Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...
BioWorld4d
Targeting recurrent ABCA4 variant with antisense oligonucleotides as new strategy for Stargardt disease
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...
Hosted on MSN29d
Sanofi taps Alloy Therapeutics for CNS antisense drug development
Sanofi has entered into a collaboration and license agreement with Alloy Therapeutics to develop an antisense oligonucleotide (ASO) therapy targeting a single, undisclosed central nervous system ...
chromatographyonline3d
Understanding the On-off Retention Mechanism of Oligonucleotides
A team from the University of Geneva and Bayer AG present their findings on the on-off retention of oligonucleotides and the ...
The Scientist13d
Making Sense of Antisense
A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as ...
ALS News Today24y
Clinical hold lifted from Phase 1 trial of ALS therapy AMX0114
With the hold lifted, Amylyx can begin screening and enrolling for its Phase 1 LUMINA trial, which will test AMX0114 in ALS ...
GlobalData on MSN7d
Imvax secures $29m to support Phase IIb glioblastoma therapy trial
Imvax has received $29m in a financing round from current investors, aimed at advancing the Phase IIb trial of its autologous ...
FierceBiotech29d
Sanofi inks $400M biobucks deal to test the mettle of Alloy's antisense platform
The earlier deal gave Sanofi a stake in Resalis Therapeutics, an Italian biotech that is advancing an antisense oligonucleotide that targets a noncoding RNA involved in obesity pathways.
Managed Healthcare Executive14d
FDA and EMA Accepts Applications for New Nusinersen Regimen
An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...
Nature1y
Spatial biology gives a clear view on gene therapy
Gene therapy is becoming a reality for the ... researchers developing oligonucleotide therapies using small RNA, antisense oligonucleotides (ASOs) or RNA interference (RNAi).
BioSpace15d
FDA Presses Pause on Atara’s Programs, Play on Amylyx’s ALS Assets
The FDA is putting Atara’s active Investigational New Drug applications on hold due to manufacturing concerns at a third-party provider while releasing Amylyx’s investigational ALS therapy from a ...