Archive for the ‘Gene Therapy Research’ Category
AveXis Reports First Quarter 2017 Financial and Operating Results … – GlobeNewswire (press release)
May 11, 2017 16:00 ET | Source: AveXis
CHICAGO, May 11, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported financial results for the first quarter ended March 31, 2017, recent corporate highlights and upcoming milestones.
We are very pleased with our progress during the first quarter and recent weeks, including the recently reported encouraging results from the closeout of the Phase 1 trial of AVXS-101 in SMA Type 1, said Sean Nolan, President and Chief Executive Officer of AveXis. Our team is focused on executing our plan to bring AVXS-101 to patients suffering from SMA Type 1 as quickly and safely as possible.
Recent Highlights
Results from the Phase 1 Trial of AVXS-101 in SMA Type 1: The Phase 1, open-label, dose-escalating study was designed to evaluate the safety and tolerability of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1. The key measures of efficacy were the time from birth to an event, which was defined as either death or at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or perioperatively, and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor milestone development survey and Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).
Presented AVXS-101 research at the Annual Meeting of the American Academy of Neurology: Jerry Mendell, M.D., director of the Center for Gene Therapy at The Research Institute at Nationwide Childrens Hospital, presented results from the Phase 1 study of AVXS-101 in SMA Type 1 during a plenary session, including video evidence of children achieving motor milestones.
Completed Type B chemistry manufacturing and controls (CMC) meeting with the U.S. Food and Drug Administration (FDA): On May 1, 2017, AveXis participated in a Type B CMC meeting with the FDA. The purpose of the meeting was to present to the agency AveXis proposed process for producing the intended commercial scale GMP-derived gene therapy product, to gain alignment with the agency on the proposed assay qualification plan, and to gain alignment on the proposed protocol for demonstrating comparability of the intended commercial scale GMP-derived product with the material administered to patients in the Phase 1 trial of AVXS-101 in SMA Type 1.
The company expects to provide an update on its further plans and development timelines following receipt of the minutes of the CMC Type B meeting, currently anticipated in early June 2017.
Joao Siffert Appointed to Board of Directors: On April 19, 2017, AveXis announced the appointment of Joao Siffert to its Board of Directors, effective upon the completion of the annual meeting of stockholders. Dr. Siffert brings important knowledge to the Board based on his experience in central nervous system drug development and regulatory expertise in both the U.S. and Europe, as well as his experience working with global health care companies.
First Quarter 2017 Financial Results
Selected Financial Information
Operating Results:
Balance Sheet Information:
Conference Call Information The AveXis conference call and webcast of April 25, 2017 was conducted in lieu of a first quarter 2017 financial and operating results conference call. AveXis will not host a conference call and webcast related to its first quarter 2017 financial and operating results. The Company expects to host its next conference call and webcast following receipt of the minutes from the CMC meeting with the FDA, expected approximately 30 days following the meeting, which took place May 1, 2017.
About SMA SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.
The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.
About AVXS-101 AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.
About AveXis, Inc. AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The companys initial proprietary gene therapy candidate, AVXS-101, is in an ongoing Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit http://www.avexis.com.
Forward-Looking Statements This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in children with SMA Type 1 and statements about the effects of SMA Type 1 on developmental milestones and timing of regulatory feedback. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the "Risk Factors" and the "Management's Discussion and Analysis of Financial Condition and Results of Operations" section of AveXis Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect AveXis results. There can be no assurance that the actual results or developments anticipated by AveXis will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, AveXis. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. AveXis cautions investors not to rely too heavily on the forward-looking statements AveXis makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). AveXis undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Non-GAAP Financial Measure In addition to disclosing financial results that are determined in accordance with GAAP, in order to understand and evaluate our operating performance, and provide a more complete understanding of factors and trends affecting our business, we also measure the increase in research and development expenses and general and administrative expenses excluding non-cash stock-based compensation expense. We believe that excluding this expense better reflects the increase in research and development and general and administrative expenses during the period, as compared to the prior period. This non-GAAP financial metric should be considered supplemental to and not a substitute for financial information prepared in accordance with GAAP. Because non-GAAP financial metrics exclude the effect of items that will increase or decrease the companys reported results of operations, we strongly encourage investors to review our consolidated financial statements and periodic reports in their entirety.
Related Articles
See the article here:
AveXis Reports First Quarter 2017 Financial and Operating Results ... - GlobeNewswire (press release)
Pfizer expands hemophilia gene therapy pipeline with $720m Sangamo deal – BioPharma-Reporter.com
Pfizer has upped its presence in the gene therapy space through collaboration with Sangamo Therapeutics on its programmes worth up to $720m.
The agreement sees Pharma giant Pfizer pay Sangamo $70m upfront, but the Californian genome editing firm could receive up to $475m in milestone payments for its lead haemophilia A gene therapy SB-525, and a further $175m for other candidates developed under the collaboration.
Sangamos gene therapies are based on its multi-platform technology, including its zinc finger nuclease (ZFN) knock-out gene tool, and both adeno associated virus (AAV) and messenger RNA delivery tech.
Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A, Pfizers president of Worldwide R&D Mikael Dolsten said in a statement.
The deal is the latest venture for Pfizer in the haemophilia gene therapy sector. The firm has a long-standing haemophilia B collaboration with Spark Therapuetics, inked in December 2014 when Pfizer also established its gene therapy research unit.
Pfizer has since acquired Bamboo Therapeutics for $150m, adding an advanced recombinant AAV vector design and production technology and several pipeline therapies, and begun assessing sites in North Carolina to expand its gene therapy manufacturing capabilities.
Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing, said Dolsten.
We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.
See more here:
Pfizer expands hemophilia gene therapy pipeline with $720m Sangamo deal - BioPharma-Reporter.com
Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in – FierceBiotech
After deals and acquisitions with Spark Therapeutics and Bamboo, Pfizer is once again looking to bolster its rare and gene therapy pipeline as it outlines a new drug pact with Sangamo.
The collaborationlicense agreement focuses on the development and eventual sale of the biotechs gene therapy programs for hemophilia A, including SB-525, one of Sangamos four lead product candidates.
This early candidate is slated to enter the clinic this quarter, centering on testing safety as well as blood levels of Factor VIII protein, and other efficacy endpoints.
Sangamo gets $70 million upfront from the Big Pharma, and could gain $475 million in biobucks and sales royalties on any medications from the collaborationthat gain approval.
Under the deal, Sangamo will take the lead on the SB-525 phase 1/2 test as well as unspecified manufacturing activities.
Pfizer, meanwhile, will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for the therapy, as well as any additional products, if any.
Sangamo will also work with Pfizer on manufacturing and technical ops using viral delivery vectors.
SB-525 works as a AAV vector carrying a Factor VIII gene construct driven by a synthetic, liver-specific promoter. The FDA has already cleared the start of human trials for SB-525, and given it an orphan drug tag.
The deal has proved powerful for Sangamo, with its shares jumping 44% after hours on the news last night.
This marks another step into the new world of gene therapies for Pfizer, coming less than a year after its $700 million buy of Bamboo Therapeutics, adding advanced recombinant adeno-associated virus (rAAV)-based gene therapies to its pipeline.
It also has a long-standing deal with Spark Therapeutics, in hemophilia, penned in 2014. Back in January, Pfizer in fact paid a $15 million milestone bonus to Spark for hitting its marks in the ongoing hemophilia B phase 1/2 trial FDA breakthrough-tagged SPK-9001.
Pfizer also has a series of preclinical gene therapies, including a neuromuscular candidate for Duchenne muscular dystrophy (DMD), as well as preclinical candidates to treat Friedreichs ataxia and Canavan disease, and a phase I candidate for giant axonal neuropathy.
