Ori doubles down on Charles River collaboration with promising new data on its automated cell therapy platform FiercePharma
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Ori doubles down on Charles River collaboration with promising new data on its automated cell therapy platform - FiercePharma
Doctors cured her sickle-cell disease. So why is she still in pain? Nature.com
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Doctors cured her sickle-cell disease. So why is she still in pain? - Nature.com
Gene Therapy Company Increases Focus on Mesothelioma Program Mesothelioma.net Blog
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Gene Therapy Company Increases Focus on Mesothelioma Program - Mesothelioma.net Blog
Sickle cell gene therapies roll out slowly : Shots - Health News NPR
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Sickle cell gene therapies roll out slowly : Shots - Health News - NPR
Patients At Last Begin Receiving Vertex-CRISPR and Bluebird Sickle Cell Gene Therapies BioSpace
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Patients At Last Begin Receiving Vertex-CRISPR and Bluebird Sickle Cell Gene Therapies - BioSpace
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Beacon Therapeutics Presents 36-Month Interim Results from Phase I/2 HORIZON Trial of AGTC-501 in Patients with XLRP - PR Newswire
Beacons Gene Therapy Shows Continued Promise in Trial TipRanks
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Beacons Gene Therapy Shows Continued Promise in Trial - TipRanks
How stem cell and gene therapies are revolutionising healthcare Express Healthcare
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How stem cell and gene therapies are revolutionising healthcare - Express Healthcare
Nanoscope Therapeutics to be Featured at Annual EUretina Congress in Barcelona PR Newswire
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Nanoscope Therapeutics to be Featured at Annual EUretina Congress in Barcelona - PR Newswire
6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company WCYB
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6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company - WCYB
Seeking a sickle cell cure: 12-year-old in DC is 1st patient in US to get new gene therapy NBC Washington
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Seeking a sickle cell cure: 12-year-old in DC is 1st patient in US to get new gene therapy - NBC Washington
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Game-changer: The Hindu Editorial on approval for gene therapy to treat sickle cell disease and beta thalassemia - The Hindu
Early trials show promise for innovative gene therapy in lung cancer treatment WJAR
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Early trials show promise for innovative gene therapy in lung cancer treatment - WJAR
Cell and Gene Therapy Manufacturing Quality Control Market Growing Trends and Technology Forecast to 2029 |... SeeDance News
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Cell and Gene Therapy Manufacturing Quality Control Market Growing Trends and Technology Forecast to 2029 |... - SeeDance News
Espaol
The genes in your bodys cells play a key role in your health. Indeed, a defective gene or genes can make you sick.
Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition.
This research is paying off, as advancements in science and technology today are changing the way we define disease, develop drugs, and prescribe treatments.
The U.S. Food and Drug Administration has approved multiple gene therapy products for cancer and rare disease indications.
Genes and cells are intimately related. Within the cells of our bodies, there are thousands of genes that provide the information to produce specific proteins that help make up the cells. Cells are the basic building blocks of all living things; the human body is composed of trillions of them.
The genes provide the information that makes different cells do different things. Groups of many cells make up the tissues and organs of the body, including muscles, bones, and blood. The tissues and organs in turn support all our bodys functions.
Sometimes the whole or part of a gene is defective or missing from birth. This is typically referred to as a genetically inherited mutation.
In addition, healthy genes can change (mutate) over the course of our lives. These acquired mutations can be caused by environmental exposures. The good news is that most of these genetic changes (mutations) do not cause disease. But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.
Depending on what is wrong, scientists can do one of several things in gene therapy:
To insert new genes directly into cells, scientists use a vehicle called a vector. Vectors are genetically engineered to deliver the necessary genes for treating the disease.
Vectors need to be able to efficiently deliver genetic material into cells, and there are different kinds of vectors. Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.
Gene therapy can be used to modify cells inside or outside the body.When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient.
When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. The vector containing the desired gene is introduced into these cells. The cells are later injected into the patient, where the new gene is used to produce the desired effect.
Before a gene therapy can be marketed for use in humans, the product must be tested in clinical studies for safety and effectiveness so FDA scientists can consider whether the risks of the therapy are acceptable considering the potential benefits.
The scientific field for gene therapy products is fast-paced and rapidly evolving ushering in a new approach to the treatment of vision loss, cancer, and other serious and rare diseases. As scientists continue to make great strides in this therapy, the FDA is committed to helping speed up development by interacting with those developing products and through prompt review of groundbreaking treatments that have the potential to save lives.
