BARCELONA, Spain I March 13, 2025 I SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations ...

The latest HPRA inspection in February 2025 was highly successful-both QC licences were renewed, and viral vector manufacturing was added to the MIA (IMP) licence. This means the Shannon site can ...

Genprex's oncology program utilizes its systemic, non-viral ... Orphan Drug Designation. Genprex's diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector ...

Dr. Forbes continued, “In forming this joint venture, MeiraGTx and Hologen have created the first neuro-AI clinical drug development ... were renewed, and viral vector manufacturing was added ...

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via ...

DB-OTO has received several designations from the U.S. Food and Drug Administration ... DB-OTO is a cell-selective, dual adeno-associated virus vector gene therapy that aims to restore hearing ...

About SRD-001 SRD-001 is an investigational gene therapy candidate that contains an adeno-associated virus serotype 1 ... with the Orphan Drug Designation. Additionally, Sardocor's pipeline ...

SENS-501: Gene therapy program to restore hearing in OTOF patients Sensorion’s SENS-501 dual vector AAV (adeno-associated ... SENS-401 has been granted Orphan Drug Designation in Europe for ...

SPK-9001 is a novel, investigation vector containing a bio-engineered ... regenerative medicines advance therapy (RMAT), as well as orphan drug designation by the FDA and PRIority Medicines ...

Apart from this, the U.S. Food and Drug Administration ... Based on vector type, the U.S. gene therapy market is divided into different types named retrovirus and gamma retrovirus, lentivirus, AAV, ...