BIOLOGICS SUBJECT MATTER EXPERT (SME) CONSULTING SERVICES

生物制品主题专家 (SME) 咨询服务

• 批准号: 10719191
• 负责人: SUSAN BENJAMIN
• 金额: $ 5.6万
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-04-15 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10719191
• 关键词: Address; Antisense Oligonucleotides; Area; Biological; Biological Assay; Biological Products; Biological Response Modifier Therapy; Cell Therapy; Cells; Chemistry; Clinical; Clinical Research; Complex; Consult; Contractor; Contracts; Data; Development; Development Plans; Electronic Mail; Ensure; Evaluation Studies; Feedback; Formulation; Government; Guidelines; In Vitro; Investigational Drugs; Investigational New Drug Application; Label; Leadership; Lentivirus; Liposomes; Logistics; Methodology; Methods; Modality; Needs Assessment; Oligonucleotides; Peptide antibodies; Performance; Persons; Pharmacology; Pharmacology Study; Pharmacology and Toxicology; Polymers; Preparation; Principal Investigator; Process; Proteins; Protocols documentation; Published Comment; Rare Diseases; Recombinant Proteins; Regulation; Regulatory Affairs; Reporting; Resources; Risk; Role; Running; Services; Small Interfering RNA; Strategic Planning; Telephone; Testing; Therapeutic; Time; TimeLine; Toxicology; United States National Institutes of Health; Viral; Viral Vector; Visit; Work; analytical method; base; design; evaluation/testing; in silico; in vivo Model; lead optimization; meetings; member; method development; model development; nanoparticle; nervous system disorder; operation; pharmacokinetics and pharmacodynamics; pre-clinical; preclinical evaluation; programs; research and development; research clinical testing; safety study; safety testing; scale up; screening; symposium; therapeutic development

The consultants will be expected to provide feedback and guidance on scientific processes and gap areas/needs assessments through in-person meetings, conference calls, and email. The consultants will also be required to provide information on the ultra-rare disease space, preferably with a focus on neurological disorders. This will assist the NIH with better understanding the requirements for product developers to move their therapeutics through the research and development stage into clinical evaluation studies as well as development strategies and alternatives. Specific consulting responsibilities will depend on the expertise of the consultants and the needs of the program. Performance Area 1: Biologics Chemistry, Manufacturing and Controls (CMC) SME(s) Subject matter experts in manufacturing of the various biological therapeutics platforms, including but not limited to, modalities such as therapeutic oligonucleotides (e.g., antisense oligonucleotide -ASOs, small interfering RNAs -siRNAs, etc.), AAV, lentivirus or other viral vector therapeutics, purified proteins, recombinant proteins, peptides, antibody-based and cell-based therapeutics, and ex vivo cellular therapies are required. Biologics CMC consultants will be expected to provide senior-level chemistry, manufacturing and controls expertise and contribute feedback and guidance on projects to the NIH and to LDT members through conference calls and by email. Please note that, for Performance Area 1, the Government will consider SME consultants with expertise in one or more manufacturing modalities. The role of the CMC SME consultant may include, but is not limited to, the following responsibilities and tasks: a) Review and comment on the adequacy of biologic manufacturing, including scale-up, analytical method development, and process development (upstream and downstream processing) approaches proposed by Principal Investigators (PIs) and CMOs b) Provide guidance on delivery methods, which may include complex formulations such as polymer, liposomes, and other nanoparticle constructs c) Provide expert advice on master and working cell and viral bank development and testing d) Provide strategy and feedback on biologics process development and manufacturing plans proposed by the team e) Provide feedback on therapeutic oligonucleotide sequences and synthesis processes proposed by the team f) Provide feedback on proposed biologics formulations g) Provide feedback on critical quality attributes, release testing, etc. h) Evaluate CMC data on a weekly or biweekly basis i) Serve as the CMC expert in order to identify potential product manufacturing challenges and suggest strategies to address these challenges j) Accompany NIH staff or visit, at NIH staff request, CROs and CMOs under contract to the NIH. k) Inspect facilities and discuss appropriateness of CRO/CMO proposed methodology l) CMC consultant may be asked to evaluate the potential tractability (including surrounding IP space) of a proposed biological therapeutic m) Collaborate on design of investigative studies in support of therapeutics development projects n) In partnership with other consultants, contractors, and the NIH staff, advise, strategically plan, and manage CMC to facilitate preclinical evaluation and safety testing, Investigational New Drug (IND) submissions, and clinical studies o) Facilitate LDT discussions via telephone and email regarding assigned CMC efforts p) Provide strategic guidance for the continued development and improvement of the BPN-Biologics, HEAL PTDP, and URGenT biologics programs Performance Area 3: Toxicology SME(s) Toxicology SME consultants will be expected to provide senior-level expertise and contribute feedback and guidance on projects to the NIH and to LDT members through conference calls and by email. The role of the consultants may include but are not limited to the following responsibilities and tasks: a) Help establish toxicology endpoints and milestones for a project, recommend bioassays to be incorporated into a testing funnel or development plan, design toxicology and pharmacology studies, and interpret data delivered by CROs. b) In partnership with other consultants, contractors, and the NIH staff, advise, strategically plan, and manage programs to facilitate lead optimization, IND enabling, and clinical studies c) Serve as a resource to the LDT for toxicology requirements, pharmacology and logistics to conduct biologics development activities for biologics from the preclinical stage through to the initial clinical stage d) Serve as the toxicology expert in order to identify potential developmental challenges and suggest strategies to address these challenges e) Collaborate on design of investigative toxicology studies in support of biologics development f) Review toxicology sections of regulatory filings provided by CROs or PIs g) Collaborate on the design of investigative studies in support of biologics development. h) Provide expert support of preclinical strategy development including that for neurological diseases i) Support in vitro and in vivo model development for neurological disease j) Support design and interpretation of in silico analyses and in vitro/ ex vivo screening strategies for oligonucleotides k) Support of design PK/PD and toxicology safety studies Performance Area 5 – Regulatory Affairs and Regulatory Operations SME(s) Regulatory Affairs consultants will provide advice and leadership in support of regulatory affairs strategy for NIH biologics programs. Consultants will be asked to provide guidance on IND-enabling studies to ensure that teams meet the guidelines and timelines of IND applications. Consultants will serve as the principal regulatory contact with NIH staff, PIs, and regulatory agencies, and may also be the primary regulatory representative to the FDA. The roles of the Regulatory Affairs and Regulatory Operations SME consultants may include but are not limited to the following responsibilities and tasks: a) Work within project teams to develop regulatory strategies, identify regulatory risks, and enable timely approval b) Advise NIH staff and project teams on issues related to regulatory strategy and identify areas of concern regarding developing regulations c) Provide input on and review specifications, methods, SOPs, protocols, reports, labeling, etc. for regulatory compliance d) Develop and manage timelines for regulatory submissions e) Work effectively to coordinate the activities of CROs and other consultants in the preparation of regulatory submissions as necessary f) Prepare and submit IND applications to the appropriate regulatory agency g) Prepare for and run regulatory agency meetings

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