The idea for gene therapya type of DNA-based medicine that inserts a healthy gene into cells to replace a mutated, disease-causing variantwas first published in 1972. After decades of disputed results, treatment failures and some deaths in experimental trials, the first gene therapy drug, for a type of skin cancer, was approved in China in 2003. The rest of the world was not easily convinced of the benefits, however, and it was not until 2017 that the U.S. approved one of these medicines. Since then, the pace of approvals has accelerated quickly. At least nine gene therapies have been approved for certain kinds of cancer, some viral infections and a few inherited disorders. A related drug type interferes with faulty genes by using stretches of DNA or RNA to hinder their workings. After nearly half a century, the concept of genetic medicine has become a reality.
These treatments use a harmless virus to carry a good gene into cells, where the virus inserts it into the existing genome, canceling the effects of harmful mutations in another gene.
GENDICINE:Chinas regulatory agency approved the worlds first commercially available gene therapy in 2003 to treat head and neck squamous cell carcinoma, a form of skin cancer. Gendicine is a virus engineered to carry a gene that has instructions for making a tumor-fighting protein. The virus introduces the gene into tumor cells, causing them to increase the expression of tumor-suppressing genes and immune response factors.The drug is still awaiting FDA approval.
GLYBERA:The first gene therapy to be approved in the European Union treated lipoprotein lipase deficiency (LPLD), a rare inherited disorder that can cause severe pancreatitis. The drug inserted the gene for lipoprotein lipase into muscle cells. But because LPLD occurs in so few patients, the drug was unprofitable. By 2017 its manufacturer declined to renew its marketing authorization; Glybera is no longer on the market.
IMLYGIC:The drug was approved in China, the U.S. and the E.U. to treat melanoma in patients who have recurring skin lesions following initial surgery. Imlygic is a modified genetic therapy inserted directly into tumors with a viral vector, where the gene replicates and produces a protein that stimulates an immune response to kill cancer cells.
KYMRIAH:Developed for patients with B cell lymphoblastic leukemia, a type of cancer that affects white blood cells in children and young adults, Kymriah was approved by the FDA in 2017 and the E.U. in 2018. It works by introducing a new gene into a patients own T cells that enables them to find and kill cancer cells.
LUXTURNA:The drug was approved by the FDA in 2017 and in the E.U. in 2018 to treat patients with a rare form of inherited blindness called biallelic RPE65 mutation-associated retinal dystrophy. The disease affects between 1,000 and 2,000 patients in the U.S. who have a mutation in both copies of a particular gene, RPE65. Luxturna delivers a normal copy of RPE65 to patients retinal cells, allowing them to make a protein necessary for converting light to electrical signals and restoring their vision.
STRIMVELIS:About 15 patients are diagnosed in Europe every year with severe immunodeficiency from a rare inherited condition called adenosine deaminase deficiency (ADA-SCID). These patients bodies cannot make the ADA enzyme, which is vital for healthy white blood cells. Strimvelis, approved in the E.U. in 2016, works by introducing the gene responsible for producing ADA into stem cells taken from the patients own marrow. The cells are then reintroduced into the patients bloodstream, where they are transported to the bone marrow and begin producing normal white blood cells that can produce ADA.
YESCARTA:Developed to treat a cancer called large B cell lymphoma, Yescarta was approved by the FDA in 2017 and in the E.U. in 2018. It is in clinical trials in China. Large B cell lymphoma affects white blood cells called lymphocytes. The treatment, part of an approach known as CAR-T cell therapy, uses a virus to insert a gene that codes for proteins called chimeric antigen receptors (CARs) into a patients T cells. When these cells are reintroduced into the patients body, the CARs allow them to attach to and kill cancer cells in the bloodstream.
ZOLGENSMA:In May 2019 the FDA approved Zolgensma for children younger than two years with spinal muscular atrophy, a neuromuscular disorder that affects about one in 10,000 people worldwide. It is one of the leading genetic causes of infant mortality. Zolgensma delivers a healthy copy of the human SMN gene to a patients motor neurons in a single treatment.
