Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
biopharma-reporter5d
Amylyx Pharmaceuticals gets FDA go ahead for phase 1 ALS trial
The Massachusetts based biotech will now press ahead with human trials for its antisense oligonucleotide (ASO) therapy, ...
PMLiVE9h
The silent treatment – researching gene silencing for Alzheimer’s, Parkinson’s and motor neurone disease
In trials at University College London Hospitals (UCLH), an ASO is being used to target the mutated gene that results in the ...
The Scientist4d
Making sense of antisense
They investigated the effects of antisense oligonucleotides targeting the regulatory RIα subunit of cAMP-dependent protein kinase (PKA). They treated human PC3 prostate cancer cells with antisense ...
Hosted on MSN19d
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 ...
Healio6d
FDA grants fast-track designation to myotonic dystrophy type 1 treatment
The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...
Nature2y
Advances in gene therapies for intractable diseases
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 ...
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 ...
Managed Healthcare Executive4d
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 ...
BioSpace6d
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 ...
The Scientist4d
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 ...