Category Archives: Genetic medicine

Gene therapy is a promising new method for treating non-small cell lung cancer (NSCLC). It allows doctors to target specific genes to prevent cancerous cells from growing and spreading.

NSCLC is a common form of cancer. It causes cancerous cells to form in the tissues of the lung. NSCLC is a serious condition. However, many people receive treatment and survive for years.

Treating NSCLC typically involves interacting with multiple specialists and receiving a combination of therapies. Specific treatment plans depend on factors that include the tumor size, type of NSCLC, and the extent of its spread to other organs.

Surgery, radiation or chemotherapy, and immunotherapy are examples of key techniques that doctors use to treat NSCLC.

Gene therapy is another promising treatment for NSCLC, which targets genes that contribute to the tumor.

There are two main approaches to using gene therapy to treat cancer:

This article focuses on the second approach to NSCLC gene therapy. Read on to learn more.

Getting genes into cells requires making vectors, which are vehicles that scientists engineer to deliver genetic materials. For example, viruses have a natural ability to deliver genetic material into cells and can act as vectors.

Scientists can deactivate parts of the virus that cause infectious diseases. They can then modify the virus to carry genetic material into cancerous cells.

One type of gene therapy for NSCLC targets tumor-suppressor genes, which are the most common gene mutation that contributes to the disease. Another approach involves restoring specific proteins to prevent disease progression.

Other possible applications include inserting genes that:

NSCLC gene therapy is a new form of treatment. However, early results are promising.

A 2017 review suggests that restoring a functional tumor-suppressing gene could slow the growth of cancer cells. Clinical trials have found that inserting tumor-suppressing genes into people who had not responded to other treatments reduced tumor size by up to 50%.

Another review in 2016 suggests that the treatment is more effective when combining NSCLC gene therapy with other therapies, such as chemotherapy or immunotherapy.

According to the American Cancer Society, doctors typically use gene therapy for advanced cancer cases.

NSCLC gene therapy is a new technique. However, it still has to meet rigorous Food and Drug Administration (FDA) standards for safety and effectiveness before a doctor can recommend it.

Gene therapies that the FDA approves are safe. However, they can have side effects, such as:

According to the FDA, gene therapies can transform medicine and provide options for people with illnesses that were previously without a cure. However, every treatment has limitations to its effectiveness.

Some limitations to gene therapy include:

Doctors will typically develop a treatment plan with people who have NSCLC depending on their health, age, and other relevant factors. Some common forms of NSCLC treatment include:

Doctors may combine these treatments to maximize their effectiveness. This will involve undergoing multiple treatments at once or back-to-back treatments, or both.

For example, doctors may use a therapy to treat cancer in one part of the body and another therapy to treat where it is spreading.

Doctors typically describe the outlook for people with cancer using the percentage of people alive at least 5 years after their diagnosis. This is the 5-year survival rate. They may further break down 5-year survival rates according to specific NSCLC diagnoses.

According to the American Cancer Society, the 5-year survival rate for people with NSCLC are:

NSCLC is a common form of lung cancer in the United States. Gene therapy for people with NSCLC is a promising new treatment that targets specific genes that contribute to disease progression. There is evidence that gene therapy can slow the growth of tumors in people with NSCLC.

Gene therapy is new, but has the potential to change the way doctors can treat cancer. Scientists and doctors must first overcome limitations, including finding reliable methods to deliver gene therapy.

Read more:
NSCLC gene therapy: Success rate, other options, and more - Medical News Today

The treatment landscape for advanced ovarian cancer has evolved from where it was ten years ago, when at that stage it was very much a standard approach of surgery, chemotherapy, and then watch and wait to one where patients receive individualized care based on the unique features of their cancer, said Warner Huh, MD, FACOG, FACS, chair of the Department of Obstetrics and Gynecology at the University of Alabama at Birmingham School of Medicine. I dont think we have ever seen so many options to help improve clinical outcomes in the modern management of ovarian cancer than we have with the introduction of personalized medicine. To say its been transformative would be an understatement.

Identifying the Right Patients for Personalized MedicineThe introduction of PARP inhibitors for the treatment of certain women with advanced ovarian cancer is one recent example of how personalized medicine is transforming the way the disease is treated. Research has demonstrated that targeted treatment with PARP inhibitor-based regimens shows greater clinical benefit in women with homologous recombination deficient tumors as HRD is associated with an increased sensitivity to PARP inhibition. This group of patients accounts for approximately 50 percent of the advanced ovarian cancer population.

HRD is the functional impairment in a key DNA damage repair pathway that hinders a cell from fixing damaged DNA. Identifying HRD may suggest that a patient is a candidate for an appropriate PARP inhibitor-based regimen, which further blocks remaining DNA repair pathways causing the cancer cells to die. Given the relationship with HRD-positivity and PARP inhibitor regimens, HRD genomic instability testing is an important component of advanced ovarian cancer management.

