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The new partnership between Sanofi and Orano Med, delivered via a new entity valued at nearly $2 billion, will focus on progressing lead-212 based targeted alpha therapies for oncology indications.
The funding aims to reduce the costs of AAV production for gene therapies, helping to accelerate treatment access for patients with rare diseases.
In this article, Dr Alaa Hamed, Global Head of Medical Affairs for Rare Disease at Sanofi, shares why the company’s enzyme replacement therapy Xenpozyme® (olipudase alfa) has promise for the rare genetic disease acid sphingomyelinase deficiency (ASMD).
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
The accelerated approval by the US Food and Drug Administration (FDA) authorises the first engineered cell therapy for a solid tumour in the US.
In this article, Roger Palframan, Head of US Research at UCB, delves into the potential of gene therapy and which modalities have the most promise, what the industry should prioritise to advance the field, as well as what is needed to develop the workforce.
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.
There is an urgent need for novel treatment options for people living with rare diseases, but carrying out clinical research in this field is notoriously difficult. As Clinical Trials Day approaches, Cezary Statuch, Head of Medical Affairs International and Japan at Alexion Pharmaceuticals, highlights how adopting patient-centric approaches can help…
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
A study has demonstrated how 3D printing can be used to manufacture tailored, safe, and effective treatments for patients with rare diseases via pharmaceutical compounding.
Data from the pre-specified interim analysis of Fabhalta® (iptacopan) indicate a statistically significant reduction in proteinuria reduction.
With no treatments approved for propionic acidaemia, Moderna’s LNP-encapsulated mRNA therapy could offer a new therapeutic option for patients, new data suggests.
At the Committee for Medicinal Products for Human Use (CHMP)’s March meeting, twelve medicines received positive opinions for marketing authorisations, including a new antibiotic and a novel oral medicine for a rare blood disorder.