ZYNTEGLO:Granted approval in the E.U. in May 2019, Zynteglo treats a blood disorder called beta thalassemia that reduces a patients ability to produce hemoglobin, the protein in red blood cells that contains iron, leading to life-threatening anemia. The therapy has been approved for individuals 12 years and older who require regular blood transfusions. It employs a virus to introduce healthy copies of the gene for making hemoglobin into stem cells taken from the patient.The cells are then reintroduced into the bloodstream and transported to the bone marrow, where they begin producing healthy red blood cells that can manufacture hemoglobin.
This approach uses a synthetic strand of RNA or DNA (called an oligonucleotide) that, when introduced into a patients cell, can attach to a specific gene or its messenger molecules, effectively inactivating them. Some treatments use an antisense method, named for one DNA strand, and others rely on small interfering RNA strands, which stop instruction molecules that go from the gene to the cells protein factories.
DEFITELIO:This drug contains a mixture of single-strand oligonucleotides obtained from the intestinal mucosa of pigs. It was approved (with limitations) in the U.S. and the E.U. in 2017 to treat severe cases of veno-occlusive disease, a disorder in which the small veins of the liver become obstructed, in patients who have received a bone marrow transplant.
EXONDYS 51:In 2016 the FDA granted approval to Exondys 51 amid some controversy regarding its efficacy; two members of the FDA review panel resigned in protest of the decision. The therapy is designed to treat a form of Duchenne muscular dystrophy caused by mutations in the RNA that codes for the protein that helps to connect muscle fibers cytoskeletons to a surrounding matrix. Exondys 51 is effective in treating about 13 percent of the Duchenne population.
KYNAMRO:Approved by the FDA in in 2013, Kynamro is designed to inhibitor effectively shut down production ofa protein that helps to produce low-density lipoprotein (LDL). Injected subcutaneously, this therapy is used to lower LDL levels in patients who have dangerously high cholesterol.
MACUGEN:Age-related macular degeneration is the leading cause of vision loss in people age 60 and older. It is caused by deterioration of the center of the retina due to leaking blood vessels. Approved in the U.S., Macugen inhibits these blood vessels from growing under the retina, thus treating the disorder.
SPINRAZA:With its FDA approval in 2016, Spinraza became the first gene-based therapy for spinal muscular atrophy. The inherited disorder is caused by low levels of SMN, a key protein for the maintenance of motor neurons. Spinraza binds to RNA from a backup gene called SMN2, converting that RNA into instructions for making fully functioning SMN proteins.
- Genetic Medicine | Internal Medicine | Michigan Medicine ... - March 26th, 2020
- Genomics and Medicine | NHGRI - March 26th, 2020
- Q&A:Transforming genetic medicine as the medical standard of care (Includes interview) - Digital Journal - March 26th, 2020
- Scientists Discover That a Squid Can Edit Its Own Genetic Code - Futurism - March 26th, 2020
- Patients with Severe Forms of Coronavirus Disease Could Offer Clues to Treatment - Howard Hughes Medical Institute - March 26th, 2020
- Bridging the gap study sequences Asian genomes to diversify genetic databases - University of Virginia The Cavalier Daily - March 26th, 2020
- Precision Medicine, Nanotechnology and the Rise of the Robot Now. Powered by - Now. Powered by Northrop Grumman. - March 26th, 2020
- Genomic assays: on the brink of revolutionising human healthcare - Drug Target Review - March 26th, 2020
- PA reactivating retired healthcare providers' licenses to treat COVID-19 patients - FOX43.com - March 26th, 2020
- How the Pandemic Will End - The Atlantic - March 26th, 2020
- You can't kill coronavirus. That's OK. - Mashable - March 26th, 2020
