The Department of Pharmacology and Toxicology within Developmental Sciences is seeking an experienced regulatory/project toxicologist and strategic leader to develop and lead the execution of nonclinical safety strategy for gene therapy programs at Spark.

The individual will serve on Compound Development Teams for pre-clinical stage programs and on Non-Clinical Study Teams for clinical/late-stage programs across the various therapeutic areas within Spark with a concentration in systemic and ocular directed therapies. The successful candidate will use scientific and drug development expertise to play an important role in the development of gene therapies to address the high unmet medical need in rare and ultrarare diseases and rapidly advance early assets though the pipeline.

To this extent the individual will work in a collaborative team environment, to develop and implement the nonclinical safety strategy in assessment of Spark’s diverse portfolio in genetic medicines, including newer modalities. Individual will partner closely with the research team leads as well as other key stakeholders within Spark (regulatory, clinical, CMC, etc.), CROs, and/or external collaborators to design, execute, interpret, and report nonclinical (GLP and non-GLP) studies. Individual will ensure timely and accurate communication of study results and interpretation to appropriate internal teams and governing committees, contribute to discussions and facilitate decision making. Individual will help author necessary global health authority (INDs, CTAs, PIPs, BLAs, etc) and clinical (protocols, IBs, ICFs, etc) documents and represent Spark at necessary global health authority meetings.

Previous experience with genetic medicine, viral vectors, gene therapy, AAV, gene editing and/or LNP drug development/toxicology/regulatory interactions or ocular toxicology/drug development/regulatory toxicology experience is strongly preferred.  The individual may manage internal and contract staff.

Responsibilities

As a member of cross functional project teams, collaborate with cross functional team members to develop and drive integrated non-clinical toxicology and pharmacology strategies.

Provide scientific leadership in the design and implementation of safety assessment strategies to support program advancement and decision-making, including design and execution of necessary regulatory/GLP/nGLP studies to support program and regulatory milestones.

Proactively manage potential safety liabilities and communicate the impact to teams and governance committees. Support the design of necessary investigative studies to identify and mitigate treatment-related toxicities.

Preparation and review of study protocols, study reports, manuscripts, etc.

Deliver high quality nonclinical summaries and other high-level regulatory documents for internal decision making and for external health authority submission (including but not limited to IND/CTA, HA briefing books, Investigator Brochures, GMO documents, etc.).

Represent Spark at global health authority meetings as needed.

Maintain current knowledge of gene therapy regulatory guidance, industry standards and recommendations, and stay current on relevant literature and methodologies specific to the non-clinical safety of gene therapy products.

Partner with the CMC organization to address any product specific issues that may impact development of a gene therapy candidate.

Education and Experience Requirements

• A PhD in toxicology or a relevant field along with a minimum of 8 years of relevant experience in the pharmaceutical/biotechnology/CRO industry related to toxicology.
• Experience in drug development and interaction with global regulatory authorities are preferred.
• Experience with cross-functional teams and capability to build productive cross-functional collaborations are required.
• Experience in writing nonclinical sections of regulatory documents and regulatory interactions.
• Experience in designing non-GLP and GLP toxicology studies in support of pipeline decision making and regulatory milestones.
• Understanding of CMC, QA, regulatory and clinical stages to guide preclinical activities setting up products for success at later stages.
• Previous experience with genetic medicine, viral vectors, gene therapy, AAV, gene editing drug development/toxicology/regulatory interactions or ocular toxicology/drug development/regulatory toxicology experience is strongly preferred.
• The successful candidate must also demonstrate strong decision-making, complex problem solving, critical data analysis and interpretation, excellent written and verbal communication skills.
• Organized and skilled in managing and prioritizing multiple responsibilities, tasks, and projects.
• Ability to lead scientists and provide scientific and business strategic leadership.
• Some business travel required (Main travel will be Health Authority meetings and CRO visists. Other travel could include conferences, off sites, etc)
• This is a hybrid role with in office presence 3 days per week