Astellas Gene Therapies
Astellas Gene Therapies is a Center of Excellence dedicated to developing genetic medicines with the potential to transform patients' lives. They focus on innovative science, an expanding AAV platform, and industry-leading manufacturing capabilities, especially targeting rare diseases of the eye, CNS, and neuromuscular systems. Their mission is to discover, develop, and deliver a broad portfolio of transformative therapies for genetic diseases worldwide, building an end-to-end gene therapy powerhouse.
Industries
Nr. of Employees
large (251-1000)
Astellas Gene Therapies
480 Forbes Boulevard, South San Francisco, CA 94080
Patents
Products
Investigational AAV-mediated gene therapy for X‑linked myotubular myopathy (MTM1 replacement)
An investigational AAV-based gene replacement therapy designed to deliver functional copies of the MTM1 gene to skeletal muscle cells to restore myotubularin expression.
Investigational AAV-mediated gene therapy for Pompe disease (GAA expression)
An investigational AAV-based therapy designed to deliver the GAA gene to skeletal and cardiac muscle to increase enzyme activity and reduce glycogen accumulation.
Investigational AAV-mediated therapy for Myotonic Dystrophy Type 1 (RNA-targeted approach)
An investigational recombinant vector therapy designed to restore functional protein expression and/or reduce toxic RNA aggregates in cells affected by Myotonic Dystrophy Type 1.
Preclinical next-generation AAV capsid program for MTM1 delivery
A next-generation capsid-enabled AAV program designed to improve delivery and efficacy for MTM1 gene delivery in X‑linked myotubular myopathy.
Investigational AAV-mediated gene therapy for X‑linked myotubular myopathy (MTM1 replacement)
An investigational AAV-based gene replacement therapy designed to deliver functional copies of the MTM1 gene to skeletal muscle cells to restore myotubularin expression.
Investigational AAV-mediated gene therapy for Pompe disease (GAA expression)
An investigational AAV-based therapy designed to deliver the GAA gene to skeletal and cardiac muscle to increase enzyme activity and reduce glycogen accumulation.
Investigational AAV-mediated therapy for Myotonic Dystrophy Type 1 (RNA-targeted approach)
An investigational recombinant vector therapy designed to restore functional protein expression and/or reduce toxic RNA aggregates in cells affected by Myotonic Dystrophy Type 1.
Preclinical next-generation AAV capsid program for MTM1 delivery
A next-generation capsid-enabled AAV program designed to improve delivery and efficacy for MTM1 gene delivery in X‑linked myotubular myopathy.
Services
End-to-end clinical development support including protocol design, safety monitoring and trial execution for investigational gene therapies.
Clinical- and commercial-scale cGMP drug substance and drug product manufacturing for AAV gene therapies, combined with QC laboratory testing and release workflows.
Partnership formation, licensing and sponsored-research collaborations with academic groups, biotech companies and patient organizations to advance gene therapy programs.
Programs to integrate patient and caregiver perspectives into research planning, clinical protocol design and educational outreach.
Provision of disease and therapy educational materials and medical information support for healthcare professionals.
End-to-end clinical development support including protocol design, safety monitoring and trial execution for investigational gene therapies.
Clinical- and commercial-scale cGMP drug substance and drug product manufacturing for AAV gene therapies, combined with QC laboratory testing and release workflows.
Partnership formation, licensing and sponsored-research collaborations with academic groups, biotech companies and patient organizations to advance gene therapy programs.
Programs to integrate patient and caregiver perspectives into research planning, clinical protocol design and educational outreach.
Provision of disease and therapy educational materials and medical information support for healthcare professionals.
Expertise Areas
- AAV gene therapy development
- Clinical trial management for gene therapies
- Bioprocessing and cGMP viral vector manufacturing
- Viral vector construct and capsid engineering
Key Technologies
- Adeno-associated virus (AAV) vectors
- Capsid engineering (next-generation capsids)
- Recombinant vector construct design
- Gene replacement therapy