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 FDF
Sponsor : Biogen
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
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.
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
Lead Product(s) : ACTX-401
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : ACTX-101
Therapeutic Area : Rare Diseases and Disorders
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...
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