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 ...
Nature2 年
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 ...
biopharma-reporter3 天
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, ...
The Scientist2 天
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 ...
ALS News Today3 天
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 ...
GlobalData on MSN3 天
Biogen’s higher dose Spinraza under review by US and EU regulators
Biogen’s applications for the higher dose regimen of SMA drug Spinraza are now under review in the US and Europe.
Research explores genetic treatment for rare arrhythmias
More information: Raul H. Bortolin et al, Antisense Oligonucleotide Therapy for Calmodulinopathy, Circulation (2024). DOI: 10 ...
BioSpace4 天
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 ...