Pfizer also gained an operating gene therapy manufacturing facility that Bamboo bought from the University of North Carolina last year.
The pharma also has several academic research agreements, including one with Kings College London to develop a series of rAAV gene therapy vectors and another with the University of Iowa Research Foundation to develop a potential gene therapy for cystic fibrosis.
And its partnered with Emeryville, CA-based Molecular Therapeutics (4DMT) to discover and develop targeted next-generation rAAV vectors for cardiac disease; it made an investment in the company a few years back.
Once seen as the next big thing in research, gene therapies have however come under pressure in recent months about just how viable they are on the market. After struggling for years to make a commercial success out of Glybera, the worlds first approved gene therapy, uniQure recently called it quits on the treatment.
The drugmaker said it wouldnt bother asking European authorities to renew the $1-million-plus gene therapys marketing authorization when it expires in October, and comes after it abandoned plans to gain an approval in the U.S. Reports from MIT Technology Review suggest only one patient ever used the med.
GlaxoSmithKline has also been struggling in Europe with its bubble boy syndrome gene therapy Strimvelis. Mindful of Glyberas cost, GSK put its price tag at half that of Glybera, at $665,000, and also offered a money-back guarantee.
Its been approved in Europe for nearly a year, but it only treated its first patient this month, according to Business Insider.
Treatment is tough as the drug is not so much manufactured as it is created for each individual patient, with a site in Italy currently the only approved site in the world for this type of manufacture, and thus the only place where patients can be treated. Only around 15 patients in Europe are believed to have the condition.
Other biotechs are however working on the manufacturing side in order to try and make these therapies more available for patients, and thus open up their viability.
There are already a number of medications on the market for hemophilia, such as from Biogen spin-off Bioverativ and Sobi, with gene therapy predicted by some also working in the space, including uniQure and BioMarin, to be the next class for treating the blood disorder.
But speaking to FierceBiotech at the start of the year, Bioverativs new chief and former Biogen exec John Cox told me that while they are to working on gene therapy approaches to hemophilia, there are reasons to be cautious: There are of course question marks over gene therapy: The obvious one is safety, because of the history here, and this is a risk-averse population, for good reason, and the other question is naturally over efficacy, and how long does it last, as well as manufacturing, scale and so on.
Were all hoping for a cure, and of course were doing work on gene therapy now, but I dont think people are looking at these now as a permanent cure; the questions are over durability, rather than cure.
He said that investors and even doctors talk a lot about gene therapy in the hemophilia space, but that if you talk to hemophilia A patients about what they really want, being able to dose, once a week [which is the target with its candidate, or even just less frequently, is what they want.
The rest is here:
Pfizer doubles down on gene therapy pipeline with $70M Sangamo buy-in - FierceBiotech
Gene Therapy Advanced by Structural Study of Potential Viral Vectors – Genetic Engineering & Biotechnology News
Scientists led by Associate Professor Vijay Reddy, Ph.D., at The Scripps Research Institute (TSRI) say they have discovered the structural details that make one virus a better tool for future therapies than a closely related virus. The team wrote inScience Advances("Cryo-EM structure of human adenovirus D26 reveals the conservation of structural organization among human adenoviruses") thatthe structure of a less prevalent species D adenovirus may work well as a gene-delivery vector because its structure doesn't allow it to wind up in the liver, thus minimizing liver toxicity. The Reddy Lab's study reportedly is the first to show the structural details on species D's surface that set it apart from another common subtype of adenovirus, called species C, which does travel to the liver.
"Greater understanding of the structures of adenoviruses from different species will help generate better gene therapies and/or vaccine vectors," said Dr. Reddy.
Using cryo-electron microscopy, the researchers discovered that while these two species of adenoviruses share the same shell-like core, they have different surface structures called decorations or loops, whichare key to a virus's behavior. They determine which receptors on human cells the virus can bind to. For species C adenoviruses, specific loops help the virus attach to blood coagulation factors (adaptor proteins) and get targeted to the human liver.Species D adenoviruses display distinctly different loop decorations. For use in gene and vaccine therapies, the virus would deliver helpful genes instead.
Species D also has one more important advantage over species C: Humans are constantly exposed to species C adenoviruses, so most people have developed antibodies to fight them off. These same antibodies would fight off the species C viruses even if they were designed for beneficial therapies. On the flip side, many of the species D adenoviruses are rare, and it's unlikely that a patient would have antibodies to fight them off, according to the investigators. That makes species D viruses better for delivering therapies, explains Dr. Reddy, who adds that scientists are already testing ways to use it to generate malaria and Ebola virus vaccines.
The researchers next plan to look at members of the other five species of adenoviruses to see if they would have useful traits as viral therapy vectors.
Follow this link:
Gene Therapy Advanced by Structural Study of Potential Viral Vectors - Genetic Engineering & Biotechnology News
Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy – PR Newswire (press release)
"With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program," said Dr. Sandy Macrae, Sangamo's Chief Executive Officer. "We believe Pfizer's end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases."
Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.
Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient's cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.
Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.
Conference CallSangamo will host a conference call today, May 10, 2017 at 5:00 p.m. ET, which will be open to the public, to discuss the details of the collaboration and the Company's first quarter business and financial results. The call will also be webcast live and can be accessed via a link the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A replay of the webcast will also be available for one week after the call.
The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 15225000. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on May 10, 2017 to 11:59 p.m. ET on May 17, 2017. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 15225000.
About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Pfizer for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.
Forward Looking Statements This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation references relating to the collaboration agreement with Pfizer, potential milestone payments and royalties under the collaboration agreement, ability of the collaboration to advance and commercialize SB-525 as a treatment for Hemophilia A, research and development of therapeutic applications of Sangamo's genomic therapy platforms, the expected timing of clinical trials of lead programs, including SB-525 and the release of data from these trials, the impact of Sangamo's clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to substantial dependence on the clinical success of lead therapeutic programs, the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.
Pfizer and Rare DiseaseRare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide,i representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuroscience, and inherited metabolic disorders.ii
Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.
Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness and meet the needs of patient families.
Pfizer Inc: Working together for a healthier worldAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. For more information, please visit us at http://www.pfizer.com. In addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
Pfizer Disclosure Notice: The information contained in this release is as of May 10, 2017. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about an investigational Hemophilia A agent, SB-525, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical study commencement and completion dates as well as the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of existing clinical data; risks associated with initial data, including the risk that the final results of the Phase I/2 study for SB-525 and/or additional clinical trials may be different from (including less favorable than) the initial data results and may not support further clinical development; whether and when any applications may be filed with regulatory authorities for SB-525; whether and when regulatory authorities may approve any such applications, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of SB-525; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.
i Rare Disease: Facts and Statistics. http://globalgenes.org/rare-diseases-facts-statistics. Accessed September 7, 2016. ii Pfizer Inc. Rare Disease. http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus. Accessed December 20, 2016.
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-and-pfizer-announce-collaboration-for-hemophilia-a-gene-therapy-300455555.html
SOURCE Sangamo Therapeutics, Inc.
See the original post here:
Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy - PR Newswire (press release)
Global Gene Therapy Technologies, Markets and Companies Research Report 2017-2026 – Research and Markets – PR Newswire (press release)
Gene therapy can now combine with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets
Profiles of 187 companies involved in developing gene therapy are presented along with 232 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.
