Startup Scout 31 May 2019 This Biotech Treats a Neurological Autoimmune Disorder Using DNA We’re jumping into Poland this week. Pure Biologics is a biotech based in Wrocław developing DNA molecules that could treat the incurable neurological condition Devic’s disease with fewer side effects than existing treatments. Mission: To develop a treatment that makes an existing treatment for Devic’s disease, plasmapheresis, more targeted and with fewer side effects than […] May 31, 2019 - 4 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 9 May 2019 Pfizer Pays up to €700M to Acquire Swiss Biotech Restoring Bone Growth in Achondroplasia Pharma giant Pfizer is acquiring the Swiss biotech Therachon, which is developing a protein drug to boost bone growth in the genetic condition achondroplasia. Pfizer will pay €303M ($340M) upfront, and up to €420M ($470M) dependent on Therachon reaching milestones in the development of its drug improving bone growth in achondroplasia. This genetic condition inhibits […] May 9, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 28 Mar 2019 Gene Therapy Exceeds Expectations in Treating Children’s Neurological Disease A gene therapy developed by the UK company Orchard Therapeutics has greatly improved the motor symptoms of children with an incurable neurological disease. The 20 children in the phase II trial suffered from metachromatic leukodystrophy, a rare genetic disease caused by the mutation of a protein that breaks down sulfur-containing molecules in cells. This leads […] March 28, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 4 Mar 2019 Biogen Acquires UK Biotech Developing Gene Therapy for Blindness The big US biotech Biogen has agreed to acquire Nightstar Therapeutics for €773M ($877M), including Nightstar’s candidate gene therapies for inherited blindness. Expected to be complete by mid-2019, Biogen’s acquisition of Nightstar bags it the UK company’s most advanced program. Currently in phase III, the therapy targets choroidemia, an incurable genetic condition which causes progressive […] March 4, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 30 Jan 2019 Danish Biotech’s Drug for Rare Metabolic Disease Aces Clinical Trial A drug, developed by the Danish biotech Orphazyme for a rare metabolic disease, has shown promising results in a phase II/III trial, reducing the disease’s progression by 74%. The 50 patients recruited into the trial suffered from Niemann-Pick disease type C, a metabolic disease in which proteins crucial for storing fat molecules such as cholesterol […] January 30, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 8 Jan 2019 Topical Gene Therapy Shows Early Promise for Inherited Skin Disease A gene therapy applied to the skin, developed by UK company Amryt Pharma, showed positive preclinical results for the treatment of a rare genetic skin disease. The disease, called recessive dystrophic epidermolysis bullosa (RDEB), is one of a group of diseases in which sufferers have mutations in proteins vital to the integrity of the […] January 8, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 7 Dec 2018 Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year A single dose of a gene therapy developed by Orchard Therapeutics can restore immune function for at least a year in patients with X-linked chronic granulomatous disease (X-CGD). The gene therapy was trialed in patients between the ages of 2 and 27 with the rare disease, which is caused by a rare genetic mutation that […] December 7, 2018 - 3 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
Interview 28 Nov 2018 Navigating the Rare Disease Space: Insights from a Danish CEO Developing drugs for rare diseases is a tough nut for biotech companies to crack. Labiotech’s Helen Albert interviewed the CEO of the Danish company Orphazyme, Anders Hinsby, at the recent BIO-Europe conference in Copenhagen, who told her about the appeals and challenges of making it in the rare disease space. Traditionally, the low number of […] November 28, 2018 - 5 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 22 Nov 2018 First Drug for Rare Inflammation Syndrome Approved by FDA The FDA has approved the first drug for the treatment of a rare condition in which the body produces too many active immune cells, developed in a Swiss-Swedish partnership. The antibody drug, emapalumab, is approved to treat primary hemophagocytic lymphohistiocytosis. This condition is a rare, often fatal syndrome in which the immune system produces too […] November 22, 2018 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 2 Nov 2018 Gut Bacteria Treatment Shows Promise for Rare Genetic Kidney Condition The Swedish biotech Oxthera reported positive preliminary Phase II results using its gut bacteria treatment, Oxabact, in patients with a rare genetic condition that causes kidney failure. The results came from an interim analysis one year into a three-year trial, where Oxthera is testing its gut microbiome treatment on patients with primary hyperoxaluria type […] November 2, 2018 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 16 Oct 2018 Drug Combination for Rare Genetic Nerve Disorder Nails Phase III A treatment developed by French biotech Pharnext has become the first clinically proven therapy for a rare genetic disorder that affects the nervous system. In a Phase III trial, the treatment reduced the level of disability of patients with Charcot-Marie-Tooth disease, a condition that affects peripheral nerves. This disease causes problems with walking, running and […] October 16, 2018 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 9 Oct 2018 Hormone Therapy for Inherited Hormonal Disorder Fails at Phase III A European Phase III trial has become a major stress for British biotech Diurnal. Its specially designed hormone therapy was no better than conventional therapies in treating an inherited hormonal disorder. Congenital adrenal hyperplasia is a rare genetic condition where patients lack crucial enzymes for making hormones. The most common form lacks 21-hydroxylase, the enzyme […] October 9, 2018 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email