Program Lead, Development - Rare Disease

Overview

The Senior/Executive Director, Development Program Lead will provide strategic and operational leadership for two early-stage rare disease programs in the hepatobiliary space. The Program Lead will be accountable for building integrated development strategies and leading their execution through critical early clinical milestones. This individual will guide cross-functional teams in establishing the right priorities, anticipating future program needs, and making thoughtful investment decisions based on emerging clinical and scientific evidence.

This role requires an experienced drug development leader who can operate effectively in ambiguity, create clarity around the milestones that matter most, and maintain engagement across programs. The successful candidate will bring meaningful rare disease and early development experience, along with the ability to integrate diverse perspectives and influence stakeholders across functions and organizational levels.

The Program Lead will work collaboratively across Research, Early Development, Clinical Research, Clinical Operations, Regulatory Affairs, Biostatistics, Drug Safety, Medical Affairs, Manufacturing, Quality, Program Management, Clinical Supply, Commercial, and Finance.

This position is hybrid based to Alnylam’s Cambridge MA office, although consideration will be given to US-remote candidates with exceptional applicable experience.

Key Responsibilities

• Lead the development and execution of integrated, cross-functional strategies for early-stage rare disease programs.
• Establish a clear program vision, strategy, and set of prioritized milestones that align scientific, clinical, regulatory, operational, and business considerations.
• Develop fit-for-purpose program plans that reflect each asset’s stage, evidence base, investment level, and future potential while maintaining readiness to accelerate if emerging data support further development.
• Lead cross-functional teams in defining proof-of-concept strategies, decision criteria, critical assumptions, and data requirements needed to evaluate therapeutic potential and inform future investment decisions.
• Integrate scientific evidence, clinical data, regulatory considerations, patient needs, and the evolving external landscape into program strategy and decision-making.
• Translate complex and sometimes incomplete information into clear recommendations, priorities, and executable plans.
• Anticipate program risks, barriers, and interdependencies; establish mitigation and contingency plans and escalate issues appropriately to support timely decisions.
• Partner with functional leaders to align resources, capabilities, and subject-matter expertise with program milestones and deliverables.
• Foster an inclusive, high-performing team environment that encourages rigorous debate, transparent communication, shared accountability, and timely decision-making.
• Prepare and present program strategy, status, risks, scenarios, and recommendations to governance bodies and senior leadership.
• Ensure key decisions, assumptions, dependencies, and program risks are clearly documented and communicated across the broader organization.
• Serve as a visible and credible champion for the programs, articulating their scientific rationale, patient impact, strategic value, and development path.

Qualifications

• Advanced degree in medicine, science, pharmacy, or a related discipline. MD, PhD, PharmD, or equivalent advanced scientific or clinical training preferred.
• 10+ years of progressively responsible experience in biotechnology or pharmaceutical drug development, including significant experience leading cross-functional development teams or complex development programs.
• Demonstrated experience in rare disease drug development preferred.
• Meaningful experience with early-stage development, including first-in-human studies, Phase 1 development, proof-of-concept planning, and/or progression into Phase 2.
• Broad understanding of the drug development process and the interdependencies among clinical development, regulatory strategy, clinical operations, safety, biometrics, nonclinical development, CMC, and program management.
• Experience developing integrated program strategies, evaluating development scenarios, and translating scientific and clinical evidence into program decisions.
• Therapeutic-area flexibility, intellectual agility, and the ability to rapidly build sufficient knowledge to lead programs in complex or evolving disease areas.
• Demonstrated ability to lead through influence, align stakeholders with differing perspectives, and drive decisions without relying on formal authority.
• Ability to navigate ambiguity, identify the decisions and milestones that matter most, and create clarity for cross-functional teams.
• Strong strategic judgment combined with a willingness to work at a detailed, hands-on level when required.

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