cancer Cancer Discovery Cancer screening Clinical Trials

Early Clinical Trial Data Show Gene Therapy Reversing Sickle Cell Anemia

After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common.  Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San
Biotechnology Gene Therapy Uncategorized

Gene Therapy May Help Brain Heal From Stroke, Other Injuries

Scientists have found a genetic trigger that may improve the brain’s ability to heal from a range of debilitating conditions, from strokes to concussions and spinal cord injuries. A new study in mice from UT Southwestern’s O’Donnell Brain Institutes hows that turning on a gene inside cells called astrocytes results in a smaller scar and – potentially – a more effective recovery from injury. The research examined spinal injuries but likely
Neurodegenerative diseases

Gene therapy may help brain heal from stroke, other injuries

Scientists have found a genetic trigger that may improve the brain's ability to heal from a range of debilitating conditions, from strokes to concussions and spinal cord injuries. A new study in mice from UT Southwestern's O'Donnell Brain Institute shows that turning on a gene inside cells called astrocytes results in a smaller scar and - potentially - a more effective recovery from injury. The research examined spinal injuries but
Gene Therapy

Mass. Eye and Ear Performs First FDA-Approved Gene Therapy Procedure for Inherited Disease

Massachusetts Eye and Ear made medical history this week by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine, as it is the first time any FDA-approved gene therapy has been given to a patient for any inherited disease. The treatment, commercially identified as Luxturna, was developed by Spark Therapeutics and approved in
Gene Therapy

New gene therapy corrects a form of inherited macular degeneration in canine model

Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable. The study, published today in Proceedings of the National Academy of Sciences, was led by Karina
Gene Therapy

Gene Therapy Researchers Find a Viral Barcode to Cross the Blood-Brain Barrier

Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS. But the small viruses that deliver therapeutic genes can have adverse side effects at high doses. UNC School of Medicine researchers have now found a structure on these viruses that makes them better at crossing from the bloodstream into the brain – a key factor for administering gene therapies at lower doses for treating
Cell Therapy

Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in Sanfillippo Syndrome

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced updated clinical data from the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH), the company’s investigational gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. The results demonstrate robust and durable clinical effects achieved throughout various timepoints post-administration.  To date,
Biotechnology Clinical Trials Gene Therapy Immunotherapies

T Cell Therapy Shows Persistent Benefits in Young Leukemia Patients

CHOP Oncologists Update Analysis in Pivotal Global Trial of CAR T-Cell Treatment for Relapsed or Refractory ALL Updated results from a global clinical trial of the CAR T-cell therapy, tisagenlecleucel, a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL), reveal that children and young adults continued to show high rates of durable, complete remission of their disease. Most side effects were short-lived and reversible, according to
Biotechnology

New explanation for why airways close in asthma holds promise for future class of drugs

 Houston Methodist researchers have a new explanation for what causes the lungs' airways to close during asthma attacks that could change the lives of the 300 million people worldwide who suffer from asthma. The discovery holds promise for developing a new class of drugs that is radically different from the steroids currently used to treat it. Led by Xian C. Li, M.D., Ph.D., and his colleagues in the Immunobiology and Transplant Science
Biotechnology cancer Clinical Trials Gene Therapy Immunotherapies

Two New Breast Cancer Genes Emerge from Lynch Syndrome Gene Study

The findings suggest that genetic screening for breast cancer should be expanded to include MSH6 and PMS2 Researchers at Columbia University Irving Medical Center and NewYork-Presbyterian have identified two new breast cancer genes. Having one of the genes—MSH6 and PMS2—approximately doubles a woman’s risk of developing breast cancer by age 60. The study, in collaboration with GeneDx, a genetic testing company, was published online today in Genetics in Medicine. The
Gene Therapy

Study advances gene therapy for glaucoma

While testing genes to treat glaucoma by reducing pressure inside the eye, University of Wisconsin-Madison scientists stumbled onto a problem: They had trouble getting efficient gene delivery to the cells that act like drains to control fluid pressure in the eye. Genes can't work until they enter a cell. Glaucoma, one of the most common blinding diseases, is caused by excess pressure inside the eye, usually due to a clog
Gene Therapy

FDA Approves Spark Therapeutics’ Luxturna

The U.S. Food and Drug Administration today approved Spark Therapeutics' 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. “Today’s approval marks another first in the field of gene therapy —
cancer Gene Therapy

German research advances in cancer and blood disorders reported in human gene therapy

Virotherapy capable of destroying tumor cells and activating anti-tumor immune reactions, and the use of engineered hematopoietic stem cells (HSCs) to deliver replacement genes that have the potential to cure blood diseases are among the key areas of gene therapy being advanced by German researchers and highlighted in a special issue of Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The issue is available free on the Human Gene
Gene Therapy Genetic Disorders

FDA Panel Approves Gene Therapy For A Form Of Childhood Blindness

An advisory board at the  Food and Drug Administration today endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood. The recommendation came in a unanimous 16-0 vote after a day full of hearings that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by
Biotechnology cancer Gene Therapy

Skewing the aim of targeted cancer therapies

Headlines, of late, have touted the successes of targeted gene-based cancer therapies, such as immunotherapies, but, unfortunately, also their failures. Broad inadequacies in a widespread biological concept that affects cancer research could be significantly deflecting the aim of such targeted drugs, according to a new study. A team exploring genetic mechanisms in cancer at the Georgia Institute of Technology has found evidence that a prevailing concept about how cells produce
Biotechnology Clinical Trials Gene Therapy

Largest study of malaria gene function reveals many potential drug targets

The malaria parasite's success is owed to the stripping down of its genome to the bare essential genes, scientists at the Wellcome Trust Sanger Institute and their collaborators have found. In the first ever large-scale study of malaria gene function, scientists analysed more than half of the genes in the parasite's genome and found that two thirds of these genes were essential for survival -- the largest proportion of essential
Biotechnology Clinical Trials Gene Therapy

Breathing in a New Gene Therapy to Treat Pulmonary Hypertension

Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with a way to reverse the damage caused by PH. This gene therapy technique comes from
Clinical Trials Gene Therapy

Altered virus may expand patient recruitment in human gene therapy trials

For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response. The discovery, published May 29 in the Proceedings of the National Academy of Sciences, is a crucial step in
Biotechnology Gene Therapy

After One Dose of Gene Therapy, Hemophilia B Patients Maintain Near-Normal Levels of Clotting Factor

At ASH Meeting, CHOP Hematologist Leads Clinical Trial in Which All Subjects Safely Maintain Factor IX Expression that Curtails Disabling Bleeding Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing
Dermatology Gene Therapy

Gene therapy for blistering skin disease appears to enhance healing in clinical trial

Grafting sheets of a patient's genetically corrected skin to open wounds caused by the blistering skin disease epidermolysis bullosa appears to be well-tolerated and improves wound healing, according to a phase-1 clinical trial conducted by researchers at the Stanford University School of Medicine. The results mark the first time that skin-based gene therapy has been demonstrated to be safe and effective in patients. The findings will be published Nov. 1