1919878_J&A.pdf

Attachment 5 Page 1

Justification for Other than Full and Open Competition Sole Source

“Source Selection Information – see FAR 2.101 and 3.104”

1. Identification of the agency and contracting activity.

a. Federal agency and contracting activity. DHHS/National Institutes of Health (NIH)/National Institute of Allergy & Infectious Diseases (NIAID)/Acquisitions Management Operations Branch (AMOB)

b. Sponsoring organization. Division of Intramural Research (DIR), Laboratory of Clinical Immunology and Microbiology (LCIM)

c. Project Officer Information:

Project Officer Name. Dr. Suk De Ravin

Mailing address. 10 Center Drive, Bethesda, MD 20892

E-mail address. sderavin@mail.nih.gov

Telephone number. (301) 251-6638

d. Contract Specialist: Bradley C. Engel, (301) 761-6795, brad.engel@nih.gov

2. Nature and/or description of the action being approved.

a. Acquisition purpose and objectives.

LCIM requires clinical grade virus vectors for gene therapy trails. This clinical grade materials permits CRISPR-mediated gene editing for targeted correction of Chronic Granulomatous Disease (CGD) in a clinical trial. The principle of the approach is to induce double strand Deoxyribonucleic Acid (DNA) breaks using an enzyme called Cas9 nuclease, which is guided to the desired site for cutting by a synthetic Ribonucleic Acid (RNA) based on sequence homology to the target site. Following the dsDNA break, the cell’s repair mechanism allows introduction of the desired sequence from a exogeneous donor template provided to the cells. The donor is delivered by a recombinant Adeno-Associate virus (AAV). The three critical components necessary to correct the stem cells are Cas9 nuclease, synthetic guide RNA and recombinant Adeno-associated virus (AAV) to deliver the correction template. We are now ready to translate this approach to a clinical protocol to treat patients and therefore require clinical grade AAV for the clinical application. Importantly, the patients with CGD suffer from frequent and sometimes deadly infections. The initiation of a clinical protocol employing the requested cGMP product will permit the determination whether this novel treatment can protect these patients from infections and potentially save their lives.

With that being said, this product is embedded into the research protocols and processes and cannot be changed at this time. This product must continue to be utilized to ensure data quality/integrity for ongoing research projects. No

Attachment 5 Page 2 substitutions can be utilized for this research. Please note this product has been approved by the Food and Drug Administration (FDA) for this particular study.

To change this product would require an all new, lengthy FDA approval.

b. Project background

The LCIM is comprised of a multi-disciplinary team that performs research on human patients suffering from diseases of the immune system or from infections caused by viral, bacterial, or fungal diseases. LCIM researchers study patients, clinical samples, animal models of diseases, cellular models and systems, and molecular tools with an aim to developing new therapies for patients with these diseases. CGD is a genetic immune disease that results often lethal infections and excessive inflammation. Despite decades of research, patients with CGD suffer from early mortality unless a suitable bone marrow transplant donor can be found. An alternate approach is to correct the patient’s own stem cells, a form of “gene therapy.” Current gene therapy approaches use retroviruses to randomly insert one or more copies of the gene (‘transgenes’) into the patients’ own stem cells. However, the long term consequences of random integration of transgenes and its expression under non-physiological promoters is unknown. To address these concerns, LCIM sought to investigate a targeted approach of gene therapy to correct the gene defect(s) in the patients’ own stem cells.

With that being said, clinical grade virus vectors are required for on-going research projects. Some of the research these virus vectors will support is as follows: Discovery of the gene mutations causing primary immune deficiencies and autoimmune disorders; bacterial pathogenesis (e.g., Mycobacterium, Borrelia, Chlamydia, Granulibacter); fungal pathogenesis (e.g., Cryptococcus, Candida, Aspergillus); viral pathogenesis (e.g., Herpes simplex and zoster, Vaccinia, Zika, Epstein-Barr); and development and testing of novel antimicrobial drugs, gene therapy, stem cell transplant, cytokines, monoclonal antibodies, and other therapeutics to modify or correct immune function, prevent infection, and reduce inflammation.

