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.

NIH-funded researchers test new gene therapy to improve visual function in mice

Researchers funded by the National Institutes of Health have developed a novel mouse model for the vision disorder Leber hereditary optic neuropathy (LHON), and found that they can use gene therapy to improve visual function in the mice. LHON is one of many diseases tied to gene mutations that damage the tiny energy factories that power our cells, called mitochondria.

6 Oct 2015

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, led to reduced symptoms and significantly extended life span in a mouse model of severe DMD. Over-expression of ITGA7 did not elicit an immune reaction, further supporting its potential as a novel treatment for DMD, according to a new study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

30 Sep 2015

Quethera receives seed investment funding to develop gene therapy for glaucoma

Quethera, a gene therapy company developing a treatment for glaucoma that could prevent blindness, has received seed investment funding led by Midven’s Rainbow Seed Fund alongside co-investor Cambridge Enterprise, the commercialisation arm of the University of Cambridge, to continue pre-clinical development of their therapy.

18 Sep 2015

CEVEC partners with Généthon to develop lentivirus packaging cell lines based on its CAP GT technology

CEVEC Pharmaceuticals GmbH, the expert in the production of tailor-made recombinant glycoproteins and gene therapy vectors, today announced a collaboration agreement with Généthon (Evry, France) for the development and exploitation of lentivirus packaging cell lines based on CEVEC’s proprietary CAP GT technology.

18 Sep 2015

Benitec Biopharma initiates new site for ongoing Phase 1/2a TT-034 trial for hepatitis C

Benitec Biopharma Limited, a clinical-stage biotechnology company developing innovative therapeutics based on its gene-silencing technology, DNA-directed RNA interference (ddRNAi), is pleased to announce it has initiated a new site for its ongoing Phase 1/2a TT-034 trial at the Methodist Health System Clinical Research Institute in Dallas, Texas.

16 Sep 2015

New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

A new collaboration between Seattle Children's Research Institute and bluebird bio, Inc., a biotechnology company based in Cambridge, Mass. and Seattle, will allow researchers to develop potentially transformative gene therapies for severe genetic and rare pediatric diseases. The collaboration supports pediatric researchers in Seattle who will work with bluebird bio scientists and the company's gene editing technology to research potential cures for genetic pediatric diseases.

11 Sep 2015

NYU's Jin Kim Montclare develops dual-delivery system to potentially treat multi-drug resistant cancer cells

The National Science Foundation recently funded research aimed at developing an engineered protein-lipid system that simultaneously delivers genes and drugs for the potential treatment of multi-drug resistant cancer cells. The dual-delivery system could also apply to diabetes and other conditions requiring a variety of therapeutic approaches.

11 Sep 2015

CEVEC launches unique cell-expression system for scalable production of gene therapy vectors

CEVEC Pharmaceuticals GmbH, the expert in the production of tailor-made recombinant glycoproteins and gene therapy vectors, today announced the introduction of its unique cell-expression system, CAP GT, for the fully scalable production of gene therapy vectors, based on the Company's proprietary CAP technology.

8 Sep 2015

USF, FARA to jointly host scientific symposium on Friedreich's ataxia

The University of South Florida will again bring together leading researchers and patients searching for a treatment for Friedreich's ataxia and related disorders at the seventh annual scientific symposium "Understanding Energy for A Cure." The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom, USF Cedar Circle, Tampa, FL 33620.

2 Sep 2015

Affinity tuning can make CAR T cells spare normal cells and attack cancer cells

A new development in engineering chimeric antigen receptor (CAR) T cells, called affinity tuning, can make the CAR T cells spare normal cells and better recognize and attack cancer cells, which may help lower the toxicity associated with this type of immunotherapy when used against solid tumors, according to a preclinical study.

1 Sep 2015

$Gene transfer therapy not beneficial for heart failure patients with reduced ejection fraction$

Gene transfer therapy not beneficial for heart failure patients with reduced ejection fraction

Gene transfer therapy aimed at correcting an enzyme abnormality involved in myocardial contraction and relaxation did not improve outcomes in heart failure patients with reduced ejection fraction, results of the CUPID 2 study show.

1 Sep 2015

Gene therapy restores visual function in mouse model of LCA1

Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months.

1 Sep 2015

CUMC vision researchers discover gene that causes myopia

Vision researchers at Columbia University Medical Center have discovered a gene that causes myopia, but only in people who spend a lot of time in childhood reading or doing other "nearwork."

1 Sep 2015

Researchers identify new virus that plays role in rare type of liver cancer

More than a cause of a simple infection, viruses are often involved in the development of serious diseases. Such is the case with liver cancer, which often develops in an organ that has been weakened by hepatitis B or C virus.

28 Aug 2015

Experimental gene therapy may prevent neuronal degeneration in patients with Alzheimer's disease

Degenerating neurons in patients with Alzheimer's disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, report researchers at University of California, San Diego School of Medicine in the current issue of JAMA Neurology.

28 Aug 2015

Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the closing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec, together with warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares.

24 Aug 2015

Stanford University launches new professional education course

Imagine a world where health care is personalized, diseases are treated or even prevented before they become debilitating, and doctor's visits include regular reviews of individual genetic profiles. These are just some of the possibilities at the intersection of healthcare and genomics.

24 Aug 2015

Alpha lipoic acid can stimulate telomerase with positive effects in mouse model of atherosclerosis

In human cells, shortened telomeres, the protective caps at the ends of chromosomes, are both a sign of aging and contribute to it. Scientists at Emory University School of Medicine have found that the dietary supplement alpha lipoic acid (ALA) can stimulate telomerase, the enzyme that lengthens telomeres, with positive effects in a mouse model of atherosclerosis.

24 Aug 2015

MCRI announces winners of cancer crowdsourcing initiative

The Myeloma Crowd Research Initiative (MCRI), an unprecedented collaboration of cancer researchers and patient advocates, today announced the two winners of its first-ever crowdsourcing and patient-led initiative to fund research in high-risk multiple myeloma, a rare malignancy of plasma cells.

19 Aug 2015

Benitec prices initial public offering of 1,500,000 American Depositary Shares

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the pricing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec and warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares, at a price of US$9.21 per ADS and US$0.01 per warrant.

18 Aug 2015

Gene Therapy News and Research

Further Reading

NIH-funded researchers test new gene therapy to improve visual function in mice

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

Quethera receives seed investment funding to develop gene therapy for glaucoma

CEVEC partners with Généthon to develop lentivirus packaging cell lines based on its CAP GT technology

Benitec Biopharma initiates new site for ongoing Phase 1/2a TT-034 trial for hepatitis C

New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

NYU's Jin Kim Montclare develops dual-delivery system to potentially treat multi-drug resistant cancer cells

CEVEC launches unique cell-expression system for scalable production of gene therapy vectors

USF, FARA to jointly host scientific symposium on Friedreich's ataxia

Affinity tuning can make CAR T cells spare normal cells and attack cancer cells

Gene transfer therapy not beneficial for heart failure patients with reduced ejection fraction

Gene therapy restores visual function in mouse model of LCA1

CUMC vision researchers discover gene that causes myopia

Researchers identify new virus that plays role in rare type of liver cancer

Experimental gene therapy may prevent neuronal degeneration in patients with Alzheimer's disease

Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Stanford University launches new professional education course

Alpha lipoic acid can stimulate telomerase with positive effects in mouse model of atherosclerosis

MCRI announces winners of cancer crowdsourcing initiative

Benitec prices initial public offering of 1,500,000 American Depositary Shares

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