Top
Advanced Zone - Healing Genes
1805
page-template-default,page,page-id-1805,page-parent,page-child,parent-pageid-1735,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-kit-3486,elementor-page elementor-page-1805

Advanced Zone

If momentum is a barometer, transformative medicine is coming. The Advanced Zone is here to help you stay current and informed. You will find continually updated links to cell and gene medicine research and information.

Featured News

Featured News

A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving

Like millions of other Americans, Victoria Gray has been sheltering at home with her children as the U.S. struggles through a deadly pandemic, and as protests over police violence have erupted across the country. But Gray is not like any other American. She’s the first …

Featured News

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader …

Featured News

CRISPR treatment inserted directly into the body for first time

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that …

Approved Therapies

Approved Therapies

Transfusion-dependent beta-thalassemia gene therapy approved in Europe

Zynteglo is Bluebird’s first drug to be approved and is the first gene therapy to be cleared for use in transfusion-dependent beta-thalassemia. Its marketing authorization from the European Commission follows landmark approvals from the Food and Drug Administration for Novartis’ Zolgensma and Spark Therapeutics’ Luxturna, …

Approved Therapies

Novartis gene therapy approved, but will come at cost of more than $2 million

Babies born with a severe form of a rare genetic condition known as spinal muscular atrophy almost always die before their second birthday. A gene therapy approved Fridayby the Food and Drug Administration promises to change that, offering a potential one-time fix for the genetic …

Approved Therapies

Mustang Bio and Nationwide Children’s Hospital Receive Orphan Drug Designation for MB-108 (Oncolytic Virus C134) for the Treatment of Malignant Glioma

NEW YORK and COLUMBUS, Ohio, May 16, 2019 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, and …

Policy & Regulation

Policy & Regulations

New FDA Model Aims to Speed More Rare Disease Treatments to Patients

The development of new therapies to treat orphan diseases is particularly perilous, due to limited patient populations, high costs, and difficulty in measuring clinical effectiveness. FDA officials are addressing these issues through the Rare Disease Cures Accelerator (RDCA) initiative in the Center for Drug Evaluation …

Policy & Regulations

New International Commission Launched on Clinical Use of Heritable Human Genome Editing

An international commission has been convened by the U.S. National Academy of Medicine (NAM), the U.S. National Academy of Sciences (NAS), and the Royal Society of the U.K., with the participation of science and medical academies around the world, to develop a framework for scientists, …

Policy & Regulations

FDA’s Efforts to Advance the Development of Gene Therapy

Gene therapy has been on the horizon for several decades and has now become a reality in the United States. There are now three approved products: two cell-based gene therapies for cancers of the blood (Kymriah and Yescarta) and one directly-administered gene therapy (Luxturna) for …

New Research

New Research

Gene Therapy Shows Promise in the Treatment of Chronic Traumatic Encephalopathy

Researchers from Weill Cornell Medical College led by Ronald G. Crystal, MD, the Bruce Webster professor of internal medicine and professor of genetic medicine and chairman, department of genetic medicine, published a new study (“Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy”) in Human Gene Therapy …

Featured News

CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001

CRISPR Therapeutics and Vertex Pharmaceuticals today reported preliminary, mostly-positive safety and efficacy data from the first two patients enrolled in two Phase I/II trials assessing their CRISPR/Cas9 gene-edited therapy CTX001 for a pair of blood disorders—the first clinical trial of a gene-editing candidate sponsored by …

New Research

Gene Therapy Vehicle for Sickle-Cell Disease Shifts Forward

A radically redesigned viral vector may improve the delivery of therapeutic genes to patients with sickle-cell disease. According to tests in animal models, the new vector is up to ten times more efficient at incorporating corrective genes into bone marrow stem cells than conventional vectors, …

Orphan Drugs & Product