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Gene Therapies Target Defective Genes/CRISPR Advances DNA Editing, Business and Industry Trends Analysis

A major goal of DNA research and the sequencing of the genetic profiles of patients is to attack, and attempt to alter, specific defective genes within the patient.  This practice is known as gene therapy, a strategy that targets defective genes (mutations) within a patient by introducing new copies of normal genes.  These new, normal genes may be introduced through the use of viruses or proteins that carry them into the patient’s body.

Internet Research Tip: Gene Therapies
A “gene therapy” is based on first identifying the fact that a patient has a specific gene mutation related to a specific disease, followed by the introduction of healthy genes into the patient’s body with the goal of altering or replacing the defective genes.  This is the Holy Grail of the biotechnology industry, as it offers the potential to cure otherwise incurable diseases.  It has also proven to be extremely difficult to carry out without significant side effects.  The American Society of Gene & Cell Therapy,, publishes extremely useful data for those researching the promise and current status of gene therapies.  Their online tools include data on clinical trials, a glossary and a list of “breakthroughs.”  The group also publishes an extremely useful newsletter.

     A major commercial hurdle was passed in late 2012, when the European Medicines Agency (EMA) approved a gene therapy to treat a rare lipoprotein lipase (LPL) deficiency.  The therapy, called Glybera, developed by Dutch biotech company uniQure, was the first approved gene therapy drug in the Western world.  One of the biggest concerns with the use of gene therapies is potential side effects.  Glybera so far appears to help patients who have specific mutations in the LPL gene, without complications from side effects.  However, patients may be required to take immunosuppressive drugs in order to use Glybera, and those drugs may cause side effects.
Glybera was commercially launched in Europe by uniQure and pharma company Chiesi Farmaceutici in early 2015.  Unfortunately, the cost for the drug is $1.4 million for a full course of treatment.  The firm announced that it would not pursue the renewal of the drug’s marketing authorization in Europe when it expired in 2017.  uniQure had one other gene therapy (a treatment for Hemophilia B) in clinical trials as of mid-2017, and four others in the pipeline.  These include treatments for Huntington’s Disease and congestive heart failure.
Recent news includes the 2022 FDA approvals of Bluebird’s $2.8 million per-patient gene therapy for a rare blood disorder, and CSL Ltd.’s $3.5 million per-patient Hemgenix to treat hemophilia B.  In 2023, approval was granted for Elevidys, a $3.2 million gene therapy for muscular dystrophy made by Sarepta Therapeutics.  The FDA also recently approved a systemic gene therapy, a treatment for spinal muscular atrophy called Zolgensma developed by Novartis (the Swiss drug firm acquired AveXis, Inc., which first developed the drug but had yet to bring it to market, for $8.7 billion in 2018).  Zolgensma is a one-time treatment for a traumatic infant muscle-wasting disease that affects one in every 6,000 to 10,000 babies worldwide.  The cost of the treatment is a staggering $2.125 million.  Novartis offers outcomes-based pricing on some drugs, in which a portion of the cost is refunded if the drug proves ineffective.  It also offers insurers the option to pay in five equal installments over five years.  Another gene therapy, Spark Therapeutics’ Luxturna for the treatment a hereditary childhood disease that causes blindness (which affects an estimated 3,500 people in the U.S. and five European countries), is $850,000 per dose.
Skyrocketing numbers of applications of gene therapy are in research in the U.S. and elsewhere for treatment of a wide variety of diseases.  Gene therapy may be highly effective in the treatment of rare immune system disorders, melanoma, Parkinson’s and cystic fibrosis.  Development of gene therapies is flourishing partly due to the “expanded access” program under the FDA which provides access to investigational drugs, biologics and medical devices for critically ill patients who are not treatable by drugs already approved and on the market.
Gene therapy is typically limited to single-treatment protocols.  A number of startups, including Ring Therapeutics (, are working with human viruses to enable repeated gene therapies.
Gene therapy is also a promising treatment for certain cancers.  In 2017, the FDA approved two CAR T-cell therapies for blood cancers.  The therapy re-engineers white blood cells to fight an antigen found in malignant cells and has fewer side effects than traditional therapies.  In November 2018, the FDA approved larotrectinib (marketed by Bayer under the name Vitrakvi), which treats malignant tumors based on genetic characteristics as opposed to their point of origin in the body.
Another breakthrough in gene therapy is CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats).  The technology focuses on the use of a DNA-cutting protein that is guided by an RNA molecule, targeted at a specific gene.  This technology enables a scientist to quickly and easily edit or re-engineer specific bits of DNA.  A defective gene can be precisely edited within the laboratory, and then reintroduced to a patient's body as a form of gene therapy with far more accuracy than previous gene therapies.  CRISPR is sometimes referred to as “genetic editing,” and it is considered to be a very significant breakthrough.  Practical applications may include treatments or even cures for sickle-cell anemia, HIV and cystic fibrosis.  Libraries of CRISPRs have been created by researchers at MIT that account for nearly all human genes.  
First demonstrated in 2012, CRISPR technologies are being used in therapies in early clinical trials for the treatment of certain cancers.  Research team at the Perelman School of Medicine at the University of Pennsylvania is working to alter the genes of ants, thereby changing their social behavior, hoping to correlate those changes to the social behavior of humans.
An experimental therapy developed by bluebird bio, Inc. to treat an inherited blood disorder called betathalassemia showed promise in a 2017 trial.  15 of 22 patients who previously needed regular blood transfusions were transfusion-free after a median period of 26 months.  The remaining seven patients needed fewer transfusions.  By 2019, the therapy, called LentiGlobin, was entering Phase 3 studies.  However, in early 2021, the FDA put LentiGlobin on clinical hold after two sickle cell disease patients developed acute myeloid leukemia and myelodysplastic syndrome respectively.  Clinical trials resumed by mid-2021.
CRISPR startups include Intellia Therapeutics (, Editas Medicine ( and CRISPR Therapeutics (  The recent success of gene therapies is helping to fuel a global surge in biotech investment, including venture capital, partnerships and outright acquisitions of promising biotech companies.  CRISPR is also being utilized in the development of modified agricultural seeds and lab-grown meats.

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