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. […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 […]

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 […]

Cell Therapy

Researchers Encouraged by Continued Signs of Tolerability and Disease Modification in Gene Therapy Trial for Sanfilippo Syndrome

Researchers from Abeona Therapeutics Inc, a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, provided, an update on clinical results through 30 days post-injection for the completed low-dose cohort (n=3) in the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) at the Orphan Drugs & Rare Disease Conference (London, UK). The first-in-man […]

Clinical Trials Gene Therapy Vaccines

Smallest-Reported Artificial Virus Could Help Advance Gene Therapy

Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report […]

Pharmaceutical Business News Pharmaceuticals

Pfizer acquires Bamboo Therapeutics to beef up gene therapy arsenal

(Reuters) – Pfizer Inc said it had acquired privately held gene therapy developer Bamboo Therapeutics Inc in a deal worth up to $645 million to boost its presence in the treatment of rare diseases. Research into gene therapy, which aims to insert corrective genes into malfunctioning cells, goes back a quarter of a century but […]

Cell Therapy Clinical Trials

Understanding Natural History Studies in Drug Development for Rare Diseases

Q &A with Michelle Berg, Vice President, Patient Advocacy, Abeona Therapeutics Inc. Developing a new drug for patients with a disease, particularly when it’s a disease that affects a relatively small number of patients,  poses unique challenges that do not apply to medical therapies for more prevalent diseases. However, an increasingly common, and particularly important […]