Ceregene, Inc
Ceregene is a private biotechnology company based in San Diego, focused on developing neurotrophic growth factor treatments for neurodegenerative disorders using proprietary gene delivery technology. The company aims to develop treatments that can slow, halt, or reverse neuronal degeneration, with a pipeline of products targeting diseases such as Parkinson’s, Alzheimer’s, ALS, and ocular diseases. Ceregene combines research, product development, and clinical expertise to bring innovative therapies into clinical trials and potential commercialization.
Industries
Nr. of Employees
medium (51-250)
Ceregene, Inc
San Diego, California, United States, North America
Products
AAV-based neurturin gene therapy candidate for Parkinson’s disease
An AAV vector carrying the gene for neurturin delivered directly into dopamine-rich brain regions to promote survival and function of dopamine neurons and potentially slow or reverse motor decline.
AAV-based nerve growth factor gene therapy candidate for Alzheimer’s disease
An AAV vector delivering the gene for nerve growth factor to cholinergic regions of the brain to support survival and function of neurons implicated in cognitive decline.
AAV-based IGF-1 gene therapy candidate for motor neuron support (ALS)
Preclinical AAV-delivered gene candidate expressing insulin-like growth factor-1 intended to enhance motor neuron function and slow progression in amyotrophic lateral sclerosis models.
AAV-based NT4/5 gene therapy candidate for retinal degenerative diseases
AAV-mediated delivery of NT4/5 gene to retinal tissue to enhance photoreceptor survival and function for conditions such as retinitis pigmentosa and age-related macular degeneration (preclinical development).
AAV-based neurturin gene therapy candidate for Parkinson’s disease
An AAV vector carrying the gene for neurturin delivered directly into dopamine-rich brain regions to promote survival and function of dopamine neurons and potentially slow or reverse motor decline.
AAV-based nerve growth factor gene therapy candidate for Alzheimer’s disease
An AAV vector delivering the gene for nerve growth factor to cholinergic regions of the brain to support survival and function of neurons implicated in cognitive decline.
AAV-based IGF-1 gene therapy candidate for motor neuron support (ALS)
Preclinical AAV-delivered gene candidate expressing insulin-like growth factor-1 intended to enhance motor neuron function and slow progression in amyotrophic lateral sclerosis models.
AAV-based NT4/5 gene therapy candidate for retinal degenerative diseases
AAV-mediated delivery of NT4/5 gene to retinal tissue to enhance photoreceptor survival and function for conditions such as retinitis pigmentosa and age-related macular degeneration (preclinical development).
Services
Preclinical development and safety testing
Nonclinical efficacy and toxicology studies including in vitro assays and in vivo animal models to evaluate candidate gene therapies prior to clinical entry.
Clinical development and multi-center trial coordination
Design and conduct of Phase I and Phase II clinical trials for neurological gene therapies, including multi-site enrollment and collaboration with clinical centers.
AAV vector design and manufacturing methods
Design and preparation methods for AAV-based vectors to carry therapeutic neurotrophic genes, including modified vectors to limit replication and enable targeted expression.
Surgical delivery method development
Development and optimization of surgical procedures for precise delivery of gene therapy vectors to specific brain regions and retinal tissue.
Preclinical development and safety testing
Nonclinical efficacy and toxicology studies including in vitro assays and in vivo animal models to evaluate candidate gene therapies prior to clinical entry.
Clinical development and multi-center trial coordination
Design and conduct of Phase I and Phase II clinical trials for neurological gene therapies, including multi-site enrollment and collaboration with clinical centers.
AAV vector design and manufacturing methods
Design and preparation methods for AAV-based vectors to carry therapeutic neurotrophic genes, including modified vectors to limit replication and enable targeted expression.
Surgical delivery method development
Development and optimization of surgical procedures for precise delivery of gene therapy vectors to specific brain regions and retinal tissue.
Expertise Areas
- Gene therapy platform development
- AAV vector engineering
- Neurotrophic factor–based therapeutics
- Preclinical safety and toxicology studies
Key Technologies
- Adeno-associated virus (AAV) vector-based gene delivery
- Targeted intracranial injection
- Intra-retinal vector delivery
- Neurotrophic factor gene therapy (NGF, neurturin, IGF-1, NT4/5)