A Single Pivotal Study of LION-101 in LGMD2I Patients
LION Therapeutics Non-confidential report
LION Therapeutics (part of Asklepios Bio) presented a request for advice on a test gene replacement therapy using AAV delivery in patients with FKRP mutations. This would use AAV9 and be delivered as a single intravenous injection into LGMD subjects with FKRP mutations.
The TACT committee provided comprehensive advice on all aspects of the proposal including the recommendation to look at multiple tissue types in pre-clinical models, clinical trial design, the selection of outcome measures, including patient-reported outcome measures and regulatory considerations.
The committee welcomed the opportunity to review the applicant’s thoughtful approach to preclinical assessment, clinical trial design based on natural history data, and clinical trial preparedness including vector production.
Name of applicant: Sharon Hesterlee Reviewed: April, 2019 in Glasgow, UK
LION Therapeutics (part of Asklepios Bio) presented a request for advice on a test gene replacement therapy using AAV delivery in patients with FKRP mutations. This would use AAV9 and be delivered as a single intravenous injection into LGMD subjects with FKRP mutations.
The TACT committee provided comprehensive advice on all aspects of the proposal including the recommendation to look at multiple tissue types in pre-clinical models, clinical trial design, the selection of outcome measures, including patient-reported outcome measures and regulatory considerations.
The committee welcomed the opportunity to review the applicant’s thoughtful approach to preclinical assessment, clinical trial design based on natural history data, and clinical trial preparedness including vector production.
