Atsena Therapeutics’ ATSN-201 Gains the US FDA’s Rare Pediatric Disease Designation for Treating Retinoschisis
Shots:
- The US FDA has granted RPDD to ATSN-201 for treating X-linked retinoschisis (XLRS). The company will receive a priority voucher on approval that could be sold or used for developing other internal product
- ATSN-201 is currently being assessed under the dose escalation & expansion P-I/II (LIGHTHOUSE) study for treating XLRS caused due to RS1 gene mutations in male patients (≥6yrs.). Recruitment is underway
- ATSN-201, a gene therapy, uses AAV.SPR (novel spreading capsid) for therapeutic gene expression in central retinal photoreceptors without the risks of foveal detachment. Preclinical studies in non-human primates showed that AAV.SPR induces transgene expression beyond subretinal injection areas
Ref: Globenewswire | Image: Atsena Therapeutics
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
