Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Botaretigene Sparoparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Johnson & Johnson Innovative Medicine
Deal Size : $415.0 million
Deal Type : Agreement
Details : Under the agreement, Janssen gains the remaining interests in investigational gene therapy AAV5-RPGR (botaretigene sparoparvovec) for the treatment of X-linked retinitis pigmentosa.
Brand Name : AAV5-RPGR
Molecule Type : Cell and Gene therapy
Upfront Cash : $350.0 million
December 21, 2023
Lead Product(s) : Botaretigene Sparoparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Johnson & Johnson Innovative Medicine
Deal Size : $415.0 million
Deal Type : Agreement
Lead Product(s) : AAV-AQP1
Therapeutic Area : Otolaryngology (Ear, Nose, Throat)
Study Phase : Phase I/ Phase II
Sponsor : Sanofi
Deal Size : $30.0 million
Deal Type : Financing
Details : Sanofi will receive a right of first negotiation for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Immunology and Inflammation and CNS targets, including AAV-AQP1, a gene therapy for xerostomia caused by Sjogren's syndrome.
Brand Name : AAV-AQP1
Molecule Type : Large molecule
Upfront Cash : Undisclosed
October 30, 2023
Lead Product(s) : AAV-AQP1
Therapeutic Area : Otolaryngology (Ear, Nose, Throat)
Highest Development Status : Phase I/ Phase II
Sponsor : Sanofi
Deal Size : $30.0 million
Deal Type : Financing
Lead Product(s) : Botaretigene Sparoparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile and efficacy assessments in this proof-of-concept study demonstrated improvements in retinal sensitivity, visual function and functional vision.
Brand Name : AAV-RPGR
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 01, 2022
Lead Product(s) : Botaretigene Sparoparvovec
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) : AAV2/5-RPGR
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Recipient : Johnson & Johnson Innovative Medicine
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : One-year findings support advancement into Phase 3 and include significant functional vision improvements observed in the time taken to walk through a vision-guided mobility maze at nine months.
Brand Name : AAV-RPGR
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 13, 2020
Lead Product(s) : AAV2/5-RPGR
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Recipient : Johnson & Johnson Innovative Medicine
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : AAV2/5-RPGR
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Ongoing Phase 1/2 MGT009 clinical trial demonstrated AAV-RPGR was generally well tolerated and produced significant improvement in vision, including improvement in retinal sensitivity and vision-guided mobility, in the dose escalation phase of the trial.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 28, 2020
Lead Product(s) : AAV2/5-RPGR
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
LOOKING FOR A SUPPLIER?