GENE THERAPY FOR GENETIC EYE DISORDERS
FROM HAND MOVEMENT TO 6/60 VISION WITH #GENETHERAPY #DRAGARWALHOSPITALS #BANGALORE #DRSUNITAAGARWAL #INDIA #MEDICALTOURISM.

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GENE THERAPY FOR GENETIC EYE DISORDERS - Video

MultiVir, a biotech developing gene therapies for cancer, filed on Monday with the SEC to raise up to $70 million in an initial public offering.

The Houston, TX-based company, which was founded in 2009, plans to list on the NASDAQ under the symbol MVIR. MultiVir initially filed confidentially on December 22, 2014. RBC Capital Markets is the sole bookrunner on the deal. No pricing terms were disclosed.

Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital's research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital, the Renaissance IPO ETF (symbol: IPO) or the Global IPO Fund (symbol: IPOSX) , may have investments in securities of companies mentioned.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

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Cancer gene therapy biotech MultiVir files for a $70 million IPO

Gentech Lesson: Gene Therapy
Momes Honors Bio EB/1: Gentech Lesson on Gene Therapy. By Sarah, Jordan, Lisa, Sydney.

By: Sarah Minster

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Gentech Lesson: Gene Therapy - Video

Colorblind monkey cured by gene therapy...
Dalton, a squirrel monkey, is cured of colorblindness by gene therapy and completes color vision discrimination test. Dalton, a squirrel monkey, is cured of colorblindness by gene therapy and...

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Colorblind monkey cured by gene therapy... - Video

NEW YORK (TheStreet) -- The next investor referendum on the resurgent gene-therapy field will arrive next month whenCelladon (CLDN)is expected to announce results from a mid-stage study of a gene therapy aimed atimproving the heart's pumping ability in patients suffering fromthe organ's advanced failure.

Gene therapy uses engineered viruses to replace defective, disease-causing genes. Celladon's lead therapy, Mydicar, is a virus engineered to insert a working gene capable of producing a protein called SERCA2a into heart-failure patients. SERCA2a is responsible for helping heart muscles contract and pump blood more efficiently. Heart-failure patients have low levels of SERCA2a and hearts that do a poor job pumping blood around the body. Celladon believes infusing Mydicar should lead to higher SERCA2a levels and improved heart function.

Must Read: 5 Stocks Warren Buffett Is Selling

Celladon went public in January 2014 at $8 per share. The stock was tradingat $24.10, down 1.8%, on Wednesday morning, after rising by more than 30% in March as investors anticipate the Mydicar study results.

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Celladon Heart-Failure Study Looms Large as Next Big Test for Gene Therapy

A simulation from the Neitz lab of what colorblindness looks like, with normal color vision on the left and red-green colorblindness on the right. Courtesy of Neitz Laboratory hide caption

A simulation from the Neitz lab of what colorblindness looks like, with normal color vision on the left and red-green colorblindness on the right.

More than 10 million Americans have trouble distinguishing red from green or blue from yellow, and there's no treatment for colorblindness.

A biotech company and two scientists hope to change that.

On Wednesday, Avalanche Biotechnologies in Menlo Park and the University of Washington in Seattle announced a licensing agreement to develop the first treatment for colorblindness. The deal brings together a gene therapy technique developed by Avalanche with the expertise of vision researchers at the University of Washington.

"Our goal is to be treating colorblindness in clinical trials in patients in the next one to two years," says Thomas Chalberg, the founder and CEO of Avalanche.

Dalton the squirrel monkey during the color vision test. Courtesy of Neitz Laboratory hide caption

Dalton the squirrel monkey during the color vision test.

The agreement has its roots in a scientific breakthrough that occurred six years ago. That's when two vision researchers at the University of Washington used gene therapy to cure a common form of colorblindness in squirrel monkeys.

"This opened the possibility of ultimately getting this to cure colorblindness in humans," says Jay Neitz, who runs the Color Vision Lab at UW along with his wife, Maureen Neitz.

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University And Biotech Firm Team Up On Colorblindness Therapy

Gene therapy for CCALD
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Gene therapy for CCALD - Video

Lumber Liquidators Scandal with Dr Mitch Gaynor on Fox Business
Subscribe to the GeneChanger YouTube: http://goo.gl/jOPcep Dr Mitch Gaynor, Medical Oncologist at Cornell Medical Center Author of "Gene Therapy Plan" on Fox Business. The Gene Therapy...

By: Mitchell Gaynor M.D.

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Lumber Liquidators Scandal with Dr Mitch Gaynor on Fox Business - Video

Gene Therapy, Non-Viral

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Gene Therapy, Non-Viral - Video

Explaining gene therapy for wet AMD
Shows how wet macular degeneration destructs vision, how secretion gene therapy works and how people can benefit.

