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-reporter7d
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, ...
Hosted on MSN21d
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 ...
The Scientist6d
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 ...
Nature3y
Diabetes: Following the science in the search for a cure
This has opened the door to antisense oligonucleotide therapy aimed at restoring beta-cell function in diabetes and potentially ‘knocking out’ other genes in a way that could support long-term ...
Imvax secures $29m to support Phase IIb glioblastoma therapy trial
Imvax has secured $29m in a financing round from current investors, aimed at advancing the Phase IIb trial of its autologous ...
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 Executive6d
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 ...
BioSpace8d
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 Scientist6d
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 ...