Key Topics Covered:
Part I: Technologies & Markets
Executive Summary
1. Introduction
2. Gene Therapy Technologies
3. Clinical Applications of Gene Therapy
4. Gene Therapy of Genetic Disorders
5. Gene Therapy of Cancer
6. Gene Therapy of Neurological Disorders
7. Gene Therapy of Cardiovascular Disorders
8. Gene therapy of viral infections
9. Research, Development and Future of Gene Therapy
10. Regulatory, Safety, Ethical Patent Issues of Gene Therapy
11. Markets for Gene Therapy
12. References
Part II: Companies
13. Companies involved in Gene Therapy
For more information about this report visit http://www.researchandmarkets.com/research/9jzl3f/gene_therapy
Media Contact:
Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com
For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-research-report-2017-2026---research-and-markets-300455149.html
SOURCE Research and Markets
Brammer invests in commercial-ready gene therapies – BioPharma-Reporter.com
Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.
The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.
We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.
Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
A Site Master File will be submitted later this year to support Brammers clients product applications.
The site originally housed Biogens clinical and commercial biologics manufacturing facility.
Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.
The Somerville site offers Brammer nearby storage and distribution capabilities.
The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.
Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.
More:
Brammer invests in commercial-ready gene therapies - BioPharma-Reporter.com
Brammer invests in commercial-ready gene therapies – OutSourcing-Pharma.com
Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.
The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.
We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.
Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
A Site Master File will be submitted later this year to support Brammers clients product applications.
The site originally housed Biogens clinical and commercial biologics manufacturing facility.
Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.
The Somerville site offers Brammer nearby storage and distribution capabilities.
The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.
Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.
Read the original:
Brammer invests in commercial-ready gene therapies - OutSourcing-Pharma.com
Clinical trial on the horizon for RP genetic therapy – AOP
Researchers hope to use advanced gene editing technology to reverse the course of retinitis pigmentosa
09 May 2017 by Selina Powell
American researchers have used the gene-editing tool CRISPR/Cas9 to reverse the course of retinitis pigmentosa (RP) and restore vision in mice.
The study, published in Cell Research, involved using the genetic tool to prevent the degeneration of the light-sensing cells.
CRISPR/Cas9 allows scientists to target specific sections of genetic code and edit DNA at precise locations.
By deactivating certain genes, researchers were able to transform rod photoreceptors into cone photoreceptors cells that are less vulnerable to the genetic mutations that cause RP.
Senior author, Dr Kang Zhang, explained to OT that traditional RP gene therapy approaches targeted a single gene or mutation.
However, he emphasised that since there were many mutations and genes causing RP, many patients were left without treatment options.
Our gene therapy approach will create a universal, cost-effective gene therapy treatment for a majority, if not all, RP patients, Dr Zhang elaborated.
Dr Zhang emphasised that a common cold virus was being used as a gene therapy vector and had a good safety profile in clinical trials.
It was expected that a preclinical study would be conducted over the coming months, and a human trial would be undertaken early next year.
Read more:
Clinical trial on the horizon for RP genetic therapy - AOP
Type 1 diabetes cured in mice using gene therapy – Medical News … – Medical News Today
Researchers from the University of Texas Health Science Center in San Antonio have found a way to cure type 1 diabetes in mice. It is hoped that the novel technique - which boosts insulin secretion in the pancreas - will reach human clinical trials in the next 3 years.
Study co-author Dr. Bruno Doiron, Ph.D., of the Division of Diabetes, and colleagues recently reported their findings in the journal Current Pharmaceutical Biotechnology.
Type 1 diabetes is estimated to affect around 1.25 million children and adults in the United States. Onset of the condition is most common in childhood, but it can arise at any age.
In type 1 diabetes, the immune system destroys the insulin-producing beta cells of the pancreas. Insulin is the hormone that regulates blood glucose levels. As a result, blood glucose levels become too high.
There is currently no cure for type 1 diabetes; the condition is managed through diet and insulin therapy. However, in recent years, researchers have investigated replacing beta cells as a means of eradicating type 1 diabetes once and for all.
Dr. Doiron and colleagues have taken a different approach with their new study. The team reveals how they used a method called gene transfer to coax other pancreatic cells into producing insulin.
Using this technique, the researchers have managed to cure type 1 diabetes in mice, bringing us one step closer to curing the condition in humans.
The gene transfer technique - called Cellular Networking, Integration and Processing - involves introducing specific genes into the pancreas using a virus as a vector.
The team notes that beta cells are rejected in patients with type 1 diabetes. With the gene transfer method, the newly introduced genes encourage non-beta cells to produce insulin, without any side effects.
"The pancreas has many other cell types besides beta cells, and our approach is to alter these cells so that they start to secrete insulin, but only in response to glucose [sugar]," says study co-author Dr. Ralph DeFronzo, chief of the Division of Diabetes. "This is basically just like beta cells."
Upon testing their technique on mouse models of type 1 diabetes, the researchers found that they were able to induce long-term insulin secretion and blood glucose regulation, with no adverse side effects.
"It worked perfectly. We cured mice for 1 year without any side effects. That's never been seen. But it's a mouse model, so caution is needed. We want to bring this to large animals that are closer to humans in physiology of the endocrine system."
Dr. Bruno Doiron, Ph.D.
Importantly, the researchers point out that the gene transfer therapy only releases insulin in response to blood sugar, so it has the potential to transform current treatments for type 1 diabetes.
"A major problem we have in the field of type 1 diabetes is hypoglycemia (low blood sugar)," says Dr. Doiron. "The gene transfer we propose is remarkable because the altered cells match the characteristics of beta cells. Insulin is only released in response to glucose."
Not only could the novel strategy yield a cure for type 1 diabetes, but the researchers say that it may also eliminate the need for insulin therapy in patients with type 2 diabetes, which arises when the body is unable to use insulin effectively.
It will cost around $5 million to test their technique in large animal models, but the researchers are confident that this can be achieved. They hope to reach human clinical trials within the next 3 years.
Learn how maternal omega-3 intake may influence the risk of type 1 diabetes in infants.
See the original post:
Type 1 diabetes cured in mice using gene therapy - Medical News ... - Medical News Today
Gene therapies assessment and therapeutics pipeline review H1 2017 scrutinized in new research – WhaTech
Details WhaTech Channel: Medical Market Research Published: 09 May 2017 Submitted by Vivian Dsena WhaTech Agency News from Wise Guy Reports - Market Research Viewed: 1 times
Gene Therapies Assessment and Therapeutics Pipeline Review H1 2017
Summary
"Gene Therapies: A Diverse Range of Technologies with a Promising Long-Term Outlook" discusses all gene therapies under the broad criteria of genetic material introduced to the cell for a therapeutic purpose. It includes not only the insertion of a gene into the cell for expression, but also gene silencing with RNA interference (RNAi) and antisense RNA, aptamers (DNA or RNA polymers which bind to a protein target), and oncolytic viruses.
GET SAMPLE REPORT @ http://www.wiseguyreports.com/sample-request/178375-gene-therapies-a-diverse-range-of-technologies-with-a-promising-long-term-outlook
Gene therapies have been in development in humans for 25 years, and a number of products have begun to enter the pharmaceutical market. However, due to various challenges and clinical trial setbacks, progress in developing this technology and achieving suitability for commercial usage has been slow.
Most are at an early stage of development, with 76% at the Discovery or Preclinical stage. However, there are currently 23 gene therapy programs in Phase III development and two at the Pre-registration stage.
This indicates that although gene therapies are beginning to reach the market after two decades of research in humans, the majority remain in relatively early development.
This report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.
Scope
Despite 25 years of clinical research, only a few gene therapies of all types have reached the market globally, and none have achieved strong clinical or commercial success -
- Why do gene therapies still occupy only a minimal market share in their respective indications?
- What can be learned from the gene therapies that have already reached the market?
