News and Trends 8 May 2020 Phase III Failure Sinks Drug for Rare Neurological Disorder The Italian biotech Newron is halting the development of the experimental drug sarizotan after it failed to meet efficacy endpoints in phase III, dashing hopes for an approved drug for Rett syndrome. Expectations had been high that the oral drug might be able to treat breathing difficulties associated with the rare genetic disorder after promising […] May 8, 2020 - 4 minutesmins - By Anita Chakraverty Share WhatsApp Twitter Linkedin Email
News and Trends 2 Apr 2020 Update: FDA Imposes No Penalties for Novartis Data Manipulation Scandal Update (02/04/2020): The FDA has concluded an investigation into a data manipulation scandal surrounding the approval of Novartis’ gene therapy Zolgensma, and has decided against sanctions. The FDA launched an investigation into the manipulated data surrounding the gene therapy application last year, and recently reported that the investigated data violations didn’t meet the threshold for […] April 2, 2020 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 31 Mar 2020 Ipsen Resumes Phase III Testing for Rare Disease Drug and Abandons Pediatric Trial The French pharmaceutical company Ipsen has restarted the dosing of its treatment for a rare bone disease in a phase III trial after obtaining questionable interim results. At the same time, the company has terminated a phase II trial of the same drug in pediatric patients with a different bone disorder following a partial clinical […] March 31, 2020 - 4 minutesmins - By Karen O’Hanlon Cohrt Share WhatsApp Twitter Linkedin Email
News and Trends 10 Feb 2020 First-in-Class Drug For Inherited Metabolic Diseases Funded with €25M The Dutch startup Azafaros has raised €25M in Series A funding to push its drug for inherited lysosomal storage disorders into phase I. The round was led by the Dutch VC firm Forbion. Other investors participating in the round included the Swiss firm BioMedPartners and the Dutch company BioGeneration Ventures, which was Azafaros’ founding investor […] February 10, 2020 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
Interview 5 Feb 2020 Harnessing Genetic Suppression to Treat Rare Disease Through quirks of genetics, some people are naturally resilient to heritable diseases. Thijn Brummelkamp, Managing Director and founder of the Dutch startup Scenic Biotech, explains how the company is using this genetic resilience to treat rare diseases and cancer. Heritable diseases result from genetic mutations that cause essential proteins in the body to malfunction. For […] February 5, 2020 - 5 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 26 Nov 2019 Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III A small molecule drug developed by the US company ChemoCentryx and its Swiss partner Vifor Fresenius Medical Care Renal Pharma (VFMCRP) was more effective at treating a rare inflammatory vascular disease than current treatments in a phase III trial. The drug avacopan was trialed in 331 patients with anti-neutrophil cytoplasmic antibody-associated vasculitis. This life-threatening condition […] November 26, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 11 Nov 2019 Mereo BioPharma’s Brittle Bone Disease Drug Stumbles in Phase II The lead drug of the UK company Mereo BioPharma has achieved mixed results in a phase IIb trial in adults with the genetic condition osteogenesis imperfecta, also known as brittle bone disease. Mereo tested different doses of its antibody drug in 112 adults with osteogenesis imperfecta in a 12-month phase IIb trial. The drug failed […] November 11, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 7 Nov 2019 €51M Nasdaq IPO Will Fund Centogene’s Rare Disease Diagnostics The German company Centogene has raised €50.6M ($56M) in an IPO on the Nasdaq Stock Exchange to fund the development of diagnostics technology that could improve the treatment of rare diseases. The fundraise fell under Centogene’s target value of €54M ($60M). A part of the proceeds will fuel the development of biomarkers that Centogene will […] November 7, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 16 Oct 2019 UK Firm Raises €51M to Repurpose Drugs for Rare Diseases Using AI The Cambridge-based artificial intelligence company Healx has raised a Series B round of €50.8M to launch an AI tool that reduces the time it takes to develop treatments for rare diseases by five years. The Series B round was led by the UK VC firm Atomico and includes the returning investor Jonathan Milner, the founder […] October 16, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
Startup Scout 20 Sep 2019 This Biotech Targets Mitochondria to Treat Inherited Diseases As we come to the end of the global Mitochondrial Disease Awareness Week, it’s time to visit Khondrion. This Dutch company is developing drugs to treat rare diseases such as MELAS syndrome by targeting defective mitochondria. Mission: To develop small molecule drugs able to treat Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) syndrome. This […] September 20, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
More News! 11 Sep 2019 Dutch Company Raises €60M to Treat Inherited Inflammatory Disease The Dutch pharmaceutical company Pharvaris has raked in €60M in a Series B round to fund the clinical development of the first oral treatment for hereditary angioedema, a condition that causes swelling in the limbs and airways. The Series B round will fuel the clinical progress of Pharvaris’ oral small molecule treatment for hereditary angioedema, […] September 11, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 4 Jun 2019 First Gene Therapy for Beta Thalassemia Approved in Europe The US biotech company bluebird bio has obtained EU conditional market approval for a gene therapy that could remove the need for blood transfusions for people with the blood disorder beta-thalassemia. Called Zynteglo, the gene therapy is aimed at treating people with beta-thalassemia who are over 12 years of age and have any but the […] June 4, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email