- 10 Groundbreaking Medical Discoveries Made in NYC - Untapped New York - Untapped New York - March 26th, 2020
- Coronavirus vaccine: when will it be ready? - The Guardian - March 26th, 2020
- Twelve Women Who Have Shaped The History of the BioHealth Capital Region - BioBuzz - March 26th, 2020
- Alnylam Pharmaceuticals and Gen Sign Distribution Agreement in Turkey for ONPATTRO (patisiran), the First-in-Class Gene-Silencing RNAi Therapeutic -... - March 26th, 2020
- The Harvard Wyss Institute's response to COVID-19: beating back the coronavirus - P&T Community - March 26th, 2020
- Football: Ryan Day and staff transitioning to work-from-home mode - OSU - The Lantern - March 26th, 2020
- How long will we have to wait for a coronavirus vaccine? - Telegraph.co.uk - March 26th, 2020
- Understanding SARS-CoV-2 and the drugs that might lessen its power - The Economist - March 16th, 2020
- Marine medicine: Understanding and treating infection types - National Fisherman - March 16th, 2020
- Rapid genetic testing becomes available to Calgary medical community - CTV News - March 16th, 2020
- Coronavirus vaccines and treatment: Everything you need to know - CNET - March 16th, 2020
- Researchers Predict Potential Spread And Seasonality For COVID-19 Based On Climate Where Virus Appears To Thrive - Herald-Mail Media - March 16th, 2020
- On the front lines of developing a test for the coronavirus - Jacksonville Journal-Courier - March 16th, 2020
- Race Is Real, But It's Not Genetic - SAPIENS - March 16th, 2020
- Genomics took a long time to fulfil its promise - The Economist - March 16th, 2020
- Why There Aren't Enough Coronavirus Tests in the U.S. - Popular Mechanics - March 16th, 2020
- UPMC ready to test for coronavirus; Collection site to open in Williamsport | News, Sports, Jobs - Lock Haven Express - March 16th, 2020
- Two Women Fell Sick From the Coronavirus. One Survived. - The New York Times - March 16th, 2020
- The New Game of Microbiology Clue: The Who, When, Where, and Why of the Novel Coronavirus - Psychiatric Times - March 16th, 2020
- Cancer Biomarker Market investigated in the latest research - WhaTech Technology and Markets News - March 16th, 2020
- GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome - BioSpace - March 16th, 2020
- GW Pharmaceuticals submits Type II Variation Application to the European Medicines Agency (EMA) to expand the use of EPIDYOLEX, (cannabidiol) oral... - March 16th, 2020
- Alzheimer's Brains Short Circuited by Defective Protein Connections - Genetic Engineering & Biotechnology News - March 16th, 2020
- New Study Shows Combining TERT Mutation Analyses With DermTech's PLA Provides Improved Sensitivity for Detecting Melanoma With Non-invasive Patches -... - March 16th, 2020
- Medical genetics - Wikipedia - January 10th, 2020
- Genetic Medicine | Department of Medicine - January 10th, 2020
- Distribution of Genes Encoding Virulence Factors and the Genetic Diver | IDR - Dove Medical Press - January 10th, 2020
- In defence of imprecise medicine: the benefits of routine treatments for common diseases - The Conversation UK - January 10th, 2020
- IDEAYA Biosciences and Boston Children's Hospital Collaborate on Preclinical Evaluation of IDE196 for Sturge Weber Syndrome - a Rare Disease... - January 10th, 2020
- Study ties gene active in developing brain to autism - Spectrum - January 10th, 2020
- Faculty and alumni appointed to state medical boards - The South End - January 10th, 2020
- Why This Thematic Healthcare Could be a January Winner - ETF Trends - January 10th, 2020
- New year health kicks are great but your environment is also vital - The Guardian - January 10th, 2020
- Biofidelity and Agilent complete successful molecular assay study for rapid and accurate detection of key lung cancer mutations - BioSpace - January 10th, 2020
- New MD Treatments the Main Goal of Astellas, Audentes Merger - Muscular Dystrophy News - January 10th, 2020
