Chiesi, the biopharmaceutical and healthcare group, has announced that the National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as a treatment for Fabry disease in adults.
mRNA pioneer Moderna wants to grow and thrive past its initial COVID-19 vaccine success. “To continue to build the best version of Moderna, we have established seven priorities for 2023,” says CEO Stephane Bancel: as the company outlines goals that range...
Curamys, a South Korean biotech developing cell and gene therapy using cell fusion technology to treat rare intractable diseases, has signed a strategic platform licensing agreement with US tech developer, MaxCyte.
Cardiff-based CatSci is investing in its oligonucleotides capability that will enable the company to make oligos from nanomole quantities up to 30 grams using solid phase synthesis.
The US Food and Drug Administration (FDA) has awarded 19 new grants and two new contracts totaling more than US$38m in funding over the next four years to support clinical trials and natural history studies related to rare diseases.
Moderna and the nonprofit Institute for Life Changing Medicines (ILCM) are collaborating to develop a new messenger RNA (mRNA) therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type 1 (CN-1), an ultra-rare disease.
A robust research and innovation ecosystem makes Italy an attractive life sciences hotspot, according to a post from an Italian trade delegation at Bio Digital 2021.
A program at the University Hospital of Tübingen is the first in Germany to use whole genome sequencing (WGS) to improve the diagnosis of rare diseases and hereditary cancers.
New York based, Neurogene, has announced a research collaboration with the University of Edinburgh for the development of a multiple-platform approach to diseases not addressable by conventional gene therapy.
A new multi-stakeholder group of experts has been set up to provide input and ideas in the context of the ongoing evaluation of the orphan medicinal products (OMP) regulation in Europe.
Paragon and Passage Bio enter a collaboration agreement to develop a dedicated manufacturing suite at the former’s Maryland facility, as the latter’s gene therapy candidates enter the clinic.
As technology has advanced, rare diseases have been met with therapies that target the cause of the condition, enabling the global market for orphan drugs to reach an expected $262bn by 2024.
Alexion has agreed to pay up to $1.2bn for Syntimmune and its lead asset SNT001, a monoclonal antibody designed to treat warm autoimmune haemolytic anaemia.
PTC Therapeutics will acquire Agilis Biotherapeutics to continue work on gene therapy for rare monogenic diseases affecting the central nervous system.
