Genome Editing a Progressing Trend

The Rush for Genome Editing

Frank Magliochetti Report

The Rush for Genome Editing

Gene-editing technology has sparked a frenzy of competitors fueled to develop the first of what may be thousands of treatments for genetic disorders.

From science fiction of movies like Jurassic Park to the reality of GMO foods, the concept of customizing plants, animals and even human genomes has mesmerized our imagination and culture for decades. This obsession is not surprising considering the remarkable impact of genetic engineering on our lives, and the promise it may hold for our future.crispr-genome-editing-franlmagliochetti-report_northandoverma

Modifying the genes of an organism is no easy task. It’s not as simple as removing the parts you do not like with an eraser. It involves making changes at a molecular level that are both complex and challenging.

One concern among scientists is the modifications made in human germline cells – i.e. the sperm or the egg. These are genetic changes that would actually be passed on through generations and the fear is that there could be unexpected side effects. Couple that with the ethical concerns of people misusing this technology to intentionally modify the genome to make “designer babies” with enhanced characteristics. These are some real concerns that are causing scientists to pause and weigh the pros and cons of use of this technology.

More and more companies are racing to stay ahead of the trend and become the leaders in the field.

In Cambridge MA, Intellia Therapeutics Inc. has a 65,000 square foot build out underway, however, it is still planning to functioning while under construction.

Intellia is one of a trio of startups in the Cambridge area working locally in the biotech niche known as CRISPR-Cas9. The gene-editing technology was frank-magliochetti-report_intelliadiscovered just four years ago.

Drug development historically has been a slow process – it often takes more than a decade to bring a medicine from lab to marketplace. But that’s changing, thanks to new machines that can screen drug targets faster and an entrepreneurial environment that’s more akin to high-tech than Big Pharma.frankmagliochettireport_genome_editing

Intellia has high-profile collaboration deals with Swiss drug giant Novartis AG and Regeneron Pharmaceuticals Inc. of Tarrytown, N.Y. It raised $108 million in an initial public offering in May. Their scientists are working on gene-editing treatments for everything including the hepatitis B virus to a rare form of blindness. If all goes well, it could move its earliest drug candidates into clinical trials within 18 to 24 months.

Competitors for Intellia include Editas Medicine and Crispr Therapeutics Inc. both completed their own IPOs this year. There are more to come: all with the heightened interest of investors. These companies are in a race to develop the first treatment based on gene editing.

Genome editing is going to be a hot trend to watch in 2017 and for many years to come.

Frank Magliochetti is Managing Partner for Parcae Capital.

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank Magliochetti Report is a new site dedicated to the industry.  He currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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Personal Healthcare Spending Trends

New Study Sheds Light on Personal Health Care Spending Trends in the U.S.

The amount of dollars the Unites Spends on healthcare surpasses every other nation on the planet; all in all accounting for 17% of the economy in the U.S.

Frank Magliochetti

People spend more for health care in the United States than in any other nation on earth and, according to results of a new study published in JAMA, they spend more on diabetes and ischemic heart disease than on any other health condition.

Health care spending continues to rise in the U.S., now accounting for 17 percent of the nation’s economy. health-spending-Frank-MAgliochetti-ReportDespite this spending, there is very little information on how spending varies by condition, age and through time. Joseph L. Dieleman, PhD, from the Institute for Health Metrics and Evaluation, University of Washington and a team of researchers hoped to estimate national spending on personal health care by various factors.

U.S. Spending on Health Care Trends

The researchers included 183 sources of data in the study. Data sources included insurance claims, government budgets, household surveys, facility surveys, and official U.S. records from 1996 to 2013. The scientists grouped ICD-9 codes to form 155 conditions, such as diabetes and ischemic heart disease, for consideration in the study.

One of the more interesting findings of the study was that many of the top 20 conditions of health care spending were chronic conditions with a relatively high prevalence and health burden – many of them were also preventable. This group of conditions included diabetes, ischemic heart disease, chronic obstructive pulmonary disease (COPD), and cerebrovascular disease, all of which are attributable to modifiable risk factors.

Total costs of care

Americans spent $30.1 trillion on personal health care during the years included in the study. The researchers looked at how Americans spent that money, estimating the costs of treating 155 conditions. They found that, at an estimated $101 billion, Americans spent the most on treating diabetes. Spending for ischemic heart disease came in second at $88 billion; spending for low back and neck pain was a close third at $87 billion.

