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Ultragenyx to Sell Rare Pediatric Disease Voucher for $210M

Shots: Ultragenyx has entered into a definitive agreement to sell a Rare Pediatric Disease PRV for $210M, provide Ultragenyx with significant non-dilutive capital to advance therapies for rare & ultra-rare diseases Rare Pediatric Disease PRV was received following FDA approval of Genglycos (pariglasgene brecaparvovec-opnr), the first treatment targeting the underlying cause of glycogen storage disease…

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Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Shots: Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa) The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…

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Regenxbio

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

Shots: The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025 As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…

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Abeona Therapeutics

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Shots: The US FDA has approved Zevaskyn (prademagene zamikeracel) for treating wounds in adult & pediatric RDEB pts based on the intra-pts controlled P-III (VIITAL) trial & has also granted RPD PRV to Abeona, which the company intends to monetize; Zevaskyn to be commercially available in Q3’25 The P-III Trial assessed Zevaskyn in RDEB pts,…

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