Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...
FierceBiotech2d
Septerna drops lead asset over bilirubin side effects 4 months after going public
Septerna stood out from the crowd of biotech IPOs last year due to the lack of clinical data to hand. Now, the data are ...
FierceBiotech2d
Solid Bio's stock soars as early DMD gene therapy data beat expectations
Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...
Patented transdermal drug candidate from Vascarta may offer a convenient & cost-effective way to treat sickle cell disease
An article in the Proceedings of the National Academy of Sciences (PNAS) Nexus journal* by Goel et al. describes studies in ...
The Pharma Letter1d
Septerna sinks as it discontinues lead asset SEP-786
US clinical-stage biotech Septerna yesterday announced its decision to discontinue the Phase I single- and multiple-ascending ...
Solid Biosciences reports initial clinical data from INSPIRE DUCHENNE trial
Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy ...
Sickle cell disease is a genetic disorder that causes lifelong suffering—here's what you need to know
Right now, approximately 20 billion red blood cells are busy traveling through your blood vessels. They are delivering oxygen ...
Solid Biosciences Stock Surges On Upbeat Duchenne Muscular Dystrophy Gene Therapy Data
Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.
Septerna discontinues SEP-786 Phase 1 clinical trial in hypoparathyroidism
Septerna (SEPN) announced its decision to discontinue the Phase 1 single- and multiple-ascending dose clinical trial of SEP-786 in healthy ...
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AstraZeneca Rises Almost 9% in a Month: How to Play the Stock
AstraZeneca AZN stock has risen 8.6% in the past month, mainly due to mixed fourth-quarter results and a positive update from ...
Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Q4 2024 Earnings Call Transcript
Q4 2024 Earnings Call Transcript February 13, 2025 Operator: Good morning, and welcome to Agios’ Fourth Quarter 2024 ...
Septerna shares plummet following clinical trial halt
The decision to halt the trial was made despite the lack of serious adverse events or indications of liver injury, cholestasis, or hemolysis in trial participants. Septerna's CEO, Jeffrey Finer, ...