Advanced ovarian cancer is a tough disease. When I see a patient is HRD-positive, I make sure she understands the results. I use it as an opportunity to share what it may mean for her care, said Dr. Huh. This conversation can also serve as a way to discuss the full treatment path ahead and set expectations for whats to come during different phases of her treatment journey.

Certain mutations that signal the presence of HRD, such as BRCA1/2 mutations, and other markers of genomic instability are identified through comprehensive biomarker testing.

In todays treatment landscape where cancer therapy is becoming increasingly personalized and biomarker-based, every woman diagnosed with advanced ovarian cancer should receive HRD genomic instability testing. Knowing that roughly half of our patients are HRD-positive, this testing can identify important information about her tumor which may better inform her treatment plan than testing for a BRCA mutation alone which is present in approximately just 25% of women with the disease, said Dr. Huh.

This puts into perspective the importance of HRD genomic instability testing. Without this test, we are not providing optimal information for informed decision-making about treatment, which is even more crucial given recent advances in medicine have brought us further than before as we aim to offer our patients hope.

However, given the various testing options across tumor types and the overall ongoing evolution in tumor-specific biomarker and genetic testing, gaps in knowledge about HRD genomic instability are complicating the testing landscape and hindering oncologists from consistently conducting the right tests immediately following diagnosis.

Elevating the Standard of Care by Addressing Gaps in KnowledgeAccording to a recent US survey 1 of 230 oncologists conducted by IntegraConnect and sponsored by AstraZeneca, many clinicians often underestimate the prevalence of HRD and the role it plays in treating cancer. The survey, which sought to better understand HRD genomic instability testing habits, barriers to testing and current knowledge gaps, showed that two-thirds of oncologists surveyed thought that fewer than 40 percent of women with advanced ovarian cancer are HRD-positive though the prevalence is much higher at 50 percent.2

The survey also revealed a common misunderstanding about the role of disease markers in ovarian cancer in this rapidly evolving space. Although three-quarters of respondents said that they were extremely or very familiar with HRD testing, 73% incorrectly said that homologous recombination repair (HRR) gene panels found in next-generation sequencing (NGS) tests help identify markers of genomic instability, when in fact, HRD genomic instability and BRCA are the only actionable biomarkers for use of PARP inhibition-based regimens known today.

HRR and HRD are both important features in DNA damage response, but the two terms are often confused. HRR is not a marker of genomic instability and is not a predictive biomarker for who may respond to PARP inhibition so an HRRm gene panel should not be used in place of an HRD test to help inform treatment decisions, said Dr. Huh.

Integrating HRD Genomic Instability Testing into the Full Treatment PlanNow, with a deeper understanding of the underlying biology of advanced ovarian cancer, oncologists have the tools to match the right treatment with the right patient.

Available tests for identifying HRD genomic instability in advanced ovarian cancer include: myChoice CDx from Myriad Genetics; FoundationOne CDx from Foundation Medicine; Caris Molecular Intelligence Comprehensive Genomic Profiling Plus (CGP+) from Caris Life Sciences; and Tempus xT from Tempus. However, 21% of oncologists surveyed did not select any of these HRD testing partners when presented with a list of 12 choices.3

These gaps demonstrate there is still more work to be done to ensure patients receive the right test as soon as possible following diagnosis to inform treatment decisions. One way to approach this is by adding a literal checklist to your clinical routine. In my practice, this checklist includes HRD genomic instability testing and germline testing at the earliest opportunity to ensure we are providing the optimal information to our patients. Simple things can make a big difference and the earlier you conduct HRD genomic instability testing, the better.

Were only at the tip of the iceberg in expanding the clinical utility of precision medicine. We can go even further to improve the treatment of advanced ovarian cancer particularly for the estimated one in two patients with HRD-positive tumors, said Dr. Huh. To sustain progress, the medical community needs to move beyond BRCA testing alone and conduct HRD genomic instability testing at the first opportunity. Without this information, physicians may not be able to optimize treatment decisions based on the patients individuals type of disease.

As we aim to work towards a cure for this devastating disease, we can enhance our care discussions by educating about comprehensive biomarker testing and ensuring that we order the right tests tests at the right time.

Learn more about incorporating HRD testing into clinical practice.

Dr. Warner Huh was compensated for his time associated with this article.

Original post:
Precision medicine in advanced ovarian cancer: Incorporating homologous recombination deficiency (HRD) genomic instability testing to transform care -...

PALO ALTO, Calif. & SHANGHAI & PRINCETON, N.J.--(BUSINESS WIRE)--LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and other major Asian markets, and BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced the first patient has been treated in a Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification and other advanced solid tumors with FGFR genomic alterations.

Infigratinib is a potent and selective FGFR inhibitor that has demonstrated compelling clinical activity across multiple tumor types with FGFR alterations, said Yizhe Wang, Ph.D., chief executive officer of LianBio. Given the disproportionately high prevalence rate of gastric cancer in China, LianBio is pursuing a region-specific development strategy focused on this area of great unmet need. This study marks LianBios first trial initiation and demonstrates our continued progress in delivering potentially transformational medicines to patients in Asia.

