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 ...
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, ...
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 ...
Retinal diseases: RNA therapeutics show promise but is India ready?
Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...
biopharma-reporter2d
Roche’s Elevidys demonstrates significant benefits for DMD patients in Phase 3
The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...
GlobalData on MSN8d
FDA lifts clinical hold on Amylyx’s Phase I ALS trial
This is the latest venture for Amylyx into the ALS field after its previous therapy Relyvrio was pulled from the market in ...
PMLiVE2d
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 ...
Ultragenyx's SWOT analysis: rare disease stock poised for growth
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), currently valued at $3.97 billion, has positioned itself as a key player in the ...
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 ...