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.

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I).

8 Feb 2016

Novel quantitative method may reduce or eliminate need for invasive biopsies

Scientists have identified a quantitative method to measure changes in biomarkers, which may reduce or eliminate the need for invasive biopsies. The method, described in the February 2016 issue of The FASEB Journal uses a novel chimera design of DNA and small DNA with a companion contrast agent to allow antibodies to cross cellular membranes.

8 Feb 2016

Cell Therapy Catapult becomes Cell and Gene Therapy Catapult

4 Feb 2016

New gene cut-and-paste methods help correct disease-causing mutation in animal model

For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease- causing mutation has been corrected.

3 Feb 2016

New study lays foundation for future gene replacement therapies to treat ALS patients

Scientists have demonstrated for the first time that it is possible to specifically modify gene expression in diseased upper motor neurons, brain cells that break down in ALS.

1 Feb 2016

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder.

21 Jan 2016

Novel gene therapy treatment proves safe, effective for factor VII deficiency

A single injection. That's all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of researchers at the University of North Carolina and The Children's Hospital of Philadelphia.

20 Jan 2016

Age-related macular degeneration: an interview with Cathy Yelf, Macular Society

Age-related macular degeneration is a condition of the macula, a tiny area of the retina at the back of the eye. Your macula is only about the size of the grain of rice, that’s about four millimeters across.

19 Jan 2016

Quad partners with University of Massachusetts to evaluate performance of novel cell separation solution

The rapid growth of cell-based immunotherapy research has led to an increase in demand for methods to isolate specific immune cell populations from heterogeneous samples with high viability, purity and recovery efficiency. Quad Technologies ("Quad"), is meeting this need with its MagCloudz™ Streptavidin Cell Separation Kits and has partnered with The University of Massachusetts Medical School to evaluate the performance of its novel cell separation solution for the enrichment and purification of CD3+ T-cells from human umbilical cord blood.

18 Jan 2016

Three new genetic associations identified for primary open angle glaucoma

Researchers from Massachusetts Eye and Ear/Harvard Medical School and Case Western Reserve University School of Medicine have led an international effort to identify three genetic associations that influence susceptibility to primary open angle glaucoma -- the most common form of adult onset glaucoma and the leading cause of irreversible blindness in the world.

12 Jan 2016

Synpromics, Cell Therapy Catapult to create stable producer cell lines for large-scale manufacture of viral vectors

Synpromics Ltd, a leading synthetic biology company, and The Cell Therapy Catapult, the UK organisation accelerating the growth of the UK cell and gene therapy industry by bridging the gap between scientific research and commercialisation, today announce the launch of a collaboration to remove a major barrier to the development of the cell and gene therapy industry by reducing the cost and increasing the scale and efficiency of viral vector manufacturing.

12 Jan 2016

Novel therapy may help in diagnosing, treating heart disease in humans

A novel therapy tested by University of Guelph scientists for treating a fatal heart disorder in dogs might ultimately help in diagnosing and treating heart disease in humans.

12 Jan 2016

Clontech Laboratories launches Guide-it CRISPR/Cas9 Gesicle Production System

Clontech Laboratories, Inc., a wholly-owned subsidiary of Takara Bio Inc., today announced the launch of the Guide-it CRISPR/Cas9 Gesicle Production System. This system provides an innovative method to deliver Cas9/single guide RNA (sgRNA) ribonucleoprotein complexes to a broad range of cell types.

11 Jan 2016

New project aims to develop CAR-T cell immuno-oncology cellular therapy for solid tumours

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, the University of Birmingham and Cancer Research Technology, the commercialisation arm of Cancer Research UK, announces today the launch of a collaboration to develop a new immuno-oncology cellular therapy based on gene modifying T cells to target solid tumours.

8 Jan 2016

Scientists discover molecular mechanism responsible for degeneration of Purkinje cells in SCA1

Scientists from Bern have discovered a mechanism which is responsible for the degeneration of Purkinje cells in the cerebellum in a neurodegenerative disease called Spinocerebellar ataxia type 1. The results of their study open up new avenues for the future treatment of cerebellum associated degenerative disorders.

7 Jan 2016

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

Gene Therapy News and Research

Further Reading

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Novel quantitative method may reduce or eliminate need for invasive biopsies

Cell Therapy Catapult becomes Cell and Gene Therapy Catapult

New gene cut-and-paste methods help correct disease-causing mutation in animal model

New study lays foundation for future gene replacement therapies to treat ALS patients

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Novel gene therapy treatment proves safe, effective for factor VII deficiency

Age-related macular degeneration: an interview with Cathy Yelf, Macular Society

Quad partners with University of Massachusetts to evaluate performance of novel cell separation solution

Three new genetic associations identified for primary open angle glaucoma

Synpromics, Cell Therapy Catapult to create stable producer cell lines for large-scale manufacture of viral vectors

Novel therapy may help in diagnosing, treating heart disease in humans

Clontech Laboratories launches Guide-it CRISPR/Cas9 Gesicle Production System

New project aims to develop CAR-T cell immuno-oncology cellular therapy for solid tumours

Scientists discover molecular mechanism responsible for degeneration of Purkinje cells in SCA1

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

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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