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News

Keep up with the latest Gene & Medicine Updates!

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 …

Approval Recommended

Approval Recommended

Gene therapy for beta-thalassemia recommended for approval in Europe

Europe’s Committee for Medicinal Products for Human Use has recommended that Bluebird Bio’s lentiglobin, or Zynteglo, gene therapy for beta-thalassemia be approved. The treatment inserts a copy of the beta-globin gene into bone marrow cells. https://www.statnews.com/2019/03/29/first-gene-therapy-to-treat-rare-blood-disease-nears-european-approval/

Clinical Trials

Clinical Trials

Diffuse Intrinsic Pontine Glioma

Phase I Study of Autologous T Lymphocytes Expressing GD2-specific Chimeric Antigen and Constitutively Active IL-7 Receptors for the Treatment of Patients With GD2-expressing Brain Tumors (GAIL-B) C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B) Phase 1 DESCRIPTION: Researchers in Texas Children’s Hospital are seeking for patients with …

Clinical Trials

Peripheral Artery Disease

Hepatocyte Growth Factor to Improve Functioning in PAD (HI-PAD) Hepatocyte Growth Factor to Improve Functioning in Peripheral Artery Disease: The HI-PAD Study Phase 3 DESCRIPTION: Northwestern University researchers are seeking 39 people with peripheral artery disease (PAD) who do not have critical limb ischemia for …

Clinical Trials

Ovarian Cancer

A Study of VB-111 With Paclitaxel vs Paclitaxel for Treatment of Recurrent Platinum-Resistant Ovarian Cancer (OVAL) (OVAL) A Randomized, Controlled, Double-Arm, Double-Blind, Multi-Center Study of Ofranergene Obadenovec (VB-111) Combined With Paclitaxel vs. Paclitaxel Combined With Placebo for the Treatment of Recurrent Platinum-Resistant Ovarian Cancer Phase …

Costs, Insurance, Payment Assistance

Costs

Legislation to Help Diagnose Children with Rare Diseases Introduced

Washington, D.C.—U.S. Senators Susan Collins (R-ME), Doug Jones (D-AL), Martha McSally (R-AZ), and Bob Menendez (D-NJ) introduced bipartisan legislation to help thousands of children with rare diseases receive a diagnosis and proper treatment more quickly. Children with rare diseases face a “diagnostic odyssey” that typically …

Costs

Novartis plans to give away world’s costliest therapy to some patients

Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug program that one patient group worried was a “health lottery” that could neglect some babies. Starting Jan. 2, Novartis’s AveXis unit which developed …

Costs

Cigna Health Services Business Pioneers an Innovative Solution to Affordably Bring Life-Changing Therapies to Patients

Breakthrough, potentially life-changing medicines offer great promise for patients and their families. However, the high prices of the medicines – millions of dollars in some cases – threaten coverage and access by patients who may benefit. Today, Cigna’s health services business is introducing Embarc Benefit …

Policy & Regulations

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, …

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