- Physicians' Education Resource Presents the 2nd Annual Precision Medicine Symposium in New York City - BioSpace - January 10th, 2020
- A Genetic Mutation Is Responsible for Mysterious Deaths in the Amish Community, Researchers Say - Gizmodo - January 10th, 2020
- Kyoto Univ.-distributed iPS cells found with abnormalities after differentiation - The Mainichi - January 10th, 2020
- The Importance of Understanding TargetProtein Interactions in Drug Discovery - Technology Networks - January 10th, 2020
- Webinar: How Providers are Harnessing the Power of Genomics to Improve Community Health - ModernHealthcare.com - January 5th, 2020
- These 2 Stocks Will Fall After the New Year - Motley Fool - January 5th, 2020
- Free Gene Therapy Available for Patients with Alzheimer's - HealthITAnalytics.com - January 5th, 2020
- Chinese Researcher Who Created Gene-Edited Babies Sentenced To 3 Years In Prison - NPR - January 5th, 2020
- Duke Researchers Garner Over $6 Million in NIH Funding to Fight Genetic Diseases - Duke Today - January 5th, 2020
- Stanford Team Proposes Automated Clinical Trial Accrual Strategy, Increased Trial Annotation - Precision Oncology News - January 5th, 2020
- Dr. Timothy Eberlein and Alvin Siteman named Citizens of the Year 2019 - STLtoday.com - January 5th, 2020
- Did Cellectis Just Provide a Glimpse of the Future of Cellular Medicine? - The Motley Fool - December 27th, 2019
- Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Health News - NPR - December 27th, 2019
- Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy... - December 27th, 2019
- What is multifocal pneumonia, the illness ESPN reporter Edward Aschoff tweeted about before his death? - USA TODAY - December 27th, 2019
- The travellers within us - Myanmar Times - December 27th, 2019
- This Start-up Might Be the Next Gene Editing IPO - The Motley Fool - December 19th, 2019
- How to bring precision medicine into the doctor's office - World Economic Forum - December 19th, 2019
- Form of severe malnutrition linked to DNA modification - Baylor College of Medicine News - December 19th, 2019
- 'Polygenic' profile could better predict disease risk for those with cancer mutations - Science Magazine - December 19th, 2019
- Here's Why You Should Avoid Betting on RPC (RES) Stock Now - Nasdaq - December 19th, 2019
- UNC Police Investigating Series of Credit Card Thefts on South Campus - Chapelboro.com - December 19th, 2019
- Personalized CF Medicine to be Tested for Rare Genetic Defects in Europe - Cystic Fibrosis News Today - December 19th, 2019
- Has Innovative Industrial Properties (IIPR) Outpaced Other Finance Stocks This Year? - Nasdaq - December 19th, 2019
- BioReference Laboratories Showcases 2019 Growth through the Addition of Cutting Edge Tests, Greater Access to Services, and Optimized Patient... - December 19th, 2019
- Detection of Secondary Metabolites as Biomarkers for the Early Diagnos | DMSO - Dove Medical Press - December 18th, 2019
- Roche concludes acquisition of Spark Therapeutics, Inc. to strengthen presence in gene therapy - GlobeNewswire - December 18th, 2019
- 10 Years Ago, DNA Tests Were The Future Of Medicine. Now Theyre A Social Network And A Data Privacy Mess. - BuzzFeed News - December 18th, 2019
- Mosaic Angelman Should Be in Differential Diagnosis of AS, Study Says - Angelman Syndrome News - December 18th, 2019
- Sarepta Therapeutics Announces $250 Million of Non-Dilutive Senior Secured Loan Financing - Yahoo Finance - December 18th, 2019
- Triplet Therapeutics Launches with $59 Million in Financing to Further its Development of Transformative Treatments for Triplet Repeat Disorders -... - December 18th, 2019
- Genetic clues of TB spread between cows and badgers revealed - Irish Times - December 18th, 2019
- The first U.S. trials in people put CRISPR to the test in 2019 - Science News - December 18th, 2019
- Care Coordination and Precision Medicine Improve Early Diagnoses - HealthPayerIntelligence.com - December 13th, 2019