November 22, 2017

Genome Editing – 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 plans to release 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.

frankmagliochetti_ParcaeCapital

Genome Editing – CRISPR

Frank Magliochetti Report:   CRISPR

CRISPR trials just may be one the most important healthcare industry breakthroughs of this generation

Frank Magliochetti

Almost every year, a new disease is introduced in the world which affects the population of our country. It would be wrong to say that people living in rural areas may be more prone to catch diseases than those who are living in the city. The fact is that no matter where you live or how Frank-Magliochetti_crisprhard you try to protect yourself from the outside world, there are some in-house diseases that may catch up to you.

Once such disease that can strike anyone at any time is cancer which arises through our genetics.   A disease that in some cases runs down from generation to generation which may strike no matter what we may do to help prevent it.  It is something we all dread.

A study conducted on genetic based cancer diseases was conducted in the US and finally was granted access for testing on humans on June 21st.   The NIH (National Institute of Health) gave the green light to start testing the CRISPR-Cas9 trial on humans so that help from T Cells could be enlisted to protect the body.

To understand the trial, first, one needs to understand what exactly CRISPR-Cas9 is. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a drug derived from Streptococcus pyogenes, which enabled the researchers to make permanent mutations. This introduces double stranded healing breaks in cells to activate the repair pathways in a human body. They adapt immunity in certain archaea and bacteria, which allow organisms to invade and eliminate genetic material.

This disease was discovered in 1989 but until 2007 its symptoms were unknown. 2016 has brought new hope for people to live a healthy life. The trial will be funded by Sean Parker, a tech entrepreneur who has a $250 million dollar cancer treatment center; The Parker Institute for Cancer Immunotherapy.

There have been other trials conducted but the problem with them was that the T Cells, which were reintroduced to a person’s body were not that effective, however, they did kill the NY-ESO-1, the T Cells stopped proliferating and became less effective. crispr-magliochetti

There are many hospitals all over the world that are holding CRISPR trails. Some trials were started at the beginning of August while others are set to start in September.

Hospitals such as Massachusetts General Hospital located in Boston have been conducting tests on genome editing since 2013. This is one of the oldest hospitals in Boston and gets close to 100,000 patient visits per year. There are even chances that MGH affiliated laboratories will also conduct genome editing trials.

Many more hospitals based in Boston have announced that with proper funding, they will start genome editing trials in 2017. Since the trials are so expensive, scientists will pick patients who are immune to Cas9 enzyme.

The discovery and trials of CRISPR is one of the best advancements in medical history. Though the patients for the trial will be chosen in small numbers, people living in fear that their life may end after four or five years due to a genetic illness like Leukemia may now have new hope of living a healthy and happy life.

The content below was aggregated from a Boston Globe opinion piece by Jim Kozubek please click the link to head to the official site to read the rest of the post.

https://www.bostonglobe.com/ideas/2016/09/03/crispr-and-capitalists/uX8NuPsva5L2iNI2DCBArJ/story.html

CRISPR and the capitalists

CRISPER-CAS9 IS A method of manipulating DNA that is transforming medicine and science. It is both popular (“molecule of the year!”) and dangerous (researchers have received threats from GMO activists). It is also wildly lucrative.

The first clinical trial of these tiny molecular scissors may begin by the end of the year. Meanwhile, two camps, the Broad Institute — where I held affiliation for nearly three years — and University of California at Berkeley and researcher Emmanuelle Charpentier, are locked in a patent showdown that will play out in early 2017 with perhaps a billion dollars at stake.
Society treats science as a public trust by funding it through taxes, but perhaps it shouldn’t do so indefinitely. One of the subliminal things that the CRISPR-Cas9 legal showdown tells us is that academic-industry partnerships may one day be mature enough that science may begin to forgo its tax base.

In 1943, President Franklin D. Roosevelt directed his wartime chief of research, Vannevar Bush, to find a way to extend funding after the war. In a document with soaring language entitled “Science the Endless Frontier,” Bush laid out the argument for robust federal funding engines through the National Institutes of Health, the creation of the National Science Foundation, and what would become a briskly expanding network of research scientists throughout the United States.
Read More – CLICK HERE

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 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.

frankmagliochetti_ParcaeCapital