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Drishti Discoveries is a private UK biotechnology company developing RNA-targeting genetic therapies for neurodegenerative and rare inherited diseases. The company combines RNA interference and gene therapy through a proprietary artificial microRNA or miRNA-adapted shRNA platform designed to achieve sustained knockdown of disease-relevant target gene expression. Its lead disclosed program is an AAV-based gene therapy for amyotrophic lateral sclerosis, and public grant records also indicate work in retinitis pigmentosa and broader multi-target neurodegenerative gene therapy. Drishti appears to be an early-stage platform biotech focused on "one-and-done" in vivo genetic medicines for diseases with major unmet need.
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