This is a follow-on requirement for additional clinical grade virus vectors for LCIM. The manufacturer of this product is Vigene Biosciences, Inc. This company sells directly to the public and does not have any resellers. Previous purchases for similar requirements were sole source acquisitions through open market and awards.

# AMBIS # Vendor Date Dollor Amount

1 1921631 Vigene Biosciences, Inc 7/19/2018 $1,365.50

2 1914871 Vigene Biosciences, Inc 6/13/2018 $1,500.00

3 1899197 Vigene Biosciences, Inc 3/8/2018 $1,500.00

4 1887946 Vigene Biosciences, Inc 1/3/2018 $1,463.00

5 1885963 Vigene Biosciences, Inc 12/13/2017 $1,323.20

6 1879116 Vigene Biosciences, Inc 10/26/2017 $1,323.20

7 1876488 Vigene Biosciences, Inc 10/11/2017 $1,463.00

8 1874471 Vigene Biosciences, Inc 9/8/2017 $3,445.00

9 1850710 Vigene Biosciences, Inc 5/9/2017 $1,155.00

10 1838334 Vigene Biosciences, Inc 2/24/2017 $1,425.00

11 1834945 Vigene Biosciences, Inc 2/3/2017 $2,785.00

12 1832106 Vigene Biosciences, Inc 1/18/2017 $1,430.00

13 1829893 Vigene Biosciences, Inc 1/3/2017 $763.00

14 1829250 Vigene Biosciences, Inc 12/22/2016 $1,155.00

15 1824417 Vigene Biosciences, Inc 11/21/2016 $2,780.00

16 1773240 Vigene Biosciences, Inc 1/5/2016 $428.00

17 1721281 Vigene Biosciences, Inc 1/28/2015 $228.00

18 1720681 Vigene Biosciences, Inc 1/26/2015 $2,534.00

19 1718991 Vigene Biosciences, Inc 1/13/2015 $1,785.00

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3. Description of the supplies or services required to meet the agency’s needs (including the estimated value).

a. Project title. Clinical Grade Virus Vectors

b. Project description.

Clinical grade virus vectors for gene therapy trails. This virus vector is utilized to deliver genetic material into cells. This virus vector is a critical aspect of research and is required for studies on targeted gene insertion in the laboratory of Dr. Harry Malech in the Genetic Immunotherapy Section of the Laboratory of Clinical Immunology and Microbiology, NIAID.

Requirement type.

Research & development (R&D) R & D support services Support services (non-R&D) X Supplies/equipment Information technology (IT) Construction Architect-engineer (A & E) services Design-build Other (specify): ____________________

Type of action.

New requirement X Follow-on to # AMBIS # Vendor Date Dollor Amount

1 1921631 Vigene Biosciences, Inc 7/19/2018 $1,365.50

2 1914871 Vigene Biosciences, Inc 6/13/2018 $1,500.00

3 1899197 Vigene Biosciences, Inc 3/8/2018 $1,500.00

4 1887946 Vigene Biosciences, Inc 1/3/2018 $1,463.00

5 1885963 Vigene Biosciences, Inc 12/13/2017 $1,323.20

6 1879116 Vigene Biosciences, Inc 10/26/2017 $1,323.20

7 1876488 Vigene Biosciences, Inc 10/11/2017 $1,463.00

8 1874471 Vigene Biosciences, Inc 9/8/2017 $3,445.00

9 1850710 Vigene Biosciences, Inc 5/9/2017 $1,155.00

10 1838334 Vigene Biosciences, Inc 2/24/2017 $1,425.00

11 1834945 Vigene Biosciences, Inc 2/3/2017 $2,785.00

12 1832106 Vigene Biosciences, Inc 1/18/2017 $1,430.00

13 1829893 Vigene Biosciences, Inc 1/3/2017 $763.00

14 1829250 Vigene Biosciences, Inc 12/22/2016 $1,155.00

15 1824417 Vigene Biosciences, Inc 11/21/2016 $2,780.00

16 1773240 Vigene Biosciences, Inc 1/5/2016 $428.00

17 1721281 Vigene Biosciences, Inc 1/28/2015 $228.00

18 1720681 Vigene Biosciences, Inc 1/26/2015 $2,534.00

Other (specify): ______________________

Proposed contract/order type:

