Investigational IONIS-HTTRx Lowers Mutant Huntingtin Protein in Early-stage Patients, Phase 1/2 Data Show

Investigational IONIS-HTTRx Lowers Mutant Huntingtin Protein in Early-stage Patients, Phase 1/2 Data Show

Publication date: Jun 04, 2019

This post was originally published on this site IONIS-HTTRx, a potential therapy for Huntington’s disease, was able to reduce the levels of mutant huntingtin (mHTT) protein in early-stage patients, according to results from a Phase 1/2 clinical trial. IONIS-HTTRx, developed by Ionis Pharmaceuticals, is designed to target and destroy all forms of mutated huntingtin protein, which means it has the potential to treat all Huntington’s patients, regardless of their individual HTT mutation. Compared with the start of the study, the median decrease in mutant HTT protein levels among patients given IONIS-HTTRX was 20% in the 10 mg dose group; 25% in the 30 mg group; 28% in the 60 mg group; 42% in the 90 mg group; and 38% in the 120 mg group. The post Investigational IONIS-HTTRx Lowers Mutant Huntingtin Protein in Early-stage Patients, Phase 1/2 Data Show appeared first on Huntington’s Disease News. The post Investigational IONIS-HTTRx Lowers Mutant Huntingtin Protein in Early-stage Patients, Phase 1/2 Data Show appeared first on BioNewsFeeds.

Concepts Keywords
Antisense Pharmacodynamics
Antisense Oligonucleotide Pharmacokinetics
Brain Clinical trial
Cerebrospinal Fluid Antisense therapy
Clinical Trial Huntingtin
Cognitive Ionis Pharmaceuticals
England Spinal muscular atrophy
Gene Therapeutic gene modulation
Genetic Antisense RNA
Headache Life sciences
Huntingtin Health
Huntingtin Protein Huntington’s disease
Huntington Branches of biology
Intrathecal Headache
Intrathecal Administration Disease
Lumbar Puncture Pain
Messenger RNA
MRNA
Mutant
Mutation
Neurological
Pain
Pharmacodynamics
Pharmacokinetics
Placebo
Protein
Spinal Canal
Spinal Cord
Tolerability

Semantics

Type Source Name
gene UNIPROT CSF2
gene UNIPROT LAMC2
disease MESH disease progression
gene UNIPROT SLC35G1
gene UNIPROT HTT
gene UNIPROT SLC6A4

Similar

Original Article

Leave a Comment

Your email address will not be published. Required fields are marked *