A number of different viral and non-viral vector types are currently in development for the delivery of gene therapies -
- What are the relative advantages and disadvantages of each vector type and which hold the most promise?
- What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?
The current pipeline for gene therapies is diverse in terms of the approaches and vectors covered; 50% are gene silencing therapies, while 31% involve the insertion of a functional gene -
- In which therapy areas is there the highest level of R&D activity for gene therapies?
- At which stage of development does the majority of pipeline gene therapies reside?
- What is the proportion of the pipeline occupied by each intervention and vector type overall?
A number of companies are currently actively developing pipeline gene therapies, including private, public and institutional enterprises -
- How do gene therapies fit into the overall portfolios of these companies?
- What is the level of involvement in gene therapy research from the top 20 Big Pharma companies?
Reasons to access
This report will allow you to -
- Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products, comprising Glybera, Kynamro, Macugen, Vitravene, Gendicine, Oncorine, and Neovasculgen.
- Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, infectious diseases, central nervous system disorders, and genetic disorders.
- Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline. Understand the level of involvement on the part of big pharma companies, and the extent to which gene therapies fit into the overall portfolios of companies in this field.
Additionally, a highly granular breakdown of companies developing multiple gene therapies is provided.
Table of Content: Key Points
Gene Therapy Overview
Types of Gene Therapy
Types of Intervention
Types of Vector
Currently Approved Gene Therapies
Glybera (alipogene tiparvovec)
Kynamro (mipomersen)
Macugen (pegaptanib)
Vitravene (fomivirsen)
Gendicine (rAd-p53)
Oncorine (rAd5-H101)
Neovasculgen (Pl-VEGF165)
Gene Therapy Production Strategies
Production of Viral Vectors
Case Study: Challenges in the Manufacture of AAV Vectors
Challenges to Gene Therapy Development
Gene Therapy Pipeline
Gene Therapy Pipeline by Therapy Area and Stage of Development
Gene Therapy Pipeline by Intervention and Vector Type
Company Positioning
Continued
ACCESS REPORT @ http://www.wiseguyreports.com/reports/178375-gene-therapies-a-diverse-range-of-technologies-with-a-promising-long-term-outlook
See the original post:
Gene therapies assessment and therapeutics pipeline review H1 2017 scrutinized in new research - WhaTech
HIV breakthrough: Scientists remove virus in animals using gene editing – Medical News Today
Worldwide, tens of millions of people are living with HIV. While scientists and medical professionals do not yet have a permanent cure for the virus, researchers have just made a breakthrough: they managed to eliminate the HIV-1 infection in mice.
According to the Centers for Disease Control and Prevention (CDC), more than 36 million people across the world are HIV positive, and approximately 1.2 million people in the United States live with the virus.
While there is currently no cure for the infection, scientists have just moved closer to finding one. Using a gene editing technology called "CRISPR/Cas9," the researchers successfully excised the HIV-1 provirus in three animal models.
A provirus is an inactive form of virus. It occurs when the virus has integrated into the genes of a cell. In the case of HIV, these host cells are the so-called CD4 cells - once the virus has been incorporated into the DNA of the CD4 cells, it replicates itself with each generation of CD4 cells.
The three mouse models used in the current research included a "humanized" model, in which the mice were genetically modified to have human immune cells, which were then infected with HIV-1.
The team was co-led by Dr. Wenhui Hu, Ph.D., associate professor in the Center for Metabolic Disease Research and the Department of Pathology at the Lewis Katz School of Medicine (LKSOM) at Temple University in Philadelphia, together with Kamel Khalili, Ph.D., Laura H. Carnell Professor and chair of the Department of Neuroscience at LKSOM, and Won-Bin Young, Ph.D, who just recently joined LKSOM.
The new study - published in the journal Molecular Therapy - builds on previous research by the same team, during which they used genetically modified rodents to demonstrate that their gene editing technology could eliminate the HIV-1-infected segments of DNA.
"Our new study is more comprehensive," Dr. Hu explains. "We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy. We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells."
Dr. Hu and team inactivated HIV-1, significantly reducing the RNA expression of viral genes in the organs and tissues of genetically modified mice.
Specifically, the RNA expression was reduced by approximately 60 to 95 percent.
The researchers then tested their findings by acutely infecting mice with EcoHIV - the equivalent of the HIV-1 in humans. Dr. Khalili explains the procedure:
"During acute infection, HIV actively replicates. With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection."
The CRISPR/Cas9 method was up to 96 percent efficacious in eradicating EcoHIV in mice.
Finally, in the third model, mice received a transplant of human immune cells, including T cells, which were then infected with HIV-1.
One of the main reasons that a cure for HIV has yet to be discovered is the virus's ability to "hide" in the genomes of T cells, where it lives latently. This is why researchers applied the CRISPR/Cas9 technology to these mice with infected T cells.
After a single round of gene editing, the viral segments were excised from the human cells that had been integrated into the mouse tissues and organs. They removed the provirus from the mice's spleen, lungs, heart, colon, and brain after only one therapy injection.
The injection was with "quadruplex sgRNAs/saCas9 AAV-DJ/8" - an improved adeno-associated viral (AAV) vector.
AAV vectors are commonly used in gene therapy, but "the AAV-DJ/8 subtype combines multiple serotypes, giving us a broader range of cell targets for the delivery of our CRISPR/Cas9 system," Dr. Hu explains.
To assess the success of the genetic interventions, the team measured HIV-1 RNA levels using live bioluminescence imaging.
This is the first time that a team of researchers has managed to halt the replication of the HIV-1 virus and eliminate it completely from the infected cells in animals.
The team also provided the first evidence that HIV-1 can be successfully eradicated and full infection with the virus can be prevented using the CRISPR/Cas9 gene editing strategy.
The study was deemed "a significant step towards human clinical trials" by the authors, and the findings represent a breakthrough in the search for an HIV cure.
"The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells. Our eventual goal is a clinical trial in human patients."
Kamel Khalili, Ph.D.
Learn how an HIV 'fingerprint' tool could greatly assist vaccine development.
Excerpt from:
HIV breakthrough: Scientists remove virus in animals using gene editing - Medical News Today
2017-2026 Global Antiviral Therapeutics Technologies, Markets and Companies Report – Research and Markets – PR Newswire (press release)
This report reviews the current state-of-art of antiviral approaches including vaccines, pharmaceuticals and innovative technologies for delivery of therapeutics. The introduction starts with a practical classification of viral diseases according to their commercial importance. Various antiviral approaches are described including pharmaceuticals and molecular biological therapies such as gene therapy and RNA interference (RNAi) as well as vaccines for virus infections. Expert opinion is given about the current problems and needs in antiviral therapy. SWOT (strengths, weaknesses, opportunities and threats) analysis of antiviral approaches is presented against the background of concept of an ideal antiviral agent.
A novel feature of this report is the use of nanotechnology in virology and its potential for antiviral therapeutics. Interaction of nanoparticles with viruses are described. NanoViricides are polymeric micelles, which act as nanomedicines to destroy viruses. Various methods for local as well as systemic delivery of antiviral agents and vaccines are described. Nanobiotechnology plays an important role in improving delivery of antivirals. Advantages and limitations of delivery of gene-based, antisense and RNAi antiviral therapeutics are discussed.
After a discussion of current therapies of AIDS/HIV and their limitations, new strategies in development of antiviral agents are described. Drug resistance and toxicities are emerging as major treatment challenges. Based on a review of technologies and drugs in development, it can be stated that there are good prospects are of finding a cure for HIV/AIDS in the next decade.
