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AACR: BTK degrader could treat CNS B-cell malignancies
New data suggests the small molecule therapeutic could also benefit autoimmune disorders with involvement in the central nervous system, such as multiple sclerosis.
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Clinical Trials
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Advancing microbiome innovation
In the fourth instalment of EPR's ‘Microbiome therapeutics: microscope to medicine’ series, Emilie Plantamura, Deputy Chief Medical Officer at MaaT Pharma, examines the promising potential of microbiome therapeutics beyond Clostridium difficile infection, particularly in the onco-haematological field.
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Antibody-immunotherapy treatment shows benefit in serious endometrial cancer
The novel combination cancer therapy facilitated “exceptional” responses in some patients enrolled in a Phase II study for endometrial cancer.
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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.
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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.
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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.
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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.
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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.
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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.
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Positive CHMP recommendation for antibiotic combination
EMA has recommended granting a marketing authorisation for Emblaveo for treatment of infections caused by multidrug-resistant bacteria.
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Novo Nordisk to buy Cardior Pharmaceuticals
The up to €1bn acquisition of Cardior Pharmaceuticals will strengthen Novo Nordisk’s pipeline in cardiovascular disease.
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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.
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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.
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Novartis acquisition to address inflammation-driven diseases
Novartis’ new acquisition aims to advance first-in-class STING treatments for inflammation-driven diseases.
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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.
