While PepGen works to persuade the FDA to let it launch a phase 2 trial of its Duchenne muscular dystrophy (DMD) therapy in ...

For more than a decade a gene therapy involving short strings of bases pairs, known as antisense oligonucleotides (ASOs), has been used to block the production of certain proteins by our body’s ...

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...

ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...

In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, ...

One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud ...

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 ...

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.

RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or ...

Biogen and Stoke partner on zorevunersen for Dravet syndrome, with a Phase 3 trial starting in 2025. Stoke gets $165 million ...