Pipeline
FDA grants Orphan Drug Designation to SignaBlok's ROP therapy
TREM-1 inhibitor is designed to selectively target inflammation via a noninvasive delivery; company plans to report preclinical data at ARVO annual meeting.
Pipeline
FDA grants Orphan Drug designation to BlueRock's RP cell therapy
Marks second designation for OpCT-001; includes tax credits for qualified clinical testing and, if approved, 7 years of U.S. market exclusivity.
Pipeline
FDA grants Orphan Drug designation to AAVantgarde's Stargardt gene therapy
Regulatory milestone is second designation for AAVB-039; includes tax credits for qualified clinical testing and, if approved, seven years of U.S. market exclusivity.
Pipeline
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.
Pipeline
Alkeus receives two rare disease FDA designations for Stargardt candidate
New statuses join gildeuretinol’s (ALK-001) previous Fast Track and Orphan Drug designations; gives company potential eligibility for priority review voucher.
Pipeline
Aldeyra reports positive topline data from RP trial on ADX-2191
The preservative-free intravitreal formulation has already received orphan drug designation from the FDA.
Pipeline