Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Leading researchers reject proposed link between adeno-associated virus 2 and hepatocellular carcinoma

The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly rejected by leading researchers in the fields of gene therapy and molecular genetics.

7 Jan 2016

Vtesse receives FDA's Breakthrough Therapy designation for VTS-270

Vtesse, Inc. announced today that the U.S. Food and Drug Administration has granted its drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial.

6 Jan 2016

UT Southwestern scientists use new gene-editing technique to prevent progression of DMD in young mice

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice.

4 Jan 2016

Researchers successfully use CRISPR to treat adult mouse model of Duchenne muscular dystrophy

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy.

4 Jan 2016

Several therapies for hemophilia A, hemophilia B and hemophilia with inhibitors to be launched between 2015-2025

Decision Resources Group forecasts that several therapies will launch for hemophilia A, hemophilia B and hemophilia with inhibitors in the United States and Europe during our 2015-2025 study period.

29 Dec 2015

Ultrafast near-infrared lasers deliver gene therapy to treat macular degeneration

Millions of adults over age 50 struggle each year with vision loss caused by damage to the retina or common macular degeneration.

24 Dec 2015

Baxalta announces initial results from ADYNOVATE Phase 3 trial for treatment of hemophilia patients

Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced initial results from a Phase 3 clinical trial of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)].

22 Dec 2015

Scientists uncover novel strategy to block cancer's spread, restore tumor vulnerability to chemotherapy

St. Jude Children's Research Hospital scientists have discovered how an aggressive tumor of the muscle and soft tissue hijacks an export pathway in cells to lay the groundwork for cancer progression and resistance to chemotherapy.

22 Dec 2015

UCL Cancer Institute win $200,000 CytoFLEX Flow Cytometer from Beckman Coulter

An internationally renowned gene therapy research team has won a $200,000 CytoFLEX Flow Cytometer from Beckman Coulter Life Sciences.

From Beckman Coulter Life Sciences - Flow Cytometry 18 Dec 2015

Combination treatment provides effective one-two punch against prostate cancer

Results from a long-term clinical trial conducted by cancer researchers at Houston Methodist Hospital show that combining radiation treatment with "suicide gene therapy," a technique in which prostate cancer cells are genetically modified so they signal a patient's immune system to attack them, provides a safe and effective one-two punch against the disease.

12 Dec 2015

Circuit awarded contract to pursue optogenetic therapies for peripheral nervous system indications

Circuit Therapeutics today announced that it has been awarded a 2.7 million dollar contract from the Defense Advanced Research Projects Agency to pursue optogenetic therapies for peripheral nervous system indications. The award, part of DARPA's Electrical Prescriptions (ElectRX) program, will enable Circuit to pursue research and development projects related to its treatment for neuropathic pain.

10 Dec 2015

Expression of particular estrogen receptor can reinstate lost memory function

The hormone estrogen helps protect memory and promote a healthy brain, but this effect wanes as women age, and even estrogen replacement therapy stops working in humans after age 65. Now researchers at University of Florida Health have used gene therapy in a rat model to show that the expression of a particular receptor can reinstate lost memory function.

9 Dec 2015

Research shows significant step toward improving outcomes, reducing treatment burden for SCD patients

Emerging therapies and promising new insights in the treatment of sickle cell disease represent a significant step toward improving outcomes and reducing the treatment burden for affected children and adults. Studies reporting these advances will be presented today at the 57th American Society of Hematology Annual Meeting and Exposition.

7 Dec 2015

New approaches to treating leukemia, lymphoma and myeloma

New, highly targeted treatment approaches for leukemia, lymphoma, and myeloma to be presented today at the 57th American Society of Hematology Annual Meeting and Exposition represent a tremendous expansion of oral and intravenous therapy options for patients with blood cancers.

7 Dec 2015

Cell Therapy Catapult’s 2015 Annual Review highlights key achievements across UK’s health and economy

The Cell Therapy Catapult, the UK organisation dedicated to the growth of the UK cell and gene therapy industry by bridging the gap between scientific research and commercialisation, today announces the release of their 2015 Annual Review.

7 Dec 2015

Gene therapy shows promise in children with Wiskott-Aldrich syndrome

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology (abstract #260).

7 Dec 2015

Gene therapy can restore immune systems of children and young adults with SCID-X1

Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare inherited disorder that primarily affects males, scientists from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, have found.

7 Dec 2015

Research reveals genetic variants linked to treatment-related complications in children with blood diseases

Research to be presented today at the 57th American Society of Hematology Annual Meeting and Exposition reveals genetic variants that are associated with disease severity and treatment-related complications in children with blood diseases.

7 Dec 2015

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.

7 Dec 2015

Memorandum of understanding signed between Cell Therapy Catapult and Kanagawa Prefecture

30 Nov 2015

Gene Therapy News and Research

Further Reading

Leading researchers reject proposed link between adeno-associated virus 2 and hepatocellular carcinoma

Vtesse receives FDA's Breakthrough Therapy designation for VTS-270

UT Southwestern scientists use new gene-editing technique to prevent progression of DMD in young mice

Researchers successfully use CRISPR to treat adult mouse model of Duchenne muscular dystrophy

Several therapies for hemophilia A, hemophilia B and hemophilia with inhibitors to be launched between 2015-2025

Ultrafast near-infrared lasers deliver gene therapy to treat macular degeneration

Baxalta announces initial results from ADYNOVATE Phase 3 trial for treatment of hemophilia patients

Scientists uncover novel strategy to block cancer's spread, restore tumor vulnerability to chemotherapy

UCL Cancer Institute win $200,000 CytoFLEX Flow Cytometer from Beckman Coulter

Combination treatment provides effective one-two punch against prostate cancer

Circuit awarded contract to pursue optogenetic therapies for peripheral nervous system indications

Expression of particular estrogen receptor can reinstate lost memory function

Research shows significant step toward improving outcomes, reducing treatment burden for SCD patients

New approaches to treating leukemia, lymphoma and myeloma

Cell Therapy Catapult’s 2015 Annual Review highlights key achievements across UK’s health and economy

Gene therapy shows promise in children with Wiskott-Aldrich syndrome

Gene therapy can restore immune systems of children and young adults with SCID-X1

Research reveals genetic variants linked to treatment-related complications in children with blood diseases

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Memorandum of understanding signed between Cell Therapy Catapult and Kanagawa Prefecture

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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