Category Archives: Gene Therapy

DAN DUNCAN CORE - Center for Cell Gene Therapy cGMP Facilities By: crystalsilvalentz … Continue reading →

Research first to compare alternative approaches to fully matched transplant for rare immune disorder (WASHINGTON - April 13, 2015) - New research published online today in Blood, the Journal of the American Society of Hematology (ASH), reports that children with "bubble boy disease" who undergo gene therapy have fewer infections and hospitalizations than those receiving stem cells from a partially matched donor. The research is the first to compare outcomes among children with the rare immune disorder - also known as X-linked severe combined immunodeficiency (SCID-X1) - receiving the two therapeutic approaches. Children with SCID-X1 are born with a genetic defect that prevents them from developing a normal immune system. Because they are prone to life-threatening infections, infants with SCID-X1 must be kept in a sterile, protective bubble and require extensive treatment for survival beyond infancy. Infants with SCID are most likely to survive if they receive a stem cell transplant from a fully matched donor - typically a sibling - a procedure that replaces an infant's diseased stem cells with healthy donor cells. Following a successful fully matched transplant, infants with SCID-X1 are able to produce their own immune cells for the first time. In the absence of a … Continue reading →

Michigan State University, UPenn and AGTCs Gene Therapy Proof-of-Concept for Achromatopsia CNGB3- Achromatopsia is caused by a gene mutation that results in extremely poor visual acuity, photophobia, day blindness and complete loss of color discrimination. It affects dogs as well... By: AGTC … Continue reading →

Advanced Topics Successful Development of Quality Cell and Gene Therapy Products FDA Presentation - Advanced Topics: Successful Development of Quality Cell and Gene Therapy Products - FDA Compliance Seminars (LIVE) at http://www.globalcomplianceseminar.com - Providing ... By: Chris Leo … Continue reading →

Gene Therapy VideoScribe By: Jessica R … Continue reading →

Being a'Night Owl' is Risky Subscribe to the GeneChanger YouTube: http://goo.gl/jOPcep Dr. Mitchell Gaynor, Gaynor Integrative Oncology / Gene Therapy Plan, joins Thom. Night owls - they have more fun - they get more... By: Mitchell Gaynor M.D. … Continue reading →

Perpetuating the surge of interest in gene therapy technology from major pharmaceutical companies, such as Pfizer Inc. (PFE) and Novartis Inc. (NVS), biopharmaceutical company Bristol-Myers Squibb Co. (BMY) announced a partnership agreement with gene therapy research and development company uniQure NV (QURE) which gives them exclusive rights over uniQures gene therapy technology development for up to 10 genetic targets, for treating cardiovascular diseases, among others. Marking the first foray of Bristol-Myers into gene therapy, the deal gives the company the sole right to develop uniQures gene therapy drugs to treat diseases such as heart failure, which is one of the most common health problems in the world today. Cardiovascular disease is expected to affect about 40 million people around the world by 2030. Bristol-Myers Squibb has an excellent and long-standing track record of success in discovering and developing treatments for cardiovascular diseases and in embracing advancing technologies for the treatment of human diseases, Carl Decicco, Ph.D., head of Discovery, R&D, Bristol-Myers Squibb said in a statement issued by the company. Collaborating with uniQure, a clear leader in the field with an innovative and validated gene therapy platform, further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases. … Continue reading →

A team of scientists from the Institute for Biotechnology and Biomedicine at the UAB has produced an alternative to the use of viruses in gene therapy. The researchers synthesised nanoparticles which act as artificial viruses, capable of surrounding DNA fragments and releasing them as therapeutic agents, with no biological risk, into the interior of the cells. Researchers of the Nanobiology Unit from the Universitat Autnoma de Barcelona Institute of Biotechnology and Biomedicine, led by Antonio Villaverde, managed to create artificial viruses, protein complexes with the ability of self-assembling and forming nanoparticles which are capable of surrounding DNA fragments, penetrating the cells and reaching the nucleus in a very efficient manner, where they then release the therapeutic DNA fragments. The achievement represents an alternative with no biological risk to the use of viruses in gene therapy. Gene therapy, which is the insertion of genes into the genome with therapeutic aims, needs elements which can transfer these genes to the nucleus of the cells. One of the possibilities when transferring these genes is the use of a virus, although this is not exempt of risks. That is why scientists strive to find an alternative. With this as their objective, emerging nanomedicines aim … Continue reading →

4 hours ago Researchers of the Nanobiology Unit from the UAB Institute of Biotechnology and Biomedicine, led by Antonio Villaverde, managed to create artificial viruses, protein complexes with the ability of self-assembling and forming nanoparticles which are capable of surrounding DNA fragments, penetrating the cells and reaching the nucleus in a very efficient manner, where they then release the therapeutic DNA fragments. The achievement represents an alternative with no biological risk to the use of viruses in gene therapy. Gene therapy, which is the insertion of genes into the genome with therapeutic aims, needs elements which can transfer these genes to the nucleus of the cells. One of the possibilities when transferring these genes is the use of a virus, although this is not exempt of risks. That is why scientists strive to find an alternative. With this as their objective, emerging nanomedicines aim to imitate viral activities in the form of adjustable nanoparticles which can release nucleic acids and other drugs into the target cell. Among the great diversity of materials tested by researchers, proteins are biocompatible, biodegradable and offer a large variety of functions which can be adjusted and used in genetic engineering. Nevertheless, it is very complicated … Continue reading →

PBR Staff Writer Published 07 April 2015 Bristol-Myers Squibb (BMS) and Netherlands-based uniQure have entered into exclusive strategic collaboration to develop gene therapies for cardiovascular diseases. The deal will give BMS exclusive access to uniQure's gene therapy technology platform for the development of new treatments. The companies will collaborate on up to ten targets, mostly concentrating on cardiovascular therapies, such as a treatment to reinvigorate congestive heart failure patients' ability to synthesize S100A1. The Dutch firm will lead discovery efforts and clinical manufacturing, while BMS will fund the research, lead development and regulatory activities as well as have sole commercialization rights under the deal. Bristol-Myers Squibb Discovery R&D head Carl Decicco said: "Bristol-Myers Squibb has an excellent and long-standing track record of success in discovering and developing treatments for cardiovascular diseases and in embracing advancing technologies for the treatment of human diseases. "Collaborating with uniQure, a clear leader in the field with an innovative and validated gene therapy platform, further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases, including cardiovascular diseases such as heart failure." As part of the deal, BMS will pay $50m upfront and $100m total in the near-term, and the company will gain … Continue reading →