X Firm-fixed-price

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Other fixed-price (specify, e.g., fixed-price award-fee, fixed-price incentive-fee): __________

Cost-plus-fixed-fee Other cost reimbursement (specify, e.g., cost-plus-award-fee, cost-plus-incentive-fee): ___________ Time and materials Indefinite delivery (specify whether indefinite quantity, definite quantity, or requirements): ___________ Other (specify): ___________ Completion Form Term form

Acquisition identification number. ABMIS 1919878 – FedBizOpps

NIAID-NOI18-1919878

c. Total estimated dollar value is $607,344.39. This price includes the following:

Product and provide Clinical Grade Virus Vectors. Must be awarded by September 7, 2018 in order to meet research deadline. Manufacture requires 10-months to produce and deliver product.

4. Identification of the statutory authority permitting other than full and open competition.

X This acquisition is conducted under the authority of 41 USC 3304(a)(1) and FAR Part 6.302-1 – Only one responsible source.

This acquisition is conducted under the authority of section 4202 of the

Clinger-Cohen Act of 1996.

This acquisition is conducted under the authority of the Services Acquisition

Reform Act of 2003 (41 U.S.C. 428a).

5. Demonstration that the proposed contractor(s) unique qualifications or the nature of the acquisition requires use of the authority cited.

a. Name and address of the proposed contractor(s).

Vigene Biosciences, Inc., 9430 Key West Avenue, Rockville, MD 20850

b. Nature of the acquisition and proposed unique qualifications of the contractor(s)

LCIM has developed extensive protocols for this virus vector and thus require the continued use of the Vigene Biosciences, Inc., virus vector, which is currently only produced by the manufacturer - Vigene Biosciences. Furthermore and most importantly, since we have been using this biological in ongoing research, we must maintain standards to ensure experimental results are not influenced by other variables. Any other source of media will require extensive testing and delay our CGD clinical trials. Also, to have successful research results, methods, and parameters must be consistent. Any changes during the study are unacceptable and may render years of existing data invalid. Salient requirements must be maintained. Finally and along with protocols/salient requirements, the FDA has approved this particular product for use in this research. LCIM cannot

Attachment 5 Page 5 change this product without lengthy, extensive delays with another FDA approval process. At this time and considering the human life aspect of this research, any change in research variables are unacceptable, the risk is too high.

This product cannot be substituted.

6. Description of the efforts made to ensure that offers are solicited from as many potential sources as practicable. Indicate whether a FedBizOpps notice was or will be publicized as required by FAR Subpart 5.2 and, if not, which exception under FAR 5.202 applies.

Pre-solicitation was publicized via FEDBIZOPPS on August 24, 2018. No responses were received.

7. Determination by the Contracting Officer that the anticipated cost/price to the Government will be fair and reasonable based on comparison to similar equipment.

The cost is within industry norms and is fair and reasonable. Documentation is filed in the acquisition folder.

8. Description of the market research conducted (see FAR Part 10) and the results, or a statement of the reasons market research was not conducted.

Several market research techniques were used to identify other possible sources, including review of acquisitions on similar items, queries of internet information sources, contact of knowledgeable individuals in the government and in industry, and review of catalogs and product literature. The results of this research indicated there is only one manufacturer/source for this product.

Market research was conducted on August 14, 2018. Some of the venues researched are as follows:

Vendor Person Description

1 Vigene Biosciences Dr. Jeffrey Hung Discussed sole source aspect

2 NIAID Federal Employee Dr. Suk De Ravin Required documentation

3 AMBIS

Researched previous history

Research revealed Laboratory utilized Leica Microsystems Inc.

portion of the research.

