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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
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.
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
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.
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
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 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