News and Trends 6 Jun 2018 German DNA Treatment Reduces Ulcerative Colitis Symptoms Sterna Biologicals has obtained positive Phase IIa data for an ulcerative colitis treatment that uses DNA to inhibit inflammatory proteins, which could add to the diverse approaches being developed to treat inflammatory bowel disease. Sterna Biologicals, based in Marburg, Germany, has clinical data showing its ulcerative colitis treatment reduced the severity of the disease compared […] June 6, 2018 - 3 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 3 May 2018 FDA Backs Gene Therapy for Fatal Neurodegenerative Childhood Disease Orchard Therapeutics has received a fourth Rare Pediatric Disease Designation from the FDA for its gene therapy candidate for metachromatic leukodystrophy. Orchard Therapeutics, based in London, has received a Rare Pediatric Disease Designation form the FDA for its gene therapy OTL-200. The treatment is for patients with metachromatic leukodystrophy (MLD), a fatal genetic disease that causes […] May 3, 2018 - 1 minutemin - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 3 May 2018 World’s First Gene Therapy Developer Raises $128M for Hemophilia and Huntington’s uniQure has raised a substantial public offering on the NASDAQ. The funding could help the Dutch biotech advance two new gene therapies after the withdrawal of its first treatment. Amsterdam-based uniQure has raised an ambitious $128M (€107M) in a public offering at the beginning of next week. The Dutch biotech develops gene therapies for hemophilia and Huntington’s disease, for […] May 3, 2018 - 2 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 25 Apr 2018 Cancer Treatment Targeting DNA Repair is Tested in Humans for the First Time Onxeo has initiated a Phase I trial testing its first-in-class cancer treatment that works by inhibiting the repair of tumor DNA. Onxeo, a French biotech, has dosed its first patient in a Phase I study testing its cancer treatment AsiDNA, the first treatment of its kind, for advanced solid tumors. The news follows the Phase […] April 25, 2018 - 1 minutemin - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 20 Apr 2018 Gene Therapy For Beta-Thalassemia Could Eliminate Need for Transfusions Bluebird bio has obtained results from two clinical studies showing its new gene therapy could eliminate the need for red blood cell transfusions in patients with beta-thalassemia. The company plans to file for an EU approval of its treatment later this year. US firm bluebird bio has results from two clinical studies showing its LentiGlobin gene […] April 20, 2018 - 2 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
Interview 18 Apr 2018 What Lies Ahead for European Cell and Gene Therapies? Cell and gene therapies have recently made big advances in providing effective treatments for a variety of conditions, from cancers to rare diseases. Nonetheless, there are still significant challenges ahead before the technologies can become a viable treatment option for most patients. What if you could treat a disease on the level of tissue and […] April 18, 2018 - 5 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 17 Apr 2018 How Low Can the Price of a Next Generation DNA Sequencer Go? The next generation of DNA sequencers has arrived, and with it, a clash of titans: Illumina, Thermo Fisher and Qiagen are all competing for the clinical diagnostics market. Next-generation sequencing (NGS) companies are redefining DNA sequencing. What began as small-scale PCR and Sanger sequencing efforts have now mushroomed into a $20Bn market with broad medical implications for […] April 17, 2018 - 5 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 4 Apr 2018 Placebo Beats a Gene Therapy for Blindness in a Phase 3 Trial GenSight has demonstrated that its gene therapy for a rare disease causing blindness can significantly improve the vision of patients. But so does the placebo. GenSight Biologics has released the results of a Phase III clinical trial testing its gene therapy GS010 in patients suffering from Leber hereditary optic neuropathy (LHON), a rare mitochondrial disease […] April 4, 2018 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
Interview 20 Mar 2018 Why It’s the Best Time to Be in Cell and Gene Therapy Cell and gene therapies are here, and they’re here to stay. We stopped to chat about the future of the field with Alexander Vos last week at Bio Europe Spring. The cell and gene therapy space has been given a big boost in the last couple of years. Since 2016, GSK’s gene therapy Strimvelis is available […] March 20, 2018 - 3 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 6 Mar 2018 Oxford Spin-out Moves Gene Therapy For Blindness Into Phase III Trial Nightstar Therapeutics will begin the first Phase III trial for a gene therapy targeting choroideremia, a rare disorder leading to complete blindness. Nightstar will launch the first-ever Phase III trial testing a gene therapy for choroideremia, a genetic disorder characterized by night blindness and gradual loss of vision that eventually leads to complete blindness. The trial, STAR, […] March 6, 2018 - 2 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 20 Feb 2018 This Gene Therapy for Vision Loss is Safe and Effective After Two Years The results of a long-term follow-up suggest that GenSight’s gene therapy for Leber hereditary optic neuropathy can provide safe and effective treatment for the genetic cause of vision loss. GenSight develops gene therapies to preserve or restore vision in patients with neurodegenerative diseases of the retina. The biotech’s lead candidate is GS010, which is currently […] February 20, 2018 - 3 minutesmins - By Alex Dale Share WhatsApp Twitter Linkedin Email
News and Trends 31 Jan 2018 Gene Synthesizing Biotech Gets $12M to Boost the Synbio Field Evonetix has closed a Series A fundraising round to support the development of its gene synthesis platform, an important tool in the emerging synbio field. Evonetix, based in Cambridge, UK, is developing a scalable, high fidelity gene synthesis approach that could be a key tool in the growing synbio field. The biotech has closed a […] January 31, 2018 - 3 minutesmins - By Alex Dale Share WhatsApp Twitter Linkedin Email