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Category Archives: Gene Therapy

FDA Awards Orphan Status To RetroSense's RST-001 For Retinitis Pigmentosa

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By Cyndi Root

RetroSense Therapeutics announced in a press release that the Food and Drug Administration (FDA) has granted Orphan Drug status to RST-001. The treatment for retinitis pigmentosa (RP) combines gene therapy and optogenetics. RetroSense developed the proprietary technology from research conducted at Wayne State University and Massachusetts General Hospital.

Sean Ainsworth, RetroSense CEO, said, We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa.

Optogenetics

Retinitis pigmentosa causes the degeneration and loss of rod and cone photoreceptors in the retina, causing severe vision loss and blindness. Currently there are no FDA-approved drugs to treat RP. RetroSenses work in optogenetics involves making the retina more light sensitive, thereby improving vision. The company expects RST-100 to have broad applications and to be useful in heredity or acquired RP.

RST-001 uses a photosensitivity gene, channelrhodopsin-2, and creates new photosensors in the retinal cells. Channelrhodopsin-2 has been shown in numerous animal studies to restore light perception and vision, and in primate studies, the agent was well tolerated. RetroSense is using optogenetics and channelrhodopsin-2 in the pre-clinical stage and hopes to begin clinical trials soon.

Retinitis Pigmentosa and Gene Therapy

Astellas and Harvard recently announced a new partnership to use gene therapy in the study for retinitis pigmentosa. The collaboration is led by Constance L. Cepko, a professor of Genetics and Ophthalmology at Harvard. Using adeno-associated virus vectors (AAVV), the team will identify and characterize genes implicated in RP.

The UKs Telegraph reported early in 2014 that researchers at Oxford University have replaced a missing gene in the retina and reversed blindness. The results startled the investigators who did not expect to see such dramatic improvements. In the study on choroideremia, inherited blindness, scientists put the missing REP-1 protein back in the retina by inserting it into the DNA of a harmless virus and then injecting that DNA into the cells beneath the retina.

Since, a third of eye diseases are hereditary, the researchers are hopeful that the treatment is applicable to various eye diseases and conditions. The research team at Oxford is developing a Phase 2 trial on the investigational therapy.

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FDA Awards Orphan Status To RetroSense's RST-001 For Retinitis Pigmentosa

Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

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CLEVELAND and COLUMBUS, Ohio >> Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle of a gene therapy to increase muscle strength and function in Becker muscular dystrophy patients. The paper, titled "A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy" describes results of the dose ascending trial to assess safety and efficacy in six patients with Becker muscular dystrophy.

All six of the patients were followed at least six months and three of the patients were followed for one year with no drug-related adverse events reported. On average, the patients showed statistically significant improvement in six minute walk distance and in stair climbing tests. Additionally, muscle biopsy histology demonstrated reductions in muscle fibrosis and central nucleation, and improved muscle fiber regeneration. Muscle satellite cells were not reduced.

Jerry Mendell, M.D., Professor at Nationwide Children's Hospital and lead author on the trial and manuscript stated, "This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease." Brian Kaspar, Ph.D., Associate Professor at Nationwide Children's Hospital, author and compensated advisor and scientific founder of Milo said, "Upon evaluating the top line data in this trial, we are particularly impressed with the effects of our gene therapeutic to express follistatin long term, not only for the functional improvement, but also in improving the state of muscle health in the patients analyzed."

The therapy, developed at Nationwide Children's Hospital by Dr. Mendell and Dr. Brian Kaspar, is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting. It could have broad ranging applications in muscular dystrophies, cancer treatment induced muscle wasting and age related muscle wasting. The clinical study, conducted at Nationwide Children's Hospital and funded by the foundation Parent Project MD, is also evaluating safety and efficacy in nine patients with inclusion body myositis. Results of the trial in inclusion body myositis will be published in mid 2015.

The underlying intellectual property was exclusively licensed in 2012 from Nationwide Children's Hospital to Ohio-based start-up Milo Biotechnology. Milo Biotechnology has received FDA orphan designation for Becker muscular dystrophy and for Duchenne muscular dystrophy.

Milo Biotechnology CEO Al Hawkins stated "This significant and long lasting clinical effect provides the basis for planning pivotal clinical studies beginning in 2015; we want to speed delivery of this important therapy to patients with debilitating muscular dystrophies."

