Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated ...

Marcel Imwinkelried, Chief Executive Officer Siegfried: “Aligned with Siegfried's strategy EVOLVE+, the new lab facility marks a key milestone in DINAMIQS' journey to becoming the leading CDMO in the ...

NGN-401 uses an adeno-associated virus serotype 9 vector to deliver a full-length functional MECP2 gene using the firm's EXACT transgene regulation technology. NGN-401 is under evaluation in the FDA's ...

In a bid to streamline viral vector manufacturing, Thermo Fisher is charting another round of layoffs this year—and ...

Diamond-Blackfan anemia (DBA) is a potentially life-threatening condition that can cause severe anemia and other abnormalities. It's a rare genetic blood disorder that occurs when the bone marrow ...

New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through ... treating 30 different genetic mutations with a single vector ...

Neurogene has a $903M market cap with two phase 1/2 assets, NGN-401 for Rett Syndrome and NGN-101 for CLN5 Batten Disease.

Malaria remains a significant global health challenge, with approximately 249 million cases reported annually. Despite being ...

RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.