4 NIAID Federal Employee Mr. Phillip Decastro Acquisition strategy

5 DCIS Reviewed orders within DCIS

Search of DCIS was conducted with the following results:

# Contract ID Vendor Name Contracting Agency Date Signed Obligation ($)

1 HHSN261201600325P VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 20‐Jun‐16 $7,860.00

2 HHSN265201700046PC VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 17‐Jan‐17 $4,160.01

3 HHSN271201500769P VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 14‐Sep‐15 $5,732.00

4 HHSN271201800080P VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 10‐Jan‐18 $5,394.00

5 HHSN275201800069P VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 5‐Dec‐17 $5,614.00

6 HHSN276201500657P VIGENE BIOSCIENCES, INC. NATIONAL INSTITUTES OF HEALTH 4‐Sep‐15 $5,372.00

Attachment 5 Page 6

Search of AMBIS was conducted with the following results:

# AMBIS # Vendor Date Dollor Amount

1 1921631 Vigene Biosciences, Inc 7/19/2018 $1,365.50

2 1914871 Vigene Biosciences, Inc 6/13/2018 $1,500.00

3 1899197 Vigene Biosciences, Inc 3/8/2018 $1,500.00

4 1887946 Vigene Biosciences, Inc 1/3/2018 $1,463.00

5 1885963 Vigene Biosciences, Inc 12/13/2017 $1,323.20

6 1879116 Vigene Biosciences, Inc 10/26/2017 $1,323.20

7 1876488 Vigene Biosciences, Inc 10/11/2017 $1,463.00

8 1874471 Vigene Biosciences, Inc 9/8/2017 $3,445.00

9 1850710 Vigene Biosciences, Inc 5/9/2017 $1,155.00

10 1838334 Vigene Biosciences, Inc 2/24/2017 $1,425.00

11 1834945 Vigene Biosciences, Inc 2/3/2017 $2,785.00

12 1832106 Vigene Biosciences, Inc 1/18/2017 $1,430.00

13 1829893 Vigene Biosciences, Inc 1/3/2017 $763.00

14 1829250 Vigene Biosciences, Inc 12/22/2016 $1,155.00

15 1824417 Vigene Biosciences, Inc 11/21/2016 $2,780.00

16 1773240 Vigene Biosciences, Inc 1/5/2016 $428.00

17 1721281 Vigene Biosciences, Inc 1/28/2015 $228.00

18 1720681 Vigene Biosciences, Inc 1/26/2015 $2,534.00

9. Any other facts supporting the use of other than full and open competition.

The clinical grade products that are needed are require specialized development, production facilities, quality control, storage and transport. This product will be used in a clinical trial in human subjects and therefore must conform to GMP standards required by the LCIM and by other US Government. FDA is the regulatory authority over this particular study and has approved this particular product. At this time no other product may be utilized within this particular venue of the study.

10. Listing of sources, if any, that expressed, in writing, an interest in the acquisition.

No other sources have expressed an interest in the proposed acquisition.

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11. Statement of the actions, if any, the agency may take to remove or overcome any barriers to competition before any subsequent acquisition for the required supplies or services.

It is the policy of NIAID to promote the use of competitive procedures whenever and wherever possible. In an effort to remove or overcome barriers to competition in the future, the market will be scanned frequently for new companies and products that could satisfy the Government’s requirement. The staff meets regularly with products manufacturers and vendors and attends manufacturer and industry training to keep current on technology. Vendor and trade magazines are scanned for new, open technologies that can satisfy future infrastructure requirements. The agency will continually re-evaluate the products currently being used and will research for the manufacturers and products that will be in the Government’s best interest as best value for future replacement products at the agency’s site and at sites the agency supports. The current situation requires a sole source justification to satisfy requirements. Since there is a continual re-evaluation to products currently being used by the agency for the present requirement, if/when alternatives become available, they will be evaluated for use.