Prof Wang Bin - Bio Gene Therapy
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Category Archives: Gene Therapy
Anna David, Prenatal Cell and Gene Therapy – Video
Anna David, Prenatal Cell and Gene Therapy
Anna David, Prenatal Cell and Gene Therapy (Research Department of Maternal and Fetal Medicine, UCL EGA Institute for Women #39;s Health) talks about her group #39;s...
By: UCL EGA Institute for Women #39;s Health
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Friedmann wins Japan Prize for gene therapy
Dr. Theodore Friedmann is a longtime faculty member at UC San Diego and a pioneer in gene therapy. / photo by Nelvin C. Cepeda * U-T San Diego
Dr. Theodore Friedmann, a pioneer in the booming field of gene therapy, has been named a 2015 winner of the prestigious Japan Prize.
A pediatrician-turned-researcher at UC San Diego, Friedmann is renowned for demonstrating in the lab that it is possible to correct a genetic defect by adding a functional gene to defective cells, a feat he and colleagues accomplished in 1968. Since then, Friedmann has been guiding the young science through controversies, ethical challenges and setbacks.
Friedmann shares the prize in "medical science and medicinal science" with Dr. Alain Fischer of the Necker Hospital in Paris, France. Fischer helped demonstrate gene therapy's clinical ability to treat a genetic immune deficiency that makes patients extremely vulnerable to infections.
Along with the recognition, Friedmann and Fischer will split a $416,600 award, a certificate and gold medal. There's also the prospect of future recognition: several Japan Prize winners have gone on to win the Nobel Prize.
Friedmann is known not only as a scientist who demonstrated gene therapy is possible, but as a thinker who has dampened the waves of excessive exuberance and despondency that often accompanies the passage of research discoveries into therapies. He has also cautioned his fellow scientists to approach gene therapy with great caution.
In 1972, Friedmann co-authored an influential article in the journal Science, "Gene therapy for human genetic disease?" proposing a program of research advancement and safety precautions with an eye to eventual therapy. In February, 2010, he coauthored an article in Science about the potential use of performance-enhancing "gene doping" in sports.
Those who didn't heed Friedmann's warnings ran into trouble. For example, in 1999 gene therapy patient Jesse Gelsinger, 18, died due to an immune reaction. Gelsinger had a mild form of a genetically caused liver disease, controlled with drugs and diet. He was enrolled to test a treatment to be used in babies with a fatal form of the disease. But Gelsinger himself had little to gain.
A mountain of bad publicity threatened to sink the field. The New York Times wrote about "The Biotech Death of Jesse Gelsinger." As a consequence, other new forms of therapy, such as stem cell treatments, have progressed more slowly to avoid a repeat.
The Gelsinger disaster has receded into the background, as safer forms of gene therapy edge closer to becoming an accepted part of medicine. Forms of gene therapy are now being tested in clinical trials to treat such different diseases as cancer, sickle cell anemia and HIV, with impressive results.
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Friedmann Named 2015 Japan Prize Winner
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Newswise Theodore Friedmann, MD, professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose original and outstanding achievements in science and technology have advanced the frontiers of knowledge and served the cause of peace and prosperity for mankind.
Friedmann is being recognized for his pioneering research and contributions to the development of gene therapy, a new field of medicine which in significant ways originated at UC San Diego. The sponsoring Japan Prize Foundation describes Friedmann as the father of gene therapy.
Sharing the 2015 Japan Prize in the field of medical science and medicinal science with Friedman is Alain Fischer, MD, PhD, director of immunology at the Necker Hospital in Paris, France. Fischer is credited with demonstrating the clinical efficacy of gene therapy by successfully treating children suffering from a severe genetic disorder that renders them extremely vulnerable to infections.
The third 2015 Japan Prize laureate is Yutaka Takahasi, PhD, professor emeritus at the University of Tokyo, who is being honored in the field of resources, energy and social infrastructure for his contributions to river basin management and reducing water-related disasters.
Each laureate will receive a certificate of recognition and commemorative gold medal. A cash award of approximately $416,600 will also be given to each prize field. Since its inception in 1985, 83 laureates from 13 countries have received the Japan Prize in a variety of fields and disciplines. Several have subsequently become Nobel Prize laureates as well.
In 1972, Friedmann, then a visiting scientist at the Salk Institute for Biological Sciences in La Jolla, and Richard Roblin, also at the Salk Institute, published a foundational article in the field, a paper in the journal Science under the heading Gene therapy for human genetic disease?