TRUSELTIQ (infigratinib) is an oral selective inhibitor of FGFR1-3 that is approved in the United States for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. It is also being further evaluated in clinical trials based on demonstration of clinical activity in patients with advanced urothelial carcinoma with FGFR3 genomic alterations. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.

The Phase 2a trial is a multicenter, open-label, single-arm study in China designed to evaluate the safety and efficacy of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification and other advanced solid tumors with FGFR alterations. The primary endpoint is objective response rate (ORR). Secondary endpoints include duration of response, safety, disease control rate, progression-free survival and overall survival.

Preclinical data have demonstrated the potential infigratinib may have for patients with gastric cancer. These results, published in Cancer Discovery, demonstrated tumor regression in multiple in vivo FGFR2 amplified gastric models.1

We believe that infigratinib could have a meaningful impact for people living with gastric cancer as well as many other cancers with FGFR alterations, and are pleased LianBio is initiating this clinical trial in China where more therapeutic options are needed to match the growing diagnosis rate, said BridgeBio founder and chief executive officer Neil Kumar, Ph.D. On the heels of TRUSELTIQ recently obtaining accelerated approval in the United States, we are hopeful that this trial will yield pivotal results in another subset of cancer patients as we continue to build our portfolio of oncology indications with the aim of reaching as many people in need as possible.

About TRUSELTIQ (infigratinib)

TRUSELTIQ (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of fibroblast growth factor receptor (FGFR) that received accelerated approval from the FDA in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. TRUSELTIQ targets the FGFR protein, blocking downstream activity. In clinical studies, TRUSELTIQ demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response. TRUSELTIQ is not FDA-approved for any other indication in the United States and is not approved for use by any other health authority, including any Chinese or other Asian health authority. It is currently being evaluated in clinical studies for first-line cholangiocarcinoma, urothelial carcinoma (bladder cancer), locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma, and other advanced solid tumors with FGFR genomic alterations.

About BridgeBio Pharma, Inc.

BridgeBio is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBios pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the companys first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com.

About LianBio

LianBios mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and other major Asian markets, through partnerships that provide access to innovative therapeutic discoveries with a strong scientific basis and compelling clinical data. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a broad and clinically validated pipeline with the potential to impact patients with unmet medical needs. For more information, please visit http://www.lianbio.com.

About the LianBio and BridgeBio Pharma, Inc. Strategic Alliance

In August 2020, LianBio entered into a strategic alliance with BridgeBio, a commercial-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, to develop and commercialize BridgeBios programs in China and other major Asian markets. This strategic relationship initially focuses on two of BridgeBios targeted oncology drug candidates: FGFR inhibitor infigratinib, for the treatment of FGFR-driven tumors, and SHP2 inhibitor BBP-398, in development for tumors driven by MAPK pathway mutations. The agreement also provides LianBio with preferential future access in China and certain other major Asian markets to more than 20 drug development candidates currently owned or controlled by BridgeBio. This collaboration is designed to advance and accelerate BridgeBios programs in China and other major Asian markets, allowing BridgeBio and LianBio to potentially bring innovation to large numbers of patients with high unmet need.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act, and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to: the timing and success of the Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification, and other advanced solid tumors with FGFR genomic alterations; the planned approval of infigratinib by foreign regulatory authorities in China and the necessary clinical trial results, and timing and completion of regulatory submissions related thereto; and the competitive environment and clinical and therapeutic potential of infigratinib; reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation: the safety, tolerability and efficacy profile of infigratinib observed to date may change adversely in ex-U.S. clinical trials, ongoing analyses of trial data or subsequent to commercialization; foreign regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; the continuing success of the BridgeBio and LianBio strategic alliance; and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBio Pharma, Inc.s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SECs website at http://www.sec.gov. Except as required by law, each of BridgeBio and QED disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. Moreover, BridgeBio and QED operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on each of BridgeBios and QEDs current expectations, and speak only as of the date hereof.

1 Guagnano, V., Kauffman, A., Wrle, S., et al. FGFR Genetic Alterations Predict for Sensitivity to NVP-BGJ398, a Selective Pan-FGFR Inhibitor. Cancer Discovery 2 (2012): 1118-1133.

Read more:
BridgeBio Pharma and LianBio Announce First Patient Treated in Phase 2a Trial of Infigratinib in Patients with Gastric Cancer and Other Advanced Solid...

The height of a childs biological parents can be a good indicator of how tall a child will be, as genetics play a prominent role in determining height. However, this can vary, and siblings with the same parents may all reach different heights. Other factors, such as biological sex, overall health, nutrition, sleep, and exercise, during developmental years all factor into height and growth.

The height a person reaches by adulthood can depend on the genes they inherit from their biological parents, although some factors may mean a child does not reach their full potential height.

Nutrition and overall health during childhood and adolescence also affect human growth and height. Over hundreds of years, the average human height has increased due to improved nourishment in children and a reduction in illness and infections.

