News and Trends 23 Aug 2022 Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome Ocelot Bio, Inc., a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of hepatorenal syndrome. Ocelot Bio is currently enrolling patients in a phase 2 clinical trial of OCE-205 in hepatorenal syndrome with acute kidney injury (HRS-AKI), […] August 23, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 22 Aug 2022 Vaderis emerges from stealth to start HTT trial Vaderis Therapeutics AG, a clinical stage biotech company focused on developing treatments for rare diseases associated with vascular malformations, has emerged from stealth. The company has also started its INSIGHT proof-of-concept clinical trial in patients suffering from hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome. About HHT HHT is a disorder where some blood […] August 22, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 19 Aug 2022 Emtora to use $16.9M grant to develop treatment for rare genetic disease Emtora Biosciences, a Texas-based clinical-stage biopharmaceutical company, has been awarded a $16.9 million non-dilutive grant from the Cancer Prevention & Research Institute of Texas (CPRIT). The funding will be used to advance the clinical and product development of its lead program, eRapa, an encapsulated form of rapamycin, for the treatment of familial adenomatous polyposis (FAP). […] August 19, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 19 Aug 2022 Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok Aeglea BioTherapeutics, Inc. says its marketing authorization application (MAA) for pegzilarginase for the treatment of arginase 1 deficiency (ARG1-D) has been validated by the European Medicines Agency (EMA). The MAA was submitted by Immedica Pharma AB, Aeglea’s commercialization partner in Europe and the Middle East. Pegzilarginase is a novel, recombinant human arginase 1 enzyme that […] August 19, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 22 Jul 2022 The coming of age story of RNAi technology The first RNA interference (RNAi) therapy was approved in 2018, after 20 years in development. What can we expect to see in the RNAi field in the future? After two decades of scientific effort, multiple devastating clinical trial setbacks, and billions of dollars spent on research and development, RNAi therapies are finally establishing themselves in […] July 22, 2022 - 7 minutesmins - By Farhan Mitha Share WhatsApp Twitter Linkedin Email
News and Trends 21 Jul 2022 Almirall launches new call to find innovative therapies for skin diseases A new call for new therapies to treat skin diseases has been launched by global Spanish-based biopharmaceutical company, Almirall, S.A. This is the seventh call through AlmirallShare – an open innovation platform for proposals to establish collaborations in dermatological research. The aim of this year’s edition is to find new partnership opportunities for assets in […] July 21, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 22 Mar 2022 Biotechs Carve out Niches in Cystic Fibrosis Treatment Space Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus that affects the lungs and other organs. Emerging biotechs such as Kither Biotech as well as the old guard including Vertex are finding new ways to tackle the inherited disease. Cystic fibrosis is best known for causing thick mucus in […] March 22, 2022 - 5 minutesmins - By Anita Chakraverty Share WhatsApp Twitter Linkedin Email
News and Trends 10 Mar 2022 Emerging Startups Take Aim at Lysosomal Storage Disorders Lysosomal storage disorders such as Niemann-Pick type C have proved difficult to tackle in the biotech industry. With recent funding rounds in the bag, Scenic Biotech and Maze Therapeutics strive to treat these conditions by activating protective genes. Lysosomal storage disorders are inherited conditions caused by mutations in metabolic enzymes found inside our cells, leading […] March 10, 2022 - 4 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 1 Dec 2021 Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones New, more targeted treatments are on the horizon for the rare autoimmune disease ANCA-associated vasculitis, with a nod towards European approval for a first-in-class drug and positive phase II results from a rival treatment. In November, the Swiss firm Vifor Fresenius Medical Care Renal Pharma (VFMCRP) received a thumbs-up from the EMA for avacopan, a […] December 1, 2021 - 4 minutesmins - By Anita Chakraverty Share WhatsApp Twitter Linkedin Email
Infographics by Merck 15 Nov 2021 Gene therapy: Making Rare Disease Therapies Less Rare While individually uncommon, rare diseases collectively impact the lives of over 300 million people worldwide. Many rare conditions are chronic, progressive, and life-threatening with limited treatment options available. Advances in gene therapy provide hope to actual cures and could revolutionize treatment prospects for patients with rare genetic diseases. This infographic shows how we can unlock […] November 15, 2021 - 2 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
Expert Advice 27 May 2020 Why Listening to Patient Organizations is Key in Drug Development As the biotech industry flourishes and therapies become more targeted and personalized, the need for drug developers to interact with patients is increasing as well. While patient organizations took a backseat in the past, they are continuously gaining importance in drug development today. Nowadays, talking with patient organizations has become paramount for successful drug development, […] May 27, 2020 - 11 minutesmins - By Larissa Warneck-Silvestrin Share WhatsApp Twitter Linkedin Email
News and Trends 19 May 2020 Update: Novartis’ Gene Therapy Zolgensma Gets Nod from EU Update (19/05/2020): The gene therapy Zolgensma, developed by the big pharma Novartis and approved in the US last year, has received conditional approval in Europe for the treatment of spinal muscular atrophy (SMA). The gene therapy is approved to treat patients with a clinical diagnosis of SMA type 1 or SMA patients that have up […] May 19, 2020 - 5 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email