The idea of gene therapy, which quickly captured the public imagination, was fueled by its appealingly straightforward approach and what Friedmann has described as its obvious correctness: Disarm a potentially pathogenic virus to make it benign. Stuff these viral particles with normal DNA. Then inject them into patients carrying abnormal genes where they will deliver their therapeutic cargoes inside the defective target cells. In theory, the good DNA replaces or corrects the abnormal function of the defective genes, rendering previously impaired cells whole, normal and healthy. End of disease.
Its not quite that simple, of course, something Friedmann and Roblin cautioned in their 1972 paper. Despite progress in the understanding of cellular functions, the roles of DNA and a series of experimental and clinical advances, the history of gene therapy has been marked by distinct highs and lows.
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Recombinant Coagulation Factors 2015: Maturation Of Recombinant Clotting Factor Pipeline And Emergence Of Gene Therapy …
DUBLIN, January 27, 2015 /PRNewswire/ --
Research and Markets (http://www.researchandmarkets.com/research/pfd99m/recombinant) has announced the addition of the "Recombinant Coagulation Factors 2015: Maturation Of Recombinant Clotting Factor Pipeline And Emergence Of Gene Therapy And Alternative Procoagulants" report to their offering. (Logo: http://photos.prnewswire.com/prnh/20130307/600769 )
A Pipeline Landscape Analysis and Comparative Assessment of Key Players
This report provides an update of recombinant coagulation factors used for controlling bleeding of hemophilia A and B and other bleeding disorders. 2013 sales figures and 2014 nine-month sales data are analyzed to evaluate commercial development of the market under the light of a strong pipeline and entry of new competitor products into the market. The profiles of drug candidates in development are presented in details not only for recombinant coagulation factors VIII, IX and VII, but also for emerging alternative procoagulants, gene therapeutics and immune tolerance inducing agents.
The competitve landscape of classical recombinant coagulation factors and of new emerging treatment modalities of hemophilia and other severe bleeding orders is analyzed. The emergence of new treatment modalities brings many new stakeholders to the field of hemophilia. Among them are the established hemophilia portofolio companies, but also new entrants from Big Pharma and Big Biotech, specialty pharmaceutical companies, biosimilar companies, and new technology providers as half-life prolongation technologies are no longer in the focus of major interest.
A short- to mid-term outlook into the field is provided and the trends driving the future of this therapeutic segment are identified and described. Preparation of this report is based on information stored in La Merie Publishing's proprietary data base and news archive, on scientific literature, and on corporate disclosures. The data are presented, analyzed and assessed in a comprehensive manner by an experienced author with an independent view writing the fifth edition of this report series.
Key Topics Covered:
1. Executive Summary
2. Recombinant Coagulation Factor Markets 2.1 Recombinant Factor VIII (rFVIII) Product Sales and Market Size 2.2 Recombinant Factor IX (rFIX) Product Sales and Market Size 2.3 Recombinant Factor VII (rFVII) Product Sales and Market Size 2.4 Recombinant Thrombin Product Sales and Market Size 2.5 Total Recombinant Coagulation Factor Market Size
3. Pipeline Update of Recombinant Coagulation Factors 3.1 Factor VIII Pipeline Changes and Drug Profile Updates 3.2 Factor IX Pipeline Changes and Drug Profile Updates 3.3 Factor VII Pipeline Changes and Drug Profile Updates 3.4 Pipeline Update for Other Recombinant Coagulation Factors 3.5 Pipeline Update for Alternative Procoagulants 3.6 Pipeline Update for Gene & Cell Therapy 3.7 Immune Tolerance Inducing (ITI) Agents
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lily and america gene therapy – Video
lily and america gene therapy
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Gene therapy trial for thalassemia at Royal Prince Alfred Hospital – Video
Gene therapy trial for thalassemia at Royal Prince Alfred Hospital
A world first gene therapy trial is currently underway at Royal Prince Alfred Hospital with the potential to cure patients living with thalassemia. Patient Stacey Wong has required long frequent...
By: SydneyLHD
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Gene therapy trial for thalassemia at Royal Prince Alfred Hospital - Video
Flagship Journal Current Gene Therapy – Video
Flagship Journal Current Gene Therapy
A short promotional video of Bentham Science High Impact Journal Current Gene Therapy.
By: Bentham Science Publishers
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Aarkstore -Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape – Video
Aarkstore -Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape
Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy profiles core insights,...
By: sangam Jain
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Switzerland: The Elixir of Life? Scientists COULD make you live to 120! – Video
Switzerland: The Elixir of Life? Scientists COULD make you live to 120!
Researchers from Bern University may have found gene therapy that can extend life by 60 percent. At the moment, the experiment is limited to fruit flies, who have just been given their third...