About Milo Biotechnology Milo Biotechnology is a clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases. Its lead program, AAV1-FS344, leads to the local expression of follistatin, a potent myostatin inhibitor. Milo's core technology was developed at and is exclusively licensed from Nationwide Children's Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and from NIAMS Rare Disease SBIR program. Visit http://www.milobiotechnology.com for more information

CONTACT: infomilobiotechnology.com

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Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

BUSINESS WIRE: Olivier Danos Joins Biogen Idec as Senior Vice President, Gene Therapy

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MITTEILUNG UEBERMITTELT VON BUSINESS WIRE. FUER DEN INHALT IST ALLEIN DAS BERICHTENDE UNTERNEHMEN VERANTWORTLICH.

Distinguished Scientist To Lead Companys Gene Therapy Research Efforts

CAMBRIDGE, Mass. --(BUSINESS WIRE)-- 23.09.2014 --

Biogen Idec (NASDAQ:BIIB) today announced that Olivier Danos, Ph.D., has joined the company as senior vice president, Gene Therapy. Dr. Danos reports directly to Douglas Williams, Ph.D., executive vice president, Research and Development, and is a member of Biogen Idecs Senior Research and Development Leadership Team.

Dr. Danos will lead the companys gene therapy research group, a team dedicated to identifying and developing new technologies for gene transfer and genome engineering. As part of his role, Dr. Danos will also identify potential collaborations that complement the companys internal capabilities and support the development of treatments across Biogen Idecs therapeutic focus areas.

Olivier is a true pioneer in the field. We believe that his expertise will help accelerate our development of a world-class gene therapy platform that combines our growing internal capabilities with collaborations with leading research organizations across the globe, said Dr. Williams. Our commitment to this important area will complement our existing capabilities in antisense, small molecule and biologics research, and enhance our ability to effectively and efficiently develop new treatments for people with serious diseases.

I have dedicated my career to advancing the use of gene therapy as a technology to develop life-saving therapies for patients, said Dr. Danos. The team at Biogen Idec shares this passion. It is their commitment to innovative science and willingness to tackle difficult medical challenges that attracted me to the company. I am excited about the opportunities before us.

Dr. Danos joins Biogen Idec from Kadmon Pharmaceuticals, where he served as senior vice president, Molecular Medicine, Synthetic Biology and Gene Regulation since 2011. In this role, he was instrumental in assembling a gene therapy program and a technology platform for the development ofcontrollablegene expression systems. Prior to Kadmon, Dr. Danos acted as the director of the Gene Therapy Consortium of the University College of London and led a gene therapy research team at the Necker Hospital Enfants Malades in Paris.

Dr. Danos also served as chief scientific officer at Genethon and senior director of research at Somatix Therapy Corporation. He has held several senior roles at the French National Centre for Scientific Research (CNRS) and at the Institut Pasteur in Paris. Dr. Danos is the former president and a founding member of the European Society of Gene and Cell Therapy.

Dr. Danos received a Masters in Genetics and Molecular Biology at University of ParisOrsay, and his Ph.D. in Biology at the Pasteur Institute and University of Paris Diderot.

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BUSINESS WIRE: Olivier Danos Joins Biogen Idec as Senior Vice President, Gene Therapy

Gene therapy, stem cell therapy trials underway

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Stem cells and gene hold promising treatment options for Parkinson's, mandate doctors across the globe, including from Mumbai. Eleven trials to test stem cell and gene therapy for treating Parkinson's are underway currently of which the one in Mumbai had to be put on hold due to regulatory hurdles.

Currently, neuro-augmentative therapies such as usage of drugs or deep brain stimulation (DBS) are being used to treat Parkinson's disorder. "The future holds hope for neuro-restorative therapies like that of stem cells or gene infusion in the Parkinson's disorder treatment. It involves restoration of brain function to normal. In the next five to seven years, this may pave the way for future," said Dr Paresh Doshi, neurologist at Jaslok Hospital, Peddar Road in Mumbai.

Regulatory hurdles and resource constraints though have led to these trials being held up in Mumbai. Dr Doshi said that trials of Duodopa therapy which involves infusion of an active ingredient gel called Levodopa in the intestines has been kept on hold at the moment at privately-run Jaslok Hospital due to regulatory hurdles. The hospital was the only centre in entire South East Asia to have been running the trial.

"Levodopa gets converted into dopamine in the body. Normal levels of dopamine control Parkinsons disorder," said Dr Doshi.

Trials to infuse stem cells from the patient's body in the patient itself had been underway in small group of patients in India, but due to inability to recruit more patients, the trial was stopped. "We could only recruit four patients for two years. However, a similar trial is underway in China and another trial which explores adipose tissue stem cells in treating Parkinson's disease is underway in South Africa," said Dr Doshi.

