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            Details:

            Direct administration of AAV-based Gene Therapies to the central nervous system (CNS) can increase transduction and expression in the CNS. However, even with direct administration, vector "escapes" the CNS and distributes to peripheral organs.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 16, 2022

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            Lonza has worked with Affinia Therapeutics to achieve a four-fold improvement in yield for the company’s lead capsid, Anc80L65. Lonza and Affinia Therapeutics are partnering with the goal of ensuring flexible manufacturing capacity for the next several years.

            Lead Product(s): Anc80L65

            Therapeutic Area: Genetic Disease Product Name: Anc80L65

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Lonza Group

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement September 08, 2021

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            Proceeds to support continued development of proprietary platform for rationally designed adeno-associated virus vectors (AAV) and advance programs into the clinic.

            Lead Product(s): AAV-based gene therapies

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: EcoR1 Capital

            Deal Size: $110.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing May 04, 2021

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            Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.

            Lead Product(s): AAV capsid-gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 27, 2020

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            The proceeds will be used to advance the company’s platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

            Lead Product(s): Gene therapies

            Therapeutic Area: Neurology Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Atlas Venture

            Deal Size: $60.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing March 31, 2020

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