Astellas Gene Therapies is developing gene therapy for the treatment of X-Linked Myotubular Myopathy.
Resamirigene bilparvovec (AT132) is an investigational gene therapy designed for the delivery of functional copies of the MTM1 gene into skeletal muscle cells of patients affected by X-Linked Myotubular Myopathy. Utilizing a recombinant adeno-associated viral vector serotype 8 (AAV8), AT132 aims to restore the expression of the myotubularin protein primarily in muscle cells.
AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA). For the current status of the drug development program, please check the latest press releases through the “Latest News” feature on the home page of this website.
Astellas Gene Therapies is developing gene therapy for the treatment of Pompe disease.
AT845 is an investigational gene therapy designed for the delivery of functional copies of the human acid alpha-glucosidase gene (GAA) into the muscles of patients affected by Pompe disease. Utilizing a recombinant adeno-associated viral vector serotype 8 (AAV8), AT845 aims to express the GAA protein directly into skeletal and cardiac muscle cells with the aim of increasing GAA enzyme activity and reducing excess glycogen.
AT845 has been granted Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Astellas Gene Therapies is developing gene therapy for the treatment of Friedreich ataxia.
ASP2016 is an investigational AAV gene therapy designed to restore functional FXN expression and increase frataxin protein levels to restore normal cellular functions.
Astellas Gene Therapies is developing gene therapy for the treatment of Myotonic Dystrophy type 1.
AT466 is an investigational recombinant gene therapy designed to insert a functional copy of the MBNL1 gene, allowing for normal protein expression in the affected cells of patients with Myotonic Dystrophy type 1. Utilizing a recombinant adeno-associated viral vector (AAV), AT466 aims to prevent the accumulation of toxic RNA aggregates and to replenish functional proteins in affected cells, thereby restoring normal cellular function.
Astellas Gene Therapies is developing gene therapy for the treatment of X-Linked Myotubular Myopathy.
KT430 is a preclinical next-generation investigational gene therapy that delivers a functional copy of the MTM1 gene via a novel MyoAAV capsid to treat X-linked myotubular myopathy (XLMTM).
Gene replacement therapy
Vectorized RNA Knockdown
THE SAFETY AND EFFICACY OF THE AGENTS UNDER INVESTIGATION HAVE NOT BEEN ESTABLISHED. THERE IS NO GUARANTEE THAT THE AGENTS WILL RECEIVE REGULATORY APPROVAL OR BECOME COMMERCIALLY AVAILABLE FOR USES BEING INVESTIGATED. THESE AGENTS ARE NOT AUTHORIZED FOR SALE IN ANY JURISDICTION.
*AT132 program is on FDA clinical hold as of September 3, 2021. The information displayed is current as of July 2023.
Research Collaborations
We believe our strong relationships with key opinion leaders, leading academic institutions, other rare disease companies and patient advocacy groups will support our product development efforts and our potential for future success.
US Healthcare Professionals who have questions or would like additional information are welcome to contact Astellas Medical Affairs at: [email protected].
Our Drug Development Process
Our clinical development programs are designed to test and demonstrate the efficacy and safety of these novel treatments. We work with the highest standards to run thoughtfully-designed, robust, and well-executed clinical trial programs that evaluate the safety and effectiveness of the investigational gene therapies.
In designing and implementing our programs, we seek a careful balance of pursuing rigorous scientific research, guarding patient safety, and working with the urgency our patients and families deserve. Our trials are intended to meet the requirements of regulatory authorities who carefully evaluate the study results before approving the therapy for use in the broader patient population.
Discovery
Early research conducted in a laboratory in which an investigational gene therapy is initially promising enough to move to the next stage of research. This can take several years.
Preclinical Research
Research conducted prior to introducing the investigational gene therapy to human participants. This research is critical to understanding the potential safety and efficacy of the investigational therapy prior to testing in humans.
Clinical Trials
Research conducted with an investigational gene therapy in humans to confirm that the therapy is safe to use in patients and works as expected.
Potential Regulatory Marketing Approval for Use in Patients
If approved by the local governing regulatory body, the product becomes commercially available to the broader patient population.
Clinical Trials
Astellas Gene Therapies actively partners with the patient and caregiver community to support clinical trials. Clinical trials are the safest and most effective way to study the potential benefits and risks of these investigational therapies.
A clinical trial is a carefully-monitored experiment that helps determine whether an investigational product or disease intervention is safe and effective for treating the condition in which it is being studied. This means there is no guarantee that a person in a clinical trial will benefit from the investigational product and there may be unanticipated risks associated with participating. The safety of participants is monitored by the clinical trial physician throughout the conduct of a trial.
For ongoing clinical trials sponsored by Astellas with investigational therapies currently in development, please visit https://clinicaltrials.gov/.
