In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

Achieved Record Revenue for 2024 and Growth of 31%;Reduced 2024 Operational Cash Burn by 35% SOLANA BEACH, CALIFORNIA / ACCESS Newswire / February 26, 2025 / ClearPoint Neuro, Inc. (Nasdaq:CLPT) (the ...

CRISPR is like an editor that can rewrite DNA letter by letter or gene by gene, to remove harmful mutations or add protective ...

PRGN-2012—which Precigen has begun to call by its generic name of zopapogene imadenovec—is a gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus ...

Ladies and gentlemen, good morning and welcome to the Taysha Gene Therapies Full Year 2024 Conference Call. At this time all participants are in listen-only mode. A brief question-and-answer session ...

A single injection of a healthy gene allowed blind children to recognize objects, shapes, and even read — offering hope for ...

Retinal gene therapy advancements may rewrite the book on treatments for age-related macular degeneration and diabetic eye diseases.

As the world recognizes Rare Disease Day on Feb. 28, Rockville-based biotechnology company, REGENXBIO is working to change ...

As FDA seeks to rehire some fired employees, Donald Trump threatens to enact tariffs on pharma companies unless they reshore manufacturing; another lawsuit hits the complex GLP-1 compounding space as ...

Early results from the company’s single-arm trial saw the therapy able to partially repair the chemical responsible for ...

New research reveals that voluntary exercise restores STAT3 levels in the amygdala, helping to prevent stress-induced ...

Qure's stock rises on Huntington's therapy progress and strong gene therapy pipeline. Learn about QURE stock's cost-cutting ...