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AHB-137, an antisense oligonucleotide targeting HBV mRNA, demonstrated safety, favorable pharmacokinetics, and rapid HBsAg reduction in healthy volunteers and chronic hepatitis B patients, supporting further clinical development.
This study unveils tofersen’s distribution and potent SOD1 reduction within the CNS of SOD1-ALS patients, utilizing autopsy tissues from clinical trial participants, confirming anticipated pharmacokinetic and pharmacodynamic effects.
The integrated analysis of the VALOR trial and its open-label extension reveals that early initiation of tofersen significantly slows functional decline, preserves respiratory capacity, and improves survival in patients with SOD1-ALS over a
GSK has announced positive Phase III results for Bepirovirsen, an antisense oligonucleotide that achieves functional cure in chronic hepatitis B patients. By targeting viral RNA and HBsAg, the drug offers a finite treatment path, potentiall
AZD2693, a liver-directed antisense oligonucleotide, safely reduces PNPLA3 expression and liver fat in homozygous PNPLA3 148M carriers with metabolic dysfunction-associated steatohepatitis, signaling promise for precision treatment in this
The phase 2 ION224-CS2 trial demonstrates that ION224, a DGAT2 antisense oligonucleotide, safely improves liver histology in MASH patients independent of weight loss, offering a potential new therapeutic approach.