By: RuptlyTV
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Switzerland: The Elixir of Life? Scientists COULD make you live to 120! - Video
Welby: Artificial Intelligence and gene therapy could hand super-rich ever more power
He added that one passage in the Bible, the Magnificat or the Song of Mary is so revolutionary he was surprised it was not banned as un-American during the McCarthy era.
His comments came as he took part in a conference at the Trinity Wall Street church in New York discussing the idea of the common good.
In an address on whether inequality matters, he argued that a message of basic equality can be traced through the Bible from the Garden of Eden to the New Testament accounts of the early church.
He said that although wealth is, in some parts of the Bible, viewed as a blessing, in others it is linked to corruption but that overall there was no right to be rich.
He added that some people had labelled passages in the Book of Acts, talking about wealth being shared between the early Christians, as communist but said this was untrue because the common ownership in the Bible was voluntary not obligatory.
Looking ahead to the next 40 years, he said: In an era in which we will see the growth of technologies like Artificial Intelligence and gene therapies, economists like Lawrence Summers foresee growing inequalities between the small minority who can maximise the benefits of new technology and the large majority who will see only stagnation in income.
We face the challenge of a society in which inequality of education or health or opportunity becomes and continues to be a life sentence to poverty.
And that is the challenge which is exactly the one that we find the prophets so concerned about.
Mr Summers wrote last year that the devastating consequences of robots and technologies like 3D printing replacing human workers would become the main story in economics and politics in years to come.
The Archbishop said: The theological understanding is that wealth is always in danger of corrupting its holders in most cases, and the corrupted become too powerful.
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Welby: Artificial Intelligence and gene therapy could hand super-rich ever more power
Minecraft: Attack of the B Team, Ep. 51 Gene therapy! – Video
Minecraft: Attack of the B Team, Ep. 51 Gene therapy!
In this episode of Attack of the B Team we put our advanced genetics knowledge to use, not good use, just use.... Really the best use for dna? https://www.fa...
By: Dr. Trotter
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Minecraft: Attack of the B Team, Ep. 51 Gene therapy! - Video
Methylmalonic Acidemia (MMA) Gene Therapy – Charles Venditti and Randy Chandler – Video
Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler
Dr. Charles Venditti and Dr. Randy Chandler discuss recent developments in gene therapy for methylmalonic acidemia (MMA) - a group of inherited disorders in which the body is unable to process...
By: GenomeTV
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Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler - Video
Regenxbio nets $30M to bring its gene therapy to clinical trial
Washington, D.C.-basedgene therapy outfit Regenxbio just raised $30 million to bringits platforminto the clinic.
The dollars will help Regenxbio generate clinical proof of concept data, as well as work toward in-licensing new programs. The companys also using the money to beef up its clinical and manufacturing processes, it said in a statement.
Regenxbio has developed what it calls NAV Technology a form of adeno-associated viral gene therapy that treats lysosomal storage disorders and ocular disease. Its got drugs in the pipeline that treat Hurler syndrome, Hunter syndrome, wet age-related macular degeneration and X-linked retinitis pigmentosa.
Heres how it works: In Hurler syndrome, for instance, children dont carry a gene that develops an enzyme called IDUA that breaks down complex sugars. These build up, and ultimately impairmental development, organ function, physical abilities and appearance. The NAV platform delivers a normal copy of the IDUA-producing gene which ultimately embeds itself into a patients DNA in a one-time doze so that patients can produce the enzyme. The research comes out of the University of Pennsylvania, and has been successful in vivo so its a matter of testing its efficacy in a real patient pool.
The companys also out-licensing this technology for other indications, with companies like Baxter, Dimension Therapeutics and Lysogene developing viral vector-based gene therapy for a number of other indications.
The Series C funding was led by Venrock and Brookside Capital, with other investors like Deerfield Management, FoxKiser and Fidelity Biosciences.
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Regenxbio nets $30M to bring its gene therapy to clinical trial
U.S. Gene Therapy Clinical Trial to treat Choroideremia initiated
PHILADELPHIA, Penn., January 20, 2015- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today it has initiated a Phase 1/2 clinical trial for the potential treatment of patients with choroideremia (CHM) utilizing its gene therapy product SPK-CHM. The Phase 1/2 trial is an open-label, dose-escalating trial designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The Phase 1/2 trial will be conducted at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania, and has plans to enroll up to 10 patients afflicted with the CHM genetic mutation.