Increases in costs of care

Spending for personal health care increased for 143 of the conditions investigated over the course of the study. Spending on low back and neck pain increased $57 billion during those 18 years, and spending on diabetes US-healthcare-costs-FrankMagliochettiincreased $64 billion during that period.

Of all the conditions included in the study, 57 percent of spending went towards the top 20 conditions. Spending on emergency care and retail pharmaceuticals rose the fastest, at 6.4 percent and 5.6 percent annual growth rate, respectively. When it came to spending on diabetes, 57.6 percent went to pharmaceuticals while 23.5 percent was for ambulatory care.

The study was important in that it was the first to provide modeled estimates of U.S. personal health care spending. The results were revealing in that they showed that diabetes, ischemic heart disease, and low back and neck pain presented the highest costs to American consumers. The study was limited in that it used population-weighted data to represent total national spending, which excludes incarcerated persons and those receiving care from a Veterans Affairs (VA) facility. The University of Washington institutional review board reviewed and approved the project.

The information presented in the study may be useful to health care policy makers and health care providers working towards making health care spending more cost effective for the conditions that most commonly affect people living in the United States.

Source

http://jamanetwork.com/journals/jama/fullarticle/2594716

Frank Magliochetti is Managing Partner for Parcae Capital

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release new sites dedicated to the industry. Frank currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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3D Bioprinter Produces Functional Skin

3D Bioprinter Prints Functional Human Skin Adequate for Transplant

3dprintedskin-frankmagliochetti-healthcareinnovationA team of researchers in Spain reports the development of a new three-dimensional (3D) printer capable of printing human skin suitable for transplant into patients. The printed skin is also suitable for testing drugs and cosmetics.

The team included several groups of researchers, including a group from the Universidad Carlos III de Madrid (UC3M) in Spain. The researchers describe their breakthrough in the scientific journal, Biofabrication.

3D Printing and Skin Production

3D printing has been around since the 1980s, when Charles (Chuck) Hull introduced the first stereolithography apparatus (SLA), but the 3D printing industry is now experience rapid growth with applications in many fields. In medicine, 3D printing holds great promise in someday giving clinicians the ability to produce personalized, complex human tissues and organs on demand. One woman has already received a 3D printed ear from one company, for example, while another frankmagliochettireport-3d-bioprintercompany provides 3D printed implants that the recipient’s body converts to bone.

Three-dimensional printing of human body parts is challenging in a number of ways. Replicating the complexity of anatomical structures is difficult. Ensuring the printed tissue survive transplantation in a living body is another problem.

The scientists in Spain have already engineered plasma-based, two-layered skin used successfully in the treatment of burns and other wounds in a large number of patients. The primary drawback to this method is that it takes 3 weeks to produce enough skin to cover an extensive burn or large wound. Another disadvantage is that the scientists must perform much of the process manually.

3D printing is similar to a desktop computer printer except that the nozzle on the 3D printer exudes biological components rather than ink. These biological components, or bio-inks, are essential to successful 3D printing of human organs and tissues.

To aid in the process, a computer controls the nozzles and flow of biological components so that the nozzles deposit the bio-inks on precise locations on the print bed.

Prints large area of skin in 35 minutes

The authors of the study describe how their 3D printing method generated a 100 x 100 centimeter area of skin in just under 35 minutes.

Like the scientists’ existing plasma-based manual method of producing skin, the 3D printing technology generates two layers of skin – the epidermis and the dermis. The printer starts by producing the epidermis, including the protective outermost layer of keratinized cells, known as the stratum corneum. Next, it prints the thicker, deeper dermis, complete with collagen-producing frank-magliochetti_bioprinter-skinfibroblasts.

The new 3D printing method is faster, but still complex. One of the authors of the report, Juan Francisco del Cañizo of the Hospital General Universitario Gregorio Marañón and Universidad Complutense de Madrid, notes:

“Knowing how to mix the biological components, in what conditions to work with them so that the cells don’t deteriorate, and how to correctly deposit the product is critical to the system.”

The research team tested the printed skin in test tubes and in immunodeficient mice. Transplantation of the printed skin into the mice helped the scientists test the long-term effects in a living animal. In both tests, the 3D printed skin was very similar to human skin and indistinguishable from the manually produced bi-layered skin from plasma.

There are two main potential uses for this 3D skin – to produce skin for research and laboratory testing of drugs and cosmetics, and to produce person-specific skin from the patient’s own cells to treat burns and other wounds. The research team is also investigating ways to use the technology to print other human tissues.

