Can CRISPR Curb Sickle Cell?

CRISPR Has the Potential to Snip Out Sickle Cell Disease

Sickle cell disease affects about 100,000 people in the United States, according to the Centers for Disease Control and Prevention (CDC), and affects millions of people across the globe. A new technology, known as CRISPR, may change all that.

CRISPR is short for “clustered regularly interspaced short palindromic repeats.” It is a group of technologies capable of editing the genes in people with inherited conditions, such as sickle cell disease.

Sickle cell disease is an inherited disorder that affects red blood cells, which transport oxygen to the rest of the body. Specifically, sickle cell disorders affect hemoglobin (Hgb), which is the protein in red blood cells responsible for transporting oxygen. A mutation in a single DNA letter (S) causes the sickle cell trait to be passed from one generation to the next. People with sickle cell disorders inherit an abnormal version of hemoglobin, known as Hgb S, which distorts the shape of the red blood cells.

Red blood cells normally have a round donut shape that allows them to carry an ample supply of oxygen, and to flow through tiny blood vessels smoothly. People with sickle cell inherit a trait that, during a sickle cell crisis, causes the normally round blood cells to resemble the C-shaped farm tool known as a sickle. The sickle cells become hard and sticky, so they clump together instead of flowing freely.

The cells are fragile and prone to rupturing, which can lead to anemia. The deformed cells also die early, which causes a constant shortage of red blood cells. The abnormal shape also means the cells can block blood vessels and damage tissue. This can cause pain, infections, a lung problem known as acute chest syndrome, stroke and other serious health issues during a sickle cell crisis and afterwards.

Current treatments involve blood transfusions, the drug hydroxyurea and bone marrow transplants. Each of these comes with risks and complications.


CRISPR is a group of gene editing technologies that allow scientists to change an organisms DNA by adding, removing or altering specific locations within the gene. Researchers created CRISPR by adapting a naturally occurring gene editing system in bacteria, which captures little snippets of an invading virus’s genes. If the virus ever attacks again, the bacteria use the snippets to create and insert a new DNA sequence into the virus, which effectively changes the virus.

The technology works the same in the lab, except to produce positive results. Scientists first remove the snippet of the “bad” gene that causes sickle cell, using CRISPR to cut the sickle cell gene (S) from a precise location in DNA, and replaces it with healthy genes. Scientists then attach healthy hemoglobin genes to a harmless virus, and then put the virus and the corrected genes it carries back in the patient’s body.

Researchers from the National Institutes of Health performed a clinical trial in which they used CRISPR to edit the genes of nine people with sickle cell disease. The lead researcher, John Tisdale, spoke about their progress and said that all of the people who had received the gene therapy had good hemoglobin levels and that none of the participants had experienced sickle cell crises.

More research is necessary before gene editing becomes a common course of treatment, but CRISPR may someday help all people overcome sickle cell disease and its complications.

Frank Magliochetti owes his professional success to his expertise in two areas: medicine and finance. After obtaining a BS in pharmacy from Northeastern University, he stayed on to enroll in the Masters of Toxicology program. He later specialized in corporate finance, receiving an MBA from The Sawyer School of Business at Suffolk University. His educational background includes completion of the Advanced Management Program at Harvard Business School and the General Management Program at Stanford Business School. Frank Magliochetti has held senior positions at Baxter International, Kontron Instruments, Haemonetics Corporation, and Sandoz. Since 2000, he has been a managing partner at Parcae Capital, where he focuses on financial restructuring and interim management services for companies in the healthcare, media, and alternative energy industries. Earlier this year, he was appointed chairman of the board at Grace Health Technology, a company providing an enterprise solution for the laboratory environment.

Mr. Frank Magliochetti MBA
Managing Partner
Parcae Capital

Genome Editing – CRISPR

CRISPR- Genome Editing:

Frank Magliochetti Report:   CRISPR

CRISPR trials just may be one the most important healthcare industry breakthroughs of this generation.  How these breakthroughs affect the overall picture of bio/pharma will be interesting to stay focused on.

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.

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.
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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.