Ultimately, Stoke Therapeutics is that classic “intriguing biotech” --hurdling to one of those proverbial “moments of truth” when the success of that lead asset provides the validation for not only ...

Five families with children who have the newly discovered rare condition recently gathered in a Washington, D.C., park.

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

The mRNA metabolism passively shapes the levels of an mRNA modification called m6A within a steady-state cell and upon stress.

BARCELONA, Spain, June 11, 2025 /PRNewswire/ -- SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today ...

Citrate is essential for the metabolism and development of neurons. A membrane transport protein called SLC13A5 plays a central role in this process and has previously been linked to a particularly ...

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

Genetic vectors are vehicles for delivering foreign DNA into recipient cells. Vectors can replicate autonomously and typically include features to facilitate the manipulation of DNA as well as a ...

SB-007 is an investigational Protein Splicing dual AAV gene therapy in development for the treatment of Stargardt disease. It is designed to restore expression of the native full-length ABCA4 ...

BARCELONA, Spain, June 11, 2025 /PRNewswire/ -- SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today ...