news
Novo Nordisk investing in rare disease with major new manufacturing site
A total of DKK 8.5 billion will fund the new production facility in Odense, Denmark, which is set to facilitate the manufacture of medicines for rare diseases.
List view / Grid view
Rare diseases
news
Novartis to advance novel Huntington’s disease therapy
As part of the agreement with Novartis Pharmaceuticals, PTC will receive an initial $1 billion to develop the potential first-in-class oral therapy.
news
Innovating LNP design to improve mRNA therapeutics
The new approach involving refinement and optimisation of ionisable lipids could accelerate the development of mRNA therapies, research suggests.
news
Sanofi collaboration to advance innovative radioligand therapies
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.
news
AAV gene therapy manufacturing gets £1.4m boost
The funding aims to reduce the costs of AAV production for gene therapies, helping to accelerate treatment access for patients with rare diseases.
article
Enzyme replacement therapy: improving outcomes in rare disease
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).
news
Gene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
news
European Commission licenses first treatment for acanthamoeba keratitis
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
news
FDA approves innovative engineered cell therapy
The accelerated approval by the US Food and Drug Administration (FDA) authorises the first engineered cell therapy for a solid tumour in the US.
article
The route to enhanced gene therapies
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.
news
CHMP meeting highlights – June 2024
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.
article
Drug approval roundup – May/June 2024
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.
article
Why patient-centricity is essential to accelerate rare disease clinical research
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…
news
CHMP meeting highlights: April 2024
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
news
Benefits of 3D printing for personalised medicine formulation
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.
