Category Archives: Genetic Therapy

By Jonathan D. Rockoff

If you havent heard of Stargardt disease, its not because it isnt serious.

The condition results in blindness, theres no approved treatment and there are few options for delaying the damage it progressively wreaks. But it only afflicts 1 out of every 10,000 children, or some 25,000 Americans, according to the American Macular Degeneration Foundation.

Nevertheless, research into a remedy is under way, spurred by the discovery, in 1997, of a gene defect that appears to play an important role in the diseases genesis. Further study is shining a light into how it operates.

The research is providing some hope that Stargardt sufferers could gain an effective treatment, like patients with another rare and previously intractable disorder caused by a single gene defect: cystic fibrosis.

I was struck with the parallels, says Joshua Boger, the founder and retired CEO of Vertex Pharmaceuticals, which recently launched a treatment for a rare form of cystic fibrosis and is working on another for a more common form of the disease.

Boger today becomes executive chairman of one of the small firms looking into a potential Stargardt therapy, signaling his confidence in the promise of research into a treatment.

The firm hes joining is Alkeus Pharmaceuticals, which expects to start studying a potential Stargardt treatment in humans later this year, or early next. Others looking into a possible remedy are Advanced Cell Technology and Oxford BioMedica, supported by the Foundation Fighting Blindness.

Its really below the radar of the big companies, so without an entrepreneur, without someone pushing this, it wasnt going to get attention, Boger says.

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Lessons From One Genetic Disorder Raise Hopes for Treating Another, Rarer One

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

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Family hangs hope for boy on unproven therapy in India

Zee News

Jaya steps up campaign for Sangma, speaks to non-Cong leaders Economic Times CHENNAI: Lobbying for PA Sangma's candidature for the presidential polls, Tamil Nadu Chief Minister Jayalalithaa today spoke to BJP leader LK Advani, Samajwadi supremo Mulayam Singh Yadav and other non-Congress leaders including Left parties. Prez polls: Jaya steps up campaign for Sangma,speaks to non-Cong leadersIndian Express Prez polls: Jaya steps up campaign for SangmaHindustan Times Presidential polls: Jayalalithaa steps up lobbying for SangmaTimes of India IBNLive.com -The Hindu -Daily News & Analysis all 71 news articles »

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The Guardian

Pakistan Blocks Twitter Over Cartoon Contest New York Times ISLAMABAD, Pakistan — The Pakistani government blocked access to the social networking service Twitter on Sunday, after publicly holding Twitter responsible for promoting a blasphemous cartoon contest taking place on Facebook, officials said. Pakistan blocks Twitter amid blasphemy fearsThe Guardian Authorities temporarily ban Twitter in PakistanLos Angeles Times Pakistan blocks Twitter access over 'blasphemous content'Economic Times TIME -AFP -Reuters India all 480 news articles »

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http://news.google.co.in/news?pz=1&ned=in&hl=en&q=Genetic+Therapy&output=rss

Zee News

IPL live score: Rajasthan elect to bat vs Mumbai Daily News & Analysis This match is a dead rubber as Kolkata's victory yesterday ensured them the 2nd place in the points table. Mumbai are coming off a big loss in their last match against Kolkata and will be looking for a morale boosting win ahead of the play-offs. IPL 5 live score: Mumbai Indians vs Rajasthan RoyalsZee News RR vs MI: Kieron Pollard gives Mumbai Indians crucial breakthroughTimes of India Rajasthan bat in final league gameESPNcricinfo.com Indian Express -Cricketnext.com all 74 news articles »

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http://news.google.co.in/news?pz=1&ned=in&hl=en&q=Genetic+Therapy&output=rss

MCLEAN, Va and BUDAPEST, Hungary, May 17, 2012 (GLOBE NEWSWIRE) -- Genetic Immunity Inc., a privately-held, biopharmaceutical company developing Langerhans cell-targeted immunotherapies, published its human proof-of-concept clinical trial, in PLoS ONE, a highly-regarded medical industry publication. The data demonstrated the induction of long-lasting memory T cell responses after a single immunization of DermaVir, the Company's lead vaccine candidate for patients infected with the human immunodeficiency virus (HIV). The publication is free to access in http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0035416.

"The results of our clinical trial confirmed our preclinical safety and immunogenicity data," said Genetic Immunity Founder, President & Chief Science Officer Julianna Lisziewicz, PhD. "These central memory T cell responses are associated with the protection from infectious diseases such as HIV, Hepatitis C and malaria. Our findings demonstrate that our Langerhans cell-targeted delivery system is suitable to induce broad and potent T cell immunity."

Regarding the result of the study, Denes Banhegyi, MD, the principal investigator of the study, added, "Decades of drug treatment can neither eliminate HIV-infected cells from the reservoirs nor reconstitute HIV-specific immunity. A broad epitope pool is key to long-term, non-progression. With a DermaVir immunization, patients can have hope that their daily drug burden could be lessened."

About the GIHU004 study:

The dose escalation study, labeled GIHU004, was conducted with three (3) topical DermaVir doses targeted to epidermal Langerhans cells to express fifteen (15) HIV antigens simultaneously in several draining lymph nodes. Antigen presentation in the lymph nodes is required to boost the immune system. Safety, tolerability and HIV-specific T cell responses against five (5) of the fifteen (15) DermaVir antigens were evaluated.

DermaVir-associated side effects were not dose-dependent and limited to mild, transient skin reactions. One of the more noteworthy results of the study was the dose-dependent expansion of HIV-specific central memory T cells with high proliferation capacity. The potent induction of Gag, Tat and Rev-specific memory T cells suggests that DermaVir boosts T cell responses specific to all 15 HIV antigens expressed from the single DNA. This is the first time that a vaccine induces memory T cell responses against all fifteen (15) antigens in human subjects.

About the DermaVir HIV Therapeutic Vaccine:

Today, HIV/AIDS is managed with life-long highly active antiretroviral therapy (HAART). This treatment, although widely accepted as the standard, raises concerns about adverse effects, resistance and the financial burden of treatment. A short fall of HAART is that it does not cure the disease because it does not kill infected cells in the latent reservoirs. Based upon our trials, DermaVir boosts and maintains killer T cells in HIV-infected human subjects. It has the broadest specificity in vaccine candidates that limits the development of resistance. Three (3) independent clinical trials, conducted in the USA and EU have consistently demonstrated the safety, immunogenicity and preliminary antiviral efficacy of DermaVir treatments.

About Genetic Immunity:

Genetic Immunity Inc., is a privately-held, U.S. biopharmaceutical company that has developed a proprietary Langerhans cell targeting delivery system which can induce long-lasting T cell immunity for therapeutic vaccinations against infectious diseases and cancer. Our proprietary system delivers a genetically targeted nanomedicine formulation that can simultaneously express 15 disease-specific antigens. All of our medicines are delivered topically using our CE-marked medical device, DermaPrep(TM).

Excerpt from:
Genetic Immunity Clinical Trial Data Published in PLoS ONE