Biotechnology Clinical Trials Gene Therapy genes

Proof of Concept Study Aims for Rapid Genotyping to Guide Epilepsy Treatment

An easy and inexpensive test for the HLA-B*15:02 allele could one day allow clinicians to quickly identify which epilepsy patients are at risk of carbamazepine hypersensitivity, according to study results presented by Gita V. Soraya at ILAE's 2018 Asian & Oceanian Epilepsy Congress in Bali. “This study has provided the proof of principle evidence for the development of a handheld device that can be used at the clinic or bedside
Gene Therapy

Sarepta Therapeutics Presented Positive Preliminary Results from the First Three Children Dosed in the Phase 1/2a Gene Therapy Micro-dystrophin Trial to Treat Patients with Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, said that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy (DMD). Dr. Mendell presented the following preliminary data on the first three patients enrolled
Biotechnology cancer Gene Therapy Immunotherapies

CAR-T Immunotherapy Eliminates Metastatic Colorectal Cancer in Mice

Immunotherapy has given patients and oncologists new options, which for some patients, has meant cures for diseases that had been untreatable. Colorectal cancer has a high mortality rate in advanced stages of the disease with few effective therapies. Researchers at the Sidney Kimmel Cancer Center (SKCC) at Jefferson Health show that a type of immunotherapy called CAR-T cell therapy, successfully kills tumors and prevents metastases in mouse models of the
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
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
Biotechnology cancer Gene Therapy rare diseases

IU Advances Fight Against Hepatitis B with ‘Virus-Cracking’ Molecules

Indiana University researchers have made an important step forward in the design of drugs that fight the hepatitis B virus, which can cause liver failure and liver cancer. It's estimated that 2 billion people worldwide have had a hepatitis B virus infection in their lifetime, with about 250 million -- including 2 million Americans -- living with chronic infection. Although a vaccine exists, there is no cure. The study, published Jan.
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
Gene Therapy Pharmaceutical Business News

Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for Gene Therapy in Epidermolysis Bullosa

Gene therapy developer, Abeona Therapeutics, announced that the FDA has granted Regenerative Medicine Advanced Therapy designation to EB-101, a gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa (RDEB). Abeona`s EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient`s own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa. The EB-101 program has
Cell Therapy Clinical Trials Gene Therapy Immunotherapies

Bioengineered Soft Microfibers Improve T-Cell Production

Columbia engineers bioengineer soft microfibers to improve T-cell production. T cells play a key role in the body’s immune response against pathogens. As a new class of therapeutic approaches, T cells are being harnessed to fight cancer, promising more precise, longer-lasting mitigation than traditional, chemical-based approaches. These “living drugs” are poised to transform medicine, with a growing number of cellular therapies receiving FDA-approval. A current bottleneck in these approaches and
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 —
Biotechnology Gene Therapy Infectious Diseases Vaccines

Discovery of key molecules involved in severe malaria

Malaria*1 is one of three major infectious diseases*2 affecting approximately 300 million people every year, accounting for about 500,000 deaths, but effective vaccine development has not been successful. Among malaria parasites infecting humans, Plasmodium falciparum (P. falciparum)*3 causes especially severe disease. In addition, acquired immunity to malaria is inefficient, even after repeated exposures to P. falciparum, but the immune regulatory mechanisms used by P. falciparum remain largely unclear. Therefore, malaria
Biotechnology Clinical Trials Gene Therapy Immunotherapies

Researchers show stress suppresses response to cancer treatments

New research shows that chronic stress suppresses the immune system's response to cancer, reducing the effectiveness of immunotherapy treatments. University of Queensland scientists say they are investigating dual therapies for patients to reduce stress signalling and improve their response to treatments. UQ Diamantina Institute researcher Dr Stephen Mattarollo said lymphoma progressed more rapidly in mouse models when stress pathways were induced to reflect chronic psychological stress. "When we used immunotherapies
Biotechnology cancer Cell Therapy Clinical Trials Gene Therapy

Pairing Cancer Genomics with Cognitive Computing Highlights Potential Therapeutic Options

A University of North Carolina Lineberger Comprehensive Cancer Center-led study has demonstrated the ability of cognitive computing to scour large volumes of data from scientific studies and databases to identify potentially relevant clinical trials or therapeutic options for cancer patients based on the genetics of their tumors. The researchers said their findings, published in the journal The Oncologist, suggest that cognitive computing applications could help physicians to stay abreast of
Biotechnology cancer Gene Therapy Prodrug rare diseases

Deadly Lung Cancers Are Driven by Multiple Genetic Changes

Blood-Based Cancer Tests Reveal Complex Genomic Landscape of Non-Small Cell Lung Cancers A new UC San Francisco–led study challenges the dogma in oncology that most cancers are caused by one dominant “driver” mutation that can be treated in isolation with a single targeted drug. Instead, the new research finds one of the world’s most deadly forms of lung cancer is driven by changes in multiple different genes, which appear to
Biotechnology Clinical Trials Gene Therapy rare diseases

Scientists make a major breakthrough to treat fibrotic diseases that cause organ failure

Researchers from Duke-NUS Medical School (Duke-NUS) and the National Heart Centre Singapore (NHCS) have discovered that a critical protein, known as interleukin 11 (IL11) is responsible for fibrosis and causes organ damage. While it is surprising that the importance of IL11 has been overlooked and misunderstood for so long, it has now been very clearly demonstrated by this work. A protein known as transforming growth factor beta 12 ("TGFB1") has
Biotechnology Cell Therapy Clinical Trials Gene Therapy Immunotherapies Infectious Diseases Vaccines

New molecule shows promise in HIV vaccine design

Researchers at the University of Maryland and Duke University have designed a novel protein-sugar vaccine candidate that, in an animal model, stimulated an immune response against sugars that form a protective shield around HIV. The molecule could one day become part of a successful HIV vaccine. "An obstacle to creating an effective HIV vaccine is the difficulty of getting the immune system to generate antibodies against the sugar shield of