Category Archives: Gene Therapy

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering. 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. 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 The voluminous literature on gene therapy was reviewed … Continue reading →

July 06, 2017 07:00 ET | Source: Shire Pharmaceuticals Group Shire submits investigational New Drug Application to FDA for Gene Therapy candidate SHP654 for treatment of Hemophilia A SHP654 aims to deliver sustained protection against bleeds for patients with hemophilia A Lexington, Mass.-July 6, 2017- Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology company focused on serving people with rare diseases, today announcedthe submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. SHP654 aims to protect hemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression.1 The IND filing for SHP654 represents the latest step forward for Shire's gene therapy program, which shows promise for both hemophilia A and B populations. "Shire is leveraging decades of scientific leadership in hemophilia to advance research in gene therapy for this community," said Paul Monahan, M.D., Senior Medical Director, Gene Therapy, Shire. "Drawing from our rich heritage, Shire is well equipped to sustainably support the development of gene therapies that aim to advance current standards of care and minimize … Continue reading →

Shire has submitted a request to the US Food and Drug Administration to test its investigational factor VIII (FVIII) gene therapy SHP654 in patients with the bleeding disorder haemophilia A. SHP654 (also known as BAX 888) aims to protect haemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression, the firm noted. Shires gene therapy programme for haemophilia A uses a recombinant adeno-associated virus serotype 8 (rAAV8) vector that selectively targets the liver. It involves the delivery of a functional copy of FVIII to the bodys liver to enable its own production of FVIII, rather than relying on a factor-based treatment. The Investigational New Drug submission is based on the results of preclinical and phase I studies showing the potential utility of its candidate, the firm noted. If Shire gets a green light for human testing it intends to study SHP654 in a global multi-center trial evaluating safety and examining the doses required to boost factor VIII activity levels and affect haemophilic bleeding. See more here: Shire applies to test haemophilia A gene therapy in humans - PharmaTimes … Continue reading →

news This latest funding round attracts two prominent new investors; Brian Kennedy and Sir Brian Souter. Ryboquin Limited, a Scottish Borders based pharmaceutical company, announces that it has closed a 1.8m equity fund raising to accelerate product development in gene therapy. In addition to support from existing shareholders, including Borders business angel group, TRI Capital and the Scottish Investment Bank (the investment arm of Scottish Enterprise), funding has also been provided by Brian Kennedy, the highly successful Scottish entrepreneur and Sir Brian Souter, the founder and Chairman of Stagecoach. Brian Kennedy will also join the Board. Founded in 2013, Ryboquin is a privately held, pharmaceutical company focused on commercialising patented intellectual property in the area of delivering gene therapy primarily in the field of human cancer medicine. Ryboquin is in partnership with Nanogenics to promote the targeted nucleic acid delivery system LipTide. The funds being raised will be used to further scientific development as well as providing funding for corporate expansion. Paul Murray, Executive Chairman, Ryboquin, says: The support from existing shareholders and the investment by Brian Kennedy and Sir Brian Souter, two esteemed leaders and hugely successful businessmen, is testimony to the potential of Ryboquin and to the work … Continue reading →

Boston Business Journal After raising $50M, virtual gene therapy startup sets up shop in Cambridge Boston Business Journal LogicBio CEO Frederic Chereau Enlarge. LogicBio CEO Frederic Chereau. LogicBio Therapeutics becomes the latest local entrant to the field of gene therapy, a method of inserting healthy genes into cells to replace missing or faulty ones. and more » Read the original post: After raising $50M, virtual gene therapy startup sets up shop in Cambridge - Boston Business Journal … Continue reading →

Author's note: The following consists of excerpts from my 45-page May 30 report on bluebird bio (NASDAQ:BLUE), Kite Pharma (NASDAQ:KITE), and Juno Therapeutics (NASDAQ:JUNO). The focus in this submission is BLUE. Please check out my Seeking Alpha profile for important information. Global Gene Therapy Market The gene therapy market is gaining popularity in the global medical community. The advent of advanced techniques for gene transfer has enabled the use of gene therapy for various new applications. Although it is still at an infant stage, its promise has led to a range of bullish estimates. Market research firm BCC Research forecasts the global market for DNA vaccines to grow at a 54.8% CAGR to $2.7 bln by 2019, while two other observers - Roots Analysis and Research and Markets - predict the gene therapy market as a whole to reach ~$11 bln by 2025. Another report from market intelligence firm Transparency Market Research forecasts that the global stem cell market will grow at a CAGR of > 20% in the next few years and said there is a rich pipeline of more than 500 cell and gene therapy products, which will drive significant capacity as the pipeline matures and progresses to … Continue reading →