Source

http://iopscience.iop.org/article/10.1088/1758-5090/9/1/015006/meta

Frank Magliochetti is Managing Partner for Parcae Capital

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release new sites dedicated to the industry. Frank currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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Telemedicine Compared to Bedside Assessment

Telemedicine for Assessing Levels of Consciousness in Comatose Patients: How Does it Compare to Bedside Assessment?

Effective care for comatose patients in intensive care units (ICUs) depends on proper intervention based on reliable assessment. Researchers recently conducted a study at Mayo Clinic Hospital in Arizona to compare the effectiveness of using telemedicine to assess levels of consciousness in comatose patients with standard bedside assessment.

Proper intervention relies on the ability to recognize changes in a comatose patient’s clinical status quickly. This had usually meant that, in order to complete an assessment, the practitioner needed to be in the same room. Advanced medical technology is changing all that and robotic medicine now allows clinicians to assess patients from across the hospital or from across the world.frankmagliochetti_Telemedice_HealthcareTrends-Innovations

Telemedicine has been around since the 1960s, when NASA built telemedicine technology into astronauts’ suits. Prior to this technology, astronauts had to rely on crewmates for an accurate diagnosis. Monitors in the suits sent biometric information about the wearer back to earth for assessment.

Computers have revolutionized telemedicine and the internet helps doctors assess patients living in remote places. This is especially helpful for patients living in underserved areas.

Despite major advances, many still worry about the effectiveness in using this technology for the most critically ill patients. A new study published in Telemedicine and e-Health should help to dispel this fear, with researchers showing that robotic telemedicine can be used successfully to complete assessments in comatose ICU patients.

Testing the Reliability of Telemedicine in the Assessment of Levels of Consciousness

Researchers enrolled 100 patients from Mayo Clinic Hospital in Arizona into the study, which occurred over a 15-month timeframe. Mean age of patient participants was 70.8 years. On average, each examination took just over 5 minutes.

Sixteen medical doctors also participated by using two scoring systems, the Glasgow Coma Scale (GCS) and the Full Outline of UnResponsiveness (FOUR) score, to assess patients’ levels of consciousness. The researchers randomly assigned two practitioners to each patient; one doctor used real-time audio and a visual robotic telemedicine system to perform the assessment and the other clinician conducted an assessment at the patient’s bedside. Each used GCS and FOUR scales.

The researchers used paired t-test and Pearson correlation coefficient (PCC) to compare the GCS and FOUR scores between bedside and Telemedicine_FrankMAgliochetti-HealthcareReportremote physician.

Differences in GCS and FOUR scores between remote and beside assessment were small. The mean Glasgow Coma Scale score at bedside was 7.5 while the mean GCS score for the remote examination was 7.23. Scores were comparable in the FOUR total scores too, with a mean bedside score of 9.63 and a mean remote score of 9.21.

The researchers also asked the clinicians about their overall satisfaction and ease of use. Ninety-five percent of remote providers rated GCS and 89% rated FOUR score as good (4/5).

Conclusions

The study is the first to evaluate the effectiveness of telemedicine in assessing patients with depressed levels of consciousness. The results suggest that doctors can reliably assess levels of consciousness in comatose patients using existing robotic telemedicine technology. Healthcare providers could adopt telemedicine to help evaluate critically ill patients in neurologically underserved areas.

“This is good news in many ways,” states lead author of the study, Amelia Adcock, M.D, in a press release issued by Mayo Clinic. “We use telemedicine frequently when evaluating acute stroke patients. This study suggests yet another way telemedicine can enhance patient care. There is a shortage of intensive care unit providers and facilities with round-the-clock patient coverage. Telemedicine can provide a way to ameliorate this shortage and improve early evaluation of critically ill patients.”

Source

http://online.liebertpub.com/doi/10.1089/tmj.2016.0225

Frank Magliochetti is Managing Partner for Parcae Capital

  • North Andover, Massachusetts

This column of posts is directed at the Healthcare Industry.  Frank plans to release new sites dedicated to the industry. Frank currently assists companies who are building, restructuring, transforming and resurrecting there business’s. An example of his client base are, Xenetic Biosciences , IPC Medical Corp, Just Fellowship Corp, Environmental Services Inc., Parsons Post House LLC, ClickStream Corporation as well as having a business talk radio show; The Business Architect on the URBN network.

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