FDA approves innovative gene therapy for MLD
Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
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Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
The new acquisition is set to boost Lonza’s large-scale biologics manufacturing capacity for mammalian therapeutics.
Novartis’ new acquisition aims to advance first-in-class STING treatments for inflammation-driven diseases.
Research suggests that to prevent common mould growth, low-dose far-UVC light could be applied as a chemical-free addition to manual cleaning of indoor surfaces.
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.
This report is dedicated to exploring some of the latest advances in pharmaceutical environmental monitoring, covering topics from contamination control and cleanroom validation through to emerging technologies for bacterial endotoxin testing.
Delivering the traditional Parkinson’s treatment via subcutaneous infusion over a 24-hour period was shown to be safe and effective, research found.
Technology innovations and a stronger focus on developing novel drugs to treat chronic diseases, are key drivers for the global mycoplasma testing market up to the next decade, research states.
Combining near-infrared spectroscopy (NIR) hyperspectral imaging (HSI) and machine learning could offer a simpler, non-invasive alternative to established analytical techniques such as chromatography, research suggests.
A novel liver therapy authorised under US Food and Drug Administration (FDA) Accelerated Approval, is a potential game-changer in the NASH treatment space.
The new sterilisation method could provide an affordable, rapid approach for preparing microbiological culture media, research suggests.
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.
A new global collaboration and exclusive option-to-license agreement between Boehringer Ingelheim and Sosei Group Corporation includes a novel precision treatment for schizophrenia.
The first South Korean vaccine manufacturing facility to achieve EU-GMP certification from the European Medicines Agency (EMA) is being expanded to support global supply of a pneumococcal conjugate vaccine.
Research suggests the rapid detection method “will enable the timely implementation of microbiological monitoring strategies for large batches of medications”.