Hepatitis viruses are described with focus on hepatitis C virus (HCV) and hepatitis B virus (HBV). Despite the presence of numerous drug candidates in the anti-HCV pipeline, and the commitment of major R&D resources by many pharmaceutical companies, it might still take several years for any new anti-HCV drugs to reach the market.
Various commercially important viruses include herpes simplex (HSV) and human papilloma virus (HPV). There a number of treatments but HSV is not destroyed completely and remains dormant and activates from time to time to cause various clinical manifestations. There is discussion about the role of HPV in cervical cancer and vaccines available now seem to be adequate in preventing HSV-induced cervical cancer.
Markets for antivirals are considered according to viruses and diseases caused by them and also according to management approaches: antiviral drugs, vaccines, MAbs and innovative approaches that include immunological and use of other technologies such as gene therapy, antisense, RNAi and nanobiotechnology. Antiviral markets are estimated starting with 2016 with projections up to the year 2026.
Profiles of 194 companies that are involved in developing various technologies and products are profiled and with 174 collaborations. These include major pharmaceutical companies (12), Biopharmaceutical companies with antiviral products (86), Antiviral drug companies (26) as well as viral vaccine companies (70). The report is supplemented with 53 tables, 15 figures and 550 references from the literature.
Key Topics Covered:
Executive Summary
1. Introduction to Virology
2. Antiviral Approaches
3. Vaccines for Virus Infections
4. Role of Nanotechnology in Developing Antiviral Agents
5. Delivery of Antivirals
6. Competitive Assessment of Antiviral Approaches
7. Influenza Viruses
8. AIDS/HIV
9. Hepatitis Viruses
10. Miscellaneous Commercially Important Virus Infections
11. Viruses with High Impact but Low Commercial Significance
12. Markets for Antivirals
13. Companies
14. References
For more information about this report visit http://www.researchandmarkets.com/research/6svl85/antiviral
Media Contact:
Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com
For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900
U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/2017-2026-global-antiviral-therapeutics-technologies-markets-and-companies-report---research-and-markets-300453036.html
SOURCE Research and Markets
http://www.researchandmarkets.com
View original post here:
2017-2026 Global Antiviral Therapeutics Technologies, Markets and Companies Report - Research and Markets - PR Newswire (press release)
Simon Atkinson appointed IUPUI vice chancellor for research – IU Newsroom
INDIANAPOLIS -- Indiana University-Purdue University Indianapolis Chancellor Nasser H. Paydar has announced the appointment of Simon Atkinson as vice chancellor for research, effective July 1.
Atkinson, Chancellor's Professor and professor of biology in the School of Science at IUPUI since 2010, has served as IUPUI's interim vice chancellor for research since August 2015. He also holds adjunct appointments in the Indiana University School of Medicine Division of Nephrology and Department of Biochemistry and Molecular Biology.
Atkinson is a cell biologist specializing in research on kidney diseases. His background spans science, medicine and business. His interactions extend beyond his core research on the kidney, including numerous scientific collaborations with investigators across campus, especially in the Herman B Wells Center for Pediatric Research at the IU School of Medicine.
"I am delighted that Simon will continue to guide IUPUI's research office," Paydar said. "His expertise, extensive leadership experience, and outstanding accomplishments in research and education greatly benefit the campus community and beyond. He will keep us on track to implement IUPUI's strategic priorities that expand research and creative activity."
Atkinson first joined Indiana University in 1994 as an assistant professor in the Division of Nephrology at the School of Medicine. He served as graduate advisor and director of the Ph.D. program in biomolecular imaging and biophysics from 2004 to 2010. He served as chair of the Department of Biology in the School of Science at IUPUI from 2010 to 2015.
In recent years, Atkinson's research team has focused on efforts to understand and treat acute kidney injury, a common and life-threatening complication in seriously ill patients, using state-of-the-art methods including multiphoton microscopy, RNA interference and gene therapy.
Atkinson is also a biomedical entrepreneur. He co-founded INphoton -- a customized, proprietary company that provided microscopy services and consulting for pharmaceutical and biotech companies in the preclinical phase of drug discovery and development. He and other IU investigators also developed the technology used by Rene Medical Inc., a startup medical device company that targets the treatment and prevention of acute kidney injury.
Atkinson's research has been funded by the National Institutes of Health and has garnered foundation and industry support. He has also held leadership roles with the American Society for Cell Biology.
"Chancellor Paydar has set ambitious goals for our researchers, and I'm committed to seeing the campus recognized as one of the leading research institutions in the nation," Atkinson said. "My colleagues in the Office of the Vice Chancellor for Research are doing exceptional work to help realize the tremendous research potential at IUPUI. I look forward to continuing my association with them."
Atkinson earned his B.Sc. in cell and molecular biology from King's College London and his Ph.D. in molecular biology from the University of Cambridge in England. He also served a postdoctoral fellowship at Johns Hopkins University School of Medicine.
Atkinson will serve as vice chancellor for up to four years.
Read more:
Simon Atkinson appointed IUPUI vice chancellor for research - IU Newsroom
Audentes Therapeutics (BOLD) Names John T. Gray, Ph.D. as SVP … – StreetInsider.com
Get daily under-the-radar research with StreetInsider.com's Stealth Growth Insider Get your 2-Wk Free Trial here.
Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced the appointment of John T. Gray, Ph.D. to Senior Vice President and Chief Scientific Officer. Dr. Gray joined Audentes in 2014 as Vice President, Research and Development, and has successfully built and led teams across the Company's molecular biology, process development, and nonclinical research and development functions.
"We are very pleased to announce John's well-earned appointment to the role of Chief Scientific Officer," stated Matthew R. Patterson, President and Chief Executive Officer. "John is recognized as an innovator who has made significant contributions to advancing the science of AAV gene therapy. Today's announcement comes as a result of the key leadership role he has played in building our world-class research organization, developing our manufacturing capabilities and advancing our pipeline of novel product candidates."
Dr. Gray joined Audentes with over 20 years of experience designing genetic therapies and vaccines, and developing manufacturing processes for those products. Immediately prior to Audentes, Dr. Gray served as the Director of Vector Production and Development at St. Jude Children's Research Hospital where he led a team devoted to advancing gene therapy vector science. He was a key contributor to the Hemophilia B gene therapy project, for which he designed the self-complementary AAV Factor IX vector expression cassette and developed the GMP production process used to manufacture the clinical vector. During his tenure at St. Jude, Dr. Gray also worked on Chimeric Antigen Receptor modified cell therapy, lysosomal storage disorder gene therapy, and multiple hematopoietic stem/progenitor cell gene therapy projects utilizing both AAV and lentiviral vectors. Prior to joining St. Jude, Dr. Gray served as the assistant director of the Harvard Gene Therapy Initiative and prior to that, worked at Pfizer Animal Health designing bacterial and viral vectors for vaccine applications. Dr. Gray has a Bachelor of Arts degree in Biochemistry from the University of California, Berkeley, and a Ph.D. degree in Biochemistry from the University of Colorado, Boulder.
Read more here:
Audentes Therapeutics (BOLD) Names John T. Gray, Ph.D. as SVP ... - StreetInsider.com
Genome studies – The Hindu
Genome studies The Hindu NCAB facilities, spread over 20,000 sqft. both in NIMS Medicity and Noorul Islam University, facilitate academic and research activities in molecular human genetics, plant and animal biotechnology, infectious diseases, genetic disorders, gene therapy ... |
Read more from the original source:
Genome studies - The Hindu
Time for a national policy on thalassaemia – The Hindu
The Hindu | Time for a national policy on thalassaemia The Hindu We were thrilled when research on gene therapy was started in India several years ago. Unfortunately, due to a lack of incentives, willingness and support, the research has come to a standstill. There are clinical trials for thalassaemia gene therapy ... |
Read the rest here:
Time for a national policy on thalassaemia - The Hindu
Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market – Assets Stock
Global PharmaSphere: Emerging Biotechnologies-Gene Therapy market 2017 Research Report offers a replete and diligent analysis of the growth rate of the PharmaSphere: Emerging Biotechnologies-Gene Therapy industry.This report scrutinize current market trends, future growth potential, dominant market growth drivers, elements impeding market growth, opportunities, market framework, market challenges, market future prognosis and best practices in the global PharmaSphere: Emerging Biotechnologies-Gene Therapy market.
