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

With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...

The deal, which features up to $385 million in milestones, gives Biogen exclusive rights to commercialize Stoke’s ...

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 ...

Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.

Nurix Therapeutics, Inc. stock slid 40% after promising NX-5948 results in CLL. NX-5948 shows efficacy in heavily pre-treated ...

Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...

No doubt 2025 will usher in many more advances in CRISPR therapies, thanks to the FDA’s new Platform Technology Designation ...

Atherosclerotic cardiovascular disease is a major health concern worldwide and requires effective preventive measures. Lp(a) (lipoprotein [a]) has recently garnered attention as an independent risk ...

Rodriguez has filed a new measure that would allow patients suffering from severe illnesses to have individualized treatment options.