Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
manilatimes1d
MeiraGTx Announces Third Quarter 2024 Financial and Operational Results and Recent Business Updates
MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the third quarter ended September 30, ...
BioSpace2d
Neurogene Shares Tank After Adverse Event Linked to Early-Stage Rett Syndrome Gene Therapy
Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated ...
Business Insider6d
Rocket Pharmaceuticals: Strong Buy Rating Backed by Promising AAV and LV Franchise Developments and Solid Financial Position
Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The firm’s clinical program is a LVV-based gene therapy for ...