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 ...

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 ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

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 ...

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 ...

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

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 ...

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.

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