This article explores methods people may use to predict height, factors that affect growth in children and adolescents, and when to speak with a doctor if growth becomes a concern.

A combination of genetics and external factors can affect how tall a child will grow.

Health experts believe that 80% of a persons height is genetic. This means the height of biological parents can be an indicator of a childs height, although this is not always a reliable predictor.

Siblings with the same parents can vary in height, and one child in the family may be taller or shorter compared to the rest of the family.

Other factors, such as nutrition, illness, or premature birth, can also play a part in height and growth and may prevent a child from reaching their full potential height.

Learn more about which factors can influence a persons height.

According to Johns Hopkins Medicine, people may wish to try the following formula for predicting how tall a child will be:

However, note that predictive methods such as these are not concrete, and a childs adult height could change depending on different factors.

Learn how to measure height accurately.

According to the Society for Endocrinology, people can usually expect the following average growth patterns in children and adolescents:

Learn about the signs and stages of puberty.

Most females will have a growth spurt in the year before they start their first menstrual period. Their feet and hands will likely increase in size first, followed by the rest of the body.

Female growth slows down after their first menstrual period, but females will usually still grow 12 in after this time.

According to health experts, males usually have a growth spurt in puberty 2 years after most females. The peak time of growth is before sperm develops, and males will grow about 9 cm a year. Males also usually have longer growth spurts than females.

According to the Society for Endocrinology, there is no set age for when males and females will stop growing. Once a person has gone through all the stages of puberty to reach adult development, their growth will slow down and stop.

Additionally, growth plates in the bones fuse together at this stage, meaning individuals will not grow any taller.

Learn about when male children might stop growing.

Learn about when female children might stop growing.

Adolescents go through puberty at different stages, so variations in growth spurts can be normal. In some cases, going through puberty at a slower rate can be due to an inherited pattern, known as constitutional delay.

If there is too much variation, such as a growth spurt not occurring or females not menstruating by the age of 16 years, then it is important to speak with a doctor for a checkup.

In some cases, unusual growth or development in a young person may be the result of an underlying medical condition, such as:

Treating the underlying condition may help improve growth. In the case of a growth hormone deficiency, people may need treatment with artificial growth hormone.

Although genetics largely determine a persons height, proper nutrition is an important factor in healthy growth and development for children and adolescents.

According to the Academy of Nutrition and Dietetics, a balanced and nutritious diet for healthy growth and development includes:

According to a 2018 study, both exercise and good sleep can help increase height, as they elevate the release of growth hormones.

Sometimes, children or adolescents may face peer pressure or bullying for being a different height than those around them. This can lead to feelings of inadequacy, anxiety, or depression.

The American Academy of Child and Adolescent Psychiatry provides the following advice for parents and caregivers to help young people deal with peer pressure:

Learn more about teen anxiety and depression.

How tall a child or teenager will grow largely depends on the genes they inherit from their biological parents.

Good nutrition, exercise, and sleep also all play an important role in healthy growth and development. Illness, infection, or premature birth may all affect whether a child reaches their potential full height or not.

In most cases, young people will not be able to have much impact on their height other than living a healthy and balanced lifestyle. In some cases, children or adolescents may have a deficiency in growth hormone, which may require medical treatment to resolve.

If a child or adolescent is not growing or developing as expected for their age and biological sex, misses a growth spurt, or females do not have their first period by 16 years, then individuals can consult with their doctor for a checkup.

Excerpt from:
How tall will I be? A guide for parents and children - Medical News Today

Precision NanoSystems, Inc. 50 655 West Kent Ave North Vancouver, BC Canada V6P 6T7 1-888-618-0031

VANCOUVER, BC, Aug. 11, 2021 /PRNewswire/ - Precision NanoSystems (PNI), a global leader in innovative solutions for genetic medicine development, will expand its global headquarters with a 75,000-square-foot facility currently under construction. Slated for completion in Q4 2022, the new site will be located at 1055 Vernon Drive, in the Evolution Block building, in the False Creek Flats area of Vancouver. PNI's new genetic medicine GMP Biomanufacturing Centre will also be housed at the site.

PNI intends to expand capabilities to include the clinical manufacturing of RNA vaccines and therapeutics.

PNI's rapidly expanding team of more than 180 life science professionals will work from the site. James Taylor, General Manager, Precision NanoSystems, says: "This is a huge leap forward in our mission to accelerate the creation of transformative medicines that significantly impact human well-being. We are proud to contribute to the Canadian biotechnology industry by enabling them to develop and manufacture the next generation of medicines."

The Biomanufacturing Centre will support PNI's client base of leading drug developers through clinical development, as well as Canada's efforts for future pandemic preparedness through the manufacture of RNA vaccines.

PNI continues to rapidly expand its clinical manufacturing organization. The Biomanufacturing Centre is led by Elaine Copsey, VP of Biomanufacturing Operations, and Lloyd Jeffs, Senior Director of Biomanufacturing Services. Hiring is ongoing to find scientists, engineers, and other skilled professionals.