Xconomy Boston Gene therapy offers the potential for a long-lasting, if not permanent, treatment for an inherited disease, but cells that divide rapidly, such as those in the liver, present a thorny problem. Because of how they insert themselves in the cells, some forms of gene therapy get diluted as the cells divide. Its a particular problem in growing children. Cambridge, MA-based LogicBio says it has developed a workaround by combining gene editing with gene therapy. The firm has raised $45 million in additional capital to help bring this technology into human testing, and it is moving from California to the LabCentral shared incubator space in Cambridges Kendall Square. LogicBio calls its technology GeneRide. The company says its approach can transfer genetic material to specific sites to repair a faulty genetic sequence. The companys focus is metabolic disorders that affect the liver in children. Published research shows that metabolic disorders of the liver can progress to injury affecting other organs. In rare cases, the severity of the disease requires a pediatric liver transplant. If GeneRide works as the company envisions, the gene therapy would offer a one-time treatment that avoids side effects. London-based Arix Bioscience (LSE: ARIX) led the Series … Continue reading →

LogicBio Therapeutics has got off a $45 million series B funding round as it eyes the cash for disease-modifying gene therapies in rare childhood diseases. London-based investment firm Arix Bioscience led the oversubscribed round in the semi-stealth biotech, with new investors OrbiMed, Edmond De Rothschild Investment Partners, Pontifax, and SBI, along with previous investors OrbiMed Israel Partners, also stumping up cash. Arix Bioscience's investment manager, Daniel OConnell, M.D., Ph.D, will join Cambridge, Massachusetts-based LogicBios board as part of the raise. This brings its total raised to $50 million, much of which will be put toward finishing off preclinical work and moving them into human tests. The biotech sets itself up as a breakthrough gene therapy company targeting lifelong cures for serious, early-onset rare diseases by combining the best of gene therapy and gene editing in a one-time treatment. It was founded in 2014 with platform technologies discovered by Adi Barzel, Tel Aviv University, Dr Leszek Lisowski, Childrens Medical Research Institute, Australia, and Professor Mark Kay at Stanford University School of Medicine. The first platform, GeneRide, is a technology that uses homologous recombination that is designed to allow site-specific transfer of therapeutic genetic material without the use of promoters or nucleases. … Continue reading →

An 11-year-old girl in Massachusetts is at the forefront of a disease so rare, that it is believed only 22 people worldwide have been diagnosed with it. Talia Duff, who was born with Down syndrome and later diagnosed with Charcot-Marie-Tooth Neuropathy Type 4J (CMT4J), is slated to be among the first to enroll in a clinical trial that is awaiting FDA approval after her parents refused to watch her fall victim to the degenerative genetic disease. Its a horrible feeling to go to a doctor and be told that theres nothing that can be done that the best you can do is try to make your child comfortable and enjoy the time you have together, John Duff, Talias dad, told PEOPLE. I learned to cherish moments in life that I would otherwise take for granted. PREGNANT MOM DELAYS CANCER TREATMENT TO PROTECT UNBORN TWINS The Duff family, which includes mom Jocelyn and older sister Teaghan, had noticed Talia struggling to crawl at around age four, and a regression in a number of other motor skills that at the time was attributed to her Down syndrome, and later to Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). Subsequent failed therapies and a diagnoses of … Continue reading →

Dimension Therapeutics is winnowing out 25% of its staff as it concentrates on three gene therapy programs, including one partnered with Bayer that has the potential to generate some badly needed milestone cash. The biotech says it has enough revenue in hand to operate for another year, adding that it can extend the runway out to the end of 2018, provided it bags about $15 million in cash in its deal with Bayer. Three years ago Dimension inked a $252 million pact with Bayer, with $20 million of that upfront. Annalisa Jenkins Counting milestone money in your business plan isnt likely to generate much confidence among investors, especially after some disappointing results and evidence of liver toxicity for its initial lead gene therapy for hemophilia B in January crushed the biotechs stock price. DTX101 which faced more advanced competitors with better data has now been shoved out of the spotlight. The biotechs market cap has now shrunk to $38 million. The lead program in the clinic now is DTX301 for rare cases of ornithine transcarbamylase (OTC) deficiency. The biotech is lining up two more programs for INDs, including DTX201 allied with Bayer. Dimension was one of several gene therapy companies … Continue reading →