FDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
The EC’s approval of the immuno-oncology treatment “will define a new standard of care for certain patients with advanced or recurrent endometrial cancer in the EU,” says principal investigator of the RUBY trial.
In this article, Dr Michael Flanagan discusses how a new class of medicines called antibody oligonucleotide conjugates (AOCs) have the potential to overcome a major challenge encountered with many established RNA-based therapeutics: delivery to tissues outside the liver. Last year, AOCs demonstrated the first-ever successful targeted delivery of RNA into…
The UK MHRA has authorised Eli Lilly's Mounjaro (tirzepatide) for weight loss and weight management in adults.
AbbVie plans to acquire neuroscience specialist Cerevel Therapeutics for $45 per share, or around $8.7 billion.
In this video, experts from Curia discuss trends in the pharmaceutical industry, including move to adopt continuous flow chemistry processes for both mRNA and small molecule manufacturing.
Improving data harmonisation and investing in the advanced therapy sector’s workforce are some of the key recommendations the sector must take to advance the field and increase patient access, a new report highlights.
A common rheumatoid arthritis drug can suppress the progression of type 1 diabetes, research shows, making it the first disease-modifying, oral treatment of its kind for the condition.
A study evaluating the combination of a PD1 inhibitor with dupilumab enabled one out of six lung cancer patients to achieve a near-complete clinical response two months post-treatment, a paper states.
A novel Factor B inhibitor of the immune system's complement pathway has been approved for paroxysmal nocturnal haemoglobinuria (PNH).
The EU-UK Specialised Committee on Participation in Union Programmes has adopted the political agreement on UK’s association to the Horizon Europe programme.
The assets Roche will gain rights to through its planned acquisition of Carmot Therapeutics include several incretin treatments with best-in-class potential in obesity and diabetes.
Currently, imetelstat is being reviewed by the FDA and EMA for the treatment of transfusion-dependent anaemia in adults with lower risk MDS.
According to research, increased in R&D investment for developing novel excipients and greater emphasis on patient-centric formulations are key drivers for the pharmaceutical excipients market.
(Bio)pharmaceutical manufacturing is changing considerably, with key innovations enabling companies to rethink R&D, manufacturing, quality control and quality assurance (QC/QA) operations. The spotlight is squarely on process analytical technology (PAT), which underpins the successful transition towards highly effective advanced manufacturing strategies. Martin Gadsby, Chairman of Optimal Industrial Technologies, looks at how this transformative approach…