Category Archives: Gene Medicine

DUBLIN--(BUSINESS WIRE)--The "Global Genome Editing Market By Technique (CRISPR, Zinc Finger Nucleases, TALENs, Restriction enzymes, Others), By Applications (Synthetic Biology, Engineering Cell Line and Organisms, Others), By Source, By End-User, By Region, Forecast & Opportunities, 2025" report has been added to ResearchAndMarkets.com's offering.

The Global Genome Editing Market is expected to grow at a brisk rate during the forecast period owing to growing number of research activities for treatment of various chronic diseases using this technology. Further, increased government funding for genomics technology around the globe, growing preference for personalized medicine and increase in R&D expenditure are fueling the market growth of genome editing.

Genome editing is a way of making specific changes to the DNA of a cell or organism. It could be used to edit the genome of any organism. It uses a type of enzyme called an engineered nuclease' which cuts the genome in a specific place. After cutting the DNA in a specific place, the cell naturally repairs the cut. It finds application in large number of areas, such as mutation, therapeutics, and agriculture biotechnology. Moreover, rise in the number of chronic and infectious diseases is likely to fuel the market for genome editing in the coming years.

The Global Genome Editing market is segmented based on technique, applications, source, end-user and region. Based on applications, the market is segmented into synthetic biology, engineering cell line & organisms, therapeutic genome editing and others. Among them, the cell line engineering is expected to witness the highest growth rate in the coming years due to increase in the number of people suffering with genetic disorders and rising government funding for stem cell research.

Based on end-user, the Global Genome Editing Market is segmented into pharmaceutical & biotechnology companies, clinical research organization and research institutes. Pharmaceutical & biotechnology companies contribute to the largest share of revenue generation for the Global Genome Editing Market. Growing establishments of biotech and pharma companies in emerging economies and growing usage of gene editing technique in research activities undertaken by them to manufacture and develop drugs for rare diseases anticipated to fuel the market across the globe.

Companies Mentioned

Objective of the Study:

Key Topics Covered:

1. Product Overview

2. Research Methodology

3. Executive Summary

4. Global Genome Editing Market Outlook

4.1. Market Size & Forecast

4.2. Market Share & Forecast

4.3. Market Attractiveness Index

5. Asia-Pacific Genome Editing Market Outlook

5.1. Market Size & Forecast

5.2. Market Share & Forecast

5.3. Market Attractiveness Index

5.4. Asia-Pacific: Country Analysis

6. Europe Genome Editing Market Outlook

6.1. Market Size & Forecast

6.2. Market Share & Forecast

6.3. Market Attractiveness Index

6.4. Europe: Country Analysis

7. North America Genome Editing Market Outlook

7.1. Market Size & Forecast

7.2. Market Share & Forecast

7.3. Market Attractiveness Index

7.4. North America: Country Analysis

8. South America Genome Editing Market Outlook

8.1. Market Size & Forecast

8.2. Market Share & Forecast

8.3. Market Attractiveness Index

8.4. South America: Country Analysis

9. Middle East and Africa Genome Editing Market Outlook

9.1. Market Size & Forecast

9.2. Market Share & Forecast

9.3. Market Attractiveness Index

9.4. MEA: Country Analysis

10. Market Dynamics

10.1. Drivers

10.2. Challenges

11. Market Trends & Developments

12. Competitive Landscape

12.1. Competition Outlook

12.2. Players Profiled (Leading Companies)

13. Strategic Recommendations

14. About Us & Disclaimer

For more information about this report visit https://www.researchandmarkets.com/r/tgb83z

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Outlook on the Worldwide Genome Editing Industry to 2025 - Featuring Pfizer, Bayer Crop Science & Editas Medicine Among Others -...

With all the attention that COVID-19-related companies have gotten over the past couple of months, it's easy to forget that there are plenty of other promising stocks out there to invest in. Although it's true that this pandemic has changed the way many companies do business, there are still tons of companies out there that are thriving during this time.

While the four companies listed below are all quite different, they have one thing in common: You've likely forgotten about their stocks due to this coronavirus situation. Let's take a look at just why you should consider adding these four companies to your portfolio going forward in 2020.

Image source: Getty Images.

