2023-04-14 08:29:09 ET
- Ocugen ( NASDAQ: OCGN ) shares gained 6% premarket on Friday after the biotechnology company reported positive prelim results from a Phase 1/2 Trial of its modifier gene therapy candidate, OCU400.
- OCU400 is a gene therapy being developed by Ocugen as a treatment for genetically diverse inherited retinal diseases (IRDs), including NR2E3 mutation.
- In the first two cohorts of the Phase 1/2 trial, seven participants with severe vision impairment due to retinitis pigmentosa associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 1010 vg/mL) or medium dose (3.33 x 1010 vg/mL) OCU400, respectively.
- Initial clinical data showed a favorable safety profile and visual improvements after treatment with OCU400 as measured by multi-luminance mobility testing and best corrected visual acuity assessment.
- The company believes these preliminary data supports potential of modifier gene therapy platform in gene-agnostic treatment of complex and heterogenous inherited genetic diseases.
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Ocugen stock gains on positive prelim date from gene therapy candidate