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.
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 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.
Under its proposed acquisition of ImmunoGen, AbbVie will gain rights to ELAHERE®, the first antibody-drug conjugate (ADC) approved in ovarian cancer.
New data from a Phase III gene therapy trial has demonstrated a 90 percent three-year overall survival rate for its participants with a high-risk bladder cancer.
Approval of the first treatment option for desmoid tumours beyond surgery and radiation has been granted by the US Food and Drug Administration (FDA).
Positive findings from a first-in-human trial means that an advanced cell therapy for progressive multiple sclerosis will be evaluated in Phase II.