Elaine Copsey says: "PNI is excited to build on our successful preclinical services to include clinical and GMP production. We are growing our expertise as one of the industry leaders and will be hiring professionals with expertise in GMP operations including RNA and lipid nanoparticles manufacturing, process development, analytical testing, quality control, sterility, and quality assurance. PNI is looking forward to working with our clients to support all stages of their drug development process resulting in expedited therapeutic batches for clinical trials and routine production."

The Biomanufacturing Centre has been partially funded with CAD 25.1 million dollars from Canada's Strategic Innovation Fund (SIF).

The Honourable Franois-Philippe Champagne, Minister of Innovation, Science and Industry, says: "We are continuing to safely restart our economyby investing in companies like PNI that will help create well-paying jobs and ensure Canada is more prepared for future pandemics. By accelerating the creation of drugs and therapeutics, PNI will contribute to the strengthening of Canada's biomanufacturing sector and to building a stronger and healthier country for all of us."

PC Urban Properties is overseeing the construction of the new site. Brent Sawchyn, CEO, PC Urban Properties, says: "PNI's new lease solidifies the False Creek Flats area of Vancouver as a growing biotech and health science hub. We're excited to welcome PNI toEvolution Block, a pioneering stacked industrial development that will help transform one of the city's oldest industrial neighborhoods into its reimagined future."

About PNIPNI is a global leader in ushering in the next wave of genetic medicines in infectious diseases, cancer and rare diseases. We work with the world's leading drug developers to understand disease and create the therapeutics and vaccines that will define the future of medicine. PNI offers proprietary technology platforms and comprehensive expertise to enable researchers to translate disease biology insights into non-viral genetic medicines.

SOURCE Precision Nanosystems

http://www.precisionnanosystems.com

Read more from the original source:
Precision NanoSystems sets time and place to open new Genetic Medicine GMP Biomanufacturing Centre in Vancouver, BC - PRNewswire

Should doctors take particular care to talk to patients about the potential dangers of COVID-19 if those patients have a problematic relationship with pot?

New research from Washington University in St. Louis suggests perhaps they should.

Diabetes, obesity and a history of smoking cigarettes are all considered risk factors for poorer COVID-19 outcomes. Warnings and tailored information are targeted to people with these conditions, and doctors are acutely aware of the elevated risks they pose.

Findings from the lab of Ryan Bogdan, associate professor in the Department of Psychological & Brain Sciences in Arts & Sciences, suggests cannabis use disorder (CUD) should be added to the list because the genetic predisposition to CUD is overrepresented in people with poor COVID-19 outcomes. More work is needed to determine if there is direct causation.

The research is in press in the journal Biological Psychiatry: Global Open Science.

As sociocultural attitudes and laws surrounding cannabis use become increasingly permissive, and COVID-19 continues to spread, we need to better understand how cannabis use as well as heavy and problematic forms of use are associated with COVID outcomes, Bogdan said.

First author Alexander S. Hatoum, a postdoctoral researcher in the Washington University School of Medicine, used genetic epidemiological models to determine that genetic predisposition to CUD is related to risk for a severe reaction to COVID-19 (i.e., being hospitalized with COVID-19).

Hatoum combined existing datasets to test whether being at higher genetic risk for cannabis use disorder was correlated to the risk of COVID hospitalization.One set of data involved 357,806 people, including 14,080 with CUD; the other involved 1,206,629 people, including 9,373 who were hospitalized with COVID. He also looked at 7 million genetic variants to assess the association between CUD and severe COVID.

Having genetic variants does not mean a person has CUD or that the person has used cannabis.

In comparing people with the variants to their COVID outcomes, the researchers found genetic liability for CUD accounted for up to 40% of genetically influenced risk factors, such as body mass index (BMI) and diabetes, for a severe COVID-19 presentation.This association suggested that heavy and problematic cannabis use may represent a modifiable pathway to minimize severe COVID-19 presentations.

The results of this study point to two possible outcomes: That a predisposition to CUD and severe COVID-19 are due to a common biological mechanism, like inflammatory conditions causing individuals to develop worse symptoms of COVID-19 and/or dependence on cannabis; or that they are associated because of a causal process.

If we know the genes that predispose individuals to cannabis use disorder, and if cannabis use disorder is a risk factor for COVID-19 hospitalization, you will see the genes influencing cannabis use disorder as predictors of severe COVID-19 cases, Hatoum said. We found that a persons genetic risk for cannabis use disorder is correlated with their risk for COVID-19, without having to ask directly about illegal substance use.

The genetic association between CUD and COVID-19 severity was similar in size to genetic correlations between COVID-19 severity and BMI, a well-known risk factor for severe COVID-19 presentations. Moreover, it was present even when accounting for genetic liability to BMI as well as other risk factors for a severe reaction to COVID-19, including metabolic traits (e.g., fasting glucose, hypertension); respiration traits (e.g., forced expiratory volume, COPD); socioeconomic status; alcohol and tobacco use; and indices of impulsivity.

