AlveoGene’s Novel, Inhaled Gene Therapy AVG-002 Receives Orphan Drug Designation from FDA for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency
03. Dezember 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
02. Dezember 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
07. November 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
23. September 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
04. März 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...
OrphAI Therapeutics receives Orphan Drug Designation for AIT-101 as a treatment for amyotrophic lateral sclerosis in the European Union
15. Februar 2024 10:38 ET
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OrphAI Therapeutics Inc.
GUILFORD, Conn., Feb. 15, 2024 (GLOBE NEWSWIRE) -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today...
Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment
12. Februar 2024 08:30 ET
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NovelMed Therapeutics Inc
FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.Ruxoprubart is an Anti-Bb antibody that selectively blocks the alternative pathway while maintaining the intactness...
La FDA Américaine Accorde la Qualification de Médicament Orphelin au PTX-252 pour le Traitement de la Leucémie Myéloïde Aiguë (LMA)
16. Januar 2024 13:30 ET
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Hyloris Pharmaceuticals SA
La FDA Américaine Accorde la Qualification de Médicament Orphelin au PTX-252 pour le Traitement de la Leucémie Myéloïde Aiguë (LMA) Le PTX-252 (précédemment appelé agent plécoïdeTM) est une nouvelle...
Orphan Drug Designation Granted to PTX-252 by U.S. FDA for the Treatment of Acute Myeloid Leukaemia (AML)
16. Januar 2024 13:30 ET
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Hyloris Pharmaceuticals SA
Orphan Drug Designation Granted to PTX-252 by U.S. FDA for the Treatment of Acute Myeloid Leukaemia (AML) PTX-252 (previously referenced as a Plecoid™Agent) is a novel molecular entity developed in...
Pleco Therapeutics receives FDA Orphan Drug Designation
16. Januar 2024 11:30 ET
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Pleco Therapeutics B.V.
The U.S Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics BV’s lead compound, PTX-252 for the treatment of AML.