International collaborations, refined gene-editing protocols, and targeted immunologic maneuvers keep the cancer gene therapy ...

Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

Based on this, we have developed a new approach to enhance the efficiency of rAAV-mediated gene expression. This approach uses co-infection with a transgene-containing vector and a second vector ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

Viralgen and the CTNNB1 Foundation have come together to manufacture an investigational gene therapy designed to treat CTNNB1 ...

Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...