The successful candidate will play a key role shaping the future of Fulcrum Therapeutics by helping lead the Biology Team to identify and enable targets through the design of robust gene and protein expression assays or identified computationally using innovative primary and iPS cell derived cell models from patients with genetically defined central nervous system (CNS) and musculoskeletal disorders. The candidate will be responsible for a talented team of creative scientists that will leverage state-of-the-art CRISPR genomic and small molecule phenotypic screening in a highly automation leveraged HTS environment. This leader will need to work across an excellent multi-disciplinary team to enable targets for small molecule drug discovery and lead the team to generate decisional, statistically robust in vitro pharmacology data to inform an innovative design chemistry team. In this leadership role, the successful candidate will be responsible for enablement of drug discovery targets with generation of intellectual property, leading discovery project team activities, presenting scientific data internally and externally, authoring study reports with regulatory documentation, contributing to high impact scientific manuscripts, identifying & managing external research collaborations, and engaging with scientific advisors and founders. In this position, this leader will be expected to work collaboratively with others across multiple disciplines while mentoring a growing team and supporting Fulcrum’s culture and core values.
- Lead the Biology team to design, execute and build quality-controlled target discovery assays and conduct in-vitro pharmacology studies using innovative human models of CNS and musculoskeletal disorders. Responsibilities include rigorous statistical analysis, data reporting, data visualization, etc.
- Ensure execution of studies aimed at building pharmacologic relationships between drug concentration, target engagement, pharmacodynamic markers and disease protein gene expression in human cell models of neuromuscular disease.
- Propose new programs/diseases/areas of science that could be considered for future portfolio expansion
- Lead team to perform target mediated mechanistic and pharmacology studies using genetic techniques including CRISPR, RNAi/shRNA, ASO, etc. in collaboration with a matrixed team of computational scientists, cell biologists, transcriptional biologists, physiologists, in vivo pharmacologists, and translational biomarker team members.
- Coordinate with collaborators and CROs, as needed, to manage in vitro experiments and set clear goals and expectations for the broader team.
- Mentors and develops individual talent capable of taking on future leadership roles. Supports an overall group culture that is agile, proactive, collaborative, and innovative.
- Ph.D (with 15+ years of drug discovery industry experience) in genetics, molecular biology, or neuroscience and track record of supervisory experience.
- Track record delivering validated drug discovery targets using CRISPR genomic and small molecule phenotypic screen. Experience with primary or iPSC- derived neurons and myotubes would be an advantage.
- Extensive experience leading a team to develop statistically robust cell-based assays to support small molecule drug discovery, HTS experience, quantitative immunocytochemistry and analysis, excellent HTS statistical or image analysis algorithms and data analytics allowing for mechanistic interrogation of small molecule pharmacology in CNS and musculoskeletal disorders is necessary.
- Extremely effective project team leader, supervisor and mentor in a matrixed environment that can motivate a high functioning team of scientists to cultivate critical path thinking and agility in drug discovery.
- Experienced team leader who effectively manages external collaborations and service providers and ability to bring pragmatic business minded approach to balancing internal and external capabilities.
- Professionally represents the group and company more broadly in collaborations, partnership discussions, external relationships and conferences.
- Proven track record of scientific contributions in target discovery using genomic and small molecule screening as evidenced by quality and quantity of publications/ presentations/ inventions and clinical candidates.
- Highly motivated cross-functional team player who is flexible with ability to work in a fast-paced environment. Ability to provide novel, innovative solutions to challenging problems. Desire to learn and tackle new challenges and initiatives. Possess an extremely high degree of scientific discretion, intuition and integrity.
- Strong written and oral communication is a requirement to support internal and external communication of data, intellectual property filings, study reports and regulatory documentation.
- Excellent teamwork in a highly matrixed environment with ability to mentor, grow leaders and maintain focus on Fulcrum’s culture and values.