Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

The Hemophilia B treatment landscape is entering a new era, marked by disruptive innovation, curative potential, and renewed global attention. Once viewed as a lifelong burden requiring constant ...

The dynamics of the Krabbe disease market are expected to shift in the coming years due to the emergence of a new pipeline, increasing awareness of t ...

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on ...

The authors created a useful collection of Rep protein variants with changes that alter the amino acid sequence, but these did not lead to clear improvements in how the virus worked. Instead, their ...

Scientists at the McGovern Institute for Brain Research at MIT and the Broad Institute of MIT and Harvard have re-engineered ...

Parasitic worms that infect humans are not interbreeding with those that infect cattle as previously thought. This is good ...

OCU410ST has received a rare pediatric disease designation for ABCA4-associated retinopathies, potentially allowing Ocugen to ...

Novel Gene Therapy is Addressing Both Type 1 and Type 2 Diabetes AUSTIN, Texas, May 28, 2025 /PRNewswire/ -- Genprex, Inc. (Genprex or th ...

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...