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How Gene Therapy Can Cure or Treat Diseases | FDA
Genetic therapies aim to treat or cure conditions by correcting problems in your DNA. Your DNA, including specific genes, contains instructions for making proteins that are essential for good health. Mutations, or changes in your DNA, can lead to proteins that do not work properly or that are missing altogether. These changes can cause genetic, or inherited, disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease.
Genetic therapies are approaches that treat genetic disorders by providing new DNA to certain cells or correcting the DNA. Gene transfer approaches, also called gene addition, restore the missing function of a faulty or missing gene by adding a new gene to affected cells. The new gene may be a normal version of the faulty gene or a different gene that bypasses the problem and improves the way the cell works.
Genome editing is a newer approach that allows precise correction or other targeted changes to the DNA in cells to restore a cells function. Genome editing can:
Gene transfer or genome editing treatments can directly modify the cells in your body, or your cells can be collected and treated outside of your body and then returned to you. For example, a doctor can remove immune system cells, cells that are part of your bodys natural defense system, or bone marrow cells from your body, modify their DNA, and then re-introduce them to your body.
The only genetic therapies that are currently approved by the U.S. Food and Drug Administration (FDA) are for a rare inherited eye condition, as well as certain types of cancer. Genetic therapies that are in development could treat or cure other inherited disorders; treat other cancers; or treat infections, including HIV.
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Genetic Therapies - What Are Genetic Therapies? | NHLBI, NIH
For Immediate Release: December 18, 2017
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The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.
Todays approval marks another first in the field of gene therapy both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses, said FDA Commissioner Scott Gottlieb, M.D. Were at a turning point when it comes to this novel form of therapy and at the FDA, were focused on establishing the right policy framework to capitalize on this scientific opening. Next year, well begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters including new clinical measures for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.
Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the U.S. Biallelic mutation carriers have a mutation (not necessarily the same mutation) in both copies of a particular gene (a paternal and a maternal mutation). The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.
Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patients vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.
The approval of Luxturna further opens the door to the potential of gene therapies, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.
Luxturna should be given only to patients who have viable retinal cells as determined by the treating physician(s). Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.
The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subjects ability to navigate an obstacle course at various light levels. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.
The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.
The FDA granted this application Priority Review and Breakthrough Therapy designations. Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The sponsor is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.
To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.
The FDA granted approval of Luxturna to Spark Therapeutics Inc. The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene
Andrea Fischer301-796-0393
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FDA approves novel gene therapy to treat patients with a rare form of ...
Ever since the dawn of mankind, diseases have plagued humans over the ages. Years of innovations and advancements in science has provided us with a deeper understanding of how diseases work. This has led to lower mortality rates and longer lifespans. But there are some diseases that just cannot be cured using traditional medicine or surgery. Gene therapy is an experimental technique that caters to patients with such diseases.
Gene therapy is a technique which involves the replacement of defective genes with healthy ones in order to treat genetic disorders. It is an artificial method that introduces DNA into the cells of the human body. The first gene therapy was successfully accomplished in the year 1989.
The simple process of gene therapy is shown in the figure below:
In the figure, the cell with the defective gene is injected with a normal gene which helps in the normal functioning of the cell. This technique is employed mainly to fight against the diseases in the human body and also to treat genetic disorders. The damaged proteins are replaced in the cell by the insertion of DNA into that cell. Generally, improper protein production in the cell leads to diseases. These diseases are treated using a gene therapy technique. For example, cancer cells contain faulty cells which are different from the normal cells and have defective proteins. Hence, if these proteins are not replaced, this disease would prove to be fatal.
Basically, there are two types of gene therapy
This type usually occurs in the somatic cells of human body. This is related to a single person and the only person who has the damaged cells will be replaced with healthy cells. In this method, therapeutic genes are transferred into the somatic cells or the stem cells of the human body. This technique is considered as the best and safest method of gene therapy.
It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes which are passed from the parents to their children. The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms. Germline gene therapy is not legal in many places as the risks outweigh the rewards.
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IVERIC bio Subsidiary Sells Assets of Gene Therapy Product Candidates for Treatment of Retinal Diseases Marketscreener.com
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IVERIC bio Subsidiary Sells Assets of Gene Therapy Product Candidates for Treatment of Retinal Diseases - Marketscreener.com