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Gene Therapy Could Restore Vision for RP Patients

An experimental gene therapy could offer new hope for patients living with retinitis pigmentosa, a group of inherited retinal diseases that can lead to progressive vision loss. Sulagna Bhattacharya, co founder and CEO of Nano Scope Therapeutics, shares how her own family’s experience with RP inspired her mission to develop new treatment options.

Bhattacharya explains why retinitis pigmentosa has been particularly challenging to treat. Because the disease can be caused by hundreds of mutations across dozens of genes, existing gene targeted approaches can only help a small percentage of patients. Nano Scope Therapeutics is pursuing a broader approach designed to restore visual function regardless of the specific gene mutation.

The company’s technology uses an engineered optogenetic molecule called opsin to make retinal bipolar cells responsive to light. Rather than repairing the damaged photoreceptors, the approach aims to turn surviving retinal cells into what Bhattacharya describes as de facto photoreceptors, potentially helping patients regain some visual abilities.

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