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            SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.

            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Biogen

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 27, 2023

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            SPINRAZA (nusinersen) is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.

            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Biogen

            Deal Size: $51.0 million Upfront Cash: $10.0 million

            Deal Type: Collaboration January 04, 2023

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            Updated preclinical safety and efficacy data will be presented for ACTX-401 (AAV9-IGHMBP2 Gene Therapy), a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders.

            Lead Product(s): AAV9-IGHMBP2 Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: ACTX-401

            Highest Development Status: Phase I/ 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 May 02, 2022

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            Alcyone’s pipeline currently includes 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation.

            Lead Product(s): ACTX-101

            Therapeutic Area: Genetic Disease Product Name: ACTX-101

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

            Partner/Sponsor/Collaborator: RTW Investments

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Funding June 09, 2021

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