news
mRNA therapy could provide intracellular protein replacement for rare disease
With no treatments approved for propionic acidaemia, Moderna’s LNP-encapsulated mRNA therapy could offer a new therapeutic option for patients, new data suggests.
List view / Grid view
Clinical Trials
news
First-in-class anaemia therapy granted expanded approval
The expanded authorisation of Reblozyl is based on a Phase III trial which showed that the treatment nearly doubled the percentage of certain transfusion-dependent anaemia patients achieving haemoglobin increase, compared to epoetin alfa.
article
Championing UK ATMP clinical trials
In this Q&A, Jacqueline Barry, Chief Clinical Officer for Cell and Gene Therapy Catapult, elucidates on how the Advanced Therapy Treatment Centre (ATTC) Network can strengthen UK ATMP clinical trials.
news
One-time gene therapy could improve wet AMD
A Phase I/IIa trial suggests that a subretinal gene therapy for wet age-related macular degeneration (wet AMD) could reduce the need for patients to receive anti-VEGF injections.
news
Novel LAI antipsychotic approved in EU
The long-acting injectable (LAI) formulation is indicated as a once-every-two-months treatment for adults with schizophrenia.
article
Clinical trials for rare diseases
In this interview, Mindy Leffler, Managing Director of Qualitative Research and Psychometrics at Emmes Endpoint Solutions, discusses the nuances of designing clinical trials for rare diseases including specific challenges related to traditional endpoints.
news
Positive CHMP recommendation for antibiotic combination
EMA has recommended granting a marketing authorisation for Emblaveo for treatment of infections caused by multidrug-resistant bacteria.
news
Novo Nordisk to buy Cardior Pharmaceuticals
The up to €1bn acquisition of Cardior Pharmaceuticals will strengthen Novo Nordisk’s pipeline in cardiovascular disease.
news
Combination treatment could minimise cancer relapse risk
Data suggests that ribociclib plus endocrine therapy could provide a new treatment option more patients with HR-positive, HER2-negative early breast cancer.
news
UK advanced therapies to get £17.9 million boost
The initiative aims to support the UK as a location of choice for advanced therapy research, according to the Cell and Gene Therapy Catapult.
news
Novartis acquisition to address inflammation-driven diseases
Novartis’ new acquisition aims to advance first-in-class STING treatments for inflammation-driven diseases.
news
AstraZeneca agrees novel $1 billion endocrine acquisition
As part of the proposed acquisition, Alexion (AstraZeneca Rare Disease) will add a therapeutic to its pipeline that has potential to “shift the treatment paradigm” in hypoparathyroidism.
news
Parkinson’s infusion treatment demonstrates advantage over oral delivery
Delivering the traditional Parkinson’s treatment via subcutaneous infusion over a 24-hour period was shown to be safe and effective, research found.
news
FDA approval could shape liver disease treatment landscape
A novel liver therapy authorised under US Food and Drug Administration (FDA) Accelerated Approval, is a potential game-changer in the NASH treatment space.
article
Developing a new treatment paradigm for IgAN
On World Kidney Day 2024, Dr Marshall Fordyce, CEO of Vera Therapeutics explores the challenges of treating kidney disease and shares why the company’s lead candidate could become the first self-administered B-cell modulation therapy.
