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- Indexed articles, last 90 days
- 20
- Latest publication
- Sep 25, 2026
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- Jul 9, 2026
Latest articles
Manufacturing aqueous siRNAs to improve patient adherence (opens the original)
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Improvements to aqueous siRNA manufacturing could lead to patients dosing themselves at home. Small interfering RNAs (siRNAs) are showing promise in treating a range of neurological, muscular, and cardiac conditions. Even a major blockbuster film has picked up on their remarkable potential. However, a significant manufacturing bottleneck limits this fast-growing modality. To make patient-friendly formulations that can be self-administered at home, siRNAs need to move from lyophilized powders req
FDA approves gene therapy for ultra-rare metabolic disease (opens the original)
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The approval is a landmark moment for Sanfilippo patients who face progressive neurological symptoms and premature death. The US Food and Drug Administration (FDA) announced its landmark approval of the gene therapy rebisufligene etisparvovec (Fayuvi, formerly known as UX111), an orphan drug for a rare inherited pediatric disease called Sanfilippo syndrome. The drug is produced by the California-based rare disease pharma company Ultragenyx. Sanfilippo syndrome, also known as mucopolysaccharide (
Muscular dystrophy AOC drug from Novartis fails clinical trial (opens the original)
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The company recently disclosed that its antibody-oligo conjugate drug failed to meet trial endpoints. Earlier this week, Novartis announced that its antibody-oligonucleotide conjugate (AOC) drug delpacibart etedesiran (del-desiran) failed to meet primary endpoints in a Phase III clinical trial. The therapy was designed for the treatment of myotonic dystrophy type 1 (DM1), an inherited neuromuscular disease. The drug works by targeting transferrin receptors in muscle tissue and then using small i
Over 15 biotech companies accused of manipulating antibody images (opens the original)
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A recent scientific report found almost 19,000 doctored images of antibodies available in the commercial market. A landmark investigation published by Northwestern University’s Reese Richardson found widespread image manipulation in the commercial antibody market. In the online article, more than 15 well-known biotechnology companies were implicated in using doctored images in their antibody catalogs. Richardson and his colleagues found almost 19,000 images online of antibody assays that had bee
Novartis halts CAR T therapy trials after patient deaths (opens the original)
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Three patients have died due to a severe autoimmune reaction following dosing with zola-cel for the treatment of autoimmune conditions. Large pharmaceutical firm Novartis confirmed that it is halting and investigating several clinical trial programs involving its chimeric antigen receptor (CAR) T-cell therapy rapcabtagene autoleucel (rap-cel). The decision follows the deaths of three patients, all of whom died following systemic inflammatory reactions. Novartis had been testing the drug to treat
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