That the genetic relationship between CUD and COVID-19 is independent of these factors raises the intriguing possibility that heavy and problematic cannabis use may contribute to severe COVID-19 presentations. As such, it is possible that combating heavy and problematic cannabis use may help mitigate the impact of COVID-19, Hatoum said.

This information needs to be incorporated into any strategy to defeat this disease, Hatoum said.

These data suggest that heavy cannabis users may have a more adverse reaction to COVID-19 and that, much like quitting tobacco smoking or reducing BMI, reducing and/or stopping heavy cannabis use may protect against severe COVID-19 reactions.

Julia Strait contributed to this story.

See more here:
Cannabis use disorder: another COVID risk factor - Washington University in St. Louis Newsroom

People who work night shifts are at increased risk of developing an irregular and often abnormally fast heart rhythm called atrial fibrillation (AF), according to a new study led by a team of international researchers.

The findings were published in the European Heart Journal.

The study is the first to investigate the links between night shift work and AF. Using information from 283,657 people in the UK Biobank database, researchers found that the longer and more frequently people worked night shifts over their lifetimes, the greater their risk of AF. Night shift work was also linked to an increased risk of heart disease, but not to stroke or heart failure.

In addition, the researchers, led by Professor Yingli Lu, of Shanghai Ninth People's Hospital and Shanghai JiaoTong University School of Medicine, Shanghai, China, and Professor Lu Qi, of Tulane University School of Public Health and Tropical Medicine, New Orleans, USA, investigated whether genetic predisposition to AF could play a role in the increased risk.

They evaluated the overall genetic risk on the basis of 166 genetic variations known to be associated with the condition but found that the genetic risk levels did not affect the link between working night shifts and AF risk, regardless of whether participants had a low, medium, or high genetic risk.

Prof. Lu said: "Although a study like this cannot show a causal link between night shifts and atrial fibrillation and heart disease, our results suggest that current and lifetime night shift work may increase the risk of these conditions.

"Our findings have public health implications for preventing atrial fibrillation. They suggest that reducing both the frequency and the duration of night shift work may be beneficial for the health of the heart and blood vessels."

The study included 286,353 people who were in paid employment or self-employed. A total of 283,657 of these participants did not have AF when they enrolled in UK Biobank, and 276,009 did not have heart failure or stroke.

Information on genetic variants was available for 193,819 participants without AF, and 75,391 of them answered in-depth questions about their lifetime employment in a questionnaire sent out in 2015. Among the participants free of heart disease and stroke when they joined the study, 73,986 provided information on their employment history. During an average follow-up time of over ten years, there were 5,777 AF cases.

The researchers adjusted their analyses for factors that could affect the results, such as age, sex, ethnicity, education, socioeconomic status, smoking, physical exercise, diet, body mass index, blood pressure, sleep duration and chronotype (whether someone was a 'morning' or an 'evening' person).

They found that people who currently worked night shifts on a usual or permanent basis had a 12 per cent increased risk of AF compared to people who only worked during the day. The risk increased to 18 per cent after ten or more years for those who had a lifetime duration of night shifts. Among people who worked an average of three to eight-night shifts a month for ten years or more, the risk of AF increased to 22 per cent compared to daytime workers.

Among participants currently working night shifts, or working night shifts for ten or more years, or working a lifetime of three to eight night shifts a month, the risk of coronary heart disease increased by 22 per cent, 37 per cent and 35 per cent respectively compared to daytime workers.

Prof. Qi said: "There were two more interesting findings. We found that women were more susceptible to atrial fibrillation than men when working night shifts for more than ten years. Their risk increased significantly by 64% compared to day workers. People reporting an ideal amount of physical activity of 150 minutes a week or more of moderate-intensity, 75 minutes a week or more of vigorous-intensity, or an equivalent combination, had a lower risk of atrial fibrillation than those with non-ideal physical activity when exposed to a lifetime of night shift work. Thus, women and less physically active people may benefit particularly from a reduction in night shift work."

A strength of the study is its size, with detailed information on over 283,000 people. In addition, it is the first study to link these data with genetic information in a population that also has detailed histories available on current shift work and lifetime employment.

Limitations of the study include the fact that it cannot show shift work causes heart problems, only that it is associated with them; some cases of atrial fibrillation may have been missed; lifetime employment was assessed only when people joined UK Biobank, was self-reported, and, therefore, may have changed or been prone to some errors; there may be unknown factors that might affect the results, and the people in UK Biobank were mainly white British and so it may not be possible to generalise the findings to other ethnic groups.

Prof. Lu said: "We plan to analyse the association between night shift work and atrial fibrillation in different groups of people. This may strengthen the reliability of these results and serve as a warning to groups working in certain types of occupations to get their hearts checked early if they feel any pain or discomfort in their chests."

