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Announces Positive Pre-Phase 3 FDA Type B Meeting and Outlines Plans to Start Pivotal Trial of jCell\u00ae for Retinitis Pigmentosa in the Second Half of 2024","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"jCyte"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Adage Capital Partners LP","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Avidity Biosciences Announces Oversubscribed $400 Million Private Placement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Avidity Biosciences"}]
Find Drugs for Genetic Disease in Phase II Clinical Development
jCell (human retinal progenitor cells) is a first-in-class allogeneic cell therapy, which is being evaluated for the treatment of Retinitis Pigmentosa.
Wakix (pitolisant) is an oral histamine-3 (H3) receptor antagonist/inverse agonist, small molecule drug. It is being evaluated for the treatment of Prader-Willi syndrome.
Larimar intends to use the net proceeds from the proposed offering to support the development of CTI-1601 (nomlabofusp), a recombinant fusion protein, for the treatment for Friedreich’s ataxia.
CTI-1601 (nomlabofusp) is a recombinant fusion protein intended to deliver human frataxin to the mitochondria of patients. It is being developed for the treatment of Friedreich’s Ataxia.
SRP-5051 (vesleteplirsen) is a next-generation peptide phosphorodiamidate morpholino oligome. It is being evaluated in phase 2 clinical trials for the treatment of patients with duchenne muscular dystrophy amenable to skipping exon 51.
Edgewise intends to use the net proceeds to support the potential U.S. commercial launch of EDG-5506 (sevasemten), an orally administered skeletal myosin inhibitor, in patients with Becker muscular dystrophy and completion of a Phase 3 trial with EDG-5506 in Duchenne.
ALK-001 (gildeuretinol acetate) is a novel molecule created as a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision. It is under phase 2 clinical development for the treatment of Stargardt Disease.
NFX‑179 topical gel is an investigational mitogen-activated protein kinase kinase (MEK) inhibitor, which is investigated for the Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1.
AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase (GCS) inhibitor which is under phase 2 clinical development to treat glycosphingolipid storage diseases, including Fabry Disease and Type 1 Gaucher Disease.
CY6463 (zagociguat), is a once-daily, oral, clinical-stage investigational medicine with the potential to positively impact both peripheral and central nervous system components of mitochondrial diseases.