AAV9 Microdystrophin Vectors

INTRODUCTION: This is a Sources Sought notice. This is NOT a solicitation for proposals, proposal abstracts, or quotations. The purpose of this notice is to obtain information regarding the availability and capability of all qualified sources to perform a potential requirement. This notice is issued to help determine the availability of qualified companies technically capable of meeting the Government requirement and to determine the method of acquisition. It is not to be construed as a commitment by the Government to issue a solicitation or ultimately award a contract. Responses will not be considered as proposals or quotes. No award will be made as a result of this notice. The Government will NOT be responsible for any costs incurred by the respondents to this notice. This notice is strictly for research and information purposes only. BACKGROUND: The National Institutes of Health (NIH) is the nation's leading medical research agency and the primary Federal agency whose mission is to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability, conducting, supporting and making medical discoveries that improve people's health and save lives. The mission of the Therapeutics for Rare and Neglected Diseases (TRND) program, which is part of the National Center for Advancing Translational Sciences (NCATS), is to facilitate therapeutic development for rare and neglected diseases. PURPOSE AND OBJECTIVES: The purpose of this acquisition is to produce three different non-GLP (Good Laboratory Practice) grade gene vectors of microdystrophin to be tested in a mouse Duchenne Muscular Dystrophy (DMD) disease model for efficacy. Once tested in the animal model, the TRND team and its collaborators can move forward with selecting an effective vector to be developed as a potential disease modifying therapy for DMD patients. PROJECT REQUIREMENTS: Three (3) different AAV9 (adeno-associated virus) microdystrophin gene vectors synthesized, cloned, manufactured (1 Mega Prep per construct) and characterized/released per required testing for animal administration: viral titer, purity, endotoxin, and any additional steps required for quality assurance. Upon award, the Government will provide the associated data indicating the specific vectors to be produced. Performance shall include: 1. Cloning of the new plasmid 2. Plasmid DNA production 3. DNA Structure and Sequence Analysis (for quality assurance purposes) 4. Production of 1 Mega Prep per construct 5. Testing & Release of each production lot ANTICIPATED PERIOD OF PERFORMANCE: Within 12 months of the date of award. DELIVERY LOCATION All items shall be delivered for inspection and acceptance to: 9800 Medical Center Dr. Rockville, MD 20850 OTHER IMPORTANT CONSIDERATIONS: The vendor will not retain any intellectual property (IP) rights on the vector lots produced nor is there any expectation that novel IP will result from this work. The sequences being used already fall under existing IP; the importance of this work is to help determine if any of them hold a true functional benefit over the others and may therefore translate to a better therapy in DMD patients. Special shipping considerations must be used to maintain product integrity. CAPABILITY STATEMENT / INFORMATION SOUGHT: Respondents must provide clear and convincing documentation of their capability of providing the products specified in this notice. Also, information must be provided in sufficient details of the respondents' (a) staff expertise, including their availability, experience, and formal and other training; (b) current in-house capability and capacity to perform the work; (c) prior completed projects of similar nature; (d) corporate experience and management capability; and (e) examples of prior completed Government contracts, references, and other related information. Respondents must provide their DUNS number, organization name, address, point of contact, and size and category of small business (e.g., 8(a), HUBZone) pursuant to the applicable NAICS code AND any other information that may be helpful in developing or finalizing the acquisition requirements. Finally, respondents are also encouraged to provide a general overview of the respondent's opinions about the difficulty and/or feasibility of the potential requirement, and any information regarding innovative ideas or concepts that may be applicable. SUBMISSION INSTRUCTIONS: One (1) copy of the response is required and must be in Microsoft Word or Adobe PDF format using 11-point or 12-point font, 8-1/2" x 11" paper size, with 1" top, bottom, left and right margins, and with single or double spacing. The information submitted must be must be in an outline format that addresses each element of the "PROJECT REQUIREMENTS" and each element of the "CAPABILITY STATEMENT / INFORMATION SOUGHT" paragraphs above. A cover page and an executive summary may be included but is not required. The response is limited to ten (10) pages. The page limit does not include the cover page, executive summary, or references, if requested. The response must include the respondents' technical and administrative points of contact, including names, titles, addresses, telephone and fax numbers, and e-mail addresses. All responses to this notice must be submitted electronically to the Contract Specialist (Facsimile responses are NOT accepted.): Matthew Antonini Contract Specialist NIH/NIDA/SSSA matthew.antonini@nih.gov DISCLAIMER AND IMPORTANT NOTES: This notice does not obligate the Government to award a contract or otherwise pay for the information provided in response. The Government reserves the right to use information provided by respondents for any purpose deemed necessary and legally appropriate. Any organization responding to this notice should ensure that its response is complete and sufficiently detailed to allow the Government to determine the organization's qualifications to perform the work. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted. After a review of the responses received, a presolicitation synopsis and solicitation may be published in Federal Business Opportunities. However, responses to this notice will not be considered adequate responses to a solicitation. CONFIDENTIALITY: No proprietary, classified, confidential, or sensitive information should be included in your response. The Government reserves the right to use any non-proprietary technical information in any resultant solicitation(s).