The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

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

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

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 ...

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

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 ...

Viralgen and Axovia are set to form a partnership to progress the development and manufacturing of gene therapy for retinal ...

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 ...