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[{"orgOrder":0,"company":"MeiraGTx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MeiraGTx to Present Data from Ongoing Trial of AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa at AAO 2020 Virtual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Janssen's RPGR Gene Therapy Shows Statistically Significant and Continued Vision Improvement in Patients with X-Linked Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Late-Breaking Phase 1\/2 Data Demonstrates Safety Profile of Investigational Gene Therapy Botaretigene Sparoparvovec (AAV-RPGR) and Sustained Vision Improvement in Patients with X-Linked Retinitis Pigmentosa (XLRP)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"MeiraGTx Announces $30 Million Strategic Investment from Sanofi; Company Pursuing Multiple Additional Strategic Opportunities","therapeuticArea":"Otolaryngology (Ear, Nose, Throat)","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Otolaryngology (Ear, Nose, Throat)","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","amount":"$415.0 million","upfrontCash":"$350.0 million","newsHeadline":"MeiraGTx Announces Asset Purchase Agreement Involving its AAV-RPGR Collaboration for up to $415 million","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]
Find Clinical Drug Pipeline Developments & Deals by MeiraGTx
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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.
Lead Product(s):
Botaretigene Sparoparvovec
Therapeutic Area: Genetic Disease
Product Name: AAV5-RPGR
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Johnson & Johnson Innovative Medicine
Deal Size: $415.0 million
Upfront Cash: $350.0 million
Deal Type: Agreement
December 21, 2023
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.
Lead Product(s):
AAV-AQP1
Therapeutic Area: Otolaryngology (Ear, Nose, Throat)
Product Name: AAV-AQP1
Highest Development Status: Phase I/ Phase II
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Sanofi
Deal Size: $30.0 million
Upfront Cash: Undisclosed
Deal Type: Financing
October 30, 2023
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.
Lead Product(s):
Botaretigene Sparoparvovec
Therapeutic Area: Genetic Disease
Product Name: AAV-RPGR
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 01, 2022
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.
Lead Product(s):
AAV2/5-RPGR
Therapeutic Area: Genetic Disease
Product Name: AAV-RPGR
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Recipient:
Johnson & Johnson Innovative Medicine
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
November 13, 2020
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.
Lead Product(s):
AAV2/5-RPGR
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 28, 2020