In January this year, medical journal The Lancet reported that after sixteen years of trials, gene therapy is showing promising results in humans. "Three genes that promote the formation of dopamine generating cells in the brain were injected in the brain bound with a viral vector in fifteen patients. The genes are intended to boost the production of dopamine, a chemical that becomes deficient in patients withParkinson's," said The Lancet report.

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Gene therapy, stem cell therapy trials underway

Plenary Session: Gene Therapy & Gene-Modified Cell Therapies – Video

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Plenary Session: Gene Therapy Gene-Modified Cell Therapies
This session will bring together several of the leading in-vivo and ex-vivo gene therapy companies in addition to pharma companies making a large bet in gene-modified cell therapy. While the...

By: Alliance for Regenerative Medicine

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Plenary Session: Gene Therapy & Gene-Modified Cell Therapies - Video

MPs to vote 'soon' on new gene therapy – pioneered by Newcastle University team

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MPs will vote soon on a new gene therapy to stop incurable diseases passing to babies ending fears the Government is ducking the controversy.

The health minister threw her weight behind a DNA-altering procedure pioneered by a team at Newcastle University, saying: This is something I want to take forward.

Jane Ellison told a parliamentary inquiry: I am now actively seeking cross-Government approval for parliamentary time in this session to bring regulations before the House.

Im extremely conscious that there are real families waiting on progress on this work. We need to keep up the momentum.

The comments are a huge boost to the Newcastle team, which has called for legislation as soon as possible, because of the number of patients waiting for treatment.

Also appearing before the Commons science select committee, the teams Professor Doug Turnbull said the worst-affected babies died within 24 or 48 hours.

He said most diseases develop in childhood, or adolescence, adding: We can do a lot to help with epilepsy and diabetes, but there is no cure.

And, on the gene therapy, This sort of approach to try to present a transmission of these sorts of diseases - would be really important.

The treatment involves replacing faulty mitochondria responsible for inherited diseases, including muscle wasting, heart problems, vision loss, organ failure and epilepsy.

Embryos are given healthy DNA from donor eggs, meaning a baby has the DNA of three people from two parents, plus less than one per cent from the donor.

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MPs to vote 'soon' on new gene therapy - pioneered by Newcastle University team

With Collaboration, Scientists Test Gene Therapy for 'Bubble Boy Disease'

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A new variation of gene therapy raises hopes for a safe and effective long-term treatment for X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening heritable disorder.

The research was produced by a collaborative research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center, along with other institutions participating in an international clinical trial that involved boys from the United States and France.

SCID-X1, dubbed bubble boy disease after a patient who lived for 12 years in a sterile bubble, is a rare genetic disorder that hinders the ability of individuals to combat infections. Because the disease is carried in an X-chromosome recessive pattern, the disorder occurs almost only in males. The resulting mutations inactivate a gene called IL-2 receptor gamma (IL2RG), severely weakening immune system functions. Left untreated, individuals who inherit the disorder usually die within a year.

Previous gene therapy trials conducted in Europe over a decade ago promised dramatic progress, until a quarter of patients developed leukemia about two to five years following treatment. Scientists found that the previously used vectorthe device for transporting the correct gene in therapyinadvertently activated oncogenes, which can cause cancer.

In this new study, the vector in use is a self-inactivating gammaretrovirus, which has a specific sequence deleted that basic research had implicated in the process of inappropriate activation of oncogenes, David A. Williams, chief of the hematology/oncology department at Boston Children's Hospital, wrote in an email.

Of the nine patients who underwent the treatment, eight had survived between 12 and 38 months after treatment. One boy died from a severe infection he was fighting at the time he enrolled in the study.

A single round of therapy restored normal disease-fighting T cell count300 cells or more per microliter of bloodin six of the eight patients. One patient underwent a second round of treatment and remains healthy despite a low cell count. The eighth patient received a hematopoietic stem cell transplant after the therapy led to less than optimal uptake of the virus and failed to stimulate T-cell production, according to Williams.

We feel the surrogate assays for safety look excellent and are very encouraged, Williams said. However, because leukemia can take years to develop (and although some of our patients are now approaching 4 years of [follow-up]) we must be cautious and continue to follow these children closely.

Williams noted that the research was the result of positive collaboration between institutions.

Work by Sung-Yun Pai and Gigi Notarangelo, funding from [Boston Childrens Hospital] (and other childrens hospitals) and [the National Institute of Health] were essential for success, he said. This is the first international collaborative trial in stem cell gene therapy, which was critical for success due [to the] rarity of [this] disease.

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With Collaboration, Scientists Test Gene Therapy for 'Bubble Boy Disease'


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