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People working night shift face increased risk of developing heart problems: Study - Hindustan Times

PHILADELPHIA, Aug. 17, 2021 /PRNewswire/ -- Coriell Life Sciences, an international leader in precision medicine, announced today its debuton the annual Inc. 5000 list, the most prestigious ranking of the nation's fastest-growing private companies. The list represents a unique look at the most successful companies within the American economy's most dynamic segment.

Our scalability enables us to deliver turnkey precision medicine solutions to people and organizations around the world.

"Having Coriell Life Sciences' growth story recognized is a tremendous honor for our entire team," says Scott Megill, President & CEO of Coriell Life Sciences. "The most important part of this story goes beyond our traction in rolling out personalized medication safety programs to help a growing number of employers and payers improve population health and lower healthcare costs. It's also about the scalability that we're introducing to the market. This scalability enables us to deliver turnkey precision medicine solutions to organizations and individuals around the globe and empower a healthier world."

Precision medicine is at a tipping point, especially as more employers and health plans offer personalized medication safety benefits to their employees, retirees, and members. CLS' Corigen Medication Safety Program is the most comprehensive medication risk management program on the market. It uses the science of pharmacogenomics (PGx) to identify which medications are the safest and most effective for individuals based on their unique DNA. In addition to minimizing adverse effects and improving individual health, it reduces healthcare costs for sponsoring organizations by minimizing the inefficiencies of trial-and-error prescribing.

"The 2021 Inc. 5000 list feels like one of the most important rosters of companies ever compiled," says Scott Omelianuk, editor-in-chief of Inc. "Building one of the fastest-growing companies in America in any year is a remarkable achievement. Building one in the crisis we've lived through is just plain amazing. This kind of accomplishment comes with hard work, smart pivots, great leadership, and the help of a whole lot of people."

To learn more, visit coriell.com.

About Coriell Life Sciences

Coriell Life Sciences (CLS), a leader in genetic science,is spearheading innovation in precision medicine to reduce healthcare costs and empower a healthier world.With advanced bioinformatics technology, CLS bridges the gap between genetic knowledge and clinical application and offers the most comprehensive medication risk management program on the market.Visit coriell.com, email info@coriell.comor follow @CoriellLife.

Media Contact:

Pamela Caruolo

For Coriell Life Sciences

pamela@caruolocommunications.com

484.574.2946

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SOURCE Coriell Life Sciences

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Coriell Life Sciences Makes its Debut on the Inc. 5000 as One of America's Fastest-Growing Private Companies - The Wellsboro Gazette

CAMBRIDGE, Mass., Aug. 11, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported recent business highlights and second quarter 2021 financial results.

This quarter we announced our shift to rapid enzymatic synthesis, or RES, for production of our closed-ended DNA, ceDNA, constructs and our signing of a lease to establish significant internal current Good Manufacturing Practice, or cGMP, manufacturing capacity. These are important steps toward our goal of extending the reach of our durable, redosable genetic medicines to patients with prevalent diseases, said Geoff McDonough, M.D., chief executive officer of Generation Bio. We will continue to advance the platform throughout the rest of the year and expect factor VIII expression data with ceDNA produced using RES in non-human primatesfor our hemophilia A program by year-end.

Business Highlights

Second Quarter 2021 Financial Results

About Generation Bio

Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral genetic medicine platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bios manufacturing scale to hundreds of millions of doses to support its mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.

For more information, please visit http://www.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platform, our research and clinical development plans, the expected timing of the submission of IND applications and preclinical data, our manufacturing plans, our expectations regarding our new facility and other statements containing the words believes, anticipates, plans, expects, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the timing and ability to complete the build-out of the companys manufacturing facility and regarding the new manufacturing process; expectations regarding the timing of submission of IND applications; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the companys cash resources are sufficient to fund the companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of our most recent annual report on Form 10-K and quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the companys views as of the date hereof. The company anticipates that subsequent events and developments will cause the companys views to change.However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.These forward-looking statements should not be relied upon as representing the companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsMaren KillackeyGeneration Bio541-646-2420mkillackey@generationbio.com

MediaAlicia WebbGeneration Bio847-254-4275awebb@generationbio.com

Lisa RaffenspergerTen Bridge Communications617-903-8783lisa@tenbridgecommunications.com

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Unaudited)

(in thousands, except share and per share data)

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Generation Bio Reports Business Highlights and Second Quarter 2021 Financial Results - GlobeNewswire

Recent changes to patient right of access policies could open the door to increased privacy challenges.

Twenty-five years in, the Health Insurance Portability and Accountability Act (HIPAA) and its related privacy and security regulations have been both celebrated and criticized. Recent developments are transforming patient right of access into a gateway for third parties. Where this transformation ultimately leads is uncertain.

One possibility is a learning health system, fueled by patient contributed data and sophisticated data science and governed with an eye to advancing population health and equity while protecting privacy and maintaining trust. Another possibility is health related corporate surveillance on steroids.