"Spark's groundbreaking announcement today brings real hope of a treatment for blindness caused by choroideremia, and promises to pave the way for treatments of other retinal diseases impacting millions of people around the World," said Dr. Chris Moen, President of the Choroideremia Research Foundation (curechm.org), the leading advocacy and fundraising organization focused on finding a cure for CHM. "The Choroideremia Research Foundation is proud to have provided key pre-clinical funding to Jean Bennett, MD, PhD and her team atthe Perelman School of Medicine at the University of Pennsylvania,that has helped bring us to the gene therapy human clinical trials being announced today."
Dr. Jean Bennett's pre-clinical work, which was funded in part by consistent financial support from the Choroideremia Research Foundation (curechm.org), demonstrated the ability of the SPK-CHM gene therapy to restore REP-1 protein production, membrane trafficking and retinal structure.
"Throughout my career's work developing genetic therapies for inherited retinal dystrophies I have had my target set on a number of different conditions, in particular, choroideremia," said Dr. Bennett, who is also one of Spark's scientific co-founders and a scientific advisor on the SPK-RPE65 clinical trials being conducted at CHOP. "The SPK-CHM program, for the first time, creates the potential for patients to use their vision for longer and see more things."
In addition to evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify appropriate endpoints for subsequent clinical trials. With SPK-CHM, Spark is leveraging the experience and technology utilized in the development of its gene therapy for Leber's Congenital Amaurosis (LCA), SPK-RPE65, including the same vector, target cells and route of administration, as well as the same manufacturing process. SPK-RPE65 is currently in a fully-enrolled pivotal Phase 3 clinical trial.
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U.S. Gene Therapy Clinical Trial to treat Choroideremia initiated
Gene Therapy: Hope For The Blind? – Video
Gene Therapy: Hope For The Blind?
In medicine, doctors often talk about treatments, but rarely about cures. Now, gene therapy is offering the potential to actually cure certain diseases and an experimental breakthrough could...
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Gene therapy-associated cancer incidence depends on vector design
Gene therapy is a promising strategy to correct hereditary disorders. The approach takes advantage of viral vectors to deliver a corrected version of the mutated gene. Adeno-associated virus (AAV) has many features that make it a favorable vector for gene therapy. In animal models, AAV-mediated gene delivery is generally regarded as safe and has demonstrated efficacy for some genetic diseases. However, a recent study reported an increase in liver cancer in mice after AAV gene therapy. A new publication in the Journal of Clinical Investigation reveals that AAV vector design influences the likelihood of developing cancer in the liver. Charles Venditti and colleagues at the National Institutes of Health looked for the development of hepatocellular carcinoma (HCC) in a large number of mice that had received AAV gene therapy. HCC was associated with the AAV vector integrating within a specific site in the genome and inducing expression of microRNAs and a retrotranposon. Moreover, AAV dose, the choice of enhancer/promoter, and timing of delivery all influenced the HCC incidence. The results of this study provide insight into features that should be considered when designing AAV vectors for gene therapy.
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TITLE:
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
AUTHOR CONTACT:
Charles Venditti National Institutes of Health, Bethesda, MD, USA Phone: 301-496-6213; Fax: 301-451-3853; E-mail: venditti@mail.nih.gov
View this article at: http://www.jci.org/articles/view/79213?key=0ff2890aa2b1c1b59bca
Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.
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Gene therapy-associated cancer incidence depends on vector design
NIH researchers tackle thorny side of gene therapy
Pre-clinical studies in mice reveal ways to reduce cancer risk with modified treatment
National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.
"Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children," said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. "This study is an important step in developing gene therapies that can be safely used to benefit patients."
Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell's DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are uniquely suited for gene therapy applications.
But one prior study did find an association between AAV and the occurrence of liver cancer. The present research addresses this problem in gene therapy for an inherited disease in children called methylmalonic acidemia, or MMA.
For 10 years, NHGRI researchers have worked toward a gene therapy to treat MMA. The condition affects as many as 1 in 67,000 children born in the United States. Affected children are unable to properly metabolize certain amino acids consumed in their diet, which can damage a number of organs and lead to kidney failure. MMA patients also suffer from severe metabolic instability, failure to thrive, intellectual and physical disabilities, pancreatitis, anemia, seizures, vision loss and strokes. The most common therapy is a restrictive diet, but doctors must resort to dialysis or kidney or liver transplants when the disease progresses.
In prior MMA gene therapy studies, researchers showed that mice bred to develop the condition could be restored to health by AAV gene therapy injection shortly after birth. The mice in the study survived into adulthood and were free from the effects of MMA.
"The corrected gene delivered by AAV is the most effective therapy we have developed so far to treat MMA," said Charles Venditti, M.D., Ph.D., senior author and investigator in NHGRI's Genetic and Molecular Biology Branch. "However, we have identified an important safety parameter related to the AAV gene therapy in our mouse models that is critical to understand before we move to human patient trials."
