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Alcyone Therapeutics
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Country
U.S.A
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Address
116 John St. Suite 300, Lowell, MA, 01852
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Telephone
(978) 709-1946

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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: ApprovedProduct 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: ApprovedProduct 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 IIProduct 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: PreclinicalProduct 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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