CSBio CSBio

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Find Drugs for Genetic Disease in Phase II Clinical Development in UNITED STATES

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            jCell (human retinal progenitor cells) is a first-in-class allogeneic cell therapy, which is being evaluated for the treatment of Retinitis Pigmentosa.

            Lead Product(s): jCell

            Therapeutic Area: Genetic Disease Product Name: jCell

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 21, 2024

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            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.

            Lead Product(s): Pitolisant Hydrochloride

            Therapeutic Area: Genetic Disease Product Name: Wakix

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2024

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            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.

            Lead Product(s): Nomlabofusp

            Therapeutic Area: Genetic Disease Product Name: CTI-1601

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Leerink Partners

            Deal Size: $150.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 14, 2024

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            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.

            Lead Product(s): Nomlabofusp

            Therapeutic Area: Genetic Disease Product Name: CTI-1601

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2024

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            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.

            Lead Product(s): Vesleteplirsen

            Therapeutic Area: Genetic Disease Product Name: SRP-5051

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 29, 2024

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            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.

            Lead Product(s): Sevasemten

            Therapeutic Area: Genetic Disease Product Name: EDG-5506

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: $240.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering January 19, 2024

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            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.

            Lead Product(s): Gildeuretinol Acetate

            Therapeutic Area: Genetic Disease Product Name: ALK-001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 10, 2024

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            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.

            Lead Product(s): NFX-179

            Therapeutic Area: Genetic Disease Product Name: NFX-179

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 13, 2023

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            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.

            Lead Product(s): AL01211

            Therapeutic Area: Genetic Disease Product Name: AL01211

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 31, 2023

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            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.

            Lead Product(s): Zagociguat

            Therapeutic Area: Genetic Disease Product Name: CY6463

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 25, 2023

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