The 1973 report, Records, Computers and the Rights of Citizens, credited with originating the term fair information practice, included an access right, which made data about individuals fully available to them, upon request, in a comprehensible form. Section 164.524 of the 2000 HIPAA Privacy Rule gave patients a right to inspect and obtain a copy of their information, with only a few exceptions. The Health Information Technology for Economic and Clinical Health (HITECH) Act updated the access right for the era of electronic health records (EHRs).

Rights on the books and in practice are, of course, two different things.

Problems soon surfaced when patients tried to exercise their access right. For example, the American Civil Liberties Union filed a complaint on behalf of patients seeking their full genetic records from Myriad Genetics. Subsequently, Congress enacted the 21st Century Cures Act, which mandated a Government Accountability Office report on barriers to access. In addition, the U.S. Department of Health and Human Services Office for Civil Rights launched a HIPAA Right of Access Initiative. Ciitizen, a consumer health technology company, published a scorecard that suggests compliance with the HIPAA right of access is finally improving.

Announced earlier this year, a proposed modification to the HIPAA Privacy Rule added the right to direct the transmission of certain protected health information in an electronic format to a third party, which provides that an individual has a right of access to direct a covered health care provider to transmit an electronic copy of protected health information in an electronic health record directly to another person designated by the individual. The individuals request would need to be clear, conspicuous, and specific. There is, however, no point-by-point specification of required elements for the request, as would be the case with an authorization.

Although examples of possible recipients are provided in the proposed modification, there are no limits on who can be a third-party recipient, and the access right redirect extends to any person or entity the individual chooses. There is request for comment about whether health care providers should be required to inform patients about the privacy and security risks of transmitting information to entities that are not covered by HIPAA.

Efforts to develop a pathway for patients to share data with researchers have also been supercharged by the Precision Medicine Initiatives (PMI) All of Us research program, which aims to enroll over one million Americans. Diversity is a priority, and so is bringing together many different types of data, including EHR data.

The technology to transmit EHR data to All of Us, and potentially to other research studies, is being developed through a publicprivate partnership known as Sync for Science (S4S). A pilot involving four EHR vendors resulted in a successful launch of connectivity at six provider sites. Given the challenges, widespread adoption may take time, but this effort is proof of principle for patient EHR sharing with researchers through application programming interfaces (APIs).

An important part of the story, in addition to technical feasibility, is the ethical and policy framework for implementation. In 2017, the Office of the National Coordinator for Health Information Technology published a report on privacy and security considerations for health care APIs. Linked to S4S, it cites the Precision Medicine Initiative Privacy and Trust Principles and Data Security Policy Principles and Framework as important guides.

The report advises that, in accordance with the principle of transparency, individuals approving data transfers should be warned that the health care providers responsibility stops once data are transmitted to the third party. As a tip for implementers, it suggests that EHR patient portals give patients a way to view and manage all third-party apps that have access to information about them, including revoking HIPAA access requests.

Interestingly, the PMI Privacy and Trust Principles begin with governance, and the first principle under governance is substantive participant representation at all levels of program oversight, design, implementation, and evaluation. The All of Us Research Program has invested in an ambassador program that integrates participant representatives in governance in line with this principle. Justifications for the All of Us ambassador program include respect for persons, relationship to trust, and the recognition that more ethical weight has been placed on transparency and individual consent than they can bear.

Combining a vision of patient driven research progress with commitments to diversity, equity, and inclusion and trust enhancing privacy, security, and governance principles is the promised land for advocates of HIPAA access right facilitated data sharing.

But perhaps the HIPAA access right facilitated data sharing could just as easily lead elsewhere. If usual patterns hold, at least initially, patient-driven data sharing may exacerbate the diversity problem affecting genomic and other research databases. Early adopters will likely come from the most privileged tier of society. This is especially true in the United States, where inequality is increasing and many less privileged groups have limited access to technology and experience social and economic insecurity that makes them justifiably averse to privacy risks.

Furthermore, critics have already raised the alarm about the flow of de-identified information permitted under HIPAA. The addition of a process that may be easy to manipulate to gain relatively unrestricted access to identifiable patient information, including sensitive genomic data, may take data privacy from leaky to hemorrhaging.

In response, the CARIN Alliance developed a voluntary code that incorporates many important protections. Unfortunately, the history of technology companies such as Facebook does not foster faith in the power of wisdom and benevolence to mitigate a move fast and break things mindset.

Tips and codes are great, but the health care sector also needs requirements. For example, an easy-to-find and easy-to-navigate dashboard within patient portals should be a must have rather than a nice to have feature for access requests directing EHR data to third parties.

In addition, the individual is no match for entities that skillfully manage attention and manipulate choices that would be contrary to their interests. Laws and regulations that reach beyond HIPAA should impose data use limitations in line with reasonable expectations, spur more robust and inclusive governance structures, and provide better protection from downstream harms such as discrimination.

Mary Anderlik Majumder is a professor with the Center for Medical Ethics and Health Policy at the Baylor College of Medicine.

This essay is part of a six-part series, entitledReflecting on 25 Years of HIPAA.

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The HIPAA Right of Access and Data Sharing - The Regulatory Review