Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...

Imvax has received $29m in a financing round from current investors, aimed at advancing the Phase IIb trial of its autologous ...

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 ...

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 ...

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 ...

This has opened the door to antisense oligonucleotide therapy aimed at restoring beta-cell function in diabetes and potentially ‘knocking out’ other genes in a way that could support long-term ...

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.

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 ...

Gene therapy is becoming a reality for the ... researchers developing oligonucleotide therapies using small RNA, antisense oligonucleotides (ASOs) or RNA interference (RNAi).

With the hold lifted, Amylyx can begin screening and enrolling for its Phase 1 LUMINA trial, which will test AMX0114 in ALS ...