Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and ...

Studies conducted in human induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs) compared various cassette configurations and informed the final design of TN-201, which incorporates a ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

Chikungunya cases have spiked substantially in La Réunion since early 2025, with 8,600 cases recorded and almost 3,000 new cases during the week of March 3 to 9, 2025 1. Similar to the 2005-2006 ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

On top of helping you quickly find the folders or files you're looking for, the PowerToys Run feature can also perform several other functions, from converting units to executing system commands. And ...

Q4 2024 Earnings Call Transcript March 5, 2025 Ocugen, Inc. reports earnings inline with expectations. Reported EPS is $-0.05 EPS, expectations were $-0.05. Operator: Good morning, and welcome to ...

Alternative splicing (AS) of pre-mRNA plays a crucial role in tissue-specific gene regulation, with disease implications due to splicing defects. Predicting and manipulating AS can therefore uncover ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

We look forward to building upon the encouraging data generated to date and engaging with health authorities to explore the registrational pathway for this promising, one-time therapy to address a ...

Adverum's inclusion in ARTEMIS of both treatment-naive and treatment-experienced patients is intended to address a broad patient population ... Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, ...

“In scripted we are looking at whether there are investments we want to make in individuals or whether there are labels that have all the resources they need,” said Bennett. “How can we help the ...