Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as ...

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Gene therapy companies have faced numerous setbacks over the last few years. What will it take for gene therapies to fulfill ...

The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR ...