Cell and Gene Therapy Catapult preclinical database

Results: 39
Marcelo Rivolta, University of Sheffield
Otic neuroprogenitors for deafness
hESC derived otic neuroprogenitors for deafness, examination of function in animal models.
Funding sources:
Not specified
Development stage:
-
Expected completion date
-
Cell type:
Not specified
Cell source:
Not specified
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Not specified
Disease area:
Not specified
Clinical indication:
Not specified
Steve Bloor, Videregen Limited
Tissue engineered autologous stem cell seeded trachea replacement
Development of a tissue engineered trachea replacement using a decellularised human trachea, seeded with autologous bone marrow derived MSCs and airway epithelial cells. For the treatment of severe structural airway diseases.
Funding sources:
Innovate UK, Private investment/VC, EU
Development stage:
Late preclinical
Expected completion date
Q1 2016
Cell type:
Not specified
Cell source:
Bone marrow and airway
Autologous/allogeneic:
Autologous
Gene modification/gene therapy:
No
Disease area:
Respiratory medicine
Clinical indication:
Structural airway diseases
Steve Bloor, Videregen Limited
Tissue engineered autologous cell seeded mucosal lining replacement
Development of a tissue engineered epithelial/ airway mucosal replacement using a decellularised mucosal scaffold, seeded with autologous derived epithelial cells. For the treatment of airway mucosal lining following mucosal lining injury e.g. trauma, cancer.
Funding sources:
Private investment/VC, Innovate UK
Development stage:
Late preclinical
Expected completion date
Q1 2017
Cell type:
Mesenchymal stem/stromal cells
Cell source:
Autologous airway mucosal
Autologous/allogeneic:
Autologous
Gene modification/gene therapy:
No
Disease area:
Respiratory medicine
Clinical indication:
Airway disease, injury
Anna David, University College London
EVERREST
Does vascular endothelial growth factor gene therapy safely improve outcome in severe early-onset fetal growth restriction? Adenovirus VEGF gene therapy will be given to women with a diagnosis of severe early onset fetal growth restriction (22-26+6 weeks of gestation, estimated fetal weight <3rd centile and not growing).
Funding sources:
Magnus growth capital investment, EU FP7
Development stage:
Late preclinical
Expected completion date
Q4 2018
Cell type:
Fetal liver mesenchymal stem cells
Cell source:
Not specified
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
N/A
Disease area:
Obstetrics
Clinical indication:
Fetal growth restriction
Steve Lee, University of Birmingham
Engineering human T cells to target the tumour vasculature through expression of a chimeric antigen receptor
Genetic modification of T cells to target the tumour vasculature. Engineering human T cells to target the tumour vasculature through expression of a chimeric antigen receptor.
Funding sources:
Not specified
Development stage:
Mid preclinical
Expected completion date
Q3 2016
Cell type:
T cells
Cell source:
Peripheral blood
Autologous/allogeneic:
Autologous
Gene modification/gene therapy:
Yes. CAR gene transfer.
Disease area:
Oncology
Clinical indication:
Multiple common solid tumours
Majlinda Lako, Newcastle University
Stem cells for biological assays of novel drugs and predictive toxicology
This is an IMI funded project aiming at deriving human iPSC lines from 500 patients with neurodegenerative disorders. I am co-leading WP3 with Dr Lyle Armstrong
Funding sources:
EU
Development stage:
Early preclinical
Expected completion date
2017
Cell type:
Other
Cell source:
Not specified
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Not stated
Disease area:
Neurology
Clinical indication:
Neurodegeneration
Majlinda Lako, Newcastle University
iPS-based disease model for AMD
Assessing the feasibility of induced pluripotent stem cells to provide a disease model for age-related macular degeneration
Funding sources:
-
Development stage:
Early preclinical
Expected completion date
-
Cell type:
Induced pluripotent stem cells
Cell source:
Not specified
Autologous/allogeneic:
Both
Gene modification/gene therapy:
Not specified
Disease area:
Ophthalmology
Clinical indication:
Blindness caused by age related degeneration of retina
Majlinda Lako, Newcastle University
Development of synthetic retina
Exploiting the power of human induced pluripotent stem cells to generate synthetic retina in vitro for cell based therapies, drug discovery and disease modelling.
Funding sources:
EU
Development stage:
Early preclinical
Expected completion date
-
Cell type:
Induced pluripotent stem cells
Cell source:
Not specified
Autologous/allogeneic:
Both
Gene modification/gene therapy:
Not specified
Disease area:
Ophthalmology
Clinical indication:
Blindness caused by age related degeneration of retina or inherited retinal disorders
Anna David, University College London
BOOSTB4
Boost Brittle Bones Before Birth. A clinical trial to compare in utero plus postnatal mesenchymal stem cell transplantation with postnatal transplantation alone, in fetuses with a molecular diagnosis of severe osteogenesis imperfecta. Phase I trial of safety and comparing with historical controls for efficacy. To be conducted in 4 EU centres of excellence: UCL/GOSH London, Leiden Netherlands, Koln Germany, Karolinska Sweden.
Funding sources:
EU Horizon 2020
Development stage:
Late preclinical
Expected completion date
31.12.16
Cell type:
T cells
Cell source:
First trimester terminations of pregnancy at Karolinska Tnstitutet, Stockholm
Autologous/allogeneic:
Allogeneic
Gene modification/gene therapy:
No
Disease area:
Congenital skeletal dysplasia
Clinical indication:
Severe osteogenesis imperfecta

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