Drug discovery through development-track, IND-enabling through phase II/III, pre/nonclinical/regulatory strategies, in-/out-licensing of assets, business planning, operations, and people development/staff mentorship.Track record: Significant contributor to >90 projects, >35 development-track decisions, >30 IND/CTN/CTNs, >5 NDA/BLA/MAAs, >25 diligences, and hundreds of supporting and integrated final reports.I'm a board-certified toxicologist with >25 years' industry experience in pre/nonclinical development as Department/Line Head, Project Toxicologist/Program Team representative, and/or Study Director. As I have multiple interactions every year with regulatory authorities (FDA, EU member countries, HC, MHRA, and PMDA), I'm confident about addressing scientific and regulatory hurdles throughout the drug approval process. A path is customized toward value-creating milestones such as IND or CTA based on the evaluation of a compound's/protein's/cell's developability at each stage of development, including the anticipated risks and options available. I've taken an early-stage biotech company from seed-stage financing into PoC phase 1/2 trials. Services-Pharmacology/Toxicology Study Design and Protocol DevelopmentManagement of GLP Toxicology Studies and non-GLP Preclinical StudiesRisk Mitigation/Management of ToxicityPharmaceutical/Biopharmaceutical ProductsDisease Areas/IndicationsRegulatory Document Writing/Preparation, and ReviewRegulatory Guidance and StrategyToxicology RepresentationDue Diligence ActivitiesMy credentials (CV/resume, bio, and leadership position) are available upon request. References available upon request.Please contact me for more details and an introductory discussion about your pre/nonclinical/toxicology needs, project scope, and timing.email: rpfeifer1@cox.netThank you!

-Provide timely and resource-efficient execution of all pre/nonclinical activities needed to advance ex vivo genetic therapies into Phase I/II/II testing (LSD/rare disease indications).-Maintain an infrastructure of CROs and leverage internal R&D and external capabilities (academic PIs, consultants) supporting unique ex vivo LV-based GT platform testing needs and timing, e.g., Gaucher (GD1, GD3, GBA-Parkinson's), Cystinosis, and Pompe.-Ensure all Preclinical aspects of pre-CTA/CTN/IND planning are fully supported, including Program Team strategy, execution of in-life PD plans, bioanalytical assay method support, and Preclinical components of Regulatory submissions.-Provide leadership and oversight of a Research & Preclinical Development (R&PCD) group.-As pre/nonclinical rep on Program Teams, support stage-appropriate strategy and progression of follow-on programs, incorporating learnings from 1st generation programs.-Provide scientific mentorship and Nonclinical regulatory expertise to R&D personnel, PTs, and senior management to support timely and strategic decision-making.-Role-model managerial and leadership values, best practices, and define a R&PCD strategy for rare disease/LSD LV-based GT platform and support a culture of innovation and operational excellence.-Provide leadership in interactions with regulatory authorities and take calculated risks for AVROBIO's long-term benefit.-Promote/create unique opportunities that build trust with individuals, other Departments and Program Teams; reward actions or behavior that engender trust.-Whenever possible, present and/or publish efficacy/safety results within the industry to enhance AVROBIO's visibility.-Deliver innovative therapies to patients; employ cutting-edge science to advance life-changing therapies; and adapt new testing methods, processes and procedures to tackle new business challenges.-Supported sale of cystinosis GT asset to Novartis Cell & Gene therapy ($87.5M).

-Provided timely and resource-efficient execution of all pre/nonclinical activities needed to advance ex vivo genetic therapies into Phase I/II testing (LSD/rare disease indications).-Created an infrastructure of CROs, including in-life testing facilities and bioanalytical services, and consultants, supporting unique ex vivo LV-based GT platform testing needs and timing, e.g., Fabry, Gaucher, Pompe, and Early development programs, with an emphasis on CRO compatibility, technical expertise, quality (GLP compliance), and responsiveness.-Ensured all Preclinical aspects of pre-CTA/CTN/IND planning are fully supported, including Program Team strategy, execution of in-life PD plans, bioanalytical (enzyme activity, dual qPCR, immunogenicity) and flow cytometric methods, estimation of FIH safety margins, and Preclinical components of Regulatory submissions.-Ensured adequate support for Business Development/Due Diligence efforts, including input on fit/prioritization relative to current portfolio, critical technical reviews and timely, clear recommendations and written output.-Eighth (8th) employee hired, led pre/nonclinical effort through Series B and IPO financing, including CTA and IND approvals for the lead programs (Fabry and Gaucher).-Played a critical role in improving the quality and consistency of transitioned development candidates through formal/informal review of proposed study designs for pilot and PoC efficacy studies.

