Oligonucleotides,_antisense

Therapeutic Gfral silencing via antisense oligonucleotides ameliorates cancer-associated cachexia and extends survival in tumor-bearing mice.

Cancer cachexia is a life-threatening wasting syndrome that can be driven by tumor-derived GDF15 signaling through the brainstem-restricted receptor GFRAL. Despite its clinical importance, central genetic interventions targeting this axis remain limited. Here, we develop a potent antisense oligonucleotide (Gfral-ASO,...

🗓️ 2026-07-28
📰 Publication: Cell Reports Medicine
Read MoreTherapeutic Gfral silencing via antisense oligonucleotides ameliorates cancer-associated cachexia and extends survival in tumor-bearing mice.

Drug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

BACKGROUND

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of survival of motor neuron (SMN) protein inducing progressive muscle weakness and atrophy due to motor neurons degeneration. Despite benefits of SMN restoration therapies in patients, motor defects are...

🗓️ 2025-10-23
📰 Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreDrug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

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