High dose TSHA-102 was generally well tolerated with no SAEs or DLTs in two adolescent/adult patients and one <a target=_blank href= ...

High Investment and Government Initiatives to Boost Rare Diseases Treatment ModalitiesRockville, MD, Nov. 13, 2024 (GLOBE NEWSWIRE) -- According to Fact.MR, a market research and competitive ...

Findings suggest that PER3 gene variants prevent adrenal adaptation to winter daylight ... often linked to increases in ...

Let's go over the whole cast of Sonic The Hedgehog characters and discuss which are the best in the franchise.

the company delivered an oral presentation highlighting preclinical data for PBFT02 at the European Society of Gene & Cell Therapy (ESGCT) conference. The presentation reviewed the robust preclinical ...

KEBILIDI is authorized to be administered using the SmartFlow Neuro Cannula. KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...

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

ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related ... a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. The deployment in the US ...

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 ...