A study published in Nature Communications revealed a new antisense oligonucleotide therapy applicable to the W1282X mutation of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis.
Following another failure in amyotrophic lateral sclerosis (ALS), Biogen Inc. will discontinue its development of antisense oligonucleotide BIIB-078 with partner Ionis Pharmaceuticals Inc. The stumble is part of a mega-collaboration the two companies began 10 years ago that has also yielded a lot of success, including the blockbuster Spinraza (nusinersen).
After struggling in the past year, Ionis Pharmaceuticals Inc. will collaborate with Astrazeneca plc to develop eplontersen for treating transthyretin amyloidosis, which is systemic, progressive and fatal. At stake for Ionis is $2.9 billion in potential sales-related milestone payments.
A Japanese study has discovered a new means of regulating endogenous gene expression in the CNS, using systemically administered antisense oligonucleotides (ASOs) in rodents, which facilitates development of ASO-based therapies for patients with neurological diseases requiring prolonged treatment.
The neurodevelopmental disorder MECP2 duplication syndrome (MDS) is caused by duplications spanning the methyl-CpG binding protein 2 gene (MECP2) locus, and researchers have shown that the MDS-like phenotype can be reversed in adult symptomatic mice using MECP2-specific antisense oligonucleotides (MECP2-ASOs).
Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) fell 21.7% to $43.59 on March 23 after its longtime partner, Roche Holding AG, decided to stop dosing the antisense oligonucleotide tominersen in a global phase III manifest Huntington's disease (HD) study. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned assessment of the drug's risk-benefit profile.
LONDON – Evox Therapeutics Ltd. sealed a $1.2 billion collaboration with Eli Lilly and Co. to apply its exosome technology to the doubly difficult task of systemically delivering RNA interference and antisense oligonucleotide drugs, to reach central nervous system targets.
Lessons learned from development of hepatitis C therapies could be applied to the underserved hepatitis B population, potentially leading to a functional cure through a combination of optimized drugs that attack the virus from different angles. That is the mission of 2-year-old Aligos Therapeutics Inc., a South San Francisco-based startup focused on liver-related diseases.
DUBLIN – The witty Twitter account @justsaysinmice, run by Northeastern University research scientist Jim Heathers, offers a very useful corrective to the misleading and unwarranted hype that often accompanies preclinical studies in mice. What looks good in murine models is all too often lost in translation, for a whole host of reasons, and never has any useful effect in patients. That’s not a concern for a group led by Thomas Thum, of the Institute of Molecular and Translational Therapeutic Strategies at Hannover Medical School in Germany, who just published in Nature Communications the outcome of what is probably the largest ever pig study in heart failure.
Philadelphia-based Aro Biotherapeutics Co. CEO Sue Dillon told BioWorld her firm has “incoming interest from other companies to apply Centyrins to other kinds of drug conjugates,” which could mean more deals with the platform like the one sealed with Ionis Pharmaceuticals Inc.