Report Synopsis:
Initially, the research study provides subtle knowledge of the PharmaSphere: Emerging Biotechnologies-Gene Therapy market composition, evaluates and overviews its multifaceted aspects & applications. It formulate both its quantitative and qualitative patterns of investigative research. The research study split the global PharmaSphere: Emerging Biotechnologies-Gene Therapy market on the basis of different parameters and assesses each section as well as sub-section of PharmaSphere: Emerging Biotechnologies-Gene Therapy market.
Do Inquiry Before Purchasing Report Here:https://market.biz/report/global-pharmasphere-emerging-biotechnologies-gene-therapy-market-icrw/42494/#inquiry
The major areas on which the report focuses on related to each firm are key competitor profile overview/description, fiscal & accounting details, business strategies and recent developments.
Key Dominant players in PharmaSphere: Emerging Biotechnologies-Gene Therapy market:
1 Dimension Therapeutics 2 Celladon Corporation 3 Bluebird bio 4 Avalanche 5 AskBio 6 Applied Genetic Technologies Corporation 7 AnGes MG, Inc. 8 American Gene Technologies International Inc 9 Amarna Therapeutics 10Advantagene
The report also scrutinates market growth potential, market scope and sales prophecy across different regions.
Region Based Analysis of PharmaSphere: Emerging Biotechnologies-Gene Therapy Market:
1 USA 2 South East Asia 3 India 4 Europe 5 China
Get Sample Copy Of Report From Here: https://market.biz/report/global-pharmasphere-emerging-biotechnologies-gene-therapy-market-icrw/42494/#requestforsample
Global PharmaSphere: Emerging Biotechnologies-Gene Therapy market is Splited into two segments product and application.
In addition,the elements that triggers and restrict the growth of the global PharmaSphere: Emerging Biotechnologies-Gene Therapy industry are mentioned and clarified in depth in this research study. It assists professionals in taking crucial business decisions to publicize their business. The study also scrutinize areas having potentialities for future market growth. It also provides information about emerging markets, profitable markets, static markets, declining markets, saturated markets or mature markets along with growth benefits.
Overall the PharmaSphere: Emerging Biotechnologies-Gene Therapy report offers complete substantial analysis of the parent market, key strategies followed by dominant industry Players, forthcoming segments, former, current and forecast market analysis in terms of volume and value along with entire research conclusions.
The Report serves as a valuable/profitable guide for the industry players and other individuals who are interested in studying the PharmaSphere: Emerging
Biotechnologies-Gene Therapy market.
For More chemicals Research Reports Visit : http://assetsstock.com/category/chemicals
Divyang has been into market research industry for last 5 years. He has a keen interest and deep knowledge of research industry. He has a stint of experience working as Research Analyst. His goals in life are simple - to stay happy, healthy and to keep writing as long as she possibly can.
View post:
Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market - Assets Stock
How Crispr Could Snip Away Some of Humanity’s Worst Diseases – WIRED
vH0z4zE),ew{KU53: $a%>9L M EyZu"Cp}1/f/o==+:#r"hjPrrtY/a+_ak~O#Ck4AGU"{VYu3,wdrR-g0NCwon}?s]<'@{d3oq[sc>lO~`0eQJrjnm{91?2G!l:F More here:
How Crispr Could Snip Away Some of Humanity's Worst Diseases - WIRED
Preclinical Data from REGENXBIO RGX-314 Gene Therapy Program for Wet Age-Related Macular Degeneration to be … – GlobeNewswire (press release)
May 04, 2017 16:06 ET | Source: REGENXBIO Inc.
ROCKVILLE, Md., May 04, 2017 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced that preclinical data from studies supported byREGENXBIOat the University of Pennsylvanias Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics and at the Johns Hopkins Wilmer Eye Institute will be shared in one presentation and four posters at upcoming conferences including the Retinal Cell and Gene Therapy Innovation Summit, the Association for Research in Vision and Ophthalmology (ARVO), and the American Society of Gene and Cell Therapy(ASGCT). These data support further clinical research regarding the use of REGENXBIOs investigational gene therapy RGX-314 for the treatment of wet age-related macular degeneration (wet AMD).
RGX-314 has the potential to be a one-time treatment for people with wet AMD by delivering high expression of anti-VEGF antibodies through the use of our NAV AAV8 vector.We are pleased to share additional positive preclinical results, which were generated by our development partners at the University of Pennsylvania and Johns Hopkins, which support our active IND, said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. REGENXBIOis on track to begin enrollment in the RGX-314 Phase I clinical trial by mid-2017 and to provide an interim trial update by the end of 2017.
Details of the upcoming presentation and posters are as follows:
Presentation at Retinal Cell and Gene Therapy Innovation Summit
Title:Preclinical gene therapy studies to select RGX-314 doses to treat wet age-related macular degeneration Presenter:Jean Bennett, PhD, Department of Ophthalmology,University of Pennsylvania,Philadelphia, PA Session date/time:Friday, May 5, 9:20 a.m. 9:30 a.m. EDT Session title:Gene Therapy, Outcome Measures, and Novel Therapies, Session 1: Preclinical Aspects Vector Design/Animal Models Room:Holiday 1-3, Hilton Baltimore, Baltimore, MD
Posters at Association for Research in Vision and Ophthalmology
Title:RGX-314, an AAV8 expressing an anti-VEGF protein, strongly suppresses subretinal neovascularization and vascular leakage in mouse models Authors:Ji-kui Shen1, Yuanyuan Liu1, Seth D. Fortmann1, Stephen Yoo3, Karen Kozarsky2, Jiangxia Wang1, Peter A. Campochiaro1. 1Ophthalmology, Johns Hopkins Wilmer Eye Inst, Baltimore, Maryland, United States; 3REGENXBIO Inc, Rockville, Maryland, United States Session date/time:Sunday, May 7, 8:30 a.m. 10:15 a.m. EDT Session title:Cytokines; Growth factors; Antiangiogenic drugs Room:Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number:B0230
Title: Subretinal delivery of RGX-314 AAV8-anti-VEGF Fab gene therapy in NHP Authors: Anna Tretiakova1, Tomas S. Aleman3, Arkady Lyubarsky3, Elaine J. Zhou4, Erik Wielechowski1, Gui-Shuang Ying2, Erin Bote1, Leah Makaron1, Stephen Yoo5, Jean Bennett3,6, Albert M. Maguire3,6, James Wilson1. 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States;2Center for Preventative Ophthalmology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania, United States;3Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, United States;4Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States;5REGENXBIO, Rockville, Maryland, United States;6Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States Session date/times:Wednesday, May 10, 11:00 a.m. 12:45 p.m. EDT Session title:Gene editing and gene therapies Room:Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number: B0164
Title: Normal parameters of the full field ERG recorded with bipolar electrodes in Cynomolgus Macaque (Macaque fascicularis) Authors: Arkady Lyubarsky1,2, Erik Wielechowski3, Tomas S. Aleman4, Albert M. Maguire1,4, Gui-Shuang Ying4, Erin Bote3, Leah Makaron3, James Wilson3, Jean Bennett1,4, Anna P. Tretiakova3. 1Center for Advanced Retinal and Ophthalmic Therapeutics, SOM Univ. of Pennsylvania, Philadelphia, Pennsylvania, United States;2Vision Research Center, University of Pennsylvania, Philadelphia, Pennsylvania, United States;3Gene Therapy Program, University of Pennsylvania SOM, Philadelphia, Pennsylvania, United States;4Scheie Eye Institute, University of Pennsylvania SOM Ophthalmology, Philadelphia, Pennsylvania, United States Session date/times: Thursday, May 11, 8:30 a.m. 10:15 a.m. EDT Session title: Retinal Function ERG studies Room: Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number: B0441
Additional information on the meeting can be found on the ARVO website:http://www.arvo.org
Poster at American Society of Gene and Cell Therapy
Title:Safety of RGX-314 AAV8-anti-VEGF Fab Gene Therapy in NHP Following Subretinal Delivery Authors:Tomas S. Aleman1, Anna P. Tretiakova2, Arkady L. Lyubarsky1, Jessica I. W. Morgan2, Elaine J. Zhou3, Erik Wielechowski2, Gui-Shuang Ying4, Erin Bote2, Leah Makaron2, Stephen Yoo5, Jean Bennett1, Albert M. Maguire1, James M. Wilson2. 1Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA,2Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA,3Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA,4Center for Preventative Ophthalmology and Biostatistics, University of Pennsylvania, Philadelphia, PA,5REGENXBIO, Rockville, MD. Session date/times:Thursday, May 11, 5:15 p.m. 7:15 p.m. EDT Session title:Neurologic Diseases (including Ophthalmic and Auditory Diseases) II Room:Exhibit Hall A & B South, Marriot Wardham Park Hotel, Washington, DC Abstract number:427
Additional information on the meeting can be found on the ASGCT website:http://www.asgct.org
Note Regarding Penn
Penn has licensed certain Penn-owned AAV intellectual property to REGENXBIO, including rights related to RGX-314.Dr. Wilson is an advisor to REGENXBIO and is a founder of, holds equity in, and receives sponsored research support from REGENXBIO.
