Life Science
Gene Therapy for Rare Eye Diseases
In partnership with VC Gene Therapy and Hiroshima University, we are advancing the application of our purely domestic genome editing tool "ZF-ND1" to gene therapy for intractable diseases such as retinitis pigmentosa.
Breaking Away from the License-Fee Money Game
As license fees rise, so do the costs of therapeutic drugs — increasing the burden on patients and driving up medical costs overall. PtBio holds an exclusive license for ZF-ND1 (Zinc Finger ND1), allowing us to avoid high license fees, with no risk of having to pay multiple rights holders.
Application to Gene Therapy for Intractable Diseases
We are currently working with VC Gene Therapy and Hiroshima University to develop a gene therapy for retinitis pigmentosa — a disease for which no effective treatment has yet been established — and to bring it toward medical application.
PtBio has signed a license agreement for ZF-ND1 with VC Gene Therapy, a company founded by Dr. Masayo Takahashi, a leading figure in retinal regenerative medicine.
Mastering ZF-ND1 requires specialized genome editing skills, but our team has overcome the hurdles to demonstrating its efficacy and safety, and is steadily progressing toward practical application.
Related information
- Partners: VC Gene Therapy / Hiroshima University / PtBio
- Field: Life Science, treatment of intractable diseases
- VC Gene Therapy: https://www.vcgt.jp/