PEP-Therapy receives FDA Orphan Drug Designation for PEP-010 for treatment of Pancreatic Cancer
March 17, 2025 09:00 ET
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PEP-Therapy
Press release PEP-Therapy receives FDA Orphan Drug Designation for PEP-010for treatment of Pancreatic Cancer Paris (France), March 17, 2025 – PEP-Therapy, a clinical-stage biotechnology company...
Nurix Announces U.S. FDA Orphan Drug Designation Granted to Bexobrutideg (NX-5948) for the Treatment of Waldenström Macroglobulinemia
March 17, 2025 07:00 ET
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Nurix Therapeutics, Inc.
First-in-class BTK degrader, NX-5948, Granted ODD for WM and assigned nonproprietary name “bexobrutideg” in newly named degrader class of drugs
Plus Therapeutics Granted U.S. FDA Orphan Drug Designation for Rhenium (186Re) Obisbemeda for the Treatment of Leptomeningeal Metastases in Patients with Lung Cancer
March 06, 2025 07:30 ET
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Plus Therapeutics Inc.
Plus Therapeutics Granted U.S. FDA Orphan Drug Designation for Rhenium (186Re) Obisbemeda for the Treatment of Leptomeningeal Metastases in Patients with L
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
January 06, 2025 07:00 ET
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NMD Pharma
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd orphan drug designation granted to the skeletal...
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
January 06, 2025 01:00 ET
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NMD Pharma
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd orphan drug designation granted to the skeletal...
AlveoGene’s Novel, Inhaled Gene Therapy AVG-002 Receives Orphan Drug Designation from FDA for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency
December 03, 2024 04:00 ET
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Alveogene
Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with few, if any, options for long-term survivalHighly encouraging preclinical data...
AAVantgarde Bio Announces FDA Orphan Drug Designation for AAVB-081 for the Treatment of Usher Syndrome Type 1B Retinitis Pigmentosa
December 02, 2024 02:00 ET
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AAVantgarde Bio
AAVantgarde Bio Announces FDA Orphan Drug Designation for AAVB-081 for the Treatment of Usher Syndrome Type 1B Retinitis Pigmentosa
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-108 (HSV-1 oncolytic virus) to Treat Malignant Glioma
November 07, 2024 08:30 ET
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Mustang Bio, Inc.
MB-108 (HSV-1 oncolytic virus) is active and well tolerated in patients with recurrent glioblastoma in ongoing Phase 1 clinical trial Preclinical data support a novel combination of MB-108 (HSV-1...
uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
September 23, 2024 07:05 ET
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uniQure Inc.
LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...
Unicycive Therapeutics Announces Orphan Drug Designation Granted for UNI-494 by the U.S. Food and Drug Administration for the Prevention of Delayed Graft Function in Kidney Transplant Patients
March 04, 2024 07:03 ET
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Unicycive Therapeutics, Inc.
LOS ALTOS, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the...