AboutREGENXBIO
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIOs NAVTechnology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Licensees are applying the NAV Technology Platform in the development of a broad pipeline of product candidates in multiple therapeutic areas.
Forward Looking Statements
This press release contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIOs research, development and regulatory plans in connection with its NAV Technology Platform and gene therapy treatments. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of REGENXBIOs development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Meaningful factors which could cause actual results to differ include, but are not limited to, the timing of enrollment, commencement and completion of REGENXBIOs clinical trials; the timing and success of preclinical studies and clinical trials conducted by REGENXBIO and its development partners; the ability to obtain and maintain regulatory approval of REGENXBIOs product candidates and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIOs product candidates; REGENXBIOs ability to obtain and maintain intellectual property protection for REGENXBIOs product candidates and technology; REGENXBIOs growth strategies; REGENXBIOs competition; trends and challenges in REGENXBIOs business and the markets in which REGENXBIO operates; REGENXBIOs ability to attract or retain key personnel; the size and growth of the potential markets for REGENXBIOs product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of REGENXBIOs product candidates; REGENXBIOs ability to establish and maintain development partnerships; REGENXBIOs expenses and revenue; regulatory developments in the United States and foreign countries; the sufficiency of REGENXBIOs cash resources and needs for additional financing; and other factors discussed in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of REGENXBIOs Annual Report on Form 10-K for the year ended December31, 2016. In addition to the risks described above and in REGENXBIOs filings with the Securities and Exchange Commission, other unknown or unpredictable factors also could affect REGENXBIOs results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). REGENXBIO undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.
Related Articles
ReNeuron presenting new data on Friday – DIGITALLOOK
Cell-based therapeutics developer ReNeuron Group announced on Friday that it would be presenting new data relating to the characterisation and scale-up of its CTX cell-derived exosome therapy candidates at a leading scientific conference in London, also on Friday.
The AIM-traded firm explained that, in a poster presentation, ReNeuron researchers and their academic collaborators at the Department of Biochemical Engineering, University College London, would address the challenges of purifying CTX-derived exosomes at scale whilst preserving their potential therapeutic attributes to ensure consistency of future manufacture.
New strategies were being presented to address the upstream cell culture processes needed to generate the exosomes and the downstream purification methods that could be applied to remove protein and DNA-based impurities from the exosomes at commercially relevant scale.
The new methods yielded a threefold increase in particle protein purity and a more than fivefold increase in particle DNA purity compared with previous purification processes.
In a further poster presentation, research teams at ReNeuron and at the UK's Cell and Gene Therapy Catapult would address the challenge of characterising ReNeuron's CTX cell-derived exosomes to ensure consistency and control during manufacture.
The company said the studies undertaken demonstrated a robust approach to optimising and qualifying assays for micro-RNA (miRNA) targets found in the exosomes.
A potential advantage of exosomes when utilised as a carrier for the delivery of therapeutic miRNAs in gene therapy was the avoidance of issues typically encountered by RNA-based drugs, such as stability in vivo and tissue targeting, ReNeurons board explained.
The application of robust characterisation and purification methods to ReNeuron's exosome populations would support the future development of the company's exosome-based therapeutic candidates across multiple potential disease indications - the initial disease target being cancer.
The data being presented at the ISCT 2017 conference represent key advances in the development of ReNeuron's CTX-derived exosome therapeutic programme, both as a potential new nanomedicine targeting cancer and as a potential delivery system for gene therapy treatments, said ReNeurons head of research Randolph Corteling.
We look forward to presenting further data from this programme in the months ahead, Dr Corteling added.
See the original post here:
ReNeuron presenting new data on Friday - DIGITALLOOK
Genetic Events in Chronic Lymphocytic Leukemia – Cordis News
In recent years was proved that the best technique in the investigation of malignant lymphocytes is the. Fluorescence in situ hybridization (FISH). In the literature it was registered, in previous years, on an international study, conducted on 109 cases of CLL, 79 cases (72.5%) who had more genetic abnormalities: the remaining 30 cases (27.5%) had normal results, using FISH. The presence of del(11q), del(17p), mutated TP53, and unmutated IGHV typically predict for poor survival. Genetic Events in Chronic Lymphocytic Leukemia
In the normal cells, suppressor P-53 gene, coding proteins that bind to DNA and regulate the expression of genes, prevents the genome mutations. A mutation of the gene P-53 will inevitably lead to a process of carcinogenesis in which the cell divides endlessly. In recent years was proved that the best technique in the investigation of malignant lymphocytes, in the processes of deletions and rearrangements of chromosome genes, is the Florescence in situ Hybridization, (FISH) technique and this method is used as an alternative to chromosomal banding, a conventional application in molecular medicine, (Nelson BP et al, 2007).
In the literature of field in molecular medicine, it was registered, in previous years, on an international study, conducted on 109 cases of CLL, 79 cases (72.5%) who had more genetic abnormalities of p53 gene; the remaining 30 cases (27.5%) had normal results, using the same technique, FISH. The majority of patients, 67% (53.79) had a single anomaly and the remaining 33% had two or three genetic abnormalities. The chromosomes 14q32-17p translocation in LLC genome, which appeared similar to some common, had demonstrated abnormalities involving IGH gene, located on chromosome14q32, (Zerdoumi A et al, 2015).
Identification of P53 gene mutations in regions of 17 chromosome of hematological neoplasm is important because these mutations have an impact on the clinical course of patients and requires an attitude adjustment therapeutic adequate. Restoring function to p53 can induce lymphoma, apoptosis. Recent, endogenous somatic gene therapy research is a basic of trial clinical and therapeutic trial.
