RaNA Therapeutics, located in Cambridge, Massachusetts, is pioneering the discovery and development of a new class of RNA-based therapeutics that selectively activate protein expression. We focus on developing treatments for diseases with a significant unmet need that are difficult or impossible to address with existing technologies. Our lead programs include the upregulation of SMN2 for the treatment of Spinal Muscular Atrophy and the upregulation of Frataxin for the treatment of Friedreich’s Ataxia. Along with these lead programs, we are targeting other rare genetic diseases, hepatic diseases, and inflammatory diseases. We are dedicated to translating cutting-edge science in the areas of gene regulation, lncRNA and epigenetics into drugs that will have a significant impact in the treatment of life-threatening diseases.
We are seeking a highly motivated and scientifically rigorous individual to join RaNA and lead our Pharmacology efforts. The successful candidate will report to Head of Translational Development and will be an integral part of the Research and Development team.
Summary of Role
The Head of Pharmacology is responsible for providing pharmacology strategy, expertise, representation and resources to all program teams and RaNA management across all therapeutic areas and stages of development. S/he will provide leadership and oversight of the Pharmacology group and will work closely with Discovery, Chemistry, Regulatory and Clinical colleagues to generate and integrate in vitro and in vivo pharmacology and pharmacogenomic data leading to the demonstration and confirmation of pharmacologic target engagement, mechanism of action (MoA) and proof of concept (PoC) efficacy or pharmacodynamic effect for novel therapeutics in translationally relevant disease models. In addition, the pharmacology organization will be responsible for exploration and validation of biomarker strategy, identification and validation, as well as study design, analysis and reporting for all phase-appropriate and registration-enabling studies. The successful group leader is expected to maintain a flat, consensus-driven team and to balance big picture, long-term and strategic considerations with tactical, detailed technical aspects in approaching and dealing with program-related pharmacology issues. This individual will be highly analytical, decisive, goal-oriented, and timeline sensitive while maintaining the highest of scientific and ethical standards. The Head of Pharmacology develops and implements strategic plans for preclinical Pharmacology and actively participates in program teams, providing expert representation to global regulatory agencies as well as collaborating with R&D leadership and assisting in the identification of new business initiatives and in-licensing opportunities. The successful candidate will establish and maintain appropriate quality systems and procedures for outsourcing, managing and reporting for Pharmacology studies and other activities in support of RaNA portfolio programs, and provide submission-ready documentation for the Pharmacology components of regulatory submissions.
- A broad understanding of the drug development process and the ability to provide expert guidance in designing and implementing nonclinical Pharmacology studies and development programs for biopharmaceuticals including protein therapies, antibodies, oligonucleotides and small-molecule therapeutics.
- Demonstrated knowledge of global regulatory requirements and expectations for nonclinical Pharmacology packages to support first in human, clinical development, and product registration, including thorough knowledge of GLP, GCP, ICH and other applicable global guidelines.
- Ability to analyze, interpret and document nonclinical Pharmacology and PK/PD data with overall responsibility to ensure that sufficient preclinical knowledge exists to validate the mechanism of action (MoA) for clinical and regulatory purposes prior to human administration.
- Experience within a team environment as both a member and a leader with strong organizational and interpersonal skills, including exceptional oral and written communication skills with a demonstrated ability to write protocols, nonclinical/clinical study reports and scientific peer-reviewed publications.
- Ability to influence through effective communication, respectful challenge, and persuasion across the Research and Development organization is essential.
- Ability to effectively represent the Company to key external stakeholders, effectively engage and present to staff at all levels, including senior management and technical and business experts clearly and at the appropriate level of detail in representing strategy, results and implications.
- Demonstrate and maintain comprehensive cross-functional and industry perspective, judgement and ability to apply understanding to individual projects and programs.