The Coming of Age Story of RNAi Technology
The first RNA interference therapy was approved in 2018, after 20 years in development. What…
02/06/2021 - 6 minutes
Rare disease
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…
27/05/2020 - 11 minutes
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…
19/05/2020 - 5 minutes
Phase III Failure Sinks Drug for Rare Neurological Disorder
The Italian biotech Newron is halting the development of the experimental drug sarizotan after it…
08/05/2020 - 4 minutes
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…
02/04/2020 - 3 minutes
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…
31/03/2020 - 4 minutes
Sponsored
by CENTOGENE
How Can We Improve the Diagnosis and Treatment of Rare Diseases?
More people are impacted by rare diseases than by cancer and AIDS combined. With 400…
17/02/2020 - 7 minutes
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…
10/02/2020 - 2 minutes
Harnessing Genetic Suppression to Treat Rare Disease
Through quirks of genetics, some people are naturally resilient to heritable diseases. Thijn Brummelkamp, Managing…
05/02/2020 - 5 minutes
Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III
A small molecule drug developed by the US company ChemoCentryx and its Swiss partner Vifor…
26/11/2019 - 3 minutes
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…
11/11/2019 - 2 minutes
€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…
07/11/2019 - 2 minutes