By: Elizabeth Rakoczy

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Explaining gene therapy for wet AMD - Video

The Alpha Clinic for Cell Therapy and Innovation | City of Hope
A new grant to City of Hope from the California Institute for Regenerative Medicine (CIRM) will make it possible for novel stem cell based therapies developed here at City of Hope to...

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The Alpha Clinic for Cell Therapy and Innovation | City of Hope - Video

Nobel Prize winners raise alarm over genetic engineering of humans.

A group of senior American scientists and ethics experts is calling for debate on the gene-engineering of humans, warning that technology able to change the DNA of future generations is now imminent.

In policy recommendations published today in the journal Science, eighteen researchers, including two Nobel Prize winners, say scientists should accept a self-imposed moratorium on any attempt to create genetically altered children until the safety and medical reasons for such a step can be better understood.

The concern is over a rapidly advancing gene-editing technology, called CRISPR-Cas9, which is giving scientists the ability to easily alter the genome of living cells and animals (see Genome Surgery). The same technology could let scientists correct DNA letters in a human embryo or egg cell, for instance to create children free of certain disease-causing genes, or perhaps with improved genetics.

What we are trying to do is to alert people to the fact that this is now easy, says David Baltimore, a Nobel Prize winner and former president of Caltech, and an author of the letter. We cant use the cover we did previously, which is that it was so difficult that no one was going to do it.

Many countries already ban germ line engineeringor changing genes in a way that would be heritable from one generation to the nextonethical or safety grounds. Others, like the U.S., have strict regulations that would delay the creation of gene-edited children for years, if not decades. But some countries have weak rules, or none at all, and Baltimore said a reason scientists were speaking publicly now was to keep people from doing anything crazy.

The advent of CRISPR is raising social questions of a kind not confronted since the 1970s, when the ability to change DNA in microrganisms was first developed. In a now famous meeting in 1975, in Asilomar, California, researchers agreed to avoid certain kinds of experiments that were then deemed dangerous. Baltimore, who was one of the organizers of the Asilomar meeting, says the scientists behind the letter want to offer similar guidance for gene-engineered babies.

The prospect of genetically modified humans is surprisingly close at hand. A year ago, Chinese researchers created monkeys whose DNA was edited using CRISPR (see 10 Breakthrough Technologies 2014: Genome Editing).

Since then,several teams of researchers in China, the U.S., and the U.K. have begun using CRISPR to change the DNA of human embryos, eggs, and sperm cells, with an eye toward applying the technology at in vitro fertility (IVF) clinics. That laboratory research was described by MIT Technology Review earlier this month (see Engineering the Perfect Baby).

Last week, in Nature, representatives of an industry group, the Alliance for Regenerative Medicine, recommended a wider moratorium that would also include a cessation of such laboratory studies, which it termed dangerous and ethically unacceptable (see Industry Body Calls for Gene-Editing Moratorium).

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Scientists Call for a Summit on Gene-Edited Babies

Gene Therapy Respiratory Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Respiratory Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape provides in depth insights into the Respiratory gene therapy. It has covered 5+...

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Gene Therapy Respiratory Insight: Trends and Challenges Analysed in Research Report - Video

Gene Therapy, Ex-Vivo and In-Vivo

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Gene Therapy, Ex-Vivo and In-Vivo - Video

Science Advances : Gene therapy rescues disease phenotype in a spinal muscular atrophy......
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model. Monica Nizzardo et al (2015), Scie...

By: KeSimpulan

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Science Advances : Gene therapy rescues disease phenotype in a spinal muscular atrophy...... - Video

Gene Therapy Gastrointestinal Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Gastrointestinal Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy ...

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Gene Therapy Gastrointestinal Insight: Trends and Challenges Analysed in Research Report - Video

Stamford, CT (PRWEB) March 16, 2015

Alliance for Cancer Gene Therapy (ACGT) the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer has achieved a major milestone, surpassing $25 million donated to innovative and breakthrough cancer research. ACGT was founded by Barbara Netter and her late husband, Edward, in 2001 with the goal of transforming cancer into a manageable, treatable disease.

Pushing the foundation across the $25 million threshold are a pair of three-year, $250,000 grants to two esteemed scientists: Meenakshi Hegde, MD, of Texas Childrens Cancer Center at Baylor College of Medicine in Houston, TX, and Christopher Jewell, PhD, at University of Maryland, College Park. Dr. Hegdes work will focus on immunotherapy, specifically adoptive cellular therapy for melanoma. Dr. Jewells research is centered on harnessing intra-lymph node gene therapy to promote tumor immunity. The grantees will develop genetically-modified T cells and cancer vaccines with the potential to stop cancer in its tracks.

Drs. Hegde and Jewell are two outstanding scientists in the vanguard of treating and defeating cancer, said Barbara Netter, ACGTs President. Their work offers tremendous hope to those battling cancer, and also to their loved ones.