At present, there are about 425 million people around the world living with diabetes, a figure that's expected to grow to 629 million by 2024. It shouldn't come as a surprise that demand for diabetes-related products will also increase as well.

Insulet (NASDAQ:PODD) is one of the leading insulin pump manufacturers on the market. Insulin pumps are small medical devices that automatically deliver small doses of insulin into the body. While not all diabetes patients need to use insulin pumps, it's much more convenient than having to manually inject insulin multiple times throughout the day, as is the case with type 1 diabetes patients.

Insulet's OmniPod insulin pump brand has seen tremendous growth over the past year. In Insulet's first-quarter financial results, the company reported $189.7 million in total OmniPod revenue. That's a 32.7% increase from the same time last year, with more than 90% of Insulet's total revenue coming from sales of the OmniPod.

While there are other insulin pump makers on the market, such as Tandem Diabetes, Insulet's OmniPod stands out for a couple of reasons. For one, it's one of the only waterproof pumps on the market. At the same time, being tubeless, it can be discreetly worn beneath clothing without anyone noticing, something that can't be said for most other insulin pumps on the market.

Gene-editing is another sector in the world of biotech that has major potential to disrupt the norms of healthcare, although most companies in this market are relatively small and still in early stages of clinical development for their programs. Editas Medicine (NASDAQ:EDIT) is one such company that has a lot of potential with its own lineup of gene-editing drug candidates.

Editas's flagship candidate, EDIT-101, targets a rare type of childhood blindness called leber congenital amaurosis (LCA), which affects the retina's ability to detect light and color. Although quite rare, affecting about 2 newborns per 100,000, there are almost no treatments available for this condition,and relatively little that can be done once it's diagnosed in a child. The only treatment on the market is called Luxturna, and it is only effective for a very specific type of LCA that only a minority of patients are diagnosed with.

Back in March, Editas announcedthat it had begun testing EDIT-101 in a phase 1/2 clinical trial with 18 human patients.. Although Editas has a number of other candidates in development -- such as its sickle-cell-disease treatment, EDIT-301 --what makes EDIT-101 so promising is its lack of competition. While there tends to be plenty of overlap between gene-editing companies and their candidates, Editas is one of the only businesses in this sector working on an LCA treatment at this time. Considering that Luxturna costs about $700,000, EDIT-101 could end up making Editas a lot of money should its clinical trials turn out well.

Image source: Getty Images.

Depending on whether you follow mining-related news, Pan American Silver (NASDAQ:PAAS) may or may not be a company you've heard of before. However, like most gold and silver miners, Pan American has been enjoying remarkably high prices for precious metals thanks to the uncertainty surrounding the global markets.

While gold and silver both have some industrial use, most of their demand comes from cautious investors looking for a place to store their money. That's because prices for precious metals tend to rise as fears surrounding the economy get worse.

This is great news for mining companies, as higher prices mean larger profit margins. When looking at Pan American's most recent fourth-quarter financial results, revenue came in at $404.4 million. That's more than double the $173.4 million reported during Q4 2018. Net earnings are now in the positive as well, sitting at $51.7 million, compared with a net loss of $63.6 million a year ago.

Pan American also finished the acquisition of Tahoe Resources, which included the rights to one of the largest silver mines in the world, the Escobal project. While the mine is currently inactive due to COVID-19 as well as a pending consultation regarding neighboring indigenous communities, Pan American's silver output is expected to drastically increase once it starts operating once more.

Insurance might not sound like a super-exciting industry, but it's easily one of the most resilient sectors in the economy. Even better, there are some specific areas of the insurance industry that are reporting compelling growth.

Trupanion (NASDAQ:TRUP) is one of the only insurance companies dedicated to providing insurance to pets. While it's easy to think that pet owners have always considered their animals to be like family, that wasn't always the case. People are spending more money now than ever on taking care of their furry friends. In 2018, the U.S. pet industry grew to $72 billion, well more than double the $29 billion seen in 2001.

As such, demand for pet health insurance has been surging, with Trupanion's quarterly revenue figures having grown exponentially over the past 10 years. Quarterly revenue for Q1 2020 came in at $111.3 million, an impressive 28% increase from the same time last year. The only mild downside to Trupanion is the fact that it's still operating at a very slight loss, about $1.1 million for the first quarter.