-Provided technical and strategic leadership in Toxicology and drug safety assessment within Research-Nonclinical Development (R-NCD) and across R&D.-Partnered with other Shire functions to communicate scientifically accurate information through presentation and publication to internal and external stakeholders, meeting appropriate legal and regulatory requirements.-Played a critical role in finalizing and implementing a new Asset Nomination Form (Q2'15) across all R-NCD functions to improve the quality and consistency of transitioned development candidates.-Generated a charter for/participated in the Immunogenicity Working Group.-As an 'ad hoc' member of Project Teams, supported stage-appropriate strategy and progression of novel post-NRPC Discovery projects and back-up programs, incorporating learnings from 1st generation programs.-As the point person for NCD, ensured adequate support of Business Development/Due Diligence efforts, including input on fit/prioritization relative to current portfolio, critical technical reviews and timely, clear recommendations and written output including study designs and endpoints, costs, and FTEs to in license; played a critical role in implementing a Due Diligence Guide (Q4'15) for R-NCD.-Supported in-licensing of a Ph III-ready asset, PF-00547659 (anti-MAdCAM mAb) from Pfizer, for IBD.

-Provided leadership and oversight of a nonclinical toxicology group (up to 7 PhD-level & 1 operational staff).-Provided expert level guidance in designing and implementing nonclinical toxicology studies and programs for humanized mAbs, soluble Fc-receptor fusion proteins, enzyme, protein, and mRNA replacement therapies, direct (AAV) gene therapies, antisense- and small-molecule-based therapeutics across all therapeutic areas (CNS/neuromuscular, renal/fibrotic, hematologic, GI/metabolic, ophthalmic, complement), for LSD/rare disease and broad indications.-Responsible for toxicology report preparation, presentation of data, and serving as internal representative for nonclinical development/safety issues on Early and Global Development Teams and to US and global regulatory agencies.-Managed scientific issues, provided strategic direction to development activities, and ensured that schedules were maintained to meet regulatory requirements.-Responsible for oversight of external toxicology contract research organizations (CRO) interactions, including GLP-level study oversight.-Facilitated a collaboration with Santaris Pharma to assess the efficacy, PK/biodistribution (BD), and neurologic safety of locked nucleic acid (LNA) antisense oligonucleotides administered intrathecally (IT).-Facilitated partnership with Sangamo to assess the efficacy, BD, and safety of a novel genetic therapy approach: site-directed, zinc finger nuclease gene editing (hemophilia B and Huntington's disease).-Preparation and/or review of nonclinical IND/CTA/CTN sections and NDA (VPRIV) and BLA (Replagal) submissions for 6 different enyzyme/protein replacement therapeutics (3 designed for IT administration, e.g., Hunter, Sanfilippo A, and MLD), an IND (anti-fibrotic) and NDAs (Xiidra and Resolor) for 3 small-molecule pharmaceuticals and a Complete Response for a peptide (Firazyr).-Facilitated smooth and efficient integration of ViroPharma staff and program/product support.

-Functioned as Drug Safety expert on exploratory, discovery, and Global Development/Learn teams for small-molecule pharmaceuticals, vaccines, and biopharmaceuticals (supported advancement of 12-15 compounds/proteins to development track, preparation of 8-12 INDs, initiation of 3 NME Phase 3 programs, and 2 NDAs/year). -Contributed to the development of 'next generation' Prevnar, Enbrel, Effexor, and Mylotarg, new products (Tygacil, Torisel, Bosulif, Viviant, Prevnar13, and Trumenba), and life cycle management (ReFacto and BeneFIX).-Planned and directed toxicology studies in collaboration with DSM scientists in Regulatory Toxicology/Pathology (Chazy, NY).-Managed an Exploratory Toxicology effort, including 7 persons (2 PhDs & 5 technical staff), including Immunotoxicology, Molecular Toxicology, and Investigative Pathology; conducted studies supporting discovery and development compounds/proteins across all therapeutic areas (Neuroscience, Inflammation, Cardiovascular/Metabolic Disease, Vaccines, Oncology/Infectious Disease, and Women's Heath & Bone/Musculoskeletal).-Provided leadership both within Wyeth and in the pharmaceutical industry concerning scientific and regulatory issues related to Immunotoxicology (e.g., served as member of ILSI HESI Immunotoxicology Technical Committee).-As Chairman of the Immunotoxicology Expert Working Group, facilitated: a novel collaboration between DSM and Inflammation Discovery to assess the safety of 'high risk' IMPs (EMA, 2007); and a Management Board decision to include FACS immunophenotyping in IND-enabling studies on a case-by-case basis.-Qualified TaqMan qPCR support for HIV plasmid (p)DNA vaccine BD studies (1 FTE for DSM included in NIH-funded $25M RFP).

-Directed and managed GLP-compliant nonclinical safety assessment studies for new chemical/molecular entities, internally or subcontracted, across a broad range of therapeutic categories; in 2001, collaborated with marketing to direct/manage a study load of ~$12 million (out of ~$25 million revenue).-Collaborated with Sponsors to implement IND/CTA-, NDA/BLA/MAA-enabling study designs/development programs.-Represented Sponsors before regulatory agencies.-Coordinated interdisciplinary study teams from shared-resource staffs: pharmacology/surgery, formulation and bioanalytical chemistry, infusion, immunobiology, clinical and anatomic pathology.-Responsible for effective and timely writing of protocols, reports, and summary/expert documents.-Provided guidance on safety issues (FDA/EU) and methods development in the area of Immunotoxicology.-Implemented corrective actions whenever required to ensure operational quality and consistency.