Now, in a long-term follow-up of the treated mice -- after mice reached about two years of age -- the researchers documented a 50-70 percent higher occurrence of liver cancer in AAV-treated mice compared with a 10 percent liver cancer rate in untreated mice. Dr. Venditti's team determined that the AAV vector triggered the cancer.
The research team performed additional experiments to detect where in the mouse genome the AAV vector delivered the corrected gene and how that related to any cancer development. In many mice that developed liver cancer, the AAV vector targeted a region of the mouse genome called Rian, near a gene called Mir341 that codes for a microRNA molecule. MicroRNAs are small, non-coding RNA molecules involved in the regulation of gene expression. When the AAV was inserted near Mir341, the vector caused elevated expression of the gene, which the researchers believe contributed to the occurrence of liver cancer in the mice. The authors note that Mir341 is found in the mouse genome, however, it is not present in humans.
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Gene therapy biotech Spark Therapeutics sets terms for $88 million IPO
Spark Therapeutics, which is developing gene therapy treatments for retinal dystrophies and hematologic disorders, announced terms for its IPO on Tuesday. The Philadelphia, PA-based company plans to raise $88 million by offering 5.5 million shares at a price range of $15 to $17. At the midpoint of the proposed range, Spark Therapeutics would command a fully diluted market value of $378 million.
Spark Therapeutics, which was founded in 2013, plans to list on the NASDAQ under the symbol ONCE. J.P. Morgan and Credit Suisse are the joint bookrunners on the deal. It is expected to price during the week of January 26, 2015.
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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Gene therapy biotech Spark Therapeutics sets terms for $88 million IPO
Center for Retinal and Ocular Therapy at Penn Expands Relationship with Spark Therapeutics to Develop Potential …
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Newswise PHILADELPHIA The University of Pennsylvania has announced an expanded relationship with Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases. As part of the expanded relationship, which included both an exclusive license agreement to certain Penn-owned intellectual property rights and a clinical trial agreement, Penn will be one of the clinical sites for a clinical trial to evaluate the treatment of a rare genetic form of progressive blindness.
This expanded strategic relationship between the two organizations is representative of Penns strong commitment towards direct engagement with the private sector to advance promising technologies, said John Swartley, Associate Vice Provost for Research and Executive Director, Penn Center for Innovation (PCI). When Penn researchers on the cutting edge of their fields are able to partner effectively with innovators in the private sector it has the potential to accelerate the advancement of exciting new approaches for the treatment of disease. PCI serves as the University of Pennsylvanias commercialization center and actively works with the private sector to foster research and development collaborations leveraging Penn technology and research.
Spark today announced the start of Phase 1/2 clinical trial for patients with choroideremia (CHM), which will take place at The Childrens Hospital of Philadelphia and the University of Pennsylvania. CHM is a rare, genetic eye disorder that causes progressive vision loss, ultimately leading to complete blindness. CHM is characterized by deletions or mutations in the CHM gene. It is a degenerative eye disease which affects males that manifests in childhood as night blindness and a reduction in visual field, followed by progressive constriction of visual fields leading, ultimately, to blindness. There is currently no approved drug treatment for the disease.
The launch of this clinical trial is the latest facet of the ongoing partnership between Spark and Penn.Expanding upon an earlier collaboration around SPK-RPE65, in December of 2014, Spark and Penn, through PCI, entered into an exclusive license agreement to certain Penn-owned intellectual property rights, including assets related to the choroideremia program. As a part of the license agreement, Penn received equity shares in Spark and may receive additional milestone payments and royalties on net sales dependent on the success of the SPK-CHM program.
The new trial is designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The investigators plan to enroll up to 10 patients afflicted with the CHM genetic mutation. In addition to evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify appropriate endpoints for subsequent clinical trials. The trial will build on the work of the clinical trial teams that have conducted trials of Sparks therapy known as SPK-RPE65, which has been observed in clinical trials to improve vision in patients with rare blinding conditions due to mutations in the RPE65 gene.
I have a particular interest in choroideremia, says Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology and director of the Center for Retinal and Ocular Therapy (CAROT) at the Perelman School of Medicine at the University of Pennsylvania, and one of Sparks scientific co-founders. I am thrilled to now be able to test our gene therapy treatments with the potential to help the many men living with this disorder.
"Editors note: The University of Pennsylvania has licensed technology involved in this research to Spark. Dr. Bennett is an inventor of this technology, and may benefit financially."
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