New Gene Insertion Therapy for Stargard Disease in Development

US-based, biotechnology company, SalioGen Therapeutics are developing a treatment called SGT-101 for Stargardt disease caused by mutations in the ABCA4 gene.

Stargardt disease is an inherited retinal condition which primarily affects central vision and usually begins to impact the individual in childhood.

SGT-1001 is based on gene coding technology which uses a special enzyme to insert the new genetic code into the recipients DNA. This is different to traditional gene editing techniques and may have significant safety advantages. SalioGen’s system also uses nano particles to deliver the new genetic material. These nano particles have a much larger carrying capacity then the virus traditionally used in gene therapy and so can accommodate very large genes like ABCA4.   SGT-1001 is an injection delivered to the retina and the company have said they plan to launch a clinical trial in the first half of 2025. This will involve a small number of participants and will probably only be at US centres.