Overall, Trupanion seems poised to see steady growth well into the future. While it's hard to find companies that are both recession-resistant and growing rapidly, Trupanion seems to be one of the few stocks that qualifies.

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4 Promising Stocks to Own That You Probably Forgot About - Motley Fool

Why can some people eat as much as they want, and still stay thin?

In a study published today in the journal Cell, Life Sciences Institute Director Dr. Josef Penninger and a team of international colleagues report their discovery that a gene called ALK (Anaplastic Lymphoma Kinase) plays a role in resisting weight gain.

We all know these people, who can eat whatever they want, they dont exercise, but they just dont gain weight. They make up around one per cent of the population, says senior author Penninger, professor in the Faculty of Medicines department of medical genetics and a Canada 150 research chair.

Dr. Josef Penninger

We wanted to understand why, adds Penninger. Most researchers study obesity and the genetics of obesity. We just turned it around and studied thinness, thereby starting a new field of research.

Using biobank data from Estonia, Penningers team, including researchers from Switzerland, Austria, and Australia, compared the genetic makeup and clinical profiles of 47,102 healthy thin, and normal-weight individuals aged 20-44. Among the genetic variations the team discovered in the thin group was a mutation in the ALK gene.

ALKs role in human physiology has been largely unclear. The gene is known to mutate frequently in several types of cancer, and has been identified as a driver of tumour development.

Our work reveals that ALK acts in the brain, where it regulates metabolism by integrating and controlling energy expenditure, says Michael Orthofer, the studys lead author and a postdoctoral fellow at the Institute of Molecular Biology in Vienna.

When Penningers team deleted the ALK gene in flies and mice, both were resistant to diet-induced obesity. Despite consuming the same diet and having the same activity level, mice without ALK weighed less and had less body fat.

As ALK is highly expressed in the brain, its potential role in weight gain resistance make it an attractive mark for scientists developing therapeutics for obesity.

The team will next focus on understanding how neurons that express ALK regulate the brain at a molecular level, and determining how ALK balances metabolism to promote thinness. Validating the results in additional, more diverse human population studies will also be important.

Its possible that we could reduce ALK function to see if we did stay skinny, says Penninger. ALK inhibitors are used in cancer treatments already, so we know that ALK can be targeted therapeutically.

The study was supported by the Estonian Research Council, the European Union Horizon 2020 fund, and European Regional Development Fund, the von Zastrow Foundation, and the Canada 150 Research Chairs Program.

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UBC scientist identifies a gene that controls thinness - UBC Faculty of Medicine - UBC Faculty of Medicine

Broadcast Date: June 18, 2020Time: 8:00 am PT, 11:00 am ET, 17:00 CET

Cell and Gene therapies continue to evolve in their use for treating human diseases. Cell-based therapies are emerging as a promising strategy for cancer, while AAV vectors have taken center stage as a gene delivery vehicle for potential gene therapy for several human diseases.

In this GEN webinar, our expert speakers, who are leading investigators in the field ofcell and gene therapy, will discuss emerging gene-edited and engineered cell therapies for cancer, as well as the next generation of AAV vectors for human gene therapy. Additionally, our speakers will cover some of the following key points:

Advancements in cell surface receptor-targeted adult stem cells, cancer cells, and T cells expressing novel bi-functional immunomodulatory proteins Demonstrate the strength of using innovative gene therapy approaches clinically Provide data and rationale for assessing combined cell- and gene-based approaches in preclinical studies

A live Q&A session will follow the presentations, offering you a chance to pose questions to our expert panelists.

Produced with support from:

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Looking toward the Future of Cell & Gene Therapies - Genetic Engineering & Biotechnology News

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic for LYNPARZA (olaparib), which was also approved today in the U.S. for adult patients with deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with enzalutamide or abiraterone. FoundationOne CDx is the only FDA-approved comprehensive genomic profiling (CGP) test for all solid tumors that incorporates multiple companion diagnostic claims.

Prostate cancer is the second most common cancer in men; 1 in 9 will be diagnosed during their lifetime.1 mCRPC occurs when prostate cancer grows and spreads to other parts of the body despite the use of androgen-deprivation therapy to block the action of male sex hormones.2 Because there have previously been limited treatment options for this specific disease area, there is generally a high mortality rate.