-Designed and executed GLP-compliant nonclinical safety and efficacy testing of pharmaceuticals to meet FDA, EU, and MHW guidelines.-Twenty-fourth (24th) employee hired, indirectly supervised manager-level scientific and administrative personnel (staff of ~100).-Provided technical evaluation of business development plans/proposals and marketed directly to Sponsors.-Accountable for fiscal management of specific programs: Program Manager for $1 million+ accounts, and individual study budgets.-Responsible for timely writing of protocols, reports, risk assessments, and summary/expert documents.-Consulted with Sponsors to implement IND/CTA, IND/BLA-enabling study designs/development programs.-Reviewed nonclinical sections of regulatory documents, integrated toxicity data with PK profiles, and represented Sponsors before regulatory agencies.-Served as Drug Safety representative on Project Teams: synthesis, formulation chemistry, metabolism/PK, bioanalytical, and pharmacology; ensured resource-efficient conduct and reporting to meet ambitious timelines.-Chairman, H&S Committee.

-Designed and directed GLP-compliant tests to evaluate product safety for FDA and MHW (devices/drugs/biologics), and EPA, FHSA, and OECD (consumer, agro- and industrial chemical) guidelines.-Responsibilities included assembly of project teams from shared-resource staffs, and business development (e.g. marketed directly to Sponsors).-Provided technical evaluation of business development proposals.-Indirectly supervised manager-level (including PhD-level) and administrative personnel (staff of ~90).-Reporting to the President, coordinated operational response of technical and QA/regulatory units.-Implemented new business initiatives: equipped surgical suite for neurological implant study, and clinical pathology with coagulation analyzer and platelet aggregometer.-Validated in vitro methods relevant to hemocompatibility and genetic toxicology testing, immunohistochemical staining (for GFAP), and implemented as SOPs.-Played an active role in attaining ISO 9001 certification.-Generated monthly revenue growth of approximately 30% (up to $500k/month).-Played a leadership role on the H&S Committee and the IACUC.

-Study Director for investigative programs relevant to carcinogen (epigenetic) risk assessment: biomarkers, short-term tests.-Supervised a laboratory with a technical staff of 5 (1 direct report).-Optimized and qualified molecular biological techniques (RT-PCR, ELISA) to monitor cytokine/growth factor gene expression in macrophages and implemented as SOPs; other techniques utilized: gel shift mobility assay, DNA sequencing/cloning, cell transfection and immunohistochemical staining (for PCNA).-Designed GLP-compliant toxicology studies (FIFRA) leading to product registration.-Served as in-house expert for government compliance (EPA, OECD) and methods development in Immunotoxicology.-Served as Chairman, H&S Committee, and on the Performance Review Task Force.

-Established and managed a laboratory characterizing the systemic actions of xenobiotics and drugs on the immune system and nonspecific host defense parameters.-Investigated the relationship of chemically induced inflammation to tumor promotion in mouse skin.-Identified strain-dependent difference in signal transduction (2-D gel phosphoprotein analysis) and generation of superoxide anion in tumor-promoter-stimulated mouse macrophages.-Secured funding (~$500k) as Principal Investigator (PI) or co-PI.-Supervised and mentored 4 doctoral candidates.-Developed curriculum/team-taught pharmacy, medical, and graduate students (Pathophysiology, Introductory & Advanced Pharmacology and Therapeutics, Introductory & Advanced Toxicology, Clinical Toxicology, and Immunotoxicology).-Prepared and submitted 12 grants/budgets to federal agencies.-Initiated the Flow Cytometry Facility.

-Established and managed a laboratory specialized for the conduct of immunotoxicological studies.-Planned conducted, and/or directed mechanisms research to elucidate the role of 17-B estradiol in sex-dependent immune responsiveness.-Identified a key role for catechol estrogen metabolites in T lymphocyte suppression.-Trained and supervised a technical staff of 7 (1 direct report).-Coordinated the program with NTP objectives: validation of immunotoxicology methods/monitoring of extramural contracts.

-Planned and conducted mechanisms research on benzene-induced lymphocytopenia and T cell suppression.-Supported in-house (TSCA) testing programs: ADME studies, inhalation, repro- and genetic toxicology; GLP training.-Identified particularly reactive thiols on tubulin as potential targets for quinone oxidation products of polyphenolic metabolites of benzene.-Designed the initial studies for the flow cytometric unit.-Trained and supervised a technical staff of 5.-Conducted human health risk assessment for benzene (e.g., testimony in support of the API position: 1980 AFL-CIO challenge of the 10 ppm TLV).