we may not be able to execute on our business plans, including our commercialization plans, meeting our expected or planned regulatory milestones, submissions, and timelines, research and clinical development plans, and in bringing our product candidates to market; and the risk that with the impact on the execution and timing of our business plans, we may not successfully execute our previously announced plans to spin off our oncology programs into an independent publicly-traded entity. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio Announces Temporary Suspension on Phase 1/2 and Phase 3 Studies of LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) - Business...

SALT LAKE CITY, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc.. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, announced today additional data further validating the prognostic power of its Prolaris test and its ability to help accurately predict which men with more aggressive prostate cancer will benefit from intensification of therapy and which patients may safely avoid such treatments. This second validation study was presented during an oral presentation at the American Society of Clinical Oncology Genitourinary Cancer Symposium (ASCO-GU) by Jonathan Tward M.D., Ph.D, associate professor in the Department of Radiation Oncology at the University of Utah.

According to estimates by the American Cancer Society, 248,530 new cases of prostate cancer are expected to be diagnosed this year in the U.S. While early screening tests have helped reduce the mortality rate, they can often result in overdiagnosis and overtreatment of a disease that is clinically insignificant. The Prolaris test can more accurately predict the aggressiveness of the cancer allowing for more precise treatment and avoidance of more intense therapies with a patients parallel morbidities.

There are many viable treatment paths for men with prostate cancer, said Dr. Tward. This new data helps distinguish the most appropriate personalized treatment path for each patient based on how their specific tumor is behaving. For some men, this means being able to avoid overtreating patients with therapies including hormone treatment that can momentously impact their quality of life, while still appropriately treating their prostate cancer.

The new data comes from a second study following previous data, recently published in Clinical Genitourinary Cancer in January 2021, that incorporated men treated surgically or with radiation therapy. This new study combined a Prolaris molecular risk score threshold with a clinical model for predicting a patients benefit from androgen deprivation therapy. Prolaris determined that about one of every two men with unfavorable intermediate-risk and one of every five men with high-risk prostate cancer are below the proposed threshold associated with aggressive disease and can therefore safely be treated with less intense therapy while maintaining the benefits of treatment. Additional key findings revealed that the Prolaris test was an accurate predictor of progression to metastatic disease.

Myriad Genetics was the first company to offer a test that directly measures the molecular biology of an individual patients prostate cancer, said Todd D. Cohen, M.D., vice president of Medical Affairs for Urology at Myriad Genetics. This study by Dr. Tward and his team is another strong validation of the prognostic power of the Prolaris test and our ongoing commitment to providing healthcare professionals with the tools needed to determine the most effective treatments and monitoring strategies for each patient.

In March 2020, the National Comprehensive Cancer Network updated its professional guidelines to include biomarker testing for unfavorable intermediate and high-risk patients with prostate cancer. With the updated guidelines, Prolaris was one of only two prognostic tests to be considered for those expanded indications. Approximately 60% of men with prostate cancer currently have insurance or Medicare access to Prolaris, and Myriad continues to work toward expanding access so that every man who is facing difficult treatment decisions will be able to utilize the full benefits of the test.

About ProlarisProlaris is a genetic test developed by Myriad Genetics that directly measures tumor cell growth. The Prolaris test paired with other clinical and pathologic variables provides the level of aggressiveness of a patients individual prostate cancer and assesses risk of death or the development of metastatic disease from prostate cancer. For more information visit: http://www.prolaris.com.

About Myriad GeneticsMyriad Genetics Inc., is a leading genetic testing and precision medicine company dedicated to transforming patient lives worldwide. Myriad discovers and commercializes genetic tests that determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across medical specialties where critical genetic insights can significantly improve patient care and lower healthcare costs. For more information, visit the Company's website:www.myriad.com.

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

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the validation study presented during at ASCO-GU by Jonathan Tward M.D., Ph.D; expanding access so that every man who is facing difficult treatment decisions will be able to utilize the full benefits of the Prolaris test; and the Companys strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2020, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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New Study from Leading University of Utah Radiation Oncologist Validates Ability of Myriad Genetics' Prolaris test to Guide Treatment for Prostate...