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Ascidian Therapeutics
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80 GUEST STREET FOURTH FLOOR BOSTON MA 02135
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Ascidian will provide Roche exclusive, target-specific rights to Ascidian's RNA exon editing technology for undisclosed neurological targets and will conduct discovery and preclinical activities.


Lead Product(s): RNA-based Therapy

Therapeutic Area: Neurology Product Name: Undisclosed

Highest Development Status: Discovery PlatformProduct Type: Large molecule

Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

Deal Size: $1,842.0 million Upfront Cash: $42.0 million

Deal Type: Collaboration June 18, 2024

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ACDN-01 is the first-ever clinical-stage RNA exon editor, which is being evaluated in preclinical studies for the treatment of Stargardt disease & ABCA4 retinopathies.


Lead Product(s): ACDN-01

Therapeutic Area: Genetic Disease Product Name: ACDN-01

Highest Development Status: IND EnablingProduct 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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The funds will be used to advance Ascidian's lead program, an RNA exon editor to halt the progression of Stargardt disease or other ABCA4 retinopathies, and additional programs in the company's pipeline.


Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Apple Tree Partners

Deal Size: $40.0 million Upfront Cash: Undisclosed

Deal Type: Series A Financing November 08, 2023

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