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 – […]

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 […]

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 — […]

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 […]

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 […]

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 […]

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 […]

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. […]

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 […]

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 […]

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 […]

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 […]

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 […]

Biotechnology cancer Cell Therapy Clinical Trials Gene Therapy

Vanderbilt-Ingram Cancer Center Patients Treated with New FDA-Approved CAR T Therapy

Vanderbilt-Ingram Cancer Center (VICC) has been selected as one of the few authorized treatment centers in the United States approved to administer the first FDA- approved chimeric antigen receptor T cell (CAR T) therapy for treatment of adult patients with a specific type of lymphoma. VICC is the only cancer center in a seven-state region […]

Biotechnology cancer Cell Therapy Gene Therapy

Insights from a rare genetic disease may help treat multiple myeloma

A new class of drugs for blood cancers such as leukemia and multiple myeloma is showing promise. But it is hobbled by a problem that also plagues other cancer drugs: targeted cells can develop resistance. Now scientists, reporting in ACS Central Science, have found that insights into a rare genetic disease known as NGLY1 deficiency […]

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 […]