This therapy and companion diagnostic approval underscores the value of comprehensive genomic profiling in advanced cancer patients as it validates our ability to identify alterations in the 14 HRR pathway genes within FoundationOne CDxs 324 gene panel that indicate a patient may be eligible for treatment with Lynparza, a process not possible through single gene or hot spot testing, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. This is an important advancement for patients with HRR-mutated metastatic castration-resistant prostate cancer, as there have previously been limited treatment options available for this specific condition.

FoundationOne CDx is the first FDA-approved broad companion diagnostic that is clinically and analytically validated for solid tumors. FoundationOne CDx is currently approved as a companion diagnostic for more than 20 targeted therapies.

LYNPARZA was approved based on the PROfound study, which was supported by Foundation Medicine and was the first phase III biomarker-selected study using a molecularly targeted treatment in men with metastatic castration-resistant prostate cancer (mCRPC) to demonstrate improved outcomes. The PROfound trial is the largest prospective study to date performing central tissue testing for homologous recombination repair (HRR) gene mutations in mCRPC patients. The clinical trial assay (CTA) is an NGS assay based on FoundationOne CDx.

LYNPARZA is jointly developed and commercialized by AstraZeneca (LSE/STO/NYSE: AZN) and Merck & Co., Inc.

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit http://www.foundationmedicine.com/genomic-testing/foundation-one-cdx.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

Source: Foundation Medicine

1American Cancer Society Key Statistics for Prostate Cancer: https://www.cancer.org/cancer/prostate-cancer/about/key-statistics.html 2 Cancer.Net. (2019). Treatment of metastatic castration-resistant prostate cancer. http://www.cancer.net/research-and-advocacy/asco-care-and-treatment-recommendations-patients/treatment-metastatic-castration-resistant-prostate-cancer

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Foundation Medicine Receives FDA Approval for FoundationOneCDx as the Companion Diagnostic for LYNPARZA to Identify Patients with HRR-Mutated...

In patients with coronavirus disease 2019 (COVID-19), higher sputum cell expression of angiotensin converting enzyme 2 (ACE2) and transmembrane protease serine 2 (TMPRSS2) was observed in certain patients with asthma while lower expression was found in patients who used inhaled corticosteroids (ICS), according to study results published in the American Journal of Respiratory and Critical Care Medicine.

COVID-19, caused by severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2), may be more severe in patients with chronic lung disease, including patients with asthma, and it appears that demographic or biological factors influence susceptibility to the infection or severity of disease. Because ACE2 and TMPRSS2 mediate viral infection of host cells, researchers reasoned that differences in ACE2 or TMPRSS2 gene expression in sputum cells in patients with asthma may identify subgroups at risk for COVID-19 morbidity.

By analyzing gene expression for ACE2 and TMPRSS2 as well as intercellular adhesion molecule 1 (ICAM-1) in sputum cells from 330 participants and 79 healthy control individuals, researchers found that gene expression of ACE2 was lower than TMPRSS2, and that expression levels of both genes were similar in patients with asthma and healthy individuals. In patients with asthma, however, men, African Americans, and people with diabetes had higher expression of ACE2 and TMPRSS2. In patients with asthma, ICAM-1 expression increased and there were fewer consistent differences related to sex, race, and ICS use. Use of ICS was associated with lower expression of ACE2 and TMPRSS2, while treatment with triamcinolone acetonide did not decrease expression of either gene or ICAM-1.

Higher expression of ACE2 and TMPRSS2 in males, African Americans, and patients with diabetes mellitus provides rationale for monitoring these asthma subgroups for poor COVID-19 outcomes, the study authors wrote. The lower expression of ACE2 and TMPRSS2 with ICS use warrants prospective study of ICS use as a predictor of decreased susceptibility to SARS-CoV-2 infection and decreased COVID-19 morbidity.

Reference

Peters MC, Sajuthi S, Deford P, et al; for the National Heart, Lung, and Blood Institute Severe Asthma Research Program-3 Investigators. COVID-19 related genes in sputum cells in asthma: Relationship to demographic features and corticosteroids [published online April 29, 2020]. Am J Respir Crit Care Med. doi:10.1164/rccm.202003-0821OC

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COVID-19-Related Genes Have Higher Expression in Certain Patients With Asthma - Pulmonology Advisor