This analysis constitutes the largest cohort of ND4-LHON patients studied after gene therapy treatment.
It confirms the good overall safety profile of the product in terms of systemic and ocular tolerability, humoral and cellular immune response and highlights a comparable safety profile for unilaterally and bilaterally treated patients.
The article, entitled "Safety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy," analyzes safety data collected from ND4-LHON patients who received single unilateral or bilateral intravitreal injections of lenadogene nolparvovec across 5 clinical studies (REVEAL, RESCUE, REVERSE, RESTORE and REFLECT) who were followed over five years after treatment.
Almost all patients received lenadogene nolparvovec at a dose of 9 x 1010 viral genomes per eye and 88% had at least 2 years of follow-up.
Systemic adverse events were mainly mild in intensity and were exceptionally related to lenadogene nolparvovec or the study procedure.
The absence of systemic issues related to lenadogene nolparvovec is mainly supported by the limited bio-dissemination of the product. Furthermore, the systemic humoral and cellular immune response was limited, acknowledging the local ocular nature of the immune response.
Also, ocular AE were mostly mild and were responsive to conventional ophthalmologic treatments.
This safety profile was consistent across patients, whether treated bilaterally or unilaterally, in terms of AE, bio-dissemination and immunogenicity. No significant differences in the frequency, nature or severity of systemic and ocular AE were found between these two groups of patients.
GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.
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