• Organizations: Atsena Therapeutics
FDA agrees to Atsena's regulatory pathway for XLRS gene therapy
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FDA agrees to Atsena's regulatory pathway for XLRS gene therapy

Expansion of LIGHTHOUSE study will support potential BLA submission in early 2028 to become the first FDA-approved treatment for the inherited retinal disease.
FDA grants RMAT designation to Atsena's XLRS gene therapy
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FDA grants RMAT designation to Atsena's XLRS gene therapy

This marks ATSN-201’s fourth designation in the FDA’s expedited drug development program, propelling it one step closer to becoming the first approved treatment for this IRD.
Atsena receives FDA Fast Track designation for XLRS gene therapy
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Atsena receives FDA Fast Track designation for XLRS gene therapy

Subretinally-injected therapeutic is powered by dual AAV vectors with potential to become first approved treatment for this rare IRD.
Atsena and Nippon Shinyaku to collaborate on LCA1 gene therapy in US
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Atsena and Nippon Shinyaku to collaborate on LCA1 gene therapy in US

Nippon Shinyaku will receive exclusive U.S. commercial rights to ATSN-101, a subretinally-injected AAV5 capsid and human RHO promoter. 
FDA grants Orphan Drug designation for Atsena's XLRS gene therapy
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FDA grants Orphan Drug designation for Atsena's XLRS gene therapy

ATSN-201 is currently being studied in the phase 1/2 LIGHTHOUSE trial among patients diagnosed with XLRS caused by mutations in the R1 gene.
FDA grants Atsena Rare Pediatric Disease designation for XLRS gene therapy
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FDA grants Atsena Rare Pediatric Disease designation for XLRS gene therapy

New designation of subretinal-injection therapeutic comes amidst the ongoing patient enrollment and dosing of the phase 1/2 LIGHTHOUSE study.
FDA grants RMAT designation for Atsena's LAC1 gene therapy
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FDA grants RMAT designation for Atsena's LAC1 gene therapy

Positive 6-month data on ATSN-101 supports designation; 12-month results expected by end of 2023.
First patient dosed in Atsena's phase 1/2 trial for XLRS
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First patient dosed in Atsena's phase 1/2 trial for XLRS

LIGHTHOUSE study is assessing the subretinal injection of ATSN-201, a novel gene therapy.
FDA clears IND for Atsena's XLRS gene therapy candidate
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FDA clears IND for Atsena's XLRS gene therapy candidate

Acceptance clears the company to begin enrollment for a phase 1/2 trial on ATSN-201.
Positive 6-month data reported in phase 1/2 trial for LCA1
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Positive 6-month data reported in phase 1/2 trial for LCA1

Phase 1/2 trial is assessing the safety and efficacy of ATSN-101, an investigational gene therapy candidate.