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 ...
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 ...
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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, ...
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 ...
Children's Mercy Kansas City Announces Breakthrough In Personalized Therapy For Rare Genetic Diseases
The methods and protocols generated in this study are accessible and can be implemented in any standard research laboratory without the need for ...
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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 ...
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FDA lifts clinical hold on Amylyx’s Phase I ALS trial
Amylyx will be allowed to run a Phase I trial after the FDA lifted a clinical hold of its investigational ALS drug.
Dyne Therapeutics, Inc.: Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 - WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular diseas ...
Is it a Good Idea to Invest in Stoke Therapeutics Stock Now?
Massachusett-based Stoke Therapeutics STOK is making rapid progress with the development of zorevunersen (STK-001), its investigational candidate for treating Dravet syndrome, a severe and progressive ...
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Advancing Gene Therapy: Enzyme Selection for Effective RNA Oligonucleotide Mapping
New gene therapy modalities, such as CRISPR guide RNA (single guide ribonucleic acid [sgRNA]) and messenger RNA (mRNA), continue to make progress in both primate and first-in-human trials. As this ...