ACGT grants are awarded to promising researchers whose work dovetails with the foundations mission: Leveraging cell and gene therapies to supplant the more harrowing cancer treatments like radiation, chemotherapy and surgery. ACGTs $25 million in grants have funded watershed research and trials such as those that activate patients own immune systems to battle cancer cells. These trials have saved the lives of cancer patients otherwise believed to be beyond treatment.

The two most recent grants continue ACGTs mission of equipping innovative scientists with the tools and support to revolutionize the fight against cancer. ACGT grants range from $250,000 to $1 million, and reward both young, promising researchers and their more established colleagues. Past recipients include such pre-eminent scientists as University of Pennsylvanias Dr. Carl June and Memorial Sloan-Ketterings Dr. Michel Sadelain; this past summer, the Food and Drug Administration (FDA) granted breakthrough status to immunotherapy treatments for leukemia developed by each of these scientists for which ACGT provided early funding.

About Alliance for Cancer Gene Therapy (ACGT) Established in 2001, ACGT (http://www.acgtfoundation.org) is the nations only not-for-profit dedicated exclusively to cell and gene therapy treatments for all types of cancer. One-hundred percent of contributions go directly to research. ACGT has funded 46 grants in the U.S. and Canada since its founding in 2001 by Barbara Netter, President, and her late husband, Edward, to conduct and accelerate critically needed innovative research. Since its inception, ACGT has awarded 31 grants to Young Investigators and 15 grants to Clinical Investigators, totaling more than $25 million in funding. ACGT is located at 96 Cummings Point Road, Stamford, CT 06902.

ACGT on Facebook: http://www.facebook.com/ACGTfoundation ACGT on Twitter: http://www.twitter.com/ACGTfoundation ACGT on YouTube: http://www.youtube.com/user/ACGTfoundation

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ACGT Surpasses $25 Million Funding Milestone with Two New Grants

Gene Therapy, Viral

By: Pedro Loureno

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Gene Therapy, Viral - Video

Gene Therapy Oncology Insight: Trends and Challenges Analysed in Research Report
Gene Therapy Oncology Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy profiles core.

By: James Jacob

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Gene Therapy Oncology Insight: Trends and Challenges Analysed in Research Report - Video

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. Thus far, the new therapy has proven safe.

"The result was stunning," said Timothy Nichols, MD, director of the Francis Owen Blood Research Laboratory at the University of North Carolina School of Medicine and co-senior author of the paper. "Just a small amount of new factor IX necessary for proper clotting produced a major reduction in bleeding events. It was extraordinarily powerful."

The idea behind gene therapy is that doctors could give hemophilia patients a one-time dose of new clotting genes instead of a lifetime of multiple injections of recombinant factor IX that until very recently had to be given several times a week. A new FDA-approved hemophilia treatment lasts longer than a few days but patients still require injections at least once or twice a month indefinitely.

This new gene therapy approach, like other gene therapy approaches, would involve a single injection and could potentially save money while providing a long-term solution to a life-long condition. A major potential advantage of this new gene therapy approach is that it uses lentiviral vectors, to which most people do not have antibodies that would reject the vectors and make the therapy less effective.

In human clinical studies, approximately 40 percent of the potential participants screened for a different kind of viral vector -- called adeno-associated viral vectors -- have antibodies that preclude them from entering AAV trials for hemophilia gene therapy treatment. This means that more people could potentially benefit from the lentivirus gene therapy approach.

Hemophilia is a bleeding disorder in which people lack a clotting factor, which means they bleed much more easily than people without the disease. Often, people with hemophilia bleed spontaneously into joints, which can be extremely painful and crippling. Spontaneous bleeds into soft tissues are also common and can be fatal if not treated promptly. Hemophilia A affects about one in 5,000 male births. These patients do not produce enough factor VIII in the liver. This leads to an inability to clot. Hemophilia B affects about one in 35,000 births; these patients lack factor IX.

This new method was spearheaded by Luigi Naldini, PhD, director of the San Raffaele Telethon Institute for Gene Therapy and co-senior author on the Science Translational Medicine paper.

For this study, Naldini and Nichols developed a way to use a lentivirus, which is a large retrovirus, to deliver factor IX genes to the livers of three dogs that have naturally occurring hemophilia. The researchers removed the genes involved in viral replication. "Essentially, this molecular engineering rendered the virus inert," Nichols said. "It had the ability to get into the body but not cause disease." This process turned the virus into a vector -- simply a vehicle to carry genetic cargo.

Unlike some other viral vectors that have been used for gene therapy experiments, the lentiviral vector is so large that it can carry a lot of payload -- namely, the factor IX genes that people with hemophilia B lack. (This approach could also be used for hemophilia A where the FVIII gene is considerably larger.)

These viral vectors were then injected directly into the liver or intravenously. After more than three years, the three dogs in the study experienced zero or one serious bleeding event each year. Before the therapy, the dogs experienced an average of five spontaneous bleeding events that required clinical treatment. Importantly, the researchers detected no harmful effects.

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New gene therapy for hemophilia shows potential as safe treatment