USA flag logo/image

An Official Website of the United States Government

Preclinical Studies Supporting Phase Ib Study of Minidystrophin Gene in AAV…

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
89390
Program Year/Program:
2008 / SBIR
Agency Tracking Number:
NS060181
Solicitation Year:
N/A
Solicitation Topic Code:
N/A
Solicitation Number:
N/A
Small Business Information
ASKLEPIOS BIOPHARMACEUTICAL, INC.
ASKLEPIOS BIOPHARMACEUTICAL, INC. 45 North Chatham Parkway CHAPEL HILL, NC 27517
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2008
Title: Preclinical Studies Supporting Phase Ib Study of Minidystrophin Gene in AAV Vecto
Agency: HHS
Contract: 1U44NS060181-01A1
Award Amount: $2,694,020.00
 

Abstract:

DESCRIPTION (provided by applicant): This proposal intends to advance the regional delivery of a chimeric adeno-associated virus (rAAV)-based, highly-truncated dystrophin (minidystrophin) gene therapy for the treatment of Duchenes Muscular Dystrophy (DMD). Experiments focus on completing preclinical studies required to initiate, the also proposed, FDA recommended Phase Ib bridging study. The proposed Phase Ib investigates regional gene delivery to the lower extremities of DMD patients. More specifically, th e studies herein evaluate vector delivery (transduction potential) and expression profiles achieved using blood vessel-mediated, regional, isolated lower limb infusion (ILP) in large animal models [the Golden Retriever Muscular Dystrophy model (GRMD) and n on-human primates (NHP)]. The GRMD model offers the additional potential to investigate the efficacy of rAAV- minidystrophin in ameliorating the nearest pathological counterpart of DMD. Our preliminary Phase 1 data indicate rAAV-minidystrophin is well tole rated and mediates expression levels as high as 2.5 transgene copies/diploid genome when directly injected in the bicep. Additionally, initial investigations of tranvascular limb delivery in GRMD and NHP demonstrate widespread transduction potential, and s ustained high levels of gene expression. These data cumulatively allude to the positive outcomes expected from the proposed preclinical studies on which the jointly proposed Phase Ib trial is contingent. A positive outcome of rAAV-minidystrophin in Phase 1 b clinical trials is expected to immediately impact DMD patients by supporting Phase II clinical trials of regional limb delivery of rAAV-minidystrophin. More importantly advancing a gene therapy for DMD offers the significant potential for prolonged ambul ation, increased exercise capacity, and reduced disease progression to a currently incurable population whose treatments remain palliative. In recognition of this potential and in light of the preliminary studies including an Interim DSMB/IDMC Phase Ia tri al report, Asklepios Biopharmaceutical has been awarded an MDA TRAC grant funding 50% of the proposed studies. This proposal intends to advance the regional delivery of a gene therapy for the treatment of Duchenes Muscular Dystrophy (DMD). Experiments focu s on completing preclinical studies in large animal models that are required to initiate, the also proposed and FDA recommended, Phase Ib bridging study that involves regional gene delivery to the lower extremities of DMD patients. Advancing a gene therapy for DMD offers the significant potential for improved quality of life to a currently incurable population whose treatments remain palliative.

Principal Investigator:

Business Contact:


resadmin@askbio.com
Small Business Information at Submission:

ASKLEPIOS BIOPHARMACEUTICAL, INC.
ASKLEPIOS BIOPHARMACEUTICAL, INC. 45 North Chatham Parkway CHAPEL HILL, NC 27517

EIN/Tax ID: 200267834
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No