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Find Clinical Drug Pipeline Developments & Deals by Alcyone Therapeutics

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          WRIB
                          Not Confirmed
                          WRIB
                          Not Confirmed

                          Details : 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.

                          Product Name : Spinraza

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 27, 2023

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Biogen

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          WRIB
                          Not Confirmed
                          WRIB
                          Not Confirmed

                          Details : 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.

                          Product Name : Spinraza

                          Product Type : Oligonucleotide

                          Upfront Cash : $10.0 million

                          April 01, 2023

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Biogen

                          Deal Size : $51.0 million

                          Deal Type : Collaboration

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                          03

                          WRIB
                          Not Confirmed
                          WRIB
                          Not Confirmed

                          Details : 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.

                          Product Name : ACTX-401

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 05, 2022

                          Lead Product(s) : ACTX-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          WRIB
                          Not Confirmed
                          WRIB
                          Not Confirmed

                          Details : 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 skippi...

                          Product Name : ACTX-101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 06, 2021

                          Lead Product(s) : ACTX-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : RTW Investments

                          Deal Size : Undisclosed

                          Deal Type : Funding

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