The DNA, (either integrated into the genome or episome external plasmid) is used to treat a disease arising as a result of mutations in chromosomal regions. In the past few years, this method has been included in the treatment of CLL, acute lymphocytic leukemia, [ALL], or multiple myeloma [MM], (Jump up^ "Gene Therapy". ama-assn.org. 4 April 2014. Retrieved22 March 2015).
The frequencies of P53 gene mutations in CLL can be categorized as individual biomarkers in proteomic and genomic profile for this type of leukemia that can be implemented in targeted patient treatment, within personalized medicine. Keywords: Gene P53, Chronic Lymphocytic Leukemia, Apoptosis, Fluorescence in situ Hybridization, Cancer. Conference: International Symposium on Clinical Neuroscience: Clinical Neuroscience for Optimization of Human Function, Orlando, USA, 7 Oct - 9 Oct, 2016. Presentation Type: Poster Presentation Topic: Abstracts ISCN 2016 Citation: UDRISTIOIU A (2016). Role of P53 gene in oncogenenesis of Chronic Lymphocytic Leukemia. Front. Neurol. Conference Abstract: International Symposium on Clinical Neuroscience: Clinical Neuroscience for Optimization of Human Function. doi: 10.3389/conf.fneur.2016.59.00101 Received: 01 May 2016; Published Online: 07 Sep 2016. * Correspondence: AURELIAN UDRISTIOIU, Emergency County Hospital Targu Jiu, Clinical Laboratory, Targu Jiu, Romania, aurelianu2007@yahoo.com
Follow this link:
Genetic Events in Chronic Lymphocytic Leukemia - Cordis News
Vivet Raises 37.5M to Develop Gene Therapies for Rare Liver Diseases – Genetic Engineering & Biotechnology News
French startup Vivet Therapeutics raised 37.5 million (about $41 million) in a Series A round of financing to support the development of gene therapies for rare inherited metabolic diseases. The firm was set up in 2016 to develop treatments based on adeno-associated virus (AAV) vector technology licensed exclusively from its close collaborator, the Fundacin para la Investigacin Mdica Aplicada (FIMA), at the Center for Applied Medical Research (CIMA) in Pamplona, Spain, and from Massachusetts Eye and Ear (MEE) in Boston.
Novartis Venture Fund and Columbus Venture Partners led the Series A investment round. Roche Venture Fund, HealthCap, Kurma Partners, and Ysios Capital also participated.Florent Gros, managing director at Novartis Venture Fund, commented, "We have searched extensively for next-generation AAV technologies and clinical applications. We are very excited by Vivet Therapeutics' clinical and commercial prospects; the company has outstanding management, assets, and capabilities."
Based in Paris, and with a wholly owned subsidiary in Spain, Vivetaims to develop gene therapies targeting disorders including Wilson disease, progressive familial intrahepatic cholestasis (PFIC), and citrullinemia.The firm is usinga novel, synthetic AAV, AAV-Anc80, to introduce genes into hepatocytes.Lead Wilson disease gene therapy program VTX801 comprises a truncated, functional version of the defective ATP7B gene, delivered directly into liver cells using the AAV vector technology. First-in-human trials with VTX801 are projected to start by the end of 2018.
Jean-Philippe Combal, Pharm.D., Ph.D., Vivet co-founder and CEO, noted, Early results from preclinical studies with VTX801 are very promising, and we are now well funded to advance this candidate into the clinic, while developing our portfolio and technologies."
Vivet's co-founders includeCombal (ex-Gensight Biologics, Sanofi),Jens Kurth, Ph.D. (ex-Anokion, Novartis), and Gloria Gonzlez-Aseguinolaza, Ph.D. (CIMA, University of Navarra).On announcement of Series A fundraising, Gloria Gonzlez-Aseguinolaza, Vivet CSO, said, By collaborating with leading institutions such as CIMA in Spain and MEE in the United States, Vivet has secured superior and novel gene therapy technologies and liver disease expertise. We believe these capabilities, combined with the international development expertise of the management team, create a company with very exciting prospects."
Read this article:
Vivet Raises 37.5M to Develop Gene Therapies for Rare Liver Diseases - Genetic Engineering & Biotechnology News
Dimension Therapeutics Inc (DMTX) Upgraded at Zacks Investment … – The Cerbat Gem
Dimension Therapeutics Inc (DMTX) Upgraded at Zacks Investment ... The Cerbat Gem Dimension Therapeutics Inc (NASDAQ:DMTX) was upgraded by Zacks Investment Research from a hold rating to a buy rating in a research note issued to ... Dimension Therapeutics Inc (DMTX) Upgraded to Buy by Zacks ... |
Read the original:
Dimension Therapeutics Inc (DMTX) Upgraded at Zacks Investment ... - The Cerbat Gem
HIV Cure Research 2017: Gene Editing Tool CRISPR Cas9 Eliminated HIV In Animals – Medical Daily
The HIV virus is adeptat evading our best efforts to flushit from the human body, hence the lack of a cure, butanew study suggests thatthis feat may one day be possible. Researchers showed they were able to eliminate HIV from infected cells in animals using the powerful gene-editing tool, CRISPR/Cas9.
In the study, now published online in Molecular Therapy, scientists at the Lewis Katz School of Medicine at Temple University (LKSOM) and the University of Pittsburgh succeeded in removing HIV DNA from the genomes of living animals a major step toward a cure, they said. The team used CRISPR gene-editing technology to remove HIV from the DNA of three different animalmodels, including a mouse that was transplanted with a humanized immune system.
Read: HIV Cure 2017? New Research Suggests Way To Theoretically Eliminate Virus From Body
"We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy, said lead researcherWenhui Hu, in a recent statement. We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells."
This breakthrough means that the team have begun to tackle a serious hurdle in the race to cure HIV. The next step would be to repeat the resultsin primatesto see if they are able to eliminate HIV DNA in latently infected T cells, and other HIV hideouts such as brain cells.
Science continues to advance more effective ways to target HIV, bringing us closer to a cure. Photo Courtesy of Pixabay
"Our eventual goal is a clinical trial in human patients, said study co-author Dr. Kamel Khalili in a recent statement.
For the study, the team built on previous proof-of-concept research from 2016 that demonstrated the ability to target and remove fragments of HIV from the genome of most tissues in experimental animals. Now, the team has taken this a step further, removing the latent HIV from DNA from the tissues of living animals. In order to measure the success of of their attempt, the team used a live bioluminescence imaging system to measure levels of HIV RNA.
In its latent form, the virus is able to lie dormant, meaning that it has stopped the production of new HIV cells, but can become reactivated at random, Aids Info reported. However, when HIV-infected cells are not actively producing the virus, HIV medication has no effect on them. This is what makes it especially difficult to completely flush the virus out of the body. According to Aids Info, finding ways to target and destroy these latent reservoirs of HIV are critical to figuring out a way to clear the virus from the body, and gene therapy is at the forefront of this search. In gene therapy treatments, such as that done in this study, scientists manipulate genes in order to either cut out or deactivate HIV in infected immune cells.
Source: Yin C, Zhang T, Qu X, et al. In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. Molecular Therapy. 2017
See Also:
HIV Breakthrough 2017: Treatment May Replace Need For Daily ART Medications, Leaves 5 Patients With Undetectable Virus Levels
HIV Breakthrough 2017: Scientists Find Protein Marker To Identify Dormant Virus-Infected Cells
See the rest here:
HIV Cure Research 2017: Gene Editing Tool CRISPR Cas9 Eliminated HIV In Animals - Medical Daily