Sarepta discontinued SRP-5051 development due to safety concerns, despite promising Phase 2 results in dystrophin expression and exon skipping. Persistent hypomagnesemia and declining kidney function ...

Crofelemer delayed-release tablets are Jaguar's FDA-approved prescription drug under botanical guidance ("botanical ...

Since 1921, when the first therapeutic peptide, insulin, was synthesized, many peptide-related accomplishments have reverberated throughout the industry, with more than 80 peptide drugs being approved ...

The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, ...

Poolbeg Pharma PLC (AIM:POLB, OTC:POLBF, FRA:POLBF), the clinical-stage biopharmaceutical company, has received positive ...

Since the FDA first approved CAR-T cell therapy in 2017, eight CAR-T drugs have emerged, six of which have been fully approved by the FDA. This groundbreaking cell therapy has brought about a series ...

Precedence Research reports that the global cell and gene therapy market size was about $15.54 billion in 2022 and is projected to reach approximately $82.24 billion by 2032. To understand this growth ...

The deal gives Cellular Intelligence control of a clinical-stage therapy designed to restore the dopamine-producing nerve cells that are lost in Parkinson’s disease. The treatme ...

Praxis Precision Medicines has received Breakthrough Therapy Designation (BTD) from the FDA for relutrigine, a sodium channel modulator intended for pediatric treatment of SCN2A and SCN8A ...

Using patient-derived cardiac tissue and stem cell-based models, the team of translational researchers demonstrated that targeting the genetic cause of disease improved cellular abnormalities and ...