Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Biogen
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Spinraza
Molecule Type : Large molecule
Upfront Cash : Not Applicable
June 27, 2023
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Biogen
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Biogen
Deal Size : $51.0 million
Deal Type : Collaboration
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.
Brand Name : Spinraza
Molecule Type : Large molecule
Upfront Cash : $10.0 million
January 04, 2023
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Biogen
Deal Size : $51.0 million
Deal Type : Collaboration
Lead Product(s) : AAV9-IGHMBP2 Gene Therapy
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : ACTX-401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 02, 2022
Lead Product(s) : AAV9-IGHMBP2 Gene Therapy
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : ACTX-101
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : RTW Investments
Deal Size : Undisclosed
Deal Type : Funding
Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders
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...
Brand Name : ACTX-101
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
June 09, 2021
Lead Product(s) : ACTX-101
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : RTW Investments
Deal Size : Undisclosed
Deal Type : Funding
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